Fall 2003 Meeting Minutes
October 20, 2003
NIH Participants:
- Elias A. Zerhouni, M.D., NIH Director
- Raynard S. Kington, M.D., Ph.D., NIH Deputy Director
- John Burklow, Associate Director for Communications, Director,Office of Communications and Public Liaison
- Ruth Kirschstein, M.D., Senior Advisor to the Director
- Jennifer Gorman Vetter, M.P.A., COPR Executive Secretary, NIH Public Liaison Officer, Office of Communications and Public Liaison
COPR Members Attending:
- James J. Armstrong
- Ted Mala, M.D., M.P.H.
- Ruth C. Browne, Sc.D., M.P.H.
- Rodrigo A. Muñoz, M.D.
- Nancye W. Buelow
- William D. Novelli
- Barbara D. Butler
- Lawrence B. Sadwin
- Frances J. Dunston, M.D., M.P.H.
- John Shlofrock
- Rafael Gonzalez-Amezcua, M.D.
- Ellen V. Sigal, Ph.D.
- Ellen E. Grant, Ph.D.
- Leonard J. Tamura, Ph.D.
- Debra S. Hall, Ph.D.
- Zelda Tetenbaum, M.Sc.
- Kimberley Hinton
- Dawna Torres Mughal, Ph.D., R.D., FADA
- Jim Jensen
- Donald E. Tykeson
NIH Director's Report
Elias A. Zerhouni, M.D.
Director, National Institutes of Health
Dr. Zerhouni welcomed attendees to the 10th meeting of the Director's Council of Public Representatives (COPR). He extended a special welcome to Dr. Thomas Ansfield, a member of the Advisory Committee to the Director (ACD), who serves as the ACD liaison to the COPR. Dr. Zerhouni added that Mr. Sadwin serves as the COPR liaison to the ACD and observed that Mr. Sadwin's and Dr. Ansfield's presence further a vital synergy between the Office of the Director (OD) and the COPR by promoting information sharing and a common purpose.
Update from the Director
Staff Changes
Dr. Zerhouni announced new directors who have taken their positions since the last COPR meeting. Story C. Landis, Ph.D., accepted an appointment as Director of the National Institute of Neurological Disorders and Stroke (NINDS). She previously served as Scientific Director of the NINDS intramural program.
Jeremy M. Berg, Ph.D., accepted an appointment as Director of the National Institute of General Medical Sciences (NIGMS). He was previously Director of the Institute for Basic Biomedical Science, and Director of the Department of Biophysics and Biophysical Chemistry at the Johns Hopkins University School of Medicine. Dr. Berg also directed the Markey Center for Micromolecular Structure and Function, and co-directed the Keck Center for the Rational Design of Biologically Active Molecules.
Dr. Zerhouni also announced resignations and retirements. Claude Lenfant, M.D., retired on Aug. 30 as Director of the National Heart, Lung, and Blood Institute (NHLBI). Dr. Zerhouni noted Dr. Lenfant's exceptional impact on the NHLBI during his long and distinguished tenure. A search team, co-chaired by Dr. Francis Collins, Director of the National Human Genome Research Institute, and Dr. T.K. Li, Director of the National Institute on Alcohol Abuse and Alcoholism, is soliciting recommendations for candidates. Dr. Zerhouni said COPR member Dr. Rafael Gonzalez-Amezcua agreed to serve on the search committee, and stressed the importance of COPR's presence in major searches. Dr. Barbara Alving, former NHLBI Deputy Director, is Acting Director.
Kenneth Olden, Ph.D., stepped down as Director of the National Institute of Environmental Health Sciences and the National Toxicology Program on July 29 but will serve until a replacement is found. Dr. Zerhouni commended Dr. Olden on a remarkable job and added that Dr. Olden will remain at NIH and continue his research.
Dr. Ellie Ehrenfeld resigned as Director of the Center for Scientific Review (CSR) in September. Dr. Zerhouni commended Dr. Ehrenfeld for an outstanding job in reshaping the NIH peer review system. Brent B. Stanfield, Ph.D., CSR Deputy Director, was named Acting Director effective Oct. 1.
Appointments of NIH Senior Staff
Richard Turman was appointed Associate Director for Budget after the retirement of Don Poppke. Mr. Turman was a longstanding leader at the Office of Management and Budget and has also worked in the private sector.
Dr. Norka Ruiz-Bravo replaces Dr. Wendy Baldwin as Deputy Director for Extramural Research. Dr. Ruiz-Bravo was chosen after an extensive review and search for an outstanding candidate who would understand the need for change in managing the grants portfolio and accelerate NIH's advance toward a more user-friendly way of applying for, reviewing, and awarding grants. She previously served as the NIGMS Deputy Director of Extramural Research.
Noteworthy Events
Dr. Zerhouni reported on the rollout of the NIH Roadmap for Medical Research at a press conference on September 30. More than 50 reporters attended, making it one of the largest NIH press conferences in recent years. A briefing of more than 150 public constituents preceded the press conference.
Developed with input from more than 300 nationally recognized leaders in academia, industry, government, and the public, the NIH Roadmap provides a framework of the priorities the NIH as a whole must address in order to optimize its entire research portfolio, and lays out a vision for a more efficient and productive system of medical research. In the post-genome era, the NIH must adapt its technologies to better address current research challenges and opportunities. The resulting 28 Roadmap initiatives are designed to speed the transfer of research discoveries from bench to bedside. They will also help make the nation's medical research capabilities more responsive to the changing scientific landscape and the demands of public accountability.
Dr. Zerhouni outlined three concepts in furthering Roadmap goals: (1) Effecting a fundamental change in the way research is conducted by promoting an interdisciplinary model, which involves importing and cross-stimulating many disciplines to solve complex problems; (2) Stimulating high-risk research by funding exceptional people with original ideas that are in the early stages of formulation; and (3) Re-engineering clinical research to create a better network of academic centers and community-based physicians to work jointly on clinical trials.
