[House Hearing, 106 Congress]
[From the U.S. Government Printing Office]



 
               SECURING THE HEALTH OF THE AMERICAN PEOPLE

=======================================================================

                                HEARING

                               before the

                            SUBCOMMITTEE ON
                         HEALTH AND ENVIRONMENT

                                 of the

                         COMMITTEE ON COMMERCE
                        HOUSE OF REPRESENTATIVES

                       ONE HUNDRED SIXTH CONGRESS

                             SECOND SESSION

                               __________

                           SEPTEMBER 13, 2000

                               __________

                           Serial No. 106-163

                               __________

            Printed for the use of the Committee on Commerce

                                


                      U.S. GOVERNMENT PRINTING OFFICE
 67-114CC                     WASHINGTON : 2000
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                         COMMITTEE ON COMMERCE

                     TOM BLILEY, Virginia, Chairman

W.J. ``BILLY'' TAUZIN, Louisiana     JOHN D. DINGELL, Michigan
MICHAEL G. OXLEY, Ohio               HENRY A. WAXMAN, California
MICHAEL BILIRAKIS, Florida           EDWARD J. MARKEY, Massachusetts
JOE BARTON, Texas                    RALPH M. HALL, Texas
FRED UPTON, Michigan                 RICK BOUCHER, Virginia
CLIFF STEARNS, Florida               EDOLPHUS TOWNS, New York
PAUL E. GILLMOR, Ohio                FRANK PALLONE, Jr., New Jersey
  Vice Chairman                      SHERROD BROWN, Ohio
JAMES C. GREENWOOD, Pennsylvania     BART GORDON, Tennessee
CHRISTOPHER COX, California          PETER DEUTSCH, Florida
NATHAN DEAL, Georgia                 BOBBY L. RUSH, Illinois
STEVE LARGENT, Oklahoma              ANNA G. ESHOO, California
RICHARD BURR, North Carolina         RON KLINK, Pennsylvania
BRIAN P. BILBRAY, California         BART STUPAK, Michigan
ED WHITFIELD, Kentucky               ELIOT L. ENGEL, New York
GREG GANSKE, Iowa                    TOM SAWYER, Ohio
CHARLIE NORWOOD, Georgia             ALBERT R. WYNN, Maryland
TOM A. COBURN, Oklahoma              GENE GREEN, Texas
RICK LAZIO, New York                 KAREN McCARTHY, Missouri
BARBARA CUBIN, Wyoming               TED STRICKLAND, Ohio
JAMES E. ROGAN, California           DIANA DeGETTE, Colorado
JOHN SHIMKUS, Illinois               THOMAS M. BARRETT, Wisconsin
HEATHER WILSON, New Mexico           BILL LUTHER, Minnesota
JOHN B. SHADEGG, Arizona             LOIS CAPPS, California
CHARLES W. ``CHIP'' PICKERING, 
Mississippi
VITO FOSSELLA, New York
ROY BLUNT, Missouri
ED BRYANT, Tennessee
ROBERT L. EHRLICH, Jr., Maryland

                   James E. Derderian, Chief of Staff
                   James D. Barnette, General Counsel
      Reid P.F. Stuntz, Minority Staff Director and Chief Counsel

                                 ______

                 Subcommittee on Health and Environment

                  MICHAEL BILIRAKIS, Florida, Chairman

FRED UPTON, Michigan                 SHERROD BROWN, Ohio
CLIFF STEARNS, Florida               HENRY A. WAXMAN, California
JAMES C. GREENWOOD, Pennsylvania     FRANK PALLONE, Jr., New Jersey
NATHAN DEAL, Georgia                 PETER DEUTSCH, Florida
RICHARD BURR, North Carolina         BART STUPAK, Michigan
BRIAN P. BILBRAY, California         GENE GREEN, Texas
ED WHITFIELD, Kentucky               TED STRICKLAND, Ohio
GREG GANSKE, Iowa                    DIANA DeGETTE, Colorado
CHARLIE NORWOOD, Georgia             THOMAS M. BARRETT, Wisconsin
TOM A. COBURN, Oklahoma              LOIS CAPPS, California
  Vice Chairman                      RALPH M. HALL, Texas
RICK LAZIO, New York                 EDOLPHUS TOWNS, New York
BARBARA CUBIN, Wyoming               ANNA G. ESHOO, California
JOHN B. SHADEGG, Arizona             JOHN D. DINGELL, Michigan,
CHARLES W. ``CHIP'' PICKERING,         (Ex Officio)
Mississippi
ED BRYANT, Tennessee
TOM BLILEY, Virginia,
  (Ex Officio)

                                  (ii)



                            C O N T E N T S

                               __________
                                                                   Page

Testimony of:
    Bennett, Catherine P., Chair, Board of Directors, Cancer 
      Research Foundation of America.............................   117
    Brady, Robert, Partner, Hogan & Hartson, on behalf of Biogen, 
      Inc........................................................   106
    Bryan, R. Nick, Professor and Chairman of Radiology, Hospital 
      of University of Pennsylvania..............................    87
    Burton, Hon. Dan, a Representative in Congress from the State 
      of Indiana.................................................   101
    Dunnick, N. Reed, Professor and Chair, Department of 
      Radiology, University of Michigan Health System............    77
    Fraser, Tomiko, National Spokesperson, Lupus Foundation of 
      America, Inc...............................................    84
    Gekas, Hon. George W., a Representative in Congress from the 
      State of Pennsylvania......................................    70
    Hillman, Bruce J., Professor and Chair, Department of 
      Radiology, University of Virginia..........................    80
    Lang, Thomas A., Senior Vice President, Strategic Product 
      Development, Serono Laboratories, Inc......................   112
    Meek, Hon. Carrie P., a Representative in Congress from the 
      State of Florida...........................................    72
    Meyers, Abbey, President, National Organization for Rare 
      Disorders..................................................   110
    Navarro, James, father of Thomas Navarro.....................   103
    Wirth, Dyann, Professor, Department of Immunology and 
      Infectious Diseases, Harvard School of Public Health.......    73
Material submitted for the record by:
    Brady, Robert, Partner, Hogan & Hartson, Counsel to Biogen, 
      Inc., letter dated September 21, 2000, enclosing material 
      for the record.............................................   138
    Bryan, R. Nick, Professor and Chairman of Radiology, Hospital 
      of University of Pennsylvania, letter dated November 20, 
      2000, enclosing response for the record....................   136
    Department of Health and Human Services, additional testimony 
      for the record.............................................   125
    Fraser, Tomiko, National Spokesperson, Lupus Foundation of 
      America, Inc., responses for the record....................   127
    Hillman, Bruce J., Professor and Chair, Department of 
      Radiology, University of Virginia, responses for the record   134
    Meyers, Abbey, President, National Organization for Rare 
      Disorders, responses for the record........................   131
    Navarro, James, responses for the record.....................   128

                                 (iii)



               SECURING THE HEALTH OF THE AMERICAN PEOPLE

                              ----------                              


                     WEDNESDAY, SEPTEMBER 13, 2000

                  House of Representatives,
                             Committee on Commerce,
                    Subcommittee on Health and Environment,
                                                    Washington, DC.
    The subcommittee met, pursuant to notice, at 10:10 a.m., in 
room 2123, Rayburn House Office Building, Hon. Michael 
Bilirakis (chairman) presiding.
    Members present: Representatives Bilirakis, Upton, Stearns, 
Burr, Ganske, Cubin, Bryant, Brown, Waxman, Pallone, Stupak, 
Green, Barrett and Capps.
    Staff present: Marc Wheat, majority counsel; Nandan 
Kenkeremath, majority counsel; Kristi Gillis, legislative 
clerk; and John Ford, minority counsel.
    Mr. Bilirakis. The hearing will come to order. My thanks to 
all of the witnesses who have taken the time to testify before 
this subcommittee. This hearing will address, as you know, 
several pieces of legislation designed to improve the quality 
of health care.
    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, 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 my Representative Carrie Meek, H.R. 762, the lupus 
research and care amendments of 1999, which expands immediate 
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 and their 
families. 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 from my area of 
Florida.
    I discussed this legislation at the full committee markup 
of the minority health disparities bill, and 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 for 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 nonclinical 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 would 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 Rights Act. This bill is named 
after his son, who was 4 years old when he was diagnosed with a 
form of cancer known as medulloblastoma. After researching 
their options, the family decided that the best course of 
action was through a nontoxic 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 the 
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 Pryce on the importance of researching 
childhood cancer. I think all of us remember that 
Representative Pryce lost their 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 biologicals 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 and 
ranking member to 3 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 the subcommittee will also give brief 
introductory remarks regarding their legislation and introduce 
their witnesses. And 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 yield to the ranking member, Mr. Brown.
    Mr. Brown. Thank you, Mr. Chairman. I would 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 is likely that today's 
hearing will not produce definitive answers. The issues 
involved are simply too complex and the implications of any 
actions we take too significant. However the questions these 
bills seek to answer, the concerns they seek to address are 
important, and it is valuable for the subcommittee to learn 
about them.
    I am also glad we will have an opportunity to review 
legislation focussing 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 
tuberculosis in the United States now in drug-resistant strains 
that are difficult to treat is a grim reminder that when a 
disease affects other nations, it is bound to affect us. 
Tuberculosis last year killed more people than in 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 are 
the wealthiest Nation in the world, we are the most influential 
force in the world. Our action sets a 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 U.S. standards literally 
can save millions of lives, prevent millions of children from 
being orphaned, prevent the social, economic and political 
turmoil these killer diseases too often engender. It is an 
opportunity and it is 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 extend a 
special welcome to Dr. Dunnick from the University of Michigan 
who is joining us to discuss 1794 at Mrs. Eshoo's request. 
Adding an institute to NIH is a major step, but Mrs. 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, more therapeutic treatments that greatly improve 
the likelihood that they will live longer and healthier lives. 
Additionally, when treatment is initiated at the early stage of 
a disease, doctors are able to rely on less expensive treatment 
options that reduce overall health care costs.
    I am glad we are taking time to access the benefit of 
establishing an institute dedicated to equipment and techniques 
that are indispensable to modern health care. I thank the 
chairman.
    Mr. Bilirakis. I thank the gentleman.
    Mr. Upton.
    Mr. Upton. Thank you, Mr. Chairman. I ask unanimous consent 
to put my full statement in the record.
    Mr. Bilirakis. Without objection, the opening statement of 
all members of the subcommittee will be made a part of the 
record.
    Mr. Upton. I would just like to say one thing verbally 
here. I am very glad that we are having these hearings, and I 
am 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 that 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 am delighted to be a cosponsor, and I yield back.
    Mr. Bilirakis. Mr. Waxman for an opening statement.
    Mr. Waxman. 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 is 
an excellent bill and 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 am 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 have been very gratified by the success of 
the Orphan Drug Act in stimulating the development of new 
treatments for rare diseases. I am pleased that we will have 
Abbey Meyers testify again before our subcommittee on this, and 
that she is 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, ``clinically superior,'' to an existing orphan 
drug; that is, drugs which are safer, more effective or provide 
a major contribution to patient care. It has 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 
with great interest to the FDA's public clarification of its 
policy toward 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 the question of how high a bar 
clinical superiority should be. There are some multimillion-
dollar orphan drugs, drugs for which 7 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 will have an opportunity early 
next year to examine these issues in greater detail than will 
be possible today.
    Finally, Mr. Chairman, I would like to submit for the 
record a series of articles and scientific reviews relating to 
the alternative cancer treatment offered by Dr. Stanislav 
Burzynski, the intended beneficiary of H.R. 3677, the Thomas 
Navarro Patient Rights Act.
    Mr. Bilirakis. Without objection, that will be the case.
    [The information referred to follows:]

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    Mr. Waxman. As ranking member of the Committee on 
Government Reform, I have attended that committee's many 
hearings to defend and endorse alternative medicine and dietary 
supplements. But I am pleased that this subcommittee, which has 
jurisdiction over these issues, has finally turned 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. So 
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 complementary 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 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.
    Mr. Bilirakis. I thank the gentleman.
    Mr. Burr to give an opening statement.
    Mr. Burr. 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 is 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 is 
unfortunate today as we meet this morning that Ms. Eshoo is in 
California under the weather, but I am 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 computer 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 have made tremendous 
progress, despite a lack of a focused effort, on our ability to 
detect at the early possible point. What we have heard, Mr. 
Chairman, from people around the country is that we can do 
better. If you give us the type of focus that it takes in 
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 putting new resources into. 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, is 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 
health care 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.
    Mr. Bilirakis. I thank the gentleman.
    Mr. Pallone for an opening statement, going on basis of 
seniority.
    Mr. Pallone. Thank you, Mr. Chairman. I will limit my 
comments to the two bills which have I cosponsored, H.R. 3677, 
the Thomas Navarro FDA Patient Rights Act, and the H.R. 1795, 
National Institute of Biomedical Imaging and Engineering 
Establishment Act.
    The first of these, the Thomas Navarro FDA Patient 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 
effects of a brain tumor. As any parent with a child in 
Thomas's situation would, the Navarros researched the treatment 
options available and found the treatment of radiation and 
chemotherapy would have extremely debilitating side effects 
which they did not want to risk. Rather, the Navarros preferred 
to have Thomas treated with antineoplaston 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 
antineoplaston treatment for Thomas in the setting of a 
clinical trial is an appropriate path to follow. They 
researched the issue, and they understand the issue, and I do 
not believe in light of the circumstances surrounding the case 
that the Navarros should be denied their right to choose.
    The issue is important not just for Thomas, t for other 
patients in similar circumstances. We shouldn't be restricting 
the rational choices and measured choices of individuals who 
choose to pursue alternative medical treatments 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 have discussed the 
importance of this legislation on a number of occasions over 
the last 2 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.
    And I want to thank the chairman for having the hearing on 
these two bills and the other that we have today. I yield back.
    Mr. Bilirakis. Mr. Stupak for an opening statement. I am 
going to try to continue on rather than break, as long as 
someone gets back in time to spell me.
    Mr. Stupak. Mr. Chairman, I thank you for holding this 
hearing, and I thank the witnesses for being here. I look 
forward to their testimony.
    I am 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.
    Mr. Bilirakis. I thank the gentleman so very much.
    Mrs. Capps for an opening statement.
    Mrs. Capps. Mr. Chairman, I thank you for holding this 
hearing, and I want to thank--extend a welcome to our 
witnesses. Today we are 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 am 
offering, enjoying bipartisan support; currently has 280 
cosponsors, many of whom serve on this committee. I am so glad 
that we are 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 am 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 grants for the delivery of essential 
services to individuals with lupus and their families. As a 
nurse, I know what an insidious disease this is. For many 
people lupus is a mild disease affecting only a few organs; for 
others can cause serious, even life-threatening problems.
    My district is home to the Scleroderma Research Foundation. 
Scleroderma is a condition that is closely linked to lupus, and 
I have seen the work of women like Sharon Monsky in Santa 
Barbara in my district, who has fought so long to raise 
awareness of scleroderma 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 this 
topic of bioengineering. These disciplines have made such 
contributions to the improvement, as our colleague has said, 
and have no research home in the current structure of NIH. And 
I support this legislation, again commending our colleagues for 
their leadership in this area.
    Finally, I know my statement submitted will be longer than 
this, but I want to say one word about my good friend and 
colleague Deborah Pryce and offer my strongest words of support 
for her resolution. Her resolution focuses on the importance of 
promoting awareness and expanding research on childhood cancer. 
This is Childhood Cancer Month, September. It is the second 
leading cause of death in children past infancy. Many childhood 
cancers can be cured, but, sadly, dozens still cannot. And I 
applaud Congresswoman Pryce's efforts in this difficult area 
and pledge any support that I can give to help get this 
legislation passed into law.
    Mr. Chairman, this is an important topic. Thank you for 
holding this hearing, and I yield back.
    Mr. Bilirakis. I thank the gentlewoman.
    [Additional statements submitted for the record follow:]
Prepared Statement of Hon. Barbara Cubin, a Representative in Congress 
                       from the State of Wyoming
    Our individual health care needs have changed over the years, but 
the bottom line is we all need care--and we want it to be the best.
    That's all the reason we need to make sure we keep moving forward 
with innovative technologies, new drug therapies, and better standards 
of care.
    We must continue to protect and nurture the different fields of 
research and development in health care because we live in an 
unpredictable and sometimes hostile bacterial environment that is 
constantly reinventing itself.
    That means that clinical trials will take on an even greater role 
in the future as we try to develop new drugs to counteract new 
diseases.
    Giving patients the proper access to these trials is therefore 
paramount to our success.
    Of course, these trials are not possible without the new drug 
therapies and biologicals that sustain them so supporting drug research 
and innovation is critical.
    By the same token, we're making technological advances that we 
never thought possible--such as MRIs, Cat Scans, laser devices, 
telemedicine, and the like.
    We must continue to push the envelope in the field of health care 
technology because it has already proven to enhance the quality of 
patient care.
    There are, no doubt, countless things we have yet to discover about 
biomedical imaging and we should continue to explore its capabilities.
    Today we're discussing specific legislative proposals that are 
designed to secure the future health of this country. I look forward to 
the discussion and stand ready to work with all of you.
                                 ______
                                 
 Prepared Statement of Hon. Tom Bliley, Chairman, Committee on Commerce
    Thank you Mr. Chairman.
    I am pleased that you are having this hearing today which continues 
the extraordinary public health work that Members of the Commerce 
Committee have done over the past six years.
    I would like to take a moment of personal privilege to state how 
proud the Members of the Committee should be. In the prior two 
Congresses, this Committee has empowered states and localities to meet 
the health care and nutritional needs of low-income residents; and 
provided relief to those hardest hit by the AIDS epidemic.
    The Health Insurance Portability and Accountability Act allows 
working Americans to change jobs without risking the loss of their 
health care insurance due to a preexisting condition. The law also 
attacked health care fraud and eliminated tax code discrimination 
against millions of small businesses and the self-employed. It also 
provided tax relief for long-term health care needs and terminally ill 
patients and their families.
    The Food Quality Protection Act and the Safe Drinking Water Act 
Amendments of 1996 modernized programs and enhanced Americans' access 
to safe, abundant, and affordable food and water.
    We established Medicare+Choice and the State Children's Health 
Insurance Program. Among other bills, we enacted the Food and Drug 
Administration Modernization Act; the Birth Defects Prevention Act; the 
National Bone Marrow Registry Reauthorization Act; the Mammography 
Quality Standards Reauthorization Act; the Women's Health Research and 
Prevention Amendments.
    This Congress, we have passed prescription drug legislation and HMO 
reform. We reauthorized funding to help those suffering from AIDS and 
moved forward on a major children's health initiative. We also have 
moved on the Breast and Cervical Cancer Treatment Act, the Health Care 
Fairness Act, the Cardiac Arrest Survival Act, the Developmental 
Disabilities Amendments, The Drug Addiction Treatment Act, the Date-
Rape Prevention Drug Act, the Health Research and Quality Act, the Pain 
Relief Promotion Act, the Organ Procurement and Transplantation Network 
Amendments, and the Nursing Home Resident Protection Amendments. We 
have also provided new options under Social Security for the disabled.
    It is a spectacular record.
    Now for the work before us today. I look forward to hearing from 
today's witnesses and to making even further progress on public health 
with the Members of this Committee. Today we will discuss global health 
concerns, new medical technologies, drug approval systems, expanding 
access to clinical trials, Lupus, and childhood cancers. Some of these 
efforts may go forward this Congress and others may not. I only ask the 
Members of the Committee to maintain their workman-like commitment to 
bipartisan improvements to our public health programs.
                                 ______
                                 
  Prepared Statement of Hon. Gene Green, a Representative in Congress 
                        from the State of Texas
    Mr. Chairman, thank you for calling this hearing today. I 
appreciate the opportunity to examine legislation that has been 
introduced to improve America's health care programs.
    There are several bills being discussed today, two of which I am a 
cosponsor of, the Lupus Research and Care Amendments of 1999 and the 
National Institute of Biomedical Imaging and Engineering Establishment 
Act.
    H.R. 762 by Rep. Carrie Meek is a bill that would expand and 
intensify research at the NIH to diagnose, treat, and eventually cure 
lupus. It also increases the funding for lupus research and education 
and establishes a grant program to expand availability of lupus 
services.
    I commend my colleague for introducing this bill and look forward 
to the testimony on this piece of legislation.
    I am also a cosponsor of H.R. 1795 by Rep. Richard Burr. This 
legislation would create an independent institute to support basic 
research in medical imaging and bioengineering. These disciplines have 
made great contributions to the improvement of medical care in the past 
two decades, yet they have no research home in the current NIH 
structure. The creation of the National Institute of Biomedical Imaging 
and Engineering would create a research environment in which new 
imaging and bioengineering technologies techniques and devices can be 
developed for clinical use more rapidly than under the present system. 
This would allow for continued rapid progress in fields such as 
genetics and molecular biology, which utilize advanced imaging 
techniques.
    I represent the medical center area and I can tell you that this 
piece of legislation would help advance the technology that can be used 
for clinical use more rapidly than under the present system.
    I look forward from hearing from our witnesses today.
                                 ______
                                 
