Director's Council of Public Representatives

Fall 2002 Meeting Minutes

October 22, 2002

NIH Participants:

• Elias A. Zerhouni, M.D., NIH Director
• Ruth Kirschstein, M.D., NIH Deputy Director
• Edward O'Neil, Ph.D., COPR Facilitator
• Jennifer Gorman Vetter, M.P.A., COPR Executive Secretary, NIH Public Liaison Officer, Office of Communications and Public Liaison
• John Burklow, Acting Associate Director for Communications, NIH, Acting Director, Office of Communications and Public Liaison

NIH Attendees:

• Institute and Center Officers of Public Liaison (OPLs)

COPR Members Attending:

• Evelyn Bromet, Ph.D.
• Nancye Buelow
• Luz Claudio, Ph.D.
• Ellen Grant Bishop, Ph.D.
• Debra S. Hall, Ph.D.
• Kimberley Hinton
• Vicki Kalabokes
• Barbara Lackritz
• Debra Lappin, J.D.
• Ted Mala, M.D.
• Isaac Montoya, Ph.D.
• Rod Muñoz, M.D.
• Rosemary Quigley, J.D., M.P.H.
• Bob Roehr
• Lawrence Sadwin
• Len Tamura, Ph.D.
• Zelda Tetenbaum
• Don Tykeson
• Doug Yee

NIH Director's Report: NIH Update

Elias A. Zerhouni, M.D.
Director, National Institutes of Health

Dr. Zerhouni welcomed everyone to the Council of Public Representatives (COPR) meeting, noting the impressive diversity of those assembled. He cited the importance of collecting the public's perspective on recent work at the National Institutes of Health (NIH) and on the implications of recent advances in biomedical research.

Dr. Zerhouni said the presence of the Council's eight founding members increased the significance of this meeting, since they will be rotating off the Council next year. He introduced officials from the NIH, Office of the Director: Mr. John Burklow, Acting Associate Director for Communications, and Mrs. Jennifer Gorman Vetter, COPR Executive Secretary and NIH Public Liaison Officer, both from the Office of Communications and Public Liaison.

Dr. Zerhouni reported on his first five months as the NIH Director. He said, at the outset, it was obvious that the learning curve would be steep and rapid due to the current phase of remarkable growth. The President and Congress have questioned the NIH's use of resources, its plans for using the resources provided by the doubling of the NIH budget, and the NIH strategic plan. Dr. Zerhouni has met with Members of the NIH Advisory Council to the Director (ACD), as well as the Scientific Directors of all the Institutes and Centers (ICs). He has made himself available to a broad range of constituencies. He has testified twice before Congress. On one occasion, he testified in response to inquiries from Sen. Arlen Specter, (R-PA), about the availability of stem cell lines for researchers. Soon after becoming Director, Dr. Zerhouni created a task force on stem cell research. The task force has taken a proactive role examining issues such as training requirements.

Dr. Zerhouni said that his top priority as the NIH Director was "to attract researchers to do the work" and fill positions in the NIH leadership. He gave a progress report on his goal. He cited the appointment of new directors to the National Institute of Mental Health (Thomas R. Insel, M.D.), the National Institute on Alcohol Abuse and Alcoholism (Ting-Kai Li, M.D.), and the recently established National Institute of Biomedical Imaging and Bioengineering (Roderic I. Pettigrew, M.D., Ph.D.). Dr. Zerhouni said that he is working diligently to fill other vacant positions. He sees an urgent need to ensure strong NIH leadership and to maintain a global view of the organization's role, focusing on the translation of scientific findings into real-world benefits.

In Congressional briefings, Dr. Zerhouni is asked on occasion to pinpoint what the NIH has done of value for the American public. He pointed out that, recently, a part of the answer can include the NIH connections to all three of the recently awarded Nobel Prizes for Science, not only in Medicine and Chemistry, but in Economics as well.

Dr. Zerhouni spoke of the important contributions made by the COPR founding members who are rotating off the Council: Dr. Luz Claudio, Ms. Vicki Kalabokes, Ms. Barbara Lackritz, Ms. Rosemary Quigley, Mr. Bob Roehr, and Mr. Doug Yee. Dr. Zerhouni was photographed with each and awarded them certificates.

