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 8-9, 2008
Dr. Margaret Turk, Board Chair, called the 37th meeting of the NABMRR to order at 8:30 a.m. Minutes from the previous meeting were approved.
May 7-8, 2009December 7-8, 2009May 3-4, 2010
As in previous meetings, Board members provided updates on their outside efforts to promote medical rehabilitation research and the activities of the NCMRR.
Dr. Ralph Nitkin provided some background on the NCMRR-funded career development networks. From the beginning, training has been a priority for the NCMRR with a particular focus on health maintenance and avoiding secondary complications, increasing progress even into the chronic phase, integrating clinical and research approaches, translating to real-world outcomes, and increasing patient demographics. Graduate students, postdoctoral fellows, and beginning faculty are supported through a variety of mechanisms at the NIH. The K12 programs provide a specific focus on training and career development in a particular professional discipline. Although the NICHD uses this mechanism widely to support programs in pediatrics and reproductive biology, NCMRR currently has three programs that focus on physiatrists, pediatric critical care, and allied health professionals. The NCMRR networks typically support 2 years to 3 years of mentored research followed by transition to independent research support. Current principal investigators of the NCMRR programs provided the Board with updates on their programs.
Dr. J. Michael Dean discussed the program in pediatric critical care, within which there is an increasing population of acute patients who require extended care. Yet the research base for implementing best outcomes lags behind practice. Within this population, pediatric and rehabilitation issues overlap because most of these children already have a disability when they come to the pediatric intensive care unit. The pediatric critical care K12 training program has an annual retreat and a national advisory committee, which includes chairs of pediatric departments and funded researchers. The overall goals of the program are to continue to support publications, good science, and the transition to sustainable NIH funding. The program also helps NCMRR enhance the link between critical care and pediatric rehabilitation. In the first five rounds of candidate submissions, this K12 program has had 70 applicants and 15 were selected. Dr. Dean highlighted some of the excellent research that has come from these trainees.
Dr. John Whyte discussed the physiatrist K12 program, which is focused on increasing the number of rigorously trained, extramurally-funded investigators in Physical Medicine and Rehabilitation (PM&R). Traditionally, PM&R departments have lacked a research tradition, lack the necessary mentors, and have a more complex scientific domain than other fields. The K12 program, co-sponsored by the Association of Academic Physiatrists, got off to a slow start but made major programmatic changes in its second funding cycle and later added a focus on pediatric rehabilitation. The program now includes a pre-applicant pathway, which is highly competitive and allows candidates to “hit the ground running”, as well as quarterly individual teleconferences with the trainees. This program has had enormous impact on the candidates, who have gone on to academic appointments, extramural funding, and continued research publications. Challenges include maintaining the pool of qualified applicants, competing with clinical fellowships, ability of PM&R chairs to provide necessary start-up and bridge support, and the heterogeneity of research topics.
Drs. Michael Mueller and Ken Ottenbacher provided background on the parallel programs that support researchers in allied health (e.g., physical therapists and occupational therapists). Dr. Mueller’s program includes three core institutions and four other training institutions. He outlined the K12 processes for selection, training, evaluation, and feedback. Candidates must have an academic appointment and a five-year commitment from their department chair. Each candidate is assigned dual mentors by the K12 program: one within the allied health specialty and one outside the specialty. Although only in its second year, the K12 program is has already had a positive effect with respect to supporting scholars and mentors and the transition to research publications and independent funding. The program is integrated with the two most appropriate professional organizations: the American Physical Therapy Association and the American Occupational Therapy Association. Current issues faced by the program leadership include educating the academic departments and the competition for independent research funding.
Dr. Ottenbacher’s K12 program represents a partnership between two mentoring institutions, which provide strong but complementary support for several key rehabilitation research areas. This support allows for centralized control of the training environment and career support of the candidates. Candidates are assigned to senior mentors who have active research programs, with support from broader mentoring teams. The program holds annual meetings in May that include scholar presentations.
As a follow up to the discussion of these programs, the Board discussed several issues. Rehabilitation also has a broad base of basic researchers that need to be integrated in these clinical departments, but leadership cannot expect the candidates to initiate these collaborations. Mentors must forge these relationships; translation is a team effort. Rehabilitation research must continue to seek opportunities based on clinical experiences and resources. Professional expertise needs to be complemented by flexibility and diversity, while still maintaining a record of productivity and specificity. The Board also considered the balance of research investment: what is the appropriate level of research trained faculty and how do we support faculty who do not quite get tenure?
