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 3–4, 2018
Robyn Watson Ellerbe
Arthur W. English
Edelle C. Field-Fote
Allen W. Heinemann
Thubi H. A. Kolobe
Kenton R. Kaufman
Craig M. McDonald
Elizabeth R. Skidmore
Ex-Officio Members Present:
Alison Cernich, NICHD
Daofen Chen, NINDS
Robert J. Jaeger, NIDILRR
Mary F. Lovley, OSERS
Matthew Scherer, DoD
Lana Shekim, NIDCD
Ex-Officio Members Absent:
James Anderson, DPCSI
Jerome L. Fleg, NHLBI
Lyndon Joseph, NIA
Chuck Washabaugh NIAMS
NICHD Staff and Visitors:
Joseph Bonner, NICHD
Leif Brierley, PPSV
Scott Campbell, AOTA
Theresa Cruz, NICHD
Shawnise Durham, NICHD
Tinera Fobbs, NICHD
Christine Hunter, OBSSR
Lyn Jakeman, NINDS
Scott Janis, NINDS
Lisa Kaeser, NICHD
Naomi Kleitman, CHN Fdn
Michael Kurilla, NCATS
Susan Marden, NICHD
Maria Nurminskaya, CSR
Louis A. Quatrano, NICHD
Agnes Rupp, NIMH
Karen Siegal, VA
JoAnn Thierry, CDC
Biao Tian, CSR
Lois Tully, NINR
Elizabeth Wilson, NICHD
The Board observed a moment of silence for President George H. W. Bush. His special role in signing the landmark Americans with Disabilities Act into law in 1990 was highlighted.
Future Board Meetings
The Board will meet May 6–7, 2019; December 2–3, 2019; and May 4–5, 2020.
Introductions of New Members
The Board welcomed five new proposed members: Arthur W. English, Ph.D.; Thubi H.A. Kolobe, Ph.D., PT, FAPTA; Barbara Lutz, Ph.D., RN, CRRN, PHNA-BC, FAHA, FNAP, FAAN; Brian Ruhe, Ph.D., CP; and Rob Wudlick. Board members introduced themselves, followed by brief introductions from NIH staff and other visitors.
Minutes from the previous meeting of May 2018 were unanimously approved.
NICHD Director’s Report
Diana Bianchi, M.D., Director, NICHD
Dr. Bianchi provided updates on the NICHD budget and the strategic planning process. She also summarized recent NICHD research initiatives.
NIH and NICHD in particular received increased funding in fiscal year (FY) 2019. NICHD’s appropriated budget for FY 2019 is $1.506 billion, an increase of 4 percent over FY 2018, when it was $1.43 billion. NIH is grateful to Congress, which appropriates the budget. NICHD used its FY 2018 increased funding to do the following:
- Supported 60 early-stage researchers to help them achieve independence—more than double the number who were supported in FY 2017
- Added $4 million to the NCMRR budget
- Increased funding by more than $2 million for small businesses to commercialize biomedical technologies such as prosthetics and other assistive devices
- Increased the NIH budget for Down syndrome research from $35 million to $58 million
- Provided $30 million to launch clinical trials to identify, treat, and care for babies exposed to opioids in utero
NICHD Strategic Planning Process
The 21st Century Cures Act requires NICHD to have a strategic plan. The strategic plan will enable internal and external stakeholders to review and refocus NICHD’s science, rebrand NICHD, clarify what NICHD does, align resources with scientific priorities, and improve the health of the population that NICHD serves.
The planning process so far has included meetings to obtain input from the Friends of NICHD and from intramural and extramural scientists. The process will be transparent, and decisions will be informed by evidence. Among the questions are whether NICHD should focus more on clinical, basic, or translational research and how to integrate NICHD’s broad scientific range and its many stakeholders.
In preparation for the strategic plan, NICHD has analyzed and divided its portfolio up by scientific and public health domains. According to this analysis, NICHD spent 59 percent of its FY 2017 budget for basic science, 15 percent for population science/epidemiology, 14 percent for screening and diagnosis, 19 percent for biomedical interventions, and 23 percent for behavioral interventions. About 1 percent was spent on training and infrastructure. (These categories are not mutually exclusive.)
NICHD spending by public health category included about 55 percent for pediatrics and child development, 15 percent for gynecology and reproductive diseases, 14 percent for pregnancy and maternal health, 18 percent for intellectual and developmental disabilities (IDDs) and physical disabilities, and 7 percent for things such as acute rehabilitation and for infrastructure. Other areas that NICHD funds include rehabilitation, autism, learning disabilities, stroke, mental illness, spinal cord injury (SCI), and traumatic brain injury (TBI).
