Four years ago, at my confirmation hearing before this Committee, I
counted among my central goals as the Director of NIH a pledge to
"encourage NIH investigators to extend their biological discoveries to
clinical settings." I am pleased to be here today to help evaluate the status of
clinical research: its importance, the unprecedented opportunities to change
the practice of medicine, the obstacles facing clinical research, and the,
several efforts the NIH has undertaken to confront these issues.
The NIH serves the nation by conducting and supporting medical research,
with the goal of improving human health. Medical research is a continuum
that spans diverse realms of laboratory research and several kinds of clinical
research. Any single weak link in this continuum reduces the likelihood
that major research findings will benefit the public in the form of new
treatment, and prevention strategies. Clinical research is a crucial element
in this endeavor.
What is clinical research?
Clinical research refers to research conducted with human beings, including
studies of specimens collected from specific patients. It encompasses some
laboratory research on the mechanisms of human disease, translational
research (in which laboratory and clinical activities are closely aligned),
clinical trials of preventive and therapeutic strategies, epidemiology,
behavioral research, and health services and outcomes research.
Because it covers so many topics, clinical research the skills and expertise
of many kinds of investigators, including individuals Whose Primary
training is in medicine (so-called "physician scientists"), dentistry, Public
health, nursing, psychology, and various laboratory sciences. Other
professionals such as dieticians, computer programmers, bio-engineers, and
technicians are also essential for clinical research. Clinical research is
conducted at a variety of sites and by a variety of entities. Academic Health
Centers (AHCs), government labs and clinics, community hospitals, State
health organizations, and managed care and pharmaceutical industry sites
are all active participants in the Nation's clinical research enterprise is
necessarily multidisciplinary.
The success of clinical research depends on funding from both Federal and
private sector sources. In FY 1996, 27 percent of competing projects at the
NIH and 38 percent of our competing research dollars were accounted for
by clinical research. (Approximately one-third of these dollars and 20
percent of these grants were invested in clinical'trials.) Data from the
Pharmaceutical Research and Manufacturers Association reveal that in
1995, the latest year for which figures are available, clinical trials accounted
for an outlay of more than S4 billion by the pharmaceutical industry,
equivalent to 35 percent of company-financed research and development.
Examples of the benefits of Clinical Research
Clinical research has changed the face of modern medicine. Fifty years
ago, at the end of World War II, physicians had little ability to effectively
treat or prevent any of the deadliest diseases. Most of the staples of modem
medicine we enjoy today were still unknown: antibiotics, vaccines for polio
and several other severe infections, most hormone replacements and steroid
therapy, effective drug therapies for cancer and psychotic illnesses, testing
for genetic disorders, coronary bypass surgery, transplanted organs, and
artificial joints. These and Other successes have encouraged public
enthusiasm for research and belief in the potency of modem medicine.
It is useful to consider in more detail the scope of changes in medical
approaches to some common diseases that have occurred in the past few
decades as a consequence of clinical research sponsored by the NIH and
others.
Atherosclerotic cardiovascular disease remains the leading cause of death
in the United States
and is increasing in incidence elsewhere in the world as the population ages.
However, since the late 1960s, the age-adjusted mortality rates for this
disease have declined greatly in all segments of the U.S. population.
Epidemiological and biochemical studies have identified risk factors,
including hypertension, obesity, lack of exercise, smoking, and high blood
cholesterol; efforts to control these factors with behavioral charge and drugs
have helped to decrease the development and mortality of heart disease.
percent Further decreases in mortality are due to more effective care of
victims of coronary artery disease. Clinical research has yielded a wealth of
diagnostic and treatment options, including cardiac catheterization,
coronary artery bypass surgery.. balloon angioplasty, thrombolytic ("clot
busting") therapy, and a vast array of new drugs to help the heart patient
control arrhythmias, and improve the strength of the heart muscle.