Dr. Zerhouni said the Roadmap has the support of the NIH Institute and Center (ICs) directors, who agreed to allocate IC funds to a common pool as part of NIH's new research direction; Roadmap initiatives are projected to account for 0.9% of the NIH budget over the next several years. The initiative recognizes that many missions are too complex to be accomplished by a single Institute, and that all ICs have a vested interest in combining resources.
Success in developing the Roadmap was due in large part to extraordinary efforts by NIH staff, whose work was complicated by a difficult budget year in 2003. He noted that COPR member Larry Sadwin participated in one of the Roadmap work group meetings and that this kind of participation from COPR members is a valuable part of COPR's work.
Dr. Zerhouni announced that, on Oct. 15, NIH received a Diversity Best Practices Leadership Award at the 2003 Diversity and Women Leadership Summit in recognition of NIH's efforts to ensure a diverse medical research workforce. NIH was the only Federal agency to receive this competitive award. Other recipients included companies such as the Boeing Company, Eli Lilly and Company, Lockheed Martin Corporation, and Booz Allen Hamilton Inc. The NIH is proud of this award and acknowledges the outstanding work of Larry Self, Director of the NIH Office of Equal Opportunity and Diversity Management (OEODM).
NIH grantees were among the winners of the 2003 Nobel Prizes for Chemistry and Medicine. The Nobel Prize for Physiology or Medicine was awarded to Drs. Paul C. Lauterbur and Peter Mansfield for discoveries involving magnetic resonance imaging. Dr. Lauterbur is a longtime NIH grantee; a majority of his funding comes from the National Center for Research Resources.
The Nobel Prize for Chemistry was awarded to NIH-funded basic scientists Drs. Peter Agre, a biologist/physician, and Roderick MacKinnon, also an M.D./Ph.D., for discoveries involving cell membrane channels.
Dr. Zerhouni said these awards illustrate the impressive advances that come from interdisciplinary research, adding that it is noteworthy that the winners in chemistry are not typical Ph.D. chemists. These awards validate NIH initiatives that seek to accelerate research advances by using interdisciplinary teams.
The Grand Challenges in Global Health initiative was recently announced, a $200 million research effort funded by the Gates Foundation in partnership with NIH and the NIH Foundation to accelerate research on grand challenges in global health. Dr. Zerhouni said this allows unappropriated funds to be used for exploring nontraditional NIH areas. Next steps include publication by the scientific board of specific scientific challenges, and issuance of a request for applications (RFA) for up to $20 million in grants.
Dr. Zerhouni reported forming a blue-ribbon panel in June to review the Intramural Clinical Program. Dr. Joel Goldstein and Dr. Ed Benz, President of the Dana Farber Cancer Institute, are conducting the review in conjunction with Dr. Michael Gottesman, Deputy Director for Intramural Research. Their recommendations will be forthcoming.
Legislative Update
Dr. Zerhouni testified on October 2 before a rare joint hearing of the Senate Health, Education, Labor, and Pensions Committee and the House Energy and Commerce Committee to present the NIH Roadmap for Medical Research. He identified several areas of congressional interest, including:
- Setting research priorities. How to set priorities in a complex institution to ensure that NIH invests in the right projects is an on-going challenge. An example of current congressional interest includes grants on sexuality research. Another area of interest was the on-going questions related to the NIH structure and organization (i.e., the issue of the proliferation of new institutes because there is a perceived need for research in a particular area or a powerful group lobbying for this specific issue). Discussion included whether this kind of proliferation was in the public interest? The public must be part of examining how NIH does business in this regard, and part of COPR's mission is to address this issue.
- Outsourcing initiative. A natural tension exists because it is not easy to outsource NIH's sophisticated services, and because NIH is responsible to taxpayers for ensuring competitive outsourcing. In a recent outsourcing competition NIH has shown the benefit from internally controlling grants management; competition is on-going in the area of facilities management.
- Changes to NIH. Congress will continue to be interested in the implementation of the Roadmap for Medical Research initiatives. Transparency is the best bulwark against inappropriate change, and COPR has a role in bringing transparency to the process.
Another topic raised was the issue of stem cell research. Dr. Zerhouni acknowledged the testimony at the hearing of the former Director of the NIH, Dr. Harold Varmus, who was extremely helpful in providing historical background about the NIH organization, and Dr. Harold Shapiro, who fairly represented the work of his committee in terms of the IoM recommendations.
Comments and Discussion
Ms. Tetenbaum asked about the process of changing NIH research grant funding from primarily single scientist grants to multidisciplinary grants. Dr. Zerhouni said the Roadmap initiatives call for interdisciplinary centers, which represents a change in the culture of institutions that receive grants.
Dr. Sigal asked about next steps after the IoM report. Dr. Zerhouni said impetus for change began before the IoM report, when NIH sought the help of several hundred scientific leaders in planning the Roadmap. There is a demonstrated need for transparency and for a disciplined agency-wide process to fund common areas, and the IC Directors have been supportive in this effort. Dr. Zerhouni said the lack of a specific process causes anxiety about the state of science and the progress that needs to be made; the improvement process must be on-going.
Dr. Mala requested elaboration on the outsourcing issue. Dr. Zerhouni agreed that the process is complicated. The intent of outsourcing is to assure taxpayers that NIH is using public funds efficiently. The initiative reviews NIH processes and puts them up for competition. If NIH is doing the best work, the processes are kept in-house. NIH is outsourcing such work as computer programming.
Mr. Tykeson suggested that the amount of money transferred to the Director's control from the ICs seems small in relation to the NIH budget and asked if it is sufficient. Dr. Zerhouni replied that the amount of money isn't as important as the fact that scientific disciplines have converged enough to focus on common areas. It is difficult in these tough economic times to ask, in midstream, to reapportion a significant portion of the budget. But Dr. Zerhouni said he is satisfied that the amount serves as seed funds. He noted that the molecular library initiative, which will cost $250–$300 million over 5 years, is something that no single institute could undertake. Dr. Zerhouni agreed that NIH must transition from where it is to where it wants to be, but the amount is not as important as the change in culture.
