Medicare Coverage Policy ~ MCAC
Executive Committee
Minutes for December 8, 1999 Meeting (For more information,
contact the Executive
Secretary.)
Attendees Harold C. Sox, M.D.
Chairperson
Robert H. Brook, MD Vice-Chairperson
Sharon Lappalainen Executive Secretary
Voting Members Thomas V. Holohan, MA,
MD, FACP Leslie P. Francis, JD, PhD John H. Ferguson, MD
Robert L. Murray, PhD Alan M. Garber, MD, PhD Michael D.
Maves, MD, MBA David M. Eddy, MD, PhD Frank J.
Papatheofanis, MD, PhD Ronald M. Davis, MD Daisy
Alford-Smith, PhD Joe W. Johnson, DC > Consumer
Representative Linda Bergthold, PhD
Industry Representative Randel E.
Richner, MPH
HCFA Representative Hugh
F. Hill III, MD, JD Ron Milhorn John Whyte, MD Harry
Burke, MD, PhD.
Open Public Session
Greg Raab, PhD Bradley Thompson, JD Larry M.
Wiesenthal, MD, PhD Robert Nagourney, MD Frank Kiesner
Elizabeth Panke, MD, PhD
Wednesday, December 8, 1999, 8:00 a.m.
The Executive Committee met and heard reports from recent
meetings of the Medicare Coverage Advisory Committee (MCAC) medical
specialty panels. The committee also considered how to provide
guidance to and substantive coordination among MCAC panels. For
example, the Committee considered the levels of evidence, types of
information needed, and the nature of issues that would be
considered by the medical specialty panels at future meetings. This
was the first meeting of the Executive Committee of the MCAC and it
began with the introduction of the panelists, a reading of the
conflict of interest statement, and the call to order.
Opening Remarks. Dr. Hill made opening remarks and
reviewed the charge of the Executive Committee. He referred to the
new coverage process that outlined when coverage decisions may be
referred to the MCAC. He stated in general, coverage issues might be
referred: 1) if it is the subject of significant scientific or
medical controversy, 2) if there is a major split of opinion among
researchers and clinicians regarding the medical effectiveness of
the service, 3) if there is a question regarding the appropriateness
of staff or setting, 4) if there is the potential to have a major
impact on the Medicare program, and 5) if it is subject to broad
public controversy.
HCFA Presentation. Ron Milhorn, provided a review of the
Coverage Group’s use of medical evidence and the development of an
evidenced-based decision making approach. He stated that Medicare
has increasingly stressed the need for published scientific studies
in order to develop coverage policies. He outlined the "Procedures
for Making National Coverage Decisions" that were published in the
Federal Register. He pointed out that HCFA was currently working on
a proposed regulation that would outline criteria to be used in
making coverage decisions, which would provide a foundation for the
development of sector specific guidance documents. He stated the
importance of items proposed for coverage demonstrates safety,
clinical effectiveness, and comparative benefit. He then outlined
the types of evidence that might be needed, stressing that the
amount and kind of evidence required will vary according to the
nature of the service or item requested for coverage. He concluded
by indicating that assessing medical evidence was very important in
making Medicare coverage decisions.
Dr. Harry Burke, MD, PhD, a consultant for HCFA, told the
committee that much of what is considered for Medicare coverage has
not undergone the FDA’s formal pre-market review process and
stressed the importance of reviewing the adequacy of the medical
evidence. His discussion centered on the demonstration of
comparative benefit and the presentation of evidence. He indicated
that new tests, treatments, or devices could be compared against
existing technologies in terms of safety, efficacy and clinical
benefit. He felt that individual patients should be allowed to
decide if the clinical benefit of the intervention is commensurate
with the risk given the severity of the disease. Dr. Burke then
discussed different types of supportive medical evidence, such as
prospective randomized clinical trials (RTC) and retrospective
studies. He proposed rating the evidence as strong, moderate, or
weak. Large, randomized clinical trials and large retrospective
clinical studies that have been replicated could be rated as strong
evidence. He thought that small RTCs, small retrospective, or
non-replicated retrospective studies could be rated as weak
evidence. For certain items, such as medical equipment, only
functional and pricing equivalence need be demonstrated. Lastly, he
felt that the presentation of evidence to HCFA should be
scientifically improved.
