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, 2011
Dr. Kimberly Anderson, Board Chair, called the 42nd meeting of the NABMRR to order at 8:30 am. Minutes from the previous meeting were approved.
Future Board Meetings
December 12-13, 2011*
May 3-4, 201
December 3-4, 20112
Leslie Gonzalez Rothi
|J. Michael Dean
Ex-Officio Members Present
|Daofen Chen, NINDS
Patricia Dorn, VA
Alan Guttmacher, NICHD
|Naomi Kleitman, NINDS
Lana Shekim, NIDCD
Lois Tully, NINR
|Michael Weinrich, NICHD|
Ex-Officio Members Absent
|Ted A. Conway, NSF
Suzanne Goldberg, NHLBI
Martin Gould, NCD
|Lyndon Joseph, NIA
Ann O’Mara, NCI
James Panagis, NIAMS
|Art Sherwood, NIDRR|
Michael Swanson, CDC
NICHD Staff and Visitors
|Beth Ansel, NICHD
Judith Cooper, NIDCD
Theresa Cruz, NICHD
Rosemarie Filart, NCRR
Marc Goldstein, APTA
Lisa Kaeser, NICHD
Mary Kim, CC
|Susan Lin, AOTA
Chanya Liv, NICHD
Michael Marge, NICHD
Sharon Miller, CMS
Ralph Nitkin, NICHD
Jo Pelham, CSR
Louis Quatrano, NICHD
|Mary Rodgers, NIBIB|
Rebecca Roth, NICHD
Mona Rowe, NICHD
Susanne Seagrave, CMS
Nate Thomas, APTA
Janice Wahlmann, NICHD
Al Wigmore, NICHD
NICHD VISION DISCUSSION
The majority of the meeting was focused on discussion of the initial five white papers published as part of the NICHD Vision process. The remaining four white papers were discussed through follow-up e-mails among Board members and are summarized in the addendum section below. Prior to the meeting, specific Board members were assigned to review and lead discussion for each paper. Within the context of these papers, the Board was particularly interested in highlighting compelling scientific opportunities and identifying possible gaps relating to medical rehabilitation and support for people with disabilities. The Board provided feedback to NICHD Director, Dr. Alan Guttmacher, on the second day of the meeting and also summarized its points in a formal e-mail, so that the remarks could be considered at the NICHD Vision Workshop on June 23-24, 2011, in Leesburg, Virginia.
Drs. Pam Duncan and John Chae, who co-chaired the NICHD Vision Plasticity workshop and authored the resulting white paper, led the Board discussion of the first paper. They briefly reviewed the NICHD Vision process to provide context for staging such a meeting and later developing a consensus document. Specifically, the chairs were struck by the breadth of the charge given to the group. As a result, they convened a pre-meeting in Washington, D.C., prior to the workshop to refine the agenda. NCMRR program officer Dr. Nancy Shinowara played a key role in this process. Given the short timeline, many of the top invitees were unavailable, which may have influenced the outcome. The workshop consisted of six working groups (two clinical, two basic, and two translational) with a healthy mix of young and established investigators. Plasticity is so broad and fundamental that it could touch on virtually every physiological process in medicine; so to keep the workshop more focused, the chairs chose to focus the white paper largely on neuroplasticity.
The Board noted that there is a strong need to look beyond isolated mechanisms and cellular phenomena. Members explained that plasticity occurs in the context of resources, environment, and social structure; biology and environment are important in shaping outcomes. The Board called for a stronger linkage between plasticity and behavior, which was the topic of another NICHD Vision white paper. The Board stressed that plasticity should be considered in the context of behavioral change and resiliency, which are crucial to rehabilitative outcomes.
The Board noted that rehabilitation is often central to the recovery of function, but that it is often eclipsed by the technologies in acute medical care. Similarly, prevention of secondary conditions is a key goal of rehabilitation and health maintenance, but is not a hot topic for researchers. Health is more than simply keeping the body alive. When discussing rehabilitation in the context of a scientific Vision, the Board added that is imperative to relate the message of “why it matters.”