Dr. Zerhouni stressed the cooperation of the ICs on this issue. A steering committee of nine of 27 directors streamlined the decision process and resolved the issue of funding in two days. Thanks to the directors' impressive leadership, 27 institutes are now heading in the same direction.
Ms. Butler raised concerns about future funding of past grants, and constituency group fears that their diseases will be slighted because each institute must give money to the pool. Dr. Zerhouni emphasized that this money will serve the entire community through corporate decision making on initiatives such as the molecular library. Constituency groups must be educated to understand that this funding is synergistic with their interests and that it is for a minimum set of important NIH activities.
Dr. Zerhouni thanked the departing COPR members for their dedicated service—Drs. Grant, Muñoz, Tamura, and Bromet, and Ms. Tetenbaum—and presented certificates of appreciation.
COPR Work Group Co-chair Report and Discussion
Dr. Zerhouni and COPR Members
Ms. Hall, co-chair of the COPR Agenda Issues Working Group (AIWG), and Dr. Tamura, co-chair of the COPR Public Input and Participation Working Group (PIPWG), recapped work group activities over the past year and outlined plans for the following day.
Ms. Hall described the evolution of AIWG's thinking in preparing the meeting agenda, noting that the group refined its focus from concentrating on how research results translate to health benefits for a specific disease (metabolic syndrome), to conducting a panel discussion to examine how to enhance public trust in the clinical research enterprise, with an emphasis on understanding the relationship between health disparities, research practices, and building trust. There would also be a report from Dr. Sigal, chair of the IoM Response subgroup. Ms. Hall recapped lessons learned from the agenda-setting process: 1) it takes time, 2) flexibility must be built in, 3) communication via teleconference and e-mail is vital and ongoing, and 4) the job could not be accomplished without the interest of Drs. Zerhouni and Kington, the leadership of Dr. Tamura and Ms. Tetenbaum, and the efficient support of Ms. Jennifer Gorman Vetter and Ms. Shelly Pollard.
Dr. Zerhouni said he was impressed by the progress made in a year on the meeting agenda and commended the co-chairs.
Mr. Sadwin asked Dr. Zerhouni to use the program as a lens for suggesting appropriate next steps.
Dr. Tamura said that PIPWG saw itself as part of the process of engaging the public in the clinical research enterprise by referencing Dr. Zerhouni's three priorities—trust, bi-directional communication, and education - keeping in mind that even the perception of a lack of transparency can erode public trust. The work group worked with many Institute Officers of Public Liaison (OPLs) to find examples of how public input is obtained, and identified specific practices that effectuate public input in the research priority-setting process. The group interviewed OPL and IC staff and amassed information. Future efforts for the group include distilling this information to identify best practices that can be recommended for replication across the Institutes and Centers. The group will also examine how the research priority-setting process works in the context of facilitating public input into that process by identifying points of public access. The focus will be on transparency, i.e., asking, how clear is this process to the public.
Dr. Zerhouni asked if the group considered meeting with public members of the advisory councils. Dr. Tamura replied that the group is investigating such meetings.
Dr. Tamura reported that the communications subgroup had developed relationships with C-SPAN and the Discovery Channel, and that Mr. Burklow would present an update. Dr Tamura expressed appreciation to Mr. Burklow, Ms. Gorman Vetter, and Ms. Pollard for their efforts and support.
Dr. Zerhouni said that communication has improved but that an ongoing strategy was needed to let the public know about NIH activities.
Mr. Tykeson suggested that, although Dr. Zerhouni has a busy schedule, it would be beneficial if Dr. Zerhouni could participate in the communications effort because greater visibility by the Director would be a valuable catalyst.
Dr. Zerhouni said that it cannot be the Director alone in this effort—multiple strategies are necessary.
Dr. Grant told Dr. Zerhouni that COPR had received tremendous cooperation from the Officers of Public Liaison in many of the Institutes.
Dr. Zerhouni introduced Mr. Marc Smolonsky, the NIH legislative liaison with Congress.
Update from the Office of Legislative Policy and Analysis
Marc Smolonsky, Associate Director for Legislative Policy and Analysis, Office of the Director, NIH
Mr. Smolonsky gave a brief legislative history, tracing NIH roots from the establishment of the Marine Hospital Service in 1798 to the passage of the Public Health Service Act (PHSA) in 1944, which created the first National Institutes of Health. The NIH Revitalization Act was enacted in 1993. There have been no major omnibus reauthorizations since then, predominantly because NIH is driven by the appropriations process.
The NIH is not self-sustaining; congressional appropriators dominate the NIH legislative universe and set NIH's course. From 1998 to 2003, the NIH budget doubled to $27.2 billion. Pending legislation contemplates much more modest increases, a situation that is expected to continue into the future.
Mr. Smolonsky identified key appropriators in the Senate and House and stressed that in 2004 there will be a significant shift from appropriations to the authorization process.
The key NIH authorities (i.e., prioritizing research through the ICs, biomedical research, grant making, peer review, training, disseminating information, human subjects protections, and soliciting public advice) come through the PHSA.
Through the authorization process, Congress authorizes programs (usually in a 3-year cycle, but programs can continue without being reauthorized if funds are appropriated), may change existing programs, may add prohibitions to the PHSA, and can create institutes at NIH without going through the standard legislative process (e.g., debate).
In 1993, Congress sent a message through the NIH Revitalization Act that NIH did not focus enough on certain populations. The Act created the offices of Alternative Medicine, Women's Health, Minority Health, and Behavioral and Social Sciences Research. It also created the National Center for Human Genome Research and expanded the authority of the Office of AIDS Research. Among other provisions, Congress established the NIH Director's discretionary fund and the IDeA program.
Only 3 years later, Congress tried but failed to set new priorities for NIH in the 1996 reauthorization attempt. It is instructive, however, to look at the proposals. The legislation would have, among other things, created an Office of Rare Disease Research, elevated the Center for Genome Research to an institute, and established a Pain Research Consortium (which was later created administratively). Congress also proposed increased research funding for Parkinson's disease and increased support for diabetes and pediatric research. These were all enacted separately as amendments to the PHSA.
