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Advisory Council Minutes
September 8, 2014
Department of Health and Human Services
Public Health Service
National Arthritis and Musculoskeletal and Skin Diseases Advisory Council
Minutes of the 84th Meeting
September 8, 2014
8:30 a.m. to 2:45 p.m.
CALL TO ORDER
The 84th meeting of the National Arthritis and Musculoskeletal and Skin Diseases Advisory Council (NAMSAC) was held on September 8, 2014, 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).
Council members present
Dr. Lynda F. Bonewald
Dr. David R. Eyre
Dr. Gary S. Firestein
Dr. Sherine Gabriel
Dr. Gary A. Koretzky
Dr. Ted Mala
Dr. Katherine Mathews (via teleconference)
Dr. Martha M. Murray
Dr. Grace Pavlath (via teleconference)
Dr. Alice P. Pentland
Dr. Gwendolyn L. Powell Todd
Dr. Anthony E. Rankin
Dr. Christy I. Sandborg
Dr. Elizabeth Shane
Mr. Alexander Silver
Ms. Elizabeth Smith (via teleconference)
Dr. Xiao-Jing Wang
Staff and Guests
The following NIAMS staff and guests attended:
Mr. Steven Austin
Dr. Carl Baker
Ms. Pamela Beheler
Ms. Elizabeth Bouras
Dr. Amanda Boyce
Mr. Gahan Breithaupt
Dr. Stephanie Burrows
Ms. Justine Buschman
Dr. Robert Carter
Dr. Faye Chen
Dr. Ricardo Cibotti
Mr. Richard Clark
Ms. Robin DiLiello
Dr. Jonelle Drugan
Ms. Elizabeth Elliott
Dr. Nancy Garrick
Ms. Gail Hamilton
Ms. Katie Joffee
Dr. Stephen I. Katz
Ms. Mary Beth Kester
Ms. Stephanie Kreider
Ms. Colleen Labbe
Mr. Mark Langer
Dr. Gayle Lester
Dr. Helen Lin
Ms. Anita Linde
Ms. Mimi Lising
Ms. Leslie Littlejohn
Dr. Kan Ma
Dr. Marie Mancini
Dr. Su-Yau Mao
Dr. Kathryn Marron
Dr. Joan McGowan
Ms. Leslie McIntire
Dr. Laura K. Moen
Ms. Regina Mong
Ms. Melinda Nelson
Ms. Anna Nicholson
Dr. Glen Nuckolls
Dr. John O’Shea
Dr. James Panagis
Ms. Vivian Pham
Ms. Reaya Reuss
Ms. Trish Reynolds
Dr. Kathy Salaita
Dr. Susana Serrate-Sztein
Dr. William Sharrock
Dr. Richard Siegel
Ms. Sheila Simmons
Dr. Kentner Singleton
Ms. Allisen Stewart
Ms. Yen Thach
Ms. Jamie Thompson
Dr. Phil Tonkins
Dr. Hung Tseng
Dr. Bernadette Tyree
Ms. Lisa Walker
Dr. Fei Wang
Dr. Xibin Wang
Dr. Yan Wang
Dr. Chuck Washabaugh
Ms. Sara Rosario Wilson
Dr. James Witter
Dr. Xincheng Zheng
Dr. Aruna Behera, Center for Scientific Review, NIH
Ms. Gwendolyn Carr, Office of the Director, NIH
Dr. Ellen Gadbois, Office of the Director, NIH
Dr. Ellen Goldmuntz, National Institute of Allergy and Infectious Diseases, NIH
Ms. Kim Holmes, IQ Solutions
Ms. Leah Howard, National Psoriasis Foundation
Dr. Kathy Hudson, Office of the Director, NIH
Ms. Anna Hyde, Arthritis Foundation
Dr. Joseph Laakso, Endocrine Society
Dr. Patrick Lai, Center for Scientific Review, NIH
Dr. Dawei Lin, National Institute of Allergy and Infectious Diseases, NIH
Dr. David Lipman, National Library of Medicine, NIH
Mr. Simit Pandya, American Academy of Orthopaedic Surgeons
Dr. John Peyman, National Institute of Allergy and Infectious Diseases, NIH
Mr. Nate Robinson, IQ Solutions
Ms. Michelle Rodrigues, SRI International
Ms. Kirstie Saltsman, IQ Solutions
Ms. Darlene Summers, Consolidated Solutions and Innovations
Mr. Richard Taffet, Ehlers-Danlos National Foundation
CONSIDERATION OF MINUTES
A motion was made, seconded, and passed to accept with no changes the minutes of the 83rd NAMSAC meeting, held on June 3, 2014.
FUTURE COUNCIL MEETING DATES
Future Council meetings are currently planned for the following dates:
February 4, 2015
June 16, 2015
September 8, 2015
February 2, 2016
June 7, 2016
September 13, 2016
DIRECTOR'S REPORT AND DISCUSSION
Dr. Katz began his Director’s Report by welcoming Council members, NIAMS staff, and guests. He thanked the following Council members whose four-year terms are expiring with this meeting:
- Dr. Lynda Bonewald, Lefkowitz Professor in the Department of Oral Biology at the University of Missouri-Kansas City
- Dr. David Eyre of the Department of Orthopaedics and Sports Medicine at the University of Washington School of Medicine
- Dr. Gary Firestein, Professor in the Department of Medicine at the University of California, San Diego School of Medicine
- Dr. Ted Mala, Director of the Traditional Healing Clinic at the South Central Foundation
- Dr. Alice Pentland, James H. Sterner Professor and Chair in the Department of Dermatology at the University of Rochester School of Medicine and Dentistry
Dr. Katz noted that the Institute is grateful to each of them for their exemplary service and their many contributions to the Council’s deliberations.
Update on Budget and Congressional Activities
Dr. Katz reminded the group that on October 1, the Federal government will begin its new fiscal year (FY). It is anticipated that FY 2015 will begin with a continuing resolution that is likely to extend through early-to-mid December. Although many discussions and votes regarding the final budget still need to occur, the Senate Appropriations Subcommittee on Labor, Health and Human Services, Education and Related Agencies has publically expressed strong support for the NIH.
On June 23, 2014, Dr. Katz and NIAMS Deputy Director Dr. Robert Carter met with the Majority and Minority Clerks of the Senate Appropriations Subcommittee on Labor, Health and Human Services, and Education. The meeting was part of a series that the Clerks requested with Institute and Center (IC) Directors to expand their knowledge about the many NIH components that do not often get the opportunity to testify at the appropriations hearings.
Also this summer, the American Academy of Orthopaedic Surgeons (AAOS) arranged for Dr. Katz to join Dr. Joshua Jacobs (AAOS Past President and former NIAMS Advisory Council member) for a full day of visits with eight different Congressional offices. Like the Appropriations Clerks, members and their senior staff were generally supportive of NIH’s mission and the contributions that biomedical research makes to the United States’ public and economic health.