Dr. Lisa Iezzoni discussed her research on disparities in health care. Much of this work depends on access to appropriate databases, because no single source (e.g., Medicare) covers the diverse population of PWD nor does a single resource include measures of functional impairments and disability. Dr. Iezzoni discussed a specific study that used to the Surveillance, Epidemiology and End Results (SEER) database from the National Cancer Institute to explore the hypothesis that PWD, who are in the later stages of cancer diagnoses, have worse outcomes. She found that PWD are 40 percent less likely to get a pap smear, 30 percent less likely to get a mammogram, and less likely to be counseled about the effects of smoking than those without disabilities. The findings were despite the priorities set forth by Healthy People 2010 and the Office of the Surgeon General on the 15th anniversary of the Americans with Disabilities Act. The leading causes of disability, as defined by Medicare status, involve mental health and musculoskeletal issues (which have superseded cardiovascular issues as the leading cause of disability). On average, Patients wait 29 months after diagnosis to get Medicare benefits after diagnosis (except for amyotrophic lateral sclerosis, which allows for immediate access to benefits); 126 of PWD have died during this waiting period, including 62 percent of those with cancer. Seeking support through the Consolidated Omnibus Budget Reconciliation Act (COBRA) would be too expensive to be a viable alternative for this population. Among the PWD diagnosed with cancer, the population is more likely to be black and to not be married. Although the work found no disparity in the stage at which breast or lung cancers were diagnosed among this population, results indicated that they were more likely to get a mastectomy rather than more conservative breast-conserving therapy. Data on chemotherapy options were not available, however women with disabilities were less likely to get radiation therapy and less likely to be offered surgical options for lung cancers than women without disabilities. Dr. Iezzoni noted that she had to submit her findings to rehabilitation journals because the traditional oncology journals chose not to publish her work.
Because of its broad expertise, the NABMRR is sometimes called on to provide an additional level of review for potential NCMRR research initiatives. Board members received background materials for such a review in the weeks prior to the meeting. Dr. Anne Krey, from the NICHD Division of Scientific Review, presided over the concept clearance process and Dr. Weinrich introduced the following research initiative.
In light of the growing interest in the orthotics and prosthetics field to develop appropriate health outcomes, the NCMRR proposed a plan to solicit R01 applications to explore the development, testing and evaluation of improved rehabilitation health outcome measures for consumers of orthotics and prosthetics. The Board pointed out that “outcomes” was a very broad concept that needed to be clearly defined in the context of this research solicitation. Prosthetics has changed dramatically in the last few years, and these changes have implications for longitudinal studies and longer-term outcomes. The Board asked about the need for expertise in materials sciences, but NCMRR staff explained that the focus of this solicitation was external prosthetics rather than on neuroprosthesis or implants, such as artificial knees or hips. The Board cautioned that the purchase of these aids is largely driven by reimbursement regulations rather than consumer preferences. The Board also encouraged the incorporation of anecdotal information and consideration of physician knowledge bases. Nonetheless, the Board felt this initiative was quite timely and fully encouraged the NCMRR to pursue research applications in this area.
Dr. Michael Weinrich indicated that the NIH is currently funded through a continuing resolution and this noted that the situation was likely to continue through March 2009. Currently paylines within the NICHD were down to the 7th percentile for R01 investigators, but new investigators will be supported up to the 12th percentile; two-year R03 and R21 grants will be supported up to the 12th percentile. There is no separate budget for the NCMRR within the NICHD appropriation and Center funding has remained flat during the last few years indicating that rehabilitation researchers are competitive with the rest of the NICHD for these limited research dollars. The situation has also limited the initiation of new funding initiatives at the NICHD, although the NCMRR did solicit applications for research partnerships to improve functional outcomes in order to bring together the necessary and appropriate research teams.
Dr. Weinrich also discussed changes in peer review at the NIH including more focused peer-review feedback, revised scoring, and limiting applications to only one revision. Reduced page limits are also being considered, with the potential for extra space for more complex clinical applications. The Board was concerned that rehabilitation researchers would be especially disadvantaged by page restrictions because of the need to explain more complex and trans-disciplinary concepts. Moreover, Board members queried whether this practice would also disadvantage new investigators who often lack a significant body of published work to reference. Dr. Weinrich suggested that perhaps the Board could help educate peer reviewers on some of the unique aspects of rehabilitation research.
Dr. Weinrich added that within the Center, Theresa Cruz has joined the NCMRR as a program analyst and will take on a role as liaison with NIH intramural programs. In light of budgetary restrictions, the Institute reduced the budget for administrative support by over 50 percent. The NCMRR feels that staff travel to professional meetings should remain a priority, but this reduction will severely limit the ability of Center staff to support networks and research/training workshops (although the latter could still be supported through the investigator-initiated R13 mechanism). He explained that the Center is also working with the National Center for Research Resources at the NIH to promote translational research through Clinical and Translational Science Awards, but added very few proposals for community-based research approaches were submitted. More information on research consortia supported through this program is available at https://www.ctsacentral.org/ .
