National Advisory Board on Medical Rehabilitation Research (NABMRR)
National Center for Medical Rehabilitation Research (NCMRR)
Eunice Kennedy Shriver National Institute of Child Health
and Human Development (NICHD)
December 6-7, 2015
Dr. Anthony Delitto opened the 51st meeting of the NABMRR at 9:05 a.m. The new Board members introduced themselves, along with other Board members, ex officios, NIH staff, and staff of other federal agencies, and visitors. Minutes of the previous meeting (May 2015) were approved unanimously by voice vote. Future meetings are scheduled for May 2–3, 2016, December 5–6, 2016, and May 1–2, 2017.
Ex-Officio Members Present:
Daofen Chen, NINDS
Patricia Dorn, VA
Ralph Nitkin, NICHD
Lana Shekim, NIDCD
Lois Tully, NINR
Ex-Officio Members Absent:
Lyndon Joseph, NIA
Ann O'Mara, NCI
Michael Swanson, CDC
NICHD Staff and Visitors:
Rosalina Bray, NICHD
Theresa Brininger, NIHCC
Theresa Cruz, NICHD
Robyn Ellerbe, APTA
Craig Fisher, APA
Rohan Hazra, NICHD
Rusti Johnson, NICHD
Lisa Kaeser, NICHD
Tiffany Knowlton, AAP
Audrey Kusiak, VA
Susan Lin, MGH
Susan Marden, NICHD
Mary Ellen Michael, NICHD
Maria Nurminskaya, CSR
Christopher Platt, NIDCD
Linda Porter, NINDS
Eve Reider, NCCIH
Christine Rogers, NICHD
Mary Rogers, NIBIB
Rajni Samavedam, BAH
Carol Sheredos, NICHD
Karen Siegel, VA
Bio Tian, CSR
Tracey Wheeler, CHNFdn
Report of the Acting NICHD Director
Dr. Catherine Spong has been Acting Director of the NICHD since Alan E. Guttmacher, M.D., retired in September 2015. First, she presented the following news from the NIH. Walter Koroshetz, M.D., was appointed Director of the National Institute of Neurological Disease and Stroke (NINDS) in June. William Riley, Ph.D., was appointed Director of the Office of Behavioral and Social Sciences Research in August. Kay Lund, Ph.D., was appointed the first Director of the Division of Biomedical Research Workforce Programs. Michael S. Lauer, M.D., is the new Deputy Director for Extramural Research, replacing Sally Rockey, who moved to the U.S. Department of Agriculture. Bruce Cuthbert, Ph.D., is the Acting Director of the National Institute of Mental Health, replacing Thomas Insel, Ph.D., who stepped down as Director to join the Life Sciences team at Alphabet (Google).
The NICHD is searching for a new director to replace Dr. Guttmacher. Dr. Spong asked the Board to encourage qualified candidates to apply by the deadline, January 4, 2016.
The NIH 5-year strategic plan was due to Congress on December 16, 2015. The NIH issued a Request for Information (RFI) on the plan and has been working to include the comments and feedback received. NIH Principal Deputy Director Lawrence Tabak, D.D.S., Ph.D., has led the effort. The plan identifies the major research themes. The NIH Institutes and Centers (ICs) use the plan to develop their own goals.
The Precision Medicine Initiative (PMI) is an individualized approach to disease based on a person’s genes, environment, and lifestyle. The PMI has already begun in the field of cancer. The PMI will also create a cohort of 1 million volunteers of all ages who will share data from their electronic medical records. Enrollment will begin in 2016. It will take up to 4 years to reach the goal of 1 million participants. The NIH has released six funding opportunities related to the cohort program. The NIH is conducting a search for a director of the cohort program. Dr. Spong asked Board members to encourage qualified candidates to apply.
The National Children’s Study (NCS) was halted last year, but Congress has allowed the NIH to redirect the funds to other projects that support the mission and goals of the NCS. In 2015, $144 million will have been used to:
- Develop tools to enhance studies of environmental influences of pediatric diseases
- Study how the environment influences development in utero (the Human Placenta Project was funded under this initiative)
- Examine environmental influences on child development by leveraging existing programs
For fiscal year (FY) 2016, the NIH has a new program, Environmental influences on Child Health Outcomes (ECHO), which will use existing cohorts to investigate the longitudinal impact of prenatal and postnatal environmental exposures on pediatric health outcomes. The Requests for Applications (RFAs) will come out soon. The NIH is also looking for a Director for the program. Dr. Spong asked Board members to encourage qualified colleagues to apply.
The NIH has created the Institutional Development Award (IDeA) program to support faculty development and research infrastructure enhancement in 23 states and in Puerto Rico that also have rural and medically underserved populations. The NIH plans to create an IDeA States National Pediatric Clinical Trials Network by using redirected NCS funding.
The NIH established the Gabriella Miller Kids First Pediatric Research Program to develop a data resource of phenotype and genetic sequence data to investigate structural birth defects and childhood cancers. Several existing cohorts will be tapped for this project. Continuation of the program to FY 2016 depends on the availability of funding.
