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Advisory Council Minutes
Department of Health and Human Services
Public Health Service
National Arthritis and Musculoskeletal and Skin Diseases Advisory Council
Minutes of the 77th Meeting
June 5, 2012
8:30 a.m. to 3:00 p.m.
CALL TO ORDER
The 77th meeting of the National Arthritis and Musculoskeletal and Skin Diseases Advisory Council was held on June 5, 2012, 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. Leslie J. Crofford (by telephone)
Dr. Harry C. Dietz III (by telephone)
Ms. Karen B. Evans (by telephone)
Dr. David R. Eyre
Dr. Linda Griffith
Ms. Michelle Hofhine
Dr. Henry M. Kronenberg
Dr. Katherine Mathews
Dr. Regis J. O’Keefe
Dr. Alice P. Pentland
Ms. Jean Pickford (by telephone)
Ms. Elizabeth Smith
Mr. Bradley R. Stephenson
Dr. Xiao-Jing Wang
Staff and Guests
The following NIAMS staff and guests attended:
Mr. Steven Austin
Dr. Carl Baker
Dr. Amanda Boyce
Ms. La’Tanya Burton
Mr. Gahan Breithaupt
Dr. Branden Brough
Dr. Eric Brown
Ms. Justine Buschman
Ms. Nazia Cheema
Dr. Ricardo Cibotti
Ms. Barbara Cohn
Mr. Richard Clark
Ms. Stephanie Craver
Ms. Robin DiLiello
Ms. Theresa Do
Dr. Jonelle Drugan
Mr. Erik Edgerton
Ms. Rhonda Edwards
Ms. Elizabeth Elliott
Ms. Sharon Fair
Ms. Barbara Footer
Ms. Gerda Gallop-Goodman
Dr. Nancy Garrick
Ms. Valerie Green
Ms. Gail Hamilton
Ms. Katie Joffee
Mr. Andrew Jones
Dr. Stephen Katz
Ms. Mary Beth Kester
Ms. Shahnaz Khan
Ms. Stephanie Kreider
Mr. Mark Langer
Dr. Gayle Lester
Dr. Helen Lin
Ms. Anita Linde
Ms. Leslie Littlejohn
Dr. Kan Ma
Dr. Marie Mancini
Dr. Kathryn Marron
Ms. Melanie Martinez
Dr. Joan McGowan
Ms. Leslie McIntire
Dr. Laura K. Moen
Ms. Regina Mong
Ms. Melinda Nelson
Ms. Anna Nicholson
Dr. Glen Nuckolls
Dr. James Panagis
Ms. Vivian Pham
Dr. Charles Rafferty
Ms. Natalie Reyes
Ms. Trish Reynolds
Dr. Louise Rosenbaum
Dr. Susana Serrate-Sztein
Dr. William Sharrock
Ms. Sheila Simmons
Ms. Theresa Smith
Ms. Allisen Stewart
Ms. Robyn Strachan
Ms. Yen Thach
Ms. Jamie Thompson
Dr. Phil Tonkins
Dr. Hung Tseng
Dr. Bernadette Tyree
Dr. Fei Wang
Dr. Xibin Wang
Dr. Yan Wang
Dr. Chuck Washabaugh
Dr. Xincheng Zheng
Dr. Josephine Briggs, National Center for Complementary and Alternative Medicine, NIH
Mr. John Burklow, Office of Communications and Public Liaison, NIH
Ms. Karen Chesbrough, Foundation for Physical Therapy
Ms. Rachael Crockett, Foundation for Physical Therapy
Ms. Celeste Crouse, KAI Research, Inc.
Ms. Kasey Duncan, Foundation for Physical Therapy
Ms. Niva Haynes, National Psoriasis Foundation
Dr. John Holden, Department of Veterans Affairs
Ms. Kim Holmes, IQ Solutions
Dr. Rajiv Kumar, Center for Scientific Review, NIH
Ms. Lora Kutkat, Office of the Director, NIH
Dr. Daniel McDonald, Center for Scientific Review, NIH
Dr. Joe Selby, Patient-Centered Outcomes Research Institute
Mr. Richie Taffet, Ehlers-Danlos National Foundation
Dr. Nancy Vazquez, National Institute of Dental and Craniofacial Research, NIH
Ms. Randi Williams, KAI Research, Inc.
CONSIDERATION OF MINUTES
A motion was made, seconded, and passed to accept with no changes the minutes of the 76th NIAMS Advisory Council meeting, held on January 31, 2012.
FUTURE COUNCIL MEETING DATES
Future Council meetings are currently planned for the following dates:
September 11, 2012
February 5, 2013
June 4, 2013
September 10, 2013
February 11, 2014
June 3, 2014
September 8, 2014
PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE (PCORI) ACTIVITIES
PCORI Executive Director Dr. Joe Selby reminded the Council that the PCORI was authorized as part of the Affordable Care Act. Created in 2010, the PCORI focuses on comparative effectiveness research (CER) as an independent, non-profit organization. It is not a branch of the Department of Health and Human Services (HHS) or NIH, although it works closely with the NIH and Agency for Healthcare Research and Quality (AHRQ). Dr. Selby stated that PCORI’s mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed health care decisions. PCORI is committed to continuously seeking input from patients and a broad range of stakeholders to guide its work.
Dr. Selby explained that 20 percent of the PCORI budget is allocated to the HHS, mostly for AHRQ activities, such as training, comparative effectiveness or patient-centered outcomes research, and disseminating the findings of that research. The remaining 80 percent of the PCORI budget supports the Institute’s mission.
PCORI features a 21-member Board of Governors that spans diverse fields of expertise within the health care arena. It also has a 17-member Methodology Committee whose members have varied scientific backgrounds and experience, particularly with regard to CER.
PCORI’s national priorities and research agenda have been vetted, finalized, and posted online. In setting its priorities, PCORI examined recommendations from a number of groups, such as the Institute of Medicine’s (IOM) 2009 list of100 specific priorities for CER, and the five broad priorities identified by the HHS Federal Coordinating Council for Comparative Effectiveness Research. PCORI also considered information from other groups, such as the Agency for Healthcare Research and Quality (AHRQ), the National Quality Forum, the National Prevention Council, and the National Priorities Partnership. After analyzing the recommendations from these various groups, the PCORI settled on the following five priorities to guide its research agenda:
- Assessment of prevention, diagnostic, and treatment options (e.g., comparisons of alternative clinical options, identifying patient differences in response to therapy, studies of patient preferences for various outcomes).
