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)
May 2-3, 2016
The 52nd meeting of the NABMRR was called to order at 9:08 a.m. Board members introduced themselves, followed by brief introductions from NIH staff and other visitors. Minutes from the previous meeting of December 6–7, 2015, were unanimously approved. Future Board meetings will be December 5–6, 2016; May 1–2, 2017; and December 4–5, 2017.
Ex-Officio Members Present:
Daofen Chen, NINDS
Joan McGowan, NIAMS
Chris Platt, NIDCD
Karen Segal, VA
Lois Tully, NINR
Ex-Officio Members Absent:
Lyndon Joseph, NIA
Cate Miller, NIDILR
Michael Swanson, CDC
NICHD Staff and Visitors:
Tim Brindle, VA
Leighton Chan, CC
Amy Colgerg, BIA
Theresa Cruz, NICHD
Robyn Ellerbe, APTA
Rosemarie Filart, PennSt
Craig Fisher, APA
Della Hann, NICHD
Lyn Jakeman, NINDS
Lisa Kaeser, NICHD
Naomi Kleitman, CHN Fdn
Jim Koenig, NINDS
Audrey Kusiak, VA
Susan Lin, MGH
Kathy Mann-Koepke, NICHD
Sue Marden, NICHD
Mary Ellen Michel, NICHD
Maria Nurminskaya, CSR
Grace Peng, NIBIB
Louis Quatrano, NICHD
John Ray, AOTA
Mary Rodgers, NIBIB
Christine Rogers, NICHD
Paul Smedberg, AAPRM
Peter Thomas, PPSV
Chuck Washabaugh, NIAMS
Erik Wolf, USAMRMC
Report of the Acting NICHD Director
Dr. Catherine Spong first presented news from the NIH. Eric Dishman has been selected as director of the Precision Medicine Initiative (PMI) Cohort Program. He most recently served as Vice President and Intel Fellow of Intel Corporation’s Health and Life Sciences Group. Matthew W. Gillman, M.D., was named Director of the Environmental Influences on Child Health Outcomes (ECHO) program and will begin this role in July 2016.
A Blue Ribbon Panel has been named to help guide Vice President Joseph Biden’s National Cancer Moonshot Initiative. The Panel will serve as a working group of the presidentially-appointed National Cancer Advisory Board and consider how to advance the themes that have been proposed for the Initiative. An online platform has been established to enable the research community and public to submit ideas about the Cancer Moonshot effort by July 1, 2016, at http://cancerresearchideas.cancer.gov . Board members were asked to circulate this information to their constituencies.
The Gabriella Miller Kids First (GMKF) program is taking applications. The program has the goal of developing a data resource for the pediatric research community of well-curated phenotype and sequence data that will help determine the biological basis of structural birth defects and childhood cancers. Applications to the GMKF fiscal year (FY) 2016 initiative, PAR-16-150 Discovery of the Genetic Basis of Childhood Cancers and of Structural Birth Defects: Gabriella Miller Kids First Pediatric Research Program (X01), are due June 17, 2016.
Dr. Spong next reported on news from the NICHD. The third annual Human Placenta Project (HPP) meeting was held in April 2016. The goal of the HPP is to understand human placental development, structure, and function in real time. This project could have implications for other organ systems and transplant medicine.
NICHD’s Office of the Director, which includes the Office of Health Equity (OHE), underwent a review of its function and structure. To better align OHE with the new structure of the NICHD, OHE grants were transferred to the Division of Extramural Research, which will broaden the NICHD focus. Work is ongoing to develop the new role of OHE. A working group has been established to identify areas of focus and a strategy for moving forward. Jean Flagg-Newton, Ph.D., is now acting director of the Office of Health Equity. Regina James, M.D., has moved to the National Institute on Minority Health and Health Disparities.
Dr. Spong gave a presentation on the Zika virus, noting that it will have implications for the rehabilitation community and for the NICHD for many years to come. Zika is a mosquito-borne flavivirus first discovered in 1947 in the Zika Forest in Uganda. The first human cases were detected in 1952. The structure of the Zika virus is similar to dengue except for a variation in its surface envelope proteins, which could shed light on how it enters cells and pave the way for new drug and vaccine targets.
The symptoms of Zika virus are short lived; 80 percent of people don’t even know they had it. The virus is transmitted by mosquitos, sexual contact, blood donation and transfusion, and organ transplant. It is associated with fetal anomalies, Guillain-Barré syndrome, and other neurological conditions. Other alarming concerns are that viral risk may not be limited to the first trimester, but could also have an effect as late as the third trimester, and that viremia in pregnancy has been found to be prolonged. Scientists at the Centers for Disease Control and Prevention (CDC) have concluded that Zika virus is a cause of microcephaly and other severe fetal brain defects. The World Health Organization declared the virus as a public health emergency. Since May 2015, there have been 500,000 to 1.5 million cases reported in Brazil alone.
There remain many research gaps related to pregnancy and pregnancy outcomes from Zika and to the neurologic sequelae it appears to cause. The NICHD is interested in the following areas:
- Risk of Zika in pregnancy
- Consequences of children born to Zika-infected mothers
- Evaluation of children born to Zika-infected mothers (symptomatic and asymptomatic)
- Rehabilitation and therapy for these children
- Rehabilitative efforts for neurologic sequelae of Zika
In response the HHS has provided guidance about travel and an unprecedented rapid funding mechanism to accept research applications on a continuous rolling basis for research on Zika. Other responses have included a CDC Zika virus registry to collect information about U.S. pregnant women with confirmed Zika virus infection and their infants and a developing Zika cohort study that would recruit 10,000 women to assess the strength of association with Zika infection during pregnancy. Dr. Spong urged Board members to receive NICHD updates via the NICHD e-newsletter and to subscribe to NICHDevelopments online.
Dr. Spong provided some legislative and budget updates for fiscal year 2016. Congress appropriated $32 billion for the NIH (a $2 billion or 6.6% increase over the FY 2015 enacted budget) and $1.34 billion for the NICHD (about 4% above last year’s total). Although the NICHD FY 2016 budget increased by $51.4 million, most of this money must go to required increases, such as the NCMRR budget ($2.9 million) and the grants payline ($11 million just to stay at last year’s level). Accordingly, only about $6 million to $7 million is available for new initiatives.
For FY 2017, the President’s proposed budget is a combination of mandatory and discretionary funds totaling $33.1 billion for the NIH. Priority areas include the Precision Medicine Initiative, the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative, and the Cancer Moonshot. The NICHD would receive $1.338 billion. It is unknown when a budget will be passed or how close it will be to the proposed budget.
The 21st Century Cures Act passed the U.S. House of Representatives by a vote of 344–77 in July 2015. The Innovations for Healthier Americans Act, which is the Senate version of the 21st Century Cures Act, was introduced in January 2016.
