[House Hearing, 108 Congress]
[From the U.S. Government Publishing Office]





 HARNESSING SCIENCE: ADVANCING CARE BY ACCELERATING THE RATE OF CANCER 
                      CLINICAL TRIAL PARTICIPATION

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

                                HEARING

                               before the

                              COMMITTEE ON
                           GOVERNMENT REFORM

                        HOUSE OF REPRESENTATIVES

                      ONE HUNDRED EIGHTH CONGRESS

                             SECOND SESSION

                               __________

                              MAY 13, 2004

                               __________

                           Serial No. 108-189

                               __________

       Printed for the use of the Committee on Government Reform


  Available via the World Wide Web: http://www.gpo.gov/congress/house
                      http://www.house.gov/reform


                                 ______

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                     COMMITTEE ON GOVERNMENT REFORM

                     TOM DAVIS, Virginia, Chairman
DAN BURTON, Indiana                  HENRY A. WAXMAN, California
CHRISTOPHER SHAYS, Connecticut       TOM LANTOS, California
ILEANA ROS-LEHTINEN, Florida         MAJOR R. OWENS, New York
JOHN M. McHUGH, New York             EDOLPHUS TOWNS, New York
JOHN L. MICA, Florida                PAUL E. KANJORSKI, Pennsylvania
MARK E. SOUDER, Indiana              CAROLYN B. MALONEY, New York
STEVEN C. LaTOURETTE, Ohio           ELIJAH E. CUMMINGS, Maryland
DOUG OSE, California                 DENNIS J. KUCINICH, Ohio
RON LEWIS, Kentucky                  DANNY K. DAVIS, Illinois
JO ANN DAVIS, Virginia               JOHN F. TIERNEY, Massachusetts
TODD RUSSELL PLATTS, Pennsylvania    WM. LACY CLAY, Missouri
CHRIS CANNON, Utah                   DIANE E. WATSON, California
ADAM H. PUTNAM, Florida              STEPHEN F. LYNCH, Massachusetts
EDWARD L. SCHROCK, Virginia          CHRIS VAN HOLLEN, Maryland
JOHN J. DUNCAN, Jr., Tennessee       LINDA T. SANCHEZ, California
NATHAN DEAL, Georgia                 C.A. ``DUTCH'' RUPPERSBERGER, 
CANDICE S. MILLER, Michigan              Maryland
TIM MURPHY, Pennsylvania             ELEANOR HOLMES NORTON, District of 
MICHAEL R. TURNER, Ohio                  Columbia
JOHN R. CARTER, Texas                JIM COOPER, Tennessee
MARSHA BLACKBURN, Tennessee          ------ ------
PATRICK J. TIBERI, Ohio                          ------
KATHERINE HARRIS, Florida            BERNARD SANDERS, Vermont 
                                         (Independent)

                    Melissa Wojciak, Staff Director
       David Marin, Deputy Staff Director/Communications Director
                      Rob Borden, Parliamentarian
                       Teresa Austin, Chief Clerk
          Phil Barnett, Minority Chief of Staff/Chief Counsel


                            C O N T E N T S

                              ----------                              
                                                                   Page
Hearing held on May 13, 2004.....................................     1
Statement of:
    Christian, Dr. Michaele, Associate Director, Division of 
      Cancer Treatment and Diagnosis, Cancer Therapy Evaluation 
      Program, National Cancer Institute; and Dr. Richard Pazdur, 
      Director, Division of Oncology Drug Products, Center for 
      Drug Evaluation and Research, U.S. Food and Drug 
      Administration, accompanied by Dr. Patricia Keegan, 
      Director of the Division of Therapeutic Biological Products    12
    Pecora, Dr. Andrew, chairman and director, the Cancer Center, 
      Hackensack University Medical Center; Dr. Robert Comis, 
      president and Chair, Coalition of National Cancer 
      Cooperative Groups; and Ellen Stovall, president and chief 
      executive officer, National Coalition for Cancer 
      Survivorship...............................................    74
Letters, statements, etc., submitted for the record by:
    Christian, Dr. Michaele, Associate Director, Division of 
      Cancer Treatment and Diagnosis, Cancer Therapy Evaluation 
      Program, National Cancer Institute, prepared statement of..    15
    Comis, Dr. Robert, president and Chair, Coalition of National 
      Cancer Cooperative Groups, prepared statement of...........    82
    Davis, Chairman Tom, a Representative in Congress from the 
      State of Virginia, prepared statement of...................     4
    Murphy, Hon. Tim, a Representative in Congress from the State 
      of Pennsylvania, prepared statement of.....................    11
    Pazdur, Dr. Richard, Director, Division of Oncology Drug 
      Products, Center for Drug Evaluation and Research, U.S. 
      Food and Drug Administration, prepared statement of........    44
    Pecora, Dr. Andrew, chairman and director, the Cancer Center, 
      Hackensack University Medical Center, prepared statement of    77
    Stovall, Ellen, president and chief executive officer, 
      National Coalition for Cancer Survivorship, prepared 
      statement of...............................................   100
    Waxman, Hon. Henry A., a Representative in Congress from the 
      State of California, prepared statement of.................     8

 
 HARNESSING SCIENCE: ADVANCING CARE BY ACCELERATING THE RATE OF CANCER 
                      CLINICAL TRIAL PARTICIPATION

                              ----------                              


                         THURSDAY, MAY 13, 2004

                          House of Representatives,
                            Committee on Government Reform,
                                                    Washington, DC.
    The committee met, pursuant to notice, at 10:03 a.m., in 
room 2154, Rayburn House Office Building, Hon. Tom Davis of 
Virginia (chairman of the committee) presiding.
    Present: Representatives Tom Davis of Virginia, Duncan, 
Murphy, Carter, Waxman, Cummings, Kucinich, Watson, Van Hollen, 
and Norton.
    Also present: Representative Garrett.
    Staff present: David Marin, deputy staff director/director 
of communications; Drew Crockett, deputy director of 
communications; Teresa Austin, chief clerk; Brien Beattie, 
deputy clerk; Susie Schulte, professional staff member; Corinne 
Zaccagnini, chief information officer; Robert White, press 
secretary; Kristin Amerling, minority deputy chief counsel; 
Josh Sharfstein, minority professional staff member; Earley 
Green, minority chief clerk; Jean Gosa, minority assistant 
clerk; and Naomi Seiler, minority staff assistant.
    Chairman Tom Davis. Good morning. A quorum being present, 
the committee will come to order.
    I want to welcome everybody to today's oversight hearing on 
cancer clinical trials. This hearing will examine the status of 
efforts to bring innovative cancer treatments to patients and 
discuss how to change the face of cancer into a less terminal 
and more treatable disease.
    The two panels of witnesses today will present testimony on 
the various factors contributing to low accrual of adult 
patients in cancer clinical trials and what efforts are being 
taken to obtain reasonable participation levels to better 
provide more treatment options for cancer patients.
    Cancer is the second leading cause of death in the United 
States, taking the lives of over a half million Americans each 
year, more than 1,500 people each day. Roughly 1.3 million new 
cancer cases are diagnosed in this country each year. These 
statistics are sobering. All of us here today know a relative 
or friend who has been diagnosed with some type of cancer. 
Anyone who has been affected by cancer understands the needs 
for more and better treatment options for patients.
    In order for new drugs and therapies to be approved by the 
Food and Drug Administration, several cancer clinical trials 
must be conducted. Clinical trials are essential for 
determining safe and effective therapies in modern medicine. 
Early detection of cancer and the application of new treatments 
available through clinical research are responsible for 
significant improvements in cancer survival rates. Clinical 
trials are designed to answer scientific questions which 
translate into better and less toxic therapies for patients. 
Trials allow doctors and researchers to gain information about 
the benefits, side effects, possible applications, and doses of 
new and existing drugs.
    In order for scientists and oncologists to make accurate 
conclusions about an experimental new drug's effect, clinical 
trials require the participation of numerous cancer patients. 
Further, research has shown that trial participants nearly 
always receive equivalent or better care than those receiving 
standard treatments, despite the experimental nature of these 
investigational treatments.
    Clinical trials can offer patients advanced treatments that 
would be otherwise unattainable. Thousands of people are helped 
each year by joining cancer clinical trials, and millions of 
people have ultimately benefited from others' participation in 
trials.
    So we pose the question to our panel of witnesses today: 
why do only 3 percent of adults nationwide enroll in centers 
when up to 20 percent are eligible? And what efforts are being 
taken to resolve the barriers to better clinical trials and 
adequate adult enrollment?
    We want to examine the different scientific, logistical, 
and financial realities that interact and impede reasonable 
participation in adult trials. The lack of patient and 
physician education about clinical trials, problems traveling 
to the trial sites, strict eligibility criteria, and third 
party payer reimbursement policies prevent a large number of 
patients from participating. As a result of these contributing 
factors, a vast majority of cancer patients fail to even 
consider clinical trials when reviewing their treatment 
options.
    We will hear today from the cancer community the urgency to 
reverse this situation and resolve the barriers to adequate 
adult enrollment in clinical trials. Clinical trials are 
essential for improving outcomes in cancer patients. By 
improving participation levels and creating more trials to new 
test therapies, we can transform cancer into a more treatable 
and less fatal disease.
    The equation is simple: clinical research leads to 
discovery of new and better therapies for cancer patients, 
helping them live longer and improving their quality of life.
    I know all of our witnesses this morning will agree that we 
need to boost participation in clinical trials. Along with 
improving accrual rates, we may need to consider improving 
other ways our health community approaches cancer. Clinical 
trials are just a single component of the cancer spectrum. I 
understand the complexity of the disease and the intricacies 
surrounding the discovery, development, and delivery of 
treatments. I look forward to a constructive dialog on this 
topic.
    The committee welcomes our witnesses for this important 
testimony today.
    I would now yield to my colleague, Henry Waxman, for an 
opening statement.
    [The prepared statement of Chairman Tom Davis follows:]

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    Mr. Waxman. Thank you, Mr. Chairman. I am pleased to 
participate in this hearing on how to accelerate progress 
against cancer. This is a topic that I've worked on for many 
years. When I was the chair of the Health Subcommittee of the 
Energy and Commerce Committee, I was responsible for 
legislation reauthorizing the National Cancer Institute. We 
worked with experts inside and outside of the Government to 
improve rehabilitation services, expand research on 
reproductive cancers, and strengthen education and grantmaking 
decisions.
    It has also been my priority to make sure that all 
Americans have access to benefits of medical progress against 
cancer, and I'm particularly proud of legislation that expanded 
access to screening for breast and cervical cancer and of 
legislation that provided Medicaid coverage for those who are 
found on screening to have these tumors.
    Today's hearing highlights how much more needs to be done. 
Over the last several decades, while rates of heart disease 
have dropped dramatically, rates of cancer have largely 
remained stable. Despite progress against a few specific 
tumors, cancer will kill an estimated 500,000 Americans in 
2004.
    The simplest and quickest way to make a dramatic reduction 
in cancer in the United States is to prevent it. Every Member 
of Congress knows that the No. 1 preventable cause of cancer in 
the United States is the cigarette. Last year a committee 
advising the Department of Health and Human Services 
recommended a simple evidence-based plan to help 5 million 
people quit smoking and save 3 million lives. The plan was 
endorsed by former Surgeons General Dr. Julius Richmond, Dr. 
David Satcher, and Dr. C. Everett Koop. Unfortunately, the Bush 
administration has shelved this report, and this Congress has 
not held a single hearing to discuss or review its 
recommendations.
    This week, New York City announced its smoking rates have 
dropped 11 percent in just 1 year as a result of Mayor 
Bloomberg's aggressive anti-tobacco policies, saving an 
estimated 30,000 lives. The response to this news should be 
obvious. The President and congressional leaders should pursue 
how to replicate these achievements across the country. But 
don't hold your breath. The National Republican Party is so 
closely aligned with the tobacco industry that the only 
hearings we have had on tobacco in the House recently have 
highlighted the alleged health benefits of smokeless tobacco, 
unbelievable as that is. That's the only hearing that has been 
held on the topic of tobacco.
    Today's hearing will focus on the challenges facing 
clinical research in cancer. Let me mention two issues at the 
outset. First, to find cures for cancer we must adequately 
support a clinical research infrastructure that can prove that 
cures work. I'm very concerned that Dr. Robert Comis, a senior 
oncologist who represents the Cooperative Groups program, will 
testify today that current finding stifles innovation; 
destabilizes key functions such as our tissue banks, data 
management, and informatics platforms; and acts as a 
disincentive to both academic and community physician 
participation in research. That's because of these current 
funding levels. Now that reductions in reimbursement for 
oncologists mandated by Congress are due to take effect, it is 
critical that NCI and Congress assure adequate funding for 
research.
    Second, to provide access to clinical trials, it is 
important that Government resources such as 
www.clinicaltrials.gov work well. This is a Web site created by 
the Congress in 1997 that is supposed to contain information 
for patients about ongoing trials for serious and life-
threatening disease such as cancer. A 2003 study by the FDA 
staff found that fewer than half of the cancer studies that are 
legally required to be listed on this Web site were actually 
listed by the companies. This lack of participation by the drug 
industry in an important resource for patients is inexcusable, 
and I'm disappointed that the PHRMAceutical Research and 
Manufacturers Association, PHRMA, which was invited to testify, 
has been unable to send a witness to this hearing.
    Today we will hear from leading health officials at the 
National Cancer Institute and the Food and Drug Administration, 
from senior cancer researchers, and from a leading 
representative of cancer patients. I thank these distinguished 
witnesses for coming today. I look forward to their testimony.
    [The prepared statement of Hon. Henry A. Waxman follows:]