Dr. Katz also joined National Heart, Lung, and Blood Institute (NHLBI) Director Dr. Gary Gibbons for a Hill briefing organized by the Marfan Foundation. Representatives Steve Israel (D-NY) and Michael Fitzpatrick (R-PA) sponsored the event, which was titled “Recent Advancements and Emerging Opportunities in Marfan Syndrome and Related Disorders Research.” That same week, Dr. Katz attended the Marfan Foundation Family Conference.
Dr. Katz reported that the NIAMS continues to work with NIAMS Coalition members to identify ways that the Institute can educate elected officials and the public about the importance of biomedical research. On the day following this Council meeting, Dr. Carter will represent the NIAMS at a briefing organized by the Lupus Foundation of America and the Rheumatology Research Foundation to update members of Congress about the Accelerating Medicines Partnership (AMP). The AMP is a public-private partnership to speed drug development by identifying molecules and pathways that are likely to respond to new therapies. The 5-year pilot phase of the effort focuses on Alzheimer’s disease, type 2 diabetes, lupus, and rheumatoid arthritis. The Institute is also working with members of the NIAMS Coalition Steering Committee to plan an October NIH Campus and lab tour for interested Congressional staffers. Dr. Katz noted that staffers value these opportunities to learn first-hand about the research supported by tax dollars.
Overview of NIH and NIAMS Activities and Plans
Personnel Changes at the NIH/NIAMS
At the NIH level:
- Mr. Pat White, former NIH Associate Director for Legislative Policy and Analysis, recently left the NIH to lead a new advocacy organization, called ACT for NIH. ACT for NIH’s goal is to reverse the dramatic declines that the NIH budget has experienced in recent years and secure steady, predictable budget growth in the future. At its meeting on February 5, 2013, Mr. White briefed the NAMSAC about the impact of the 2012 elections on NIH’s authorizing and appropriating committees.
- NIH Director Dr. Francis Collins has appointed Dr. Amy Patterson, former NIH Associate Director for Science Policy, as NIH’s Associate Director for Biosecurity and Biosafety Policy. Dr. Patterson will lead NIH’s participation in the complex area of “dual use research of concern,” which is the term used for life-sciences research that, if misapplied, could pose a considerable threat to the public health, the environment, or national security. Dr. Patterson will also be deeply involved in new efforts to address the growing public health problem of antibiotic resistance.
- Dr. Story Landis is retiring as Director of the National Institute of Neurological Disorders and Stroke (NINDS) this month. The NIAMS has had many interactions with Dr. Landis over the years, especially in the area of muscular dystrophy research. Dr. Landis has led the NINDS since 2003 and was the NINDS Scientific Director from 1995 to 2003. Dr. Walter Koroshetz, the Deputy Director of NINDS since 2007, will serve as Acting Director while the NIH conducts a national search to fill the position.
At the NIAMS level, Dr. Glen Nuckolls of the NIAMS Division of Musculoskeletal Diseases has accepted a position as Program Director in the NINDS Extramural Program’s Neurogenetics Cluster. Dr. Nuckolls has contributed greatly to the NIAMS for the past 19 years—8 years in the NIAMS Intramural Research Program and 11 years in the Extramural Program, first as a Scientific Review Officer and then as the Director of NIAMS’ Muscle Disorders and Therapies Program. In 2008, he served as Acting Director of the NIAMS Division of Extramural Research Activities (DERA) while the Institute searched for a permanent DERA Director.
Update on NIH/NIAMS Activities
Dr. Katz, who is involved with the NIH Scientific Management Review Board (SMRB), noted that the SMRB was recently charged by Dr. Collins with recommending ways to optimize NIH’s pre-college programs and initiatives that align with the NIH mission and ensure a continued pipeline of biomedical science students and professionals. A working group has been formed and charged with: (1) examining the evidence base for successful pre-college biomedical science programs; (2) determining the attributes, activities, and components of effective pre-college biomedical science programs, including the role and relative importance of training programs for teachers; (3) identifying points in the pre-college biomedical workforce pipeline where NIH’s efforts could be applied most effectively, given finite resources; and (4) defining ways for NIH to improve the evidence base for effective pre-college biomedical science programs.
The SMRB also has recently established a working group to recommend strategies that the NIH can implement to reduce the amount of time between submission of a grant application and receipt of the award. Data show that the time from application to award is approximately 1.2 years. The average time from application submission to the receipt of summary statements for Center for Scientific Review (CSR) reviewed applications is 4.5 months. Dr. Katz assured Council members that he will continue to keep them informed about these activities.
In his blog, Dr. Collins has posted an outline of NIH’s overall strategy for testing several pilot projects to address the changing needs of the biomedical workforce. Participating Institutes and Centers (ICs) are using different approaches. For example, the National Cancer Institute (NCI) is inviting investigators to consolidate their NCI grants into one award with a 7-year project period and up to $600,000 in direct costs per year. The National Institute of General Medical Sciences (NIGMS) is considering a program that would consolidate an investigator’s NIGMS funding into one 5-year grant. Dr. Katz noted that later in this meeting’s agenda, Dr. Susana Serrate-Sztein, Director of the NIAMS Division of Skin and Rheumatic Diseases, would be presenting the draft concept of NIAMS’ plans related to this effort. The Institute’s proposed approach targets investigators who have received their first competitive renewal, or type 2, grant.
Dr. Katz reported that after an extensive public comment period, the NIH released its Genomic Data Sharing policy on August 27, 2014. Developed to promote data sharing as a way to speed translation while protecting the privacy of research participants, a key tenet is that researchers will seek the broadest possible sharing permissions from participants for future research use of their data. The policy, which will be in effect starting with the January 25, 2015, receipt date, will apply to all NIH-funded, large-scale human and non-human projects that generate genomic data.
Dr. Katz briefly discussed NIH plans and forthcoming policies regarding the importance of including sex differences in the design and reporting of preclinical studies. The exact details and timelines will become available as the NIH begins rolling out the policies over the coming months. The overarching goal is to respond to the growing body of evidence that the sex of the animals or cells being studied may influence outcomes. The new policies will formalize the importance of considering the sex of the animals or cells being studied when developing research questions, designing experiments, analyzing data, and reporting results.
Dr. Katz expressed gratitude to the Council members who provided input into the latest edition of the NIAMS Long-Range Plan, which will be available on the NIAMS website. Council input gathered after the June 3, 2014, NAMSAC meeting has been incorporated into the Plan, as has feedback from researchers and NIAMS Coalition member organizations. The 2015 version of the NIAMS Long-Range Plan will: (1) communicate the Institute’s perspective on research needs and opportunities within the NIAMS mission, (2) encourage scientists to submit their best research ideas in the form of investigator-initiated grant applications, and (3) serve as a resource for all who are interested in NIAMS activities.