Mr. Smolonsky identified congressional committees involved in the process, key players in the Senate and the House, and congressional caucuses with interests in specific diseases or research. The congressional process, as it relates to NIH, is truly bipartisan.
The 108 th Congress has already held three authorization hearings for a reauthorization bill that will be considered in the spring. One of these was Dr. Zerhouni's appearance at a rare joint House/Senate hearing, which was a success for NIH.
Controversial issues before Congress are stem cell research, cloning, fetal tissue research, sexuality studies, and politics and science. Of concern is the Toomey Amendment, which was introduced on the House floor and focused on five sexuality study grants. It lost by two votes. This was important because Congress was considering defunding peer-reviewed research; i.e., grants that had already been awarded.
Potential issues that will be addressed in the reauthorization process are:
- DARPA-like authority. Should the NIH have Defense Department-like authority that allows it to cut through red tape in research and fund studies more directly and quickly?
- Bioterrorism. A big issue in the reauthorization process.
- Priority setting. Congress is constantly concerned with how NIH sets its priorities; for example, why the NIH funds one disease over another.
- Director's authority. Congress will examine whether it is sufficient to manage corporate NIH in a system where each center or institute receives its own appropriations.
- Peer review. Congress will ask whether the process is too cumbersome and prevents high-risk research.
- Structure. The focus will be on the number of institutes and centers.
- Small states. Many states do not receive funds; often they may not have research hospitals or university research centers that are successfully competing for NIH grants.
- Recruitment and training.
- Human subjects protection.
- Centers of Excellence. A common congressional solution to areas of research it considers underfunded.
Comments and Discussion
Dr. Sigal asked how Congress will set priorities and if there is anything COPR can do. Mr. Smolonsky said Congress is influenced by constituency groups, which should work together and think about how they interact with NIH. There is more risk than benefit to a reauthorization bill.
Dr. Mala asked for more information about the relationship between the IDeA program and small states. Mr. Smolonsky explained that the IDeA program is designed to help small states by providing seed money for developing institutional skills to help organizations in those states compete for NIH funding. Congress has been reluctant to force NIH into a geographic distribution of money, but legislators are frustrated by the lack of geographical distribution.
Mr. Sadwin said that all states benefit from good research. Mr. Smolonsky suggested that the Roadmap for Medical Research will help resolve geographical inequities.
Senator Jensen suggested that acceptance comes from not pushing too hard on controversial issues and letting them evolve. Mr. Smolonsky agreed that this is true in the ideal world but in Congress political evolution has reached a ceiling for certain areas of research; it is incumbent on NIH to be engaged to make things to happen.
Dr. Torres Mughal asked for advice on how to effectively reach and talk to legislators. Mr. Smolonsky replied that what would be more effective is to have biomedical advocacy and constituency groups collaborate and work together among one another.
Dr. Sigal suggested that there are overarching issues important to all advocacy groups. She said COPR is uniquely suited to encourage collaborative thinking. She added that NIH is where it is today because of constituency groups but, for some large reauthorization issues, group thinking is absent. Mr. Smolonsky agreed that constituency groups have played and will continue to play a vital role for NIH, but cooperative group thinking is necessary to be effective in the current political climate.
Ms. Hall raised the issue of the importance to the public and health care providers of the Roadmap as it relates to translating research into clinical practice.
Dr. Zerhouni said that when something new arises it is natural to try and fit it into existing frameworks. But NIH is not interested in responding with structures (e.g., ICs). The changing research paradigm is relationships and information transfer. Academic health centers were effective in dealing with acute health problems, but chronic health problems around the globe require a system of information transfer to share the benefits of research with physicians at each site. In this process, trust and best practices must be maintained and transferred easily across networks so participants can be recruited for research. This represents a philosophical change that will revolutionize the way research is done.
Dr. Muñoz raised the issue of the relationship among the research establishment, patients, and clinicians, suggesting that distance among these three groups must be decreased through collaboration.
Dr. Zerhouni replied that there are effective models, citing the Cystic Fibrosis Foundation, which works with 190 clinical sites in communities that connect to one data registry. This takes great cooperation from physicians. It is a best practice model that has led to increased life expectancy each year. NIH must create new research partnerships—a base of communities willing to partner with NIH and a base of credentialed specialized physicians who are qualified for such a partnership.
Dr. Zerhouni introduced Debra Lappin, an attorney and founding COPR member and a former member of the NIAMS Advisory Council. Calling her committed to the mission of NIH and an alumnus emeritus of COPR, Dr. Zerhouni said that Ms. Lappin, a member of the IoM Committee, would give an inside look at the IoM report and the issue of NIH organizational change.
Presentation on National Research Council/Institute of Medicine (IoM) of the National Academies Report
Debra Lappin, J.D., A COPR Alumni and IoM Committee Member
Ms. Lappin explained that Congress commissioned the NIH-funded study in response to rising concern about the NIH organizational structure. The Committee was composed of distinguished members with a wide breadth of expertise. Ms. Lappin noted that, despite the Committee's independent thinkers, there was a remarkable convergence with the NIH Roadmap for Medical Research.
The Committee's charge was to consider the following questions: How should NIH be organized to identify and respond with greater agility to emerging public need and scientific opportunity, is NIH equipped to be accountable to the pubic and Congress, and how can we ensure that public input informs strategic planning and research priority setting? The study was driven by three concerns: whether NIH had become too large and decentralized to respond to the needs of a rapidly changing science; that Congress felt the need to develop a new institute when there was a perceived research need; and that science was changing so dramatically that new infrastructures were required in which the multidisciplinary and multi-institutional concepts must be augmented with new interdisciplinary and inter-institutional concepts.
The Committee, recognizing that an organization can become entrenched in its own culture and lose the ability to adapt, confronted the major question: How should the NIH be optimally configured to meet 21st century scientific needs?
The Committee rejected the idea of a major consolidation of ICs, concerned that clustering the ICs would add a new layer of management. The Committee also found no ready set of natural dimensions for clustering, concluding that a loose federation of units with a high degree of autonomy and close ties to its constituency is NIH's bedrock.
