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Document 1 of 41.

September 13, 2000, Wednesday

SECTION: CAPITOL HILL HEARING

LENGTH: 23028 words

HEADLINE: HEARING OF THE HEALTH AND ENVIRONMENT SUBCOMMITTEE OF THE HOUSE COMMERCE COMMITTEE

SUBJECT: HEALTHCARE LEGISLATION

CHAIRED BY: REPRESENTATIVE MICHAEL BILIRAKIS (R-FL)

WITNESSES:

DOCTOR R. NICK BRYAN, HOSPITAL OF UNIVERSITY OF PENNSYLVANIA;

DOCTOR N. REED DUNNICK, UNIVERSITY OF MICHIGAN HEALTH SYSTEM;

DOCTOR BRUCE J. HILLMAN, UNIVERSITY OF VIRGINIA;

TOMIKO FRASER, LUPUS FOUNDATION OF AMERICA, INC.;

DOCTOR DYANN WIRTH, HARVARD SCHOOL OF PUBLIC HEALTH;

JIM NAVARRO

ROBERT BRADY, HOGAN AND HARTSON;

ABBEY MEYERS, NATIONAL ORGANIZATION FOR RARE DISORDERS;

THOMAS A. LANG, SERONO LABORATORIES, INC.;

CATHERINE BENNETT, CANCER RESEARCH FOUNDATION;

LOCATION: 2123 RAYBURN HOUSE OFFICE BUILDING, WASHINGTON, D.C.

BODY:
REP. MICHAEL BILIRAKIS (R-FL): The hearing will come to order. My thanks -- is this working? I didn't think so. Is someone checking into this? Testing. Now it's on. My thanks to all of the witnesses who have taken the time to testify before the subcommittee. This hearing will address, as you know, several pieces of legislation designed to improve the quality of healthcare. Today, we will hear about H.R. 2399, the National Commission for the new national goal -- the Advancement of Global Health Act.

This legislation, introduced by my friend -- all of our friend -- Representative George Gekas of Pennsylvania, would establish a commission to recommend a national strategy to coordinate public and private sector efforts toward the global eradication of disease. The commission would specifically address how the United States may assist in the global control of infectious diseases through the development of vaccines and the sharing of health research information on the Internet.

Also on the first panel, we will hear testimony on H.R. 1795, the National Institute of Biomedical Imaging and Engineering Establishment Act. This legislation, introduced by Representatives Richard Burr and Anna Eshoo, members of this panel, would establish a National Institute of Biomedical Imaging and Engineering at the National Institutes of Health. Finally, our first panel will address legislation introduced by my Florida colleague, Representative Carrie Meek. H.R. 762, the Lupus Research and Care Amendments of 1999, expands federal lupus research activities and authorizes the Secretary of Health and Human Services to make grants for the delivery of essential services to individuals with lupus in their family.

Congresswoman Meek has been a tireless proponent of this legislation, and I would also be remiss if I failed to mention the advocacy efforts of Sandy Freer (ph), from my area of Florida. I discussed this legislation at the full committee markup of the Minority Health Disparities bill. I look forward not only to the testimony today, but to advancing this very important legislation. Our second panel will include testimony on H.R. 4242, the Orphan Drug Innovation Act. This bill amends the Federal Food, Drug, and Cosmetic Act to allow sponsors of a drug for a rare disease or condition, so- called "orphan" drugs, to ask the Secretary of Health and Human Services to provide written recommendations for the non-clinical and clinical investigations, which must be conducted with a drug before it may be approved as a new drug or licensed as a biological product.

It also authorizes the Secretary to provide recommendations on whether such a drug is for a disease or condition which is rare in the United States. I'd also like to welcome Mr. Jim Navarro, a concerned parent, to our second panel. He will be discussing H.R. 3677, the Thomas Navarro FDA Patient's Rights Act. This bill is named after his son, who was four years old when he was diagnosed with a form of cancer known as medulloblastoma. After researching their options, the family decided the best course of action was through a non-toxic, FDA- approved clinical trial. The FDA denied Thomas access to this clinical trial because he had not first undergone and failed treatment by chemotherapy and radiation, which can have, as we all realize I think, serious side effects for children of that age.

H.R. 3677 precludes the FDA from establishing a clinical hold on the basis that there is a comparable or satisfactory alternative therapy available if a patient is aware of the other therapy and aware of the risk associated with an investigational drug, yet still chooses to receive the treatment. Finally, in honor of Childhood Cancer Month, we will hear testimony in support of a resolution sponsored by Representative Deborah Price on the importance of researching childhood cancer. I think all of us remember that Representative Price lost her little daughter a few months ago.

The testimony and the resolution focus on the importance of promoting awareness of and expanding research on childhood cancers. The resolution would encourage medical trainees to enter the field of pediatric oncology, encourage the development of drugs and biologics to treat pediatric cancers, and promote medical curricula to improve pain management. The resolution would also support policies that reduce barriers to participation in clinical trials. I welcome all of our witnesses, including our colleagues, to this hearing. And to cover as much ground as possible, I would ask members to limit their opening statements.

Under the rules, I can limit opening statements, other than chairman or ranking member, to three minutes, and I would appreciate it if you would hold them to within that period of time. And I would also note that some members of Congress, who are not members of this subcommittee, will also give brief introductory remarks regarding their legislation and introduce their witnesses. I just hope that this hearing will shed light on a number of important public health issues, and that we can devote most of the time to our witnesses.

With that, I now yield to the ranking member, Mr. Brown.

REP. SHERROD BROWN (D-OH): I thank the chairman. I'd like to welcome our witnesses also. Thank you for joining us. We have an ambitious agenda this morning. Among the six bills, we will consider two that would affect access to medications, H.R. 4242 and H.R. 3677. In the case of both bills, it's likely that today's hearing will not produce definitive answers. The issues involved are simply too complex, and the implications of any action we take too significant. However, the questions these bills seeks to answer -- the concerns they seeks to address -- are important and it's valuable for the subcommittee to learn about them.

I'm also glad we'll have an opportunity to review legislation focusing on lupus and childhood cancers.

Both types of illnesses devastate and too often take young lives, and neither has received the attention that they both deserve. But in the interest of time, I want to focus my comments on two of the other bills we will consider this morning, H.R. 2399 and H.R. 1795. I fully support the efforts of my colleague, Mr. Gekas, to establish the improvement of global health as a national priority, because global health should be a national priority for several reasons. Global health and the health of Americans are linked. Americans travel abroad. The world travels here. Lethal infectious diseases cross borders.

The reemergence of, for instance, tuberculosis in the United States, now in drug resistant strains that are difficult and expensive to treat, is a grim reminder that when a disease affects other nations, it's bound to affect us. Tuberculosis last year killed more people than any year in history -- 1,100 Indians die every day from tuberculosis. A second reason that global health should be a national priority is because the United States is a world leader. We're the wealthiest nation in the world. We're the most influential force in the world. Our actions set the precedent. Our inaction sets a precedent.

The United States is in a unique position to save lives, to save families, to save children all over the world. An investment that is modest by US standards literally can save millions of lives, prevent millions of children from being orphaned, prevent the social, economic, and political turmoil that killer diseases too often engender. It's an opportunity and it's a privilege that our nation should embrace. The other bill I want to mention briefly is H.R. 1795, which would establish an Institute for Biomedical Imaging and Engineering within the National Institutes of Health. Unfortunately, my colleague Mrs. Eshoo couldn't be here this morning, but I wanted to acknowledge her outstanding leadership and the leadership of Mr. Burr on this measure.

I want to extend a special welcome to Dr. Dunnick from the University of Michigan, who's joining us to discuss 1795 at Mrs. Eshoo's request. Adding an institute to NIH is a major step, but Ms. Eshoo and Mr. Burr make a compelling case for it. Advancements in medical imaging technology have led to stunning breakthroughs in the early detection and the treatment of many diseases. By identifying these diseases early and without invasive procedures, patients are often able to receive less painful and more therapeutic treatments that greatly improve the likelihood that they'll live longer and healthier lives.

Additionally, when treatment is initiated at the early stage of a disease, doctors are usually able to rely on less expensive treatment options that reduce overall healthcare costs. I'm glad, Mr. Chairman, we're taking the time to assess the benefit of establishing an institute dedicated to equipment and techniques that are indispensable to modern healthcare. I thank the chairman.

REP. BILIRAKIS: I thank the gentleman.

Mr. Upton, for an opening statement.

REP. FRED UPTON (R-MI): I would ask unanimous consent to put my full statement in the record and just --

REP. BILIRAKIS: Without objection, the opening statement of all members of the subcommittee will be made a part of the record.

REP. UPTON: I would just like to say one thing verbally here. I'm very glad that we're having these hearings, and I'm particularly happy that we are focusing on H.R. 672, the Lupus Research and Care Amendments Act. Sadly, my sister-in-law suffered from this disease and died last year from this illness, so I know how important it is to commit ourselves to finding a cure for this devastating disease. I commend my colleague from Florida for offering her bill and I'm delighted to be a co-sponsor, and I yield back.

REP. BILIRAKIS: Mr. Waxman, for an opening statement.

REP. HENRY WAXMAN (D-CA): Thank you, Mr. Chairman. Let me begin by saying how pleased I am that H.R. 762, the Lupus Research and Care Act, is receiving our attention. It's an excellent bill. It deserves our support. Lupus is also one of the dozens of autoimmune diseases, which are the subject of my own bill to establish an office of autoimmune diseases at NIH, which has already passed this committee and the House. But today, I'm principally concerned about H.R. 4242, the Orphan Drug Innovation Act.

As the author, with Senator Metzenbaum of the Orphan Drug Act, I care deeply about the issues raised by this legislation. For many years, I've been very gratified by the success of the Orphan Drug Act in stimulating the development of new treatments for rare diseases, and I'm pleased that you're going to have Abbey Meyers testify again before our subcommittee and that she's willing to come. Market exclusivity is the foundation of the Orphan Drug Act, but we created exceptions to that exclusivity in the law. The bill before us would limit the scope of exclusivity granted to drugs proven, quote, "clinically superior," end quote, to an existing orphan drug. That is, drugs which are safer, more effective, or provide a major contribution to patient care.

It's been alleged that the bill is intended to anoint a winner in a commercial dispute, but the bill raises an important and legitimate question. What is the right balance between preserving exclusivity, encouraging competition, and encouraging affordable access to these lifesaving drugs? As a first step to the best answer, I was looking forward to with great interest to the FDA's public clarification of its policy towards clinical superiority. There are questions about its consistency and its relationship to a generic approval process for biotech drugs. This subcommittee requires some clear answers.

They have significant implications for patient health and for access to reasonably priced breakthrough drugs. But this morning I learned that the FDA has withdrawn its witness and testimony. While this may be the result of late notice to the administration, it nevertheless ensures today's hearing will be of less assistance in guiding our deliberations. I would add that orphan drug policy deserves a hearing on its own. There are 25 million Americans suffering from over 6,000 rare diseases. There is a great deal of unfinished business for Congress. There is a question of how high a bar clinical superiority should be.

There are some multimillion dollar orphan drugs -- drugs for which seven years of exclusivity is unjustified and serves only to boost prices and profits, putting those lifesaving therapies out of the reach of many patients. And just as important, there is the urgent need for more orphan disease research at NIH and FDA. I sincerely hope that we'll have an opportunity early next year to examine these issues in greater detail than will be possible today. Finally, Mr. Chairman, I'd like to submit for the record a series of articles and scientific reviews relating to the alternative cancer treatment offered by Dr. Stanislav Berzinski (ph), the intended beneficiary of H.R. 3677, the Thomas Navarro Patient's Rights Act.

REP. BILIRAKIS: Without objection, that will be the case.

REP. WAXMAN: As ranking member of the Government Reform Committee, I've attended that committee's many hearings called to defend and endorse alternative medicine and dietary supplements. But I'm pleased this subcommittee, which has jurisdiction over these issues, is finally turning its attention to them. Developing new forms of cancer prevention, detection, and treatment never ends. Ensuring patients have access and accurate information about their treatments is also vital. We must keep an open mind about innovative or unconventional approaches to cancer treatment and prevention, but our first priority must be ensuring access to treatments which are proven to be the best chances of curing patients.

And, second, our priority must be rigorous testing of new therapies, including complimentary and alternative therapies, to determine their safety and efficacy. The problem with H.R. 3677 is that it undercuts these goals. If research is under a clinical hold, you can be sure that there are unresolved questions about the conduct of that research. But this bill would shield such research from scrutiny, discourage practitioners from cooperating in rigorous research, and lessen our chances of ever knowing for sure whether an alternative treatment actually works or not.

And at a time when research and patients alike complain that IRBs (ph) are overburdened and informed consent is not always truly informed, this bill would increase the chances that patients are put at inappropriate risk, not lessen them. I join my colleagues in welcoming our witnesses, and I look forward to their testimony.

REP. BILIRAKIS: I thank the gentleman.

Mr. Burr, to give us -- share his opening statement with us.

REP. RICHARD BURR (R-NC): I thank the chairman, and I thank the chairman for this hearing. Mr. Chairman, we have a lot on our plate this morning and it's all extremely important. I will focus just briefly on the NIBIEE bill, which Ms. Eshoo and I have introduced, which currently has 169 cosponsors. It's unfortunate today, as we have this hearing, that Ms. Eshoo is in California under the weather, but I'm sure if she were here, she would speak out very loudly in support of this legislation that she and I and others on the Hill and throughout the country have worked on.