The founding COPR members presented a plaque to Dr. Ruth Kirschstein in appreciation of her guidance of the COPR in its first years while she was the NIH Acting Director.

Report on the NIH Road Map Initiative

Dr. Zerhouni

Dr. Zerhouni gave a presentation entitled "A Vision for Medical Discovery in the 21st Century." He emphasized the importance of seeking public input for the NIH strategic vision. He reviewed the evolution of the NIH budget, which is expected to grow over the next two decades. He emphasized the need to accompany growth with wisely applied resources, to meet the challenges ahead. The increased budget, said Dr. Zerhouni, stems from the recognition that changes in biomedical research have increased in both cost and complexity. For example, a single bioimaging array experiment can cost $3,000 - $4,000. However, technological advances have reduced the time required for gene identification from years, just a decade ago, to a period of 20 minutes.

Biomedical research is a priority for the entire United States, not just for the NIH. According to projections, investment in research facilities and faculty at U.S. medical schools will triple within the next 5 years, compared to 1990 levels. An increased capacity to conduct research, to prevent, treat and cure disease, will determine the success of the U.S. research enterprise.

Increased investments lead to more applications submitted for review and more grants awarded, and they stimulate non-federal investment in research. Over the past 30 years, in the Pharmaceutical Research and Manufacturers (PhRMA), the companies have tripled their research and development expenditures, creating more than 200,000 new jobs and 1,300 new, enduring biotechnology companies. Until 1991, the NIH spent more on biomedical research than the entire pharmaceutical industry. In that year, private industry funding exceeded federal spending. Today, private industry is developing an ever-increasing number of drug targets and mechanisms, as reflected by the many new medications on the market. Private biomedical investment has reduced Federal biomedical spending to a secondary source of funding.

The unusual convergence of many of the life science areas leads explorations in new directions, said Dr. Zerhouni. Existing ideas are being redirected with new tools, technologies, and observations - sparked by the development of new approaches, such as, high-throughput screening for selection, transgenic animals and mutational analysis, and progress in structural biology. The fields of genetics and genomics are driving these discoveries. They now allow us to understand the evolutionary forces that influence gene patterns and expression. The past 10 years have produced tremendous changes in biomedical research. For the first time in human history, we have the "parts list" of nature. To master our destiny, we must continue to combat the burden of disease and accelerate the pace of discovery. The NIH is the vanguard of this effort.

The complexity of biological systems is a major barrier to questions facing biomedical science. Reductionist approaches may be unable to solve that complexity. Technical limitations hinder dynamic visualization at the subcellular level. Challenges remain in the exploration of cell trafficking, signaling, and other complex molecular systems and their controls. The "signal-to-noise" ratio limits the computational analyses of these systems. The complexity of these and other molecular systems requires us to come up with better techniques to understand them.

The NIH's Road Map Initiative outlines a vision for NIH research that identifies the scientific challenges and roadblocks to progress and indicates future directions for research. The NIH as a whole must take responsibility, because any single Institute cannot undertake this enterprise. In defining a vision for NIH research strategies, three broad priorities have emerged:

First, the NIH must explore new pathways to discovery and develop revolutionary methods of research. It must accelerate and expand existing technologies and resources, particularly proteomics and large-scale array technologies for the genomic era. We must increase the number of, and access to, molecular research libraries and press forward with methods for the structural study of complex molecules. Mathematical modeling and computer simulation of complex systems must also expand. Developing revolutionary methods of research will also include integrative approaches to systems biology, quantitative analyses of dynamic, genome-wide patterns of expression, epigenetics, differential gene expression, comprehensive analyses of complex molecular networks, and regenerative medicine.

Second, the NIH must promote the research teams of the future, by overcoming structural and cultural barriers to "team science." Aiding this effort will be the establishment of interdisciplinary undergraduate and graduate programs and the creation of better mechanisms for funding of multidisciplinary teams without compromising investigator "R01" research. Better support is needed for high-risk/high-impact research. The NIH should actively explore ways to leverage private-sector partnerships, to optimize resources. The system's key is still the creative individual.

