Medicare Coverage Policy ~ MCAC
Executive Committee
Transcript of June 6, 2000 Meeting
(For more information,
contact the Executive
Secretary.)Note:This transcript has not been edited and HCFA
makes no representation regarding its accuracy. 00001
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11 HEALTH CARE FINANCING ADMINISTRATION
12 Medicare Coverage Advisory Committee
13 Executive Committee Meeting
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19 June 6, 2000
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21 Baltimore Convention Center
22 One West Pratt Street
23 Baltimore, Maryland
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1 Panelists
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3 Chairperson
4 Harold C. Sox, M.D.
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6 Vice-Chairperson
7 Robert Brook, M.D.
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9 Voting Members
10 Thomas V. Holohan, M.A., M.D., FACP
11 Leslie P. Francis, J.D., Ph.D.
12 John H. Ferguson, M.D.
13 Robert L. Murray, Ph.D.
14 Alan M. Garber, M.D., Ph.D.
15 Michael D. Maves, M.D., M.B.A.
16 Frank J. Papatheofanis, M.D., Ph.D.
17 Ronald M. Davis, M.D.
18 Daisy Alford-Smith, Ph.D.
19 Joe W. Johnson, D.C.
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21 HCFA Liaison
22 Hugh F. Hill, III, M.D., J.D.
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24 Consumer Representative
25 Linda A. Bergthold, Ph.D.
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1 Panelists (Continued)
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3 Industry Representative
4 Randel E. Richner, M.P.H.
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6 Executive Secretary
7 Constance Conrad, R.N.
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1 TABLE OF CONTENTS
2 Page
3 Opening Remarks
4 Constance Conrad, R.N. 7
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6 Review of Comments on MCAC Interim Operating
7 Procedures
8 Summary of MCAC Interim Operating Procedures
9 Recommendations
10 Harold C. Sox, M.D. 11
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12 HCFA Presentation
13 Hugh F. Hill, III, M.D., J.D. 20
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15 Open Public Comments & Scheduled Commentaries
16 Sandra Sherman 27
17 Jerome B. Connolly, P.T. 31
18 Marshall Stanton, M.D. 37
19 Alfred Chiplin 44
20 Debra Jensen, M.D. 47
21 Kevin M. Connolly 52
22 Jodi Chappell 57
23 Thomas Mesken 63
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1 TABLE OF CONTENTS (Continued)
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3 Remarks from Director, Office of Clinical Standards
4 and Quality
5 Jeffrey Kang, M.D. 68
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7 Open Committee Deliberation 71
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9 Open Public Comments 108
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11 Final Recommendations 117
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13 LUNCH 135
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15 Presentation and Review of Medical Surgical
16 Procedures Panel
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18 HCFA Presentation
19 John J. Whyte, M.D., M.P.H. 136
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21 Report of the Chair
22 Alan M. Garber, M.D., Ph.D. 154
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1 TABLE OF CONTENTS (Continued)
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3 Open Public Comments & Scheduled Commentaries
4 Barbara Woolner 174
5 Debra Jensen, M.D. 180
6 Kevin M. Connolly 184
7 Francie Bernier, R.N.C., B.S.N. 190
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9 Open Committee Deliberation 196
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11 Open Public Comments 221
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13 Final Recommendations/Ratification 228
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15 HCFA Announcements/Information 234
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1 PANEL PROCEEDINGS
2 (The meeting was called to order at 8:35
3 a.m., Tuesday, June 6, 2000.
4 MS. CONRAD: Thank you, good morning.
5 Welcome panel chairperson, members and guests. I
6 am Constance Conrad, Executive Secretary of the
7 Executive Committee of the Medicare Coverage
8 Advisory Committee. The Committee is here today to
9 discuss procedural aspects of future public
10 meetings of the medical specialty panels of the
11 MCAC and to hear reports from the Medical and
12 Surgical Procedures Panel meeting of April 12th and
13 13th, during which biofeedback and pelvic floor
14 electrical stimulation in the treatment of urinary
15 incontinence were deliberated.
16 At the conclusion of the afternoon
17 session today, if the Executive Committee ratifies
18 the Medical and Surgical Procedures Panel
19 recommendations, it will officially transmit that
20 recommendation to HCFA. HCFA will develop a
21 coverage policy within 60 days of the receipt of
22 that recommendation.
23 The following announcement addresses
24 conflict of address issues associated with this
25 meeting and is made a part of the record to
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1 preclude even the appearance of impropriety. To
2 determine if any conflict existed, the Agency
3 reviewed the submitted agenda and all financial
4 interests reported by panel participants. The
5 conflict of interest statute prohibits special
6 government employees from participating in matters
7 that could affect their or their employer's
8 financial interests. The Agency has determined
9 that all members may participate in the matters
10 before the Committee today.
11 With respect to all other participants,
12 we ask in the interest of fairness that all persons
13 making statements or presentations disclose any
14 current or previous financial involvement with any
15 firm whose products or services they may wish to
16 comment on.
17 And now I would like to turn the meeting
18 over to Chairman Harold Sox, who will ask the
19 Committee members to introduce themselves.
20 DR. SOX: Well, the Committee members
21 are known to each other but they're not necessarily
22 known to members of our audience, so I would like
23 each person to identify themselves by name and also
24 state where they're from and what they do very
25 briefly, so that the audience will understand.
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1 Randel, would you like to start please?
2 MS. RICHNER: Randel Richner, Boston
3 Scientific, vice president of reimbursement and
4 outcomes planning.
5 DR. JOHNSON: Joe Johnson, chiropractor
6 in private practice, Paxton, Florida.
7 DR. BERGTHOLD: I'm Linda Bergthold, and
8 I'm a consultant and researcher, and I am the
9 consumer representative to the Executive Committee.
10 DR. DAVIS: I'm Ron Davis. I'm a
11 preventive medicine physician. I work at the Henry
12 Ford Health System in Detroit, where I direct a
13 center for health promotion and disease prevention.
14 DR. PAPATHEOFANIS: I'm Frank
15 Papatheofanis. I'm an assistant professor of
16 radiology at the University of California in San
17 Diego.
18 DR. MURRAY: Bob Murray. I'm an
19 attorney and biochemist at Lutheran General
20 Hospital in Park Ridge, Illinois, technical
21 director of the clinical laboratory.
22 DR. BROOK: Robert Brook. I'm an
23 internist and professor of medicine at UCLA, and
24 head of Rand Health.
25 DR. ALFORD-SMITH: I am Daisy
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1 Alford-Smith. I am the director of the County
2 Department of Human Services in Akron, Ohio.
3 DR. GARBER: I am Alan Garber. I'm a
4 general internist at the Department of Veterans
5 Affairs and a professor of medicine at Stanford.
6 DR. FERGUSON: John Ferguson,
7 neurologist, former director of the consensus
8 program at the NIH and now a private consultant.
9 DR. HOLOHAN: Tom Holohan, internist,
10 hematologist oncologist, associate chief medical
11 director at VA Headquarters, and chief of patient
12 care services for the Veterans Administration.
13 DR. FRANCIS: Leslie Francis. I'm
14 professor of philosophy and professor of law, and
15 adjunct professor of medicine at the University of
16 Utah.
17 DR. MAVES: I'm Mike Maves. I'm the
18 president of the Consumer Healthcare Products
19 Association and a practicing otolaryngologist at
20 Georgetown.
21 DR. HILL: I am Hugh Hill, and as you
22 heard, I'm the acting director of coverage and
23 analysis, and I am the federal representative to
24 the panel.
25 DR. SOX: I am Hal Sox. I am a general
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1 internist. I chair the department of medicine at
2 Dartmouth-Hitchcock Medical Center.
3 Well, today's meeting basically has two
4 parts to it. The first is to give the Executive
5 Committee a chance to reflect on the responses that
6 it has received regarding the interim Medicare
7 Coverage Advisory guidelines that we ratified at
8 our March meeting. We have both had a chance to
9 get some written feedback from organizations and
10 individuals, as well as to use the guidelines in
11 evaluating procedures for incontinence. So the
12 first half of today's meeting gives us a chance to
13 reflect on what we've done and to decide whether or
14 not we need to make any significant modifications
15 on the basis of this feedback and the single
16 experience we have had in applying these guidelines
17 to a real world task.
18 I think each committee member should be
19 thinking about whether this is the right time to
20 make fundamental changes in what we proposed or
21 whether to make changes and to emphasize certain
22 aspects of the procedure that may not have gotten
23 full attention during the incontinence review by
24 the Med-Surg panel. So the question is, do we make
25 big changes on the basis of feedback plus one
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1 experience or do we make small changes that are
2 aimed at trying to make the process fair and to
3 give everybody a chance to hear from everybody who
4 has an opinion about the process.
5 So, to get that process rolling, I am
6 going to quote selectively from our, from the
7 guidelines as much for the benefit of the audience
8 as for the panel, who by now should have them
9 pretty well memorized. Then we are going to be
10 hearing from some of the leaders at HCFA about
11 their take on the experiences of the past three
12 months. Then we are going to hear from a number of
13 people who signed up to give presentations for
14 about an hour. And then after a break, we will
15 have a chance to debate really, what we should do,
16 if anything, at this point. That will bring us up
17 to lunch and then in the afternoon we will discuss
18 the recommendations of the Med-Surg panel regarding
19 procedures for incontinence. So that is sort of a
20 blueprint for what we're going to do today.
21 Let me quote sort of selectively from
22 this document that we've developed and ratified on
23 March 1st. The document has two purposes
24 basically; one is to give some general guidelines,
25 not rigid rules, but general guidelines about how
.00013
1 to evaluate evidence, and it focuses first of all
2 on whether the evidence is sufficiently strong to
3 draw a conclusion about whether a potential
4 procedure in fact is effective in patients. And
5 then secondly, if there is evidence, valid evidence
6 that it's either effective or not effective, how
7 big is the effect, is this a breakthrough
8 technology, something that makes a small
9 contribution at the margin, or something that in
10 fact is less effective than currently covered
11 procedures but has some benefits that might be
12 advantageous to selected patients. The second part
13 of the document deals with procedure, makes some
14 suggestions about how the panels of MCAC can
15 function most effectively.
16 So first on the evaluation of evidence,
17 it states that the MCA panels should explore many
18 sources of evidence in assembling the body of
19 evidence to be used in their deliberations. The
20 sources might include peer reviewed scientific
21 literature, the recommendation of expert panels,
22 and unpublished data used to secure FDA approval.
23 The quality of evidence from these various sources
24 will vary, and the panel should weigh that evidence
25 according to its quality.
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1 So the first question is then, is the
2 evidence adequate? The panels must determine
3 whether the scientific evidence is adequate to draw
4 conclusions about the effectiveness of the
5 intervention in routine clinical use in the
6 population of Medicare beneficiaries. There is a
7 lot contained in that sentence. The assessment of
8 the adequacy of evidence is a sine qua non of
9 essentially all modern approaches to evaluation of
10 medical technologies, and I want to underscore
11 that. There are many efforts in many different
12 venues for evaluating medical technology. They all
13 have as a common feature assessing whether the
14 evidence is adequate to draw conclusions about
15 whether the technology is effective, so this panel
16 is mainstream in adopting that approach rather than
17 on the cutting edge.
18 Defining what constitutes adequate
19 evidence is the critical step, and that includes
20 both the validity of the evidence and its general
21 applicability to the population of interest. And
22 the question about validity turns principally on
23 the question of whether the study systematically
24 underestimates or overestimates the effect of the
25 intervention because of possible bias or other
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1 errors in assigning patients to the intervention
2 and control groups.
3 Now, the best way to avoid systematic
4 bias that leads to over or underestimating the
5 effect of an intervention is to assign subjects
6 randomly to the intervention or control group, and
7 if the number of subjects is sufficiently large,
8 the process of randomization essentially insures
9 that any effect that's seen is due to the
10 intervention rather than some other variable that
11 might be causing differences between the two
12 groups. Therefore, the randomized trial represents
13 the easiest way to be sure that you're dealing with
14 valid evidence.
15 There are other forms of evidence that
16 may be considered in which controls are present,
17 such as case control studies, cohort studies and
18 the like. Because subjects are not randomly
19 assigned to intervention and control group, there
20 is always a possibility that some other variable
21 causes people to either get the intervention or to
22 be in the control group, and it is that variable
23 rather than the intervention itself that affects
24 the outcome. And so it raises the possibility of
25 misinterpreting the evidence and assigning credit
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1 for the success of the intervention to the
2 intervention when in fact it's some other variable
3 that's at fault. And that's the reason why,
4 insofar as possible, randomized trials make it
5 easier on panels like this to make up their mind.
6 Although they do not have randomized
7 controls, all well designed observational studies
8 include some form of control. This is certainly a
9 fundamental point. Controls may consist of an
10 implicit or explicit control group. A body of
11 evidence consisting solely of studies with no
12 controls whatsoever, where they're based on
13 anecdotal evidence, testimony or case series, is
14 never adequate for making a decision about whether
15 something is effective. That's a very strong
16 statement and one that this panel has endorsed.
17 However, in many cases the panel will
18 determine that observational evidence is sufficient
19 to draw conclusions about effectiveness. This
20 panel would prefer to have randomized trial
21 evidence but it has said to itself, you must
22 consider other forms of evidence when randomized
23 trial evidence is not available. But when these
24 circumstances apply, the panel must describe
25 possible sources of bias and explain why they felt
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1 they could draw a conclusion despite the potential
2 for bias.
3 The second major point beside validity
4 is external validity. Do the studies apply to the
5 Medicare population? A study performed in 25 year
6 olds, it may be a stretch to assume that that's
7 going to apply to a group of 75 year olds. So
8 external validity means, do the studies apply to
9 the population in question. And the second major
10 criterion for evaluating the evidence, is the size
11 and direction of the health effect it
12 demonstrates. Does the intervention improve health
13 outcomes or does it make them worse, and by how
14 much are health outcomes improved or made worse.
15 And the Committee at its last meeting endorsed a
16 way to categorize the size of the effect ranging
17 from breakthrough technology in which the
18 improvement is so large that the intervention
19 becomes the standard of care, to noneffective,
20 which means that it has no effect or even
21 deleterious effects on health.
22 The second part of our report dealt with
23 suggestions for panel operations, and I will just
24 very briefly go over each of those in turn. The
25 first had to do with the structure of the evidence
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1 provided to the panels. The panel, the Executive
2 Committee asserted that panels should receive well
3 organized high quality background information
4 before beginning their deliberations. The evidence
5 should be summarized in a report, not simply
6 presented as a collection of data or primary
7 studies. And we saw an example of that with the
8 incontinence study, in which an evidence based
9 practice center that is associated with Blue
10 Cross/Blue Shield carried out the evidence report,
11 which the panel considered in drawing its
12 conclusions.
13 The second major point about panel
14 operations is that panel members should take an
15 active role in preparing the evidence report, they
16 shouldn't just be passive receivers of the report
17 but in fact the panel chair should play an active
18 role in framing the questions that the evidence
19 report must address, and the panel must answer, and
20 we'll hear about how that was done with the
21 incontinence study. We assert that several panel
22 members should participate actively in designing
23 the evidence review and preparing the evidence
24 report, so that there are few built-in experts on
25 the panel who understand the evidence intimately.
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1 And finally, that the panel should assign two
2 members to be sort of primary reviewers of the
3 evidence and report, and to go over in depth,
4 perhaps spending more time on it than every panel
5 member would have time to do.
6 The third recommendation is that the
7 evidence report should get external review by
8 several experts in order to be sure it is complete
9 and that it is free from bias, and that's a way of
10 both protecting the panels against making mistakes,
11 as well as protecting the advocates of the
12 technology from a biased report that might be
13 unfavorable to their interests.
14 Finally, explanation. A panel must
15 explain its reasoning in coming to its conclusions
16 and that explanation should be in writing. And in
17 fact, we stated that prior to acting on a panel
18 recommendation such as the Med-Surg panel
19 recommendation on incontinence, that the Executive
20 Committee, this group will have the following
21 operational documents: First of all, a meeting
22 transcript, which in this case I gather ran to 500
23 pages or 500 megabytes or something like that, and
24 also a meeting summary prepared by HCFA staff and
25 reviewed by the panel chair for approval, and the
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1 summary shall include the explanation of the
2 panel's vote.
3 So that was our report, and again, the
4 question before us during the first half of this
5 meeting today is did we get it wrong, should we
6 make some changes, should me emphasize certain
7 elements of the report that may not have been
8 followed to everybody's satisfaction in the first
9 report.
10 So, I guess before we proceed now to
11 hear from folks who signed up to comment on this
12 report, we're going to hear from the folks in HCFA
13 and Hugh, do you want to lead?
14 DR. HILL: Yes, if I may. I just very
15 briefly want to welcome and thank you all. In the
16 interest of time, I will not respond or iterate all
17 the comments that we received, that we collected
18 for the Committee in the operations procedures
19 mailing that you got. But on behalf of the Agency,
20 the administrator, my boss, Dr. Jeffrey Kang, who
21 may yet be able to join us briefly, I want to
22 welcome you and thank you very much for
23 participating, and especially to the panel for its
24 hard and good work on this.
25 The summary statement I want to make is
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1 that HCFA remains strongly supportive of the MCAC
2 and the Executive Committee and its work, strongly
3 supportive and very appreciative. In addition to
4 the comments that we received, a general theme that
5 came up repeatedly was a question of whether we
6 want the Medicare Coverage Advisory Committees to
7 be telling us whether or not they think something
8 should be covered. And our answer to that at the
9 present time is that we are very interested in the
10 MCAC telling us about its analysis of the
11 scientific evidence. We hope that that analysis is
12 informed by clinical and methodological expertise.
13 Basically I'm reiterating Dr. Kang's letter to the
14 Committee that was sent out, I believe in January.
15 And one other brief comment is we
16 continue to get questions about whether or not the
17 Medicare Coverage Advisory Committee and the
18 Executive Committee is, in some people's terms,
19 quote, setting the bar too high, closed quote,
20 questions about whether or not the threshold that
21 has to be crossed by technology in terms of
22 evidentiary standards and proof is too great to be
23 met. Let me reiterate what we've said before and
24 what we've said consistently is that the threshold
25 determination is a matter of our responsibility; we
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1 look to the MCAC for comments about the hierarchy
2 of evidence, and analysis of comment of evidence at
3 different levels within that hierarchy, but we have
4 not abdicated our responsibility, nor have we tried
5 to shrug it off for making the ultimate coverage
6 decisions ourselves.
7 We will be talking about the urinary
8 incontinence panel this afternoon, I will make no
9 further comments about what happened there and what
10 we, the way we structured the questions and that
11 sort of thing. We are prepared to talk about that
12 should that become necessary, but as a notice, just
13 a heads up, I want to point out to the Committee
14 something that I think most of the people in the
15 audience already know, that we have published a
16 notice of intent. Both the Committee and the
17 public has expressed some of the same interests
18 that we have in having guidelines for what we mean
19 by reasonable and necessary as we try to continue
20 to be increasingly open and consistent and fair in
21 our coverage decision-making process. And this
22 step is a major one; we hope it will lead to a
23 notice of proposed rule making, which will define
24 what we mean by reasonable and necessary, or give
25 some criteria for that.
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1 We are not asking for a formal response
2 from the Medicare Coverage Advisory Committee or
3 even this learned Executive Committee about that
4 notice of intent, but the comment period on it is
5 being extended and your informed, given the wisdom
6 you've shown in this, is especially important to
7 us; statements and suggestions will be very much
8 appreciated. And that's all I have right now.
9 MS. CONRAD: Thank you, Dr. Hill. Well,
10 at this time, let us move ahead with public
11 comments. And each, I have a list of speakers here
12 and we have determined by the number of speakers
13 and the amount of time available to us, each
14 speaker may have six minutes. It's nine o'clock
15 now. About -- excuse me. Yes, Bob?
16 DR. BROOK: Since we have a minute
17 before nine, can I ask the HCFA representative one
18 question?
19 MS. CONRAD: Please.
20 DR. BROOK: I am just wondering one
21 question; if the purpose of the panels is only to
22 provide evidence, to assess the evidence, can I ask
23 a question about why, since another government
24 agency, the Agency for Health Care Policy and
25 Research, had already issued an evidence based
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1 report on urinary incontinence in 1996, were those
2 two topics sent to MCAC? Was it because you felt
3 that the report by the Agency was inadequate, out
4 of date? I mean, there seems to be some confusion
5 here about the roles of various agencies in the
6 government.
7 DR. HILL: That's more than a one-minute
8 answer. In the charter for this committee and its
9 subpanels, we mention several criteria for when
10 subjects would be sent to this committee. And one
11 of those is impact on the program; we've talked
12 about others, where something's controversial,
13 there's a split of opinion among scientists in the
14 area and that sort of thing. But in this case,
15 tracing it back as far as I can tell, it was
16 decided before I came on board because of its
17 impact on the program. And we heard testimony at
18 the UI panel about the significance of this to the
19 Medicare population which reinforced our belief
20 that it is important to the program.
21 DR. BROOK: I'm sure the Agency felt the
22 same way. The question is, again, I come back:
23 Was this -- it would be nice for this panel to
24 know, because that's part of what we need to decide
25 both for the morning and the afternoon, is there
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1 was presumably a first class evidence based report
2 that had been released. Was it determined --
3 because that determines our role. If our role is
4 only to look at evidence in the classical sense,
5 then that report was already there and you could
6 have just taken that report and used it, and made
7 it your coverage decision. And that's what -- I'm
8 trying really to understand the ground rules so I
9 can listen to this public discussion before we go
10 further, because this is what's confusing me.
11 DR. HILL: We have had discussions about
12 whether or not questions that come before the
13 panels should have technological assessments in
14 front of them, whether the panel should have TEC
15 assessments to look at in their evaluation of the
16 evidence. We are looking for obviously, more than
17 what the TEC assessment gives us when something
18 comes to the panel, and we're looking for your
19 informed expertise beyond that. I think Alan had
20 another response, based on his experience with the
21 UI panel.
22 DR. GARBER: Well, Bob, I think it's a
23 very fair question and it does deserve more than a
24 minute, but I'm sure this is going to be one of the
25 points of discussion this afternoon. If I could
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1 just briefly summarize, I was involved in the
2 process of helping to refine the questions and
3 wasn't participating in the initial formulation of
4 this whole area as a topic. But my understanding
5 is, first of all, the AHCPR report did not directly
6 address the same questions, although it was in the
7 same broad area, number one. And number two, there
8 had been a fair number of publications since the
9 AHCPR report was written. Now one could still
10 question the wisdom of choosing to do this topic,
11 but at least there was a rationale for why the
12 AHCPR report might not be the last word at the
13 current time for the questions that HCFA wished to
14 consider.
15 DR. HILL: And I also point out that as
16 ARCAS reminded us, this was not a full $500,000
17 18-month evidence report that we asked for as a
18 supplement from them. This was a limited report on
19 the randomized control trials that were available
20 and a layer of evidence that was looked at in a
21 more limited and briefer fashion, in part in the
22 interest of time.
23 DR. SOX: Thank you for that question,
24 Bob. I'm sure it's one we will return to, because
25 it in part raises the question about a process
.00027
1 that's intended to provide clinical guidance to
2 physicians perhaps in the absence of good evidence
3 on the one hand, and a process of making
4 recommendations about that evidence on the other,
5 and at least for now, our assignment is to make
6 comments about the adequacy of the evidence, not
7 really to give guidelines to physicians about how
8 to practice under circumstances where the evidence
9 may not be full and complete. So with that brief
10 exchange, why don't we move on here? Do you have
11 the first speaker, Connie?
12 MS. CONRAD: I certainly do. The first
13 scheduled speaker is Sandra Sherman, representing
14 the American Medical Association. Following her
15 will be Jerome Connolly.
16 MS. SHERMAN: Good morning. I believe
17 you've already been provided with a copy of a
18 letter that Dr. E. Ratcliffe Anderson, the AMA
19 executive vice president and CEO, sent to HCFA on
20 May 9th, offering comments on the interim
21 recommendations. I just want to underscore a few
22 of the key points in Dr. Anderson's letter.
23 First, we want to make clear our view
24 that the interim recommendations have significantly
25 improved the process for consideration of issues
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1 referred to the MCAC. The MCAC represents a major
2 leap forward in the methods to be used by Medicare
3 for the development of national coverage policies.
4 We applaud the Committee's and HCFA's focus on
5 evidence based decisions, and we were pleased by
6 the significant level of participation in the April
7 panel meeting by the national medical specialty
8 societies.
9 The interim recommendations document
10 focused on the question, is the evidence concerning
11 effectiveness in the Medicare population adequate
12 to draw conclusions about magnitude of
13 effectiveness relative to other items and
14 services. The Executive Committee indicated that
15 the standard of excellence for evidence reports
16 would include the best work in the private sector,
17 e.g., Blue Cross/Blue Shield, by professional
18 organizations, e.g., ACP, ASIM, and for other
19 federally sponsored panels, e.g., the evidence
20 based practice centers, technical support for the
21 U.S. Preventative Services Task Force.
22 At the April panel meeting, however, it
23 seemed as if the recommendations were being
24 interpreted as stating that the only evidence
25 worthy of consideration is that contained in peer
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1 reviewed scientific literature. The MCAC panel was
2 essentially asked to disregard clinical guidelines
3 that had been developed by the Agency for Health
4 Care Policy and Research, and expert opinions
5 developed by national medical societies.
6 The second point addressed in our letter
7 is that continued application of the Executive
8 Committee recommendations in such a narrow fashion
9 will prevent the MCAC deliberations from achieving
10 the desired high standards of comprehensiveness and
11 balance. The effectiveness of many procedures that
12 are covered by Medicare today for aged and disabled
13 beneficiaries has not been demonstrated in peer
14 reviewed scientific literature. And even where the
15 effectiveness of treatments has been demonstrated
16 in a study population under study conditions, it is
17 unlikely that effectiveness in routine clinical use
18 in the Medicare population will have been
19 demonstrated in scientific journals.
20 It is clearly important that MCAC panels
21 focus on a critical evaluation of the available
22 scientific literature on the effectiveness of
23 procedures proposed for Medicare coverage. It is
24 equally important, however, for the panels to
25 critically evaluate other clinical information.
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1 Inclusion of a service within a clinical guideline
2 that is accepted by the medical community as the
3 standard of care is an important consideration.
4 Omission of such clinical information from MCAC
5 deliberations could lead to Medicare's failure to
6 cover important and effective diagnostic and
7 therapeutic options.
8 The issues that HCFA is most likely to
9 refer to the MCAC are those that are
10 controversial. If the questions surrounding these
11 issues could be unequivocally answered by a
12 technical assessment of published scientific
13 evidence, HCFA would not need to refer them to the
14 MCAC. What generally makes these issues
15 controversial is that the published studies do not
16 conclusively answer the question of effectiveness
17 for Medicare beneficiaries.
18 Finally, our letter describes several
19 suggested revisions to the document. In
20 particular, within the section on adequacy of
21 evidence, the AMA recommends that language be added
22 regarding how the panel should weigh and consider
23 clinical guidelines, standard text books, review
24 articles, and other clinical evidence that may be
25 presented. Grading systems clearly give more
.00031
1 weight to evidence from well designed clinical
2 trials than what might be regarded as expert
3 opinion. Nonetheless, when the data available from
4 the scientific literature is insufficient to draw
5 conclusions, expert opinion regarding the adequacy
6 of available clinical information should be
7 considered.
8 The AMA also recommends that several of
9 the panel members be tasked with assessing the
10 completeness and accuracy of the evidence report.
11 If panel members know of studies, guidelines,
12 consensus statements, or other information that
13 should be but is not included, they should be
14 encouraged to provide this information in time for
15 the evidence report to be revised. Thank you.
16 MS. CONRAD: Thank you, Miss Sherman.
17 Jerome Connolly, to be followed by Marshall
18 Stanton.
19 MR. J. CONNOLLY: Members of the
20 committee, good morning. My name is Jerome
21 Connolly. I'm a physical therapist of 28 years,
22 having graduated from the Mayo Clinic School of
23 Physical Therapy in 1972. I currently serve as the
24 senior vice president for health policy for the
25 American Physical Therapy Association, and I have
.00032
1 no current or past conflict of interest to be
2 disclosed at this time.
3 I was told originally that I'd have
4 between seven and ten minutes, so I only have six,
5 so I'll speak a little more quickly than I probably
6 ordinarily would. Plus the fact that I drove from
7 Washington this morning in a driving rainstorm and
8 I haven't had a chance to use a men's room yet, so
9 I will probably only use six minutes, maybe only
10 five and a half.
11 On behalf of APTA and its almost 69,000
12 members, I wish to thank you for the opportunity to
13 address you today. APTA commends HCFA for its
14 attempt to implement an open coverage process, a
15 process that should allow the general public, the
16 health professions, and the health care industry to
17 play an important role in the development of
18 Medicare coverage decisions. Having participated
19 in the Medicare Coverage Advisory Committee process
20 relative to urinary incontinence in April, a
21 participation which at times APTA found quite
22 frustrating, APTA has several recommendations that
23 it would like to offer in an effort to make the
24 process and the experience more useful, more
25 productive, and more credible. And these have been
.00033
1 conveyed to you and to Dr. Hill in a letter that we
2 have submitted.
3 First, we recommend that the catalogued
4 information to be sent to the panel should be sent
5 well in advance, to allow panelists a sufficient
6 amount of time to request, to read, and to digest
7 materials. Due to time constraints imposed on
8 verbal testimony, it is difficult to provide
9 extensive comment to the panel without the aid of
10 written correspondence; however, written comments
11 are only beneficial if the panel members read them
12 prior to deliberation. APTA appreciates the fact
13 that HCFA solicits written comments from the
14 public. The process should provide assurance that
15 the written comments submitted will be distributed
16 to the panel, and will be done in a time -- and
17 that distribution will be done so in a timely
18 manner.