- Improving health care systems (e.g., improved support of patient self-management, coordination of care for complex conditions, comparing alternative strategies for workforce deployment).
- Communication and dissemination research (e.g., understanding and enhancing shared decisionmaking, alternative strategies for dissemination of evidence).
- Addressing disparities (e.g., understanding differences in effectiveness across groups, understanding differences in preferences across groups, reducing disparities by implementing findings from patient-centered outcomes research).
- Accelerating patient-centered outcomes research and methodological research (e.g., improving study designs and analytic methods of patient-centered outcomes research, building and improving clinical data networks, methods for training researchers and patients to participate in patient-centered outcomes research, facilitating the study of rare diseases).
Two weeks prior to this Council meeting, PCORI released four funding announcements that correspond to the Institute’s first four priorities, totaling $96 million in research funds. It is expected that awardees will be announced by the end of 2012. Examples in the funding announcements were drawn from focus groups, and they emphasize outcomes that matter to patients. There are eight PCORI review criteria: (1) impact of the condition on the health of individuals and populations, (2) innovation and potential for improvement, (3) impact on health care performance, (4) patient-centeredness, (5) rigorous research methods, (6) inclusiveness of different populations, (7) research team and environment, and (8) efficient use of research resources. Study section reviewers will be expected to apply these criteria to each PCORI application.
Dr. Selby then briefly outlined each of the four PCORI funding announcements.
- the assessment of options for prevention, diagnosis, and treatment, includes $46 million in funding for the first round.
- improving health care systems
- communication and dissemination research
Dr. Selby said that the content of these funding announcements is very broad and could include any condition, treatment, or type of intervention. He added that patients and other stakeholders will be placed on the study sections reviewing applications to PCORI funding announcements and will comprise 30 percent of the study section membership.
Dr. Selby explained that PCORI envisions two complementary approaches to funding impactful research. The first approach is a broad, "bottom up" approach that begins with PCORI issuing broad funding announcements. Researchers and stakeholders generate questions and apply criteria in proposals, and then peer review identifies the best applications based on adherence to the criteria. The second approach is a targeted or "top down" approach in which PCORI and stakeholders generate questions responsive to the criteria. PCORI then issues targeted funding announcements, researchers and stakeholders develop responsive proposals, and then again, peer review identifies the best applications based on adherence to the criteria.
Dr. Selby concluded his remarks by noting that PCORI is looking to leverage its resources through collaborations with other groups, including the NIAMS. He anticipates that there will be jointly funded research initiatives in the near future.
In response to a question from Dr. Katz regarding data for reimbursement decisions, Dr. Selby emphasized that the language in the statute establishing PCORI makes it clear that it is not a policymaking body, although decision makers may use PCORI research. Simply put, PCORI’s job is to increase the amount of evidence with a primary target of patients and caregivers.
A council member commented that there appears to be significant overlap with PCORI’s work and efforts of the Centers for Disease Control and Prevention (CDC), and asked about partnering with the CDC. Dr. Selby noted that PCORI representatives would be meeting with the CDC shortly and that surveillance, specifically surveillance for treatments (i.e., how they are changing over time and how they vary across the country), is one area of significant overlap between them.
Others asked about the process for selecting patients as lay members to serve on PCORI study sections. Dr. Selby explained that PCORI recognizes the need to develop a training program for these study section lay members —one that is PCORI-specific and provides training on research methods as well as an orientation to PCORI review criteria (with a particular emphasis on the effectiveness and best methods for engaging patients and stakeholders). PCORI is working with other organizations that have trained patients in this capacity (e.g., the National Breast Cancer Coalition, U.S. Food and Drug Administration [FDA], and the National Cancer Institute [NCI]), and it hopes to use patients that have prior experience serving as reviewers on study sections from these and other organizations.
Council member Dr. Regis O’Keefe, Chair of the Department of Orthopaedics and Rehabilitation at the University of Rochester Medical Center, noted that he expects an extraordinary response to PCORI from health care systems and academic medical centers. He asked if there is a plan for increasing the investment in the PCORI. Dr. Selby acknowledged that the current funding environment for research is difficult and suggested that PCORI can partner with a wide range of funding entities, including foundations and industry.
Dr. Katz commented that some of PCORI’s activities transcend many diseases or conditions. Translating knowledge into behavioral change, for example, covers a broad range of diseases, and there may be a role for PCORI in this area. Dr. Selby agreed citing a number of examples of cross-cutting themes in PCORI solicitations, such as the appropriate management of chronic pain in patients with chronic conditions, the coordination of care across settings, and the general approach to patients with multiple comorbidities. It is hoped that many of the projects that PCORI funds, which are based on one condition or patient population, will produce findings that are generalizable to other areas.
A Council member noted that many patient advocacy groups are focused on rare diseases and asked if the PCORI views these groups as being able to help the Institute with patient recruitment. Dr. Selby concurred and added that the PCORI is hoping to establish a rare diseases advisory group.
Council member Dr. Lynda Bonewald, Lefkowitz Professor in the Department of Oral Biology at the University of Missouri-Kansas City School of Dentistry, noted that CER has generated significant interest in for-profit health care delivery and asked Dr. Selby about the interplay between for-profit and non-profit health care delivery groups that are eager to learn and benefit from CER. Dr. Selby commented that the PCORI does not favor for-profit and non-profit groups in terms of funding. If researchers have partnerships with large health care systems when they apply for PCORI funding, it is seen as a positive sign that the research is more likely to be disseminated.
Council member Mr. Bradley Stephenson, an attorney at law and a member of the Muscular Dystrophy Association’s National Task Force on Public Awareness, voiced support for the "bottom up" approach to conducting research because it allows for many different types of proposals, especially those focused on rare diseases. He noted that the "top down" approach may be better for examining more common conditions and treatments, and may be useful for identifying areas in which the health care system can save the most money. Dr. Selby agreed, noting that the PCORI was established under the assumption that comparative effectiveness research findings will lead to cost savings.
Council member Dr. David Eyre, Professor in the Departments of Orthopedics and Sports Medicine at the University of Washington School of Medicine, suggested that a Web-based system for disseminating information to the public will become increasingly important as the PCORI evolves and asked about its plans to implement one. Dr. Selby noted that the PCORI’s Communications Director is a specialist in this area, and that the PCORI is thinking along these lines, and even beyond the Internet.
