DEPARTMENT OF HEALTH AND HUMAN SERVICES
PUBLIC HEALTH SERVICE
NATIONAL ARTHRITIS AND MUSCULOSKELETAL AND SKIN DISEASES ADVISORY COUNCIL
MINUTES OF THE 99th MEETING
September 10th, 2019
8:30 a.m. to 3:00 p.m.
I. CALL TO ORDER
The 99th meeting of the National Arthritis and Musculoskeletal and Skin Diseases Advisory Council (NAMSAC) was held on September 10th, 2019, at the National Institutes of Health (NIH) Campus, Building 45, Conference Rooms E1/E2. The meeting was chaired by Dr. Robert H. Carter, Acting Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).
Council members present:
Ms. Karen M. Ball, President and CEO, The Sturge-Weber Foundation
Dr. Joan M. Bathon, Professor and Chief of the Division of Rheumatology, Columbia University College of Physicians and Surgeons
Dr. Jill P. Buyon, Director, Division of Rheumatology, Department of Medicine, New York School of Medicine, and Director, NYU Lupus Center
Dr. Elizabeth H. Chen, Professor, University of Texas Southwestern Medical Center
Mr. Vincent Del Gaizo, Director of Strategic Partnerships and Patient Engagement, Childhood Arthritis and Rheumatology Research Alliance (CARRA)
Dr. Michael Econs, Glenn W. Irwin, Jr., Professor of Endocrinology and Metabolism; Director, Division of Endocrinology and Metabolism; and Professor of Medicine and Medical and Molecular Genetics, Indiana University School of Medicine
Dr. Said A. Ibrahim, Vice Chair for Strategy and Development and Chief, Division of Healthcare Delivery Science and Innovation, Department of Healthcare Policy and Research, Weill Cornell Medicine
Dr. Judith A. James, Chair and Member, Arthritis and Clinical Immunology, Oklahoma Medical Research Foundation
Dr. Ethan A. Lerner, Associate Professor of Dermatology, Massachusetts General Hospital
Ms. Rosemary J. Markoff, Co-Chair, Scleroderma Foundation National Advocacy Committee
Mr. William J. Mulvihill, The Mulvihill Advisory Group
Dr. Anthony Oro, Eugene and Gloria Bauer Professor of Dermatology, Associate Director and Professor, Center for Definitive and Curative Medicine, Department of Dermatology, Stanford University School of Medicine
Dr. Stephen J. Tapscott, Professor, Fred Hutchinson Cancer Research Center
Dr. Michael J. Yaszemski, Professor, Orthopaedic Surgery and Biomedical Engineering, Mayo Clinic
Staff and Guests
The following NIAMS staff and guests attended:
Dr. D. Lee Alekel
Dr. Gayle Lester
Dr. Danilo Tagle, Associate Director for Special Initiatives, National Center for Advancing Translational Sciences
Dr. Bruce Tromberg, Director, National Institute of Biomedical Imaging and Bioengineering
II. CONSIDERATION OF MINUTES
A motion was made, seconded, and passed to approve the minutes of the 98th NAMSAC meeting, held on June 5, 2019.
Ms. Nelson noted an upcoming request regarding members’ potential conflicts of interest. The request will come in October. Members must respond within 30 days of receipt in order to participate in any Council activities.
III. FUTURE COUNCIL MEETING DATES
Future Council meetings are currently planned for the following dates:
February 4, 2020
June 9, 2020
September 1, 2020
January 26, 2021
May 18, 2021
August 31, 2021
IV. COUNCIL OPERATING PROCEDURES
Ms. Nelson reviewed the Statement of Understanding between NAMSAC and NIAMS. The Director of NIAMS has authority delegated from the Secretary of the U.S. Department of Health and Human Services (HHS) to make grants. Those awards must undergo peer review and be recommended by this Council. Policy requires staff to bring certain applications to the Council’s attention, among them: those involving animal welfare and human subject concerns, foreign grants, and items brought up by members of the Council. As part of the September meetings, Council will generally engage in an expedited concurrence process, which means a subset of the Council will review a slate of grant applications for the current year’s funding. Ms. Nelson noted that the Statement of Understanding is always available in the Electronic Council Book.