The NIH has issued a Common Rule Notice proposing revisions to regulations that govern research. Comments are due by January 6, 2016. Changes include a plan to streamline institutional review board (IRB) reviews, simplify consent documents, and increase privacy and security safeguards for research with biospecimens and data.
The NIH is clarifying expectations regarding rigor and transparency in research. As of January 25, 2016, all grant applications must include a scientific premise, a rigorous experimental design, consideration of relevant biological variables such as sex, and authentication of key biological and chemical resources. Dr. Spong asked the board members to circulate information regarding these principles to their colleagues.
Dr. Spong then shifted to news from the NICHD. The NIH Medical Rehabilitation Coordinating Committee (MRCC) is developing a comprehensive rehabilitation research plan. Comments on the plan were due on December 11, 2015. The conference, Rehabilitation Research at NIH: Moving the Field Forward, will take place May 25–26, 2016. The meeting will identify gaps in rehabilitation research at the NIH, provide an opportunity for input into the development of the NIH Rehabilitation Research Plan, and discuss infrastructure needs.
The Human Placenta Project (HPP) received $46 million in FY 2015 and published two RFAs; applications were due on December 18, 2015. The goal of the HPP is to understand the structure and function of the placenta in real time. The NIH will hold the third HPP meeting April 14–15, 2016.
PregSource™ is a crowdsourced interactive study that will provide information about pregnancy to the women who use the study’s app and allow them to compare their own experiences to the experiences of other pregnant women. Women will provide information about their pregnancy, helping to develop a detailed natural history of pregnancy. Nineteen partner organizations, including seven NIH Institutes, are helping to develop the app.
Dr. Spong provided some legislative and budget updates. The 21st Century Cures Act passed the U.S. House of Representatives in July and the bill has been referred to a Senate committee. Congress had not passed a budget for FY 2016 as of December 7, 2015, with a continuing resolution in effect through December 11, 2015.
Dr. Spong then invited discussion from the Board. One Board member asked how long the NCS funding would continue. Dr. Spong responded that some FY 2015 projects received 5 years’ funding up front. The NICHD anticipates using NCS funds for the ECHO and IDeA programs in FY 2016 and some projects could receive funding for up to 7 years. Another Board member asked whether the PMI would include rehabilitation projects. Dr. Spong said that the PMI is partnering with some rehabilitation organizations and that anybody can volunteer for the cohort. Dr. Ralph Nitkin encouraged rehabilitation investigators to get involved with the PMI.
Later during the Board meeting, Lisa Kaeser, NICHD Office of Legislation and Public Policy, provided some additional updates on these NICHD initiatives. The ECHO program is supported by funds from the NCS initiative. The 7-year program will draw on existing pediatric cohorts to answer questions about environmental exposures on pediatric development. The IDeA States Pediatric Clinical Trial Network is a 5-year program to train pediatric clinical trial experts at institutions in 23 states that receive fewer NIH dollars. The program will allow children to take part in clinical trials nearer to their homes instead of having to travel to another state. The network will include an operation and data coordination center. Any institution can contact the center to help recruit pediatric clinical trial participants. Dr. Cernich encouraged Board members to let eligible colleagues know about these opportunities. One way to get more money into rehabilitation research is to apply for funding from these programs. NCMRR staff can provide advice to applicants.
Report of the NCMRR Director
Dr. Alison N. Cernich first discussed the NIH-wide Rehabilitation Research Plan currently under development. According to current legislation, the NICHD Director is charged with developing a periodic and comprehensive research plan in consultation with the NCMRR director, the NABMRR, and the MRCC. The current process began in October 2015 with a request for public comment. The review of the public comments will continue through December 2015. Priorities and action items will be released in January 2016. The full research plan will be drafted and presented to the NABMRR at its May 2016 meeting. There will be a notice for public comment in June 2016. The final plan is expected to go to Congress by September 2016. Seventeen ICs have signed on to the plan. Board members were asked to provide input and suggestions. The draft agenda for the NIH Rehabilitation Research Conference (May 25-26, 2016) is available and speakers have been invited. The conference is of interest to anybody who has received NIH funding for rehabilitation research. The conference will include short presentations and a town hall meeting.
Dr. Cernich highlighted the Medical Rehabilitation Research Infrastructure Network (MRRIN). It consists of six centers funded by the NICHD, the NINDS, and the National Institute of Biomedical Imaging and Bioengineering (NIBIB). The network provides access to state-of-the-art research facilities, courses and workshops, mentorship and consultations, pilot grants, and other collaborative opportunities in key research domains. The current cohort of centers provides access to expertise in:
- Analysis of large data sets
- Biomechanics and modelling of movement
- Technology assessment and product development
- Clinical trial design
- Regenerative medicine
- Neuromodulation: clinical applications
Dr. Cernich provided some updates on the NCMRR. The Center is reviewing its portfolio and will likely focus less on diagnostics and more on rehabilitation. The NCMRR budget has been stable since 2014, when the Center was provided with access to a stable, set-aside budget as a percentage of the NICHD extramural total. This allowed the Center to be more proactive about seeking research collaborations with other NIH Institutes and pursing research opportunities. In the current FY, about 55% of NCMRR funding went to traditional research projects (e.g., R01s, R21s, R03s), 18% to small business grants, 15% to training and career development, and 12% to multi-component projects.