Osteoporosis afflicts 25 million Americans, most of them older women, and
the loss of bone mass due to osteoporosis contributes to 1.5 million
fractures annually; such fractures are a major cause of admission to
hospitals and nursing homes and a major financial burden. Over the last
decade there has been a revolution in thinking about osteoporosis. A major
insight comes from tile recognition that osteoporosis and fractures we not a
necessary consequence of aging. NIH support for clinical studies of
nutrition and physical activity interventions have provided strong evidence
that fractures can be prevented and bone loss reduced even in older
individuals - for example, with, calcium and vitamin D supplements. -More
recently, studies of postmenopausal women, with osteoporosis have led to
the identification Of several classes of medications. including estrogen, that
appear to slow the actions of bone-resorbing cells and thereby improve
bone strength.
An estimated 16 million people in the United States have one of the several
forms of diabetes mellitus, a chronic disease that affects how our bodies use
food for growth and energy. Following the discovery of insulin and its first
use in the treatment of diabetes in the early 19205, progress was slow,
especially towards effective treatments for the many serious complications
accompanying the disease. In recent years, advances in diabetes research
have led to better ways to manage diabetes and treat its complications, It
has been recognized that there are two major forms of diabetes: Type 1, or
insulin-dependent diabetes mellitus, that commonly begins in childhood,
and the more common Type II, or non-insulin-dependent diabetes mellitus
that appears during adult life. Examples of advances to control these forms
of the disease include. new forms of purified insulin, such as human insulin
produced through genetic engineering; better ways for doctors to monitor
blood glucose levels and for people to test their own blood glucose levels at
home; development of external and implantable. insulin pumps that deliver
insulin, replacing daily injections; better ways of managing diabetic
pregnancies; new drugs to treat Type II diabetes and better ways to manage
this form of diabetes through weight control; laser treatment for diabetic
eye disease, reducing the risk of blindness; evidence that intensive
management of blood glucose substantially reduces microvascular
complications of diabetes --such as eye, kidney, and nerve damage; and
demonstration that antihypertensive drugs called ACE inhibitors prevent
delay kidney failure in people with diabetes. Still, diabetes is a leading
cause of chronic ill-health and mortality in this country, and definitive
therapies continue to elude us.
The partially successful efforts to control the AIDS pandemic, even in the
absence of an effective vaccine, has depended heavily on epidemiological,
behavioral, and pathophysiological, as well as basic viral and
immunological research. Recent advances with antiviral drugs and immune
augmentation have been strongly influenced by methods to measure virus
levels accurately in the blood of infected patients, slowing that the
treatments are capable of rapidly reducing virus levels and that virus levels
are indicators of beneficial effects on well-being and longevity in clinical
trials. Clinical research has also played important roles in the definition of
the many manifestations of this new disease, development of improved
therapies for its complications (including opportunistic infections and
unusual cancers), and recent studies of genetic resistance to HIV infection
and AIDS that results from mutations in newly-discovered genes encoding
viral receptors.
What are the clinical research opportunities of the future?
Medical research has visibly transformed the practice of medicine over the
past fifty years, but
even greater benefits may be possible in the next fifty years, if we are
positioned to capitalize on
the many profound developments that have recently occurred in
fundamental science --- especially
in genetics, structural biology, molecular and cell biology, computer
science, and imaging
technologies.
Studies of the human genome and the genomes of many other organisms--hold great promise for
approaches to medicine not even imagined a decade
ago. The complete DNA sequence of human beings will make it possible to
define a unique signature for every gene. This situation has already been
achieved for several bacteria and yeast, providing new opportunities for
combating infectious diseases and understanding fundamental cell
processes. Rapidly evolving technologies, comparable to those used in the
semi-conductor industry, will allow scientists to build detectors that trace
hundreds or thousands of these gene -signatures in a single experiment.
Scientists will use the powerful new tools to reveal the secrets of disease
susceptibility and the mechanisms by which diseases develop, create broad
new opportunities for preventive medicine, and even provide unprecedented
information about the origin and migration of human populations.
