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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Public Health Service
National Institutes of Health
John E. Fogarty International Center
for Advanced Study in the Health Sciences
Minutes of the Advisory Board
Forty-eighth Meeting
Table of Contents
Attendance
- Call to Order
- Consideration of Minutes of Previous Meeting
- Review of Requirements for Confidentiality and Conflict
of Interest Procedures
- Future Meeting Dates
- Report of the Director
- FIRCA: A Historical Overview
- An Independent Not-For-Profit Initiative to Develop Drugs for Neglected Diseases: A Project of Médecins Sans Frontiéres
- Discussant
- Closed Portion of the Meeting
- Review of Application
- Adjournment
The meeting of the Advisory Board of the John E. Fogarty International Center for Advanced Study in the Health Sciences (FIC) was convened on Tuesday, May 22, 2001, at 8:35 a.m., in the Conference Room of the Lawton Chiles International House, National Institutes of Health (NIH), Bethesda, Maryland. The meeting was open to the public from 8:35 a.m. to 12:40 p.m., followed by the closed session for consideration of grant applications until adjournment at 2:30 p.m. Dr. Gerald T. Keusch, Director, FIC, chaired the overall meeting, and Dr. Sharon Hrynkow, Deputy Director, FIC, chaired the applications review.
Board Members Present:
Dr. Margaret A. Chesney
Dr. Ezekiel Emanuel
Dr. Cutberto Garza
Dr. Steven E. Hyman
Dr. Patricia Marshall
Dr. Jane Menken
Dr. Jaime Supulveda
Dr. Catherine M. Wilfert
Board Members Absent:
Dr. Barry R. Bloom
Dr. Mary Claire King
Dr. Cora B. Marrett
Mr. Thomas J. McAndrew
Dr. Theodore Reich
Dr. Robert E. Shope
Members of the Public Present:
Dr. Mariam Claeson, The World Bank, Washington, D.C.
Dr. Joy deBeyer, The World Bank, Washington, D.C.
Dr. Dean T. Jamison, University of California at Los Angeles
Dr. Prabhat Jha, The World Bank, Washington, D.C.
Dr. Chris Lovelace, The World Bank, Washington, D.C.
Dr. Anthony R. Measham, The World Bank, Washington, D.C.
Ms. Angela Sharpe, Consortium of Social Science Associations, Washington, D.C.
Dr. Gavin Wright, Triangle Research Institute, Chapel Hill, North Carolina
Federal Employees Present:
Dr. Martin Alilio, FIC/NIH
Ms. Judith Levin, FIC/NIH
Mr. Amar Bhat, FIC/NIH
Dr. Jeanne McDermott, FIC/NIH
Dr. Joel Breman, FIC/NIH
Dr. Kathleen Michels, FIC/NIH
Dr. Kenneth Bridbord, FIC/NIH
Dr. Mark Miller, FIC/NIH
Mr. Bruce Butrum, FIC/NIH
Mr. Richard Miller, FIC/NIH
Ms. Jennifer Cabe, FIC/NIH
Dr. Rachel Nugent, FIC/NIH
Dr. Lois K. Cohen, NIDCR/NIH
Dr. Sherri Park, NICHD/NIH
Mr. Eric Dakake, FIC/NIH
Ms. Charlotte Quinn, FIC/NIH
Ms. Irene Edwards, FIC/NIH
Dr. Theodore J. Roumel, OD/NIH
Ms. Mollie Fletcher, FIC/NIH
Ms. Minerva Rojo, FIC/NIH
Dr. Phyllis Freeman, FIC/NIH
Dr. Joshua Rosenthal, FIC/NIH
Dr. Pierce Gardner, FIC/NIH
Dr. Luis A. Salicrup, FIC/NIH
Ms. Lee Ann Gschwind, FIC/NIH
Dr. Barbara Sina, FIC/NIH
Ms. Anne Hill, FIC/NIH
Ms. Janice Solomon, FIC/NIH
Dr. Karen Hofman, FIC/NIH
D. Shveta Srivastava, FIC/NIH
Ms. Cherice Hollaway, FIC/NIH
Ms. Natalie Tomitch, CSR/NIH
Dr. J. Allen Holt, FIC/NIH
Mr. Al Villatoro, FIC/NIH
Dr. Sharon Hrynkow, FIC/NIH
Ms. Helen Wegman, NHLBI/NIH
Mr. Andrew Jones, FICNIH
Dr. Sandy Warren, CSR/NIH
Dr. Gerald T. Keusch, FIC/NIH
Mr. Randolph Williams, FIC/NIH
Dr. Richard Krause, FIC/NIH
OPEN PORTION OF THE MEETING
I. CALL TO ORDER
Dr. Gerald T. Keusch called the open session of the Advisory Board meeting to order at 8:35 a.m. on Tuesday, May 22, 2001. He welcomed Board members, NIH staff, and guests. Dr. Keusch welcomed and introduced Dr. Jane Menken, a new Board member attending her first Board meeting. He noted that Dr. Robert E. Shope, also a new Board member, had participated in the Board's Research Awards Subcommittee the previous day but was unable to attend the present meeting. He also noted that Dr. Theodore Reich, who is recovering from laryngeal surgery, was unable to attend the Board's meeting. Dr. Keusch congratulated Drs. Cutberto Garza and Catherine M. Wilfert on the graduation of their respective son and daughter from medical school. He introduced several guests and presenters attending the meeting.