Dr. Cernich provided some ranking of funding across specific research domains and conditions. During FY 2011–2015, the NCMRR supported grants in prosthetics and robotics, pediatrics, pain, gait, wheelchair, and behavioral issues, among other areas, with a clear growth in the pediatric domain. The research covered conditions such as stroke, traumatic brain injury (TBI), spinal cord injury, cerebral palsy, and amputation.
Currently, the NCMRR has four health science administrators, a health specialist, a program support specialist, and an administrative assistant. The Center is looking at ways to expand staff, including through fellowships and internships, including a current internship to an impressive person with disabilities.
In the discussion session that followed, one Board member asked whether the funding levels that Dr. Cernich discussed included only NCMRR dollars, or did it include rehabilitation research co-funding from other ICs. Dr. Cernich said that the figures include only NCMRR dollars. Another Board member asked how often the NCMRR co-funds projects and Dr. Cernich noted that the NCMRR currently co-funds 27 projects. A Board member asked how much funding the NCMRR was currently leveraging by partnering with other ICs, but Dr. Cernich indicated that she did not know but would present that information to the Board in the future.
NICHD Data and Specimen Hub (DASH)
Dr. Rohan Hazra, who is overseeing this activity within the NICHD provided some background. NICHD DASH was launched in the summer of 2015 and currently includes an RFI seeking comments on ways to enhance the timely sharing of high-quality data generated by NICHD-funded clinical studies. Dr. Hazra invited Board members to respond to this request. The purpose of DASH is to maximize the NICHD’s investment in 2,000 clinical research studies, provide a way to share all types of data more efficiently and seamlessly, and comply with federal and NIH data-sharing and data-access policies
The NICHD DASH homepage has had 3,000 visits, and there are 160 users. Most visitors so far have been looking at the data that is already there but have not submitted new data. One purpose of the NICHD DASH is to change the culture so that investigators want to share data. Staff members are working with NICHD Branches to upload data to the site. Before making the data available, staff must anonymize the data by removing all of the 18 identifiers defined by the Health Insurance Portability and Accountability Act (HIPAA) and ensure that all permissions are in place. NICHD DASH currently has data from 14 NICHD-funded studies. And several dozen more are waiting to be incorporated once they have been de-identified. The aim is to upload one or two new studies every month. Dr. Mary Ellen Michel is the NCMRR staff contact for rehabilitation research contributions to NICHD DASH. NICHD DASH is now working to provide investigators a way to submit their own data and to view and request associated biospecimens that capability should be available in early 2016. NICHD DASH will also provide links to related data repositories outside the NICHD.
Submission of data from NICHD-funded studies is encouraged but not required. Investigators submitting data must submit certification from their institution that the study IRB has approved the submission. To submit data, the investigator will obtain institutional certification from his or her institution. The data is placed in a data archive until it is de-identified. Once the data is de-identified, the NICHD Branch that funded the study will sign off on the data’s inclusion, making the data available. Biospecimens will require a more stringent scientific review.
Investigators requesting data must submit an online data request form and a data use agreement co-signed by the lead investigator and the investigator’s institution. The NICHD DASH Data Access Committee will determine whether the request is approved. Investigators who use the data will be required to acknowledge the data’s source. Data donors will get a report of how their data has been shared—information that they could include on their curriculum vitae and possibly in connection with tenure decisions. The NICHD DASH home page has resources for investigators, including the full NICHD DASH policy, the data use agreement, and the data request checklist.
In the discussion that followed, one Board member asked whether NICHD DASH is open only to NICHD-funded projects. Dr. Hazra said that it contains only data from NICHD-funded studies, but any researcher can request to use the data. Another Board member asked whether there are ways to streamline the process. Dr. Hazra said that retrofitting data to include in NICHD DASH has been time-consuming. The staff is trying to streamline the process because otherwise there will be a backlog of studies to retrofit to the database. A Board member asked whether NICHD DASH would accept large datasets containing raw data. Dr. Hazra said that the staff would find a way to include the data regardless of the size of the database. NICHD DASH is storing data on the cloud, so there is unlimited capacity. A Board member asked whether studies involving pediatric trauma and rehabilitation will be included in NICHD DASH. Dr. Cernich said that the NCMRR and the NINDS fund pediatric brain and spinal cord injury studies, and trauma studies that would be included. A Board member asked whether NICHD DASH can interface with repositories containing gene expression data. Dr. Hazra said that NICHD DASH will contain phenotypic data and will interface with other repositories, but it can be difficult to link data. A Board member asked whether data could be shared from projects in which the informed consent document did not specifically address data sharing with participants. Dr. Hazra said that it is possible to share data when the informed consent document is silent on the issue. The submitting institution, perhaps in the form of the IRB, must agree that it is appropriate to share the data with NICHD DASH in that case. A Board member asked how the existence of NICHD DASH could be disseminated more widely. Dr. Hazra said that NICHD placed a notice in the NIH Guide, e-mailed constituent groups, and is doing presentations in the research community. The NICHD has not yet advertised widely, because NICHD DASH is just ramping up. Each time that NICHD DASH receives a new study, announcements will be disseminated, helping to get the word out. Three studies related to rehabilitation will be entered into DASH. The rehabilitation community will be contacted when those are uploaded.