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    Chairman Tom Davis. Thank you very much. Do any other 
Members wish to make statements? If not, any statements can be 
put in the record.
    Mr. Murphy.
    Mr. Murphy. Sure.
    Chairman Tom Davis. The gentleman is recognized.
    Mr. Murphy. Mr. Chairman, thank you for holding this 
hearing on cancer screening trials. We all know few issues have 
as negative an impact upon so many as cancer. Though all of us 
have not been diagnosed, we certainly all hold the roots of 
this disease and we all know someone close to us who has or has 
had some impact of cancer.
    While modern medicine has brought us a long way toward 
winning this battle, anyone who has a parent or child or friend 
diagnosed with cancer knows all too well we have not come 
anywhere near far enough. Increased participation in cancer 
clinical trials would significantly increase the discovery of 
newer cancer treatments with the ability to keep cancer 
patients feeling better while undergoing treatment, add years 
to their lives, and even cure them of the disease altogether; 
however, we will never have enough adult volunteers if patients 
continue to be misinformed or not encouraged to participate.
    It is certainly sad to me to read the survey cited that the 
National Cancer Institute revealed that 85 percent of cancer 
patients are either unaware or unsure of participation in 
clinical trials is an option, and of the few who are aware of 
the trials, most of these individuals believe clinical trial 
treatment would be less effective than standard care, or that 
their insurance would not cover the cost, so something has to 
be done to change the public's perception of these trials.
    I know in southwestern Pennsylvania the University of 
Pittsburgh Medical Center has received two grants to study 
these barriers and improve minority participation in clinical 
trials. This grant money is being used to essentially bring the 
trials to the patient through the utilization of 
teleconferencing, video equipment in outlying hospitals, 
providing modes of transportation to bring patients to trials 
and treatment, and working with other cancer centers and 
hospitals outside of the University of Pittsburgh Medical 
Center system.
    Improving access and public perception of cancer clinical 
trials may seem to be an overwhelming task, but the doctors in 
my District are committed and excited about the cause.
    I'm looking forward to the witnesses' testimony on the 
various steps groups are voluntarily taking to achieve higher 
rates of participation, as well as some of the problems they 
are confronting in their efforts.
    In addition, I appreciate the committee's efforts to raise 
national awareness of this issue crucial to the future of 
medical innovation.
    Thank you, Mr. Chairman.
    [The prepared statement of Hon. Tim Murphy follows:]

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    Chairman Tom Davis. Thank you very much.
    The gentlelady from California, Ms. Watson?
    Ms. Watson. I, too, want to add my thanks to you, Mr. 
Chair, for bringing this issue up. I'm sure that Congressman 
Waxman will remember that way back in the 1980's a group of us 
got together in the California Legislature, a group of females, 
when we found that the only cancer testing was done on males, 
and breast cancer was coming more into high profile.
    My statement ends up this way: when you are making good 
public policy, it takes years because you have to educate. So I 
thank you for gathering the witnesses here that will educate us 
to let us know that clinical trials are a must if we are going 
to make a dent in cancer.
    Thank you, Mr. Chairman.
    Chairman Tom Davis. Thank you very much.
    We have a great selection of witnesses today. In our first 
panel we have Dr. Michaele Christian of the National Cancer 
Institute, along with Dr. Richard Pazdur from the Food and Drug 
Administration. They are going to provide the committee with an 
overview of the Federal Government's role in cancer clinical 
trials and highlight efforts the Government is taking to 
increase participation in clinical trials.
    It is the policy of the committee that we swear all 
witnesses, so just rise with me and raise your right hands.
    [Witnesses sworn.]
    Chairman Tom Davis. Thank you very much. We are very 
privileged to have both of you here today. Your entire 
testimony is a part of the record and has been read, and what 
I'd like to do is we have some lights that will be in front of 
you. The green light goes on for 4 minutes, then you get an 
orange light for a minute, and then red is the end of 5. Try to 
sum up, and then we can move right to questions. Your entire 
statement is in the record.
    Dr. Christian, we will start with you and then to Dr. 
Pazdur. Thanks for being with us.

   STATEMENTS OF DR. MICHAELE CHRISTIAN, ASSOCIATE DIRECTOR, 
  DIVISION OF CANCER TREATMENT AND DIAGNOSIS, CANCER THERAPY 
EVALUATION PROGRAM, NATIONAL CANCER INSTITUTE; AND DR. RICHARD 
 PAZDUR, DIRECTOR, DIVISION OF ONCOLOGY DRUG PRODUCTS, CENTER 
     FOR DRUG EVALUATION AND RESEARCH, U.S. FOOD AND DRUG 
ADMINISTRATION, ACCOMPANIED BY DR. PATRICIA KEEGAN, DIRECTOR OF 
        THE DIVISION OF THERAPEUTIC BIOLOGICAL PRODUCTS

    Dr. Christian. Good morning. Thank you Mr. Chairman, 
Representative Waxman, and Members for the opportunity to 
discuss NCI's efforts to deliver innovative and effective 
cancer treatments to the public.
    I am Michaele Chamblee Christian, a medical oncologist and 
associate director of the Division of Cancer Treatment of the 
National Cancer Institute.
    I have slides which are going to be hard to see, I think, 
on these screens, but you have hard copies. Cancer is actually 
more than 100 complex diseases and as depicted on this first 
slide, cancer cells have many potential targets and pathways 
and processes that have been subverted by the malignant 
process. These differ in different patients and different tumor 
types, yet, because of our investment in basic research and 
advances in our understanding of biology, we now have a growing 
list of new agents and chemical entities in clinical trials and 
unprecedented opportunities to make significant progress in the 
treatment and prevention of cancer.
    NCI funds an extensive clinical trial system, including 
over 3,000 clinical trial sites, more than 13,000 clinical 
investigators that accrue over 30,000 patients each year to 
trials. We study 138 investigational or experimental drugs. And 
this system has been very effective at developing the 
treatments and defining the standards of care for patients we 
treat today, and contributing to the fact that more patients 
with a diagnosis of cancer are living longer today.
    We also have 88 formal clinical trials agreements within 
companies the biopharmaceutical industry, which is the source 
of most of the promising new agents in clinical development 
today.
    Because of these extensive relationships, NCI is in a 
unique position to sponsor clinical trials of combinations of 
investigational agents owned by different companies. NCI has 
worked with over a dozen industry collaborators to arrange more 
than 20 trials of novel investigational combinations to date, 
and more are in development. Many of these regimens would not 
have been evaluated until one or more of the agents had 
received FDA marketing approval, potentially resulting in years 
of delay.
    Why is this important? You have copies of the slides. We 
are going to try to do this without the slides. The cartoon 
that I was going to show you depicts one of the common problems 
and challenges with the targeted therapy of cancer, and that is 
that there are multiple branching and redundant signal pathways 
that control the behavior of cancer cells, and it is widely 
believed that many of the most promising new molecularly 
targeted agents will demonstrate their optimal utility in 
combinations that inhibit or modulate multiple targets in these 
critical pathways blocking progression of cancer, so we need to 
be able to give these simultaneously, and that's why these 
combinations are important.
    I wanted to tell you about the components of the clinical 
trials program. There is an extensive early clinical trials 
program which is comprised of phase one and phase two clinical 
trials done via contracts and grants at academic centers. There 
are numerous translational research components that conduct the 
correlative of laboratory studies on blood and tumor specimens 
from patients so that in clinical trials we may learn not only 
whether a treatment works but how it works, so that we can 
select future patients better for treatment.
    The map that you saw up there at one point represents the 
distribution of these clinical trial sites around the country 
and shows a very broad and I think good distribution of these 
sites.
    The next slide should have been on Cooperative Groups 
funding, since funding for clinical trials is an issue, and 
points out that during the period from fiscal year 1998 to 2003 
funding to the Cooperative Groups program increased by 62 
percent.
    NCI was asked to comment on why Cooperative Groups are not 
fully funded at the levels recommended by peer review, and I 
wanted to point out that the Cooperative Groups grants are 
amongst the largest in NCI's portfolio. In addition, each phase 
three trial that we sponsor costs anywhere from $2 million to 
$10 million, depending on its size. Our groups undergo peer 
review once every 6 years, where plans for the next 6 years are 
reviewed, along with the requested budget. However, this is a 
projected plan and a provisional budget because it is not 
possible to predict which clinical trials will actually be 
conducted 4 or 5 years in the future. So peer review 
recommendations are one component of effective coordination and 
stewardship that NCI staff consider in arriving at a funding 
plan.
    In the next slide I wanted to show you that during this 
same period accrual to clinical trials also rose dramatically, 
by 24 percent in the phase three program and, importantly, by 
58 percent, as shown in the light blue bars, in the early 
clinical trials program. That's important because that's where 
many of these promising new agents and combinations that will 
be evaluated in phase three trials are initially studied.
    So my final slide just points out that there are a number 
of ongoing initiatives at NCI to broaden access to clinical 
trials for patients and to facilitate physician participation. 
The slide lists a number of them.
    I wanted to just comment also in closing, that NCI is 
committed to effectively integrating its clinical trials 
mechanisms in order to make smarter use of the available 
resources and to ensure that we are optimally positioned to 
take advantage of emerging scientific and medical 
opportunities, speed accrual to the highest priority clinical 
trials, and accelerate the delivery of promising new approaches 
to cancer prevention and treatment to the American public.
    I thank you for the opportunity to testify, and I will be 
happy to answer any questions.
    Chairman Tom Davis. Thank you, Doctor.
    [The prepared statement of Dr. Christian follows:]