II. CONSIDERATION OF MINUTES OF PREVIOUS MEETING
The minutes of the Advisory Board meeting of February 6, 2001, were considered and accepted unanimously.
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III. REVIEW OF REQUIREMENTS FOR CONFIDENTIALITY AND CONFLICT OF INTEREST PROCEDURES
Dr. Keusch thanked the Board members for providing their updated Confidential Financial Disclosure Reports to the FIC.
IV. FUTURE MEETING DATES
The following meeting dates were confirmed:
Tuesday, September 18, 2001 (*changed from September 11, 2001)
Tuesday, February 5, 2002
Tuesday, May 21, 2002
Tuesday, September 10, 2002
The proposed meeting dates for 2003 are:
Tuesday, February 11, 2003
Tuesday, May 20, 2003
Tuesday, September 16, 2003
Dr. Keusch asked Board members to inform Ms. Irene Edwards, Executive Secretary, of any scheduling conflicts they may have with the future dates. All subcommittees of the Board will meet on the Monday preceding each Board meeting.
V. REPORT OF THE DIRECTOR
Dr. Keusch invited FIC staff members to report on the FIC budget and selected programs. The written Report of the Director was distributed previously to Board members, was available at the meeting, and is appended to these minutes as Attachment 2, Written Report of the Director.
Fiscal Year 2002 Budget
Mr. Richard Miller, Executive Officer, FIC, presented an update on the NIH and FIC budgets for Fiscal Year (FY) 2002. Mr. Miller noted that the Administration recognizes, and is giving priority to, the commitment to double the NIH budget over a 5-year period. The President's budget for FY 2002 proposes approximately $23.0 billion for the NIH, or about a 13.5 percent increase over the FY 2001 budget. The final FY 2001 budget totals approximately $20.3 billion, or about a 13.7 percent increase over FY 2000.
For the FIC, the President's budget for FY 2002 proposes approximately $56.5 million, or about an 11.8 percent increase over FY 2001. The FIC's final FY 2001 budget is approximately $50.5 million, or about a 16.6 percent increase over FY 2000. Mr. Miller noted that the percentage increase proposed for FY 2002 is about the same for almost all NIH institutes and centers (ICs), with the exception of new funding for the new National Institute of Biomedical Imaging and Bioengineering (NIBIB), a larger increase for the National Center for Research Resources to support extramural construction, and a large increase for the Office of the Director associated with the Women's Health Initiative.
FIC's FY 2001 budget includes support for three new initiatives: the International Studies in Health and Economic Development (ISHED) Program; the International Clinical, Operational, and Health Services Research Program; and the Epidemiology Program. The President's budget for FY 2002 will enable FIC to expand slightly the Fogarty International Research Collaboration Awards (FIRCA) Program and to launch three additional initiatives: the International Tobacco and Health Research and Capacity Building Program, the Stigma Research Initiative, and the Incorporating New Genetic Tools in Global Health Strategies Program.
Mr. Miller noted that between FY 1992 and FY 2002 (the President's budget), FIC's appropriated level of funding has grown from approximately $20 million to slightly less than $60 million. Appropriated funds for non-AIDS programs have increased from approximately $14 million to $38.4 million, while those for AIDS-related programs have increased from approximately $5.6 million to $18.1 million. Collaborative funds from FIC's co-funding partners within and outside the NIH, in support of FIC programs, have increased from less than $1 million in FY 1992 to an estimated $14 million to $15 million in FY 2002. Representative of these partnerships is the significant funds FIC receives from the National Center for Minority Health and Health Disparities (formerly the NIH Office of Minority Research) to support the Minority International Research Training (MIRT) Program.
In closing, Mr. Miller said that the process for completing the FY 2002 budget is "moving along." On April 4, Dr. Keusch participated in a panel on Special Populations and Health Disparities before the House Appropriations Subcommittee. On May 16, Dr. Ruth Kirschstein, Acting Director, NIH, and the IC directors participated in the House hearings on the FY 2002 NIH budget, and on May 23, they will participate in the Senate hearings.
