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Ad Hoc Group for Medical Research Funding – FY 2003 Proposal
Executive Summary
Revolutionizing the Future of Medicine: The Federal Investment in medical research through
the National Institutes of Health (NIH) promises to revolutionize how we diagnose, treat, and
prevent and cure disease. Congress and the Administration have accelerated the fulfillment of
this potential by committing, in a bipartisan fashion, to double the NIH budget over the five-year
period FY 1999 to FY 2003. Three primary reasons for doubling NIH’s budget include the many
health challenges that still confront us, the burgeoning scientific opportunities that are now
available, particularly as a result of the scientific achievement of sequencing the human
genome, and the large economic benefits that accrue as we make progress against diseases.
For these reasons, the Ad Hoc Group for Medical Research Funding joins with the President
and the Congress in supporting an appropriation of $27.3 billion for NIH in FY 2003, to achieve
the bipartisan goal of doubling NIH by FY 2003.
Extending Research from the Genome to the Patient: NIH’s support for basic research has
resulted in unprecedented opportunities in genetics and an increased emphasis on the
application of fundamental knowledge to specific diseases through clinical research.
Providing the Infrastructure for Continued Discovery: Investigator-initiated research is the
engine that drives scientific creativity and productivity. However, to realize fully the potential of
our scientists’ creativity, additional support is also required for a variety of mechanisms that
support a state-of-the-art research environment. A stable, well-maintained research
infrastructure includes support for training, facilities, instrumentation, imaging, and information
technology.
Maintaining the Public’s Trust: NIH has a thorough merit-review system for deciding which
research proposals to fund and which areas of science and health needs to pursue. It also has
systems to monitor the research conducted by grantees to ensure it is carried out and reported
ethically, accurately, and accountably.
Sustaining the Momentum of Research to Confront Future Challenges: Recent NIH
investments have helped create new diagnostic methods, new treatments, new vaccines, and
new cures. Doubling the NIH budget will provide the additional resources needed to enable
American scientists to address the burgeoning scientific opportunities and new health
challenges that continue to confront us. When the doubling of the NIH budget is achieved, there
will still be many disease burdens facing us, and NIH-supported scientists will remain our best
hope for solving them. It is therefore essential to sustain the momentum of NIH-funded research
so that it continues to be able to meet the ever-evolving responsibility of improving the health of
all Americans.
Introduction
Imagine walking into the doctor’s office for your
annual check-up. Instead of going through a
battery of uncomfortable and expensive tests, a
drop of your blood is placed on a microchip.
Within minutes, you are told not only the state of
your health today, but also whether you are at
risk for any particular disease or illness in the
future. And to ward off that threat, your doctor
prescribes a drug or even a vaccine designed
with your genetic make-up in mind.
Science fiction? This could become science
fact, especially when you consider the onceunimaginable gains that have been achieved our
health.
years. NIH research played a major role in
lowering the death rates for heart disease by 48
percent and stroke by 61 percent during this
same period. An estimated 80,000 more people
with cancer are alive each year compared to four
decades ago. NIH research has aided in the
development of new medications that reduce or
eliminate the symptoms of schizophrenia in 80
