108th Congress

Testimony Before the Subcommittee on Health Committee on Energy and Commerce

Elias A. Zerhouni, M.D.
Director
National Institutes of Health
U.S. Department of Health and Human Services
June 2, 2004

Mr. Chairman, Members of the Subcommittee, I am Dr. Elias Zerhouni, the Director of the National Institutes of Health. I am pleased to appear before you today to provide an overview of the NIH research portfolio and discuss priority setting.

NIH’s mission is to conduct research that will lead to better methods of diagnosing, treating, preventing, and curing disease. The research that we support has resulted in improvements in detecting disease, better therapies, and more effective vaccines.

As one example, our ability to fight infectious diseases is greater than ever before. Consider how research advances enabled the world to quickly identify and contain the SARS virus. We will produce treatments and vaccines for other diseases, such as West Nile Virus, in record time. We have also developed a single dose, fast-acting experimental vaccine to prevent one of the most feared viruses of all, Ebola. The vaccine has proven successful in animals and human trials are under way.

In the area of mental illness, NIH-supported researchers recently discovered genes associated with schizophrenia, a tragic illness that affects 1 percent of the adult population. This research, which brings us closer to better treatments for this disorder, was cited by Science Magazine as the number two scientific “breakthrough of the year” in 2003.

Much of our progress is attributable to basic research advances furthering the understanding of human biology. NIH and its collaborators have sequenced the human genome, one of the greatest scientific achievements in history. Now we are moving forward with research into molecules and proteins to gain knowledge leading to new therapies that will alter the way medicine is practiced and result in even greater improvements in public health. We are on the cusp of an era of medical practice that will identify and prevent diseases before the symptoms appear.

In short, we are living much longer and significantly better as a result of biomedical research. And while we have come very far, we have even farther to go. Despite our extraordinary successes, there is still a great deal we do not know about human biology. In areas where we have reduced death and suffering, we can do even more. Consider the case of cardiovascular disease. One of the greatest public health success stories of the last half-century is the dramatic reduction in mortality from stroke and heart disease. In the year 2000, the number of deaths from cardiovascular disease was nearly 40 percent less than we had projected in the same year. Research identifying risk factors and new therapeutic interventions to control the risks were largely responsible for this remarkable lifesaving achievement. Yet cardiovascular disease still accounts for about 38 percent of all deaths in the United States every year. If we sustain our research effort, imagine how many more lives we can save over the next 50 years.

The Nation remains committed to the support of biomedical research. Congress and the President appropriated a little more than $28 billion to NIH in FY 2004, and the President’s Budget Request for FY 2005 is $28.7 billion, a $729 million or 2.6 percent increase. Of the funds appropriated in FY 2004, an estimated $5.6 billion will be spent on cancer research and $4.9 billion on neurosciences research. We expect to spend $3.6 billion on research affecting women’s health and $3.2 billion on pediatric research. We plan to spend $1.6 billion on biodefense research, and $2.4 billion on cardiovascular research. Another major investment is aging research, which will receive $2.3 billion. Vaccine development research will total $1.4 billion.

There are several ways to view the NIH budget. The most relevant picture is the snapshot of the individual Institute and Center budgets because Congress appropriates funds on the basis of allocations to these 27 organizations. In FY 2004, their budgets range from $4.7 billion for the National Cancer Institute to $65 million for the Fogarty International Center.

Another common way to view NIH’s budget is by the funding ‘mechanisms’, such as grants, contracts, cooperative agreements, or in-house programs. About 80 percent of the NIH budget is awarded to extramural research institutions throughout the United States. The largest grant mechanism is our Research Project Grants, which comprise 54 percent of NIH’s budget in FY2004, or $15.1 billion. Another important funding mechanism, research centers, supports groups of investigators working on common disease or research areas. This mechanism accounts for 9 percent in FY 2004, or $2.6 billion. Another vital mechanism is our research training programs, which comprise 3 percent of our total budget, and will help ensure that we will have the skilled workforce needed in the future to continue making progress in research. Our intramural research program accounts for 10 percent of our total budget, or $2.7 billion.