UPDATE ON NIH PAIN ACTIVITIES
Dr. Katz introduced this presentation by noting that he is committed to engaging patients and public representatives in the Institute’s various activities and making sure that these viewpoints are reflected in all of its deliberations. The NIAMS shares this value with many other federal government agencies. When Congress established the Interagency Pain Research Coordinating Committee (IPRCC) with the passage of the Affordable Care Act, the law stipulated that the Committee’s membership included six members of the general public who are representatives of the leading research advocacy and service organizations. Two individuals who are well known to the NIAMS are members of the IPRCC: Terrie Cowley, President of the TMJ Association (temperomandibular disorders) and Tammy Liller, President of the National Fibromyalgia Partnership and former NIAMS Advisory Council member.
As a result of the provisions of the Affordable Care Act, the IOM released a report calling for coordinated national efforts of public and private organizations to create a cultural transformation in how the nation understands and approaches pain management and prevention. Dr. Katz then introduced Dr. Josephine Briggs, Director of the National Center for Complementary and Alternative Medicine (NCCAM), who provided additional details on the IOM report and the NIH Pain Consortium’s activities.
Dr. Briggs explained that the Pain Consortium was established seven years ago to enhance pain research and promote collaboration among researchers across the many NIH Institutes and Centers (ICs) that have programs and activities addressing pain. Five IC Directors belong to the Pain Consortium’s Executive Committee, including Dr. Briggs, who characterized the Pain Consortium as an active and effective trans-NIH group. The Pain Consortium sponsors an annual meeting that brings in leading pain researchers and issues funding announcements regularly to increase pain research. Despite a greatly improved understanding of central pain pathways, pain management has not improved significantly.
The IOM report had the following recommendations for the NIH: (1) designate a lead Institute at the NIH (the National Institute of Neurologic Diseases [NINDS] has been selected) responsible for moving pain research forward, and increase the support for and scope of the Pain Consortium, (2) improve the process for developing new agents for pain control, (3) increase support for interdisciplinary research in pain, (4) increase the conduct of longitudinal research in pain, and (5) increase the training of pain researchers. The overwhelming conclusion from the report was that current processes for pain management were not optimum. The report cited an estimate that 100 million Americans suffer from chronic pain. The IPRCC’s charge is to: (1) develop a summary of advances in pain care research supported or conducted by the federal agencies, (2) identify critical gaps, (3) make recommendations to ensure that the activities of the NIH and other federal agencies are free of unnecessary duplication of effort, (4) make recommendations on how best to disseminate information on pain care, and (5) make recommendations on how to expand partnerships between public entities and private entities to expand collaborative, cross-cutting research.
Dr. Briggs noted that there has been a 5-fold increase in unintentional drug overdose death rates since 1990, and that two-thirds of this increase has occurred since 2000. There have also been striking increases in the rates of prescription painkiller sales, deaths, and substance abuse treatment admissions in the last 10 years. In response, the Pain Consortium launched an effort to strengthen education in pain management for physicians and other health care providers. It is led by the National Institute on Drug Abuse (NIDA), but all ICs with major programs in pain have contributed to the contract funding mechanism. The 11 health professional schools designated as Centers of Excellence in Pain Education (CoEPEs) will be developing a curriculum resources for pain education. Dr. Katz noted that the NIAMS has participated in the CoEPEs initiative with the belief that at an early stage, students should be exposed to pain management approaches.
Pain-related issues are of particular interest to NCCAM, which has a focus on non-pharmacologic approaches to pain management. Dr. Briggs reminded the Council that NCCAM’s legislative mandate is to study complementary and alternative treatments, and in particular, to study their integration into conventional health care. She noted that the primary reason individuals turn to complementary and alternative medicine is for pain management with back, neck, joint (arthritis) pain, and headache being some of the leading reasons.
In the area of pain management—particularly for back pain—a body of evidence is slowly accumulating for certain complementary and alternative health practices, including spinal manipulation, massage, yoga, tai chi, mindfulness, and other meditation strategies. Dr. Briggs noted that the Department of Defense (DoD) and Veterans Administration (VA) are facing sizable pain management challenges and problems with prescription drug abuse among returning veterans from current wars. The DoD and VA are using a variety of complementary and alternative approaches, particularly meditation and yoga, in pain management. Dr. Briggs briefly noted examples of high-quality clinical trials that show some benefit of these self-care and pain management interventions in combination with pharmacologic treatments.
Another area of relevance is the mechanism of placebo analgesia, which is an active area in the NCCAM portfolio. The NCCAM is learning a fair amount about the ways in which endogenous pain modulatory pathways may be activated by both placebos and some of the previously mentioned modalities. Descending pathways that modulate pain are often affected by placebo responses, physician reassurance, or context, and this is an area that the NCCAM is pursuing.
The NCCAM is also in the process of establishing an intramural pain program. With Dr. Katz’s assistance, the Center has recruited a leading pain researcher to serve as its new Scientific Director and provide trans-NIH leadership in intramural pain research.
Dr. Katz asked about the issue of acute-to-chronic pain and approaches to managing it. Dr. Briggs explained that there is a subgroup of people with acute pain that becomes chronic without a continuing evidence of tissue damage or activation. The Pain Consortium has been trying to advance understanding of this biological conversion. It is clear that certain regions in the brain change in chronic pain sufferers. Dr. Briggs commented that this problem remains a challenge, and there is not a strong understanding in this area, other than that the initiating stimulus is no longer needed for the individual to perceive pain.
Drs. Gayle Lester (Health Scientist Administrator in the NIAMS Division of Musculoskeletal Diseases) and Jim Witter (Health Scientist Administrator in the NIAMS Division of Skin and Rheumatic Diseases) are members of the Pain Consortium.
Dr. Briggs emphasized the importance of acknowledging that pain is an incredibly difficult and ubiquitous clinical problem, and that the approaches to addressing it must be multifaceted. She noted that the Patient-Reported Outcomes Measurement System (PROMIS) initiative has developed useful tools for measuring pain. She added that, in research, it is critical to more effectively capture the extent to which pain interferes with function, as opposed to just measuring pain on a scale of 1-10.
Dr. Katz reminded the group that NCCAM is part of the Osteoarthritis Initiative (OAI), which includes a focus on pain and pain management. Dr. Joan McGowan, Director of the NIAMS Division of Musculoskeletal Diseases, added that the NIAMS, NCCAM, and several other ICs are collaborating to address the issue of low back pain. One of the needs that has been identified is research diagnostic criteria. A subgroup of the Pain Consortium is working to develop research diagnostic criteria that facilitate the entry of patients into clinical trials and the categorization of those patients that would further enable PCORI’s support of clinical trials and CER.