The Board also discussed the need to encourage interdisciplinary research, especially collaborations between basic and clinical scientists. Currently, members noted that scientists operate in silos, which need to be broken down to really develop significant therapeutic advances. They added that translational applications should be considered up front in order to anticipate the necessary steps to move research discoveries toward clinical use. They suggested that introducing “team science” to young investigators would facilitate this goal, as well as building a sense of responsibility to promote multi-disciplinary science. Given that building research communities is as much a social as a scientific issue, the Board suggested that the NIH could assist this transformation by creating new funding mechanisms that require collaboration. It was noted that the field of rehabilitation provides a wonderful model of how to break silos, and that the topic of plasticity is an excellent case in point.
The Behavior white paper discussion was led by Drs. Dennis Hogan and Denise Tate, who noted several shortcomings in the paper regarding children with physical disabilities. The working group consisted mostly of psychologists, who focused on normal child development and mostly cognitive and behavioral outcomes. Board members explained that by neglecting physical disabilities, the white paper overemphasized educational outcomes (e.g., test scores) rather than considering functional outcomes and activities of daily living. The group noted that normative studies tend to focus on the middle 90 percent, eliminating the top and bottom 5 percent of continuous variables, and then go on to follow these “normal” trajectories over time. This research strategy tended to exclude the study of children with disabilities. They added that continuous variables are often impractical measures for disability; blindness, deafness, and mobility deficits are largely binary states. The white paper identified early childhood as a crucial time for genetic and epigenetic factors to influence cognitive development and later educational outcomes. However, the Board noted that there are huge variations in norms with respect to age, and that many disability trajectories are not age-related. The white paper largely focused on negative, scarring events, but failed to describe promotion of healthy interventions and mitigating factors. Although the Board supported the white paper recommendation for new measures of the environment, they would also encourage higher level studies of gene-by-environment interactions as another scientific opportunity.
Within the context of behavior, the Board identified several additional scientific opportunities, including the following: role of culture in defining norms, role of assistive technologies, the role of the built environment, accessibility, aging with a disability, role of the school system (often the main context of child interventions), and bioethics. The Board agreed that integrated modeling using common data elements and repositories is important. However, members expressed concern over how people with disabilities will be classified and categorized in the common data elements of the Electronic Health Records (e.g., by diagnosis or by functional abilities). Participants noted that the National Institute of Neurological Disorders and Stroke (NINDS) is currently promoting the use of common data elements to integrate research studies and that there is an NINDS genetics repository for the collection, pooling, and distribution of genotypic and phenotypic data.
The Board disagreed with the strong deterministic attitude of the paper, as trajectories are knocked off track in individuals with disabilities. It added that there are special opportunities to explore how trajectories are altered, who does well and who does poorly (and why), and how to effectively intervene.
Dr. Kim Anderson led the discussion on the Development white paper. The Board found the white paper comprehensive, but noted that it focused mainly on normal development. The Board felt that knowledge of normal development could be used to enhance interventions following injury or disease. Members added one missing scientific opportunity was the use of genotypic and phenotypic data to understand why some people respond to rehabilitation interventions and others do not. Furthermore, they said information from humans could be used to develop more appropriate animal models.
The Board observed that there was a large overlap between the Development and the Plasticity papers, particularly with respect to stem cells, technology development, genomics, and the role of environmental factors. While they felt the emphasis on animal models was appropriate, they thought there should be more emphasis on translational studies. The Board also discussed that while knowledge of underlying genetic defects is important, it does not guarantee an effective therapy (e.g., sickle-cell disease). The Board also suggested development of biomarkers for plasticity as a potential scientific opportunity and explained that it would be beneficial to the field to know how interventions affect the nervous system, as well as the development of prognostic biomarkers for risk of decline or for recovery.
Diagnostics and Therapeutics
Dr. Leslie Gonzales-Rothi led the Diagnostics and Therapeutics white paper discussion. The goal of this workshop was to identify scientific opportunities in the areas of biomarkers, techniques, and approaches in the field of diagnostics and therapeutics. The Board found the paper difficult to synthesize, because it reads like four separate reports, loosely divided into maternal-fetal-pediatric and neural rehabilitation topics. The former set of topics identified bi-generational scientific opportunities that link maternal, prenatal, and postnatal health with pediatric outcomes. The Board noted a strong emphasis on the fact that children are not small adults and therefore present new and challenging research opportunities. The Board particularly endorsed the goal to “gently and powerfully evaluate the status of the brain sooner,” which applies to both the pediatric and rehabilitation populations. Members noted that the ability to assess loss sooner would be revolutionary in aiding prognosis and treatment.