Our budget can also be viewed by funding of specific diseases, several of which I have already mentioned. Research projects can often contribute to advances in multiple diseases; thus, our estimates of research expenditures by disease necessarily contain overlap and are not mutually exclusive.

The public can also view the NIH budget from the perspective of the success rate of grant applications. However, looking at the budget from this perspective results in many misleading conclusions. Success rate alone is not indicative of the number or size of grants being funded; the number or quality of grant applications received in a given year; and research mechanisms that NIH may be funding other than grants. In addition, our estimates of the projected number of applications submitted and the number actually awarded have undergone significant revisions from earlier predictions, creating some difficulties in making grant budget projections.

Over the decades, the allocation of NIH dollars has adapted to public health needs. In fact, much of our spending focus recognizes the shift in disease burdens that has taken place in recent decades. For example:

• NIH is increasingly targeting chronic diseases, which have overtaken acute conditions as the Nation’s leading health problem.
• We are responding to a new epidemic - obesity - which, if continued unabated, threatens to undermine our progress against disease in similar ways to tobacco use. Part of this response has been a proposed trans-NIH funding increase of 10 percent allocated for obesity research in the FY 2005 President’s Budget.
• We are quickly expanding our research efforts to protect the Nation against lethal bioterrorist acts by identifying the threats and developing vaccines, diagnostics, and therapeutics to address them.
• We are committed to NIH’s infectious disease research on problems such as AIDS, SARS, West Nile Virus, influenza, malaria and tuberculosis.
• We remain committed to research on other long-standing problems, such as the health disparities that exist among racial, ethnic, and disadvantaged populations.

As the most influential force in the U.S. biomedical research community, NIH exercises its leadership by continually surveying public health needs and the scientific landscape to identify new biomedical research areas that require attention. Simultaneously, we search for emerging scientific opportunities. To maintain the vibrancy of our scientific enterprise, NIH also actively supports strong basic and clinical research training programs. Our programs are unique in both igniting and complementing private sector research and development efforts.

NIH undertakes studies for which the risks are too high or the financial incentives too low to attract private investment. Tailoring therapies for the special needs of vulnerable populations and evaluating treatments for rare diseases are other NIH-led investigations where the intervention of a public agency is essential. With the massive responsibility of advancing knowledge across such a wide landscape, whenever possible NIH marshals efforts of academic institutions, industry, research organizations, disease foundations, and patient groups to maximize its efforts.

This focus on vulnerable populations and rare diseases is an essential part of NIH’s mission, and must be a component of priority setting. Instilled in all of us at NIH is the human dimension that drives us to help the helpless, whether their suffering is from a disease that affects millions or a disease that affects only a few. This is why I became a physician, and it is why I was eager to come to NIH.

To maintain a research portfolio that balances public health needs and scientific opportunities, NIH seeks input through multiple channels, including the Advisory Committee to the Director and the NIH Council of Public Representatives. NIH uses an unparalleled peer review system involving its Center for Scientific Review as well as separate vetting programs within each Institute and Center. These programs are part of a two-tiered system of advisory bodies and specialized review committees that guarantees funding of the best applications from among the nearly 50,000 research and training applications reviewed annually.

NIH’s priorities are driven, in part, by the ideas and opportunities presented to us through the grant applications we receive. By placing most of our resources in investigator-driven research, NIH ensures that federal dollars track the latest science. But allowing the scientific community to drive research is only one factor in how NIH sets priorities.

Determining research priorities is a complex, multifaceted process. One cannot easily quantify the various factors and questions that surround priority setting at NIH. Some of the variables in the determination of resource allocations include public health needs and the burden of disease, scientific opportunities, the quality of research proposals, the experience of applicants, and the ability to sustain research through adequate staffing and infrastructure. These factors are often lost in the public debate about NIH funding, in which the discussion is simplified by focusing attention on apparent funding inequities between the toll of certain diseases and the amount spent on research about those diseases.