19 Secondly, when a technology assessment
20 is done, and HCFA solicits written comments from
21 the public, it is imperative that the public has
22 access to the technology assessment for a
23 reasonable period of time preceding the deadline
24 for comments. In this case, the assessment was
25 posted on the web almost a week after the deadline
.00034
1 to comment had passed. This made it impossible for
2 the public to address by written analysis issues
3 raised in the technology assessment. To achieve an
4 open public forum, HCFA must allow the public to be
5 privy to, and provide written comment on relevant
6 materials prior to the meeting. Otherwise, the
7 public is disadvantaged and its efforts to provide
8 meaningful comprehensive input are thwarted.
9 APTA's presenter, Cynthia Feldt, requested that
10 APTA's analysis, written analysis of the
11 assessment, be distributed to the panel during her
12 testimony, but that request was denied.
13 Third, in an effort to achieve an
14 objective process, it is imperative also that the
15 panel be sent, and not just have access to upon
16 request, a variety of materials that reflect
17 diverse opinions when such material is available.
18 For example, the panel only received the Blue
19 Cross/Blue Shield technology assessment, which
20 expressed only one viewpoint. In addition to the
21 assessment, it would have been beneficial for the
22 panel to have received the AHCPR clinical practice
23 guidelines, because they contained considerably
24 different, yet valid, viewpoints. If HCFA is
25 expecting the panel to make an impartial decision
.00035
1 at the meeting's conclusion that day, more balanced
2 materials and perspective should be provided to the
3 panel well in advance of its deliberations.
4 Fourth, the questions posed to the panel
5 by HCFA should be broader than those used in
6 April. These questions were very narrow in scope,
7 and therefore only allowed consideration of a
8 limited number of studies that carried out a very
9 specific comparative analysis of treatment. APTA
10 believes that the adoption of an evidence based
11 standard for coverage should not preclude the
12 consideration of either the expert clinical
13 testimony presented at the meeting, panelists' own
14 individual knowledge or clinical experience, or the
15 personal experiences of consumers. Without
16 allowing consideration of this input, panel members
17 are essentially being told that the relevant
18 clinical experience, even their own clinical
19 experience, what has gone in this case, in April,
20 for the past 50 years, is not noteworthy.
21 According to Sackett, et al., evidence
22 based medicine means integrating clinical expertise
23 with the best available external clinical evidence
24 from systematic research. He goes on to say, EBM
25 builds on and reinforces but never replaces
.00036
1 clinical skills, clinical judgment and clinical
2 experience.
3 Fifth, to insure the public of receiving
4 due process, it is essential that the panel members
5 have more latitude when formulating their opinions
6 on coverage issues. Amending the questions to be
7 inclusive rather than exclusive would accomplish
8 this goal. Therefore, APTA recommends that in
9 addition to framing more broadly constructed
10 questions, that the panel members be allowed to and
11 specifically requested to call upon their
12 individual clinical expertise and experience. An
13 appropriate question to be posed to the panel would
14 be worded as follows: Is the scientific evidence
15 adequate, when combined with clinical evidence,
16 clinical experience and consumer input, to allow a
17 conclusion to be drawn that the intervention has a
18 reasonable chance of benefitting the patient? In
19 other words, we're talking about reasonable and
20 necessary here. In other words, the basis for the
21 panel's conclusion and recommendation should be
22 evidence based, based on the preponderance of both
23 scientific and clinical evidence. Preponderance of
24 evidence, not beyond a reasonable doubt. Such an
25 approach would be consistent --
.00037
1 MS. CONRAD: Time please.
2 MR. J. CONNOLLY: -- With the writings of
3 Sackett, et al., in evidence based medicine, how to
4 practice and teach EBM, which is now in its fifth
5 printing, which states, EBM is not restricted to
6 randomized trials and meta-analysis.
7 So we would say in conclusion, that the
8 process we participated in is not yet quite to the
9 level that it needs to be, that it does contain
10 some fundamental flaws, Dr. Sox, and in answering
11 that question, there are some fundamental flaws,
12 and the process can be and should be modified and
13 improved before any decision reached by using this
14 process is implemented. Thank you again for the
15 opportunity to comment.
16 MS. CONRAD: Thank you, Mr. Connolly.
17 Marshall Stanton, representing Medtronic
18 Incorporated is next, followed by Alfred Chiplin
19 please.
20 DR. STANTON: Thank you. Connie, have
21 the panel members received copies.
22 MS. CONRAD: I'm not sure.
23 DR. SOX: We certainly received a letter
24 from you.
25 DR. STANTON: My name is Marshall
.00038
1 Stanton, I am the industry representative the
2 MCAC's Medical Surgical Procedures Panel. I am
3 currently medical director and vice president of
4 therapy development for the cardiac rhythm
5 management division of Medtronic. Prior to this, I
6 was a practicing cardiac electrophysiologist at
7 Mayo Clinic in Rochester, Minnesota, holding an
8 academic post there for ten years.
9 The MCAC Executive Committee has
10 emphasized that its interim recommendations for
11 evaluating the effectiveness of medical therapies
12 and diagnostics will be a living document subject
13 to modifications in substance and tone. I would
14 like to take this opportunity to report on my
15 observations from the first meeting of the Medical
16 Surgical Panel, and suggest how the experience of
17 this panel might improve the guidance document and
18 the MCAC process.
19 I think we all agree that ideally, the
20 MCAC process should be predictable, timely,
21 accountable and consistent, while still maintaining
22 the flexibility necessary to make the process of
23 practical use. While a number of excellent points
24 were raised at the recent Medical Surgical Panel
25 meeting that considered biofeedback and pelvic
.00039
1 floor stimulation for urinary incontinence, I
2 believe that the panel's rigid interpretation of
3 the guidance document led to deliberations and
4 results that fell short of the Executive
5 Committee's stated goals.
6 The Executive Committee has suggested
7 that a variety of evidence, including
8 recommendations from experts, could be considered
9 adequate for a positive panel recommendation.
10 However, the Medical Surgical Panel interpreted the
11 guidance document to require conclusive scientific
12 evidence from multiple large randomized control
13 trials with consistent positive outcomes. The
14 process virtually ignored other forms of evidence.
15 The panel took no account of the views and
16 recommendations of specialty societies, consumers
17 or practitioners, and it ignored the results of
18 clinical practice in its determination that there
19 was insufficient evidence.
20 In addition, the questions that were
21 posed left the panel too constrained to be of any
22 practical use. The purpose of an advisory
23 committee, like the Medical Surgical Panel, should
24 be to provide its clinical perspective on the value
25 of a diagnostic or therapy. Instead, the exclusive
.00040
1 focus of the panel on the adequacy of the
2 scientific evidence and the Blue Cross/Blue Shield
3 TEC reports left some panel members voting no,
4 while stating they believed the therapy should be
5 covered. Requiring the panel to vote on the
6 question of adequacy of study design, consistency
7 of results, applicability to the Medicare
8 population, and applicability beyond the research
9 setting before proceeding to an opinion on the
10 potential benefit of the therapy, places a serious
11 and in some cases insurmountable obstacle into the
12 process, virtually excluding further rational
13 discussion of evidence.
14 Many procedures and technologies that
15 are widely accepted as standard of care will not
16 meet the standard of conclusive scientific evidence
17 from multiple randomized control trials. The
18 notion that public health policy making will
19 require rigorous scientific proof, will result in a
20 disservice to beneficiaries. If we confine the
21 panels decision making to evidence that satisfied
22 the P less than .05 perspective, it will be an
23 opportunity lost for Medicare beneficiaries.
24 Therefore, I believe the outcome of the
25 Medical Surgical Panel meeting shows that the
.00041
1 guidance document needs to be clarified to further
2 strengthen the ability of the panels to give
3 positive recommendations to therapies they believe
4 are beneficial for certain patients, but may not be
5 supported by conclusive scientific evidence. By
6 definition, the standards established to judge
7 adequacy of data in the guidance document seem to
8 preclude uncontrolled observational evidence,
9 including expert testimony and disease registries.
10 The guidance document is not sufficiently explicit
11 that various levels and forms of evidence are
12 acceptable, and this resulted in a panel meeting
13 that merely rubber stamped a Blue Cross/Blue Shield
14 TEC report.
15 We are naive if we believe that at a
16 time when HCFA is emphasizing an evidence based
17 coverage process, panel votes will not weigh very
18 heavily in their final decision. A no vote that
19 results in a noncoverage decision by HCFA may have
20 significant impact on beneficiaries. Noncoverage
21 decisions at the national level mean that no
22 beneficiary will be able to access the therapy at
23 any point in the treatment continuum, even as a
24 therapy of last resort, and there is no practical
25 right to appeal.
.00042
1 As a physician, I want to emphasize that
2 in clinical practice, treatment decisions for
3 patients are made based upon an assessment of the
4 literature, not on a meta-analysis requiring a
5 significance level of P less than .05. If the
6 latter were the case, few treatments would be
7 initiated.
8 It was remarked that the standards used
9 to make coverage decisions are entirely different
10 from those used in the clinical setting. I
11 understand the point that different levels of
12 evidence may be required to make coverage decisions
13 for large populations. However, I think we have to
14 remember that the Medicare beneficiary population
15 is made up of individual patients who together with
16 their physicians, make decisions about appropriate
17 treatments. If some of those treatments are not
18 available, then it directly interferes with options
19 available in the clinical setting. Coverage
20 decisions and clinical practice are directly
21 related in the real world.
22 It is worth noting some of the work of
23 other organizations charged with evaluating
24 evidence and making recommendations regarding the
25 use of certain services in clinical practice. For
.00043
1 example, it might be useful to look at the work of
2 the American College of Cardiology and American
3 Heart Association in developing practice
4 guidelines. When developing pacemaker guidelines
5 the committee emphasized that, quote, for certain
6 conditions for which no other therapy is available,
7 the indications for device therapy are based on
8 expert consensus and years of clinical experience,
9 and are thus well supported even though the
10 evidence was ranked at a level C. An analogous
11 example is the use of penicillin in pneumococcal
12 pneumonia, where there are no randomized trials and
13 only clinical experience.
14 MS. CONRAD: Time please.
15 DR. STANTON: The Executive Committee
16 should give highest priority to insuring that the
17 Medicare coverage process will work for
18 beneficiaries and the clinical community. This
19 means developing a process that utilizes the
20 expertise of the panel members to look beyond
21 randomized control trials to other appropriate
22 methods of evaluating evidence.
23 I will stop there, thank you.
24 MS. CONRAD: Thank you, Dr. Stanton.
25 May we have Alfred Chiplin please, and following,
.00044
1 Debra Jensen.
2 MR. CHIPLIN: Good morning, ladies and
3 gentlemen. I am Alfred Chiplin, with the Center
4 for Medicare Advocacy, and I represent several
5 other beneficiary advocacy organizations whose
6 names are listed in my testimony. I'd also say at
7 the beginning that our organization, one of them I
8 represented, the National Senior Citizens Law
9 Center and the Center for Medical Advocacy, were
10 involved in the initial litigation called Jameson
11 versus Bowen, which began to open up at least
12 through the settlement process in that lawsuit,
13 open up a window to exploring and making more
14 available to beneficiaries, information about the
15 national coverage process.
16 Along those lines, we applaud the moves
17 that have been made to continue to open up that
18 process, but we do have some concerns. With the
19 interim recommendations as they are designed, with
20 the laudable goal of assisting the MCAC panel in
21 evaluating the formal request for national coverage
22 determinations, they also place an insurmountable
23 burden on beneficiaries who are often asked to
24 prove the safety and effectiveness of medical
25 services, items and procedures. We ask you to
.00045
1 reevaluate your recommendations for the following
2 reasons:
3 Many services and items currently under
4 review by HCFA have already received approval from
5 the FDA and/or are already covered by other
6 insurance carriers. The onerous evidentiary proof
7 demanded by the interim recommendations ultimately
8 hurts rather than helps Medicare beneficiaries by
9 delaying access to services their own physicians
10 found reasonable and necessary for their care.
11 Beneficiaries often suffer negative legal
12 consequences or die as a result of a lengthy
13 coverage process, which often takes years to
14 complete, while HCFA decides whether to cover the
15 item, service or procedure prescribed by treating
16 physicians. The requirements that outside experts
17 be used in certain situations to further evaluate
18 the evidence presented to the review panels of
19 experts exacerbates the delay problem. Delays
20 cause further disparities between Medicare and
21 private insurance coverage. Together, the
22 requirements exacerbate the disparity between what
23 is covered by Medicare and what is covered under
24 private insurance practices.
25 Finally, the interim recommendations
.00046
1 inappropriately place the burden of proving
2 effectiveness and reasonableness on those seeking
3 coverage. This unfairness is magnified by the
4 preference in the interim recommendations for
5 clinical trials, the most time consuming and costly
6 of scientific data collection. Reliance on
7 clinical trials, especially where other clinical
8 evidence is available to support coverage,
9 increases the time and cost involved in making the
10 coverage decision and discourages innovation. An
11 efficient coverage determination process should
12 recognize the range of clinical evidence to support
13 the coverage of items and services, and recognize
14 that for some items and services, clinical trials
15 are not appropriate. It should allow Medicare
16 beneficiaries to receive Medicare payment for
17 services and procedures, devices and technologies,
18 that have been approved by the FDA where
19 appropriate, and found by the beneficiary's
20 physician to be reasonable and necessary for the
21 treatment of that beneficiary's illness or
22 condition. We thank you very much for the
23 consideration of these very important points.
24 MS. CONRAD: Thank you so much. Debra
25 Jensen, and the next will be Kevin Connolly.
.00047
1 DR. JENSEN: Good morning. My name is
2 Debra Jensen and I am the vice president of
3 regulatory affairs, quality assurance and clinical
4 research for EMPI. EMPI is a manufacturer and
5 distributor of electric therapy and orthopedic
6 rehabilitation products. Clinical studies
7 conducted on EMPI's Innova pelvic floor electrical
8 stimulation device for the treatment of
9 incontinence were among those reviewed by the MCAC
10 Medical and Surgical Procedures Panel in April.
11 EMPI strongly supports and remains committed to
12 HCFA's efforts to create a more open and
13 predictable process for making Medicare coverage
14 decisions. However, we have significant concerns
15 about the panel and the Executive Committee
16 operation in this time of transition.
17 Our discussions with other stakeholders
18 have demonstrated that EMPI is not alone. Many
19 organizations, clinicians and professional
20 societies share our concerns regarding the evolving
21 process, and echo our frustration with the
22 deliberations and outcome of the April panel
23 meeting. In an effort to improve this important
24 process, we would like to offer the following
25 observations and comments regarding the evidence
.00048
1 standard, the duties of the Medicare Coverage
2 Advisory Committee, and the role of public
3 comments, especially those of professional medical
4 societies.
5 The interim recommendations for
6 evaluating effectiveness adopted by the MCAC
7 Executive Committee were designed to provide a
8 framework that would promote consistency within and
9 between panels, and promote accountability to the
10 public by providing a consistent framework for
11 decision making. While the Executive Committee was
12 well intentioned and should be applauded for their
13 commitment to the principles of evidence based
14 medicine, we question whether this document was
15 consistent with the mission defined for them in the
16 MCAC charter and within the framework of the April
17 21st, 1999 Federal Register notice announcing
18 HCFA's process for making coverage decisions.
19 According to these documents, the role
20 of the MCAC is to provide the Agency with
21 recommendations on whether a technology or service
22 can be considered reasonable and necessary, and
23 then to make recommendations on national coverage.
24 MCAC referrals are made when the technology or
25 service being considered is the subject of
.00049
1 significant scientific or medical controversy. The
2 recommendations ratified by the Executive Committee
3 and utilized in the April Medical and Surgical
4 Panel meeting appear to be in conflict with the
5 MCAC charter, that provides for a process designed
6 to review controversial technologies or services.
7 We believe that the process as
8 originally envisioned and laid out in the charter
9 in the Federal Register notice is a useful and
10 appropriate method for reviewing controversial
11 technologies. From our perspective, the outcome of
12 the April meeting was diminished, however, because
13 the questions and deliberations focused solely on
14 the scientific rigor of the randomized control
15 trials as reviewed by Blue Cross/Blue Shield while
16 minimizing any discussion regarding other
17 interpretations of the data and more importantly,
18 the clinical experience.
19 It is our opinion that the coverage
20 determination process can be approved if HCFA, this
21 Committee, and the specialty panels, are refocused
22 on the original goals defined for the MCAC. In
23 order for the Executive Committee and the panels to
24 be consistent with their charter and the processes
25 defined by the Agency, we respectfully suggest the
.00050
1 following for your consideration:
2 Refocus the Executive Committee and the
3 panel on the goals that were originally defined in
4 the charter and the coverage guideline published in
5 the federal notice, that is, to provide coverage
6 advice. Secondly, the interim recommendations for
7 evaluating effectiveness may be useful in
8 determining if a technology needs to be referred to
9 the MCAC. Once the technology is referred to the
10 MCAC, these questions alone are not enough and
11 should not be used as a no vote criteria for
12 further discussion. HCFA makes a referral to the
13 MCAC when an issue is either the subject of
14 significant controversy in the medical or
15 scientific community, has the potential to have a
16 major impact on the Medicare program, or is the
17 subject of fraud and public controversy. Given
18 this, it is redundant to ask the panelists to
19 reanswer a question pertaining solely to the
20 adequacy of scientific literature if as was the
21 case with PFS and biofeedback.
22 The reason the technology is being
23 referred to the MCAC is because it was already
24 determined that a controversy exists.
25 Unfortunately, the panel members in the April
.00051
1 meeting were not allowed to meld the scientific
2 evidence, clinical experience and medical judgment,
3 to make a recommendation regarding national
4 coverage for either biofeedback or PFS.
5 Given the need to assess the controversy
6 surrounding a given technology, the deliberations
7 of a panel therefore, should be more global in
8 nature, and allow for discussion and evaluation of
9 the total body of evidence, including technology
10 assessments, clinical guidelines, the testimony of
11 clinical experts, professional medical societies,
12 technical experts, and the scientific data obtained
13 from nonrandomized trials. HCFA published a
14 criteria for the evaluation clinical evidence, and
15 specifically included expert consensus; it was not
16 limited to peer reviewed literature. HCFA has not
17 articulated a rational explanation for changing
18 this policy for technologies that have been
19 referred to the MCAC. Therefore, an MCAC
20 recommendation that was based on analysis deviating
21 from this criteria would be of little use to HCFA
22 in developing a coverage determination consistent
23 with its own regulations and policies.
24 The panel needs to be provided any and
25 all comprehensive reviews of the scientific
.00052
1 literature. Simply providing the HCFA contracted
2 Blue Cross/Blue Shield assessment and not providing
3 other independent opposing assessments, such as the
4 government funded AHCPR guidelines raises questions
5 of bias and possible conflict of interest, and also
6 compromises the quality of the panel's
7 deliberations.
8 Finally, some words about the process.
9 We must require as outlined on under backup
10 regulations --
11 MS. CONRAD: Time please.
12 DR. JENSEN: -- that the public be given
13 adequate time to review, comment and testify on
14 issues relating to the technology assessments. It
15 is our sincere hope that these suggestions are
16 helpful to you as you refine your process. Thank
17 you for your thoughtful consideration.
18 MS. CONRAD: Thank you, Dr. Jensen.
19 Kevin Connolly please, and the next scheduled
20 speaker is Nicolette Horbach.
21 MR. K. CONNOLLY: I am Kevin Connolly,
22 CEO of SRS Medical Systems. We manufacture
23 biofeedback and stimulation products. I want to
24 thank the committee for giving me the opportunity
25 to present today. Does the committee have the
.00053
1 two-page written statement that I submitted?
2 When I was told that I would have the
3 opportunity to address both sets of issues, I put
4 together separate presentations, but they pretty
5 much amplify points in those written statements.
6 As you can see, I'm pretty positive
7 about this process in general, but I'm here because
8 obviously I believe the process can be improved.
9 Specifically, I think there were certain problems
10 with the April meetings. In the interest of
11 clarity, I'm going to limit my comments to
12 biofeedback, also because biofeedback is a covered
13 service and discontinuation of coverage would have
14 a great deal more significance to most people.
15 My understanding is that the purpose of
16 MCAC is to determine coverage in areas where the
17 evidence is conflicting. In the case of
18 biofeedback, however, I maintain that most of the
19 evidence has been positive. And most
20 significantly, HHS's own Clinical Practice
21 guidelines established biofeedback as a standard of
22 care, but the April meetings hardly considered
23 those guidelines. As you can see, the guidelines
24 reached a very different conclusion than the Blue
25 Cross TEC report regarding biofeedback
.00054
1 effectiveness.
2 For a variety of reasons, it seems that
3 the Blue Cross TEC report became effectively the
4 only evidence considered. Now, I think the main
5 reason was that it was the only substantive
6 evidence that considered the question of
7 comparative effectiveness. Now I know HCFA has
8 decided to analyze most procedures this way and I
9 think it's a very informative way to analyze them,
10 but I do think you have to be very careful when you
11 apply a comparative approach. If you compare two
12 procedures with the use of different populations,
13 you could end up comparing apples with oranges,
14 even if both procedures have the same clinical
15 purpose.
16 A number of the panel members were
17 frustrated by the fact that the question to be
18 voted, in their mind, changed several times, until
19 it seemed that only a negative vote was possible,
20 which rendered their clinical knowledge and expert
21 opinion superfluous. Likewise, medical societies
22 all wondered why they were invited, since what they
23 said didn't seem to be part of the evidence that
24 was considered.
25 Now I know this isn't a popularity
.00055
1 contest, but there are two points about this slide
2 worth making, the sheer number of expert opinions
3 involved, and the unanimity of their opinion. I'm
4 not sure you could get all these societies to agree
5 about any other procedure. All the evidence listed
6 here was theoretically included in the review
7 process, but in fact, none of it was effectively
8 considered. Incidentally, all of this evidence
9 supports biofeedback effectiveness.
10 Now we come to the reason I was invited
11 here for the morning. I have some suggestions. My
12 main suggestion, following everybody from Dr. Sox
13 on, is that the committee consider all appropriate
14 evidence. In the case of biofeedback, there were
15 some randomized control trials, although they were
16 used in part to limit other evidence. And one of
17 the things I think that you're going to be faced
18 with and one of the questions I think a lot of
19 people in April were asking is why there weren't
20 good studies.
21 I think the answer to that has some
22 implications. First, if a procedure is regarded as
23 a standard of care, like biofeedback, there haven't
24 been any compelling reasons to run. Second, if the
25 study is going to be run by private industry,
.00056
1 someone has to benefit financially. Most of the
2 companies in biofeedback are like SRS, very small.
3 We don't have the facilities to run studies, and we
4 wouldn't benefit financially even if we ran them,
5 because there is no meaningful intellectual
6 property left to be had; it's all prior art.
7 So, I think you should recognize that
8 the only financial incentives are for doing exotic
9 technologies like the Metronic Inner Stim, or for
10 proprietary pharmaceuticals. So I recommend that
11 the future panels consider the full range of
12 relevant evidence, and that the role of the
13 literature review be to simply organize information
14 for the panel. I believe the panel, and not an
15 analysis of literature should make determinations
16 of effectiveness. Otherwise, frankly, what are
17 they there for?
18 I know there are concerns as to how long
19 the panels might take to make decisions this way,
20 but I believe the process can be managed in a
21 timely way if the literature review is crisp and
22 inclusive and if subject matter experts are
23 involved. I believe it's critical to include
24 subject matter experts at all levels of the review
25 process. Just by way of comparison, as far as I
.00057
1 know, the Agency formerly known as AHCPR, all their
2 guidelines were prepared by a panel of subject
3 matter experts and as far as I know, their
4 conclusions are uncontroversial.
5 Now, I don't pretend to know what
6 specific factors HCFA should use to determine
7 coverage. My suggestion here is simply that
8 whatever those factors are, that you standardize
9 them and you make their weighting public. That
10 way, everybody knows the basis for the decisions.
11 My slide shows one example. At the
12 bottom right, though, is one thing I would like to
13 point out, which is, I do think if a procedure has
14 a history of coverage, that HCFA should analyze its
15 own data with regard to outcomes. I remain very
16 positive about this process. Thank you for
17 allowing me to present; I hope this was helpful.
18 MS. CONRAD: Thank you, Mr. Connolly.
19 Nicolette Horbach, followed by Tom Mesken.
20 MS. CHAPPELL: Nicolette is actually
21 stuck on a detour, so I'm going to present her
22 statement, read from it, and she will probably be
23 walking in momentarily, and you may ask her
24 questions about it. My name is Jodi Chappell. I'm
25 manager of regulatory affairs at the American
.00058
1 Urogynecologic Society.
2 I am pleased to provide the following
3 comment on behalf of AUGS and eight other
4 professional health care organizations. In the
5 interest of time, I will not read the exact list;
6 you have been provided the letter, and we have
7 provided it today with the additional inclusions.
8 These groups in coalition represent approximately
9 294,000 clinicians involved in the treatment for
10 urinary incontinence. We as a coalition support
11 and commend the efforts of HCFA and the MCAC
12 Executive Committee to provide guidance for an open
13 and consistent Medicare coverage decision process.
14 The procedures outlined in the interim
15 recommendations released in March represent a
16 positive step in the evolution and development of
17 an open national coverage decision making process.
18 We do have several concerns regarding the
19 interpretation and use of these interim
20 recommendations, based on the Medical and Surgical
21 Procedures Panel hearings in April. During the
22 hearing, narrow questions left the panel with no
23 other possible answer than no, because there are
24 few studies that make such an exact comparison.
25 The reason for such few studies is that
.00059
1 pelvic muscle rehabilitation or for that matter,
2 all rehabilitative techniques, must use some form
3 of biofeedback in order to be delivered
4 effectively. All scientific groups and research
5 efforts, such as the AHCPR guidelines, and HCFA's
6 own technology assessment report, do accept that
7 there is substantial scientific evidence that
8 rehabilitative techniques are successful, and have
9 a direct health impact in managing urinary
10 incontinence.
11 Rather that asking the April panel to
12 evaluate the adequacy of the evidence and the
13 efficacy of the intervention, it seemed obvious
14 that HCFA tailored the question to the panel in
15 such a way that prohibited the members from
16 answering yes to the effectiveness of the
17 rehabilitative interventions for the Medicare
18 population. The questions that were asked by HCFA
19 were specific to a limited number of studies that
20 met the certain criteria laid out by the technology
21 assessment for evidence based practice.
22 We believe that the adoption of
23 stringent standards for coverage should not
24 preclude consideration of either the expert
25 clinical testimony presented at the meeting, the
.00060
1 panelists' own individual knowledge and clinical
2 expertise, or the personal experiences of the
3 consumers. Moreover, this seems inconsistent with
4 the Executive Committee's procedures outlined in
5 March. In limiting its focus to peer reviewed
6 scientific literature only, the Medical and
7 Surgical Procedures Panel was essentially asked to
8 disregard clinical guidelines that had been
9 developed by the AHCPR, and expert opinion
10 developed by the national medical specialty
11 societies. For the panels of the MCAC to make fair
12 impartial decisions at the conclusion of these
13 meetings, more balanced materials and perspectives
14 should be provided to panels prior to
15 deliberations.
16 In practice, when the evidence from
17 analytic studies is poor or lacking, more relevance
18 is given to observational and/or descriptive
19 studies. The literature contains numerous
20 observational and descriptive studies which
21 demonstrate the efficacy and effectiveness of
22 biofeedback and electrical stimulation in the
23 treatment of pelvic floor disorders. The
24 effectiveness of the vast majority of procedures
25 that are covered by Medicare today for its aged and
.00061
1 disability beneficiary has not been demonstrated in
2 peer reviewed randomized trials. However, such a
3 level of scientific evidence is likely never to be
4 available for every intervention. Because such
5 evidences does not exist, HCFA questions the
6 rationale for its reimbursement.
7 We agree that continued application of
8 the Executive Committee's recommendations in such a
9 narrow fashion will prevent the MCAC deliberations
10 from achieving the desired high standards of
11 comprehensiveness and balance. Omission of
12 clinical evidence and clinical guidelines from the
13 deliberations of the MCAC could lead to an adverse
14 harmful coverage decision developed through an
15 indefensible process, and to Medicare's failure to
16 cover important and effective diagnostic and
17 therapeutic options.
18 The consequences of developing a
19 noncoverage policy for biofeedback and electrical
20 stimulation will most likely result in the lack of
21 appropriate conservative therapy interventions for
22 needy patients, especially women and the elderly; a
23 possible resurgence in the use of surgery and/or
24 drug therapy, which are most costly measures, as
25 the primary and initial modes of intervention;
.00062
1 patients who lack access to effective
2 therapeutics. The lack of poor coverage could
3 result for useful and effective behavioral and/or
4 rehabilitative intervention for Medicare patients
5 suffering from urinary incontinence.
6 The issues that HCFA is most likely to
7 refer to the MCAC are those that are the most
8 controversial. Coverage decisions should not be
9 based solely on the perspective of one faction
10 within the medical community, but rather on the
11 balance of scientific and clinical information and
12 therefore, the input of both the scientific and
13 clinical communities need to be considered and
14 valued.
15 We understand that this process
16 represents a learning experience for HCFA in
17 determining the methods to be used to evaluate the
18 effectiveness of new medical products and services
19 based on the adequacy of evidence and the magnitude
20 of clinical benefit. Not only should the formal
21 outcome of the Medical and Surgical Procedures
22 Panel not be ratified, but more importantly, the
23 flawed process must be corrected in order to
24 eliminate the prospect and perception that the
25 process was less than open and objective.