percent of patients, just as it has made possible
vaccines against infectious diseases that once
killed and disabled millions of children and
adults. Most recently, medical researchers have
been marshaled to help the nation respond to
new menaces, including bioterrorism and drugresistant infectious diseases.
Research supported and conducted by the
National Institutes of Health (NIH) - the world’s
pre-eminent medical research organization may soon lead to the kind of personalized
diagnosis, prevention, and treatment of disease
envisioned above, if the recent past is any
indication. Due in large part to an intensive
medical research effort begun shortly after World
War II, this country has built a remarkable health
record. Life expectancy in 2000 reached an alltime high of 76.9 years; in 1970, it was 70.8
Presidents and Congresses have longrecognized the promise and value of investing in
medical science. But that support took on even
larger proportions when, in 1998, Congress
made a bipartisan commitment to double the
funding for the National Institutes of Health in
five years (FYs 1999-2003). This bold and
unprecedented action reflected both the health
challenges facing our Nation, and the
unparalleled opportunities science holds for
meeting those challenges.
The more than 300 organizations of the Ad Hoc Group for Medical
Research Funding, representing patients, volunteers, research institutions,
scientists and medical professionals, wholeheartedly agree that research
offers the best hope for saving lives, reducing human suffering, and
lowering health costs. To those ends, the Ad Hoc Group strongly urges
our nation’s leaders to provide an appropriation of $27.3 billion for NIH in
fiscal year 2003.
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The Rewards of Research
The Challenges that Remain
Research supported and conducted by NIH has
had a profound and far-reaching impact on
society in many important ways, serving as a
catalyst for new products and technologies,
creating skilled jobs, and contributing to the
nation’s economic growth. But above all else,
the central tenet of medical research is to
safeguard and improve the lives of all our
citizens.
Despite these impressive achievements, much
work remains to be done. There still is a
formidable list of diseases for which there are no
cures or effective treatments, and we are
continually confronted with new challenges to
good health: the emergence of new, lifethreatening diseases and infectious agents; the
increased health risks associated with a rapidly
aging population; racial and ethnic disparities in
the prevalence and severity of many diseases;
and social, environmental and behavioral factors
that undermine good health.
In that regard, NIH research is paying enormous
dividends. Americans are living longer, more
productive lives; infant mortality rates have been
reduced; and communicable diseases that once
were thought incurable have been all but
eliminated. And the quality of those added years
of life also has improved, as evidenced by the
steady decline, by 1 - 1.5 percent per year over
the last two decades, in the number of elderly
experiencing developmental, physical and
mental disabilities.
Chronic diseases like cancer, cardiovascular
disease and diabetes continue to claim the lives
of millions of Americans each year, wasting
precious human resources and draining billions
of dollars from the nation’s economy.
Economic Burden of Illness
Annual Cost
Disease/Condition
(in billions)
Injury
$338
Alcohol Abuse and Dependence
$185
Heart Diseases
$183
Disability
$169
Mental Disorders
$161
Smoking
$138
Drug Abuse
$110
Alzheimer’s Disease/Dementia
$100
Obesity
$99
Diabetes
$98
Cancer
$96
Chronic Pain
$79
Arthritis
$65
Digestive Diseases
$56
Stroke
$43
Source: “Disease-Specific Estimates of Direct
and Indirect Costs of Illness and NIH Support,”
NIH, February 2000.
NIH research has produced safe, cost-effective