Dr. Katz also provided an update on the NIAMS Intramural Research Program Long-Range Plan. He reminded Council members that at the June 3, 2014, NAMSAC meeting, Council member Dr. Gary Koretzky, Dean of Weill Cornell Graduate School and Senior Associate Dean for Research at Weill Cornell Medical College, described the NIAMS Intramural Research Program’s planning process. NIH-wide, the Intramural Research Program is developing a 10-year vision for how laboratory science expertise, clinical resources, and population-based programs can be used to advance basic biomedical research and clinical applications. NIAMS’ component of the plan has been sent to the NIH Director’s Office, where it will be integrated into a single NIH report that will be discussed at the December meeting of the Advisory Committee to the NIH Director (ACD). Dr. Katz noted that a subgroup of Council members have been asked to review the administrative role of the NIAMS Intramural Research Program Scientific Director (Dr. John O’Shea).
Dr. Katz emphasized that the NIAMS strives to inform Council members about a range of NIAMS and NIH endeavors. Council members are relied upon to advise the Institute on broad policy issues. He stated that the NIAMS is committed to providing the Council with the background necessary for each member to be knowledgeable about the Institute’s research and research support activities and functions. Dr. Katz noted that later in the meeting, Council members would be invited to discuss how the Institute might keep them better informed regarding NIAMS efforts and its scientific portfolios.
Overview of NIH and NIAMS Activities and Plans
NIH wide, the Intramural Research Program (IRP) is developing a 10-year vision for how it can continue to use its valuable laboratory science expertise, clinical resources, and population-based programs to advance basic biomedical research and clinical applications. Council member Dr. Gary Koretzky (Dean of Weill Cornell Graduate School and Senior Associate Dean for Research at Weill Cornell Medical College) has been working closely with Dr. John O’Shea (Scientific Director of NIAMS’ Molecular Immunology and Inflammation Branch), Dr. Joseph Craft (Paul Beeson Professor of Medicine, Yale School of Medicine, and Chair of the NIAMS Board of Scientific Counselors), Dr. Katz, and other NIAMS staff to focus the Institute’s contribution to this effort.
Council members were encouraged to review a recent editorial in the Proceedings of the National Academy of Sciences that describes challenges facing the U.S. biomedical research enterprise. The authors’ assessment of the workforce and funding issues facing the academic research community, as well as recommendations to address them, is generating considerable discussion both at the NIH and in the wider community.
The NIH strives to ensure that all applicants are treated fairly by the peer review process. In response to research suggesting discrepancy in success rates for NIH R01 grant funding between white and African-American applicants, NIH’s Center for Scientific Review (CSR) is looking for rigorously constructed, well-designed, and creative proposals for: (1) new methods to detect bias in peer review, and (2) strategies to strengthen fairness and impartiality in peer review. Proposals are due at the end of the month.
With regard to NIAMS information dissemination efforts, several NIAMS staff members have spoken with various media outlets since the February Council meeting. Last month, Drs. Jim Witter (Health Scientist Administrator in the NIAMS Division of Skin and Rheumatic Diseases) and Gayle Lester (Health Scientist Administrator in the NIAMS Division of Musculoskeletal Diseases) joined colleagues from the National Center for Complementary and Alternative Medicine (NCCAM), National Institute on Aging (NIA), the Mayo Clinic, the Arthritis Foundation, and the American College of Rheumatology to answer questions about OA, RA, and gout during a Twitter chat. Ms. Liz Szabo, a medical reporter with USA Today, hosted the event, which reached more than 21 million Twitter users. Dr. Katz reported that the AMP also generated considerable media coverage in addition to the Capitol Hill events he described previously. In addition, Dr. Katz participated in a Washington Journal segment during which he highlighted ongoing research and recent advances.
Highlights of Selected Recent Scientific Advances
- A paper from Dr. Renate Pilz of the University of California, San Diego, describes the identification of a thyroid hormone receptor variant that is important for bone formation. The binding of thyroid hormone to the variant receptor triggers a series of events that eventually helps to increase the growth of osteoblasts and protects the osteoblasts and osteocytes from death. When combined with what is already known about the complex effects of thyroid hormone on bone, these latest findings suggest that the alternative, membrane-based signaling pathway might have considerable therapeutic potential (Sci Signal. 2014 May 20; 7(326):ra48. doi: 10.1126/scisignal.2004911. PMID: 24847117).
- Dr. Florent Elefteriou and colleagues, most of whom are at Vanderbilt University, determined that the skeletons of mice lacking functional neurofibromin in the cells that produce bone and cartilage resembled those of people who have the disease neurofibromatosis. After the investigators determined that neurofibromin deficiency results in inadequate levels of an enzyme called alkaline phosphatase, they attempted to increase the amount of enzyme to ameliorate some of the disease’s skeletal consequences. The synthetic form of alkaline phosphatase that restored bone mineralization in their mouse experiments had been developed to treat people with defects in the gene for alkaline phosphatase. Called asfotase-alpha, the treatment has already been shown to improve the bones of children with this genetic disease, a condition known as hypophosphatasia (Nat Med. 2014 Aug; 20(8):904-10. doi: 10.1038/nm.3583. Epub 2014 Jul 6. PMID: 24997609).
- Researchers are identifying structural differences that predict skeletal fragility in postmenopausal women whose bone mass—while lower than normal—is not so low that it is characterized as osteoporosis. A team of investigators led by Council member Dr. Elizabeth Shane, Professor of Medicine and Vice-Chair for Clinical and Epidemiological Research at Columbia University, used high-resolution imaging and mathematical modeling methods to analyze bones in the distal radius of the arm and the distal tibia of the leg. Women in the fracture group had fewer plate-like structures and fewer connections between rod-like structures in their arm bones compared with women with similar bone mineral density measurements without a history of fractures (J Bone Miner Res. 2014 May;29(5):1101-9. PMID: 24877245).
- An article from Dr. Krista Vandenborn’s team at the University of Florida describes a magnetic resonance spectroscopy method for assessing the fat content in the lower limb muscles with measures (Neuromuscul Disord. 2014 Jul;24(7):574-82. doi: 10.1016/ j.nmd.2014.03.013. Epub 2014 Apr 13. PMID: 24798221). Another paper, this one from Seward Rutkove of Harvard Medical School, describes improvements to an electrical impedance myography approach that allows researchers to more accurately assess muscle quality by decreasing the interference from subcutaneous fat (Clin Neurophysiol. 2014 May 17. pii: S1388-2457(14)00256-9. doi: 10.1016/j.clinph.2014.05.007. [Epub ahead of print] PMID: 24929900). Results from both approaches correlate with lower limb function in boys who have Duchenne muscular dystrophy, a disease in which skeletal muscle tissue is replaced by fat and fibrous connective tissue.