.00063
1 Ultimately, elderly patients, mostly women, will
2 suffer if needed services are not available to them
3 or covered by Medicare because the bar for
4 inclusion was set inordinately and unnecessarily
5 high. Thank you for your time.
6 MS. CONRAD: Thank you, Miss Chappell.
7 The next speaker is Tom Mesken, president of
8 Medical Alley.
9 MR. MESKEN: Good morning, members of
10 the Committee. My name is Tom Mesken, president of
11 Medical Alley. Medical Alley is a 15 year old not
12 for profit trade association, whose members are
13 from all sectors of health care.
14 I wanted to follow up on your invitation
15 from the last meeting to provide some suggestions
16 on your interim recommendations document. We
17 greatly appreciate the opportunity to be part of
18 the discussion and dialogue on shaping the
19 evolution of the Medicare coverage process. I'm
20 not going to base my comments so much on the
21 Medical and Surgical Procedures Panel meeting at
22 all, quite frankly, and instead offer three
23 specific suggestions on your document as it relates
24 to the process.
25 The first item falls under the area of
.00064
1 the document called evaluation of evidence,
2 adequacy of evidence, and external validity. Our
3 suggestions are the following: Number one, the
4 Executive Committee or the document should define
5 the terms typical practice setting and general
6 practice setting. Secondly, a panel should be able
7 to state whether the results of a study or studies
8 validate receiving an intervention for the Medicare
9 population or subgroups of that population in
10 particular practice settings, and explain their
11 reasoning.
12 Our rationale for this is that the
13 interim recommendations document states that the
14 panels will need to explain their reasoning on
15 whether an intervention is likely to apply in the
16 general practice setting. This could be taken to
17 say that the service must have studies which
18 validate its use in the general practice setting.
19 The questions may not even envision nor advocate
20 using all studies to develop their studies
21 accordingly. Given the Agency's capabilities to
22 determine what is an appropriate setting, the panel
23 should be empowered to discuss this aspect of
24 external validity accordingly.
25 Our second suggestion under the item of
.00065
1 panel operation is the structure of evidence
2 provided to the panel. We suggest that the process
3 of garnering the information for an evidence report
4 should include the opportunity for an early
5 discussion between the panel's information
6 collectors and appropriate external advocates of
7 the service. The rationale for that is that the
8 Executive Committee calls for panel member
9 involvement in the evidence report to insure that
10 the evidence report covers a sufficient scope of
11 studies, considers relevant alternative
12 interventions, and can be useful to the panel in
13 other respects.
14 In this same vein, the process of
15 collecting information for the report can only be
16 enhanced when there is an opportunity for those who
17 are most familiar with the evidence which surrounds
18 a service to share their materials and knowledge of
19 relevant studies. We can certainly appreciate the
20 Committee's interest in acquiring material that
21 will allow for independent judgments. Yet, a
22 conversation of this nature can also serve to
23 facilitate better communications on the relevance
24 and implications of the various pieces of evidence
25 that has taken place under the public testimony
.00066
1 portion of a panel meeting. Furthermore, such a
2 conversation enhances the perception that the
3 process is open, impartial and balanced.
4 Our final suggestion under suggestions
5 for panel operations and a panel explaining its
6 conclusions in writing, our suggestion is that when
7 a panel explains its conclusions in writing, it
8 should explicitly address what role, if any, each
9 of the following health outcomes played in its
10 determinations: Mortality, morbidity, functional
11 status, quality of life, and patient experience.
12 We certainly agree with the Executive Committee
13 that requiring the panel to explain its conclusions
14 in writing will help insure the integrity of the
15 MCAC procedures and judgments, and make the
16 Committee's reasoning process more explicit and
17 open, and provide internal and external
18 accountability. We also agree that it is desirable
19 that the panels specifically describe any
20 additional research that would be required to
21 strengthen that evidence.
22 That said, we believe that by asking the
23 panels to explicitly address the role of each of
24 these suggested outcome measures, the greater
25 specificity that would become part of the
.00067
1 explanation will only serve to enhance the benefits
2 that the Executive Committee sees in calling for
3 conclusions to be provided in writing.
4 The final comment I would like to make,
5 and really it is not so much -- you have heard a
6 number of comments today about the panel's ability
7 to hear evidence, a variety of sources of evidence
8 and to take action on that, based on the experience
9 of the Medical and Surgical Procedures meeting. A
10 lot of those comments have obviously been directed
11 to this Committee, but quite frankly, it is our
12 perspective that it is really the Agency's
13 responsibility to cast those questions for you, so
14 I will focus my comment to the federal
15 representative of the Committee and suggest that we
16 are very disappointed that the Agency has failed to
17 signal to the MCAC Executive Committee that it
18 wants the panels to be able to provide their
19 clinical expertise and judgment on all ranges of
20 evidence. Quite frankly, we think that the Agency
21 is a fiddler here and it is they that should be
22 setting the questions. Thank you very much.
23 MS. CONRAD: Thank you very much. At
24 this time I would like to introduce Dr. Jeffrey
25 Kang, director of the Office of Clinical Standards
.00068
1 and Quality, who would like to say a few remarks.
2 DR. KANG: Good morning. I apologize,
3 because I didn't have any prepared remarks, but I
4 would like to take this opportunity to make a few
5 comments. My hopes had been to stay here for the
6 entire meeting, but I am actually involved in some
7 testimony tomorrow which I have to prepare for.
8 I would like to say a couple of things.
9 First, in the way of announcements, I understand
10 Dick Coyne did announce the new director for the
11 Coverage and Analysis Group, Dr. Sean Tunis, and he
12 is here in the audience. He comes to us from
13 Lewin, where at Lewin he was working on the
14 development of clinical trials to inform coverage
15 and reimbursement issues, but also has an extensive
16 history at the Office of Technology Assessment, in
17 looking at health care technologies.
18 I would like to take this opportunity to
19 thank Hugh Hill for acting in the position. I
20 think that given everything that we have been
21 trying to accomplish in the last year, we have made
22 remarkable progress. I understand from the
23 testimony though, that we have a long ways to go,
24 but I really thank Dr. Hugh Hill for his leadership
25 here. I would like to point out that he actually
.00069
1 will not be lost to the Agency though, as he is
2 going to be the deputy director for our Program
3 Integrity Group with Penny Thompson. I think
4 that's very important, because as many of you know,
5 our Program Integrity Group looks at fraud and
6 abuse issues, but also runs our local contractors
7 and is responsible for local coverage decisions.
8 So I think having clinical input there is going to
9 be very important in the future.
10 I understand Dr. Hill has already
11 mentioned briefly about the notice of intent, and
12 we are -- given the complexity of this issue, we
13 did decide to add a third step to the process, a
14 notice of intent would be to a proposed rule, which
15 would lead to a final rule with regard to Medicare
16 coverage. My only observation there is given kind
17 of a lot of the interest there, we decided to
18 extend the comment period for another 30 days until
19 mid-July, and also are going to move towards a town
20 hall meeting to actually get public comments and
21 obviously, I'm sure Dr. Hill has already mentioned
22 this, we would love to get your all perspectives on
23 what's in that notice.
24 I should say just by way of, I know the
25 staff is going to work this afternoon to address
.00070
1 many of the issues that have been raised this
2 morning. I just want to say that at a larger
3 level, I am aware of the interests in having the
4 Medicare Coverage Advisory Committee and the panels
5 actually talk or give us advice around the actual
6 coverage decision and not be as limited to the
7 narrow issue of the evidence. I think that is a
8 place where we would like to go. Part of the
9 problem has been, though, we've missed what the
10 criteria are, and that these two things are very
11 interactive. And one of the things that I would
12 like to ask of not only the people in the public
13 but also people here the on the advisory committee,
14 if you can please bear with us, we are acutely
15 aware of these issues, and just bear with us. We
16 are trying to make all the efforts we can to make
17 the coverage advisory committee work in the
18 interest of Medicare beneficiaries and of science.
19 And I just wanted to say that this will actually be
20 one of the first issues that Dr. Tunis and I will
21 be trying to tackle for future panels, and the
22 Executive Committee.
23 So in some ways, I just want -- I
24 appreciate your efforts here, I know that it's been
25 rough going, but quite frankly, we are making this
.00071
1 up as we go along, and I appreciate you all working
2 with us on this. It's going to take some time. I
3 do believe though, in the long run, this is the
4 right thing to do, this sort of forum and these
5 sort of meetings, and open discussion of the
6 evidence, and trying to sort out what is adequate
7 evidence and then what do you do in situations
8 where there's insufficient evidence but a decision
9 needs to be made, are issues that we're going to
10 have to wrestle with together.
11 Thank you very much.
12 MS. CONRAD: Thank you, Dr. Kang. Let's
13 take a short break. I want to see you back here at
14 10:15 please.
15 (Recess taken.)
16 MS. CONRAD: We have a quorum.
17 DR. SOX: The next part of the morning's
18 agenda is entitled open committee deliberations,
19 and what we're going to do during that time is talk
20 about how the Committee will respond to, first of
21 all, the comments that have come in about its
22 process, and then also the prospect of our moving
23 from a group that advises HCFA on evidence to a
24 group that actually makes coverage
25 recommendations. I have asked Dr. Kang to take the
.00072
1 floor again to try to give us as clear an
2 indication of what's going to happen over the next
3 six to 18 months, so that we can plan as a group
4 about how to respond to both the public comment as
5 well as the prospect of broadening our
6 responsibility somewhat. So Jeff, if you would?
7 DR. KANG: Thanks, Harold. I'm sorry, I
8 may not have been completely clear. Because I
9 think there's a short-term issue here and there's a
10 long-term issue, and I think in the short run, that
11 we have to -- what we have intact here at the
12 Medicare Coverage Advisory Committee is a process
13 to look at the evidence, and then advise HCFA on
14 that and I also think just for the purposes of
15 continuing our work, that that's really what we
16 have to focus on.
17 The one thing I do want to assure you
18 all and the public, is that when we, HCFA gets your
19 advice on the evidence, we do not just look, quite
20 frankly, at that, but we look at the entire record
21 and that gets submitted. And those kinds of
22 things, the entire record will really be forming
23 whatever coverage decision that we make. And what
24 has always been difficult here for us, quite
25 frankly, is the lack of a rule like I referred to
.00073
1 in my comments, of what the criteria are, and that
2 really is our job, to wrestle with that.
3 I do think though, that in the long run,
4 we should be moving towards this advisory committee
5 as a forum for advice on actual coverage issues.
6 And now with the publication of the notice of
7 intent, I do think that we need to engage with you
8 all in beginning to think through how would we get
9 that accomplished from a procedural standpoint.
10 And part of the dilemma that I run into, that
11 notice of intent and those criteria are not final.
12 To the extent that it's a moving target, it gets
13 really very difficult here also. So, one of the
14 things that we had tried to do in the notice of
15 intent was really actually make, hinge all the
16 decision making points really on clinical issues,
17 which really is I think in the purview of, the
18 clinical judgment, which is in the purview of this
19 advisory committee, leading to reimbursement
20 judgments to us at HCFA.
21 At any rate, I think that the
22 interactions and beginning to transition to this
23 are going to be extraordinarily complicated, and my
24 thought here is that when Dr. Tunis is on board,
25 that I think we, HCFA will be willing to work with
.00074
1 a small subgroup or work group of the Medicare
2 Coverage Advisory Committee, to begin to think this
3 through, and then obviously whatever document comes
4 out of that, we would share to the public with, you
5 know, appropriate notice and comment and
6 discussion, et cetera. But I think that is, if
7 you're willing, that's what I would suggest as a
8 process to get from here to there in the long run.
9 But again, in the short run, I think
10 because we are going to have coverage decisions
11 that we have to deal with today and in the near
12 future, that we kind of stick with what we have now
13 with regard to your discussion of the evidence, and
14 then we obviously will internalize that and make
15 some sort of coverage decision.
16 DR. SOX: Thank you, Jeff. That's very
17 helpful. What I would like to do for the remaining
18 40 minutes or so is to discuss two issues. Then
19 we'll hear from public comment on our discussion
20 and then we will make a decision, and then we will
21 have lunch.
22 The two issues are, first of all, how do
23 we prepare this committee for going beyond its
24 current charge, which is simply to give HCFA advice
25 about the quality of the evidence, to one in which
.00075
1 we take into account the clinical evidence, a term
2 yet to be really carefully defined, and make
3 recommendations about coverage. That's going to be
4 a while before we start to do that, but we have to
5 apply, if possible, the same methodologic,
6 systematic methodology to evaluating what people
7 have commented on as, quote, the clinical evidence,
8 unquote, as we have for the scientific evidence.
9 So that's the long-term issue, preparing for a
10 transition, when we will actually be making
11 coverage recommendations, that will take into
12 account both clinical evidence as well as the
13 scientific evidence.
14 We will talk about that first, perhaps
15 hopefully briefly, and then we will go on to a
16 discussion of a process by which we will, if
17 necessary, modify our procedures, taking into
18 account the comments that have pointed out
19 opportunities for improvement in our evaluation of
20 the scientific evidence. So, we've got to fix
21 that, if need be, in the short run, while preparing
22 in the long run for a broad role that will require
23 us to be systematic and thoughtful and rigorous
24 about evaluating what many folks at the podium here
25 are calling clinical evidence.
.00076
1 So, let's first of all talk about how
2 we're going to prepare ourselves for this
3 transition to a broader role in which we take into
4 account both clinical evidence, as well as
5 scientific evidence. And I guess I'm really
6 thinking of the process, what are we going to do to
7 get ready for this? So let's talk about that for
8 about 20 minutes, and then we can talk about how we
9 adjust, if necessary, our approach to evaluating
10 the scientific evidence. So who would like to
11 begin? John?
12 DR. FERGUSON: Is it appropriate here --
13 I think that part of the problem as I see it in
14 many of these presentations was that the questions
15 that HCFA posed to the panels were based on our
16 interim report, and I, in my sense, there was a
17 sort of built-in semantic hurdle, and I, in this
18 interim report upon which these questions were
19 based, and I would just like to make a few
20 comments.
21 I really looked at this hard and it
22 bothered me, and it bothered me listening to the
23 comments when I listened to this part of the
24 incontinence thing, and the word was adequate. In
25 my view, adequacy is in the eye of the beholder,
.00077
1 sort of like beauty, and in looking in the
2 dictionary, the dictionary always gives two
3 definitions. Adequate is sufficient, but also,
4 adequate is barely sufficient, mediocre, passable,
5 okay, sort of like a C.
6 And basing -- I think that our interim
7 document is wonderful, and I agree that evidence
8 needs to be adequate and so on, and randomized
9 trials are the gold standard and so on. But what
10 it amounted to as I see it was that the questions
11 were posed in a way to force a yes or no on the
12 panel about adequacy, and that did not -- it was
13 sort of like a hurdle, and they couldn't get to the
14 discussion of the actual evidence.
15 Now I have some suggestive modifications
16 of the interim report, it's a very mild one, if you
17 want to hear it now.
18 DR. SOX: Why don't we wait, and either
19 talk about that later, or you can provide that as
20 written input to a group that might be working to
21 improve the document. Let's talk now about a
22 process for preparing ourselves for this broader
23 charge we have just heard about from Jeff. Alan,
24 do you want to comment.
25 DR. GARBER: I really have two sets of
.00078
1 comments and I could reserve one of them. I think
2 it is pertinent to discuss how the MCAC Executive
3 Committee's recommendations were actually
4 implemented by the Medical and Surgical Procedures
5 Panel in moving forward, because I'm not sure that
6 all of us have the same understanding of what
7 occurred. Certainly, my understanding doesn't gibe
8 perfectly with that of some of the public speakers
9 today, and that might give us some perspective on
10 how to go forward in terms of broadening the
11 mission.
12 The second is really right on the topic
13 of how to make this transition. I would propose,
14 first of all, that we have used the term clinical
15 evidence or clinical expertise, used in a myriad
16 ways, and I think it's safe to say that no two
17 speakers have used it in the same way, nor has
18 anybody been very precise about what that means.
19 It has been used implicitly to mean anything from
20 opinion or anecdote to evidence that wasn't
21 obtained as part of a trial. But in fact, a
22 coverage decision actually encompasses issues even
23 broader than that, and I don't think in the next 20
24 minutes, we can take a big stab at taking into
25 account all of the types of information that might
.00079
1 be needed for a coverage decision.
2 And I think that HCFA was very wise in
3 steering us away from making direct coverage
4 recommendations at this point in time, even though
5 it may be something we should be doing in six
6 months or a year. They were wise in doing so,
7 because they have not specified all the factors
8 that should be considered in a coverage decision,
9 nor I dare say, do most of us have the expertise
10 necessary to do a credible job at that at this
11 point in time.
12 So what I would like to propose is that
13 we discuss a little bit about the types of
14 information that are considered by the panels under
15 the current recommendations, and I think that the
16 current recommendations issued by Executive
17 Committee are actually far broader and for more
18 flexible than some of the speakers have implied.
19 DR. SOX: Randel?
20 DR. RICHNER: I would like to get this
21 back on sort of a practical level here. I agree
22 with what Alan is saying essentially, you know,
23 that we have to have a robust criteria, et cetera,
24 for making coverage recommendations. But it
25 concerns me gravely that he's mentioning six months
.00080
1 to a year before we ever get to that point. This
2 process was started quite a while ago. In fact, in
3 1998 was when we began this whole process of
4 thinking about how can we improve the coverage
5 process to make it more transparent. And to think
6 that we won't be able to make coverage
7 recommendations for another year appalls me.
8 Even in England, with NICE, they can
9 make a coverage decision in about four months,
10 which is clearly not what we have here. I have
11 many issues about what has happened, but basically
12 I want to say that it's important that we look at
13 this in the spirit of what was intended, and that
14 is to take all of us here and to give
15 recommendations to HCFA that they will be able to
16 use to make sensible recommendations for Medicare
17 beneficiaries. And that means using, you know, an
18 academic approach in a sense, looking at the
19 objective criteria, et cetera, but we're here
20 because we come from a lot of different
21 perspectives, and we need to all bring that to the
22 table to decide what is best for a Medicare
23 beneficiary. We have forgotten that somehow, and
24 it just concerns me.
25 We have to get back on track. We need
.00081
1 to have the process clearly defined. There is ways
2 to do that in a practical manner that we can all do
3 it, and we need HCFA's leadership and guidance to
4 do that. And so far, all I'm hearing are code
5 words, and you know, and we've lost our intent.
6 I'm passionate about this because it just seems
7 that if you read all these letters that we have,
8 everyone is concerned that we have lost what the
9 focus is here.
10 DR. SOX: Thank you. Daisy?
11 DR. ALFORD-SMITH: Yes. I have some
12 concern. I think my questions are still along the
13 line of what is the role for HCFA, versus what is
14 the role for this particular executive body, and I
15 am almost coming to the conclusion that perhaps we
16 need to really think as to whether we should be
17 making recommendations to HCFA at all, but indeed,
18 perhaps HCFA should come before us in some way to
19 present their justifications and then have this
20 body either review, approve or provide some input.
21 But I think we're switching hats in some way in
22 terms of who are the administrators and who are the
23 policy makers, versus an advisory body. And I
24 think until we resolve that, there is always going
25 to be some level of conflict in terms of what we
.00082
1 can really bring regarding our own areas of
2 expertise, and ultimately with some type of
3 recommendations to the official body, and that is
4 HCFA.
5 DR. SOX: Thank you. Yes, Bob.
6 DR. MURRAY: I would like to comment on
7 Alan's question or Alan's proposal just a few
8 moments ago, that we focus on what we mean by
9 clinical. And I think that there's a bit of a
10 problem in that clinical seems to be juxtaposed
11 with scientific, as though scientific evidence were
12 collected in a laboratory away from patients and
13 clinical is what is done in a clinic, it's face to
14 face with patients, hands-on evidence resulting
15 from hands-on treatment. I don't see it that way.
16 I understood as these interim guidelines
17 were being developed that the word scientific meant
18 any evidence collected according to the scientific
19 method. That is, it is objective, it is
20 duplicative, it is capable of duplication, and I
21 think that is really where I believe these
22 guidelines are excellent, that they focus on the
23 objective evidence. It can be clinical objective
24 evidence; I don't if I'm erroneous in that
25 assumption, but I would like to hear if others
.00083
1 believe that the focus should be scientific
2 method.
3 DR. SOX: Thank you. Alan, do you want
4 to respond or if not, then Tom first.
5 DR. HOLOHAN: Thank you. I was waiting
6 for somebody else to say that first so I wouldn't
7 be viewed as the curmudgeon that I was at the first
8 meeting.
9 It seems to me in both the presentations
10 and the reading that the term clinical evidence has
11 been applied to, and I'm quoting from some of the
12 statements, both written and verbal, expert
13 opinion, personal experience of consumers, the
14 establishment of a standard of care, and where a
15 standard of care exists, there is no necessity to
16 do scientific study. The history of medicine is
17 replete with examples of procedures, services,
18 various interventions, that were provided not on
19 the basis of evidence, but on the basis of
20 so-called expert opinion, which have been shown
21 ultimately to be of no benefit or to even be
22 harmful.
23 The best most recent example is the
24 establishment of a standard of high dose
25 chemotherapy in stem cell support for breast
.00084
1 cancer. There is no question that the personal
2 experience of some consumers supported that, people
3 who believed that this treatment benefitted them.
4 There is really no dispute that in many members of
5 the medical community, this was believed to be so
6 useful that it was unethical to do a randomized
7 trial. We had 30,000 patients subjected to this
8 treatment, only 1,000 were part of studies, and
9 yet, when all of the prospective randomized control
10 trials were in, excluding the one in which the data
11 were found to be falsified, the evidence indicated
12 that this was not beneficial to survival.
13 The various case series could be argued
14 to be clinical evidence, and certainly the expert
15 opinion of the bone marrow transplant community
16 generally was in support of this. But the fact is,
17 it didn't work. And I don't see that the simple
18 expert opinion, if it's not based on evidence,
19 whether we call it clinical evidence or scientific
20 evidence, if expert opinion is not based on
21 objective evidence, then one man's opinion is just
22 as good as any other, and I would argue that
23 something that's gratuitously affirmed can just as
24 easily be gratuitously denied.
25 DR. SOX: So it's an important reminder
.00085
1 that anecdote can lead to adoption of things that
2 don't work, and potentially harm them.
3 DR. HOLOHAN: The plural of anecdote is
4 not data.
5 DR. SOX: Alan?
6 DR. GARBER: Yeah. I think at this
7 point it really would be relevant to say a few
8 words about the role of clinical evidence in the
9 Medical Surgical Panel's proceedings, and I want to
10 clear up a couple of misconceptions along the way.
11 No panel member that I heard ever said that they
12 wanted data from multiple well designed randomized
13 control clinical trials, not were they ever
14 instructed to consider only those kinds of data.
15 What they were instructed to do and what I believe
16 they made a good faith effort to do, was to answer
17 the questions that HCFA posed to the panel. And
18 they were free to consider all kinds of evidence
19 and in fact, the evidence presented to them was not
20 limited to randomized control clinical trials, it
21 wasn't limited to good studies.
22 The evidence that was presented to them
23 or distributed to them was selective, and the
24 Executive Committee advised that the panel chair
25 should assist HCFA in selecting materials to
.00086
1 distribute, and I want to remind those of you who
2 were at the last Executive Committee meeting and to
3 tell the rest of you, that we discussed how much
4 information should be presented.
5 The first two panels that met received
6 voluminous amounts of material, and there was a
7 general belief that panelists would not be able to
8 assimilate that amount of information in any
9 meaningful way, so there had to be some
10 discretion. Does that mean that the evidence, that
11 the literature distributed to the panel was the
12 right set? No, of course we can't be sure it was
13 right, and we're learning as we're going along. If
14 I had it to do over again, I would have insisted
15 that the AHCPR guidelines be distributed in the
16 initial packet. But let me point out that, as was
17 basically suggested by the Executive Committee,
18 there were multiple opportunities for panel members
19 to suggest that materials be added, including a
20 conference call. And in fact, one of the people
21 who complained that some material wasn't
22 distributed was on the conference call and didn't
23 mention it at the time.
24 So yes, there can always be questions
25 about whether the correct set of literature was
.00087
1 included. I believe we could have done a better
2 job, knowing what I know now. I don't think that
3 we could have done a better job knowing what I knew
4 then. And to the extent that the public did not
5 get adequate time to respond, that the report was
6 not issued on schedule, that's a problem that
7 should not be repeated, and we hope it won't happen
8 again. But keep in mind that this is the first
9 time through this process, so a certain amount of
10 understanding and forbearance would probably be
11 appropriate.
12 Now as I said, a variety of materials
13 were distributed, and there was a large catalog of
14 written materials. The evidence report provided by
15 Blue Cross/Blue Shield considered a vast body of
16 evidence, but focused, appropriately I believe, on
17 better designed studies that were most directly
18 relevant to answering the question that was put
19 before the panel.
20 There was a third way in which other
21 kinds of evidence could be presented, and that was
22 during the public testimony and the documents that
23 public commenters have presented to the panel.
24 They were helpful, and the instructions that Connie
25 Conrad actually gave to the public were to make
.00088
1 sure that whatever you present is going to be
2 pertinent to the question. She didn't say what
3 type of information had to be provided, that it had
4 to be clinical trials, it had to be published peer
5 reviewed literature, nothing of the sort, just that
6 it had to be directly relevant to the questions
7 that the panels were posed with. And that was in
8 the interest of keeping the discussion on track to
9 address the questions we had.
10 So as a result, in fact, many kinds of
11 evidence were presented. The public speakers for
12 the most part, I thought did a terrific job in
13 informing the panel. The panel didn't necessarily
14 vote the way that they wanted them to, but I think
15 it's interesting that the Executive Committee
16 document was constructed to give the panels a great
17 deal of discretion in deciding how much evidence is
18 enough. It never said that randomized control
19 trials had to be considered and as you will recall
20 from our previous discussions, we wanted to make
21 sure that there was a clear understanding that
22 observational studies might be adequate, other
23 kinds of information would be adequate, as long as
24 the panel believed that they could draw conclusions
25 about the questions from the data. If they
.00089
1 believed that the information presented was from
2 studies that were too poorly designed to draw
3 conclusions, they might reject them.
4 What I saw happening at the Med-Surg
5 panel meeting was that the panelists listened
6 carefully to all kinds of information, which was a
7 wide variety of quality, at least from the point of
8 view of answering the questions, and they did
9 reject some kinds of data that public speakers,
10 advocates had wanted them to consider. It's not
11 that they ignored it, but my understanding is that
12 the people who voted no on the questions, that is
13 the questions regarding adequacy, had concluded
14 that the data and the studies just weren't
15 sufficient. So no one had told them it had to be
16 randomized control trials; that was their
17 judgment. And as an Executive Committee, we can
18 give them detailed instructions about what kinds of
19 information would be adequate to draw conclusions,
20 or we can let them reach their own conclusions, and
21 I believe they reached their own conclusions the
22 way that we had intended them to draw their own
23 conclusions. I'm not sure that everybody who was
24 on the panel, or people who weren't on the panel
25 had they served on the panel, would have voted the
.00090
1 same way that all the panel members did, or the
2 majority of the panel members did, but that's how
3 this kind of process works. If you're dealing with
4 a controversial area, not everybody will agree on
5 the interpretation of the evidence, and you saw
6 that in the discussion and the votes of the panel.
7 But the point is that I believe that the
8 panelists did consider a wide variety of evidence,
9 so-called clinical evidence, i.e., that falls short
10 of randomized control clinical trials, yet in order
11 to feel comfortable in answering the question, they
12 seemed to be looking for a higher standard of
13 evidence than was present in the literature.
14 DR. SOX: Before we go on here to more
15 speakers, we only have about 15 more minutes, and
16 sometime around half hour from now we've got to
17 decide what we're going to do next. So with
18 respect to -- I'm going to make a proposal that we
19 can respond to if it seems worthy. I think we need
20 to reconvene a methods working group, to meet and
21 try to prepare this Committee with a series of
22 procedures and guidelines about how to deal with
23 the problem of making coverage decisions, which is
24 going to include not only scientific evidence, but
25 clinical evidence and other issues that Alan has
.00091
1 alluded to, without identifying them at this time.
2 So, I am basically proposing that we do have a
3 methods committee to deal with this problem, that
4 we give them roughly six months to try to solve the
5 problem, that it's probably going to be a more
6 intensive effort than the first one, because it is
7 going to deal with standards of evidence that are
8 perhaps less well established than those involved
9 in evaluating controlled trials.
10 MS. RICHNER: Is there some reason that
11 you have chosen six months?
12 DR. SOX: Beg your pardon?
13 MS. RICHNER: Why would you choose six
14 months?
15 DR. SOX: Well, Randel, the reason for
16 proposing that is, A, I think it's going to take a
17 while, but secondly, Jeff has told us that the time
18 table for us making coverage decisions, whether you
19 like it or not, is not going to be in the next four
20 months, it's going to be more like nine to 12
21 months, or even longer.
22 MS. RICHNER: So then what would happen
23 then in the interim, to the panels?
24 DR. SOX: In the interim, we are going
25 to do what HCFA has asked us to do for the urinary
.00092
1 incontinence, which is to advise them about the
2 quality of the evidence that bears on the question
3 of coverage. So right now we have a fairly limited
4 task, one that is going to change to a more
5 comprehensive and responsible task in the future,
6 and we need to get ready for that, but we're not
7 going to do it right away. That's why I thought a
8 reasonable amount of time was in order to get
9 ready, but that could change if Jeff gives us
10 orders that we're going to start making coverage
11 decisions sooner than that. I think Mike was next
12 and then Bob, and then we need to move on.