vaccines against a number of diseases,
including hepatitis B, Haemophilus influenzae
type B, pneumococcus and pertussis, that have
the potential to save millions of lives a year.
In May 2001, the FDA approved Gleevac™ for
use in treating a serious blood cancer known as
chonic myelogenous leukemia. This drug –
developed as a result of more than 40 years of
NIH-funded research and a successful
collaboration with industry – is the first of its kind
to be approved: a drug that targets specific
molecules in cancer cells, leaving healthy cells
unharmed.
Newly developed medications for schizophrenia
have reduced psychiatric hospitalizations nearly
30 percent between 1996 and 1998. By reducing
dangerous and unwanted side effects, these
drugs have increased patient treatment
compliance rates and resulted in estimated
savings in hospitalization costs of $1.7 billion
annually.
The aging of our population is creating
enormous challenges in health care. Nine years
from now, “Baby Boomers” will begin to turn 65.
By 2050 the number of Americans age 65 and
over will have more than doubled and the
number of Americans over age 85 will have
increased five-fold. With increasing age comes
increasing risk of disease and disability. Society
must find ways to preserve the physical,
cognitive, and social functioning of our older
citizens, and when they do fall ill, we must work
to ensure the availability of effective treatments
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for conditions such as Alzheimer’s disease,
osteoporosis, arthritis, chronic pain, blinding eye
diseases, depression, and other health problems
prevalent in the elderly.
Mental illness continues as a national concern,
particularly among our younger citizens. In the
United States, 1 in 10 children and adolescents
suffer from mental illness severe enough to
cause impairment. The Surgeon General
released a report in January 2001, announcing a
“national crisis” in children’s mental health. The
report calls for the further development of
scientifically proven prevention and treatment
services.
Infectious diseases, the second leading cause
of death worldwide, present an ever-growing
threat to millions of Americans with the
emergence of infectious agents like hepatitis C,
and the re-emergence of diseases like
tuberculosis, malaria and cholera in more
virulent and drug-resistant forms.
Health conditions, often involving intricate
biological factors, that are manifested in
behaviors – often at an early age – such as
obesity, drug and alcohol addiction, smoking,
sexually-transmitted diseases, violence and teen
pregnancy, take an enormous health, social and
economic toll on the nation. Increased
behavioral research has the potential to
substantially reduce these and other health
problems including heart disease, lung disease,
cirrhosis, neurological disorders, developmental
disabilities, and many others.
Greater risks befall minorities and the poor
who have not shared equitably in the progress in
the nation’s overall health, and experience
continuing disparities in the incidence of illness
and death from cancer, hypertension, diabetes,
infant mortality and AIDS.
Responding to the Increased Threat of Bioterrorism
Research has been underway for years to improve America’s capacity to respond to bioterrorism threats
or attacks. For example, more than a year ago, the National Institute of Allergy and Infectious Diseases
began a study to determine the effectiveness of the smallpox vaccine at different levels of potency.
In response to increasing concerns over potential bioterrorism threats or attacks, NIH recently
announced several new, more intense research initiatives, and Congress has added additional
bioterrorism funding. Many of these new programs will build on past studies and findings, applying the
latest technology and methods in order to expedite research on high priority diseases such as smallpox
and anthrax. The goals are to:

Seek new medications and faster, more accurate diagnostics for other diseases that could pose a
bioterrorism threat.

Apply the latest technologies to map the gene sequences of particular infectious organisms.

Establish multidisciplinary research units to investigate diseases that could be used by terrorists and
rapidly disseminate their findings.
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Economic Productivity and
Job Creation
The JEC found that of the 21 most important
drugs introduced between 1965 and 1992, 15
were in part the result of federally-funded
research, and seven were the direct result of
NIH research. The success and prosperity of
the U.S. pharmaceutical industry has long
depended upon NIH-supported science for
"enabling discoveries" and for training the skilled
workers needed to develop cures and therapies.
The revolutions in molecular biology and in
genomics can only strengthen this reliance.
The economic returns from medical research are
an additional reason for supporting increased
NIH funding. A May 2000 Ernst and Young
study entitled The Economic Contributions of the
Biotechnology Industry to the U.S. Economy
found that the biotechnology industry employed
150,800 people in 1999. Adding the purchases
from other related companies and the spending
of their employees, the biotechnology sector
generated an additional 287,000 jobs in 1999.
Product sales for the biotechnology industry
grew by 33 percent that same year for a total of
$20.2 billion.
Fundamental Science: The
Foundation of Health Research
According to the Congressional Joint Economic
Committee (JEC), high-tech and research-driven
industries, including biotechnology and the
pharmaceutical industry, have contributed
directly to one-third of our nation’s economic
growth over the past decade.
New technologies, new methods of inquiry, new
scientific perspectives - these are the building
blocks of medical research in the 21st Century,
and NIH is the major supporter of fundamental
science. We often think of NIH as being
responsible for producing what we know about
specific diseases. But NIH also is a driving force
for progress in science more generally – for
advances in the conduct of research that allow
us to better examine many specific diseases.
Health Care Cost Savings
Cardiovascular disease provides a dramatic
example of the economic return possible from a
disease prevented, ameliorated or cured.
According the JEC, savings resulting from the
reduction in cardiovascular mortality and ensuing
longevity and economic productivity are
estimated at $1.5 trillion a year. If one-third of
this reduction in disease and resulting economic
benefit can be attributed to medical research, the
return on the research investment could be $500
billion annually, nearly 28 times the size of the
FY 2002 NIH budget.
To provide just a handful of examples, NIH has
played a central role in developing advanced
imaging technologies, animal models of disease
and health, high-end computational facilities,
state-of-the-art instrumentation, non-invasive
diagnostic techniques, shared databases,
sophisticated assessments of health disparities
in ethnic minorities, improved prediction of risk
factors for a variety of conditions, new ways of
analyzing longitudinal data, and innumerable
other accomplishments that have allowed our
Nation’s scientists to pursue new research
directions that even a short while ago were
unimaginable.
The May 2000 report commissioned by the Mary
Woodward Lasker Charitable Trust, Exceptional
Returns: The Economic Value of America’s
Investment in Medical Research, states that
medical research that reduced deaths from
cancer by just one-fifth would be worth $10
trillion to Americans – double the national debt.
In fact, the cancer death rate has dropped more
dramatically for children than for any other age
group. Up to 70 percent of all children with
cancer can now be cured.
As outlined on the following pages, two trends
that have emerged as a result of NIH’s support
for basic research are the increasing importance
of genetics and of clinical research in the
research agenda for all of the NIH institutes and
centers.
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GENETICS
From Molecular Medicine to Human Health
Less than 50 years after the landmark discovery of the structure of DNA, the Human Genome Project - an
international collaboration led by NIH’s National
Human Genome Research Institute - reported the
initial sequence and analysis of the working draft of
Finding the Genes that Contribute to Disease
the DNA sequence of the human genome. Just as
and Illness
Watson and Crick’s discovery ushered in the era of
molecular biology in research, the completion of the
 The National Institute of Environmental
human genome heralds a revolution in the practice
Health Sciences is conducting studies to
of medicine.
identify critical variations in genes that affect
an individual’s response to toxins.
Deciphering the human genetic code – what has
been described as the “instruction book for human
biology” – surely would not have been possible
without a sustained commitment of federal funding.
Consider that prior to launching this collaborative
effort, it took scientists nine years to find the gene
for cystic fibrosis; under the Human Genome
Project, scientists precisely described a gene for
Parkinson’s disease within nine months.
Obtaining the complete sequence of the human
genome is only the prelude for things to come. For
the full impact of advances in genetics to be felt in
the practice of medicine, major challenges must be
addressed. Much work remains to understand how
the human genome carries out its many functions.
But the consequences for the diagnosis, prevention,
and treatment of disease will be profound.

National Institute of Mental Health research
has led to the discovery of a genetic
vulnerability to schizophrenia.

A recent study from the National Institute of
Neurological Disorders and Stroke indicates
that a specific gene can dramatically
improve the ability of adult neurons to
regenerate.