Dr. Spong invited discussion from the Board. A Board member asked whether it was understood why people do not recover fully from Guillain-Barré syndrome caused by Zika. Dr. Spong said it was not known at this time why the syndrome does not resolve fully, as is usually the case in non-Zika caused cases.
Report of the NCMRR Director
Dr. Alison Cernich first discussed the trans-NIH Rehabilitation Research Plan currently under development. Pursuant to congressional legislation, the NICHD director, in consultation with the NCMRR director, the Board, and the Medical Rehabilitation Coordinating Committee (MRCC), is charged with developing a comprehensive plan for conducting and supporting medical rehabilitation research. Dr. Cernich reported that the public comments were very helpful and resulted in two new sections added, Translational Science and Building Research Capacity and Infrastructure. The approach has changed to have a more living document with fewer time limits to allow for greater flexibility as priorities change. The plan will be presented to the NIH Institute and Center (IC) Directors in June, and the final plan is expected to go to Congress in September 2016. At this meeting, Board members will provide input on the objectives of the plan and how progress should be reported.
The NIH Rehabilitation Research Conference will be held May 25–26, 2016, on the NIH campus. Response has been excellent, with 425 people registered. NIH Director Dr. Francis Collins will open the conference. Speaker invitations are underway, and the proceedings will be produced for publication and dissemination. The presentations will focus on gaps and opportunities in the field.
Two NCMRR grantees have been recognized with the Presidential Early Career Award for Scientists and Engineers and will be honored at a White House ceremony. The grantees are Elizabeth Skidmore, Ph.D. (Closing Gap in Stroke Rehabilitation: Early Intervention for Cognitive Disability) and Ervin Sjedic, Ph.D. (The Aspirometer: A noninvasive tool to detect swallowing safety and efficiency). The researchers were 2 of only 15 NIH recipients of this prestigious award.
The trans-NIH MRCC continues to meet monthly and is beginning work on analyzing and monitoring the NIH Rehabilitation Research Plan. The committee is working to incorporate federal partners and potential collaborators into the meetings. Recently, both the National Science Foundation (NSF) and the U.S. Agency for International Development (USAID) came to discuss collaborative opportunities.
The Medical Rehabilitation Research Infrastructure Network is off to a good start. It will fund $5 million in grants to support infrastructure in the field. An administrative supplement grant was recently awarded to the University of Alabama at Birmingham to coordinate activities among the six centers and provide a unified portal to the research community.
Initiatives proposed for 2018, and for which the Board will consider approval at this meeting, include the NCMRR Early Career Research Award, Research to Address Sleep Disorders in the Context of Medical Rehabilitation, and Effects of Adaptive Fitness Programs for Individuals with Disabilities.
Dr. Cernich emphasized the NCMRR’s intensive outreach efforts to numerous federal agencies and nongovernmental organizations to introduce the NCMRR as a source of collaboration. Some of the meetings have included:
- An Food and Drug Administration (FDA) meeting regarding medical devices in rehabilitation
- Discussions with the National Institute on Disability and Rehabilitation Research (NIDILRR) to help align the Institute’s strategic plan with the NIH Rehabilitation Research Plan
- Meeting with the NSF regarding a collaborative workshop in August and a national robotics initiative
- A USAID meeting on disability mobility
- A discussion with the Access Board for writing standards for medical devices and standards for the Americans with Disabilities Act
- Interactions with the Department of Veterans Affairs (VA) regarding veterans’ health
Nongovernmental outreach is mainly directed to professional organizations to gain an understanding of where they fund training and research.
Dr. Cernich discussed co-funding, showing a comparison of NCMRR outgoing and incoming co-funding between FY 2011 and FY 2015. The NCMRR received a $2.9 million increase for FY 2016 and will be able to use these funds for quality grants. The comparison was notable for illustrating the increased collaboration between the NCMRR and other NIH ICs over the past 4 years. This will continue as the NCMRR actively looks to fund projects in other ICs. The Funding mechanisms are predominantly R01s and U01s and include cofunding to nine other ICs. Conversely, the NCMRR has received cofunding from five ICs including significant support for the research infrastructure networks.
NCMRR staffing remains at the same level (four health science administrators, one health specialist, one program support specialist, and one administrative assistant). Carol Sheredos and Mary Ellen Michel are both retiring, and the Center has begun an active search for their replacements. A new undergraduate volunteer, Dean Allen, has been recruited from the American Academy for the Advancement of Science program, and a search is ongoing to fill the Diversity in Research, Executive and Administrative Management (DREAM) Internship with a person with disabilities.
A Board member asked how the relationships differ between co-funding with NIH ICs versus working with outside agencies. Dr. Cernich said it is easier to work within the NIH because of similar review and funding procedures. But working with outside groups often requires negotiating through respective missions, administrative procedures, and funding timelines. The Center is looking for ways to leverage some projects funded by outside groups but also avoid duplication. For example, the Center is currently looking at ways to work with the VA and the Department of Defense to support the emerging area of regenerative rehabilitation. There are also collaborations with the NSF in the area of robotics. The NCMRR has been very active with strategic planning with the NIDILRR this year to advance and target the research in key areas of disability research. Another Board member asked about pediatric rehabilitation. Dr. Cernich said there are discussions with NSF about pediatric rehabilitation devices and the NIH will host a conference to promote opportunities in this area.
NICHD Division of Extramural Research
Dr. Della Hann, Associate Director for Extramural Research and Director of the NICHD Division of Extramural Research, first reviewed the NIH and NICHD budgets. In the NIH FY 2017 President’s budget request of $33.14 billion, the extramural program accounts for $26.9 billion, or 83%. The major part of the extramural program portfolio is research grants. This is also true for the NICHD; the majority of the $1.07 billion is spent on research.
The critical question is how to fund the best science at the NICHD. There are some concerning trends. In 2015, the NIH had about 55,000 grant applications. Although this number of applications has been steadily increasing over the past few years, the budget has not increased, thus the success rate has been falling. For FY2015 the NIH success rate was 18%, and the NICHD 2015 success rate was down to 11.5%.
NICHD application trends since 2007 show a significant increase in R01 and R21 awards, which indicates that the NICHD has a lot of science of interest to investigators. In addition, the requested direct cost of NICHD awards is $1.6 million compared to $1.2 million for those of the NIH as a whole. One reason for this disparity is that the NICHD funds significantly more human studies than the NIH average (65 % versus36%), and human studies are more expensive. These direct costs for NICHD grants continue to rise because including young or disabled people in research studies often requires additional research expertise and support.
NICHD paylines for R01 awards have decreased since 2000. The payline is now at the 9th percentile, which does not compare well with the other ICs. Moreover, compared with other ICs, the NICHD has a higher reliance on a definitive, rather than a discretionary payline, which means that many applications for good science in the discretionary zone are not being supported by the NICHD. A first step for improving the NICHD payline is to tighten the referral guidelines to better communicate NICHD priorities and provide criteria for discretionary funding.