NICHD Training Committee and NCMRR Priorities
Dr. Ralph Nitkin indicated that the NICHD has been reevaluating training and development programs and convened a task force that included NICHD staff, colleagues from other parts of the NIH, and NICHD researchers. Particular challenges for the NCMRR include finding the best mix of funding mechanisms for training and career development, contrasting K12 networks versus individual K awards, the appropriate range of clinical specialties, and what outcome measures should be used to document success of specific programs and support mechanisms.
Dr. Nitkin discussed some recommendations from the NICHD task force. Currently, the Institute supports training grants and individual fellowships for predoctoral and postdoctoral scholars, career development awards to facilitate the transition from postdoctoral to faculty, and mentored support for early-career faculty through individual awards and for specific clinical fields K12 networks. About 5 to 7% of the NICHD budget goes currently goes to the various training mechanisms—a percentage comparable to what ICs of similar size are spending; the NICHD task force recommended that the Institute, at a minimum, maintain the training budget, however, more resources should be allocated for late-stage postdoctoral and early faculty levels, particularly for those with non-clinical degrees.
The NICHD relies on institutional T32 and K12 awards far more than other ICs do. The task force recommended that the NICHD rebalance the portfolio by shifting funds from institutional awards to individual awards and to better fit the NICHD mission. The task force recommended giving priority to diversity, including underrepresented minorities and people with disabilities, by increasing the individual F31 and F32 fellowships. The task force also recommended using a more multifaceted formula for gauging the success of NICHD training programs, including tracking the number of grantees who continue in research-related careers, the funding that they win, and their publications.
Dr. Nitkin asked the Board to comment on whether the NCMRR has the appropriate mix of support mechanisms for training and career development, how it should balance institutional K12 networks versus individual K awards, whether the right mix of professions are being supported by K12s, what is the value-added of K12 networks, how do we evaluate success in terms of current needs, capacity, and support mechanisms, and workforce constraints, and at what point do we consider a sunset strategy for current support networks especially with the growing constraints on career development resources in general.
The presentation on training and career development raised a lot of discussion with the Board. One member commented that one critical issue in evaluating the programs is how to define success and that the measures of success may vary by discipline. Another member pointed out that individual K awards provide 5 years of support, while institutional K12 networks only provide (direct) support to the candidates for 2 or 3 years, and that it takes 5 years to train a clinician. A Board member asked whether other ICs have evaluated their training support and Dr. Nitkin indicated that all the ICs are wrestling with these questions and trying to balance the individual and institutional programs. Moreover, the question was raised about whether there would be enough academic jobs available to justify training these levels of scholars.
A Board member asked whether placing more of an emphasis on individual K awards could result in more funding for rehabilitation research. Dr. Cernich said that the NCMRR would rebalance its individual and institutional awards, but the total rehabilitation research funding within the NICHD would likely remain the same. Dr. Nitkin suggested that the NCMRR would follow the lead of rest of the Institute unless the Board can make a case that the NCMRR is sufficiently different and should take a different approach. A Board member said that the NCMRR is different; rather than looking for a cure, rehabilitation aims to improve the quality of life and perhaps this critical metric is missing in the evaluation of rehabilitation researchers.
One Board member asked how effective the institutional K12 programs are across the NICHD. Dr. Nitkin cautioned that it is difficult to compare programs, because they serve a range of career levels and clinical specialties. However, on at least two metrics—remaining in research and applying for and receiving grants—rehabilitation researchers did as well as or better than scholars from K12 programs. A Board member said that the clinical impact of the research is an important outcome to consider when evaluating the success of these programs. Dr. Cernich said that it is difficult to find a way to measure the impact that a researcher has on patient outcomes and solicited the Board’s ideas about appropriate metrics. Dr. Nitkin added that research findings can take a long time to impact clinical practice.
One Board member indicated a preference for institutional K12 programs because they include interdisciplinary training, which is especially important in rehabilitation research and that the NCMRR should customize the awards programs to the needs of rehabilitation research. Dr. Nitkin indicated that the NCMRR could develop local metrics above and beyond the Institute-wide measures to evaluate programs. Dr. Cernich said that there could be a change in strategy to focus on more interdisciplinary approaches, but the goal of developing capacity in rehabilitation research would remain.
A Board member asked whether there is any difference in the quality of the applicants between the individual and institutional K12 awards, but Dr. Nitkin indicated that the analysis has not been done. The Board member added that individual awards allow the trainees more choices about where to train while network K12 awards are limited to specific locations. A Board member suggested that institutional K12 awards allow trainees to establish wider professional networks within their fields, which can be invaluable in the long run. Dr. Cernich agreed but said that it is possible to give those on individual awards opportunities for networking and interdisciplinary training. The U.S. Department of Veterans Affairs (VA) gives only individual awards but convenes trainees for mentoring, networking, and interdisciplinary training. Dr. Michel said that because rehabilitation is trans-NIH, it may be possible for NCMRR awardees to attend gatherings of K awardees from the NINDS, the National Institute of Diabetes and Digestive and Kidney Diseases, or other ICs with whom an NCMRR awardee may share research interests. Board members were encouraged to submit written comments to Drs. Cernich and Nitkin; the board may form a subcommittee to draw up a plan for the training program.