Prepared Statement of Hon. Anna G. Eshoo, a Representative in Congress 
                      from the State of California
    Thank you, Mr. Chairman, for holding today's hearing. With six 
bills before us today, we have a lot on our plates. However, I'd like 
to focus my comments on the bill that my colleague Mr. Burr and I have 
introduced, H.R. 1795 to establish a National Institute of Biomedical 
Imaging and Engineering at the National Institutes of Health (NIH).
    Mr. Chairman, dramatic advances in imaging and bioengineering have 
revolutionized medical practice in recent years. Development of new, 
noninvasive imaging techniques, such as Magnetic Resonance Imaging 
(MRI) and Computed Tomography (CT), has allowed for earlier detection 
and diagnosis of disease, dramatically improving the quality of 
healthcare. But, the next generation of breakthroughs will be longer in 
coming, or in some fields, may not come at all unless we modernize the 
structure at NIH.
    Today, these disciplines, which have made unmatched contributions 
to the improvement of medical care in the past two decades, have no 
research home in the current NIH structure. Research at the current 
institutes at NIH is based on molecular biology, which is fundamentally 
different from research in imaging and bioengineering. If we are to 
ensure the continued development of new techniques and technologies, 
these disciplines require an identity and research home at the NIH that 
is independent of the existing institute structure.
    H.R. 1795 would fill a critical void at the NIH by creating an 
independent institute to support basic research in medical imaging and 
bioengineering. It would create a research environment in which new 
imaging and bioengineering technologies, techniques, and devices can be 
developed for clinical use much more rapidly than under the present 
system. By doing so, H.R. 1795 replaces disorganization with 
efficiency, effective management, accountability, and an improved 
scientific focus.
    At a time when Congress has committed to doubling the NIH budget, 
we must ensure that research dollars are expended more effectively and 
efficiently and that the fields of medical science that have 
contributed the most to the detection, diagnosis, and treatment of 
disease in recent years receive appropriate emphasis. Establishment of 
a National Institute of Biomedical Imaging and Engineering at the NIH 
would accelerate the development of new technologies, improve 
coordination and efficiency throughout the Federal government, reduce 
duplication, lay the foundation for a new medical information age, and 
provide a structure to train the young researchers who will make the 
pathbreaking discoveries of the next century.
    I'm proud to join Representative Burr in sponsoring H.R. 1795 and 
I'm pleased that the Committee has finally turned its attention to it. 
I encourage my colleagues' support at tomorrow's full committee markup 
and I look forward to passage by the full House.
                                 ______
                                 
    Prepared Statement of Hon. John D. Dingell, a Representative in 
                  Congress from the State of Michigan
    Mr. Chairman, we have six bills and resolutions before us today, so 
I will be brief.
    H.R. 2399 would establish a national commission to examine how the 
United States can most effectively use our scientific and technical 
expertise to tackle disease on a global basis. This is an ambitious 
undertaking. A panoply of public and private sector organizations spend 
considerable time and resources on projects that collectively span 
virtually all global health issues. Nevertheless, the commission could 
generate useful information, which in turn could optimize the use of 
scarce resources and which could lead to improvements in global health.
    H.R. 762, the Lupus Research and Care Amendments, is an excellent 
piece of legislation that has been developed by my good friend and 
colleague, Representative Carrie Meek. As we will learn, lupus is a 
debilitating and sometimes fatal autoimmune disease that 
disproportionately afflicts women, particularly women of color.
    H.Res. 576 by Representative Pryce is another measure worthy of our 
support. Children are not simply ``little adults.'' I note that our 
colleague, Representative Forbes, has a similar bill, H. Con. Res. 115.
    H.R. 1795 would create the Institute of Biomedical Engineering at 
the National Institutes of Health (NIH). I note the fine work that our 
colleagues, Representatives Eshoo and Burr, have done on this bill and 
believe that it deserves our careful consideration, although NIH has 
some concerns with the bill.
    H.R. 3677 causes me great concern, and I can not support it as 
drafted. This bill would dramatically alter the basic role of the Food 
and Drug Administration (FDA) in the supervision of human subject 
protection in the development of experimental drugs. FDA must play a 
fundamental role in protecting the public in both the development and 
approval of drugs. The FDA Modernization Act, which has provisions on 
this matter, is less than three years old, but we should examine the 
investigational drug process to ensure that it is serving the public in 
the best possible manner. The moral, ethical, and safety issues 
presented in the informed consent process are particularly important in 
the case of experimental drugs and their use on persons with serious 
and life threatening diseases.
    Finally, I oppose H.R. 4242, the Orphan Drug Innovation Act. We 
will hear testimony opposing this bill from the National Organization 
for Rare Disorders (NORD). NORD represents approximately 25 million 
Americans with more than 6,000 ``orphan'' diseases. The orphan drug law 
has been a success. Anyone wishing to enact significant changes in this 
important health statute bears a heavy burden. I do not believe the 
proponents of this bill have met that test.
    Thank you, Mr. Chairman.

    Mr. Bilirakis. Mr. Gekas and Mrs. Meek are here to testify 
on behalf of their legislation briefly and also 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, but 
we do want to make this vote. Would like to return? I will be 
here.
    Mrs. Meek. With your pleasure and Mr. Gekas's, I would 
rather go forward, if I may.
    Mr. Bilirakis. What would you prefer to do?
    Mr. Gekas. I do not mind proceeding.
    Mr. Bilirakis. Well, then, proceed. The only thing is I am 
liable to cut you off.
    You are recognized, the gentleman from Pennsylvania.

     STATEMENT OF HON. GEORGE W. GEKAS, A REPRESENTATIVE IN 
            CONGRESS FROM THE STATE OF PENNSYLVANIA

    Mr. Gekas. 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 gole 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, 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 interests, 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 is what the purpose of it is. That is 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 Dyann Wirth, of the Department of 
Immunology and Infectious Diseases, Harvard School of Public 
Health, and has a slew of publications 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, and I am sure she will impress you. 
Unfortunately she has impressed me so much that I am leaving 
right now to go vote, but I know what she will testify and 
commend her to you.
    [The prepared statement of Hon. George W. Gekas follows:]
    Prepared Statement of Hon. George W. Gekas, a Representative in 
                Congress from the State of Pennsylvania
    Mr. Chairman, and Members of the Committee, I would like to thank 
you for giving me the opportunity to testify at this hearing on such an 
important matter.
    The latter part of the twentieth century saw the dismantling of the 
U.S.S.R., the fall of the Berlin Wall, and a worldwide blossoming of 
democracy. To many this seemed an impossibility at the time. Most of 
the world rightly places the credit for these events in the hands of 
our United States. Our leaders and our government put forth endless 
efforts to achieve these ends successfully. While these accomplishments 
are extraordinary, it is time to turn our attention to another 
seemingly impossible goal.
    With the dawning of the new millennium, the time has come to focus 
our national energy and resources on efforts to eradicate all disease 
across the globe. This is indeed an awesome goal that may not be 
attainable in itself, but its purpose could lead to new treatments of 
diseases, and new approaches to controlling them.
    Beyond the humanitarian reasons for promoting this idea, the U.S. 
has enlightened domestic goals as well. Eradicating global disease 
would protect American citizens, improve the quality of life worldwide, 
enhance our economy, and advance American interests across the globe. 
Achieving this goal would impact every aspect of our society, not just 
the field of health care.
    In order to reach the objective of eradicating disease globally, I 
have introduced H.R. 2399, the Advancement of Global Health Act, which 
would establish a commission, the National Commission for the New 
National Goal: The Advancement of Global Health. The commission's task 
would be to recommend a strategy for the global eradication of disease. 
The United States has the resources, through the National Institutes of 
Health (NIH) and the National Science Foundation, to take the lead in 
expanding health research information globally, especially with the 
recent explosion of Internet technology. Additionally, the commission 
would assist the Center for Vaccine Development at the NIH to achieve 
global control over infectious disease. A one-time $1 million 
allocation would be granted to the commission to coordinate and attract 
other sources of funding both domestically and abroad for the purpose 
of achieving these objectives.
    The fifteen-member commission would be charged with coordinating 
governmental, academic, and public and private health care entities for 
the purpose of global disease eradication. The commission would then be 
required to submit a final report to Congress within one year of its 
establishment, with its recommendations for legislative, 
administrative, and other appropriate actions. The wide-sweeping goals 
of this legislation could encompass ideas like the simple act of 
teaching a youngster how to properly wash his or her hands, reaching 
across to the more complex, such as medical advances learned from space 
exploration.
    It gives me great pleasure to have Dr. Dyann F. Wirth here today to 
further discuss the idea of eradicating global disease. Dr. Wirth 
earned a Ph.D. in Cell Biology and Biochemistry from the Massachusetts 
Institute of Technology. She currently serves as a professor at the 
Harvard School of Public Health in its Department of Immunology and 
Infectious Diseases. Dr. Wirth is a widely published author in numerous 
esteemed scientific and medical journals. She also worked for ten years 
as an NIH Study Section Member on Tropical Medicine and Parasitology, 
which is Dr. Wirth's particular area of expertise. Again, I am glad to 
have her with us today.
    The intelligence and imagination of American researchers and 
scientists did not fail us in our attempts to preserve and promote 
freedom and democracy across the globe. It is now time for us to 
unleash these same great minds towards the goal of improving the 
quality of life for everyone. We must summon all of the resources at 
our command in our efforts to eradicate disease from the face of the 
Earth.
    Again, Mr. Chairman, I thank you for this hearing on this very 
important matter that can change the lives of countless individuals.

    Mr. Upton [presiding]. I thank the gentleman from 
Pennsylvania. Now I recognize Mrs. Meek, and I didn't know that 
you were here when I gave my opening statement, but I commended 
you for your good bill, and I am delighted to be a cosponsor 
and delighted to recognize you now.

 STATEMENT OF HON. CARRIE P. MEEK, A REPRESENTATIVE IN CONGRESS 
                   FROM THE STATE OF FLORIDA

    Mrs. Meek. Thank you, Mr. Chairman. I am 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 is time that it be passed, Mr. 
Chairman, and of course I am 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 stuck 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 is add additional services for lupus victims.
    But I am here this morning because I am pleased and 
privileged and blessed to have a young lady who is sitting at 
the table, the testimony table, to testify for lupus, a very 
beautiful, lovely lady, Tomiko Fraser. She is the spokesperson 
for the Lupus Foundation, and I do not have to take your time 
to tell you all of the demerits, I would say, or all of the 
real terrible effects of lupus. I lost a sister to it. I have 
lost so many friends to lupus. It is a young woman's disease, 
and as a result of that, I think as this committee you have 243 
cosponsors behind this bill.
    And I want to have my statement placed in the record, Mr. 
Chairman, and with your permission.
    Mr. Upton. Without objection, your entire statement will be 
put in the record, and, again, we thank you for your leadership 
on this very important issue that touches so many American 
families in every State.
    [The prepared statement of Hon. Carrie P. Meek follows:]
Prepared Statement of Hon. Carrie P. Meek, a Representative in Congress 
                       from the State of Florida
    Good morning, Chairman Bilirakis, Ranking Member Brown and my other 
colleagues. It's a pleasure to be with you. Thank you for holding this 
hearing and inviting me to appear before your Subcommittee to discuss 
my lupus bill, H.R. 762. My bill would authorize additional funding for 
lupus research and treatment programs. Providing such additional help 
for lupus victims is not a Democratic issue or a Republican issue. It's 
an American issue. My bill has broad bipartisan support (243 cosponsors 
from both sides of the aisle).
    I won't take the Subcommittee's time to lay out all of the details 
of H.R. 762. In a nutshell, this bill would authorize spending of up to 
$75 million for lupus research and $75 million for lupus care and 
treatment programs.
    I want to recognize and introduce to the Subcommittee the beautiful 
young women sitting next to me, Tomiko Fraser. Tomiko is a nationally 
recognized spokesmodel and actress. She is the first African-American 
women to be a spokesmodel for Maybelline. Tomiko's sister has lupus. 
Tomiko has seen firsthand the devastation that lupus has caused for her 
sister and she speaks eloquently about what it is like for her sister 
to live with lupus. She makes a powerful case for why we need the 
additional funding for lupus research and treatment that my lupus bill 
authorizes.
    Tomiko, I salute you and your sister for your courage and thank you 
for your commitment to obtaining additional relief for lupus victims 
and their families.
    As many of you know, I know firsthand the heartache that lupus 
causes, I lost a sister to lupus and have seen many others suffer from 
this disease. We all know the debilitating pain and fatigue that lupus 
often causes, pain and fatigue that makes it difficult for persons with 
lupus to maintain employment and lead normal lives. We also know the 
profound impact that this disease has on family members of those with 
lupus.
    Mr. Chairman, lupus victims and their families need more help and 
they need it now. Congress and the President can provide it by passing 
and signing H.R. 762. We can do it, and we must do it this year, but, 
to make this happen, I need your help to persuade the leadership to 
bring this bill to the floor immediately. I won't be satisfied, and 
none of us should be, until we get this bill to the House floor, pass 
it overwhelmingly, pass it in the Senate and have it signed by the 
President.
    I urge the Subcommittee and the full Commerce Committee to mark 
this bill up immediately and implore the Leadership to bring this bill 
to the floor on the Suspension Calendar at once so that it can pass the 
House this Session. Thank you, Mr. Chairman.

    Mrs. Meek. Thank you, Mr. Chairman. And I would love to 
hear Tomiko, but I must go and vote.
    Mr. Upton. Well, you can come back.
    Mrs. Meek. All right.
    Mr. Upton. At this point, as no members are coming back 
from the vote that is currently ongoing, I would 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 
the University of Pennsylvania; Dr. Reed Dunnick, professor and 
Chair of the Department of Radiology, 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 is, of course, at the table already, and 
obviously the national spokesperson for the National Lupus 
Association; and Dr. Dyann Wirth, professor at 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, and 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 am 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 would like to keep this to 5 minutes each or less. 
And Dr. Wirth, we will start with you. Thank you for being here 
with us this morning.

STATEMENTS OF DYANN WIRTH, PROFESSOR, DEPARTMENT OF IMMUNOLOGY 
 AND INFECTIOUS DISEASES, HARVARD SCHOOL OF PUBLIC HEALTH; N. 
  REED DUNNICK, PROFESSOR AND CHAIR, DEPARTMENT OF RADIOLOGY, 
    UNIVERSITY OF MICHIGAN HEALTH SYSTEM; BRUCE J. HILLMAN, 
  PROFESSOR AND CHAIR, DEPARTMENT OF RADIOLOGY, UNIVERSITY OF 
     VIRGINIA; TOMIKO FRASER, NATIONAL SPOKESPERSON, LUPUS 
 FOUNDATION OF AMERICA, INC.; AND R. NICK BRYAN, PROFESSOR AND 
 CHAIRMAN OF RADIOLOGY, HOSPITAL OF UNIVERSITY OF PENNSYLVANIA

    Ms. Wirth. Mr. Chairman and members of the subcommittee, 
thank you for the opportunity to testify today. My name, as you 
already know, is Dyann Wirth. I am a professor at Harvard 
University School of Public Health and the Department of 
Immunology and Infectious Diseases, and I am 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 sciences 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's bill which we have just heard about on the advancement 
of global health, H.R. 2399. This bill, if enacted 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 of 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 is 35 percent of the deaths in the world today. That 
means during the duration of this hearing, 1,500 people will 
die from infectious disease. 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. The January 2000 unclassified report from the 
CIA's National Intelligence Council entitled ``The Global 
Infectious Disease Threat 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 U.S. and global security over 
the next two decades. These diseases will endanger U.S. 
citizens at home and abroad, threaten U.S. 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 is now more important than ever. I will 
talk just briefly about malaria because that is where my 
interest and passion is. Among adults living in high-
transmission areas, malaria is considered a chronic disease. A 
single bout can incapacitate someone for weeks, and despite 
massive efforts to eradicate this disease in the 1950's, 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 
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 is important to 
seize the opportunities of these scientific advances to 
accelerate and defeat malaria worldwide.
    But equally important as progress in research and public 
support and awareness of these major health threats, in order 
to conquer malaria, AIDS, malaria, 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.
    [The prepared statement of Dyann Wirth follows:]
Prepared Statement of Dyann Wirth, Professor, Harvard University School 
                            of Public Health
    Mr. Chairman and members of the Subcommittee, thank you for the 
opportunity to testify today. My name is Dyann Wirth. I am a Professor 
at the Harvard University School of Public Health Department of 
Immunology and Infectious Disease. I am 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 (JSC) is a coalition 
of four life-science societies representing more than 25,000 
researchers in the fields of genetics, cell biology, biophysics, 
biochemistry and molecular biology. The JSC was formed in late 1980s to 
bring scientists together to advocate for federal funding for basic 
biomedical research. Eric Lander of MIT chairs JSC, and among its 20 
board members are Nobelists J. Michael Bishop, Paul Berg and Harold 
Varmus.
    Founders of the JSC realized that there was a great need for the 
United States to invest in biomedical research and called for the 
doubling of the NIH budget. Since that time, Mr. Chairman, the 
Congress' visionary support of the NIH has lead us to the dawn of a new 
age in science. I have no doubt that the coming decade will be 
remembered for major discoveries enabled by the mapping of the human 
genome which will lead to the prevention and cure of diseases. The JSC 
also worked with Congressman Gekas to introduce the Congressional 
Biomedical Research Caucus to the Halls of Congress. Today, the 
Biomedical Research Caucus has been called by Chairman John Porter and 
former Speaker New Gingrich among others, ``the most influential Caucus 
in Congress.''
    I am here today to express the support of the Joint Steering 
Committee for Congressman Gekas' bill the National Commission for the 
New National Goal: The Advancement of Global Health Act, H.R. 2399. 
this bill, if enacted into law would create a Presidential/
Congressional commission to investigate how we as a Nation can most 
effectively seize the myriad scientific opportunities presented by 
modern advances in genomic 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 third Millenium it is within the grasp of human 
capability 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: scientific potential is there; it 
requires only political will to make it reality.
    My particular experience is malaria, but as devastating as malaria 
is, it is just one of 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 means that over the duration of this hearing 1,500 
people will die from an infectious disease--over half of them children 
under five.
    According to the WHO the seven infectious diseases that caused the 
highest number of deaths in 1998 are AIDS, TB, malaria, hepatitis B and 
hepatitis C, diarrheal diseases, and measles. Of these, TB and 
hepatitis are renewed threats because they are becoming increasingly 
resistant drug resistant. But, malaria alone is estimated to cause up 
to 500 million clinical cases and 2.7 million deaths each year, 
representing 4 percent to 5 percent of all fatalities globally. 
Tragically, every 30 seconds a child somewhere in the world dies of 
malaria. As you know, most of these deaths occur in developing 
countries where extreme poverty and lack of access to basic health 
care, adequate sanitary and essential drugs can seal the fate of 
children before they're born. However, the enormous volume of travel 
and trade today have made, infectious diseases blind to national 
borders.
    A January, 2000, unclassified report from the CIA's National 
Intelligence Council, entitled ``The Global Infectious Disease Threat 
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 assesses 
claims, and I concur, that ``New and reemerging infectious diseases 
will pose a rising global health threat and will complicate US and 
global security over the next 20 years. 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 in which the United States has significant 
interests.''
    Research into the prevention, treatment and control of tropical and 
infectious disease are now more important than ever to the US and the 
world. I will address malaria as an example because I know more about 
malaria than about other global health threats.
    Among adults living in areas of high transmission, malaria is best 
thought of as a chronic, debilitating illness that robs its victims of 
years of productivity. A single mosquito bite can transmit one of the 
four parasites that cause malaria, setting in motion bouts of fever, 
chills, and nausea that can recur for weeks. According to a 1993 World 
Bank Report, malaria represents a global public health burden second 
only to tuberculosis among infectious diseases of the 2-3 million 
children who die of malaria each year, most of them live in Africa, 
continent already overwhelmed by poverty and internal conflict. Those 
who survive can suffer chronic anemia and/or immune suppression that 
leave victims vulnerable to other fetal diseases.
    Despite massive efforts to eradicate in the 1950s, today than at 
any other time in history. More than 500 million people are infected 
with malaria worldwide; one-fourth of the world's population is at risk 
for infection. Better implementation of currently available control 
measures, including the use of insecticide, and better and more 
rational use of existing drugs, should be the goal in the short term; 
in the long-term, new research interventions are desperately needed. 
Cutting-edge technology has led to the development of new paradigms--
the genome of the most important malaria parasites is being sequenced, 
DNA vaccines are being developed and tests of methods to prevent 
transmission by the mosquito are being explored. We must seize the 
opportunity presented by these scientific adversities to accelerate the 
defeat of malaria worldwide. But equally important as progress in 
research is public support and awareness of this major health threat. 
In order to conquer malaria, we need a global strategy that includes 
American leadership and resources to invest into continued research 
into prevention and treatment. I must also point out the great need for 
action with regard to HIV/AIDS in Africa. Africa remains the epicenter 
of the pandemic, bearing the largest disease burden, with 70 percent of 
people living with AIDS worldwide, 83 percent of global AIDS deaths, 
and 95 percent of the world's AIDS orphans. AIDS is reversing decades 
of progress from important public health efforts, lowering life 
expectancy, and significantly affecting daily life form millions of 
Africans. This situation cries out for leadership.
    The JSC supports efforts to encourage research and development on 
vaccines to combat malaria, tuberculosis, AIDS and other infectious 
diseases causing and to ensure that existing vaccines are accessible to 
populations in developing countries most impacted by these diseases. 
These efforts will require partnerships among federal agencies, 
industry, non-profit foundations and other NGOs, the World Bank, and 
international organizations to combat the scourge of infectious 
diseases. This Commission could vastly accelerate the pace of these 
efforts.
    Specific mechanisms might include, enhanced R&D tax credits and new 
tax credits for sales of vaccines, contributions to international 
organizations such as the Global Alliance for Vaccines and 
Immunizations (GAVI) for the purchase and distribution of vaccines in 
developing countries, and measures that will improve the public health 
infrastructure in developing countries in order to expand 
immunizations, prevent and treat infectious diseases, and build 
effective delivery systems for basic health services.
    As we begin the 21st century, we are blessed with unimaginable 
opportunities to build on breakthrough research to control and prevent 
global infectious disease. This is not just altruism to reduce the 
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 
disease is a win-win for the country: by helping others across the 
world we are also launching the best defense to protect the health of 
our Nation's people. We hope you will seriously consider passage of 
H.R. 2399.
    Thank you for the opportunity to present the views of the Joint 
Steering Committee for Public Policy. I would be happy to answer your 
questions.