The Committee explored new organizational strategies that included a trans-NIH capacity for response, a program to encourage high-risk/high-reward research, a major reorganization of clinical research activities, and strengthening of the Office of the Director. These were seen as ways to enhance NIH's ability to respond to science and new opportunities.
Ms. Lappin elaborated on each strategy:
- Trans-NIH capacity for planning and implementation to allow NIH-wide responses to compelling emerging areas. This entails regular NIH-wide planning and priority setting, and increased accountability of the Director to Congress. The Director must therefore have increased responsibility and authority, some of which will be accomplished by ongoing and stable funding from the ICs at an initial rate of 5%, increasing to perhaps 10% in 5 years. This funding, which does not take away from R01s but allows reprioritization to address public needs, will represent a new NIH acting as a whole. This new approach will demand that advocacy groups come forward as a new kind of NIH partner.
- High-risk/high-reward research that envisions creating a Director's Special Projects Program (DSPP) and urging the intramural research program (IRP) toward higher risk and innovation. The DSPP and IRP would complement the peer review system for the substantive body of NIH research.
- Clinical research. NIH is seen as the leader in balancing scientific opportunity against public need. NIH is the convener for orchestrating the removal of translational blocks. The NIH goal is to facilitate the passing on of information—to the next partner in clinical research—to ease the way for research teams to take a scientific concept through the clinical research passage to improve public health. Appointing a deputy director of clinical research is recommended to facilitate the process of coordinating intramural and extramural activities, and to build on and consolidate existing units and programs.
- Strengthen the OD. If the Director is to be held accountable, he must have the leverage to lead. This requires giving the Director an adequate budget to support the management role, operations budget, and the DSPP, and the budget authority to organize trans-NIH strategic planning.
Areas of accountability and administration include measuring COPR's impact and using data and information management systems to estimate spending priorities.
The advisory council system must be strengthened dramatically to gain consistency and independence. Members should be selected based on expertise, not a political agenda, that fairly represents the public; councils should include scientific members from organizations not funded by NIH institutes. There should be members from outside institutions who will push for new directions.
Centralization of administrative functions must proceed slowly and cautiously and not undermine the ability to administer, oversee, and conduct science. Among the administrative issues to be addressed is the tenure of IC and NIH Directors. There was support in the Committee for a 5-year, 360° review of directors in a new formalized way. The NIH Director should have a 6-year renewable term.
Structural changes (i.e., changing the number of institutes and centers) should revolve around a public process; there should be no sacred cows in terms of combining or changing the status of existing institutes. The Committee recommended a merger of the National Institute on Drug Abuse (NIDA) and National Institute on Alcohol Abuse and Alcoholism (NIAAA), and suggested combining the National Human Genome Research Institute and the NIGMS. Another suggestion involved examining the National Cancer Institute's special status.
The Committee concluded that what makes NIH great is its ability to attract outstanding leaders and scientists. Congress and the public anticipate a new level of accountability from NIH and the NIH must be ready to respond. Ms. Lappin suggested that COPR should be able to give Dr. Zerhouni important advice in light of this report.
Comments and Discussion
Dr. Browne asked what COPR's response should be in educating the public at this critical juncture for NIH. Ms. Lappin said this is up to COPR.
Ms. Tetenbaum asked if the Committee was on-going. Ms. Lappin said the Committee's work is complete.
Ms. Hall asked for more elaboration on removing translational blocks and leadership. Ms. Lappin said it is a tough question but trust in the enterprise means something is being done that makes a difference in people's lives. NIH is the obvious organization to orchestrate public trust in the clinical enterprise.
Mr. Tykeson, acknowledging the Committee's conclusion that it would be too expensive to change the structure of NIH , asked Ms. Lappin what the Committee would have suggested if it had the luxury of starting from scratch in organizing the NIH. Ms. Lappin said the structure most considered was clustering but that was finally rejected because it might lead to a subset of clusters that would demand even more money for a growing bureaucracy.
Mr. Armstrong asked Ms. Lappin to estimate how much weight the report will carry with Congress regarding high-risk research. Ms. Lappin said congressional scrutiny is driven by what serves the public need and that it remains to be seen whether this will stimulate Congress to allocate funds for trans-NIH planning.
Dr. Tamura asked how the Committee assessed the state of the system. Ms. Lappin referred him to the back of the report, which lists public testimony. Town meetings were also held.
Dr. Kington introduced the afternoon panel discussion, "Examining How to Enhance Public Trust in the Clinical Research Enterprise," with an emphasis on understanding the relationship among health disparities, research practices, and building trust.
Presentation on "Race, Trust, and Tuskegee: Professional Ethics, Broken Trust, and Health Disparities"
Matthew Wynia, M.D., M.P.H., Director, the Institute for Ethics, American Medical Association
Dr. Wynia recounted a story about the study of an AIDS vaccine in 5,009 people at high risk. Among vaccine recipients, 5.7% contracted the infection, in contrast to 5.8% of placebo recipients, so the vaccine did not work. But among African Americans the vaccine reduced infection rates from 8.1% to about 2%. The catch: only 314 African Americans enrolled in the study and only 13 became infected, so this may have been a statistical anomaly.
There are many reasons why minorities may be less likely to participate in research, and mistrust of the health care system and the research enterprise is one of them. Minorities more often mistrust the health system and this mistrust is grounded in realities that medical researchers need to acknowledge as valid. Mistrust has many negative effects throughout health care, including increased litigation, increased disenrollment, and possibly lower health status.
The integrity of the medical profession depends on trustworthiness. Relationships in health care and medical research are based on trust because of patients' vulnerability. We build trustworthiness in the hope that trust will follow.
There is a reciprocal relationship between interpersonal trust (between individual doctors and patients) and institutional trust (such as trust in the medical profession and medical research). Patients will not see a doctor if there is not some baseline institutional trust, and trust in institutions grows through positive interpersonal interactions. Building personal trust with the doctor validates initial institutional trust and builds on it.
