Department of Health and Human Services
Public Health Service
National Arthritis and Musculoskeletal and Skin Diseases Advisory Council

Minutes of the 81st Meeting
8:30 a.m. to 3:10 p.m.

UPDATE ON THE HEALTH CARE SYSTEM COLLABORATORY: A COMMON FUND PROGRAM

Dr. Josephine Briggs, Director, National Center for Complementary and Alternative Medicine, provided an update on the Health Care System Collaboratory, noting that this Common Fund program’s goal is to strengthen the national capacity to implement cost-effective large-scale research studies that engage health care delivery organizations as research partners. Specific goals of the program are to:

This initiative is focusing on pragmatic trials, which differ in substantial ways from the classic NIH-sponsored efficacy trial in that the questions are tested in real-world settings, not in the tightly controlled classic structure used for efficacy-based trials. The aim is to identify questions to be tested that are simple enough so as not to require a complex structure for their implementation. These trials will help to develop monitoring outcome assessments that will utilize electronic health records and will not forgo the rigor of a trial that comes from randomization.

The first set of projects is now ending the feasibility phase and will hopefully be moving into the implementation phase. Dr. Briggs then described the following demonstration projects:

Dr. Briggs commented that these trials hold the potential for changing patient care and are being implemented with more modest budgets than are frequently required for projects of this scale. The solicitation for the second round of projects is open.

This type of research poses a number of challenges. These include waiver of consent and related ethical and regulatory questions, optimizing trial designs for group or cluster randomized trials, preserving effective public-private partnerships and addressing data-sharing issues, and ensuring synergism between NIH and Patient-Centered Outcomes Research Institute (PCORI) processes (this project was developed in close collaboration with PCORI leadership, and its related studies likely will be similar to some of the projects PCORI will be undertaking).

Discussion

Dr. Gabriel, who recently led the Methodological Core at PCORI, explained that it is critical that PCORI and the Collaboratory together launch some of these investigations in the context of health care delivery systems to address important issues related to clinical research. She noted that PCORI has been facing similar challenges to those outlined by Dr. Briggs. Dr. Gabriel commented that there is great value in these types of collaborations and indicated that PCORI would be interested in working even more closely with the Collaboratory to share best practices and approaches. Dr. Briggs noted that the Collaboratory’s Coordinating Center is developing a website that will include position papers on the ethical and regulatory issues associated with each of the projects, models, and information on lessons learned. Information on extracting reliable data from electronic health records will also be posted online.

In response to a question from Dr. Gabriel about the need for pragmatic trials, Dr. Briggs commented that some of the ways in which the NIH develops disease-specific expertise will be well served by continuing to invest in real-world effective research. She added that the Health Maintenance Organization Research Network, which includes roughly two-thirds of the health care systems participating in the Health Care System Collaboratory, covers approximately 13 million individuals.

Dr. McGowan informed the Council that the NIH (the National Institute on Aging, in particular) and PCORI have been collaborating on an initiative focusing on falls and frailty in the elderly. Dr. Briggs noted that a similar collaboration on back pain is forming, and that there is great optimism that the NIH can work synergistically with the investments made in PCORI.

1. CALL TO ORDER

   The 81st meeting of the National Arthritis and Musculoskeletal and Skin Diseases Advisory Council was held on September 10, 2013, at the National Institutes of Health (NIH) Campus, Building 31, Conference Room 6. The meeting was chaired by Dr. Stephen Katz, Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).

   Attendance

   Council members present

   Dr. Lynda F. Bonewald
   Dr. Harry C. Dietz
   Dr. David R. Eyre
   Dr. Gary S. Firestein
   Dr. Sherine E. Gabriel
   Ms. Michelle Hofhine
   Dr. Ted Mala
   Dr. Katherine Mathews
   Dr. Martha M. Murray
   Dr. Regis J. O’Keefe
   Dr. Alice P. Pentland
   Dr. Edward A. Rankin
   Dr. Elizabeth Shane
   Ms. Elizabeth Smith
   Mr. Bradley R. Stephenson
   Dr. Julio L. Vergara
   Dr. Xiao-Jing Wang

   Staff and Guests

   The following NIAMS staff and guests attended:

   Staff

   Ms. Alexandra Adams Mr. Steven Austin Dr. Carl Baker Ms. Susan Bettendorf Ms. Elizabeth Bouras Dr. Amanda Boyce Mr. Gahan Breithaupt Ms. Justine Buschman Dr. Robert Carter Dr. Faye Chen Dr. Ricardo Cibotti Mr. Richard Clark Ms. Stephanie Craver Ms. Theresa Do Dr. Jonelle Drugan Mr. Erik Edgerton Ms. Elizabeth Elliott Ms. Barbara Footer Dr. Nancy Garrick Ms. Valerie Green Ms. Gail Hamilton Dr. Chao Jiang Mr. Andrew Jones Dr. Stephen Katz Ms. Mary Beth Kester Ms. Stephanie Kreider Ms. Colleen Labbe Mr. Mark Langer Dr. Gayle Lester Ms. Anita Linde Ms. Mimi Lising Ms. Leslie Littlejohn Dr. Kan Ma Dr. Su-Yau Mao Dr. Marie Mancini

   Dr. Kathryn Marron Ms. Regina Mong Dr. Joan McGowan Ms. Leslie McIntire Dr. Laura K. Moen Ms. Clairisse Mullsteff Ms. Melinda Nelson Ms. Anna Nicholson Dr. Glen Nuckolls Dr. James Panagis Ms. Vivian Pham Dr. Charles Rafferty Ms. Reaya Reuss Ms. Trish Reynolds Ms. Kathy Salaita Dr. Susana Serrate-Sztein Dr. William Sharrock Ms. Sheila Simmons Ms. Theresa Smith Ms. Allisen Stewart Ms. Robyn Strachan Ms. Yen Thach Ms. Jamie Thompson Dr. Phil Tonkins Dr. Hung Tseng Dr. Antonella Nadia Tullio Dr. Bernadette Tyree Dr. Fei Wang Dr. Xibin Wang Dr. Yan Wang Dr. Chuck Washabaugh Ms. Sara Rosario Wilson Dr. James Witter Dr. David Zielinski Dr. Xincheng Zheng

   Guests

   Ms. Kimberly Beer, Arthritis Foundation
   Dr. Josephine Briggs, National Center for Complementary and Alternative Medicine, NIH
   Mr. Michael Bykowski, Consolidated Solutions and Innovations
   Ms. Kim Cantor, Lupus Foundation of America
   Dr. Janine Clayton, Office of Research on Women’s Health, NIH
   Dr. Alison Davis, National Institute of General Medical Sciences, NIH
   Mr. Dale Dirks, Health and Medicine Counsel of Washington
   Ms. Judy Dulovich, Office of the Director, NIH
   Ms. Rhonda Edwards, Office of the Director, NIH
   Ms. Dana Ferrari, American Assoication of Colleges of Osteopathic Medicine
   Dr. Seymour Garte, Center for Scientific Review, NIH
   Dr. Mark Guyer, National Human Genome Research Institute, NIH
   Ms. Patti Brandt Hansberger, Office of Legislative Policy and Analysis, NIH
   Ms. Petra Harvey, Osteogenesis Imperfecta Foundation
   Ms. Kim Holmes, IQ Solutions
   Ms. Leah Howard, National Psoriasis Foundation
   Dr. Rajiv Kumar, Center for Scientific Review, NIH
   Dr. Rebecca Minnillo, Society for Investigative Dermatology
   Mr. Simit Pandya, American Association of Orthopaedic Surgeons
   Ms. Michelle Rodrigues, SRI International
   Dr. Kirstie Saltsman, IQ Solutions
   Dr. Lawrence Tabak, Office of the Director, NIH
   Mr. Richie Taffet, Ehlers-Danlos National Foundation
   Ms. Esther Weiss, Office of the Director, NIH

2. CONSIDERATION OF MINUTES

   A motion was made, seconded, and passed to accept with no changes the minutes of the 80th NIAMS Advisory Council meeting, held on June 4, 2013.