    Mr. Upton. Thank you, Dr. Wirth.
    Dr. Dunnick, welcome to Washington's version of the ``Big 
House.''

                  STATEMENT OF N. REED DUNNICK

    Mr. 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. 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 4 years as a staff 
radiologist in diagnostic radiology at the National Institutes 
of Health.
    The 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 applications 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 health care. 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 it is 
needed.
    The imaging community has not proposed the establishment of 
a new institute lightly. We recognize and agree that the bar 
for structural change should be set high. For that reason we 
looked to the National Academy of Science's Institute of 
Medicine in their 1984 report titled Responding to Health Care 
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 area 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 amount of moneys carried out by 
radiology departments throughout the country account for less 
than 1 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.
    [The prepared statement of N. Reed Dunnick follows:]
Prepared Statement of N. Reed Dunnick, Professor and Chair, Department 
             of Radiology, University of Michigan Hospitals
    Good morning. Thank you for this opportunity to share my support 
for H.R. 1795 with you. My name is Reed Dunnick, and I chair the 
Radiology Department at the University of Michigan. Prior to coming to 
Michigan in 1992, I served on the faculties at Stanford and Duke. In 
addition, I spent four years as a staff radiologist in the Diagnostic 
Radiology Department at the National Institutes of Health.
    Like everyone in my discipline and throughout the imaging sciences, 
I was extremely pleased that the Congress 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 applications 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 
health care. The logical next step suggested by this Congressional 
finding is the creation of a National Institute of Biomedical Imaging 
and Bioengineering as proposed in H.R. 1795. Like my colleagues Dr. 
Hillman and Dr. Bryan, I have been involved in imaging research at the 
NIH for many years. 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.
    During that period, numerous plans have been tried. In 1982, for 
example, most extramural research in imaging was transferred from the 
National Institute of General Medical Sciences to the National Cancer 
Institute with the understanding that NCI would support imaging 
research beyond that related to cancer. However, the NCI has focused 
almost exclusively on cancer imaging.
    On the intramural side, the Laboratory of Diagnostic Radiology 
Research (LDRR) was transferred from the Office of the Director of the 
NIH to the Clinical Center a couple of years ago. This move was an 
improvement but far from a solution because the Clinical Center lacks 
the research mandate and resources to allow LDRR to achieve its full 
promise.
    Despite good will on both sides, these initiatives did not solve 
the problems faced by either extramural or intramural imaging 
investigators. Only after such repeated efforts failed did the imaging 
community conclude that the NIH structure is not compatible with an 
effective imaging research program and that creation of a new institute 
is justified and necessary. 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 at the NIH should be set high. For that reason, we looked to 
neutral, expert guidance, which we found in the National Academy of 
Sciences' Institute of Medicine. In a 1984 report titled Responding to 
Health Needs and Scientific Opportunity: The Organizational Structure 
of the National Institutes of Health, issued in response to a 
Congressional directive, the IOM concluded that new institutes should 
be created only in unique circumstances when the following five 
criteria are met:

(1) the activity is compatible with the mission of the NIH;
(2) it can be demonstrated that the research area in question (defined 
        either as a disease or health problem or as a biomedical or 
        behavioral process related to a health problem) is not 
        receiving adequate attention;
(3) there are reasonable prospects for scientific growth in the 
        research area;
(4) there are reasonable prospects of sufficient funding for the new 
        organization;
(5) 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 
clearly that this field is compatible with the mission of the NIH and 
that there are reasonable, even extraordinary, prospects for scientific 
growth. The NCI has increased its level of support for the Biomedical 
Imaging Program in recent years. Even with this increased commitment of 
resources, however, extramural support for imaging research carried out 
by Radiology Departments continues to account for substantially less 
than one percent of the NIH budget--a level that is inadequate for an 
``Extraordinary Opportunity for Investment.'' 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.
    Two years ago, again in response to a Congressional mandate, the 
IOM issued another report that addressed the question of structural 
change. In its 1998 report titled Scientific Opportunities and Public 
Needs: Improving Priority Setting and Public Input at NIH, the IOM 
recommended that ``The U.S. Congress should use its authority to 
mandate specific research programs, establish levels of funding for 
them, and implement new organizational entities only when other 
approaches have proven inadequate.'' The imaging community agrees with 
this recommended limitation on Congressional action and believes firmly 
that the proposed National Institute of Biomedical Imaging and 
Bioengineering is consistent with it.
    In addition, and just as important, the proposed institute is also 
completely consistent with the NIH's own rationale for the elevation of 
the National Center for Human Genome Research to institute status in 
1997. According to the statement issued by the NIH to announce the 
creation of the National Human Genome Research Institute, ``As an 
institute, NHGRI can more appropriately interact with other Federal 
agencies, and develop collaborations with industry, academia, and 
international organizations.''
    The same is true for imaging. The proposed institute would 
coordinate imaging research throughout the federal government, a 
mission that cannot be accomplished successfully by a lower level 
office or organization lacking sufficient institutional stature and 
authority to deal effectively with cabinet-level departments. 
Similarly, industry-government collaborations are essential to the 
development of new imaging technologies. The proposed institute would 
assist industry in setting research priorities and assessing potential 
fields for development. The National Electrical Manufacturers 
Association, which represents the companies that produce imaging 
devices, supports this proposal.
    This matter of coordinating imaging outside the NIH is important. 
Other federal agencies such as NSF, NASA, the intelligence agencies, 
and the Departments of Defense, Commerce, and Energy all support 
imaging research. There is little or no coordination of these efforts. 
In one key area, telemedicine, the General Accounting Office found a 
couple of years ago that more than 35 separate federal organizations in 
nine different departments have sponsored telemedicine initiatives. 
Despite a major federal investment of $646 million over three years, 
the GAO found that ``no formal mechanism or overall strategy exists to 
ensure that telemedicine development is fully coordinated among federal 
agencies to serve a common purpose.'' According to the GAO, the Joint 
Working Group on Telemedicine, created in response to a directive from 
the Vice President, encountered serious difficulties even in developing 
a federal inventory of telemedicine programs.
    This lack of coordination not only encourages duplication and 
inefficiency, it impedes the transfer of imaging technologies from 
research projects to the public health care sector. The proposed 
institute could do much to ensure that federal research dollars are 
expended more efficiently and productively.
    The National Institute of Biomedical Imaging and Bioengineering 
would also provide a focused effort to meet the medical needs of the 
information age. It has been estimated that computerization 
requirements in the coming decade will double just to keep pace with 
developments in all segments of society. The largest component of that 
need will be in the area of medical imaging information. In addition to 
the enormous amount of imaging-related, unprocessed information 
generated, there will be a need to process and analyze the huge date 
sets produced by medical imaging to improve the medical value of those 
data to the referring physicians who will access the network. An 
imaging institute would support research focused on the acquisition, 
transmission, processing, and optimal display of images.
    Mr. Chairman, breakthroughs in medical imaging have revolutionized 
the way in which physicians detect, diagnose, and treat disease in the 
past two decades. Imaging holds the promise of further advances that 
will move us into an era of non-invasive 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 National 
Institute of Biomedical Imaging and Engineering Establishment Act.

    Mr. Upton. Thank you very much.
    Dr. Hillman.

                  STATEMENT OF BRUCE J. HILLMAN

    Mr. Hillman. Good morning. I am Dr. Bruce Hillman, Chair of 
the Department of Radiology at the University of Virginia, and 
I am also a Chancellor of the American College of Radiology. 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 likely 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 to disease. Imaging 
technologies will precisely determine what faction 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, MRI, and image-guided interventional methods have 
most frequently occurred outside the U.S. 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 connected 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.
    Among 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 costs the American public receives for its 
expenditure on cancer imaging.
    The results of major definitive ACRIN trials of such 
technologies as digital mammography, a screen for breast 
cancer, and CAT scan 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 2 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 entity devoted to biomedical imaging, 
bioengineering, and related fields will necessarily be of the 
size that will 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.
    [The prepared statement of Bruce J. Hillman follows:]
Prepared Statement of Bruce J. Hillman, Chair, Department of Radiology, 
               University of Virginia School of Medicine
    Good morning. I am Dr. Bruce Hillman, Chair of the Department of 
Radiology at the University of Virginia School of Medicine and a 
Chancellor of the American College of Radiology. I greatly appreciate 
your invitation so that I may testify in support of H.R. 1795, the 
National Institute of Biomedical Imaging and Engineering Establishment 
Act.
    During the past thirty years, no specialty of medicine has advanced 
technologically so much as medical imaging. And no specialty of 
medicine has so drastically and positively changed the way we detect, 
diagnose, stage, and treat disease. The introduction during that time 
period of such computer-based technologies as CAT scanning and MRI, and 
the use of imaging to guide a host of interventional procedures has 
revolutionized medicine. As a result, the preferred diagnosis and 
treatment methods for a broad range of diseases--cancer, heart disease, 
orthopedic injuries, and infectious diseases, to name just a few--has 
become less invasive, less expensive, and less risky for patients.
    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 
likely to be much greater than currently. Virtually everyone agrees 
that medical imaging must be a critical element in this new paradigm.
    Medical imaging is the ``non-invasive biopsy'', the method of 
disease quantitation, the guidance for new treatments still to be 
developed that will form the underpinnings of molecular medicine. Under 
this scenario, new medical imaging technologies will detect alterations 
in the genetic or molecular makeup of cells that have the potential to 
progress to disease. We then will employ imaging technologies to 
precisely determine what fraction of cells are so affected. Finally, 
medical imaging technologies will be integrated with new therapeutic 
methods to either guide or monitor treatment, so that we can much more 
precisely than ever before ensure that only diseased cells are treated 
while preserving normal tissue.
    The basic knowledge exists to begin to implement this vision. 
However, for this optimistic and exciting prophecy to come to fruition, 
we will need to invest in the development and assessment of new imaging 
technologies. We are ill-equipped to do so under the current NIH 
organizational structure that focuses the work of existing institutes 
on specific organ systems and diseases.
    Imaging technologies, with few exceptions, are multi-potential. 
They are applicable to many organ systems and diseases. Their value in 
detection, diagnosis, staging, and treatment of disease cuts across 
traditional institute lines. The leadership of existing institutes do 
not generally have training or expertise in imaging. The requests for 
applications and the public announcements that are the principal 
instruments used by institutes to guide research in their respective 
fields view imaging more as a tool to address disease-specific 
questions than as a focus for research in its own right. The critical 
research training of new young imaging investigators, when it is 
supported at all, is forced into unnatural pathways that address 
imaging technology research obliquely rather than head-on.
    Thus, 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 and basic 
research into new technologies that have emerged in recent times--such 
as CAT scanning, MRI, and image-guided interventional methods--have 
most frequently occurred outside the U.S. where the logistics and 
funding of basic research into medical imaging can be handled in a more 
straightforward 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;
 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;
 provide a home for 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 the vision of molecular medicine that I have detailed 
        earlier;
 through relationships the new institute will develop with 
        regulatory agencies and industry, facilitate technology 
        transfer, allowing important innovations to more rapidly be 
        employed in medical care;
 and very significantly, 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.
    Among my other responsibilities, I am the chair of the American 
College of Radiology Imaging Network, or ACRIN. ACRIN is a National 
Cancer Institute-funded cooperative group that conducts clinical trials 
of medical imaging technologies at community practices and academic 
health centers across the country. The overriding goal of ACRIN is, 
through clinical trials, to gather information that will extend and 
improve the quality of the 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 costs the American 
public receives for its expenditures on medical imaging. The results of 
major, definitive, ACRIN trials of such technologies as digital 
mammography for screening for breast cancer, CAT scanning for the 
detection of early lung cancer, and the use of PET scanning for 
patients' response to chemotherapy 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 the National Heart, Lung, and Blood 
Institute, or the National Institute for Neural Diseases and Stroke--
institutes whose purview includes organ systems and diseases where 
imaging plays a large and critical role--nor, for that matter, 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 unification of imaging technology assessment activities under 
the aegis of the proposed Institute obviates these concerns. It ensures 
that assessment activities critical to both the health and financial 
well-being of the American public will be orderly, strategic, coherent, 
and efficient and that they will best advise us on how to most wisely 
expend our resources on imaging technologies as they are employed in 
medical practice.
    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. 
An entity devoted to biomedical imaging, bioengineering, and related 
fields would 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 those I have detailed in this testimony, I 
hope that you will vote favorably on HR 1795 and pass it on to the full 
House of Representatives for its consideration.

    Mr. Bilirakis. Thank you, Dr. Hillman.
    Ms. Tomiko Fraser is the national spokesperson for the 
Lupus Foundation of America.
    Thank you very much for being here today.

                   STATEMENT OF TOMIKO FRASER

    Ms. 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, 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 forty-three Members of the U.S. 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 nonprofit 
health care 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 benefits 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 6- and 
$10,000 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 revenue due 
to lost wages when individuals with lupus are unable to work.
    The Lupus Research and Care Amendments Act of 1999 is a 
bipartisan effort to address an urgent national health care 
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 lead to a cure 
for lupus. By accelerating medical research for lupus now, 
Congress will reduce future health care 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 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 effects 
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 times more 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 the 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 3 to 5 years, visiting 5 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 effects of the disease.
    It is time for action. A majority of Members of the U.S. 
House of Representatives, a total of 243, are cosponsor 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.
    [The prepared statement of Tomiko Fraser follows:]
   Prepared Statement of Tomiko Fraser, National Spokesperson, Lupus 
                         Foundation of America
    Good Morning Mr. Chairman, and Members of the Subcommittee. I 
appear before you today representing the Lupus Foundation of America on 
behalf of 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, 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 a 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 & Care 
Amendments Act of 1999. Congresswoman Carrie Meek, who lost a sister to 
lupus, introduced this legislation. 243 members of the U.S. 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 
health care facilities with a pool of funds so they could offer life-
saving medical care to poor or uninsured people with lupus.
    This grant program will help local communities hardest hit by 
lupus, especially in medically under-served 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 benefits 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 revenue due to lost wages when individuals with lupus 
are unable to work.
    The Lupus Research and Care Amendments Act of 1999 is a bipartisan 
effort to address an urgent national health care 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 health care 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 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 effects 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 times more 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% of lupus cases begin in childhood. Unfortunately, 
lupus is more severe in children. Nearly 70% of children with lupus 
have kidney disease, as opposed to 30% of adults who develop lupus. 
Whereas half of those with adult-onset lupus have organ-threatening 
disease, nearly 80% of those with childhood-onset lupus go on to 
develop organ-threatening conditions.
    Lupus strikes women in their child-bearing 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 already have 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 effects of this disease.
    It's 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. Now I will be happy to answer 
your questions.

    Mr. Bilirakis. Thank you very much, Ms. Fraser.
    Dr. Bryan.

                   STATEMENT OF R. NICK BRYAN

    Mr. 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 would like to thank 
the sponsors of the bill, Mr. Burr and Mrs. Eshoo, for their 
leadership and efforts on this issue.
    You have 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 NIH.
    Prior to coming to Penn, I served for 2 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 Gallen, 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 in 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, for instance, the National Cancer Institute has 
authorized significant expansion of the extramural biomedical 
imaging program.
    The NIH Biomedical Engineering Consortium, known as BECON, 
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 Bioimaging, Bioengineering and 
Bioimformatics in the Office of the Director of the NIH. The 
new office is to provide focus for and facilitate work in our 
fields.
    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 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 focus is 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 existing disease and organ system institutes to 
divert resources from their primary 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 organizations 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 
is 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 technologists, 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 as the 
development of better systems for delivery of health care. This 
institute would be good for patients, physicians, and the NIH 
itself.
    I urge the subcommittee to approve this bill. I would be 
pleased to answer any questions.
    [The prepared statement of R. Nick Bryan follows:]
Prepared Statement of R. Nick Bryan, Chairman, Department of Radiology, 
               University of Pennsylvania Health Systems
    Good morning. My name is Nick Bryan. I have been a radiologist 
specializing in neuroradiology for more than 25 years. I currently 
serve as Chairman of the Department of Radiology and Eugene P. 
Pendergrass Professor of Radiology at the University of Pennsylvania. 
Prior to coming to Penn, I served for two years as Director of 
Diagnostic Radiology and Associate Director, Radiologic and Imaging 
Sciences Program, at the Warren G. Magnuson Clinical Center at the 
National Institutes of Health.
    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.
    I appreciate this opportunity to share some of my experiences as an 
imaging researcher and NIH staff member with you and to express my 
support for H.R. 1795, the National Institute of Biomedical Imaging and 
Engineering Establishment Act. I would like to thank you, Mr. Chairman, 
and the Committee leadership for holding this hearing and the sponsors 
of the bill, Representatives Burr and Eshoo, for their leadership and 
efforts on this issue.
    It is important to note that all of radiology and imaging supports 
this initiative. More than 40 separate professional organizations 
representing physicians, radiologic technologists, bioengineers, and 
imaging scientists have joined coalitions that support H.R. 1795. The 
total individual membership of these organizations is well over 
100,000.
    This is perhaps the most exciting time in the history of medical 
imaging and, indeed, all of medicine. Breakthroughs in imaging have 
allowed physicians to eliminate much surgery, including virtually all 
exploratory surgery, and to diagnose disease at earlier and earlier 
stages of development, when treatment is most effective. Because of 
advances in imaging, patients receive more effective treatment, avoid 
painful, expensive, and often dangerous surgical procedures, and live 
longer.
    The National Institutes of Health is the premier medical research 
institution in the world and has been at the center of pathbreaking 
research in most areas of medicine. In imaging, however, the NIH is 
not--and under its present structure cannot be--the catalyst of imaging 
innovation. The various institutes are focused on specific disease 
processes or organ systems, but imaging cuts across those lines and is 
broadly applicable to virtually all diseases and organ systems. 
Consequently, imaging is used as a tool in all the institutes, but 
there is no home at the NIH for the basic research that is essential to 
develop new imaging techniques and technologies for the 21st century.
    The basic science of imaging and bioengineering, it must be 
remembered, is fundamentally different from that of the existing 
institutes at the NIH. Imaging is based on mathematics and physics, not 
the biological sciences that underly most of the research in the 
current institutes. Imaging and bioengineering are unique scientific 
fields at the NIH and are also critical to future advances in the 
delivery of high quality health care.
    While at the NIH, I directed intramural research efforts in imaging 
in the Clinical Center. During my tenure, we were able to consolidate 
several disparate imaging departments into the unified Imaging Sciences 
Program (ISP), 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, the ISP 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 imaging research proposals was ultimately dependent on the 
ability of ISP researchers 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. Even when imaging researchers are 
successful, which sometimes requires artificially tailoring proposals 
to create the appearance of disease- or organ-specific research, the 
institutes are likely to assume practical control of projects and, in 
all probability, recast the research to fit their own missions.
    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 for imaging. 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 has taken steps to make 
imaging research a more visible part of its portfolio. The National 
Cancer Institute, for example, has designated imaging as one of only a 
few ``Extraordinary Opportunities for Investment'' and has authorized 
significant expansion of the extramural Biomedical Imaging Program. The 
Biomedical Imaging Program at the NCI, under the extremely able 
leadership of Dr. Dan Sullivan, has benefited from growing staff 
resources and new research initiatives.
    In addition, the Bioengineering Consortium, known as BECON, 
sponsored a conference in June 1999 titled ``Biomedical Imaging 
Symposium: Visualizing the Future of Biology and Medicine.'' 
Participants produced an ambitious research agenda for imaging science 
that calls for focused efforts in a number of fields:

--multidisciplinary research;
--imaging technology, probes, and contrast agents;
--education and training;
--clinical trials and informatics
--greater cooperation among the NIH, the Food and Drug Administration, 
        the Health Care Financing Administration, and private industry 
        to improve the speed with which new imaging technologies, 
        probes, and contrast agents are transferred to clinical 
        practice.
    The 1999 BECON symposium was actually the second NIH-sponsored 
conference in recent years devoted to imaging research. In 1994, the 
NIH brought together more than 40 top researchers from inside and 
outside government at a Conference on Developing a Long-term Plan for 
Imaging Research. Conference participants developed a set of 
recommended research goals in 33 separate areas of basic science, basic 
and applied technology, and organ-based clinical research.
    Finally, 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, NIH. According to the 
Vacancy Announcement seeking candidates to direct the OBBB, the 
Director of the new Office ``will provide a focus for stimulating and 
coordinating the development of biomedical engineering, bioimaging and 
bioinformatics activities among the 25 Institutes and Centers (ICs) at 
the NIH; and will facilitate the overall planning, development, and 
implementation of NIH biomedical engineering, bioimaging and 
bioinformatics research programs and activities.''
    Unfortunately, all of these initiatives suffer from one of two 
fatal flaws. First, the NCI efforts apply real resources to imaging, 
but all of the research is on cancer imaging. Cancer imaging clearly 
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 focus is on 
cancer or any other individual disease.
    ACRIN, the cooperative clinical trials group chaired by Dr. 
Hillman, offers a clear example of the shortcomings of this approach. 
ACRIN represents a significant and wise investment, but this 
application of resources could produce so much more if imaging 
technologies beyond cancer were included.
    The ACRIN approach, valuable as it is, actually shortchanges cancer 
research as well as the broader field of imaging. Evaluating the use of 
existing techniques and technologies for the diagnosis and treatment of 
breast, prostate, and other cancers will produce important knowledge 
that will result in incremental improvements in patient care. But the 
real breakthroughs that will produce quantum leaps forward are likely 
to occur through the development of wholly new imaging modalities that 
do not result from disease-specific research.
    The second group of NIH initiatives--the 1994 conference, the BECON 
symposium, and the creation of the OBBB--represents the second fatal 
flaw. These initiatives 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. 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 be, as I was as Director of the 
Imaging Sciences Program, dependent on the goodwill and interest of the 
other institutes. Again, these are institutes that do not have imaging 
as primary missions and which are faced continually with competing 
claims for scarce resources from within their primary research 
constituencies.
    The disciplines represented in the existing institutes use imaging, 
but they cannot accommodate the basic science of imaging itself. It is 
unrealistic, and perhaps even inappropriate, to expect the existing 
disease and organ system institutes to divert resources from their 
primary missions in order to support basic research to advance the 
science of imaging.
    Without grant-making and decision-making authority, these NIH 
efforts to improve coordination can be marginally successful at best. 
No one would suggest that the search for better treatments for cancer, 
diabetes, or other diseases should be undertaken by organizations that 
lack the capability to make research grants--or that progress in these 
vital areas should be dependent on the ability of researchers to 
convince other institutes to divert a portion of their resources away 
from their chief responsibilities. Yet that is precisely the approach 
the NIH has taken to imaging. In consequence, while the NIH might 
consider the recommendations from the 1994 and 1999 imaging conferences 
to be priorities, there is not much evidence that significant action 
has been taken on this research agenda. The present organization of the 
NIH makes such action unlikely.
    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, only institutes possess those attributes.
    Institutes, for example, can issue Requests for Applications (RFAs) 
and Program Announcements (PAs) to initiate research projects and 
direct resources toward investigations on specific subjects that offer 
particular opportunities for scientific advances. Such institute-
initiated research projects represent a substantial portion of the 
institutes' extramural research portfolios. Even smaller and mid-sized 
institutes can exert considerable influence on the direction of 
research. A recent analysis showed that the National Institute on Aging 
(NIA), with only the 10th largest budget among the institutes and major 
centers at the NIH, issued seven RFAs covering a wide variety of topics 
with a total price tag of more than $16 million in Fiscal Year 1999. In 
the same year, the NIA collaborated on 16 additional RFAs in which 
other institutes were the primary sponsors. Without an institute, 
imaging lacks this fundamental capability to guide and support the 
research in this field.
    Research training is another key issue. Training grants ensure the 
continuity of a pool of trained researchers in the institutes' fields 
of research. Under the current structure, training opportunities in 
imaging are generally limited to grants that focus on the use of 
imaging in connection with specific diseases and organ systems rather 
than on training imaging scientists to conduct the basic research that 
will produce new modalities. An analysis of NIH data on T32 grants, the 
most common NIH training awards, found only three imaging awards of the 
148 active T32 grants in the National Heart, Lung, and Blood Institute. 
Moreover, the National Institute of Neurological Disorders and Stroke 
(NINDS), which is the institute most closely related to my work in 
neuroradiology, had no active imaging grants among its 44 T32 awards.
    The National Institute of Biomedical Imaging and Bioengineering 
would help to ensure that the brightest young radiologists and imaging 
scientists have opportunities to obtain research training. Such 
opportunities are largely non-existent under the present system.
    For all of these reasons, I 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 molecular biology as well as to the development of 
a better system for the delivery of health care. The proposed would be 
good for patients, physicians, and the NIH itself. I urge the 
Subcommittee to approve H.R. 1795, and I would be pleased to answer 
questions.

    Mr. Bilirakis. Thank you so much, Dr. Bryan.
    I will 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 under way by the NIH Lupus and Minority Studies, or 
LUMINA. We believe lupus has a genetic basis, and it appears 
that the gene suspected of causing lupus may be more prevalent 
among women of color.
    Mr. 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 is correct.
    Mr. Bilirakis. We do not know of any other reason, though, 
other than the fact?
    Ms. Fraser. Not yet, we are still checking.
    Mr. Bilirakis. In terms of the administration's support or 
nonsupport of the legislation, my understanding--and I may be 
wrong, and if I am, I wish to be corrected, but I think it is 
significant--that they have problems with title 2. The 
administration has problems with title 2. 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 is 
okay with the chairman.
    Mr. Bilirakis. Okay. Yes, we would 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, and that would be very significant.
    Dr. Wirth, can you tell us the organizations, or at least 
some of the organizations, you are familiar with that are 
working on the issue of global disease eradication?
    Ms. 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 is 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.
    Mr. Bilirakis. I know--I am a Rotarian. Do not attend very 
many meetings these days for business reasons, but in any case 
they have worked on eradicating polio around the world, as you 
know.
    Ms. Wirth. That is correct.
    Mr. Bilirakis. And that is working and has worked very 
well; has not it?
    Ms. Wirth. Uh-huh. 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 is implementing a very important 
step, implementing the sort of research in 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.
    Mr. 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?
    Ms. Wirth. I think so. And 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.
    Mr. Bilirakis. Do we have now--and if not, is that the 
reason maybe for the national commission--do we have the proper 
coordination? For instance, I don't know, how has Rotary gone 
about it all to know exactly where to go? Have they 
coordinated?
    Ms. Wirth. That is right. I think one of the things that 
the National Commission could do is to have a focus point for 
this kind of work in the United States. I think in the case of 
Rotary and other nongovernmental 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.
    Mr. Bilirakis. Well, let me ask you this. If this 
commission is formed, the administration feels that CDC and 
NVPO, the National Vaccine Program Office, should be included 
in the composition of the commission's membership. And also 
that the FDA, as the agency overseeing vaccine safety and 
approval of new vaccines, should also have a role in it if the 
bill is enacted. Your opinion?
    Ms. 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 are dealing 
very specifically with vaccine issues. As you know, there are 
broader issues of implementation including drug development and 
development of other controlled, measured environmental and 
insecticides at some point.
    Mr. Bilirakis. 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.
    Mr. Brown. Thank you, Mr. Chairman.
    Dr. Wirth, I am 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 on breakthrough research to control and 
prevent world disease.
    Dr. Gro Brundtland of the World Health Organization--I 
would argue that the World Health Organization has done 
phenomenal work over the last 20 years. Nonetheless, she said 
talking about tuberculosis--and I know your expertise is more 
malaria, and I want to get to that in a second, but she said it 
is not medical eradication--dealing with tuberculosis is not a 
medical problem, it is a political problem. But--and I think 
back just less than a year ago in December 1999, the Government 
of India, working with nongovernment 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 1 day, which tells me 
that Mr. Gekas's bill goes in the right direction in this 
country. Our country should show a great deal more leadership 
in dealing with issues that surely we can.
    Tuberculosis and malaria both do not get the attention from 
the big drug manufacturers in their research arms that they 
should. The drug companies seem much too interested, in my 
mind, in ``me too'' drugs, in drugs that are more cure for 
baldness than for tuberculosis, malaria, lupus, a whole host of 
diseases where there simply is not the moneys, potential profit 
available. There is not 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.
    Shift gears to Walter--Walter Reed has done especially and 
the Defense Department has done especially good research in 
malaria. We underfund Walter Reed. We fund organizations like 
NIH, a wonderful government agency. We want to double its 
budget in the next 5 years, yet we do not fund CDC very well, 
which its budget is about one-sixth of the NIH. And we do not 
fund the Walter Reed research arm of the Defense Department 
particularly well, putting it mildly.
    Is the only real hope for a malaria vaccine, TB vaccine, 
better treatment of those diseases--TB, as you know, you need 
to take a pill every day for 6 months, which in countries with 
military occupation, in places like Chiapas in southern Mexico, 
people are afraid to go by the military checkpoint to get their 
TB pills every day. And even though we can cure it, it is 
difficult because of that. Is the only hope for a TB vaccine or 
malaria vaccine better medicines to treat those two diseases? 
Must there be government funding because the drug companies 
won't do it? And what do we need to do? Talk about malaria. 
What do we do with Walter Reed in the Defense Department, and 
what do we do with NIH and CDC on malaria? Even though it is 
not a great issue for me to go back to my district in Ohio and 
say, I am working on malaria and TB, it does not 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?
    Ms. Wirth. I share your respect for Walter Reed and the 
work they have done over the last several years in developing 
antimalarial 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 profits like drugs for 
diseases in this country. The drug companies will not develop 
them. And I think we are going to have to--it is going to 
require governmental intervention and governmental funding.
    I recommend that Walter Reed certainly receive funding, 
that the NIH receive funding for basic research and for 
translational research, something I think that the NIH has 
become very interested 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 tool we have. We still 
face the challenges, 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 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.
    Mr. Brown. Thank you.
    One last brief question. Should Walter Reed and the CDC be 
included in this bill, both?
    Ms. Wirth. Yes. I think that is an excellent idea.
    Mr. Brown. Okay. Thanks.
    Mr. Bilirakis. Mr. Burr to inquire.
    Mr. Burr. Thank you, Mr. Chairman.
    Dr. Wirth, I don't know that you misspoke, I don't think 
you need to apologize. Sherrod and I participated in the same 
hearing in International Relations on the threat of global 
infections, a debate over whether it was a public health issue 
or whether it is a national security issue, and I think we can 
all agree it is probably (d) All of the above.
    Clearly the World Health Organization and other 
international organizations that are targeted toward health 
issues have been effective on some things. Clearly there are 
other things where health care professionals have pointed out 
the deficiencies that exist; and with deficiencies in place, we 
cannot be assured of successful immunization or successful 
eradication of diseases that ultimately we see as a threat, not 
only here but spreading throughout the globe.
    And your reference to AIDS in Africa is a very good one. It 
is important that we recognize that that spread, as it begins 
to happen in Asia, is of a magnitude that we have never seen 
before, potentially; and that every effort that we can make, 
not relying on any one entity, is in fact the policy that we 
should adopt.
    And I appreciate your 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 turn to you, Dr. Bryan.
    Who benefits? Who benefits from the creation of an 
institute for biomedical imaging?
    Mr. Bryan. Well, the people who benefit the most will be 
the patients.
    Mr. Burr. Isn't that who it is all supposed to be about?
    Mr. Bryan. That is exactly right.
    Mr. Burr. For any person who is on the fence about this 
issue as to whether we should create this, if they stopped for 
a minute and thought, who is this about; if their answer was, 
the patients, then the answer is, vote for this bill.
    Mr. Bryan. I would agree.
    Mr. Burr. Is it safe to say--and I open this up to anyone--
as we identify breakthroughs in technology, that we can also 
expect health care costs to possibly decline because if we 
detect earlier, our treatments may be less intensive as it 
relates to a period of time; and if you looked 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?
    Mr. Hillman. That has been the history of the development 
of imaging technologies: that, in fact, they do detect disease 
earlier, they do replace more morbidity-inducing, more illness-
inducing technologies. And over time I believe that imaging 
technologies have been cost-saving and also improve patient 
outcomes.
    Mr. Dunnick. I would like to make two comments in response 
to that. First, when the DRGs were established a number of 
years ago, my assumption was that the medical centers would try 
to reduce the number of ancillary tests 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 will 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 use 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 in what we 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 will make 
dramatic changes in decreasing the cost of health care.
    Mr. Burr. Can any of you address a specific disease where, 
say, in the last decade the imaging improvements have changed 
in----
    Mr. Dunnick. Absolutely. Trauma would be the first response 
to that. The patient comes into the emergency room, and in fact 
it does not even have to be a traumatic injury. It can be a 
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 noninvasively. In the trauma setting 
specifically, we can now identify not only the problem, but in 
many cases, quantify it, which enables more conservative 
therapy.
    So it has resulted in a dramatic decrease in the number of 
patients that have to go to the operating room.
    Mr.  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 
that not only in imaging, but in other areas of medical 
breakthroughs, we are 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 further development of new innovations, new 
treatments, new imaging that might detect disease earlier?
    Mr. Bilirakis. Important question, but brief answers, 
please.
    Mr. Hillman. Yes, there are two things that this new 
institute will be able to do better than 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 has been 
problematic under the current NIH structure.
    The other is that we will develop relationships directly 
with the regulatory agency and payers to quickly move these 
technologies into practice.
    Mr. Burr. I thank the witnesses. I thank the chairman. I 
yield back.
    Mr. Bilirakis. Dr. Ganske.
    Mr. Ganske. Thank you, Mr. Chairman, for having this 
hearing. I think there are several bills that we are talking 
about that have merit, and while they may not be the biggest 
health care 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 is one of these bills 
that I think relates to that and that is the Orphan Drug Act 
which created incentives for drug companies to develop 
therapies for rare diseases by awarding a period of 7 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 orphan 
drug's 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 and physician choice of therapy.
    Now, after approval an original orphan drug, whenever a 
subsequent orphan drug with a clinically superior improvement 
has also been approved and awarded exclusivity, FDA totally 
restarts the 7-year exclusivity clock for the drug as a whole, 
and in this way the improved drug shields the original drug 
from competition, even when--after the original drug's 
exclusivity period is over. In these instances, companies that 
have developed new competing versions of the same drug to treat 
the disease in anticipation of the expiration of the original 
7-year exclusivity are unfairly denied access to the market for 
an additional 7-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 surely keep prices higher. That is why I and 
others have been fighting an extension of--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.
    Mr. Bilirakis. And I thank the gentleman. If he would yield 
to me maybe 30 seconds of his time before me yields----
    Mr. Ganske. I will.
    Mr. Bilirakis. You know we have a dilemma here in terms of, 
let's say, NIH funding. Let's just talk NIH funding, and the 
dilemma is, should we in this so-called ivory tower determine 
the amount of money for research that ought to go to specific 
diseases? I mean, the experience that we have had on this 
committee has been just amazing, the number of diseases that I 
am sure most of us, if any of us, although some are medical 
doctors, were not even aware of.
    Just some terribly sad stories that we are going to hear, 
and we are going to hear certainly even on the next panel, and 
the plea for more funding for Parkinson's, more funding for 
lupus. We can just go on and on. Mohammad Ali was here pleading 
for more funding for Parkinson's.
    So the thought has been that we just do not know enough of 
actually what is taking place up there in terms of research and 
how close they may be to a breakthrough and that sort of thing; 
and should we be telling them, rather than just giving the 
money or doubling the money as Mr. Brown has indicated?
    Any opinions in that regard, because I consider that quite 
a dilemma. We have come to maybe a conclusion.
    I have not talked with Mr. Brown on his feelings on that 
subject. I don't remember that we have in any case. But any 
feelings in that regard? Just very quickly, please.
    Mr. Bryan. Mr. Chairman, I think you do have indeed a major 
challenge, and that is the responsibility you all accept as our 
public representatives. I think that your directive is to 
provide broad strokes of direction to institutes such as the 
NIH. And I do think you have to leave some of the details to 
them.
    Mr. Bilirakis. Yes, sir?
    Mr. Dunnick. I think in terms of H.R. 1795, what we are 
really talking about is not necessarily more funding, but 
reorganization to establish focus and priority setting.
    Mr. Bilirakis. Which is basically what Ms. Fraser has 
testified to and what Mrs. 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 did not really 
know a lot about it before my sister was infected with the 
disease. And as I learned more about it and I learned that 
there are so many Americans, 1.4 million infected 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.
    Mr. Bilirakis. I just wanted to sort of share with you the 
dilemma that we have and the difficulty sometimes. Did you want 
to add something quickly?
    Ms. Wirth. Yes. Very quickly, I come from the 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 is very important that NIH 
be given as much free rein to follow the advances as they come.
    But I also think it is important that the interests of 
individuals, who perhaps cannot 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.
    So I think it is very important that that be heard at NIH 
at least in an advisory and perhaps not absolutely directive 
way.
    Mr. 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?
    Mr. Bryant. Mr. Chairman, thank you. Just some very brief 
statements and perhaps a question of Dr. Bryan. I think I am 
thinking more of the medical research at Pittsburgh, and you 
are down the road a little bit I guess in the other direction. 
But perhaps you know something about this.
    I agree with Dr. Wirth in terms of that NIH ought to be 
given a broad rein--range, I guess, in which to make their 
decisions and less input from those of us who come into contact 
with a lot of these difficult situations and have to--can't 
really pick and choose. We are not knowledgeable either to make 
those determinations.
    But on the other hand, I think there is some need for input 
from outside, as you point out, some representation, and I 
guess to a degree we do that.
    It seems to me--and maybe I am not using the setup of NIH 
correctly, but I have heard the representation on their board 
or perhaps the doctors' panels that help set these priorities. 
Perhaps maybe we could have a better play in what groups are 
represented there--what specialties, what doctors, what 
diseases are represented there. And that would be a way of 
again giving them broad powers, but yet we in Congress being 
able to make sure that one disease is not given priority over 
another one for the wrong reasons.
    Second--and my last comment in this area, and I am going 
toward something that I just mentioned earlier--I have been 
working really 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--a percent of our young children. 
It is Duchenne's muscular dystrophy; I was at a fund-raiser for 
them about a week ago in Memphis, and I am told that that is a 
disease in which 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.
    I think our priorities also ought to be, in addition to all 
the other priorities, trying to find cures for those diseases 
regardless of the size of the population affected; those 
diseases that are getting close to being solved, cured. That, 
to me as a layperson, a nonmedical person, makes some sense, 
that if we are getting close--because that can open the doors 
to other related diseases, I would think. I would think 
Duchenne's muscular dystrophy would have some very close 
cousins in terms of diseases that could be affected in a 
positive sense.
    So, Dr. Bryan, I am asking you cold, do you know anything 
about that particular disease in terms of are we making 
progress there?
    Mr. Bryan. I am familiar; I am not an authority on that 
disease. But you are correct, it is a disease that affects a 
relatively small population, but in a devastating fashion. And 
remarkable advances have been made, mostly in understanding the 
genetics and etiology of the disease.
    I think the dilemma is one that is difficult. Your 
committee has to face the public needs, define 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 priority from a public 
perspective, but then I think you have to take into account the 
experts and the peer review system to help decide when you 
actually support a particular research project area.
    Mr. Bryant. Quickly, does anyone have an additional 
comment? Thank you for being here and thank you, Mr. Chairman.
    Mr. Bilirakis. And I thank the gentleman.
    We will excuse this panel at this time. We customarily 
furnish written questions, and we request written responses. We 
would appreciate your assistance if you are all willing, to do 
that in a timely fashion, Ms. Fraser, sooner rather than later, 
particularly on the question that I raised----
    Ms. Fraser. That will not be a problem.
    Mr. Bilirakis. Thank you very much.
    The second panel consists of--was 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 FDA on this 
matter?
    Mr. Doleski. I work for the FDA.
    Mr. Bilirakis. Well, you do not want to testify at all? 
Technical responses?
    In any case, you will be here for the testimony and the 
questions so that you can take those back too? I appreciate 
that.
    Why don't you give us your name, sir, for the record?
    Mr. Doleski. Dave Doleski, D-O-L-E-S-K-I, Legislative 
Analyst with the FDA.
    Mr. Bilirakis. I am going to introduce Mr. Robert Brady, a 
partner with Hogan & Hartson. They are here on behalf of 
Biogen; Ms. Abbey Meyers, President of the National 
Organization of Rare Disorders; Mr. Thomas A. Lang, Senior Vice 
President, Strategic Product Development, Serono Laboratories, 
Rockville, Maryland, and he is accompanied by Nick Ruggieri, 
Vice President of Governmental Affairs; Ms. Catherine Bennett, 
Chair, Board of Directors, Cancer Research Foundation of 
America.
    And I would now yield with the committee's indulgence to 
Mr. Dan Burton, who is not on this committee, but who chairs of 
course another very significant committee, who will introduce 
Mr. Navarro and at the same time take 2 to 3 minutes to talk 
about his legislation. You are recognized.