- Work by Dr. Judith James and her group at the University of Oklahoma Health Sciences Center brings the field a step closer to developing biomarkers that predict lupus flares. The investigators compared 52 molecules in blood samples from lupus patients whose disease flared during the study, from patients who did not flare, and from healthy volunteers. Compared with patients who have clinically stable disease, lupus patients with impending flares had higher levels of proinflammatory cytokines and lower levels of regulatory mediators, suggesting that the balance between inflammatory and regulatory factors is altered before a lupus flare. The findings could result in earlier and more effective treatment, or even prevention, of lupus flares (Arthritis Rheumatol. 2014 Jul;66(7):1888-99. doi: 10.1002/ art.38573. PMID: 24578190).
- A team of investigators led by Dr. Mariana Kaplan of NIAMS’ Intramural Research Program recently identified some of the molecules that make the cells that line the blood vessels more susceptible to the clots and cholesterol plaques that cause heart attacks and strokes in patients with systemic lupus erhythematosus. Previous research demonstrated that an innate immune system response to invading microorganisms—the formation extracellular fibers called neutrophil extracellular traps (NETs)—is enhanced in lupus patients, even in the absence of pathogens. The current research has shown that the high concentrations of NET fibers and associated enzymes trigger oxidative changes to high density lipoproteins (the type of cholesterol typically thought of as “good”) (Arthritis Rheumatol. 2014 May 16. doi: 10.1002/art.38703. [Epub ahead of print]. PMID: 24838349).
- A research team led by Dr. Farshid Guilak, in collaboration with investigators in the Department of Orthopaedic Surgery at Duke University, determined that inhibiting the inflammatory cytokine interleukin-1 (IL-1) prevents cartilage degeneration and joint inflammation following injury in a mouse model of post-traumatic osteoarthritis. These results add to the body of evidence that suggests the blockage of inflammation immediately following a joint injury could halt or slow the degenerative changes that lead to osteoarthritis, thereby preventing or delaying the pain and disability that characterize later stages of the disease (Arthritis Res Ther. 2014 Jun 25; 16(3):R134. [Epub ahead of print] PMID: 24964765).
- A team lead by Johns Hopkins University’s Dr. Jennifer Elisseeff developed a strategy to bind hyaluronic acid, a lubricating molecule naturally found in the fluid that bathes the knee joint, to a protein fragment that interacts with cartilage. Tests on tissues and in animals show that the altered cartilage bound hyaluronic acid twelve-fold more readily than normal cartilage, and the bound lubricant reduced friction as effectively as the viscosupplementation currently given to treat patients. Although the strategy still needs further testing before it can be used with patients, the approach suggests a new way to ease the pain of arthritic knees, shoulders, elbows, and hips without the repeated injections needed today (Nat Mater. 2014 Aug 3. doi: 10.1038/nmat4048. [Epub ahead of print] PMID: 25087069).
- Researchers have long wondered why itch can be relieved by other skin sensations, or counter-stimuli, such as cold, heat, and pain. A new study has identified a neural circuit that explains, at least in part, how menthol’s cooling sensation relieves itch. A research team led by Dr. Sarah Ross at the University of Pittsburgh, in collaboration with colleagues from across the globe, found that a specific group of inhibitory nerve cells—called the B5-I cells—interacts with menthol-sensing cells to trigger the release of dynorphin, a chemical that blocks the itch signal (Neuron. 2014 May 7;82(3):573-86. doi: 10.1016/ j.neuron.2014.02.046. Epub 2014 Apr 10. PMID: 24726382).
- Skin cancer incidence is still on the rise, despite the fact that the link between skin cancer risk and chronic UV exposure is well known. A new study sheds light on the biological basis of tanning addiction and may lead to new prevention strategies and ultimately, reduce skin cancer incidence. A research team led by Dr. David Fisher at the Massachusetts General Hospital showed that in mice, daily UV exposure at doses sufficient to cause sunburn triggers keratinocytes to release ß-endorphin, an endogenous opioid that addicts the mice to UV light. The UV-treated mice showed a higher threshold for pain than untreated mice, as well as signs of addiction, such as opioid tolerance and dependence (Cell. 2014 Jun 19;157(7):1527-34. doi: 10.1016/j.cell.2014.04.032. PMID: 24949966).
- Ongoing work in the NIAMS Intramural Research Program is improving the understanding of inflammation pathways and how certain mutations can lead to chronic inflammatory signaling. Dr. Raphaela Goldbach-Mansky and an international team of researchers have described the genetic cause of a novel pediatric autoinflammatory disorder called stimulator of interferon gamma (STING) associated vasculopathy with onset in infancy (SAVI). SAVI is characterized by systemic inflammation, especially in blood vessels, as well as blistering rashes and severe scarring of the lungs (N Engl J Med. 2014 Aug 7;371(6):507-18. doi: 10.1056/NEJMoa1312625. Epub 2014 Jul 16. PMID: 25029335).
Highlights of NIAMS Information Dissemination Efforts
As part of the Institute’s efforts to engage with patients and the general public, the NIAMS launched a new page on the social media site Google Plus. Concurrently, the NIAMS established a dedicated channel on YouTube, Google’s video-sharing subsidiary. Google Plus and YouTube will provide a single, convenient platform for NIAMS stakeholders to view NIAMS video resources and other informational content. Complementing existing Facebook and Twitter pages, Google Plus broadens NIAMS’ reach to members of the “millennial generation.” It also has the benefit of improving the placement of NIAMS web content in the Google search engine, allowing many more people who are looking for credible sources of health information to quickly find the Institute’s resources.
Dr. Katz reported that “Life: Magnified,” a display of 46 biologic images that have been colorized and magnified up to 50,000-fold, can be seen at Dulles International Airport. He presented a slide showing one of the photographs featuring skin cancer research conducted by NIAMS grantees Dr. Markus Schober of New York University and Dr. Elaine Fuchs of Rockefeller University. The exhibit closes in November 2014, and the images are available online.
As part of the Institute’s effort to showcase the important work that researchers are able to conduct with NIAMS funding, members of the scientific community are being asked to submit scientific visuals that can be used in a variety of the Institute’s print and digital communications to increase the public’s appreciation for and understanding of research in diseases related to the bones, joints, muscles, and skin. More information about the types of photographs and videos that the NIAMS is looking for is outlined in Dr. Katz’s Director’s Letter in this month’s Shorttakes—the compilation of NIH and NIAMS news items that the Institute’s Office of Science Policy, Planning and Communications (OSPPC) prepares for each Council meeting. Dr. Katz noted that additional information about many of the activities, personnel changes, outreach efforts, and scientific discoveries that he discussed in his Director’s Report can be found in Shorttakes and elsewhere on the NIAMS website.
Dr. Shane explained that her research on skeletal fragility in postmenopausal women described by Dr. Katz in his Director’s Report was supported by NIAMS through a U01 grant. It enabled her team to use a novel scanner to characterize the microarchitecture of bone and enabled collaboration with bioengineering colleagues at Columbia University to better understand what it is about bone quality that is different among fracture patients and non-fracture patients. Women in the study were well matched in terms of bone density, age, and all other parameters. Those who had a fracture had fewer trabecular plates, which are associated with bone strength, as well as more rods and less connectivity between rods. This work has led to a greater understanding of what happens in the trabecular compartment that can make women more susceptible to fractures. She suggested that this work could be used to help determine which women with modestly low bone mineral density are at greatest risk for fracture so that therapies can be targeted appropriately.