In the last two decades, we have learned that genetic changes lie at the root
of all cancers. We are now working to identify all of the genes responsible
for the establishment and growth of cancer. To be able to peer into a single
cell and read its molecular signature will allow researchers to determine
what is different between a normal cell. and a cancer cell and will lead to
the development of highly specific, sensitive molecular markers for cancer
detection, These -findings will provide the clinical tools for determining, at
the earliest possible stage of cancer development, those tumors that will
respond to therapy, which therapies they will respond to. whether a
particular cancer will grow quickly or slowly, and whether or not it will
metastasize.
Advances in high performance computing are revolutionizing the practice
and teaching of medicine. Perhaps one of the most fascinating examples of
this is the Visible Human Male and Female, the world's first "computerized
cadavers." Detailed images of the bodies of a male and female who donated
their bodies to science have been compiled into a computer database,
providing complete, anatomically detailed, cormputer-generated 3-D
representations of the human body. More than 850 licensed users have
developed remarkable applications for the data, as well as technologies to
create Visible Human-Like representations of live patients. Doctors can
now practice procedures on surgical simulators. Non-invasive cancer
screening techniques, such as virtual colonoscopy, are being developed,
which may eliminate the need for costly, uncomfortable invasive
procedures.
Imaging technologies also promise to change the way we diagnose disease
and follow responses to innovative therapies, replacing more costly and
invasive approaches. For example. because atherosclerosis and congestive
heart Failure (CHF) develop over decades without symptoms, their true
prevalence is unknown, and identification of those at greatest risk of
myocardial infarction, stroke, or overt CBF has yet to be achieved. We also
do not yet know the events leading to atherosclerotic plaque rupture and
loss of cardiac- muscle function in CHF. Noninvasive cardiac MRI
techniques can already image coronary arteries, map blood flow through the
peripheral vascular system, and measure cardiac wall strain with accuracy
approaching, or equal to, now standard methods, echocardiography and
coronary angiography. Use of MRI imaging in large population studies
may reveal pathways of progression from presymptomatic to overt disease,
gender and racial differences in disease progression, and biological markers
of imminent risk. Equally dramatic changes are occurring through the use
of new imaging devices in the study of diseases of the brain, including the
neurodegenerative disorders of aging, multiple sclerosis, psychiatric
illnesses, drug abuse, and trauma.
Accelerated methods for studying the three-dimensional structure of
proteins, including those proteins that serve as targets for drugs against
disease, are likely to have profound effects on approaches to drug
development in the public and private sectors. Rational drug design has
already influenced the production of the protease-inbibitors used in the
treatment of HIV infection, A firm understanding of disease mechanisms
and well-designed clinical trials Will be required to reap the benefits of
these coming changes in therapeutics.
Advances in biomaterials and bioengineering also Offer new health
improvements in the near future in the form of artificial tissue and organs,
devices for monitoring disease, and new methods for delivering therapies.
For example, scientists are developing a promising new drug delivery
system--tiny, drug-filled beads that, when administered orally, stick to cells
in the digestive tract and slowly degrade to release the drugs into the
bloodstream. The beads may one day be used for the oral administration, of
drugs that are recently given only by injection and -could be used to deliver
a wide variety of substances, including DNA-based vaccines, cancer
chemotherapy, hormones, and gene therapy.
The take-home message is simple: every one of these advances and
opportunities they engender are dependent on a sustained and heightened
clinical research effort.
What are the barriers to effective clinical research?
Despite these extraordinary past successes and remarkable future
opportunities, clinical research
in this country appears to be threatened by several factors, some relatively
new and some recognized for more than a decade.