Dr. Keusch said that the FIC has targeted its planned initiatives in FY 2002 to the $6 million increase proposed for FIC in the President's budget and that other initiatives discussed previously with the Board will be on hold until additional resources become available. He noted that FIC's greatly strengthened relationship with other ICs has resulted in a significant amount of co-funding and is a strong and favorable indicator of FIC's continued progress.
International Studies in Health and Economic Development Program
Dr. Rachel Nugent, Program Officer, Health and Economic Development Research Program, Division of International Training and Research (DITR), FIC, reported that the FIC received a large number of diverse research proposals in its first competition for awards under the ISHED Program. Based on the Board's recommendations, the FIC adopted a two-tiered approach to fund 10 proposals. Five proposals were fully funded, and five received developmental funds. The research supported is geographically diverse, with three awards for studies in Asia, five for studies in Africa, and two for studies in Latin America. The research topics include the relationship between health and economic productivity at the microeconomic level (seven awards); the linkage between health inequality and social status (one award); the impact of HIV/AIDS on businesses in South Africa (one award); and the impact of malarial interventions on national economies (one award). Dr. Nugent noted the significant support of FIC's partners in this program, which include the NIH's National Institute on Aging, National Institute on Mental Health (NIMH), National Institute of Child Health and Human Development, Office of Behavioral and Social Science Research, and the World Bank's Global Development Network. The FIC intends to advertise the program again for a second competition in 2003. A networking meeting of the current U.S. investigators and collaborators will be held in conjunction with the annual meeting of the Global Development Network, in Brazil in December 2001.
Discussion. Commenting on FIC's role in addressing methodological issues concerning health and disease outcomes, Dr. Nugent said that the researchers are considering broad, longitudinal measures (e.g., social status) of health outcomes within the context of economics. Dr. Keusch emphasized the need for an effective system for assessing burden of disease, and he encouraged discussion of this topic by the Board. Dr. Mark Miller, Associate Director for Research, FIC, noted that the U.S. Department of Health and Human Services (DHHS) has established a trans-DHHS committee, which includes FIC representation, to systematically address assessments of disease burden and health outcomes and to suggest new methodologies and standards for measurement. He noted that the measure used most often, disability-adjusted life years (DALYs), has been adopted by default and is criticized by many health professionals. This suggests the need for a broader assessment of disease burden assessment and new research on alternative methodology to develop an effective and appropriate measurement system that can be more universally recommended.
The Board commented on the importance of worldwide input and linkage with the World Bank and international economists for developing countries participating in the ISHED program. Dr. Keusch noted the Bank's interest in the program, the contribution of the Global Development Network to the program, and FIC's continuing efforts to foster linkages and networks between public health personnel and economists.
The Board also commented on the importance of the methodological issues and commended the FIC on its involvement in discussions of the issues. The Board noted the various types of data that are being collected (e.g., health survey data, biological markers, genetic information) which are relevant to measuring health outcomes. Dr. Keusch noted the need for research training and support to strengthen research capacity in this area in developing countries. The Board emphasized that the discussions should include NIH experts in biomarkers and genetics, as well as consideration of the costs and ethics of collecting and storing DNA samples.
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International Clinical, Operational, and Health Services Research and Training Award
In February, the FIC, in collaboration with five other ICs, issued a Request for Applications (RFA) for the International Clinical, Operational, and Health Services Research and Training Award (ICOHRTA). The due date for applications was April 27. Dr. Keusch noted that the award is focused on behavioral science. In consultation with the Board, the FIC has conceived this award as an initiative to promote clinical research expertise in developing countries and, even broader, to achieve a seamless transition between clinical, operational, and health services research. The objective is to create linkages among these activities and the individuals and institutions which support and conduct them. The ultimate goal is to improve health by implementing research results through policy and practice. The FIC intends to fund one new ICOHRTA program in a different health area each year over the next 5 years. In the first competition, the FIC received 25 applications and expects to fund awards in FY 2001. Dr. Keusch highlighted the ICOHRTA program as an example of FIC's change in thinking and strategy, to focus on targeted programs in collaboration with other ICs in areas of mutual interest.
FIC's Office of Grants Management
Dr. Keusch noted that, since 1987, the FIC's portfolio has expanded from three categories of training programs to a wide range of different programs offering both investigator-initiated awards (R01s) and training grants. To handle the increasing number and complexity of FIC awards, FIC has established an Office of Grants Management within the FIC's Office of the Director. Dr. Keusch introduced the director of the office, Mr. Bruce Butrum.