Earlier this year, an NICHD task force undertook a global review of the Institute’s extramural training programs. The percentage of total expenditures for training and career development has remained at a steady 6% over the last 20 years, and this is comparable with that of other ICs of similar size. The task force found that the NICHD is low in proportion to other ICs with respect to support for individual F and K awards, similar in support of institutional T awards, and markedly higher in support of K12 awards ( institutional career development programs). The task force recommended shifting support from institutional K12 awards to individual K awards and increasing success rates for K99/R00 transition awards. Data have shown that the individual scholars have a higher level of productivity then those supported by K12 programs. In addition, NIH policy now requires raising K08 and K23 salaries to $100,000, which further strains the career development line. For FY 2017, the NICHD plans to keep the overall training budget at 5% to 6%, gradually reducing K12 awards while, increasing the number of individual K awards, and absorbing the mandated salary increases in K08 and K23 awards.
Dr. Cernich noted that the four NCMRR K12 programs would be re-competed one last time with some gradual reductions. She added that the NCMRR does have slightly more discretion than the rest of the NICHD because of its dedicated budget. A Board member asked about potential NICHD strategies for increasing funding and whether austerity measures are being considered so that the investigators will still be able to do what they need to do, but perhaps in a reduced way. Dr. Hann said that the NICHD has a cap of $500,000 annual direct costs for investigator-initiated grants and is now poised to become more strategic about setting funding priorities. The NICHD’s mission is very broad, and the science and support strategies differ among programs so that the Institute has to remain flexible.
A Board member asked whether the young investigator payline should be extended to the 20-30 percentile range to produce more active careers given the data showing scholars supported by individual awards are more successful. Dr. Hann said that although that strategy may seem appealing, there is only a finite amount of money, so dedicating more support for young investigators would further jeopardize middle-tier and senior investigators. She also pointed out that middle-tier investigators are especially fragile because they are up for tenure and need awards.
A Board member asked whether there is a rule of thumb for setting aside a specific percentage of funding for clinical trials. Dr. Hann said that is not done because the NIH has difficulty defining clinical trials, and specific trials may be buried among the aims of specific grants.
Discussion of NCMRR Priorities
Dr. Cernich asked for the Board’s input about the priorities that have been identified that are targeted to the Rehabilitation Research Plan and the NICHD Vision document. She explained that the NCMRR’s statutory mission is to concentrate on physical disability, while other NICHD branches focus on secondary conditions, such as cognitive impairment. Dr. Cernich asked the Board to comment on these priorities and indicate whether any priorities have been missed. She emphasized that these priorities are important because they will shape what kind of research the NCMRR can reach for with its discretionary funding.
Priority 1: Objective prognostic and surrogate markers most relevant to rehabilitation treatment response, monitoring functional progress, and tailoring interventions to the individual needs of the person with disabilities
In this discussion, a Board member noted that identifying biomarkers could subsume every type of study looking at functional outcome. It might be better to target only markers of rehabilitation treatment response, although that may or may not be the same thing as a functional outcome. Dr. Cernich said that the challenge is the inability to anticipate from where the markers are going to come. If the NCMRR is too restrictive, it deprioritizes some of the other objective markers that would prove useful. The Center did not want self-report measures because it has a lot of them, so the balance was specifically geared toward objective measures rather than subjective measures; that was the intent for the restriction in this priority.
Priority 2: Activity-mediated approaches to promote functional plasticity and sensory integration, including the optimization of regenerative techniques, electrical stimulation, therapeutic exercise, and pharmacological treatments
In the discussion, a Board member asked whether functional plasticity should be better defined to be less broad. Dr. Cernich said the challenge in restricting it to neural plasticity is that the NCMRR cannot rule out the interface between neural plasticity and plasticity of other systems. Another Board member asked about the end goal, which seems to be about improving functional performance. A third Board member suggested changing the words “sensory integration” to “sensory-motor plasticity.”
Priority 3: Rehabilitation research that addresses key lifespan transitional issues such as the steep physical and developmental transition from adolescence to adulthood and the transition into adulthood and independence
In this discussion, a Board member asked for an example of aging-related transitions and suggested stroke. Dr. Cernich said that they had identified four transitions but were told to pick one because choosing all four would not be prioritizing. The thought was that aging would be the primary focus for transition. The concentration would be on adolescence to adulthood, which would also include transition from pediatrics to adult health services. If the NCMRR covers adolescence, the Center could cover the development and maturation issues as people grow, such as changes needed in prostheses. Dr. Ralph Nitkin added that this is not a comprehensive statement. It is a way to pick the most topical, important areas that would justify reaching outside the normal funding line. Dr. Spong agreed that this effort is to identify some specific high-priority topics and that the priorities are not set in stone forever but represent opportunities for the NCMRR now. Dr. Cernich explained that if an application in aging came in at the 8th percentile (i.e., within the NICHD payline), the NCMRR would be able to fund it; however, if it came in at the 14th percentile (beyond the payline and not represented among the high-priority topics), the Center would not be able to support it. The NCMRR is looking at where research needs to be stimulated and especially priorities that are not represented in the portfolios of other ICs. The intent is for these priorities to be published to highlight current under-represented and unique research opportunities that the Center would like to promote.
Priority 4: Scalable strategies and technologies to promote, monitor, and sustain outcomes in real-world settings (e.g., home, community, workplace, and school), including the use of engineering and social science approaches to understand environmental supports and barriers, monitor individual participation, understand health service access, and support health and independence
In this discussion, a Board member suggested developing analytics to ensure that the data are analyzed in a way that is decipherable. Dr. Cernich agreed that the data generated must be translated into something useable. Another Board member suggested reducing “promote, monitor, and sustain outcomes” to only one thing that the NCMRR is most interested in and suggested that “monitoring outcomes” would be a good first step. Dr. Cernich asked whether the Board recommended narrowing simply in terms of limiting to monitoring or whether it would be to restrict the approaches described in the second sentence of the document.
A Board member asked whether the outcomes referred to in the priority referred to function or quality-of-life. Dr. Cernich said that they did not restrict the types of outcomes because various populations have different challenges, so there could be different pertinent outcomes depending on the context. The intent here was to be broad enough to cover the entire rehabilitation community. A Board member asked whether scalable strategies were really the major focus. Dr. Cernich indicated that the focus was scalability, particularly in terms of being able to use these measures more broadly. For example, sensor technology could be used not only for monitoring wheelchair use but also to help minimize the risk of injury. That technology could be scaled not only for spinal cord injury (SCI) but also for other injuries.
A Board member asked whether there is actually any advantage in narrowing these priorities. Dr. Nitkin responded there is only a limited amount of money, so the idea is to put forward the greatest short-term needs. Another Board member suggested that if that if the greatest short-term needs are the goal, then the priorities are still way too broad and applicants would always find a way to make their projects fit. Dr. Nitkin said that each round would consist of only a few applications in a narrow window, which should constrain the possibilities. Dr. Cernich noted that the first draft was much broader, and this draft best combines a “broad but narrow” approach. Dr. Nitkin observed that this is a reactive document, which requires transparency about why the NCMRR is giving a few proposals priority funding.