Open Public Comments and Scheduled Commentaries. During
the morning session panelists heard from 3 scheduled speakers.
Greg Raab, PhD and Brad Thompson, JD, from the Health Industry
Manufacturers Association (HIMA) shared their views on the Medicare
coverage criteria. HIMA applauded HCFA for publishing "Procedures
for Making National Coverage Decisions" and for establishing the
MCAC. Dr. Raab stressed the importance of coverage criteria because
of its direct influence on the capacity of many medical device
companies to undertake and develop new products. The HIMA
representative indicated that the proper forum for developing
coverage criteria for the Medicare program is the Federal rulemaking
process and not the deliberation of the MCAC. He reminded the panel
that its charter states that the MCAC was established as a technical
committee, not to recommend policy for the Agency. Dr. Raab then
highlighted key principles that HIMA believes should be included in
the Medicare coverage criteria regulation: 1) the patient should
come first and economic factors should play no role, 2) coverage
decisions should be guided by clinical evidence that is reasonable,
clinically relevant, and collaboratively developed, 3) Medicare
should amend its current policy on investigational devices, 4) HCFA
should not make national non-coverage decisions unless the product
or service is not effective or that it causes patient harm, and
lastly 5) HIMA believes that coverage restrictions, through
appropriateness reviews, should be grounded in clinical evidence and
frequently upgraded.
Larry M. Weisenthal, MD, PhD of Weisenthal Cancer Group gave his
perspective as a laboratory-based private practitioner and as a
medical oncologist. He addressed 4 specific issues as they related
to his experience: 1) comparative evidence, 2) the importance
defining the relevance of evidence to be considered, 3) consider
conflicts of interest of those presenting evidence, and 4) the need
for the proposed service and to consider the risk of not providing
that service. He then illustrated each of his points by providing
examples. He stressed the importance of matching the therapy to the
patient, so that the patient receives the best treatment and
indicated that definitive studies are needed.
Open Committee Discussion – Levels of Evidence. Several
questions and concerns were raised by panelists that were directed
towards the various presenters. Many issues were related to the
inclusion of cost as a factor and to the extent of the MCAC’s
authority to discuss levels of evidence. Dr. Hugh Hill was asked to
clarify the role of the Executive Committee in helping panels to
consider the evidence.
Dr. Alan Garber presented a document that he authored called
"Standards of Clinical Evidence and their Application." He described
what others have done in this area and mentioned options for the
EC’s consideration. He also cited a chapter from an Institute of
Medicine publication "Methods of Technology Assessment." He urged
the adoption of a 2-step process: 1) rating the quality of the
medical evidence, and 2) question improvement/advantages to health
outcomes.
Panelists agreed that the issues presented to them were difficult
and acknowledged the need for a structured background paper for
rating of the evidence and for deciding if the evidence was
adequate. Other panelists expressed similar concerns: how much
evidence was enough, how to identify the right evidence, and how to
rate it? One panelist suggested the formation of a workgroup that
would provide a consistent approach to the medical evidence that is
presented before MCAC panels. Other panelists supported the idea and
felt the approach should be put together in the form of a document
that would be available to the public. One panelist felt that no
medical specialty panel should be conducted until such information
had been put together. In addition, the panelist felt that the
framing and ordering of questions posed to the committee had a
profound effect on the thinking of the panel. He then recommended
the development of a structured sequence to questions. Lastly, it
was proposed to operationalize the process of reviewing and rating
of the medical evidence with the goal of achieving consistency,
uniformity and correctness of MCAC decisions. Many panelists queried
HCFA as to what extent cost might or might not be taken into
consideration. Some felt that cost was very important, others felt
that is was not part of what the MCAC could consider. The panelists
agreed that on the issue of cost, they would need clarification.
HCFA Presentation. John Whyte, MD, spoke about the
evaluation of new technologies. He provided an overview of the
process by which coverage decisions are made and urged the panel to
engage in a dialogue about how HCFA should use medical evidence to
make coverage decisions. He stated that the challenge for the Agency
was to determine when there is sufficient evidence to cover a new
technology. He stated that HCFA wants to be assured that the new
technology is safe, effective, benefits outweigh risks, there is
evidence of improved outcomes, and has added value. Regarding the
issue of costs, he indicated that there are many aspects to costs,
both opportunity and additive costs. He concluded by stating that
Medicare covers those items that are reasonable and necessary for
the improvement of the lives of the beneficiaries and he encouraged
the committee to engage in a dialogue on these issues.