3. FUTURE COUNCIL MEETING DATES

   Future Council meetings are currently planned for the following dates:

   February 11, 2014
   June 3, 2014
   September 8, 2014
   February 4, 2015
   June 16, 2015
   September 8, 2015

4. DIRECTOR'S REPORT AND DISCUSSION

   Dr. Katz opened the meeting by welcoming Council members, NIAMS staff, and guests. He thanked the following Council members, whose 4-year terms were expiring with this meeting:

   • Dr. Hal Dietz, the Victor A. McKusick Professor of Medicine and Genetics at the McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins University School of Medicine.
   • Dr. Regis O’Keefe, Chair of the Department of Orthopaedics and Rehabilitation at the University of Rochester Medical Center.
   • Ms. Jean Pickford, Executive Director at the Foundation for Icthyosis and Related Skin Types.
   • Mr. Bradley Stephenson, an Attorney-at-Law and Member of the Muscular Dystrophy Association’s National Task Force on Public Awareness.
   • Dr. Julio Vergara, Distinguished Professor in the Department of Physiology at the University of California, Los Angeles, School of Medicine.

   Dr. Katz noted that the Institute is grateful to each of the outgoing Council members for their exemplary service and many contributions to the group’s deliberations. Ms. Pickford was unable to attend this Council meeting, and sent a note indicating that it has been a privilege and honor to serve on the Council and expressing confidence that the patients who benefit from NIAMS’ efforts are in good hands.

   Personnel Changes at the NIH and NIAMS

   At the NIH level, Dr. Jon Lorsch has joined the NIH as the new National Institute of General Medical Sciences (NIGMS) Director. He comes to the NIGMS from the Johns Hopkins University School of Medicine, where he was a Professor in the Department of Biophysics and Biophysical Chemistry.

   At the NIAMS level, Dr. James Katz has joined the Institute as the new Director of the Rheumatology Fellowship Training Program (the NIAMS manages this Program for the NIH). Dr. Katz previously served as the Director of the Division of Rheumatology at George Washington University, and will also be leading the NIAMS Community Research and Care Branch. Ms. Reaya Reuss has joined the NIAMS as a Legislative Liaison and Science Policy Analyst in the Office of Science Policy, Planning and Communications (OSPPC). For the past two years, Ms. Reuss’ work has focused on congressional relations, first with the Health Resources and Services Administration’s (HRSA) Office of Legislation, and most recently with the National Institute of Allergy and Infectious Diseases’ (NIAID) Legislative Affairs and Correspondence Management Branch.

   Update on Budget and Congressional Activities

   Dr. Katz reported that the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education, and Related Agencies has marked up and reported on its version of the fiscal year (FY) 2014 Labor-HHS-Education Appropriations bill. The bill would provide $31 billion for the NIH, which is essentially the same as the President’s Budget request. This amount is significantly higher than funding for the current year—the amount proposed by the Senate for the NIAMS is $537 million, which represents an increase of approximately $32 million over the FY 2013 level. At the time of this Council meeting, the FY 2014 House markup has been postponed. It is expected that the NIH will begin FY 2014 under a continuing resolution.

   Despite the current fiscal picture, the NIAMS must continue to identify research areas or activities that are ripe for investment but are not being pursued through the regular investigatorinitiated grant process. Dr. Katz noted that this Council meeting would feature a discussion on initiatives that the Institute would like to put forth for consideration in FY 2015 to promote research in arthritis and musculoskeletal and skin diseases.

   Dr. Katz reminded Council members that the NIAMS receives many more high-quality investigator-initiated grant applications than it could possibly fund. Applicants with scores that would have been paid automatically in previous years are now missing the payline and need to resubmit their applications. Several professional societies are exploring ways to provide shortterm funding to keep laboratories open until they can re-apply for longer-term support. For example, last month the American Society for Bone and Mineral Research (ASBMR) pledged $1 million in research funding for a new Grants in Aid Program (GAP). The ASBMR expects to support 20 members who have submitted a peer-reviewed grant application to a major funding sponsor within the last 15 months and received a full review but were not funded. The American Academy of Dermatology is exploring a similar initiative, and the Rheumatology Research Foundation has an ongoing program for investigators who are applying to the NIH for mentored patient-oriented or clinical career development awards.