A Board member suggested that each priority begin with a short phrase, such as “real world situations” or “sensory motor function,” that more clearly defines the priority at the very beginning.
Priority 5: Identifying, preventing, and treating key secondary conditions (e.g., pain, musculoskeletal dysfunction, deconditioning, skin breakdown, cognitive impairment, and sleep disturbances) that are associated with impairments and physical disabilities
A Board member suggested including examples of behavioral issues as secondary conditions, such as agitation or depression.
Because of its broad expertise, the Advisory Board is sometimes called on to provide an additional level of review for NCMRR research initiatives. Background material for this process was distributed to Board members prior to the meeting. Concept review and clearance of all proposed Requests for Applications (RFAs) or Program Announcements (PAs) are required by the NIH. Dr. Nitkin presented three concepts for the Board’s review.
NCMRR Early Career Research Award
The purpose of this award would be to provide junior investigators (less than 7 years in post-terminal training) in rehabilitation fields access to an award mechanism that does not require preliminary data. Dr. Nitkin described the award as not quite a K award but rather the next step to gather pilot data to help get the first major research grant. With the anticipated reduction in K12 programs, there is a need to provide this kind of support for beginning independent investigators.
A Board member asked whether these awards would be a separate pool from the R21 grants. Dr. Nitkin responded that it would represent a different pool; some applicants may have some preliminary data, but this mechanism would allow them to gather sufficient data to submit a competitive research application. A Board member asked how this pool of applicants would overlap with current K awardees. Dr. Nitkin indicated that this would be targeted for investigators who are no longer eligible for traditional career development awards. A Board member suggested adding nursing to the list of rehabilitation fields eligible to apply. The Board accepted this concept with the suggested modification.
Research to Address Sleep Disorders in the Context of Medical Rehabilitation
The purpose of this initiative would be to encourage research on the occurrence of sleep disorders in patients with chronic disabling conditions and the impact on overall functions.
A Board member suggested adding developmental disorders and pediatric issues to the list of chronic disabling conditions. An attendee suggested changing “Sleep Disorders” in the title to “Sleep-Wake Disturbance” to bring a broader perspective. Some things are not disorders yet, and the hope is to catch them before they become disorders, so sleep dysfunction might be another term to use in the text. Dr. Nitkin said that using “Sleep-Wake” in the title might be too narrowing, but he agreed it should be highlighted in the text. Dr. Cernich noted that this initiative has been coordinated with the National Center for Sleep Research. A Board member asked about the link between sleep disorders and chronic pain. Dr. Cernich indicated that there are separate programs looking at sleep and pain across the NIH. The Board accepted this concept with the suggested modifications.
Effects of Adaptive Fitness Program for Individuals with Disabilities
The purpose of this initiative would be to study the effects of adaptive fitness programs on the health of individuals with disability in the context of their community.
A Board member suggested including risk and protective factors that might contribute to barriers to effective exercise. Dr. Cernich felt this was beyond the scope of the initiative, which was developed in response to comments about the dearth of research on the accessibility of fitness programs in the community. There is another NIH program announcement (PA) that might be looking at risk and protective factors in mid-life adults, so there might be some opportunity to partner.
Dr. Nitkin added that the focus here is adapting, that is, how does one shift gears to support people with disabilities The NCMRR realizes that it is difficult for people with disabilities to get into community programs, and that some of the barriers are environmental. Although a primary focus of the effort is identifying barriers and solutions, there might be opportunities to collaborate with other studies about underlying mechanisms of fitness and health. A Board member suggested that the NCMRR should specify that this initiative applies to both adults and children. The Board accepted this concept as presented.
Cerebral Palsy (CP) Workshop on Basic and Translational Research
Dr. Jim Koenig, from the National Institute of Neurologic Diseases and Stroke (NINDS), explained that CP represents a heterogeneous group of neurodevelopmental disorders that affect movement, muscle tone, posture, and sometime cognition, and that it is one of the more common childhood problems. Current therapies focus on improving movement and reducing secondary conditions. One significant challenge of CP is that it is usually not diagnosed until at least age 2, which limits interventions that might reverse CP pathophysiology at a time when the developmental window is still most open. The structures underlying CP remain unclear but probably involve brainstem and spinal cord motor pathways. One encouraging development is recent evidence that supports an involvement of genetic factors.
In 2014, the NIH hosted a workshop on research gaps in the treatment of CP which highlighted:
- The knowledge/translation gap between basic and clinical research in CP
- A communication and collaboration gap among researchers, patients, and clinicians
This led the NIH to convene a second workshop that focused on gaps and achievements in basic and translational CP research. That workshop was held on March 24–25, 2016, and is available as a videocast on the NIH website.
The workshop identified four major themes that could have high impact on CP research:
- The developing brain and responses to injury: information from animal models
- Neuroprotection, including pharmacological strategies and stem cell–based therapies
- Neuroimaging strategies, for earlier and more accurate diagnosis
- Promoting neuroplasticity and adaption for improving function
The workshop suggested a more global approach to studying brain changes because early neurodevelopmental insults may result in subtle alterations in the development of the entire brain. Effects downstream on the spinal cord might and even the peripheral nervous system may also be important. Applying more advanced imaging techniques will supply information about what is actually happening in the brains of people with CP, which could open a door to precision medicine and more targeted approaches to optimize therapy and functional outcomes.
The workshop participants identified a number of knowledge gaps. Among the main ones were:
- Development of relevant CP circuitry from brain to spinal cord to muscle, with particular focus on the cerebellum and placental interactions.
- Neurorestorative effects of combined therapies. There may be no single intervention, so research should focus on finding combinatorial approaches.
- Prognostic imaging biomarkers, including imaging of the peripheral nervous system, muscle, and placenta (The placenta is included because many of the insults that are associated with CP occur in utero)
- Promotion of improved research-clinical-consumer interactions for rapid development of therapies and the amassing of more information about what treatments are effective for which CP populations.
The workshop participants recommended a number of ways that the NIH could advance CP research. These included:
- Using animal models to study CP pathophysiology
- Integrating imaging into all facets of CP research, encouraging the development of CP biomarkers and prognostic information
- Accelerating bench-to-bedside strategies, expanding public-private partnerships
- Enhancing training of the next generation of CP investigators and clinicians
One potential future activity would be for the NIH to conduct a workshop on devices and intervention, an area that was underserved by the previous two workshops. Dr. Koenig asked the Board to think about other topics for workshops and whether there are additional initiatives in CP research for the NIH to consider.