Third, the NIH must re-engineer the clinical research enterprise. Innovative methods in clinical research need to be developed. Clinical research procedures and policies need to be standardized. Dr. Zerhouni proposed the establishment of a national clinical research data resource, national standards for clinical research data, and a national computational infrastructure. We need to find ways to facilitate access to data, such as molecular libraries for testing.

Dr. Zerhouni ended his presentation and asked for comments.

Comments and Discussion

Dr. Zerhouni responded to a comment noting the absence of the behavioral sciences from his list of areas in need of expansion and improvement. He assured the listener that the Institute budgets and needs are under careful review.

Dr. Mala asked for ideas on how busy clinicians might become involved in clinical research. Citing the large decrease in cancer hospitalizations, Dr. Zerhouni noted the movement of clinical care away from hospitals and into the community. However, the information infrastructure to monitor this trend is missing. Some Institutes are working on ways to involve community physicians, but we need the ability to standardize information gathering. These difficulties exist in the academic health centers and throughout the community. Mr. Roehr raised the possibility of using large public and private institutions—such as the Veterans Administration and Kaiser Permanente—to mine data.

Dr. Zerhouni said that the NIH wants to stimulate multidisciplinary education, not just in top-flight universities, but in 4-year colleges, focusing on general science rather than microbiology. New multidisciplinary pathways to training need to be developed in behavioral science, social science, and psychology, as well as the biological sciences. Professional schools must be encouraged to train people in ways that are less categorical.

Dr. Zerhouni responded to comments made by Ms. Lappin concerning the access to data and materials. He cited the importance of protecting intellectual property, adding that the issue is seen differently by different government agencies, e.g., the Food and Drug Administration (FDA) and the Centers for Disease Control and Prevention (CDC). However, once protections are in place, data sharing should be encouraged.

Update on the NIH Response to the Report of the COPR Working Group on Human Research Protections

Wendy Baldwin, Ph.D., Deputy Director for Extramural Research, NIH
Lana Skirboll, Ph.D., Associate Director for Science Policy, NIH

Drs. Baldwin and Skirboll reported on the NIH response to COPR's report on Human Research Protections (HRP). The report identified many of the stresses on the current system of protections. NIH is taking steps to strengthen the system. This will create burdens for researchers and require increased education.

Chief among these steps is the creation of the Human Subjects Research Enhancement Program, which designates money for improving the HRP system in many areas targeted by the COPR report. So far, the NIH has spent $25 Million on the program and made 142 awards.

Dr. Baldwin was encouraged by the Institutes' response. For example, interest is growing for awards in the behavioral sciences, for improving the informed consent process, and for ethics training. The applications process is getting underway.

The NIH is developing tools for research participants, researchers and administrators, including an "online chat room" where users can post questions. Dr. Baldwin cited increasing support for research on ethical issues, informed consent, and behavioral interactions in the research process. The growing interest, she said, reflects a recognition that the power of research has yet to be focused on the understanding of research itself. A recent announcement for a short-term ethics-training program has received a slow response, said Dr. Baldwin, but interest has been increasing. The program helps investigators understand bioethics.

Various stakeholders have met on the issue of conflicts of interest and stimulated the sharing of community insights, discussing ways in which these issues have been addressed within other institutions. A report from that meeting is forthcoming.

Comments and Discussion

Responding to a question about the extent to which the NIH has contemplated public access to research results, Dr. Baldwin said the NIH has not considered access to data during a clinical trial, because this would disrupt the research process. She acknowledged that providing this access could be important to informed consent. Ms. Buelow reiterated that the public is very interested in data sharing and wants to take part in the research process.

COPR Members expressed the need for improved dissemination of the HRP report, possibly through its publication in a peer-reviewed journal. Dr. Baldwin promised to give the matter thought and make further suggestions.

Role of the NIH in Implementing the Privacy Rule

Dr. Skirboll informed the group of the NIH's efforts to reach out to the research community with information and resources for implementing the Health Insurance Portability and Accountability Act (HIPAA). These efforts encompass a research-oriented website, brochures for general and specific audiences, speaking engagements, and trained NIH staff. The Office for Civil Rights—the Agency responsible for oversight and enforcement of HIPAA—has asked the Department of Health and Human Services (DHHS) to develop educational materials for researchers. The NIH, along with the FDA, the CDC, the Agency for Healthcare Research and Quality, and the Office of Human Research Protections is leading a cooperative effort to develop this material. DHHS committees and NIH working groups assist in the coordination effort.