Mr. Butrum briefly described the organization and functions of the grants office and two new procedures implemented by the grants staff. Prior to January 2001, FIC's grants activities were performed by DITR program staff. The grants office, established in January, consists of the Grants Management Officer, two grants management specialists, and a half-time program support assistant. The grants office staff ensure that grants are awarded and official files are maintained properly in accordance with Federal guidelines; advise FIC staff on grants policy and procedures; help develop RFAs and revise program announcements; and plan to help foreign grantees by giving presentations and materials on grantsmanship and the administration of grant funds.
Two new procedures already implemented by the grants staff are (a) the recycling of start dates for grants, and (b) the expediting of awards. By recycling the start dates of competing and non-competing awards, the FIC can distribute the fiscal and scientific administration of grants throughout the fiscal year. Staff already have been able to recycle 80 percent of FIC's non-competing awards. To expedite the process of getting FIC grant funds to awardees, the FIC is (i) requesting informal "just-in-time" information (i.e., required information to fund grants) from grantees and (ii) making restricted awards before resolving all administrative issues. Mr. Butrum noted that these and other changes being implemented by grants staff will significantly improve FIC's grant operation, planning, and management in FY 2001 and future fiscal years.
Noting that the new grants office is part of FIC's maturation, Dr. Keusch thanked the FIC staff for their extraordinary performance during the past 2 years in successfully developing, implementing, and administering FIC's rapidly expanding portfolio of programs. He noted that the grants staff is a wonderful advance for the FIC and that the staff has very positive working relationships with FIC's program and administrative staff.
International Tobacco and Health Research and Capacity Building Program
Ms. Anne Hill, Research Assistant, Division of Advanced Studies and Policy Analysis (DASPA), FIC, presented an update on FIC's tobacco research and training initiative. The FIC has targeted tobacco control as one of its first initiatives in the noncommunicable disease arena because of the magnitude of the problem and the unlimited spending of the tobacco industry to promote its products nationally and internationally. Previously, the FIC stimulated the formation of a trans-NIH group to define the needs for a comprehensive tobacco control program, including priorities for research and training. Ms. Hill noted that the ICs' response to FIC's invitation was tremendous and that, over the past 2 years, the trans-NIH group has crafted a program that focuses on both the needs of scientists and institutions in developing countries and NIH interests. The unique research interests and needs of low- and middle-income nations were clarified in discussions with scientists from developing countries at a group meeting held in conjunction with the 11th World Conference on Tobacco OR Health, held July 2000.
In cooperation with five ICs, the FIC is preparing a comprehensive RFA to solicit proposals for the new International Tobacco and Health Research and Capacity Building Program. Ms. Hill noted that the World Health Organization's (WHO) Tobacco Free Initiative has been enlisted as a cosponsor of the RFA. The NIH's RFA is expected to be issued within the next few weeks. The total funds available to support meritorious applications are estimated at $3.5 million each year, for a total of approximately $18 million over 5 years, of which the FIC will be contributing approximately $2.5 million. More than 50 percent of the direct costs associated with each award will support research or capacity building in low- and middle-income nations. The FIC anticipates funding six to eight grants. Five key research areas are identified in the RFA: epidemiological and surveillance research, susceptibility and risk, biobehavioral and social research, intervention research, and policy-related research. Equal emphasis is being given to research and to the strengthening of capacity and infrastructure in developing countries. The due date for applications is October 26, 2001.
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Discussion. Dr. Keusch noted that this initiative is a first example of FIC's integration of research and training from the beginning of an R01 award and the stipulation of a strong training component. This step was taken because of the lack of scientific background on issues of tobacco and health in developing countries and the need to develop an informed public health constituency and relevant policy research to combat the economic dependence of countries on the sale of tobacco products.
The Board applauded the FIC and its collaborative partners for launching such a comprehensive, innovative, inclusive, and forward-looking initiative. The Board noted that the combination of biobehavioral, intervention, and policy research is needed for other health problems as well (e.g., weight management) and that this approach is just beginning to be addressed in other venues [e.g., at the Institute of Medicine (IOM)]. Dr. Keusch said that the FIC will be striving to encourage a larger coalition of institutions and agencies to participate in the initiative. The FIC already has discussed tax and economic issues with the World Bank and enlisted the interest of the Rockefeller Foundation in sponsoring some networking meetings. The Board encouraged the FIC to seek the participation of the U.S. Department of Agriculture.
Ms. Hill further clarified that the initiative addresses both prevention and cessation of tobacco use and that the RFA focuses in certain sections on young adolescents or children. While gains in cessation of smoking have been made in the United States and the overall prevalence of smoking has declined in Mexico, for example, the commencement of smoking, as well as other risk-taking behaviors, is increasing dramatically among youth in many countries.