A Board member asked about the most promising CP interventions on the horizon. Dr. Koenig discussed innovative devices, some of which are simple and not expensive. The problem with cell-based pharmacological therapies is that they take so long to develop while the path is much quicker with devices that can be employed immediately. A Board member, noting that early diagnosis is important, asked whether more aggressive therapies would be available if the diagnosis were made earlier. Dr. Koenig said one of the best interventions is active parenting once the child is identified as at risk in the early developmental stages. Actively working with a child on walking gives the child a chance to respond to manipulation in a more meaningful way and at a time when the nervous system might be most receptive. Unfortunately, active participation requires early diagnosis and acknowledgment of the problems, and often parents may be reluctant to acknowledge it, or they believe their child will outgrow it. A Board member noted that it would take a lot of people doing a lot of research to get a handle on this because of the heterogeneity of the disorder, which creates challenges in the context of experimental models.
A Board member asked whether there was a mechanism to ensure peer-review panels appreciate the clinical aspects and not take too rigid a view that is not appropriate for this condition. Dr. Nitkin responded that it would be wonderful to have a special study section, but applicants must also take the responsibility to educate their own peer reviewers. The field must submit more applications, which will signal to the NIH that this is a burgeoning field that justifies more reviewers with the requisite expertise to conduct an appropriate review.
A Board member asked about potential damage to the brain from cell therapies. Dr. Koenig said that the therapies seem to be safe, but the exact mechanism is not clear. Until research directs them to the most likely cell therapy, scientists won’t know the answer to that question. A Board member suggested that there are some interesting delivery systems using neurotherapies to more targeted areas. Dr. Koenig said this was an important point, but early neurotherapies will not be useful without early diagnosis. To intervene early and change the programming, doctors first need to have the diagnosis. The more immediate impact maybe in the device strategy, but ultimately the CP field needs both strategies working together.
Next Steps on the NIH Rehabilitation Research Plan
Dr. Cernich gave an overview of the research plan, saying that the expectation is to have the edited plan up on the NCMRR website just prior to the May conference at the NIH, with adequate opportunity for email comments. The plan will have an Executive Summary and an Introduction that discusses the need for rehabilitation research, incorporates cost data, gives background on the NIH’s investment in rehabilitation research highlighting collaborative areas, and describes current medical rehabilitation research activities at the NIH. The next section, titled Opportunities, Needs, and Priorities, will lay out the objectives of the plan. Dr. Cernich asked the Board to keep in mind that the document is meant to cover the breadth and depth of the NIH. The NCMRR is not endorsing one area, and the plan must be broad enough to engender support of the other ICs to ensure collaboration and promotion of the research plan.
Dr. Cernich next outlined some of the changes that had been made to the document. Timeframes were removed, short- and long-term objectives were collapsed, and sub-bullets were removed to give the document more flexibility to adapt over time. Some of the objectives were revised, and two sections were added in response to public comment: Translational Science and Building Research and Infrastructure. Because comments indicated that the Technology Use and Development section was too detailed and restrictive, the section was revised to be more general. The final section, Coordination with Other Federal Agencies, will be written to reflect how active the NCMRR has been in this area.
Dr. Cernich stated that, in the afternoon, the Board and visitors would be engaged in breakout sessions to further evaluate the document, specifically considering five areas:
- Suggestions for the final revision of the proposed objectives, particularly whether anything has been missed
- Metrics to be used for reporting on an annual basis to the Board (i.e., What does the community want to see that reflects progress.
- An RFA cannot be done for every objective because of budget constraints, but what metrics would be meaningful that does not involve an RFA?)
- Initiatives or programs that could be leveraged to accelerate the proposed objectives; the Board was asked to be very creative in this area
- Potential partners to engage to achieve the objectives
- New research or initiatives that might drive interest in research that supports the objectives
Dr. Cernich said that comments from the Board and from the workshop in May would be incorporated into the final document presented to the IC Directors.
Programmatic Tools and Strategies
Dr. Nitkin provided the Board with some pragmatic information to help guide their discussion of how to implement the Rehabilitation Research Plan. This included identifying the specific goals and opportunities, differentiating research from policy or implementation, taking appropriate resources into account, incentivizing professional interactions, considering what novel approaches and new tools are needed, defining short-term and long term goals, balancing investment and costs against potential impact, and defining what success would look like. A range of tools could be employed, including webinars, courses, conferences, workshops, white papers, PAs, RFAs, training and career development, infrastructure and databases, and wide-ranging collaborations. Traditionally, people consider PAs and RFAs, but Dr. Nitkin cautioned that not everything can be solved with an RFA, so all available tools should be kept in mind.
Dr. Nitkin gave an overview of PAs and RFAs. PAs, which highlight a specific research need or opportunity, generally extend over 3 years. This timeframe provides an opportunity to revise and resubmit applications that were initially unsuccessful. No actual funds are set aside because PAs compete for regular IC paylines and resources. RFAs highlight a more specific research need and funds are set aside for the most meritorious applications. The review panel is specifically tailored to the goals of the RFA. However, the funding set-aside for the RFA detracts from the overall research payline, which is already quite depressed; the RFA also offers a short turnaround time, which generally favors more seasoned investigators already situated in those research domains. Moreover, unsuccessful applicants to the RFA cannot revise and reapply. Finally, there is no supportive research infrastructure to support and maintain new research initiated by successful applicants to the PA or RFA.
Dr. Nitkin provided some possible positive outcomes of research initiatives. Conferences, white papers, or funding announcements might create a “buzz” in the research community. Critical mass, infrastructure, and resources might be strengthened by development of research tools, novel collaborations, and greater access to patient populations. Funding announcements might stimulate unique collaborations among researchers.
Dr. Cernich informed the Board that the NCMRR could develop criteria for tracking research grants and productivity especially in response to particular research domains. She asked the Board for input on whether there are different or additional metrics that would be more meaningful to their communities. She urged each breakout group to consider the Research Rehabilitation Plan in detail and provide suggestions on how to move forward.
Summary and Review of Breakout Sessions from Day 1
Board members reviewed the suggestions from the breakout sessions as they related to the following specific sections of the Research Rehabilitation Plan.
Rehabilitation across the Lifespan
Board members liked the distinction between efficacy and effectiveness and appreciated that the introductory paragraph highlighted important issues in terms of ethnic and gender identify. This section seemed the ideal place to further develop the idea that cultural diversity may affect participation. There is a lot in the opening paragraph about lifespan and diversity. The bullets cover lifespan; however, there is not much about diversity. Specific suggestions included:
- Item 1 – Consider including mechanistic language.
- Item 2 – Put lifestyle and health behaviors in the major bullet and use the physical activity point in the description.
- Item 3 – The sequelae looked redundant because the symptoms and sequelae were not up front. These are not secondary sequelae, so the disability is secondary to a condition, disease, or disorder. The sequelae will vary based on the primary concern. Preventing disability should be the larger point, with a call-out to prevention of secondary conditions.
