Dr. Margaret Stineman, Board Chair, called the 22nd meeting of the National Advisory Board on Medical Rehabilitation Research (NCMRR) to order at 8:45 a.m. Members of the Board, Ex-officio members, and visitors were invited to introduce themselves.
May 24-25, 2001 (note change)December 3-4, 2001May 2-3, 2002December 5-6, 2002
Dr. Duane Alexander discussed the implementation of new programs to support loan repayment for trainees in targeted areas. Already such programs are in place for contraceptive and reproductive health, and future programs will include pediatrics and more general aspects of clinical research. The National Institutes of Health (NIH) budget was finally passed in late December. The NICHD got a 13.7 percent increase to $976 million (slightly behind the overall NIH increase of 14 percent). Congress remains committed to doubling the NIH budget within five years; this represents the third year of that cycle. The NICHD increase will not translate into significantly more grants funded because of the inflationary costs of ongoing grants. The NICHD "success rate" should remain at about 27-28 percent of submitted applications. Dr. Alexander also announced that Dr. Owen Rennert, a pediatric researcher from Georgetown University, has assumed the position of Scientific Director and Head of Intramural Research at the NICHD.
Dr. Michael Weinrich discussed some staff changes at the NCMRR. Beth Ansel has become the Director of the Clinical Practices program and Chris Jennings has joined the support staff, and Debbie Welty will be leaving the NCMRR to work in the Center for Scientific Review of the NIH. Carol Sheredos is also working with the NCMRR on a special, one-year fellowship. The Center is currently recruiting someone to direct the emerging program in pediatric trauma.
Dr. Weinrich highlighted some of the recent research initiatives. The Regional Network program has begun with four strong centers, which are discussed in more detail below. Several applicants responded to recent requests for applications (RFAs) in the area of areas of Innovative Therapeutics and Pilot Clinical Trials for Pediatric Brain Injury. A few applicants responded to the request for a contract to develop a web site to promote engineering solutions among the rehabilitation community. The RFA for clinical trials planning grants in the area of Pediatric Rehabilitation is still open; and an RFA for clinical trial networks in traumatic brain injury will appear soon. Despite these initiatives, the greatest potential for growth in the NCMRR remains the development of investigator-initiated proposals, which are the backbone of the NIH biomedical research.
Dr. Weinrich also discussed recent meetings sponsored by NCMRR. These include workshops on Stroke and Hip Fracture and on Home Mechanical Ventilation, which involved collaborations with the Health Care Financing Administration (HCFA). The NCMRR also initiated sessions at the World Congress on Disabilities on Measurement of Pediatric Disability and on Pain Management. The NCMRR workshops help promote high-quality research, identify gaps in knowledge, and bring data to the attention of policy makers.
Finally, Dr. Weinrich described new procedures to facilitate reimbursement for travel expenses. For standard reimbursement, Board members should sign their travel orders on-site and hand Chris Jennings their hotel receipts and airline stubs (or corresponding paperwork from electronic tickets). Dr. Weinrich promised that reimbursements will be processed within the NICHD within the first weeks after the meeting.
Because of its expertise, the Board was called on to provide an additional level of review for specific NCMRR initiatives. Dr. Laurence Stanford, Director, Division of Scientific Review, NICHD, led this part of the discussion. Background material on each of the following five proposals was distributed.
The Board endorsed the development on an RFA on Clinical Studies on the Use of Augmentative and Alternative Communication for Cognitive-Linguistic Deficits. Members felt that patients could benefit from emerging technologies and encouraged increased access to these advances. They suggested that the subject population include patients who have survived central nervous system cancer and encouraged increased communication with industry in the development and adaptation of relevant devices.