13 DR. MAVES: Actually, I appreciate that,
14 and I rise to support that. As Alan's co-chair on
15 the Med-Surg panel, I think the last panel,
16 regardless of sort of your opinion of the outcome,
17 was handled in a much better fashion, and I think
18 the MCAC interim operating procedures helped us to
19 put together a panel process that at least seems by
20 testimonial to have gone better than the first two
21 panels. I wasn't at those panels so I can't
22 directly compare.
23 I do think it's important, and I sort of
24 view this as an evolutionary process, and I think
25 probably, Harold, your comments are in the line of
.00093
1 evolution rather than revolution, and I certainly
2 would not be in favor of throwing out the document
3 in its entirety. I do think it's important to have
4 a couple points though.
5 One of the things that I sense that we
6 need to make sure of, is that this is an open
7 process with an element of due process. After all,
8 this is the government and to a certain extent I
9 think we need to make sure that, if you will, the
10 minority opinion or if you will, the weakest voice
11 is heard in this debate. And I actually think that
12 at the panel that we had in April on urinary
13 incontinence, I understand that we heard every
14 speaker, every speaker was allowed a time to
15 present. There may be some timing problems which
16 didn't allow them to point there comments to us as
17 appropriately as they should have. But again, I
18 think these those kinds of problems can be
19 addressed in an iterated situation, and certainly
20 represent improvement in the process without
21 throwing out the basic sort of framework of where
22 we are.
23 I do think we need to approve some
24 things, and I mentioned these to some HCFA staff at
25 a meeting. We indicated that I believe when there
.00094
1 is an AHCPR guideline, or AHRQ guideline now,
2 that's appropriate and relevant, that ought to be
3 included in the packet, and I think we have largely
4 addressed that. I think it's important for the
5 panelists to receive opposing viewpoints and
6 document. One of the problems I had, we were sort
7 of only given the con side of the argument, and we
8 had to wait until the panel to get the pro side of
9 the argument. It would have been better, I think,
10 to have had documents, particularly I think from
11 the medical societies.
12 As the person from the AMA has
13 indicated, the societies put a great deal of time
14 and effort into looking at these, and I think they
15 form a basis of expert opinion that again, may not
16 rise to the same level or weight as a controlled
17 randomized trial, but nonetheless represents a body
18 of information I think is important in these
19 deliberations. We need to find some mechanism, and
20 I would applaud your decision to have a panel come
21 together to look at how we recognize the
22 information. I think the document actually allows
23 that to be stratified and I think if the panelists
24 at the meeting in April go away with the feeling
25 that they were not heard, that is an incorrect
.00095
1 perception. I think in point of fact, they were
2 heard, and I can recall specific speakers that made
3 very poignant comments, very pointed comments, but
4 as Alan indicated, when you added those together in
5 the weight of the evidence, they certainly were not
6 as conclusive as some other evidence that we had at
7 that time.
8 My last point was just simply, and it's
9 sort of pleasing to see that Dr. Kang has agreed to
10 allow us to look at the issue of coverage, because
11 I think looking at the issue of coverage, although
12 it may be more difficult, is an argument that is
13 probably more to the heart of the real work I think
14 that HCFA wants us to do, than just the science,
15 and the comments that were given at the urinary
16 incontinence panel seem to reflect that. The
17 answer to the question was no, but as I think I
18 indicated in my comments, if this were a family
19 member had this problem, would you recommend it to
20 the family member, then obviously the answer would
21 have been far different. So I think the idea of
22 proceeding with a panel, Dr. Sox, is one that's
23 probably an appropriate thing to do at this time.
24 But I would caution the Executive Committee not to
25 sort of throw the baby out with the bath water. I
.00096
1 think we've got a good process, it needs to be
2 tweaked, it will be different in a year, and for
3 those who are in this process, I think you have to
4 be patient with us as we go through this process.
5 DR. SOX: Bob?
6 DR. BROOK: I agree that we need a
7 methods working group, but I'm a little concerned
8 with what I have heard, and the last statement
9 really makes me upset, that we're going to approve
10 something where the evidence supports doing
11 nothing, but we want our own family members to get
12 it. That is so disturbing that I really believe
13 it's almost unethical to do that, and I'll use
14 really harsh words.
15 Now, there's definitely something wrong
16 with the process that we're engaged upon. First of
17 all, I want to go on the record as saying these two
18 documents represent an analysis of the literature,
19 mostly concentrated on trials with no synthesis.
20 In the time I guess they had to do them, because
21 they were pushed on the one side to be timely and
22 on the one side to be complete. I can't judge
23 whether they represent a good or bad product, but
24 they represent that product.
25 My understanding of what this product
.00097
1 would like if the panel had it, and the public had
2 it, would be all of the evidence that's available.
3 In the absence of randomized trials, I would have
4 expected cohort studies, case control studies,
5 simple follow-up studies, certainly guidelines, the
6 kind of stuff you saw presented here ought to have
7 been part of the document the panel got, and I
8 would urge that be referred to the methods group
9 for consideration. That's what we do when we do
10 the appropriateness work at Rand. If there is
11 basically randomized trials, there's no need to do
12 all the other stuff, but the bottom line is it
13 ought to have been part of that, so that's my
14 issue.
15 Now, I have a second problem, and the
16 second problem comes back to Daisy's problem of
17 what the hell we're doing here, and I don't know
18 what we're doing here anymore. If HCFA refers to
19 us procedures where none of the literature here
20 that I reviewed shows any harm, then if only
21 somebody perceived they had benefit from it because
22 they are satisfied in visiting their doctor and
23 being stimulated, then it should be covered,
24 because we have no right to deal with costs.
25 So if we are being -- the example of
.00098
1 bone marrow transplant is judging a procedure that
2 has a hell of a lot of harm versus a whole lot of
3 benefit, in trying to weigh big benefits and big
4 harms, when they're big on both sides, to reach
5 some judgment on coverage. Here we were faced
6 with, or the panel was faced with something that
7 there is no -- nobody has put forth a case that
8 biofeedback or stimulation produces harm. It may
9 waste time and cost money, it may produce poor
10 inheritance to the children and less tax dollars to
11 the federal government, but there's nothing in
12 anything that we've gotten here that it produces
13 harm.
14 I can't see how we can consider those
15 procedures under the rulings of this Committee in
16 the absence of considering cost, because I mean if
17 we just get two patients coming here and saying
18 they got better, and even if it's a Horthon effect,
19 how are we going to judge this? And that's what I
20 think this ethical dilemma is all about. Medicine
21 has side effects, surgery has side effects, try it
22 for six weeks and see if you get better, a simple
23 question, and in the absence of cost you should
24 cover everything. This would have covered vision
25 rehabilitation, every kind of rehabilitative
.00099
1 service, physical therapy, your knee hurts, you
2 know, why not get a knee brace and try physical
3 rehabilitation. I'm concerned with this problem,
4 I'll state that categorically, and I think we have
5 to deal with that issue.
6 Now, the second part of the problem is
7 the fact that if it is indeed correct that it's in
8 nobody's interest to produce information, if we
9 vote no to ever produce new information about this
10 procedure or these techniques that would benefit a
11 large number of people, because there's no
12 company's financial interest, then I don't think
13 ethically we can deny coverage, in the absence of
14 the federal government saying that we're going to
15 use part of that $2 billion of extra money that
16 went to the NIH to actually do the studies quickly
17 to answer the question. Because we are now in a
18 situation saying that we have an orphan technology
19 that nobody will approve that people are using, and
20 basically there is testimony it works, and we have
21 no strategy to actually provide the knowledge that
22 this is going to work. So we need to force the
23 government to make some consistent policy here if
24 we are going to operate in a way that makes sense.
25 The FDA is in a very different
.00100
1 circumstance relative to drugs, and we need to do
2 something that makes some consistency here as we do
3 this. So, those would be both my general and
4 specific comments about what needs to be done.
5 DR. SOX: Thanks, Bob. Linda, I think
6 you were next.
7 DR. BERGTHOLD: Yeah. I wanted to go
8 back a little bit to Dr. Maves and Randel's
9 comments. I sat on the very first panel, the
10 multiple myeloma panel, and I don't know how many
11 of you know that actually, I believe HCFA has
12 issued some coverage determinations about that.
13 It's on the web site. I don't even know if this
14 Executive Committee knew that.
15 Our panel really struggled with the
16 process and Tom can attest to that. I think we
17 have improved the process a lot through the use of
18 the interim guidelines, and I do see this as a work
19 in progress and want to keep working on it. And
20 while I am concerned about the public, the public
21 is not the same as the industry manufacturers who
22 make the products. The public is not the same as
23 the advocacy groups who necessarily advocate for
24 that particular product. And you know, I'm
25 struggling, I think all of the consumer reps are
.00101
1 struggling with our role on this, but I see my role
2 as a very tough one, and that is sort of looking at
3 the overall good for beneficiaries.
4 I would say that what happened with the
5 multiple myeloma panel, as awkward and messy as it
6 was, will result in better care for Medicare
7 beneficiaries, because people will not be given
8 painful very complex procedures that are really not
9 going to extend their lives in the same way they
10 were before our panel met. And to me, that's the
11 bottom line, and the bottom line is we are deciding
12 about things, and maybe the incontinence issue is
13 just not at the same level of life or death, so it
14 makes it more complicated to talk about it. But on
15 the multiple myeloma issue, to be telling people
16 that they are not going to indeed live more than
17 three more months with a procedure and it's going
18 to be extremely painful and affect their quality of
19 life, and the evidence shows that they're not going
20 to survive this, that to me is an issue that these
21 panels really need to be taking up, and hurrying it
22 does nobody any good.
23 So I feel like we ought to -- I mean, I
24 feel an urgency about it, Randel, but not enough of
25 an urgency to do things that would be mistakes and
.00102
1 that would harm patients in the long run. So I
2 think we ought to keep working on this, and
3 recognize that we're in a process that we are
4 inventing this process as we go along, and we're
5 going to have to accept that we're going to make
6 some process errors.
7 DR. SOX: Thank you. We just have a
8 couple minutes before we give a chance for public
9 comment. I want to make sure that anybody who
10 hasn't already had a chance to speak gets a chance
11 to speak, however briefly that might be. Anybody
12 else? Leslie?
13 DR. FRANCIS: I just want to be sure
14 that we add to this, there are really two issues
15 here. One is the substance of the panel
16 recommendation and the other is the openness of the
17 process, and I would hope that when you appoint a
18 working group, you think about -- that group thinks
19 about the second half of our guidelines as well as
20 the first half, because I was not part of the
21 research panel, but my sense is that a lot of the
22 upset about it isn't the substantive criteria, it's
23 whether stuff got out there fast enough for people
24 to talk about, which actually goes against
25 Randel's.
.00103
1 DR. SOX: Okay. Well, before we enter
2 into the period of public comment, I'm just going
3 to summarize by saying that we're going to
4 reconvene a methods subpanel or subcommittee, and
5 it's going to have two charges. The first one, I
6 think the most immediate one is to respond to
7 issues that have been raised about the process for
8 evaluating evidence; in other words, how we do our
9 assignment as HCFA has asked us up until now. And
10 hopefully, we can fix some of the issues that came
11 out in the incontinence panel procedure quickly so
12 that subsequent panels that deal with advising HCFA
13 on evidence can do so with benefit of learning from
14 the lessons of the Med-Surg panel. That's number
15 one.
16 And then the next step will be to tackle
17 the big issue as I raised earlier, of how we make
18 coverage decisions that are systematic,
19 transparent, even handed, consistent from
20 technology to technology. That will be a bigger
21 issue, but one I think we need to take on only
22 after we make a fix on the issues that have been
23 raised about evaluating the evidence, which I think
24 will be fairly straightforward.
25 So that's the proposals. I am going to
.00104
1 ask you to think about it and hopefully affirm,
2 after we have a period for public comment.
3 MS. RICHNER: I would like to say one
4 more thing, is there time? I mean, certainly I'd
5 like to defend my statement about the timing. Once
6 again, it's related to process. I fully appreciate
7 all of the issues associated with openness, careful
8 evaluation of all of the evidence, I appreciate our
9 time that we're going to be spending on developing
10 the methodology and this subcommittee. That's not
11 the point. The point is that we haven't had clear
12 guidance from HCFA from the very beginning in terms
13 of what our role is, and a lot of that has to do
14 with coverage decisions.
15 And one more point that's very
16 important. Dr. Hill in his opening statement when
17 he was asked why was this particular technology,
18 urinary incontinence, sent to this Committee, he
19 said because of the impact on the program. I do
20 have to say one thing. To me, that is very
21 telling. Once again, that brings up what Bob Brook
22 just said. What that is, using coverage to control
23 volume to me sort of defeats what we're all about
24 here. There's lots of way that HCFA can control
25 volume beyond using the coverage process for that.
.00105
1 Thank you.
2 DR. BROOK: I would just like to go on
3 record that I don't agree with that. The most
4 effective way to control volume is coverage, and
5 the most effective way -- and somebody eventually
6 down the road, and that's why I'm sure that HCFA
7 didn't deal with this up front -- when there are a
8 large number of people that can use labor intensive
9 services over a long period of time, that have
10 extraordinarily small benefits, somebody is going
11 to have to step up to the plate and say this is not
12 covered, you can get it outside the system but it's
13 not covered. So I don't agree with you, Randel,
14 about what the issue is. I do agree that we are
15 confused at this moment, and because of our role
16 here in what we're doing and therefore, this
17 confuses the whole process, even to the whole
18 question of what the evidence is.
19 DR. SOX: Okay. Before we move in to a
20 chance for public comment, maybe I could ask Jeff
21 or Hugh to comment on this discussion and give us
22 some guidance about whether we're going in the
23 right direction here, from your perspective as the
24 Agency that we are trying to advise.
25 DR. HILL: Well, I will take the first
.00106
1 stab at that and then Jeff, maybe you can clarify.
2 I am not confused at all. We in the charter said
3 quite clearly that we wanted the Executive
4 Committee and the MCACs to comment on the
5 scientific evidence. The charter also says that
6 the committees may be asked about other things, but
7 it says they will be asked about the scientific
8 evidence. And Dr. Kang has indicated to us a
9 future direction, but for now we're asking for
10 clarifications on the evidence, and I appreciate
11 the committee's willingness to go forward with us,
12 and look forward to where we're going in the
13 future, and I hope that you will set up a
14 subcommittee and a working group that will work
15 towards that.
16 DR. SOX: Jeff, do you have anything to
17 add?
18 DR. KANG: Yeah. This has been very
19 helpful for me to hear the conversation here.
20 First of all, just in answer to Randel's question,
21 coverage is not all about controlling volume. What
22 coverage is about is whether or not the benefits
23 should be available; that's the threshold
24 question. Then once the benefit is available to
25 Medicare beneficiaries, then there are whole
.00107
1 subsequent issues about trying to control volume.
2 But I think the threshold question, and this is in
3 the notice of intent, and maybe what might be
4 useful in some future MCAC meeting is for us to
5 come and present that notice of intent, because it
6 begins to flesh out the questions that both
7 Dr. Alford and Dr. Brook is wrestling with, what is
8 the role. But I think coverage really is about the
9 threshold question as to whether the benefit should
10 be available, and then there are subsequent
11 questions about volumes and appropriateness on an
12 individual case by case basis.
13 I agree with Dr. Hill that short term,
14 your charge is very explicit. We want you to focus
15 on the evidence. This largely has been because of
16 the lack of criteria, and part of our dilemma, and
17 I understand Bob's impatience with us, but part of
18 our dilemma is we have not been clear about the
19 criteria, and we're working on that.
20 The only thought, Harold, for your work
21 group is in listening to discussions, I actually
22 wanted to follow up with what Dr. Murray and I
23 think it was Tom actually, Dr. Holohan was saying,
24 I think there is some confusion about scientific
25 evidence versus clinical evidence, and I think we
.00108
1 would really appreciate your advice on that issue.
2 Are we, from an evidentiary state, is it about the
3 scientific methodology, or in fact is this the gray
4 area of kind of consensus and expert opinion and
5 recommendations, so that, I think would be very
6 helpful also. I don't know if you were referring
7 to that in terms of when you say the process
8 itself, but I think it's a different question in
9 the process, a very substantive question about
10 what's good evidence.
11 DR. SOX: Thank you. Well, the podium
12 is now open. We have about 10 minutes or so for
13 anybody in the audience who wishes to comment on
14 whether we're heading in the right direction or
15 veering dangerously off the path.
16 Anybody who wants to speak, please get
17 up behind the mike, and that will give a chance for
18 me to gauge how many people want to talk, so that
19 we can allot the time fairly.
20 DR. STANTON: Marshall Stanton. I want
21 to start by complimenting Dr. Garber on his
22 chairing of the Med-Surg Panel. I think that he
23 allowed a lot of open discussion that was very
24 useful for everybody that was there, both
25 concerning the urinary incontinence question, and
.00109
1 also, he allowed us to discuss some of the process
2 and some of the frustration.
3 I will differ a little bit with Dr.
4 Garber in at least not letting people here leave
5 with the impression that everybody was satisfied
6 with the process. And I don't want to put words in
7 his mouth but I want to make sure that people
8 realize there was a lot of frustration with the
9 process, and I would encourage those who are
10 interested to look at the transcript at the end of
11 the first day and the beginning of the second day
12 particularly.
13 Dr. Garber also made comments about the
14 conference call where people were given the
15 opportunity to request additional information if
16 wanted. Now I for one, being a cardiologist at
17 that time, and participating in that conference
18 call, did not understand enough about the urinary
19 incontinence area to be able to know which pieces
20 of evidence I would want to request. I certainly,
21 from my academic upbringing and scientific
22 background, know how to look at evidence when it's
23 presented, but I'm dependent on the other experts
24 on the panel and on HCFA to present a balanced view
25 of the literature that's out there, and the
.00110
1 opinions that are out there, and not just provide
2 the Blue Cross/Blue Shield TEC report.
3 Regarding Dr. Sox's formation of the
4 methods subcommittee, I think that's a very good
5 idea, but considering listening to everybody's
6 opinions, how diverse this group is, I would
7 encourage that subgroup to be diverse as well, and
8 I think it would be important to have industry
9 representation on that group.
10 And lastly, I just want to make a quick
11 comment on Dr. Holoran's rejection of expert
12 opinion. I found that interesting but a bit
13 bothersome. I think many people certainly respect
14 the opinions of experts when conclusive data are
15 absent, and I find it interesting that I think this
16 Executive Committee as well as the panels are
17 panels of experts that were brought together to
18 render expert opinion.
19 DR. SOX: Thank you very much. Center
20 mike please?
21 MS. WOOLNER: My name is Barbara
22 Woolner, and I am a clinician, I am not a
23 scientist. I would like to make two points only.
24 Number one, I would like to reiterate
25 what Dr. Stanton has just commented on, and urge
.00111
1 you to look at the dissatisfaction of the panel
2 members, particularly on the second day. The first
3 hour of the entire meeting was spent on their
4 dissatisfaction and frustration with the process.
5 I would like to make one other comment
6 about the AHCPR comments that were omitted and
7 point out that earlier, I believe Dr. Hill or
8 Dr. Garber were asked this morning why they
9 enlisted the Blue Cross/Blue Shield Technology
10 Evaluation Center to issue a report, and their
11 reason was that they wanted to look at the new
12 evidence when in fact, the six studies that were
13 looked at were done in 1993, 1993, 1996, 1996,
14 1986, and 1983. Certainly, there was not a lot of
15 new evidence. Thank you.
16 DR. SOX: Left mike, two minutes.
17 MS. SHERMAN: Sandra Sherman again, from
18 the AMA. I just want to underscore what Dr. Kang
19 was talking about in terms of the importance of the
20 criteria that HCFA is going to establish for making
21 Medicare coverage policy and the interaction of the
22 criteria that are going to be used with the
23 evidence that this Committee is going to consider.
24 Certainly we want the basic effectiveness of
25 procedures that are proposed for coverage to be
.00112
1 demonstrated through scientific methods and
2 available in scientific literature. But when the
3 panels have to extract from that science to
4 determine how effective would this procedure be in
5 the Medicare population, in our aged and disabled
6 beneficiaries, when we look at criteria that HCFA
7 is proposing in its notice of what is the impact,
8 not just on mortality and morbidity, but on quality
9 of life.
10 Is a certain procedure that's proposed
11 for coverage more convenient for patients, does it
12 improve compliance with prescribed therapies?
13 These kind of questions are going to require you to
14 have some expert opinion. They are not going to be
15 demonstrated, I don't believe, in the scientific
16 literature. Basic effectiveness, yes, but these
17 other questions, you're going to need to take a
18 broader look at what's available.
19 DR. SOX: Next, left mike.
20 MR. MESKEN: Tom Mesken, Medical Alley.
21 There seems to be some question among the Committee
22 about what its role is, and I'm sure everybody can
23 cite different things from the charter, and the
24 preamble to the interim recommendation, but in the
25 charter it explicit says that panels may be asked
.00113
1 to develop recommendations about specific issues of
2 Medicare coverage and/or to review and comment upon
3 proposed or existing Medicare coverage policies.
4 And in the discussion paper, in its preamble, the
5 Agency says that HCFA views the materials being
6 developed as helping to insure that MCAC panels
7 have complete discussion around the questions posed
8 to them by HCFA.
9 And I appreciate that you want to set up
10 a methods subcommittee to address these issues of
11 both the process and the larger coverage issue, but
12 I think whether it's the leadership of this
13 Committee or otherwise, they should put strong
14 pressure on the Agency to get explicit instruction
15 to address this so-called gray area, as Dr. Kang
16 suggests. Your ability to do that comes from the
17 Agency, and for them to put it back on you and say,
18 well, help us think about what gray area is, does
19 get to the point of can you address gray area or
20 not. And I think that you need to put strong
21 pressure on the Agency to do that. To do otherwise
22 is to wait for criteria regulation, which will
23 probably never see the light of day. Thank you.
24 DR. SOX: Thank you. Next?
25 MR. J. CONNOLLY: Jerome Connolly, with
.00114
1 APTA. Just very briefly, Dr. Kang has indicated
2 that there is some confusion and acknowledged that
3 there is some confusion over what is scientific
4 versus what is clinical, and I think if you review
5 the transcript, that there was confusion on the
6 panel members' behalf, that in fact they could not
7 use clinical evidence, they could not use clinical
8 expertise, their own clinical judgments. They did
9 not feel the capability or latitude to call upon
10 that clinical experience in making the judgment and
11 answering the very narrow questions.
12 There was a primer provided by Dr. Zarin
13 prior to their discussion that indicated what
14 scientific evidence and how it was developed, and
15 that was the background and framework by which this
16 panel operated. The panel was not familiar with
17 the AHCPR clinical practice guidelines before they
18 came in here. Many of them acknowledged during the
19 meeting that this was the first time they had heard
20 about them. So in a few hours in the course of a
21 day, to try to become familiar with clinical
22 practice guidelines that members of the scientific
23 and medical community have reviewed and said have
24 good clinical applicability and utility, and to not
25 be familiar with those in order to make a decision
.00115
1 is not really fundamentally correct.
2 Dr. Garber has indicated that the
3 correct set of information was not distributed. He
4 acknowledged that if he had it to do over again, I
5 think he would do it differently and I believe that
6 he would, and said he has.
7 The questions were too narrow.
8 Everybody has said that. The question was changed
9 the morning of the meeting, the first meeting on
10 biofeedback, and in fact, the technology assessment
11 was not posted on the web site allowing written
12 comment until after the written comment period had
13 expired. These are not small refinement issues.
14 These are not issues to tweak. These are
15 fundamental issues that need to be brought up and
16 need to be changed, and any decision that you make
17 or you allow to go forward, allow to be ratified on
18 the basis of a process that is fundamentally flawed
19 will be a fundamentally flawed decision. Thank
20 you.
21 DR. SOX: Last speaker?
22 MR. GEIGLE: Thank you. My name is Ron
23 Geigle. I'm a writer on technology issues, and I'm
24 representing only myself. And I think I have a
25 philosophical point. I've attended almost all MCAC
.00116
1 meetings, including almost -- I missed one panel
2 meeting. And the issue obviously comes down to
3 adequacy of evidence, and the philosophical issue
4 that troubles me as a consumer is, and I think this
5 especially applies to the methods panel Dr. Sox is
6 talking about is, what is the answer when we
7 consistently conclude that the evidence is
8 inadequate? Do we always say no?
9 Because I think a lot of, as I listen to
10 clinicians speak, and have done a lot of research
11 over the years, randomized control clinical trials
12 are generally not available because if they are
13 available, there is going to be obviously
14 continuing disagreement in many cases. Then as I
15 listen to the panels, it's not just randomized
16 control clinical trials, it's extensive randomized
17 control clinical trials; it's randomized control
18 clinical trials that have consistent findings where
19 there is no potential for bias, and the potential
20 for bias was a critical element in the urological
21 panel.
22 So therefore, I ask you to think about
23 as part of the methods, the issue of when the
24 evidence is quote-unquote inadequate, is the answer
25 no, or does the federal government allow what Dr.
.00117
1 Brook was saying, have some responsibility to jump
2 into this and try to help resolve this, whether
3 it's NIH or elsewhere? Many of the people on the
4 urological panel said, why isn't there more
5 randomized control clinical trials, where is the
6 evidence, why isn't is being done? What do we do
7 when we run into that?
8 And one final point is, this is not just
9 an issue for MCAC. The first, in a decision tree
10 on the NOI on coverage, the first question relates
11 to advocacy of evidence. If no, we stop. What do
12 we do about that?
13 DR. SOX: Thank you. Before we move
14 back into the deliberation of the panel, I would
15 like to call upon anybody in the office who wishes
16 to help us define clinical evidence and distinguish
17 it from scientific evidence, to write to us and
18 help us, because that's going to be one of the
19 tasks that the methods work group undertakes, if
20 this Committee decides to go that route. So this
21 is an open invitation to help this Committee try to
22 define what's meant by clinical evidence.
23 Well, what I would like now is to here a
24 motion from the Committee about proposed procedures
25 for going forth to deal first of all, with
.00118
1 improving our procedures for evaluating evidence,
2 but then also moving on to the larger issue of
3 procedures for actually making coverage decisions.
4 MS. CONRAD: Before you proceed with
5 that, I am obligated to read something for the
6 record, as follows: For today's committee meeting,
7 voting members present are Robert Brook, Thomas
8 Holohan, Leslie Francis, John Ferguson, Robert
9 Murray, Alan Garber, Michael Maves, Frank
10 Papatheofanis, Ronald Davis, Daisy Alford-Smith,
11 Joe Johnson. A quorum is present. No one has been
12 recused because of conflicts of interest. You may
13 continue, Dr. Sox.
14 DR. SOX: Thank you. I have to remind
15 the nonvoting members of the panel that they are
16 not eligible to engage in the discussion of the
17 motion that we will discuss, but hopefully they
18 have had adequate opportunity to give us their
19 input before the discussion.
20 So I would like to hear a motion that we
21 can discuss and hopefully act upon. Ron, you're
22 one of our star motion makers. Do you want to give
23 it a shot?
24 DR. DAVIS: Well, I would move that we
25 form a methods subcommittee to address the issues
.00119
1 that you just mentioned a few moments ago, as our
2 court reporter could read back to us.
3 DR. MURRAY: Second.
4 DR. SOX: Okay. So we have a motion to
5 form, or reform a methods subcommittee, to deal
6 with both short term issues of improving our
7 ability to evaluate the evidence and then as a
8 second assignment, to prepare us with procedures
9 and approaches to evaluate clinical evidence and
10 other issues that may bear on our responsibility to
11 make coverage decisions, something that is still a
12 ways off, but for which we need to be prepared.
13 So, time for discussion of that motion.
14 Anybody have discussion? Frank, then Bob.
15 DR. PAPATHEOFANIS: I wanted a
16 clarification on one of the potential
17 responsibilities of this working group, and that
18 goes back to the framing of questions that each of
19 the panels are asked to address. I remember from
20 one of our initial meetings, that my understanding
21 was that HCFA would provide us as panels with
22 questions, but we would be able to provide some
23 feedback to HCFA until we finally came to some
24 consensus. And there seems to have been a little
25 bit of an imbalance, or at least a perception of
.00120
1 imbalance in the types of questions that we're
2 asked, to the point where a lot of the letters
3 seemed to suggest from the public that the
4 questions were setting up a no answer.
5 And so, a question I have is, is part of
6 this working group going to be charged with the
7 responsibility of working with HCFA in refining the
8 questions and selecting appropriate ones?
9 DR. SOX: I perceive that this committee
10 is going to read every single letter that's come in
11 commenting on our methods, both generally as well
12 as in respect to the Med-Surg Panel, and is going
13 to consider each one of them and whether to adopt
14 it or not. So -- and certainly, that's among the
15 issues that were raised by those who wrote to us.
16 Bob?
17 DR. MURRAY: First, I would like to say
18 that I support the motion and intend to vote for
19 its passage. But secondly, I would just like to
20 comment that many of the comments that we have
21 heard this morning urge that the Executive
22 Committee develop guidelines that are more open and
23 allow consideration of a wide variety of opinions
24 as well as evidence. I would like to comment that
25 the reason we have the interim guidelines is that
.00121
1 the first two panels that considered questions did
2 so with no guidance, and as a result, the Executive
3 Committee refused to ratify, or decided not to
4 ratify the votes or the recommendations of the
5 panels.
6 I believe that the guidelines need to be
7 prescriptive, and I would urge that the working
8 group keep that in mind, and that they be clear so
9 that the process remains predictable and
10 consistent.
11 DR. SOX: Thank you. Leslie?
12 DR. FRANCIS: I just want to comment
13 that I hope that the working group will be balanced
14 in the folks who are on it, and that there will be
15 opportunity for going back and forth between the
16 working group and the Executive Committee as this
17 process continues.