- Item 5 – Delete “new” from the care delivery models for lifespan transitions.
- Item 6 – Consider revising “healthcare delivery” to “care delivery” models.
Opportunities for Collaboration and Partnerships
Board members suggested reaching out to other groups that are building infrastructure including the North American Research Committee on Multiple Sclerosis, iConquerMS, the Interactive Autism Network, and other groups working on fibromyalgia and muscle disease, such as Cure SMA and CureDuchenne. It would also be beneficial to contact caregiving organizations, such as the Coalition on Caregiving, the Family Caregiver Alliance, the National Caregiving Alliance, and larger groups that advocate community living. Dr. Cernich noted that some of these networks have patient-based registries. It would be helpful to partner with them to determine the needs of the community to make the outreach more patient-centered.
Family and Community
Board members suggested retitling this section to “Community and Family.” The introduction should reflect the existence of individuals who are living alone without care support. The language should state that individuals live within the context of a community that may include, for example, a family or a social group. Family and formal and informal caregivers should be highlighted. Suggestions related to the bullet points included:
- Item 2 – Include stressors, challenges, and benefits and language related to positive aspects and resilience.
- Item 3 – Look at maximizing benefits and accomplishments, resilience, and role fulfillment here.
Add the following shorter-term objectives:
- Educate and engage family and community through joint goal development.
- Develop technology to assist family and community in delivery of care.
- Develop comprehensive analyses for economics of rehabilitation, including consideration of secondary health conditions. Determine cost-effectiveness and potential for reimbursement.
- Item 1 – Shorten this item to the impact of sociodemographic influences on the outcomes of rehabilitation interventions. Change “success” to “outcomes of,” reference “community resources” instead of “geography,” and describe the caveat list as “factors including but not limited to.”
- Item 2 – Include the person with disability with the caregiver and the care provider in partnership. Include examples in call-out and shorten the point.
The Board discussed the economics of rehabilitation and the potential for reimbursement, perhaps with the Patient-Centered Outcomes Research Institute or the Centers for Medicare and Medicaid Services (CMS) as a partner. A Board member suggested that data on objective measures of cost effectiveness might change policy. Dr. Cernich cautioned that this is a research plan, not a policy plan. The focus must be on research, with the hope that it might influence policy at some point. But the language of the document must be about what the science is going to achieve. Dr. Nitkin noted that there are economists on NIH staff. He observed that the document should not take too short a horizon because rehabilitation might not necessarily pay off in the first year.
Technology Use and Development
Board members found the introduction comprehensive but in need of editing. A comment should be added related to security and privacy for the end user, either in this section or in the Research and Design Methodology section. Suggestions related to the bullets included:
- Item 1b and 2b – Add “assessment” to the series.
- Item 1c – Spell out “assistive technologies” at first use.
- Item 3 – Consider whether the term “device fabrication” is too specific.
Add the following shorter-term objectives:
- Determine cost-effectiveness of new technologies (build into applications?).
- Develop standards for readiness levels for new technology (similar to Department of Defense technology readiness levels).
- Item 1 – Delete “research” at the end of the sentence.
- Item 2 – Consider “needs and preferences” of users and funding initiatives to identify needs of individuals with disabilities.
Board members asked whether a more holistic approach would be beneficial for talking about how individuals respond to technology and actively participate with high-tech devices. This is particularly important for new, expensive technologies that are out of reach for most people. This approach also raises issues about health disparities. Board members suggested incorporating readiness levels into applications to indicate where the technology is in the translational pipeline and providing metrics for how well the investment in new technology is moving it into clinical use. Dr. Cernich pointed out that the technology readiness level and the state of technological development are in parallel, but much of this is retrospective. If a company decides it is no longer going to develop that technology, it is out of the Board’s and NIH’s control.
The Board debated whether such metrics even belong in a research plan, with some suggesting that research is an investment, so it is important to show where things are in the pipeline, while others questioned the metrics’ inclusion in a research priority plan. Dr. Cernich said that currently the plan does not contain metrics on how to measure success. It is a challenge because the same type of metric may not be appropriate across the entire portfolio. The model that is picked must fit the design of the portfolio. A Board member advised caution in deciding how to determine success because that will determine accountability. Dr. Cernich said that baseline analysis of the portfolio in terms of where it is and what should be targeted is being conducted. The Board will need to determine whether there have been RFAs in a particular area and whether they have been successful. These are prospective goals to grow over the next 5 years, but there certainly needs to be a back-end document that indicates what the analysis is going to be based on.
The Board listed a number of potential metrics including numbers of grants submitted, grants successful, publications and impact score, citations, workshops, media mentions of rehabilitation research, treatments implemented as routine use, social media mentions of rehabilitation research, and awards and promotions received as the impact of NIH funding.
Research Design and Methodology
Board members made the following suggestions:
- Add “Transdisciplinary research must be encouraged.”
- Item 1 – Replace “provide” with “expand.” Correct typo “interventions and approaches.”
- Item 2 – Change common metrics to reflect the need for identification of standards (e.g., common data elements, NIH Toolbox).
- Item 3 – Consider adding privacy and security.
Add the following objectives to Item 3:
- Develop new registries and experiential databases of what has worked and what has not.
- Develop quality-assurance and quality-control guidelines for databases.
Add the following objective:
- Develop research designs that incorporate patient goals and consumer feedback.
- Item 1 – Delete “better” from “better characterization.”
- Item 3 – Move this item to the Building Research Capacity and Infrastructure section.
Dr. Nitkin noted that there has been discussion about databases and expanding registries and looking at what has or has not worked. Quality assurance guidelines will be developed, especially for functional measures. He agreed that patient-centered goals and consumer feedback are important in research design.
The Board made the following suggestions:
- Item 3a – After “adaptation,” delete “and response to illness or injury or to rehabilitation intervention” and add “with respect to genetics/genomics/epigenetics.”
Add the following to Item 3:
- Examine pharmacogenetic/pharmacogenomic/epigenetic interactions with respect to function.
- Examine epigenetic responses to illness or injury.
- Examine epigenetic responses to rehabilitation interventions.
A Board member asked whether studies that can actually do good epigenetics should be prioritized to ensure that epigenetic studies would have sufficient power and be more targetable. Another Board member suggested it would not be good to restrict studies this way but rather let the investigators decide.
Building Research Capacity and Infrastructure
The Board made the following suggestions:
- Add the words “basic scientists” before the words “physician scientists.”
Add as the last sentence:
- “Consideration of cost-effectiveness as well as potential for reimbursement needs to be considered.”
- Item 5 – Add the words “more effective” before the word “mechanism.”
Add the following items:
- Educate prospective reviewers regarding realistic expectations for rehabilitation trials based on a variety of methodological designs.
- Develop team science approaches to rehabilitation research.
- Develop methods and strategies to inform the research community about the results of research capacity and infrastructure.