 STATEMENT OF THE HON. DAN BURTON, A REPRESENTATIVE IN CONGRESS 
                   FROM THE STATE OF INDIANA

    Mr. Burton. Thank you, Mr. Chairman. And I hope you will 
grant me just a minute or 2 latitude because I think some of 
the things that I would like to say are very important.
    To my classmate and chairman of this committee, Chairman 
Bilirakis, it is nice to be with you. I think it is the first 
time in the 18 years that we have been here that I have 
appeared before your committee.
    Mr. Bilirakis. I am sure that is true. And, Dan, I am sure 
the thought has crossed your mind, there are not too many of us 
left.
    Mr. Burton. No, and unfortunately we just lost one of our 
classmates.
    Mr. Bilirakis. Yes, we just lost one of ours.
    Mr. Burton. Anyhow, I appreciate your holding this hearing 
and allowing us to testify on H.R. 3677, the Thomas Navarro FDA 
Patient Rights Act.
    The United States of America is a country based on 
freedoms, and among the freedoms guaranteed through our 
Constitution are freedom of speech, freedom to practice the 
religion of our choice and a free press. However, we are not, 
as individuals, guaranteed the freedom to make a life-and-death 
decision in the area of medicine.
    Imagine our own government forbidding your child access to 
a nontoxic treatment, a nontoxic 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, to be deaf, to make him sterile, 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 chancing no 
treatment and possibly death or toxic treatment and possibly 
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 attacked by the FDA in an attempt to stop your work. 
Attacked by the very agency that is supposed to encourage and 
promote research.
    Imagine submitting the BT-29 protocol so that a 4-year-old 
boy can be treated with a nontoxic cancer therapy whose safety 
has been established. A treatment which has 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. That is exactly what has happened to 
Dr. Burzynski down in Texas.
    Many 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 will leave the full story of Thomas' specific 
condition for Mr. Navarro to talk about.
    Two years ago the parents of another little boy, Dustin 
Kunnari, testified before our committee, the Government Reform 
Committee, about FDA's gatekeeping on clinical trials. Dustin 
had the same form of cancer as little Thomas Navarro. Dustin 
was the last person the FDA allowed to receive antineoplastons 
without having first failed chemotherapy and radiation. He is 
healthy and cancer-free today and without the devastation of 
chemotherapy and radiation side effects.
    Over the last 3 years, the Committee on Government Reform 
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 the government's 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, who 
decides? Is it the role of the U.S. 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 FDA Patient Rights Act is the 
first step in restoring medical freedom. It is the first step 
in taking the decisionmaking out of the hands of the government 
and putting it back in the hands of the individual where it 
belongs, an informed decision.
    Mr. Chairman and members of the committee, H.R. 3677 has 43 
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 
sold their house. They completely depleted all of their 
resources--in trying to solve the problem of their boy. And it 
is a heart-rending 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 son. They 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 the 
battle for his son's life, 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 
do not mind, to Mr. Navarro.
    Mr. Bilirakis. Thank you, Dan. Thanks for your interest in 
this subject, all issues in America.
    The Chair will now yield to Mr. Navarro, who uses as his 
address: Ronald McDonald House, Room 1101, 405 East 73rd 
Street, New York, New York.
    Mr. Navarro, please proceed, sir.

 STATEMENTS OF JAMES NAVARRO, FATHER OF THOMAS NAVARRO; ROBERT 
  BRADY, PARTNER, HOGAN & HARTSON, ON BEHALF OF BIOGEN, INC.; 
    ABBEY MEYERS, PRESIDENT, NATIONAL ORGANIZATION FOR RARE 
  DISORDERS; THOMAS A. LANG, SENIOR VICE PRESIDENT, STRATEGIC 
 PRODUCT DEVELOPMENT, SERONO LABORATORIES, INC.; AND CATHERINE 
    P. BENNETT, CHAIR, BOARD OF DIRECTORS, CANCER RESEARCH 
                     FOUNDATION OF AMERICA

    Mr. Navarro. 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 5-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 like to first go on record saying that my son's 
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 than 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 which are 
good or greater than those treatments currently available for 
treating pediatric cancer.
    We were faced with a decision almost a year ago, which 
changed our lives forever, when our son Thomas was diagnosed 
with medulloblastoma, which is a nonsurvivable type of cancer. 
Thomas was rushed to surgery within hours to remove a 4 by 6 
centimeter tumor from his cerebellum.
    After surgery, we were faced with the decision of follow-up 
therapy. We discovered in short order that the standard follow-
up therapies, radiation and chemotherapy, both had severe and 
irreversible side effects. These included the possibility that 
he would become blind, deaf, and sterile; that Thomas would 
develop hormonal deficiencies that would have stunted growth; 
that he 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, nontoxic means 
of treating our son. We found a treatment that showed a great 
promise for treating medulloblastoma 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 efficacy 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 
actions to save the Thomas Navarros 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 heat. 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 to you 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 others that would stand in opposition to this bill 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 treatment that he has been forced to take. And I can 
only tell you it has been a very long and hard year.
    Thank you for letting me speak.
    [The prepared statement of James Navarro follows:]
                  Prepared Statement of James Navarro
    Good morning Mr. Chairman, my name is Jim Navarro. I am the father 
of 5 year old, Thomas Navarro, for whom this Bill is named. I have been 
asked to speak of the benefits this Bill would present. I would first 
like to go on record saying that my son's health has not stood still 
for the slow wheels of Government to move, and thus this Bill may 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 in 
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 
than 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 which are as good or greater than those treatments currently 
available for treating pediatric cancers.
    We were faced with a decision almost one year ago, which changed 
our lives forever. When our son, Thomas, was diagnosed with 
Medulloblastoma, Thomas was rushed to surgery within hours to remove a 
4 X 6-cm tumor on his cerebellum. After the surgery we were faced with 
the decision of follow up therapy. We discovered in short order that 
the standard follow up therapies, radiation and chemotherapy, both had 
severe and irreversible side effects. These side effects included the 
possibility that he would become blind, deaf, and sterile. That Thomas 
would develop hormonal deficiencies would have stunted growth, that he 
would have an immediate and progressive loss of IQ. And that he could 
develop secondary cancers as a result of the treatment.
    We immediately began our search for a safer, non-toxic means for 
treating our son. We found a treatment that showed great promise for 
treating Medulloblastoma. 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 manufacture's information 
that the drug companies put in the boxes, they state, ``safety and 
effectiveness in pediatric patients have 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 efficacy 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 in jail for being ``bad 
parents.''
    Based on your own experience Mr. Chairman, what actions must I take 
today to get you and your committee to take the required actions to 
save the Thomas Navarros of tomorrow?
    During the course of the last year, my family has lost everything--
our home and business in Arizona. It has been because of the kindness 
and generosity of others, especially the support of Citizen's For 
Health, that Thomas has been able to receive medical care. HR 3677 
introduced by Congressman Dan Burton, now has 43 cosponsors. I implore 
you take this issue up and get HR 3677 passed into law.
    I would be pleased to entertain any questions from the Committee.

    Mr. 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 has got to be done on a 
bipartisan basis. That is your reason for imploring Mr. Waxman, 
who frankly has just been very much interested in health care 
all through the years. I know I have worked with him on this 
committee for many, many years. And I do not really know 
personally what his position is on the legislation. I guess 
staff here does, but we are going to do everything we possibly 
can.
    Mr. Navarro. Mr. Chairman, if I might add real quick: It is 
interesting, 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. Yet the 
irony is, here before me are the consent forms to the treatment 
Thomas is going through now, which state, ``Permission to for 
participation of child in research.''
    He is not in a protocol because they do not have a 
protocol. They really do not know quite what they are going to 
do with him. It is a hit and miss, and as they would say, a 
crapshoot. In fact, one of the things that is sad that 
disturbed me, especially after hearing Dr. Pazdur testify that 
the rate of success was 70 and 80 percent--and this is coming 
out of the horse's mouth--however, with standard therapy, there 
is less than 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 fared the toxic side effects better than the other 
children in the ward, they are anxious to start using high, 
high-dose radiation and chemotherapy five to six times greater 
than 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 
nontoxic and nonlethal. It is our right as Americans to have 
that freedom.
    Mr. Bilirakis. Thank you, Mr. Navarro.
    Mr. Brady. Mr. Brady? 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.

                   STATEMENT OF ROBERT BRADY

    Mr. Brady. Thank you, Mr. Chairman. I will be quite brief 
and just summarize my comments.
    I am Bob Brady. I am here appearing this morning 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 am 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 benefit to patients 
or promoting innovation. The Orphan Drug Act has been an 
unparalleled success. Any changes to the act should be made 
only after careful analysis and consideration by fully informed 
Members of the Congress in the context of the entire law.
    The FDA and its Office of Orphan Products 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 than one here at the table, supporting this 
provision.
    The Orphan Drug Act shouldn't be amended piecemeal by an 
amendment hastily packaged together with noncontroversial 
measures at the end of a legislative session. It would 
undermine 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 the judgment of FDA that it is clinically superior 
for safety reasons to the prior drug approved in this 
marketplace. That is 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, after 25 years it has 
been the least controversial piece 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 7-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 product 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 
Congress' intent that there be new and innovative treatments 
developed for millions of Americans who suffer from rare 
diseases.
    Orphan drug policies 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 among the current marketplace.
    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 Act 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 are not 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 than 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.
    [The prepared statement of Robert Brady follows:]
    Prepared Statement of Robert P. Brady on Behalf of Biogen, Inc.
    I am Robert P. Brady, and I appear here this morning on behalf of 
Biogen, Inc. (Biogen). I am a partner in the law firm of Hogan & 
Hartson L.L.P., in Washington, D.C. I have practiced law for 25 years, 
and spend almost all my time on matters involving pharmaceutical and 
biotechnology laws including the Orphan Drug Act. I would like to thank 
you for the invitation to Biogen to testify before this Committee.
    At the outset I would like to summarize the key points of my 
testimony. 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 benefit 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 whole Orphan Drug Act. The FDA office of Orphan Products 
Development, which has done such a conscientious and successful job in 
implementing the Orphan Drug Act, should be consulted and its views 
taken into account. Moreover, Biogen knows of no patient advocacy 
groups supporting H.R. 4242, including the National Organization of 
Rare Disorders (NORD) which is the chief consumer advocacy organization 
for orphan drug research and development and was instrumental in the 
development of the Act. The Orphan Drug Act should not be amended piece 
meal, by an amendment hastily packaged together with non-controversial 
measures at the end of a legislative session. It would undermine 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.
    Biogen is a biotechnology company based in Cambridge, MA and 
manufacturer of a product for the treatment of relapsing forms of 
remitting multiple sclerosis (MS) that was approved in 1996. Biogen's 
product (Avonex') was approved as an orphan drug and thereby 
received a grant of seven years of marketing exclusivity. Two important 
medical facts about Avonex' must be kept in mind. It is the 
only MS drug approved for both reducing the number of exacerbations and 
slowing disease progression. Also, the FDA concluded that it was 
clinically superior to the existing MS product due to greater safety. 
As a result patients have benefited greatly from this approval.
    Biogen is strongly opposed to H.R. 4242 for the following reasons:
    The Orphan Drug Act is one of the most effective laws enacted by 
Congress with full bipartisan support in the last twenty 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. The Orphan Drug Act has been an unqualified 
success. In particular, it spurred the development of breakthrough 
drugs for Multiple Sclerosis, Cystic Fibrosis, Hemophilia, Leukemia, as 
well as over 100 other rare diseases. During the ten years before the 
law, only ten drugs were approved by the Food and Drug Administration 
(FDA) for the treatment of rare diseases. Since the law was enacted, 
however, about 200 orphan drugs have been approved and about 1,000 are 
in the pipeline. Clearly, any modification of the Act must be the end 
result of a deliberative process that increases incentives to develop 
breakthrough treatments and, most importantly, benefits patients.
    This is not the case with H.R. 4242--it would undercut the 
overwhelming success of the Orphan Drug Act with no real benefit to 
patients. 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 incentive by limiting it to particular aspects of the 
orphan product. Narrowing this key incentive would not only hurt 
companies that make orphan drugs but would also undercut Congress's 
intent that there be new and innovative treatments developed for the 20 
million Americans who suffer from rare diseases.
    The FDA has skillfully implemented the Congressional intent of the 
Orphan Drug Act in a manner that balances the seven year marketing 
exclusivity incentive with the need to foster the public health goals. 
One way in which FDA has balanced this issue is through the development 
of a regulation defining scientific/medical criteria under which an 
orphan drug which is the same as an orphan drug already approved for 
marketing can be determined to be ``clinically superior'' and, 
therefore, allowed to come to market in spite of any remaining 
marketing exclusivity granted to the original approved product. (See 21 
C.F.R. Sec. 316). FDA, the scientific and medical expert in this area, 
has defined three such criteria to determine clinical superiority. 
These criteria were thoughtfully and deliberately developed through 
notice and comment rulemaking by the FDA. All interested parties had an 
opportunity to present their views. Ultimately, the FDA crafted a well 
reasoned definition of clinical superiority.
    Critically important since the finalization of this rule almost a 
decade ago, FDA has carefully exercised its scientific and medical 
judgment in implementing this rule in a manner that is truly in the 
best interests of patients. When another orphan drug truly is 
clinically superior, FDA has allowed it to go to market so that 
particular patients will benefit. It has done so sparingly, however, 
because the FDA has correctly concluded, based on its extensive 
experience, that, absent a real showing of clinical superiority, 
preserving the seven year marketing exclusivity incentive is vitally 
important to the development of new orphan drugs and that will help 
even more patients suffering from orphan diseases.
    These regulations are sound and fair. Companies are rewarded when 
they produce a clinically superior drug that represents an innovation 
above the current marketplace. Biogen satisfied these regulations when 
the FDA found that its product Avonex was ``clinically superior'' to 
another existing beta interferon product in 1996.
    Serono Laboratories (Serono) is testifying on behalf of H.R. 4242 
today. They manufacture a drug for multiple sclerosis that is the same 
as Biogen's MS drug. Serono would like to get their drug onto the 
American market, but they are blocked by the market exclusivity of 
Biogen's multiple sclerosis product, which does not expire until May 
17, 2003. The Orphan Drug Act and the FDA implementing regulations 
currently provide a way for Serono's product to get to market: a 
showing of ``clinical superiority'' based on appropriate scientific 
data.
    Serono, or any other company, should not be held to a lesser 
standard than its competitors in the marketplace. To date Serono has 
not demonstrated in head to head comparative trials that its product is 
safer or more effective than the other beta interferon products on the 
market for relapsing remitting multiple sclerosis. In 1999, the FDA 
found, based on data submitted to the FDA by Serono, that Serono's 
product was not clinically superior to the other similar multiple 
sclerosis products on the market. Therefore, it is barred from the U.S. 
market until the Biogen marketing exclusivity expires in May 2003. 
Because Serono does not represent an innovation, patients are not being 
denied a new or improved therapy. Serono is seeking to change these 
requirements because Serono has not been able to satisfy them.
    Serono's situation highlights one of the key problems with H.R. 
4242. Under the bill, Biogen's market exclusivity for its multiple 
sclerosis drug would be limited to blocking from the market only those 
products that cause fewer injection site reactions and less skin 
necrosis. However, a product which is less safe than Biogen's by 
causing more site reactions and skin necrosis--such as Serono's 
product--would be, under this bill, eligible for approval by the FDA. 
Biogen does not understand how it benefits patients to allow a drug on 
to the market during the exclusivity period that is neither safer nor 
more effective.
    The Orphan Drug Act's market exclusivity is not a barrier to 
approval of a subsequent product that is not the same drug. A 
subsequent drug for the same indication may be found to be not the same 
drug if it is either chemically different or clinically superior. 
Serono's Rebif product meets the statutory and regulatory standards for 
``same drug'' because chemically it is the same drug for the same 
disease as both Avonex and Betaseron. Because Rebif chemically is the 
same drug as Avonex, in order for Serono to receive marketing approval 
prior to 2003, Serono must demonstrate that it is clinically superior 
to Avonex. The FDA's clinically superior criteria protects the drug 
development incentive, while permitting the introduction of better 
products to treat serious illness.
    Aside from the serious policy concerns with H.R. 4242, another 
fundamental flaw is that it is, in part, unconstitutional. As presently 
introduced, the bill would apply to any drug designated on or after 
January 1, 1990. By going back and retroactively narrowing the scope of 
the market exclusivity, this would constitute an unconstitutional 
taking under the Fifth Amendment. This legislation would not only 
effect the property rights of Biogen but property rights of many other 
companies with orphan drug designations that have made significant 
economic investments based on an expectation of market exclusivity. 
Biogen has attached a legal opinion on the unconstitutionality of the 
bill that was previously sent to the Committee.
    For Congress to make a change to a law as successful and important 
to the health of the American people, as the Orphan Drug Act, one would 
expect that there would be strong support from the biotechnology and 
pharmaceutical industries and patient advocacy groups. Biogen knows of 
no outside parties that favor the H.R. 4242. There are no patient 
advocacy groups supporting this bill. The National Organization of Rare 
Disorders (NORD), which is the chief consumer advocacy organization for 
orphan drug research and development and was instrumental in the 
development of the law, is opposed to the bill. The views of NORD and 
other patient groups should not be ignored.
    The stated purpose of this hearing today is to consider legislation 
that would secure the health of the American people. The Orphan Drug 
Act has already fostered the development of numerous breakthrough 
treatments for rare disorders and helped countless persons. H.R. 4242 
will threaten this continued development and risk the security of the 
health of the American people with rare disorders.

    Mr. Bilirakis. Thank you, Mr. Brady.
    Ms. Meyers, please proceed, ma'am.

                    STATEMENT OF ABBEY MEYERS

    Ms. Meyers. Thank you, Mr. Chairman.
    For those of you who do not 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 its 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 when more than one sponsor is interested in the same 
drug. And so we caution you not 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 innovator drug. You can do that in several 
ways. You can get an orphan drug approved for a different 
disease.
    For example, if beta Interferon was approved for cancer or 
something else, they could get it on the market and it could 
compete in the marketplace. Or you could prove that it is 
chemically or structurally different than 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 
is safer or more effective or a major contribution to patient 
care. In the case of Avonex and Betaseron, for example, Avonex 
showed that you need less injections every week, it had fewer 
side effects and did not 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 7 years of exclusive marketing 
rights; and it is only through regulations that the current 
definitions of ``same'' and ``different'' was created. And I 
want to tell you that 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 
incentive of the Orphan Drug Act. And also be aware that the 
Orphan Drug Act's 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 
climbing Mount Everest every day. And then they lose their 
eyesight, so they can't see to fill up their syringe. It is a 
major improvement to patient care when you only need 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 beta Interferon? If Serono--and we have the greatest 
respect for Serono, but if Serono believes that its drug is 
either safer or more effective or that it is 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 Betaseron, 
they should take their proof to the courts. They should not be 
hiring lobbyists 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 will 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.
    [The prepared statement of Abbey Meyers follows:]
 Prepared Statement of Abbey Meyers, President, National Organization 
                           for Rare Disorders
    Mr. Chairman and members of the Committee, the National 
Organization for Rare Disorders (NORD) is the consumer organization 
that worked for passage of the Orphan Drug Act (ODA) of 1983, and we 
continue to closely monitor its implementation today. NORD represents 
approximately 25 million Americans with more than 6,000 rare ``orphan 
diseases'', and we are very pleased to be here today. Thank you.
    As you know, the Orphan Drug Act is one of the most successful 
pieces of health legislation ever enacted by Congress. Today, 
approximately 1,000 orphan drugs have been designated by the FDA, and 
over 200 of them have been approved for marketing in the United States. 
Recognizing the public health impact of the American law, other nations 
have implemented orphan drug statutes including Japan, Singapore, 
Australia and the entire European Union.
    It is extremely important that Congress not undermine the intent of 
the law, which is to encourage the commercial development of treatments 
for small populations of patients. We do not support H.R. 4242, the 
Orphan Drug Innovation Act, for many reasons, and we urge the committee 
not to approve the legislation for further consideration by Congress. 
Let me explain why we do not support H.R. 4242.
    Early in the evolution of the American Orphan Drug Act, it became 
necessary to define the words ``same'' and ``different''. In other 
words, as defined by FDAs carefully crafted regulations, if a 
manufacturer of a similar orphan drug can prove that their drug is 
chemically different or clinically superior, even though it contains 
the same active ingredients as the original orphan drug, the FDA will 
approve it for marketing.
    There are several ways to prove that a drug is ``clinically 
superior'': either by providing FDA with scientific data proving that 
it is either safer or more effective, or that it represents a ``major 
contribution to patient care''. The latter is usually an obvious 
improvement, such as developing an oral version of an injectable drug--
so that patients no longer have to suffer painful injections--or 
developing a long-acting version of a drug that must otherwise be taken 
several times each day, for example. Thus, the orphan drug regulation 
defining ``same'' and ``different'' not only promotes, but encourages 
development of new improved versions of marketed orphan drugs while 
PRESERVING the chief incentive of the ODA.
    Since most orphan drugs have no competition because companies are 
generally not interested in investing the huge sums necessary for 
research and development of a drug that will have a very small market, 
the ODA offers an important incentive to encourage orphan drug 
innovation.
    Companies can receive seven years of exclusive marketing rights for 
both the innovator drug and the clinically superior follow-on drug.
    Mr. Chairman, from time to time there are orphan drug ``races'' 
when more than one company is developing the same orphan drug for the 
same disease, and the law purposely creates a winner-take-all contest. 
This is the very core of the success of the ODA because it prevents 
competition for seven years, and ensures that a manufacturer of an FDA-
approved orphan drug will recoup its investment and make a profit. But 
losers of the race sometimes ask Congress to change the law because 
they want an exception for their drug. Thankfully Congress has been 
wise enough not to allow this, knowing that tinkering with the Act 
could destroy it.
    In this case, Congressman Thornberry's bill aims to redefine FDA's 
definition of ``same'' and ``different'', and to codify it into law, 
based on the mistaken belief that people with rare diseases do not 
already have access to clinically superior orphan drugs. However, H.R. 
4242 will NOT enhance patient choice because current regulations not 
only permit, but encourage competition when a therapeutic advantage can 
be scientifically proven.
    We believe that H.R. 4242 would disincentivize companies to develop 
clinically superior orphan drugs and biologics, and it would allow 
companies to seek approval for clinically inferior products. Moreover, 
H.R. 4242 would reduce the exclusivity of orphan drugs and biologics 
that have demonstrated they are clinically superior, because it would 
limit exclusivity to the innovation that enabled a clinically superior 
product to reach the market.
    There are no benefits to patients if H.R. 4242 becomes law. There 
are only benefits to companies that want to break the innovators 
exclusivity, but that exclusivity is the very backbone of the Orphan 
Drug Act which has since 1983 saved the lives of millions, and improved 
the quality of life for countless others.
    The current regulations, which are based on sound scientific 
knowledge and common sense, were written to promote innovation and to 
allow consumers access to clinically superior orphan drugs. They are 
fair to consumers and fair to companies. The ODA is good public health 
policy and continues to be one of the most successful pieces of health 
legislation ever written.
    We are profoundly grateful to Congress for enacting the Orphan Drug 
Act, and for preserving its integrity since 1983. If you write the 
Thornberry bill into stone, it will require an act of Congress to 
change it when new medical technologies emerge in the future. If you 
leave the orphan drug regulations alone, the FDA can easily fine tune 
the rules, if and when that becomes necessary.
    Mr. Chairman and members of the Committee, ``if it ain't broke, 
don't fix it.'' We urge you NOT to enact H.R.4242.