I don't think I can sum it up any better than the committee brief for this hearing. In their description of the NIBIEE bill, it said breakthroughs in imaging, such as magnetic resonance imaging and computed tomography, have revolutionized the practice of medicine in the past quarter century. But those technologies are inadequate in diagnosing some diseases. What that statement says is that we've made tremendous progress despite a lack of a focused effort on our ability to detect at the earliest possible point. What we've heard, Mr. Chairman, from people around the country is we can do better. If you give us the type of focus that it takes, and resources, we can come through with an earlier detection of disease, and we can give physicians who are treating disease many more options because of that early detection.

What NIBIEE does is create an institute of health for biomedical imaging at the NIH -- the same NIH that every member of this panel and most members of this Congress are committed to put new resources in. What we want to make sure when we make that commitment to the American people for additional resources to chase the disease that affects every family in this country, that we make sure that biomedical imaging is one of the concentrated focuses of the NIH because we know that early detection will give us more options and will give patients more options. Mr. Chairman, I would urge my colleagues today to ask as many questions of the witnesses that are here to testify on this bill, but in the end to also be supportive of this legislation.

This is extremely important that we get it done, and we get it done now. We spend a lot of time talking about healthcare policy. This is a place where we can in fact make sure that our options are greater down the road, and I applaud the chairman, and I yield back.

REP. BILIRAKIS: I thank the gentleman.

Mr. Pallone, for an opening statement -- Well, I'm going on the basis of seniority.

REP. FRANK PALLONE (D-NJ): Thank you, Mr. Chairman. I'll limit my comments to the two bills which I have cosponsored, H.R. 3677, the Thomas Navarro FDA Patient's Rights Act, and H.R. 1795, the National Institute of Biomedical Imaging and Engineering Establishment Act. The first of these, the Thomas Navarro FDA Patient's Rights Act, deals with the rights of patients and parents to make informed choices about medical treatment. The bill's namesake, young Thomas Navarro, has unfortunately been suffering from the affects of a brain tumor. As any parent with a child in Thomas' situation would, the Navarro's researched the treatment options available, and found that treatment of radiation and chemotherapy would have extremely debilitating side effects, which they did not want to risk.

Rather, the Navarro's preferred to have Thomas treated with antineoplastine therapy, a therapy surrounded by some controversy, that is under clinical trial. The Food and Drug Administration has, however, refused to allow this to happen on the basis that his parents have not yet tried the radiation and chemotherapy path. I cosponsored the bill because Thomas Navarro's parents should be allowed to decide for themselves whether or not the antineoplastine treatment for Thomas in the setting of a clinical trial is an appropriate path to follow. They researched the issue. They understand the issue. And I do not believe, in light of the circumstances surrounding the case, that the Navarro's should be denied their right to choose.

The issue is important not just for Thomas, but for other patients in similar circumstances. We should not be restricting the rational choices and measured choices of individuals who choose to pursue alternative medical treatment, whose possible outcomes they fully comprehend. The second bill I want to mention, the National Institute of Biomedical Imaging and Engineering Establishment Act, is an excellent piece of legislation that I hope all of my colleagues on this subcommittee will support. I've discussed the importance of this legislation on a number of occasions over the last two years with medical professionals from the radiology department of the University of Medicine and Dentistry of New Jersey, and other medical professionals from my home state.

All of them have stressed the importance an institute for imaging research within NIH can play in promoting further breakthroughs in a field that has already vastly changed the practice of medicine for the better. I want to thank the chairman for having a hearing on these two bills and the others that we have today. I yield back.

REP. BILIRAKIS: I thank the gentleman.

Mr. Stupak, for an opening statement. I'm going to try to continue on here, rather than break, so long as someone gets back in time to spell me.

REP. BART STUPAK (D-MI): Mr. Chairman, I thank you for holding this hearing. I thank the witnesses for being here. I look forward to their testimony. I'm a cosponsor of H.R. 762 and H.R. 1795, and for the sake of time, and I want to hear the witnesses, I yield back the balance of my time.

REP. BILIRAKIS: I thank the gentleman so very much.

Ms. Capps, for an opening statement.

REP. LOIS CAPPS (D-CA): Mr. Chairman, I thank you for holding this hearing, and want to extend a welcome to our witnesses. Today, we're going to be discussing several worthy pieces of legislation, all focused on securing the health of the American people. Because of the nature of our hearing today, I want to remind you and other members of the bill, H.R. 353, the ALS Treatment and Assistance Act. This is a bill that I'm offering, enjoying bipartisan support -- currently has 280 cosponsors, many of whom serve on this committee. I'm so glad we're having a hearing today on so many important pieces of legislation. I commend you for making that happen.

And my hope is that this committee can address H.R. 353 before the 106th Congress is over. I want to state my strong support in this setting for H.R. 762, the Lupus Research and Care Amendments of 1999. I'm so pleased that our colleague, Carrie Meek, is here -- the author of this bill, which would authorize the Secretary of Health and Human Services to make grounds for the delivery of essential services to individuals with lupus and their families. As a nurse, I know what an insidious disease this is. And for many people, lupus is a mild disease, affecting only a few organs. For others, it can cause serious, even life threatening, problems.

My district is home to the Sclera Derma Research Foundation. Sclera Derma is a condition that is closely linked to lupus, and I've seen the courageous work of women like Sharon Monski (ph) in Santa Barbara -- my district -- who has fought so long to raise awareness of sclera derma and lupus diseases, which disproportionately affect women and can have life and death consequences. So, I applaud the subcommittee for recognizing this legislation. I also want to acknowledge the legislation sponsored by my colleague, Anna Eshoo, which was described by our colleague, Richard Burr, H.R. 1795.

This legislation will fill a critical void at the NIH by creating an independent institute on the topic of bioengineering. These disciplines have made such contributions to the improvements, as our colleague has said, and have no research home in the current structure of NIH. I support this legislation, again, commending our colleagues for their leadership in this area. Finally, I know -- my record submitted will be longer than this -- but I want to say one word about my good friend and colleague, Deborah Price, and offer my strongest words of support for her resolution. Her resolution focuses on the importance of promoting awareness of, and expanding research on, childhood cancers.

This is Cancer Childhood Month -- September. It's the second leading cause of death in children past infancy. Many childhood cancers can now be cured, but sadly dozens still can't.

And I applaud Congresswoman Price's effort in this difficult area and pledge to offer support -- any that I can give to help get this legislation passed into law. So, Mr. Chairman, this is an important topic. Thank you for holding this hearing, and I yield back the balance of my time.

REP. BILIRAKIS: I thank the gentlelady.

Mr. Gekas and Mrs. Meek are here to testify on behalf of their legislation briefly, and also to introduce the witnesses. What is your pleasure? Should we go to you now, or would you like to return? I don't want to really rush you. We do want to make this vote. Would you like to return? I'll be here. Pardon?

REP. CARRIE MEEK (D-FL): I would rather go forward, if I may.

REP. BILIRAKIS: Go forward now. What would you prefer to do? Well, then, proceed. The only thing is I'm liable to cut you off. I don't want to miss this vote if no one is here to spell me.

You're recognized, the gentleman from Pennsylvania.

REP. GEORGE GEKAS (R-PA): Thank you. The opening remarks made by the chairman and the ranking member, in describing the bill which I have placed before you, were more than adequate in describing the purport of the legislation. I simply want to add to that the fact that the national goal for the 20th century is well known to everybody -- the one that we just completed -- that goal was thrust upon us. It was the repulsion of totalitarianism and the reestablishment and preservation of democracy across the globe. That was the national goal thrust upon us.

Now we have an opportunity in the next century to assume leadership in, what I envision to be and others do, the national goal for the 21st century -- namely, the eradication of disease worldwide. Why is that important? Not only for the humanitarian and altruistic rationale that are the foundation for such a project, but also in the enlightened self-interest of our country, which is the leader in all of these disciplines that are so vital to the health of the world. In that enlightened self-interest, we not only protect our people in the future from these diseases and other catastrophes that might occur, but at the same time, we create jobs, we create interest, we develop new technologies, new pharmaceuticals, and all the other necessaries to further envision a world without disease with our country in the forefront.

That's what the purpose of it is. That's why our witness is so important. She has testified before our biomedical research caucus, as one of the leading lecturers in her field, and you will see from her testimony that she will be a vital force if we implement the legislation which I have offered. She is Professor, Dr. Dyann Wirth, w - i - r - t - h, of the Department of Immunology and Infectious Diseases, Harvard School of Public Health, and has a slue of publications of which she is the author. She is renowned in her field. She impressed the members of the Hill, the Capitol, who engaged in the Biomedical Research Caucus series. I'm sure she will impress you.

Unfortunately, she has impressed me so much, that I'm leaving right now to go vote. But I know what you will testify, and commend her to you.

REP. UPTON: Thank the gentleman from Pennsylvania.

Now I recognize Mrs. Meek, and I don't know that you were here when I gave my opening statement --

REP. MEEK: Yes, I was.

REP. UPTON: I commended you for your good bill, and I'm delighted to be a cosponsor, and would be delighted to recognize you now.

REP. MEEK: Thank you, Mr. Chairman. I'm pleased to be before the committee again, and I want to thank you for bringing this hearing up again. 762 is an extremely important piece of legislation. It's time that it be passed, Mr. Chairman. And, of course, I'm hoping that this committee will see fit to pass it out and send it to the floor as quickly as possible so that no one else will have to wait for the kind of assistance that this Lupus Research and Care Act will bring. The Lupus Foundation has really talked with the Congress through this, Mr. Chairman. They have assiduously watched this bill for many, many years, and I do hope we can get it passed. What it will do will add additional services for lupus victims.

But I'm here this morning because I'm pleased, and privileged, and blessed to have a young lady who's sitting at the table -- the testimony table -- to testify for lupus -- a very beautiful young lady, Tomiko Fraser. She is the spokesperson for the Lupus Foundation. And I don't have to take your time to tell you all of the demerits, I would say -- or all of the real terrible affects of lupus. I've lost so many friends to lupus. It's a young woman's disease. And as a result of that, I think of this committee, you have 243 cosponsors behind this bill. And I want to have my statement placed in the record, Mr. Chairman, with your permission.

REP. UPTON: Without objection, your entire statement will be on the record. And, again, we thank you for your leadership on this very important issue that touches so many American families in every state.

REP. MEEK: Thank you, Mr. Chairman. And I'd love to hear Tomiko, but I must go and vote.

REP. UPTON: Well, you can come back. At this point, no members coming back from the vote that is currently ongoing. I'd like to invite the first panel to come to the table. They include Dr. Nick Bryan, Professor and Chairman of Radiology at the Hospital of University of Pennsylvania, Dr. Reed Dunnick, Professor and Chair, Department of Radiology at University of Michigan -- go Blue, beat UCLA this weekend -- Dr. Bruce Hillman, Professor and Chair, Department of Radiology, University of Virginia, Ms. Tomiko Fraser, who of course is at the table already and obviously the national spokesperson for the National Lupus Association, and Dr. Dyann Wirth, Professor, the Department of Immunology and Infectious Diseases at Harvard.

I just want to say before we start that there are a number of things ongoing this morning -- a number of committees and subcommittees that are meeting. We have a very important issue on the House floor, that being the marriage penalty tax as well, where I'm going to have to return to engage myself in that debate a little bit later this morning. Your statements are made part of the record in their entirety. We'd like to keep this to five minutes each.

And, Dr. Wirth, we will start with you. Thank you for being here with us this morning.

DR. DYANN WIRTH: Mr. Chairman and members of the subcommittee, thank you for the opportunity to testify.

REP. UPTON: You might just pull the mike just a little closer for people in the back. That'd be great. Thank you.

DR. WIRTH: My name, as you already know, is Dyann Wirth. I'm a professor at the Harvard University School of Public Health in the Department of Immunology and Infectious Diseases, and I'm here today on behalf of the Joint Steering Committee for Public Policy, which has worked closely with Representative Gekas in his outstanding efforts in support of biomedical research. The Joint Steering Committee for Public Policy is a coalition of four life-science societies, representing more than 25,000 researchers.

I am here today to support -- to express the support of the Joint Steering Committee for Congressman Gekas' bill, which we've just heard about, on the advancement of global health, H.R. 2399, which if put into law, would create a presidential congressional commission to investigate how we as a nation can most effectively seize the scientific opportunities presented by modern advances in research to eradicate many of the diseases that are plaguing millions of the world's people. We support this bill because we believe that in this next millennium, it is within the grasp of human capacity to accelerate the role of basic biomedical research and the translation of that research to the benefit of the world's least fortunate people.

Now is the time. This is an attempt to focus all the tremendous scientific energy in the United States on fighting diseases throughout the world. This is a noble endeavor for the United States. We have the means to do this, and I believe we should make it a priority. My particular experience is in malaria.

But as devastating as malaria is, it is just one of the several infectious diseases that are not only killing millions, but costing billions. According to the World Health Organization, infectious diseases account for more than 13 million deaths a year. That's 35 percent of the deaths in the world today.