While this paper shared several recommendations with the other white papers (e.g., central data archive for rare patients, use of adaptive trials, role of pharmacogenetics, and need for combination therapies), there were several unique aspects that the Board supported. Specifically, this paper addressed the importance of forming relationships between academia and industry to promote translation of therapies. The Board appreciated inclusion of the following issues: patient-reported outcomes (e.g., NIH initiative on the Patient-Reported Outcomes Measurement Information System); epigenetics of susceptibility to disease; patient and caregiver as part of the research team to provide a bi-directional flow of information; outcome measures that are reliable, sensitive, clinically viable, and related to mechanisms of recovery; and the use of computational and engineering models to inform research design. The Board discussed the importance of good outcome measures and the need to translate them to the clinic in order to influence real-world practice. Finally, the Board felt that access to databases and individual patient records could provide a better estimate of the numbers of disabled populations.
The Reproduction white paper discussion was led by Dr. Lynn Gerber. The Board expressed disappointment that disability was largely neglected in this paper, despite the scientific opportunities that it could provide. It added that consideration of reproductive health as part of overall health was also lacking. The Board encouraged investigation of function in the context of reproduction, because quality of life, societal interactions, and disability may be major factors in determining reproductive health.
The Board emphasized that the issue is not merely that disability was neglected, but that there are specific scientific opportunities involving the intersection of reproduction and disability. They provided this example: in women with disabilities, symptoms vary across the hormonal cycle (e.g., spasticity in cerebral palsy increases during the menstrual phase). Other opportunities mentioned include special considerations for women with disabilities with respect to advanced maternal age, the need for Caesarean sections, in vitro fertilization and the ethics of genetic counseling, fertility rates, the relationship between the pregnancy and bone health, and the use of contraceptives. In the clinical realm, the Board explained that issues with the built environment and inadequate dissemination of information to clinicians may restrict access to reproductive care for people with disabilities.
The Board highlighted several overarching themes related to people with disabilities and rehabilitation research that members felt were integral to the NICHD Vision discussion. Themes included: ethical issues regarding access to knowledge, human subjects, informed consent, linking of data, and privacy; the importance of including people with disabilities, racial and ethnic minorities, immigrants, and those of low socio-economic status in research studies; health disparities due to functional disabilities; the importance of a common vocabulary and common data elements; and breaking down silos to promote interdisciplinary research and improve interactions among researchers (basic and clinical) and the public. Participants recognized that there are many scientific opportunities in the interplay of genetic, epigenetic factors with the biological environment, especially relevant to people with disabilities that should be investigated in a systematic way. For example, when investigating obesity in children, there are specific questions related to those with disabilities (e.g., accurate height and weight measurements, validity of those measurements and body-mass index for people with disabilities, and unique strategies for promoting health in children with disabilities). The Board also emphasized the need for clinically relevant translation and implementation of research findings and for the flow of information from lab to clinic to be bi-directional. The Board noted the importance of bioinformatics and access to large datasets, and the need for new modeling, methodology, and statistical strategies. Finally, the Board emphasized the importance of researchers continuing to justify “why it matters.”
Throughout the white paper discussions, the need for process changes was highlighted. The Board recognized that there is a need to overhaul the academic tenure process so that it acknowledges the contribution of collaborative activities and safety and optimization trials, which are all needed to promote translation of research finding into the clinic. The Board said it would also like to see more leadership training for scientists. Members added that translational research could be facilitated by promoting communication among local Institutional Review Boards and federal entities, such as the NIH and the Food and Drug Administration.
The Board provided its input to the NICHD Vision process through discussion with the NICHD Director on the second day of the Board meeting and in a subsequent e-mail to Dr. Guttmacher dated June 22, 2011. The e-mail included the following comments.
The Board was especially pleased with the inclusion of functional status; differences/disparities across populations; bioethics; analytical and measurement tools and methods; bioinformatics; biotechnology and bioengineering; implementation science; and training and mentoring. The Board felt that the Vision papers were especially strong with respect to analytical and measurement tools and methods, and to the importance of biomarkers. The Board agreed with the call for enhanced bioinformatics resources, including the formation of and access to large data repositories. The Board concurred with the call for the development of analytical and computational opportunities, including complex modeling as a common platform.