18 DR. SOX: Yeah. I envision first a
19 representative committee, and I would point out for
20 everybody that both the consumer representative and
21 the industry representative were on the previous
22 working group, so there was balance. But I
23 envision one group that's small enough to get
24 something done, but getting feedback on a
25 continuing basis from the whole committee. Other
.00122
1 questions or comments? Bob?
2 DR. BROOK: I wanted just a
3 clarification. Alan, in the report of the two
4 subcommittees, if I remember and I don't have the
5 document in front of me, it said something like the
6 evidence was inadequate to support doing procedure
7 X or Y. It wasn't a question of this is the level
8 of evidence that's available, it was that it's
9 inadequate, just inadequate. So was that, the
10 reason you responded that way because HCFA demanded
11 an adequate or inadequate response, as opposed what
12 the preventive task force has done, and other
13 panels have done with A, B, C, D, E, and 1, 2, 3, 4
14 and 5?
15 DR. GARBER: We were certainly trying to
16 answer the questions, so the narrow answer to your
17 question is yeah, we were guided by what the
18 question was that HCFA proposed. But at the same
19 time, as I understood the panelists' thinking on
20 the matter as they publicly expressed, it was that
21 they thought they couldn't draw conclusions, in
22 part because -- there were randomized trials by the
23 way, and we will be talking about this more this
24 afternoon I presume -- but there were issues of
25 conflicting results and also questions about
.00123
1 various kinds of biases like ascertainment bias,
2 which might have made it impossible to determine
3 whether the treatment was really effective. So I
4 think the individual panelists didn't go through
5 study by study to say their reasons.
6 DR. BROOK: I want to come back to the
7 general concept, because I am trying to figure out
8 what has to be done with this working group motion,
9 and that's why I'm asking you this question.
10 Virtually every other expert group that's looked at
11 evidence, when they have rated evidence, has graded
12 the level; they've never come up with evidence is
13 adequate or inadequate, they've graded the level of
14 evidence, so that the evidence might be that this
15 is only supported by clinical opinion or behavior,
16 or this is not supported even by that. But you
17 just said that the statement as read in the minutes
18 of this meeting, is the evidence is inadequate.
19 And I wondered, because I'm just wondering how that
20 came out as opposed to a gradation statement.
21 DR. GARBER: That's the way it was
22 posed. Let me say that not every expert group uses
23 that scoring system that the preventive services
24 task force uses. And a difficulty in applying that
25 scoring system is it gets a little confusing when
.00124
1 you have studies that are randomized but have
2 various kinds of biases, because it's a very
3 approximate rating system and when you have
4 conflicting results, although that's usually a
5 strength of evidence issue. But, I personally
6 think it would be worth exploring from the methods
7 group, I think it's worth exploring doing formal
8 grading or not, don't forget that if they had
9 concluded that the evidence was adequate, there
10 would have been a second exercise of assigning it
11 to one of the seven categories. And I thought that
12 the reasoning in doing it this way was primarily
13 that it wasn't necessary to go into the gradation
14 steps if the panelists felt they couldn't draw
15 conclusions about effectiveness. But it would have
16 helped to specify the reasons for it, I agree, if
17 they had been grading it.
18 DR. SOX: So at present we have a system
19 for grading effect size. It sounds like we should
20 put on the table the issue of a system for grading
21 the adequacy of the evidence, to make a conclusion
22 about whether something works or not, and that's
23 another task for this work group.
24 DR. GARBER: But I think that if you
25 were to say you must put the adequacy of the
.00125
1 evidence into, say, U.S. preventive Services Task
2 Force categories, there would have been fairly
3 straightforward, I believe, if I understand the
4 categories correctly, because there were multiple
5 randomized trials on both of these. There were
6 flaws in things like blinding and outcome
7 measurement that at least were critiqued during the
8 meeting, and then they have mixed results on
9 effectiveness. And you might alter the balance by
10 saying you excluded or included different subsets
11 of studies, but it wouldn't be that hard to go back
12 and come up with it.
13 DR. SOX: Well, anything we could do to
14 improve the transparency of our deliberations, I
15 think is a plus.
16 DR. GARBER: Right. But let me just add
17 to Bob's suggestion that this Committee should
18 think of all the ways that they can improve
19 transparency, and I think the grading scheme is one
20 set, but the other is that we could have something
21 that's more specific about where the critical areas
22 are, where we would need more information so we
23 know if a particular new study is likely to resolve
24 the problem, or to give some guidance as to what
25 kind of study would need to be done. And I think
.00126
1 we could do a good job of that if we made that an
2 explicit part of the process.
3 DR. SOX: Bob, do you want to respond to
4 what Alan said?
5 DR. BROOK: I would just wanted to,
6 based on this, I would like this working group to
7 do two things then, and I don't know how your
8 motion quotes that: One is to actually help
9 prepare for HCFA, because under the current rules,
10 it sounds like we're helping make the coverage
11 decision. I'm going to interpret this in the near
12 term that way. So we want to produce the best
13 written document that would help people at HCFA
14 understand the evidence so that they can make a
15 decision about whether to cover or not cover a
16 procedure. So we want that document to be fair,
17 unbiased, open, all of the things that we talked
18 about. We ought to reexamine what we did to do
19 that.
20 I would urge that we take a second
21 step. We would also like to develop a guidance to
22 the panels to say that in the case that the
23 evidence is inadequate, not because there are
24 substantial negative trials or negative results but
25 that it's just inadequate, that the panel must
.00127
1 discharge its responsibility by suggesting who
2 needs to do what or what needs to be done to
3 produce an evidence base that this process then
4 could be revisited and this topic decided again or
5 examined again, with a time frame and a specific
6 work plan. I would like to move us to a point
7 where we don't discharge our responsibilities by
8 saying, hey, we haven't studied this enough. I
9 don't know if that's in the scope of what we're
10 supposed to do or not, Hal, but that would be an
11 interesting document that should go to HCFA in
12 terms of the second part of this.
13 DR. SOX: I think all of us would like
14 to have a better evidence base for medical practice
15 and that suggestion would hopefully put some
16 pressure on somebody to provide that evidence
17 base. Yes, John?
18 DR. FERGUSON: Is it my understanding
19 that this methods subgroup will address possible
20 changes to the interim document in trying to come
21 to grips with the evidence and the coverage issues,
22 this methods group will consider modifications of
23 this to enhance the process?
24 DR. SOX: That's right. I think
25 everybody agrees that the present document is a
.00128
1 good beginning and it needs to be tweaked, for lack
2 of a better term, to make it better in helping us
3 to advise HCFA about the quality of the evidence.
4 And then it probably needs some fairly major
5 changes as we prepare for our broader assignment
6 from HCFA, which is to make coverage
7 recommendations.
8 DR. FERGUSON: I want to be sure that my
9 comment is related to making sure that this is
10 enhanced to help this document. The second thing
11 is, the questions that HCFA poses to the panels, I
12 think I would like to recommend or suggest if it's
13 not already implied, that this methods group also
14 consider these questions, and to work with HCFA on
15 the kinds of questions.
16 I say this with some, I won't say
17 emotion or experience, but because in the consensus
18 program at the NIH, the questions were paramount.
19 We spent the majority of planning trying to arrive
20 at what questions would bring out the best
21 discussion of the evidence, and so I think that's a
22 very important part of this methods group.
23 DR. SOX: Yes, Bob?
24 DR. MURRAY: I have heard nothing in the
25 motion that would rescind any of the current
.00129
1 guidelines, so I just wanted to clarify. It's my
2 understanding that any panels that meet between now
3 and the time when the new guidelines are approved,
4 will still operate under the current guidelines; is
5 that correct?
6 DR. SOX: That's correct, and I think
7 that the working group, methods working group,
8 ought to have a pretty tight time line.
9 DR. BROOK: I don't believe that.
10 DR. SOX: What?
11 DR. BROOK: I disagree with that and I
12 was just wondering, to beat the process, that this
13 document, the next document that looks like this,
14 ought to include the kinds of pieces of information
15 -- this is just a list of the randomized trials
16 that were done, or the exclusion of the randomized
17 trials. That's what was given to the panel in
18 terms of background information. I think that's
19 inadequate in terms of -- unless it's a field that
20 has had multiple randomized trials, that's all
21 that's in this document, and it's inadequate, from
22 the perspective of the testimony we heard today,
23 and I would urge that -- I don't know where the
24 stage is for the next document or the next meeting,
25 that this document needs to be a broader document
.00130
1 that goes to the panel before the panel meets,
2 period. It's not state of the art. If the
3 randomized trials were there, we wouldn't be in
4 business. So I disagree. I mean, I basically
5 heard that we have an inadequate written process
6 here and for that reason, either this document gets
7 expanded for the next panel rapidly, or we delay
8 the next panel. Sorry about that.
9 DR. SOX: Thank you, Bob. I'd like to
10 see if there's any more comments, and then we can
11 take a vote. Tom, did you want to comment?
12 DR. HOLOHAN: Just a comment to
13 Dr. Brooks. Virtually all of the evidence, save
14 one piece of published information, given to the
15 myeloma panel, was not randomized trial.
16 DR. BROOK: But that evidence wasn't
17 synthesized any way that anyone can understand it.
18 So the question is, can we produce a synthesis that
19 people can understand that contains all of the
20 evidence, that labels the evidence for what it is.
21 I mean, I'm not arguing that the panel ought to
22 make -- by the way, I'm not disagreeing, I'm not
23 saying how the panel ought to make its decision
24 about the adequacy of the evidence, I think our
25 guidance is perfect on that. What I would like to
.00131
1 know is that no matter if you're the person, the
2 consumer, the manufacturer or the panelist, when
3 somebody reads a synthesis of the information, that
4 they will say that this is something that I have
5 faith that has been done in an open complete
6 process, period. And not to have the guidelines,
7 not to have the endorsements, not to have all this
8 material synthesized in some way as part of the
9 package.
10 That is evidence, and it's even included
11 under our interim guidelines that that's evidence.
12 That is a mistake. And I think that's how we got
13 into trouble. I did not expect to see a
14 randomized, a list of descriptive literature from
15 randomized trials when I read this document after
16 -- you know, my documents came after the panel
17 met, so I did not expect to see that document,
18 based on our interim document. It didn't correlate
19 with what we had said.
20 DR. SOX: Well, number one, this
21 committee is going to start meeting quickly and
22 hopefully come to conclusions that will affect
23 subsequent operations of the MCAC panels in a
24 timely fashion. And secondly, you Bob, will have
25 an opportunity to decide whether we've done an
.00132
1 adequate job when we bring the revised document to
2 a vote, which hopefully will be soon.
3 Any other comments before we vote?
4 MS. CONRAD: Okay. Let me repeat the
5 motion as I understand it. You're going to vote on
6 establishing a methods subcommittee which is a
7 working group to deal with the current Medicare
8 Coverage Advisory Committee process. As an aside,
9 that includes modifying the interim procedures, the
10 interim recommendations guideline, and to help
11 establish formation of the questions that HCFA
12 addresses to you.
13 DR. SOX: So far, so good, but also
14 moving on to discuss and develop procedures for the
15 broader issues that are raised by an expanded
16 assignment of our MCAC to give coverage advice to
17 HCFA.
18 MS. CONRAD: All in favor? Those in
19 favor please, a show of hands?
20 (All members with the exception of Dr.
21 Brook voted in the affirmative.)
22 MS. CONRAD: Opposed?
23 (No opposed votes.)
24 MS. CONRAD: Abstain?
25 (Dr. Brook abstained.)
.00133
1 DR. SOX: Do we have a requirement to
2 ask for people to explain their votes.
3 MS. CONRAD: We do for the panels, but I
4 don't think it's necessary here.
5 DR. SOX: Good. Yes, Ron.
6 DR. DAVIS: Would it be appropriate for
7 me to quickly raise a related issue? It's kind of
8 a generic issue about process and really doesn't
9 fit as neatly in the afternoon discussion. The
10 title of our Committee suggests that we have a
11 fairly broad remit, Medicare Coverage Advisory
12 Committee, and this subcommittee that is going to
13 be formed is going to be exploring the issue of our
14 remit being expanded and getting more into the
15 coverage recommendations. And I feel that to
16 properly discharge our responsibilities, it's
17 important that we are generally aware of all the
18 important decisions and documents and activities on
19 Medicare coverage that are occurring at HCFA, and I
20 feel that in some cases we've fallen short of
21 that. For example, I wasn't aware of the notice of
22 intent that apparently was published by HCFA a
23 month or so ago, or maybe it was earlier than
24 that. And maybe we were notified and I just missed
25 it, but I was also not aware that any action had
.00134
1 been taken as a follow up to the multiple myeloma
2 issue that we spent a half day or so considering in
3 the Executive Committee. And I suspect that there
4 are other important activities and decisions and
5 documents being made about Medicare coverage more
6 broadly. And even if there are activities going on
7 behind the scenes that don't directly relate to our
8 formal responsibilities, I think it would behoove
9 us to be well educated and well informed about
10 what's going on.
11 So if other people on the Executive
12 Committee share my concern, the suggestion that I
13 would offer is that we would request that HCFA
14 staff keep us informed on a proactive basis on
15 various documents that are put out for public
16 comment, various decisions that are made,
17 especially decisions that follow up on actions that
18 have been on our table for consideration. And this
19 could be done by mailing us material, by e-mailing
20 us notices, by letting us know where on the web
21 site these things may appear. I personally don't
22 have time to be checking the HCFA web site on a
23 daily or weekly basis, so that's my comment.
24 DR. SOX: Do you want to respond?
25 DR. HILL: Yes. Your chairman has
.00135
1 already indicated the need for that, so I'll offer
2 you a three-part response, in view of the time.
3 You're right, we hear you, and we're working on it.
4 DR. SOX: So basically, when you get to
5 where you want to be, we will kind of understand
6 the broader context of HCFA decision making.
7 DR. HILL: As best we are able to
8 articulate it.
9 DR. SOX: Okay. Is there anything you
10 want to say about logistics of lunch?
11 MS. CONRAD: I am informed that I
12 neglected to read the results of the last vote into
13 the record, and I must do so at this time. We had
14 all affirmative, all for votes except for one
15 abstention, so the vote, the motion is carried.
16 DR. SOX: We are going to reconvene in
17 precisely one hour.
18 (Luncheon recess taken at 11:44.)
19 MS. CONRAD: We have a quorum; let's
20 go.
21 DR. SOX: This afternoon is going to be
22 a discussion of the Med-Surg recommendations about
23 incontinence, and just for the information of the
24 committee, what I'm aiming at is a motion that will
25 basically, a blanket motion to approve the
.00136
1 recommendations of the Med-Surg panel with the
2 possibility that if an individual member of the
3 committee feels strongly, we can pull out different
4 of the subquestions that the committee considered
5 for individual discussion, but I'm hoping we can
6 deal with what the committee did with one motion.
7 So for those of you who have some particular bone
8 to pick with some aspect of it, be ready to
9 identify that aspect of it for special
10 consideration.
11 The first part of the session will begin
12 with a presentation by HCFA staff, and who is going
13 to do that?
14 MS. CONRAD: Dr. John Whyte, please.
15 DR. WHYTE: Thank you, Connie, and thank
16 you, Dr. Sox, for the time address the panel. What
17 I'm going to do over the next 15 minutes is to talk
18 about how we came to the overall topic of urinary
19 incontinence, how we narrowed it to these two
20 topics, the process we used relating to the
21 technology assessment, the formulation of the
22 assessment questions, and discuss a little about
23 the AHCPR guidelines. I'm going to talk about how
24 we disposed of materials, and how we came to the
25 panel with questions.
.00137
1 So to start with how we came to the
2 overall topic of urinary incontinence, and as you
3 all know, it's a significant source of morbidity
4 for the Medicare population, and the diagnostic and
5 therapeutic options have not been definitively
6 studied and researched. So originally, when
7 someone asked this morning, why did we decide to
8 even tackle the topic of biofeedback, as well as
9 the topic of pelvic floor electrical stimulation,
10 the reality is that we really wanted to look at the
11 overall topic of urinary incontinence; how do you
12 treat it, what are the various therapeutic options,
13 are there a continuum of options, and where do they
14 all fit. And this would include pharmacologic
15 agents, surgery, bulking agents, biofeedback,
16 sacral stimulator, pelvic floor electrical
17 stimulators, as well as other behavioral
18 modifications, which as you can imagine, was quite
19 an ambitious undertaking.
20 But what we learned from the first two
21 panel meetings was that we needed to limit the
22 number of topics that could be addressed in the
23 two-day meeting. So what we decided at a staff
24 level was to limit the first incontinence panel
25 meeting to two topics. We chose the issue of
.00138
1 biofeedback primarily because there was a
2 difference in coverage policies. If you look at
3 our April 1999 Federal Register notice, we say that
4 when the service is subject to inconsistent local
5 coverage policies, we may take on that issue for
6 national coverage determination. Biofeedback is
7 one of those issues where there is local carrier
8 discretion.
9 We also decided at that time to look at
10 pelvic floor electrical stimulation, since there
11 had been several requests over numerous years to
12 readdress the issue of noncoverage. As opposed to
13 biofeedback, pelvic floor electrical stimulation
14 was essentially noncovered. And finally, we have
15 decided there are other areas of urinary
16 incontinence left to address, and that actually
17 will be addressed at the next Medical Surgical
18 Procedures Panel. So in answer to questions on
19 urinary incontinence and biofeedback, it was a
20 broad issue of incontinence that we wanted to look
21 at, because we realized it has a significant
22 morbidity issue for beneficiaries and if it could
23 improve the quality of life, we would be interested
24 in doing so.
25 Can everyone hear me? How's this?
.00139
1 Better?
2 So let's spend a few minutes discussing
3 the process relating to the technology assessment.
4 Based on the interim recommendations of the MCAC
5 Executive Committee, we decided to order a
6 technology assessment, and our policy for external
7 assessments is to go through the AHRQ, the Agency
8 for Health Research and Quality, formerly known as
9 AHCPR, which has 12 evidence based practice centers
10 throughout the country.
11 Now based on the comments that we
12 received in the mail as well as what we heard at
13 the previous panel meeting and this morning, there
14 appears to be some confusion as to what the Blue
15 Cross/Blue Shield TEC, technology evaluation
16 center, is, and why it was chosen to do this
17 assessment. TEC is one of 12 evidence based
18 practice centers, TEC was founded in 1989 by the
19 Blue Cross/Blue Shield Association, and since then
20 has produced over 400 technology assessments.
21 Now when people hear TEC is part of the
22 Blue Cross/Blue Shield Association, they often feel
23 there is an inherent bias towards a negative
24 assessment. It's important to note that the staff
25 persons are noninsurance executives but rather are
.00140
1 primarily physicians and other health services
2 researchers with advanced degrees in statistics,
3 whose stated mission, I'll just read from it, is to
4 produce rigorous high quality scientific
5 assessments of medical effectiveness. The goal at
6 TEC is to provide plans and subscribers, which
7 includes Kaiser, with the best available evidence.
8 And again, it's important to note that TEC does not
9 consider costs, nor does it make coverage
10 recommendations.
11 Now HCFA in some consultations with the
12 AHRQ, but primarily HCFA determined that TEC would
13 be the most appropriate center to develop the
14 evidence report, primarily because TEC had
15 previously done assessments on these modalities.
16 It is a common practice for AHRQ to select an EPC
17 that has conducted the initial assessment when a
18 new evaluation is requested. And I really want to
19 emphasize that a previous assessment, despite what
20 people have said, does not predict nor does it
21 prejudice the outcome of a subsequent assessment.
22 We made it abundantly clear throughout the process
23 that this was to be a de novo assessment.
24 Now some critics have argued that there
25 should have been a group of experts on incontinence
.00141
1 who did the technology assessment. We would
2 disagree with this premise. Technology
3 assessments, as you all know, are based on
4 systematic reviews of literature. The experts in
5 methodology and health services research should do
6 these type of reviews. Unbiased researchers are
7 often the best individuals to perform these roles,
8 so it's really unfair to criticize the TEC
9 assessment. So it's really unfair to criticize the
10 TEC assessment as a biased assessment.
11 Now critical to the assessment is the
12 formulation of the assessment questions, and I want
13 to spend a significant amount of time on this
14 particular issue. Again, the assessment questions
15 were determined by the HCFA staff with consultation
16 of the TEC staff, and for biofeedback, the
17 assessment question was: For urinary incontinence
18 patients, does adding biofeedback to pelvic muscle
19 exercises result in greater improvement in health
20 outcomes than the use of pelvic muscle exercise
21 alone?
22 Now you can ask, why did we choose this
23 question, and that's a reasonable question to ask
24 about our question. We know that biofeedback and
25 pelvic muscle exercises work compared to nothing,
.00142
1 and we know that pelvic muscle exercises alone work
2 compared to nothing, but we do not know the
3 effectiveness of biofeedback and pelvic muscle
4 exercise works in comparison to pelvic muscle
5 exercises alone, in other words, what is the added
6 benefit of the biofeedback component. And that's
7 really what it has come down to, and I am going to
8 talk in a few minutes about how the AHCPR
9 guidelines relates to that question.
10 So again, we know that biofeedback and
11 pelvic muscle exercises works compared to nothing,
12 and that's what the AHCPR report addressed. We
13 know that pelvic muscle exercises alone works. So
14 it's not an unreasonable question to ask, what is
15 the added benefit of biofeedback to pelvic muscle
16 exercises.
17 Now it's also important that we clarify
18 as to what we mean by biofeedback, because there
19 are different definitions out there, and it may not
20 be a single unifying definition, but let me tell
21 you the definition that we used, because that
22 relates to the assessment, because throughout the
23 day, to be honest, people are sometimes having
24 different conversations or are talking different
25 things, and we're not always in disagreement. So
.00143
1 hopefully, through this talk, we can really focus
2 on what the assessment is about and what the
3 questions have been.
4 So for biofeedback, the definition that
5 we chose which relates to the assessment is a
6 therapy that uses either an electronic or
7 mechanical device that relays visual and/or
8 auditory evidence of pelvic floor muscle tone.
9 This is done in an effort to improve the awareness
10 of pelvic floor musculature and to assist patients
11 in pelvic muscle exercises.
12 Now, the selection of the assessment
13 question that I just discussed, as well as the
14 definition of biofeedback, have been a stimulus for
15 continued discussion. Now we would assert that
16 biofeedback, remember, always involves pelvic
17 muscle exercises. You can't have biofeedback
18 without pelvic muscle exercise. Now some persons
19 have suggested that pelvic muscle exercises always
20 involves biofeedback and we do not support this
21 premise, primarily because of how we define
22 biofeedback for this meeting.
23 There are other types of biofeedback
24 which do not use an actual mechanical device, such
25 as verbal feedback, digital probe, and actually the
.00144
1 AHCPR guidelines state on page 36 of their report
2 that pelvic muscle exercises can be done with or
3 without biofeedback, and I'll quote from it:
4 Pelvic muscle exercises may be used alone or
5 augmented with bladder inhibition, biofeedback
6 therapy, or vaginal retraining.
7 For pelvic floor electrical stimulation,
8 there were three questions. Compared to placebo,
9 is treatment with pelvic floor electrical
10 stimulation efficacious in reducing incontinence?
11 What is the efficacy of pelvic floor electrical
12 stimulation as compared to pelvic floor muscle
13 exercises or alternative nonsurgical treatment?
14 Does the addition of pelvic floor electrical
15 stimulation to pelvic floor muscle exercises result
16 in improved outcomes above that obtained with
17 pelvic muscle exercises alone?
18 So there were different questions for
19 there. Pelvic floor electrical stimulation wasn't
20 being just viewed as adjunctive therapy but also as
21 a primary therapy, so they are questions that we
22 are different questions that we were interested in
23 for each modality.
24 Now let me address the AHCPR guidelines,
25 because they have been discussed throughout this
.00145
1 morning's meeting as well as at the previous
2 meetings. Now the first report was issued in 1992
3 and updated in 1996, and I'm going to discuss
4 really what are the guidelines and what they said.
5 First, they are guidelines, they are not
6 the same type of systematic review of literature
7 that was done in this assessment, and that's an
8 important point to emphasize.
9 Second, the guidelines, as I mentioned
10 earlier, focused on the use of biofeedback and
11 pelvic muscle exercises compared to nothing, so
12 when the guideline said that biofeedback assisted
13 pelvic muscle exercises are effective, they were
14 saying that biofeedback assisted pelvic muscle
15 exercises are effective as opposed to nothing.
16 They did not actually address in any great detail
17 the contribution of biofeedback to biofeedback and
18 pelvic muscle exercise, which was the focus of this
19 meeting.
20 And I'll quote from page 38, because you
21 don't have to believe me. On page 38 it says:
22 Further controlled trials are needed to assess the
23 conditions in which biofeedback provides an added
24 benefit to pelvic muscle exercises alone.
25 Now contrary to what some people have
.00146
1 said, we do not disagree with the AHCPR
2 guidelines. If we had asked the panel if
3 biofeedback and pelvic muscle exercises are
4 effective compared to nothing, the answer would
5 most likely have been yes, but we did not ask that
6 question. Rather, we asked again, is biofeedback
7 and pelvic muscle exercises more effective than
8 pelvic muscle exercises alone? We wanted to
9 determine how much of the benefit is due to pelvic
10 muscle exercises alone and how much is due to the
11 addition of biofeedback to pelvic muscle exercises.
12 And again, for pelvic floor electrical
13 stimulation, our questions were more numerous than
14 the AHCPR guidelines since we looked at the
15 effectiveness of pelvic floor electrical
16 stimulation compared to placebo, compared to pelvic
17 muscle exercises or alternative nonsurgical
18 therapies, as well as compared to pelvic muscle
19 exercises alone in combination with pelvic muscle
20 exercises.
21 As someone else mentioned this morning,
22 it's also important to note that their has been an
23 enhanced body of literature since these guidelines
24 initially came out. So not only are there
25 different questions, but there's also different
.00147
1 literature.
2 I just wanted to discuss the exclusion
3 articles which have sometimes been overlooked. In
4 an effort to be fair and comprehensive, we prepared
5 a table of exclusion articles, and by exclusion
6 articles, I mean we abstracted several articles
7 that were included in the assessment since they
8 were primarily studies that involved historical
9 controls or pre-post designs. We also included
10 several articles that were typically quoted and
11 cited, and these were extracted in the exact same
12 manner that was used for the assessment.
13 I want to address the assertion by
14 several members this morning that we only
15 considered randomized clinical trials, and that is
16 very inaccurate. For those of you who have the
17 assessment in front of you, there are grids that
18 are set up and if you have it, I ask that you refer
19 to it, because what you will see on the one grid,
20 they talk about group allocation and you will see
21 it will say randomized, nonrandomized, randomized,
22 single blinded trial, K series, K series, K series,
23 questionnaire, cohort study. So, the point is that
24 there was an entire body of literature that was
25 looked at. To say that we only allowed randomized
.00148
1 clinical trials to be examined is a fallacy.
2 In the previous discussions of the
3 Executive Committee, you all talked about a
4 hierarchy of evidence, and it's important to keep
5 that focus, that there is a hierarchy. That does
6 not mean that other information, clinical
7 expertise, anecdotal experience, is ignored. It is
8 considered and it was encouraged to be considered;
9 that does not mean it was weighted in an equivalent
10 type of point scale as other types of studies. So
11 I really want to emphasize that point, and I
12 challenge these people that say that we only looked
13 at randomized clinical trials to actually look at
14 the assessment, because that assessment is a
15 synthesis of the body of literature to answer the
16 question that we formulated at the beginning of the
17 assessment.
18 Now we mailed the technology assessment,
19 the exclusion tables, and articles that were
20 extracted, to the members of the panel as well as
21 the two guests that we invited to participate on
22 the panel. And we did indeed receive a significant
23 amount of materials from various persons, some at
24 our request, often the specialty societies. We
25 asked for their input. And at a staff level, we
.00149
1 have reviewed and catalogued all of these
2 materials, and did make every item available on the
3 catalog to every member of the panel, and we did
4 discuss the catalog in a conference call prior to
5 the meeting, as well as described it in a cover
6 letter that went out to the panel. All the panel
7 had to do was ask for an item and we would send it
8 to them, and indeed, we did send several items to
9 several members of the panel.
10 We made the decision at a staff level
11 based on the Executive Committee interim
12 recommendations in consultation with the panel
13 chair, that the catalog was the most fair way to
14 handle the information that was sent. We were sent
15 a very large volume of information from people; we
16 felt it would overwhelm the panel if we sent all of
17 that information, especially based on discussions
18 of the first two panels. We felt that the most
19 fair way was to catalog it; we did not want to be
20 the arbitrator and decide what we would send and
21 what we would not send, because in that way, we
22 would never please anyone, you know, why didn't we
23 send this report?
24 So what we did was say we will catalog
25 everything and then to say if you need something,
.00150
1 you let us know. And the reason why we may not
2 have pointed out specific items on the catalog
3 during the conference call, it would not be fair to
4 say, well, this is more important, you might want
5 to request this, and not look at other items.
6 Now, the issue about the time frame and
7 how the report got on the web, this was all done in
8 a compressed time frame and we are trying to do
9 better, but it really is consistent with the time
10 frame of other federal agencies as well as other
11 federal advisory panels, roughly a two-week time
12 period to receive information is a reasonable
13 period of time.
14 Now along with materials I just
15 mentioned, we also sent questions for the panel to
16 address. These questions were largely based on the
17 interim recommendations of the Executive Committee,
18 again, in consultation with the chair of the panel,
19 and I'll just read you what the questions were very
20 briefly.