V. DIRECTORS REPORT
Dr. Carter’s report covered NIH and NIAMS personnel and policy changes, an update on the NIH and NIAMS budgets, an overview of this meeting’s agenda, and one NIAMS-funded scientific advance.
Dr. Carter noted the passing of NIAMS Associate Director for Management and Operations, Mr. Gahan Breithaupt, on August 31, 2019. Mr. Breithaupt’s death came within a year of the passing of NIAMS Director Dr. Stephen Katz. Dr. Carter noted Mr. Breithaupt’s often-unnoticed work behind the scenes. He characterized the deaths as “first our boss, and then our right-hand man” with over 80 years of institutional knowledge and federal service between them. Despite these significant losses, NIAMS staff conducted Institute business without interruption and handled some complicated tasks including the launch of the new Back Pain Consortium. Dr. Carter thanked staff for their resilience, dedication, and loyalty.
Dr. Carter noted that, despite completion of their four-year terms, Council members Lerner, Mulvihill and Tapscott will continue to serve on the Council for an additional 180 days under an administrative extension. He identified NAMSAC’s newest permanent members: Ms. Karen Ball, Dr. Joan Bathon, Dr. Elizabeth Chen, Dr. Said Ibrahim, and Dr. Jennifer Westendorf.
Since NAMSAC’s last meeting, two Institute Directors have announced their retirements: Dr. Paul Sieving of the National Eye Institute and Dr. Linda Birnbaum of the National Institute of Environmental Health Sciences. NIH recently welcomed Dr. Debara Tucci as the new Director of the National Institute on Deafness and Other Communication Disorders.
NIAMS continues to await news of who its new Director will be. The Search Committee has completed its task, and as of this meeting the decision is resting with NIH leadership. Progress continues on filling the position of Director of the Division of Extramural Research, as well as the Clinical Director of the Intramural Program. Dr. Carter acknowledged Dr. Bob Colbert’s service as the Acting Clinical Director and Dr. Gayle Lester’s efforts as Acting Director of the Division of Extramural Research.
Dr. Carter noted the passage and signing of a spending resolution lifting the budget caps that would have triggered sequestration in fiscal years 2020 and 2021. With the new federal fiscal year beginning on October 1, Appropriations committees in both chambers of Congress are working on HHS funding bills, and NIH expects to begin FY2020 with a continuing resolution. NIH will do its best to make awards once it has a sense how long the continuing resolution is likely to last. Congressional support for NIH remains strong.
In follow-up to a presentation that he gave at the September Council, Dr. Carter reminded Council members that despite the budget increases of recent years, NIAMS had a greater increase in funds committed for non-competing grants. This leaves less money for competing grants, despite the overall budget increase. Furthermore, the upward trend in non-competing costs is projected to continue for FY2020. NIAMS leadership is considering ways to moderate the impact of this increase, including increased use of the R56 bridge award for one or two years of funding for applications that just missed the FY 2019 payline. NIAMS greatly appreciates NAMSAC’s input on this and other budget issues over the years. Consistent with Council’s guidance, NIAMS is committed to funding as many investigators as possible.
Dr. Carter brought two NIH policy issues to the attention of the Advisory Committee. The first has to do with an updated policy regarding use of fetal tissue obtained from elective abortions. Dr. Carter referred those interested to a Notice (NOT-OD-19-137) dealing with the pertinent requirements and mentioned that relevant applications receiving a fundable score in the primary review are now required to undergo an additional review by a department-level Ethics Advisory Board, likely adding considerable time before the research may be funded. The second policy item originated with NIH Director Dr. Francis Collins’ intent to “[break] up the subtle (and sometimes not so subtle) bias that is preventing women and other groups underrepresented in science from achieving their rightful place in scientific leadership . . . .” Dr. Collins said he would decline to take part in scientific meetings and other high-level conferences in which inclusiveness is not evident in the agenda and challenged other scientific leaders to do the same. Dr. Carter indicated his support of this commitment, adding that NIAMS leadership will only accept invitations to speak at scientific conferences and meetings that follow this diversity policy.