   Highlights of Selected Recent Scientific Advances

   • Dr. Mayumi Ito and colleagues at the New York University School of Medicine reported that Wnt proteins play a role in fingernail growth and in digit regeneration. Although many lizards and other amphibians can regenerate lost digits and even entire limbs, this ability in mammals is limited to the very tips of fingers and toes. The work by Dr. Ito and colleagues shows that Wnt activation in the nail epithelium is essential for nail regeneration, bone formation, and the innervations of wounded tissue following amputation. They also explain why regeneration in mammals is restricted to the most distal appendages. The researchers found that when amputation removes the entire nail bed area and its nail-forming stem cells, it also removes tissue that produces the molecule Wntless (which is essential for Wnt activation; in its absence, regeneration does not occur) (Nature. 2013 Jul 11;499(7457):228-32. doi: 10.1038/nature12213. Epub 2013 Jun 12. PMID: 23760480).
   • An international team of investigators, including NIAMS grantee Dr. Daniel Cohn of the University of California, Los Angeles, have found evidence that Wnt1 is one of the ligands involved in bone building. They studied two families with children who had severe low bone mass. In one family, the children had osteogenesis imperfecta; the children in the other family had been diagnosed with early onset osteoporosis. Instead of having mutations that altered type I collagen, the most common cause of osteogenesis imperfecta, the patients carried different mutations in the Wnt1 protein. When the researchers conducted experiments in mice to identify which tissues and cell types produce this molecule, they found that some Wnt1 came from bone-forming osteocytes. However, the highest levels were from bone marrow, specifically from hematopoietic progenitors of the B lymphocyte lineage. This work contributes to the growing body of evidence indicating that hematopoietic cells play an important role in regulating bone formation. It implicates Wnt1 as a key signaling molecule that mediates these effects and further underscores the intricate connections between bone and the hematopoietic and immune systems (N Engl J Med. 2013 May 9;368(19):1809-16. PMID: 23656646).
   • Another advance connecting bone mass and the immune response comes from Dr. Robert Colbert’s group in the Pediatric Translational Research Branch within the NIAMS Intramural Research Program (IRP). These researchers are working to understand the cellular and molecular mechanisms underlying the pathogenesis of spondyloarthritis, which is a spectrum of chronic immune-mediated inflammatory diseases. Defects in the HLA-B27 gene play a critical role in the pathogenesis of many spondyloarthropathies, including ankylosing spondylitis, which is characterized by inflammation and bone loss, especially in the spine. Dr. Colbert’s team has provided evidence that defects in HLAB27 trigger production of the immune system protein IL-1alpha, and that elevated levels of IL-1alpha stimulate production of the bone-degrading cells called osteocytes. This is one of the first studies linking HLA-B27 to altered bone homeostasis (Arthritis Rheum. 2013 Aug;65(8):2123-31. doi: 10.1002/art.38001. PMID: 23666508).
   • To understand the effect of muscle load on the formation of the bone-tendon insertion site, Dr. Stavros Thomopoulos’ team at Washington University in St. Louis studied the connection between a shoulder muscle and the foreleg of young mice. They concluded that muscle paralysis—as would occur during voluntary immobilization due to an acute injury or because of damage to the nerves that control muscle movement—changed the mineral composition and microscopic organization of the tendon-bone junction. This led to a weaker connection where the tendon inserts into the bone. This model may prove to be important for studying neonatal brachial plexus palsy, a condition which occurs when the collection of nerves around the shoulder are damaged during birth (Bone. 2013 Jul;55(1):44-51. doi: 10.1016/j.bone.2013.03.010. Epub 2013 Mar 29. PMID: 2354286)9.
   • Dr. Kurt Spindler of Vanderbilt University and colleagues found that within 6 years of their first surgery, approximately 19 percent of the 900 patients in the Multicenter Orthopedic Outcomes Network (MOON) cohort study underwent at least one additional operation on the knee that had already been repaired, while 10 percent of patients needed surgery on the other knee. Younger patients were more likely to require subsequent surgeries—a finding that may be due to these patients being more active, less likely to comply with post-operative instructions, or having biological differences that predispose them to injury. The rate of repeat surgery also was higher in patients treated with a cadaver (allograft) ligament than one from their own body. Results of this study support the orthopedic literature showing that anterior cruciate ligament reconstruction is a safe and effective procedure in active individuals and provides patients, parents, providers, coaches, and trainers with additional valuable information (Am J Sports Med. 2013 Jul;41(7):1534-40. doi: 10.1177/0363546513490277. Epub 2013 May 30. PMID: 23722056).
   • Although there have been advances in rheumatoid arthritis (RA) treatments, oral diseasemodifying anti-rheumatic drugs (DMARDS) are still the gold standard for treating RA patients. However, to benefit from these drugs, it is important for patients to take their medications as prescribed. Drs. Christian Waimann, Maria Suarez-Almazor, and colleagues from the University of Texas MD Anderson Cancer Center studied medication use in an ethnically diverse and predominantly low-income population over 2 years. They found that only one-fifth of RA patients took their DMARD medications as prescribed at least 80 percent of the time and, of all the doses of medication available during the study period, less than two-thirds were taken correctly. This study is the first to measure drug adherence in RA patients over the long term. Patients who had better mental health status and were not widowed or separated were more likely to adhere to their medication regimen—findings that will help health care providers identify subsets of their patients who might benefit from more intensive efforts to ensure that they fully benefit from available treatments. (Arthritis Rheum. 2013 Jun;65(6):1421-9. doi: 10.1002/art.37917. PMID: 23728826).
   • Mycophenolate mofetil (MMF) is an immunosuppressive medication used to treat lupus patients, particularly those who have kidney inflammation. Investigators in France, Boston, and Houston, led by Dr. Tamar Aprahamian (Boston University), studied the impact of MMF in lupus-associated atherosclerosis in a mouse model of lupus that develops atherosclerosis when fed a cholesterol-rich Western diet. When treated with MMF, the mice had not only decreased symptoms of lupus, but also decreased atherosclerotic lesions, which were indicative of less severe cardiovascular disease. Although more extensive studies are needed to better understand the link between atherosclerosis, inflammation, and autoimmune disease, these findings point to MMF as a potentially valuable drug to prevent the development of atherosclerosis in patients with lupus (PLoS One. 2013;8(4):e61042. doi: 10.1371/journal.pone.0061042. Epub 2013 Apr 8. PMID: 23577189).
   • Recessive dystrophic epidermolysis bullosa (EB) is a blistering skin condition that is immensely painful and places patients at high risk of infections, scarring, malnutrition, and skin cancer. Drs. David Woodley and Mei Chen of the University of Southern California tested the efficacy of topical and intravenous administration of a recombinant collagen VII protein in mouse models of recessive dystrophic EB. Dr. Katz noted that systemic therapies in particular could have a huge impact on the quality of life for EB patients, making findings about the safety and potential efficacy of the intravenous route of administration particularly exciting. Drs. Woodley and Chen found that although topical application corrected the defects in existing wounds, the compound could not penetrate intact skin to prevent the formation of new blisters. However, related experiments revealed that recombinant collagen VII might have general utility as a topical therapy to accelerate wound healing and reduce scarring in other clinical settings (Mol Ther. 2013 Jul;21(7):1335-44. doi: 10.1038/mt.2013.87. Epub 2013 May 14. PMID: 23670575).

   Update on NIH and NIAMS Activities

   Dr. Katz reminded the group that at its June meeting, NIAMS Deputy Director Dr. Robert Carter provided an overview of the Institute’s recent efforts to assess whether NIAMS-funded Centers are currently configured to meet the rapidly evolving needs of the NIAMS scientific community. Since posting the final report on its website, the NIAMS has published a Request for Information (RFI) to solicit comments on the plan as the Institute considers new funding strategies for the Centers programs. The comments received suggest that the report has been well-received by the community. Dr. Katz attributed this to the outstanding efforts of the Centers Evaluation Working Group, which identified and articulated some common goals that the NIAMS will reflect on moving forward. He noted that Council members Dr. Alice Pentland (James H. Sterner Professor and Chair in the Department of Dermatology at the University of Rochester School of Medicine and Dentistry) and Mr. Bradley Stephenson were Working Group participants. A small Centers Leadership Group has been created within the NIAMS to implement the necessary steps for the design, solicitation, review, award, and administration of a new set of Centers. This group is being led by Drs. Joan McGowan (Director of the NIAMS Division of Musculoskeletal Diseases) and Susanna Serrate-Sztein (Director of the NIAMS Division of Skin and Rheumatic Diseases) and is charged with developing strategies to configure Center structures that, consistent with the principles set forth in the Working Group’s report: (1) support collaborative, transdisciplinary team science and essential resources; and (2) maintain flexibility to address critical emerging scientific challenges that require unique approaches. Dr. Katz noted that the Council will be kept informed as these activities progress. The goal is to develop the first Funding Opportunity Announcement (FOA) in FY 2015, and issue the first set of awards in FY 2016.

