News & Events

Advisory Council Minutes

January 2003 (historical)

Minutes of the 49th meeting
January 23, 2003

Department of Health and Human Services
Public Health Service
National Arthritis and Musculoskeletal and Skin Diseases Advisory Council
8:30 a.m. to 2:00 p.m.

  1. CALL TO ORDER

    The 49th meeting of the National Arthritis and Musculoskeletal and Skin Diseases Advisory Council (hereinafter referred to as the "Council") was held on January 23, 2003, at the National Institutes of Health (NIH) Campus, Building 31, Conference Room 6.


    Attendance

    Council members present
    Mr. Chris Allen
    Dr. Gunnar Andersson
    Dr. John P. Atkinson
    Dr. Paul R. Bergstresser
    Ms. Priscilla Ciccariello
    Dr. Bess Dawson-Hughes
    Dr. Sue K. Donaldson
    Dr. Michael Frank
    Ms. Victoria Kalabokes     		Dr. Cato Laurencin
    Ms. Jean Mandeville
    Dr. Richard Moxley
    Dr. Robert Oglesby
    Dr. Francesco Ramirez
    Ms. Mary Elizabeth Replogle
    Dr. Dennis R. Roop
    Dr. Linda J. Sandell
    Dr. Charles S. Via
    Chairman

    Dr. Stephen I. Katz

    Executive Secretary

    Dr. Cheryl A. Kitt

    Staff and Guests

    The following National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) staff and guests attended:

    Staff
    Dr. Deborah Ader
    Dr. Janet Austin
    Dr. Julia B. Freeman
    Dr. Elizabeth Gretz
    Dr. Steven J. Hausman
    Ms. Jane Hymiller
    Ms. Margaret Kerza-Kwiatecki
    Dr. Charisee Lamar
    Dr. Gayle Lester
    Ms. Anita Linde
    Dr. Peter Lipsky     		Dr. Richard Lymn
    Dr. Joan A. McGowan
    Dr. Alan Moshell
    Dr. James Panagis
    Ms. Wilma Peterman
    Dr. Susana A. Serrate-Sztein
    Dr. William J. Sharrock
    Ms. Helen Simon
    Ms. Robyn Strachan
    Dr. Bernadette Tyree
    Guests

    Dr. Lee Simon
    Dr. Fei Wang
    Ms. Mary Woolley

    In addition, other NIAMS staff members and guests were present for portions of the meeting. Dr. Katz, Director of NIAMS, chaired the meeting.

  2. CONSIDERATION OF THE MINUTES

    The minutes of the 48th Council meeting, held in September 2002, were accepted.

  3. FUTURE COUNCIL DATES

    Future Council meetings are planned for the following months:

    May 22, 2003
    September 25, 2003
    January 29, 2004
    June 3, 2004
    September 20, 2004

  4. DIRECTOR'S REPORT AND DISCUSSION

    Dr. Katz reported the highlights of recent NIAMS activities. He noted that more detailed updates for several activities are posted on the NIAMS Web Site as "NIAMShorttakes," including: (1) trans-NIH and NIAMS initiatives and (2) NIH Director Elias Zerhouni's "Roadmap Initiative".

    Changes in Council Members

    Outgoing members of the Council include Ms. Ciccariello and Drs. Donaldson, Liang, Roop, and Sandell. These outgoing members attended this meeting because new Council members have been approved but not yet appointed.

    Personnel Changes

    Dr. Katz announced two personnel changes within NIAMS. Dr. Fei Wang, formerly of the National Heart, Lung, and Blood Institute, will serve as the new Muscle Biophysics and Cell Biology Program Director. Ms. Margaret Kerza-Kwiatecki, Associate Director for Management and Operations, will retire on April 3, 2003.

    Update on the Budget and Congressional Activity

    Dr. Katz announced that NIAMS' funding success rate for Fiscal Year (FY) 2002 was slightly more than 23 percent. The budget table and plans, as well as interim paylines, are posted on the NIAMS Web Site.