In collaboration with their European
colleagues, scientists funded by the National
Institute of Child Health and Human
Development and the National Institute on
Deafness and Other Communications
Disorders have identified regions of four
chromosomes that appear to be linked with
autism.
Much of the progress in understanding and treating
human diseases has come from research using
experimental animals, especially the mouse.
Animal research will play an important role in helping scientists understand the biological functions of
genes. Because mice and humans share virtually the same set of genes, the sequence of the mouse
genome will provide a critical perspective to help scientists understand the human genome. Scientists will
not only be able to find genes easily in the sequence of the mouse genome, they also will be able to test
possible therapies for humans in mouse models of disease.
Genomic medicine holds the ultimate promise of revolutionizing the diagnosis and treatment of many
illnesses. Some scientists predict that using genetics to predict individual risks of disease and
responsiveness to drugs will reach the medical mainstream in the next decade or so. The development of
so-called “designer drugs,” based on a genome approach to targeting molecular pathways that are
disrupted in disease, will become more common soon after.
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CLINICAL RESEARCH
From the Bench to the Bedside
Medical research often is described as a continuum, from basic research to clinical research. But the
journey from basic research to human health is not an automatic, linear evolution. Studies must be
undertaken at several stages, beginning with translational research to explore whether there are possible
health applications for basic research findings, and moving through clinical research – including clinical
trials – to the actual use in clinical practice.
Past investments in basic research supported by NIH have produced a wealth of knowledge about the
fundamentals of human health and disease. But this accumulation of knowledge must be put to use to
fulfill NIH’s health mission and to realize the full value of the nation’s research investments. An integral
part of NIH’s mission is to move the unprecedented amount of information generated by basic research
from the hands of the laboratory scientist to the practitioner.
By supporting clinical research as well as basic research, NIH is ensuring that new knowledge can be put
to use in preventing, diagnosing and treating disease and disability.
Moving knowledge from the laboratory bench to the patient is neither easy nor inexpensive. The scope,
size and pace of modern medical research is nowhere more apparent than in clinical research, particularly
clinical trials, which requires setting up teams to design research protocols, recruit participants, conduct
the trails, and analyze the data. The Ad Hoc Group supports increased funding for additional clinical
research infrastructure, including general clinical research centers (GCRCs) - a national network of
approximately 75 centers, usually located within teaching hospitals, with specialized resources and
infrastructure to support clinical investigations - and community-based clinical research.
Clinical Research Across the Nation:
Translating Basic Research into Effective Therapies

The National Institute of Diabetes and Digestive and Kidney Diseases is continuing studies to develop
prevention strategies through its Type 1 Diabetes TRIALNET and has issued two requests for applications
to develop clinical trials for the prevention and treatment of Type 2 diabetes in children and adolescents.

The National Institute on Drug Abuse has launched a Clinical Trials Network to test promising
pharmacological and behavioral therapies across a wide range of community-based treatment centers
and diverse patient populations.

The National Institute of Neurological Disorders and Stroke is supporting a network of acute stroke
centers that will provide rapid treatment to patients and serve as a clinical laboratory for related studies,
including the testing of new medicines

The National Institute of Allergy and Infectious Diseases continues to support programs to build the
infrastructure for clinical trials, including the Immune tolerance Network, the AIDS Clinical Trials Group,
and the HIV Vaccine Trials and Prevention Trials Networks.

The National Institute of Arthritis and Musculoskeletal and Skins Diseases and the National Institute on
Aging are collaborating with other NIH institutes and centers and pharmaceutical companies on the
Osteoarthritis Initiative, and public-private partnership to provide resource for clinical research.
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Research Infrastructure Required
To Produce Discoveries
Setting Priorities,
Ensuring Accountability
Investigator-initiated research is the engine that
drives scientific creativity and productivity. NIH
fosters this creativity through its investment in
research project grants. However, to realize fully
the potential of our scientists’ creativity,
additional support is also required for a variety of
mechanisms that support a state-of-the-art
research environment. A stable, well maintained
research infrastructure includes not only
innovative research tools and facilities, but also
investments in people, education and
technology.
NIH is the largest and most comprehensive
medical research enterprise in the world,
supporting scientific study at more than 2,000
universities, medical schools, hospitals and
research institutions spread throughout the
country; supporting training of over 15,000
investigators annually for careers in science; and
conducting research in its own intramural
laboratories. Through its extramural research
program, NIH enlists the talents of approximately
50,000 scientists. The roster of individuals who
have received NIH support in the past includes
97 Nobel Prize winners, five of whom made their
prize-winning discoveries while working in NIH
laboratories.

Training - The next generation of
researchers needs adequate stipend and
tuition support. Current levels of support
provide little incentive for young scientists to
pursue careers in research. Efforts to
increase the participation of minorities in
research careers should be expanded and
enhanced.

