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U.S. Department of Health and Human Services National Institutes
of Health
Fiscal Year 2007 Budget Request House Subcommittee
on Labor - HHS - Education Appropriations
Dr. Elias A. Zerhouni, Director
April 6, 2006
(Slides used during the presentation: NIH
and the Transformation of Medicine, (PDF
- 4.79 MB))
Mr. Chairman and distinguished members of the subcommittee, it
is an honor and a privilege to appear before you today to present
the National Institutes of Health (NIH) budget request for Fiscal
Year 2007 and discuss the priorities of NIH for this year and beyond.
Budget Request
The request for NIH is $28.4 billion in FY 2007, the same as the
FY 2006 level for the Agency. The budget request will support the
research programs managed by NIH's Institutes and Centers. At this
budget level, NIH will increase the biodefense research program
by $110 million for Advanced Development. Support for the Pandemic
Influenza Preparedness Plan will increase by $17 million. We have
also chosen to carefully invest in several trans-NIH strategic
initiatives. The NIH Roadmap, an incubator for new ideas and initiatives
that will accelerate the pace of discovery, increases by $113 million.
We allocated $40 million to the Institutes and Centers to launch
the Genes, Environment and Health Initiative to accelerate discovery
of the major genetic and environmental factors for diseases that
have a substantial public health impact. We have also directed
$15 million to the new "Pathway to Independence" program
to increase our support of new investigators.
I will focus my testimony on the return of the investment in NIH
for the American people. In particular, I will discuss how discoveries
fueled by this investment are transforming the practice of medicine.
We can now clearly envision an era when the treatment paradigm
of medicine will increasingly become more predictive, personalized
and preemptive. We will strike disease before it strikes us with
the hope of greatly reducing overall costs to society. We expect
to move away from the costly and predominantly curative model of
today, which requires us to wait for the disease to occur before
intervening. I will share with you the strategic vision of NIH
and discuss the many management innovations we have implemented
to ensure optimal stewardship of taxpayers' resources.
Selected Accomplishments of NIH and their Impact on Health
The achievements of NIH and our private sector partners in medical
research are difficult to overstate. According to the latest report
on the Nation's health from the Centers for Disease Control and
Prevention (CDC), life expectancy continues to rise, now at an
unprecedented 78 years for the total U.S. population. Since 1950,
the age-adjusted death rate for the total population declined by
a remarkable 43 percent. Life expectancy has increased by one year
in every five for the past 30 years. Americans are not only living
longer, they are healthier. For instance, the disability rate of
American seniors dropped by almost 30 percent in the past 20 years,
owing to a range of scientific advances.
The following are samples of the many advances driven by the investment
in NIH.
Advances in Cardiovascular Disease and Stroke
Thirty years ago, it was common for a man or woman to suddenly
die of a heart attack or stroke between the ages of 50 and 60.
Had this trend continued unabated, today more than 1.6 million
lives would have been lost per year. Fortunately, today the toll
is much less. The death rates from cardiovascular disease have
declined by 63 percent and by 70 percent for stroke. Were it not
for the ground-breaking research on the causes and treatment of
heart disease, supported in large part by NIH, including recent
developments such as drug coated stents, safe levels of blood pressure
and cholesterol lowering therapies, heart attacks would still account
for 1.2 to 1.3 million deaths per year instead of the actual 515,
000 deaths experienced today. The estimated total cumulative investment
in cardiovascular research at the NIH per American over the past
30 years, including the doubling period, is about $110, or about
$4 for each American per year over the entire period.
Advances in Cancer
The mortality rates of cancer, the second leading cause of death
in the United States, have been falling for several years, and
this year, for the first time in history, the absolute number of
cancer deaths in the United States has decreased. More effective
therapies have led to improved outcomes for more than 10 million
American cancer survivors. With the increase in budgets between
1999 and 2003, the National Cancer Institute has stimulated a paradigm
shift in cancer therapy. We are seeing the emergence of targeted
therapies, with the unprecedented ability to use specific molecular
targeting to treat tumors with novel agents. We can also detect
and treat cancer at earlier stages. The National Cancer Institute's
(NCI) Early Detection Research Network (EDRN), launched in 1999,
has identified a number of biomarkers that allow for the earlier
detection of breast, prostate, colon, lung and other cancers. This
year, NCI, in collaboration with the Human Genome Research Institute,
has launched a cancer genome pilot project to help further our
understanding of the basic biology of cancer and identify additional
treatment targets. The estimated total cumulative investment at
the NCI per American over the past 30 years, including the doubling
period, is about $258, or about $9 per American per year over the
entire period.