NICHD research grants over the past 10 years have led to publications in scientific journals across a range of scientific domains and fields. NICHD’s large extramural programs have influenced practice guidelines. For example, about 26 percent of publications produced by the Maternal-Fetal Medicine Units Network are cited in professional practice guidelines.
NICHD funding also has a measurable impact on technology development, as evidenced by the number of patents that cite NICHD research.
NICHD held a meeting of the Strategic Planning Working Group in October, NICHD staff have formed working teams, and NICHD held a live interactive webinar for stakeholders. Anyone with questions or ideas can email them to NICHDStrategicPlan@nih.gov or connect with NICHD on Facebook and Twitter . NICHD will also issue a request for information. More information on the strategic plan is available on the NICHD website.
Action Item: Board members should provide input on the following questions:
- What are the most important research priorities for NICHD over the next 10 years?
- What is the most important thing that NICHD could accomplish for the public, patients, and health care providers over the next 5 to 10 years?
- What emerging technologies and techniques are likely to affect the types and methods of research conducted in the next 5 to 10 years?
Noteworthy NICHD Research Initiatives
NICHD Young Investigators Conference. The conference took place in August as a “bootcamp” for M.D.s who have an interest in research. This year the conference was extended to include other NICHD-relevant clinical domains such as medical rehabilitation. The training included a mock study section and information about setting up a wet lab. Attendees networked with NICHD program staff and learned about lesser known grant mechanism and career-development opportunities. The conference is meant to encourage physician scientists to continue the research portion of their careers.
Trans-NIH Pediatric Research Consortium (N-PeRC). N-PeRC will explore gaps and opportunities in the pediatric research portfolio across NIH. One idea is to make better use of the Best Pharmaceuticals for Children Act by identifying drugs that should be studied for use in children. Among other tasks, N-PeRC will create a pediatric genomic data commons and support training to increase the pediatric workforce.
Helping to End Addiction Long-termSM (HEAL) Initiative. NIH doubled its funding of opioid misuse/addiction and pain research from $600 million to $1.1 billion between FY 2016 and FY 2018, thanks to a funding boost from Congress. NICHD is using HEAL funds to support research on neonatal opioid withdrawal syndrome. NCMRR is involved in HEAL pain treatment alternatives studies. Funding opportunity announcements (FOAs) will be coming out in the next few months.
The Task Force on Pregnant Women and Lactating Women (PRGLAC). PRGLAC has completed its work and sent its report to Congress and to the Secretary of Health and Human Services. The report’s recommendations include a call to remove pregnant women from the Common Rule’s list of vulnerable populations. Listing pregnant women as vulnerable has limited scientific knowledge of therapeutic product safety, effectiveness, and dosing for pregnant and lactating women.
INvestigation of Co-occurring conditions across the Lifespan to Understand Down syndromE (INCLUDE). NIH has set aside $23 million to fund INCLUDE, bringing NIH funding for Down syndrome research to $60 million. INCLUDE will study conditions that are more common among people with Down syndrome, such as Alzheimer’s disease, and will also study why people with Down syndrome are resistant to some conditions, such as colon cancer. The program will assemble a large cohort of people with Down syndrome to participate in studies across the life span and will include people with Down syndrome in existing clinical trials.
New Institute and Center (IC) Directors
Bruce J. Tromberg, Ph.D., has been appointed the director of the National Institute on Biomedical Imaging and Bioengineering. His laboratory will be located within NICHD.
Helene M. Langevin, M.D., will take over as the director of the National Center for Complementary and Integrative Health.
A Board member asked which ICs are the leaders in funding pediatric research. Dr. Bianchi said that the National Cancer Institute expends the most dollars on pediatric research, but its budget is larger than other Institutes. NCI expends about 7 percent of its budget on pediatric research. The National Institute of Dental and Craniofacial Research and the National Institute of Mental Health contribute about one-quarter of their budgets to child health research. There are other ICs that allocate 10 percent to 20 percent of their budget to child health research.
Action Item: Dr. Bianchi will provide a breakdown of the ICs’ spending on pediatric research by percentage of total budget.
A Board member asked how much of the strategic plan should focus on the near versus the long term. Dr. Bianchi said that there are some issues that NICHD must deal with now, such as treating neonates for opioid addiction. Maternal mortality is also a pressing issue that must be addressed now. That said, much of what NICHD funds is geared toward the long term. For example, NICHD funds research on pregnancy, and a healthy pregnancy provides the child with the potential for long-term health.
Action Item: Board members could contribute to the strategic plan by suggesting an innovative idea of their own and sending it to Dr. Cernich. For example: If
NICHD offered to give them $10 million in funding, how would they spend it? Dr. Nitkin suggested thinking about what is missing from the members’ field of research and asking what collaborations could be formed to fill those needs.