Dr. Katz commented that the Bone Quality Project, led by Dr. Gayle Lester (Health Scientist Administrator in NIAMS’ Division of Musculoskeletal Diseases), has been initiated to advance the qualification of biomarkers for drug development and patient management in osteoporosis. It aims to evaluate osteoporosis biomarkers obtained from retrospective clinical trials and qualify them for use in drug development. This activity involves collaboration with the Foundation for the NIH (FNIH), American Society for Bone and Mineral Research (ASBMR), the U.S. Food and Drug Administration (FDA), and industry partners.
MODERNIZATION OF CLINICAL TRIAL ACTIVITIES AND OTHER HUMAN SUBJECTS RESEARCH
Dr. Hudson discussed how the NIH is trying to modernize the policies governing clinical trials. She reminded the group that NIH’s goals in this regard are to: (1) increase data sharing, (2) evolve relationships between researchers and participants, and (3) enhance efficiency/reduce burden on investigators and institutions conducting human subjects research. These goals are being pursued through the reform of human subjects research policies, advances in data sharing policies, and enhancements to clinical trials (particularly in terms of modernizing NIH’s clinical trials monitoring and oversight systems).
Dr. Hudson presented a timeline outlining the progression and evolution of policies to protect participants in human subjects research. She noted that in 1981, the policy for the U.S. Department of Health and Human Services (HHS) was finalized and there has been no significant advancement in HHS’ policy since that time. In 1991, due in large part to aggressive efforts of NIH staff, 17 Federal agencies signed on to the Common Rule for the protection of human subjects in research. Work is now underway to update the Common Rule, given the fact that the nature of research has changed in many ways (e.g., the type of research conducted, the risks posed to research participants, and the scale of research).
Overarching goals associated with trying to reform the Common Rule include enhancing the protection of/respect for research subjects and increasing the efficiency of the oversight processes. There has been no change in the legal/regulatory requirements for informed consent since 1981, and yet the documents associated with informed consent continue to increase in length and complexity. Dr. Hudson noted that steps can be taken to streamline this process.
Soon after President Obama came into office, he directed senior leaders to modernize the Common Rule. An Advanced Notice of Proposed Rulemaking (ANPRM) was issued in July 2011 and proposed the following seven key reforms to human subjects protection regulations:
- Refine the existing risk-based framework
- Utilize single Institutional Review Boards (IRBs) for domestic multi-site studies
- Establish a single source of guidance on federal regulations
- Establish mandatory data security and information protection standards
- Establish a systematic approach to the collection and analysis of adverse events
- Extend protections to all research conducted at institutions receiving federal funding
- Informed consent (require consent for use of data and specimens)
Dr. Hudson discussed the proposal to utilize single IRBs for domestic multi-site studies, explaining that multiple IRB reviews do not appear to enhance the protection of participants.
Additionally, multiple IRB reviews increase costs and cause excessive delays. It has been suggested that moving to a single IRB of record can potentially focus the attention of the IRB of record in a beneficial way.
Because reforms to the Common Rule must pass through a number of Federal agencies. Dr. Collins has asked that the NIH examine what it can do through NIH policy to encourage or require the use of a single IRB for multi-site studies. The NIH already uses a single IRB in a number of contexts. For example, the NCI uses a central IRB that has a relatively fast turn-around time. The National Children’s Study has been using a central IRB, the NINDS uses a multi-site infrastructure for clinical research called NeuroNEXT, and the Clinical and Translational Science Award (CTSA) program has proposed using a single IRB process.
Dr. Hudson then discussed the informed consent portion of the ANPRM, which proposed and asked for public comments on the notion of asking people for their permission to use their clinically collected data and/or specimens in research (primary or secondary). There is a clearly emerging strong patient preference to set some parameters early on regarding how broadly one’s data and biospecimens can be shared. Dr. Hudson suggested that asking for permission to use patients’ data/specimens can facilitate science. She explained that the research enterprise is bigger than ever, with more data, more collaboration, and more sharing. Obtaining broad consent will facilitate the sharing of specimens, combining data from many studies, and enhancing researchers’ ability to re-connect with study subjects. Asking for permission is a powerful way of showing respect to study subjects. Dr. Hudson also noted that “non-identifiability” is a myth today; it is now relatively straightforward to re-identify individuals in specific cohorts. Asking for consent also makes the research endeavor more of a partnership between the investigators and subjects.
Dr. Hudson told the story of Henrietta Lacks and the use of her biopsied cells without her knowledge to generate the first widely used human cell line. The NIH worked with the family and researchers to develop a controlled access policy to this cell line.
NIH’s Genomic Data Sharing Policy was published in final form on August 28, 2014, and will be implemented in January 2015 for FY 2016 funding. It expands the current genome-wide association studies (GWAS) policy to large-scale non-human and human genomic data. NIH-funded researchers are expected to share genomic research data, and for human data, researchers should obtain consent for data to be used for future research purposes and shared broadly.
Dr. Hudson reminded Council members that roughly 12% of the NIH budget is used to support clinical trials. NIH’s Clinical Trials Working Group (CTWG) has been examining the entire series of events required to conduct and report on clinical trials. A number of ICs have implemented policies requiring specific types of data sharing in different clinical trials programs, including sharing of patient-level data from clinical trials. The NIH does not have a single repository for this type of data, and this will be an increasing need in the future. The Institute of Medicine (IOM) is looking across the government and private sector to examine what work is needed to advocate for and require the sharing of patient-level data from clinical trials. IOM’s report on this subject is expected to be released in December 2014.
The CTWG has made a number of recommendations, including streamlining IRB review by requiring the use of a single IRB for multi-site studies, and optimizing the value of trials by requiring results reporting for all NIH-funded clinical trials. Dr. Hudson cited a 2012 study published in the British Medical Journal showing that of NIH-funded clinical trials published within 100 months of completion, fewer than 50% are published within 30 months of completion. NIH’s own data show the same trend. A more recent study in PLOS showed that studies supported by industry are posted to clinicaltrials.gov more frequently than those not supported by industry.