After some discussion the Board endorsed the development of an RFA on Timing, Intensity and Duration of Rehabilitation for Stroke or Hip Fracture. Board members questioned the connection between these diverse conditions, but Dr. Weinrich pointed out that they coexist in rehabilitation facilities and are the leading categories of HCFA reimbursements. The Board stressed the importance of defining "usual treatment," especially in the context of current HCFA guidelines. There may also be a distinction between those most amenable to treatment and those more severely affected; likewise, considerations of front-loaded versus more long-term treatments. Although it is unlikely that these studies can be integrated into current HCFA demonstration projects, they need adequate resources so that they can be done right. There are individual differences within the patient population, and quite a breadth of outcome variables.
The Board supported the need for an RFA on Innovative Research Approaches to Reducing Secondary Conditions Associated with Primary Disabilities, but wanted some clarification on the breadth of relevant conditions. The members were assured that the priority would be those secondary conditions that have the greatest occurrence (e.g., pain, fatigue, depression, and bowel and bladder control) and are most amenable to intervention. The focus would be on physiological mechanisms and treatment strategies, rather than prevention or demographic data (which are more in the domain of the Center for Disease Control and Prevention). The Board encouraged the NCMRR to consider the physiological-environmental link in some of these conditions and to be sure that the secondary conditions are defined in medical terms rather than as patient "issues". The National Institute on Disability and Rehabilitation Research supports some centers that study secondary conditions, but the initiative proposed here has a unique emphasis on physiological mechanisms. Overall, the Board endorsed this concept, but stressed the need to prioritize and focus the scope of the research initiative.
The Board supported the need for an RFA in Pharmacological Approaches to Enhance Neuromodulation and Rehabilitation. This is a broad research area, but has exciting connections to emerging basic neurobiological findings. It is worth considering creative ways to support the pharmacological studies and potentially atypical therapeutic approaches, possibly through collaboration with HCFA.
The final initiative considered by the Board was an RFA on Innovative Bioengineering Initiatives in Rehabilitation, which is focused on tissue and prosthetic design. This topic would not include studies regenerating organs such as liver, but rather tissue reconstruction and prosthetic interfaces. This initiative could include tissue-level studies and studies in appropriate animal models. The National Institute for Neurological Disorders and Stroke currently has a large program in neural prostheses, and the National Institute of Arthritis, Musculoskeletal and Skin Diseases supports related work in muscle, bone and skin research. The Board endorsed the need for the NCMRR to develop this RFA in tissue bioengineering, which focuses on rehabilitative issues.
At this point in the meeting, Dr. Margaret Stineman welcomed the special visitors to this meeting including several alumni who had served on the Board in previous years. She encouraged each person to say a few words about their connection to the NCMRR and medical rehabilitation in general.
Dr. Henry Betts provided some personal remarks on the origins of the NCMRR, as well as the political climate for early advocacy in the area of medical rehabilitation. At that time, medical schools were more focused on acute illness and did not put a high priority on study of the elderly and "cripples". Physiatry was a second-class specialty. To stimulate research and clinical trials for people with disabilities, medical schools sought to form a link with the highest level of scientific inquiry, as represented by the NIH. The initial research agenda, developed by physiatrists and orthopedists, was promoted through the networking skills of the Mary Lasker, the prominent biomedical patron who had a seminal role in the formation of several NIH Institutes. Mary Switzer and others supported the training of more physiatrists, as well as programs to facilitate the employment of people with disabilities. Within universities, medical departments worked to get clinicians and basic researchers to collaborate and to address aspects of medical rehabilitation. Leaders of the rehabilitation community met with the directors of various NIH Institutes and began lobbying Congress for the formation of an NIH Institute focusing on medical rehabilitation. The NCMRR was established in 1990, located within the National Institute of Child Health and Human Development (NICHD).