The recruitment, training and sustenance of clinical investigators, especially
of physician-
scientists, prominent among the long-term problems. Medical students and
young physicians in training continue to show a high interest in research
career$, with 26.6 percent of respondents to a 1996 Association of
American Medical Colleges-sponsored survey of medical students
indicating their intended first career choice was a full-time academic
appointment with research involvement, Despite this abiding interest, the
recruitment of young investigators is hampered by the large debt burden
borne by recent graduates, by the difference between incomes of clinical
investigators and those of colleagues who have chosen to enter private
practice, and by the perception that there is a high rate of failure in clinical
research careers, due in part to the difficulty of obtaining financial support
for research proposals, Clinical investigators also report a discrepancy in
academic advancement between those who enter clinical research and those
entering basic research, where accomplishments in the laboratory tend to
come sooner and are often held in higher regard by academic promotion
committees. Clinical research suffers from an
insufficient number of clinical research-specific training programs
nationwide, particularly the
that incorporate formal, didactic course work in areas such as protocol
design, statistics, medical ethics, and regulatory issues, Finally, there is also
a need for more successful clinical researchers who can same as role
models for their successors.
Clinical research has also been made more difficult by the advent Of
managed care. Because of the costs of conducting research and the need to
serve indigent patients, academic health centers (AHCs) typically charge
higher fees than their non-academic counterparts. In an effort to reduce
costs, managed care organizations are thus generally reluctant to refer
patients to AHCs, and when they do, they typically pay only a limited
portion of the costs of providing care. The resultant redactions in clinical
income has led to increased pressures on research faculty to turn their
attention away from research a-id to bring in clinical revenues. The end
result of this spiral of change is a decrease in the infrastructure and
personnel to conduct clinical research, a consequence little recognized by
the American public. This trend has recently been documented by a study
showing that AHCs, in areas with the greatest growth of managed care,
have been adversely affected in the competition for NIH research grants.
Finally, further medical progress requires a sound and modernized physical
infrastructure. -Accordingly, continuing investment and support in such
infrastructure elements as new buildings and refurbished facilities, and
state-of-the-art instrurnentation and information technology must be a key
component of the Nation's overall strategy for sustaining its capacity for
world class health research, There is substantial evidence that the research
infrastructure is eroding in many research arenas, including clinical
research.
What methods are being used by the NIH to evaluate the status of clinical
research?
Over the past four years, the NIH has been attempting to define the
problems that afflict clinical research and to develop a program for
alleviating them. We have also benefited from the views of other
organizations and individuals. (For example, long-standing concerns about
the number of individuals pursuing careers in clinical investigation
prompted the Institute of Medicine (IOM) to initiate a study in 1991 to
address career paths for clinical investigators, leading to their important and
useful report, "Careers in Clinical Research: Obstacles and Opportunities"
in 1994.) One of my first actions as NIH Director was to appoint Dr.
Lawrence Shulman, a former NIH Institute Director, to serve as my
emissary for clinical research and to seek the opinions and suggestions of
those investigators who are actively engaged in clinical research. A panel
headed by Dr. Gordon Williams from Harvard Medical School was
convened to examine the review of applications for clinical research grants
as performed by the NIH Division of Research Grants (DRG). In 1995, I
established The NIH Director's Clinical Research Panel, which includes
representatives from major U.S. medical centers and private industry and is
chaired by Dr. David Nathan, President of the Dana Farber Cancer Center.
The Panel was charged with broad responsibilities to review the status of
clinical research in the United States and to make recommendations to
improve its effectiveness. I asked the Panel to examine many specific
aspects of clinical research, including its financing and the toles of the
General Clinical Research Centers (GCRCs) and the Warren Grant
Magnuson Clinical Center (CC) at the NIH, as well as the recruitment and
training of clinical researchers and peer review of clinical research. (The
Panel has made a number of recommendations, and we are already
implementing many of them.) To define the needs of clinical research in the
context of specific diseases, I have stimulated the development of
workshops, conferences, and panels centered on certain clinical problems.
Prominent among these meetings have been workshops on Parkinson's
disease, autism, diabetes mellitus, and spinal cord injury.