The Board encouraged the FIC to target the capacity-building efforts beyond public health officials to include legislators and economists, who can articulate fiscal reforms (e.g., earmarked taxes) to decrease tobacco use. Special efforts also are needed to understand and control tobacco use among young girls and to influence policymakers to ban tobacco advertisements. The Board suggested that the Rockefeller Foundation could provide consultation on these dynamics of tobacco control.
Stigma Research Initiative
Dr. Keusch described the development of FIC's initiative to stimulate research on stigma. The need for this initiative became apparent in discussions with Dr. Steven E. Hyman and his colleagues at NIMH on opportunities for collaboration in mental health research and in consultation with Dr. Arthur Kleinman during his week-long visit to FIC. Dr. Keusch noted that stigma is a problem in public health. Beyond being a violation of human rights, for which legal or judicial solutions are typically sought, stigma has a major impact on public health. It affects a person's willingness to seek diagnosis for a health problem, to access available care, and to participate in biomedical or behavioral research.
In cooperation with NIMH, the FIC invited other ICs to from a trans-NIH working group to address research issues in stigma. Dr. Keusch noted that the other ICs were very responsive and, over the past 4 months, the working group decided to consider developing a research program in stigma. As a first step, the FIC and NIH partners will convene an international conference on stigma, to be held in Bethesda, Maryland, on September 5-7, 2001. Ms. Emily Smith of DASPA and Dr. Kathleen Michels of DITR have jointly taken on the coordination functions for this initiative. The multi-IC working group is planning for 250 participants, with the goal that at least 100 will come from developing countries. The objectives are to summarize the state of knowledge on stigma, the mechanisms or determinants of stigma, the role of stigma as a determinant of health, knowledge gaps, and research methodologies that are available or needed. The goal is to develop a research agenda that could be implemented through a collaborative RFA for research and training. The participants will focus on HIV/AIDS, schizophrenia, epilepsy, drug and alcohol abuse, and physical deformities. They also will address the role of the media and the "two faces" of stigma-negative stigma and positive stigma (e.g., against tobacco use, alcohol or drug abuse, or suicide). Dr. Keusch noted that information on the conference (e.g., announcements,Web-based conference applications, agenda) will be available by the end of May.
Discussion. The Board congratulated the FIC on its vision in developing this initiative. Two key questions for discussion at the conference were noted: How will the results of research on stigma be translated into changing the attitudes of the public? How will success (i.e., less stigma) be measured and evaluated? Dr. Hyman noted that stigma is an enormous problem for individuals who have different diseases (e.g., HIV/AIDS, cancer, epilepsy) and is an important and complex intellectual and research issue. Dr. Keusch commented that no new theoretical approaches have been developed during the past 30 years to understand stigma behaviors.
Genetics Initiative
Dr. Karen Hofman, Acting Director, DASPA, FIC, described steps FIC has taken recently to develop a genetics initiative which would help bridge the divide in genetics research between developing and industrialized countries. First, the FIC invited NIH stakeholders, including other ICs, to form a trans-NIH group to address the type of genetic research currently funded in developing countries. Dr. Hofman noted that the group has met three times and is very interested in collaboratively initiating funding for genetic research and for addressing ethical, social, and legal issues pertaining to this research. Second, the FIC organized and chaired a workshop, entitled "Addressing the Global Divide in Genetic Science: A Consultation with Scientists to Assess the Needs for Research and Training Programs in the Developing World," which was held in Vienna, Austria, on May 16, in conjunction with the International Congress of Human Genetics. The workshop participants identified a range of research needs, expressed serious concerns about the sustainability of genetic research programs in developing countries, and emphasized the need to address ethical issues. The FIC will report on these discussions to the trans-NIH group. Dr. Hofman also mentioned that the FIC will expand its consultations to include others who would be involved in collaboratively supporting and conducting research and training.
Discussion. In response to a query from the Board, Dr. Hofman said that no research or training priorities have yet been determined. The Board cautioned against diluting a potentially small program across a range of activities.
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VI. FIRCA: A HISTORICAL OVERVIEW
Dr. Richard M. Krause, Senior Advisor, FIC, summarized the history and accomplishments of FIC's FIRCA program. As the program approaches its 10th year, the FIC is beginning an evaluation of its impact. Dr. Krause noted that the basis for the program originated in 1988 when, at the request of Dr. James Wyngaarden, then Director of NIH, the FIC initiated a Latin American Initiative to foster collaborative research opportunities and to stimulate research grant applications from Latin American scientists. In 1991, with the collapse of the U.S.S.R., the FIC extended the regional initiative to Eastern and Central Europe to seed long-term collaborative ties between U.S. institutions and counterparts in this region. In 1992, the FIC initiated the FIRCA mechanism as a new extramural grant award to fund these regional activities. The award provided supplemental funds ($20,000 a year for 2-3 years) to NIH-supported U.S. investigators for collaborations with scientists in the two regions.