Dr. Ralph Nitkin introduced the four regional research networks. The networks are an important new initiative for the NCMRR, which was actually stimulated by discussions with the Advisory Board. The NICHD is committing almost $4,000,000 per year to support medical rehabilitation research networks located in the West, Midwest, South, and Northeast, respectively. According to RFA, networks were to be built on major collaborations among three institutions, with the potential to connect with researchers from other facilities within the U.S. region. Each network must support multidisciplinary research cores, information transfer, and pilot projects, with the goal of facilitating ongoing projects and stimulating the development of future research activities in medical rehabilitation. Ultimately, the network must demonstrate the potential for increasing the quality and quantity of research applications. The NCMRR was very pleased with the quality of responses to this special initiative and the broader effect that this has had on energizing the medical rehabilitation community.
Dr. Marjorie Anderson described RehabNet-West, which has a very broad geographical outreach and particular focus in basic and clinical neurobiology. Dr. Peter Patrick provided some background on the southern ERRIS (Enhancing Rehabilitation Research in the South) network, which has particular expertise in the quantification of disablement. Dr. Zev Rymer described the midwestern Center for Advanced Research on Neurorehabilitation, which is currently focused on bioengineering and neurosciences. Dr. John Whyte described the northeast Cognitive Rehabilitation Research Network, which provides expertise in the treatment of traumatic brain injury and stroke. More details on each of these networks and contact information can be obtained through the NCMRR web site at http://www.nichd.nih.gov/about/org/ncmrr/.
Immediately after lunch, four brief talks highlighted various areas of research currently supported by the NCMRR.
Dr. Leonardo Cohen, of the National Institute for Neurological Disorders and Stroke intramural, program discussed brain imaging and neuroplasticity. Neuroplasticity refers to the way that the nervous system adapts to changes in the environment. Functional neuroimaging shows which brain regions are activated during certain tasks, but provides little information about the regions' specific role in performance. During recovery from stroke, there is generally an increase in the size and number of regions that become activated. Magnetic resonance spectroscopy can be used to examine neurochemical changes, especially those involving neurotransmitters. Transcranial magnetic stimulation can be used to stimulate specific brain regions to see what movements are affected (e.g., twitching of a finger). The brain shows remarkable plasticity in its ability to respond to injury or dysfunction. In the case of blind individuals who have developed the ability to read Braille, visual centers get reprogrammed to respond to tactile sensation. Other recent studies demonstrate the ability of "constrained use" therapy to refine activation regions in stroke patients. One caveat is that studies of these individuals must consider the possibility that the initial pathophysiology has subtly altered the brain anatomy, or that the individual may have adopted alternative cognitive or motor strategies to perform a given task.
Dr. Hunter Peckham, from Case Western Reserve University, discussed some exciting new advances in the development of neural prostheses for the restoration of motor function. A neural prosthesis refers to any device that interfaces directly with the nervous system to augment function. This may include: motor prostheses for limb, respiration, or bowel and bladder function; sensory prostheses for hearing or vision; or even strategies for suppressing undesired movements (e.g., spasticity). Dr. Peckham showed some remarkable videos of implanted sensors and stimulators that improve reach and grasp in individuals with upper body dysfunction. More recently, his lab developed a network of leg implants to stimulate a sequence of muscle contractions that allow a lower-limb paralyzed individual to stand up.
Dr. Robert Nerem, from Georgia Tech University, discussed tissue engineering. Current research focuses not only on implantation and substitution, but also on stimulating remaining tissue to support regeneration and remodeling. Early successes in tissue engineering centered on skin. Research continues to be driven by advances in basic cell and tissue biology. Issues in tissue engineering include cell sourcing, controlling cell functioning, interactive biomaterials, three-dimensional constructs, scaling-up, preservation, and immune acceptance. Stem cells offer the possibility of material that can differentiate into a variety of cell types. At this point, tissue engineering has been more successful in restoring structural integrity than more subtle aspects of tissue function.
Dr. Mary Law, from McMaster University in Canada, discussed studies in the participation of children with disabilities. About 6.5 percent of children have some disability that limits their participation in daily activities, and this isolation becomes more severe as they reach school age. Current research focuses on the interaction of environmental factors, family, and specific child issues. Initial studies suggest that key factors in the enhancement of participation include: minimizing "barriers"; presence of supportive relationships; supportive home environment; decreasing financial and time impact; demographics; self-perception; nature of physical, cognitive and communication disabilities; presence of validating frameworks; and policy decisions.