    NIH-Wide Activities

    Dr. Katz provided updates on two NIH-wide activities. The first, Dr. Zerhouni's 5-Year Roadmap Initiative, is under development. For this Initiative, White Papers are being prepared to identify areas in which research is needed. Topics addressed by these white papers include: (1) new pathways to discovery, (2) multidisciplinary research teams of the future, and (3) reengineering the clinical research enterprise. On January 30, 2003, Dr. Katz was to meet with Dr. Zerhouni and many extramural advisors and other Institute Directors to discuss the needs for the reorganization of the clinical research enterprise. Dr. Zerhouni plans to present information from the Roadmap Initiative planning activities at upcoming Congressional hearings.

    The second NIH-wide activity discussed by Dr. Katz was the extramural Loan Repayment Program. In 2002, 964 applications were submitted to the four programs within the Loan Repayment Program, 714 of which were paid. As of 2003, applicants are no longer required to have NIH support, but they must receive federal or nonprofit organizational support. Applications for 2003 were due January 31.

    More students and young investigators in the field of biomedical research need to be made aware of the Loan Repayment Program. To increase awareness of this Program, the American College of Rheumatology sent letters to all participants in its training programs inviting them to apply. Dr. Katz made a plea for other organizations to do the same.

    Recent Scientific Activities

    The U.S. Food and Drug Administration (FDA) recently suspended 27 gene therapy trials worldwide (about one-half were conducted in the United States) because of cases of leukemia reported among patients receiving gene-based therapies.

    The first report of the Lower Extremity Assessment Project (LEAP) was published in the New England Journal of Medicine in late 2002. The objectives of this study were to compare clinical and functional outcomes of patients undergoing amputation versus reconstruction, to identify early clinical predictors of successful limb salvage and good functional outcomes, and to identify characteristics of the patients' environment that affect these outcomes. To date, few differences have been found between outcomes for the amputation group and outcomes for the reconstruction group. About one-half of patients in each group returned to work within 2 years of surgery. Neither the severity of injury nor the presence of other injuries in the same or other leg significantly impacted outcomes.

    Another NIAMS-supported study found that social experience influences joint replacement decisions. Dr. Mary Charlson and colleagues at Cornell University surveyed African-American and white joint replacement surgery patients. Forty-two percent of African-American respondents and 65 percent of white respondents indicated that they knew someone who had this type of surgery for pain. Lack of personal contact with individuals who have had this type of surgery may contribute to underutilization of joint replacement surgery by African Americans.

    NIAMS-supported research has found that a large numbers of individuals suffering from facioscapulohumeral muscular dystrophy have a defect in chromosomal allele 4QA. Other NIAMS-supported research led to the development of an approach to reversing muscle degeneration in a mouse model of Duchenne muscular dystrophy. This approach involves the use of a vector with low immunogenicity to introduce the missing dystrophin gene into diseased muscle tissue. In addition, the NIAMS, the National Institute of Child Health and Human Development, and the National Institute of Neurological Disorders and Stroke Muscular Dystrophy (MD) Task Force recently held a meeting that focused on translational opportunities in MD research.

    Other highlights of NIAMS-supported research include:

    • Dr. Michael Brenner and colleagues at Harvard University recently published a study of mast cells in arthritic joints.


    • Dr. Richard Gallo and colleagues recently published results of a study of beta defensins in atopic dermatitis in the New England Journal of Medicine. Low levels of beta defensins were found in skin affected by atopic dermatitis compared to skin affected by psoriasis. This finding suggests that bacterial infection is involved in atopic dermatitis.


    • Melanoma researchers have identified a master regulator molecule (MITF) that controls BCL2. The BCL2 molecule interferes with apoptosis of melanocytes.


    Intramural Research at NIAMS

    NIAMS intramural researchers have determined that high doses of an extract of the root of the thunder god vine may relieve symptoms of rheumatoid arthritis (RA) with minor side effects. Larger, long-term trials are needed to confirm this finding.

    Intramural researchers collaborating with researchers at Brown University have found that Type 1 interferons (alpha and beta) stimulate production of infection-fighting Type 2 interferon. This process is believed to occur through the STAT 4 signal protein.

    Recent NIAMS Activities and Plans for the Future

    The United States and Canada are cooperating on a study of fracture repair techniques. The goal of this study is to determine the best approach for repairing fractures of the tibia (the most common long-bone fracture). Nine hundred patients will participate in this study through various centers in the United States, Canada, and one center in Europe. The main research question is whether or not to ream out bone marrow when a pin is placed in the bone.