Board members recognized that there was a refreshing emphasis on training and mentoring, and a call for a change in culture. Nearly all the workshops emphasized the importance of breaking down the barriers and silos that divide the various basic and clinical sciences and of embracing transdisciplinary research. There was a strong call for the dismantling of barriers between basic and clinical scientists and a commitment to develop and utilize a common language to facilitate communication. Finally, there was a strong call for the development of a corporate culture research versus the prevailing individualized culture across federal institutions and the academia.
In reviewing and discussing these white papers, the Board also felt that there were a number of opportunities that could contribute significantly to the Vision process. Although functional status was identified as a cross-cutting issue, only two of the initial five workshops discussed this important domain. Even though the concept of quality-of-life was mentioned in several white papers, the International Classification on Function domains, such as activities and societal participation, were generally absent. The Board encouraged the exploration of the mechanistic relationships between the usual disease and pathophysiology parameters and the more clinically relevant outcomes of impairment, activities, societal participation, quality-of-life, as well as moderating the influences of behavior and the environment.
With respect to differences and disparities across populations, the Board explained that there is an important scientific opportunity to studying this in the context of persons with disabilities. Just as children should not be treated as “little adults,” persons with disabilities have their unique set of medical and physiologic issues and should be treated accordingly. Yet, the Board said that none of the workshops identified persons with disabilities in the application of their scientific opportunities; instead, the issue of differences and health disparities appear in other cross-cutting issues. For example, biomarkers and bioinformatics were strongly emphasized in all the white papers, but none actually mentioned any disability-specific variables. In discussing transdisciplinary research, the Board found that a variety of specific scientific and clinical disciplines were mentioned, but that rehabilitation was omitted even though rehabilitation is specifically included in the mission statement of the NICHD. In the discussion of prevention, the Board found no mention of prevention of disabilities nor of secondary conditions.
The Board also recognized that an important concern for many of these white papers was the inadequate emphasis on translation and implementation science (i.e., T1 from laboratory science to animal studies; T2 from animal studies to first human implementation; T3 from first human implementation to pivotal trials and optimization studies; and T4 from pivotal trials to actual clinical practice). The Board was especially concerned that most of the workshops focused on T1 and T2 level studies, less on T3, and almost nothing on T4. The Board believed this reflected the predominant culture of the scientific community, which may actually be a major barrier to clinical and real-life translation.
The Board said that while bioethics was noted as a cross-cutting issue, there was relatively little attention to ethical issues. Specifically, it had concerns about ensuring adequate knowledge of interventions for research participants, protection of privacy, and consent issues.
The Board recommended the addition of section entitled “Why it matters” to the white papers to emphasize significance and relation to health of Americans. The Board also recommended addition of a section entitled “Process opportunities” to address policies, regulations, and institutional (academic, industry and federal) relationships.
Addendum: After the Advisory Board meeting, the Board discussed (through e-mail) the remaining four NICHD Vision papers: Developmental Origins, Environment, Cognition, and Pregnancy and Pregnancy Outcomes, which were released after the May 2011 meeting. The Board’s comments were included in a formal e-mail to Dr. Guttmacher dated June 22, 2011, which highlighted the following issues.
Board members explained this as a compelling White Paper that weaves an integrated story of genomic, epigenomic, and metagenomic influences on the human organism. However, they added that the paper is over-focused on perinatal events, and that it neglects additional opportunities for probing the same biological mechanisms at latter stages. While the paper provided an outstanding discussion of the cell and molecular principles, the group felt that it provided limited discussion of clinical strategies for interventions during embryonic, gestational, and/or perinatal stages. E-mails from the Board described the unique opportunities in populations such as children with critical illness or persons with significant disability, who have suffered severe perturbations from a “normal developmental trajectory.” Members added that studies in younger populations may provide more detailed understanding of the primarily genomic factors, while studies in older populations would help understanding the role of superimposed environmental exposures on the ultimate health trajectory. The Board noted that progress in understanding the integration of genomic, epigenomic, and metagenomic influences on the trajectory of human development will require concerted efforts to build transdisciplinary teams who have the requisite intellectual skills to link research findings together into a comprehensible context. Moreover, the research must move beyond the traditional reductionist approaches in order to develop new ideas, teams and tools, as suggested by the white paper report.