Dr. Hudson noted that clinical trial data sharing/dissemination is required by law for “applicable” clinical trials. The NIH has developed proposed regulations to implement this law and will be seeking public comment soon. The NIH is also contemplating additional measures to ensure that the results of every trial conducted with NIH dollars are shared. Dr. Hudson reviewed the public benefits associated with sharing data from clinical trials (e.g., posting to clinicaltrials.gov), noting that all of them contribute to enhancing public trust in clinical research:
- Enhancing patient access to information about open and enrolling trials
- Preventing unnecessary or unwitting duplication of trials, especially those found to be unsafe
- Honoring ethical obligations to participants
- Mitigating reporting bias (non-publication of negative results)
- Informing future research and funding decisions
- Increasing access to data about marketed products
- Facilitating the use of findings to improve health
Dr. Hudson explained the scope of the FDA Amendments Act (FDAAA). Through the compliance provisions of the FDAAA, the NIH must verify submission of information before releasing any remaining funds for a grant or funds for a future grant (grant and progress report forms must certify information has been submitted) and must provide notice of non-compliance. The FDA is authorized to levy monetary penalties, and the NIH is considering ways to increase compliance. Dr. Hudson noted that Phase 1 trials, observational studies, and studies that do not involve drugs, biologics, or devices are not covered by the FDAAA. At the time of her presentation, Dr. Hudson noted that there were 5,864 NIH applicable clinical trials registered in clinicaltrials.gov. Of these, 2,728 should have posted results. She explained that 1,119 (41%) have posted results, while 1,609 (59%) have not (i.e., these trials are more than 2 years post completion and have not posted results). These data illustrate the need for NIH ICs to work together to ensure that the results of applicable clinical trials are posted in a timely manner. Dr. Hudson noted that some supplementary funds from the Director’s Discretionary Fund are being used to enhance and streamline the process for submitting data to clincialtrials.gov.
Dr. Koretzky expressed support for NIH’s efforts in this area. He asked if the NIH has plans to ensure that the data that are posted to clinicaltrials.gov are reviewed in some manner. He also expressed concern that a move to a single IRB may overshadow unique institution- or investigator-specific issues that could be better addressed by the institution itself. Dr. Hudson observed that sometimes, even data that have been peer reviewed are found not to be reliable. To some extent, there is a community responsibility to understand how the data were generated and its validity. It is likely that there will be some opportunity for self-correction built into the process. A way to annotate data is also being considered. With regard to Dr. Koretzky’s concerns about a move to a single IRB, Dr. Hudson commented that institution- or investigator-specific issues might be negotiated before the study begins. She explained that the goal is for the specific responsibilities and requirements under the human subjects protections regulations to be handled in a centralized fashion. Dr. Katz pointed to the need to develop some type of annotation system for clinicaltrials.gov (e.g., to indicate the publication status of a study) and noted that the information housed on clinicaltrials.gov is publicly available. Oversight of this annotation system will require significant effort.
Council member Dr. Sherine Gabriel, Dean of the Mayo Medical School, agreed with Dr. Hudson’s previous remarks regarding how multiple IRBs do not enhance patient protection. She asked if there is any NIH guidance available to help institutions move towards a centralized IRB model. Dr. Hudson commented that an active engagement with institutions regarding roles and responsibilities on the part of NIH is important, as is collecting information from single-IRB systems to learn from their experiences. She also noted that the NIH is funding some research on how to develop reliance agreements between institutions; some of these data may be available to help inform the move to a centralized IRB model.
Council member Dr. Christy Sandborg, Professor of Pediatrics at Stanford University, recounted her experience with a NIAMS-supported, 23-site study on an FDA-approved drug for treating lupus in the pediatric population. The approval time for the 23 separate IRBs ranged from 2 months to more than 18 months. She noted that some IRBs voiced conflicting concerns; this delayed the research considerably. Dr. Sandborg voiced strong support for moving towards a centralized IRB approach, particularly for large, multi-site clinical trials.
Mr. Alex Silver, Chairman of the Jackson Gabriel Silver Foundation and a member of the Council, described the Epidermolysis Bullosa Clinical Research Consortium, which was formed to reach consensus and synchronize IRB processes among research institutions. He noted that the costs to non-profit organizations associated with getting procedures in place just so that research can be conducted are extremely high. He also reminded the group that single-IRB models promote and enhance collaboration among institutions and allow groups such as his to allocate more funds to research activities.
Dr. Gary Firestein noted that he has been involved in trying to move the University of California Consortium to use a single, reliant IRB. He suggested that many of the challenges in doing so are local in nature (e.g., some institutions feel that their local IRBs provide unique oversight that would not be provided by a centralized IRB). The Consortium has agreed to a rotating central IRB solution, which has allayed many of these issues. Dr. Firestein commented that a top-down approach may be necessary at the NIH level (e.g., indicating to an institution that it has to use a central IRB if it wants to participate in/obtain funding for a study). He reminded the group that the primary task of an IRB is not to conduct formal scientific reviews of protocols (which usually have already been vetted by the FDA and an NIH study section), but rather to assess risk and benefit and protect study participants.
Council member Dr. Anthony Rankin, Chief of Orthopaedic Surgery at Providence Hospital and Clinical Professor of Orthopaedic Surgery at Howard University, asked about establishing a single source of guidance for Federal regulations. Dr. Hudson explained that the many different agencies that support human subjects research all abide by the Common Rule but each has issued separate guidance documents. Ideally, a single source of guidance will be developed with consistent terms, definitions, etc. It will be challenging, however, to align all relevant agencies in this regard.
Dr. Richard Siegel, Clinical Director of the NIAMS Intramural Research Program, applauded efforts examining the submission process to clinicaltrials.gov. He expressed concern that the outcome screens for interpreting data on the site (which are formatted as data tables) are not truly explanatory and suggested that more of a summary statement-type explanation of the results might be more appropriate. Dr. William Sharrock, Health Scientist Administrator in NIAMS’ Division of Musculoskeletal Diseases, commented these types of clinical trials issues are similar to those associated with GWAS. He reminded the group that making changes to clincaltrials.gov and databases such as NCBI’s Database of Genotypes and Phenotypes (dbGaP) requires significant time and effort. The submission and controlled access processes impose burdens on the investigator community, the grantee community, and the NIH; more resources will likely be needed to streamline these processes. He also noted that there are a number of emerging datasets not funded by NIH that should still be shared, and that many journals now require that investigators document sharing of their data before allowing their work to be published.
COMMENTS & CONNECTIONS ON PUBMED COMMONS
Dr. Lipman opened his presentation by explaining that PubMed Commons was developed based on feedback from IC advisory boards, well-known experts, and others in the scientific community who expressed a desire to include comments and/or ratings on papers listed in PubMed. PubMed Commons is limited to those researchers who have authored publications already included in PubMed. It began as a pilot project in October 2013; today, there are roughly 7,000 members who have provided almost 2,000 comments.
In addition to limiting comments only to authors of publications in PubMed, cornerstones of PubMed Commons include:
- No anonymous accounts and no pseudonyms
- Rating of comments, but not of publications
- Post-comment moderation only
- Creative Commons license
- Staged, evaluated piloting (i.e., every several months, the NCIB will prepare a report to IC Directors and Dr. Collins with any suggested changes)
Dr. Lipman explained that at its onset, there were different notions of what value could be gained from comments in PubMed Commons (e.g., evaluating the quality of a paper, identifying and bringing attention to potential problems, identifying papers that may not have received enough attention on the part of the community, providing links to relevant available datasets, etc.). The scientific contributions of comments to date have included discussion of future research, interpretation of results (including implications of replications and refutations), generalizability and application of research results, and enriching and updating the scientific record by author and community curation.