Clinical research and training in the NIH intramural program
A crucial component of our campaign to enhance the Nation's clinical
research enterprise is focused specifically on the NIH intramural activities,
as carried out principally within the Clinical Center (CC). For 40 years, the
CC has been the embodiment of the NIH mission to improve the Nation's
health through medical research. It provides a critical venue for the
translation of basic research into clinical practice and vice versa. It has
traditionally drawn patients and clinical trainees from all over the world
who come for the study and treatment of myriad diseases and disorders,
ranging from chron1c infection, metabolic diseases, and cancers to rare
hereditary diseases. Unlike most of the AHCs at which most extramural
Clinical studies are conducted, the NIH intramural program is not directly
threatened bit recent changes in provision of health care that limit
recruitment of research subjects and make clinical research programs
unaffordable, As a result, clinical research within the NIH intramural
program will likely become even more important to the Nation than it has
been in the past.
The most visible evidence of our efforts to renew the intramural clinical
research effort is the recently initiated construction of a new facility, the
Mark O. Haifeld Clinical Research Center (CRC), to replace many
components of the existing CC, whose physical deterioration has been well
documented. Support from the Administration and Congress has provided
the funds that allow us to proceed with the detailed planning and
construction of the CRC. The CRC will encompass a state-of-the-art
research hospital with 350 beds, allied clinical facilities, and adjacent
research laboratories for work, that is closely, intertwined with clinical
activities, a traditional strength of the existing CC.
The prospect of a new CRC has re-energized clinical investigation at the
NIH. Governance of the existing CC is benefiting from a thorough
evaluation of its administrative practices, carried out in 1996 by a team of
intramural and extramural experts, led by Dr. Helen Smits, then Deputy
Administrator of the Health Care Financing Administration. I have also
initiated efforts to ensure that the intramural program continues to recruit,
retain and train the best clinical researchers, I appointed an NTH Committee
on the Recruitment and Career Development of Clinical Investigators in
order to review the current status of istramural clinical research activities on
the NM campus. That committee's recommendations indicated that
increased resources should be made available for clinical research, and
suggested that improvements in tenure and promotion procedures be
instituted to support clinical investigators. It was also recommended that
more clinical Researchers serve on the Boards of Scientific Counselors and
on ICD Promotion and Tenure Committees. These recommendations are
being implemented by a Clinical Research Revitalization Committee..
composed of Scientific and Clinical Directors and clinical investigators.
The CC is pioneering the use of information and telemedicine technologies
to increase the pool
of care providers available to particpate in clinical research, especially
clinical research protocols. Two telemedicine suites are currently under
development at the CC with the potential to create a "virtual clinical
research center at the NIH. This would link rural communities to the CC
and major academic centers throughout the country. Thus, with over 900
active clinical research protocols sponsored by the various ICDs, and an
additional 250 collaborative research protocols, the CC is using
telemedicine for patient recruitment, for patient follow-up, and for new
collaborations with primary-care investigators in remote locations. finally,
in order to enhance intellectual exchange among medical centers, selected
CC Grand Rounds are now televised live to more than 50 medical centers
nationwide, and remote participants may phone in during the question-and-answer sessions.
Historically, the CC has also been profoundly important to the continued
supply of new clinical investigators, especially physician-scientists. In fact,
many--perhaps most--of today's stars in extramural clinical research trained
at some point in their careers in the NIH intramural program. The CC, with
its large number of studies and physicians from specialty areas, is also in a
strong position to supplement ongoing efforts at some AFCs to recruit and
train clinical investigators. To that end, two years ago the CC initiated a
new curriculum in clinical research for physicians, physician assistants,
nurses, and Ph.D. scientists, open. to both intramural and extramural
investigators. The syllabus for the course will be placed on the World Wide
Web soon and a textbook is under development. Finally, in response to an
innovative recommendation by the Clinical Research Panel, the NIH has
initiated a new program to bring medical students to the NIH CC for one or
two years of intensive training in clinical research. Over eighty students
responded to the initial advertisement, and the ten students selected are
already at work on the NIH campus. We hope to expand this program if it
continues to prove successful, and we anticipate that it may serve as a
model for use at several AHCs around the country.