Board members emphasized the need to disseminate information on research findings to the professionals who are training the care providers because that is how to educate the clinicians who are seeing the patients. Other suggestions included looking at how people are collaborating across sciences, determining who the new investigators entering the field are, and bringing people with disabilities into the research field.
Use of Common Data Elements (CDEs) for Rehabilitation. Particularly SCI
Dr. Lyn Jakeman, NINDS, explained that since the NIH launched the Big Data to Knowledge initiative in 2012, there has been a shift from sharing just genomic data to sharing all data. Federal agency awardees are required to share data; the NIH data sharing plan released in 2015 included encouraging “the use of established repositories and community based standards.” Developing CDEs is one of the first steps in this process.
Data sharing requires a uniform language to ensure that data are findable, accessible, interoperable, and reusable. A data element is information that describes a piece of data to be collected in a study, whereas a CDE is a data element that is common to multiple data sets across different studies. A CDE must be dynamic, because standards evolve over time, but still stable enough to allow for interoperability. While there are a number of NIH CDE initiatives, which can be found on the NIH website, this discussion would focus on the NINDS initiative.
The goal of the NINDS CDE project is to identify common definitions and standard report forms for clinical research of neurological diseases and disorders. The NINDS identified a number of areas for CDE compilation such as epilepsy, multiple sclerosis, stroke, and SCI. The process for developing CDEs is to first invite the input of disease-specific experts, then form domain-specific working groups to identify and prepare data element definitions and case report forms (CRFs), make classifications, and provide guidelines and recommendations. There is a public review prior to the publishing of the results. All NINDS-funded researchers are encouraged to ensure their clinical data are compatible with the CDEs.
An attendee asked whether there was a risk of the experts lapsing into their own jargon. Dr. Jakeman said that is a risk, but the CDEs go through more than one review, which counteracts this tendency. A Board member asked who the stakeholders are. Dr. Jakeman said that there are multiple stakeholders including clinicians, consumers, academia, and industry, but the actual decision-making tends to be driven by the clinical side because they see the patients. A Board member asked how protected health information is dealt with and how patients are tracked. Dr. Jakeman said that creating CDEs is a first step, and they are not looking at what happens to the data. This is a worldwide initiative, and every country has different rules and solutions. A globally unique identifier (GUID) is proposed, but at this point all this is beyond the CDE process.
A Board member asked how the data set is matched with the CMS data set. Dr. Jakeman said one goal is to harmonize with some electronic health records, but not CMS. The GUID will help because each person is uniquely identified, so there won’t be multiple identifications of the same individual. Ethicists are working on these questions. An attendee noted a concern about using the data once it is in a repository, suggesting the need to start using the data from the repository that has been created to see how it works and provide information about how to make it better.
Dr. Jakeman resumed her presentation by describing the NINDS SCI CDE process. Ninety worldwide expert volunteers were recruited. There were issues about the international mission regarding clinical use versus research use. The goal was to incorporate the full continuum of the World Health Organization International Classification of Functioning and include data elements associated with personal factors and environmental factors. Nine working groups were identified: demographics, neurological assessments and outcomes, spinal cord imaging, participation and quality of life, pain, care, functional assessments and outcomes, electrodiagnostics, and psychological outcomes. The recommendations were incorporated into CRFs and data definitions and published. SCI CDE efforts are continuing. A new expert working group developed CDEs for pediatric SCI, which are published on the website and will be published as a paper. An oversight committee of both veteran and new members reviewed all SCI CDEs for omissions and new evidence, and there is continued incorporation of new and revised International Spinal Cord Society international data sets. These international data sets have a common name, and this is the beginning of making big data sharable.
Harmonization efforts for SCI CDEs are ongoing with a number of organizations. NIDILRR has been a challenge because they need to use data from 38 years ago, so the NINDS is looking at how to cross talk with old data. Perhaps there will need to be two different data element descriptions, one of which matches the NINDS data element. For an example of how to use the CDEs, Dr. Jakeman directed attendees to the NINDS CDEs site at https://commondataelements.ninds.nih.gov. Some disease-specific rehabilitation CDEs have been developed for rehabilitation research, but they have been described by research and registry experts as “woefully inadequate.”
An attendee asked how one could suddenly shift to a key variable. Dr. Jakeman said they could create elements to match a key variable. CDEs are like a box of Lego®s—we provide the blocks and you use them as you see fit. A Board member asked how the database could be used to influence the role of practitioners because of the differences between clinical and research applications. Dr. Jakeman said there is not currently a shared repository for SCI. The challenge is the quality of the use of CDEs because the NINDS is not micromanaging its CFRs as well as they should. An attendee pointed out the difficulty that has been seen in the international community because of the difference between clinical and research collections. Whole areas of data have been identified that were not being collected, and now the data sets must be validated.
Dr. Cernich noted that this was a case study of SCI specifically, but some things are common across conditions and outcomes. The question for rehabilitation is: where do we need to position ourselves to encourage the use of CDEs shared across rehabilitation approaches and be able to validate those data so we have reference points? What is the clinically meaningful difference? Dr. Jakeman said that the CDE effort has been disease specific, and rehabilitation has been left out of the process, even though rehabilitation crosses all disorders. Rehabilitation input and participation are needed in developing cross-disease CDEs that the rehabilitation field deems important. Dr. Cernich noted the challenge at the NIH of harmonizing across proprietary or private data systems. In an investigator-driven system, people are determining their own CDEs, and mandating the use of common ones could seem restrictive. The balance is finding what is common, what is valid, what to do to establish efficacy, and how to use it across disease populations. The tension is the potential for restriction versus the potential for cross-disease comparison. Dr. Jakeman said the concept is not intended to be limiting, but rather just to provide a common language.
NICHD Support of Training Activities
Dr. Nitkin listed the types of NIH grant mechanisms across the training and career development pipeline:
- Graduate students: Ph.D., M.D., and M.D./Ph.D. programs (T32 and F31)
- Postdoctoral fellows (T32 and F32)
- Transition from postdoctoral fellow to faculty (K99/R00)
- Beginning faculty (K01, K08, K23, K25, K12)
The recent NICHD Training Report noted that the NIH training budget has declined by about 30% over the past 10 years. Moreover, there was a recent decision to increase salaries on individual K08 and K23 awards from $75,000 to $100,000. The NICHD is currently invested in institutional T32 and K12 awards far more than any other IC, despite data showing that individual F and K award scholars have better outcomes than scholars trained on T32 or K12 programs. The NICHD’s heavy commitment to institutional T32 and K12 programs in mainstream disciplines limits opportunities to cultivate scientists in novel and emerging areas.
The NCMRR supports four K12 awards: two for physical or occupational therapists, one for physiatrists, and one for rehabilitation bioengineers. The awards provide 2- to 3-years’ salary support along with centralized mentoring, which adds an extra level of support and creates a cohort effect. In contrast, individual K awards provide greater institutional diversity and flexibility, are not limited to specific clinical specialties, and offer 5 years of local mentored salary support. In general, the individual K awards provide more candidate accountability and more diversity.