Motions, Discussions and Recommendations –Levels of Evidence.
The committee voted regarding the following motions as they
related to the discussion of levels of medical evidence. A quorum of
12 voting members was present, no member was recused from
participation due to conflicts of interest:
On the first motion, HCFA should develop a tutorial in order to
educate MCAC members on how to evaluate medical evidence, this
motion was tabled by a vote of 9 in favor and 3 opposed. The motion
was tabled so that the second motion may be considered.
On the second motion, the EC should offer advice to HCFA on a
structured approach used to assemble medical evidence on which the
MCAC medical specialty panels would make their deliberations, the
motion carried by a unanimous vote, all were in favor.
On the third motion, the EC should authorize the Chair to appoint
a subcommittee to work on the structured approach stated above, the
motion carried by a unanimous vote, all were in favor.
Each panelist was invited to voice their comments on what they’d
heard. Panelists agreed that they should advise HCFA to develop a
training package for panel members that would cover the principles
of medical evidence so that MCAC members would be prepared to engage
in this process. Panelists also indicated that the result of the
subcommittee’s work should be brought back to the EC for full
discussion and an opportunity for public participation at the next
meeting.
Report of Medical Specialty Panel Recommendations. Drs.
Ferguson and Murray, Chair and Vice-Chair of the Laboratory and
Diagnostic Services Panel reported that panel’s recommendation on
Human Tumor Assay Systems (HTAS). They stated that the panel was
presented with many different tests from several companies involving
many cancers and many drugs. Both felt that because of the variety
of technologies and the number of speakers, it was difficult to have
adequate discussion time devoted to each type. Dr. Ferguson also
indicated that some of the presentations were repetitive and others
were not contributory. Overall, he felt the agenda was too full and
that the questions were difficult to pose in the form of motions for
voting. He was unsure about whether the coverage proponents had
received all of the critique information in advance so that they
might be prepared to respond. He also indicated the paucity of RTCs
for laboratory tests. Both indicated that the medical literature on
the use of HTAS in chronic lymphocytic leukemia was reasonable, but
for other cancer indications, it was suggestive but not as
persuasive. Both had suggestions for better organizing the panel
package and for revising/restating the questions posed.
Open Panel Discussion. Panelists agreed that the amount
of information presented on HTAS issue was daunting. One panelist
indicated that the evaluation of laboratory tests is fundamentally
different and more difficult than the evaluation of treatments.
Other panelists stated that the literature supporting laboratory
tests usually does not provide receiver-operating characteristic
(ROC) curve analysis, which is a measure of the sensitivity and
specificity of the test. Without having this information, it is
difficult to examine the performance of different studies or
different tests. Other panelists had a number of similar comments
regarding how should the literature be evaluated, the availability
of survival data, and the content and format of the panel’s report.
Report of Medical Specialty Panel Recommendation. Drs.
Holohan and Leslie Francis, the Chair and Vice-Chair of the Drugs,
Biologics and Therapeutics (DBT) Panel gave a report of that panel’s
recommendation on the combination of autologous stem cell
transplantation and high dose chemotherapy in the treatment of
multiple myeloma. Dr. Holohan pointed out that he held a dissenting
opinion on the recommendation from that of the DBT panel. He felt
that the panel’s conclusions were ill advised in that they were not
based upon the evidence that was provided. He stated that the
background materials consisted of literature reprints. Dr. Holohan
felt that the questions posed to the panel were vague and the time
allowed for consideration was limited. He agreed with the previous
report in that presentations were not always direct or to the point,
and there were a number of inconsistencies in the information
presented. Overall, he had serious reservations about the final
conclusions of the panel. Dr. Francis concurred.