Training of clinical investigators at extramural sites
The changing frontiers of medical science require a continuing supply of
personnel from many disciplines, prepared to understand the implication of
current discoveries and to apply them to future clinical research
opportunities. Some of our efforts to accomplish these goals in the NIH
intramural program are described in the preceding section. A wide variety
of programs designed to develop the careers of clinical investigators
through our extramural activities have also long been part of the NIH
fabric.
Today, we must train more clinical investigators, but, more importantly, we
must train them better. We must attract talented people to the challenges of
clinical research and then provide them with the critical skills that will
enable them to perform first-class clinical research. Unfortunately, it is not
always easy to know which programs work. well. Training programs, by
definition, take many years to bear fruit and thus, years to gather the critical
mass of data that permits adequate assessment. Nevertheless, NIH is
committed to steps that we believe are likely to strengthen research training
and career development and to ensure the recruitment of investigators from
many disciplines, as well as under-represented groups, into clinical
research. To these ends, NIH is mounting a multifaceted approach, one that
involves the re-emphasis modification, and expansion of existing programs
as well as the design of new programs to meet specific needs,
This effort must span a variety of fields of research and encompass a broad
range of clinical scientists, including Ph,D. students and recipients who are
primarily laboratory-oriented, but also interested in the mechanisms of
human diseases, and M.D.-Ph.D graduates of our highly successful Medical
Scientist Training Program, Several training programs focused specifically
on young clinicians will be expanded. We will encourage AHCs to
organize existing resources into high quality training experiences in clinical
research in order to attract an increased number of highly motivated
candidates. Institutions will need to build strong clinical research programs
by attracting faculty who have the didactic and research talents necessary
for training clinical researchers. It is expected that such programs would
recruit clinicians into a patient-oriented research fellowship either at the end
of their general medical or surgical training or during the Research
fellowship portion of their subspecialty training. Individual training
positions will also be expanded to provide three to five years of support for
physicians and dentists who wish to engage in a period of closely
supervised clinical research career development. Candidates for these
programs generally will have completed their general residency and be
ready for a research training experience.
A key feature to these training initiatives is the emphasis on a didactic
portion that will ensure that the clinical investigator is well grounded in
research design, data-management and analysis along with issues of
protection of human subjects and confidentiality of records. We must
prepare this coming generation of clinical researchers to be competitive in
seeking research grant support and be fully aware of the complexity of
conducting sound clinical research.
The NIH's distributed network of 74 General Clinical Research Centers
(GCRCs), usually located at academic medical centers across the country,
play a key role in clinical research by providing both the infrastructure f"or
the support of inpatient and outpatient research and effective sites for
training and junior career development in clinical research. The purpose of
the GCRCs is to support the clinical research infrastructure for investigators
who receive their primary, research support from the other components of
the NTM, other Federal and State agencies, and the private sector. In FY
1995, the research support provided by various NIH components to nearly
8,000 OCRC-based investigators exceeded $1 billion.
Within the academic institutions, the GCRC staff.-through their
professional activities in clinical in
research, experimental design, nutritional and nursing support, biostatistics.
ethics, computer systems management--provide a focal point for training
medical students and fellows, as well as updating experienced faculty in
new developments in clinical research. Spread across 30 states, the
CTCRCs provide support for individuals through general and minority
clinical associate programs. These programs provide a way for Junior
faculty to pursue a mentored research training experience. In some
institutions, formalized GCRC lectures have attracted large audiences of
medical professionals, whole in others, more detailed curricula have been
provided for preselected students for intense immersion in subjects related
to patient-oriented research and clim'cal research trial design. In some
institutions, such career development opportunities lead to advanced
degrees in areas of specialization.
NIH is working with other agencies to issue an announcement for
applications for individual post-doctoral support and a short-term
institution-based program for scientists who want to specialize in bioethics.
Training programs may address community involvement in research
protocols, informed consent privacy of records, and other topics, including
issues related to minority patient populations and minority researchers.
Most medical and dental students have accumulated considerable debt by
the time they have -finished their training. This debt burden poses a real
barrier to young physicians and dentists interested in pursuing research
careers. This is particularly true for under-represented minorities.