This sector represents the beginning of a dialogue with the Advisory Board to determine how responsive the NCMRR has been to the needs of the rehabilitation community and whether it is time to update the research priorities. Dr. Nitkin summarized the first ten years of research supported by the NCMRR. Initial funding focused on training grants because of the need to build a cadre of basic and clinical researchers focusing on medical rehabilitation issues. The research and training portfolio of the NCMRR has grown steadily to its current level in fiscal year 2000, of 190 grants totaling $37,711,704; this represents 5.4 percent of the NICHD budget. Although there have been some important workshops and research initiatives, the majority of this funding is driven by the development of successful investigator-initiated applications.
The research agenda for the NCMRR was developed through a series of meetings among persons with disabilities, scientists, clinicians, and engineers, which culminated in the Hunt Valley report of 1990. Dr. Nitkin reiterated the seven research priorities and provided some indication of the types of grants that the NCMRR has supported in each of these areas. He reminded the audience that the funded research is largely a reflection of investigator-initiated applications that were able to pass muster in the rigorous arena of NIH peer review. Moreover, this list only represents research supported by the NICHD, but not other rehabilitation-relevant research that may have been supported by other relevant institutes of the NIH.
Based on a simple perusal of grant titles, approximately 10 percent of the NCMRR research during the first ten years of its existence falls in the area of improving function and mobility. This includes research on wheelchair usage; coordination and control of arm movements; gait analysis; therapeutic footwear; surgical and drug treatments for spasticity; body strength and exertion; constrained-use therapy; and improving respiratory control.
Approximately 9 percent of NCMRR research can be classified under promoting behavioral adaptation to functional losses. This includes research on: psychosocial adjustments; health promotion, wellness, and exercise; impact on caretakers and family members; special issues for women with disabilities; geriatric issues; participation of children with disabilities; cognitive deficits associated with multiple sclerosis; sexuality; compensatory strategies for memory and cognitive deficits; improving access to certain environments; and virtual reality and other computer tools.
Approximately 6 percent of NCMRR research can be classified under assessing the efficacy and outcomes of medical rehabilitation therapies and practices. This includes research on: treatment for pain and depression; interventions for urinary tract infections and bladder dysfunction; neuroimaging in stroke and traumatic brain injury; decision-making and access to health care; economic constraints; tendon surgery for cerebral palsy; pharmacological treatments to improve respiratory function; and recovery from hip fracture.
Approximately 15 percent of NCMRR research can be classified under developing improved assistive technology. Research in this area particularly benefits from the fact that a small percentage of the NIH budget is set aside to support small business innovation research. Research in this area includes: wheelchair design; prosthetic hands, feet, and knees; improved fitting and monitoring of orthotics and prosthetics; design of specialized recreational equipment; neuroprostheses/microsensors for brain, nerves, muscle, or joints; functional electric stimulation of muscle or nerve; external control of bowel and bladder function; communication aids and computer interfaces; and improved environmental control.
Understanding whole body system responses to physical impairments and functional changes is a broad areas that accounts for about 28 percent of NCMRR funding. This includes: motor cortex changes associated with stroke and brain injury; spinal cord; plasticity and regeneration; muscle atrophy and decreased bone density; causes of pain and potential treatments strategies; cognitive and behavioral changes (e.g., attention, memory); reproductive function and sexual response; comorbidities and mortality; spasticity and muscle coordination; skin ulceration; tissue/biomaterial interface; sympathetic and parasympathetic function; and training strategies involving robotics and computers.
Research in developing more precise methods to measure impairments, disabilities, and societal and functional limitations accounts for about 9 percent of the NCMRR budget. This includes: demographics; archives of disabilities; brain imaging for diagnostics and prognosis; improved measures of well-being, satisfaction and quality-of-life; special assessments for children with disabilities; measurement of bone, muscle, and limb function; risk factors; and ethnographic and socioeconomic analyses.