A Board member suggested that the strategic plan should include an effort to implement scientific findings into clinical practice more quickly. Dr. Bianchi said that some members of the NICHD Strategic Planning Working Group have implementation science expertise and that there will be an implementation strategy within the plan. The strategic plan will incorporate NCMRR’s strategic plan and the strategic plans of other NICHD entities. The goal is to tie the plans together within the NICHD plan.
NCMRR Director’s Report
Alison Cernich, Ph.D., Director, NCMRR
Dr. Cernich provided updates on trans-NIH, NICHD, and NCMRR initiatives and policies.
Funding is available this fiscal year for the HEAL Initiative, with an emphasis on nonpharmacologic treatment of pain. FOAs will focus on biomarkers, novel targets for treatment, and omics.
The Acute to Chronic Pain Signatures (A2CPS) program will include longitudinal studies to better predict who will convert from acute to chronic pain. The Common Fund will provide $40 million for A2CPS.
The NIH Back Pain Consortium (BACPAC) Research Program is a translational research initiative to address the need for effective and personalized therapies for chronic low back pain. BACPAC, part of the HEAL Initiative, has active FOAs.
The Brain Research through Advancing Innovative Neurotechnologies® (BRAIN) Initiative has FOAs available. NCMRR has funded a number of BRAIN Initiative studies this year.
The All of Us Research Program, a longitudinal study aiming to enroll 1 million participants, has added the American Association on Health and Disability as a community partner. AAHD will help recruit people with disabilities to take part in the study. The All of Us Research Program has also established a disability task force to develop a disability survey that participants will complete. Dr. Cernich, who serves as the subject matter expert for the task force, is also helping ensure that children are included in the program and that proxy consents are available. The program will begin making data available in 2019.
NCMRR Initiatives and Highlights
NCMRR will begin working on its rehabilitation research plan. The initial draft should be available in May 2019. The final draft will be published in December 2020. NCMRR must submit the plan to Congress.
NCMRR is working in partnership with the Centers for Disease Control and Prevention (CDC) to incorporate disability questions into the Pregnancy Risk Assessment Monitoring System (PRAMS). PRAMS data is widely used to study birth outcomes, but there are gaps in the understanding of the specific reproductive health care needs of women with disabilities.
The Mayo Clinic was awarded the NIH–Department of Defense (DoD) Limb Loss and Preservation Registry contract. NABMRR member Kenton R. Kaufman, Ph.D., is the principal investigator.
NCMRR and the National Institute of Neurological Disorders and Stroke (NINDS) received funding to conduct a systematic review of the literature on whether physical activity can improve the health of wheelchair users. The aim is to increase the focus on health and wellness of wheelchair users. The Agency for Healthcare Research and Quality and the Oregon Health & Science University are conducting the review.
Dr. Cernich has been working with the World Health Organization (WHO) on Rehabilitation 2030, which is working on a global rehabilitation research agenda. In January, the Pan American Health Organization will meet on the NIH campus to identify the infrastructure that will be needed so that research can be done on rehabilitation services delivery in countries in the Americas. NCMRR is also working with the Global Partnership for Assistive Technology.
NIH rehabilitation research funding has been growing and is now approaching $600 million. NCMRR funding for FY 2019 is $72.7 million. About 69 percent goes toward research projects, 12 percent to training and career development, 10 percent to small business projects, and 8 percent to multicomponent projects.
NCMRR makes its greatest investments in the categories of pediatrics, prosthetics and robotics, and wheelchairs. When NCMRR investments are viewed by health condition, the greatest investment is in stroke. NCMRR also invests in SCI, TBI, cerebral palsy, and amputation.
As part of NIH’s enhanced clinical trial stewardship, NICHD has a new monitoring and oversight policy to ensure that its funded clinical trials achieve their objectives. Among other changes, NICHD will now work with investigators to establish milestone plans for each clinical trial. The new Human Subjects System will incorporate clinical trial and human subjects information into a single system. As of January 15, 2019, all grant applications for human subjects research must include individuals of all ages, from children to older adults, unless there is a scientific or ethical reason not to include them.
NIH has released an Assisted Referral Tool to help applicants choose the appropriate study section for their applications. Applicants can paste their aims into the tool, and the study sections that best match will appear.
The Medical Rehabilitation Research Resource Network has several pilot opportunities available.
A Board member commented that there is a clinical practice guideline that measures function that may be useful for the All of Us Research Program survey on disabilities.
A Board member asked who is collecting the data on functional outcomes for the international programs. Dr. Cernich said that WHO will collect that data using an electronic tool that is based on the International Classification of Functioning, Disability and Health.
A Board member asked whether the R03 award amounts would be increased. Dr. Cernich indicated that NCMRR increased the amounts only for the Early Career Researcher Award.