Development of a National Database for Clinical Research Using Gene Transfer

Dr. Skirboll reported on the development of the Genetic Modification Clinical Research Information System (GeMCRIS), a national database for clinical research on gene transfer. GeMCRIS will serve as an analytical tool for a diverse group of users, including Federal Agencies, National Advisory Committees, investigators, policy makers, patients, and the public. A Data Safety Monitoring Review Board will be formed, to review cross-trial reports of adverse events.

GeMCRIS will allow for systematic analysis of data across clinical trials and will enhance the application of knowledge acquired from research. The database is being developed in phases, under the purview of a steering committee that is composed of representatives from the NIH Office of Biotechnology Activities and ICs with gene transfer portfolios, and in collaboration with the FDA.

GeMCRIS will facilitate the analysis of safety information from all gene transfer clinical trials for the NIH and the FDA. It will provide reports that will inform diverse user groups, including IRBs, local data safety monitoring boards, investigators, and research participants and their families. GeMCRIS will also be useful for other areas of clinical research, and for the National Library of Medicine, General Clinical Research Centers, National Gene Vector Laboratories, and the CDC, all of which have shown interest in using the database. GeMCRIS may represent the beginning of a core format for reporting adverse events for all Federal agencies and others engaged in clinical research.

Dr. Skirboll noted that privacy protections are important, citing the example of Jesse Gelsinger's father, who completely lost his privacy in the wake of the teenager's death during a gene transfer experiment. Because information submitted to the NIH database is available through the Freedom of Information Act. Dr. Skirboll anticipates that the database will receive reports that have been stripped of patient identifiers to the extent that this is possible.

Comments and Discussion

Dr. Skirboll was asked whether the FDA would subject private research to the same requirements imposed upon federally funded studies. Dr. Skirboll expects FDA to make these decisions.

Report and Discussion on COPR Planning Session

Dr. Zerhouni, Dr. O'Neil, and COPR Members

Edward O'Neil, Ph.D., a professor of family and community medicine and dental public health, and director of the Center for the Health Professions at the University of California, San Francisco, facilitated the COPR Planning Session, and reviewed the outcomes of pre-meeting discussions with COPR members. In confidential interviews, he explored the opinions of the COPR members about COPR's role within the NIH. He asked COPR members some key questions:

• What do you see as COPR's role at the NIH and for the country?
• What issues are of particular importance to you?
• What ways does COPR work best?
• Where could it be improved?

From the interviews, he said, "no particular stain of advocacy" had emerged. Dr. O'Neil summarized the interviews and the pre-meeting discussions. He found that members view COPR's role as having three main elements.

First, COPR is to provide a voice for public input to the NIH and its Director. All but two members see this as a central role. All members recognize the need to incorporate public concerns into NIH strategies, but they are uncertain how it should be done. Members have different opinions on the proper role of COPR. They contemplate a role that is primarily advisory in nature, as well as one that takes an activist stance. On the whole, members are concerned about duplicating the work of the NIH Offices of Public Liaison (OPLs). They have concerns about the public's attitude toward the policy process in science. All see the important relationship between the needs of the public and the NIH agenda.

Second, COPR is to carry the NIH message to the various public groups represented by the Council. Some members do not consider this role important, and COPR members have uneven interests and abilities to serve this networking function. Those carrying the NIH message would like more guidance. Members recognize that they must integrate their roles as a public voice with their outreach responsibilities. They seek adequate management and support that is accounted for systematically - with real evidence.

Third, COPR members must participate in focused work that addresses the goals and concerns of the NIH and COPR. Members agree: the best work to date is the report produced by the COPR HRP workgroup. To be successful, such work requires an extraordinary commitment, adequate resources and encouragement from the NIH.