DIRECTOR’S REPORT AND DISCUSSION
Dr. Katz welcomed Council members, NIAMS staff, and guests, and opened his Director’s Report by inviting Council members to review the NIAMS ShortTakes online, a resource that contains information about NIH initiatives, highlights of Capitol Hill/Department of Health and Human Services/NIH activities, news about personnel changes at the NIH and NIAMS, updates on NIAMS communications and outreach efforts, and Dr. Katz’s "Director’s Column." In the current ShortTakes, Dr. Katz’s "Director’s Column" focuses on the Institute’s new Clinical Director, Dr. Richard Siegel, who will be overseeing the clinical and translational research within the NIAMS Intramural Research Program (IRP), and supervise the clinical staff assigned to NIAMS investigators performing clinical and translational research, the NIH Rheumatology Fellowship Training Program and the NIH Community Health Center. Dr. Siegel was recruited to the NIAMS about 11 years ago as an investigator and was awarded tenure in 2009. Dr. Siegel has made numerous contributions in the area of tumor necrosis factor (TNF) receptor signaling, and is a member of the American Society for Clinical Investigation and the Association of American Physicians.
Personnel Changes at the NIH/NIAMS
At the NIH level, NIH Director Dr. Francis Collins has announced the selection of Dr. Gary Gibbons as the new Director of the National Heart, Lung, and Blood Institute (NHLBI). Dr. Gibbons was the Founder and Director of the Cardiovascular Research Institute at Morehouse School of Medicine, Chair of the Department of Physiology, and Professor of Physiology and Medicine at Morehouse. His laboratory will be located within the National Institute of Minority Health and Health Disparities (NIMHD). Dr. M. Roy Wilson has been selected as the new NIMHD Deputy Director for Strategic Scientific Planning and Program Coordination. Dr. Wilson has served as Chancellor of the University of Colorado, President of the Texas Tech University Health Sciences Center, and Dean of the Creighton University School of Medicine.
Within the NIAMS, the Office of Science Policy and Planning and the Office of Communication and Public Liaison have been combined as Branches within the new NIAMS Office of Science Policy, Planning, and Communication (OSPPC). This reorganization makes permanent an informal operating procedure that has been in place at the Institute for the past year. The NIAMS believes that this new structure will enable it to leverage the expertise of its staff to share knowledge, streamline the decisionmaking and reporting processes, and maximize its resource utilization. Ms. Anita Linde has been selected as the Director of OSPPC.
Ms. Mary Beth Kester has officially joined OSPPC and will serve as a Health Science Policy Analyst. Ms. Kester will facilitate a number of NIAMS’s strategic planning, program evaluation, and legislative activities. She comes to the Institute from the National Institute of Biomedical Imaging and Bioengineering (NIBIB), where she served as the Planning and Evaluation Officer and the Legislative Officer.
Ms. La’Tanya Burton is the new Chief Information Officer of NIAMS and Chief of the Scientific Information Technology Branch. Ms. Burton comes to the Institute from the Department of Commerce and the National Oceanic and Atmospheric Administration (NOAA). Dr. Katz thanked former NIAMS Chief Information Officer Mr. Robert Rosen and former Acting Chief Information Officer Mr. Melvin Broadus for their service and support.
Dr. Xincheng Zheng has joined the NIAMS as a Scientific Review Officer within the Institute’s Scientific Review Branch. Dr. Zheng comes to the NIAMS after seven years in the pharmaceutical industry, where he managed many aspects of pre-clinical drug development. Dr. Zheng has served as a peer reviewer on several NIH review committees and has been a recipient of several NIH Small Business Innovation Research (SBIR) awards.
Update on Budget and Congressional Activities
Dr. Katz reminded the group that the NIAMS keeps an updated version of its funding plan on the Institute’s Web site. The plan has changed recently, and the payline will be through the 12th percentile for established investigators and through the 15th percentile for new investigators. In the interest of improving communications with its research community, the NIAMS will soon be providing data about the number of applications that it receives and funds as a function of the scores these applications received during the peer review process. The National Institute of General Medical Sciences (NIGMS) has posted this type of information for many years and the NCI has begun to share this type of information for fiscal year (FY) 2011.
On March 20, 2012, the House Appropriations Subcommittee on Labor, HHS, and Related Agencies held a hearing. Dr. Collins was a primary witness at this hearing and was accompanied by Dr. Tom Insel, the Acting Director of the National Center for Advancing Translational Sciences (NCATS). Most of the Subcommittee’s questions pertained to the NCATS. The Senate Subcommittee held its hearing on March 28, 2012, and Dr. Collins was accompanied by a number of IC Directors. At this hearing, there was considerable discussion about the challenges that lie ahead given current budget constraints.
On May 18, 2012, the NIH published a notice regarding a new policy on special council review procedures which are to be piloted during the closed session of each IC advisory council meeting in May and June. Dr. Katz explained that part of the President’s budget proposal for FY2013 includes a provision for NIH to scrutinize applications from principal investigators (PIs) who have more than $1.5 million in total costs from NIH grants. This proposed policy arose from the dialogue that the NIH launched with the research community in autumn 2011 about options for managing NIH resources in austere times. The NIAMS may need to implement this policy as soon as September 2012. He emphasized that this policy would not represent a cap on total NIH funding for individual PIs. Some of the most productive NIH investigators lead significant research teams and programs that may require annual budgets of more than $1.5 million in total costs. Also, some types of research, such as large, complex clinical trials, commonly trigger this type of review.
Dr. Katz reported that on April 4, 2012, President Obama signed the law PL 112-105, the Stop Trading on Congressional Knowledge (STOCK) Act. This law prohibits members of Congress, their employees, and federal employees from using non-public information for personal benefit. The STOCK Act is the first of several pieces of legislation that provide greater government oversight. Pending bills would require increased reporting on government programs or would restrict agencies’ budgets for travel to meetings and conferences.
At the request of the Coalition for Imaging and Bioengineering Research (CIBR), Dr. Katz participated in a panel discussion on Capitol Hill about osteoarthritis research. The event was entitled "Medical Imaging and Arthritis: A New Look at the Athlete in All of Us." The panel discussion featured CIBR President Dr. Renée Cruea, Dr. Michael Recht (a professor at New York University), and Mr. L.C. Greenwood (a patient and former All-Pro football player for the Pittsburgh Steelers).
Dr. Katz also made other Congressional visits with the President of the American Academy of Dermatology to highlight the importance of medical research. Each of the offices visited expressed support for investing in biomedical research, even though the tight financial climate is having an impact on these national priorities.