A Board member asked whether NIH was considering restoring the previous administrative cuts. Dr. Nitkin said that NIH is sensitive to the effects that administrative reductions could have on clinical trials. NIH wants to ensure that clinical trials are valid, but it must also spread the funding around as much as possible.
National Center for Advancing Translational Science (NCATS) Support of Research Infrastructure
Michael Kurilla, M.D., Ph.D., NCATS
The Clinical and Translational Science Awards (CTSA) Program was created to facilitate translational projects and to improve the efficiency and effectiveness of clinical trials. The CTSA program provides translational science training with a focus on multicenter trials. Its goals are to engage patients in the translational process, promote the inclusion of special and underserved populations in translational research across the lifespan, and advance the use of cutting-edge informatics.
There are 59 CTSA hubs, which receive UL1 cooperative agreement awards and KL2 career development awards. They may also receive TL1 institutional training awards. The KL2 awards provide postdoctoral and junior faculty time to do research. The TL1 awards provide training for postdoctoral and predoctoral fellows.
The CTSA Program activities include the following:
- The Trial Innovation Network (TIN) to improve the process of conducting clinical trials. The TIN has three components: the Trial Innovation Centers, the Recruitment Innovation Centers, and the CTSA hubs.
- The Streamlined, Multisite, Accelerated Resources for Trials (SMART), an Institutional Review Board (IRB) platform, reduces the time to initiation of multisite research and supports nationwide collaborations through a single IRB authorization agreement.
- The Accrual to Clinical Trials (ACT) Network improves the process for identifying participants for clinical trials via a nationwide network of sites that share electronic health record (EHR) data.
- The National Center for Data to Health (CD2H) enables data sharing by integrating data across institutions. This includes developing EHR systems, clinical data warehouses, and common data models.
- The CTSA Program Collaborative Innovation Award (CCIA) is meant to enhance collaboration across the CTSA Program. One example is the Development, Implementation, and Assessment of Novel Training in Domain-Based Competencies (DIAMOND) program, a collaborative learning space for clinical research professionals with an “ePortfolio” system to encourage workforce development and connection similar to LinkedIn.
Among the CTSA pilot projects are one to test the efficacy of a low-resistance exercise protocol with blood flow restriction for older adults awaiting total knee replacement surgery, a dose–response trial of aerobic exercise after a stroke, and the development of a “smart bandage” containing stretchable electronics that will be able to measure physiological signals and control robotic limbs.
A Board member asked how the CTSA Program hubs and NCATS decided to make collaboration a key part of their research program. Dr. Kurilla said that translational science is necessarily multidisciplinary and that more researchers are becoming involved in transdisciplinary interactions.
A Board member asked whether NIH would consider making individuals with a Ph.D. in physical therapy eligible for the predoctoral TL1 award, since such individuals are not given the time to get research training. Dr. Kurilla said that NCATS allows the institutions to decide how to distribute those awards.
Dr. Cernich asked what lessons were learned in the rollout of the SMART IRB program. Dr. Kurilla said that many of the institutions that rely on a central IRB do not know that their IRBs may need to be involved, for example, to resolve conflict-of-interest questions or oversee safety reviews for work done at their institution. NCATS has also learned that investigators may not understand what they give up when working with a central IRB. NCATS continues to work to resolve issues, including whether trials that are minimal risk need a central IRB.
A Board member asked about the smart bandage and whether partners are emerging to sponsor state-of-the-science meetings and similar activities. Dr. Kurilla said that NCATS is moving to a cloud platform that will provide more opportunities for institutions to work together. NCATS is also trying to create a system to match groups with similar interests.
Nomination for Chair-Elect
Nominations were sought for the next chair-elect and to serve as next chair after the May 2019 meeting. Three NABMRR were nominated: Kenneth Ottenbacher, Ph.D., OTR; Richard Ellenson; and Edelle Field-Fote, Ph.D., PT, FAPTA. Later in the afternoon a vote was taken and Dr. Field-Fote was elected.
Update on Rehabilitation Research Plan Analysis
Jennifer Jackson, Ph.D., NCMRR
Dr. Jackson presented the portfolio analysis and asked the Board to provide feedback on whether these are the data needed to track progress on the NIH Rehabilitation Research Plan. Each project was categorized in two tiers.
Tier 1 included:
- Rehabilitation across the lifespan
- Community and family
- Technology use and development
- Research design and methodology
- Translational science
- Research capacity and infrastructure
Tier 2 included:
- Disease-related basic
- Applied science (translational or clinical)
- Unable to categorize
In FY 2017, NIH funded 1,441 rehabilitation research grants totaling $502.5 million, compared with 1,394 grants totaling $466.3 in FY 2016. About 81 percent of the funding went to research projects including about 41 percent for research design and methodology, 19 percent for translational science, and 19 percent for technology use and development. The category with the least funding was the community and family.