NABMRR Subcommittee Update: Rehabilitation Research Priorities
The trans-NIH MRCC has developed a draft set of research priorities, which a subcommittee of the Board reviewed. Dr. Gerard Francisco presented the draft plan to the Board, along with comments from the subcommittee. The plan focused on the priorities of rehabilitation across the lifespan, family and community, technology, and research design and methodology.
Priority 1: Research Across the Lifespan
The draft priorities recommend more investigations of rehabilitation interventions for children, particularly:
- Taking what is already known about adults and adapting it to children
- Focusing on the transition from childhood to adulthood
- Adapting devices and other technology so that users can continue to use the devices as they grow
The priorities also recommended moving towards understanding the effects of disability and functional limitations across the lifespan:
- Studying the natural history of the condition
- Identifying strategies for care and delivery of care for individuals with disabilities throughout life
- Determining factors that affect health care delivery during the aging process
- Integrating considerations of aging into device and assistive technology development
- Advancing understanding of the role of modifiable lifestyle and health behaviors on the effectiveness of rehabilitation interventions or on disability prevention
- Clarifying the effects of disabilities and functional limitations
- Examining symptoms and sequelae associated with disability
The subcommittee concluded that the first priority, rehabilitation across the lifespan, was too broad. The subcommittee suggested ways to focus the priority, including investigations into how to tailor treatments to developmental levels or maturation, the benefits of exercise beyond strengthening and aerobic capacity building, health disparities in the pediatric population, and combination treatments such as combining drugs with exercise. The subcommittee said that there was too great an emphasis on nutrition and that more work was needed to incorporate the effects of multiple chronic conditions on people with disabilities.
In the discussion that followed, a Board member asked whether neurodegenerative diseases and the role of rehabilitation were considered. Dr. Cernich said that the MRCC used the term “lifespan” to avoid referencing specific conditions because these priorities would be used across the ICs. However, neurodegenerative conditions were discussed as these priorities were drawn up. Another Board member asked about the impact of changes in health care policy that restrict access to rehabilitation, reduce the length of stay in acute rehabilitation, and make subacute rehabilitation harder to access. Dr. Francisco said that this is important, and there was mention of health economics in the fourth priority. A Board member suggested examining the effect of exercise on functional outcomes such as participation in school and especially the economic impact of preventing secondary complications. Another board member agreed, also highlighting the impact of exercise on cognitive abilities and a range of other skills.
Priority 2: Family and Committee
The second priority includes examining the impact of sociodemographic influences such as geography and education on rehabilitation success, developing self-management strategies to use in community settings, and examining the stressors and challenges of caregivers. The subcommittee approved of this priority because it is responsive to a report by the Blue Ribbon Panel. The MRCC asked whether this priority should be included in the life span section, but Dr. Francisco said that the subcommittee preferred this as a separate priority. There was some discussion of the need to develop interventions to address the stressors of disability and rehabilitation and how to improve compliance.
In discussing this priority, one member asked whether the effectiveness of the interventions would be measured. Dr. Cernich said that the NIH is already doing so and will consider the effectiveness of the intervention, the effectiveness of adapting current interventions with new technologies, and the effectiveness of developing new interventions. A Board member said that this priority should include the economic impact of disability. Caregivers may need to leave work and lose their own health insurance and they may be forced to rely on subsidies, increasing the nation’s health costs even more. Dr. Nitkin commented that different strategies may be required within various communities and he asked whether that had been taken into account in the plan. Dr. Cernich said that the research priorities plan is meant to be a very broad description of what will be done. How it will be done will require greater specificity and will come later. Defining the communities would be too specific for this document.
Priority 3: Technology and Device Development
The third priority included advancing the use of telehealth in rehabilitation assessment, delivery, and adherence monitoring; advancing the use of assistive technology and noninvasive sensors; exploring novel platforms to deliver care to remote sites; developing mobile health apps in rehabilitation science; providing an evidence base for device fabrication and implementation for individuals with disabilities; supporting the development of neuroprostheses; and encouraging the use of computational models for the design and development of new technologies. The subcommittee found this section to be strong and on target. Board members agreed and made no additional comments.
Priority 4: Research Design and Methodology
The fourth priority was to improve the design and methodology of rehabilitation investigations, especially in the area of clinical trials; to make precision medicine relevant to rehabilitation medicine; and to advance understanding of “-omics,” including genomics and proteomics. The subcommittee had many comments on this priority area. The subcommittee recommended the use of databases and registries as mechanisms for discovery, encouraged the funding of multisite collaborative trials, encouraged study of the effectiveness of interventions in pilot studies before moving to larger trials, and recommended economic analysis of proposed interventions. The subcommittee also commented on the precision medicine portion of the section, recommending that patient-driven outcomes be made concordant with the patient-driven outcomes of other funding agencies and that funding supplements be used as a way to include people with disabilities in more research.