Dr. Lipman walked Council members though a demonstration of the PubMed Commons system. He explained that comments, including information on when the comment was made and a rating system (i.e., “helpful” or “not helpful”), on a paper are viewed below the abstract in PubMed. Members of PubMed Commons can rate comments, reply to comments, invite an author to join PubMed Commons, link to comments, share comments, and report problems. Dr. Lipman noted that it is possible to search PubMed for papers that only have comments tied to them and presented some examples of comments that have been made for papers in PubMed.
Moving forward, key strategies for PubMed Commons include increasing the profile of good comments, building a community of “commenters,” encouraging authors to respond to comments on their work, and implementing a pilot program for journal clubs. Dr. Lipman also noted that if any of the NIH reporting mechanisms allowed for a “comments in PubMed Commons” slot, it may encourage its use. He concluded his remarks by presenting a slide showing the PubMed Commons home page and encouraging Council members and NIAMS staff to visit www.ncbi.nlm.nih.gov/pubmedcommons/ for additional information.
Dr. Katz commented that PubMed Commons should become significantly more successful and robust than Faculty of 1,000. He suggested that increased efforts to publicize PubMed Commons should be explored.
Dr. Koretzky asked if users can see comments on a paper through a basic PubMed search, or if users have to go to PubMed Commons first to find comments. Dr. Lipman explained that comments can be seen through a PubMed search; going to the PubMed Commons site is not necessary, and all comments are publicly available. In response to a question, Dr. Lipman explained that only a few comments are being added on a daily basis.
Dr. Gabriel asked about the proportion of PubMed Common comments that are critical of a paper compared with the proportion that are positive in nature. Dr. Lipman indicated that more of the comments are somewhat critical (e.g., pointing out some issue with the paper in question) and that there are a number of neutral comments that often link to associated data. To date, only a very few comments have been blocked, based on explicit criteria that are listed on the PubMed Commons home page.
UPDATE ON THE SUPPLEMENTS TO ADVANCE RESEARCH (STAR) PROGRAM
Dr. Serrate-Sztein reminded Council members that the NIAMS STAR award for early established investigators (EEIs) is a supplemental award program to facilitate the transition from a project to a research program (Council members were introduced to the STAR Award Program at the June 3, 2014, NAMSAC meeting). NIAMS’ goal in this regard is to promote innovation and the exploration of high-risk ideas by providing flexible funding. Dr. Serrate-Sztein explained that EEIs are investigators who have renewed their first R01 (Type 2) application in the current fiscal year. STAR awards are intended to allow EEIs to: (1) explore new areas and high-risk ideas, (2) expand and improve laboratory capacity, (3) accelerate resource-intensive projects, (4) mentor junior scientists, and (5) have funding stability.
A NIAMS STAR Working Group, chaired by Dr. Serrate-Sztein, was formed and charged with delineating key aspects of the STAR Award Program, such as eligibility criteria, a requirement for institutional support, the review process, and the evaluation process and criteria. The Working Group included six Council members and four NIAMS staff members. Two conference calls were held to discuss early drafts of a document outlining the STAR Award Program, followed by an e-mail discussion of a subsequent draft of the document.
Dr. Serrate-Sztein noted that the most-discussed item by Working Group members was the initial eligibility criteria, which would have limited the program to EEIs with their first Type 2 and no other NIH support. Identified advantages to this approach included: (1) a small number of eligible applicants will have a lower impact on the R01 payline, and (2) a smaller number of applications is more suitable for administrative review. The approach has the disadvantage of excluding EEIs with the first Type 2 plus other NIH support who may be better poised to establish a research program. However, broadening the eligibility criteria could have a greater impact on the R01 payline; and a larger pool requires a more stringent selection/review criteria process. After significant deliberation, the Working Group agreed that other NIH funding would be allowed, including a second R01. The STAR award eligibility criteria were revised as follows:
- The cohort starts with Principal Investigators (PIs) who received their first AR R01 in 2009 as a “new investigator.”
- The PI must have renewed the first AR R01 (Type 2 AR R01).
- Other NIH funding is allowed.
All NIAMS STAR award applicants will be asked to submit a 3-page essay that includes: (1) a brief overview of research accomplishments to date and how the EEI came to their current project, (2) a short- and long-term cohesive vision for their research program, (3) the significance and potential impact of their research, (4) how they plan to develop their research program, (5) a description of how the STAR Award will allow them to move forward in their research program, and (6) highly innovative and/or high-risk aspects. In addition to the essay, a biosketch, and a curriculum vitae, STAR Award applicants are required to provide a letter from the Chair of their department describing how the EEI and their research program will flourish in their institution. The letter must indicate strong support of the candidate’s research program—the strength of the institutional recommendation will be considered as a factor in the review of the nomination.
Dr. Serrate-Sztein explained that the NIAMS STAR award process is modeled after the Method to Extend Research In Time (MERIT) award (i.e., individuals are nominated by NIAMS staff, outside opinions and perspectives are sought as needed to provide additional expertise, and applications are reviewed and recommended by Council). She then outlined the NIAMS STAR award application evaluation criteria as follows:
- The significance and potential impact of the STAR award on expanding the scope of the R01 research proposal and the field as a whole.
- Evaluation of the innovation, risks, and challenges of the new activity, including whether the research is in an area of high importance.
- The ability of the investigator to create a research program based on their history of innovation and productivity.
- The commitment of the EEI on the field of research based on their past research activity and the supplied short- and long-term visions.
- The strength of the institutional recommendation letter.
A number of factors will be considered in annual post-award evaluations. Examples include consideration of what the STAR award enabled the EEI to do that he or she would not have otherwise done, supporting evidence that these accomplishments were as a direct result of the STAR award, the development of technologies and recently made scientific discoveries, and recent publications and a description of their impact. Additional factors include patent applications and issuances, the development of new collaborations, recently acquired funding for their research program (both from the NIH and other sources), and new mentees and their contribution to the program. Before concluding her remarks, Dr. Serrate-Sztein noted that the STAR Working Group’s report was included in the Electronic Council Book.
Dr. Firestein (a member of the NIAMS STAR Award Working Group) congratulated NIAMS on the STAR Award Program and commented that the flexibility with regard to institutional support will be critical, whether it is phased, salary, or relief of other duties. Dr. Koretzky (also a member of the Working Group) agreed, noting that institutions will need to find unique, creative ways to support their STAR awardees; hopefully, these approaches can be shared. He emphasized the need to reward investigators who have been successful (e.g., in obtaining a second R01) because it can predict those individuals who might use STAR award funding most effectively and efficiently. It also introduces greater competition for the award, which, he commented, is positive as long as the success rate is high. Dr. Shane voiced strong support for the flexibility introduced by the STAR Award Program, noting that individuals who have received two R01 awards are particularly likely to be successful in this program.