    Twenty centers from the Pediatric Rheumatology Research Network will enroll children with recent onset lupus in a study of the efficacy of statins (cholesterol-lowering agents) in delaying progression of cardiovascular disease in these children, to prevent cardiovascular disease.

    NIAMS recently funded: (1) 5 new neuropsychiatric lupus studies, (2) 8 studies of heritable disorders in connective tissue, and (3) 5 new studies of the use of adult stem cells to treat diseased bone and muscle tissue. An intramural center supported by multiple Institutes is being established to characterize approved stem cell lines and train investigators to conduct stem cell research.

    NIAMS is organizing roundtable discussions to prepare for its annual planning retreat in April 2003. The roundtable discussions will focus on clinical research in rheumatic, , skin, bone, and musculoskeletal diseases. Proposed initiatives generated by these meetings will be discussed at the Council Meeting in September 2003.

    Discussion

    Dr. Frank noted that the Loan Repayment Program should target students before they become fellows. Currently, there are too few students taking board exams for pediatrics and orthopaedic surgery. The Loan Repayment Program may encourage more students to enter these fields. Innovative research developments also may help attract students to fields experiencing shortages, as has occurred in the field of rheumatology. Dr. Frank indicated that the American Board of Pediatrics has a system for tracking medical students. He agreed to ask the President of the American Board of Pediatrics about using this system to track participation in the NIH Loan Repayment Program.

    Dr. Katz indicated that the NIH plans to review the application rate for the Loan Repayment Program in the near future, particularly for fields in which there is a shortage of researchers. Dr. Moxley recommended developing a 5-year plan to evaluate approaches for promoting the Loan Repayment Program. This evaluation should identify the best times during the education process to communicate about this Program, and the key individuals at universities and other locations who can reach potential candidates for this Program. An NIH committee will meet to define indicators of success for the Loan Repayment Program, and approaches for tracking these indicators.

  5. BRINGING RESEARCH CLOSER TO HOME: THE IMPORTANCE OF STRONG PUBLIC OUTREACH FOR HEALTH RESEARCH

    Ms. Mary Woolley, President of Research!America, discussed her organization's goals and activities, as well as opportunities for collaboration with NIAMS. Research!America is a nonprofit advocacy organization committed to making medical/health research a national priority, and to increasing medical research funding at a rate consistent with scientific opportunity. Several scientific and legislative leaders serve on the Research!America Board. Currently, 450 organizations are Research!America members, including the Arthritis Foundation, Lupus Foundation, National Alopecia Areata Foundation, National Osteoporosis Foundation, and the Scleroderma Research Foundation.

    Research!America assists member organizations in: (1) developing strategies for public outreach, (2) assessing public knowledge and awareness about research, (3) measuring and identifying barriers to public support of research, (4) gauging support for specific policy measures and legislation through public opinion polls, and (5) identifying compelling messages and messengers.

    Research!America tracks public awareness of, and support for, medical/health research through opinion polls conducted across states. Since 1992, Research!America has conducted opinion polls in most of the 50 states, and expects to poll all 50 states by the end of 2003. Findings of the polls, to date, suggest strong support for medical/health research within states, with little variability between states. Research!America has used these findings to develop a case for doubling national spending on government-sponsored scientific research over 5 years (61 percent of poll respondents support this proposal). Opinion polls conducted by this organization further indicate that 78 percent of respondents believe that it is very important for the United States to remain the world leader in medical research. Ms. Woolley added that focus group research suggests that the leadership of the United States in the field of medical research is important to its citizens because it increases their access to state-of-the-art care. Survey respondents also believe that medical research is very important to their state's economy. Research!America polls also found that: (1) 68 percent of respondents nationwide believe that the elimination of health disparities is very important, (2) 61 percent support therapeutic cloning but only 17 percent support reproductive cloning, and (3) 80 percent support responsible use of animals in research necessary for progress. In addition, a recent national survey, conducted by Research!America, found that 89 percent of respondents believe that biomedical research is extremely important in preparing for biological and chemical terrorism, and 83 percent are prepared to pay for this research through taxes. Another poll conducted during election time found that medical research was the second highest priority among voters, after education (and more important than creation of jobs and homeland security).