   The NIAMS has been working with the National Institute of Neurological Disorders and Stroke (NINDS), the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the National Heart, Lung, and Blood Institute (NHLBI), and other members of the Muscular Dystrophy Coordinating Committee (MDCC) to update the National Action Plan for the Muscular Dystrophies. The MDCC (of which Dr. Katz is a member) is soliciting public input on accomplishments relevant to the specific objectives in the existing Action Plan, remaining opportunities, and suggestions for new research objectives. Comments are being sought from researchers, health care providers, patients and their families, and all others who are interested in understanding the disease mechanisms, improving diagnostic and screening methods, developing therapies, and otherwise improving the lives of the people whom these diseases affect. Council members were invited to share their thoughts using a form available on the NINDS Web site before December 20, 2013.

   Dr. Katz reported that the NIAMS is also updating its Long-Range Plan, the current version of which runs through the end of FY 2014. As with the previous plan, the goal is to provide a document that will propel research by informing the Institute’s priority-setting process while enabling it to adapt to the rapidly changing biomedical and behavioral research landscapes. The Long-Range Plan outlines the Institute’s perspective on research needs and opportunities within the NIAMS mission and serves as a resource for all who are interested in NIAMS activities. He noted that the Long-Range Plan is the topic of his Director’s Letter in this month’s Shorttakes. Later in this Council meeting, Ms. Anita Linde, who directs NIAMS’ OSPPC, provided additional information on the Institute’s planning process.

   Dr. Katz then provided the Council with highlights of the Institute’s outreach and dissemination efforts. On July 10, 2013, at the request of the Society for Women’s Health Research (SWHR), Dr. Carter participated in a Capitol Hill event where he provided remarks regarding NIH research and the etiology and treatment of rheumatic diseases in women. The briefing was sponsored by Representative Lois Capps (D-CA) and co-sponsored by the SWHR and the American College of Rheumatology (ACR). Dr. Katz noted that the NIAMS often has opportunities to partner with the NIH Office of Research on Women’s Health (ORWH) on studies to uncover the bases of sex differences and devise effective strategies to treat and prevent them. Dr. Janine Clayton, NIH Associate Director for Research on Women’s Health and Director of the ORWH, addressed the Council later in the meeting.

   Recently, Dr. Katz was part of a small group of IC Directors who met with HHS Secretary Kathleen Sebelius to discuss how NIH investments in medical research are changing people’s lives. The Secretary was enthusiastic about the work that the NIH and NIAMS supports, and spent part of the day with NIAMS investigator Dr. Raphaela Goldbach-Mansky, Acting Chief of the Translational Autoinflammatory Disease Section, and a patient who has benefitted from Dr. Goldbach-Mansky’s work on neonatal onset multisystem inflammatory disease (NOMID).

   During this meeting, Council members were also given an update on the NIAMS National Multicultural Outreach Initiative, an effort to ensure that the results of the Institute’s research investments and its health messages reach all Americans. Through this initiative, the NIAMS is working with national partners to develop and disseminate culturally appropriate messages and materials about bones, joints, muscles, and skin for racial, ethnic, and underserved populations. Dr. Katz thanked Council members Dr. Ted Mala (Director of the Traditional Healing Clinic at the South Central Foundation), Mr. Stephenson, and Ms. Pickford for their involvement in this effort.

   Dr. Katz acknowledged NIAMS staff members who have been featured by various popular press and online media outlets:

   • NIAMS Scientific Director Dr. John O’Shea was quoted in a MedPage Today article regarding salt and autoimmune disease. He placed the research that the article was describing into a public health context, noting the importance of maintaining a healthful diet regardless of whether researchers ultimately determine a connection between salt consumption and the risk of developing an autoimmune disease.
   • Also from the NIAMS IRP, Dr. Mahendra Rao, Chief of the NIAMS Laboratory of Stem Cell Biology, appeared in the PBS NewsHour feature "Liver Buds Show Promise, but Growing New Organs is Still a Long Way Off."
   • Dr. Gayle Lester, Director of the NIAMS Extramural Clinical Osteoarthritis and Diagnostic Imaging Program, was featured on an Academy of Women’s Health blog segment about non-pharmacologic management of knee osteoarthritis.
   • NIAMS Senior Advisor Robyn Strachan participated in an American Association of Retired Persons (AARP) video showcasing the NIH as the #1 Winner of the 2013 AARP Best Employers for Workers Over 50 Award. The video illustrates how the NIH strives to improve the health of not only the American public, but also the people who work there every day (the video can be viewed on both the AARP and NIAMS Web sites).

   Discussion

   Council member Dr. Lynda Bonewald, Lefkowitz Professor in the Department of Oral Biology at the University of Missouri-Kansas City School of Dentistry (and ASBMR President), provided additional information on the ASBMR GAP initiative. She noted that new investigators are being lost, and senior investigators are being forced to downsize their laboratories across the country. As described by Dr. Katz in his Director’s Report, the GAP initiative is intended to serve as a bridge for investigators who just missed the funding payline. This year, the ASBMR will offer 20 $50,000 grants to help bone researchers fill that gap until they can resubmit their applications and hopefully be funded by the NIH. Dr. Katz applauded this effort and commented that it will be very helpful to the community.

   Council member Dr. Sherine Gabriel, Dean of the Mayo Medical School, William J. and Charles H. Mayo Professor, and Professor of Medicine and Epidemiology at the Mayo Clinic, noted that the ACR has had a successful bridge funding program in place for several years. Additionally, in her prior role as a co-Principal Investigator (PI) of a Clinical and Translational Science Award (CTSA), Dr. Gabriel and colleagues implemented a similar bridge program for a different community of young investigators that has been very successful.

5. THE NIH BIG DATA TO KNOWLEDGE (BD2K) INITIATIVE

   Dr. Mark Guyer, Deputy Director, National Human Genome Research Institute (NHGRI), explained that big data can be defined many ways, but most definitions of the term share two main characteristics: (1) it exceeds the capacity of unaided human cognition for its comprehension, and (2) it strains current technology capacity and is therefore CPU-bound, bandwidth limited, and/or storage limited. Three sources of data fall into the big data category:

   • Large datasets that are produced as community resources by a small number of generators (e.g., the human genome sequence, the Common Fund Molecular Library High-Throughput Screening Program, etc.).
   • Individual investigators producing large amounts of data (e.g., genome-wide association studies).
   • The many datasets produced by individual investigators which, if one could operate across them, provide significant opportunities for data mining and discovery.

   Through the BD2K initiative, the intent is to capture all types of data of interest to the NIH at large. In June 2011, NIH Director Dr. Francis Collins established the Data and Informatics Working Group (DIWG), which falls under the auspices of the NIH Advisory Committee to the Director. The DIWG produced a report in June 2012 that serves as the basis for the BD2K initiative. The report highlights a number of major problems that need to be addressed: (1) locating the data; (2) providing access to the data; (3) extending policies and practices for data sharing; (4) organizing, managing, and processing biomedical big data; (5) developing new methods for analyzing big data; and (6) training researchers who can use biomedical big data.