Advances in HIV/AIDS
Without the development and testing of antiretroviral drugs, there
would be no hope for patients with HIV/AIDS. The development of
Highly Active Antiretroviral Therapies primarily resulted from
the work of a large cadre of NIH-supported scientists and their
counterparts in the pharmaceutical industry. Their discoveries
about the cellular mechanisms of the disease have transformed AIDS
into a manageable disease, preventing hundreds of thousands of
hospitalizations and early deaths. To date, 21 antiretroviral drugs
and 4 combination formulations have been approved by the FDA. Many
more less toxic AIDS drugs are currently in development. Today,
fewer than 50 HIV-infected babies are born each year in the United
States, sparing 16,000 to 20,000 children from AIDS through the
use of antiretroviral drugs to prevent mother-to-child transmission.
Mother-to-child transmission rates in developing countries have
declined by 40 percent with the use of drug therapy. With the introduction
of these new drugs, economists estimate the aggregate potential
value of improved survival has been nearly $400 billion for those
infected through 2000. They estimate the aggregate potential value
for all past and future cohorts of individuals infected with HIV
is almost $1.4 trillion.
With the additional resources provided during the doubling of
the NIH budget, we launched the Vaccine Production Program (VPP)
Laboratory to efficiently translate candidate research vaccines,
including HIV vaccines, into useable products. Since its inception
in 2001, this program has overseen the manufacture of over 29 bulk
pharmaceutical compounds formulated into 14 different vaccine products
for HIV, as well as West Nile, SARS and Ebola Virus, and expanded
our network of clinical trial sites across the globe. This program
is enabling NIH to serve the needs of the American people in an
age of global risks of infectious diseases.
Advances Against the Threat of Pandemic Influenza
Thanks to fundamental advances in viral genomics and genetic engineering,
NIH has been able to help in the development of countermeasures
against both seasonal and pandemic influenza viruses. We now have
a vaccine against the H5N1 virus and will develop a second one
in conjunction with CDC. Without such a vaccine, and others under
development and testing, we would be completely defenseless against
the potential pandemic that threatens the entire world. We are
investing in research and development to hasten the production
process by converting from egg-based to cell culture-based vaccines.
We are developing novel vaccine approaches using a variety of molecular
biological techniques, and we launched discovery efforts for new
anti-viral compounds against pandemic flu. We initiated a project
to identify the genomes of thousands of human and avian influenza
viruses, and, to date, 831 influenza genome sequences from human
isolates have been deposited in NIH's GenBank, allowing researchers
across the world to better understand influenza viruses and develop
countermeasures.
Development of Biodefense Research
Since 2001, NIH has directed more than $10 billion toward protecting
the American public from bioterrorism. The 2001 intentional release
of anthrax underscored the reality of a bioterrorism threat posed
by other Category A agents such as smallpox, plague, tularemia,
hemorrhagic fevers, and botulinum toxin. NIH responded swiftly.
Promising vaccine candidates for Ebola and smallpox are currently
in clinical trials. Identification of the three-dimensional structure
of the anthrax toxin complex is fueling the search for compounds
that block the toxin's effects, and the discovery of the key mechanism
of Ebola virus cell entry prompted experiments demonstrating that
Ebola infection could be blocked in laboratory tests. We continue
to build a national biodefense research infrastructure that will
position the Nation to respond even more quickly and precisely
to bioterrorism.
Advances in Diabetes and Related Illnesses
Nearly 21 million Americans have diabetes, a disease that can
cause damage to multiple organs and lead to death. Without NIH
research, the improvements of the past two decades in the therapies
for diabetes would not have occurred. Through large prospective
trials, made possible by the doubling of our budget, we have assessed
the relative value of drug based approaches versus weight loss
and physical activity, and showed it is possible to reduce the
risk of type 2 diabetes by 58 percent with lifestyle modifications
alone.
Diabetes can also result in vision loss. Four million American
adults suffer from diabetic retinopathy, the outcome of damage
to the tiny blood vessels in the light-sensitive retina lining
the inside of the eye. Nearly a million have the advanced vision-threatening
stage of the disease. The National Eye Institute completed a series
of landmark clinical trials to develop novel treatments for diabetic
retinopathy. Without these new treatments, 450,000 patients who
have advanced disease today would otherwise likely be blind in
5 years. As a consequence, of those currently at risk, only 27,000
would progress to legal blindness, and only 9,000 would become
blind today. In addition to reduced suffering and disability, the
economic savings from these treatments will reach as much as $1.6
billion per year.