Dr. Martha Banks began a discussion of this topic by indicating that rehabilitation research, particularly in the psychosocial domain, is mostly based on studies of men and boys and lacks sensitivity to important women’s issues, such as reproductive health and abuse. Research grounded in the medical model does not adequately consider the meaning of disability, social and cultural factors, the role of social networks, and shifting demographics. Disability status also impacts how people seek employment, develop healthy behaviors, and cope with religious and spiritual images. Dr. Banks discussed the role of personal resilience in managing a disability, seeking assistance, adapting, and surviving. In the discussion that followed, the Board related Dr. Banks’ remarks to other research that has been supported by NCMRR in the area of women with disabilities.
Dr. Michael Weinrich provided some background for this discussion, noting that although access to care, medical insurance, education, and physical activity are key issues for individuals with CP, these are really advocacy issues and not well suited for NIH research proposals. The NIH has a huge portfolio on the neuropathological basics of early brain damage and molecular and cellular approaches, but the NCMRR is more focused on rehabilitation strategies and supports a few studies in that area. Longitudinal studies tend to be expensive, time-consuming, and slow. Because CP is a heterogeneous disorder and the underlying pathology remains unclear, and we lack studies that consider the transition of developmental disabilities into adulthood are lacking. Moreover, we lack a pool of qualified investigators, who can separate the “hope from the hype”. Dr. Weinrich encouraged the Board to focus on research opportunities and priorities in order to determine which approaches would hold the most promise for CP efforts. He also discussed how Congressional initiatives in other developmental disorders such as Down Syndrome supported collaborations between the NIH, the Centers for Disease Control and Prevention (CDC), and professional and advocacy groups.
Drs. Daofen Chen and Deborah Hirtz provided some background on National Institute of Neurological Disorders and Stroke (NINDS) research on CP. In the years 2001 to 2008, the NIH received 236 proposals involving CP, which were distributed across 13 of the Institutes. In total, 77 of these applications were funded, 50 percent by the NICHD and 30 percent by the NINDS. NINDS proposals were mostly focused on neuropathology of and risk factors for CP, as well as clinical strategies for treating brain damage and refining the most appropriate target populations.
Drs. Laura Tosi and Mindy Aisen, from the Cerebral Palsy International Research Foundation, provided additional background on CP research progress. CP is an umbrella term for injury that occurs in utero, at birth, or at any time up to 2 years to 3 years of age. In addition to the motoric issues, CP may include epilepsy, autism, and hearing or visual problems, but most of the children are of normal intelligence. The incidence of CP is 2 to 4 per 1,000, a figure that has increased over the last two decades. Life expectancy is almost normal for those who retain walking ability, but is worse for those who are mobility impaired.
The demographics of CP and the transition into adulthood for individuals with CP are less clear. CP seems to be associated with premature aging, often involving pain and osteoporosis. Adults with CP may lose the ability to ambulate due to fatigue or inefficiency of gait. In addition, problems arise with deterioration of hip and knee joints, scoliosis, and stenosis. But it is unclear if physical therapies accelerate these bone and joint problems or whether there are strategies to delay these secondary complications and retain capacity and function. Most people with CP are pushed out of therapy at age 18-21 years and then lose access to health services. Orthopedists, who are more trained in sports medicine, lack the knowledge base to treat these issues in adults.
Neurorehabilitation generally takes the view that structure is destiny, but there is increasing evidence that some therapies can make a difference in functional progress. Training in CP should not be thought of as re-learning a task but rather learning it for the first time, and in the context of a developing nervous system that is adjusting to an early insult. Potential research opportunities include non-invasive imaging for early diagnosis, improved understanding of the mechanisms of neurological damage and plasticity, neuroprotective and neuroregenerative treatments, conserving/protecting/restoring musculoskeletal and neurological function, and applying the principles of gait training for spinal cord injury and stroke to this population of children.
The Board discussed the need to partner with other federal agencies, such as the Department of Defense on research related to CP. Members added that there are many intersecting themes that could build on the expertise that the Pediatric Critical Care and Rehabilitation program brings to the NCMRR. Other research questions related to aging with CP include: Is the appearance of premature aging really just a consequence of increased fatigue and pain? The Board also suggested a systematic review of the literature and an improved model for the progression of secondary disabilities. Within the context of adults with CP, it is unclear what health care supports exist for them and whether those that reach adulthood are inherently more functional. Members agreed that it would help to draw more adults with CP into research studies perhaps through recruitment to current clinical centers.