Highlights of Selected Recent NIAMS-supported Scientific Advances
- Dr. Katz noted that 2012 marks the 20th anniversary of the discovery of the JAK-STAT cell signaling pathway by cell biologists Drs. George Stark and James Darnell. In the years since this discovery, research by many other scientists, along with the technological advances for genome-wide association studies, have contributed to a much better understanding of how these JAKs and STATs influence immunity. Advances in understanding the JAK-STAT pathway have allowed researchers, including NIAMS Scientific Director Dr. John O’Shea, to develop ways to inhibit various JAK proteins. In 1994, Dr. O’Shea discovered JAK3, which is the first and most widely-studied example, in a partnership with Pfizer. This work led to the development of tofacitinib, a JAK inhibitor that is in late-stage clinical trials for various autoimmune diseases. Last year, an FDA advisory panel voted in favor of approving tofacitinib for rheumatoid arthritis patients who have not responded to one or more other medications. Concerns about long-term side effects prompted the panel to recommend follow-up studies if the drug is approved, and the FDA is expected to issue its ruling in August.
- A new study from Dr. Raphaela Goldbach-Mansky of the NIAMS IRP, along with many collaborators, shows that anakinra, a medication approved for the treatment of rheumatoid arthritis, is effective in stopping the progression of organ damage in people with neonatal multisystem inflammatory disease (NOMID). NOMID is a rare, debilitating genetic disorder and has been a focus of research within the NIAMS IRP for some time (Arthritis Rheum. 2012 Jan 31. DOI: 10. 1002/art.34409. [Epub ahead of print] PMID: 22294344).
- In a promising preclinical study from the University of Pittsburgh, researchers have identified an agent that, in laboratory tests, protected the skin and lungs from fibrosis, a process that can lead to organ failure and even death because the damaged tissue becomes scarred and dysfunctional. The team, led by Dr. Carol Feghali-Bostwick, demonstrated that a fragment of the protein endostatin, or E4, could protect fibrosis-prone human skin cells, and the skin and lungs of mice from scarring. This peptide could reverse scarring that had already occurred, which would be a tremendous advance if it can be extrapolated to humans (Sci Transl Med. 4, 136ra71 (2012); DOI: 10.1126/scitranslmde.3003421).
- Myotonic dystrophy type 1 is a form of muscular dystrophy characterized by progressive muscle degeneration or weakness and is caused by excessive repetition of a short DNA sequence in the DMPK gene. Whereas unaffected individuals have up to 50 repeats in the sequence, myotonic dystrophy patients have hundreds to thousands of repeats. This mutated gene produces toxic RNA that becomes trapped in the nuclei of cells of patients with myotonic dystrophy type 1. A study by Dr. Thomas Cooper from Baylor College of Medicine describes a new strategy aimed at eliminating this toxic RNA. The research designed and tested a type of molecular drug called gapmers, which are composed of chemically modified DNA sequences that bind the abnormal repeats in the RNA and trigger their destruction and elimination. The gapmers acted only on the toxic RNA and did not promote the degradation of normal products from the DMPK gene (Proc Natl Acad Sci USA. 2012 Mar 13;109(11):4221-6. Epub 2012 Feb 27. PMID: 22371589).
- Studies from a large group of collaborators, including Drs. Kathy Moser and Patrick Gaffney of the Oklahoma Medical Research Foundation, using genome-wide association studies have explored a gene in the region surrounding the neutrophil cytosolic factor 2 (NCF2) gene. The investigators discovered that the NCF2 gene is abnormal in some children and adults with lupus. Little is known about the role NCF2 plays in lupus, but the discovery of a new gene variant that may contribute to the development of lupus through neutrophils provides a new target for researchers to explore (Proc Natl Acad Sci USA. 2012 Jan 10;109(2):E59-67. Epub 2011 Dec 27. PMID: 22203994).
- Although two-dimensional areal bone mineral density, as measured by DXA, is regularly used to guide decisions about who should receive treatment for osteoporosis, its value is limited for predicting future vertebral fractures, the most common type of osteoporotic fractures. Dr. Tony Keaveny from the University of California, Berkeley and colleagues analyzed data from a subset of men who were participating in the long-standing Osteoporotic Fractures in Men (Mr. OS) Study. The findings revealed that three-dimensional bone mineral density measured by computerized tomography, and its derived vertebral strength, are stronger and more robust predictors for new vertebral fractures in older men than the DXA measurements. Most of the men in this study were white; future studies are needed to confirm this finding in more diverse populations of men and women (J Bone Miner Res. 2012 Apr;27(4):808-816. PMID: 22190331).
- Studies from Drs. Jeffrey Katz and Elena Losina of Brigham and Women’s Hospital have shown a dramatic increase in the number of total knee replacements over the past 10 years. The researchers believe this increase cannot be fully explained by population growth and obesity and suggest that other factors must be involved. The increase in surgeries was especially pronounced among younger patients, suggesting that this change may be a result of a growing number of knee injuries and expanding indications of total knee replacements (J Bone Joint Surg Am. 2012 Feb 1;94(3):201-7. PMID: 22298051).
- Two papers by Dr. Thomas Rando’s laboratory at the Palo Alto Institute for Research and Education address the regulatory roles of microRNAs. MicroRNAs are not transcribed. Mature skeletal muscles composed of multinuclear cells called fibers are encapsulated in tubes of collagen and satellite cells reside in the space between the fiber and the collagen. These satellite cells are thought to be precursor cells for more adult cells and can be generated after certain stimuli. In order for a pool of these cells to be ready when needed for muscle repair, satellite cells are kept in a state of quiescence (like hibernation) that is common to many stem cell populations. When muscle is damaged, the cells divide into two: one cell reverts back to a quiescent satellite cell and one continues to divide into cells that will develop into mature cells. This process is tightly controlled by many factors that control gene expression. The Rando laboratory showed for the first time that microRNAs can regulate the activation of these quiescent stem cells. This has been a long-term interest in the area of muscular dystrophy (Cell Stem Cell. 2012 Mar 2;10(3):327-36. PMID: 22385659) (Nature. 2012 Feb 23;482(7386):524-8. DOI: 10.1038/nature10834. PMID: 22358842).
- Research teams are pursuing different strategies to understand and control muscle differentiation and repair. Investigators at the University of Minnesota demonstrated the capability of muscle progenitor cells that were derived from human embryonic and human induced pluripotent cells to engraft and improve muscle function in a mouse model of Duchenne muscular dystrophy. The cells were modified to become muscle stem cells by transferring the PAX7 gene into them, which controls muscle cell fate during embryonic development. The treated muscles exhibited partial recovery of strength. Most of the transplanted cells grafted and became mature muscle cells with multiple nuclei, but some PAX7-expressing cells with individual nuclei remained. This represents a potential promise in terms of being able to generate these types of cells to regenerate stem cells (Cell Stem Cell. 2012 May 4;10(5):610-9. PMID: 22560081).