Board members noted that this is currently a very important research topic and commended workshop organizers and participants for their efforts in addressing this topic. They added that while the paper is quite comprehensive in the range of exposures, it is also limited in certain perspectives. Notably, the definition of “environment” used in this white paper omits the structural environment, which particularly affects the functioning of people with physical disabilities and other limitations. Members explained that the white paper heavily emphasized toxins and chemicals in the environment, but that it should be more inclusive of other environmental threats (compare with Developmental Origins and Pregnancy white papers).
Board members felt that the paper should begin with a broader discussion of the ”environment,” describing the multiple levels associated with the natural, physical, social, and cultural environment and potential influences on child health and optimal development. They also felt that the paper would benefit from a brief review of the literature describing current knowledge and gaps in research in order to frame the issues that are currently not being addressed, especially given the respective mandates of the NICHD and the National Institute of Environmental Health Sciences. Members hoped to see a much broader discussion of the issues associated with various aspects of the structural (built) environment; technological, social, and cultural communication; economic and policy elements; and the interaction between policy and outcomes. The Board also wanted the paper to acknowledge that environment can either interfere or enhance prevention of both primary and secondary disabilities, with a special focus on obesogenic factors that contribute to obesity, potential ethnic and health disparity factors, injury prevention, and environmental factors that contribute to positive health and resilience.
Board members indicated that the workshop focused exclusively on developmental cognition, and thus addressed “habilitation” but not rehabilitation. Given the mission of the NICHD to support the health, productivity, independence, and well-being of all people throughout the lifespan, the Board felt that a major limitation of this white paper was the lack of focus on acquired cognitive disorders, especially in the context of aging, disease, or injury. On the other hand, members noted that the paper contained some very interesting and appealing discussion of the need to: develop registries and create common databases to study individuals with developmental disorders; establish better links between researchers and professionals in the community; use evolving technologies to further characterize normal cognitive trajectories; and the development of preventative recommendations for optimizing cognitive health.
Pregnancy and Pregnancy Outcomes
The Advisory Board explained that, as with the Developmental Origins workshop, participants in this workshop utilized a more inclusive definition of environment that extended across the lifespan (i.e., “a sequence of socially defined events and roles that the individual enacts over time” and that these events occur in “relation to the structural, social, and cultural environment of the individual”). However, Board members felt that the discussion was largely presented in the context of the “able-bodied” population, and (especially given the mission of the NICHD) lacked any consideration of the health and functioning of persons with disability. The Board noted that pregnancy in the context of debilitating conditions such as spinal cord injury, traumatic brain injury, multiple sclerosis, or adult cerebral palsy required unique clinical considerations and offered additional scientific opportunities for exploring biological interactions. They added that the white paper would benefit from incorporating findings from the NICHD meeting on Pregnancy in Women with Physical Disabilities, January 25-26, 2010, including such issues as pregnancy outcomes, pregnancy-associated risks to mother and baby, and strategies for overcoming clinical barriers (see http://www.nichd.nih.gov/about/meetings/2010/Pages/12610.aspx for more information on the meeting).
ELECTION OF THE NEXT CHAIR-ELECT
At the end of this meeting, Dr. Kim Anderson finished her term as chair, and Dr. John Chae moved up from chair-elect to chair. Elections were held to determine the next chair-elect. Dr. Pamela Duncan was elected by unanimous vote. Dr. Chae will continue to serve as the Board liaison to the National Advisory Child Health and Human Development (NACHHD) Council.
REPORT OF THE NCMRR DIRECTOR
Dr. Michael Weinrich gave the NCMRR Director’s Report to the Board. He informed the Board of the difficult financial position that the NIH is currently experiencing. He said he anticipates low paylines, low travel funds, and limited opportunities for the NCMRR to sponsor conferences in the coming year. Despite this outlook, he pointed out that the NIH did well compared to other federal research agencies with respect to budget cuts. NCMRR staff provided brief updates on their respective travel and meeting activities. Dr. Weinrich indicated that in response to requests from the research community to promote the NCMRR into a stand-alone institute, the NIH Director, Dr. Francis Collins, commissioned a blue-ribbon panel to examine rehabilitation research support across the NIH. Dr. Weinrich also indicated that the American Physical Therapy Association (APTA) sponsored a briefing on Capitol Hill that was well attended by congressional staff and featured keynote presentations from Drs. Alan Jette, Richard Shields, and NICHD Director Alan Guttmacher. He added that Carol Sheredos is now circulating a weekly, federal-wide news briefing from the NCMRR that highlights information on disability and rehabilitation issues.