Dr. Katz explained that, based on a review of NIAMS data over the last few years, it appears likely that 5-6 individuals will be eligible for a STAR award on an annual basis. He asked Council members if they felt well situated to review these applications; no Council members expressed any concerns in this regard. In response to a question regarding the portability of the STAR award, Dr. Serrate-Sztein explained that if an awardee moves to a new institution, the new institution’s commitment to the award will be considered.
2016 SCIENTIFIC INITIATIVES
Dr. Katz explained that the NIAMS continues to identify research areas and activities to further the NIAMS mission. He and Drs. McGowan and Serrate-Sztein invited Council members to review the descriptions of the following NIAMS extramural program initiatives proposed for 2016:
- Resource Based Centers for Rheumatic, Skin, and Musculoskeletal Disease Research
- Centers of Research Translation (CORT) Re-Issue
- NIAMS Clinical Trial Planning Cooperative Agreement
- NIAMS Solicitation of Pre-Applications for Clinical Trials
- NIAMS Clinical Trial Implementation Cooperative Agreement
- NIAMS Clinical Trial Phased Award Implementation Cooperative Agreement
- Clinical Observational Studies (COS) in Musculoskeletal, Rheumatic, and Skin Diseases
- NIAMS Small Grant Program for New Investigators
- Opportunities for Innovative Research in Musculoskeletal, Rheumatic, and Skin Diseases Leading to High-Impact Clinical Studies
DISCUSSION OF CLOSED SESSION TOPICS
Dr. Katz asked Council members for input on what topics should be discussed during the closed sessions of NAMSAC meetings. Dr. Serrate-Sztein also asked Council members for their preferred format of receiving information about NIAMS activities (e.g., focusing on an entire program or focusing in detail on a few particular aspects of a program) and what topics would be most informative overall.
Dr. Eyre suggested that it would be helpful for Council members to have more of a “landscape” view of what the NIAMS is funding and what the Institute anticipates as future directions. Dr. Shane agreed, noting that it would also be helpful to have a better understanding of the distribution of the different types of grants that are being funded across the NIAMS mission by discipline (e.g., identifying the proportion of NIAMS grants focused on skin). She commented that it can be difficult to get an overview of the Institute’s entire portfolio and expressed some concern regarding a lack of clinical investigation in the area of bone disease. Dr. Katz commented that different disciplines are in different stages of development, but that the NIAMS could work in this area to provide this information to Council members.
Dr. Shane noted that during the last NAMSAC meeting, Council members were informed that the proportion of K awardees who went on to receive R award funding was substantially lower for musculoskeletal researchers than those focused on the areas of skin and rheumatology. She asked if there were potential barriers that musculoskeletal researchers face in terms of receiving NIH funding. Dr. Katz noted the Institute generally has not received many K applications from the bone and mineral community. The NIAMS is working to help K awardees identify appropriate mentors and career opportunities and convenes an annual conference for K awardees. The next such meeting is scheduled for December 4-5, 2014; additional information will be presented to the Council following this meeting.
Dr. Koretzky suggested that it is less valuable to hear about specific research projects at the Council level. He added that information regarding the Institute’s goals for new initiatives or programs would be useful for the Council, followed later by an analysis on whether or not these goals have been achieved. He suggested that this approach be used to inform the Council on all NIAMS funding mechanisms. Dr. Katz reminded Council members that detailed discussions on upcoming initiatives are not permitted; however, once an initiative has been approved, these discussions can take place during the open or closed sessions of Council meetings. Dr. McGowan explained that the original concept for closed session discussions was to provide Council members with a detailed description of what exists in the NIAMS portfolios.
Dr. Gabriel commented that if the Council’s role is to help inform the Institute’s future directions, understanding trends and successes/non-successes would be helpful and better prepare the Council for this role. Dr. Shane agreed and voiced support for learning about how each NIAMS portfolio fits into the Institute’s overall landscape. Dr. Katz noted that some of these topics are more appropriately presented and discussed in open sessions of NAMSAC meetings. He explained that the NIAMS will continue to try to provide a global analysis of what the Institute is funding to Council members.
Dr. Sandborg suggested that being more informed on which programs and initiatives are succeeding and which are not would be helpful to the Council. Dr. Firestein suggested that the Council is interested understanding the impacts and outcomes of NIAMS programs, particularly from the perspective of new investigators. He advocated for using some of the time in closed session for critically examining whether the programs that have been/are being implemented are achieving their goals.
Council member Dr. Gwendolyn Powell Todd, a patient, health advocate, and educator, suggested that there be more of an emphasis on organ diseases and rare diseases, and a description of where they fit into the NIAMS landscape. Dr. Katz noted that there is a strong emphasis on rare diseases in the NIAMS Intramural Research Program; findings from this work have helped inform progress made in other disease areas. He added that rare diseases are an integral component of the NIAMS mission.
Dr. Gabriel explained that it is difficult to “consume” the entire landscape of NIAMS activities and develop meaningful recommendations to inform the strategic direction of the Institute. However, if the challenging work of determining what metrics and important considerations are associated with outcomes is carried out, it will help Council members in using the landscape to provide useful guidance to the NIAMS. Dr. Koretzky agreed, adding that Council members should be provided with specific information on the explicit goals of NIAMS programs and whether these goals have been met. This would also help Council members with their deliberations on proposed new initiatives.
Dr. Katz clarified that discussions regarding the NIAMS landscape, updates on current activities, and discussions regarding the success/non-success of specific programs can be held during the open sessions of NAMSAC meetings. It may be more appropriate to discuss issues in closed sessions when specific names are attached to certain programs. In response to a question, Dr. Katz explained that the NIAMS is re-tooling its P30 program; additional information will likely be presented during the open session of a future Council meeting.
Dr. Susana Serrate-Sztein noted that the NIAMS is about to evaluate progress against some of the objectives that were set forth in the Institute’s 2007 Lupus Research Plan. In formulating the future iteration of this Plan, the NIAMS is trying to articulate future goals that relate to specific short- and long-term outcomes and outputs. This approach may be extended to other programmatic areas within the NIAMS, and will be shared with Council members during the open session of a future NAMSAC meeting.
BOARD OF SCIENTIFIC COUNSELORS REPORT
This portion of the meeting occurred during closed session.
CONSIDERATION OF APPLICATIONS
In closed session, the Council reviewed 750 applications requesting $824,634,649 in total costs and recommended 750 for $824,634,649 in total costs.
The 84th National Arthritis and Musculoskeletal and Skin Diseases Advisory Council Meeting was adjourned at 2:45 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.
Laura K. Moen, Ph.D.
Stephen I. Katz, M.D., Ph.D.