    Polls also have identified a lack of knowledge about medical research among the public. The majority of respondents to most state polls cannot name a medical research facility in their area. Only 2 percent of respondents knew that the NIH is the government agency that funds most medical research in the United States. Knowledge about the National Science Foundation (NSF) also was low in this poll. Ms. Woolley encouraged increased NIH/NSF collaboration to reduce these public knowledge deficits. Knowledge about scientific awards also was low, with only 47 percent of respondents indicating that they knew about the Nobel Prize (52 percent for those with college degrees), 23 percent indicating knowledge of the Intel Award, and 2 percent knowing about the Lasker Award.

    Low public awareness of scientific issues is due, in part, to the lack of coverage of these issues in the media. For example, an analysis of op-ed articles in major national newspapers (pre-9/11) revealed that only 3 percent of these articles dealt with health issues and 1 percent dealt with science and technology issues. Members of the media indicate that few health/science op-eds articles are submitted, which suggests a lack of public interest in these topics. In addition, polls indicate that 74 percent of scientists do not engage in public outreach. Research!America surveys found that scientists do not engage in public outreach because: (1) they do not have time (49 percent), and (2) they think outreach makes no difference (41 percent). Only 14 percent of scientists, however, responded that they believe other parties are doing an adequate job of informing the public about science. Research!America is promoting increased interaction of the scientific community with the media through its 435 Project: Bringing Research Closer to Home. Some activities of this Project are media/science roundtables, leadership forums, and advocacy workshops for scientists. Ms. Woolley suggested several ways that scientists can promote research to the public, including: (1) remaining accessible and accountable; (2) discussing research with others directly and via the Internet; (3) communicating with policymakers through invitations to visit research facilities and "thank-you" letters for grant awards; (4) engaging with responsible critics by participating in town meetings and making media appearances; (5) proactively developing partnerships with voluntary health organizations; and (6) encouraging leaders of science-based organizations to model, expect, train for, recognize, and reward effective public outreach on the part of scientists.

    Research!America also is working to increase federal spending on medical research in the United States. For example, Research!America has collaborated with the NIH for several years and established a lobbying group focused on doubling the NIH budget over 5 years. Currently, less than 6 cents of every health care dollar is spent on medical research (1 cent on preventive research), and most of these funds come from private organizations rather than tax dollars. Research!America also produces a series of 1-page information sheets, entitled "Investment in Research Saves Lives and Money" for policymakers, members of the media, and the public. These sheets provide information on the impact of research funding in states and communities, and on key national resources. Research!America is beginning to develop information sheets that address diseases under the purview of NIAMS and, as part of this development process, will be seeking input from NIAMS staff.

    Discussion

    Dr. Katz noted that General Norman Schwartzkopff recently wrote an article for the Washington Times about waging war against illness. Ms. Woolley added that, nationwide, the number of newspaper articles about health and science are decreasing in number.

    Dr. Moxley indicated that scientists may hesitate to submit letters or articles to newspapers for fear of being misrepresented or appearing to promote their own research. Leaders of institutions involved in medical research need to help overcome this culture of reticence by supporting young scientists who want to become involved in public outreach. Scientists also need training in conducting public outreach activities. Ms. Woolley is encouraging voluntary health organizations that provide grants to make public outreach a requirement of funding. Research!America can train these grantees to conduct public outreach in a cost-effective manner.

    Dr. Ramirez added that public statements about ongoing research sometimes unduly raise the public's hopes. When the research does not deliver what the public expects in a short timeframe, it is seen as a failure.

    Dr. Sandell questioned whether patients' interests in health information (e.g., information they collect through Web searches) might provide an outreach opportunity for researchers. Ms. Woolley responded that this is an outreach opportunity if the research community develops appropriate approaches for reaching these patients, who may be overwhelmed, anxious, skeptical, or suspicious. She added that researchers must consider word usage (e.g., "teaching hospitals" is more attractive to the public than "academic centers").

    Dr. Katz recommended inviting Ms. Woolley to another Advisory Council meeting to discuss Research!America's surveys of clinical trial participation.