That means during the duration of this hearing, 1,500 people will die from infectious diseases. Malaria alone kills 2.7 million people each year. Tragically, every 30 seconds, a child somewhere in the world -- probably in Africa -- dies of malaria. The enormous volume of travel and trade have made infectious diseases blind to national borders, and this has been recognized. A January 2000 unclassified report from the CIA's National Intelligence Council entitled, "The Global Infectious Disease Threats and its Implications for the United States," suggests that in modern warfare, infectious diseases are likely to account for more military hospital admissions than battlefield injuries.

The report claims, and I concur, new and reemerging infectious diseases will pose a rising global health threat, and will complicate US and global security over the next two decades. These diseases will endanger US citizens at home and abroad, threaten US armed forces deployed overseas, and exacerbate social and political instability in key countries and regions where the United States has an interest. Research into prevention, treatment, and control of tropical and infectious diseases are now more important than ever. I will talk just briefly about malaria, because that's where my interest and passion is. Among adults living in high transmission areas, malaria is best thought of as a chronic disease. A single bout can incapacitate someone for weeks.

And despite massive efforts to eradicate this disease in the 1950s, there is more malaria in the world today than there ever has been in history. One fourth of the world's population is at risk of the infection. Clearly, we need better implementation of the tools that we have in the short term, but our tools are not adequate. New research interventions are desperately needed. Cutting edge technology has led to the development of new paradigms. The genome of the most important parasite is being sequenced. We have DNA vaccines, new technologies, and it's important to seize the opportunities of these scientific advances to accelerate and defeat malaria worldwide.

But equally important as progress in research is public support and awareness of these major health threats. In order to conquer malaria, AIDS, other infectious diseases, we need a global strategy that includes American leadership and resources to invest in continued research into prevention and treatment. As we begin the 21st century, we are blessed with unimaginable opportunities to build on breakthrough research to control and prevent global infectious diseases. This is not just altruism to reduce suffering of the world's most needy. This is also a question of national security and health for the United States and its citizens.

Renewed investment in the treatment and prevention of global infectious diseases is a win-win for the country. By helping others across the world, we are launching the best defense to protect the health of our nation's people. We hope that you will seriously consider passage of H.R. 2399, and thank you very much for the opportunity to present.

REP. UPTON: Thank you, Dr. Wirth.

Dr. Dunnick, welcome to Washington's version of the big house.

DR. N. REED DUNNICK: Thank you. Good morning. Thank you for this opportunity to share my support for H.R. 1795 with you. I also appreciate the leadership shown by Mr. Burr and Ms. Eshoo in support of this legislation. I am Reed Dunnick, and I currently serve as the Chair of the Department of Radiology at the University of Michigan. Prior to coming to Michigan in 1992, I served on the faculties at Stanford and at Duke. In addition, I spent four years as a staff radiologist in diagnostic radiology at the National Institutes of Health.

Congress has recognized the structural impediments to imaging research that exist at the NIH in the conference report on H.R. 3194, the Consolidated Appropriations Act for Fiscal Year 2000. The language in that conference report is a good summary of the current situation at the NIH. Continued advances in biomedical imaging and engineering, including the development of new techniques and technologies for both clinical applications and medical research, and the transfer of new technologies from research projects to the public health sector, are important. The disciplines of biomedical imaging and engineering have broad application to a range of disease processes and organ systems, and research in these fields does not fit into the current disease and organ system organizational structure of the NIH.

The present organization of the NIH does not accommodate basic scientific research in these fields, and encourages unproductive diffusion of imaging and engineering research. Several efforts have been made in the past to fit imaging into the NIH structure, but these have proved to be inadequate. This congressional report is correct. The current structure of the NIH does not promote basic research in medical imaging and bioengineering -- two disciplines that have become critical to improving healthcare. The next logical step suggested by this congressional finding is the creation of a national institute of biomedical imaging and bioengineering, as proposed in H.R. 1795.

The imaging science community has worked with the NIH leadership for three decades to fit imaging into the existing NIH organizational structure of individual institutes dedicated to specific disease processes and organ systems. However, nothing short of an institute will be effective in stimulating and coordinating biomedical research to the extent that is needed. The imaging community has not proposed the establishment of a new institute lightly. We recognize and agree that the bar to structural change should be set high. For that reason, we looked to the National Academy of Sciences' Institute of Medicine in their 1984 report entitled, "Responding to Healthcare Needs and Scientific Opportunity: The Organizational Structure of the National Institutes of Health."

They recommend a new institute when each of the following five criteria are met. One, the activity is compatible with the mission of the NIH. Two, the research in question is not receiving adequate attention. Three, there are reasonable prospects for scientific growth. Four, there are reasonable prospects of sufficient funding. And, five, the proposed structural change will improve communication, management, priority setting, and accountability. The proposed National Institute of Biomedical Imaging and Bioengineering is consistent with these criteria. In identifying imaging as one of its top research priorities, the NCI has stated that this field is compatible with the mission of the NIH.

The NCI has indeed increased its level of support for biomedical imaging in recent years. However, even with this increase in resources, the amounts of monies carried out by radiology departments throughout the country account for less than one percent of the NIH budget. Finally, the proposed institute, which would include a division to coordinate imaging research throughout the federal government, would certainly improve communication and management in a field in which these qualities are sorely lacking.

Mr. Chairman, breakthroughs in medical imaging have revolutionized the way in which physicians detect, diagnose, and treat disease. Imaging holds the promise of further advances that will move us into an era of noninvasive medicine. To reach that goal, however, we need to create a climate that promotes discovery and innovation in imaging, just as the NIH provides such a climate for other fields. For that reason, I urge the committee to support the establishment of the National Institute of Biomedical Imaging and Engineering. Thank you.

REP. UPTON: Thank you very much.

Dr. Hillman.

DR. BRUCE HILLMAN: Good morning. I am Dr. Bruce Hillman, Chair of the Department of Radiology at the University of Virginia, and I'm also a Chancellor at the American College of Radiology.

REP. UPTON: You might want to just bring that mike just a little bit closer, again for the people in the back.

DR. HILLMAN: That'll work better that way. I am grateful to the committee, especially to the bill's sponsors, Mr. Burr and Ms. Eshoo, and my own Congressman, Chairman Bliley, for the invitation to testify in support of H.R. 1795, the National Institute of Biomedical Imaging and Engineering Establishment Act. Medicine now stands at the threshold of a new and exciting revolution in how we think about disease. It is the molecular revolution, wherein we will develop the tools needed to diagnose and treat disease at its earliest stages, when the chances of success are lightly to be much greater than currently.

Medical imaging must be a critical element in this new paradigm. Medical imaging is the noninvasive biopsy that will detect alterations in the genetic or molecular makeup of cells that have the potential to progress the disease. Imaging technologies will precisely determine what fraction of cells are affected. Finally, medical imaging technologies will be integrated with new therapeutic methods to either guide or monitor treatment, so that only diseased cells are treated, while preserving normal tissue. The basic knowledge exists to begin to implement this vision.

However, the current means by which the NIH institutes address imaging research is as a stepchild -- a part of the research portfolio of nearly all of the institutes, but the principal focus of none. As a result, basic research into the development of new imaging technologies has been subject to overlap and duplication, inefficient use of resources, and lost opportunities. The initial invention of and basic research into new technologies that have emerged in recent times, such as CAT scanning, MRIs, and image-guided interventional methods, have most frequently occurred outside the US, where the logistics and funding of basic research into medical imaging can be handled in a more straightforward and integrated fashion.

The establishment of a new institute of biomedical imaging and bioengineering would correct many of these structural problems. The institute would address the needs of imaging research directly and comprehensively. It would provide a home and focus for the fundamental disciplines of computer sciences, physics, and engineering, that are so inextricably related to progress in imaging research. It would foster basic imaging research lacking in the current NIH structure, that would lead to the more efficient development of the new, broad-ranging technologies applicable to my vision of molecular medicine.

Through relationships the new institute will develop with regulatory agencies and industry, it would facilitate technology transfer, allowing important innovations to more rapidly be employed in medical care. And very significantly, the new institute would foster the more rapid assessment of new technologies as they enter practice to ensure that their use is appropriate and cost effective. This last aspect of the institute's proposed functions is critical, and is yet another example of how the current institutional structure insufficiently addresses the needs of the American public.ong my other responsibilities, I am Chair of the American College of Radiology Imaging Network or ACRIN. ACRIN is a National Cancer Institute-funded cooperative group, that through clinical trials, gathers information to extend and improve the quality of lives of cancer patients. ACRIN is a remarkable endeavor that evaluates the effectiveness of imaging technologies in improving health outcomes for individual patients, and measures the balance of benefit and cost the American public receives for its expenditure on cancer imaging.

The results of major definitive ACRIN trials of such technologies as digital mammography for screening for breast cancer, and CAT- scanning for the detection of early lung cancer, will guide medical practice and reimbursement in the years to come. The National Cancer Institute should be applauded for its vision in establishing ACRIN less than two years ago. Yet again, these same technologies that ACRIN will study, and many other current and future technologies, are broadly applicable to diseases other than cancer. There is no counterpart to ACRIN at any of the other institutes.

Even if there were, the fragmentation of imaging technology assessment on such arbitrary grounds would be wasteful, inefficient, and leave important gaps. The structural inadequacies that hinder imaging research can be rectified only through an institute. Institutes are the standard NIH administrative units for areas of such significant scientific research. Any institute devoted to biomedical imaging, bioengineering, and related fields will necessarily be of a size that would make any organizational unit short of an institute inappropriate.

Related research occurs in numerous federal agencies, such as the Departments of Defense and Energy, and the National Science Foundation. A subordinate administrative unit would lack the stature necessary to coordinate research involving imaging outside of NIH. For these reasons, and for those I have detailed in this testimony, I hope you will vote favorably on H.R. 1795, and pass it on to the full House of Representatives for its consideration. Thank you.

REP. BILIRAKIS: Thank you very much, Dr. Hillman.

Ms. Tomiko -- is that correct? Ms. Tomiko Fraser is the national spokesperson for the Lupus Foundation of America. Thank you very much for being here today, Ms. Fraser. Please proceed.

MS. TOMIKO FRASER: Good morning, Mr. Chairman and members of the subcommittee. I appear before you today representing the Lupus Foundation of America, on behalf of the 1.4 million Americans who have lupus erythematosus, a devastating disease that causes the immune system to attack the body's own cells and organs. Unfortunately, one of the victims of lupus is my younger sister, Shneequa (ph), who has a very serious case of lupus that affects her brain. The disease has been so devastating to Shneequa that she must receive around-the-clock care at a skilled nursing facility.

That is why I have agreed to serve as the national spokesperson for the Lupus Foundation of America. I want to help educate all Americans about the devastating impact lupus has on its victims. I urge Congress to pass H.R. 762, the Lupus Research and Care Amendments Act of 1999. Congresswoman Carrie Meek, who lost a sister to lupus, introduced this legislation. Two hundred and forty three members of the US House of Representatives are cosponsors of H.R. 762. The legislation authorizes a $23 million increase to the current funding level for lupus medical research supported through the National Institutes of Health.

It also authorizes $75 million to fund a grant program. This program would provide local governments, community hospitals, and other non-profit healthcare facilities with a pool of funds so they could offer lifesaving medical care to the poor or uninsured people with lupus. This grant program will help local communities hardest hit by lupus, especially in medically underserved areas, including rural and urban communities where often there is a shortage of medical facilities to treat people with lupus. Lupus deserves special funding consideration. Lupus is the prototypical autoimmune disease.

Research on lupus benefit all autoimmune diseases that disproportionately affect women. Autoimmune diseases are the fourth leading cause of disability among women. Lupus is an expensive disease to treat. The cost to provide medical care for a person with lupus averages between six and ten thousand dollars annually. The Lupus Foundation of America estimates the economic impact of lupus on the federal Treasury to be several billion dollars every year. These costs include disability income payments to the tens of thousands of lupus victims disabled every year by the disease.

They also include the cost of government-sponsored medical care provided through the Medicare and Medicaid programs, and uncollected tax revenues due to lost wages when individuals with lupus aren't able to work. The Lupus Research and Care Amendments Acts of 1999 is a bipartisan effort to address an urgent national healthcare crisis that inflicts an enormous burden on individuals, families, the business community, the federal government, and society. Many scientific opportunities exist, but current funding levels can support only one in four of the promising studies submitted for funding, that eventually will lead to a cure for lupus.

By accelerating medical research for lupus now, Congress will reduce future healthcare costs, and save billions of dollars for the Social Security and Medicare Trust Funds in future years. Lupus is a complicated and mysterious disease that needs extensive study. Presently, there is no cure for lupus, nor do researchers fully understand what causes the disease. We do not know why lupus alternates between periods of remission and periods of the disease activity called "flares." We do not know why the disease can remain mild in some individuals and become life threatening in others.

What we do know, Mr. Chairman, is that lupus has a devastating impact on its victims and their families. We know that lupus causes debilitating health affects, including extreme joint pain and swelling, constant fevers, overwhelming fatigue, horrible skin rashes, organ failure, and a host of other devastating symptoms. Lupus destroys the quality of life for many of its victims. The disease can severely damage the kidneys, heart, lungs, and other vital organs.

Lupus disables one in five of its victims, often at a very young age. And, tragically, every year thousands of lupus victims die from complications of the disease.