   In its report, the DIWG made several recommendations with respect to scientific data (the Working Group was also charged with examining administrative data and the data management systems at the NIH—these are being addressed through a separate Common Fund initiative). These include setting up a variety of approaches for making the data findable and available (e.g., data catalogs and software catalogs that provide links to the data and make datasets themselves citable).

   Dr. Katz noted that an additional problem is associated with collecting the data from investigators and housing it in some type of central database. Dr. Guyer agreed, noting that one of the overarching comments made by the DIWG is that there is a need for culture change at the NIH and in the scientific community with regard to terms of data sharing. To accomplish data sharing, there need to be cultural solutions demonstrating that this is an important and responsible activity. Incentives for sharing the data and developing the technology to allow them to do so are needed. The DIWG also recommended the development of new technologies and new approaches for manipulating, managing, storing, analyzing, and visualizing these large data sets. It also recommended placing a heavier emphasis on the development of quantitative skills in the training of biomedical researchers. The group’s final recommendation was that the NIH needs to invest the money needed to implement these activities. Dr. Guyer explained that in the concluding paragraph of its report, the DIWG summarizes that these issues are now and increasingly relevant to every IC and addressing them should be a trans-NIH activity, and a funding commitment will be needed.

   In response to the Working Group’s report, Dr. Collins established:

   • A new position in the NIH Office of the Director, the Associate Director for Data Science
   • A high-level, trans-NIH group, the Scientific Data Council
   • The BD2K initiative.

   NHGRI Director Dr. Eric Green has been appointed as the Acting Associate Director for Data Science while the NIH searches to fill the position on a permanent basis. The Scientific Data Council includes trans-NIH representation and will provide programmatic leadership and coordination of data science activities. Joint responsibilities of the Associate Director for Data Science and the Scientific Data Council include: (1) serving as the trans-NIH intellectual and programmatic hub for data science, (2) oversight of the BD2K initiative, (3) coordination with data science activities beyond the NIH, (4) long-term NIH strategic planning in data science, (5) playing a key role in data sharing policies and oversight, and (6) coordination with parallel administrative data efforts. Dr. Guyer emphasized that none of these efforts is intended to replace current ongoing activities within the individual ICs.

   Dr. Guyer explained that the BD2K is a major trans-NIH initiative that aims to be catalytic and synergistic, with the goal of enabling a quantum leap in the ability of the biomedical research enterprise to maximize the value of the growing volume and complexity of biomedical data. BD2K aims to develop the new approaches, standards, methods, tools, software, and competencies that will enhance the use of biomedical big data by supporting research, implementation, and training in data science and other relevant fields. Dr. Guyer noted that many of the challenges and the solutions developed in the context of big data will be appropriate and useful for data collection and analysis in general.

   BD2K will begin in FY 2014 and will be funded through an initial 7-year plan that starts with $27 million in FY 2014 and increases to slightly more than $100 million per year by FY 2016 (and continuing through FY 2020). A novel funding model is being used that features early front loading contributions by the Common Fund and increasing IC contributions. Complete budgetary "adoption" by ICs is expected by FY 2020 to ensure sustainability.

   Dr. Guyer outlined the four primary programmatic areas that BD2K will address: (1) facilitating broad use of biomedical big data, (2) developing and disseminating analysis methods and software for biomedical big data, (3) enhancing training for biomedical big data, and (4) establishing Centers of Excellence for biomedical big data to develop data science research. To obtain input from the community, two RFIs have been issued to date, one on the development of a data catalog and one on software needs. Dr. Guyer then discussed a series of upcoming BD2K workshops that will focus on the programmatic areas outlined above.

   The first FOA has been issued for the first round of BD2K Centers, which are essentially entirely investigator initiated. A second round of Centers will be solicited for next year and will be designed based on findings from the first round.

   In closing, Dr. Guyer commented that the biomedical research enterprise is undergoing a major phase change with respect to big data and data science. This change is bringing about trans-NIH challenges that require trans-NIH solutions that will include multifaceted cultural changes.

   Discussion

   Dr. Dietz asked about the extent to which users of big data will be represented on the newly established Scientific Data Council. He pointed to the critical need for the phenotypic information associated with many different sources of big data to be quality controlled and granular. Dr. Guyer explained that NIH’s approach recognizes that there are two major types of expertise needed for dealing with big data—those who develop the solutions and the users. Both groups need to be incorporated into these efforts. For example, at each of the BD2K workshops, at least one-half of the invitees are data users. The Scientific Data Council includes members who are not informaticists and represent intramural programs.

   Council member Dr. Gary Firestein, Professor in the Department of Medicine at the University of California, San Diego School of Medicine, noted the importance of including not only "highend" users, but also the "rank and file" users. He commented that one key to this effort will be making big data accessible to the lower-end users. Dr. Guyer agreed, noting that one specific aim of BD2K is addressing the inability of the average biomedical researcher to fully take advantage of the technological capabilities that are now available for data generation.

   Dr. Katz asked if the country has enough individuals with the required expertise to lead this effort. Dr. Guyer reminded the group that there is a call for training in this area to improve the number of experts in this field. He added that one challenge facing these efforts relates to competing with industry for individuals who have expertise with big data.

6. ENHANCING DATA REPRODUCIBILITY INITIATIVE

   Dr. Lawrence Tabak, Principal Deputy Director, NIH, briefed the Council on the ongoing Enhancing Data Reproducibility Initiative, an effort to enhance the reproducibility and transparency of research findings. He noted that the reproducibility and transparency of research findings have been described as an issue in multiple publications recently. Reproducibility and transparency concerns have been observed in both clinical and preclinical research, although his presentation focused on preclinical research.

   One of the primary papers that brought this issue to the forefront was published in Nature Reviews and Drug Discovery. The authors found that roughly two-thirds of in-house projects conducted in the pharmaceutical industry could not replicate data published by others. With preclinical studies in particular, insufficient reporting of methodological approaches is common. Dr. Tabak noted that this does not necessarily mean that the studies were conducted improperly or that the results are suspect, just that the methodology was not reported adequately. He provided an example of a series of hundreds of published studies related to stroke, Parkinson’s disease, and multiple sclerosis. Very few studies indicated whether the assessment was blinded, and none indicated sample size calculation. Dr. Tabak described a survey of top journals and publications that assessed quality criteria of the papers published. Although more than 60-70 percent of quality criteria related to dose-response and clinical outcomes were met, very few studies articulate important measurements such as blinding or randomization.

   Dr. Tabak noted that the NINDS and National Cancer Institute (NCI) convened workshops in 2012 to enhance their focus on data reproducibility (a summary of the NINDS workshop was published in Nature later that year). The results of these workshops were brought to the IC Directors and a thorough discussion was held, culminating in Dr. Collins forming an ad hoc group (led by Dr. Tabak) to develop approaches for addressing these issues.