  6. FDA ACTIVITIES IN RHEUMATIC DISEASE AND PAIN

    Dr. Lee Simon of the FDA Center for Drug Evaluation and Research (CDER) discussed activities of this Center and new drug developments to treat arthritis, and musculoskeletal and skin disorders. CDER monitors drug development and testing, including adverse events, during the drug approval and labeling process. CDER does not conduct clinical studies, set drug prices, evaluate pharmacogenic impact studies, select products to study, or force companies to market drugs. Several divisions within CDER focus on treatments of interest to NIAMS, including analgesic, anti-inflammatory, and ophthalmic drug products; devices; biologics; skin treatments; dental therapies; (anesthetics and critical care); cardiovascular therapies; kidney therapies; and imaging.

    The average time it takes to develop a new drug is 9 years. After development, it takes an average of 14 months to approve a drug, and only 6 months to approve a high-priority, life-saving drug. Dr. Simon described CDER's Anti-Inflammatory, Analgesic, and Ophthalmic Drug Products approval process. This process involves review of investigational new drug applications; evaluation of pivotal and supporting trials examining drug benefits, and treatment safety issues; continued review of drug safety reports after the drug is approved and marketed; development of drug labels; consultation with other divisions, centers, and regulatory bodies; and interaction with stakeholders (e.g., responding to petitions and queries). CDER also develops guidance for therapeutic research and development.

    The development of guidances may involve collaboration with multiple voluntary organizations and the NIH, formation of a steering committee of both lay and medical experts, and meetings to define state-of-the-art treatment. Guidance for analgesic development was completed in 1992, guidance for rheumatoid arthritis (RA) therapy development was completed in 1998, a draft of guidance for osteoarthritis (OA) therapies was completed in 1999, and an internal draft of guidance for systemic lupus erythematosus therapies is in its third iteration. Guidance for the development of therapies for other conditions also is being developed. CDER is considering whether functional assessment should be a requirement for RA therapy development. Functional assessment would involve an evaluation of patient symptoms, progression of joint damage revealed in x-rays, and disability. CDER also is considering whether guidance for OA therapy development should require an a priori improvement in symptoms before structural improvement (as revealed in x-rays) is evaluated. CDER has decided to include patient-reported outcomes and measures of function and neuropathy in guidance for pain therapies.

    Dr. Simon described the expertise and activities of individuals involved in the approval of Humira, a new RA treatment. Activities included data mining, ascertaining a pain scale, determining the correlation between pain and function, and evaluating diverse safety signals observed during clinical trials. For example, the mining and examination of safety data indicated that both Humira and methotrexate may increase risk for Non-Hodgkin's lymphoma and autoimmune reactions. Data also revealed that Humira treatment alone may cause immunogenic reactions in some patients, but this risk is reduced with concomitant chemotherapies. Humira will not be covered by Medicare, but the developers of this drug may make it available free-of-cost to some high-risk patients.

    CDER is planning the following activities:

    • An ophthalmology drug development clinical trial fellowship in cooperation with George Washington University. CDER is investigating the possibility of the NIAMS participating in this fellowship program by providing clinical rheumatology training.

    • A research plan for clinical trials and pain that focuses on patient-reported outcomes, in collaboration with National Institute of Dental and Craniofacial Research.

    • A merging of CDER and the Center for Biologic Evaluation Research (CBER).

    • A review and comparison of existing databases of treatment safety information. More therapeutic safety data need to be collected (e.g., data on skin cancer incidence in RA patients, longitudinal data) and existing data need to be improved and augmented.

    Discussion

    In response to participant inquiries, Dr. Simon indicated that, until recently, CDER did not have access to enough quality data on untreated RA patients to examine the independent contributions of RA and RA treatments to the development of lymphoma. Participants noted, however, that a large Swedish study of RA patients revealed a very high incidence of lymphoma (particularly among patients with the most active disease) that was independent of drug therapy. Recent CDER analyses of a large database of approximately 8,000 RA patients suggest that RA therapies contribute to the development of lymphoma in some patients. Dr. Simon indicated that this database was superior because it was produced by a controlled trial and covered a large, genetically diverse population that was followed over time with different therapies. More databases of this quality need to be developed and made available to CDER.

    A representative from the Center for Medicare and Medicaid Services (CMS) indicated that CMS wants to ensure that future prescribing practices are based on clinical benefit as well as cost. CMS examines reasonable, necessary, inpatient access to treatments. Congress has presented bills that propose CMS reimbursement of outpatient treatments.