21 For biofeedback, is the scientific
22 evidence adequate to draw conclusions about the
23 effectiveness of biofeedback as an adjunctive
24 therapy to pelvic muscle exercises in routine
25 clinical use in the Medicare population, for the
.00151
1 following three indications? We had broken it down
2 to stress incontinence, urge incontinence and
3 post-prostatectomy incontinence.
4 Then for pelvic floor electrical
5 stimulation, we asked them: Is the scientific
6 evidence adequate to draw conclusions about the
7 effectiveness of pelvic floor electrical
8 stimulation compared to placebo, compared to pelvic
9 muscle exercises or alternative nonsurgical
10 therapies, and then a combination of pelvic floor
11 electrical stimulation and pelvic muscle exercises,
12 compared to pelvic muscle exercises alone.
13 And then we asked them to consider when
14 they looked at the data, the adequacy of the study
15 design and the consistency of the results, the
16 applicability to the Medicare population, and
17 applicability beyond the research setting. What we
18 were trying to do is to develop consistent
19 questions that we would ask for every panel, so
20 that's the reason why these questions were phrased
21 that way.
22 The second point deals with the issue of
23 the health effect that some of you talked about
24 earlier. If the evidence is adequate to draw
25 conclusions, because remember, the first question
.00152
1 is, is the evidence even adequate to draw
2 conclusions? So if the answer is no, then you
3 can't go on to the second question. If the answer
4 is yes, you then say, if the evidence is adequate
5 to draw conclusions, what is the size, if any, of
6 the overall health effect? And then there's
7 various categories on the seven point scale that
8 all of you know from having helped us devise those
9 seven points.
10 Now I want to point out some issues
11 about the assertion that somehow we changed the
12 question or made it more narrow. And I really need
13 to point out that there was never a change in the
14 intent of the question. There was a clarification
15 based on some confusion as to what we meant by
16 biofeedback and originally the question read, if I
17 can get it for you --
18 MS. WOOLNER: Do you want a copy?
19 DR. WHYTE: No, I have it. Thank you,
20 Barbara. Is the scientific evidence adequate to
21 draw conclusions about the evidence of biofeedback
22 in routine clinical use in the Medicare population,
23 et cetera. The difference that, between the two
24 questions, was the addition of three words, or four
25 words, as an adjunctive therapy. And the reason
.00153
1 why we made that clarification, because as phrased
2 it became apparent to us that some people assumed
3 or wished that we meant biofeedback and pelvic
4 muscle exercises compared to nothing, but as you
5 know from this discussion, this is not what we
6 intended, by adding those words, as an adjunctive
7 therapy, to clarify it.
8 For pelvic floor electrical stimulation,
9 we had broken it down into three categories, the
10 pelvic floor electrical stimulation compared to
11 placebo, compared to pelvic muscle exercises, and
12 then in combination.
13 And again, there's been an intimation
14 that we narrowed the question to focus a no vote,
15 and that's simply not the case. These
16 modifications were not meant to be more restrictive
17 but rather, more clarifying. And it's also
18 important to note that these questions are
19 consistent with the technology assessment
20 questions. I read to you early on what the
21 technology assessment question was, the issue as an
22 adjunctive therapy.
23 Dr. Simon, who made opening HCFA remarks
24 at the presentation at the biofeedback panel,
25 clearly point out that it's viewed as an adjunctive
.00154
1 therapy, so to point out that somehow we only meant
2 biofeedback and pelvic muscle exercises compared to
3 nothing, really is inconsistent with what we have
4 said all along.
5 Those really are the points that I
6 wanted to bring all of you up to date on. At this
7 point in time I am going to defer to Dr. Garber to
8 actually discuss the content that was discussed at
9 the meeting, and after Dr. Garber, if that's
10 allowable by the chair, myself or others would be
11 happy to answer any questions you have, or I could
12 answer them now, however you want to handle it.
13 DR. SOX: Does the panel have any
14 questions for John before we proceed to Alan's
15 report of the committee? Alan, why don't you go
16 ahead?
17 DR. GARBER: Well, I understand that my
18 task is to give a concise statement that explains
19 everything about the panel's reasoning even though
20 the panel never really had a chance to individually
21 explain all aspects of their reasoning. It may be
22 a little ironic in that I could not and did not
23 vote on any of the questions, so I cannot be said
24 to be speaking for myself about reasoning, but I
25 will try to reconstruct what I think was going on.
.00155
1 Let me just make a couple of background
2 statements that I think are relevant to the members
3 of the Executive Committee who will at some point
4 presumably this year also be in the chair's role on
5 their committees, because a very important aspect
6 of this process is the chair's input and other
7 panel members' input into areas like formulation of
8 the question and selection of the literature for
9 distribution. We've already spoken about selection
10 of the literature, I've already said my mea culpas
11 about that, and I'll leave it at that.
12 But about the formulation of the
13 question, I was consulted about the questions and I
14 asked myself a few things in trying to evaluate
15 whether this was a reasonable question to put
16 before the panel. One of them was, is this a
17 reasonable question to answer in coverage, for the
18 purpose of coverage decision making, that is, could
19 an answer to this question be helpful to HCFA, and
20 I decided that I am not really the right person to
21 judge, that's HCFA's question and one should give
22 them a lot of leeway. But I did ask, is this a
23 separable service, is there some reason why you
24 might want to ask this question, and though I'm not
25 an expert, it seemed to me that was plausible and I
.00156
1 deferred to their greater expertise about that
2 issue, and feel that I should question that very
3 hard.
4 The second aspect, is it a reasonable
5 question to ask of the literature? And if you knew
6 before you started that there wouldn't be any
7 reasonable literature on this, that is, studies
8 that address this question, that strikes me as not
9 something that is worthy of the panel's time even
10 if it is a reasonable coverage question. And in
11 this case, there was a substantial literature,
12 including randomized trials and many other kinds of
13 studies addressing the question, at least in
14 biofeedback -- actually all aspects of the
15 questions, but in the case of biofeedback, was it
16 effective as an adjunct to pelvic muscle
17 exercises? So indeed, there was a literature, so
18 it was feasible.
19 And then there are questions, is the
20 question that's being posed to the panel clear and
21 consistent with the instructions from the Executive
22 Committee, and I thought here that was quite true
23 with the proviso that it did need to be changed as
24 John described, to make it a little clearer what
25 was meant by biofeedback, because one thing that
.00157
1 became very clear in the public testimony is that
2 there are many different forms of biofeedback, and
3 a manual examination where the provider feels a
4 muscle contracting can be one form of biofeedback,
5 but what HCFA had in mind was more mechanical and
6 electrical assistance as part of the process. So
7 that all seemed reasonable.
8 Now there is also a question, did HCFA
9 and me as panel chair consult widely enough early
10 enough about the formulation of the question, and I
11 think there is probably substantial room for
12 improvement. It would have been nice to get a lot
13 of the input that we had received during the panel
14 meeting at an earlier stage of the process so that
15 the questions could have been refined if suitable.
16 But I actually thought that the questions were
17 clear and appropriate, at least in broad terms.
18 The second issue is about expertise on
19 the panel. It's important to point out, and John
20 mentioned this, that although the panelists come
21 from a wide variety of backgrounds, it was clear
22 there was a need for expertise in the continence
23 area on the panel. A panelist from another
24 standing MCAC panel, Lisa Landy, who is a
25 urogynecologist, was brought on to the panel for
.00158
1 this meeting. She apparently uses these
2 techniques, has a lot of hands-on familiarity.
3 Another regular member of our panel is a
4 gynecologist who has experience with them. HCFA
5 also brought in a nonvoting continence specialist
6 to be a member of the panel, and I dare say that
7 she and Lisa Landy spoke more than anybody else,
8 each one of them spoke more than anyone else by a
9 substantial amount, and they were heard. I think
10 people took what they said very seriously. So
11 don't get the idea that content expertise was
12 ignored or overlooked, although it certainly was
13 the case that not all the panelists voted the way
14 that the clinical experts in the field might have
15 preferred.
16 There is a second operational issue and
17 that is the review of both the evidence review and
18 the panel's deliberations as a whole. Hugh Hill
19 had mentioned beforehand that it would not be
20 possible to fully implement the external review
21 previsions that the MCAC Executive Committee had
22 recommended and still stay within the time frame
23 that we had set as goals, and so that part was not
24 done, but we had the two designated members of the
25 panel to review this material in detail, Lisa Landy
.00159
1 and Les Zendel, who I believe is trained as a
2 geriatrician and has substantial experience with
3 the treatment of incontinence. So they were sort
4 of the panel's designated experts who reviewed the
5 material.
6 John described what the questions were
7 and to give you an idea, every comparison was
8 subdivided into three clinical indications, urge
9 incontinence, stress incontinence, and
10 post-prostatectomy incontinence, and then we have
11 this rather complex day on the pelvic floor
12 stimulation, where there were a variety of
13 comparisons, comparisons to placebo -- well, you've
14 heard it all, but what that meant is on the second
15 day we were asked the questions for basically nine
16 different pairs of indications and which treatments
17 were being compared.
18 Let me add, by the way, that one of the
19 reasons I thought the question on biofeedback was
20 reasonable is it seemed to correspond directly to
21 what the Executive Committee had laid out among the
22 comparative effectiveness criteria, that is, to
23 compare it to other treatments in making the
24 determination, at least as part of the process.
25 So, what happened in terms of the
.00160
1 committee deliberations? As John said, the
2 evidence review that was conducted by the Blue
3 Cross/Blue Shield Association TEC center did
4 incorporate both nonrandomized and randomized
5 trials. And I should say in passing that I have
6 participated in authoring an evidence based
7 practice center report, I'm part of the
8 UCSF-Stanford evidence based practice center, and I
9 have served as a formal reviewer for other EPC
10 reports, and I would say that this report was very
11 much of the same quality as all the other EPC
12 reports. There was nothing particularly to
13 distinguish it, except it was done on a shorter
14 time frame and it was shorter, it was a briefer
15 document.
16 And in contrast to Bob Brook's
17 impression of the report, I felt it was very
18 carefully done and in fact many of the people who
19 spoke publicly and criticized aspects of the report
20 actually commended it on its thoroughness and
21 completeness, and indeed it was a synthesis. The
22 one thing that Bob mentioned that it didn't have,
23 and it really didn't have, was a review of the
24 guidelines. I looked in vain for anything in the
25 Executive Committee report that said the evidence
.00161
1 report should include a summary of guidelines; I
2 didn't see that there.
3 I actually think it adhered closely to
4 what we asked for, and I don't mean to imply it was
5 a perfect document, but it was reviewed very
6 extensively by a number of people, and I would urge
7 the Executive Committee to look at that very
8 carefully and where you see flaws, omissions,
9 things that were not done properly, to send back
10 comments to HCFA staff and to the rest of the
11 Executive Committee, because we need to know what
12 needs to go into these evidence reports. But as I
13 said, I thought it was every bit the typical
14 evidence based practice center report, and did not
15 see any obvious omissions. Now that's not to say,
16 by the way, that I have enough expertise to know if
17 studies were overlooked or to know about highly
18 specific details of the studies that formed the
19 foundation for this report, but I thought in format
20 and general content, it was roughly what we were
21 working for.
22 Now the panel, the voting members of the
23 panel, turned out to be nearly unanimous on all the
24 questions that were posed with a couple of
25 exceptions. The first day on biofeedback, Lisa
.00162
1 Landy and a nonvoting member and another voting
2 member strongly disagreed with the claim that the
3 evidence was inadequate. And I think that what
4 Lisa's main point was, and I mention her because
5 she had the most complete explanation and I thought
6 she put it very well, was that clinically the
7 distinction between pelvic muscle exercises plus
8 biofeedback and pelvic muscle exercises alone was
9 not meaningful, and she did not think that's what
10 the question should have been. And I believe that
11 the other people who voted that the evidence was
12 adequate or expressed opinions to that effect
13 agreed with that.
14 I did not hear among the panelists any
15 claims that the literature was very compelling.
16 And the reason I think that's true is that there
17 was a large catalog of flaws in the studies and the
18 panel had to consider whether these flaws were
19 serious enough to call into question the
20 conclusions of the studies. And the second reason
21 is that many studies were negative and some were
22 positive, and the panelists had to weigh that.
23 They heard very informative
24 presentations from public speakers, including some
25 from authors or participants in some of the
.00163
1 studies, which helped clarify the study designs.
2 For example, one study that I in particular found
3 relatively compelling was on the pelvic floor
4 stimulation, one by Sand, and from the published
5 version of the study it was unclear whether there
6 was a flaw in follow-up and how patients were
7 assigned when they were lost to follow-up, and that
8 was clarified by the author himself at the meeting,
9 and the study turned out to be stronger than it
10 appeared to be from the published form.
11 But I believe that the panelists
12 concluded that in total on both days, and for each
13 indication, the evidence was not adequate. There
14 was more support for biofeedback than for the
15 pelvic floor stimulation, and that really very
16 clearly reflects the volume of the literature and I
17 think to some extent the quality of the studies
18 that were done.
19 And of the indications, if I can
20 generalize, and this may not be completely
21 accurate, I think -- well no, this part is true,
22 that post-prostatectomy incontinence was very
23 poorly studied and panelists made a point of saying
24 that of all of the indications, and this is for
25 both biofeedback and pelvic floor stimulation, of
.00164
1 all the indications, the evidence was virtually
2 nonexistent for effectiveness in treating
3 post-prostatectomy incontinence. There was more
4 discussion on the other indications.
5 And furthermore, aside from Lisa Landy's
6 yes vote on the first indication for pelvic floor
7 stimulation, there were no affirmative votes about
8 adequacy of evidence for any of the indications on
9 pelvic floor stimulation. And I believe that the
10 discussion of biofeedback, or the voting on
11 biofeedback, was a little more mixed and the
12 discussion was more heated on biofeedback, because
13 there was this concern about the phrasing of the
14 question and in addition there were more studies
15 that lend some support, even though the panelists
16 seemed to feel that the total evidence did not
17 enable them to draw conclusions.
18 Let me reiterate, and this is somewhat
19 my own inference rather than based on direct
20 statements, it's not that they felt that there had
21 to be multiple randomized trials, but they seemed
22 to believe that in this area, it was important to
23 have a fairly rigorous study design because of the
24 number of outcomes in urinary incontinence and so
25 on. If you had biased ascertainment, for example,
.00165
1 there wasn't a strict criterion for deciding when
2 events occurred, how you measured outcomes, it
3 would be very easy to be misled by placebo effects
4 and any other number of confounders, so I believe
5 that that was their reasoning.
6 So I think that there was a lot of
7 sympathy for one issue that did come up in
8 discussion, and that is the difficulty in finding
9 the funding to do adequate studies. And although
10 much has been made of the statements that some
11 people said that they would have voted to cover
12 even though they didn't think there was enough
13 evidence, on my review of the transcript, I think
14 only two people said anything like that, that is,
15 if they had considered the clinical evidence, they
16 would have voted to cover. But they didn't explain
17 what they meant by clinical evidence and frankly,
18 I'm not sure if we had time to probe it, if it
19 really would have supported the statement that they
20 made for those two people. So I think that there
21 is a very interesting issue there about why they
22 said that, and maybe this goes back to what we call
23 clinical evidence and how it should be used, but
24 the panelists who voted that the evidence was not
25 adequate seemed to be quite confident in their
.00166
1 conclusion.
2 Can I turn it over to Michael, because
3 he may have some additional perspectives on that?
4 DR. SOX: Here you are.
5 DR. MAVES: You know, Alan, I basically
6 agree with your report and as I indicated before, I
7 thought it was a much improved process at least
8 from the historical perspective of where we had
9 learned a little bit about the first two panels,
10 and I really want to commend Alan for I think
11 really trying to make this as fair and open a
12 process as is possible.
13 I would agree with him, I think much has
14 been made over the phrasing of the questions and
15 the fact that the question was altered slightly. I
16 don't think that had a substantive effect on the
17 end result of the deliberation, and I think
18 hopefully we can learn a little bit from this and
19 perhaps make the process better in the end. I do
20 think as I indicated before, that it is important
21 to have a roundness of information and not just
22 controlled randomized clinical trials, but to
23 obviously listen to what's going on in the medical
24 practice community, listen to what's going on with
25 clinicians, and we had some people in that
.00167
1 audience, and particularly those from the specialty
2 societies, they were extremely distinguished
3 individuals. I think that they were heard, I don't
4 want to make that misstatement that they weren't,
5 but I think the process and I think what Dr. Sox
6 has outlined hopefully will allow us to more
7 formally integrate that into the deliberative
8 process of the panels.
9 DR. SOX: I would like to give an
10 opportunity for members of the panel to ask
11 questions of fact to Alan or Michael, not to
12 express opinions, just clarification questions, and
13 then we will move on to the open comment. Any
14 questions of fact? Ron?
15 DR. DAVIS: In looking through the
16 minutes of the meeting, I notice there was a
17 question posed where the vote wasn't indicated, and
18 this is on the minutes on the meeting for
19 biofeedback, it's page 4, middle of the page, and
20 just above the bolded heading. The question is
21 listed and there is no vote indicated, so that has
22 to be indicated. I presume it's like --
23 DR. GARBER: Ron, are you talking about
24 the post-prostatectomy indication there, just above
25 panel comments on their votes?
.00168
1 DR. DAVIS: Yes.
2 DR. GARBER: That was unanimous
3 negative, and I'm sorry that we didn't catch that.
4 DR. SOX: Any other questions or points
5 of fact? Yes, Bob?
6 DR. MURRAY: In the TEC summary on page
7 20, I note the pages aren't numbered, but it's the
8 last paragraph under the section Review of Section,
9 Stress Incontinence, the sentence reads: In
10 summary, and these are the words of the author, it
11 is not possible to draw conclusions from this body
12 of evidence on whether the addition of biofeedback
13 to PME results in improved outcomes as compared to
14 PME alone.
15 My question to you, Alan, is: Do you
16 feel that the members of the panel did an
17 independent assessment of the studies that were
18 presented, or were they simply ratifying or
19 agreeing with his statement? And I will tell you
20 the reason that I ask that question. I found some
21 of the evidence rather persuasive that there is no
22 benefit. The best studies found no benefit, the
23 weaker studies were equivocal, so on balance I
24 found the evidence persuasive against, yet the
25 author says it's not possible to draw conclusions.
.00169
1 What was the feeling on the panel?
2 DR. GARBER: That's an excellent
3 question, and I'm trying to remember. I think I
4 did hear one or two statements similar to yours,
5 Bob, that the evidence went the other way, but
6 there was a lot of discussion about positive
7 evidence too. And you know, I hate to speculate
8 about how much people read and absorbed of this,
9 but judging from the questions the panelists asked,
10 I felt confident that at least a number of them did
11 very detailed readings of at least the key studies
12 and did not rely solely on the evidence report.
13 And let me also say though, that it was clear that
14 the evidence report had a great deal of credibility
15 and received a great deal of weight among the
16 panelists, and that's one reason why it was so
17 important to insure that it be comprehensive and
18 fair.
19 DR. SOX: Anything else?
20 DR. BROOK: Can I ask a question of
21 Alan?
22 DR. SOX: Please, Bob.
23 DR. BROOK: Alan, since this report has
24 come out and all the comments, have you received
25 anything indicating there were significant pieces
.00170
1 of good studies that were not included in this
2 report?
3 DR. GARBER: Well, there have been some
4 claims like that. Let me just tell you that the
5 issue that's more of an issue in my mind than
6 overlooked studies is really knowing what was going
7 on in the studies that were reviewed. On a lot of
8 these studies, you know, many of us have reviewed
9 papers before, and I guess I was surprised that
10 there were probably correct things that were done
11 in these studies that did not appear in the
12 published versions. And anyone who has gone
13 through the literature, in some fields anyway,
14 would be surprised to see how often that occurs.
15 DR. BROOK: Let me go back to the first
16 question. As far as you know as chair of this
17 committee, there is no specialty society or nobody
18 that has come forward with a paper that meets the
19 methodologic criteria of this TEC assessment that
20 was overlooked?
21 DR. GARBER: Not to my knowledge.
22 DR. BROOK: That's a statement of fact,
23 to your knowledge and to any of the committee
24 members that were on the committee, that's the
25 case?
.00171
1 DR. GARBER: Yeah. Now there are people
2 who would make the claim that there were. I'm
3 saying that there is none that I have seen that
4 would.
5 MS. RICHNER: I think I read in one of
6 the reports that there was an article published in
7 March of 2000, either biofeedback or whatever, that
8 was significant for this process. I don't have it
9 in front of me.
10 DR. GARBER: Well, yeah. As I said,
11 there have been claims. The ones that were brought
12 to us that were not included that I saw did have
13 flaws. That doesn't mean that there isn't some
14 study out there that is just recently published or
15 will be published to meet the criteria. Bob's
16 question was about to my knowledge, were there
17 things? I'm not saying that -- that doesn't mean
18 something out there exists.
19 DR. BROOK: Let me ask the second
20 question. Somebody pointed out that there are, I
21 went back and looked at it, and I missed the fact
22 that there were one or two of these studies that
23 were nonrandomized in one of the tables. This
24 report is about a few number of studies, something
25 like 11 to 15 total is what it looks to me in these
.00172
1 tables. As far as you knew as chair, the summary
2 of the TEC report, the questions that were asked,
3 you believe that the assessment of those studies
4 were fair, complete, unbiased, and as far as you
5 know, nobody has come forward, the authors, or
6 nobody has come forward to say you guys
7 misrepresented what we did? In other words, you
8 haven't gotten anything -- there is nothing in all
9 these letters that we got.
10 DR. GARBER: There are undoubtedly
11 members of the audience who will tell you that.
12 DR. BROOK: No, no. We have gotten no
13 specific -- to be honest, I have seen zero
14 specific -- I have read all the letters -- zero
15 specific comments that the abstraction of the
16 study, the way it was described, the limitations
17 that were described, the positive features of any
18 of these studies, there's no specific detail that
19 I've read, and I just wondered if there is any body
20 of evidence anywhere floating around that would
21 indicate that people were concerned over how these
22 studies were described.
23 DR. GARBER: I wouldn't go that far. I
24 think people would dispute how the studies were
25 described. I think the panelists felt comfortable
.00173
1 with how the studies were described. And as anyone
2 who has been involved in the process of abstracting
3 studies, summarizing them, pulling them knows,
4 there are aspects of this process that are judgment
5 calls. Like what criteria you use to decide which
6 studies to include and exclude, how far you go
7 using information that's not available in the
8 published studies and about how they were
9 designed. And the way I would characterize this is
10 I have not heard major defects that aren't matters
11 of judgment calls, as opposed to things that are
12 clearly done wrong in the report.
13 DR. BROOK: And when you've heard these
14 comments of defects, have they been sort of on both
15 sides equally in terms of you, this was really more
16 positive and this was really more negative?
17 DR. GARBER: No. The vast majority of
18 comments that we have received have come from
19 people who disagree with the conclusions of the
20 panel.
21 DR. BROOK: I'm not talking about the
22 panel.
23 DR. GARBER: But that's the people who
24 have expressed substantive concerns about what's in
25 this report.
.00174
1 MS. CONRAD: I wonder if we could hear
2 from our public commenters.
3 DR. SOX: It's time for the public
4 comment. And would you like to announce the first
5 person please?
6 MS. CONRAD: The first speaker is
7 Barbara Woolner, followed by Debra Jensen please.
8 DR. SOX: As much as possible, I hope
9 that those who stand to comment will try to address
10 the issues that were raised by both Dr. Whyte and
11 Dr. Garber, so as much as possible -- we know we've
12 had a lot of written comment, what we want is stuff
13 that's kind of on this discussion. Thank you.
14 MS. WOOLNER: My name is Barbara
15 Woolner, and I am a clinician. I am certified in
16 biofeedback incontinence care. I have been
17 practicing this for 12 years. I stand today before
18 you as representative of the Continence Coalition,
19 a group of urologic nurses and wound ostomy
20 incontinence nurses who have banded together for
21 the benefit of patients suffering from
22 incontinence.
23 I did present before the Medical
24 Surgical Panel last month, actually in April, and I
25 actually made three points. I'm not going to go
.00175
1 through -- you got my testimony that's written here
2 today right before lunch. Don't look at it,
3 because what I'm going to say is not there, most of
4 it at any rate.
5 I would like to some of the things that
6 Dr. Whyte and Dr. Garber have said, not all
7 together disagreeing, but maybe enhancing a little
8 bit. There was a question by Ms. Richner about the
9 study that's floating around out there that might
10 have to do and that might have been relevant to
11 this whole issue had it been available for the TEC
12 report. That is a very well designed study, and I
13 say that because my colleagues, my scientific
14 colleagues tell me so, by Carolyn Samselle, who is
15 a nurse researcher, and it was published in March
16 of 2000 and it was regarding pelvic muscle exercise
17 alone, and pelvic muscle exercise alone, done with
18 her very rigid criteria, and I actually know this
19 lady.
20 She got a 27 percent improvement in
21 patient symptoms, which is not terribly good,
22 because if you look back at the studies that were
23 included in the TEC report, they give you overall
24 biofeedback, 61 percent, 89 percent, 91 percent, 54
25 percent, and 76 percent. And I know for a fact
.00176
1 because I know two of the people that produced a
2 couple of these studies, particularly Patricia
3 Burns in 1993. She had a nonsignificant effect in
4 her pelvic muscle exercise group versus her
5 biofeedback group alone. I know for a fact that
6 the pelvic muscle exercise group, though not
7 reported in her outcome, was a group of patients
8 that did get a very very specific muscle testing
9 with a very skilled nurse practitioner. The
10 biofeedback they received, however, was done by a
11 person who was not trained in biofeedback, who was
12 merely a technician, so I really think there was a
13 lot of fault with this study.
14 Pat Burns herself will tell you that she
15 did use biofeedback EMG to evaluate the patients in
16 her pelvic muscle exercise group alone and as a
17 clinician I will tell you that once a patient sees
18 a screen and sees what's happening, they learn very
19 quickly, sometimes in a manner of seconds. So you
20 can't say that that was a pelvic muscle exercise
21 alone group.
22 In terms of other things that support
23 what I'm saying, Bump in 1993 reported on 27 women,
24 and I'm pulling this from my head, not my paper.
25 He found that 50 percent of the women that he asked
.00177
1 to perform a pelvic muscle contraction could not do
2 so on verbal instruction alone. And in fact, the
3 majority of instruction in this country is from a
4 physician who hands a patient a piece of paper and
5 says go home and do this while you're sitting at
6 stop signs, or you're sitting on the toilet, and
7 there is in fact no good instruction to pelvic
8 muscle exercise.
9 In a study that I reported on at the
10 last panel because I was thinking that they would
11 pay some attention to really good clinical work, we
12 found in over 200 patients that 65 percent of the
13 patients that we evaluated both manually and with
14 EMG had either abandoned or had failed pelvic
15 muscle exercise alone. I thought that was very
16 significant, and I still do.
17 Actually, getting back to the Blue
18 Cross/Blue Shield TEC report, I think one of the
19 things that we have all overlooked is that report
20 did say that there was some efficacy to
21 biofeedback. They didn't say it wasn't
22 efficacious. However, I think that we need to look
23 at the fact that while HCFA has really avoided
24 mentioning this fact, that it was efficacious, and
25 while all the professional organizations have been
.00178
1 looking at problems with the process, we have all
2 forgotten what happens with the patient.
3 I would like to just bring this point up
4 and have you be a little forward thinking about
5 what's going to happen if you choose to ratify this
6 vote from the last panel. This is a very expensive
7 proposition. This data was developed by Tai Wei Yu
8 in 1995, it was reported in '98, and according to
9 him, the expenditure for incontinence in persons
10 over the age of 65 was approximately $27.8 billion
11 per year, and almost half of that was due to
12 consequences of untreated incontinence.
13 Consequences. These are UTIs, these are falls,
14 these are skin irritations, prolonged hospital
15 stays, and additional skilled nursing facility
16 admissions. Of that $13.5 billion, 92 percent of
17 that money was spent on these adverse consequences,
18 92 percent.
19 Of the other 8 percent, it was broken
20 down in this manner: Surgery got the majority in
21 the red, I don't have a pointer, but the large
22 block there of the pie is surgical treatment.
23 Evaluation got the next largest part of the pie.
24 Pharmacological and behavioral treatments got less
25 than 0.1 percent of the treatment that was paid out
.00179
1 to treat incontinence.
2 If you hear nothing of what I've said
3 today, please hear this. Do not let these
4 technologies fall victim to a process that is in
5 evolution. The Continence Coalition joins its
6 professional colleagues, consumer groups, and other
7 concerned individuals in asking you to withhold
8 ratification of this vote by a panel which was
9 forced to judgment on a comparative question that
10 cannot at this time be answered purely in a
11 scientific way. Thank you.
12 MS. CONRAD: Thank you very much.
13 MS. WOOLNER: Oh, one point. I still
14 had a green light.
15 Let me just say that be ratifying this
16 vote, we are actually giving HCFA permission to
17 withdraw coverage, and I would like you to know
18 that there are a number of states that have
19 reasonable coverage for biofeedback. So, I ask
20 you, can you just take it away? Is there not a
21 process by which you have to review the information
22 and the safety of a treatment or a technology from
23 all of these states: Alabama, Alaska, Arizona,
24 California, Connecticut, Georgia, Hawaii, Idaho,
25 Indiana, Kansas, Maine, Massachusetts, Maryland,
.00180
1 Michigan, Missouri, Mississippi, New Hampshire, New
2 Jersey, North Dakota, North Carolina, Nevada,
3 Oklahoma, Oregon, Pennsylvania, Utah, South Dakota,
4 South Carolina, Tennessee, Utah, Virginia,
5 Washington and Wisconsin. Thank you.
6 MS. CONRAD: Debra Jensen please; the
7 next speaker will be Kevin Connolly.