Lupus is not an equal opportunity disease. Ninety percent of the victims of lupus are women. Also, lupus is more common among women of color. Lupus is two to three more times likely to affect African Americans, Hispanics, Asians, and Native Americans than Caucasian women. Lupus also appears to be more serious among African American women. Approximately 20 percent of lupus cases begin in childhood. Unfortunately, lupus is more severe in children. Nearly 70 percent of children with lupus have kidney disease, as opposed to 30 percent of adults who develop lupus.

Whereas half of those with adult-onset lupus have organ threatening disease, nearly 80 percent of those with childhood-onset lupus go on to develop organ threatening conditions. Lupus strikes women in their childbearing years, between the ages of 15 and 44. This is one of the most devastating realities of lupus. It destroys the quality of life during a time when young women should be enjoying their best health. Many people with lupus suffer three to five years, visiting five or more doctors before they receive a correct diagnosis. Many medical schools do not provide family physicians with sufficient training to diagnose lupus.

By the time some lupus patients are diagnosed, especially in poor or rural communities, irreversible damage to vital organs may have already occurred. This increases the need for expensive treatments, such as kidney dialysis or transplantation. Medical researchers have made progress, and there is great hope for new discoveries. Still, most lupus patients are frustrated that the disease remains incurable. As you can imagine, Mr. Chairman, lupus is not an easy disease to live with. Over a million American families are struggling to cope with lupus every day of their lives. I know this personally from watching my sister suffer from the devastating affects of the disease.

It is time for action. A majority of members of the United States House of Representatives -- a total of 243 -- are cosponsors of H.R. 762. I urge that this legislation be brought to the floor of the House for a vote as soon as possible. Thank you for the opportunity to represent the victims of lupus at today's hearing, and I will be happy to answer any of your questions, and thank you for the extra time.

REP. BILIRAKIS: Thank you very much, Ms. Fraser.

Dr. Bryan.

DR. R. NICK BRYAN: Good morning. My name is Nick Bryan. I currently serve as Professor and Chairman of the Department of Radiology at the University of Pennsylvania. I really appreciate this opportunity to share some of my experiences as an imaging researcher and a former NIH staff member with you, and to express my support for H.R. 1795. I would like to thank you, Mr. Chairman, and the committee leadership for holding this hearing, and I'd like to thank the sponsors of the bill, Mr. Burr and Mrs. Eshoo, for their leadership and efforts on this issue.

You've already heard about the importance and uniqueness of biomedical imaging and engineering, and I will not belabor the point. I will instead focus on what I view as current structural inadequacies to support this field in the NIH. Prior to coming to Penn, I served for two years as director of diagnostic radiology and associate director, imaging sciences program at the Warren G. Magnuson Clinical Center at the NIH. During my tenure, and with superb support from Dr. John Gallon (ph), director of the Clinical Center, we were able to consolidate several disparate imaging departments into a unified imaging sciences program, which elevated the status of imaging research on the NIH campus, and began to lay a foundation for an advanced research program.

In the final analysis though, I felt the imaging sciences program could not be wholly successful, mainly because the very structure of the NIH makes such an endeavor problematic. Research authority and resources reside in the institutes, not in programs at the Clinical Center. As a result, the success of our imaging research was ultimately dependent on the ability of me and my colleagues to convince one or more of the institutes -- institutes whose primary missions and priorities are in areas other than imaging -- to divert funds from their main activities and commit those funds to imaging research.

I accepted the position at the Clinical Center knowing that it involved a significant challenge, but in the hope and in the belief that an effective imaging research program could be developed within the parameters of the NIH structure. In fact, at that time I was skeptical about the need for a new institute. My experience, however, gradually changed my opinion, and convinced me that the existing NIH organization will not work optimally for imaging and bioengineering. Ultimately, my decision to leave the NIH owed much to the inherent obstacles to imaging research that are built into its structure.

It should be recognized that the NIH does acknowledge the importance of imaging, and has taken steps to make imaging research a more visible part of its portfolio. As you heard, and as for instance, the National Cancer Institute has authorized significant expansion of the extramural biomedical imaging program. The NIH bioengineering consortium, known as BECN (ph), sponsored a conference in 1999 entitled, "Biomedical Imaging Symposium: Visualizing the Future of Biology in Medicine." This year, the NIH, in response to a Congressional mandate, has begun to organize a new office of bioengineering, bioimaging, and bioinformatics in the Office of the Director of the NIH.

The new office is to provide a focus for and facilitate work in our field. Unfortunately, all of these initiatives suffer from major flaws. First, the NCI program applies real resources to imaging, but the research is limited to cancer imaging. Cancer imaging is clearly important and should be an extremely high priority. But imaging, as I have said, is not disease or organ-system specific. It has applications far beyond cancer -- applications that are neglected when the research focuses on cancer or any other individual disease. Initiatives such as BECON and OB3, as it is called -- the new office -- constitute a useful effort to identify research opportunities and focus attention on imaging, but they bring little in the way of actual research dollars to imaging research.

They represent a strong commitment by the NIH to identify potentially fruitful areas of research, but no commitment at all to supporting that research. The director of the OBBB will have to do what I did. He or she will have to pass the hat by the current institutes for contributions, and I am certain that the donations will be insufficient to support a robust imaging research program. In fact, it is unrealistic and perhaps even inappropriate to expect the existing disease and organ system institutes to divert resources from their primarily missions in order to support basic research to advance the science of imaging.

For these reasons, I believe that the creation of a National Institute of Biomedical Imaging and Bioengineering is essential to promote the development of new imaging techniques and technologies. In order to flourish and grow consistently at the NIH, a scientific field requires an organization with the mandate, the responsibility, the authority, and the resources to direct and drive investigation in that field. In the NIH structure, institutes possess those attributes. I would like to conclude by noting that my opinions are not alone. Nearly all of radiology and bioengineering supports this initiative.

During the current year, I am also privileged to serve as Chairman of the Board of Directors of the Radiological Society of North America. The RSNA is the largest radiological organization in the world, with a membership of more than 30,000 radiologists, physicists, and allied scientists. The RSNA and more than 40 other professional organizations representing physicians, radiologic technologies, bioengineers and imaging scientists, have joined coalitions that support H.R. 1795. The total individual membership of these organizations is well over 100,000.

All of us believe that this is the time to create a National Institute of Biomedical Imaging and Bioengineering to support a field of inquiry that is central to continued progress in advanced research in biomedicine, as well to the development of better systems for delivery of healthcare. This institute would be good for patients, physicians, and the NIH itself. I urge the subcommittee to approve this bill. I'd be pleased to answer any questions.

REP. BILIRAKIS: Thank you so much, Dr. Bryan. Well, I'll start off the questioning.

Ms. Fraser, why does lupus seem to affect women of color more often than Caucasian women?

MS. FRASER: Well, this is a subject of a research project currently underway by the NIH, Lupus and Minority Studies or Lumina (ph). We believe lupus has a genetic basis, and it appears that the genes suspected of causing lupus may be more prevalent among women of color.

REP. BILIRAKIS: So we sort of know that or have come to that conclusion on the basis of studies that are taking place.

MS. FRASER: Yes, that's correct.

REP. BILIRAKIS: We don't know of any other reason other than the fact --

MS. FRASER: Not yet. No. But we're still checking.

REP. BILIRAKIS: In terms of the administration's support or non- support of the legislation, my understanding is -- and I may be wrong, and if I am, I wish to be corrected, but I think it's significant -- that they have problems with Title II (ph). The administration has problems with Title II. Are you aware of that?

MS. FRASER: I would be happy to answer that, but I would like to submit a written response to that question, if that's okay with the chairman.

REP. BILIRAKIS: Okay. We'd like to have that from you, by all means, because it would be very helpful in terms of not only moving the legislation through, but we also try to work with the minority in most cases to work things out ahead of time. That would be very significant.

Dr. Wirth, can you tell us the organizations, or at least some of the organizations, that you're familiar with that are working on the issue of global disease eradication?

DR. WIRTH: Well, there are several organizations in the world. The World Health Organization is very active in this area. But the World Health Organization is more of an implementation organization rather than a research organization. They have a very small research arm. Really the United States is really the only -- the United States National Institutes of Health, and to a certain extent, the NSF, are really the only organizations that have the knowledge base and the research base to bring that to bear on these important tropical diseases. There's some work in Europe funded by the European Union. But, again, I think the United States really has the leadership role and we need to maintain that.

REP. BILIRAKIS: I'm a Rotarian.

I don't attend very many meetings these days for obvious reasons. But in any case, they have worked on eradicating polio around the world, as you know.

DR. WIRTH: That is correct.

REP. BILIRAKIS: And that is working and has worked very well, hasn't it?

DR. WIRTH: Yes. Polio actually will be eradicated in this hemisphere this year. And there have been many groups involved in that and certainly the rotary has been involved particularly in the last several years. And again, I think that's implementing a very important step implementing the, sort of research and discoveries that have been made over the years primarily here in the United States.

So I think there are many steps to eradicating global disease. We have to get those vaccines and drugs that we have to the people who need them and that is very important. But we also need for many of these diseases to develop new interventions. The tools we have just aren't working.

REP. BILIRAKIS: Okay, so the World Health Organization is just not doing the job and you feel that the National Commission that Mr. Gekas is a proponent of, would do the job?

DR. WIRTH: I think so and I think it would particularly establish the United States in its natural leadership role in this area. I think that we need political leadership at this point to bring to bear on this problem. We have the skill set in the United States to develop these interventions and to implement them but we need to take that leadership role in the world.

REP. BILIRAKIS: Do we have now, and if not, then is that the reason, maybe, for the National Commission? Do we have the proper coordination? For instance, I don't know how (inaudible) has Rotary, for instance, gone about it all to know exactly where to go and have they coordinated...

DR. WIRTH: That's right. I mean, I think that one of the things the National Commission to do would be to have a focus point for this kind of work in the United States. I think in the case of Rotary and other non-governmental organizations, they have sort of gone about it themselves. Having to go to different agencies to different interest groups to begin to find out about it and then to become involved with the implementation.

REP. BILIRAKIS: Well, let me ask you this. If this commission is formed, the administration feels that CDC, NVP (ph), National Vaccine Program House, should be included in the composition of the commissions membership. And also that the FDA is the agency overseeing vaccine safety and the approval of new vaccines should also have a role of this bill if it is enacted. Your opinion?

DR. WIRTH: I think that those, particularly the CDC and the FDA would be appropriate to become involved in this. They have implementation roles and I think the National Vaccine Program is also one that certainly could be involved. They're dealing very specifically with vaccine issues, as you know, there are broader issues of implementation including drug development and development of other controls, measures, environmental, insecticides, and stuff like that.

REP. BILIRAKIS: Thank you, any other opinions regarding that particular legislation that you all may want to offer?

All right, that being the case the Chair yields to Mr. Brown.

REP. BROWN: Thank you, Mr. Chairman.

Doctor Wirth, I'm sorry I missed your testimony, I was voting. But I read your testimony and you said, as we begin the 21st Century we are blessed with unimaginable opportunities to build break-through research to control and prevent global infectious disease.

I would argue that World Health Organization has done phenomenal work over the last 20 years, but none the less she said, she was talking about tuberculosis in this case and I know your expertise is more malaria and I want to get to that in a second. But she said that it's not a medical irradiation of -- dealing with tuberculosis is not a medical problem it's a political problem.

But I think back and just in less then a year ago in December of 1999, the government of India working with a non government organizations around the world, including the World Health Organization, including all kinds of groups from this country, had a national immunization day and immunized 134 million children in one day. Which tells me that Mr. Gekas bill goes in the right direction and this country, our country, should show a great deal more leadership in dealing with issues that surely we can.

Tuberculosis and malaria both don't get the attention from the big drug manufacturers and their research arms that they should. The drug companies seem much to interested in, in my mind, in 'me too' drugs and drugs that are more a cure for baldness then a cure for tuberculosis, malaria, lupus, the whole host of diseases where there simply isn't the money, the potential profit available. There isn't a lot of profit in malaria or TB especially diseases that hit this country not very hard and hit the poorest countries with the poorest citizens, especially hard.

I'll shift gears to Walter Reed. Walter Reed has done especially -- and the defense department has done especially good research in malaria. We under fund Walter Reed, we fund organizations like NIH, a wonderful, wonderful government agency.

We want to double its budget in the next five years, yet we don't fund CDC very well. Which it's budget is about one sixth of the NIH and we don't fund the Walter Reed research arm of the defense department particularly well, putting it mildly.

Is the only real hope for malaria vaccine, TB vaccine, better treatment of those diseases? TB as you know, you need to take a pill everyday for six months, which countries with military occupation in places like Chiapas in Southern Mexico, people are afraid to go by the military checkpoints to get their TB pills everyday. And the difficulty -- even though we can cure it, it is difficult because of that.

Is the only hope for a TB vaccine and malaria vaccine, a better medicine to treat those two diseases, must it be government funding because the drug companies won't do it? And what do we need to do? Talk about malaria because that is your expertise. What do we do with Walter Reed within the defense department? What do we do with NIH and CDC on malaria?

How do we get, even though its not a great issue for me to go back to my district in Ohio and say, I'm working on malaria and TB. It doesn't matter much directly today to citizens of this country, it will down the road and that is a whole other issue, but what do we do with places like Walter Reed?

DR. WIRTH: All right. I think, you know, share your respect for Walter Reed and the work they have done over the last several years in developing anti malarial drugs. They really are the only group that has consistently maintained a research program even in spite of very limited funding.