Dr. Carter reviewed the meeting’s agenda, making the following points.
- There is a clear need for NIAMS and the National Institute for Biomedical Imaging and Bioengineering (NIBIB) to work together in advancing research on devices and imaging.
- The NIAMS Strategic Plan for FY2020-2024 is close to completion. Dr. Carter thanked those Council members who reviewed and commented on the various drafts of the Plan. The Plan’s theme is “promoting the unexpected”—fertilizing discoveries and ideas that cannot be anticipated at the present moment.
- NIAMS greatly appreciates the Back Pain Consortium (BACPAC) working group of Council.
- NIAMS has been a longstanding partner with the National Center for Advancing Translational Sciences (NCATS) on the Tissue-on-a-Chip initiatives. Dr. Carter noted the leadership of Dr. Fei Wang in working with NCATS.
- NIAMS welcomes Council’s feedback on the use of Notices of Special Interest (NOSI), a way an Institute can express interest in receiving applications on a specific topic without setting aside dollars associated with a Request for Applications.
- A recent perspective article in Journal of Bone and Mineral Research by Leder et al. reflects the importance of balancing what is known about the effectiveness of drugs that are prescribed to prevent osteoporotic fractures with the scientific opportunities pertaining to the use of these medications.
- NAMSAC would receive a report of the Board of Scientific Counselors and hear a presentation about the Institute’s small business programs during closed session.
Dr. Carter noted a paper by Croft et al. showing that two distinct fibroblast populations are organized in different layers of the joint tissue and drive different pathological features of rheumatoid arthritis (RA). The paper built on data accumulated from Accelerating Medicines Partnership (AMP) on RA and Lupus program. As background, Dr. Carter mentioned three foundational papers recently published in Nature Immunology that show how single-cell profiling technologies can shed light on potential pathogenic mechanisms.
AMP will be a topic at the February 2020 NAMSAC meeting, when a working group of Council will present lessons learned from the AMP RA/Lupus network and how AMP can serve as a model for other large interdisciplinary research projects. Dr. Carter also previewed two other topics for the February Council meeting: a roundtable discussion with members of the research community on the types of clinical trials that NIAMS can and should support, and a discussion about how to better get out the word around Funding Opportunity Announcements (FOAs).
VI. WEARABLE AND BEDSIDE BIOPHOTONICS FOR PERSONALIZED HEALTH
Dr. Carter introduced Dr. Bruce Tromberg, Director of the National Institute of Biomedical Imaging and Bioengineering (NIBIB). Dr. Tromberg noted his previous role at the University of California (UC) Irvine Beckman Laser Institute and Medical Clinic and at the UC Irvine Cancer Center program. Dr. Tromberg believes that NIBIB should be at the center of the NIH’s intramural and extramural engineering activities. He said NIBIB’s role is to integrate emerging technologies. Engineering and physical science in biology and medicine have been growing enormously, and biomedical issues have become a top priority across schools of engineering across the United States in the past 15-20 years. He characterized “physician-eering” as a growing enterprise. He noted increased NIH funding to Biomedical Engineering and Engineering Departments since 2000. NIH funding to Engineering Departments has increased sixfold since 2000, even as funding of NIH has increased only twofold. 12% of NIH’s budget goes to bioengineering, a 55% increase since 2008. NIBIB’s invention rate (i.e., patents) is about 4.5 times greater than that of the NIH average, and NIBIB sees a high leveraging effect as defined by downstream research and development (R&D) investments.
Intra-NIH partnerships are a means to effectively leverage appropriated funds. Dr. Tromberg noted strategic opportunities in 1) sensing and imaging, 2) digital health, and 3) cutting-edge biology technologies. NIBIB’s strategic vision can be summed up in the phrase “Engineering the Future of Health.” Success here would result in understanding, preventing, and detecting disease; extend healthspan and lifespan; personalize diagnosis and treatment; reduce costs and barriers to access; and drive economic growth. NIBIB’s building blocks to achieve these aims include modeling and machine intelligence, engineered biology, sensors and point of care devices, imaging technologies, and therapeutic devices.