Using tier 2 analysis, 52 percent of the funding went to applied clinical projects, 25 percent to disease-related projects, 11 percent to applied translational science projects, 9 percent to basic science projects, 2 percent to infrastructure, and 1 percent to projects that could not be categorized.
There were 55 patents associated with 25 of the 1,441 FY 2017 grants. Twenty-six percent of the publications resulting from the FY 2017 grants were cited by a clinical trial or a clinical guideline.
Action Item: Board members should send their suggestions, feedback, and questions regarding the portfolio analysis.
A Board member asked how these data can be used to measure the impact of the research plan and to develop the next plan. Dr. Cernich said that the data help to identify gaps. For example, the portfolio has a large number of clinical projects; should there be more basic science projects? The analysis also may suggest the need to foster particular collaborations, such as between basic scientists and clinicians. There has been an increase in new applications in some areas but not others. For example, the number of Phase III clinical trials has not changed, nor has the number of small clinical trials that convert to large trials, so new efforts may be necessary there.
A Board member asked why some categories receive less funding than others. Is that due to a lack of good submissions? Dr. Cernich said that it may be possible to look at how much funding is in response to targeted funding opportunities, but the analysis could be too time-consuming to undertake.
An attendee noted that there are fewer collaborations involving occupational therapy (OT) compared with physical therapy, and inquired whether that was because of funding. Dr. Cernich said that the disparity does not appear to be related to funding. OTs tend to collaborate within the allied health fields. There may be a need to spur more collaborations with other specialties. An attendee said that it would be important to identify the barriers to OTs doing research and forming collaborations. A Board member added that there is inherent “noise” in the data that NCMRR cannot remove; for example, an OT with a Ph.D. might identify as a neuroscientist.
A Board member suggested that NCMRR could save itself work by having the investigators code their own projects. Dr. Cernich said that investigators are already asked to provide a lot of information on their applications. NCMRR will cull the data that is needed from the investigators’ biosketches.
NINDS Workshops and Activities in Rehabilitation
SCI 2020: Launching a Decade of Disruption for Spinal Cord Injury Research
Lyn Jakeman, Ph.D., NINDS
NINDS will host SCI 2020 February 12–13, 2019, at NIH. SCI as a field has made significant advances over the past 10 years, including in treatments such as neuromodulation. This meeting will identify the progress and gaps in the field and identify the top SCI research priorities for the next 5 to 10 years. The conference will encourage greater collaboration among researchers and consumer groups.
Bridging the Translational Gap in Stroke Recovery and Rehabilitation Research
Scott Janis, Ph.D., NINDS
This workshop was held in November 2018 to identify gaps and opportunities in stroke recovery through translational research. The aim was to encourage high-impact trials that will improve practice.
Over the past several years, the number of randomized control trials in stroke rehabilitation has grown rapidly, but the trials focus more on the chronic phase. Nearly all are small, single-site trials. NINDS created StrokeNet with 29 regional centers and has partnered with 400 stroke hospitals to encourage larger, multisite trials that allow patients to move from a treatment trial to a recovery trial and then to a prevention trial.
At the meeting, attendees identified problems and opportunities, dividing opportunities into “accessible,” “achievable,” and “aspirational.” Accessible opportunities included having humans use wearable technologies in recovery trials and establishing consistency in data collection across study sites. Achievable tasks included defining compensation versus recovery in animal models and establishing a rehabilitation control group that could serve multiple recovery trials. Aspirational opportunities included developing prognosticatic biomarkers.
The attendees also discussed what opportunities they might be missing and how they can collaborate with community partners. Attendees indicated that they were interested in home-based telerehabilitation. Attendees said that the major reasons for not having more large-scale stroke recovery and rehabilitation trials were insufficient infrastructure and personnel and the difficulty of accessing patients. The most pressing needs were defined as follows:
- Better alignment of definitions and data
- Opportunities to develop better collaborations between basic preclinical/clinical recovery and rehabilitation
- Biomarkers to predict treatment responsiveness that can be measured across species
- Studies of the optimal intensity and duration of therapies in the subacute period
- Collaborations with community partners and other stakeholders
Dr. Nitkin said that one problem is that peer reviewers do not give implementation and translational science projects very high scores, making it difficult to get these studies funded.
Dr. Cernich said that one of the interesting aspects of the meeting was that basic scientists who work on animal models could compare notes with clinicians with a similar focus in humans.
Limb Loss and Limb Preservation Registry
Joseph Bonner, Ph.D., NCMRR
Dr. Bonner said that the Registry will collect data on the limb loss population that are needed to do research and to allocate resources. This data gap exists because the responsibility for patients with amputations is spread across the health care system.