And in fact, I think that the solution to these diseases is going to require a very large governmental component because the pharmaceutical industry, as you say, is driven by developing drugs that are important for this country. These are important drugs for this country but the diseases of tuberculosis and malaria and many other diseases found in tropical countries just will never have profit like drugs for diseases in this country.

The drug companies will not develop them and I think were going to have to -- it is going to require governmental intervention and governmental funding. I recommend that the Walter Reed, certainly receive funding. That the NIH receive funding for basic research and for transnational research. Something I think the NIH has become very interested in and very active in.

And in terms of CDC; CDC is our implementation arm. Once we have these tools we have to get them out and in fact, for many diseases we could certainly improve the situation today just by better implementation of the tools we have. We'll still face the challengers but certainly better implementation through CDC is important. So I recommend support for all of these organizations and let me correct myself if I misspoke.

I certainly have a great deal of respect for the World Health Organization but I think that they need help and they need leadership from the United States. Their budget is very small compared to the budget of the NIH for example. And I think they provide a forum but I think they need help from us and I think we can assume the leadership role in these diseases.

REP. BROWN: One last question, brief question. Should Walter Reed and the CDC be included in this bill, both?

DR. WIRTH: Yes. I think that is an excellent idea.

REP. BROWN: Okay, thanks.

REP. BILIRAKIS: Mr. Burr to inquire.

REP. BURR: Thank you, Mr. Chairman.

Doctor Wirth, I don't know that you misspoke so I don't think you need to apologize. The Chair and I have participated in the same hearing in international relations on the treat of global infections. A debate over whether it was a public health issue, whether it was a national security issue, and I think we can all agree it's probable "d", all of the above.

Clearly the World Health Organization and other international organizations that are targeted towards health issues have been effective on some things. Clearly there are other things where there are other things where health care officials have pointed out the deficiencies that exist. And with deficiencies in place, we can't be assured of successful immunization or successful eradication of diseases that ultimately we see as a threat, not only here but as a spreading threat throughout the globe.

Your reference to aids in Africa is a very good one. Its really important that we understand that the threat is that spread begins to happen in Asia, is a magnitude that we have never seen before, potentially. And every effort we can make, not relying on any one entity, is in fact the policy that we should adopt. And I appreciate you allowing me to editorialize just a little bit.

Let me move to some of the other witnesses if I can, because I do have some real interest in another piece of legislation.

Let me just turn to you, Doctor Bryan. Who benefits, who benefits from the creation of an institute for biomedical imaging?

DR. BRYAN: Well the people who benefit the most will be the patients.

REP. BURR: Isn't that who its all supposed to be about?

DR. BRYAN: That is exactly right.

REP. BURR: I mean, if for any person who is maybe on the fence on this issue as to whether we should create this. If they stop for a minute and thought, whose this about? If their answer was the patient then the answer is vote for this bill.

DR. BRYAN: I would agree.

REP. BURR: Is it safe to say, and I open this up to anybody, that as we identify break troughs in technology that we can also expect healthcare cost to possible decline? Because we detect earlier, our treatments may be less intensive as it relates to a period of time and if you look at the patient from that standpoint the quality of the care we deliver might in fact be better because we have put them through less?

DR. BRYAN: That has been the history of the development of imaging technologies, that in fact they do detect the disease earlier, they do replace more morbidity inducing, or illness inducing technologies and over time, I believe imaging technologies have been cost saving and also improved patient outcome.

DR. DUNNICK: I'd like to make two comments in response to that. The first, when the DRG's were established a number of years ago, my assumption was that the medical centers would try to reduce the number of ancillary test being performed. In fact, just the opposite occurred. We went to more ancillary testing in an effort to get to the answer faster, which in the long run would reduce the cost of medical care.

My second comment is a reflection of my own experience. When I was a medical student, my first research project was with influenza and we tried to use immunization to protect against that disease. We used death as the end point. Fortunately we were using mice as an animal model to test that.

As we move along, radiology has become very good at identifying disease processes, being able to quantify them in many cases and so we can use changes in imaging assessment as the end point for testing this.

We are now at what I call the era of molecular imaging or functional imaging. Where we can actually detect changes before they become manifest with routine testing. This allows us to see the changes, see whether treatment is effective before the disease has gotten out of control. I think these make dramatic changes in decreasing the cost of healthcare.

REP. BURR: Can any of you address a specific disease where say in the last decade the imaging improvements have changed in a --

DR. DUNNICK: Absolutely.

Trauma would be the first response to that. A patient comes into the emergency room and in fact it doesn't even have to be a traumatic injury, it could be the patient with abdominal pain. And the conventional way to treat that would be first to do an operation to open the abdomen and find where the pathology is.

We can do that non-invasively. In the trauma setting specifically, we can now identify not only the problem but in many cases quantify it, which enables more conservative therapies. So it has resulted in a dramatic decrease in the number of patients that have to go to the operating room.

REP. BURR: Let me ask one last question with the Chairman's indulgence. One of the fears that I have is that we are successful and not only in imaging but in other areas of medical break trough's we're successful. Technological improvements have not necessarily been rewarded through the reimbursement process in this country, specifically Medicare.

If in fact our reimbursement system does not recognize the cost of technology and the cost of this research. What will that do to the further development of new innovations, new treatments, new imaging that might detect this disease earlier?

REP. BILIRAKIS: Important question but brief answers please.

DR. HILLMAN: There are two things that this new institute will be able to do better then we are currently. One, as I indicated that it will have an assessment component that will run clinical trials in a timely fashion to provide the information to guide reimbursement. In fact that's been problematic under the current NIH structure.

The other is that we will develop relationships directly with regulatory agencies and payers to quickly move these technologies into practice.

REP. BURR: I thank the witnesses, I thank the Chairman. I yield back.

REP. BILIRAKIS: Doctor Ganske.

REP. STEVE GANSKE (R-IA): Thank you, Mr. Chairman, for having this hearing. I think that there are several bills that we are talking about that are half merit. And while they may not be the biggest healthcare issues that congress is facing such as prescription drugs or patient protection legislation, or even for that matter a bill that this committee will be doing shortly on providing relief for Medicare, in particular I hope relief for rural hospitals.

I just completed my series of town hall meetings back in the district and I get asked a lot about the high cost of prescription drugs and I find that there are one of these bills that I think that relates to that and that is the orphan drug act. Which created incentives for drug companies to develop therapies for rare diseases by awarding them a period of seven years of market exclusivity to a product approved for an orphan indication.

I find the testimony of Mr. Thomas Lang to be convincing. He says in his testimony, recently FDA has adopted a policy position related to the scope of a clinically superior drug exclusivity that actually undermines the incentives for companies to continue to innovate for additional improvements in these areas. As noted earlier, FDA's policy also raises questions of fairness, alternate product availability, and patient / physician choice of therapy.

Now after an approval of an original orphan drug, whenever a subsequent orphan drug with a clinical superior improvement has also been approved and awarded exclusivity, FDA totally restarts the seven- year exclusivity clock for the drug as a whole. In this way the improved drug shields the original drugs from competition even after the originals exclusivity period is over. In these instances companies that have developed new competing versions of the drug to treat the disease in anticipation of the expiration of the original seven-year exclusivity, are unfairly denied access to the market for an additional seven-year period.

I think this has pertinence to the high cost of prescription drugs and congress even in the short time period that we have left should significantly look at the Thornberry Bill, H.R. 4242, because I think that additional extensions of exclusivity will surly keep prices higher. That's why I and others, have been fighting a patent extension for the drug Claritin.

And so, Mr. Chairman, I again thank you for holding this hearing. I thank the people for testifying. I have another hearing that is ongoing at this moment that I will be going to and I will yield back.

REP. BILIRAKIS: And I thank the gentleman. If he will yield to me maybe 30 seconds of his time before he yields. I would like to -- you know we have a dilemma here in terms of lets say, NIH funding, lets just talk NIH funding. And you know the dilemma is should we in this so-called ivory tower, determine the amount of money for research that ought to go for specific diseases.

I mean what the experience that we've had on this committee has been just amazing the number of diseases that I'm sure that most of us, if any of us, although maybe some of our medical doctors, were not even aware of. Just some terrible sad stories and we are going to hear certainly some even in the next panel and the plea is more funding for Parkinson's, more funding for Lupus, more funding we can just go on and on. Muhammad Ali for instance, was here pleading for more funding for Parkinson's.

So the thought has been that we just don't know enough of actually taking place up there in terms of research and how close they may be to a break through and that sort of thing. Should we be telling them rather then just basically given them the money and doubling the money as Mr. Brown indicated.

Any opinion's in that regard, because I consider that really quite a dilemma. We've come to maybe a conclusion -- I had a talk with Mr. Brown on his feelings on that subject, I don't remember we've had in any case. But any feelings in that regard? Just very quickly, please.

DR. HILLMAN: Mr. Chairman, I think you do have indeed a major challenge and that's the responsibility that you all accept as our public representatives. I think that your directive is to provide broad strokes and directions to institutes such as the NIH and I do think you have to leave some of the details to them.

REP. BILIRAKIS: Yes, sir?

DR. DUNNICK: I think in terms of the H.R. 1795, what we are really talking about is not necessarily more funding but reorganization to establish focus and priority setting.

REP. BILIRAKIS: Which is basically what Ms. Fraser has testified to and what Ms. Meek's Bill does, right?

MS. FRASER: Yes, I just want to say that we just want to level the playing field pretty much. Lupus, I really didn't know a lot about it before my sister was effected with the disease and I learned more about it and learned that there are so many Americans, 1.4 million, that are effected with it. I think it is a disease that should be on the forefront right now, not putting anybody else's cause down or their testimony but we just want to level the playing field is why we are here.

REP. BILIRAKIS: Okay. I just wanted to sort of share with you the dilemma that we have and the difficulty sometimes.

Did you want to add something very quickly, Doctor Wirth?

DR. WIRTH: Yes, very quickly. I come from, sort of training, where I feel getting basic training and understanding fundamental mechanisms is very important to understanding disease. So, in general, I think it's very important that NIH be given as much free reign to follow the advances as they come.

But I also think it is important that the interest of individuals who perhaps can't sit at a table like this are represented in the area of biomedical research.

And I think without influence from the public to help direct the NIH to areas of importance, I mean the area of importance I clearly consider very important is global health and rarely is there anyone sitting at this table with direct experience in it.

And so I think it is very important that that be heard at NIH, at least in an advisory, perhaps a not absolutely directive way.

REP. BILIRAKIS: Thank you. I see that Mr. Bryant who was here earlier and had to leave has returned.

Did you have any questions of this panel, Ed?

REP. BRYANT: Mr. Chairman, thank you. Just some very brief statements and perhaps a question of Doctor Bryan, and I'm not sure -- I think I'm thinking more of the medical research at Pittsburgh and your down the road a little bit, I guess in the other direction, but perhaps you know something about this.

I agree with Doctor Wirth in terms that NIH ought to be given a broad reign, range I guess, in which to make their decisions and less input from those of us that come into contact with a lot of these difficult situations and have to -- they really can't pick and choose. We're not knowledgeable either to make those determinations. But on the other hand I think there is some need for input outside, as you point out, some representation and I guess to a degree, we do that.

It seems to me, and maybe I'm not using the setup of NIH correctly, but I have heard that the representation on their board or perhaps the doctors panels that help set these priorities, perhaps we could have a better play in what groups are represented there, what specialties, what doctor, what diseases, are represented there. And that would be away, again, of giving them broad powers but yet, we in congress, being able to make sure that one disease is not give priority over another one for the wrong reasons.

Secondly, and my last comment in this area, and I'm going toward something that I just mentioned earlier. I have been really working closely with a group in Memphis in terms of a disease that again does not address a large part of our population but a lot of our, well, to some of our young children, it's Dishing (ph) Muscular Dystrophy.

I was at a fundraiser for them about a weekend ago in Memphis and I am told there that is a disease where there have been great advancements made, and I think a lot of that has come out of the University of Pittsburgh or the Pittsburgh area. And that perhaps our priorities also ought to be, in addition to all of the other priorities we have, trying to find cures for those disease regardless of what the population effected, the size of the population effected.

Those disease that are getting close to being solved, cured, and that to me as a lay person, non-medical person, makes some sense. Because if we are getting close, because that can open the doors to other related diseases I would think. I would think the Dishing Muscular Dystrophy would have some very close cousins out there in terms of diseases that could be effected in a positive sense.

So, Doctor Bryan, again I'm asking you cold, do you know anything about that particular disease in terms of are we making progress there?

DR. BRYAN: I am familiar, I'm not an authority on that disease, but you're quite correct that it's a disease that effects a relatively small population but in a devastating fashion and remarkable advances have been made. Mostly in understanding the genetics and ideology of the disease.

I think the dilemma is one that is difficult. Your committee has to face the public needs to find areas where you think emphasis should be placed. But then, I think, to be honest, one has to defer to our peer review system, which the NIH has a superb peer review system, where the experts have to adjudicate whether in fact it is time, whether the knowledge is there, the technology is there, the feasibility is there, to actually at that time fund the additional research in that area.

So I think you all have to define already from a public perspective but then you have to, I think, take into account the experts in the peer review system to help decide when you actually support a particular research area.

REP. BRYANT: Very quickly, anyone else have an additional comment? Thank you for being here.