In follow-up discussion, one Board member asked how much support there is within the NIH for pilot clinical trials and whether there is funding for a pilot trial to get preliminary data in order to organize a larger trial. The Board member also asked whether investigators should be encouraged to develop interventions that are more health oriented as opposed to advancing basic science. Dr. Francisco said that there is a need to understand better the safety and efficacy of interventions and the variables that might affect those interventions. From that perspective, it makes sense to study a smaller sample before moving to a larger trial. Dr. Cernich said that the NIH is reevaluating how it conducts large clinical trials to ensure that they can meet recruitment goals, among other things. Another point is that pragmatic or adaptive trials may be better for rehabilitation research, and sometimes reviewers are less open to those. The NCMRR is open to suggestions, comments, and questions from Board members on this issue.
A Board member suggested consideration of who the audience for the trial is; a trial to inform an intervention takes a different approach from a trial to influence policy or payment. There is not enough money to do it all, so the decision about the study’s purpose should be made in the planning stage. Another Board member asked whether the primary aim of the NCMRR is to affect science or insurance company policies and payments. Dr. Cernich said that it is important to keep in mind that this is an NIH-wide plan and that the NIH mission is to promote public health and reduce disability through scientific discovery. A Board member indicated that science is useful when it can be used to provide treatments or interventions to people with disabilities, and asked if there was a way for the NIH to reach out to payers so that discoveries can be translated to clinical use. Dr. Cernich responded that the NIH does regularly meet with and bring these issues to payers, health care systems, legislators, and other federal agencies but that the NIH can only do so much and then the payers have to take it from there. Dr. Francisco said that there is a lot of great science but no infrastructure to support getting a clinical application to patients, but Dr. Cernich said that the NIH can only try to persuade other entities.
A Board member raised a point made earlier to include the impact that disabilities have on the entire family. Another point is to consider interventions within the context of the changing health care system. A Board member said that it will be difficult to follow patients long-term when private insurers are limiting the amount of therapy that patients can receive. Another Board member said that it may be best to consider the economic aspect of everything that the Board does. It is possible to drive good science while being cost-conscious by carefully selecting projects and taking approaches such as promoting community partnerships. Dr. Cernich agreed that the end user must be kept in mind in any NIH research, but the research plan must also cover basic research. This is not only about human trials but also about basic science and animal trials, which are needed to understand mechanisms. A Board member agreed but said that the NCMRR’s unique function is to emphasize the human element in research.
The subcommittee said that the plan contained too little on cardiovascular, pulmonary, cancer, and musculoskeletal rehabilitation, and that greater emphasis was needed on treatment and secondary conditions and on plasticity and exercise. The subcommittee added that there were more policy statements than research priorities. Subcommittee members also said that there should be a section in the preamble related to current funding in some of these areas and that the gaps should be pointed out. Dr. Cernich indicated that the subcommittee’s work will help refine the plan and she asked Board members to share any further comments that they had by December 11, 2015. The NCMRR will be talking to federal partners about coordinating this research effort.
Feedback from the RFI on Rehabilitation Research Priorities
Ms. Rosalina Bray, NICHD Office of Science Policy and Communication (OSPAC), reviewed the comments received on the NIH-wide rehabilitation research priorities. The RFI was released on November 3, 2015, and invited public comments up to December 11, 2015. Although the response period had not quite closed at the time of the presentation, this briefing summarizes what had been received up to that point. The NIH had received 28 submissions from a range of individuals and organizations. Respondents were asked to comment on proposed research priorities that included the four areas that Dr. Francisco had covered in his presentation: rehabilitation across the lifespan, family and community, technology use and development, and research design and methodology.
Respondents were also asked to comment on some research topics that included:
- Potential benefits, challenges, and areas of consideration for the proposed priority areas
- Compatibility of the proposed priorities with the NIH mission
- Compatibility of the framework of the NIH Rehabilitation Research Plan with available models of rehabilitation research
- Comprehensive trans-NIH research themes in rehabilitation that have not been captured in the priority areas
- Future opportunities or emerging research needs
OSPAC will review the responses to look for agreement, concerns, or recommendations with the priority areas. OSPAC is coding recommendations to either priority areas or topic areas and coding themes under concerns and issues. Analysis thus far indicates that most commenters supported the framework and its compatibility with the NIH mission, but most suggested changes or the inclusion of additional areas of research. There was strong support for basic and applied sciences, but many respondents emphasized clinical research and behavioral and social sciences. Respondents noted emerging opportunities in data science and technology that would accelerate data collection and analysis. There were recommendations to change the review process and procure more funding.
About 70% of the commenters said that they had concerns with the priority areas. Among the concerns expressed were little mention of subfields, not enough emphasis on training of researchers, little mention of funding in specific areas, and not enough emphasis on determination of the effectiveness of rehabilitation interventions. Among the comments were that basic scientists dominate the review panels, that there is a need for interventions for geriatric and cancer patients, that there is a need to determine the effectiveness of current rehabilitation interventions, that there is a need to specify functional targets, and that there is a need for economically sustainable programs for the future.