Facilities - More cutting-edge research
requires more state-of-the-art labs. For
example, pioneering new research into viral
pathogens requires very costly Level 4
laboratories, which have increased safety
and security measures.

Instruments - Increasingly sophisticated
equipment is required for today's research.
One time investments in large
instrumentation such as mass
spectrometers can be used by multiple
researchers from a variety of disciplines.

Imaging - New imaging methods have
revolutionized diagnostics and treatment
design. For example, new high powered
magnetic resonance technology allows
scientists to witness the real-time effects of
drugs or behavior on patients’ brains.

Information technology - increasingly
powerful computing has become a mainstay
of advanced medical research as research is
producing more complex data.
Bioinformatics is fusing medicine and
information technology to help diagnose,
monitor and treat disease.
Entrusted with the nation’s largest investment in
medical research, NIH is responsible for
ensuring that the taxpayer’s dollars are allocated
prudently, consistent with national priorities, and
in a fashion that holds recipients accountable for
meeting stringent legal and ethical standards for
the protection of human subjects, the care and
well-being of animals used in research
experiments, and the handling of biohazards. To
those ends, NIH has in place a carefully
constructed system for setting research
priorities, and a tiered review process to ensure
that those grant applications of highest scientific
merit are funded.
NIH sets priorities through a complex series of
evaluations and judgments among its more than
two dozen research institutes and centers, each
of which has a different research focus. The first
level of priority-setting is guided in large part by
the annual appropriation for each institute and
center. The next occurs when applications for
funding are initially reviewed for their scientific
merit by a panel of outside experts. Applications
that score sufficiently high are then assigned to
the most appropriate institute or center, where
they undergo a second evaluation by an advisory
council composed of scientists and a crosssection of public representatives.
Universities, medical schools, teaching hospitals,
independent research institutions, and federal
laboratories, take seriously their responsibilities
to ensure that Federal research funds are used
carefully to advance science. The recent
strengthening of the oversight system involving
protection of human subjects in research has
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caused investigators and the institutions in which
they work to re-focus their attention to ensuring
that human beings who are the subject of
research are treated ethically and responsibly.
Institutions and investigators must also adhere to
federal laws and regulations governing conflict of
interest, scientific misconduct, care and
treatment of animals, and the handling of
hazardous materials.
Sustaining the Momentum
Of Discovery
NIH funding has helped to create the new
diagnostic methods, treatments, vaccines and
other preventive measures and cures that we
described above. But there still will be many
challenges ahead. An ongoing commitment to
sustaining the momentum of NIH-funded
research will remain our best hope for solving
them.
One measure of whether the nation's medical
research system is setting the right priorities and
funding the best scientists to work on the most
important scientific problems in a cost effective
manner is to compare the frequency of medical
breakthroughs generated by NIH-supported
scientists to those generated by scientists
supported by other nations. By this measure,
NIH has been the world's leader in medical
research, and America has the world's most
highly acclaimed and emulated medical research
system.
Even with the additional funding provided by the
current doubling effort, there still are two highquality unfunded research proposals for every
one that gets funded — any of which could be
the idea that leads to the cure for Alzheimer’s,
cancer, or diabetes.
Medical research is a long-term process. The
scientific “breakthroughs” trumpeted by the
newspapers and television are the result of years
– sometimes decades – of basic and clinical
research. Ensuring future breakthroughs
requires sustained investments to attract and
retain first-class researchers and to create and
maintain state-of-the-art laboratories.
If NIH research is to continue to meet the everevolving challenge of improving the health of all
Americans, we as a nation must continue our
commitment to medical research. The Ad Hoc
Group urges Congress and the Administration to
finish the job of doubling NIH by appropriating
$27.3 billion in FY 2003. We look forward to
working with the Executive and Legislative
branches to increase support for this critically
productive enterprise that enhances the quality
of life for all Americans in the years to come.
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