About 16 percent of the NCMRR funds supported training research scientists in the field of medical rehabilitation. This includes: individual fellowships for postdoctoral fellows; institutional training grants for graduate students and postdocs; mentored awards specifically targeted to those in rehab fields; mentored awards to expose clinicians to research opportunities; and national development and training program for physical medicine and rehabilitation departments.
About 7 percent of the NCMRR budget defies simple categorization in any of the seven areas above. This group includes more basic research approaches and support of research infrastructure.
Drs. Margaret Stineman, David Gray, George Zitnay provided some individual comments on the NCMRR research portfolio and led a lively discussion involving a significant number of current and former Advisory Board members, as well as others present at the meeting. A sampling of those responses follows. Dr. Stineman also introduced a letter from fellow board member, Samantha Scolamiero, who was not able to attend this meeting. Samantha wanted to highlight issues of access and barriers that individuals with disabilities face, even in the context of attending these very Board meetings.
What is the role of the NCMRR? What is unique about the study of rehabilitation? The study of rehabilitation must include such issues as function, participation, involvement, and quality-of-life. The World Health Organization struggled with the concept of quality-of-life in the context of disability; they also took a broader view of "health". But quality-of-life is enormously complex and personal, often involving issues of satisfaction and subjective well-being.
Board members and other attendees expressed disappointment that the Center has not evolved into a freestanding NIH Institute, as has happened in a few other cases. The National Center for Complementary and Alternative Medicine grew faster than the NCMRR; their single goal was the development of evidence-based research on complementary and alternative health issues. Some felt that the NIH peer-review system should also consider a more holistic view of medicine when reviewing applications in rehabilitation. How has NCMRR influenced the research priorities of other NIH Institutes?
The summary of NCMRR research in the seven priority areas represents a significant accomplishment. But some expressed disappointment that so few grants cut across the dimensions of pathophysiology, impairment, function, disability, and limitation. The Center should also track the result of the special research initiatives. For instance, have these initiatives built a constituency? A major goal of rehabilitation remains the increasing participation of individuals with disabilities. New theories for rehabilitation science should be incorporated into the research plan for the NCMRR. At what level(s) should interventions be planned: pathophysiology, impairment, functional limitation, disability, or societal limitation?
Should NCMRR researchers have special requirements to address quality-of-life issues or the impact of their studies on the enablement/disablement process? Perhaps researchers should apply the "so what?" test. But these evaluation standards would not be appropriate for evaluating more basic research approaches.
The Center needs to minimize the artificial boundaries between basic, clinical, and applied research fields. NCMRR researcher needs to continue focusing on the efficacy and effectiveness of rehabilitative interventions, with disability measurements that go beyond the FIM (Functional Independence Measure). There is a need to adapt measurement technologies to examine dynamic changes, especially across treatments and environments. Center-supported studies have to evaluate specific outcomes and be sensitive to changes in rehabilitative practice; research should also consider the changing state of persons with disabilities. Could the NCMRR benefit from studies in other countries? There is continuing need to focus on access to health care for individuals with disabilities, such as cancer screening. In the area of training, are there enough suitably qualified scientists? How can the Center keep physicians involved in research?
The Center also needs to analyze what it has accomplished. Where can the Center make the most impact and improve the lives of persons with disabilities? The NCMRR needs to make big plans and have major goals. Progress in research is not linear; often there is a lag-time and only about 10 percent hits the mark. How can the NCMRR package rehabilitative medicine so that able-bodied individuals would appreciate the goals? Enabling America had more of a treatment and intervention orientation. An NCMRR plan needs a sense of balance between research opportunities and needs. Possibly the Center could increase communication of research advances with the disabled community.
The meeting was adjourned at 5:30 p.m., however this discussion will continue at the next Advisory Board meeting.