   Dr. Tabak indicated that inadequate training and evaluation, and perverse reward incentives (e.g., the culture of "publish or perish") all contribute to the issues facing the NIH and the research community at large with regard to the transparency and reproducibility of research findings. He then outlined five principles for addressing these underlying issues: (1) raise community awareness, (2) enhance formal training, (3) improve the evaluation of applications, (4) protect the integrity of science by adopting more systematic review processes, and (5) increase stability for investigators.

   Dr. Tabak offered two additional suggestions for consideration: (1) consider the use of guidelines and/or checklists to systematically evaluate grant applications, and (2) consider the advisability and approach to supporting replication/reproducibility studies or centers.

   Discussions with IC Directors have elucidated important factors to consider as the pilots are designed, implemented, and evaluated. These issues include the many difficulties and differences in implementation across fields and research areas, the effects on experienced versus early career researchers, the costs of housing and managing additional data, the potential for added burden to the review process, and the difficulty associated with publishing negative results (Dr. Tabak noted that this point ties into BD2K efforts to develop a metadata catalog of all NIHsupported research that would allow investigators to access aggregated data from studies with positive and/or negative findings). A number of ICs are participating in a variety of pilot studies to address the principles outlined above.

   Discussion

   Dr. Elizabeth Shane, Professor of Medicine and Vice-Chair for Clinical and Epidemiological Research at Columbia University College of Physicians and Surgeons, asked if increasing the focus on ethics training might have the unintended consequence of de-emphasizing training on appropriate scientific experimental design. Dr. Tabak noted that others have raised this concern and commented that this initiative is intended to focus on ethics training through an evolutionary approach (as opposed to a revolutionary approach). It may be that ethics training deserves its own module rather than adding it into training on experimental design.

   Dr. O’Keefe asked about dealing with the pharmaceutical industry, particularly in cases where there is the perception that complications may be under-reported. Dr. Tabak noted that the NIH does try to engage the pharmaceutical industry through various partnerships that focus on shared interests and common goals (particularly with regard to pre-clinical work). It is hoped that having an increased emphasis on addressing the issues related to data reproducibility and transparency as researchers work with the pharmaceutical industry will garner some positive effects.

   In response to a question from Dr. Bonewald regarding how investigators can access data on previous attempts to reproduce the results of a given experiment, Dr. Tabak noted that the field is not yet at this point. However, the path forward involves capturing data sets as they are produced from laboratories around the country to create a catalogue. Tools will be developed to allow investigators to identify, extract, and in real time integrate previous work from various laboratories. Dr. Bonewald noted that this is also an issue for reviewers (e.g., when a reviewer personally knows of multiple failed attempts to reproduce the results from a study that is cited in an investigator’s grant application).

   Council member Dr. Xiao-Jing Wang, Professor in the Department of Pathology at the University of Colorado, Denver, asked about parameters for measuring the scientific impact of published studies. Dr. Tabak responded that the NIH is working on measures for providing insight into the importance/value of a given publication, although much work remains to be done in this area.

7. NATIONAL MULTICULTURAL OUTREACH INITIATIVE

   Dr. Carter and Ms. Mimi Lising (NIAMS OSPPC) provided an update on the status of the NIAMS National Multicultural Outreach Initiative (NMOI). Dr. Carter explained that, through the NMOI, the NIAMS is: (1) raising awareness in multicultural communities about the availability of NIH, NIAMS, and other federal resources to help people with conditions of the bones, joints, muscles, and skin; (2) emphasizing that research is the foundation for progress; and (3) supporting and involving organizations in multicultural outreach. The third activity was driven in part by hearing from health advocacy groups that additional tools were needed to reach multicultural communities. The four underserved populations targeted include African Americans, Hispanics/Latinos, American Indians/Alaska Natives/Native Hawaiians, and Asian Americans and Pacific Islanders.

   Dr. Carter then presented the NMOI timeline. The first phase, from 2010-2011 involved stakeholder research, conducted through focus groups, to identify a health product and develop language and themes appropriate for the communities. These efforts played a significant role in shaping further development of the NMOI. In 2012, pilot testing of two health planners was carried out. Based on the results of that pilot study, health planners for each of the four population groups were developed for 2013. At the same time, the electronic toolkit (or etoolkit) of resources was developed in an effort to provide on-line materials.

   The four health planners, one tailored for each of the multicultural populations, included 12 months of resources, reminder stickers to track appointments and other self-care activities, and photographs and culturally tailored message content to reflect the diverse populations. The planners were distributed nationally from January 3 to March 31, 2013. The focus of the outreach was on intermediaries, including NMOI Work Group members, NIAMS Coalition members, and national organizations that have access to multicultural communities. Outreach was conducted through a variety of mechanisms, including e-blasts, social media, exhibits, and direct mailings.

   Ms. Lising reported that the Institute was very pleased with how well the planners were received. Overall, 1,463 orders were placed for 80,560 copies of the health planners. The greatest number of orders came from community-based organizations, health advocacy groups, and multicultural entities, representing 36 percent of all shipped copies. In addition, 41 NIAMS Coalition organizations, representing both national offices and local chapters, placed orders. Ms. Lising reported that there were more than 123,000 contact points (i.e., any view, access to publications, or other exposure of any kind to the NMOI outreach effort) from specific outreach efforts. The health planner distribution generated more than 3,000 unique website visits, and nearly 1,000 inquiries to the NIAMS Information Clearinghouse from individuals and organizations. The health planners were delivered to almost every state in the nation and to five U.S. territories.

   After the planners were distributed, the NIAMS conducted an evaluation of their effectiveness. Ms. Lising indicated that intermediaries reacted positively toward the planners, finding them informative, culturally appropriate, and useful as patient-centric educational tools. They especially noticed and appreciated that each of the four planners was tailored to a specific multicultural community. They reported that the populations they serve connected well to the cultural specificity, especially the photographs. Stakeholders indicated that, by far, the stickers were one of the most useful aspects of the planners for prompting self-care actions to assure medications do not run out and that appointments are met. The stickers also encourage easy tracking for people with limited literacy and physical abilities. Recipients found that tracking their good and bad days was particularly helpful, especially in communicating with their health care providers. Intermediaries conveyed the usefulness of the English/Spanish bilingual planners, but also expressed a strong need for resources in additional languages, including Chinese, Korean, and Vietnamese..

   Ms. Lising commented that the e-toolkit was developed to assist organizations with promoting the health planners in their communities. It contains resources and downloadable versions of the health planners, a promotional flyer, sample tweets and Facebook posts, a fact sheet, a newsletter article, and an image gallery of all the photographs used in the health planners. One of the more popular tools on the e-toolkit is the image gallery, which contains many copyright-free multicultural photographs.

   The original impetus for this project was to support the outreach efforts of NIAMS’ partners to multicultural populations. With this in mind, a community-engagement approach was employed to develop and distribute the planners. Significant community input was obtained throughout all stages of the planner development and review process. In addition, five workgroups of multicultural experts provided strategic guidance and input throughout the planner development to ensure they were culturally relevant, patient-centered, and practical. Ms. Lising acknowledged Council members Dr. Mala, Mr. Stephenson, and Ms. Pickford for their contributions to the Multicultural Initiative leadership group. In addition, NIAMS’ federal colleagues provided contact information and resources for the Federal Resources section of the planners, and they were vital partners in promoting the planners through their networks.