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    Chairman Tom Davis. Dr. Pazdur, thanks for being with us.
    Dr. Pazdur. Mr. Chairman and members of the committee, I am 
Richard Pazdur, M.D., the Director of the Division of Oncology 
Drug Products at the Center for Drug Evaluation and Research at 
the Food and Drug Administration. Dr. Patricia Keegan, the 
Director of the Division of Therapeutic Biological Oncology 
Products at CDER, is accompanying me today to answer any 
questions on biological products.
    I am pleased to be with you today to discuss what our 
agency is doing to accelerate the delivery of innovative cancer 
treatments to meet the needs of cancer patients and their 
families. FDA's mission is to ensure that new cancer drugs are 
safe and effective. We also facilitate access to promising 
therapies for seriously ill and dying patients when no other 
treatment is available.
    Since the FDA last testified before this committee in June 
2000, a number of important cancer drugs have been approved and 
are helping cancer patients. Of particular note are the number 
of innovative drugs that are targeted to specific parts of the 
cancer cells. These new therapies are a glimpse of the future 
of cancer therapy and should be a source of encouragement to 
the American public and to cancer patients and their families.
    FDA has numerous programs in place to help speed the 
development and approval of promising drugs to cancer patients. 
Let me briefly mention some of these programs.
    Under the accelerated approval route, FDA can approve drugs 
for serious or life-threatening conditions. These drugs 
demonstrate the potential to address unmet medical needs based 
on a surrogate end point that is ``reasonably likely'' to 
predict clinical benefit.
    Second, priority review is intended to direct overall FDA 
review attention and resources to the evaluation of 
applications for products that have the potential for providing 
significant therapeutic advances.
    Third, a drug sponsor may request fast track status. This 
designation facilitates the investigational development and the 
approval of drugs that provide significant advancements in the 
treatment of serious or life threatening diseases. These 
programs have been instrumental in shortening the time to 
approval for many promising cancer treatment drugs; however, 
the FDA is aware that there is growing concern that many of the 
new basic science discoveries made in recent years may not 
quickly yield more effective, affordable, and safe medical 
products for patients. This is because the current medical 
product development path is becoming increasingly challenging. 
During the last several years, the number of new drugs and 
biological applications submitted to the FDA has declined 
significantly. The number of innovative device applications has 
also decreased.
    In response, on March 16, 2004, the FDA released a report 
entitled, ``Advancing America's Health: Advancing Medical 
Breakthroughs.'' We refer to this FDA report as a critical 
path. This timely paper calls for academic researchers, product 
developers, and patient groups to work with the FDA to identify 
ways to modernize tools for speeding approval, innovative 
products to the market to improve public health.
    The report provides FDA's analysis of the current pipeline 
problem, the recent slow down instead of the expected 
acceleration in innovative medical therapies reaching patients. 
FDA is planning an initiative that will identify and prioritize 
the most pressing development problems, and, second, the areas 
that provide the greatest opportunities for rapid development 
and public health benefits. This will be done for all three 
dimensions along the critical pathways, namely: safety 
assessment, evaluation of Maryland utility, and product 
industrialization. We will work together with stakeholders to 
identify the most important challenges.
    Concurrently, FDA will refocus its internal efforts to 
ensure that we are working on the most important problems and 
intensify our support of key projects.
    Thank you for the opportunity to discuss these important 
issues with you. I would be happy to answer any questions.
    [The prepared statement of Dr. Pazdur follows:]