Although burden of disease should not stand alone as a factor in setting priorities, there are indications that NIH funding generally tracks disease burden data. A study published in the New England Journal of Medicine five years ago concluded that there is a significant - although not absolute - correlation between the burden of disease and NIH funding. The genome project, development of instrumentation, training in clinical research, and new developments in basic science all have high values in the treatment of specific diseases, even though they lack a disease-specific orientation. Nonetheless, the study is evidence that NIH resources reflect the burden of disease in measurable terms.

Do these successes mean we are doing everything we can to ensure that the NIH research portfolio is balanced, is focused on the most urgent needs, and is based on irrefutable data? Let me answer that question with the following observation: Great organizations can maintain greatness only by continuous reassessment and adaptation.

I believe that we cannot be static. NIH must enhance the current process for determining priorities and allocating resources as part of a balanced research portfolio across the Agency and within each Institute and Center. The system of funding research by allocating resources directly to disease, organ, or special-population-based Institutes and Centers has served NIH and the public well. We plan to continue this approach to funding programs at the Institute and Center level. But science is changing, driven by new technologies and discoveries. Modern research is often best conducted by teams, which may include mathematicians, chemists, physicists, engineers, bioimagers, computer scientists, behavioral scientists, and physicians, and which may cut across the expertise of many different NIH Institutes and Centers. Several fertile areas of research - genomics, proteomics, molecular engineering - serve all fields of endeavor and cannot be pigeonholed according to specific diseases.

As the Institute of Medicine noted last year in its review of the structure of NIH, consideration should be given to refinements in the priority setting process and the management of our portfolio. There is a particular need for new and sustained approaches to evaluating NIH’s crosscutting science. While maintaining the support for existing Institute and Center research programs, I think we should consider ways of using resources that may not be controlled by a single Institute or Center, but by a priority-setting process with input from outside and inside NIH. I am encouraging each Institute and Center to evaluate their own priority setting and portfolio management processes and seek best practices or other methods of enhancing their systems. I have also asked the Institute and Center directors to strive to pool resources, as they have done in research areas such as obesity and neuroscience.

An expanded approach to priority setting would enable NIH to ensure balance in our research portfolio, identify appropriate cycles of change, maintain proper turnover rates for grants and provide much more accountability to Congress and the public. Under such processes, we would identify crosscutting research that requires common investments from the various NIH Institutes and Centers. This approach must include a regular horizon scan of all research so that we can have sufficient information to manage the NIH research portfolio.

Two years ago, soon after I arrived as the Director of NIH, we convened a summit of the Nation’s scientific experts to determine obstacles to the advancement of research and methods to overcome them that could not be addressed by any single Institute or Center. Teams comprising the NIH’s leadership, working with their counterparts in the extramural scientific community, discussed new ideas. From these deliberations, the NIH Roadmap emerged. The Roadmap is focused on three goals: Identifying new pathways of discovery; Building the research teams of the future; and Re-engineering the Clinical Research Enterprise. As a modest but significant step forward, the Roadmap is supported by voluntary funding from all of our Institutes and Centers, with the goal of supporting research that will benefit all NIH programs and research into multiple diseases.

As I said, the Roadmap is a modest attempt at progress. It has an initial investment of less than 1 percent of NIH’s total budget. The Roadmap is an example of a better-integrated mechanism for priority setting at NIH. My expectation is that we will build on the Roadmap, and it will serve as a model for future determinations of resource allocations.

In summary, I believe the confidence of the American people in NIH to lead biomedical research has been and will continue to be deserved. Our processes for identifying priorities and ensuring sound science have worked well. But reassessment and adaptation should occur and lead to a priority setting process that has greater public input, is more transparent, and lead to a research portfolio that will keep NIH at the leading edge of biomedical research.

I intend for the process to contain the following essential elements:

1. A transparent process characterized by a defined scope of review with broad input from the scientific community and the public.
2. A solid database of information, including uniform disease coding and accurate, current and comprehensive information on burden of disease.
3. An institutionalized process of regularly scheduled evaluations based on current best practices to be used by Institutes and Centers.
4. The ability to weigh scientific opportunity against public health urgency.
5. A method of assessing outcomes to enhance accountability.

Thank you for the opportunity to testify. I will be pleased to answer your questions.