Biophotonics represents a $70 billion per year industry. It can be divided into diagnostic and therapeutic categories. Emerging areas include diffuse optics, non-linear optics, and multiphoton microscopy. Dr. Tromberg described how technological advances have made diffuse optics more accessible. In the field of non-linear optics, the interaction of near-infrared lasers and collagen create new opportunities in skin imaging. He described his own work on techniques and findings in two-photon excited fluorescence and second harmonic generation in skin. The team at UC Irvine has invested considerable effort in exploring various processes and structures in the skin. This has applications for dermatology, as dermatologists are interested in examining skin at sub-micron depths. Images can be used to predict the future incidence of ulcers. Skin metabolism can be quantified on a per-cell basis. Longitudinal studies using imaging tools have resulted in better understanding of hemoglobin changes over time. These are but a few examples of how biophotonics can be used, and barriers to access to these new technologies should be minimized.
Dr. James asked how common multiphoton microscopy for looking under the skin is around the country, and whether the technology may be applied to conditions such as systemic lupus or sclerosis. Dr. Tromberg said, following validation, the techniques will be expanded; investigators and practitioners will need to think outside the box because technology developers will be looking for opportunities to collaborate. Ms. Markoff asked whether the technique has been tried as an alternative to punch biopsies in scleroderma. Dr. Tromberg did not believe that it had been tried but was not certain. Dr. Lerner asked for an elaboration about the status of imaging of nerve and immune cells in skin. Dr. Tromberg’s team has created images of structures they believe to be immune cells, though they are not yet certain of this. He acknowledged that imaging work related to neural processes is extremely important. Dr. Ibrahim noted the challenge of transitioning new technologies into clinical care and asked how Dr. Tromberg planned to accomplish this. Dr. Tromberg said the most important concepts regarding translation of technologies to patient care boil down to patience and persistence. Multi-institution consortia can be used to provide validation, support, and gravitas to these translational efforts. In response to a question from Ms. Ball, Dr. Tromberg emphasized the importance of partnering with foundations. Dr. Oro asked for comment on the presented technology versus more conventional biopsies. Dr. Tromberg said it will take time to validate the technology. A big challenge is incorporating the technology into the real lives of clinicians and patients. Among other challenges is one of designing a product that a physician can use during a patient interaction. In closing, Dr. Carter noted the axiom that most scientific advances come about as a result of technological advances.
VII. STRATEGIC PLAN FINAL REPORT
Ms. Caughman, Chief of the NIAMS Scientific Planning, Policy, and Analysis Branch, provided the report. Work on the NIAMS FY2020-2024 Strategic Plan commenced about one year prior to this meeting. A Request for Information about areas of the current plan that should be updated was released in fall 2018, followed by seven listening sessions with more than 100 participants. Disease- and tissue-specific research objectives as well as cross-cutting scientific themes were developed as a result. The NAMSAC Strategic Plan Working Group released their recommendations in spring 2019 and presented them at the June Council meeting. The Strategic Plan was revised as a result of NAMSAC input. Ms. Caughman emphasized Dr. Carter’s point that the Institute’s intent is to create a document that will catalyze new discoveries and position NIAMS to embrace the unexpected and capitalize on unanticipated opportunities. The Plan highlights NIAMS’ longstanding commitment to creating a research environment that fosters transformative new ideas through support for meritorious investigator-initiated research grants and collaborative scientific partnerships, as well as responsible management and stewardship of public resources. Public input was sought over the summer of 2019. Revisions to the plan are noted in a table available to NAMSAC members. The Strategic Plan was reviewed by the NIH Office of the Director and was found to be in compliance with the NIH Common Template for Strategic Plans. A September 2019 release of the Strategic Plan is anticipated. Ms. Caughman thanked NAMSAC members for their help and input.