As another example of payoff from recent NIH research, end-stage
renal disease (ESRD)-kidney failure requiring dialysis or transplantation,
a complication of diabetes and high blood pressure- results in
direct federal expenditures of approximately $20 billion per year.
Through the 1980s and 1990s, the incidence of ESRD nearly doubled
each decade, but in the last five years overall rates have stabilized—and
even declined in certain population groups. This improvement has
been driven by monitoring for proteins in urine to prevent kidney
disease or detect it in its early stages. Compared with earlier
projections, the savings in federal health care expenditures are
approximately $1 billion dollars per year.
Without the investment in medical research, people with diabetes
would be living shorter, less productive, and less hopeful lives.
Advances in Image-Guided Microsurgery
Increases in the NIH budget allowed new investments in the use
of imaging technologies like CAT scanning, MRI or ultrasonography
for the development of new microsurgical techniques. These minimally
invasive therapies are changing the fate of many patients, including
patients with Parkinson's disease, through deep brain stimulation.
These new techniques are also promising to revolutionize the treatment
of epilepsy, a disease that affects over 2.7 million Americans.
As we move forward with such research, we expect that surgery will
become less invasive, more precise and less dangerous, with far
less operative complications.
Advances in Health Information for Scientists and the Public
The National Library of Medicine of the NIH provides the American
public with high quality, reliable information. The NIH web sites
(www.nih.gov) are now recognized
by independent organizations as the most successful health related
web sites, with over 2 million queries per day. Millions of patients
and their families regularly consult NIH web sites for up to date
information in English and Spanish, a capability made entirely
possible by the doubling of the NIH budget. The web-based ClinicalTrials.gov
represents a landmark effort to provide information to patients
and physicians across the country on NIH-funded clinical trials.
NIH also leads the research field in developing information technology
for biomedical research. No biomedical scientist develops a project
without first consulting the suite of powerful informational research
tools available through the NIH National Library of Medicine's
PubMed, a growing digital archive of peer-reviewed research articles
and scientific databases.
New Research Tools
NIH researchers have pioneered powerful new research tools and
methods such as high throughput DNA sequencing, protein identification
with mass spectrometry, gene expression arrays, the determination
of thousands of new protein structures, and imaging technologies
which were simply unavailable before the doubling of the NIH budget.
A great illustration of the impact of these advances has been the
identification of the cause of the SARS virus in less than a month
and the current tracking of pandemic flu viruses. These tools have
greatly accelerated the research process itself, spurred progress
and spawned new discoveries in all areas of biomedical research.
Perhaps nowhere else have these technological advances in imaging
and genotyping elicited more excitement than in the field of mental
and behavioral health, elucidating genes linked to schizophrenia,
depression, bipolar disorder and anxiety. These discoveries are
allowing for the first time direct visualization of brain structure
and function to study the brain circuitry involved in thinking
and a range of behaviors.
New Diagnostic and Therapeutic Technologies
Some of NIH's successes can be measured in new medical technologies.
Advances in research are driving an increase in the number of technologies
being licensed to companies for commercialization. In FY 2004,
there were thousands of active licenses between federally funded
research institutions and companies worldwide. Out of these technologies,
several thousand companies are making many new products that have
an immeasurable impact on public health. Today, from NIH funded
research, more than 300 new drug products and vaccines targeting
more than 200 diseases—including various cancers, Alzheimer's
disease, heart disease, diabetes, multiple sclerosis, AIDS and
arthritis—are in clinical trials. These outcomes are accomplished
through the on-going network of successful collaborations with
our colleagues in private industry.
The Changing Landscape of Disease
Disease and injury are constant threats to humankind and are never
static. New diseases can emerge at any time, such as HIV/AIDS,
SARS, Pandemic Flu, obesity or many other conditions. Bioterrorism
did not figure significantly in the NIH agenda in 2001, but is
now a top priority of the agency. Twenty years ago the impact of
Alzheimer's disease was not fully appreciated, nor were its causes
known.
As the result of our success in preventing and treating acute
and short term conditions such as heart attacks, stroke, cancer
and many infectious diseases, we are living longer. Our increasingly
older population faces the new challenge of multiple chronic conditions
which now consume about 75 percent of healthcare expenditures.
This shifting burden of health care from acute to chronic diseases
is perhaps the greatest challenge we face.
Health care costs in the United States have risen to more than
$2 trillion. The amount spent on health care per person has doubled,
from $3,461 in 1993 to $7,110 today. The causes of health care
inflation are varied and complex, requiring different, nation-wide
solutions.