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    Chairman Tom Davis. Thank you both. Let me start. We'll 
take 5 minutes.
    Dr. Christian, the witnesses on our second panel today are 
going to cite lack of physician education as a barrier to 
adequate accrual in clinical trials. You talked briefly, but I 
wonder if you could elaborate on what efforts the National 
Cancer Institute has taken to inform physicians about the 
cancer trial support unit program. I mean, it seems to me 
that's where the word needs to get out. People go see their 
doctor, they get the diagnosis, they want a list of options. I 
think you'd just be teaming with people who want to get in on 
the latest and be a part of this, but it really starts and ends 
with the physician that they're treating who really gives them 
their menu of options.
    Dr. Christian. Well, I think that is absolutely correct. 
The NCI and the Cancer Trial Support Unit have worked with a 
number of organizations, the Coalition for Cooperative Groups 
being one, since they are intimately involved in that, but also 
with the American Society for Clinical Oncology and others, to 
make physicians aware of the opportunities to participate in 
trials through the Cancer Trials Support Unit. They also use 
electronic systems----
    Chairman Tom Davis. Let me ask this. In continuing medical 
education, do you have requirements of that? I don't know if 
you have it in every State, but in most States is this part of 
it?
    Dr. Christian. To my knowledge it is not part of continuing 
medical education requirements. We do offer sessions, however, 
at national meetings so that there are educational 
opportunities there, but it is not a requirement, per se.
    Chairman Tom Davis. I mean, it just seems to me when you're 
going in every year to get updated, that menu option, it's 
pretty easy to include it. If I were a physician, I'd sure want 
to know it and be able to give my patients that option, 
particularly some of those that don't have a lot of good 
options, as you're working on.
    Dr. Christian. Can I just elaborate also?
    Chairman Tom Davis. Yes, please.
    Dr. Christian. We have also, working with the Cooperative 
Groups and others, invested quite heavily in patient education, 
because having patients be aware of clinical trials and 
requesting those opportunities of their physicians, I think is 
another way to approach this problem, and so we have, I think, 
all made major efforts to improve the education of patients 
about these opportunities.
    Chairman Tom Davis. I think you touched briefly, but Dr. 
Comis of our next panel is going to testify that the National 
Cancer Institute only reimburses physicians about $2,000 per 
case to perform research in community-based physician 
practices, while studies suggest the cost is closer to $4,000. 
Is it possible, working with NCI's budget, to increase the 
allocation to offset these costs? Physician reimbursements are 
low on everything. This is an area where you really don't want 
to do it on the cheap.
    Dr. Christian. Well, the cost at a site for conducting 
clinical trials has been a subject of great interest and 
concern to us. During the 5-year period that I showed on my 
slide, there was a 62 percent increase in funding to the group 
system overall. But importantly, I think, funding to the sites 
actually doubled during that period. So while $2,000 remains 
perhaps meager, it represented a substantial increase in what 
we had been able to fund previously. So we are continuing to 
seek ways to increase funding and to think about how best to 
allocate the clinical trials resources that we have overall so 
that we get the work done.
    There are a number of groups--Dr. Comis and I work on 
several of them--that are actually trying to nail down much 
more precisely what the real components of cost are so that we 
can make, I think, more effective judgments in terms of how we 
are able to fund.
    Chairman Tom Davis. I don't mean to single you out. Our 
reimbursements that we allocate to physicians under Medicare 
and Medicaid are pitiful, so I hear you.
    Dr. Pazdur.
    Dr. Pazdur. I just wanted to jump in. Having done clinical 
trials for almost 20 years, there is a difference between 
knowing if they exist and actually doing them. OK? And I think 
people have to understand that the enrollment of a patient when 
one is conducting a busy practice is a very time consuming 
activity. It requires infrastructure to be present. It requires 
data managers. It requires research nurses. So there has to be 
an infrastructure within a clinician's practice that will allow 
him to do the clinical trials that he views to be of 
importance.
    It is an issue, and I think when we take a look at the 
whole issue of why patients do not participate in clinical 
trials, to leave out the physician component is one that would 
not be appropriate. It is, as you pointed out, the physician 
that ultimately will be prescribing the therapy that the 
patient will be getting, and he has to have the appropriate 
incentives and also the infrastructure that will allow him to 
place patients on trial.
    Chairman Tom Davis. Let me ask you, just so I know, 
understand, how many investigational products fail in clinical 
trials? I mean, the ratio compared to products approved, any 
idea? Ball park?
    Dr. Pazdur. I could--the vast majority of them. It's a 
small--probably our critical pathway states that one--if you 
take five drugs that are being developed in their very earliest 
stages, probably one will make it to the market.
    Chairman Tom Davis. OK. Thank you very much.
    Mr. Waxman.
    Mr. Waxman. Dr. Christian, the Clinical Trials Cooperative 
Groups Program at NCI, they play a key role in clinical 
research against cancer. I know you're familiar with that. 
Studies by these networks of researchers have led to major 
advances in survival, particularly in the pediatric cancers, 
but we have a chart showing that the funding for this program 
has not increased in the last several years. And Dr. Comis, who 
is chair of the Coalition of National Cancer Cooperative Groups 
is going to testify that current funding stifles innovation, 
destabilizes key functions such as our tissue banks, data 
management, and informatics platforms, and acts as a 
disincentive to both academic and community physician 
participation. You can see from that chart there is a decrease 
for leukemia, gynecologic oncology, breast, and bowel cancer. 
How do you respond to the view that this important research 
program is threatened?
    Dr. Christian. Well, I can't see the details there, but my 
slide also covered, I think, some of those years, and, as I 
point out, we actually substantially increased funding over a 
significant portion of that time. Now, with that said, I think 
that there is no doubt that the costs for enrolling a patient 
at a site still are not being reimbursed at the proper rate. I 
think that is something that we continue to need to address.
    You know, I think that there are many components of the 
clinical trials program. There is the Cooperative Groups 
Program, which is extremely important, as you point out, but 
there are many other critical elements, too, and I think that, 
given all of the new targeted agents that are coming into the 
clinic, the early clinical trials are extremely important, too, 
in advancing regimens to the point that they can go to phase 
three trials, and, indeed, many of those have been funded at 
much higher rates and, in fact, new resources have been created 
to try to get those new novel agents into a point where they 
can go to phase three trials, so----
    Mr. Waxman. Do you agree that with less money and rising 
expenses the Cooperative Groups will have a harder time 
recruiting patients and doctors to their clinical trials?
    Dr. Christian. I think that with less money at each site 
that would be true, and our approach to that actually is to 
look at the entire clinical trials program and find ways to 
better integrate, to make the cost of putting patients on 
clinical trials lower by having consistent and standard 
approaches to data collection and other things that cost time 
and money. Data managers, as Dr. Pazdur pointed out, are one 
important component.
    So we are trying to look across the system and find places 
where we can better integrate, conserve resources, and then 
allocate them to the places that need them the most so that we 
can, indeed, raise the funding at the sites where the research 
is actually being done.
    Mr. Waxman. Given the NCI's emphasis on the genetics of 
cancer and the importance of tissue banks, what is NCI doing to 
enhance the role of the tissue banks run by the Cooperative 
Groups?
    Dr. Christian. Well, NCI actually has put forward a request 
recently for proposals for funding for tissue banks. We are 
planning not only to increase the funding for tissue banks 
because we agree that it is a really critical component of this 
research, but to actually stabilize that funding by awarding 
grants specifically to that purpose. So, rather than just 
general Cooperative Groups funding, we are going to provide 
additional funds specifically targeted to tumor banks in our 
Cooperative Groups.
    Mr. Waxman. As NCI reorganizes itself to deal with the 
challenges of the future, I think it is important not to 
undermine those resources such as the Cooperative Groups that 
are the bedrock of our clinical research efforts, and I'm sure 
you agree with that, as well.
    Dr. Christian. I agree absolutely.
    Mr. Waxman. Dr. Pazdur, clinical trials are a key tool in 
our fight against cancer, but they also can provide one of the 
few sources of hope for those people who have failed standard 
treatment options. The Web site, www.clinicaltrials.gov, is a 
searchable, online registry of clinical trials that patients 
with serious or life-threatening disease and illness and their 
providers can use to see if they are eligible for 
participation. I'm sure you're familiar with this Web site.
    Now, Federal law requires that the sponsor of any 
effectiveness study conducted under an investigational new drug 
proposal register with the data base within 21 days of the 
start of the patient enrollment. In April 2003, FDA reported 
that many sponsors of clinical studies of cancer treatments had 
not submitted the information to the registry. While 91 percent 
of NIH- and NCI-sponsored studies had been posted, only 47 
percent of industry-sponsored studies had been posted, even 
though FDA had released a detailed guidance for industry in 
March 2002. Do you think increased industry compliance would 
benefit patients?
    Dr. Pazdur. The answer to your question is emphatically 
yes. We also are greatly concerned about the low participation 
of industry in listing their trials on www.cancertrials.gov. We 
have taken a concerted effort to try to find why, what is the 
reason, and I really don't have a good reason at that time if I 
could say, you know, this is the reason why industry is not 
placing studies on the Web site. One would think they would 
have every reason to place their trials on. If we're talking 
about poor accrual to clinical trials, industry can't cry about 
poor accruals to clinical trials if they are not putting it on 
the Web site.
    In our own division and at the FDA, in general, after every 
phase two meeting and industry meeting we have a written bullet 
that is part of the minutes to that meeting that specifically 
informs the sponsor of the existence and their obligation to 
list the Web site. That is a written part of the minutes of 
every end of phase two meeting.
    We've taken concerted efforts to talk to patient groups, to 
encourage patient advocates to advocate for participation of 
commercial sponsors to list their trials. In addition to that, 
we've taken a concerted effort of talking to industry about 
this.
    There may be some concerns of confidentiality in listing 
clinical trials on www.clinicaltrials.com. I really don't buy 
that, Mr. Waxman.
    Mr. Waxman. Well, I'm disappointed we couldn't get a PHRMA 
representative come in and testify today. It's ironic that, 
while they're refusing to comply fully with the Government Web 
site, PHRMA has a Web site, and on that PHRMA Web site there 
are--it's called, ``New medicines and development,'' and on the 
PHRMA Web site they'll often list clinical trials that they're 
not listing on the Government Web site, but on their Web site 
they frequently don't include eligibility details on clinical 
trials or provide contact information for the trials. I don't 
know why companies may list information about studies in a form 
that's not useful to the patients, because if you read about 
it, you want to find out more about it, there's no contact 
information.
    What is FDA going to do to enforce participation in the Web 
site?
    Dr. Pazdur. At this point we obviously have a process where 
we are communicating with the sponsors. We are trying to find 
out reasons why they are not putting the trials on this. We 
have begun an education program, as I pointed out, where at 
every meeting we are asking them and informing them of their 
obligation to do so.
    In addition to that, we have a massive process during 
professional education to encourage physicians and to encourage 
patients to utilize this resource. We are somewhat limited on 
what we can do. We can educate, we can talk.
    Mr. Waxman. Well, thank you very much.
    Chairman Tom Davis. Thank you very much, Mr. Waxman.
    Judge Carter.
    Mr. Carter. Thank you, Mr. Chairman.
    Richard Nixon was President of the United States in 1970 
and he declared war on cancer. He said our goal was to cure 
cancer in our lifetime. Well, he's dead. We haven't even come 
close. We spent $52.5 billion under the theory the Government 
could come up with a solution, and it is my understanding--and 
please correct me if I am wrong--that if you view the overall 
war on cancer, it has been a pretty bad failure. If you compare 
it to other wars in health areas, like heart disease, for 
instance, there has been much more success. Are we doing 
something wrong in our direction on the war on cancer as you 
see it? That's the question that's really concerning me, 
because where I come from there's a lot of folks dying of 
cancer. They don't see a whole lot of hope and they don't see a 
whole lot of success.
    Do either one of you want to comment on why this--$52 
billion and 35 years--hasn't succeeded in any way?
    Dr. Pazdur. I hear you. OK. I sympathize wholeheartedly, 
having had patients and family members with this disease. I 
think it is important for us to understand that cancer is not 
one disease. As Michaele pointed out, at this time we can say 
it is 100 diseases, but probably it is even many more diseases 
on a molecular basis.
    The riddle of cancer I feel is far, far more complicated 
than, for example, the problems that we face with HIV 
infections where we know the ideology, we know the virus that 
is causing the problems. For most cancers we have very limited 
or rudimentary knowledge of what causes the cancer, and even 
sometimes the natural history and the variations.
    For example, even what we call breast cancer probably is 
hundreds and hundreds of different diseases. The problem is the 
science. We have to make scientific advances, and that is on 
the basic level. We are beginning to do so, I feel, by 
understanding the genetic causes of cancer. We have seen some 
drugs that have dramatically changed the face of some diseases. 
While you are entirely right, if you took at look at the entire 
scope of cancer, one would have to look at it somewhat 
pessimistically. However, there are some diseases--for example, 
CML, a disease that when I began my medical career that was 
uniformly fatal with drugs that we used from the 1950's to 
treat it, which now we have a drug called Glevic, which 
transformed that disease into probably a chronic disease. One 
can't use the word ``cure'' at this time with many of these 
diseases, but clearly was an important medical development.
    Why did we develop this drug? What gave us the 
capabilities? It was the basic understanding of the genetics of 
the disease, the molecular pathogenesis of the disease, and 
marrying that basic understanding of the disease with a therapy 
that interacted here. I think that is going to be the 
overriding principle as we take a look at newer drugs, 
understanding the disease on a molecular basis and then drugs 
that are targeted toward molecular abnormalities or molecular 
deficiencies.
    This is an area that I think is evolving, and I am somewhat 
pessimistic with the past, but on the future I am very 
optimistic that we are exactly--or we are now beginning to have 
the tools that will enable us to move forward.
    Mr. Carter. About 3 weeks ago Lance Armstrong has a cancer 
survivor group in Austin, TX, and I went to that, and it was 
very uplifting about all these cancer survivors, but when you 
cut through it the definition of a cancer survivor is they 
survived the first round, really. There's an awful lot of 
people there that, although they are in remission right now, 
they face the distinct possibility of seeing the cancer again, 
and it ultimately killing them. Where I stand from, cure of 
cancer means you go in, you get treated, and you're not going 
to have cancer, that cancer at least, again.
    So we haven't reached that point, I understand, on almost 
anything but maybe childhood leukemia maybe where we've got a 
handle on it to some extent, so I'm just concerned about that 
much money and that much time and that little success, and I'm 
wondering, is there something innovative we can look at that 
would stimulate the challenge of the market go to out to win 
cancer? I firmly believe that if I could invent something to 
cure cancer I could be, you know, richer than Microsoft, and I 
think we ought to be able--somebody ought to be able to come up 
with some incentive to do that.
    A question I want to raise--and I realize my time is gone, 
Mr. Chairman, a few more minutes--the question I want to raise 
is the issue on the development of drugs. Is that becoming a 
stumbling block to giving incentive to private enterprise to 
take on full-fledged the challenge of cancer treatment. It 
takes you 9 or 10 years to approve a cancer drug, gives you 
another 9 years to recover your cost if you're in the business 
of inventing or creating that cancer drug. Could we change our 
intellectual property laws to give a longer time after approval 
where you still have the assurance of a trademark, a patent 
which might enhance the ability of private industry to invest 
the kind of money they are going to invest to go into fighting 
cancer? How do you feel about that?
    Dr. Christian. I'd like to comment on that, and then I'd 
like to comment also on your previous question.
    You know, I think that intellectual property issues are 
certainly a problem, particularly for combination treatments, 
particularly when we want to look at a much broader range of 
cancers than the more narrowly focused FDA approval pathway, 
for example. And, you know, I think it is possible that 
incentives might encourage industry to work more broadly with 
NCI, with each other, etc., so that we can do the combinations 
and do the broader development that will speed up this whole 