Numerous studies have shown that African Americans commonly have a profound mistrust of medical research and a concern that they will be used as guinea pigs. This mistrust occurs in highly educated African American populations and reflects a collective memory of a history of unethical experimentation on African Americans such as the Tuskegee study of untreated syphilis. A significant minority of African Americans hold extreme levels of mistrust of the medical profession; believing, for example, that sickle cell screening is done to reduce the African American population.
The Tuskegee study (1932–1972) is an example of the failure of professional ethics in research on African Americans. Participants were lied to about the nature of the study. They were told they would receive free treatment for "bad blood," despite the fact that the objective of the study was to prevent subjects from receiving treatment and bring the men to necropsy (post-mortem) to evaluate the "effects of untreated syphilis on the negro male." The legacy of the Tuskegee study endures, researchers have concluded, in part because "the racism and disrespect for black lives that it entailed mirror black people's contemporary experiences with medicine." Today, 80% of minorities are more likely to believe that health professionals, whether they mean to or not, treat minority patients differently than they treat white patients.
In summary, Dr. Wynia suggested:
- Trustworthiness is central to professional integrity and trust confers health benefits.
- Many minorities mistrust medical research.
- There are historic and contemporary reasons for this mistrust that reflect profound failures of professional ethics.
- To improve outcomes, reduce health disparities, and build integrity, medical researchers must work to demonstrate their trustworthiness to minority populations.
Ways to demonstrate trustworthiness include:
- Recognize and focus on the issue.
- Develop standard, well-validated tools to assess trust in the research enterprise.
- Build on personal trust, which is still strong; people tend to trust their own doctors.
- Involve the community, such as through community-based participatory research (CBPR).
- Communicate the value of research within minority communities.
Finally, note that the Tuskegee Study could not enroll enough research subjects until advertisements said that "government doctors" were conducting the study. Never forget that trust in medicine, research and the government has been quite strong in the past, but it must be re-built.
Dr. Kington then introduced Vence Bonham, who recently joined the National Human Genome Research Institute from Michigan State University.
Presentation on Enhancing Trust in the Clinical Enterprise
Vence L. Bonham, J.D., Senior Advisor to the Director for Societal Implications of Genomics and Chief of Education and Community Involvement Branch, National Human Genome Research Institute, NIH
Mr. Bonham focused on research he was involved in at Michigan State University, representing about 250 individuals.
Mr. Bonham said the research, Communities of Color and Genetics Policy Projects, provides insights for enhancing trust in the clinical enterprise and biomedical research. The project aimed to engage African American and Latino communities of diverse socioeconomic levels in dialogs relating to genome research and its resulting technology, and to develop recommendations for laws, professional standards, and policies on the use and application of genome research and technology.
Dialog groups were identified and engaged in public discourse over 5 weeks. Their recommendations were outlined and the groups met again to review the dialog report. All 15 individual dialog reports were distilled into a summary report that was presented in meetings with policy groups (such as legislators) around the country.
Areas of concern identified by the dialog groups included education and trust.
Education:
- Must be in a culturally relevant language and form.
Trust:
The following recommendations came from the study:
Education:
- It is the responsibility of the Federal government to fund broad public education efforts regarding genetic technologies, research, and current policy issues.
Trust:
- There is a need for more racial and ethnic diversity in advisory committees for lay and scientific members.
- There is a need for networking between advisory groups and advocacy organizations to share findings and recommendations.
- Federal funding must be maintained at a high enough level related to private funding to minimize the profit motive's impact.
- Institutional Review Boards (IRBs) must address the protection of groups (i.e., communities and populations) and individuals, with an emphasis on minorities. This raises a question about how these different groups should be identified.
Lessons learned about enhancing individual participation and building trust in the research enterprise:
- It is important to reach out directly and work with targeted communities. Researchers must engage the community to understand how to work in that community.
- Long-term relationships must be developed with organizations and community leaders; parachuting in does not work.
- It is necessary to go beyond obvious community leaders to include individuals who represent important grassroots coalitions.
- Issues of trust as part of the research structure must be continually examined.
Presentation on Building Trust: Engendering Research Communities
Sharon B. Wyatt, R.N., C.S., Ph.D., Co-Principal Investigator, Jackson Heart Study Examination Center, Professor of Nursing, University of Mississippi
Dr. Wyatt cited former Surgeon General Dr. David Satcher as saying that none of us can argue with the national fact that we must do better to reach those with disparities in health: the uninsured, the uninformed, the uninspired, and the untrusting. How we do that is the issue at hand.
Noting that only in the last decade has the research enterprise made substantive and serious efforts to assure that the traditionally disenfranchised are equitably included in research to overcome health disparities, Dr. Wyatt suggested that research has become increasingly difficult to conduct and, as the NIH Roadmap has identified, requires new approaches and partnerships.
Dr. Wyatt described the Jackson Heart Study (JHS), the largest epidemiological cohort study of heart disease risk factors and causes in African Americans, as an exemplar of current approaches in building trust and overcoming disparities in health through use of a community driven model (CDM):
- The study extended the reach of researchers beyond the building to form partnerships among minorities and majority institutions in the community. The challenge was the time required to forge this partnership. Research outcome is driven by data; community outcome is driven by relationships.
- The CDM is generalizable to other sites. The CDM engages the community and researchers/practitioners as co-investigators; i.e., partners in planning, not ancillary reactors to preconceived plans.
- The CDM involves three overarching themes illustrated in the JHS study:
- Interpreting the concerns of the JHS family, particularly the guardedness that is a reflection of the way this population lives. Appreciating these concerns affects issues of consenting, the amount of study information that should be provided, and the community's need to have (and trust) guarantees about the researchers' intentions.
- Gathering the community as co-investigators. Researchers need to collaborate with their community co-investigators to identify and develop protocol elements. In the JHS, the consent brochure is extensive and there is a video and a signed commitment by the investigator that the data will only be used as listed in the consent form. Researchers must be aware that family is special in the African American community and an emphasis is needed on building a family-like relationship between the community and the researchers and institution. To this end, a Council of Elders was formed to provide a supportive presence for the community and the researchers.