    Mr. Bilirakis. Thank you, Ms. Meyers.
    Mr. Lang.

                   STATEMENT OF THOMAS A. LANG

    Mr. Lang. Good morning. My name is Tom Lang. I am 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 evergreening, 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 
evergreening policy affects all drugs governed by the Orphan 
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 orphan drug evergreening is 
not a single-product issue.
    Serono is a strong supporter of the Orphan Drug Act. 
However, we have recently encouraged an anomalous and confusing 
interpretation of the FDA orphan drug regulations which results 
in orphan drug exclusivity evergreening. ``evergreening'' 
refers to FDA's granting of a new 7-year orphan drug 
exclusivity period for the entire drug substance upon the 
approval of a clinically superior version of the same drug, 
rather than protecting only the innovative feature exhibited by 
the second drug.
    The results are to close the market to competition beyond 
the initial 7 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 limit the scope of exclusivity for new versions of 
previously approved products in a manner consistent with H.R. 
4242. Like the Orphan Drug Act, the Waxman-Hatch Act seeks to 
create incentives for continued research on improved drugs and 
product improvements. The Waxman-Hatch Act, as one would 
expect, rewards only innovative features with exclusivity, 
rather than shielding the entire drug substance from 
competition when its original exclusivity period has run. This 
serves as evidence of Congress' intent and provides a basis for 
supporting the principle in H.R. 4242.
    FDA's handling of one particular product has resulted in 
several very strained policy positions on the part of FDA. In a 
letter to Serono, dated November 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 
were eligible for an abbreviated new application.
    Mr. Chairman, there is no rationale whatsoever for 
preventing competition from products that are supported by full 
NDAs and BLAs. Subsequently, Serono became aware of an instance 
where FDA has taken what appears to be a different position 
than that previously described, a position which actually is 
consistent with H.R. 4242, in a letter to Genentech.
    Serono believes orphan drug exclusivity evergreening can be 
resolved by FDA or Congress by simply limiting the second 
clinically superior drug's scope of orphan drug exclusivity to 
the superior characteristic that distinguished it as clinically 
superior. This solution would probably 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 initiatives, 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 incentives. 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 drug's exclusivity; 
Second, it would obtain 7 years exclusivity for the improved 
feature; and Third, 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 have expired.
    In summary, the current evergreening 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 2 years. Nevertheless, in Serono's opinion, FDA 
continues to administer the exclusivity principle in an 
inconsistent and unclear manner. The evergreening policy is now 
riddled with ad hoc exceptions not found anywhere in the 
statute or in the regulations. We, therefore, believe that 
clarifying legislation is warranted.
    Again, I would like to thank the committee for the 
opportunity to testify on this important matter affecting the 
incentive to develop improved drugs for rare diseases. We 
appreciate the committee's attention and consideration, and I 
would like to thank Dr. Ganske for his earlier comments.
    [The prepared statement of Thomas A. Lang follows:]
Prepared Statement of Thomas A. Lang, Senior Vice President, Strategic 
                   Product Development, Serono, Inc.
                             introduction.
    Good morning, my name is Tom Lang. I am Senior Vice President for 
Strategic Product Development at Serono, Inc. I would like to thank the 
Chairman, and other members of the Committee, for the opportunity to 
testify on the issue of orphan drug exclusivity ``evergreening,'' which 
is addressed by H.R. 4242. At this time, I want to make it clear to the 
Committee that Serono believes this issue needs to be addressed 
irrespective of the impact on our products. FDA's current evergreening 
policy affects all drugs governed by the Orphan Drug Act (ODA) and 
represents a policy issue that demands your attention. We fully support 
the remedy posed by H.R. 4242; however, we would be equally supportive 
of an administrative remedy accomplishing the same goal.
    As a global leader in biotechnology with a number of drugs and 
biologic products already approved in the U.S. and many more in our 
research pipeline, Serono is a strong supporter of the Orphan Drug Act. 
We recognize the need to provide strong incentives to develop drugs for 
rare diseases, and indeed, many of our drugs have orphan drug 
designations as well as orphan drug exclusivity. However, we have 
recently encountered a problem with FDA's anomalous and confusing 
interpretation of the Orphan Drug regulations, which results in orphan 
drug exclusivity ``evergreening.'' In this context, ``evergreening'' 
refers to FDA's granting of a new seven year orphan drug exclusivity 
period for the entire drug substance upon the approval of a second, 
clinically superior, version of the same drug, rather than protecting 
only the innovation exhibited by the second drug. This results in 
closing the market to competition beyond the initial seven years of 
exclusivity intended by Congress.
    This is an unwarranted extension of orphan drug exclusivity. It 
presents troubling policy issues of fairness, impediments to price 
competition that would benefit consumers, and the delay in availability 
of alternative therapies for patients.
                              background.
    The phenomenon of orphan drug exclusivity ``evergreening'' is a 
result of FDA's interpretation of its regulations giving effect to the 
ODA, and not a result contemplated or intended by Congress. The ODA 
itself created incentives for drug companies to develop therapies for 
rare diseases by awarding a period of seven years of market exclusivity 
to a product approved for an orphan indication. During these seven 
years, FDA may not approve other applications which are for the ``same 
drug'' and the same disease or condition. Congress intended that after 
this period of exclusivity, the public would benefit from increased 
treatment options as well as price competition among various products 
in these areas. The ODA overall has been a significant success in 
driving research for rare diseases.
    However, the statute is silent as to improved versions of 
previously approved orphan drugs. In the regulations adopted by FDA to 
implement the ODA, the agency created a mechanism by which a second 
product could also be approved during the period of market exclusivity 
awarded to the first drug. Such exceptions are made where a second drug 
is deemed to be clinically superior to the first orphan drug. FDA's 
intention in creating this mechanism was to maintain incentives for 
companies to continue research on orphan drugs, and to reward 
additional advancements by allowing them earlier access to an otherwise 
closed market. While this objective is laudable, FDA has recently 
chosen to implement it in a very problematic fashion.
                       fda's exclusivity policy.
     Recently, FDA has adopted a policy position related to the scope 
of a clinically superior orphan drug's 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 and physician choice of therapy.
    Now, after approval of an original orphan drug, whenever a 
subsequent orphan drug with a clinically 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 drug from competition, even after 
the original drug's exclusivity period is over. In these instances, 
companies that have developed new competing versions of the same 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. Under FDA's current policy, this total 
period of exclusivity barring the entry into the market of competing 
product versions could theoretically be as long as fourteen years if 
two drugs were approved; as long as twenty-one years if three drugs 
were approved, and so on. This possible extension, or ``evergreening'' 
of the original drug's exclusivity period by restarting the clock for 
the entire drug substance when a second or third clinically superior 
version is approved is the problem sought to be addressed by the 
Thornberry Bill (H.R. 4242).
        orphan drug evergreening is not a single product issue.
    FDA has designated at least five drugs as orphans, based solely on 
their demonstration of superiority over a previous version.

 Avonex' (recombinant beta interferon la for 
        relapsing remitting multiple sclerosis), exclusivity based on 
        clinical superiority to Betaseron.
 Sandostatin LAR' Depot (generic name octreotide for 
        several orphan indications, primarily transplant rejection)--
        long acting formulation, exclusivity based on superiority in 
        dosing to original drug.
 Nutropin Depot' (recombinant human growth hormone 
        for pediatric growth hormone deficiency) dosing superiority, 
        long acting formulation.
 Benefix TM (Coagulation Factor IX for hemophilia) - 
        Recombinant version judged safer than two previous versions.
 Prolastin' (Alpha 1 proteinase inhibitor for 
        emphysema), long acting formulation superior in dosing 
        convenience to original orphan drug.
    All five were considered to have presented a safety or dosing 
improvement over the original version of the drug. The active chemical 
entity in all five cases is the same as the original drug. The older 
versions were all approved as safe and effective products and are still 
being marketed. Thus, a new competitor in these other disease areas 
would also be expected to face an evergreening problem, based on FDA's 
policy.
other areas of food and drug law limit the scope of exclusivity for new 
               versions of previously approved products.
    As with the Orphan Drug Act, the Waxman-Hatch Act seeks to create 
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 than shielding the drug 
substance from generic competition when its original exclusivity period 
has run. This serves as evidence of Congress' intent, and provides a 
basis for supporting the principle in H.R. 4242.
  fda's position of only allowing abbreviated applications to compete 
               with an expired orphan lacks a rationale.
    FDA's handling of our product has resulted in several very strained 
policy positions on the part of the agency. For example, in a letter to 
Serono dated November 8, 1999 (copy attached), FDA indicated that while 
it would not allow our product to be marketed in competition with the 
original drug approved for this orphan drug indication, it would 
approve a generic version of the original product to come on the market 
if it were eligible for an abbreviated new drug application (ANDA.)
    In Serono's opinion, this position indicates that FDA in fact 
agrees in principle that the exclusivity that was awarded to the second 
clinically superior drug should not prevent competition with the 
original product whose exclusivity has lapsed. However, FDA makes an 
arbitrary determination that only ANDA drugs can compete, but not drugs 
that are supported by full new drug applications (NDAs and BLAs). There 
is no rationale whatsoever for preventing competition from products 
that are supported by full NDAs and BLAs.
  fda's action in a subsequent case is actually consistent with h.r. 
                                 4242.
    Recently, Serono became aware of an instance where FDA has taken 
what appears to be a different position than with our product, and one 
which appears to be consistent with H.R. 4242. In a letter to Genentech 
dated October 28, 1999 (copy attached), FDA advised the company that 
new long acting formulation of recombinant growth hormone (Nutropin 
Depot) has been designated as an orphan, but that the orphan 
designation ``applies only to the long acting formulation,'' rather 
than to the entire drug substance. This means that the Nutropin Depot 
improved formulation, once approved, would achieve orphan protection 
for seven years, but its exclusivity only would cover the improvement, 
and manufacturers wishing to introduce additional versions of the 
conventional dosage form would not be blocked. Thus, in this instance, 
FDA's position appears to be totally consistent with H.R. 4242.
                 solution to ``evergreening'' problem.
    Fortunately, the ``evergreening'' problem is one that has an 
extremely simple solution. Orphan drug exclusivity evergreening can be 
resolved by FDA or Congress by simply limiting the second, ``clinically 
superior'' drug's scope of orphan drug exclusivity to the superior 
innovation, feature, or characteristic that distinguished it as 
clinically superior. This solution would properly reward the innovation 
found in the clinically superior drug, while still allowing competition 
with an expired original drug, as intended by the law. Again, FDA could 
remedy this problem itself, without legislation, by simply modifying 
its current policy as to the scope of exclusivity associated with a 
``clinically superior'' orphan. This would be consistent with other 
exclusivity-related legislative initiatives, such as the Waxman-Hatch 
Amendments, and patent law as well.
    the solution proposed in h.r. 4242 would retain incentives for 
                  innovation in orphan drug research.
    Limiting the scope of exclusivity of a clinically superior orphan 
drug to its clinically superior feature still leaves the drug sponsor 
with an adequate incentive. A clinically superior drug would gain three 
significant rewards. First, it achieves the benefit of being allowed 
onto the market immediately despite the originator drug's exclusivity. 
Second, it obtains seven years' exclusivity for the improved feature. 
Third, it will be able to market its product as clinically superior. 
These are substantial rewards and incentives. These substantial 
incentives make it unnecessary to keep the market closed to other 
products wishing to compete with previous versions whose exclusivity 
may have expired.
                               conclusion
    In summary, the current evergreening policy unnecessarily denies 
patients and physicians alternative therapeutic options. In our view, 
given the way it is being administered, it actually inhibits innovation 
and deters competition, and creates anomalous windfall extensions of 
drug exclusivity. We have attempted to work with FDA to resolve this 
issue for two years. Nonetheless, in Serono's opinion FDA continues to 
administer the exclusivity principle in an inconsistent and unclear 
manner. The evergreen policy is now riddled with ad hoc exceptions not 
found anywhere in the statute or in the regulations. This has caused 
significant confusion for industry. We therefore believe that 
clarifying legislation is warranted to avoid policy that we believe was 
never intended by Congress. That is why we support H.R. 4242.
    Again, I would like to thank the Committee for the opportunity to 
testify on this important matter affecting the incentive to develop 
improved drugs for rare diseases. We appreciate the Committee's 
attention and consideration.

    Mr. Bilirakis. Ms. Bennett, please.

                STATEMENT OF CATHERINE P. BENNETT

    Ms. 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. We are a 
national nonprofit 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 Pryce 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 us have had a personal experience with 
cancer. We have seen it attack a family member, a friend, a 
coworker, 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 No. 
1 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 
is about 100 classrooms filled with children who won't start 
school next September. September is significant in that it is 
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 in resolution which 
seeks to raise awareness about the realities of childhood 
cancer and make suggestions or recommendation about where 
Congress could 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 
develops 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. Four 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 through 
research through clinical trials. They have become the standard 
of care for pediatric oncology patients with approximately 70 
percent of the children who are diagnosed participating. This 
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 support such 
policies. Additionally, H. Res. 576 encourages support for 
policies that encourage the development of new drugs in 
biologics. As members of this committee know, the Food and Drug 
Administration Modernization Act provides additional incentives 
to encourage greater private investments and research by 
providing some additional 6 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 is worth 
reevaluating the policies reflected in that statute.
    While we look to the future with hope that we will see the 
day when 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 improving the 
quality of life for these patients. The horrors of cancer are 
many, but it is hard to imagine anything more tortuous than a 
parent witnessing their child in pain. Yet many will tell you 
that they have been forced to stand helplessly by while their 
children are enduring invasive and painful treatments.
    The resolution points out a recent study which revealed 
that 89 percent of children with cancer experienced 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 in 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 that 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.
    [The prepared statement of Catherine P. Bennett follows:]
    Prepared Statement of Catherine P. Bennett, Chairman, Board of 
            Directors, Cancer Research Foundation of America
    Thank you, Mr. Chairman and members of the Subcommittee. I 
appreciate this opportunity to testify on an issue that is of great 
personal importance to me--cancer awareness, treatment, and research.
    I am here representing the Cancer Research Foundation of America, 
as Chairman of their Board of Directors. For those of you not familiar 
with CRFA, we are a national, non-profit health organization whose 
mission is the prevention of cancer through scientific research and 
education. Founded in 1985 by Carolyn Aldige, the Foundation has funded 
research by more than 200 scientists at more than 100 leading 
universities and medical centers, and is one of only ten non-federal 
agencies whose grant review process is approved by the National 
Institutes of Health. And, I am pleased that 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. 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 have seen it attack a family member, a friend, a coworker, 
or we have been diagnosed ourselves. This dreaded and pervasive disease 
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 cancer 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 each year, some 
2,300 children will die from the disease. That's about 100 classrooms 
filled with children, who won't start school next September.
    September is also significant in that it is recognized as National 
Childhood Cancer Awareness Month. So it is appropriate that the 
Committee has house resolution ______, 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 about where Congress can help 
ensure that more children live to start a new school year.
    The statistics in the resolution demonstrate the challenge we face:

 The incidence of cancer among children is rising by one 
        percent each year.
 One in every 330 Americans develops cancer before age 20.
 Cancer constitutes about 8% of deaths between ages 1 and 19.
 And, as I mentioned earlier, 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. Forty years ago, 
        a diagnosis of childhood cancer was a death sentence, but today 
        almost 70 percent of children diagnosed with the disease will 
        survive. This success rate can be attributed to research 
        through clinical trials. In fact, clinical trials have become 
        the standard of care for pediatric oncology patients, and about 
        60 percent of children that are diagnosed with cancer 
        participate. That compares with only 3% of adult cancer 
        patients and 1.5 % of Medicare patients.
    It makes sense to build on these efforts by making sure that 
opportunities for childhood cancer research are funded, that we attract 
the best and brightest scientists to pediatric oncology, and that as 
many children as possible participate in and benefit from the 
discoveries made through clinical trials. H. Res. ______ suggests that 
Congress support policies consistent with these goals.
    Additionally, H.Res. ______ encourages support for policies that 
encourage the development of new drugs and biologics. As the Members of 
this Committee know, the Food and Drug Administration Modernization Act 
of 1997 provided an incentive to encourage greater private investment 
in research on the use of drugs to treat pediatric diseases. 
Specifically, the Act provides an additional six months of market 
exclusivity to sponsors of new or approved drugs if they conduct 
pediatric studies that may produce benefits for children. Despite the 
good intentions of this law, the policy has not proven effective in 
stimulating research or providing additional information about drugs 
that may prove useful in treating pediatric cancer. It is worth re-
evaluating this policy and its implementation by the FDA so that we can 
ensure that children are not left out of the tremendous advances in the 
treatment of disease that new drugs and biologics can provide.
    While we look to the future with hope that we will see the day when 
no child becomes the innocent victim of cancer, we must face the 
reality that children today are suffering and dying. We must also focus 
our attention on improving the quality of care and of life for these 
patients. The horrors of cancer are many, but it is hard to imagine 
anything more torturous for a parent than witnessing their child in 
pain. Yet, many parents will tell you that they have been forced to 
stand by helplessly while their child endured invasive and painful 
treatments. As H.Res. ______ points out, a recent study revealed that 
89 percent of children with cancer experienced 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 
reasons 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. H.Res. ______ is supportive of such curriculum as part of 
medical training.
    The battle against childhood cancer is being hard fought by those 
that 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--to their cause. I believe 
H.Res. ______ 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 this subcommittee to lend its support to this legislation.
    Again, I appreciate the opportunity to participate in today's 
hearing and to speak to this issue during Childhood Cancer Awareness 
Month.
    Thank you, Mr. Chairman.