Open Committee Discussion. One panelist asked if they
were in direct opposition to what their panels had recommended? They
also questioned the role of the Chair as a guide to their panel,
because it appeared to be unclear. Dr. Holohan indicated that he
dissented with his panel’s recommendation. Dr. Francis indicated
that she voted in favor the panel’s recommendations but with a great
deal of reluctance. Other EC members questioned the current practice
of oncology with respect to stem cell transplantation’s use in
multiple myeloma patients receiving high dose chemotherapy. Some
panelists had questions relating to cost and cost analysis. Others
questioned the data analysis and raised issues about process.
Open Public Session. During the afternoon session,
panelists heard from the public, 4 of the 5 scheduled speakers were
present.
Dr. Robert Nagourney, founder of Rational Therapeutics, a
hematologist and oncologist provided his testimony on the subject
HTAS. He indicated that he is an investigator of a HTAS technology
that is based on cell-death events or apoptosis. He asserted that
apoptosis is the proper endpoint of study for HTAS. He believed that
the reason the panel could not come to a conclusion was because too
many technologies, endpoints, measures and results were presented.
He stated only studies using cell death endpoints correlated
strongly with survival outcomes. He strongly felt that the results
of HTAS that used cell proliferation endpoints should not have been
combined with cell-death endpoints. He recommended that the
conclusions of the panel should not be ratified because it would put
an end to investigational work in this area.
Dr. Larry Weisenthal of Weisenthal Cancer Group, recommended that
the EC ratify the panel’s recommendation and read from the
transcript of the November meeting. He cited many sections of the
transcript where panelists had stated that HTAS show promise for
clinical utility. He asserted that HTAS should be covered and that
the data presented was supportive. He urged the EC to consider this
and focus on what was in the transcript of the meeting.
Mr. Frank Kiesner, president of Oncotech Inc., stated two points.
First, he asked that the panel not accept Dr. Nagourney’s position.
Second, he stated that although it was very important to focus on
the process, the judgement of the LDS panel was sound and should be
upheld. He felt that the EC should exercise caution about the
message that would be sent out in the event of non-ratification.
Dr. Elizabeth Panke, Director of Genetica Laboratories related
her experiences with HTAS both professionally as a practicing
pathologist and personally as an ovarian cancer patient. She told of
her diagnosis and of her decision to use HTAS to help in her
treatment decisions. She stated that samples of her malignant cells
were sent to two different laboratories that offered HTAS testing.
Each lab used a different endpoint. When the results came back, one
lab report indicated that her cancer was not resistant to the drugs
tested, the other lab report indicated that her cancer was resistant
to the drugs tested except for the combination of cisplatin and
gemcitabine. After receiving this combination as treatment, she has
improved. She concluded that because of the disparities between lab
reports, more studies are needed before broad-based coverage of HTAS
should be recommended.
Motions, Discussions and Recommendations – Reports of Medical
Specialty Panel Recommendation. The committee voted
regarding the following motions as they related to the reports of
the medical specialty panel recommendations. A quorum of 12 voting
members was present no member was recused from participation due to
conflicts of interest:
For the report of the Laboratory and Diagnostic Services Panel,
the motion: 1) the EC should take no action, 2) thank the panel for
its conscientious work, 3) ask the panel to reconsider these matters
after the EC and HCFA have established a consistent process for
panel review and assessment of the evidence, and 4) each of the
various HTAS should be reviewed on an item-by-item basis. The motion
carried with 8 in favor and 4 opposed. Those opposed did not agree
that the panel should revisit the issue again and preferred to see
ratification.
For the report of the Drugs, Biologics and Therapeutics Panel,
the motion: 1) the EC take no action, 2) thank the panel for its
conscientious work, and 3) ask the panels to reconsider these
matters after the EC and HCFA have established a consistent process
for panel review and assessment of the evidence. The motion carried
with 9 in favor and 3 opposed. Those opposed did not agree that the
panel should not revisit the issue again and preferred to see
ratification.
Adjournment, Wednesday, December 8, 1999, 4:07 p.m. The
meeting adjourned at 4:07 p.m.
|
I certify that I attended the meeting of the Laboratory
and Diagnostic Services Panel on November 15 and 16, 1999,
and that these minutes accurately reflect what
transpired.
_________________________________ Sharon K.
Lappalainen, Executive Secretary, HCFA
|
|
I approve the minutes of this meeting as recorded in
this summary.
______________________________ Harold C. Sox,
M.D. Chairperson |
|