Mr. Mulvihill suggested repositioning certain pieces of text (e.g., the Institute’s mission, the Plan’s purpose) so that they appeared earlier in the document. Dr. Oro suggested composition of a short document to provide additional transparency for lay members of the public interested in the Strategic Plan. Dr. Buyon emphasized that the concept of the Strategic Plan as a starting point, not the end, resonated with her.
VIII. BACPAC WORKING GROUP
Dr. Carter noted that the HEAL (Helping to End Addiction Long-Term) initiative is still working on its budget, and some decisions regarding the NIH Back Pain Consortium (BACPAC) have not yet been finalized. Dr. Derr of NIH said the HEAL initiative is NIH’s way of addressing the opioid crisis. The initiative has two broad goals: improving treatments for opioid misuse and addiction, and enhancing pain management by understanding the biological underpinnings of chronic pain and accelerating the discovery and development of non-addictive treatments of pain. BACPAC is the NIAMS-led component of HEAL; it uses a bio/psycho/social approach to understand chronic low back pain, the most common complaint among adults with chronic pain and the most common non-cancer reason for prescribing opioids. BACPAC is creating an integrated model of chronic low back pain to better understand the mechanisms of chronic low back pain and response to treatment. To this end, researchers will do deep phenotyping of patients to better understand the sub-phenotypes, look for new targets, develop new technologies or refine existing ones, and develop and validate algorithms that will allow healthcare providers to tailor treatments to patients’ sub-phenotypes. Four Funding Opportunity Announcements were issued as part of BACPAC.
Council member Dr. Bathon noted that the Working Group met twice by phone. She described the charge of the Working Group, which was to review proposals submitted in responses to the various Funding Opportunity Announcements and advise the Council about their findings. She identified overall recommendations in implementing BACPAC: engage patients and stakeholders across the Consortium; incorporate spinal stenosis expertise into the overall program; incorporate some elements of lower-scoring, unfunded proposals into the program, perhaps through pilot projects; consider a rigorous study addressing how to sequence treatments and complement existing interventions; and ensure that BACPAC works with other elements of the HEAL initiative to ensure interoperability of data elements and outcome measures.
Dr. Buyon asked for comment on the database and investigators’ willingness to contribute to it. Dr. Derr noted the Data Integration, Algorithm Development, and Operations Management Center will collect data in real time. When Dr. Buyon suggested providing funding to elements of non-selected proposals, Dr. Derr noted that BACPAC has money for pilot and feasibility studies and can support collaborative studies. With regards to integrating unfunded components into the program at the very beginning, Dr. Carter noted that there are NIH-wide challenges regarding the optics of funding low-scoring proposals.
IX. TISSUE - ON - A - CHIP
Dr. Carter introduced Dr. Danilo Tagle, Associate Director for Special Initiatives at the National Center for Advancing Translational Sciences (NCATS). Dr. Tagle described his presentation as a high-level overview of the program. NCATS’ mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. In 2012, the Microphysiological Systems Program (i.e., the Tissue Chips for Drug Screening initiative) was launched with the central goals of developing an in vitro platform modeling human cells and tissues and combining them with advances in stem cell biology, microfluidics and bioengineering to evaluate the efficacy, safety, and toxicity of promising therapies. The ten major organ systems of the body are represented. NCATS wants the products to be as physiological relevant as possible, genetically diverse, and pathologically meaningful. The original focus was on safety signals as an entry point to clinical testing. The hope is that the models can better predict adverse events and safety in humans than preclinical animal models can. The program is a partnership among FDA (as a non-funding partner), DARPA, and NIH. Dr. Tagle used the examples of organ systems to describe how the models are developed and function. He described the partnerships within the program, which includes other federal agencies, industry, and academia. Partners are building frameworks that will increase confidence in the validity of the models. The last phase of validation is industrialization, where the chips will be used to test proprietary sets of compounds. Regarding diseases and tissues of interest to NIAMS, a team at Pittsburgh is working on osteoarthritis and a team at Columbia is testing a model of muscle and vascular disease. A partnership with NASA is increasing knowledge about the effects of aging, as many of the changes that occur with age also occur more rapidly in microgravity; two of these studies focus on sarcopenia (University of Florida) and post-traumatic osteoarthritis (MIT). Future initiatives include co-culture of many differentiated iPSC-derived cell types per tissue architecture and composition, integration of different tissue chips to form human body on chip, genome editing to introduce various polymorphisms on isogenic iPSC lines, developmental/pediatric response to drugs/toxins, and rare diseases. NCATS has issued an RFA to foster research demonstrating the utility of clinical trials on a chip; NIAMS is participating in this initiative.