    Mr. Burr [presiding]. Thank you, Cathy, and welcome. The 
Chair would take this opportunity to recognize himself, as soon 
as he gets his thoughts. And we apologize that there are 
members trying to get back. And it is the intent of the 
committee to continue with the hearing rather than take a break 
for lunch because of the afternoon schedule. Here we think it 
is not only more beneficial to us, but also to you to go ahead 
and allow those members to 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.
    Mr. Burr. Can you be a visionary for us just a minute and 
look out once the human genome project is complete, once we 
have mapped 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 in the future?
    Ms. Meyers. Well, it is very complicated because it is 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 
be able to personalize drugs for the particular genetic defect 
that has occurred in individuals. So we will have custom-made 
bio technology drugs to address the specific defect in that 
gene.
    Mr. Burr. Which means the population, that because they are 
going to be subsets of disease, the population that they are 
going to target is going to be tremendously small.
    Ms. Meyers. Minuscule.
    Mr. Burr. 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 is defined at 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 small populations, most of which will come from 
biotechnology.
    Mr. Burr. 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 am not talking 
about the debate that we are at at this table, I am 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 are going to charge $100- or $200,000 
a year for that treatment, you are 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 is even hard to find companies that are willing 
to make drugs for 3- or 400,000 Americans.
    Mr. Burr. In today's research environment?
    Ms. Meyers. Yes.
    Mr. Burr. Ten years from now, in tomorrow's research 
environment where you have got a map that leads to you a point 
that it took you 5 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 you have that there is 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 as under the Orphan 
Drug.
    Ms. Meyers. It is true, but, you know, the bigger these 
companies are getting with their mergers and their 
acquisitions, I mean, they are interested in Viagra, they are 
not even going to be 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 a billion 
a year.
    Mr. Burr. 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 think it will be, yes.
    Mr. Burr. Did you ever envision under the Orphan Drug law 
that--let me ask it a different way: Do you think it is 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--7-year exclusivity?
    Ms. Meyers. Yes.
    Mr. Burr. 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 economically or 
structurally different.
    Mr. Burr. Two different companies.
    Ms. Meyers. Two different companies.
    Mr. Burr. Should the same company have the ability to 
reapply for whatever changes, get a new 7-year exclusivity 
agreement?
    Ms. Meyers. Yes. And that also happened with Genzyme's drug 
for genetic disease for Gaucher's disease where they did 
improve it. It had been made out of a natural blood substance 
or something, and then they made a biotechnology version and 
they got another 7 years. Yes, they should, because the main 
incentive is to develop a better drug. And it worked.
    Mr. Burr. 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 is not--
that is 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 data base to chase that 
small population drug, you really do have an inherent ability 
of one company to continue to restart the clock. I am 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 problem. And I think that we have got to 
focus out on the future and ask ourself what do we need to do 
in preparation for the changes. 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. But it is 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 prove all the safety and 
effectiveness of a brand new drug, and then they are allowed on 
the market to compete with the drugs whose exclusivity has 
expired. That is what could happen here. Betaseron, the first 
beta interferon, their exclusivity has expired. If any company 
can prove that their beta interferon is the same as Betaseron, 
they would get on the market.
    Mr. Burr. We have 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 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 goals standard of the FDA, their process, and 
the innovative treatments that Dr. Brezinski and others have in 
the marketplace today, and certainly I have been involved for 4 
years in Dr. Brezinski's treatments. And hopefully we have--
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. Brezinski on the 
data that was needed for us to get expansions. But I don't want 
to address Dr. Brezinski's treatment specifically, because one 
thing I want you to understand is that Members of Congress are 
not here to practice medicine. But we are here to try to 
address the structure that is 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 us have affected in our 
families.
    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 HCFA, and that we can get patients back to the 
forefront of the health care delivery system in this country. 
We spent a lot of time arguing whether it is reimbursements or 
whether it is doctors or whether it is hospitals or whether it 
is insurers, and really more time about the process than we do 
about the outcome. I understand you are only concerned with the 
outcome. That is all you should be concerned with. We have 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 am 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 have 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 the 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' oncologist made it very clear that we are 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 have been efforts made 
to bring it to the forefront where it can 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 parents 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 have 
spoken to parents of children that have been destroyed by 
radiation and chemo. And I am thinking in particular of the 
young man from Houston, Texas who today, at 19 years old, is 
deaf, dumb, blind, strapped to a wheelchair, has an arm's 
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 am 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 have even 
applied for a compassionate use exemption. Now the doctor that 
we chose to go to in Houston has a protocol for 
medulloblastoma. He has treated with the blessings of the FDA 
children with medulloblastoma. 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 recurrent 
measurable tumor.
    If this is a pure glass of water, and my agency is in 
charge of making sure that water is available that 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 he would be 
reluctant to drink the water.
    And in Thomas' 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 about other 
therapies that take, to be honest, a lifetime to recover from?
    Mr. Burr. 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 had passed with unanimous bipartisan support from this 
House and from the Senate and was signed into law by the 
President. We are still waiting to see the full changes as they 
are implemented from that legislation. It is 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 is 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 have been notified that we have 
a series of votes. And because I know that that will throw 
further the end of the turmoil of members' schedules this 
afternoon because of the knowledge of their schedules, I am 
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 am 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.
    [Whereupon, at 12:45 p.m., the subcommittee was adjourned.]
    [Additional material submitted for the record follows:]
  Additional Testimony for the Record of the Department of Health and 
                             Human Services
    The Food and Drug Administration (FDA or Agency) has serious 
concerns about H.R. 3677, Thomas Navarro Patients Rights Act because 
this bill will seriously weaken the Agency's ability to protect 
individuals who volunteer to participate in clinical trials, one of its 
primary statutory missions. The Agency would be precluded from 
protecting a segment of the population from insufficient information, 
misinformation and, at worst, fraud. The Federal Food, Drug and 
Cosmetic (FD&C) Act created a process that carefully balances the need 
for scientific support for new products with the overriding need to 
protect human subjects. H.R. 3677 upsets this balance and could lead to 
harm or possibly death for some patients.
    Under the FD&C Act, FDA has been provided the authority to regulate 
the use of investigational (unapproved) drugs. As an element of this 
authority, FDA regulates clinical human investigation conducted by a 
sponsor. Under statutory and regulatory authority, sponsors wishing to 
use investigational, unapproved drugs in human patients must file an 
Investigational New Drug application (IND) with FDA. This IND is either 
an IND that is designed to involve a number of patients or can be filed 
as a single patient IND to treat one patient.
    The Food and Drug Administration Modernization Act of 1997 (FDAMA) 
codified and expanded FDA's programs to provide access to unapproved 
products in section 561 of the FD&C Act. This was done after extensive 
review and consideration of the needs of patients. The intent was to 
provide access to therapies for which there was no effective, approved 
therapy and particularly for patients who had failed existing approved 
therapy. The provisions are clear in requiring the individual 
physician, FDA and the sponsor of the investigational drug to go 
through a series of steps before allowing the drug to be made available 
to the patient. These ultimately require a balancing of the risks and 
benefits of the proposed administration of the investigational drug. As 
part of the access program, the sponsor is still required to file 
either a single patient IND or an IND to treat a small number of 
patients.
    FDA is obligated to evaluate an IND submission within 30 days and 
make a determination if the clinical investigation should proceed or 
the investigation should be put on clinical hold. The primary focus of 
this decision is the safety of the patients who may receive the 
unapproved drug. The evaluation includes a determination of the nature 
and level of risk to the patients who may be the subject of the 
clinical investigation, design of the clinical investigation, various 
qualifications of the investigators, the disease being investigated and 
other information about the drug. The same evaluation is done for 
submissions that may be considered single patient INDs or emergency 
INDs, although FDA makes the decision in a considerably shorter time 
period than 30 days and in the case of an emergency IND usually within 
24 hours or less.
    FDA has, in rare instances, placed on clinical hold a proposal to 
administer an investigational drug about which little was known to 
treat a serious or life threatening disease when there was known 
effective, or life saving, therapy for that condition. We did so 
because those subjects would have been exposed to an unreasonable and 
significant risk of illness or injury. This is also required under FDA 
regulations governing clinical holds. (Title 21, Code of Federal 
Regulations Sec. 312.42). The risk in these cases was not simply the 
possible untoward effects of the investigational agent but also the 
risk of substituting a known effective therapy with something that 
might have no effect. Although these circumstances occur rarely and are 
dwarfed in numbers by those clinical trials allowed to proceed, the 
ramifications of being unable to put a particular investigation on hold 
would be dramatic.
    FDA understands the seriousness of placing any investigation on 
clinical hold and does so only after following a careful process that 
includes deliberation with internal experts and, if necessary, 
consultation with external experts about the particulars of the disease 
state and potential for available therapies.
    The ability to put an investigation on clinical hold is the primary 
mechanism FDA has to protect human subjects who are being asked to 
participate in a clinical investigation and asked to be part of an 
experiment with unapproved investigational drugs. H.R. 3677 contains 
several provisions that could impair the ability of FDA to put clinical 
investigations on hold even if a patient's safety is being compromised 
and could prevent FDA from taking action specifically for an individual 
patient who may be at a high level of risk.
    Section 2 (a) of H.R. 3677 amends section 505(i)(3) of the FD&C Act 
by limiting the ability of FDA to impose a clinical hold on an 
investigation. The new provision would prohibit a clinical hold even if 
another therapy is determined to be safer because it is a known 
curative therapy. The entire basis for this prohibition on FDA is that 
the patient has, in effect, waived their right to informed consent. The 
patient, by declaring in writing that s/he is ``aware of the comparable 
or satisfactory alternative therapy . . . aware of the risk involved in 
receiving the drug in the investigation, and chooses to receive the 
drug notwithstanding such risk and notwithstanding the comparable or 
satisfactory alternative therapy'' could prevent the imposition of a 
clinical hold even for safety reasons which may be directly related to 
the individual patient's safety.
    The bill creates a situation in which research subjects may be 
presented with incomplete and/or biased information by investigators 
who often have substantial personal, financial or professional 
interests at stake in the research. FDA is concerned that the consent 
obtained under these circumstances will not be truly informed consent 
because it will not be based on a thorough and objective explanation of 
the risks and benefits of both the unapproved investigational product 
and the proven treatment that is being foregone.
    H.R. 3677 does not even require that the sponsor administering the 
unapproved investigational drug provide all the information to the 
patient typically contained in an informed consent. The idea that a 
patient could essentially sign away their rights to adequate health 
care to anyone who is able to convince them to do so, without oversight 
and without any assurance of proper information, is offensive and 
unethical. Even absent mal-intent, the most well intentioned 
investigator is unlikely to have access to all that is known about an 
unapproved investigational drug very early in its development.
    Furthermore, since FDA has been provided the authority to oversee 
the use of unapproved investigational drugs, the Agency is the one 
central location that collects data on those unapproved investigational 
drugs. FDA is often the only party in possession of information from 
different sponsors that could impact the decision as to whether an 
unapproved drug should be used in a clinical investigation. There is no 
means of ensuring that the patient actually knows all of the risks 
involved. Reliance on the sponsor of the investigational drug to 
provide all the known risks is misplaced since that particular sponsor 
may not have access to all of the information known about a drug. Thus, 
through no fault of the sponsor, complete information on risk may not 
be provided to the patient considering the use of the unapproved 
investigational drug.
    The importance of clinical hold as an Agency tool to help ensure 
the protection of human subjects in clinical trials is illustrated by 
the recent example of the Jesse Gelsinger case. At age 18, Mr. 
Gelsinger volunteered to participate in a clinical trial of a gene 
therapy product and died shortly after the administration of the 
product. The investigator who recruited Mr. Gelsinger to the trial did 
not follow the terms of his protocol and did not reveal important 
safety information during the informed consent process, so that Mr. 
Gelsinger's consent was not fully informed. When FDA learned of this 
subject's death, the Agency put this trial on clinical hold so that no 
other patients would be subjected to the dangers inherent in this 
trial.
    Under H.R. 3677, FDA would have no ability to protect subjects even 
if the Agency had information that practitioners were using dishonest 
means to coerce patients into participating in trials in lieu of taking 
proven therapy. If H.R. 3677 is enacted and such ethically suspect 
practices are allowed to proliferate, it also could seriously undermine 
the public's confidence in the process of conducting biomedical 
research in this country with dangerous consequences to the public 
health.
    There are other technical drafting problems with H.R. 3677 which 
are not included in this submission. Even if technical changes were 
made, however, FDA would still have concerns with the impact of the 
proposed legislation.
    FDA has a long, and successful, history of expediting access to 
investigational agents for those who patients with serious and life 
threatening illnesses who have no satisfactory therapy available to 
them. We also have a long and successful history of protecting patients 
who are most vulnerable, including those with life threatening 
illnesses who are desperately seeking help and hope.
                                 ______
                                 
                                Lupus Foundation of America
                                                   November 9, 2000
The Honorable Michael Bilirakis
Chairman, Subcommittee on Health & Environment
U.S. House of Representatives
Committee on Commerce
2125 Rayburn House Office Building
Washington, DC 20515
    Dear Mr. Chairman: In response to your letter of November 2, 2000, 
I am pleased to submit the attached answers to the questions prepared 
by members of the Subcommittee on Health & Environment. Please contact 
me if I can provide additional information.
    It was a pleasure to appear before your Subcommittee on behalf of 
the 1.4 million Americans with lupus. I want to express my sincere 
gratitude to you and the Members and staff of the Subcommittee for your 
support of the Lupus Research & Care Amendments of 2000 Act.
            Sincerely,
                                              Tomiko Fraser
                                              National Spokesperson
        Answers to Questions submitted by Subcommittee members.
    Question 1. Why does lupus seem to affect women of color more often 
than Caucasian women?
    Response. This is the subject of a research project currently 
underway by NIH. (Lupus in Minorities Study, or LUMINA) We believe 
lupus has a genetic basis and it appears that the genes suspected of 
causing lupus might be more prevalent among women of color.
    Question 2. Are manifestations of lupus more serious among African-
Americans than among Caucasians?
    Response. African Americans have more kidney involvement than 
Caucasians. Researchers may have located a gene believed to cause 
kidney disease in African American lupus patients.
    Question 3. Can lupus be prevented, or the health impact minimized 
in any way?
    Response. Unfortunately, you can't prevent lupus. However, you can 
take steps to minimize health effects of lupus, such as adopting a 
healthy lifestyle, such as avoiding stress, getting plenty of rest, 
eating well, light exercise, and following your doctor's advice. 
Diagnosing lupus early, and seeing a doctor regularly can minimize 
damage to vital organs. The earlier lupus is diagnosed and treated, the 
more likelihood of preventing the need for more expensive treatment.
    Question 4. Do we know why lupus strikes mostly young women?
    Response. The exact cause of lupus is unknown, but researchers 
believe lupus has a genetic basis. Hormonal influences may explain why 
lupus affects mostly women in their childbearing years. But we don't 
know if there is something about women that makes them more vulnerable 
to developing lupus, or if there is something about men that protects 
them from lupus.
    Question 5. Why is lupus so expensive to treat?
    Response. Lupus requires constant medical attention by a number of 
specialists, requiring many tests to monitor the status of the immune 
system. Because the disease usually presents multiple symptoms, people 
with lupus must take many medications. It is common for people with 
lupus to take over a dozen medications. In addition, monitoring lupus 
activity requires many expensive tests to determine the functioning of 
the immune system and many vital organs, such as the kidneys, lungs, 
heart, and brain.
    Question 6. What impact does lupus have on the work force or 
corporate community?
    Response. One in five people with lupus is disabled. Many victims 
of lupus must cut back their work hours, or change jobs to reduce 
stress. This results in lost productivity for corporations, and costs 
millions of dollars. In addition, disability costs rise to cover people 
with lupus.
    Question 7. What is The financial impact of lupus on the rest of 
the family?
    Response. It's difficult to measure, but it has to be significant. 
On average lupus costs $6,000 to $10,000 annually to treat, but some 
victims incur costs of several thousand dollars a month. This level of 
expense can have an enormous impact on the family budget.
    Question 8. Does the environment play a role in causing lupus?
    Response. Yes, while lupus has a genetic basis, we know that 
certain environmental factors trigger disease activity, including UV 
light, infections, certain chemicals and drugs, and stress. There is 
much interest in this area of research, particularly in regard with the 
role breast implants may have in causing an autoimmune reaction.
    Question 9. Are we seeing more cases of lupus today than in the 
past?
    Response. It's hard to determine the exact number of lupus cases. 
We don't know if lupus is on the rise, but doctors are getting better 
at diagnosing the disease, which may be the reason we are seeing more 
cases of the disease. However, some researchers do believe that lupus 
is increasing among young women.
    Question 10. Can lupus be treated with less expensive herbal or 
complementary therapies?
    Response. There is considerable interest in this area. Some 
therapies, when used in combination with a doctor's care, can help 
reduce symptoms, but they are no substitute for traditional medicine. 
Lupus is a dangerous disease that needs constant monitoring by a 
trained doctor. The FDA has given fast track designation to a new 
therapy for lupus using the hormone, DHEA.
    Question 11. Are there new treatments in the pipeline?
    Response. Yes, there are several therapies undergoing clinical 
trials that will address various manifestations of lupus, but the 
disease is so complicated, no magic pill will ever cure lupus. It will 
take much more research. Some therapies include biologics that can 
block the immune system from producing autoantibodies.
    Question 12. What is the outlook for a cure for lupus?
    Response. While there has been progress, a cure is not on the 
horizon. We are getting better at treating symptoms of the diseases and 
patients are living longer, but there is no cure for the foreseeable 
future.
                                 ______
                                 
 Response of James Navarro to Questions of the Subcommittee on Health 
                            and Environment
    Question 1. Mr. Navarro, in your opinion, who should decide the 
course of medical treatment for your son: you, or the FDA?
    Response. This answer requires very little thought. The Food and 
Drug Administration (FDA) is an agency made up of many different people 
from many schools of thought. These people many times as we have 
discovered, get caught up in defending a position, or a particular 
discipline of medicine and often forget the ``PATIENT'S RIGHT TO 
CHOOSE''. Often, the patient is ignored entirely. We as parents of a 
terminally ill child, have only one agenda, and that is to see our son 
survive, with dignity and quality of life. Agencies and doctors are 
often driven by egos and money, as we have tragically experienced 
during our journey with Thomas. Nobody and I mean nobody, will ever 
have his best interest at heart, more so than his mother, Donna, and I 
do.
    Question 2. My understanding is that the FDA denied Thomas access 
to a clinical trial because he had not first gone through and failed 
chemotherapy and radiation. Why did you decide to forgo this treatment?
    Response. Before I can answer this question, we must first clarify 
a very important fact which is quite often ignored, that fact is that, 
``RADIATION AND CHEMOTHERAPY HAVE EVER BEEN APPROVED BY THE FDA FOR USE 
IN TREATING PEDIATRIC CANCERS'', it has merely become the unopposed 
standard of care for treatment, because anyone who stands in opposition 
to its use is either crushed as far as career advancement goes, or is 
dismissed as a charlatan as history has bore out. Chemotherapy alone 
represents a $107,000,000,000.00 ($107 Billion) a year industry. Our 
history unfortunately has always born out that profit always comes 
before people.
    It is important to remember that ``STANDARDS OF CARE'' does not 
mean that a treatment has been thoroughly tested and adequate evidence 
reviewed by the FDA or other Federal Agency to assure safety and 
efficacy. A ``STANDARD OF CARE'' is not and should not be an 
endorsement by a Federal agency that has never evaluated the data. 
``STANDARDS OF CARE'' are protocols that doctors in cancer research 
centers have developed according to their own research as the best 
treatment option according to their perspective in how to treat cancer.
    Now, to be more specific, our reasons for not wanting to use these 
``STANDARDS OF CARE'', on our son were much simpler. The simple fact is 
these treatments are extremely dangerous to use, and more often than 
not, end in permanently damaging or even killing the patient. Quite 
simply, if it is all a ``crap shoot'', we as parents are compelled to 
start with a treatment which will do the least amount of harm first. 
The following is a list of just some of what Thomas would suffer as a 
result of being treated by Radiation and Chemotherapy: FATIGUE, NAUSEA, 
VOMITING, ABDOMINAL CRAMPING, HAIR LOSS, LOSS OF IQ, LOSS OF HEARING, 
LOSS OF MEMORY, FLUID IN THE MIDDLE EAR, HYPOTHYROIDISM, SPINAL GROWTH 
DEFICIT, HYPOPITUITARISM, SECONDARY TUMORS, LOW LEVEL HORMONES (For the 
rest of his life), RADIATION NECROSIS, KIDNEY NECROSIS, AND DEATH FROM 
EITHER RADIATION POISIONNG OR CYTOTOXIC POISIONING. These are just some 
of the possibilities we faced with Thomas. You must also understand 
that every child we have met during Thomas's journey that was treated 
using the ``STANDARD OF CARE'', is now dead, the most recent we buried 
just two weeks ago. The choice we made was not a difficult one. We 
chose life. We knew through careful research that there were other 
possibilities for Thomas, we never thought our government would stand 
against us in making those choices.
    Question 3. According to the administration's written testimony, 
the intent of current law is to protect ``our most valuable citizens, 
those who are desperately ill'' and that the FDA believes this bill 
would undermine FDA's ability to help assure reasonable safety and 
effectiveness of subjects in clinical trails and informed consent for 
patients given access to experimental therapies.'' In that 
Administration statement, with what do you agree or disagree?
    Response. The opening statement of this question reminds me of the 
old axiom; ``THE ROAD TO HELL IS PAVED WITH GOOD INTENTIONS.'' First, 
all citizens of this great country should be considered valuable, not 
just the sick, and in keeping with that thought, it is we the people 
who should be allowed to decide what is best for us. Second of all, the 
Agency's roll is to monitor safety and effectiveness, not to sit in 
judgment, deciding who can and can't introduce new promising therapies: 
as is the case with the treatment we choose for Thomas. For almost 
twenty years, the FDA has stood as a roadblock to progress, instead of 
helping to advance a therapy which shows great promise in treating the 
specific cancer that Thomas suffers from. Third, as far as informed 
consent goes, this is a process that we personally have seen abused and 
the FDA and HHS does nothing to correct it. There needs to be better 
accountability at the FDA and amongst the Doctors. Until or unless that 
happens, no expanded authority in the decision making process should be 
considered. The role of both the doctor and the FDA should be to assist 
us in making decisions by providing us with all of our treatment 
options and offering their best advice. It is not the role of the FDA 
or the doctor to take control of our lives and forcing us to submit to 
their will.
    Question 4. H.R.3677 precludes the FDA from establishing a clinical 
hold on the basis that there is a comparable or satisfactory 
alternative therapy if the patient is 1) aware of the other therapy; 2) 
aware of the risk associated with the investigational drug; and 3) 
chooses to receive the drug. How would this bill affect you and others 
you may have met during the ordeal you have faced in getting your son 
the treatment you have decided to be best? Do you see ways in which 
this bill might be abused by charlatans pushing treatments that do not 
work?
    Response. Again we see in the opening statement, the FDA, usurping 
a patient's right to choose. Understand that ALL CANCER THERAPIES ARE 
EXPERIMENTAL AT THIS TIME IN OUR SEARCH FOR THE CURE, and to deny a 
patient access to their personal choice of treatment based on a 
personal bias is IMMORAL. The bill does not open the door for 
charlatans to abuse the law anymore than current laws do. This bill 
would have allowed us access to the therapy of our choice, a therapy 
that would not have destroyed our son as conventional treatment would. 
Also understand that if we had been allowed access to it and it had not 
worked, we would have stepped our mode of treatment to a more 
aggressive treatment like chemotherapy or radiation. But to be FORCED 
into a treatment from the onset is wrong. The FDA will still be allowed 
to put clinical trials on hold that have safety concerns. It is the 
role of the FDA to advance science monitoring the safety of trials 
involving human subjects. It is not the role of the FDA to pick one 
trial over another, or to stand in the way of advancing science by 
excluding access to a clinical trial for a ``STANDARD OF CARE'' that 
has never been through an FDA approval process for the specific 
condition or age group in question.
    Question 5. In a news account entitled ``PARENTS FIGHT TO SAVE 
SON'', published by Wired News, the article stated that ``When the 
Navarro's decided they wanted their son to be treated by Burzynski, the 
FDA denied them permission, ruling that the treatment could only be 
used as a last resort. FDA officials threatened to take Thomas into 
protective custody if the Navarro's denied him traditional treatment.'' 
Do you have any documentation you can provide the Committee that the 
FDA, or any other government agency, was going to place your son in 
protective custody?
    Response. First of all, I would like to state for the record that I 
was unaware of this article until I read of it in your questions from 
the subcommittee. With that said, I found a copy of the article and 
read it. Now in answer to your question:
    It is true that the FDA denied Thomas access to treatment at the 
Burzynski Clinic, citing that there was no scientific, ethical, or 
moral basis for allowing Thomas access to treatment when an existing 
``STANDARD OF CARE'' was already available. It didn't matter to them 
that the ``STANDARD OF CARE'' had such devastating and deadly results. 
It didn't matter to them that in the ``STANDARD OF CARE'' two of the 
three recommended chemotherapy drugs stated on the package insert, 
``this product not proven safe or effective in the pediatric 
population.'' It was their way of doing business, and to that end their 
word was final.
    In following their logic for a moment, if you have used the most 
damaging and deadly treatment first, what is left of the patient to 
treat if the patient has been rendered permanently damaged, or worse, 
if the patient has died? At this point, treatments that would offer as 
great or greater a hope have been rendered useless by the FDA's 
decision making process. In effect, ``THEY HAVE THROWN THE BABY OUT, 
WITH THE BATH WATER''.
    It is incorrect that the FDA threatened to take Thomas into 
protective custody. In actuality, When my wife and I made the decision 
not to subject Thomas to the standard of care, Thomas's oncologist back 
in Arizona swore out a complaint, alleging CHILD ABUSE, CHILD 
ENDANGERMENT, AND MEDICAL NEGLECT for refusing a treatment which had 
discovered would leave our son retarded, deaf, sterile, blind, with 
stunted growth, and even dead. WHAT IS WRONG WITH THIS PICTURE? It is 
our undaunted opinion, that the current ``STANDARD OF CARE'' is 
LEGALIZED CHILD ABUSE. What loving parent would knowingly and willing 
subject their child to this type of torture with no guarantee of 
success, knowing that there were other treatments available, that 
didn't do this type of damage that hadn't been tried first.
    Question 6. Critics at the FDA state that by pursuing alternative 
therapies that are ``not FDA approved'', you are placing your ``son at 
greater risk of death'' than if he first pursued FDA approved therapies 
that include radiation and chemotherapy, that may render your son to be 
retarded. How would you respond to your critics at the agency?
    Response. First, I would like to remind my critics at the FDA of an 
oath they took many years ago. And I quote, ``FIRST, DO NO HARM'', it 
is the beginning of the Hippocratic Oath. With that said, let me start 
by saying, I agree, unapproved therapies can present risks. Some are 
great and some are small. The risk is no greater than the risks taken 
by using Radiation and Chemotherapy, which again I will remind you are, 
`` NOT FDA APPROVED FOR TREATING PEDIATRIC CANCERS''. They are standard 
treatments used, which ``IMPLIES APPROVAL'', it does not mean that have 
been approved going through the ``NORMAL APPROVAL PROCESS''. LET US NOT 
CONFUSE THE TWO! If under any other means, if I were to go out with 
forethought and blind my son, or deafen my son or by some means, cause 
him to become retarded I would be arrested, tried, and thrown into 
jail, and rightfully so, but it is done everyday in the treating of 
childhood cancer with NO ACCOUNTABILITY . And not allowing my son to be 
subjected to these medieval treatments, makes me a bad parent in the 
eyes of the ``STANDARD OF CARE COMMUNITY''. There is nothing in my life 
that I love greater than my children, and they will be no doctor's 
``LAB RAT'', not while I breath.
    I hope this has clarified any questions you might have. Please feel 
free to contact me if you have any further questions.
                                 ______
                                 