process, so I think that there are issues there that are 
probably worth looking at.
    I mean, we, for example, have been following the Best 
PHRMAceuticals for Children Act to see what the impact of that 
might be on drug development, and I think that will give us 
some notion, so I think intellectual property is probably an 
issue that warrants further thought.
    With regard to where we are with cancer, though, you know, 
I do want to point out that there are, you know, a growing list 
of tumors where we actually do cure patients and do prolong 
survival by adding treatment to surgery. You know, I think 20 
years ago the list was negligible and now there is a growing 
list of situations where we actually do cure patients, and many 
more where we prolong their lives. So your pessimism I 
understand, but we have made progress, and I think, like Rick, 
that, given the array of new entities that we have to use and 
to develop at this moment, molecularly targeted entities, I am 
more optimistic than I have been in 20 years of therapeutics 
development that we actually may make fundamental molecular 
strides.
    Of course, the case that he gave with Glevic and chronic 
myelogenous leukemia is one example. The reason that the 
numbers remain so daunting is that the major solid tumors--lung 
cancer, colon cancer--are much more complex molecularly, and so 
we are going to take a little bit longer, I think, to sort out 
what the relevant targets there are.
    But it is very interesting--in two major medical journals 
this past month, ``The New England Journal of Medicine'' and 
``Science,'' there were articles about a mutation found in 
patients with lung cancer which may help explain how we should 
better use some of these new targeted agents that we are 
investigating. It was the first such article, so we are really 
very optimistic, again, that we are entering a period where we 
really are going to understand the molecular nature of these 
tumors and how to treat them.
    Chairman Tom Davis. Thank you very much.
    Mr. Carter. Thank you, Mr. Chairman.
    Chairman Tom Davis. Yes, sir, Mr. Murphy.
    Mr. Murphy. Thank you, Mr. Chairman. I have a lot of 
questions. Let's see what I can get through here.
    First of all, there has been some frustration in the 
Hillman Cancer Center in Pittsburgh trying to recruit minority 
members for these studies--these are more than just racial 
minorities. These are also socioeconomic minorities--getting 
them to sites. Statistically speaking, do you believe there are 
significantly fewer minorities in studies on cancer trials 
nationwide? Is that a concern? Are there other things we need 
to be doing to recruit people of various income levels, a wide 
range of income levels, etc.?
    Dr. Christian. There are some situations with NCI-sponsored 
clinical trials where minorities are not accrued in proportion 
to the numbers that one would expect based on the prevalence of 
the cancer, for example. That's particularly true of African 
American men. It's true of Asian men and women. It's true of 
Hispanic men and women. And there are a lot of efforts ongoing, 
both in NCI-sponsored trials and at the cancer centers, 
University of Pittsburgh being one that has a funded grant 
actually to look at what some of the barriers are to accrual 
for racial and ethnic minorities and for people of lower 
socioeconomic status and for the elderly, which are also under-
represented on clinical trials. So this is an issue that is 
important to us, and we are approaching it from a variety of 
different perspectives.
    In our cancer centers I think, for reasons that are not 
altogether clear to me, there are some even greater 
disparities, so they have an even harder time than we do to our 
national trials accruing what would seem to be a representative 
and reasonable number of minorities.
    I chair actually the Task Force for the American Society of 
Clinical Oncology on Health Disparities and Workforce 
Diversity, and there are many issues that have to do with trust 
in communities, which have to do with the very, very low 
representation of minority medical professionals who might be 
able to reach these communities in more effective ways, so 
there are many issues. I think it is an important problem and 
one that we need to continue aggressively to try to address.
    Mr. Murphy. Let me move on to another area here that is 
important, and that has to do with the issue of people who may 
have insurance. Some States like California require insurance 
companies to cover clinical trials, and many other insurance 
companies will simply say they're not going to cover anything 
experimental. I'm not sure whether other States in addition to 
California have these laws. My question is this: has there been 
a cost/benefit analysis on these things, because there are 
times when people feel they are desperate and they want to try 
anything and the insurance company may say, ``No, we're not 
going to cover that. There's simply no efficacy to support this 
and it appears to be very expensive.'' Have we done any sort of 
analysis overall? Is that ongoing?
    Dr. Christian. There have been a couple of studies looking 
at the incremental cost of care on clinical trials which have 
shown that they are not significantly higher than care 
delivered outside of clinical trials.
    Mr. Murphy. What does ``significantly higher'' mean?
    Dr. Christian. I don't remember what the actual dollar 
amounts were. Do you remember that?
    Mr. Murphy. Rough idea? Percentage?
    Dr. Christian. Yes, I can certainly provide those precise 
details, but the estimate is around 5 percent.
    We have attempted to look at those incremental costs, but I 
think in addition there are other important things to consider 
here. Patients are going to be treated, and the treatments in 
the community may or may not be as well developed, as well 
based in evidence, and so there are costs associated with 
delivering----
    Mr. Murphy. Plus, you have differences in communities that 
may have a university medical center versus what might be in a 
community that might not be near some of those more advanced 
research.
    Dr. Christian. I mean, there are community participants in 
clinical trials, but there are also the vast majority of 
patients who are treated outside of clinical trials who are 
just treated in private practice settings. There, the evidence 
on which the treatments are based may or may not be as sound as 
that in the clinical trials. So I think there are a lot of 
things you have to take in account, but the cost concern, I 
think there is growing evidence that it should not be an 
impediment to providing those opportunities for patients 
through their health insurance coverage.
    Mr. Murphy. Thank you. That's all I have at this point. 
Thank you.
    Chairman Tom Davis. Thank you.
    Mr. Cummings.
    Mr. Cummings. Thank you very much, Mr. Chairman. I want to 
also thank you for holding this hearing. It is a subject I am 
very much interested in.
    Dr. Christian, in my District I have Johns Hopkins, and I 
also have the University of Maryland Hospital, and one of the 
things that, in trying to get people involved in trials, not 
just for cancer, but trials, period, African Americans, is that 
there is a tremendous--and I think you mentioned it a moment 
ago in response to a question--a tremendous distrust. People 
cite the Tuskegee experiment. My mother, for example, says at 
one of those hospitals, if she is in dire straits on her death 
bed, don't stop there. And that's because they have seen and 
had rumors at least of African Americans who were experimented 
upon, and it causes them not to want--many people not to want 
to be a part of any kind of experiment. And so even when the 
argument is made that some of what they may have heard in the 
past isn't absolutely true, what seems to happen, just when we 
get to a point like, for example, with AIDS, for people to 
finally get right there at the doorstep to participate, 
something happens.
    We have a situation we will be having a hearing here on the 
18th where another hospital in my District tested some 2,500 
patients and, come to find out, for AIDS and Hepatitis C, and 
come to find out the results, the machinery was malfunctioning. 
People knew it was malfunctioning and nobody did anything about 
it. So then when they hear that kind of information it makes it 
even worse, so they say, ``Wait a minute. You're talking about 
AIDS and Hepatitis C, things that could be life threatening, 
and you mean to tell me I went to--'' people have gone to the 
hospital, gotten results that may not be accurate, and nobody 
even waved a red flag.
    So I was wondering, and I think that this is a situation 
that probably exists all over the country. And then, of course, 
with the whole issue of health care disparities, in the 
Congressional Black Caucus one of our No. 1 issues is health 
care disparities. African American people, as you well know, 
are dying early, suffering needlessly, and, as a matter of 
fact, just recently I was in an audience of about--I'd say 
about 400 African Americans in my District, and I asked how 
many of them believed in the last 5 years--within the last 5 
years that they had a relative to die earlier than they should 
have because of some medical foul-up or to suffer serious 
injury because of something like that, and three-fifths of them 
raised their hand. I can cite situations in my own family, at 
least four or five, that I know things like that have happened.
    So the question becomes: how do you deal with--how does--
and we in the Congress, we not only have these hats that we 
wear here sitting on these panels, but we also take up the 
bully pulpit trying to get our constituents to do the right 
thing. Even when we pass legislation, we still want them to get 
out there and take the colon test and to do all those things to 
stay healthy. How do you say and how do you guarantee--not 
guarantee, but how do you assure people it's OK, particularly 
when they have these thoughts embedded in the DNA of every cell 
of their brain?
    Dr. Christian. Well, I appreciate the opportunity to talk 
about this a little bit. I grew up in a medical family and 
Tuskegee was very real to us, too, and it was a real 
phenomenon. But I think that there have been many years since 
Tuskegee and there are many new protections for patients. I can 
assure you for NCI-sponsored trials, for example, that they are 
reviewed exhaustively by experts in the management of the 
cancer that we're talking about, by people at the National 
Cancer Institute, and by institutional review boards and others 
to ensure that the nature of the research is reasonable and 
ethical. That said, there clearly are health disparities across 
the entire spectrum of medical care.
    I actually tell patients that on a clinical trial patients 
get the same treatment as everybody else. It is prescribed. It 
is written in a protocol. Exactly what needs to be done is 
written there and patients are treated according to that 
protocol. And we audit those sites. We go to the place and we 
look at the records and we make sure patients were treated the 
way they were supposed to be treated on the protocol, so there 
are even additional protections.
    So there are many protections in place that I think make 
participation in an NCI-sponsored clinical trial a safe and 
reasonable thing. I think it is important for minority 
communities to benefit from the opportunity to participate and 
from the knowledge that is gained. There are concerns about 
whether there may be differences in the way various racial 
groups handle drugs, for example. We won't know that if we 
don't study them. We won't know that if we don't participate in 
trials. And so when the new drug is then approved for the 
treatment of some cancer, we won't know whether that actually 
benefits us as it does the rest of the population. I think it 
is important for us to be a part of that and to have the 
opportunity to have those treatments and to, you know, 
aggressively pursue those opportunities so that we are sure at 
least in that venue that we are getting comparable care.
    So that's what I tell patients. I don't think patients 
should feel coerced to do it, but I think the opportunity 
should be made available to them.
    Mr. Cummings. Thank you.
    Chairman Tom Davis. Thank you very much.
    Let me just ask one final question, Dr. Pazdur. You raised 
concern in your testimony that the development of new cancer 
treatments will make clinical trials more costly and 
complicated. How do we address the concern if the criteria for 
enrollment in trials becomes based more on a patient's 
biological characteristics than clinical ones?
    Dr. Pazdur. Well, I think the eligibility criteria of the 
protocol will say patients must over express a particular 
enzyme, for example, and then patients will be enrolled that 
have that particular expression of the target that is aimed at. 
I look at this as a benefit to patients, to drug development, 
and to drug regulation because one of the problems obviously 
that we have in oncology today is even or most effective drugs 
are relatively modest in their response rate or in their 
clinical efficacy, so if we can predefine an enriched 
population that is more likely to benefit from the drug, that 
efficacy will be greater expressed, we'll have higher response 
rates, we'll have higher timed progression and better survival. 
Therefore, I look at it as a positive aspect for the whole area 
of drug development, and I don't really look at it as an 
obstacle at all. I look at it as a positive thing.
    Chairman Tom Davis. OK. Thank you both. Dr. Christian, 
thank you, as well. You have been very helpful, and I will 
allow you to go now and we'll go to our second panel. Thank you 
very much.
    Before we do that, I want to welcome my colleague from New 
Jersey, Representative Scott Garrett, to our hearing. We'll 
take a 10-minute break because we just switched, and then, Dr. 
Garrett, I'll allow you to introduce one of our panel members.
    [Break.]
    Chairman Tom Davis. The committee will come back into 
order.
    We are pleased to have our colleague from New Jersey here 
with us today. Mr. Garrett, you are recognized.
    Mr. Garrett. Thank you, Mr. Chairman. I appreciate the 
opportunity to introduce Dr. Andy Pecora, who is a 20-year 
veteran of the war on cancer. When I first met him, I knew a 
little bit about his background, now I know a whole lot more 
about his background and I would like to share that with the 
panel.
    He is a graduate of Seton Hall back in 1979. He went on to 
receive his medical degree from the University of Medicine and 
Dentistry in New Jersey, the Great State of New Jersey, in 
1983. He completed his residency over at New York Hospital, 
Cornell Hospital Systems, and then moved on to the Memorial 
Sloan Kettering Cancer Center and completed a fellowship in 
hematology and oncology in 1989, after which he moved on to 
Hackensack University Medical Center to serve as director of 
hematology and oncology in the adult blood and marrow 
transplant program, and now has recently been promoted to 
chairman and director of the Cancer Center at Hackensack 
University Medical Center.
    He is a diplomat of the American Board of Internal Medicine 
with subspecialties in hematology and subspecialties in 
oncology, and he has received numerous awards and honors, some 
of which include the Women's Guild Premedical Academic 
Achievement Scholarship back in 1978. In 1979 the Academic 
Excellence Award in Biology. In 1983 the Doctors Milton and 
Rose Petrokowski Award for Overall Excellence in Patient Care. 
And then the Outstanding Teacher Award from the Department of 
Internal Medicine at Hackensack University Medical Center in 
1989. And for all doctors I think this is important--he was 
selected as one of the best doctors in America in 1997, 1998, 
and 2003, and then received the American Cancer Society 
Physicians in the Forefront Award and the Susan G. Komen Breast 
Cancer Foundation Award as a hero in the fight against breast 
cancer. He received the EBMTESMO Award at the International 
Conference on High Dose Chemotherapy in Breast and Ovarian 
Cancer.
    His professional positions include such things as the 
scientific advisor for the companies ProNeuron and ProVirus. In 
addition, he has co-founded and served as chairman and chief 
executive officer of Progenitor Cell Therapy and now serves on 
the Board of Directors of the American Society of Bone Marrow 
Transplant, and previously on the International Society of 
Hemotherapy and Graft Engineering, the Hackensack University 
Medical Center IPA, and served as chairman of the medical board 
and board members of the Affiliated Physician Network.
    In addition to these things, he has served as chairman of 
the Transplantation Committee on the International Society of 
Hemotherapy and Graft Engineering and served as a member of the 
Cancer Institute of New Jersey Protocol Advisory Committee.
    Recently, he was appointed to the Steering Committee on the 
Transplant Treatment Trials Group, and he is a fellow of the 
Academic Academy of Medicine of New Jersey and a fellow of the 
American College of Physicians and American Society of Clinical 
Oncology and American Society of Oncology.
    Finally, the doctor has been involved in numerous research 
projects in an effort to improve the outcome of patients with 
cancer. His recent work includes the production of stem cell 
products that are free of contaminating malignant cells using 
technology including CD-34 selection and ex vivo expansion. He 
has led several national trials in the field of transplantation 
and has published numerous peer reviewed articles and abstracts 
and has presented the results of his research at many national 
and international scientific meetings.
    Probably most importantly, he is married and has three 
great children and resides in the fair State of New Jersey in 
the beautiful town of Ridgewood.
    We welcome you to the panel.
    Chairman Tom Davis. Thank you very much, Dr. Pecora. Thank 
you very much. We are joined here today with Dr. Robert Comis, 
who is president of the Coalition of National Cancer 
Cooperative Groups and is a professor of medicine and director 
at the MCP Honoman University Clinical Trials Research Center 
in Philadelphia; and Ms. Ellen Stovall, who is a 28-year 
survivor of two bouts with cancer and president and CEO of the 
National Coalition for Cancer Survivorship.
    This panel of witnesses is going to provide the committee 
with their perspective on the seriousness of low accrual levels 
in cancer clinical trials and what efforts are being taken to 
improve outcomes in cancer patients. They are obviously a very 
distinguished panel. We appreciate all of you being with us.
    It is our policy to swear everyone in, so if you would rise 
with me and raise your right hands.
    [Witnesses sworn.]
    Chairman Tom Davis. Thank you.
    What we will do is your entire statement is already part of 
the record. Dr. Pecora, I'll start with you. Try to do it 
within 5 minutes. You have your lights there that turn orange 
after 4 minutes, red after 5, and then we'll go straight down 
the line and then we'll move to questions. Thank you all very 
much for being with us.