8 DR. JENSEN: Good afternoon. My name is
9 Debra Jensen and I represent EMPI as their vice
10 president of regulatory affairs and clinical
11 research. EMPI is a manufacturer of biofeedback
12 and pelvic floor stimulation devices.
13 Once again, we appreciate the dedication
14 of the panel, the Agency and their staff for their
15 efforts to develop a fair and equitable coverage
16 process. But as we have previously communicated,
17 we were deeply disappointed in the outcome of the
18 April meeting of the Medical and Surgical
19 Procedures Panel on urinary incontinence. Despite
20 working with the Agency for the past six years on
21 the issue of coverage for our pelvic floor
22 electrical stimulation devices, we are no closer to
23 resolution of this issue than when we started our
24 discussions with the Agency.
25 While we can appreciate the difficulties
.00181
1 in implementing a new process such as the MCAC,
2 quite frankly, we are tired of being a guinea pig.
3 We are concerned that fair coverage policies for
4 this technology and also biofeedback will be
5 further delayed while you make this up as you go
6 along and continue to refine your work in
7 progress. It is obvious from this morning's
8 discussion that much work remains in order to make
9 the process work for the benefit of Medicare
10 beneficiaries. Thus, you as members of the
11 Executive Committee are in a difficult position
12 regarding what conclusions should be drawn about
13 the recommendations made by the Medical and
14 Surgical Procedures Panel concerning the adequacy
15 of the scientific evidence supporting these
16 technologies.
17 In recognition of the importance of
18 clinical evidence, I would like to present a brief
19 summary of the relevant evidence presented to the
20 Med Surgical Panel about pelvic floor stimulation.
21 Pelvic floor electrical stimulation was presented
22 as a technology that has been studied extensively
23 in a clinical setting and was found by the AHCPR
24 guidelines to have the same level of evidence as
25 other incontinence treatments such as surgery and
.00182
1 collagen implants, both of which are covered
2 Medicare benefits.
3 PFS was presented as a technology that
4 is already the standard of care, as evidenced by
5 the unanimous support of all of the medical
6 societies that presented at the April panel meeting
7 and were involved in the treatment of urinary
8 incontinence. PFS was presented as a technology
9 that has been recognized by over 300 private
10 insurance carriers as a covered benefit.
11 The point was also made in the April
12 meeting that Blue Cross/Blue Shield in conducting
13 their technology assessment did not appreciate the
14 subtle differences in the various stimulation
15 devices when they wrote their report. Dr. Sand,
16 the author of the randomized placebo control trial
17 that Dr. Garber mentioned earlier very adequately
18 explained how these differences in technology were
19 a factor in the outcome.
20 In light of the difficulties
21 acknowledged this morning and the clinical evidence
22 presented at the April meeting, we encourage this
23 Committee and HCFA to look at recent past coverage
24 decisions made by the Agency to guide the ultimate
25 coverage policy determination for pelvic floor
.00183
1 electrical stimulation. For instance, a review of
2 recent coverage decisions shows that the Agency has
3 acted without ratification by the MCAC panels. In
4 the case of external contrapulsation therapy, the
5 Agency felt that there was a lack of evidence and
6 requested additional studies by the manufacturer.
7 Industry complied by conducting one study of less
8 than 150 patients, and a positive national coverage
9 decision was granted.
10 In the case of augmentative and assisted
11 communication devices there was evidence to the
12 issue was left to carrier discretion. No
13 noncoverage decision was issued. In fact, the
14 Agency plans to issue new national coverage
15 guidelines soon for these guidelines, according to
16 a recent article in Inside HCFA. Unlike
17 augmentative and assisted communication devices,
18 pelvic floor stimulation does have evidence to
19 support its efficacy. Based on this, it appears
20 that the Agency's policy with regard to durable
21 medical equipment is inconsistent in both the
22 application of the standard of evidence as well as
23 their guidelines for MCAC referral and ultimately
24 for coverage decisions.
25 We believe that we have demonstrated
.00184
1 that PFS is a safe and effective treatment and that
2 a positive national coverage decision is
3 warranted. Without evidence that our technology is
4 unsafe or that it offers no benefit for anyone, the
5 national noncoverage policy implemented in 1994 is
6 inappropriate and should be lifted. Based on the
7 information that is provided to the Agency, or that
8 has been provided to the Agency over the past six
9 years and the testimony before the MCAC relative to
10 the scientific evidence, the technology's clinical
11 utility, and the support of the professional
12 societies, we believe there is adequate evidence
13 that PFS is a reasonable and necessary service for
14 Medicare beneficiaries.
15 It seems that everyone this morning
16 agreed that the process used to review PFS and
17 biofeedback was problematic. Therefore, in the
18 interests of fairness, we respectfully request that
19 the panel not ratify the results of the Med-Surg
20 panel. Thank you.
21 MS. CONRAD: Thank you, Dr. Jensen.
22 Kevin Connolly. The next speaker will be Francie
23 Bernier.
24 MR. K. CONNOLLY: I am Kevin Connolly,
25 and am still CEO of SRS, at least for the moment,
.00185
1 and we manufacture biofeedback and stimulation
2 products, and I want to thank the committee for
3 allowing me to make a second presentation.
4 Since Dr. Sox has asked us to address
5 Dr. Whyte's and Dr. Garber's comments, I'm going to
6 start with a slide that didn't make it into my
7 presentation otherwise, which is, Dr. Whyte spent a
8 lot of time talking about the definition of
9 biofeedback, but the really key thing here in these
10 studies, this slide was put together to show that
11 basically the use of randomized control studies as
12 a methodology does not in itself guarantee a
13 well-constructed study.
14 But the key point that I'm making here
15 is that the definition of PME that's used in these
16 studies versus what's used in the real world is
17 vastly different. The Samselle study that you
18 quoted is much more typical of how PME is used in
19 real clinical practice. Companies like
20 Kimberley-Clark prepare a little sheet saying
21 here's how you do these exercises; you give that to
22 the patient, that's PME. So the idea of this
23 intensive coaching, you know, vaginal exams, EMG
24 exams, that's rolling downhill with the wind to its
25 back on a sunny day. Arguably, the benefit of
.00186
1 biofeedback is that it offers a simple standardized
2 way of getting people to do these exercises well,
3 that obviates the need for all of those things.
4 Now, hopefully, I will still have time
5 to do my presentation. I'm going to skip through a
6 lot of this stuff but I will just touch on it
7 quickly. The evidence listed here was
8 theoretically included in the review process, but
9 in fact, none of it was effectively considered.
10 And one of the other issues that has come up
11 repeatedly is the use or the role of subject matter
12 experts. Now, since part of what we are here to do
13 is make recommendations, my recommendation is a
14 multidisciplinary panel at this level is very
15 effective, but subject matter experts, I think are
16 required for the analysis, because only they can
17 look deep enough into the studies that are cited,
18 only they can look at the premise of the study to
19 see whether it's valid in terms of being consistent
20 with clinical practice.
21 And I think that you have to understand
22 the role of the clinical practice guidelines. In
23 the real world, they define the standard of care.
24 And where you have a multidisciplinary group that
25 doesn't have the background in the subject area,
.00187
1 they are not necessarily going to know to ask for
2 this. And I think one of them said that. And I
3 think it's disingenuous to say it was on the list
4 of materials. As far as the clinicians are
5 concerned, if you want to put together an
6 authoritative evaluation, you have to start there
7 and diverge from it, but --.
8 We have already covered the fact that
9 the guidelines found a very different conclusion
10 but as Dr. Whyte acknowledged, nobody's really
11 disputing the fact that biofeedback is effective.
12 The question really comes down to a comparative
13 one. This is the study that I wish had been in
14 there, and was excluded on the basis of the
15 comparative effectiveness issue. And the reason I
16 have this up here is not just because its
17 conclusion is so definitive, but because one of the
18 authors who was cited in the TEC report has
19 declared that this is the best constructed study
20 that's ever been done in this area.
21 And obviously we covered this this
22 morning, but as you can see, panel members were a
23 bit frustrated, and I don't think that content
24 expertise was overlooked, but if you ask Lisa
25 Landy, it was just outvoted.
.00188
1 I'm running out of time. Can I have a
2 little more time? Dr. Whyte and Dr. Garber had a
3 lengthy time to be able to make their case. Can I
4 just have a few more?
5 MS. CONRAD: You want two more minutes?
6 MR. K. CONNOLLY: Okay. I'm going to
7 just skip to the most important part of this thing,
8 which is, I think a lot of the issues came down to
9 this question, the question of the question, as we
10 phrased it, so I would like to examine whether this
11 was an appropriate question. As I said this
12 morning, in actual clinical practice, these are
13 sequential, they are not competitive procedures.
14 And HCFA's own experience at a regional level in
15 covering them, there are explicit exclusion
16 criteria. Patients have to have failed with more
17 conventional treatments, including PME.
18 Now, there is some amount of plasticity
19 to the staging of this, but I don't think too many
20 UI experts would disagree with this basically.
21 Now, the real action for HCFA is on the side,
22 because that's where you start paying out dollars,
23 after patients have failed on the left side.
24 And just very quickly to go over what
25 biofeedback is and what its clinical function is
.00189
1 for UI, the key point is it's only used when it's
2 needed. This is where it's needed, not for the
3 folks on the left, but for the patients on the
4 right. The Bump study that another one of the
5 presenters referred to, found with urodynamic
6 studies that not only do half the people not do
7 pelvic floor exercises correctly on the basis of
8 verbal instruction, but half of them do it in a way
9 that's clinically counterproductive, i.e., doing
10 things that are likely to increase an incontinence
11 problem.
12 In light of this, in light of the fact
13 that biofeedback is not a first line intervention,
14 and explicit exclusion criteria exists, and in
15 light of the fact that that's actually how it's
16 used, I think that the panel should have been asked
17 a question more like this: Does the evidence
18 support a sizable health effect from the use of
19 biofeedback for those UI patients who have failed
20 to respond to PME alone?
21 MS. CONRAD: Time please. Thank you.
22 MR. K. CONNOLLY: I'm sorry I can't get
23 to my next point, because it's really one of the
24 most important ones.
25 DR. SOX: The committee always has the
.00190
1 option of asking you later on during discussion.
2 MR. K. CONNOLLY: All right, thanks.
3 MS. CONRAD: Francie Bernier please.
4 MS. BERNIER: I am Francie Bernier. I
5 am a clinical consultant and on staff at Rose
6 Medical Center in Denver. I've had a large
7 continence program in Denver for the past eight
8 years.
9 In 1948, Dr. Arnold Kegel described the
10 use of pelvic floor exercises to treat urinary
11 incontinence and other pelvic floor dysfunction.
12 His research provided the beginning for nonsurgical
13 rehabilitation programs to treat incontinence.
14 Over the last 50 years, Kegel exercises have been
15 prescribed and offered as the first line treatment
16 for incontinence. Although the medical profession
17 understands the usefulness of this therapy,
18 physicians have never been taught how to do
19 adequate pelvic floor muscle contractions. As
20 clinicians offering continence care know, just
21 handing an exercise instruction sheet to a patient
22 or telling a patient to just squeeze the Kegel
23 muscle, has never provided adequate instruction.
24 You are faced with the challenge of
25 deciding the fate of biofeedback and the probes for
.00191
1 electrical stimulation used in the treatment of
2 incontinence. The decision was based on the TEC
3 report, which claims there is a lack of scientific
4 evidence to support utilization of this therapy.
5 During the meeting, during the previous meeting,
6 the question posed to the panel was changed three
7 times, demonstrating a lack of understanding of
8 what was to be decided. Additionally, most of the
9 panel members were not familiar with the supportive
10 scientific literature or therapeutic value of a
11 continence program. The panel agreed with the TEC
12 report and therefore decided there was a lack of
13 randomized double blind placebo controlled
14 studies. The incomplete and damaging information
15 the TEC report provided has put the coverage of
16 this therapy in jeopardy.
17 The U.S. Department of Health and Human
18 Services created the Agency for Health Care Policy
19 and Research, AHCPR. AHCPR was established in 1989
20 under Public Law 101-239 to enhance quality,
21 appropriateness, effectiveness of health care
22 services and access to these services. AHCPR
23 carries out its mission by conducting and
24 supporting the general health services research,
25 including medical effectiveness research findings
.00192
1 and guidelines, and dissemination research findings
2 and guidelines to health care providers, policy
3 makers, and the public.
4 The TEC report clearly ignores the AHCPR
5 guidelines, which reviews all of the literature and
6 reports its findings on strength of evidence. For
7 the sake of time, I'm not going to go into all of
8 the strength of the evidence. However, just to
9 mention that the strength of evidence for
10 biofeedback, pelvic muscle exercise, and bladder
11 inhibition augmented by biofeedback, was given a
12 strength of evidence of A, and pelvic floor
13 electrical stimulation was given the strength of
14 evidence of B.
15 Another concern voiced by our clinician
16 was the lack of review of very appropriate studies
17 by the TEC report. With so many clinical studies
18 demonstrating usefulness of this therapy, it seems
19 amazing the studies reviewed by this group
20 eliminated those which the expert field reports is
21 statistically significant information to support
22 the use of biofeedback and electrical stimulation
23 for the treatment of incontinence.
24 Additionally at the previous meeting,
25 one panel member chose not to vote. He reported he
.00193
1 was not provided the information to make an
2 informed decision. He claimed he was not even
3 aware the AHCPR guidelines were available. He
4 appeared obviously angered, confused and
5 disappointed, and frustrated with the situation.
6 It appeared that other panel members felt the same
7 way.
8 Interestingly, one statement was made on
9 behalf of the panel. Without question, we all
10 agree, we would have this therapy in our office.
11 Looking at the lack of randomized studies on
12 biofeedback directed pelvic floor exercises, it
13 makes the clinicians wonder how a study like this
14 can be done. The TEC report excluded the
15 observational studies that report the outcomes of
16 the continence programs. They excluded very strong
17 and thorough studies on pelvic floor rehabilitation
18 as compared to drug therapy. There have been
19 randomized double blinded placebo controlled
20 studies on electrical stimulation, reported by
21 Dr. Pearcy and at the April meeting.
22 Over 20 million Americans are calculated
23 to be experiencing urine loss today. These people
24 are over the age of 65 for the most part.
25 Treatment options are limited to medication,
.00194
1 surgery and behavioral therapy. The use of the
2 medications carry a risk of severe side effects
3 affecting daily life. Patients report such
4 significant side effects as intense dry mouth,
5 inhibiting their ability to speak. Other side
6 effects are constipation, which is known to
7 hospitalize patients in some instances, dry mucous
8 membranes which contribute to vision problems and
9 general malaise. Most patients abandon the use of
10 the medications to the due to the side effects of
11 the drugs.
12 Additionally, these medications are very
13 costly. Most of the elderly population are
14 Medicare subscribers. For those on a fixed income,
15 the cost of Ditrepam XL for a one-month supply is
16 over $200, or $2400 per year. Drug therapy is cost
17 prohibitive. With no side effects from biofeedback
18 and electrical stimulation, why is this not offered
19 as a first line therapy for patients? The cost of
20 therapy is generally half the cost of Ditrepam XL.
21 Surgery is even more expensive. The complication
22 rate, morbidity and mortality rate is very high in
23 the elderly.
24 Finally, consider the 1993 report from
25 the Alliance for Aging, which reported that $22.5
.00195
1 billion could be saved by the year 2000, if the
2 incidents of incontinence were reduced by 20
3 percent. This report also described how $80
4 million could be saved annually if surgical
5 patients also appropriate for behavioral therapies
6 were treated with behavioral interventions.
7 The April meeting demonstrated a bias
8 and predetermined outcome of biofeedback and
9 electrical stimulation. I and the hundreds of
10 patients my colleagues and I have successfully
11 treated hope you will thoroughly take a look at the
12 major concerns we voice today. Take into account
13 the many medical, nursing, physical therapy
14 organizations which support the therapy, and please
15 keep these codes and treatments available for
16 patients who suffer from urinary incontinence.
17 Thank you.
18 MS. CONRAD: Thank you.
19 DR. SOX: Thank you very much. I was
20 asked to say that Dr. LeFevre, who did the TEC
21 report, is actually on the telephone someplace and
22 if we have questions for him, we can ask them and
23 he will be able to respond in a way that we can all
24 hear.
25 Dr. Hill wanted to make a brief remark
.00196
1 regarding the assertion about the effect of our
2 decision on coverage.
3 DR. HILL: Thank you, Chairman Sox. I
4 wanted to make sure that it was understood that the
5 effect of ratification by the Executive Committee
6 of the panel's recommendations functions as advice
7 to HCFA. It will inform our coverage decision. We
8 will have to weigh that recommendation with all the
9 other information. It doesn't result in an
10 automatic taking away of coverage. I won't
11 prejudice by speculation what the outcome would be,
12 but we could decide to cover, noncover, local
13 discretion, we can leave it up to local discretion,
14 we can cover with limitations, we have a number of
15 options open to us, and this will be a piece of
16 advice that we consistently said fits into the
17 matrix that we have to use to make the decision
18 that is ultimately our responsibility. Thank you.
19 DR. SOX: Thank you. We now come down
20 to the point of having a proposal to discuss, and
21 perhaps I could take a minute to lay out what I
22 thing are the options.
23 The first is that we could approve the
24 recommendations of the panel, either separately for
25 biofeedback and for pelvic floor stimulation, or do
.00197
1 them together. And if we were to vote that way, we
2 would say that the process was good, it was close
3 to or as close to as we can get for our first time
4 around, to the process that we outlined as an
5 Executive Committee, and that the committee made a
6 correct interpretation, that is to say that the
7 evidence really is inadequate to make a statement
8 about whether these technologies are effective or
9 not in the way that the question is framed.
10 The second thing that we could do would
11 be to disapprove the recommendation of the panel,
12 which I think means that we felt that the process
13 they used was a reasonable process that covered the
14 evidence, but that they simply made the wrong call,
15 that in fact there is adequate evidence to make a
16 decision about whether this works or not.
17 The third option would be to send the
18 thing back to the panel, which I would suggest
19 would be because the process was flawed in
20 important ways that if corrected, would lead to a
21 high probability of a changed recommendation if it
22 came back to us.
23 So in a way, I think we first of all
24 have to address the question, was the process a
25 good process and one that would allow a decision
.00198
1 about whether the evidence is adequate or not, and
2 if we decide that the process is a good process
3 then we have to decide whether the committee in
4 fact made the right call in stating that the
5 evidence is inadequate to make a decision about
6 whether it works or not. Alan or Mike, do you want
7 to comment on that formulation, whether that makes
8 sense to you, and how you might suggest that we
9 proceed here?
10 DR. GARBER: Yeah, Hal, if I understand
11 how the way that you defined it, it does come down
12 to breaking it up between whether the process was
13 good and whether the panel followed the process,
14 and I think that's perfectly fine.
15 DR. SOX: Michael?
16 DR. MAVES: I feel that is the
17 appropriate way to look at it, and I would concur.
18 DR. SOX: Well perhaps then, unless
19 somebody else has a serious objection, why don't he
20 we talk for a little while about whether the
21 process as outlined by Alan and Mike was a
22 reasonable process and one that in fact would allow
23 them to make an informed decision up or down about
24 whether the evidence is adequate. So let's
25 concentrate now on the question about process for a
.00199
1 while, and just see whether we have a consensus on
2 whether the process was adequate or not. Ron?
3 DR. DAVIS: Just a question that picks
4 up on your question. We had a nice explanation
5 from Dr. Whyte about the reasons why the questions
6 were framed the way they were. I'm wondering
7 whether that explanation was provided to the panel
8 so they had an equal understanding of that?
9 DR. SOX: Alan, can you respond?
10 DR. GARBER: That's a good question, and
11 the way I would answer it, we had a conference call
12 before the panel meeting, and I had a pretty clear
13 idea of that. It was not said in so many words all
14 at one time the way John Whyte did it today, but
15 the pieces were there at least for the panel
16 members. I'm not as certain that that was done for
17 the general public, but we got it from the
18 conference call.
19 DR. DAVIS: So procedurally, I would
20 suggest that we take care to explain why the
21 questions are worded the way they are for both the
22 panel members and any members of the public who are
23 involved in the process or in attendance.
24 DR. SOX: Are you satisfied with Alan's
25 explanation or do you think the committee didn't
.00200
1 understand the question and was kind of off base to
2 start with, based on what you've heard so far?
3 DR. DAVIS: With all that we've heard
4 today, I'm reasonably satisfied that the process
5 was a good one, not a perfect one, not without some
6 flaws that can be improved upon in the future, but
7 I think overall, they did a very good job and good
8 enough in my mind that we can move forward in
9 making a decision.
10 DR. SOX: Randel?
11 MS. RICHNER: I think when I reread the
12 transcript, including I think Dr. Epstein's
13 discussion, was very relevant to this discussion,
14 and I think beyond the point of whether the
15 questions were understood was once again, getting
16 back to what their purpose was in terms of whether
17 it was a coverage decision or evaluating that very
18 narrow question of medical evidence. Because I
19 mean, when I read Dr. Epstein's note, it's very
20 very relevant to this discussion, and I think
21 everyone needs to think about, once again, what is
22 important for the Medicare beneficiary and whether
23 or not that was achieved in that first panel.
24 For instance, when Arnie said one is,
25 you know, the whole idea about making coverage
.00201
1 decisions. One is, we're going to cover procedures
2 where there is clear scientific evidence indicating
3 the procedure is effective or efficacious, and you
4 would like both. The second runs orthogonal to
5 that; it says in the face of broad consensus from
6 medical experts that a procedure is effective,
7 we'll cover it absent evidence that it's not
8 effective, so long as the clinical down sides are
9 minimal. What's making everyone uncomfortable is
10 that the scientific question that is designed to
11 lead to the former approach, as opposed to the
12 latter approach. We should have asked two
13 questions: Is the scientific evidence adequate to
14 show that the procedure works? The answer was no.
15 Is the scientific evidence adequate to show that
16 the procedure doesn't work? The answer was no.
17 And that's pretty relevant to this, and
18 I hope the essence of what you have just proposed
19 brings in that flavor as well.
20 DR. SOX: As you noted, some people
21 raise the question about whether the scientific
22 evidence was adequate enough to say it doesn't work
23 or doesn't add anything.
24 Well, I'm eager to sort of cut to the
25 chase, so I guess I'd like anybody who really feels
.00202
1 as if the process was seriously flawed and would
2 have made it difficult for the panel to reach an
3 appropriate decision about the adequacy of the
4 evidence to speak up, so that we can kind of cut to
5 the chase in this discussion. John?
6 DR. FERGUSON: This is kind of hard to
7 explain, perhaps, but I don't think that the
8 process is flawed in the larger sense, in that we
9 have an interim report which is sort of a
10 blueprint, and we have an evidence gathering that
11 was done and presented and given to the panel, and
12 we have a series of questions asked by HCFA to the
13 panel.
14 However, semantically, I think what
15 happened makes the process in this particular case
16 flawed, and the reason or reasons that I think so
17 are this: By asking the question, is the evidence
18 adequate, it sort of assumed, adequate for a
19 positive answer is implied, and forced the panel
20 new a yes or no vote. In my view, that seemed to
21 obstruct rational discussion and deliberation of
22 the evidence. And in that sense, in my view, the
23 process was flawed.
24 I would like to mention that it seems to
25 me that both the evidence reports for biofeedback
.00203
1 and for pelvic floor stimulation, actually did
2 reach conclusions, in which there were conclusions
3 stated, yet the panel was not really allowed or
4 because of the semantics and the way the question
5 was asked, to reach conclusions, they were forced
6 to go yes or no on the adequacy. So I feel that
7 that was a snarl in the process, which I mean on
8 paper in the large sense, is a good process and I
9 believe, I am very much a believer in evidence
10 based medicine and the necessity to have good
11 studies. But in this case the way the questions
12 were phrased and basing it on this interim report,
13 to me led on a process that did not allow the panel
14 to debate the evidence, and that I think is not
15 proper.
16 DR. SOX: Alan, you and Michael were
17 there. Do you want to comment on John's
18 assertion?
19 DR. GARBER: I don't agree with the
20 position that the interim recommendations in any
21 way limited debate on the questions that John
22 raised. And there are many ways to approach this,
23 and I thought about this a great deal too, and I'm
24 sympathetic with John's point. One approach we
25 could have taken as a panel that wasn't quite
.00204
1 allowed by the interim guidelines, would have been
2 to say what we thought the effect was, and I think
3 this is what John was alluding to, even if we
4 thought the evidence was inadequate. But the more
5 I thought about it, the more meaningless I thought
6 that any such statement would be, simply because if
7 you don't think you can draw conclusions because
8 the scientific evidence, either the studies have
9 tremendous biases, and I'm not referring to this
10 specific topic now but thinking in terms of general
11 recommendations for how the panels should operate,
12 if you really think the evidence base isn't
13 adequate, what is the meaning of saying there is a
14 slight benefit, that's our point estimate, and the
15 confidence regions include some horrible detriment
16 and some greatly large benefit. So, I'm struggling
17 with the same issue as John. I think this is
18 something that we will have to revisit again, but I
19 actually think that the interim recommendations
20 worked well in this context, and I don't think that
21 had we chosen a different approach that the panel
22 would have reached a different conclusion. And
23 again, that's surmise based on what I heard the
24 panelists say. Mike?
25 DR. MAVES: I would agree. I think that
.00205
1 the answers to the questions would have been the
2 same. I do think it was interesting and part of
3 the discussion really revolved around what was the
4 effect of the intervention, and a number of the
5 medical specialty societies gave opinions, and I
6 think the range was somewhere in the four to five
7 range on our seven point scale. We never actually
8 got there as a committee in deliberation. And I
9 think that, again, would be something that the
10 Committee looking at the process might want to
11 wrestle with a little bit, is there some advantage
12 perhaps to integrating that. But as Alan has
13 indicated, with the body of evidence that was
14 reviewed, and I think for a lot of us looking at
15 those studies and going over that material, it was
16 certainly not as clear as one like to see. I think
17 the answer would end up being the same even if you
18 had gone around the other way.
19 But I do think as a matter of process in
20 the future, perhaps looking a little bit at what is
21 the effect of this, if you back off a little bit
22 and say, just give me an estimate of the treatment
23 effect, you know, without a yes or no answer on
24 scientific evidence, might help the process a
25 little bit and again, might give a roundness and
.00206
1 some idea. I think someone mentioned earlier,
2 should we let the individuals know what kind of
3 work needs to be done in the future to jump this
4 hurdle. That might be very very helpful to those
5 that are trying to get this technology accepted and
6 covered by HCFA.
7 DR. SOX: Bob?
8 DR. BROOK: Well, one, the process is
9 much better than it was for the last panel. Two,
10 I'm a little sad that the evidence based report is
11 not more readable in terms of an executive summary
12 so the public can really understand what the
13 evidence shows. I don't think that this report is
14 readable, so it's going to be hard for HCFA to use
15 it in any positive way. It could have gone through
16 a process of summarizing, like evidence based
17 medicine does for different diseases, and it could
18 have been a chapter on urinary incontinence, these
19 kind of issues and that kind of a format, and I
20 think that would have been easier to understand at
21 the end.
22 We've heard about problems in the
23 process, timeliness and whatever, but I've been
24 really dismayed that after 50 years of introducing
25 all this technology, the level of science is so
.00207
1 poor, and it's being used so poorly by the people
2 that have come before us. That really is what has
3 dismayed me more than anything else. I mean, if
4 this is a sequential procedure, fine, but if you
5 have a randomized trial at the beginning and you
6 know that half are going to fail, you still will
7 pick up that difference if you power it enough.
8 So, these are not insurmountable questions, and
9 it's not like assigning people to randomized
10 cardiac surgery.
11 So, I've been impressed that no matter
12 how much I have tried to pull out of any of these
13 letters or testimony, I don't think see any
14 evidence that comes to me to argue that the process
15 did not identify the evidence, that it did not deal
16 with this. So, in that regard, I think the process
17 is fine.
18 I am a little bit sad, however, that it
19 almost appears like a setup, and let me give you
20 the example. If indeed, pelvic floor exercises,
21 for instance, are being dealt with by handing out a
22 piece of education, and if indeed there are lots of
23 clinicians who know that women who get this fail,
24 that's true, and you've got a lot of testimony for
25 that. And if you know that you can then apply
.00208
1 biofeedback, for instance, to that group, and you'd
2 get a lot of people that succeed, but you also
3 know, these are also facts, then the question is,
4 how is the material that we produce here going to
5 be used? That's the real problem and that's the
6 dilemma, and I think we need to caution HCFA in
7 terms of either saying, it's your responsibility or
8 somebody's responsibility to answer the real world
9 question of, does pelvic floor exercises for
10 example, used in the way it's used in the
11 community, versus that with biofeedback used in the
12 way it's used in the community, make a difference?
13 I see no evidence that there has been any studies
14 to answer that question.
15 And the scariness of all this is that I
16 don't see any evidence that when we finish our
17 deliberations, anybody will do this in the next
18 decade, and that to me is the dilemma. And I at
19 least, if we approve this report, and I don't see
20 how we cannot, with basically the testimony we have
21 heard today, we would basically want to add some
22 caveat that there has to be some responsibility
23 here to quickly and definitely address some of
24 these questions, and in order to discharge our
25 responsibilities socially and in a responsible way,
.00209
1 because I think Tom is right.
2 I believe that if somebody had any one
3 of these conditions, with so little of a benefit
4 risk to the patient, no risk, that people would
5 want to try everything they possibly could to get
6 rid of this problem before they went to drugs or
7 surgery, a large number of people would want that,
8 and that there's probably some added benefit to
9 this, but unfortunately, nobody has shown it.