The 5-year contract was awarded to Mayo Clinic in September 2018. The principal investigator, Dr. Kaufman, is recruiting a scientific advisory panel to decide who should be recruited and which data to collect. Dr. Cernich and Erik Wolf, Ph.D., of the DoD are forming the government oversight committee.
The contract is expected to open in mid-2020 with an initial focus on retrieving information from ERH. For logistical reasons, the Registry will not interface with the DoD registry, but the data will be comparable. The plan is for the Registry to eventually support itself.
Dr. Kaufman said that that the Registry will have data from hospitals, providers, and patients. The Registry will begin by collecting data that other organizations already have, including information from the American Joint Replacement Registry.
Liaison to NICHD Advisory Council
Kenneth Ottenbacher, Ph.D., OTR, University of Texas Medical Branch
Dr. Ottenbacher, the Board’s liaison to the National Advisory Child Health and Human Development (NACHHD) Council, provided an overview of the NACHHD’s structure and reported on its latest meeting. He noted the broad range of NICHD’s mandate, including child development, contraception, and population dynamics, among other areas.
Summaries of the public portions of the meeting are available on the NACHHD website. The Council also holds a closed session to conduct second-level reviews of grant applications that are recommended for funding but are outside the payline and other special actions. The NACHHD also reviews applications when the principal investigator has more than $1 million in direct funding.
An NABMRR member asked whether the NACHHD is a Federal Advisory Committee Act board. Dr. Cernich said that it is and that the open portion is videocast and archived on the NIH site.
An NABMRR member added that the NACHHD recently added a “voice of the participant” segment that is very compelling. The September meeting featured a young man who has been HIV positive since birth. Dr. Cernich said that the segment shows the human impact of NICHD research.
NIH requires concept review and clearance of all Requests for Applications. The NABMRR is one entity authorized to conduct this review and clearance.
Dr. Theresa Cruz made a brief presentation for a proposed request for basic and clinical research in pediatric rehabilitation. This proposal was stimulated by analyses of the NIH Research Plan for Rehabilitation, current NIH efforts in this area, other NIH Strategic Plans, and potential synergies with NIH’s federal partners. NICHD and NCMRR in particular are well positioned to lead this research effort. After brief discussion, the Board provided unanimous support for this concept.
Updates on the NIH BRAIN Initiative
Theresa Cruz, Ph.D., NCMRR
The NIH BRAIN Initiative began 5 years ago as a public–private partnership to develop better tools for understanding the brain at the cellular level. The work that NIH is doing within the multiagency initiative focuses on the circuits of the brain and how they control things such as movement and memory. This knowledge could one day be used to improve brain function and to explain how disordered circuits lead to neurological, mental, and substance use disorders.
The budget for the Initiative has grown from about $46 million in FY 2014 to $400 million today. The work has brought 130 new investigators to NIH, including experts in optics, engineering, circuit design, and material sciences who are now working with neuroscientists. Projects that may have particular relevance to rehabilitation research include the following:
- A second-generation rabies viral vector that is nontoxic to host neurons and enables long-term study of a group of neurons
- A new imaging technique, magnetic particle imaging, that could detect internal hemorrhaging following a brain injury
- A study to improve the use of deep brain stimulation so that the implanted prosthesis senses brain activity and adjusts stimulation as needed
As funding for the Initiative continues to increase, NIH has paused to assess its work so far. NIH formed the Advisory Committee to the Director BRAIN Working Group 2.0 and its Neuroethics Subgroup to do this evaluation. Between August and November, the Working Group held four public workshops in four cities to review the Initiative’s progress. The Neuroethics Subgroup is working to help create a neuroethics roadmap.
The International Brain Initiative Inventory held a workshop in July to develop a global inventory of brain initiatives and to discuss how the Inventory could be most useful to the scientific community. The National Science Foundation is the lead representative for the United States at international conferences.
The BRAIN Initiative at NIH has a new website containing all of the current funding opportunities. The Initiative is working to increase the diversity of the workforce by using the postdoctoral fellowship (F32) award mechanism as well as the more advanced Pathway to Independence (K99) career development mechanism. Within the context of these BRAIN initiatives, women are considered as an underrepresented group.
NIH is currently searching for a director of the NIH BRAIN Initiative. A search committee has recommended a short list of candidates.
Dr. Nitkin asked whether the BRAIN Initiative tends to consider the spinal cord and the peripheral nervous system. Dr. Cruz indicated that it tends to include the former but not the latter. The Stimulating Peripheral Activity to Relieve Conditions (SPARC) program covers the peripheral nervous system.
Dissemination and Implementation Research at NIH
CAPT Christine Hunter, Ph.D., ABPP, Office of Behavioral and Social Science Research
Dr. Hunter said that it generally takes 17 years for research advances to reach patients. This is a problem that implementation science is meant to solve.