             National Organization for Rare Disorders, Inc.
                                                  November 15, 2000
The Honorable Michael Bilirakis
Chairman
Subcommittee on Health and the Environment
US House of Representatives
2369 Rayburn House Office Bldg.
Washington, DC 20515
    Dear Chairman Bilirakis: Thank you for your letter of November 2, 
containing additional questions from the September 13, 2000 hearing on 
H.R. 4242, the Orphan Drug Innovation Act. My answers to the questions 
follow:
    Question 1. Do you believe that shielding the original orphan drug 
from competition for longer than 7 years is good policy?
    Response. No. The Orphan Drug Act of 1993 provides seven years of 
exclusive marketing rights to the sponsor of an orphan drug, and no 
manufacturer should have more than seven years without competition. 
However, there are many orphan drugs that have had no competition after 
seven years on the market simply because no other companies have made 
an effort to compete after the innovator's exclusivity expired.
    On the other hand, the European Union enacted the Orphan Medical 
Products Regulation in December 1999, providing ten years of 
exclusivity to orphan drug manufacturers. Since this is a new law, we 
do not yet know how successful it will be in comparison to the U.S. 
law. In other words, is ten years of exclusivity a better incentive 
than seven years? Will more companies develop orphan drugs in Europe 
than the United States because American incentives are not as strong? 
We believe it is premature for Congress to consider revising (either 
contracting or expanding) orphan drug exclusivity in the United States 
at this time.
    Question 2. Do you believe that this policy needs clarification?
    Response. Under very limited circumstances the FDA may allow the 
manufacturer of a ``similar'' orphan drug to reach the American market 
before an innovator's orphan drug exclusivity expires IF it can prove 
that the follow-on drug is ``different'' from the original orphan drug. 
A manufacturer must prove that its drug is chemically or structurally 
different from the first orphan drug, or that it is superior because it 
is more effective or safer, or represents a ``major contribution to 
patient care.'' FDA's regulations defining these requirements were 
established in 1992. We believe the regulations have worked very well 
for many years, and they do not need to be revised at the current time.
    Please note that the orphan drug regulations were printed nine 
years after the Orphan Drug Act became law, they went through extensive 
public comment periods, and they have been time tested since 1992. Mr. 
Chairman we believe these regulations effectively carry out the spirit 
and intent of the law, and they should NOT be written into law because 
minor changes would subsequently require an act of Congress. 
Regulations are easier to change when the need arises, and the public 
is encouraged to comment and provide input into revised regulations.
    Question 3. On February 7, 2000, you sent a letter to FDA 
Commissioner Henney stating, ``As you probably know, we have been 
concerned about the blockage of a new version of beta interferon form 
multiple sclerosis. FDA seems to believe that the product is the same 
as both Avonex and Betaseron, even though those two products have 
already been determined by both FDA and a federal court to be 
`different.' This puts Serono in a no-win situation, even with its full 
BLA and having been initially determined to be `the same' as Betaseron 
under the Orphan Drug Act.'' Why did you write that the FDA 
implementation of the Orphan Drug Act puts Serono in a no-win 
situation?
    Response. If you read my entire letter of February 7, 2000 to FDA 
Commissioner Henney, you will clearly see that the question I raised 
involved FDA's refusal at the beginning of this year, to decide whether 
Avonex was the ``same'' as Betaseron or the ``same'' as Avonex, but I 
asserted it could not be the same as BOTH. The letter was an effort to 
force the agency to decide which ONE of those two drugs Rebif is the 
same as.
    Let me explain. At the beginning of this year, FDA determined that 
Rebif is the same as the two competing versions of the multiple 
sclerosis treatments, Betaseron and Avonex. Betaseron was the first 
version of beta interferon to reach the market, but FDA subsequently 
decided that Avonex was a ``clinically superior'' orphan drug, and 
therefore should be made available to multiple sclerosis patients 
before Betaseron's exclusivity expired. The manufacturer of Betaseron 
went to court to stop Avonex from reaching the market, claiming that 
its orphan drug exclusivity would be violated. The court decided that 
FDA's decision was correct and that Avonex is a ``different'' drug 
because it was ``clearly superior,'' and thus could be approved by the 
agency despite Betaseron's exclusivity.
    Late last year, FDA told Serono that it could not approve its 
multiple sclerosis treatment, Rebif, because it was the ``same'' as 
both Betaseron and Avonex. I wrote to Commissioner Henney in February 
explaining that FDA cannot claim Rebif is the ``same'' as both of these 
drugs because the FDA had already determined, and a federal court had 
agreed, that Betaseron and Avonex are not the same drug. Serono was in 
a no-win situation because the company would have to prove that Rebif 
was ``different'' than two different drugs, not one. I urged the 
Commissioner to determine which of those two Multiple Sclerosis 
treatments Rebif was the ``same'' as. I did not urge the Commissioner 
to cast a vote toward one drug or the other; we simply wanted a 
decision to be made. Later this year FDA did make the decision that 
NORD was asking the FDA to make, and the agency decided that Rebif is 
the ``same'' as Avonex. Apparently Serono disagrees with that decision.
    Note. The answer to the following three questions is written below:
    Question 4. Can you reconcile your written testimony today with 
your February 7 letter to FDA Commissioner Henney where you state, in 
reference to 7-year market exclusivity enjoyed by Biogen, ``However, 
after seven years expire, competitors should be allowed on the market 
without undue delay, and the beta interferon scenario, as a precedent, 
very troubling.''
    Question 5. Would you consider your February 7, 2000 letter to FDA 
Commissioner Henney to be at variance with your testimony today?
    Question 6. What caused you to change your mind?
    Response. My testimony on September 13 conforms to the statements 
made in the February 7 letter to Commissioner Henney. The beta 
interferon scenario was very troubling because there was no way that 
Rebif could be the ``same'' as both Avonex and Betaseron! It could only 
be the same as ONE of those drugs.
    I believe that FDA was wrong for not making a decision on this 
matter much sooner, and for keeping their decision confidential when it 
was made. Unfortunately, FDA is required under law to keep all 
information confidential unless a drug sponsor agrees to make it 
public. It was only after Serono gave written permission to FDA to talk 
to me about Rebif that I was told the agency's scientific analysis 
concluded that Rebif is the ``same'' as Avonex, and not the same as 
Betaseron. This means FDA cannot approve Rebif for sale in the United 
States until the exclusivity of Avonex expires. If Serono disagrees 
with this decision, the onus is on the company to scientifically prove 
their drug is not chemically the ``same'' as Avonex, or to prove their 
drug is ``clinically superior'' to Avonex. I understand that Serono is 
conducting clinical trials now in an effort to prove clinical 
authority.
    Question 7. The testimony of your organization was requested by 
Biogen, the company that has been trying to thwart Serono's legislative 
activities in this area. Have you had any contacts with Biogen or its 
agents on this matter?
    Response. I have had no contact with Biogen on this or any matter. 
I am not surprised that Biogen recommended that we testify at the 
hearing because we are usually invited to testify at all hearings 
concerning the Orphan Drug Act. In fact, I was quite surprised when I 
learned there would be a hearing about orphan drugs, and we were not 
invited to testify. I finally received a telephone request from your 
staff a few days before the hearing and was delighted to change my 
plans and go to Washington, DC. I had no idea that Biogen made this 
recommendation, and let me assure you NORD is not conspiring with 
anyone to keep Serono off the market.
    Since Biogen had no way to know what I would say in NORD's 
testimony, it was certainly considerate of them to suggest that NORD be 
included. However, people in Washington generally know that NORD is 
always fair and unbiased, it is the quintessential patient advocacy 
organization, it does not favor one drug over another, one company over 
another, and NORD can always be relied on to defend the Orphan Drug Act 
and its regulations.
    Question 8. Do you believe that co-marketing during the original 
orphan's exclusivity period, protection for that innovation, and the 
opportunity to market that advantage are enough of an incentive to 
foster research and development investment to improve orphan drugs? If 
not why not?
    Response. This is a very complicated question, but the basic answer 
is: Exclusivity is the most important incentive of the Orphan Drug Act, 
and any weakening of exclusive marketing rights would greatly undermine 
development of future treatments for rare diseases. If you allow orphan 
drug exclusivity to be violated because a manufacturer makes a minor 
change to a drug that has no substantial benefit to patients, then you 
will have diluted the ODA's chief incentive, and fatally weakened the 
law.
    Mr. Chairman, when a pharmaceutical company decides which drugs to 
invest in, they prefer to focus R&D on the least risky products. This 
is why so many drug companies invest billions of dollars in research 
and development of ``me-too'' drugs, compounds that are very similar 
and vary only slightly from other marketed drugs that are very 
successful and profitable. They practically copy the original compound 
and make some minor chemical or structural changes so that they do not 
violate the innovator's patent, and then they get the drug on the 
market to compete with the original drug. Historically this is why we 
have so many beta blockers on the market for hypertension, so many 
anti-inflammatories for arthritis, and soon there will be many drugs 
for erectile dysfunction.
    This is the way the marketplace works so well for drugs that are 
used by large numbers of people. But orphan drugs affect few people, 
manufacturers know there is a limited potential market to buy their 
drug, and they want assurance that a competitor will not take part of 
their market away. The industry did not manufacture orphan drugs before 
the Orphan Drug Act was enacted (e.g., only ten orphan drugs were 
brought to market in the 10 years before the Orphan Drug Act became 
law); but since 1983, over 200 orphan drugs have reached the American 
market.
    These companies will tell you they would not have invested in 
development of their orphan drug if they were not guaranteed seven 
years of exclusivity.
    In the very few instances when FDA has approved a ``similar'' 
orphan drug before the exclusivity of the first drug expired, it has 
only occurred when manufacturers AGREED to SHARE exclusivity, or the 
follow-on company PROVED SCIENTIFICALLY that their drug is clinically 
superior.
    In the case of the three versions of beta interferon the first 
version (Betaseron) caused a very serious injection site reaction 
necessitating surgery. The second version (Avonex) does not cause this 
very serious adverse reaction, so it was allowed on the market. This is 
a safety advantage, and it would have been unjust to prevent Avonex 
from reaching Multiple sclerosis patients. Rebif on the other hand has 
not yet proven that it is clinically superior to Avonex, FDA has 
determined that Rebif is scientifically the ``same'' as Avonex, but it 
also causes the same serious injection site reactions as Betaseron.
    The onus is now on Serono to prove that their drug is clinically 
superior to Avonex. The company is conducting a clinical trial right 
now, comparing Avonex to Rebif. We believe that the scientists at FDA 
will then be able to determine whether Rebif is clinically superior to 
Avonex. Thus Congress should await the results of that trial and allow 
FDA to make that determination. We believe that tinkering with the law 
now, and diluting the exclusivity incentive of the Orphan Drug Act, 
will weaken the law and remove any incentive for manufacturers to 
develop clinically superior orphan drugs that patients need.
    Question 9. Do you believe FDA's policy should distinguish between 
drugs that are clinically superior based on improved safety or efficacy 
from those based on being a major contribution to patient care?
    Response. We believe FDA's current regulations defining clinical 
superiority are right on target, and they should be left as they are. 
If and when FDA decides to change the regulations, the agency is 
required to publish the proposed revisions in the Federal Register, and 
patients will then have an opportunity to express their point of view. 
Today the agency will only approve a competing orphan drug if it can 
prove that it is safer, more effective, or is a major contribution to 
patient care. Patients want and need these therapeutic improvements. 
The current regulations act as an important incentive to companies to 
develop better orphan drugs.
    Question 10. Do you object to Congress providing FDA with guidance 
on handling clinically superior drugs, an issue not currently covered 
in the statute?
    Response. We do not believe it is necessary or warranted for 
Congress to provide guidance to FDA on handling ``superior'' orphan 
drugs. Firstly, we recommend that Congress ought to leave these 
decisions up to the physicians and dentists at FDA. Secondly, although 
``clinical superiority'' is not specifically mentioned in the statute, 
it was absolutely proper and necessary for the agency to develop this 
regulation because the terms ``same'' and ``different'' in the statute 
had to be defined in regulations.
    The FDA's orphan drug regulations define the terms ``same drug'' 
and ``different drug'' because recombinant DNA technology made this 
necessary. Seventeen years ago when the law was written we were dealing 
solely with chemical compounds that could easily be differentiated. 
Today, however, many new treatments are developed through biotechnology 
engineering and the differences between biologics is very difficult to 
discern. One simply cannot define the chemical structure of a biologic 
and determine if it is the same or different from a ``similar'' 
biologic. Most of these products are copies of proteins or enzymes that 
human bodies naturally make. Moreover, Congress cannot develop a 
formula that fits every clinically significant difference, and which 
factors should be considered. For example, is a three-hour intravenous 
infusion clinically superior to a six-hour infusion? Does a lower price 
for a follow-on orphan drug represent clinical superiority? Once you 
ask questions like these, you open a very big can of worms.
    In conclusion Mr. Chairman, the patient community is indebted to 
Congress for enacting the Orphan Drug Act in 1983. We advise that if 
``it ain't broke, don't fix it,'' nor its regulations. Serono is doing 
the right thing now; they are conducting clinical trials to try to 
prove scientifically that its drug is superior to other competing 
drugs. This process is available to them under the current Act and its 
regulations. NORD's concern is not which company is right or wrong, 
nor, how much profit they may lose when FDA denies them early marketing 
approval, but whether patients are suffering because of lack of access. 
There are three good FDA approved treatments for multiple sclerosis 
available to American patients today, and at best any clinical 
superiority that Serono will claim will not indicate that Rebif is a 
cure for multiple sclerosis. If it were, FDA would approve it! Rebif 
may be a treatment that is superior in some ways, but inferior in 
others. Only science can tell us, and we await results of its clinical 
trials comparing it directly to Avonex.
    Please do not hesitate to contact me if you have any other 
questions.
            Very truly yours,
                                            Abbey S. Meyers
                                                          President
                                 ______
                                 
                             University of Virginia
                                    Department of Radiology
                                                  November 10, 2000
Congressman Michael Bilirakis
Chair, Subcommittee on Health and Environment
U.S. House of Representatives
Rayburn House Office Building, Room 2125
Washington, DC 20515-6115
    Dear Mr. Bilirakis: The following are my responses to the questions 
in your letter of November 2, concerning my testimony on HR 1795.
    Question 1a. Why is an institute necessary to solve the problems 
associated with biomedical imaging research at the NIH?
    Response. That imaging research is spread over 16 institutes and 
centers means that there is no single institute charged with 
responsibility for support of basic research to develop new imaging 
techniques and technologies with broad applications to the diseases and 
organ systems that are the focus of the existing institutes. 
Consequently, such research, which is critical to advances in imaging, 
receives little or no support from the NIH. There is also no 
coordination of imaging research at NIH. Major opportunities are lost, 
as they are not apparent in the content of the institutes' disease 
focus. There is duplication, as there is little coordination of imaging 
opportunities. Given the declining ability of both academic departments 
and industry to finance imaging research, an institute directed 
specifically at imaging, with a comprehensive plan, able to prioritize 
and pursue opportunities is essential.
    Question 1b. Could the Bioengineering Consortium (BECON) that the 
NIH has established, or a coordinating committee of some type be an 
alternative?
    Response. Such alternatives fall short of what is needed in both 
scope and authority. An institute has the capability of addressing the 
diverse needs that are essential to successfully addressing the 
nation's needs with respect to medical imaging, including the authority 
to fund grants, set a research priorities through the request for 
applications process, and enable research training. This level of 
comprehensiveness does not exist through any other mechanism.
    Question 2. The National Cancer Institute has made imaging a top 
research priority and has put much more money into this field. Can't we 
solve the problem by further increasing the amount of money that NCI 
commits to imaging?
    Response. While more NCI money certainly can increase imaging 
research in cancer, it does not address the need to advance imaging 
research more broadly in support of the nation's health. Indeed, it is 
this ``silo'' approach to imaging that is so ineffective. Imaging is 
applicable across a broad range of organ systems and diseases and 
needs, as such, to be addressed more directly.
    Question 3. Other groups have come to the Congress requesting that 
new institutes be created. How can Congress distinguish among these 
proposals?
    Response. The Institute of Medicine has advised Congress in a 1984 
report titled Responding to Health Needs and Scientific Opportunity: 
The Organizational Structure of the National Institutes of Health--on 
when it should consider establishing a new institute. The proposed 
Institute for Biomedical Imaging and Bioengineering fulfills all of 
these criteria and is also consistent with a second IOM report, which 
was written in 1998 and titled Scientific Opportunities and Public 
Needs: Improving Priority Setting and Public Input at NIH. 
Establishment of the proposed institute will advance research that will 
have a positive impact on the public health while, at the same time, 
reducing inefficiencies and duplication. Dr. Reed Dunnick discussed 
both IOM reports at greater length in his testimony.
    Question 4. What role can new imaging technologies play in the 
advanced research in molecular biology and genetics that is conducted 
by the other institutes at the NIH?
    Response. Both advances in established imaging technologies and the 
emergence of a host of new technologies promise spectacular 
contributions to our understanding of the early phases of such 
important disease processes as cancer and heart disease. Medical 
imaging is, in essence, the ``noninvasive biopsy'' that can provide 
insight into how subcellular structures are altered by disease in both 
their morphology and function. Imaging technologies already are being 
used for these purposes. Their importance in investigation, diagnosis, 
and treatment will grow over the near term.
    Question 5. What makes the basic scientific research involved in 
imaging and bioengineering different from the scientific research at 
the disease and organ-system institutes?
    Response. The most fundamental difference is in the nature of the 
training and expertise of the individuals involved. Medical imaging 
technology development requires the skills of physicians, computer 
scientists, physicists, mathematicians, information technology 
specialists, and engineers. The research is interdisciplinary and cuts 
across disease and organ system lines.
    I appreciate the opportunity to further comment on the need for an 
Institute for Biomedical Imaging and Bioengineering. Please let me know 
if you wish me to address these or other issues further.
            Sincerely,
                                               Bruce J. Hillman, MD

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