  STATEMENTS OF DR. ANDREW PECORA, CHAIRMAN AND DIRECTOR, THE 
CANCER CENTER, HACKENSACK UNIVERSITY MEDICAL CENTER; DR. ROBERT 
   COMIS, PRESIDENT AND CHAIR, COALITION OF NATIONAL CANCER 
  COOPERATIVE GROUPS; AND ELLEN STOVALL, PRESIDENT AND CHIEF 
 EXECUTIVE OFFICER, NATIONAL COALITION FOR CANCER SURVIVORSHIP

    Dr. Pecora. Thank you. Thank you, Mr. Garrett, for your 
kind words. First and foremost, thank you, Chairman Davis and 
distinguished committee members, for providing me the 
opportunity to participate in this important hearing that 
hopefully will result in actions leading to improve outcomes 
for people afflicted with cancer. I have been active in the war 
on cancer for over 20 years, participating in basic science, 
clinical trials, and now cancer care administration. I welcome 
the opportunity to share my ideas on why and how increasing 
participation in clinical trials, regardless of the status of 
innovation, will improve outcomes for people suffering with 
cancer.
    We all well know that over the past 35 years Government, 
industry, and the public have spent billions of dollars to 
create and operate the agencies that oversee and fund efforts 
in basic and clinical discovery aimed to improve outcomes for 
people battling cancer. As a result, substantial advances have 
been made in the understanding of the biology of cancer and, as 
a consequence, new and more effective treatments have emerged.
    Unfortunately, even with this focus and extensively funded 
effort, cancer remains a serious problem. This year in the 
United States, alone, cancer is expected to claim more than 
500,000 lives; thus, criticism of our current system exists. In 
a recent article in ``Fortune'' magazine authored by Clifton 
Leaf, significant criticism is levied at the cancer research 
community claiming the culture is dysfunctional and that the 
search for knowledge has supplanted the search for cures, they 
lead to discoveries of marginal benefit regardless of a great 
expense of time and money. I believe, however, that these 
claims are only partially correct, and that, as a consequence 
of our national effort, it is now within our reach to turn 
cancer in most cases into a chronic disease much like diabetes, 
while searches for prevention and cures continue.
    As Mr. Leaf eloquently points out in his article, we must 
make the entire system of discovery and application of new 
agents more efficient. Clearly, continued improvements in our 
understanding of the underlying root causes of cancer and 
methods of detection, of efficacy, and safety are essential. Of 
equal importance however is active and robust participation by 
people with cancer in clinical trials. No matter how promising 
a therapy appears in laboratory testing, it is only through 
clinical trials that safety and effectiveness can be 
established in people. Simply stated, no person or computer 
program is capable of predicting whether a new treatment will 
work and be safe in people. In my current experience, it is not 
the lack of good ideas that is slowing progress in our quest to 
cure cancer, but it is much more a result of the slow pace of 
completing active clinical trials.
    In 2003, there were approximately 1,700 ongoing clinical 
trials, of which the NCI sponsored 1,200. Despite this large 
number of trials, only 3 percent of adult patients 
participated, while 20 percent were eligible. Low participation 
in clinical trials slows the continuum of drug development from 
initial concept to FDA approved products, and as a consequence 
impedes improvements of outcomes for people with cancer.
    In addition, poor participation at clinical trials 
lengthens the new drug approval process, estimated now at 10 to 
12 years, and has the cascade effect of increasing new drug 
development cost, now estimated at $800 million, inflating the 
cost of drugs to consumers once approved, and, worst of all, 
limiting the number of new agents that make it through the 
process of the discovery pipeline.
    Advances in knowledge which will lead to better questions 
should continue to be supported, but at the same time we need 
to improve participation in clinical trials. So what can we do 
about it? Lack of participation is due to several factors that 
can and should be addressed. A lack of public knowledge of 
availability of clinical trials and a growing public bias 
against participation due to poor outcome high profile cases is 
a key factor. Government should do everything it can to educate 
the public on the value and importance of participating in 
clinical trials.
    In an era of shrinking reimbursement for clinical care, 
funding needs to be established for clinical programs, both 
hospital and office based, to support the required 
infrastructure, including research staff and informatics, to 
participate in clinical trials. Reductions in their growing 
regulatory burden, including centralization of institutional 
review boards, streamlining adverse event reporting, and 
minimizing regulation resulting in increased cost and 
complexity without compromising patient safety or privacy must 
also be accomplished.
    Finally, insurance reimbursement for clinical trial cost 
needs to be addressed nationally. In my home State of New 
Jersey, I was a member of the New Jersey working group to 
improve outcomes in cancer patients. Our group was successful 
in convincing the insurance companies covering New Jersey 
residents to voluntarily reimburse for approved clinical trial 
related expenses. This could serve as a model for a national 
effort.
    Another important aspect to improve outcomes for people 
with cancer is to have more clinical trials available. This can 
be accomplished by increasing the efficiency of moving clinical 
trial concepts through the approval process before they become 
available to the public. The current system should be more 
efficient and held to more businesslike timelines for results. 
Specifically, the Cooperative Groups in the national cancer 
review process takes too long, at times years, and should have 
efficiencies mandated by Government, since it is Government 
that supports these efforts. Moreover, encouraging and 
rewarding the national Cooperative Groups to work together on 
questions that require large number of patients to answer is 
essential.
    Finally, the issues of creating an environment--
intellectual property protection, FDA approval support, etc.--
for multiple agents to be tested together for effectiveness 
prior to the FDA approval process needs to be addressed.
    In summary, I believe this is an exciting time for those 
engaged in the battle against cancer. The fruits of our efforts 
over the past 35 years are just beginning to be realized. It is 
clearly no time to retreat or claim defeat, but instead refocus 
our energies to make the entire system more efficient, less 
expensive, and more user friendly. Our family and friends 
afflicted with cancer deserve our collective best effort. In 
doing so, participation in clinical trials should increase, 
resulting in meaningful answers and better answers sooner for 
those battling this dreaded disease.
    Thank you. I am happy to answer any of your questions.
    [The prepared statement of Dr. Pecora follows:]