Thank you, Mr. Chairman.

REP. BILIRAKIS: And I thank the gentleman. Well we will excuse this panel at this time. We customarily furnish written questions and we request written responses and if you are all willing, of course, to do that in a timely fashion.

Ms. Fraser, sooner rather then later, particularly in the question that I raised.

MS. FRASER: That won't be a problem.

REP. BILIRAKIS: Thank you, thank you very much.

The second panel consists of, scheduled at least, Mr. Jack McCormick, Deputy Director of the Office of Orphan Drugs, for the Food and Drug Administration. Is Mr. McCormick here? No. Is someone else going to be here to represent the FDA in this matter?

(Off mike response.)

REP. BILIRAKIS: Well, you don't want to testify at all, technical responses?

(Off mike response.)

REP. BILIRAKIS: All right. Okay, in any case you'll be here for the testimony and for the questions that take place so that you can take those back too?

(Off mike response.)

REP. BILIRAKIS: All right, I appreciate that. Why don't you give us your name, sir, can you do that, maybe so we can have it in the record?

(Off mike response.)

REP. BILIRAKIS: Did you get that? No. Please speak up.

(Off mike response.)

REP. BILIRAKIS: Got that? Thank you, you guys are better then we are up here. And to introduce -- I'm going to introduce the other - well, I'll introduce Mr. Robert Brady is a partner with Hogan & Hartson, they're here on behalf of Biogene (ph).

Ms. Abbey Meyers, President of the National Organization for Rare Disorders, New Fairfield, Connecticut. Mr. Thomas A. Lang, Senior Vice President, Strategic Product Development Serono Laboratories in Rockville, Maryland, and he is accompanied by Mr. Nick Ruggieri (ph), Vice President, Government Affairs. Ms. Catherine Bennett, Chair, Board of Directors Cancer Research Foundation of America.

And would now yield with the committees indulgence to Mr. Dan Burton, who is not on this committee but who chairs another, of course, another very, very significant committee, who will introduce Mr. Navarro and at the same time take two to three minutes to talk about his legislation.

REP. BURTON: Thank you, Mr. Chairman, and I hope you maybe grant me just a minute or two latitude because I think some of the things that I would like to say are very important.

To my classmate and Chairman of the committee, Chairman Bilirakis, it's nice to be with you. I think it's the first time in the 18 years that we have been here that I have appeared before your committee so it's nice to...

REP. BILIRAKIS: I think that's true and, Dan, I'm sure the thought has crossed your mind, there aren't too many of us left, are there?

REP. BURTON: No and unfortunately we just lost one of our...

REP. BILIRAKIS: Yeah, we just lost one of our...

REP. BURTON: Anyway, I appreciate you holding this hearing and allowing us to testify on H.R. 3677, the Thomas Navarro FDA Patients Rights Act. The United States of America is a country based on freedoms and among the freedoms guaranteed through our constitution, our freedom of speech, freedom to practice a religion of our choice, and a free press.

However, we're not as individuals guaranteed to make a life and death decision in the area of medicine. Imagine our own government forbidding your child accesses to a non-toxic treatment. A non-toxic treatment with full human subject protection through clinical trials that has already saved the lives of other children. Imagine being told that you must subject your child to treatments that may cause him to be blind, be deaf, to make him sterol, to stunt his growth, to give him hormonal deficiencies, to lower his IQ and to give him secondary cancers.

Imagine having your choices reduced to changing no treatment and possible death or toxic treatment and possible creating a special needs child with no guarantee of success all at a time when another treatment is available. Imagine learning that the treatment that the FDA wants your child to receive, that two of the three drugs in the, quote, "standard protocol" of approval. Drugs clearly state on their package inserts, not proven safe or effective in the pediatric population. Now that is exactly what Donna and Jim Navarro have been faced with.

Imagine being a doctor who has treated cancer patients successfully for over 20 years. Imagine being repeatedly being attacked by the FDA in an attempt to stop your work. Attacked by the very agency that supposed to encourage and promote research. Image submitting to the BT 29 protocol so the four-year-old boy can be treated with a non-toxic cancer therapy whose safety has been established. A treatment which you have saved the lives of other children with the same type of cancer. Imagine this government agency putting that protocol on hold because of other existing treatments. Now that is exactly what has happened to Doctor Berzinski (ph) down in Texas.

Many of you have heard the story of little Thomas Navarro. You may have seen his story in People Magazine, in the New York Post, or on CNN. His father, Jim Navarro, is here today to testify and I'll leave the full story of Thomas's specific condition for Mr. Navarro to talk about.

Two years ago the parents of another little boy, Dustin Canarri (ph), testified before our committee regarding the FDA's gate keeping on clinical trials. Dustin had the same form of cancer as little Thomas Navarro. Dustin was the last person that the FDA allowed to receive antineoplastines without having first failed chemotherapy and radiation. He is now healthy and cancer-free today and without the devastation of chemotherapy and radiation side effects.

Over the last three years the Government Reform Committee has conducted five hearings looking at cancer treatments and access to care. Unfortunately, Thomas Navarro is just one of thousands of Americans who have been excluded from clinical research because of the FDA. He is just one of the thousands of children who are denied access to the parents treatment of choice because of the governments agency has made a life or death decision for the family and not allowed them the freedom to choose.

The heart of this whole issue, Mr. Chairman, is whose going to be deciding. Is it the role of the United States Government to make a treatment decision or is it the right of the patient and the family to make an informed treatment choice? H.R. 3677, the Thomas Navarro Patients Right Act, is a first step in restoring medical freedom. It is the first step in taking the decision making out of the hands of the government and putting it back in the hand of the individual where it belongs, an informed decision.

Mr. Chairman and members of the committee, H.R. 3677 has forty- three cosponsors from both sides of the aisle, democrat and republican. I respectfully request your help in getting this bill passed during this congress.

I am now pleased to introduce Jim Navarro, Mr. Chairman. And once again I want to thank you very much, my colleague, for holding this hearing. Jim testified at our June hearing and shared with us the challenges that they faced as a family dealing with a cancer diagnosis and the federal agency that has forced them into a corner.

They have spent almost all of their money, I think they have sold their house, I think they've completely depleted all of their resources in trying to solve the problem of their boy. And it is a heart-rendering story and I know Jim is going to go into it in detail. He is here to testify about this bill. Jim and Donna Navarro are intelligent, conscientious parents; they love their boy. They have stood firm in the battle to find the best and safest treatment for their child and Jim is a brave man. Fighting a battle on two fronts. While he is in a battle for his son's life, he just recently discovered he is in a battle for his own life. Several months ago Jim was diagnosed with prostate cancer.

So, Jim, we wish you the best and we pray for you and your boy and with that, Mr. Chairman, I would like to yield, if you don't mind, to Mr. Navarro.

REP. BILIRAKIS: Well thank you, Dan. Thanks for your interest in this subject and of course in all issues in America and the Chair now will yield to Mr. Navarro, who uses as an address, Ronald McDonald House, room 1101, New York, New York.

Mr. Navarro, please proceed, sir.

MR. JIM NAVARRO: Thank you, sir. That is home as you stated, because as the Chairman stated, there is no longer a home for us.

Good morning, Mr. Chairman, my name is Jim Navarro and I am the father of five-year-old Thomas Navarro for whom this bill is named. I have been asked to speak on the benefits of this bill and I would first like to go on record as saying that my sons health has not stood still while the slow wheels of government move and thus this bill will not help my son.

It is too late to bring hope to our family. Hope that the FDA would stand down and allow my son access to our first choice of treatment. We were forced to begin the FDA's preferred treatment this summer. This bill will however, help thousands of others. This bill was conceived as a result of the FDA's unwillingness to allow Thomas access to a treatment which had a higher rate of success then the treatment offered through conventional means.

This bill will however, bring hope to others. Others who like us have been denied access to treatments that show promise and give a chance of survival. Treatments that are good or greater then those treatments currently available for treating pediatric cancers.

We were faced with the decision almost a year ago, which changed our lives forever. When our son, Thomas, was diagnosed with meningeal blastoma, which is a non-survivable cancer, Thomas was rushed into surgery within hours to remove a four by six-centimeter tumor from his cerebellum. After surgery we were faced with a decision of follow up therapy. We discovered in short order that the standard follow up therapy, radiation and chemotherapy both had sever and irreversible side effects.

These side effects included the possibility that he would become blind, deaf, and sterol. That Thomas would develop hormonal deficiencies that would have stunted growth, that would have had an immediate and progressive loss of IQ, and that he would develop secondary cancers as a result of the treatment itself.

We immediately began our search for a safer, non-toxic means of treating our son. We found a treatment that showed a great promise for treating miginal blastoma, only to discover that our son would be denied access to the treatment by the FDA. The doctor was not allowed to treat my son because the FDA did not approve his access to the treatment. Yet the FDA has never approved radiation and chemotherapy for treating pediatric cancers.

In fact, if you read the manufacturer's information that the drug companies put in the box, they state, safety and effectiveness in pediatric patients has not been established. This sentence in and of itself should cause concern. The FDA has no problem forcing this therapy on my son and thousands of others even though the safety and efficiency has not been established in children.

In fact, if you are the parent of a terminally ill child, your child can be taken away from you for experimentation. And as parents, if you do not cooperate with this madness you can be thrown into jail for being bad parents. Based on your experience, Mr. Chairman, what actions must I take today to get you and your committee to take the required action to save the Thomas Navarro's of tomorrow?

During the course of this last year, my family has lost everything. Our home, our business, even our state of residency, which although it is hot, it is a dry heap. It has been because of the kindness and generosity of others, especially the support of Citizens for Health that Thomas has been able to receive medical care.

H.R. 3677, introduced by Congressman Dan Burton, now has 43 cosponsors and I implore you to take this issue up and get H.R. 3677 passed into law. Thomas is very hard to recognize now as a result of conventional therapy and I would like to encourage, Mr. Waxman and any others that would stand in opposition to come see him. What he looked like before and what he looks like now.

Thomas's fight for his life now includes fighting against the very treatments that he has been forced to take and I can only tell you its been a very long and hard year. Thank you for letting me speak.

REP. BILIRAKIS: Thank you very much, Mr. Navarro, I do -- well, what can one say. We want to be able to accomplish something here. We want to be able to pass Dan's Bill or essentially Dan's Bill, but it's got to be done in a bipartisan basis. That's your reason for imploring Mr. Waxman, who frankly is just been very much interested in healthcare all through the years. I know I have worked with him on this committee for many, many years and I don't know really what his position is on the legislation, I guess staff here does, but we are going to do everything we possible can. Thank you very much, sir.

MR. NAVARRO: Mr. Chairman, if I might add real quickly. It's interesting that when I testified last time in Chairman Burton's hearings and spoke to a number of the directors of the FDA outside in the halls, they kept trying to reiterate the great successes of conventional therapy in Thomas's case. And yet the irony is, here before me are the consent forms to the treatment that Thomas is going through now, which say, permission for participation of child in research.

He is not in a protocol because they don't have a protocol. They really don't know quite what they are going to do with him, it's a hit and miss, and as they would say, a crap shoot. In fact, one of the things that it said that disturbed me, especially after hearing Doctor Pasdor (ph) testify, that the rate of success was 70 and 80 percent and this is coming out of the horses mouth.

However, with standard therapy there is less then a 30 percent chance of curing these malignant brain tumors in young children. Furthermore, young children treated with radiation therapy for brain tumors may experience serious and irreversible long-term side effects from the radiation. And yet yesterday, the doctors announced to us that because Thomas has faired the toxic side effects better then the other children in the ward, their anxious to start using high, high dosed radiation and chemotherapy five to six times greater then they have used on him so far.

And to be honest, sir, he is tired of fighting the drugs. We need to have the freedom to seek out a treatment that is non-toxic and non-lethal. It is our right as Americans to have that freedom.

REP. BILIRAKIS: Thank you, Mr. Navarro.

Mr. Brady, Mr. Brady, your up, sir. Obviously the written statement that you all submitted is made a part of the record and we would prefer that you might, sort of supplement it or what ever.

MR. ROBERT BRADY: Thank you, Mr. Chairman, I'll be quite brief and just summarize my comments. I am Bob Brady, I am here appearing on behalf of Biogen, Inc., a biotechnology company from Massachusetts. I am a partner in the law firm of Hogan & Hartson where I have been practicing food and drug law, focusing on pharmaceutical matters for 25 years, including the implementation of the Orphan Drug Act.

Let me summarize my points and then I'm just going to focus on two or three of them. If enacted H.R. 4242, the Orphan Drug Innovation Act, would actually undercut the carefully crafted incentives of the Orphan Drug Act without providing any real benefits to patients or promoting innovation. The Orphan Drug Act has been an unparalleled success. Any changes to the Act should only be made after careful analysis and consideration by fully informed members of congress in the context of the entire law.

The FDA and its office of orphan's product development, which has done a conscientious and successful job in implementing the law to date should be consulted and its views taken into account. Moreover, Biogen knows of no patient advocacy groups supporting this law nor do we know of any other organization other then one here at the table supporting this provision.

The Orphan Drug Act should not be amended piecemeal by an amendment hastily packaged together with non-controversial measures at the end of a legislative session. It would undermined the foundation of the Orphan Drug Act so a single company can market a product that has not been shown to be clinically superior to orphan drug products already on the market.