Dr. Chen noted the importance of obtaining both muscle and connective tissues in the case of sarcopenia. Dr. Tagle said only muscle is being cultured at this point. Dr. Buyon noted the importance of stem cells in the cultures. Dr. Tagle said this input would be incorporated into the systems. In response to a question from Dr. Buyon, Dr. Tagle said a big focus of the program from the beginning was development of fetal stem-cell alternatives. There are a lot of opportunities here, particularly with patient-derived iPSCs. Dr. Lerner asked for comment on the possible relative success of the program compared with rat and dog models. Dr. Tagle that animal models were able to predict neurotoxicity 30-40% of the time while brain on a chip saw a 90% prediction rate. Pharmaceutical companies and the FDA are beginning to think of tissue on a chip as a possible third species requirement. In response to Dr. Carter’s question, Dr. Tagle agreed that clinical trials on chips necessitate not only tissue on a chip but also diseases on chips. Dr. Econs noted the possibility that, given false positives in screening, possibly useful compounds could get erroneously ruled out. Dr. Tagle agreed and noted that pharmaceutical companies also are revisiting compounds that may have been erroneously ruled out because of false positives related to toxicities in animal studies.
X. NOTICES OF SPECIAL INTEREST (NOSI)
Dr. Carter introduced Ms. Amy Mistretta of the NIH Office of Extramural Research. Ms. Mistretta said NIH is transitioning from program announcements to NOSIs. NOSIs will point to pre-existing Funding Opportunity Announcements. She described the trends around RFAs and Program Announcements (PAs) since 2014. The NOSI is not a new idea; several ICs used them over the past 5-6 years. In the past two years, the Office of Extramural Research within the Office of the NIH Director made an effort to transition away from non-parent PAs to NOSIs. PAs that have already been released will remain active until their expiration. Ms. Mistretta described the features of completed NOSI. ICs will be able to update their topics of interest on their webpages. NOSIs move through OER review much more quickly. Several requirements must be fulfilled in a properly formatted NOSI. Ms. Mistretta displayed an exemplar NOSI published by NHLBI. NIH is working to ensure that the scientific community is aware of the transition to NOSIs. Going forward, NIH will continue to use PARs (PAs with special receipt, referral, or review considerations), PASs (PAs with set-aside funds), and RFAs. She encouraged NAMSAC members to reach out to OER or NIAMS staff if they have questions.
In response to a question from Dr. Carter, Ms. Mistretta acknowledged there have been cases where Institutes have felt that other Institutes are encouraging applications that are covered by their own areas of concern. Dr. Lester noted that the NOSI is very similar to a program announcement. In response to Dr. James’ question, Ms. Mistretta said OER operates a glossary of all the pertinent acronyms in this area. Dr. James said she had a hard time understanding how the NOSI helps investigators. Dr. Carter replied that though, NIAMS does not use NOSIs very often, in the cases where they have, there’s been in impact to the community.
XI. CONCEPT CLEARANCE
Dr. Lester provided the report on a concept that was put forth by Dr. Heiyoung Park, NIAMS’ representative to the Office on AIDS Research. The concept’s intent is to encourage individuals doing musculoskeletal/autoimmune research to better understand the effects of HIV/AIDS and its treatment on NIAMS’ mission-area diseases. NIAMS is required to spend a certain amount of money on HIV/AIDS initiatives. This investment is monitored by the Office of AIDS Research. Staff sought NAMSAC approval of the following concept:
Promoting Research Opportunities on HIV/AIDS in NIAMS
The purpose of this announcement is to inform potential applicants to National Institute of Arthritis and Musculoskeletal Diseases (NIAMS) of special interest on the area of HIV/AIDS-associated comorbidities that are within the mission area of NIAMS.