- Community outreach. Taking newly gained knowledge back to the community redefines the traditional researcher/subject relationship and forges another level of partnership; a partnership for community health education and awareness.
Throughout the presentation, Dr. Wyatt shared the thoughts of participants, using their own words in the vernacular.
Despite this effort, there has been a mixed level of success:
- On the positive side, gaining knowledge for themselves and their families gives community members an incentive to participate and creates a sharing atmosphere that overcomes differences. The annual follow-up response is greater than 95%.
- On the negative side, recruitment is always a problem.
- Obstacles to building trust remain. There is a built-in inequitable power distribution that makes shared control in a CDM difficult. The nature of the scientific enterprise, which is data driven, often gives participants the sense that "all you want is our data." In addition, the difficulty of changing researchers' methods of inquiry, timetables, and resource allocation can preclude investment in community building. Finally, although building trust is on-going, trust remains fragile. Once established, trust can never be taken for granted and requires constant vigilance. Conflicts over meaning, language, values and beliefs, assumptions, and promises are inevitable, necessitating the on-going joint development of operational norms.
Community-driven possibilities for engendering trusting research communities:
- Develop models for community-driven research and multi-method evaluations.
- Fund studies that attend to how researchers build their research communities.
- Provide training for investigators and NIH staffand reviewers to facilitate broad-based, multiethnic studies.
Next, Dr. Kington introduced Myrl Weinberg, President of the National Health Council.
Presentation on Introducing Research into Communities
Myrl Weinberg, C.A.E., President, National Health Council
Ms. Weinberg described the National Health Council (NHC) as a nonprofit umbrella organization of 110 health-related organizations representing 100 million people. The NHC also numbers among its members pharmaceutical biotech companies, the AARP, and Hospice.
Rather than repeat ideas advanced by previous speakers, Ms. Weinberg highlighted major points:
Literacy and trust:
- Trust and trustworthiness are closely tied to communication. Health literacy issues (people cannot read, or they use words differently in their cultures or in rural areas) can account for recruiting difficulty because people do not know about the studies.
- The NHC conducts market research and asked people how they viewed genetic research to find out which words are effective. The research found that people did not have the language to process such technical concepts.
Building trust in the community:
- It takes a serious time commitment to develop the respect of people and their families.
- Identify community leaders, the most valuable community resource.
- Get involved in the community. Going beyond medical research to general issues that are important to the community is the basis of building trust.
- Create new roles. Identify and train people who can serve as trusted intermediaries between researchers and the community, such as social workers. This can be more effective than a legalistic consent document; it will improve the researchers' knowledge of the patients' perspective and what is important to them.
- Involve the community from the beginning. Every IRB should include people from the community. They are among the most valuable resources and they should be involved in the initial stages of research in their communities.
Research our own research:
- Find good models so we know what activities work when we recruit, and try to engage the community in research. An organization that does this is the Association for the Accreditation of Human Research Protection Programs (AAHRPP), which works to identify how communities and potential research participants are involved, how are they kept safe, and how their information is kept confidential.
Dr. Zerhouni thanked the speakers and asked them to define trust. The speakers gave the following definitions:
- Dr. Wynia: Trust is an expectation of future cooperation. Domains of trust include knowing that you are dealing with someone who is competent, will ensure confidentiality, encourages shared decision-making, and makes full disclosure. But this does not help us measure trust; it would be valuable to develop mechanisms for measuring trust. As new ways are developed to conduct community-based research, it would be helpful to be able to see if they affect trust in a measurable way.
- Dr. Wyatt: Trust is the expectation of being safe as predicated on past experience.
- Mr. Bonham: Trust is the faith, the belief, and the knowledge that you will not be harmed.
- Ms. Weinberg: Trust is feeling safe because of knowing that the doctor/researcher "has my best interests at heart."
Dr. Zerhouni, quoting a Japanese philosopher, added that trust is the ability to predict someone's behavior. It is a short word encompassing a complex reality.
Panel Discussion Question and Answer
Dr. Sigal recounted recent difficulty recruiting an African American who had been in a clinical trial to be an advisor to another clinical trial. It was difficult to find such a person and medical directors told her that it was exceptionally difficult to recruit African Americans for clinical trials. Dr. Sigal found that there was a collective memory about Tuskegee, and a potential recruit told Dr. Sigal that her entire family asked her not to volunteer.
Dr. Kington said the reported racial distrust has not led to a consistent failure to enroll. Statistics have shown that there is not a big difference in response rates by race; so there is a disconnection between reports of distrust and the lack of a consistent failure to enroll in studies.
Dr. Grant agreed that there are positive enrollment experiences, citing the Boston Black Women's Study. She said HHS has its own faith-based initiative and some lessons learned were that barber shops and beauty salons are valuable sites for educating the community. After holding a meeting that no one attended, it was clear that a first step is going into the community and engaging involvement. Dr. Grant said she liked the Council of Elders idea but suggested that participants' words should not have been left in the vernacular. Dr. Wyatt said the participants requested this, saying, "Don't whitewash our dialect."
Mr. Bonham gave an example of the African American community's involvement in genetics research, citing the NHGRI prostate cancer family project, which was done with African Americans.
Dr. Zerhouni recognized Dr. Stephen Katz, Director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and recalled that Dr. Katz had suggested that there is no such thing as one community of African Americans, but that many factors are involved from community to community.
Dr. Wyatt agreed, having seen this in the Jackson Heart Study, which involves three diverse counties.
Dr. Katz added that a critical element in building community infrastructure is building trust, a process that involves an on-going series of interactions. The success of the Roadmap for Medical Research section on clinical re-engineering depends on building trust in communities, something that will take time and commitment. NIH is concerned with public trust at all levels and with all communities.
Ms. Butler asked about work being done in the area of researcher bias. Dr. Wynia said there is much interest in improving physician communication, and last year physician accreditation required cross-cultural communication skills for new physicians. Dr. Wynia sees this as the least threatening way to approach this sensitive issue. Stereotyping can occur because physicians are rushed and this can appear to be racism. Physicians shut down if this is called racism but will talk about cross-cultural communication skills.