Dr. Michael Weinrich provided a brief update on activities in TBI research. The Congress has finally taken an interest in TBI research, especially as it relates to war injuries. This has resulted in several new initiatives including $300 million to the Department of Defense to study Post-traumatic Stress Disorder and TBI and to set up clinical trials on these and other topics. Within the NCMRR, Dr. Beth Ansel leads a TBI clinical trials network, which attempts to standardize treatments through the acute period and improve the linkage between acute and post-acute care. The network has also focused on the classification of TBI patients and on developing more comprehensive outcome measures. Current clinical trials at the NIH include a National Heart, Lung and Blood Institute (NHLBI) trial on hypertonic saline for coma patients and a National Institute of Neurological Disorders and Stroke study of neurological emergency treatment. The NCMRR supported a small study of acetylcholine for acute and post-acute TBI, but it did not make sense for the Center to try to support larger TBI trials so for the time being it will focus on investigator-initiated awards.
The NCMRR is also supporting a joint conference on combinational therapies using pharmacological strategies that helped advance treatments for cancer and HIV; the Center is also involved in creating a trans-agency Web site to document current activities on TBI. There is also a collaboration between NIH intramural programs and the Uniformed Health Services.
The Board discussed the need to develop behavioral platforms to assess treatments and functional progress. They questioned why Congress turned to the Department of Defense to lead this research effort rather than the NIH, and inquired how the NIH could help provide the necessary expertise to this effort. A particular issue with the studying TBI associated with war injuries is that soldiers are disproportionally drawn from rural America, where it is harder to get geographic coverage and maintain contact for follow-up studies.
Funding constraints within the NICHD require the NCMRR to dramatically reduce administrative costs. As a result, the May 2009 Advisory Board session will be conducted as virtual meeting and will probably impact at least one of the Center’s two annual meetings in the future. Obviously, this format will alter dialogue and the flow of information among Board members and friends of NCMRR. For the May 2009 meeting, NCMRR staff will work to develop a much briefer and focused agenda that would be appropriate for the virtual format. However, this format will make it more difficult for the Board to interact with ex officio members and other relevant agencies and organizations. Board members are encouraged to continue to interact with NCMRR staff through emails, phone calls, and other communications throughout the year.
The Board also discussed the responsibility of NCMRR-sponsored research grants to promote positive rehabilitation outcomes. Members also noted a need to develop appropriate data sets and population surveys, especially those that focus on disability status, outcomes, and children’s issues. Such databases are especially relevant to the NICHD, where the Institute Director, Dr. Duane Alexander, serves on inter-agency database groups. The Board also revisited the issue of providing a definition for “rehabilitation” and “disabling conditions”. One Board member suggested that stroke research might be a good place to start translating research into practice because of its strong research base and its potential to begin to define optimal treatment strategies for improving function.
Dr. Duane Alexander, NICHD Director, thanked the Board for its continued participation and wise counsel and advice. He indicated that the NIH research budget has been flat for the past four years, and that, if you figure in inflationary rates of 3 percent to 4 percent, this figure actually represents negative growth. He explained that he did not yet have projections for Fiscal Year 2009, and that the NIH remains on a continuing resolution that holds the NICHD at Fiscal Year 2008 funding levels. He hoped the new budget would be passed in March shortly after the inauguration. In addition, the NIH might get some additional support as part of the economic stimulus package with an investment in health science research. The research management and support budget (which covers staff salaries, travel to meetings, and office equipment) suffered a 60 percent reduction across the NICHD.
Despite these financial constraints, he explained that the NIH is working to preserve the new applicant pool with specific targets of 1600 new investigators funded. This goal will require some NIH institutes to set up more liberal paylines for new investigators. New legislation on pediatric medical devices has passed; the NICHD will work with NHLBI to develop the required report. Stem cell research, which is currently restricted to a few specific cell lines, will probably be expanded by the new administration. The National Children’s Study, supported by a specific allocation to the NIH Office of the Director, expands from $69 million in Fiscal Year 2007 to $110 million in Fiscal Year 2008 and $190 million in Fiscal Year 2009. The Study will enroll the first round of subjects as a pilot cohort in January 2009. Dr. Alexander also reminded the Board that the Pediatric Critical Care and Rehabilitation program provides an important bridge between acute care and longer term care in pediatrics, balancing the needs of people with disabilities and unique childhood issues.
Board members followed up with some specific questions to Dr. Alexander. They asked whether the legislation on Pediatric Medical Devices was targeted to small business (SBIR) grants, but Dr. Alexander indicated that it would also include investigator-initiated R01 grants. They also discussed to what extent the National Children’s Study will include children with disabilities and how they would be identified. They encouraged Dr. Alexander to support improving federal data and statistical data bases to include more appropriate measures of disability and relevant outcome measures, but conceded that this effort gets more difficult due to current reductions in research support. They also discussed whether proposed reductions in the page limits and numbers of revisions for NIH research proposals would differentially impact rehabilitation researchers.
The meeting was adjourned at 12:05 p.m.