Commenters had the following specific suggestions:
- Include the definition, classification, and measurement of rehabilitation treatments as an important and neglected area of methodology development
- Include a multifaceted approach to disability reduction
- Emphasize interventions for disability and participation restrictions, not just impairment reduction
- Prevent or ameliorate lifelong disability through early identification and intensive therapy
- Train more rehabilitation researchers from a variety of health fields
- Develop quality indicators, including measures of the impact of interventions
- Include people with disabilities on research teams
OSPAC will prepare the final summary and present it on January 10, 2016. Dr. Cernich urged Board members to spread the word that the comment period for the rehabilitation research plan is open. The plan can reflect the community’s thinking only if the community participates.
Rehabilitation Resource Centers: New Awardees and Dissemination
Dr. Nitkin provided some background on the resource program. In response to suggestions from the Board, Rehabilitation Resource Centers were developed in 2000 to help build the research infrastructure in medical rehabilitation and enhance research capacity. Initially, the NICHD committed $4 million annually to establish networks in four geographic areas, with a big emphasis on mentoring.
During the next 5 years (2005–2010), the regional constraints were removed so that researchers could connect with a center in any region. The centers provide expertise in specific domains related to rehabilitation research, including courses, workshops, mentorship, and pilot grants. With additional support from the NINDS and the NIBIB, six networks were funded.
The program was renewed for 2010-2015, with a significant turnover in centers. Through continued support from the NINDS and the NIBIB, seven centers were funded – and they provided broad expertise across the range or rehabilitation approaches and expertise. A common website portal was developed to provide access to the seven programs.
For the next phase, 2015 to 2020, the NCMRR specifically encouraged the development of programs that would focus on clinical trial design, engineering and environment, individualized medical rehabilitation and dynamic reassessment, and applied behavioral supports for rehabilitation research and healthy outcomes. Again with a healthy turnover, six centers were funded, which now provide expertise in:
- Analysis of large data sets
- Biomechanics and modeling of movement
- Technology assessment and product development
- Clinical trial design
- Regenerative medicine
- Clinical applications of neuromodulation
Each center provides courses, workshops, and access to expertise in critical research domains. In the past funding round, the centers funded promising pilot projects and prompted applications for funding to more than a dozen other NIH Institutes and other federal agencies.
Dr. Nitkin indicated that the Board should consider how much longer the NCMRR should continue to provide specialized infrastructure for rehabilitation researchers and what metrics should be used to measure their effectiveness. Dr. Cernich added that the NCMRR wants to ensure that this investment pays off, and she asked how to spread the word to the research communities to ensure that the resource widely accessed. The NCMRR is also encouraging the centers to be even more proactive and creative about seeking these connections. Dr. Nitkin indicated that outcome metrics were written into the grant; this could include measures such as the number of requests for consultation that they have received, the number of grants submitted as a result of the center’s work, and the number of successful applications.
A Board member asked how many contacts came from within the institution hosting the center versus the larger rehabilitation community. Dr. Nitkin said that the pilot grants have to go out to investigators outside their institutions. A Board member asked whether there is a simple way for potential researchers to contact the centers and whether the NCMRR could facilitate this process. Dr. Cernich indicated that the NCMRR is working to transfer responsibility for maintaining the common portal to the new centers with increased functionality. A Board member suggested that the centers could communicate success stories as a good way to build a following in the research community. Dr. Cernich agreed and said the NCMRR had recently met with representatives of the six centers; there were very enthusiastic about working together and had already set up a shared online workspace to share documents. Moreover, the established centers that successfully renewed are sharing their best practices and outcome metrics with the three new centers. A Board member asked whether there is a way to work with the National Institute on Disability, Independent Living, and Rehabilitation Research (NIDILRR) with respect to shared research infrastructure and Dr. Cernich indicated that she could talk to NIDILRR about that. Another Board member suggested that the rehabilitation research plan priorities could provide guidance on the value added of rehabilitation research infrastructure and at what point the centers should be phased out.
Nominations for Chair of the NABMRR
Tony Delitto will be stepping down as Board chair after the May 2016 meeting, so nominations were sought for the chair-elect, who would take over as chair after the May meeting. After a brief discussion, two nominations were put forth and in a close vote by private paper ballot, Amy Bastian was selected.
Discussions with Board: Public Comment and Input
Dr. Alexander Leonessa of the National Science Foundation (NSF) indicated that interagency agreements could be used to enhance funding for projects. For example, the NSF could support a clinical feasibility study to develop a larger clinical study. The NSF also does outreach; it gave a network a $1 million supplement to teach strategies to recruit and retain people with disabilities. Dr. Leonessa suggested further discussion during the interagency meeting scheduled for the following day after the Board meeting. A Board member complimented the leadership of the Board and the NCMRR, saying that the agency had successfully completed work with the NIH Blue Ribbon Panel and that rehabilitation research is on a good path that can lead to success. No additional public comments were made during this discussion period.