Ms. Weinberg suggested that research institutions develop systematic, organized ways of interacting with communities that have measurable objectives. Institutions must cull these successful strategies and replicate them.
Ms. Hall noted that this morning the group spoke of human subjects research and this afternoon it is speaking of people. She added that notions of mistrust can cut across a wide group of people of all ethnicities. She described a need to educate community researchers and called for the community to be a partner in research.
Dr. Mala acknowledged that "every researcher must pay for the sins of his or her forebears," and emphasized the problem he has seen in the Native American community of researchers who come into the community, do the research, and are never heard from again. He suggested that mistrust will be overcome when research results are brought back home.
Dr. Dunston said that, to promote public trust in clinical trials, researchers must demonstrate competency in dealing with participants and engendering trust. Should this be a core competency related to funding grant proposals?
Dr. Gonzalez-Amezcua said socioeconomic considerations often drive involvement in research. Such disparity should not exist, and this topic should be addressed.
Dr. Torres Mughal asked whether there is information on trust in minority groups.
Dr Wynia said there was some, but there is more information about trust in terms of how individuals view personal physicians. Among Latinos, for example, there appears to be a high level of trust in doctors and too much trust in Latino physicians. All minorities have a lower level of trust in the health care system. They tend to trust their doctors, but not researchers. This is why the distinction between trust and trustworthiness is so important; the research enterprise must be trustworthy.
Mr. Bonham said there is limited data on trust and research.
Dr. Muñoz said he had anecdotal evidence that the Latino population in his area evidences mistrust of clinical research.
Ms. Hinton expressed appreciation to the speakers for their presentations and said her own experience has been in HHS service organizations with AIDS. She felt the key is to respect the person sitting in front of you; this is not so much a trained competence but an innate respect. She added that, despite her own education and understanding of the research process, she sees Johns Hopkins trying to recruit and something inside tells her that she should not volunteer because, "What if?" As an African American woman, she added, when she visits the doctor the first thing that builds trust is respect. If professionals don't listen because she does not have an M.D. after her name, it does not work.
Dr. Dunston noted that the group had been discussing trust at a relational level (person to person) and wanted to know about the broader sense of public trust—confidence from the public in the NIH as an institution.
Ms. Weinberg suggested that they are intertwined. Building trust from the ground up at the relational level leads to trust in institutions.
Dr. Mala described his visit to Dr. Katz's inner-city clinic. The clinic was established because patients weren't coming to NIH, so the researchers took the clinic to the community. Dr. Mala reported being impressed with the staff and the positive attitude about this effort to bring NIH to the community.
Dr. Katz thanked Dr. Mala and said that it was Dr. Peter Lipsky of the Intramural Research Program who realized that NIAMS is an institute that deals with women and minority health, but sensed that this minority population was not reflected in patients being seen in the Clinical Center. He decided to go out into the community. He met with Hispanic and African American community leaders, including representatives from Howard University, to explain that NIH wanted its intramural program to be accessible to the community. This was a good practice and could be broadened to other initiatives.
Dr. Zerhouni said that the dimension of public trust is greater than the minority context. He said there are delays and recruiting problems across the board and charged COPR with taking the larger view and looking at broad generic problems that stifle the discovery pipeline.
Ms. Tetenbaum noted that patient accrual is not the only problem.
Dr. Zerhouni agreed, saying patient accrual was an example. He added that NIH must do the experiment because that is how science advances.
Dr. Wynia suggested that education is weakly related to trust because sometimes an educated person is less trusting. He mentioned the difficulty of framing a research program for grant funding for CBPR when you are not sure of the questions because you have not had the money to go out into the community. He felt there was no obvious way to do CBPR with traditional funding procedures.
Ms. Hall returned to the question of consent forms, saying that researchers need to be educated about what language works in the community.
Ms. Butler asked about the feasibility of approaching nonprofits and teaching them how to explain research projects.
Ms. Weinberg considered this an excellent idea because nonprofits are trusted community sources, but suggested it is limited because what works and what doesn't have not been identified. She wished that plain language could be used for guidelines like HIPAA (The Department of Health and Human Services (HHS) issued the Standards for Privacy of Individually Identifiable Health Information (the Privacy Rule) under the Health Insurance Portability and Accountability Act of 1996 (HIPAA) to provide the first comprehensive Federal protection for the privacy of personal health information.). She gave an example of the Alpha-1 Registry. Individuals with alpha-1 can register confidentially, and volunteers from patient groups contact these individuals and explain available research. Then it is up to the individuals to contact the researcher if they want to participate. The Registry successfully recruits participants because it is a trusted intermediary.
Dr. Katz mentioned two initiatives germane to the discussion:
- Harmonizing and simplifying research regulatory language to do away with 15-page consent forms. Dr. Zerhouni is using the power of his office in this effort, and the community must get involved.
- Support for the idea that volunteer organizations are important partners in this effort. This does not require money, it requires time and commitment.
Wrap-up and Next Steps
Dr. Zerhouni said it is clear that the evolving core issue of trust must be addressed and asked for next steps.
Dr. Sigal suggested that outreach efforts by the institutes be integrated in this effort.
Dr. Gonzalez-Amezcua suggested that more education is needed for the public to realize NIH's impact on their lives. Trust will increase as the public learns more about NIH.
Dr. Zerhouni thanked all participants.
Ms. Gorman Vetter asked COPR members to put the following tentative dates on their calendars for upcoming meetings:
2004
- April 29–30, with the reminder that a new member orientation must be scheduled on Wednesday, April 28
- October 25–26
2005
- April 21–22 or 28–29
- October 17–18 or 24–25
Ms. Gorman Vetter will check the Director's schedule and get back to all members.
Dr. Mala commended the Anchorage Native News to members and said that it contained a picture of COPR. Copies were available.
Dr. Zerhouni called the meeting one of the best and well focused that he had attended. He said COPR has an important role to play, that much rests in its hands, and that he appreciates the time that members give NIH.
The meeting adjourned at 4:28 p.m.
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