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    Chairman Tom Davis. Thank you very much.
    Dr. Comis.
    Dr. Comis. Congressmen Davis, Waxman, and members of the 
committee, I want to thank you for this opportunity to testify 
on behalf of 8,000 Cooperative Groups members from cancer 
centers, community practices, and patient advocacy groups 
across the country. Most importantly, we should all thank the 
courageous patients who enter our clinical trials. They are the 
real pioneers who move the frontiers of cancer treatment 
forward.
    There are two distinct forces in cancer clinical trials--
those studies directly supported by industry and overseen by 
the FDA, and those studies supported by the NCI which are 
conceived, designed, and executed in academic and community 
practices throughout the Nation. Typical industry supported 
trials are directed toward drug approval; Cooperative Groups 
trials are designed to evaluate new approaches and establish 
new evidence-based standards of care. We estimate that 
approximately 50,000 patients participate in clinical trials 
yearly. The NCI-funded Cooperative Groups account for about 
half, or 25,000 of those patients. The Cooperative Groups have 
always played a key role in the Nation's cancer research 
system. We develop curative therapies for childhood cancers, 
improve the post-surgical survival for patients with breast and 
colorectal cancer by 25 to 30 percent, and show that cancer 
can, indeed, be prevented in high-risk patients.
    As importantly, though, the publicly funded system allows 
us to ask and answer questions that challenge the mainstream. 
Our studies evaluating high-dose chemotherapy for breast cancer 
showed that this extraordinarily expensive and toxic treatment 
was of no clear benefit, saving the country hundreds of 
millions of dollars and patients unquestionable toxicity.
    Much has changed since President Nixon declared the war on 
cancer. The understanding of the biology of cancer has 
increased tremendously. The public and private sector has 
invested huge resources in the development of biologically 
directed therapies, and new targeted agents are entering the 
oncology practice in our phase two and three trials.
    The Cooperative Groups have adjusted to the opportunities 
and challenges created by these changes. We are investigating 
the newest molecules and approaches. Virtually all of our 
studies now include laboratory correlative studies which 
attempted to find why something does or does not work. In order 
to do this, we've established excellent tissue banks and 
laboratory programs in cancer centers throughout the country 
which collect, store, and analyze tissue specimens and 
correlate biology with clinical events occurring in our 
controlled clinical trials. But we must do more to ensure that 
patients have the opportunity to benefit from our work.
    First, let me address the issue of accrual of adults onto 
cancer clinical trials. We estimate that only about 3 to 5 
percent of adult cancer patients participate in clinical 
trials. This number was confirmed prospectively in our survey, 
which we did along with Ellen's group, which also revealed that 
only 15 percent of patients were aware that participation was 
even an option. That survey also reinforced the critical role 
of the oncologist in informing and educating patients about 
this option. Increasing awareness, dispelling misconceptions, 
engaging physicians are key elements of the solution to the 
accrual problem.
    These considerations led the Coalition of National Cancer 
Cooperative Groups to launch a national awareness campaign 
along with ``Newsweek,'' which is in its 4th year, develop Web-
based tools to facilitate trial searches, and work with the 
American Society of Clinical Oncology in developing both 
recognition and educational programs for physicians.
    Indeed, the efforts of the Coalition and others have born 
some fruit. There has been a 30 percent increase in overall 
accrual onto cooperative group studies from 1997 to 2002, from 
about 20,000 patients a year to about 26,000 patients, but more 
needs to be done. However, the system is stressed even at this 
level of accrual. The Cooperative Groups have been and remain 
chronically under-funded. Two extensive reviews of the system 
in the mid 1990's recommended that the Cooperative Groups be 
funded at the full peer recommended level. We continue to be 
funded at approximately 60 percent of that level, and funding 
has been flat for the last 3 years. This stifles innovation, 
destabilizes key functions such as our tissue banks, data 
management platforms, and acts as a disincentive to both 
academic and community physician participation.
    Keep in mind that about 60 percent of accrual comes from 
community-based practices. The NCI reimburses $2,000 per case 
to perform the research at the site. It is estimated in the 
ASCO survey that the actual cost is more like $4,000 to $6,000 
per case. The ability for both academic and community sites to 
continue in Government-sponsored work will be increasingly 
challenged, particularly when the full effect of the Medicare 
Modernization Act of 2003 takes place in 2005.
    The entire system is being buried under a regulatory 
mountain. It is estimated that about 30 percent of clinical 
trials research dollars goes toward ensuring regulatory 
compliance.
    Our studies are overseen by about 1,600 separate IRBs. HIPA 
compliance complicates our laboratory work. The current 
discussions about off-label drug use in oncology could have a 
huge impact on our studies, which try to explore new 
indications and uses for targeted agents as they become 
available.
    We all believe that there is an important balance between 
the need for innovation and the critical societal concerns, but 
the balance must ultimately be struck for the advantage of all 
who suffer from cancer.
    The Cooperative Groups remain totally committed to 
providing high-quality care and new opportunities for cancer 
patients, but rest assured the development of the newer cancer 
treatments will make clinical trials more complicated and more 
costly and accrual will remain a major concern.
    The Cooperative Groups chairs have developed a white paper 
entitled, ``Harnessing the Science: A Proposal to Improve the 
Publicly Funded Cancer Clinical Research System,'' which I have 
submitted
for the record, which outlines our thoughts on what can be done 
to ensure the continued vitality and importance of the 
Cooperative Groups in the publicly funded system, which is so 
critical to our cancer patients in the Nation.
    Thank you.
    [The prepared statement of Dr. Comis follows:]

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    Chairman Tom Davis. Thank you very much.
    Ms. Stovall, thanks for being with us.
    Ms. Stovall. Thank you, Mr. Chairman. Good morning. I am 
Ellen Stovall, president and CEO of the National Coalition for 
Cancer Survivorship, and I am a 32-year, two-time survivor of 
cancer.
    NCCS is the Nation's oldest survivor-led organization for 
people with all type of cancer. Our mission is to advocate for 
quality cancer care for all Americans, including the 10 million 
cancer survivors alive today in this country.
    I'd like to thank you for the opportunity to testify to the 
important work of this committee.
    I have a very direct and personal experience with cancer 
clinical trials. When I was first diagnosed in 1971, I actually 
started treatment on the day that President Nixon signed the 
National Cancer Act. My father literally thought I would be 
cured in 7 years, as the whole world would be, of cancer. 
Unfortunately, I was unable to participate in a cancer clinical 
trial promising a new therapy for the type of Hodgkin's Disease 
that I had because I was deemed ineligible for the trial, as I 
had just had a baby. Today, that therapy has become the 
standard of care for Hodgkin's Disease, and when I was re-
diagnosed with the same disease 12 years later I was able to 
take the drugs that had been in that clinical trial some 12 
years earlier, so I really do understand the progress that is 
made through clinical research.
    I cite this experience to draw your attention to two key 
issues: first, restrictive standards for trial enrollment 
unnecessarily prohibit many patients from entering a trial; 
and, second, clinical trials do serve as the means of testing 
new therapies and moving the standard of care forward step by 
step, extending or even saving many lives that would have been 
lost to cancer.
    There are many reasons why the rate of participation in 
trials is low. Both physicians who enroll their patients in 
trials and those individuals who agree to receive their care in 
a trial encounter several barriers to participation. All of 
those obstacles must be addressed if we are to improve the 
clinical trial system.
    The community has made great strides in increasing the 
awareness that care in a clinical trial may, indeed, be the 
best treatment option for a cancer patient, but our educational 
mission is incomplete. We still have to address the fears of 
some regarding the risks associated with trials, as well as the 
reservation of others about the value of trials. This is 
perhaps most acutely felt in under-served communities where 
socioeconomic, cultural, ethnic, and language disparities 
present even more barriers. Cancer patients may have to make 
sacrifices to enroll in a trial, and they want to believe that 
their time and energy are well spent in a valuable research 
endeavor.
    Over the last several years, some researchers and companies 
have taken the step of involving advocates early in the 
clinical trial design process. The FDA has also made great 
progress, particularly in the last few years, of involving 
advocates at earlier and earlier stages of drug development. 
Those efforts have generally been rewarded because advocates 
have embraced those trials and encouraged participation in 
them. Examples of this are breast cancer advocates' involvement 
with the design of the hterceptin', trials, and 
multiple myeloma advocates' involvement more recently with 
trials for Velcade'. For more than a decade, NCCS 
and a number of other patient advocacy organizations and 
professional societies like ASCO collaborated in a legislative 
effort to address the failure by third party payers to pay for 
the routine patient cost in trials. After many years of 
unsuccessful legislative effort, we were able to persuade the 
Clinton administration to issue an Executive memorandum 
instructing Medicare to allow all beneficiaries, those with 
cancer as well as other life-threatening diseases, to 
participate in high-quality clinical trials such as those 
sponsored by Federal programs or under the oversight of FDA. 
With this change in the leadership role of Medicare in health 
policy, reimbursement has seemingly become less of an obstacle.
    While cost is a primary concern, of no less concern is the 
fact that so few doctors recommend a clinical trial for their 
patients as a viable treatment option. Clinical research is 
expensive, requiring an extensive infrastructure both at the 
central point of control that is the research centers providing 
overall management of the trial and at the level of the 
individual provider. Research requires sophisticated, dedicated 
personnel such as research nurses, as well as the means for 
data collection and management, not to mention additional time 
commitment from physicians involved.
    For many years, cancer clinical researchers have made clear 
that the rate of payment from NCI for their participation is 
inadequate, despite some modest increases over the last few 
years. Privately funded research has overtaken that sponsored 
by NIH and other Federal sources because industry is willing 
and able to pay the full cost of research, whereas the 
Government's funding lags behind.
    As you probably know, over the last two decades cancer care 
has truly moved into the community. As much as 80 percent of 
cancer care is provided by community oncologists around the 
country. This system has been welcomed by cancer patients who 
prefer to record their care near their homes, thereby avoiding 
the dislocation that occurs if they must travel. Obtaining care 
in the community does not eliminate a patient's ability to 
receive care in a trial. As we just heard from Dr. Comis, as 
many as 60 percent of clinical trial enrollees are referred to 
trials by community doctors.
    The fact remains that only a small percentage of adults are 
enrolled. We are hearing disturbing reports from community 
oncologists that, as a result of changes in Medicare 
reimbursement for cancer care that were included in the MMA, 
they may be forced to reassess their participation in clinical 
trials altogether. The MMA reformed the system of payment that 
was over-paying for chemotherapy drugs, a reform that we all 
agree was necessary. The bill also made a temporary adjustment 
in the payment for expenses associated with delivering 
chemotherapy, but the concern of all of us who care about 
quality cancer care is that in 2005 this new law will reduce 
total payments to cancer care that such an extent that services 
offered by the community oncologists will have to be reduced, 
and clinical trial participation may be among the first things 
to go.
    We often describe the system of cancer care in the United 
States as the best in the world, and yet a series of reports 
from the Institute of Medicine's National Cancer Policy Board 
proclaimed great inconsistencies in the quality of care and the 
lack of any systematized way of assuring access to it. We do 
know that a good deal of this disparity could be corrected if 
more people were involved in clinical research and were assured 
access to a high quality clinical trial as a matter of first 
course rather than last resort.
    Our country is unusual in our ability to provide high-
quality care, including care in a trial in a community, but the 
system is suffering from so many strains that I fear all these 
factors will create such a stress that it may be impossible to 
carry on clinical research in the future that people could have 
access to.
    NCCS and others have been engaged for more than a decade in 
efforts that will ensure that clinical trials are an integral 
part of cancer care in this country. We are dedicated to that 
outcome, and we look forward to cooperating with this committee 
and others in ensuring that it continues in the future.
    Thank you.
    [The prepared statement of Ms. Stovall follows:]