Currently, NIH has authorization for loan repayment programs in the
intramural research program, and we operate three such programs. These
include the AIDS Loan Repayment Program, which supports medical
professionals carrying out AMS research and patient care at NIH- the
Clinical Research Loan Repayment Program.. which supports financially
disadvantaged and under-represented minorities at NIH; and the General
Research Loan Repayment Program, which largely supports senior post-doctoral fellows and
individuals recently recruited to tenuretrack positions,
In general, these programs pay up to $20,000/year of educational debt and
taxes. Programs last for two to three years and are renewable in some
cases.
While some have also suggested expanding loan repayment programs to
extramural institutions, this would require a legislative change. In
conjunction with entry level research training programs, the NIH loan
repayment programs have served as important tools for recruiting high
quality candidates for careers in clinical investigation,
The clinical research enterprise, like all research areas, is dependent on the
fair and equitable review of applications submitted to the NIH. The
majority of the clinical trials and much of the other clinical research is
reviewed within the Institutes and Centers. This ensures that these
applications are reviewed by groups specifically constituted for a single
review task and all members are likely to be skilled at reviewing clinical
projects. The remainder of the reviewing responsibilities for clinical
research applications resides in the Center for Scientific Review (formerly
the Division of Research Grants), which provides revieves for most
unsolicited research projects of all types at the NIH.
In its analysis of DRG's efforts to review applications for clinical research
grants, the Williams Committee noted that the success rates for such
applications were likely to be lower than expected when considered by a
review group that does not see a high volume of clinical applications. To
address this concern, Dr. Elvera Eluenfeid, the newly appointed Director of
CSR, has made the issue of the review of clinical research a major focus.
She has met with representatives of numerous clinical professional
societies, the Association of American Medical
Colleges, and medical school deans and clinical department chairs. She has
recruited Dr. Michael Simmons, a distinguished clinical investigator, to
serve as a liaison with the extramural clinical research community. Drs.
Ehrenfeld and Simmons have also met with directors and staff of each
involved Institute to discuss this issue and potential solutions.
CSR will soon experiment with some new special emphasis panels.-one,
with a core of members expert in clinical trial design, execution, and
evaluation, for review of applications to conduct clinical trials, and others to
review clinical applications that are currently reviewed in study sections
with a low density of clinical applications. To further ensure equitable
review for investigators applying for clinical research grants, an
experienced reviewer of clinical research ..will be available to provide
guidance to study sections,with a low proportion of clinical applications.
The impact of managed care
AHCs remain responsible for the lion's share of our national biomedical
research effort and stand uniquely poised to transfer basic research
knowledge into improved medical therapies, With changes in health care
delivery, collaborations between managed care and ABCs have become
increasingly important, For such collaborations to succeed, the leaders of
the managed care industry and the companies that purchase such care for
their employees must be persuaded of the importance of medical research to
clinical practice. Experience shows that high quality health care and
research go hand-in-hand, and organizations that conduct research are
generally viewed as providing the best care. As collaborative ventures
between managed care companies and academia proliferate, both partners
stand to benefit, with a health dividend for all Americans.
A number of activities under way at the NIH are focused on strengthening
ties between managed care and academia, and fostering a better
understanding between these communities - Oyer the course of this past
year, I have established a productive dialogue about clinical research with a
number of potential partners, including representatives of managed care
organizations (MCOs) and member groups of the American Association of
Health Plans (AAHP). To act as a liaison among NIH Institutes, AHCs, and
MCOs, I initiated an NIH Fellowship in Managed Care, currently filled by
Dr. Edward Wagner, a physician-scientist from the Puget Sound Health
Plan. I have also established an NIH Managed Care Workgroup, comprised
of representatives from each Institute and Center, which will serve as a
central resource for coordinating collaborative efforts with the managed
care community - The Workgroup,will share strategies employed by
various ICDs, develop proposals for grass-roots experiments in clinical
research, and design innovative approaches for linking AHCs with managed
care partnerships. One model of a successful, partnership, announced this
past year, was developed between the National Cancer Institute (NCI) and
the Department of Defense (DoD), permitting beneficiaries of DoDs health
benefits program access to NCI-sponsorcd clinical trials. This agreement
should have the dual advantage of increasing the quality of health care for
DoD employees, by providing them with promising cancer therapies, and
helping to secure an adequate supply of patients for NCI's clinical trials.