   Ms. Lising noted that 2014 NMOI products will include: (1) electronic and hardcopy versions of the 2014 health planners; (2) additional multicultural images for the image gallery; (3) downloadable one-page fact sheets based on health planner messages; and (4) 11 fact sheets translated into Chinese, Vietnamese, and Korean languages. She suggested that Council members visit Community Outreach Initiative for more information on the NMOI and other NIAMS multicultural efforts.

   Discussion

   Dr. Mala thanked the Institute for its efforts in this area, noting that the health planners have been very well received. He also applauded the NIAMS for making these resources available online and suggested that some type of reminder cards be printed that direct individuals to the NMOI website. Dr. Katz indicated that the Institute will likely print hardcopy versions of the planners for a few more years, after which it will transition to an electronic-only format. Dr. Mala recommended that the Institute partner with the National Library of Medicine, which also has a strong history of outreach to minority groups.

   Mr. Stephenson noted that he reached out to some of his contacts at organizations serving communities along the U.S.-Mexico border to inform them of the NMOI and the health planners. Some of these organizations placed orders for the planners.

   • Develop capacity to leverage resources of major integrated health care systems for largescale clinical research studies.
   • Test and improve methods to extract research quality data from electronic health information systems.
   • Strengthen relevance and translatability of research results to “real world” health practice.
   • Develop and test more cost-effective models for large-scale randomized clinical trials.
   • Preventing hospital acquired infections: Do intensified antibacterial bathing measures reduce hospital-acquired infections? This cluster randomized trial was conducted in collaboration with the Hospital Corporation of America including 50 hospitals and 375,000 patients (the paper from this trial was published recently in the New England Journal of Medicine and showed a dramatic benefit of targeted decolonization in intensive care unit settings over standard care).
   • Colorectal cancer screening: Does a simple intervention implemented through Federally Qualified Health Centers improve colorectal cancer screening? This is a cluster randomized trial including 18 clinics and 10,000 patients.
   • Suicide prevention: A trial comparing suicide prevention programs. Can patients who admit to suicidal thoughts in a health care encounter be successfully randomized to one of two management strategies and do interventions reduce subsequent suicide attempts? This trial includes 16,000 patients from two major health care systems.
   • Lumbar Spine imaging: Does insertion of epidemiological information into imaging reports reduce subsequent diagnostic and therapeutic interventions? This cluster randomized trial includes 128 clinics and 135,000 imaging reports.
   • Nocturnal blood pressure control: Does taking anti-hypertensive medications at night reduce cardiovascular events? This trial includes 6,000 patients from University of Iowa and Duke University primary care clinics.
   • Collaborative care pain management model: Study on the impact of integrating psychosocial support for patients with chronic pain on pain measures and opioid use. This trial includes 6,000 patients from several hundred practices from three Kaiser regions.
   • Longer dialysis duration: Does increasing dialysis duration reduce mortality? This cluster randomized trial includes 402 dialysis units and 7,000 patients.

8. WHY SEX AND GENDER MATTER FOR HEALTH AND BIOMEDICAL RESEARCH

   Dr. Janine Clayton, NIH Associate Director for Research on Women’s Health and Director of the ORWH, reminded the group that the ORWH was founded in 1990 and is now 23 years old. The ORWH is an entity within the NIH Office of the Director and it leverages its investments through partnerships and working in collaboration with the ICs. The ORWH continues to support and advocate for the inclusion of women in research. It also extends its focus beyond simply including women in research—Dr. Clayton explained that women need to be included in studies so that the data developed and the result of those studies can be applied to women as well as men. The ORWH is also working to expand career development programs for women in biomedical careers and continues to remain the focal point for NIH’s research agenda on women’s health and sex and gender factors.

   Dr. Clayton explained that the Strategic Plan for Women’s Health Research is an NIH-wide strategic plan led by the ORWH. This Plan is implemented primarily through partnerships with ICs. An interdisciplinary approach has been a component of the Office from its inception to address the fact that women’s health and women’s health care can be very fragmented because of the way women are cared for in the medical system.

   This year, the ORWH issued an administrative supplement program for human and animal studies to facilitate adding additional groups of subjects (e.g., the opposite sex) to studies when only one group was included in the original study. This program has been very well received, with the ORWH funding 35 of these supplements. The Research Enhancement Award Program (REAP) is a trans-NIH program that has been in existence for many years. Through REAP, the ORWH partners with IC colleagues to fund grant applications that have just missed IC funding paylines. Recent REAP awards to NIAMS investigators have included projects on osteoclast fusion, osteoporosis in women, cutaneous dermatomyositis, cutaneous lupus erythematosus, ondemand drug delivery for RA, and a model for a new anti-RA drug. ORWH funds are for 1 year and up to $100,000, and the NIAMS has committed to fund the out years for these grants.

   Dr. Clayton described some of ORWH’s interdisciplinary programs, including the Specialized Centers of Research on Sex Differences (SCOR), which focuses on sex differences through integrated basic, clinical, and translational research; and Building Interdisciplinary Research Careers in Women’s Health (BIRCWH), which is an institutional mentored career development program designed to increase the number of women’s health researchers.

   The ORWH established an R21 program—Advancing Novel Science in Women’s Health Research (ANSWHR)—5 years ago with the goal of promoting innovative, interdisciplinary research to advance women’s health research and the study of sex/gender differences. Dr. Clayton noted that a number of NIAMS grantees are supported through ANSWHR. Other ORWH-NIAMS collaborations include the Osteoarthritis Initiative (OAI), Lupus Federal Working Group, and the NIH Osteoporosis and Related Bone Diseases National Resource Center. Dr. Katz commented that ORWH involvement in the OAI was critical in moving this project forward. Dr. Clayton noted that the ORWH has contributed roughly $10 million to the OAI, and its investment in the OAI has clearly helped get the message out to the community regarding gender differences in osteoarthritis. She noted that the Office helped to develop OAI Online, an international resource that has roughly 2,800 registered users from 105 countries, more than 15,229 datasets downloaded, and 457 image sets distributed. The ORWH also cofunds a number of projects in the areas of lupus, bone health, pain, and RA.

   Research on women’s health is essential for a number of important reasons. There are many significant sex factors in diseases/organs related to reproduction, as well as those not related to reproduction. Important information may be missing because sex and gender are sometimes not included in research design. These issues are guiding much of ORWH’s work moving forward. Dr. Clayton explained that the classification of "sex" is derived from the chromosomal complement, while "gende"” represents a person’s self-representation as a male or female. Both sex and gender affect health, behavior, and perception, and thus both are critically important in biomedical research.