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    Chairman Tom Davis. Thank you all very much. I think the 
committee has some questions, so I'll start with Judge Carter.
    Mr. Carter. Thank you, Mr. Chairman. And I thank all of you 
for being here. Ms. Stovall, please don't be mad at me for what 
I said about Lance Armstrong, but it was a shock to me to 
realize that the definition of a survivor was survive each 
time, and as you pointed out in your testimony, you said, ``I'm 
a two-time survivor.'' To me a survivor is it's all over and it 
won't happen again.
    Ms. Stovall. It would be nice if that were true.
    Mr. Carter. Yes, and I think that's the goal we are looking 
for. And a question I also have, I question what's going on, 
and maybe you can give me an answer. It seems to me that the 
research that we're doing in the areas of cancer is how to 
fight the tumor. Do you know, in these clinical research 
experiments that are being done, are we doing anything to 
inoculate for cancer, to come up with a genetic engineering to 
fight cancer? Are we still in the same direction we were in 
1970, how to fight a tumor? Does anybody know the answer to 
that?
    Dr. Pecora. I'm happy to comment on that. I think it is 
two-fold. I think, one, and sort of a generic way of speaking, 
is there is a focus to make the tumor go away, and that's a lot 
of giving medicines, doing surgery, radiation, but I think 
there is a growing and equal emphasis of keeping it from coming 
back and understanding how and what you need to do.
    You're right. I mean, there are strategies now aimed at 
using certain approaches to make tumors go away and then using 
cancer vaccines as an example to prevent it from coming back. 
When you have therapies that are only of modest benefit, you do 
the best you can. There are a whole new class of agents now--
you heard about them this morning--that's changing the whole 
cancer paradigm that I'm not even sure how to answer that 
question, because it may be you don't need to make the tumor go 
away and the person could have a happy and healthy life, like 
Glevic that you heard about. It doesn't cure CML, but it makes 
that clonive cell go away for a very long period of time, maybe 
forever.
    So the answer to that question is changing, but I do think 
your concern about keeping it from coming back is on the minds 
of people who do this sort of thing.
    Mr. Carter. You know, one of the things, just human 
nature--and I have no expertise in this at all, just human 
nature and comments--in Texas MD Anderson has a great 
reputation in Texas. Anybody that has cancer in Texas will try 
to go to MD Anderson Hospital. I'm sure there are people at 
Brackovitch Hospital in Austin, oncologists that can do a great 
job in treating. That's the publicly perceived--it is perceived 
all the way--it's really a private hospital now--it is 
perceived by the people in Austin to be a public hospital 
because it was at one time our public hospital. If given the 
choice, they will go to MD Anderson, which is funded heavily by 
public funds, and it is perceived to be a great scientific 
research center, a private hospital, if you will. It is 
perceived that way and everybody would want to go to MD 
Anderson because they think they have success.
    I think that's part of what your clinical trial situation 
is. People perceive this as another Government program rather 
than--do you get my drift? When you're talking about the 
National Cancer Institute, well, they've been at it for 50 
years with $52 billion worth of money spent. They're just 
another Government program. And if I can go to MD Anderson and 
participate, that's fine. In fact, I think MD Anderson actually 
runs some of your programs. But do you understand? It's the 
public perception.
    I would be willing to bet we have a better turnout at MD 
Anderson in Texas than you would some place else. I'd be 
willing to bet the farm on that. So a whole lot of what you 
have is the public perception that Government is failing in the 
war on cancer and that it is going to take private involvement 
to succeed in the war on cancer.
    Dr. Pecora. Well, I did make a statement that I will stand 
on. I think that the Government side of the equation can be 
more efficient, and I think that it takes too long to get 
things through the process, and I think we have created a 
bureaucracy that you heard about that is impeding discovery and 
making it harder to do.
    I've learned more in the last 2 years as a cancer 
administrator than I ever learned as a cancer investigator 
about why we're not getting more people into clinical trials, 
and many of them are business issues. They're not science 
issues. These are the things that--one of the reasons I wanted 
to come here today was to address this with Congress, because 
you fund these efforts. So I think if you--the money for you is 
to look at these things and to try to drive efficiencies into 
the system.
    Mr. Carter. Absolutely, and that's one of the reasons I 
mentioned it to the last panel about the intellectual 
properties issues. The incentives--and let's face it, we live 
in a world where we're all trying to make a living, and the 
incentives to go out and meet these challenges and advance 
private capital in meeting these challenges, in my opinion, 
needs to be encouraged. The Government can't fight this war 
forever. We fought it and we can use some help, I guess is what 
I'm saying.
    Dr. Pecora. There's one other thing I want to say before--I 
don't want to monopolize the microphone, but another misnomer, 
this concept of clinical research, people hear ``research'' and 
it has all these connotations that we heard one of the 
Congressmen speak to before. In cancer care, when the outcome 
is dismal, clinical trials should be the standard of care, not 
that there's something better and they're trying this out. I 
mean, that's a bad way of looking at it, particularly when the 
likelihood, as we get smarter about the mechanisms of the 
disease, how to read if something is effective or not are 
improving at light speed. The likelihood of you having a better 
outcome by participating in trial is going to go up 
proportionately.
    So what we need to do as a Nation is to push cancer trials 
out into the community offices, into the hospitals that aren't 
the MD Anderson's of the world, because that's not where all 
patients are treated. That's where the minority of patients are 
treated.
    Mr. Carter. That's right.
    Dr. Pecora. We have to get this in the doctors' offices, 
and in order to do that we're going to have to support them. 
We're going to have to provide them funding for research 
nurses, data managers, and we're going to have to simplify the 
process or it is not going to happen.
    Mr. Carter. We're going to have to convince the public's 
perception that they'll get that equal treatment in that 
doctor's office that they would get in this famous--supposedly 
famous cancer center. And that's the perception you've got to 
overcome on these clinical trials, in my opinion.
    My time is up. Thank you, Mr. Chairman.
    Chairman Tom Davis. Thank you very much.
    Mr. Garrett, would you like to ask any questions?
    Mr. Garrett. Thank you. I appreciate the testimony of 
everyone.
    A couple of quick questions. First of all, with respect to 
New Jersey and on the insurance side of the issue, you 
indicated how you were able to convince the insurance industry 
to provide coverage for the expenses of clinical trials. How 
did you do that? What is the status nationally, if you know? 
And what is the recommendation as far as facilitating that on a 
national basis?
    Dr. Pecora. Well, how we did it in the State is we got the 
major cancer program directors and people involved in various 
aspects of clinical trials to come together in a forum and we 
brought in the CEOs and other representatives of the insurance 
industry and showed them the data. We made the claim, ``Look, 
you know, you're paying for things that are marginally 
effective. Wouldn't you want to pay for something that could be 
better for your subscriber?'' And they got it. And they got it 
to the point where they did it voluntarily.
    I can't speak to the rest of the Nation. I know there's 
efforts around the country, but I'm not privy to that 
information.
    Dr. Comis. Maybe I can help clarify. This is a very 
complicated issue. In the Harris Survey that I mentioned before 
we asked several questions about personal barriers to 
participation--travel, taking time off from work, insurance 
coverage, etc.--and the thing that was on the very top of the 
list, 60 percent of the 6,000 cancer patients that we surveyed 
said that they were afraid their insurance companies wouldn't 
pay.
    Then we went to the 4 percent of patients who did actually 
participate and asked a final question which was: in the end, 
did your insurance company pay? And 86 percent said yes.
    Now, how hard it was to get there we didn't ask, or how 
difficult it was to navigate the system, but this insurance 
barrier issue is a major perception barrier, at least, and 
there are now--Ellen may know the exact number. I think there 
are 19 States now that have legislative solutions, there are 
about three States that have non-legislative solutions. 
Medicare said they would pay. I think it is incumbent on us, 
and this is what we try to do in a lot of our materials is to 
get the word out that, in fact, your insurance companies have 
said that they would pay, that States are backing you, and if 
you are having trouble you have to use that information to make 
sure that you have access to clinical trials.
    Mr. Garrett. And, following upon this, then, if I may, Dr. 
Comis, you made the comment--and you did, too, Andy--as far as 
the responsibility of greater education, but to me isn't the 
education responsibility on the part of the doctor, the 
oncologist? Isn't he supposed to know this, and isn't he the 
one that's supposed to convince the patient that clinical 
trials are necessary and, if he has been in the practice for a 
while, that he should know the statistics that he can rattle 
off that he can put his patient at ease?
    Dr. Pecora. Right. Well Mr. Carter I think hit the nail on 
the head. It is very hard to battle public perception. I am a 
practicing physician. I see cancer patients. I still put people 
on clinical trials. I do it every day, and it is hard. And the 
reason it is hard is because people come in with preconceived 
notions, and the minute you start talking about a clinical 
trial all of the sudden warning bells go off.
    You know, we have high-profile cases in our country, the 
Jesse Geisinger case, going back historically to the Tuskegee 
experiment, the whole concept of what IRBs are doing now. It 
is, I think, pushing people in the general direction of being 
suspicious and having a bias against clinical trial, and I 
don't think at the individual physician level that's going to 
be reversed very readily. I think it has to be a societal 
issue.
    Dr. Comis. Maybe I can followup on that, because we've 
studied this a lot. I agree totally. In the end, it's the 
interaction between the doctor and the patient that decides 
when somebody goes on. And, in fact, I mentioned in my remarks 
that only 15 percent of the patients in the survey were aware 
that they could participate, 85 percent weren't.
    If you look at the people who were aware, a quarter went 
on. And if you look at the difference between the quarter that 
went on study and the three-quarters that didn't, it was all 
the doctor. The doctor helped educate them about trials, helped 
find a trial for them. The doctor and the staff worked on this 
together.
    I agree. There are two components on how we have to 
approach this. One is to increase awareness and decrease 
misconceptions on the part of the patients and the public, but 
the other thing is to facilitate the involvement of the doctor 
in the process.
    You know, it's not just reimbursement. It takes time. It 
takes staff time, patient time to do this, and we have to get 
the resources out to the sites, particularly the community 
sites that are really committed to do this, and the resources 
are not there and the challenges are great.
    Ms. Stovall. And I just want to add that, you know, I put a 
lot in my testimony, my submitted testimony, about physician 
reimbursement issues which, Mr. Chairman, you touched on, and 
it is not about the income of doctors or what doctors make, 
it's about how we value the time they spend with their patients 
and their families. When we have been over-paying for the 
chemotherapy that they provide and grossly under-paying for the 
time that they spend counseling families and patients about 
treatment decisions which are more and more complex as time 
goes on with the new science, I think that we really have to 
look at our reimbursement system and put the dollars where we 
value the doctors' time.
    Mr. Garrett. Thank you.
    Chairman Tom Davis. Thank you. Thank you very much.
    Mr. Duncan, do you have any questions?
    Mr. Duncan. Well thank you, Mr. Chairman. Thank you for 
calling this hearing.
    One thing I'm curious about, I have read in the ``Wall 
Street Journal'' and other places sometimes that it takes--I've 
seen figures of $650 to $850 million to get a typical drug 
approved in this country, and it sometimes takes 10 or 12 
years, and I've also read that in no other developed nation 
does it take anywhere close to as long. I remember the ``Wall 
Street Journal'' had on its front page several years ago this 
small company in Illinois had a breast cancer detection pad 
that had been approved in every other country where they had 
asked for approval, most of them within days or weeks, but they 
had been--I think it was 9 years, and they still weren't 
approved in this country, and they had some quotes from cancer 
specialists saying that thousands of lives have been lost 
because that had happened.
    I'm just wondering. I assume that none of you can say 
anything critical about the FDA or maybe they'd get back at you 
later, but are we doing any better on any of that stuff? Why is 
it that it takes so much longer and so much more money to get 
approved here as compared to any other developed nation? I 
mean, you can go overboard on anything, and I'm just wondering 
about all that.
    Can any of you say anything----
    Dr. Pecora. I'd like to comment on that.
    Mr. Duncan [continuing]. Without getting in trouble?
    Dr. Pecora. Well, you always get in trouble.
    Mr. Duncan. OK.
    Dr. Pecora. But I do think that the FDA talked a little bit 
about this critical path document that they put out, and 
personally, as someone involved both on the academic side and 
on the corporate side, I find them getting more and more user 
friendly, and I do think that some of the initiatives will 
decrease time to discovery and cost to discovery, particularly 
as we get better at screening for toxicities and putting the 
right kind of people on trials, i.e., people who have the 
potential for benefit.
    But I see the major stumbling block becoming this issue 
about clinical trials. This is not going to go away. This is 
going to get worse. People in the community, people who are 
doing this, you're not going to answer a question of whether or 
not something is safe or effective until you test it on a 
person, period. And until we fix this system, which is going in 
the wrong direction, that's going to maintain that high cost of 
discovery, the 10- to 12-year timeline. You heard plenty of 
testimony today to attest to that, and I think that's where the 
emphasis should be now.
    Ms. Stovall. I just want to add on to that. I mean, I 
remember Dr. Pazdur, who was on your first panel, remarking at 
several meetings over the last few years that we've attended 
that when a really good drug, a really novel therapy comes to 
the FDA, you see it move very, very fast. I don't know what 
that means if they're not exciting therapies that are coming to 
the FDA what the countervailing point would be. But I do know 
that the FDA--the burden on the FDA in terms of peer review 
capabilities, well-trained specialists to review oncology 
products is just not what it needs to be in terms of capacity 
building. I think that's another area for this committee 
perhaps to examine in its future deliberations. I think that 
would be a very interesting thing to pursue.
    Mr. Duncan. Well, what happens? If it takes, you know, 
hundreds of millions or years to get a drug approved, then 
obviously what you do--this is one of the main reasons why the 
drug industry has ended up in the hands of a few big giants, 
because a small company can't handle that. And then also the 
small companies don't have the connections within the FDA. 
Chairman Burton said in here one time that 9 out of the last 14 
FDA commissioners work for the big drug companies now. I don't 
know if that's true or not, but, boy, there just is a lot of 
things going on apparently that--I mean, people wonder why 
drugs cost so much, and that's--this seems to me to be why. It 
is our own--we've let the Government get too big and too 
bureaucratic, and if we don't cut this down a little bit and 
speed this process up and make it where a small company has a 
chance again, these drug prices are just going to go up even 
more, it seems to me.
    Thank you, Mr. Chairman.
    Chairman Tom Davis. Thank you very much.
    Let me just try to wrap a few questions up. First of all, 
let me just pick up on where Mr. Duncan left off. If there's 
one or two things Government could do to really help this 
process along, would it be the funding dollars, would it be 
speeding up the regulatory process, would it be reducing the 
paperwork and the bureaucracy that you have to go through and 
patients have to go through, would it be information 
dissemination? Let me start, Ms. Stovall. You've taken a 
leadership role on this from the patients' perspective. From 
your perspective, what do you see? And then let me ask 
everybody what they see from their perspective. We're talking 
about Government's role. So much of this is private.
    Ms. Stovall. Well, I think as patient advocates we do look 
to Government and the very, very important role of both the FDA 
and the NIH and CMS, frankly, play in this whole, you know, 
landscape of both developing new drugs, the research approval, 
and then finally paying for them and getting them to people.
    I would like to see better coordination among these 
agencies, working more collaboratively, having some of the 
regulatory barriers removed, having a bit of a more 
transparency to the FDA processes that I believe could truly 
make things work better, better training of reviewers, more 
attention paid to that whole process, including I think 
something that has been mentioned but largely not examined very 
closely, and that is institutional review board reforms, 
because I think the regulatory burdens on the system, as Dr. 
Comis and others have mentioned, are at this point very 
onerous, and really helping lawyers more than they are helping 
patients succeed in getting these new therapies.
    Chairman Tom Davis. I like that one. That's not even, I 
mean, that's something that is within this committees 
jurisdiction and is not even big dollars. It's just trying to 
be efficient with what we do.
    Ms. Stovall. It's just making more efficient the things 
that we already have in place, because I do think that the 
protections that Government offers are very important to 
patient care but shouldn't be burdensome.
    Chairman Tom Davis. Thank you.
    Dr. Comis, do you have any thoughts on that?
    Dr. Comis. Yes. I would followup with three areas. One is I 
think the Government--the NCI has to recognize that it needs to 
fund these things at an acceptable level, at a level it can be 
done. For the group system, $150 million out off a $4.3 or $4.4 
billion budget, I mean, that's like chump change. So, I mean, 
people have to decide whether they want the Government to be 
involved in this. And they have to be, because we can ask 
questions that a company can't.
    The second thing is we have to be able to interact and 
develop relationships between the public side and the private 
side. The private side has developed all the drugs, and we have 
to be able to work with them very, very effectively, and there 
are a lot of barriers to that need to be broken down.
    As well, I think that the layering of the Government 
bureaucracies have to be harmonized between FDA, NCI, etc.
    And, last, I'd followup with one other huge thing that 
could happen, and it does relate to the OHRP, the regulatory 
office. If that office wrote a letter to the 1,600 IRBs that we 
deal with tomorrow and said, ``We will accept the 
recommendation of the central IRB that has been sponsored by 
the NCI,'' that would open up the whole deal.
    Those three things could really make a huge difference.
    Chairman Tom Davis. Thank you very much.
    Dr. Pecora, any thoughts?
    Dr. Pecora. Yes. It's somewhat repetitious, but for the 
Government which funds all of these efforts to take a product 
development, like you want to make a product mindset, and look 
at all of the issues that we've discussed and see where 
Government can intervene in a way that continues to protect 
patient safety, continues to protect patient privacy but gets 
rid of the bureaucracy and the inefficiencies, centralizing 
IRBs, getting rid of the craziness we have with the way we do 
adverse event reporting, and there's a list of things that I 
don't want to repeat in the interest of time that can and 
should be done.
    Chairman Tom Davis. Well, let me ask you this. Do you think 
that the central IRB that has been developed by the National 
Cancer Institute has proven to be effective in eliminating 
duplicative applications in the review process?
    Dr. Pecora. I think it will. I think it has been and I 
think it will, and I think centralization of IRBs in the 
country would be wonderful.
    Chairman Tom Davis. Well, let me just ask this. In terms of 
getting the doctors involved in this, because that's really 
your pressure point here--people get diagnosed by their 
doctors. What are my options? What's the best way to get that? 
Is continuing medical education an option here for oncology? 
You do an hour talking about what is involved here, what are 
the options, how they can counsel patients? Or is there a 
better way to get that word out? Because it seems to me if we 
do a better job of that, we're going to have plenty of people 
lining up to be part of these trials.
    Dr. Pecora. It will only happen if you match that with the 
resources they need to do it, and they don't have it right now.
    Chairman Tom Davis. OK.
    Dr. Comis. But I also think that--you know, I mentioned in 
the body of my talk that the Coalition or the Cancer 
Cooperative Groups are working closely with ASCO, the American 
Society of Clinical Oncology, in education. You know, I think 
that we have to--you know, ASCO is perfectly positioned to try 
to take the lead in this educational process along with us, 
and, in fact, there have been some innovative approaches with 
regards to recognition awards at the annual meeting, and also 
with we're developing a series of meetings to try to have the 
25 or 30 percent of the practices that are really great at this 
educate the people who are really interested but can't see a 
way how to do it. And over the course of the next 3 years we 
hope to have several meetings and a syllabus that arises from 
that.
    I think we have to focus on the doctors who are interested 
in doing this but don't seem to have the wherewithal to do it, 
but it can't be done without resources.
    Chairman Tom Davis. Ms. Stovall, let me ask you--you 
answered that, but also, as you look around and you network 
with patients and so on, are we seeing any geographical issues 
on this, as well, or any demographic issues in terms of who is 
getting notified, who sees the options, and who is lining up?
    Ms. Stovall. Well, to answer your last question first, I 
really think that if you looked at the map that Michaele 
Christian put up originally with all the dots on it about where 
places are funded to do the work, there's a big gap right in 
the middle of the country where there aren't too many dots, and 
this represents a lot of farmland, it represents a lot of rural 
America and a lot of poor people.
    I want to add on to that the disparity again is created 
both in the inconsistency in the way treatment is provided and 
offered to patients, but also the health care disparities. The 
uninsured and under-insured are terribly disadvantaged by not 
having access.
    I really think that the point again, building on what Bob 
just said about physicians, physician education, training, all 
of that is wonderful. If we do not fix the reimbursement system 
that's being dismantled with the current MMA, we really are 
going to see even more disparity in care than we are seeing 
now. And that's not just with clinical research, that's with 
all kinds of treatment, because doctors are just going to go 
out of business, and it is just a pure and simple fact.
    Chairman Tom Davis. And that's a problem----
    Ms. Stovall. It's a serious problem.
    Chairman Tom Davis [continuing]. Across the medical field. 
It's not just here, it's everywhere.
    Ms. Stovall. Right.
    Chairman Tom Davis. You get the Government buying so much 
health care, and that's how we think we save money.
    Let me ask you this, too. Has the FDA's accelerated 
approval, their expanded access, priority reviews, and fast 
track policies--do you think they've approved and shortened the 
length of the approval process as a whole? And do patients 
receive drugs and therapies more quickly under these policies? 
Or do you think it is just a lot of rhetoric?
    Ms. Stovall. I have seen improvement, and I think it is 
because I know that patient advocates are actually in there and 
they are involved and they are constantly putting pressure on, 
as well. They have the most to gain or lose from what happens 
with new therapies that come through the FDA. So I would say 
yes, there has been improvement, and I think particularly under 
Dr. McClellan when he was there and Dr. Pazdur specifically we 
saw tremendous improvement.
    Dr. Comis. I agree with that, and I think that the drugs 
that appear active are getting in the hands of the physicians 
and patients quicker, and I think that most as importantly, you 
know, we're regulated by two bureaucracies, the NCI bureaucracy 
and the FDA bureaucracy, and we need to harmonize those things 
and, in fact, Rick Pazdur and Michaele and those of us from the 
extramural environment are working on trying to do those 
things. So it will be very, very important to facilitate this 
interaction between the public side and the private side of the 
system.
    Chairman Tom Davis. Well, let me say to all of you thank 
you for, first of all, your testimony. I think it has been 
very, very helpful to us. I hope it has been helpful to the 
previous panel, as well, as they take notes on this and try to 
improve and see what we can do about it. But I also thank you 
for what you're doing in the fight against cancer. You are in 
the front lines, all of you. You have a little bit different 
roles, but what you're doing is very, very important and I want 
to thank you for that.
    We now adjourn the hearing.
    [Whereupon, at 12 noon, the committee was adjourned, to 
reconvene at the call of the Chair.]
    [Additional information submitted for the hearing record 
follows:]

[GRAPHIC] [TIFF OMITTED] T5598.054

                                 
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