Dr. Krause noted that the FIRCA program has been praised highly since the beginning by U.S. scientists, the Congress, and the IOM. Besides the research support provided, collaborating scientists in Russia have noted the program's value in conveying the concept of peer review and providing funds directly to investigators. The FIRCA has served as a model for other programs developed, for example, by the Howard Hughes Medical Institute and the U.S. Civilian Research and Development Foundation.
Referring to a handout provided to the Board, Dr. Krause noted that the FIRCA program has grown from 35 funded awards in FY 1992 to 109 awards in FY 1999. During this time, the program also was extended to other regions (Africa, Asia/India/Middle East, the Caribbean, and Western Europe). In FY 1999, the FIC initiated the AIDS-FIRCA to support research collaborations with scientists in all these regions and in Canada. A list of FIRCA grant recipients, FY 1992-97, who are members of the National Academy of Sciences (NAS), was prepared by the NAS and indicates the high visibility of the program and the range of science supported.
In closing, Dr. Krause posed several questions to consider when evaluating the impact of the FIRCA program. For example: Have the foreign scientists moved on to positions of reasonable security in their country? How productive have the foreign scientists been? Do they still need to collaborate with U.S. scientists to maintain their creativity and support? Has the program helped to strengthen the foreign scientists' institutions? Should the program be continued or modified (e.g., to focus on specific scientific areas, award funds directly to the foreign scientist)? Is the program funded adequately? Is the total number of awards sufficient?
Dr. Keusch said that the FIC is beginning the FIRCA evaluation in response to the Board's expressed interest in evaluation and will pursue the evaluation in consultation with the Board. He noted that regional initiatives continue to be important at the FIC. A new initiative is being considered, for example, in Latin America in light of the success of the FIC-Pan American Health Organization fellowship program.
VII. AN INDEPENDENT NOT-FOR-PROFIT INITIATIVE TO DEVELOP DRUGS FOR NEGLECTED DISEASES: A PROJECT OF MÉDECINS SANS FRONTIÉRES
Dr. Yves Champey, Project Coordinator, Not-for-Profit Initiative (NfPI) to develop drugs for neglected diseases, Médecins Sans Frontières (MSF), introduced the NfPI, which MSF is considering as a means to develop new and affordable medicines for neglected diseases. The principles include cost-effective research and product development in the public's interest achieved through collaboration and support of the public and private sectors. He noted that the presentation before the Board was the first public discussion of NfPI and that development of the initiative is a "work in progress." Joining Dr. Champey in the presentation were Dr. Hellen Gelband, Consultant, MSF, Washington, D.C., and Dr. Nicolas de Torrente, Director of the MSF New York office.
Dr. Torrente briefly described MSF's interest in this initiative. Founded in 1971, MSF is an independent medical humanitarian organization with 19 offices around the world. MSF conducts medical assistance programs for the most disadvantaged people in times of crisis (e.g., as a result of warfare, famine, displacement, epidemics). In recent years, the volunteer doctors have become increasingly frustrated by the unavailability of drugs to treat patients (i.e., nonexistent drugs for specific diseases, too-high-priced drugs, and ineffective and toxic treatments). In response, 2 years ago, MSF launched its Access to Essential Medicines Campaign. Directed from the Geneva MSF office, the campaign was strengthened financially and publicly by the monies and attention MSF received on winning the 1999 Nobel Peace Prize. The campaign includes efforts to reduce the cost of AIDS medications in developing countries and to secure the production of drugs no longer produced (e.g., for sleeping sickness). An essential component of the campaign is developing the means to redress the lack of drugs for neglected diseases. The Drugs for Neglected Diseases Working Group, consisting of experts from many different disciplines and fields, has been exploring various alternatives, including the NfPI.
Dr. Champey expanded on the concept of the NfPI. He noted that the most neglected diseases of particular interest to the NfPI are those affecting large, but often geographically clustered, groups of people, but which receive little public attention (e.g., trypanosomiasis, leishmaniasis). At least initially, diseases such as tuberculosis or malaria, already the focus of national and international attention, would not be tackled, although they might be in the future. Dr. Champey emphasized that, for diseases such as trypanosomiasis and leishmaniasis, a new approach is needed that combines development of new, more effective, and less-expensive drugs; transfer of research knowledge gained in public institutions to drug innovation by industry; advocacy on behalf of patients in very poor and remote geographic regions; and the financial, scientific, and technological resources of governments in both the north and south.