Dr. Nitkin emphasized the need to be realistic about workforce constraints and the availability of jobs for career-development scholars. It is time to evaluate whether there is a continued need for specialized training and career development programs for rehabilitation in general and in special clinical domains in particular, as well as a continued need for networks, or whether there are other strategies and models to consider. Factors to help guide this consideration include the quality of the applicant pool, the scholars’ commitment to academic and research careers, the quality and impact of K12 publications, the role of the individual K awards in relation to getting grants from other federal agencies and foundations, and whether there is value added to K12 programs.
Dr. Nitkin noted that while the productivity of scholars on individual K awards has been impressive, the scholars supported by rehabilitation K12 programs have also done well. Based on the recent Training Report, the NICHD has made a decision to transition away from institutional K12 awards, but the Institute will continue to offer opportunities in training and career development by providing detailed content on is website and through programs such as: the Training in Grantsmanship for Rehabilitation Research (TIGRR) program; the Early Career Development group at the American Congress of Rehabilitative Medicine ACRM); and NCMRR participation in workshops, national meetings, and regional conferences.
Dr. Nitkin asked the Board whether the NCMRR should consider additional opportunities to bring young scholars together and help maintain the cohort effect and mentoring that K12 awards provide.
A Board member asked whether it would make more sense to try and maintain the K12 program success because the data on K12 awards demonstrate good outcomes. Dr. Nitkin said that the NCMRR must be a citizen of the larger NICHD, which is evolving and adapting. The NCMRR agreed to re-compete the four K12 awards during the upcoming year for one final 5-year cycle because there was not sufficient time to engage the professional community and plan for this change in training support.
The Center needs to think about the best use of its training and career development resources and how they coordinate with rehabilitation training programs in other ICs. The NCMRR must think about whether it should continue to maintain specialized programs or build capacity more broadly. As part of its investment decision, the Center must also consider whether it needs additional workshops or other forms of support. The Center’s suggestion is to shift its investment because it has seen with 20-year data that the return on investment and the retention are better in the individual K awards than in the K12s.
A Board member noted that it is possible to substitute something for the loss of the cohort effect, but ultimately people must find their own peers. A Board member asked whether the rehabilitation-based K12s have done worse or better than other K12s. Dr. Cernich said they are comparable. A Board member was concerned about the loss of the important national mentoring that K12s provide. Dr. Nitkin observed that in the early days of the NCMRR, the K12 award was a foundational need. Now there are other opportunities, such as the TIGRR workshop and ACRM Early Career programs to provide mentoring. An attendee asked how well rehabilitation K01s do competing against other K01s. If not well, perhaps the NCMRR K12 has a special niche. Dr. Cernich said that the current NCMRR K12 awards are directed only to certain clinical professions and not so much to research as part of training. The NCMRR does not want to abdicate but must determine the point at which professional associations take the responsibility to invest in research career development infrastructure and incorporate research into their curricula. Dr. Nitkin acknowledged that academic departments also have financial constraints for supporting research. Not many departments offer a clinician the support to build a research career.
A Board member was concerned about limiting access by going to a wider group of professions. Dr. Nitkin noted that K12 networks are choosy about which institutions have adequate research infrastructure and commitment to support the student scholars. The Board member suggested looking at where the scholars are coming from and breaking it out among disciplines.
Dr. Cernich said that the NICHD data have made it impossible to support the K12 awards in their current form. Ultimately, the K12 program will be much reduced. A big concern with the K12s is limiting them to so few professions, which may limit perspectives and not serve the needs of the broader rehabilitation field. For example, there are no K12s in neuroplasticity or computational modeling. Moreover, the NCMRR provides mentoring and training through the research Infrastructure network, which is a $10 million annual investment. An important question for the Board is, how do we propel the field forward? How do we partner with associations to move forward?
Dr. Nitkin added that another consideration must be whether these investigators have a future in the field because there is not the turnover of academic positions anymore. Dr. Spong observed that when these programs began, there was a need for mentors. But now the fields have matured and there are people at other institutions who can mentor trainees. The NICHD is clearly an outlier in its number of K12 programs, which might mean the Institute is behind the times. A Board member who was once the recipient of a K12 award agreed that the quality of applicants is higher for individual K awards and that the field is mature enough to maintain the useful parts of the K12 awards, such as mentoring.
Dr. Cernich thanked the Board for the excellent discussion. She agreed that it was key to understand which aspects of the K12 awards should be preserved and what mechanisms are available to do that. Another consideration would be how to work with the professional associations to ensure that research remains a core capacity. Dr. Cernich suggested forming a subcommittee on training to look more deeply at this issue, talk to the K12 directors, and propose the best way to go forward. The working group might also consider where the gap is after the K or R21 award because that is a very vulnerable time for investigators.
Comments from Retiring Board Members
Dr. Roger Smith recalled some important things that have come to fruition since the Blue Ribbon Panel reviewed the NICHD, specifically a budget with more flexibility to try new things and the nearly completed Rehabilitation Research Plan. He urged the Board to keep in mind that rehabilitation research is different and has different requirements and challenged the Board to continue to be mindful of small businesses in the rehabilitation area. Dr. Smith concluded by noting that much has been discovered because of people with disabilities, yet a common exclusion criterion in trials is having a disability. There will come a time when it will seem very strange that trials did not include people with disabilities. Dr. Smith thanked his colleagues for such a stimulating 4 years.
Ms. Marilyn Spivak was not present due to a family medical emergency, but she sent her written farewell to the Board. She recalled how elated she was to be invited to join the Board 4 years ago and commended the commitment of the staff, which is so highly respected, and the gains made in just a year under Dr. Cernich’s and Dr. Nitkin’s leadership. She said that because she experiences the effects of disability both in her work and in her family, she knows first-hand the impact of chronic disease on the quality of life. Interventions are not a luxury. There is a need for access to treatment for all, not just those who can afford it. She ended by saying how grateful she was to serve on a Board that fosters such passionate discussion and such positive change.
Closing Comments and Solicitation of Topics for December Meeting
Dr. Cernich asked the Board for topics to be covered at the next meeting. Suggestions were the status of the Rehabilitation Research Plan, including an update about the NIH Rehabilitation meeting later in May, and opportunities in rehabilitation science as they relate to PMI.
Dr. Cernich said that there would be a summary of the conference. She suggested that any additional suggestions for topics be sent to Dr. Nitkin and asked Board members for names of rehabilitation scientists who are taking a precision medicine approach. Dr. Nitkin asked the Board members to spread the word about the issues and opportunities for the field. Dr. Cernich noted that the presentations at this meeting would be made available and thanked the Board members for their valuable counsel.
The meeting was adjourned at 11:50 a.m.