Let me speak one moment about the Biogen product, which is a product to treat multiple sclerosis, which was approved and is the only multiple sclerosis drug approved for two indications to treat this terrible disease. And it has been approved by judgement of FDA that it is clinically superior for safety reasons to the prior drug approved in this market place. That's important because that will be a point of discussion here during the rest of this morning's testimony.

The Orphan Drug Act is one of the most effective laws enacted by congress with full bipartisan support in the last 20 years. Especially in terms of the lives it has enhanced, the pain and suffering it has diminished, and the hope it represents to Americans with rare diseases. I might also add parenthetically that after 25 years it has been the least controversial of FDA law ever enacted in terms of subsequent debate and litigation, suggesting that it was well done to begin with and remains properly implemented.

However, H.R. 4242, would undercut the overwhelming success of this act. The key incentive of the act is a seven-year period of marketing exclusivity for the first product to be approved as an orphan drug. H.R. 4242, would significantly narrow the scope of this exclusivity by limiting it to particular aspects of the orphan products subsequently approved.

Narrowing this key incentive, especially for a product which has not shown any clinical superiority, would not only hurt companies that make orphan drugs but would also undercut congresses intent that there be new and innovative treatments developed for millions of Americans who suffer from rare diseases.

The orphan drug policy, first passed by congress and implemented by FDA have been fair. Companies are rewarded when they produce a clinically superior drug that represents an innovation above the current market place. Biogen in fact, satisfied this standard in the law in the mid 1990's when it was found to be clinically superior to the existing multiple sclerosis product.

Serono Laboratories is testifying here today on behalf of this bill. They manufacture a drug for multiple sclerosis that is the same as Biogen's multiple sclerosis product. Serono would like to get their drug into the American market but they are blocked by the market exclusivity of Biogen's product, which does not expire until May of 2003.

The Orphan Drug Act and the FDA implementing regulations currently provide a way for Serono's product to get to market. Precisely the same way that Biogen's product got to market in 1996, which is to prove clinical superiority to the two existing products that are already available to multiple sclerosis patients today. This is not a situation where there aren't products available to patients. There are two interferon products already approved by FDA in this area. Serono or any other company should not be held to a lesser standard then the products that are already on the market.

Thank you very much, Mr. Chairman, for allowing me this brief statement and I am prepared to answer any questions you may have.

REP. BILIRAKIS: Thank you, Mr. Brady.

Let's see, Ms. Meyers, please proceed, ma'am.

MS. ABBEY MEYERS: Thank you, Mr. Chairman. For those of you who don't know us, the National Organization for Rare Disorders is the consumer organization that advocated for passage of the Orphan Drug Act and we continue to monitor it's implementation. We do not support H.R. 4242 and let me say at the outset, we have no relationship with Biogen. Biogen has never donated to NORD, this is totally independent.

Most orphan drugs have only one sponsor and that is very important to understand because this situation comes up very rarely then when more then one sponsor are interested in the same drug. And so we caution you to change the Orphan Drug Act in any way based on something that happens so rarely.

You can get the same drug, orphan drug, on the market to compete against the innovative drugs, you can do that in several ways. You can get an orphan drug approved for a different disease. For example, if (abada ?) interferon was approved for cancer or something else, you could get it on the market and it would compete in the market place.

Or you can prove that it is chemically or structurally different then the first drug to get on the market. Or you can show that it has clinical superiority. Clinical superiority means that you have to prove that it's safer or more effective or a major contribution to patient care.

In the case of abenex (ph) and (betaserine ?), for example. Abenex showed that you would need less injections every week, it had fewer side effects and didn't cause one particular side effect. And so it was a major contribution to patient care and you needed only one injection a week.

So the current law protects the major incentive of the Orphan Drug Act, which is seven years of exclusive marketing rights. And it's only through regulations that the current definition of same and different was created. And I want to tell you the Orphan Drug Act, passed in 1983 but those regulations weren't written and published until 1992. So we waited many years and there was a lot of public input on the development of those regulations.

The Thornberry amendment, we feel, would destroy the backbone of the law because it would undermine the major incentives of the Orphan Drug Act. And also be aware that the Orphan Drug Act success has been replicated all over the world. The European Union just passed an orphan drug law, Japan has one, Singapore, every country in the world admires what we have been able to do here.

So we need to keep the incentive in place that would spur other manufacturers to develop clinically superior orphan drugs. In the case of multiple sclerosis, for example, people have very poor muscular control giving themselves an injection is like climbing Mount Everest every day. And then they loose their eye site so they can't even see to fill up the syringe. It is a major contribution to patient care when you have something where you need only one shot a week and a nurse can come to your house to do it.

So what do we see in this situation here with this argument over Abata Interferon? If Serono, and we have the greatest respect for Serono, but if Serono believes it's drug is either safer, or more effective, or that its clinically superior, or even if they want to say that their drug is the same as the original orphan drug so it would be able to get on the market today, if it could prove it is the same as Beta Seron, they should take their proof to the courts.

They should not be hiring lobbyist to come down here and ask you to change the law. It is wrong for company after company, year after year to come to you and ask for an amendment to the Orphan Drug Act. It works and if it ain't broke don't fix it. I'll be glad to answer any of your questions that you may have about the law. And we say again, we do not support this amendment.

REP. BILIRAKIS: Thank you, Ms. Meyers.

Mr. Lang.

MR. THOMAS A. LANG: Good morning. My name is Tom Lang, is this on?

REP. BILIRAKIS: Pull it closer but it's on, yes.

MR. LANG: My name is Tom Lang, I'm Senior Vice President for Strategic Product Development at Serono Inc. I want to thank the committee for the opportunity to testify on the issue of orphan drug ever greening, which is addressed by H.R. 4242.

Serono believes this issue needs to be addressed irrespective of the impact on our products. FDA's current ever-greening policy effects all drugs governed by the Orphaned Drug Act. We fully support the remedy posed by H.R. 4242 whether or not it would apply to any of our products. Furthermore, Serono believes that orphan drug ever greening is not a single product issue.

Serono is a strong supporter of the Orphan Drug Act. However, we have recently encountered an anomalous and confusing FDA interpretation of the orphan drug regulations, which results in what we are calling orphan drug exclusivity ever greening. Ever-greening refers to FDA's granting of a new seven-year orphan drug exclusivity period for the entire drug substance upon the approval of a clinically superior version of the same drug rather then protecting only the innovative feature exhibited by the second drug.

The results are to close the market to competition beyond the initial seven-years of exclusivity intended by congress. This raises troubling policy issues of fairness, impediments to price competition that would benefit consumers and delays in availability of alternative therapies for patients. Mr. Chairman, congress needs to decide exactly what the scope of exclusivity should be for improved versions of originator orphan drugs.

We note that other areas of food and drug law limits the scope of exclusivity for new versions of previously approved products in a manor consistent with H.R. 4242. Like the Orphan Drug Act, the Waxman / Hatch Act seeks to increase incentives for continued research on approved drugs and product improvements.

The Waxman / Hatch Act, as one would expect, rewards only the innovative feature with exclusivity rather then shielding the entire drug substance from competition when it's original exclusivity period has wrung. This serves as evidence as congress' intent and provides a basis for supporting the principal in H.R. 4242.

FDA's handling of one particular product has resulted in several very strained policy positions on the part of FD Agency. In a letter to Serono, dated in November of 1999, FDA indicated that while it would not allow an NDA or a BLA product to be marketed in competition with the original drug it would allow a generic version of the original product to come on the market if it was eligible for an abbreviated new application.

Mr. Chairman, there is no rational whatsoever for preventing competition from products that are supported by full NDA's and BLA's. Subsequently, Serono became aware of an instance where FDA has taken what appears to be a different position then that previously described. A position which actually is consistent with H.R. 4242 in a letter to Genentech (?).

Serono believes orphan drug exclusivity ever greening can be resolved by FDA or congress by simply limiting the second clinically superior drug scope of orphan drug exclusivity to the superior characteristic that distinguished it as clinically superior. The solution would properly reward the improvement found in the clinically superior drug while still allowing competition with the expired drug as intended by the law.

This would be consistent with other exclusivity related legislative initiative, such as the Waxman / Hatch amendments and patent law as well. Limiting the scope of exclusivity of a clinically superior orphan drug to its clinically superior feature still leaves the drug sponsor with adequate incentive.

A clinically superior drug would gain three significant rewards as follows.

First, it would achieve the benefit of being allowed on the market immediately despite the originator drugs exclusivity. Second, it would obtain seven-year exclusivity for the improved feature. And thirdly, the company would be able to market its product as a clinically superior product.

These are substantial awards and incentives. These incentives make it unnecessary to keep the market closed to other products wishing to compete with previous versions whose exclusivity has expired.

In summary, the current evergreen policy actually inhibits innovation, deters competition, and creates an anomalous windfall extension of drug exclusivity. We have attempted to work with the FDA to resolve this issue for two years. Nevertheless, in Serono's opinion, FDA continues to administer the exclusivity principal in an inconsistent and unclear manor.

The evergreen policy is now riddled with add hock exceptions not found anywhere in the statute or in the regulations. We there fore believe that clarifying legislation is warranted. We thank the committee for the opportunity to testify on this important matter effecting the incentive to develop improved drugs for rare diseases. We appreciate the committee's attention and consideration and I would like to thank Doctor Ganske for his earlier comments.

REP. BILIRAKIS: Ms. Bennett, please.

MS. CATHERINE BENNETT: Thank you, Mr. Chairman, and members of the subcommittee. I appreciate this opportunity to testify on an issue of great personal importance to me, cancer awareness, treatment and research.

I am here representing the Cancer Research Foundation of America as chairman of its board of directors. CRFA is a national non-profit health organization whose mission is cancer prevention through scientific research and education. Since its founding in 1985, the foundation has funded research by more than 200 scientists at more than 100 leading universities and medical centers, and it is one of the only 10 non-federal agencies whose grant review process is approved by the National Institutes of Health.

Within the last year, CRFA has increased its focus on childhood cancers with the establishment of Hope Street Kids, a foundation created under the umbrella of CRFA following the loss of Caroline Price Walker. The mission of Hope Street Kids is to eliminate childhood cancer through advocacy, education, and cutting edge research and to help sustain and support children with cancer and their families during and after treatment.

Unfortunately, most of us have had a personal experience with cancer. We've seen it attack a family member, a friend, a co-worker, or we have been diagnosed ourselves. I was diagnosed with breast cancer in 1993. It is a dreaded and pervasive disease that claims the lives of more than 500,000 Americans each year and it is a disease that knows no racial, ethnic, economic or gender boundaries. Perhaps what is even more disturbing is that cancer also does not discriminate based on age.

Many of us think of it as a disease of the elderly or middle aged. But, we must also recognize that cancer is the number one cause of death by disease for children. Each year, more than 12,000 children are diagnosed with cancer and some 2,300 children will die from the disease. That's about 100 classrooms filled with children who wont' start school next September.

September is significant in that is it recognized as National Childhood Cancer Awareness Month. So, it is appropriate that the committee has House Resolution 576, sponsored by Congresswoman Deborah Pryce, on its agenda. I am pleased to testify in support of this resolution, which seeks to raise awareness about the realities of childhood cancer and make suggestions and recommendations about where Congress can help ensure that more children live to start a new school year.

The statistics in the resolution demonstrate the challenges we face. The incidence of cancer among children is rising by 1 percent each year. One in every 330 Americans develop cancer before age 20. It constitutes about 8 percent of deaths between the ages of 1 and 19. And as I mentioned, it is the leading cause of death by disease in children.

It is clear to me that we cannot dismiss this disease as rare or ignore the substantial loss of life for which childhood cancer is responsible. In my mind, even one child lost to cancer is unacceptable. The good news is that progress has been made. Forth years ago, a diagnosis of childhood cancer was a death sentence. Today, almost 70 percent of children diagnosed will survive. Nonetheless, that means 30 percent do in fact succumb.

The success rate can be attributed in part to research and clinical trials. They have become the standard of care for pediatric oncology patients with approximately 70 percent of the children who are diagnosed participating. It makes sense to build on these efforts by making sure that opportunities for childhood cancer research are funded and that we attract the best and brightest to pediatric oncology and that we make sure that as many children as possible have access to the Centers of Excellence and clinical trials. The resolution suggests that Congress supports such policies.

Additionally, H.Res. 576 encourages support for policies that encourage the development of new drugs and biologics. As members of this committee know, the Food and Drug Administration Modernization Act provided additional incentives to encourage greater private investment and research by providing some additional six months of market exclusivity to sponsors of new or approved drugs if they conduct pediatric studies.

Despite the good intentions of this law, the policy has not proven as effective in stimulating research or providing additional information about drugs that may prove useful in pediatric oncology. I believe it's worth reevaluating the policies reflected in that statute.

While we look to the future with hope that we will see the day where no child becomes the innocent victim of cancer, we must also face the reality that children today are suffering and are dying. We must focus our attention on proving the quality of life for these patients. The horrors of cancer are many, but it is hard to imagine anything more torturous for a parent witnessing their child in pain. Yet, many will tell you that they have been forced to stand helplessly by while their children endure invasive and painful treatments.

The resolution points out a recent study revealed 89 percent of children with cancer experience substantial suffering in the last month of life. Why in this day of modern medicine and technology is this necessary or acceptable? In my view, it is not.