10 That's the sad part of this, that unfortunately, it
11 just ain't there. And you coming up and showing
12 slides and all this other kind of stuff, I'm sorry,
13 you didn't make -- to me, the public did not, the
14 advocates of this procedure did not meet the case
15 of the evidence, that there's evidence there to do
16 this, from the standards of evidence that one uses
17 in this field.
18 But the problem is that since this is
19 not a billion dollar drug market, where comes the
20 incentive to get those things done quickly so that
21 we don't really harm people by getting rid of a
22 therapy which if you really tested it out in real
23 world circumstances well, you would show a marginal
24 benefit that's worth funding? That's the problem
25 I'm faced with.
.00210
1 But the process is, I think -- I have
2 seen no evidence to support, even though I was very
3 critical this morning, I see no evidence to
4 support, from both this afternoon and morning
5 session, that there is anything here that would
6 indicate that we should overturn the panel's
7 deliberations.
8 DR. SOX: Well, you could make a case,
9 Bob, that the fact that this panel exists, that we
10 have a transparent process for evaluating the
11 literature is the key point of departure for
12 improving the situation that you're decrying, and
13 that we all recognize as a big problem, but that's
14 kind of a side point.
15 DR. BROOK: Well, I'll just make one
16 last point. It happens for most services that
17 involve functioning, rehabilitation, these kinds of
18 things, it happens over and over again, and there's
19 no constituency to study them, and there's no
20 funding from the government to study them. It
21 would almost be really nice for this Committee to
22 say to HCFA, we ought to cover it because the
23 government is being irresponsible to fund the
24 studies to do this, and as long as we have these
25 things, there is no evidence to support funding it,
.00211
1 but it ought to be funded anyway because the
2 government is socially irresponsible.
3 DR. SOX: I want to try to keep the
4 conversation on point, if I can. Again, I'm
5 looking for somebody to speak up and say this is a
6 seriously flawed process, it's so flawed that if we
7 send it back to the committee, that there is a
8 fairly high probability they would come to a
9 different conclusion, if those flaws were fixed.
10 And I'm not hearing anybody so far who's willing to
11 say that. And if they are not, then I think we
12 ought to move on to the point of making a formal
13 motion to decided whether or not the evidence is
14 adequate based on the process, which we seem to be
15 implicitly if not explicitly endorsing. Leslie?
16 DR. FRANCIS: I want to ask a couple of
17 questions about that, but before I do that I want
18 to say that I think we all understand that one
19 question about the process is, was it limited in
20 ways that later processes might not be, and that's
21 part of what the working group is going to be
22 doing, it's going to be opening things up more.
23 The questions that I have about whether
24 it was flawed in its own terms, given the kind of
25 process it was and that we set up for you to do, is
.00212
1 first of all, in the report, the AHCPR report that
2 didn't get to people, was there evidence of the
3 kind you would have considered that people didn't
4 have, and was there an adequate amount of time for
5 a variety of commentators and so on to try to bring
6 that evidence to the committee? Because it does
7 seem to me that if there was evidence out there
8 that you didn't get for some reason, that would
9 mean the process was flawed on its own terms. And
10 I just want reassurance on the answers to those
11 questions.
12 DR. GARBER: Leslie, there are flaws and
13 there are flaws, and I already said, I think it
14 didn't work that well; if I had to do it over
15 again, I would have made sure that the panel
16 received a copy of the report. Did that flaw in
17 the process affect the outcome? I don't believe it
18 did so at all. I think that it would have been
19 good background material; I don't think any primary
20 matter data were missing by the lack of the
21 availability of the AHCPR report. And it's been
22 pointed out repeatedly, that was addressing a
23 slightly different topic. So the AHCPR report
24 might have had different significance if the
25 question had been biofeedback plus exercise
.00213
1 compared to placebo, or nothing. So no, I don't
2 think that affected the outcome, even though I
3 believe that the process was flawed in a minor way.
4 DR. FRANCIS: Thank you, you answered
5 the question.
6 DR. SOX: John.
7 DR. FERGUSON: Just a comment. I think
8 that the panel could have come to the conclusion of
9 inconclusiveness, that the data was inconclusive,
10 or they could have come to a conclusion that the
11 data was, did not point in the direction of
12 positivity, or that it was suggestive but not good
13 enough, but they weren't able to do that because of
14 what I think is sort of a false barrier of voting
15 on adequacy. Now maybe I'm all wet, but it seemed
16 to me that the evidence was adequate upon which to
17 base a conclusion, and the conclusion could have
18 been it's not very good evidence. And they weren't
19 allowed to do that, and that to me -- or at least
20 it didn't seem to me that they -- and I'm not
21 blaming you, I think it was a combination of the
22 way the questions were structured, plus this
23 blueprint which I think needs a little bit of
24 tweaking. So I think that that is not quite
25 proper, that they weren't allowed, or weren't -- it
.00214
1 wasn't structured in such a way that they could
2 evaluate the evidence and say the evidence is poor
3 or the evidence is good.
4 DR. SOX: Alan, do you want to comment?
5 DR. GARBER: Like I said before, I think
6 John has a point, and we'll have to struggle with
7 how to deal with these kinds of situations. In
8 terms of the bottom line, I don't think this had
9 any effect, but we have to be sensitive to this
10 issue about where the evidence is so-so, we might
11 be very confident, if I could rephrase part of what
12 John said, or paraphrase him perhaps, we might have
13 been confident that there was no significantly
14 large effect. That's -- and I'm not saying that's
15 the case here, but that's the kind of conclusion me
16 might reach, the evidence is murky, we're quite
17 confident there's no large effect, and it could be
18 detrimental. That's the kind of situation we might
19 want to handle with a different procedure than we
20 used here, and I think we should be aware of that.
21 I don't think that rises to the level of a
22 fundamental flaw in the process, as it applied in
23 this case.
24 DR. BROOK: But I do think, I mean, it
25 does raise the question, I think we are all groping
.00215
1 with this, why does the government choose to fund a
2 $100 million study of carotid enterectomy and not a
3 $500,000 study of urinary incontinence? And it
4 does raise the question, and I think to discharge
5 our responsibility in the area where Alan at least
6 has described the studies as being flawed,
7 difficult, not realistic, in general poor, that we
8 ought to make some statement that this is something
9 from the clinical testimony that they heard that by
10 our action, we do not mean to suggest that this
11 therapy ought to be relegated to leeches, that it
12 looks like there's something here, and we think the
13 government ought to pursue this vigorously to try
14 to see if there is ways of producing the evidence
15 quickly to either refute or substantiate that
16 claim.
17 There's enough solid clinicians who
18 really are reputable human beings, that argue that
19 we missed the effect, and just to drop this with
20 this statement would do a hell of a lot of damage
21 to the field. That's what I'm really sad about in
22 terms of where we are at, and I will predict that
23 that's what, if there's any impact at all of what
24 we will do, it will be that, and it may be the
25 wrong impact.
.00216
1 DR. SOX: Well, we're certainly learning
2 that the playing field is not level and that
3 certain technologies, because they're produced by
4 relatively poorly capitalized operations have a
5 recurrent system of not as good a chance of having
6 an adequate test as those that are produced by well
7 capitalized organizations. Hugh, did you want to
8 say something?
9 DR. HILL: Some of my plastic surgical
10 colleagues tell me that for a narrow defined
11 population in certain situations, leeches are still
12 quite useful.
13 DR. BROOK: That's true by the way,
14 especially fingers.
15 DR. HILL: I just want to say, it looks
16 like you're heading towards a vote or some
17 conclusion about the process itself, and pardon me
18 for being legalistic, I think that's my role here
19 though. You're not going to vote on the process?
20 Okay, well that ends that.
21 But let me just say this briefly about
22 the ratification. The panel is asked in the
23 charter to review and ratify panel reports, and
24 submit the report to HCFA. And so, I would hope
25 that you would decide whether or not to ratify
.00217
1 based on whether given the questions and the
2 process and the vote of the subpanel as a closed
3 system, was it internally acceptable? You could
4 refuse to ratify as some form of protest about the
5 questions we asked or whatever, ratify is well
6 defined at this point, or you could ratify with
7 comments that would indicate your feeling as a
8 panel about us and about the result in your advice
9 to us separately from that.
10 But I also wanted to point out that on
11 the presentation of any new evidence, if this does
12 happen to stimulate new evidence or if it's coming
13 in anyway, requestors can request reconsideration
14 the day after we issue a decision on the basis of
15 new evidence, and we'll have to look at it again.
16 DR. SOX: Okay. Well, I'm really eager
17 to move on, so if there is anybody else who wants
18 to make the case that the process was flawed in a
19 way that would lead to a different decision if it
20 was to be reconsidered, now is the chance to speak
21 up, because what I'm hearing is general consensus
22 that it was an adequate process and that we need to
23 move on to a decision about whether or not to
24 endorse the committee's recommendations. Randel?
25 MS. RICHNER: I just want to make sure
.00218
1 that it's on record that I think that the process
2 was flawed, and I think that it's important to note
3 that I think that the whole issue of what our
4 mandate is as a committee needs to be clarified and
5 needs to go on record. I think that the coverage
6 criteria is critical. I think HCFA needs to give
7 guidance as to what kinds of questions we need to
8 answer, and it's much broader than just looking at
9 the adequacy of the scientific evidence. And I
10 think that in a sense you did what you were
11 supposed to do in a narrowly defined way for that
12 particular panel meeting on April 12th and 13th,
13 but it didn't do service to, or justify or help the
14 overall mandate on our mission of what we were
15 supposed to do.
16 I am still absolutely surprised that all
17 of these different associations all agree to
18 support this. In my clinical experience and my
19 industry experience, you rarely see that, and there
20 has to be some kind of weight put on that kind of
21 endorsement.
22 DR. SOX: Well, let's move on then.
23 Alan, actually I'd like your advice and Mike's
24 advice about whether we should have a motion for
25 biofeedback as an adjunct to pelvic muscle
.00219
1 exercises, and a separate one on electrical floor
2 stimulation, or whether to do them both together.
3 Do you have an opinion?
4 DR. GARBER: I have a procedural
5 suggestion, which is to take it as a whole and if
6 the panelists in the discussion indicate that they
7 feel there's some reason to distinguish them at
8 that point, to separate them. But I think it's
9 unlikely that the Executive Committee would vote to
10 ratify one and not the other.
11 DR. SOX: In that case I would like to
12 call for a motion.
13 DR. BERGTHOLD: Can I ask a question of
14 process? I notice that we have open public
15 comments before we take --
16 DR. SOX: Yeah. The plan will be to
17 have a motion, to have discussion, then we'll have
18 public comment and then come back to brief
19 discussion and vote. But I want to get something
20 on the table so we can have a conversation. Ron?
21 DR. DAVIS: I would like to make a
22 two-part motion, if I could. One would be just to
23 get the issue out on the table and to facilitate
24 action, that the Executive Committee ratify the
25 recommendations of the panel.
.00220
1 DR. SOX: Okay, there is a motion. Is
2 there a second?
3 DR. HOLOHAN: Second.
4 DR. DAVIS: Can I mention the other part
5 of the motion, or a second motion?
6 DR. SOX: Please do.
7 DR. DAVIS: Picking up on Bob Brook's
8 suggestion that the Executive Committee encourage
9 HCFA to open a dialogue with appropriate funding
10 agencies to discuss the need for good research on
11 the treatment of incontinence, that would help
12 inform future decisions and actions on Medicare
13 coverage.
14 DR. SOX: I think we should treat those
15 as two separate motions. Is there a second to the
16 second motion?
17 DR. BROOK: Second.
18 DR. SOX: Let's not talk about the
19 second motion, let's just talk about the first
20 motion for a while, because my sense is the second
21 one, probably everybody's going to think that's a
22 good idea, so let's focus on the first motion,
23 which is to ratify the panel's recommendation.
24 One question I guess we ought to address
25 right away is whether we should pull any particular
.00221
1 element of their actions out of this blanket
2 motion, because the evidence for it looks like it
3 might be treated differently than the rest of the
4 evidence. Anybody want to pull a piece of this out
5 for separate consideration? Good. Okay.
6 Let's talk about the motion, which is
7 basically to endorse the committee's conclusion
8 that the evidence is inadequate to draw a
9 conclusion about the effectiveness of these
10 procedures. Yes, Bob?
11 DR. MURRAY: Will we have an opportunity
12 to explain our vote after the vote is taken, so we
13 can put comments new the record? If so, I'll
14 withhold my comments until then.
15 DR. SOX: Not only opportunity, but I
16 think requirement.
17 DR. MURRAY: I'll save my comments then.
18 DR. SOX: Well, hearing no discussion, I
19 think it's now probably time for us to go into
20 public session and hear from folks who are here who
21 would like to make comments before we actually take
22 a vote. So anybody who would like to make a
23 comment on the discussions so far, please step to
24 the microphones.
25 MS. CHRISTIAN: Hi. My name is Martha
.00222
1 Christian. I am a health policy and clinical
2 outcomes panelist for EMPI. I just have a couple
3 of concerns in evaluating the process. One of the
4 things I spent a lot of time with doing is looking
5 at the MCAC charter and the federal guidelines for
6 the whole MCAC process. One of things that those
7 documents are very clear on is that the role of
8 this Committee is to make advice on coverage. In
9 fact, the vote that it actually specifies in those
10 guidance documents, that the panels are supposed to
11 make vote on national coverage. From that
12 perspective, the Committee failed in its duties as
13 defined by HCFA to vote on coverage, so you didn't
14 vote on the right question, and part of that is
15 because HCFA didn't give you guys the right
16 question to answer. So I would have to object to
17 that, and feel the process is fatally flawed
18 because of that.
19 Secondly, I'm also very concerned
20 following this morning's decision to have a
21 subcommittee. I think that's a wonderful idea;
22 this is a great process, it has great opportunity
23 to make sure that the process is inclusive and open
24 and we can get some good advice and input into the
25 coverage decision making process. However, to
.00223
1 subject PFS and biofeedback, and to make a decision
2 based out of this process that has been determined
3 that, one, we didn't do it quite right, that there
4 were some problems, it's a work in process, we're
5 sort of inventing the process as we go along, it's
6 really unfair to our technologies to make decisions
7 based on a process that isn't as good as it could
8 be, and future technologies are going to have the
9 benefit of a better process, and that would concern
10 me greatly both as a representative of a technology
11 but also as someone who has parents who are
12 Medicare beneficiaries.
13 As a person who's a geriatric former
14 long-term care administrator, I see the effect of
15 these policies every day in my career and I'm very
16 concerned that Medicare beneficiaries are going to
17 be harmed greatly by the fact that this technology
18 isn't going to get a fair hearing that's consistent
19 with what's going to happen down the road. Keep up
20 the good work, keep working on your process, but
21 don't penalize these technologies because we aren't
22 quite where we should be. And, I guess I would
23 encourage you not to ratify this, or at least say
24 that the process was screwed up. Thank you.
25 DR. SOX: Thank you. Middle mike,
.00224
1 please.
2 MS. CHAPPELL: My name is Jodi Chappell,
3 I'm manager of regulatory affairs for AUGS, and we
4 would agree with the process that you are, it is in
5 evolution and we support that, and we want to
6 continue our work, to work with HCFA and work with
7 the MCAC committees and panels to insure that a
8 clinician's point of view is represented.
9 I wanted to also commend Randel for
10 paying close attention to the transcripts,
11 especially the end of the first day and the
12 beginning of the second day, there was a lot of
13 verbal communication back and forth among the
14 panelists, and this might -- I have not reviewed
15 the minutes of the meeting, I have not seen that,
16 and I understand they were posted, but I have not
17 found those yet. But the review of the minutes by
18 some panelists were concerned that those comments
19 and dialogue were not reflected, so I would just
20 urge you to read that dialogue.
21 In addition, the questioning, the
22 concerns that Dr. Whyte was talking about, the
23 question that was posed, I would like to reiterate
24 a comment by panelist Dr. Lisa Landy regarding her
25 feedback on the questioning. The original question
.00225
1 posed to the panel in advance of the proceedings
2 was, is the scientific evidence adequate to draw
3 conclusions about the effectiveness of
4 biofeedback? This is what I base my primary review
5 on, and she served as a primary reviewer. As the
6 presentations proceeded the morning of the first
7 day, I realized the question had been changed to be
8 of a more narrow scope. The question was altered
9 to, is the scientific evidence adequate to draw
10 conclusions about the effectiveness of biofeedback
11 as an adjunct to pelvic muscle exercises?
12 This may seem like an insignificant
13 alteration, but actually changed the entire course
14 of the panel proceedings. This precluded
15 discussion of the efficacy of biofeedback assisted
16 pelvic muscle exercise as an intervention. The
17 focus was redirected to analysis of adequacy of
18 scientific evidence, comparing two types of
19 intervention, as opposed to clinical efficacy. The
20 scientific literature clearly supports the efficacy
21 of biofeedback.
22 Due to the panel being unaware of this
23 change until the day of, or if they were, they
24 weren't totally. I understand Dr. Garber
25 understood it and maybe -- and I appreciated your
.00226
1 comments about being told about it -- but I would
2 hope that all the panelists would clearly
3 understand the question. Due to these flaws, and I
4 support the definition that we need to come up with
5 some standardized terminology and we support those
6 efforts, and anything we can do to support HCFA and
7 the MCAC on that standardizing the terminology,
8 clinical evidence, adequacy, even biofeedback
9 itself, we would be supportive of. Thank you for
10 your time.
11 DR. SOX: Thank you very much. Left
12 mike?
13 MR. J. CONNOLLY: Jerome Connolly,
14 American Physical Therapy Association. There has
15 been some concern suggested that, or some concern
16 indicated why there aren't more studies, and I
17 think this goes right to the heart of the issue
18 again, of the narrowness of the question. And
19 there's so little at stake here relative to the
20 Medicare dollar that I'm wondering why this is an
21 issue that is requiring this much time and this
22 much effort, because I am not sure that a whole lot
23 of dollars are going out of the Medicare coffers to
24 pay for biofeedback enhanced pelvic muscle exercise
25 to warrant this kind of attention.
.00227
1 Yet there are a few studies relative to
2 this specific narrow question of comparative
3 analysis, a head-to-head study, that was asked.
4 And I think it goes right back to the question that
5 Jodi just reiterated that Dr. Landy had mentioned
6 in her letter. And the changes that were made to
7 the question, they seemed insignificant, but it was
8 a significant alteration. And Dr. Ferguson picked
9 up on that, in that it changed the entire course of
10 the proceeding in terms of the discussion. And I
11 think that that really needs to be weighed heavily,
12 because if you're talking about going forward on a
13 process that isn't necessarily fundamentally fair
14 or inclusive, or thorough, and in fact the primary
15 reviewer indicates that the discussion was changed
16 considerably by the nature of the question, then I
17 think you really need to consider where you are in
18 this process.
19 So it would seem to me that given this
20 from a primary reviewer, that you may want to
21 consider a remand, because if in fact the primary
22 reviewer were here today, I wonder if she would be
23 wondering if she was in the same meeting that we're
24 talking about on April 12th and 13th. Because it
25 seems to me if the primary reviewer is confused or
.00228
1 indicates the discussion was altered, that perhaps
2 there could be, there could be a substantial
3 chance, maybe even a likelihood upon remand, that
4 further discussion and different kind of
5 discussion, and the panelists would utilize the
6 clinical experience, the expertise, and the opinion
7 of clinical experts that they were not and did not
8 feel allowed to use during that proceeding on April
9 12th and 13th.
10 DR. SOX: Thank you. If there is no
11 more comment from the floor, we are now discussing
12 a motion. Are there any comments before we go to a
13 vote? Alan?
14 DR. GARBER: Well, I think that if there
15 had been a major substantive change in the question
16 at the last minute, that would indeed call into
17 question the validity of the process. And I
18 believe that that quote from Dr. Landy is correct,
19 but I have to point out that the changes in wording
20 were as far as I could tell solely of a clarifying
21 nature. The evidence report, which Dr. Landy had
22 well before the meeting, and which she was charged
23 with reviewing, was very clearly structured toward
24 the question of the additional effect of
25 biofeedback to pelvic muscle exercise. I found her
.00229
1 comment very thoughtful and helpful, but it was
2 difficult to believe that one could have thought
3 the comparison was primarily against no treatment
4 at all, that it had ever been conceived as that,
5 recognizing of course that the language was not
6 perfectly clear at the outset, and there was room
7 for ambiguity.
8 And let me just reiterate, this is why I
9 think that formulating the questions should be done
10 early and with broad consultation with a lot of
11 people. But I don't think that was the reason that
12 she voted one way and the rest of the panel voted
13 another way on the majority of the questions. And
14 by the way, none of what she said applied to the
15 pelvic floor stimulation component of the
16 assessment.
17 DR. SOX: Other comments before we
18 vote? Hearing none, I'll turn to Connie to make
19 sure we do this right.
20 MS. CONRAD: Okay. There is a motion
21 that the Executive Committee ratify the Medical
22 Surgical Procedures Panel recommendation from April
23 12th and 13th. Do we have a vote? In favor?
24 Hands?
25 (Dr. Brook left the meeting before the
.00230
1 vote was taken.)
2 (All remaining panelists voted
3 affirmative, except Dr. Ferguson and Dr. Johnson.)
4 MS. CONRAD: Against?
5 (Dr. Ferguson and Dr. Johnson voted in
6 the negative.)
7 DR. SOX: Let's move on now to --
8 DR. FERGUSON: Can I be sure -- oh,
9 that's right.
10 DR. SOX: If you have an explanation of
11 dissenting votes, it's an opportunity; I don't know
12 if it's a requirement, but it's certainly an
13 opportunity.
14 DR. FERGUSON: I am not noted for my
15 curmudgeonness, but I'll reiterate my main concern,
16 was that the questions were worded in such a way,
17 to some extent based on the way our interim report
18 was, that in it in effect blocked the panels from
19 evaluating evidence and forming their own
20 conclusions, because they were forced to vote yes
21 or no on adequacy.
22 I think in the biofeedback portion, the
23 questions were narrower than they could have been
24 and perhaps should have been, but my understanding
25 from what I have been able to talk with the people,
.00231
1 the word PME alone is kind of an oxymoron in the
2 sense that biofeedback is often used to inform and
3 allow people to use PME.
4 And what applies to both the areas from
5 what I can see, conclusions were drawn on the TEC
6 reports for both of these, stimulation and
7 biofeedback, and how did they do that if the
8 evidence wasn't adequate. And I think that in the
9 biofeedback portion, not apprising the panel of the
10 AHCPR report was a mistake. But basically it was
11 the formulation which did not allow the panel to
12 address what I would consider necessary to debate
13 the evidence.
14 DR. SOX: Thank you. Joe, do you wish
15 the say something about your vote?
16 DR. JOHNSON: Yes. The vote against
17 ratifying, on page 4 of the minutes, it says under
18 panel comments on their votes, and I quote:
19 Panelists expressed views that if the question had
20 been posed to suggest a decision based on the
21 belief from clinical experience rather than
22 scientific evidence, the results of the voting may
23 have been different. Several panelists offered the
24 feeling that if this were a coverage decision,
25 their votes would have been different as well,
.00232
1 because biofeedback does prevent or deliver
2 effective treatment and is efficacious. And I
3 think that vote would be consistent with
4 Dr. Landy's, Dr. Bradley's, as well as the numerous
5 professional organizations and public testimony
6 that brought forth comments.
7 DR. SOX: Thank you. Well, let's move
8 on to the second motion. Ron, perhaps you could
9 restate it just to remind us.
10 DR. DAVIS: I tried to clean it up a
11 little bit, it's still a bit long, but here it is:
12 That the Executive Committee encourage HCFA to open
13 a dialogue with appropriate funding agencies to
14 discuss the need for good research on treatments
15 for incontinence to better inform future decisions
16 by HCFA on Medicare coverage of those treatments.
17 DR. SOX: That motion has a second, so
18 we can discuss it. Alan?
19 DR. GARBER: I just want to suggest a
20 friendly amendment. In the beginning where he said
21 about the research, support for research, can you
22 insert support for?
23 DR. DAVIS: That's fine.
24 DR. SOX: Any other comments?
25 DR. FRANCIS: I want to make a comment
.00233
1 that is in part an explanation of a positive vote
2 on the prior motion, which is that I certainly
3 understood the vote for ratification as being a
4 vote about the panel's judgment about the adequacy
5 of the evidence, and not at all about a coverage
6 recommendation to HCFA. And so it seems to me in
7 that spirit, that the second motion is particularly
8 important.
9 DR. SOX: Thank you. Well, if there are
10 no further comments, why don't you restate the
11 motion as amended?
12 DR. DAVIS: That the Executive Committee
13 encourage HCFA to open a dialogue with appropriate
14 funding agencies to discuss the need for support
15 for good research on treatments for incontinence,
16 to better inform future decisions by HCFA on
17 Medicare coverage of those treatments.
18 DR. SOX: I think we're ready for a
19 vote. Connie, will you do that?
20 MS. CONRAD: Sure. Could I see a show
21 of hands for those for the motion?
22 (All members present voted
23 affirmatively.)
24 MS. CONRAD: Unanimous? Thank you. The
25 motion carries.
.00234
1 DR. SOX: Well, it's then time to move
2 on. The last item on the agenda is HCFA
3 announcements and information.
4 DR. MURRAY: Dr. Sox, I thought that we
5 were going to have an opportunity to make comments
6 on the vote that was made before.
7 DR. SOX: I'm sorry.
8 DR. MURRAY: I will keep this very very
9 brief. I think that as the conversation, as the
10 discussion has gone forward, I see an analogy to
11 the area of practice with which I'm much more
12 familiar, and that's laboratory testing, and we
13 have struggled through negotiated rule making to
14 develop NCDs for many laboratory tests. And
15 basically what we're talking about here is a
16 service that's analogous to a test, or has some
17 parallel. And that is that there are diagnoses
18 which justify, which provide medical justification
19 for the use of that test, and there are diagnoses
20 which do not provide medical justification. And I
21 think all that we said today is that the diagnosis
22 of urinary incontinence is not in and of itself
23 justification for the use of this service; we're
24 not saying that the service does not have a place,
25 we're not saying that the service is of no value;
.00235
1 all we're saying is that urinary incontinence in
2 and of itself is not adequate medical
3 justification.
4 If there is and ICD-9 code or if there
5 is a diagnosis of urinary incontinence following
6 failure of PME, that is a totally different
7 question, and one which would probably elicit a
8 different answer, and I would expect that the
9 proponents of this therapy would bring this to HCFA
10 and say, well, if urinary incontinence in and of
11 itself, you know, initial urinary incontinence is
12 not adequate medical justification, then failure of
13 PME, failure of other therapies, and they would ask
14 for acceptance of that diagnosis as medical
15 justification, and my expectation is that they
16 would get a favorable hearing at HCFA.
17 DR. SOX: Hugh?
18 DR. HILL: Just real briefly. I had
19 planned at this point to give you some of the
20 feedback that Ron had pointed out, that we have
21 been remiss in not giving you along the way, and
22 simply point out that the multiple myeloma decision
23 had been issued based on the TEC assessment that we
24 got rather than sending it back to the panel as you
25 had instructed.
.00236
1 And the other panel finding in December
2 that you asked to go back to panel, refusing to
3 ratify it, was the human tumor assay for cancer
4 chemotherapeutic sensitivity. All of the
5 requestors joined in asking us to please withdraw
6 that request from the decision making process. At
7 least one of the requestors has indicated his
8 intention to resubmit separately a request for a
9 decision on that, so it may come up again.
10 The only panel that we currently have
11 planned subjects for, and the date is not yet firm
12 for and the subjects are not yet firm, is another
13 meeting of the same panel whose report you were
14 reviewing today, on sacral nerve stimulation for
15 urinary incontinence, I know you're looking forward
16 to reviewing that again, and electrical stimulation
17 for wound healing.
18 And since this is my last meeting with
19 you, I want to take advantage of the opportunity to
20 very briefly thank you very much for your service
21 on this. I've enjoyed wrestling with some of you
22 and strolling with others, and I very much
23 appreciate your public service in this regard.
24 Thank you.
25 DR. SOX: Well, Hugh, you have a very
.00237
1 distinguished place in the history of what many
2 people think is a very important effort on the part
3 of HCFA, and as you go on to your other assignments
4 in HCFA, you know, you'll leave back a lot of fond
5 memories about your leadership and understanding.
6 DR. HILL: Thank you.
7 (Applause.)
8 MS. CONRAD: Thank you. Could I have a
9 motion that the meeting be adjourned? No. Linda?
10 DR. BERGTHOLD: I would just like to ask
11 HCFA to consider convening the Executive Committee
12 at some point in the fall to have an open public
13 discussion about many of the issues that we've
14 talked about today, absent a panel decision. I
15 think we all feel that we don't have enough time to
16 substantively talk about issues, about sort of
17 issues about what is clinical evidence, about a lot
18 of this work. And perhaps it will follow on some
19 of this subcommittee work, but you know, we feel a
20 need for more training, and I don't think I need to
21 make a motion about it. I think several of us
22 agreed at lunch that --
23 DR. HILL: Without a motion, we are
24 already working on that, and we can't promise you
25 in the fall, but we're working on it.
.00238
1 DR. BERGTHOLD: For the public's sake, I
2 would just like to say that while this is a messy
3 process, it's an open messy process, and to
4 remember that it used to be a closed messy process.
5 MS. CONRAD: Do I have a motion to
6 adjourn?
7 DR. GARBER: So move.
8 MS. CONRAD: Do I have a second?
9 ALL PANELISTS: Second.
10 DR. SOX: We are adjourned.
11 (The Executive Committee meeting
12 adjourned at 3:08 p.m.)
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