- Another NIAMS-funded stem cell advance suggests a strategy to direct stem cells to enhance bone formation. Dr. Nancy Lane, University of California at Davis Medical Center, and colleagues took stem cells and bound them to a bisphosphonate, resulting in the stem cells differentiating into bone cells. If the hybrid molecules used in this study, or related compounds, prove to be safe and effective in humans, this study would represent a breakthrough in treating osteoporosis (Nat Med. 2012 Feb 5;18(3):456-62. PMID: 22306732).
NIH/NIAMS Activities and Plans for the Future
Dr. Katz provided the Council with an update on the new NIH Center for Regenerative Medicine (NIH-CRM). The Center is a Common Fund program, and its Web site will serve as a resource for the intramural and extramural scientific communities. He acknowledged the participation of Dr. O’Shea in this effort.
The NIH is also examining ways to enhance utilization of the Clinical Center by extramural researchers. As Chair of the Clinical Center Governing Board, Dr. Katz has been involved in an effort to broaden the Bench-to-Bedside Program at the Clinical Center to include extramural investigators, provided that they partner with intramural scientists. The NIH recently received input from the extramural community in the form of a Request for Information on potential partnerships with intramural clinical investigators.
In March 2012, the NIAMS launched a new page on Facebook, the popular social media Web site, as a companion to the Institute’s social media presence on Twitter. Facebook will be used as a platform to feature health information and publications, news stories, resources, research events, and other activities from the Institute. The NIAMS Facebook page will also feature videos that the NIAMS has posted to YouTube.
The Institute continues to work closely with the NIAMS Coalition. Last month, the NIAMS hosted a teleconference for Coalition members interested in effective strategies for sharing the value of research. The teleconference was attended by almost 30 NIAMS Coalition members from 2 dozen organizations. Dr. Katz recognized new Coalition Co-Chair Ms. Kim Cantor of the Lupus Foundation of America, and Ms. Annie Kennedy of the Muscular Dystrophy Association, who has extended her term.
The NIAMS has engaged in several activities with individual Coalition members and organizations. In March, the Institute hosted two separate tours of the NIH campus and NIAMS intramural laboratories for more than 45 patients and family members who are associated with the American Academy of Orthopaedic Surgeons and the National Psoriasis Foundation. Dr. Katz explained that these visits are embraced by the public and serve as a valuable means of informing the public about NIAMS and NIH activities.
Dr. Bonewald noted that, from the investigator’s viewpoint, higher paylines are obviously better. If the payline is higher; however, they could indicate that the Institute is not receiving enough applications. She asked whether competitive projects fall between the 6th percentile and the 25th percentile, and if the NIAMS payline should be somewhere in the middle. Dr. Katz explained that each IC treats this matter differently. For example, the NCI payline is through the 7th or 8th percentile, and pays a large percentage of the applications beyond the 8th percentile. The NIAMS strives to set a payline that allows for some leftover funds that can be used as discretionary money to pay for applications that the Institute views as critical.
Dr. Kronenberg stated his assumption that the differences between ICs’ paylines was dependent on the amount of money each sets aside for incentive programs and other activities, as opposed to the number of applications it receives. Dr. Katz commented that some ICs are more focused on funding Centers and others are more focused on research project grants (RPGs). Another factor is the variation in the amount of each IC’s commitment to non-competing projects for the next year, which, in turn, affects how many new applications the Institute can fund. For the NIAMS, the amount available for competing applications has ranged from $69 million to $90 million. Additionally, the number of applications has generally been increasing (although last year, the number of R01 applications was flat), which in turn lowers the success rate.
Dr. Bonewald recalled a comment from a previous Council meeting that there was no significant difference between productivity or success rate for grants through the 20th percentile. She expressed concern about the process of only allowing two submissions (A0 and A1), and asked if this was part of the reason for the lower number of R01 applications. Dr. Katz clarified that investigators are not excluded from the system after two submissions; they are allowed to come back with a different application. He noted that, since the decision to eliminate A2 and A3 applications in 2009, more A0s are being supported.
Council member Dr. Xiao-Jing Wang, Professor in the Department of Pathology at the University of Colorado, Denver, pointed out that the difference between an application scoring in the 13th or 14th percentile compared with the 5th percentile could simply be a reflection of the individuals on the study section reviewing that application. Dr. Katz commented that the NIH is looking at these issues. There is a new Acting Director at the Center for Scientific Review (CSR), and the search is ongoing for a new CSR Director.
Dr. Kronenberg reflected that inadequate NIH funding makes it extremely difficult, if not impossible, to find the "right" answer to these questions. He commented that the scientific enterprise is changing and the trend is discouraging. This could result in many individuals selecting a different career path. Dr. Katz added that the NIH and its ICs may have to consider different approaches in the future to maintain the research enterprise.
NIH COMMUNICATIONS PLAN
Mr. John Burklow, Director of the NIH Office of Communications and Public Liaison (OCPL), updated the Council on efforts to strengthen NIH communications. When Dr. Collins became NIH Director, he emphasized the need to make the strongest possible case for the NIH, and that sentiment was shared by previous NIH Directors. Mr. Burklow noted that there has probably never been a more important time for people to have appreciation for biomedical research and, specifically, NIH-supported research.
Next year will mark 10 years since the end of the doubling of the NIH budget and it is important to communicate to the American public what they are getting back from their investment in the NIH. The communications climate and landscape have changed. In the last 5 years, there have been fundamental changes in the ways people receive and share information, and information is becoming increasingly fragmented. This requires that the NIH take a more cohesive approach.
Mr. Burklow noted that the many NIH’s many stakeholders, who sometimes have competing interests, must have a common goal and move cohesively towards that goal. A recent Research!America survey asked the question, "What is the name of the government agency that funds most of the medical research paid for by taxpayers in this country?" Most respondents indicated that they did not know or thought it was the FDA. Only 9 percent identified the NIH. However, positive strides are being made. Mr. Burklow noted that 10 years ago, only 5 percent of respondents to a similar survey identified the NIH in response to this question.
One of the fundamental needs that must be addressed to maintain public support for biomedical research is increasing the public’s understanding of the connection between research and health. On a consistent basis, Congress has asked the NIH for information on its activities. The challenge is to communicate the value of NIH-supported research with maximum impact, while striking a balance between promoting NIH’s activities when communicating to the public, but not at the expense of individual ICs.
