The United States is preeminent in medical research. Since World War II this country has fashioned a medical research system—with government, academia, and industry at its core—that is a source of great national pride and the envy of the world. The federal government is the single largest sponsor of this research, committing more than $16 billion of public funds in the current fiscal year (1998). Of this total, the vast majority—$13.6 billion—is appropriated to the National Institutes of Health (NIH). Given the size of this public investment and the likelihood that it will be increased significantly in the immediate future and given the mission of NIH—"to uncover new knowledge that will lead to better health for everyone"—it should come as no surprise that there is intense interest in how NIH sets its priorities, that is, how it allocates its sizable budget. How could it be otherwise? Every one of us wants to live a long and healthy life. Every sick person—woman, man, or child—wants researchers to find new ways to make him or her well or to improve the quality of life for those who are disabled, regardless of whether the ailment is common or rare, acute or chronic, life-threatening or self-limiting.

We must acknowledge that setting priorities at NIH is an awesome task. Not only must the leadership of NIH answer to the executive branch and to the U.S. Congress, it must work with all of its constituencies—scientists, health care providers, patients, voluntary health groups and patient advocates, and industry executives—before making its fateful decisions. The quality and quantity of excellent science that it has supported, the widespread respect for it in and out of government, and its favored position in the annual congressional appropriations process signify that, over time, NIH must be doing many things right. Yet, we must also acknowledge that the recent request from Congress that the Institute of Medicine conduct an independent assessment of research priority setting at NIH and that the evaluation be completed within 6 months signifies, just as clearly, that there is at least a perception that some things are not right. It is apparent that some segments of the public, in general, and its representatives in Congress, in particular, are dissatisfied enough to ask for ways to improve the current process.

This committee was charged with examining four issues related to setting priorities at NIH: allocation criteria, the decision-making process, mechanisms for public input, and the impact of congressional directives. Despite the nearly impossible constraints imposed by the study's time line, we took this broad charge seriously. To grasp the approach that NIH currently uses, we heard in person from the director of NIH, most of the institute directors, and many of the directors of offices housed within the director's office. To understand the tensions surrounding public input, we held a full-day public meeting at which we received verbal and written testimony from patients, advocacy and interest groups, foundations, and professional societies. To understand Congress's rationale for requesting the study, we interviewed legislative aides from key offices. To expand our collective knowledge base, we reviewed many current and past publications relevant to our charge.

As we listened and deliberated, several things became clear to me. First, the country has extraordinarily high expectations of NIH. To some, NIH has become a virtual surrogate for the U.S. Department of Health and Human Services, being encouraged to expand its purview well beyond research. We heard from no one who wants to dismantle NIH; we heard from many who wanted their "cause" to be embraced by NIH; we heard from all that NIH must excel at everything it does because what it does is so important to the hopes and aspirations of people everywhere.

Second, there is a sense that NIH has evolved mechanisms for judging scientific opportunity and merit that surpass its capabilities for assessing and being influenced by public health needs. Estimating research spending by disease and developing metrics for spending according to disease burden (e.g., incidence, mortality, disability, and cost) must be done more systematically and more thoroughly than they are currently done because not doing so leads some to conclude, incorrectly I believe, that NIH cares more about curiosity than cure, more about fundamental science than clinical application.

Third, there is frustration on the part of some groups about not being listened to and heard by NIH. We heard repeatedly that some institutes, and particularly the Office of the Director, lack mechanisms for orderly, regular public input and outreach. As the authority of the director over priority setting has increased, the demand to influence that office has become louder.

Fourth, there is a lack of understanding about how NIH priority setting "works." NIH has not crafted simple communications that make its priority-setting processes as transparent as possible to its many publics. NIH has not developed sufficient communications tools to satisfy people that allocation decisions are made on the basis of equity and justice, as well as scientific opportunity.

Each of these observations has been converted to recommendations that reflect the committee's consensus. Our recommendations address each of the committee's charges, but there is a single theme that runs through them. It is that NIH must revamp its approach to public input and outreach—at every level—without delay. This will strengthen the priority-setting process in many ways. It will underscore that openness is as important to the process as such other valued qualities as expertise, innovation, and objectivity. It will provide NIH leaders more ways to demonstrate that they share the public's view that NIH exists to improve health through research. It will enhance the public's understanding of the complexities of decision making at NIH. Finally, it will give Congress additional confidence that it can delegate priority setting to NIH leadership knowing that a broader range of views will be sought and welcomed before decisions are made.

I would like to thank the many people who have made it possible for this report to be completed on schedule: first and foremost, the members of the committee who met and overcame the many challenges of our difficult task with a commendable blend of experience, energy, collegiality, and wisdom; second, the staff of the Institute of Medicine, without whom we would have foundered; third, the leadership of NIH, who educated us about this agency; and fourth, and perhaps most important, the public, who reminded us of the purpose of NIH and of the democratic ideals that must permeate effective stewardship of a federal agency.

Leon E. Rosenberg, M.D. Chair