   At a recent Institute of Medicine workshop (that also was supported by the ORWH) titled "Sex- Specific Reporting of Scientific Research," four important themes emerged: (1) identifying the sex of populations in journal publications (include sex of origin cells, tissues, and animals in basic research), (2) sharing sex-identified raw data to facilitate meta-analyses, (3) providing "extra credit" in review for manuscripts that include sex-specific information, and (4) requiring sex-stratified analyses. These efforts will require collaboration on the part of journal editors, government funding agencies, industry, basic researchers, professional societies, and other stakeholders.

   Dr. Clayton noted that the American Physiological Society has added to its instructions to authors, indicating the need to include the sex of the cells and the animal models used in the Materials and Methods section. She also explained that the ORWH is collaborating with the FDA on an on-line course on the science of sex and gender in human health. There are a variety of other resources that also speak to the importance of including these issues in medical and professional curricula (e.g., a May 2013 Health Resources and Services Administration report on women’s health curricula).

   Dr. Clayton concluded her remarks by emphasizing that examining sex and gender can improve not just women’s health, but the health of the entire population. She identified four cross-cutting areas in ORWH’s strategic plan that will be guiding the Office moving forward:

   • Advance the understanding of biological sex differences.
   • Apply new technologies to maximize research potential and impact.
   • Expand understanding of health and disease in women.
   • Foster partnerships to conduct and translate research.

   Discussion

   Dr. Katz noted that each of the ICs contributes to a report on the inclusion of women in clinical studies that is submitted to Congress on behalf of the ORWH (this report is also included in the NIH Director’s Biennial Report).

9. NIAMS LONG-RANGE PLAN FISCAL YEARS 2015-2019

   Ms. Linde provided Council members with an overview of the process for updating the current version of NIAMS’ Long-Range Plan, which was developed with considerable input from Council members and runs through the end of FY 2014. The next plan will take effect in FY 2015 and run through FY 2019. She explained that NIAMS refers to this plan as a "Long-Range Plan" instead of a "strategic plan" because its intent is to provide a broad outline across the spectrum of the Institute’s extramural research programs that captures both what it is currently investing in, as well as looking forward to areas of emerging research across the arthritis, musculoskeletal, and skin disease fields. The Long-Range Plan is used to communicate the Institute’s perspective in terms of research needs and scientific opportunities for the coming 3-5 years and is designed to serve as a tool and resource for all who are interested in NIAMS’ activities. The Long-Range Plan is not meant to be prescriptive; rather, it is meant to help communicate what the Institute sees as research needs and to spark an ongoing dialog with its various stakeholder communities.

   Like the current version, the updated Long-Range Plan will include some cross-cutting topics that transcend NIAMS’ disease- and tissue-specific areas. These include health disparities, training and career development, and information dissemination. In these areas, the NIAMS is leveraging what is happening at the trans-NIH level and is working to define the important components that impact most directly on the Institute’s research communities and affiliated organizations. Disease- and tissue-specific topics included in the current Long-Range Plan that are anticipated to be replicated in the updated plan include arthritis and rheumatic diseases, skin biology and diseases, bone biology and diseases, muscle biology and diseases, and musculoskeletal biology and diseases.

   The NIAMS will be providing periodic opportunities for Council members to provide input and feedback. During the next few months, a web-based RFI will be issued to obtain input from the community. A series of listening sessions will be held and will include both scientific and lay representatives to solicit more specific input about opportunities going forward. The Long- Range Plan will also be discussed at the November NIAMS Coalition Outreach and Education Day. Another update will be given to the Council at its February 2014 meeting, followed by a more substantive discussion in June, when a draft Long-Range Plan is expected to be ready. Once formulated, the draft Long-Range Plan will be posted online and another RFI will be issued. It is hoped that by this time next year, the final Long-Range Plan will be ready for presentation to the Council and posting online.

   Ms. Linde noted that there are two key overarching questions guiding the planning process for the NIAMS Long-Range Plan. First, what research opportunities in the existing plan should be modified because of progress over the past 5 years? And second, what emerging research opportunities should be added to the plan? The ideas gathered during NIAMS roundtable discussions and scientific planning retreats will also be considered for inclusion in the plan. Additionally, the Institute will be leveraging a series of activities and existing resources from other trans-NIH plans such as the NIH Health Disparities Plan, the NIH Strategic Plan for Women’s Health Research, etc.

   Discussion

   Dr. McGowan reminded Council members that the Institute depends on them and their respective communities to provide input and comments on the Long-Range Plan, particularly with regard to new and emerging areas of opportunity. She asked Council members to respond to the RFI, and to provide specific comments on strategically enhancing access to and utilizing big data. Dr. Katz emphasized the importance of the Long-Range Plan, noting that it serves as a communication vehicle to the public and to the Institute’s professional communities to provide a sense of the breadth and depth of its interest in various areas. Ms. Linde added that the Long- Range Plan is included in the Institute’s communications with Congress.

   Dr. Pentland asked about how professional research societies have worked with NIAMS in the past to shape the Long-Range Plan and if there are ways that these societies can better inform the planning process. Dr. Katz explained that the Institute began developing its Long-Range Plans in 1999 and that input from the professional societies has been critically important. It is also important for the Institute that the professional societies disseminate the information to their members for additional comment and feedback. Dr. Serrate-Sztein agreed, adding that input from both professional and patient/voluntary organizations has greatly improved the quality of the Long-Range Plan.

10. 2015 SCIENTIFIC INITIATIVES

    Dr. Katz explained that the NIAMS is continuing to identify research areas and activities that are ripe for investment. Before the concluding the open session of the Council meeting, Dr. Katz invited Council members to review the descriptions of the following proposed FY2015 NIAMS extramural program initiatives:

    • Consortium on Signaling Networks to Critical Targets (CONNECT)
    • The Circadian Cycle in Musculoskeletal, Skin, and Rheumatic Diseases and Tissues
    • Strategies To Improve Understanding of Atypical Femur Fractures and the Role of Osteoporosis Therapy
    • From GWAS to ENCODE and Beyond — Bridging the Gap With Critical Cells and Tissues of Direct Relevance to Rheumatic, Skin, and Musculoskeletal Diseases
    • The Skin-Brain Axis: The Neuroendocrine Influence on Cutaneous Biology
    • Re-Issue of the Ancillary Studies to Large Clinical Projects
    • Reissue of the NIAMS Clinical Trial Outcomes and Instrument Development Grant Program (U01)

11. BOARD OF SCIENTIFIC COUNSELORS REPORT

    This portion of the meeting occurred during closed session.

12. CONSIDERATION OF APPLICATIONS

    In closed session the Council reviewed a total of 812 applications in closed session requesting $824,153,251 in total costs and recommended 812 for $824,153,251 in total costs. 80 applications were considered by early concurrence.

13. SPECIAL ACTIONS

    This portion of the meeting occurred during closed session.

14. BASIC BONE BIOLOGY AND BONE DISEASES PORTFOLIO ANALYSIS

    This portion of the meeting occurred during closed session.

15. ADJOURNMENT

    The 81st National Arthritis and Musculoskeletal and Skin Diseases Advisory Council Meeting was adjourned at 3:10 p.m. Proceedings of the public portion of this meeting are recorded in this summary.

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