Similar partnerships have been negotiated between the NCI and the
Department of Veterans Affairs and between the NHLBI and the Health
Cam Financing Administration.
This past July, the Board of Directors of the AAHP, with over 1000
member organizations, adopted a new policy intended to encourage health
plans to permit their beneficiaries access to NIH-sponsored clinical trials.
The Board indicated that plans participating in clinical trials should
consider covering the routine patient care costs incurred during the course
of those studies. A joint NIH-AAHP negotiating team will convene next
month to develop a consensus regarding the central working principles
underlying this arrangement. We look forward to achieving the benefits to
clinical research that these developments promise. Earlier this summer. I
also held an introductory meeting with purchasers of health care--the
representatives of those 'large companies that employ significant numbers
of Americans and pay for the health care benefits their employees receive--to discuss the value of
research in the continuum of first-rate medical care.
Partnerships with industry
Clinical research activities sponsored by the NIH have traditionally required
close relationships with the pharmaceutical and biotechnology industries.
NIH trains many of the people who work in those industries, NIH-supported basic research
commonly serves as the point of departure for the
development of new products by industry, discoveries in the public and
private sectors are frequently exchanged through technology transfer
agreements, and financial support of research flows in both directions (with
NIH providing funds to industry through Small Business Innovation
Research and Small Business Technology Transfer grants and industry
providing resources to intramural NIH investigators through Cooperative
Research and Development Agreements and to our extramural investigators
through sponsored research agreements).
The relationship between NIH and the pharmaceutical industry is of
particular importance for clinical research because of shared interests in the
training of pharmacologists and chemists, in the design and conduct of
clinical trials, and in the discovery of new therapeutic agents. This summer
I met with representatives of several large pharmaceutical companies to
discuss some of these issues and to explore ways in which NIH and the
industry can work more effectively together. To this end,NIH will soon
convene a series of collaborative forums, with representatives from
government, industry, managed care organizations, and academia, to
discuss the training of clinical pharmacologists, new methods for drug,
evaluation and drug development, and the conduct of clinical trials.
Outreach
NIH is also expanding its efforts to educate the Public about the crucial
importance Of clinical research for the future health of the Nation. it is clear
that both the public and policy makers, as well as State and local
governments, need to be kept aware of the health and economic benefits of
research as a public good. I have sought means to enhance communication
of NM activities more broadly to patients and their families, to traditional
providers, and to the health care plans where more than half of all
Americans now receive their health care. The NIH World Wide Web site,
which includes sites for each Institute, Center and Division, contains a
wealth of information about various activities, including interim reports
from the Panel on Clinical Research. Efforts are under way to establish a
web site that will provide public access to a more extensive database of
NIH-supported clinical research protocols staged at all extramural sites. I
anticipate that these web sites will enhance public knowledge about
advances in research, stimulate participation in NIH-supported clinical
trials, and accelerate the translation of research findings into widespread
medical practice.
Conclusion
In this testimony, I have attempted to describe some of the many new
initiatives that the NIH has launched in the past few years to sustain and re-invigorate the
Nation's clinical research effortsIt is essential that today's
remarkable laboratory findings-findings that reflect decades of Federal
research investment--be translated into improved diagnosis, treatment, and
prevention of disease. Through activities of cur intramural and external
programs and through exercise of our leadership in the medical research
community, we intend to expand our partnerships with AHCs, universities,
pharmaceutical and biotechnology industries, and health care providers.
We pledge to use these partnerships to ensure that the infrastructure to
support clinical research and its training programs will provide the
American people with those extensions of "biological discoveries to clinical
settings" that are required to achieve our traditional goal: improved health
through science.
I would be pleased to answer any questions you may have.