Mr. Burklow described four primary strategies that the NIH is starting to use:
- Fortify the NIH. Efforts are underway to examine all of the ways in which the NIH communicates with the public to ensure that it is being done in a cohesive way that allows the public to understand what the NIH is and what it does. The OCPL is developing a toolkit for NIH staff and, eventually, for NIH grantees, that will include PowerPoint slides, key messages, resources, and templates for Web sites. Currently, there are 1,743 different public NIH Web sites, and there are ongoing efforts to create more consistent in presentation.
- Mobilize NIH stakeholders. This involves working with the grantee institutions, advocacy groups, and professional societies, and giving them the tools and resources they need so that they can communicate with all of their constituents.
- Educate policy makers. Mr. Burklow noted that the OCPL works collaboratively with the NIH Office of Legislative Policy and Analysis (OLPA) and with the grantee institutions, to highlight their connections with the NIH. When grantee institutions have local events showcasing NIH-supported research , it is important for them to emphasize the role of NIH funding.
- Leverage traditional and social media. Any activities publicized with traditional media should be amplified through social media.
There has been some discussion about changing the NIH logo, which was developed in 1975. It is likely that a new one would feature the letters "NIH" without a graphic element, to make the logo stronger and simpler.
Despite the perception that the NIH is not well known, there were roughly 4,000 stories in the media in April 2012 that mentioned it. There may have been many more, but often, reporters and editors are not able to include all funding information in a story because of space constraints. Mr. Burklow emphasized the importance of communicating to the public in ways that they will remember. He noted that the NIH and Dr. Collins have been featured prominently in a number of television specials and documentaries on health-related topics. In addition, Dr. Collins appeared on The Colbert Report to discuss the obesity epidemic in this country.
Mr. Burklow acknowledged that the NIAMS has taken the lead in many communications-related activities, particularly in working with and informing stakeholders, and Congressional and multicultural outreach.
Council member Ms. Michelle Hofhine, a patient advocate and Vice President of Marketing at Accredited Home Care Services, noted that she had no idea what the NIH was 18 years ago, when her daughter was diagnosed with osteogenesis imperfecta. In her experience, physicians know about the NIH, but do not know what the NIH does. She expressed enthusiasm and strong support for the public tours of NIH that inform visitors about the NIH mission and activities. Mr. Burklow said that approximately 300,000 research professionals around the country are funded, in part, by the NIH, and that the Council could serve as a conduit of information about the NIH to the public.
Dr. Katz asked about the impact of the appearance of Dr. William Gahl, Director of the NIH Undiagnosed Diseases Program, on 60 Minutes May 20, 2012. Mr. Burklow noted that his office has received some mail regarding that episode, but did not have specific viewership information. These types of stories are very effective in conveying information and connecting people with the NIH.
Dr. Mathews noted that physicians are increasingly being told to provide patients with education materials and suggested that brief disease- or condition-specific items that feature the NIH logo prominently could reach large numbers of patients. She also suggested that text appear in these materials indicating that the NIH uses research dollars to support the advancement of care for the particular disease or condition in question.
Council member Dr. Alice Pentland, James H. Sterner Professor and Chair in the Department of Biology at the University of Rochester School of Medicine and Dentistry suggested that promoting the message of the NIH using taxpayer dollars to cure disease would unify many current fragmented activities.
Mr. Stephenson noted that each of the 27 ICs has a communications department and asked about consolidating them into one office. Mr. Burklow explained that one might think that centralization would be more efficient, but there is a programmatic need for each IC to have its own communications office. Previous efforts to consolidate communications offices, and even budget offices, across the NIH have not been successful. The NIH OCPL works very closely with the IC communications offices.
Dr. Linda Griffith of the Department of Biological and Mechanical Engineering in the Biological Process Engineering Center at Massachusetts Institute of Technology suggested that it would be beneficial to have a slide describing the NIH that she and her colleagues could use when giving presentations to lay audiences. Mr. Burklow agreed, and noted that there are plans to incorporate this into the toolkit being developed by the OCPL.
Dr. Kronenberg voiced support for revamping the NIH logo and asked about the reaction from large ICs that may have mixed feelings about featuring the NIH logo more prominently than their own logo. Mr. Burklow noted that there was a meeting in May during which IC Directors expressed support for the increased emphasis on the NIH logo. There is a general sentiment that it is time to modernize NIH’s communications and make it easier for the public to understand and appreciate its work.
OVERVIEW OF 2012 NIAMS EXTRAMURAL SCIENTIFIC RETREAT
Dr. Katz reminded the Council that the Institute holds its Scientific Retreat every year to discuss certain areas of interest to the NIAMS. Dr. Susana Serrate-Sztein, Director of the Division of Skin and Rheumatic Diseases, explained that this year’s Scientific Retreat focused the scientific topics of induced pluripotent stem cells (iPSCs), atopic dermatitis, and advancing research in tendon and ligament biology. The Retreat also included sessions on leveraging and strategic funding collaborations, as well as clinical trials portfolio analysis and NIAMS goals for clinical research.
iPSC Opportunities in NIAMS Diseases
Dr. Serrate-Sztein explained that the NIAMS decided to revisit the issue of iPSC research because of recent technological advances in the field that allow for a greater examination of current needs, gaps, and opportunities. Topics of discussion included:
- What NIAMS diseases/conditions and research areas are poised to take advantage of recent advances in iPSC technologies, and would adoption of iPSC approaches accelerate progress? What are the current limitations in the use of iPSCs for these applications?
- Are there appropriate in vitro cellular models for common and rare diseases of interest to the NIAMS that could be generated from patient-specific iPSCs?
- Are any of these models ready for: (1) studies of disease pathogenesis, (2) functional studies of gene variants, (3) high-throughput drug screening, (4) drug toxicity studies, and (5) studies of gene repair?
- What are the technological hurdles, and are special resources required to overcome them?
- What are the opportunities for leveraging and partnerships?
Best practices/goals, opportunities, and challenges were discussed. Best practices/goals include streamlining communication and enhancing collaboration throughout the community, standardizing terminology and protocols, and establishing common regulatory and technology transfer protocols. With regard to opportunities, Retreat participants identified monogenic diseases, disease-specific and/or personalized tissue models, and centralized resources (e.g., repositories, cell manufacturing facilities, iPSC control lines). Challenges include developing efficient and reliable cell reprogramming and differentiation techniques and protocols, cell sorting and purification, safe and efficient genetic "correction" methods, complex diseases, immune response, and therapeutic delivery methods.