The principles being defined for the NfPI include establishing an independent entity with a social vision and most of the attributes of the pharmaceutical industry, but without a profit motive; ensuring that patients have equitable access to the research results of modern medicine; engaging in capacity building and technology transfer; and adopting a rational, integrated approach that includes global health policy and planning. The major focus would be needs- and knowledge-based research and development (R&D) of innovative compounds, pursuit of novel combinations of existing drugs, and reformulation of existing products for easier use. Interactive capacity building and technology transfer, from the south to the north and vice versa, would be complementary. Dr. Champey emphasized that money is only part of the answer to solving the problem of neglected diseases. Collaboration through north-south interaction; broad and multiple R&D partnerships; and national, multinational, and international organizations must be strong (in numbers and quality) for the initiative to succeed.
Dr. Champey described the organization, management, and funding proposed for the NfPI. MSF aims to develop a new paradigm, or business model, which involves engaging the public research institutions (e.g., the NIH) in identifying knowledge that is ready to transfer to drug development and inviting (and paying) the pharmaceutical industry to contribute its specific technological expertise (in personnel and tools) to develop the drugs. Guiding the effort would be a small group of talented people, who would work through collaborating institutions and industry, and an independent management team. The legal status of NfPI will be addressed with respective countries, and MSF will establish a board of directors and scientific advisory committees.
Dr. Champey noted that, to pursue the initiative, MSF must be assured of a minimum of 5-year financing, to demonstrate that sustainable, long-term, predictable, and reliable sources of funding can be provided. MSF envisages a mix of public and private funding (perhaps two-thirds and one-third, respectively), consistent support, and in-kind contributions. MSF would assume a lead role, but the entity would be independent.
In closing, Dr. Champey thanked the FIC for the opportunity to present the concept of the NfPI publicly and invited the Board's comments and critique. He noted that MSF will continue similar consultations during this initial information-gathering stage, to inform individuals and organizations about NfPI, obtain reactions, assess the potential for collaboration and synergy with other initiatives, and explore funding possibilities.
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VIII. DISCUSSANT
Dr. Keusch invited Dr. Arthur Amman, President, Global Strategies for HIV Prevention, San Rafael, California, to present his perspective on the NfPI, drawing on his substantial experience and leadership in both industry and clinical and prevention research in developing countries. Dr. Amman fully supported the concept of the NfPI and urged that it move forward quickly under the leadership of MSF which, he noted, has the credibility and international reputation needed for such an effort.
Dr. Amman emphasized that the reason for discussing an NfPI is not because of a failure in drug development, but because drug development has been so successful. The power and profitability of the pharmaceutical industry are indicative of its success in fulfilling all aspects of drug discovery and meeting health care needs. Despite the benefits for many people, the cost of drugs has become a universal complaint. The cost of drugs to treat all diseases in the United States will reach an estimated $1.4 trillion by 2011-a conservative estimate. And, most drugs are to treat disease, not cure or prevent disease. Patients in developing countries have been essentially "left out" because they cannot afford the drugs, or drugs that they need are simply not profitable enough to produce. Some of the costs the patients bear in drug discovery and testing (e.g., providing blood samples, participating in clinical trials) arguably could be returned to them (e.g., through reduced drug prices, development of drugs for neglected diseases). Dr. Amman emphasized that, if the benefits of new discovery are to be realized in developing countries, the recent changes that government has made to favor pharmaceutical companies (e.g., in patenting, accelerated drug approval resulting from the biotechnology revolution, regulatory reform, incentives, intellectual property rights, acceptable profit margins) must be reconsidered. He also emphasized that the price of drugs, or the use of generic drugs, is not the sole issue; a focused effort is needed to discover and develop drugs specifically for diseases in developing countries.
Dr. Amman summarized several major issues that will have to be addressed in organizing the NfPI. Examples of these concerns are: defining and maintaining the independent status of the organization versus the collaborative nature of the initiative, identifying private/public funding and other sources of funds (e.g., fees, tariffs, taxes), achieving self-sustainability of a not-for-profit entity, providing appropriate and effective incentives to attract scientists and other participants, assuring a strong legal standing internationally, accommodating or modifying international patent laws, transferring public intellectual property into private intellectual property, and handling conflict-of-interest issues.
In closing, Dr. Amman noted that his comments were meant to be provocative, in the positive sense, in relation to the potential of the NfPI. He encouraged MSF to continue to pursue consultations on the initiative and noted that the obstacles, which primarily concern funding and legal issues, are known but are not insignificant.
