Thursday, April 6, 2006
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 following are samples of the many advances driven by the investment in NIH.
Advances in Cardiovascular Disease and Stroke
Advances in Cancer
Advances in HIV/AIDS
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
Development of Biodefense Research
Advances in Diabetes and Related Illnesses
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
Advances in Health Information for Scientists and the Public
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
New Diagnostic and Therapeutic Technologies
The Changing Landscape of Disease
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
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
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.
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
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
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.
Elias A. Zerhouni, M.D.
NIH Director, Elias A. Zerhouni, M.D., leads the nation’s medical research agency and oversees the NIH’s 27 Institutes and Centers with more than 17,000 employees and a fiscal year 2004 budget of over $28 billion. The NIH investigates the causes, treatments, and preventive strategies for both common and rare diseases helping to lead the way toward important medical discoveries that improve people's health and save lives. More than 80% of the NIH’s funding is awarded through almost 50,000 competitive grants to more than 212,000 researchers at over 3,000 universities, medical schools, and other research institutions in every state and around the world. About 10% of the NIH’s budget supports projects conducted by nearly 6,000 scientists in its own laboratories, most of which are on the NIH campus in Bethesda, MD.
Dr. Zerhouni, a well-respected leader in the field of radiology and medicine, has spent his career providing clinical, scientific, and administrative leadership. President George W. Bush nominated him to serve as the 15th Director of the National Institutes of Health, and he began in May 2002. Since then, Dr. Zerhouni oversaw the completion of the doubling of the NIH budget; initiated a strategic vision for the agency called the NIH Roadmap for Medical Research; established an NIH-wide research initiative to address the obesity; accelerated efforts in health disparities research; championed public access to NIH-funded research results; supported the Neuroscience Blueprint; streamlined NIH’s executive decision-making process; and named nine new Institute and Center Directors, and filled many other senior-level positions.
Prior to joining the NIH, Dr. Zerhouni served as executive vice-dean of Johns Hopkins University School of Medicine, chair of the Russell H. Morgan department of radiology and radiological science, and Martin Donner professor of radiology, and professor of biomedical engineering. Before that, he was vice dean for research at Johns Hopkins. He became a member of the National Academy of Sciences Institute of Medicine in 2000. Dr. Zerhouni has won several awards for his research including a Gold Medal from the American Roentgen Ray Society for CT research and two Paul Lauterbur Awards for MRI research. His research in imaging led to advances in Computerized Axial Tomography (CAT scanning) and Magnetic Resonance Imaging (MRI) that resulted in 157 peer reviewed publications and 8 patents.
Last Revised: May 11, 2006