    Dr. Atkinson asked about the degree to which FDA staff can publicize data collected in treatment trials. Dr. Simon replied that, in general, the FDA can publicize only published information. FDA staff cannot publish independently because of proprietary concerns. FDA guidances, however, may include the results of proprietary data analyses. Drug developers also may agree to publish results of their analyses, or the FDA may obtain permission from drug developers to publish results of proprietary data analyses. A new initiative at the FDA encourages this last option. Once the FDA publishes results of a data analysis, the data may be made available to investigators outside the FDA in an anonymous format. Investigators may obtain these data through the Freedom of Information Act process, which may take up to 8 months.

    Mr. Allen discussed the possibility of pharmacoeconomics driving drug prices in the United States, as they do in other countries. Dr. Simon indicated that pharmacoeconomics is not yet a well-developed field. Countries with socialized medicine may use pharmacoeconomics to control access to, or use of, certain treatments.

    In response to an inquiry by Dr. Andersson, Dr. Simon indicated that no orthopaedic surgeons currently serve on the Arthritis Advisory Committee for CDER, but they have served in the past. Several orthopaedists, however, serve on CDER's Devices Advisory Committee and other Advisory Committees. The Arthritis Advisory Committee does not address tumor necrosis factor alpha inhibitors, but often invites internists and other specialists to address questions about nonsteroidal anti-inflammatory drugs (NSAIDs).

    Dr. Frank asked whether trials have been conducted to compare similar therapies developed by different companies. The large NIH tamoxifen/raloxifene trial is one example of this type of trial. The NIAMS also is conducting trials of combination therapies that would not be conducted by industry. FDA can recommend that pharmaceutical companies conduct comparative trials (e.g., comparison of COX-2 inhibitors with NSAIDS).

    Dr. Dawson-Hughes raised the issue of placebo use during clinical treatment trials. In most of these trials, the current standard treatment rather than a placebo must be used in the control group. In the case of clinical trials for the treatment of chronic disease, placebo may be used for short periods of time (no more than 4 months).

  7. REVIEW OF MEMORANDUM OF UNDERSTANDING

    Dr. Hausman reviewed the various sections of this MOU with the Council. According to this MOU, Council members can review and approve funding applications prior to a Council meeting. The Council approved the Memorandum of Understanding (MOU) for the Council's operating procedures.

  8. NIAMS PARTICIPATION IN TRANS-NIH GENETICS/GENOMICS INITIATIVES

    Dr. William Sharrock provided an overview of the various trans-NIH initiatives that address genetics/genomics. Most information about these initiatives is available on the NIH Web Site. Dr. Sharrock noted that most genetics/genomics efforts are large, expensive, and not investigator-initiated. Most NIH Institutes, including NIAMS, now are collaborating on initiatives that build on the Genome Project and develop genomic technology and public resources.

    The sequences for the genomes of humans, mice, and other simpler organisms are complete and publicly accessible; and scientists are working on the identification of sequences for the genomes of other animals. Mouse and human genomes can be compared. This capability needs to be further developed and utilized in future studies to identify the biologically important components of the genome.

    The following trans-NIH genetics/genomics activities are or may be supported by NIAMS:

    • Annotating the genome (determining which parts of the genome are biologically important and what their role is), including:

      • Identifying protein coding regions with the: (1) development of bioinformatics, (2) expansion of the Unigene database, (3) development of the Mammalian Gene Collection (MGC) that will include complete copies of each cDNA that can be isolated. The MGC has clones for more than 10,000 human genes and 6,000 mouse genes.

      • Identifying the regulatory regions of the genome and other functional elements (that turn the gene "on" and "off").

      • Attributing biological functions to features identified in the genome.

    • Developing tissue-specific libraries (e.g., of mouse limbs), which are needed to develop custom microarrays.

    • The evolutionary conservation gambit of comparative genomics, which has identified noncoding RNAs.

    • Recovering mutations (altering a region of the genome to determine the consequences). For example, several mutant mice have been developed as part of a study of developmental defects. Many of these mutant mice have skeletal system defects. A Request for Applications also is being developed to support insertional mutagenesis research.

    • Developing in vitro expression vectors to synthesize proteins produced by specific genes.