The reason for inadequate pain relief for children and cancer patients may be many, but one can be found in the lack of training for pain management received by physicians in their medical training. We can begin to address this issue by expanding knowledge among medical personnel to help them recognize the signs of pain and treat them effectively. The resolution is supportive of such curriculum as part of medical training.

The battle against childhood cancer is being hard fought. But, those who know the horrors of this disease, and many of them will be in Washington this week to do what they can to raise awareness and recruit Congress and others, whoever will listen in fact, to their cause. I believe Mrs. Pryce's resolution is a good first step that indicates a congressional understanding of the issues at hand and provides an outline for what a successful policy aimed at defeating childhood cancer should entail.

I encourage the subcommittee to lend its support to this legislation and again appreciate the opportunity to participate today. Thank you very much.

REP. BILIRAKIS: Thank you, Cathy, and welcome.

The chair would take this opportunity to recognize himself as soon as he gets his thoughts. And, we apologize, but there are members trying to get back and it's the intent of the committee to continue with the hearing rather than to take a break for lunch. Because of the afternoon schedule here, we think it's more beneficial not only to us, but also to you to go ahead and allow those member who can make it back to ask questions.

Ms. Meyers, let me ask you some specific questions.

Things have changed significantly since we originally put together the Orphan Drug Legislation, haven't they?

MS. MEYERS: Yes, yes.

REP. BILIRAKIS: Can you be a visionary for us just a minute and look out once the Human Genome Project is complete, once we have mapped sort of the genetic outlay of the human structure, how many of what we classify as rare diseases today do you think that researchers will be out there trying to find the key to the cure for it in the future?

MS. MEYERS: Well, it's very complicated because it's not just a matter of everybody, for example, with muscular dystrophy, having the same genetic defect. There are many different genetic defects that may result in Duchenne's muscular dystrophy.

And down the road, in about 20 years, the way I foresee it, the way the scientists do, is that they will able to personalize drugs for the particular genetic defect that has occurred in individuals. And so, we will have custom-made biotechnology drugs to address the specific defect in that gene.

REP. BILIRAKIS: Which means the population, that because they're going to be subsets of disease --

MS. MEYERS: Right.

REP. BILIRAKIS: -- the population that they're going to be targeted are going to be tremendously small.

MS. MEYERS: Miniscule, yes.

REP. BILIRAKIS: So, is it safe for the members of this committee to assume that a large share of the pharmaceutical applications that will go in are going to be under the Orphan Drug Legislation because the population defined that 200,000 people, if I remember correctly, we will clearly be chasing a multitude of things under that population?

MS. MEYERS: It will be a growing number of treatments for the full population, most of which will come from biotechnology.

REP. BILIRAKIS: Is there any reason that we as a committee, and as an institution, should in any way, shape or form look out at that 200,000 person number knowing the changes that are going to take place through the genetic mapping and at least debate, or possibly change that number to be more reflective of where we think exclusivity should be in the future? I'm not talking about the debate that we're at at this table. I'm trying to think out a number of years.

MS. MEYERS: Well, looking back over the 17 year history of the Orphan Drug Act, the problems that have arisen have not arisen around the size of the population. The problems that have arisen are pricing problems. And even drugs for very tiny numbers of people, if you're going to charge $100,000 or $200,000 for that treatment, you're going to make a lot of profit.

And so, if there are any changes to the Orphan Drug Act, and one of them was introduced, and actually passed the House and Senate by Mr. Waxman in 1990, and it was vetoed by President Bush, and that was aimed at shortening the period of exclusivity for blockbuster drugs.

So, I would not lower the size of the population because 200,000 is not a huge number. And believe me, it's even hard to find companies that are willing to make drugs for three or 400,000 Americans.

REP. BILIRAKIS: In today's research environment?

MS. MEYERS: Yes.

REP. BILIRAKIS: Ten years from now in tomorrow's research environment where you've got a map that leads you to a point that it took you five years now to hopefully do research to find, my question was not should we, it was should we at least have a debate on it? Should we bring in individuals out of biotechnology, and pharmacological research, to discuss what do you see down the road? Are you going to be chasing diseases because of the information that you have that are a population of 5,000, and 7,000 and 12,000? Which means that the majority of the stuff that we do will be classified under the Orphan Drug.

MS. MEYERS: It's true. But, you know, the bigger these companies are getting with their mergers and their acquisitions, I mean, they're interested in Viagra. They're not going to be even looking at these types of diseases. The latest one is something about removing facial hair. I mean, these kinds of markets are so huge the big companies don't want to look at a drug with an estimated sales under $1 billion a year.

REP. BILIRAKIS: Well, clearly, you make a point that is probably an accurate one today, if through these advances it is much easier for them to design that drug of the future, as you said a custom designed drug, it may be a whole different situation.

MS. MEYERS: I still say yes.

REP. BILIRAKIS: Did you ever envision under the Orphan Drug Law that, let me ask it a different way. Do you think it's right under the Orphan Drug Law that a company could have an approval, could have their exclusivity, at some point during that period of exclusivity, they made an enhancement to the product? They reapplied and were approved for whatever reason at FDA and got a new year seven year exclusivity?

MS. MEYERS: Yes.

REP. BILIRAKIS: Did you envision when the Orphan Drug Law came about that that was something that would happen?

MS. MEYERS: First of all, biotechnology was in its infancy. We couldn't imagine what would happen with biotechnology. But, we saw early on in 1985, Genentech got approval for human growth hormone. And a few months later, Eli Lilly came on with a different version of human growth hormone. And, it created exactly this situation. FDA approved Eli Lilly's second version of human growth hormone saying it was chemically or structurally different.

REP. BILIRAKIS: Two different companies?

MS. MEYERS: Two different companies.

REP. BILIRAKIS: Should the same company have the ability to reapply and for whatever changes get a new seven year exclusivity agreement?

MS. MEYERS: Yes. And, that has also happened with Genzime's (ph) drug for a genetic disease, for Gaucher's (?) Disease, where they did improve it. It had been made out a natural blood clump for something and then they made a biotechnology version and they got another seven years. Yes, they should because the main incentive is to develop a better drug and it worked.

REP. BILIRAKIS: Do you see any problem with the fact that the company who has the current exclusivity certainly has a tremendous advantage because they have the data? That's not data that is shared within the community of researchers that are out there. And if in fact nobody wants to invest the money to create that database to chase that small population drug, you really do have an inherent ability of one company to continue to restart the clock. And, I'm not saying that it happens today.

I think that to some degree in health care, we have to start getting visionary in this institution. We do a poor job at crisis management, but that seems to be the only thing that we try to address now, is the crisis management of today's problems. And I think that we've got to focus out on the future and ask ourselves what do we need to do in preparation for the changes. And I would only suggest to you that I see a potential problem there as a member of Congress.

I see the ability for one company to continue to restart the clock almost like FDA used to do in their application process when they changed investigators. And when they wanted to slow down the process, they asked for a new piece of information and the new 180 days started. And that became more the norm than the exception.

MS. MEYERS: Right.

REP. BILIRAKIS: But, it's a question that I raise.

MS. MEYERS: I agree with you. It could be a potential problem. But, the fact is when the exclusivity on the first drug expires, any other company can get on the market and make that first drug, like a generic drug. Except for one thing, Congress has never passed a law that allows the FDA to approve generic biologics.

And so, they have to do all of the research, all of the clinical research, and approve all the safety and effectiveness of a brand new drug. And then, they're allowed on the market to compete with the drugs whose exclusivity has expired. That's what could happen here.

Beta serine (?), the first beta interferon, their exclusivity has expired. If any company can prove that their beta interferon is the same as beta seron, they would get on the market.

REP. BILIRAKIS: We've clearly got some work that we know we need to do. I want to turn to Mr. Navarro.

Mr. Navarro, I don't want you to think that in any way, shape or form that members of this committee, and members of Congress, haven't struggled not just this year, but for a number of years to try to find the right balance of the gold standard of the FDA, their process, and the innovative treatments that Dr. Berzinski (ph), and others, have in the marketplace today.

And certainly, I've been involved for four years in Dr. Berzinski's treatments. And hopefully, this committee has contributed greatly to the process forward of the current clinical trials that he has, the expansion of those trials as a liaison between FDA and Dr. Berzinski on the data that was needed to get expansions.

But, I don't want to address Dr. Berzinski's treatments specifically because one thing I want you to understand is that members of Congress are not here to practice medicine. We are here to try to address the structure that's needed for everybody to receive the quality of care that they deserve.

In doing that, I have found it to be very difficult because, quite honestly, many of the patients that visit me with the personal stories of their fight don't come back the next year. That makes a very, very big impact on every member of Congress, I can assure you, as it does the families, of which many of affected in our families.

And my hope is that we can be visionary. We can look at some of the treatments that exist out there. And that we can form a partnership between medicine and FDA, and medicine and NIH, and medicine and HICVA. And, that we can get patients back to the forefront of the health care delivery system in this country.

We spend a lot of time arguing whether it's reimbursements, or whether it's doctors, or whether it's hospitals, or whether it's insurers. And, really more time about the process than we do about the outcome. I understand. You're only concerned with the outcome. That's all you should be concerned with. We've got to deal with everything else.

But, let me ask you specifically as it relates to your son, is it your understanding from the health care professionals that treated your son that there was no conventional treatment that was FDA approved, be it chemotherapy or anything else that they had suggested, that was specifically FDA approved for pediatrics?

MR. NAVARRO: You have to understand that radiation, and chemotherapy, and I'm going to pick just on Thomas's disease for a minute, has not been approved for the very reason that it doesn't produce successful results.

I've had the opportunity in the last year to speak to more parents than I care to remember that are the parents of dead children who presented to me their medical records, the results of the chemotherapy, the results of their radiation. And, it became very clear very early on that this was a very, very dangerous option that I did not want to take with Thomas.

In the case of chemotherapy, you have to understand that chemotherapy is a cytotoxic poison. And Thomas's oncologist made it very clear that we're going to basically stretch you to your limits because it is unnatural for a parent to voluntarily poison their child in the hopes that it will create a cure.

And we have had to go through this process with Thomas, and watch him endure the poisoning with great frustration and anxiety realizing that there was a non-toxic chemotherapy available that other children had been allowed to use and yet Thomas had been denied access to that.

That particular therapy has been with us for almost 30 years. And for the last 18 years, there had been efforts made to bring it to the forefront where it could be approved. But, again, it has been continually blocked.

And the point that I have been trying to make for the last year is if I have to choose between two unapproved therapies, which both are, why as a parent do I not have the right to go with the therapy that will do the least amount of damage to my son and then if it doesn't work step up to a more aggressive therapy?

I've spoken to parents of children that have been destroyed by radiation and chemo. And I'm thinking in particular of a young man from Houston, Texas, who today at 19 years old is deaf, dumb, blind, strapped to a wheelchair, has a arms length list of side effects. And yet, now that his parents need help in his maintenance and care are denied access to that because this child who is, picture him strapped to a chair, he can't see, speak or hear, can't move, can't function, is deemed a danger to the other patients, therefore he can't go into a group home. I'm still after almost a year trying to figure out how he becomes dangerous if he can't operate under his own power.

And, again, my frustration with the FDA is we've even applied for a Compassionate Use Exemption. Now, the doctor that we chose to go to in Houston has a protocol for meningeal blastoma. He has treated with the blessings of the FDA children with meningeal blastoma. But, I found it odd that as more and more children came through the process well that all of a sudden there was a new step put in place saying we can no longer allow you to treat these children until they first go through radiation and chemotherapy and fail and have reoccurred measurable tumor.

Now, if this is a pure glass of water, and my agency is in charge of making sure that water is available and is healthy to all, and I allow someone to come over here and pour a substance into it and say well, Mr. Brady, we want you to have clean water, but before you can drink this clean water, someone is going to be allowed to taint it. I think you'd be reluctant to drink the water.

And in Thomas's case, how can we know? If the FDA is truly and sincerely interested in progress and medical and scientific breakthroughs, how can we ever know if the treatment we wanted for him is successful if we taint the baseline with other therapies that take, to be honest, a lifetime to recover from?

REP. BILIRAKIS: I hope you will accept my answer, which is I don't have one. I can't explain it to you. But, I will assure you that this committee has not quit. The members on this committee have not quit to try to one, wade through the modernization of the Food and Drug Administration, which we completed in 1997, which people gave us no hope could be done and which passed with unanimous bipartisan support from this House and from the Senate and was signed into law by the president.

We're still waiting to see the full changes as they're implemented from that legislation. It's not always a fast process. But, much of that is by design in this system. I hope you understand that we will continue to strive to make sure that we have the answer for you and for the other parents that I know will be here in the future whether it's on this treatment or another treatment because we will never build a system that is perfect. But, we will never be content with what we have. We will always strive for something better.

The unfortunate thing is I've been notified that we have a series of votes. And because I know that that will throw further into the turmoil of member's schedules this afternoon because of the knowledge of their schedules, I'm going to take this opportunity to apologize to all of you because I know that we will have a lack of participation if we take 45 minutes off and try to reconvene.

I'm going to leave the record open for written questions to come to each of you from any members of the subcommittee and I hope you will respond to those written questions with answers for the purposes of the record. Let me once again on behalf of the chairman thank you for your participation in this hearing.

This hearing is now adjourned.

END

LOAD-DATE: September 15, 2000

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