Dr. Weinrich highlighted the following scientific opportunities and activities:
- Rehabilitation research in the United States is largely hospital- and clinic-based, despite the fact that chronic treatment of disability may be better served by community interventions. We know that exercise is beneficial, regardless of where it takes place. Research into environmental and behavioral factors to promote physical therapy, in conjunction with other therapies in the community is needed.
- Chronic traumatic encephalopathy (CTE), a neuronal degenerative disease traditionally observed in boxers and football players, is now being observed in the brains of younger people. These dramatic findings have implications for young people returning to contact sports after a concussion, as well as for soldiers exposed to repeated concussive injuries in battle. Many research questions exist, including: what are the underlying genetic factors and what are the appropriate animal models?
- Participating in the nanomedicine initiative at the NIH, Dr. Weinrich was exposed to intriguing work that has exciting implications for vision rehabilitation. Photosensitive ion channels that change configuration when exposed to light can be inserted into retinal ganglion cells in order to provide a unique opportunity to restore some visual functioning to patients with macular degeneration or retinitis pigmentosa. These approaches also broach a broader question of how to teach the nervous system to adept to altered neural inputs to the brain.
PROMOTING HEALTHY OUTCOMES IN CHILDREN WITH DISABILITIES: REDUCING OBESITY AND OTHER SECONDARY CONDITIONS
Dr. James Rimmer provided the Board with some background on promoting healthy outcomes in children with disabilities. Rates of obesity have been increasing in both the non-disabled and disabled populations. The majority of these findings come from national surveys and self-report, which presents some problems with respect to overestimates of height and underestimates of weight. In his own research, Dr. Rimmer found that that biased self-report leads to a twofold underestimation of obesity rates, compared to actual height and weight measurements taken by clinicians. He added that the ability to get accurate measures is especially difficult for children with disabilities because accessible instruments are often unavailable and impairments (e.g., contractures, wheelchair use) may make accurate measurement difficult.
In addition, Dr. Rimmer explained that even when accurate measures are collected, the interpretation and meaning of these data are unclear. For example, body-mass index (BMI) may not be a meaningful measure of fat distribution for the disabled population. Data have shown that BMI values in the normal range for the healthy population could be associated with obesity when they occur in subjects with spinal cord injury. Likewise, cerebral palsy subjects with relatively low BMIs, actually have obese levels of fat mass. Thus, BMI maybe be a good surrogate measure of body fat in the non-disabled population, but these associations do not hold among disabled populations.
He acknowledged that while the cause of the obesity epidemic is multi-factorial, decreased physical activity is a contributing factor. Children with disabilities are often limited by their access to playgrounds, recreational equipment, and sports teams. Power-wheelchair use is also on the increase. To prevent negative spirals, he felt that research is needed in motivation and delivery of care, especially in home-based and community-based interventions.
Dr. Rimmer’s presentation spurred some discussion of scientific opportunities as a way to entice obesity researchers to address obesity in disabled populations. For example, the distribution of body fat is different in the disabled and non-disabled populations (especially as fat replaces inactive skeletal muscle rather than depositing in the abdomen). This situation may influence metabolic processes, such as glucose tolerance and the onset of diabetes, and could be an important area for further research.
REPORT FROM THE NICHD DIRECTOR
Dr. Alan Guttmacher addressed the Board with NICHD and NIH updates. He congratulated the NCMRR on reaching its 20th anniversary. Over the last year, Dr. Guttmacher was active in promoting rehabilitation research at the NIH and beyond. He also highlighted the hearing on Capitol Hill that was arranged by the APTA (mentioned above). He said he also spoke at the annual meeting of the Association of Academic Physiatrists in Phoenix, Arizona. Dr. Guttmacher discussed the formation of the blue-ribbon panel endorsed by NIH Director Francis Collins to assess rehabilitation research across the NIH with particular focus on the role of the NCMRR. He added that Drs. John Chae and Rebecca Craik have been invited to chair the panel.