    • Developing resources for the study of single nucleotide polymorphisms (SNPs), including the:

      • SNP Consortium

      • Haplotype map to examine the clustering patterns, in which specific polymorphisms are inherited in human populations.

    • Developing inbred strains of laboratory mice for systematic phenotyping and isolation of the gene loci that influence specific phenotypes.

    • Developing genomic tools.

    Some challenges to progress in the field of genetics/genomics for the prevention and treatment of disease include: (1) developing resources with centralized management, including a large clinical cohort to address complex disorders; (2) regulatory, ethical, legal, and social concerns about patient privacy and stigmatization of individuals and populations; (3) forming real partnerships between the National Human Genome Research Institute (NHGRI) and other Institutes; and (4) linking genotype to phenotype in humans.

    Discussion

    In response to participant inquires, Dr. Sharrock indicated that all MGC clones are available through the Image Consortium. Both individual clones and collections of clones are available through contracted suppliers.

    Ms. Ciccariello asked about opportunities for the NIH to collaborate with voluntary organizations to provide genetic/genomic information to policymakers and the public. Dr. Sharrock indicated that this information is available on various NIH and other Web sites, and in articles in scientific journals. The Genome Web Site, for example, has a public information section. A central repository of information on genetic/genomic activities has not been developed for the general public. Dr. Sharrock indicated that the NHGRI is interested in outreach to the general public. He recommended that Ms. Ciccariello contact the NHGRI to discuss a possible collaboration with voluntary health organizations to develop a central repository of genetic/genomic information. The NIH Center for Bioinformatics should be involved in this effort. The Genetics Alliance and the National Organization of Rare Disorders also would be valuable partners for this effort. The National Coalition for Health Professional Education and Genetics (NCHPEG) may be another important partner. Dr. Donaldson recommended that voluntary health organizations interested in developing a central repository of genetics/genomics information join this organization, which has a Web Site at www.nchpeg.org. The NCHPEG currently is evaluating the accuracy of various educational materials that discuss genetics.

  9. BIENNIAL REPORT ON NIAMS PROGRESS IN MONITORING WOMEN AND MINORITY ISSUES IN GRANT APPLICATIONS

    Dr. Lamar of the NIAMS Women's Health and Health Disparities Program distributed and discussed an FY 2002 Report of NIAMS Progress in Monitoring Women and Minority Issues in Grant Applications. This report addresses NIAMS compliance with the NIH requirement for inclusion of women and minorities in clinical research. Advisory Council members approved this report.

    In FY 2001, NIAMS funded 181 new studies that included human subjects. Twelve of these awards needed to resolve problems with inclusion of women and minorities. Eight of these awards had to resolve both gender and minority inclusion issues, and four needed to resolve issues related to minority inclusion only. In FY 2002, NIAMS funded 167 new studies with human subjects. Only 4 of these awards had to resolve problems with inclusion of women and minorities.

    NIAMS currently funds 1,290 competing and noncompeting awards, of which 761 involve human subjects. The NIAMS population tracking system for clinical research includes 143 protocols involving approximately 44,000 enrolled subjects. Five of these protocols are Phase III clinical trials. Sixty percent of the enrolled subjects are women because of the large proportion of women affected by the diseases addressed by NIAMS. In the FY 2003 protocols, 85 percent of enrolled subjects are white, 6.5 percent are African American, 6 percent are of unknown race/ethnicity, 2.5 percent are Hispanic, 1 percent are Asian, 1 percent are multiple races/ethnicities, and less than 1 percent are American Indian/Alaskan Native, Hawaiian, or Pacific Islanders.

  10. CONSIDERATION OF APPLICATIONS

    The Council reviewed a total of 571 applications in closed session requesting $115,075,745 and recommended for $94,239,668.

I hereby certify that, to the best of my knowledge, the foregoing summary and attachments are accurate and complete.

Cheryl A. Kitt, Ph.D.
Executive Secretary, National Arthritis
and Musculoskeletal and Skin Diseases
Advisory Council

Director, Extramural Program
National Institute of Arthritis and
Musculoskeletal and Skin Diseases

Stephen I. Katz, M.D., Ph.D.
Chairman, National Arthritis
and Musculoskeletal and Skin
Diseases Advisory Council

Director, National Institute of
Arthritis and Musculoskeletal and
Skin Diseases