The HIV/AIDS pandemic has been a challenging US public health issue. The NIH has provided the largest public investment in HIV/AIDS research globally. In line with overall NIH efforts, NIAMS would like to promote observational, translational, and basic/pre-clinical research focusing on how the presence or treatment of HIV/AIDS impacts systemic rheumatic, musculoskeletal and skin disease progression and pathogenesis with a focus on utilization of existing HIV cohorts and repositories.
XII. P2P UPDATE
Dr. Faye Chen of NIAMS provided the update on the NIH Pathways to Prevention (P2P) workshop on Appropriate Use of Drug Therapies for Osteoporotic Fracture Prevention. The goal of the NIH Office of Disease Prevention (ODP) P2P workshop was to synthesize and interpret evidence and develop an action plan to move the field forward. She characterized the P2P process as long, usually taking 2-3 years. The National Institute on Aging (NIA) and NIAMS are the scientific co-leads with ODP on this effort. A workshop was convened in October 2018. As a result of this workshop as well as a systematic review of available materials on the topic, an unbiased expert panel issued its report with recommendations to advance the field. This report was discussed among the federal partners in summer 2019. The report of the federal partners was still under review as of this Council meeting, but should be published in November/December 2019. Dr. Chen provided a rationale for the holding of a workshop on osteoporotic fracture prevention. Effective therapies are not used as much as they could or should be, perhaps due to insufficient knowledge about how to use them appropriately. Fracture rates have leveled off in recent years following a steady decline, alarming the osteoporosis community which considers this a crisis situation. The workshop was built around four key areas: short-term use of drug therapies; long-term use of drugs; drug holidays as a means of decreasing adverse events in long-term use of bisphosphonates and denosumab; and patient and clinician factors that could impact the use of and adherence to osteoporosis drugs. Importantly, use of and adherence to drug therapies is low. The independent panel derived five research areas that could move the field forward, having to do with innovative study designs; multi-component interventions with reduced adverse events but improved efficacy; prevention of rare but serious adverse events; drug holidays and sequential therapies; and barriers to access of drug therapies. The federal partners also developed goals in the short, medium and long term, as well as action items for closing the knowledge gaps. As the report was still under review, Dr. Chen was unable to go into details about the federal partners meeting.
Dr. James expressed concern that, while recommendations and research would help, it will take 5-10 years from now to see their benefits. She asked about implementation of strategies to prevent falls and to help physicians and patients use medications appropriately in the short term. Dr. Chen said fall prevention was discussed at the federal partners’ meeting. NIAMS has signed onto an NIH dissemination and implementation FOA. Noting that this P2P effort was undertaken in response to interest from then-members of the NAMSAC, Dr. Carter’s intention for this presentation was to show current NAMSAC members that the process is still moving forward. The amount of research that is called for is overwhelming. This is a clear public health need. Dr. Westendorf said part of the challenge is increasing awareness beyond the bone community. Mr. Del Gaizo said that, as effective treatments are put into practice, people begin to fear the treatment more than the condition. Dr. Econs noted the challenge associated with taking a drug to prevent something as opposed to treating something; he compared it to challenges that the cardiology community faces regarding statin use.
The Advisory Council stood in recess at 12:24 p.m., to reconvene in closed session.
XIII. BOARD OF SCIENTIFIC COUNCELORS REPORT
This portion of the meeting occurred during closed session.
XIV. SUCCESS STORIES: SBIR AND STTR PROGRAM
This portion of the meeting occurred during closed session.
XV. SPECIAL ACTIONS
This portion of the meeting occurred during closed session.
En bloc concurrence was unanimously approved for 750 primary and 445 secondary applications. 50 applications were taken to Early Concurrence. The total cost requested in the first year for all applications was $575,263,256.
The 99th National Arthritis and Musculoskeletal and Skin Diseases Advisory Council Meeting adjourned at 3:00 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.
Robert H. Carter, M.D