We are in a race against the overwhelming human and economic consequences
of disease. We can win this race, but only if we use research discoveries
to transform medicine as we know it. Thanks to recent research
advances, we can foresee a future of more effective medical treatment
that might be less expensive than current practices.
Strategic Vision for NIH: from Curative to Preemptive Care
We are in an era of great scientific opportunity. Advances in
our understanding of basic human biology allowed NIH to sequence
the human genome by 2003, two years ahead of schedule, and to complete
the Haplotype Map, showing the variation between individual humans,
in October 2005, also ahead of plans. One of the greatest scientific
achievements in history, the genome blueprint, along with work
in systems biology and proteomics, are driving a revolutionary
period in the life sciences. We are on the brink of transforming
medical treatment in the 21st Century. Our hope is to usher in
an era where medicine will be predictive, personalized and preemptive.
Toward this goal, NIH is strategically investing in research to
further our understanding of the fundamental causes of diseases
at their earliest molecular stages so that we can reliably predict
how and when a disease will develop and in whom. Because we now
know that individuals respond differently to environmental changes
according to their genetic endowment and their own behavioral responses,
we can envision the ability to precisely target treatment on a
personalized basis. Ultimately, this individualized approach, completely
different than how we treat patients today, will allow us to preempt
disease before it occurs.
Consider, for instance, how better predictive and personalized
treatments could improve the safety and effectiveness of drugs.
As we know, drugs do not fall into the "one size fits all" category.
The same drug can help one patient and harm another. Recent research
shows that we will be increasingly able to know which patients
will benefit from treatment and which patients might be harmed.
This field of study is known as pharmacogenetics. Using the latest
genomic data, enabled by the doubling of the NIH budget, the NIH
established a Pharmacogenetic Research Network which is studying
the interactions of drugs and molecules as well as the biological
processes that eliminate compounds from the body. In the first
five years of this program, the researchers in this network made
numerous discoveries.
For example, they learned that 10 percent of the North American
population exhibits a genetic variation that puts them at high
risk for life-threatening reactions to irinotecan, a cancer drug.
We now know that patients with this variation should be given lower
than prescribed doses of this successful drug, thus potentially
saving their lives.
NIH researchers also discovered variations in a gene involved
in the body's response to more than half of all medications.
Understanding these differences could explain critical individual
as well as racial and ethnic differences in drug responses. Other
genetic variations discovered by the NIH network will have an impact
on asthma treatment, the risk of sudden death from irregular heartbeats
and the proper use of blood thinning medications to avoid deadly
bleeding complications.
In another example of emerging personalized medicine, cancer researchers
have developed a test that helps determine the risk of recurrence
for women who were treated for early stage, estrogen-dependent
breast cancer. This information can help a woman and her doctor
decide whether she should receive chemotherapy in addition to standard
hormonal therapy. This test has the potential to change medical
practice by sparing tens of thousands of women each year the unnecessary
and harmful side effects associated with chemotherapy at large
potential cost savings.
Rapid Advances in the Genomic Era
Because of a hundred fold reduction in the cost of genomic technology,
we can now study, at affordable costs, the differences between
patients who have a disease and their normal counterparts. Recently,
this revolutionary approach led to the discovery of two previously
unsuspected factors that can identify who is at risk and how to
protect patients from age-related macular degeneration, an increasing
cause of blindness in our aging population, with over 7 million
Americans at risk. Last month, a key transcription factor that
may be responsible for a large percentage of cases of diabetes
was discovered.
These breakthroughs form the basis of our budget request for the
Genes and Environment Initiative, supported by Secretary of Health
and Human Services Michael Leavitt, because it will give us the
unprecedented ability to discover, over the next three years, the
potential causes of the 10 most common diseases afflicting the
U.S. population. With this funding, if approved, we will also launch
a technology development effort for enabling scientists to measure
many types of environmental exposures at the individual level.
Taken together, these efforts will lead to better understanding
of the environmental and genetic factors in the development of
many diseases.
Imagine a world where we will be able to tell each patient whether
they need to take action to preempt altogether the development
of costly and painful diseases. Imagine telling them that they
do not need to take expensive medications for life because they
are not at risk of disease. A more predictive, personalized and
preemptive form of medicine is no longer just a dream, but a vision
to strive for as rapidly as we can.
Management Innovations
NIH has an enormous and growing scope of mission. We conduct or
support research on over 6,600 diseases and conditions, from the
most common to the rarest. In 2005, more than 43,000 research grant
applications went through our rigorous two-tiered review process,
with about 22 percent of applications ultimately receiving funding.