Dr. Guttmacher described Dr. Collins’ creation of a new Institute at the NIH to begin operation on October 1, 2011. The goal of the National Center for Advancing Translational Sciences (NCATS) would be to advance the discipline of translational science and catalyze the development and testing of novel diagnostics and therapeutics across a wide range of human diseases and conditions. He explained that NCATS will improve processes in the therapeutics development pipeline by experimenting with innovative approaches along the pipeline utilizing an open-access model, choosing therapeutic projects to test innovative approaches, and by promoting interactions to advance regulatory science. It is also hoped that the NCATS will catalyze development of novel diagnostics and therapeutics by encouraging collaborations across all sectors, by providing resources to enable therapeutic development, and by enhancing training in relevant disciplines. He stressed that the NCATS would facilitate—not duplicate—other translational research activities supported by the NIH; complement—not compete with—the private sector; and reinforce—not reduce—the NIH’s commitment to basic research.
Dr. Guttmacher updated the Board on recent budget developments. In fiscal year (FY) 2010, the NICHD was appropriated funds of $1.327 billion, $699.3 million of which was used to support funding of 1,680 research project grants (RPG). The RPG success rate was ~15.2 percent, up slightly from 14.9 percent the previous year. The FY 2011 budget was recently passed with reductions across many federal agencies. He explained that the reduction for the NICHD
(-0.9%) translates to an estimated $11.7 million less in FY 2011 than in FY 2010.
Dr. Guttmacher described the ongoing Vision process at the Institute. He noted that the nine topic workshops had all met and the resulting white papers are (or will soon be) available for comment. He said that feedback from the public is highly sought and may be delivered via the NICHD Vision Web site at http://www.nichd.nih.gov/vision/. He also indicated that a large meeting would be held in June 2011, consisting of a diverse group of external experts and NIH leaders, to discuss and refine the draft Vision statement. Then the NACHHD Council would have an in-depth discussion during the September 2011 Council meeting to add final revisions. He noted that the goal is for the final NICHD Scientific Vision statement to be completed and published in a leading journal and posted online by the end of 2011 and that dissemination and strategic planning will follow in calendar year 2012.
The update segued into a dialogue between Dr. Guttmacher and the Board, led by Dr. Chae, whereby the Board provided initial feedback on the NICHD Vision papers in the areas of Plasticity, Behavior, Development, Diagnostics & Therapeutics, and Reproduction. Details are presented earlier in these minutes, but, briefly, the Board commended Dr. Guttmacher for his efforts in undertaking this Herculean task. They reiterated their support for research in several cross cutting areas, such as biomarkers, data depositories, bioethics, and trans-disciplinary training and research.
The Board also highlighted several missed opportunities. Function, quality-of-life, and participation status, although large contributors to overall health, were largely missing from the papers. Members felt that the NICHD community has a great appreciation that children are not small adults, but that, likewise, the disabled population has unique research needs of its own. People with disabilities should not merely be included in larger studies without special consideration of scientific questions developed around their needs. They explained that the inclusion of disability-specific variables in large datasets and bioinformatics may help promote disability research. They noted that the prevention of disease was discussed, but not in the context of disability and secondary complications. Discussion of translational research was largely restricted to strategies to move from bench to animal, while widespread clinical use should be the ultimate goal.
The Board added that it would like to see a stronger integration across the Vision themes. For example, they felt the behavioral aspects of rehabilitation, both physical and cognitive, need to be combined with therapeutics (e.g., stem cells and pharmaceuticals). Similarly, behavioral outcomes need to be assessed in a gene-by-environment interaction analysis. These types of research approaches will require the formation of integrated research teams using new analytic and statistical tools. Finally, the Board emphasized the need to articulate to the general public why research is needed.
RETIRING BOARD MEMBERS
The Board acknowledged the retirement of four Board members—Drs. Kim Anderson, Lynn Gerber, Linda Robinson, and Gail Whiteneck—and entertained parting remarks from those present. Next fall, the Board will be joined by the following new members: Carmen Green, M.D. (University of Michigan); Todd Kuiken, M.D., Ph.D. (Rehabilitation Institute of Chicago); Nicholas LaRocca, Ph.D. (National Multiple Sclerosis Society); and Jonathan Wolpaw, M.D. (Wadsworth Center).
NEW BUSINESS FOR NEXT MEETING
The next NABMRR meeting will celebrate the 20th anniversary of the NCMRR. The featured talks will focus on the Medical Rehabilitation Research Infrastructure Network. Future discussion topics include the representation of disability in the National Children’s Study and what is the “science” of rehabilitation.
The meeting was adjourned at 12 noon.
Ralph M. Nitkin, Ph.D. Date
Executive Secretary, NABMRR
Kim Anderson, Ph.D. Date