Implementation science is the study of methods to promote the integration of research findings and evidence into health care policy and practice. Implementation science includes dissemination research (the study of the distribution of information and interventions to a public health or clinical practice audience) and implementation research (the study of the use of strategies to adopt and integrate evidence-based health interventions into clinical and community settings to improve patient outcomes).
One of the lessons of implementation science is that researchers must embrace the reality that fidelity—the adherence to a research treatment protocol—will not be perfect. The question is how to achieve the desired outcome in the absence of perfect fidelity.
Another lesson from implementation science is that recruitment and retention among high-risk participants is time-consuming and challenging. Researchers should understand the importance of forming partnerships with community organizations to achieve engagement of patients, policy makers, and other stakeholders. Researchers should ask stakeholders what research outcomes they want.
Dissemination and implementation happen naturally as health care providers adapt how they deliver treatments. Researchers should evaluate these natural experiments to see whether they have changed outcomes.
Researchers must embed implementation and dissemination questions earlier in the experimental design process, asking questions such as who will implement a treatment and which patients will want it.
Dr. Hunter also reviewed some current funding opportunities for dissemination and implementation studies. NIH has funded more than 200 implementation science grants since 2006.
A Board member asked how much of the failure to implement research findings into clinical practice is due to limits to reimbursement. Dr. Hunter said that some studies have shown that even when a service is reimbursable, it is not necessarily implemented.
A Board member asked about de-implementation of things that do not work. Dr. Hunter said that there is increased interest in de-implementation and that it is now mentioned in grants.
A Board member said that it is important to determine the attitudinal and architectural barriers to changing behavior and to find out how to remove them. Community partners can help remove the barriers that prevent patients from changing their behaviors.
A Board member said that implementation research must note all of the patient and provider barriers to following a certain treatment plan. Dr. Hunter said that provider barriers include the provider’s workflow, access to resources, and attitudes, and that it is important to understand how the provider perceives the barriers.
A Board member asked whether there are examples of best-practice success stories or if effectiveness measures could be built into FOAs? Dr. Hunter said that there are success stories, but that common measures of success do not exist yet, partly because the definition of success depends on the situation. Much more work would be required to define the range of success for patients, providers, and organizations.
A Board member asked whether there is evidence showing that research findings are being incorporated into practice and are making a difference in patients’ lives. Dr. Hunter said that she is not familiar with the rehabilitation field but that there is evidence of effectiveness in other fields. However, simply publishing findings is unlikely to bring a change in care.
Dr. Cernich said that one question is whether the system is ready to accept a change. The system may need more preparation, and personnel may need more training. NCMRR will continue to fund this area to give researchers and trainees the resources to translate research findings to care.
CDC: Health and Disability
JoAnn Thierry, Ph.D., CDC
Dr. Thierry described the work of the Disability and Health Branch (DHB) of the National Center on Birth Defects and Developmental Disabilities. She displayed an infographic that laid out the Center’s core principles and their goals.
Among other activities, the Branch funded 19 state disability and health programs to increase inclusion of people with disabilities in public health programs and reduce health disparities of people with disabilities.
The evidence on people with disabilities is limited, so the Branch focuses on filling the largest data gaps. One DHB project focused on evidence-based diabetes self-management strategies for people with IDDs, which aims to reach people with IDDs, their caregivers, and their providers. Problems with diabetes self-management are one of the major reasons that people with IDDs are hospitalized. The Branch is also developing a “community of practice” to document best practices for implementation of programs for people with disabilities.
CDC’s National Center on Health, Physical Activity and Disability (NCHPAD) is working to identify evidence-based models, programs, practices, and policies and adapt them for children and adults with mobility limitations. NCHPAD is publicizing best practices related to inclusive physical activities, nutrition, and obesity prevention strategies in community settings.
CDC partners with the Special Olympics on the Healthy Athletes program, which provides screening and referrals for follow-up care to athletes participating in Special Olympics events. In addition to benefitting people with IDDs, the program gives participating providers experience interacting with people with IDDs.
CDC also supports the Special Olympics Healthy Communities program. Athletes receive a scorecard to bring home and follow to benefit their health. Another program partners with communities to include people with disabilities in healthy activities. For example, one community built an accessible climbing wall.
The CDC Disability and Health Data System aims to provide demographic data on people with disabilities within each state. The dataset includes 30 health topics.
Dr. Cernich said that NCHPAD has spurred NIH supported research. For example, NCMRR has developed R01s to investigate the effects of physical activity on the health of people with disabilities. CDC surveillance work informs NCMRR’s work to improve the health of people with disabilities.