More than 80 percent of the NIH budget supports extramural research
at 3,100 institutions around the world, employing about 200,000
scientists and other research personnel. Another 10 percent of
the budget goes into the NIH intramural program, consisting of
approximately 6,000 scientists, where work is focused on public
health priorities and cutting edge research. The hub of the intramural
program, the NIH Clinical Center on the Bethesda campus, is the
world's largest dedicated clinical research complex.
NIH is spending $95 per American this year on medical research,
and we need to make every dollar count. With the growth and increasing
complexity of the agency, NIH has aggressively moved to transform
its management strategies and decision-making processes. To streamline,
harmonize and better coordinate decisions that affect the entire
agency, in 2003, I established the NIH Steering Committee, composed
of nine Institute Directors who serve on a rotating basis. Six
working groups support the Steering Committee. This new governance
structure has enabled greater coordination and harmonization between
the 27 Institutes and Centers at NIH.
NIH has addressed the need for more robust means to oversee the
vast NIH research portfolio, and plan and launch trans-NIH initiatives.
While the NIH successfully developed important trans-NIH initiatives
such as the Roadmap for Medical Research, the Strategic Plan for
Obesity Research, and the Neuroscience Blueprint, the agency is
now implementing even more rigorous and transparent processes and
developing cutting-edge tools to analyze, assess and manage the
array of research it supports. This will provide better information
to support planning and priority-setting in areas of shared Institute
and Center interests. To reinforce these accomplishments, NIH is
establishing a new office within the Office of the Director—the Office
of Portfolio Analysis and Strategic Initiatives (OPASI).
Review of our programs by the Office of Management and Budget
under the congressionally mandated Government Performance and Results
Act (GPRA) provides evidence that our programs are effective. We
have been rated in the top 15 percent of federal organizations.
NIH's effective performance is reflected in recent scores as measured
by the OMB Program Assessment Rating Tool (PART). In the FY 2007
PART, the Buildings and Facilities Program and the Intramural Research
Program both received the highest possible rating of effective,
with scores of 96 percent and 90 percent, respectively. On the
FY 2006 PART, the NIH Extramural Research Program achieved a similarly
high 89 percent. These high scores demonstrate exemplary management
and substantial progress toward meeting NIH performance measures.
To date, approximately 90 percent of NIH's budget has been PARTed
and rated effective.
Translating Discoveries into Better Medical Treatment
Rapidly translating our discoveries from the bench to the bedside
is a top priority of the NIH. The opportunities have never been
greater to use modern research methodologies such as genomics,
proteomics, metabolomics, high sensitivity biochemical methods
and other novel strategies to bring new insights to the study of
human populations and more rapidly achieve the goal of making medicine
predictive, personalized and preemptive.
To accelerate progress, NIH recently introduced the institutional
Clinical and Translational Science Award (CTSA). The CTSA program
will stimulate institutions across the country in transforming
Clinical and Translational Science in the U.S.A. to
(1) captivate, advance, and nurture a cadre of well-trained multi-
and inter-disciplinary investigators and research teams; (2) create
an incubator for innovative research tools and information technologies;
(3) synergize multi- and inter-disciplinary clinical and translational
research; and (4) accelerate the application of new knowledge and
techniques to clinical practice at the front lines of patient care.
Training a New Generation of Scientists
New visions require new talent. In times of constrained budgets
the most important action NIH needs to take is to preserve the
ability of young scientists with fresh ideas to enter the competitive
world of NIH funding. To that effect, NIH has launched the new "Pathway
to Independence"
program which will support, for each of the next five years, 150
to 200 recently trained scientists conducting independent, innovative
research.
In Summary
Our Nation's investment in biomedical research has dramatically
improved health outcomes. The return on the investment of the American
people at NIH is nothing short of spectacular. Thanks to the support
of Congress, we are able, through our science, to respond in record
time to emerging threats such as SARS, Pandemic Flu and biodefense
needs. We have learned how to decrease the incidence of many diseases
and other disabilities for old and young Americans. The estimated
total cumulative investment at the NIH per American over the past
30 years including the doubling period is about $1,334 or about
$44 per American per year over the entire period. In return, Americans
have gained over six years of life expectancy and are aging healthier
than ever before.
The President and Congress have wisely invested in biomedical
research. We are acutely aware that NIH research is often the only
hope for millions of people afflicted by disease. In the battle
for health, NIH also believes that it needs to accelerate the pace
of progress, as it is only through a fundamental transformation
of medicine that solutions to the rising burden of healthcare will
be found.
I will be happy to answer any questions you may have.
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