Other important issues that emerged from the members' discussions on the previous day:

• Safety of human research subjects
• Health disparities
• Public understanding of the role and value of biomedical research
• Public input into new research directions

Informal discussions have worked well in the past, particularly where work focused on a single issue and individual members worked directly with the NIH. The Council's diverse membership is an asset. Other strengths include COPR's access and relationships, the agenda-setting process, and the ability to bring the NIH to local communities.

The Council cited areas in need of improvement. Members should reach a consensus about COPR's role and expectations of its work. Members would like the Director to incorporate his expectations for COPR into his broader strategic vision for the NIH. Members also want to understand COPR's distinctive role as the "capstone" in the system of public input. If public input is valued, then the agenda-setting and decision-making processes must be clarified and tied to the Director's agenda.

Members would also like clarification on how to use NIH resources to carry out the Council's work. COPR seeks clarification on how its work is disseminated and used within the NIH, and what its relationship is to other NIH public advisory bodies. Once COPR's role and agenda have been clarified, the need for meetings can be assessed (i.e., how many per year and appropriate timing). Adequate staff support is necessary, and accountability between COPR and staff needs to be defined.

Next, Dr. O'Neil presented the "COPR Action Agenda:"

• Develop testimony for the IOM Committee's Review of the NIH Structure and submit to the NIH Director, who will forward to the IOM Committee
• Continue setting COPR's priorities, in cooperation with the Agenda Work Group, whose co-chairs will also serve on the Executive Issues Work Group and COPR liaison
• Enhance communication with the members of other public groups and councils, in an effort to look at public participation practices within the NIH and to do so by:
  • Sending a COPR representative to the ACD Meeting on December 5, 2002
  • Drafting a report examining other public participation practices
  • Exploring the possibility of a summit on public input across the NIH

COPR Member Comments and Discussion

Ms. Kalabokes and Dr. Mala reported that the COPR Working Group on Health Disparities has received valuable assistance from the various OPLs.

Dr. Muñoz raised the issue of retaining public trust in the research enterprise. The members made various suggestions for enhancing the NIH's public image. Dr. Mala suggested posting "one-liner" advertisements in public places, such as on public transportation. The ads could highlight some NIH achievements and research activities. (e.g., "9,864 research grants this year alone. NIH: Protecting Your Health") Ms. Hinton agreed that the NIH needs to tout its own accomplishments. She envisioned a booklet providing the history of the Institutes, with a timeline of achievements. (e.g., "NIH: The First 50 Years"). Dr. Kirschstein noted that something similar was developed in 1987 when the NIH celebrated its centennial. The 1987 publication of the NIH history and the NIH Almanac would be wonderful resources for this type of information. They are available for any COPR member who requests them.

It was suggested that a speaker could be invited to a COPR meeting to talk about the history of medicine and the history of the NIH. Mr. Burklow mentioned that Dr. Victoria Harden, NIH Historian and Director of the Dewitt Stetten, Jr. Museum of Medical Research, might be good choice for such a talk. The COPR Agenda Working Group will consider inviting Dr. Harden to speak.

Concerning the issue of public trust, Ms. Lappin said that the Conflict of Interest conference, mentioned earlier by Dr. Skirboll, was a great success. An overarching theme was retention of the public trust, and the conference addressed many issues from the COPR Human Research Protections (HRP) Report.

Mr. Yee expressed concerns about reaching a diverse audience with NIH messages and activities. Many of the people he deals with are not college graduates, and it is important that messages be targeted to them.

Ms. Quigley agreed that surveys show the public has difficulty in trusting biomedical researchers, but that those who are sick still invest a great deal of hope and remain willing to participate in clinical research.

Dr. Skirboll said that in order to retain public trust, the goal must be to ensure the trust of the community. Most surveys, she said, show support for scientists relative to other professions. Dr. Kirschstein concurred with these remarks, saying that Gallup polls have shown that many Americans think the best hope and benefit for their health lies in biomedical research.

Wrap-Up and Scheduling of Upcoming COPR Meetings

The following future meeting dates were set:

2003:

• April 24-25
  
• October 20-21

2004:

• April 26-27
  
• October 18-19

Dr. Zerhouni thanked all those present for their interest and participation. The meeting was adjourned at 4:30 p.m. on Tuesday, October 22, 2002.

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