Discussion. Dr. Keusch noted that the concept of the NfPI is very positive and collaborative and fits well with FIC's long-term goal of translating research into improved health globally. He emphasized that industry is an appropriate partner and needs to be encouraged to focus on the health needs of developing countries. He commented that, in contrast to 20 years ago, research in academia is supported by multiple public and private sources and that the collaborative process envisaged for the NfPI will be complex and must be carefully thought out. Without a clearly defined process, NIH's role and leverage, for example, could be diluted. Dr. Keusch agreed with Dr. Amman that MSF has taken a significant step in identifying a niche and that the evolution of the concept and process is now extraordinarily important.
Dr. Theodore J. Roumel, Assistant Director for Management and Operations, Office of Technology Transfer, Office of the Director, NIH, commented on the interaction among the NIH, academia, and industry. He noted that the key issue in drug development is not patents, but licensing, and that, to "prime the pump" of research, researchers should be rewarded for their innovations. He said that the NIH has no legal authority over universities' licensing of drug products, but does issue exclusive, limited licenses for NIH-developed technologies (currently, about 1,200 active licenses) and retains the rights to continue the research. He agreed that the organization of the NfPI entity is a critical consideration, and he suggested that a "full-service organization" will require substantial funding support that may not come easily from the public sector.
The Board expressed enthusiasm for the concept and timing of the NfPI. The Board suggested several additional topics for MSF's further consideration. These included the lack of adequate networks for distributing pharmaceutical drugs in developing countries; ability to access industry's library of compounds; use of selective licensing to encourage industry participation; and development of licensing agreements that would return benefits to the public. An industry representative applauded the initiative as a focused, pragmatic approach to solving identified problems. He encouraged MSF to delineate more clearly the specific issues to be addressed (e.g., development of new drugs, reformulation of drugs, distribution of drugs); the interests and commitments of scientists, host governments, policymakers, and other participants; and the specific issues, funding possibilities, and sustainability of the separate efforts targeting trypanosomiasis and leishmaniasis.
Some members of the Board expressed the need for an initiative focused on prevention, rather than treatment, of disease. It was suggested that, in developing countries, prevention may be more successful than drug development and that directing the amount of time, organization, and funds that will be devoted to the NfPI to prevention would prove to be more worthwhile. The Board strongly encouraged MSF to include the development of vaccines and a focus on children, as well as adults, in the initiative.
In response, Dr. Champey said that the MSF working group has decided not to include vaccines in the NfPI because the activities of drug development (e.g., the science, technology, distribution, and commercialization) are very different from those of vaccine development and because the NfPI has a greater chance of succeeding if it is focused. The initiative also would not specifically focus on children, but would include children burdened with the specific diseases. Dr. Champey also noted that MSF will focus on the transfer of new knowledge to drug development, collaborating with other organizations for the manufacturing and distribution of drugs. MSF also will assess the state of knowledge and the gaps in the science of drug development for neglected diseases as a first step before launching the NfPI. He agreed that a prevention effort is needed, but emphasized that drug development is important and can be "a large part of the answer" to neglected diseases such as leishmaniasis, which was successfully treated with drugs during the 1940s-60s in Africa.
Dr. de Torrente commented on the benefits MSF offers to the NfPI through its experience, focus, and programs. He noted that MSF's extensive experience with trypanosomiasis in Africa includes both prevention (e.g., vector control) and treatment. He also noted that MSF has successfully engaged industry in restarting production of some drugs for sleeping sickness and that MSF will ensure distribution of these drugs through its network of programs worldwide. To mobilize resources for the NfPI, MSF plans to focus attention on patients with neglected diseases and encourage public-sector responsibility for public health.
IX. CLOSED PORTION OF THE MEETING
This portion of the meeting was closed to the public in accordance with the determination that it was concerned with matters exempt from mandatory disclosure under Sections 552b(c)(4) and 552b(c)(6), Title 5, U.S. Code and Section 10(d) of the Federal Advisory Committee Act, as amended (5 U.S.C. Appendix 2).
There was a discussion of procedures and policies regarding voting and confidentiality of application materials, committee discussions, and recommendations. Members absented themselves from the meeting during discussion of and voting on applications from their own institutions or other applications in which there was a potential conflict of interest, real or apparent. Members were asked to sign a statement to this effect.
X. REVIEW OF APPLICATION
A total of 67 scored competing applications were reviewed by the FIC Advisory Board at the May 22, 2001, Board meeting. The applications were in the following programs:
The Board concurred with the initial review group recommendations for the 67 applications.
XI. ADJOURNMENT
The meeting was adjourned at 2:30 p.m. on May 22, 2001.
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