Scientific Presentation—Activity and Participation: The Keys to Healthy Outcomes
Elizabeth Skidmore, Ph.D., OTR/L, FAOTA, FACRM, University of Pittsburgh
Dr. Skidmore’s research focuses on activity and participation as a key to improving health. Activities are things that people with disabilities need or want to do and include personal care, functional mobility, household management, recreation, and working. Participation includes choice and control, meaningful engagement, access and opportunity, social connectedness, inclusion, and membership.
Activity limitations and participation restrictions affect health, as research on people with diabetes, back pain, stroke, and heart disease has shown. Activity limitations are more prevalent in underrepresented racial and ethnic populations and in rural and low-income communities. The disparities exist from when individuals are admitted to rehabilitation programs and through discharge and follow-up.
Historically, activity and participation were viewed as distal health outcomes, not potential mediators of health. Today, the association between activity/participation and health are becoming better understood. For example, one study followed individuals who had suffered a mild stroke, received in-hospital care, and gone home without the need for further rehabilitation. At the end of 6 months, 87 percent reported that they continued to have limitations in meaningful and essential daily activities. The study suggests that restoration of physical function by the provider does not necessarily restore activity and participation for the patient. This is an area that should receive further investigation.
Participation restrictions are associated with low levels of physical activity and higher levels of social isolation. A failure to intervene may contribute to much greater chronic disease, secondary conditions, and disability. This is a costly problem that cannot be ignored.
A better understanding of the role of activity and participation in individual health is needed. Dr. Skidmore said that one study found that participation was a robust predictor of emotional health 6 months after a stroke. The study also found that baseline emotional and physical well-being are predictors of participation.
Dr. Skidmore conducted a pilot study in which people who suffered strokes were given activity-focused training that was associated with greater improvements in activities of daily living. When these people were compared with those who received the usual inpatient rehabilitation, the study found that the training resulted in significantly better executive function and mood. In addition, the study found that people who received the activity training maintained a lower level of apathy. Dr. Skidmore emphasized that “activity” is more than physical activity; it also means engagement in meaningful activities and with other people. The next step is to find out whether there is a causal relationship between greater activity and improved executive function.
Social capital intervention studies (i.e., studies of interventions to improve social support and social participation) find that these interventions have a favorable impact on overall health, mental health, physical health, mortality, and use of health-related resources.
One barrier to this research is the need for better ways to measure activity and participation and for studies to better understand behavior and the biological consequences. New interventions to stimulate activities and participation are needed.
Ninety-three percent of frail adults show that they can engage in meaningful activities, leading to improvements in health and the ability to stay at home longer. Studies are now looking at home health activities that can reduce hospitalizations, emergency care, and health care costs.
There is a need to look beyond the one-intervention-to-one-outcome model to having complex interventions that address the array of health issues that an individual may have. The United Kingdom has guidance on developing complex interventions, and the Patient-Centered Outcomes Research Institute is also developing guidance.
Dr. Cernich said that even animal models show that the social aspect of rehabilitation is key to recovery. An animal that is isolated after a stroke will quickly die.
A Board member said that the National Institute on Disability, Independent Living, and Rehabilitation Research is interested in funding work on activity and participation. He encouraged people to apply for funding for these types of studies.
A Board member said that the NIH portfolio analysis showed that there were relatively few grants related to the community and family domain. However, community and family may be a secondary focus of many grants. This member suggested that the next portfolio analysis code community and family as a joint focus when appropriate. Dr. Skidmore said that it would be difficult to have a joint focus when coding the portfolio. There is value in basic science, early translational science, and work on community and family.
Dr. Nitkin asked how Dr. Skidmore measured participation. The patients likely knew what the investigators wanted to hear. How can that be measured in a valid way? Dr. Skidmore said that investigators have used different methods to measure participation, including looking at Global Positioning System data. Measuring participation is similar to operationalizing pain. It has taken time to operationalize pain, and it will take time to operationalize participation.
A Board member said that it is important to determine what benefit the participant believes that he or she has received from participating in a research study.
A Board member said that having complex interventions can be difficult, in part because investigators “protocolize” so many elements that clinicians are not allowed to change. This may not be realistic in rehabilitation, where there are so many treatment subtleties. Dr. Skidmore said that there are ways to design complex intervention trials to get good fidelity, but they take longer to develop, and the people who run them need to be trained. One approach that her group takes is to develop its fidelity guidelines with the clinicians.
Dr. Cernich said that the mental health field has developed rigorous assessments of counseling sessions. If counseling sessions can be studied in a rigorous way, rehabilitation should be able to do the same.
Dr. Lanig and Dr. Cernich thanked Board members for their input. Dr. Cernich asked that Board members email her with any questions about the meeting. The meeting was adjourned at 11:45 a.m.