Atopic Dermatitis (AD)
Dr. Serrate-Sztein commented that, despite the large market for the development of AD drugs, progress in this area has not been as rapid as hoped. Available commercial products that have been approved by the FDA provide relief to many AD patients but there are no significant treatments that cure the disease. Topics presented during this session included:
- What are the opportunities for accelerating research in AD that can lead to new therapeutic targets?
- What are the main factors that contribute to severe AD?
- What translational research opportunities have emerged from recent studies on the biology of AD?
- What are the opportunities, needs, and barriers to proof-of-concept trials in AD?
- What challenges arise with conducting trials in specific patient populations (e.g., children, variation in disease severity), and how can they be overcome?
- How should comorbidities be addressed, and what insights do they provide?
Retreat participants discussed: (1) mechanisms of disease/disease susceptibility (e.g., barrier disruption, immune dysfunction); (2) clinical presentation and clinical research (e.g., comorbidities, phenotyping complications, challenges with pediatric trials, quality of life outcomes); (3) future research directions (e.g., identification of primary disease etiology, better characterization of barrier defects and immune system effects on barrier function); and (4) emerging opportunities for intervention (e.g., molecular messengers in immune function, regulators of epidermal barrier and cell-cell junctions). Dr. Serrate-Sztein noted that in May, the Society for Investigative Dermatology (SID) held a meeting that included a session on AD and the need for national and international collaboration to reach agreement on new outcome measures that could be used in clinical trials. That meeting also touched on the need for biomarkers and the simultaneous study of the mechanisms of disease within the context of clinical trials. The NIAMS intends to work with the SID and experts around the world to advance the research agenda for AD.
Advancing Research in Tendon and Ligament Biology
Dr. McGowan noted that basic tendon and ligament research impacts the other NIAMS portfolios to a large extent. She reminded the group of the tremendous burden of injuries to these tissues in the population. These injuries often occur in young people participating in sports-related activities, and they have consequences after the repair of these tissues. Topics addressed during the session included:
- What are the opportunities for exploring the basic biology of tendons and ligaments?
- How can knowledge of tendon and ligament developmental biology inform tissue engineering and regenerative medicine approaches?
- Are failures and limitations of the current "bedside" approaches to tendon and ligament injuries pointing to gaps in our understanding of the basic biology of these structures?
- What are the most immediate clinical needs, and how can these gaps be addressed by investment in basic science?
- What are the obstacles in exploring the basic biology of tendons and ligaments?
- Does this area have a sufficient workforce/pipeline in basic research relevant to tendons and ligaments?
Discussion on this topic led to the identification of basic research opportunities, such as tendon and ligament structure and mechanical properties, pre-injury degeneration, and tendon development and healing. Obstacles in this area include developing better animal models and the small size of the tendon and ligament research community. Retreat participants identified a number of clinical needs, such as repair and rehabilitation strategies and strategies to repair large tears of the rotator cuff, the role and appropriate use of growth factors, understanding of the role of inflammation, and understanding why engineered grafts do not behave like normal tendons and ligaments. The NIAMS is considering what can be done with the information from these discussions to build bridges between fundamental biologists and the more applied scientists who are working in tendons and ligaments.
In response to a question from Dr. Bonewald, Dr. McGowan noted that the NIAMS plans to work with the Orthopaedic Research Society (ORS) and possibly hold a meeting that would attract fundamental cell biologists and developmental biologists who could provide expertise in addressing these issues. Dr. Bonewald noted that the ORS and American Society for Bone and Mineral Research have been exploring opportunities for collaboration, to move shared aspects of their areas of interest forward. Advancing research on tendon and ligament biology may represent such an opportunity.
Leveraging and Strategic Funding Collaborations
Dr. Serrate-Sztein noted that the impetus for this discussion was the challenging budget landscape and the Institute’s desire to re-examine the way in which it collaborates with others within the NIH, within the federal government, and with external organizations to maximize the use of research dollars, particularly in areas where there is a common research interest. This topic was discussed in the context of the benefits and challenges associated with collaborative funding, pilot project/seed funding, sustaining registries, infrastructure for planning groups, and industry expertise and resources in therapeutic and biomarker research.
Retreat participants discussed collaborative funding strategies within NIH with the cooperation of multiple ICs to understand complex diseases/comorbidities, the co-development of initiatives, and the Common Fund. They indicated that leveraging funding from non-federal, not-for-profit organizations can provide a "proving ground" for innovative concepts and could represent a potential source of bridge funding for new investigators seeking NIH support. In terms of public-private partnerships, Retreat participants noted the importance of involving funding partners and patient advocacy groups. The OAI was highlighted as an example of obtaining buy-in from industry leaders, unique resources provided by industry partners, and the NIH’s important leadership role.
Clinical Trials Portfolio Analysis and NIAMS Goals for Clinical Research
Dr. McGowan reminded the group that the NIAMS has been examining its clinical trials portfolio for some time and has designed a series of new initiatives to better enable the most impactful clinical trials to be submitted and considered by the NIAMS. Topics considered during the Retreat included:
- How does the Institute define the impact of completed studies? What are the most meaningful measures of success or predictive performance indicators?
- Are there over- or under-represented areas in the NIAMS portfolio?
- What approaches could be used to determine whether the NIAMS is supporting the most important studies and how to achieve that goal?
- What is NIAMS’s role in promulgating results of significant clinical trials and studies?
Retreat participants noted that, beyond the most important goals—influencing clinical practice and public health policy—clinical trials may inform future research, particularly if they contain some mechanistic studies. Dissemination of trial outcomes was also discussed, and it was noted that the requirement to provide results to ClinicalTrials.gov may not be sufficient. Retreat participants also identified three questions to consider before accepting a clinical trial application: (1) If this trial is successfully completed, will findings guide the field, and will they provide more information than just the disease being studied? (2) How timely is this trial—will events make it meaningless? (3) Do investigators have evidence (e.g., from electronic medical records) that they can recruit enough patients for a meaningful study?
CONSIDERATION OF APPLICATIONS
The Council reviewed a total of 822 applications in closed session requesting $1,151,308,126 in total costs and recommended 822 for $1,151,308,126 in total costs.
EMERGING TOPICS ON IMMUNE-MEDIATED SKIN DISEASES
This portion of the meeting occurred during closed session..
The 77th National Arthritis and Musculoskeletal and Skin Diseases Advisory Council Meeting was 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.
Laura K. Moen, Ph.D.
Stephen I. Katz, M.D., Ph.D.