Focus on Pain
Dr. Linda Porter of the NIH indicated that the NIH Pain Consortium ensures that pain research is coordinated across the NIH. There are 27 ICs involved in the consortium and Dr. Walter Koroshetz, the Director of the NINDS, is the chair of the executive committee. The Pain Consortium was established in 1996 and reinvigorated by former NIH Director, Dr. Elias Zerhouni, in 2003. The Consortium has developed clinical research resources, issued RFAs for pain research, and worked to expand NIH pain research funding. It also developed common data elements to harmonize data across studies of lower back pain and is working to harmonize data for overlapping chronic pain conditions such as temporomandibular joint disorder and migraine headaches. There continues to be a need for common elements for clinical trials. The Consortium funded an open-source patient data registry that Stanford University is running where patients input their own data. The registry can help establish the prevalence of pain conditions and their outcomes.
The Consortium has worked with pain experts who have advised them on areas of research needs. One such collaboration brought together a research effort funded by the NIH Institutes that supported neuroscience research for $25 million over 5 years to focus on neuropathic pain. The Consortium also supported a consensus conference at which experts outlined efficacy and safety recommendations for opioid use with chronic pain patients. NIH pain research funding has increased over the past 10 years and in FY 2015, the NIH was spending $414 million on chronic pain research.
The Patient Protection and Affordable Care Act (PPACA) contained a bill related to pain care that called for an Institute of Medicine (IOM) report; the 2011 report, Relieving Pain in America: A Blueprint for Transforming Prevention, Care, Education, and Research, which highlighted the need to expand pain research. The PPACA also called for the establishment of the Interagency Pain Research Coordinating Committee (IPRCC), composed of federal and private partners. The committee meets twice a year to coordinate pain research across the federal government. The IPRCC put together the Interagency Pain Research Portfolio database for pain research funded at all federal agencies, to help coordinate and plan future pain research and help develop a federal pain research strategy. The database contains information on 1,300 grants, is accessible to the public, and can be broken into thematic areas.
The IPRCC is working on a 5-year federal strategy to identify gaps in pain research, define next steps, and prevent duplication of efforts across federal agencies. The strategy, which will be aligned with the National Pain Strategy, will define research needs and create a plan to move forward. The IPRCC expects to complete the strategy by the end of 2016.
NIH Rehabilitation Research: Opportunities for Pain Research
Dr. Cernich asked the Board members for advice regarding NCMRR pain research. She asked whether the NCMRR should join the Military/Veterans Pain Initiative request for proposals and, if the NCMRR does join, which projects might be best to fund. A Board member agreed that the NCMRR should join the military initiative. The best projects would be those that include many modalities. Another Board member also indicated that it would be helpful to research complementary treatments, as a first step toward having them become reimbursable.
A Board member encouraged the NCMRR to investigate neuromusculoskeletal pain, which is a significant source of patient pain and Dr. Cernich agreed. Several ICs are interested and could join in funding efforts in this area. Dr. Nitkin added that the NCMRR is not seen as a primary funder of pain management research but that making a statement about the importance of specific areas of pain management research can bring in more applications.
A Board member said that the NCMRR should emphasize both that pain management and rehabilitation beginning in the acute phase, and that early interventions should include a multidisciplinary approach. Another Board member indicated that posture and gait training can alleviate lower back pain and that more research could be done in that area. A Board member said that some areas of investigation include amputees and pain, how individuals who cannot talk can communicate their pain, and how assistive technology can reduce pain. Dr. Cernich indicated that the NCMRR could join projects within the pain initiative that fit the needs that the Board has identified as important.
Dr. Cernich asked whether there were other issues about pain and rehabilitation that that the NCMRR should consider. A Board member said that cancer pain is one area that could be singled out. Another issue concerns gender differences in pain. Some neuroimaging has shown that there are gender-specific responses to pain. Another member indicated interest in pain perception based on childhood experiences, both positive and negative. Does girls’ increased participation in athletics change how girls manage pain in a positive way? Does early physical abuse affect the experience of pain in adulthood, and is there a gender difference here? A meeting participant said that it would also be important to study how the pain associated with a childhood disability could affect pain later in life.
Board members agreed that it is important to understand how pain can affect rehabilitation. For example, pain causes balance problems, and Parkinson’s disease patients have pain, but pain is not one of the outcome measures in rehabilitation of people with Parkinson’s disease. A Board member said that major discoveries about health have been made because of the study of people with disabilities. Studying people with disabilities could provide a way to better understand the basic science of pain in the general population.
Dr. Cernich said that some of the issues suggested by the Board included encouraging inclusion of pain as an outcome in rehabilitation trials, investigation of pediatric pain and rehabilitation, and inclusion of the biopsychosocial model in pain research. Neuropathic pain has also been identified as an area of investigation. A Board member said that the Patient Reported Outcomes Measurement Information System (PROMIS) is an efficient way to include many variables, such as depression, into a study on pain.
New Business and Agenda for Next Meeting
The following topics were suggested by Board members:
- Disability outcomes initiatives
- NIH common data elements
- IDeA States Pediatric Clinical Trial Network
- Rehabilitation research plan draft
- NCMRR metrics for success that go beyond number of grants and publications
Dr. Cernich encouraged Board members to send her suggestions about items that should be discussed or any other suggestions for future meetings.
The meeting adjourned at 11:45 a.m.