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 5-6, 2014
Dr. Nicholas LaRocca, Board Chair, called the 48th meeting of the NABMRR to order at 9:00 am. Future Board meetings will be: December 1-2, 2014; May 4-5, 2015; December 7-8, 2015.
Roger O. Smith
Marilyn Price Spivack
Ex-Officio Members Present:
|Daofen Chen, NINDS
Jerome Fleg, NHLBI
Alan Guttmacher, NICHD
Stuart Hoffman, VA
|Lyndon Joseph, NIA
Cate Miller, NIDRR
Roger Miller, NIDCD
|Ralph Nitkin, NICHD
Lynn Padgett, NCI
Lois Tully, NINR
Ex-Officio Members Absent:
|Ted A. Conway, NSF
Martin Gould, NCD
|James Panagis, NIAMS
Michael Swanson, CDC
NICHD Staff and Visitors:
|Ruth Brannon, NIDRR
Gene Civillico, FDA
Theresa Cruz, NICHD
Tinera Fobbs, NICHD
Sarah Glavin, NICHD
Margaret Glos, NIH CC
Audrey Hill, NICHD
Michael Hoffman, FDA
Adnan Jalil, APTA
Shirley Groer, VA
Rusti Johnson, NICHD
Lisa Kaeser, NICHD
John Kane, CMS
|Cille Kennedy, ASPE
Naomi Kleitman, CHNF
Anne Krey, NICHD
Susan Lin, AOTA
Michael Marge, NICHD
Mary Ellen Michel, NICHD
Stuart Moss, NICHD
Edward Nyman, FDA
Jo Pelham, CSR
Vivek Pinto, FDA
Lou Quatrano, NICHD
|Mary Rodgers, NIBIB
Mona Rowe, NICHD
Carol Sheredos, NICHD
Shawn Sherman, FDA
Nancy Shinowara, NICHD
Robert Silverstein, PPSU
Cathy Spong, NICHD
Katie Stout, MRMC
Priyanka Suno, NHLBI
Biao Tian, CSR
Nancy White, APTA
Dr. Ralph Nitkin highlighted the agenda for the meeting. Dr. LaRocca then asked each Board member and visitor to introduce him- or herself. Representatives of the Food and Drug Administration (FDA), National Institute on Disability and Rehabilitation Research (NIDRR), and Center for Medicare and Medicaid Services (CMS) were in attendance to present talks at the meeting.
Dr. LaRocca asked the Board whether they had any comments or questions on the minutes from the prior meeting, and if not, to approve of the minutes. He said that the minutes indicated how much was accomplished during that meeting. There was a motion to approve the minutes; it was seconded, and a vote to approve the minutes passed.
Report of the Acting NCMRR Director
Dr. Ralph Nitkin, Acting Director of the NCMRR, provided an update on traumatic brain injury (TBI). The NCMRR is collaborating with the National Institute of Neurological Disorders and Stroke (NINDS) and the National Football League to support clinical studies and postmortem specimen analysis. In addition, the NCMRR is supporting two R21 research grants that explore the use of specially instrumented helmets to record and assess concussion. The Center is on the steering committee for the Federal Interagency TBI Research (FITBIR) Network to improve coordination of TBI efforts across government agencies, and is represented on the steering committee for a Department of Defense program on TBI biomarkers.
Dr. Nitkin noted that the NCMRR is also collaborating with the NINDS on clinical trials for stroke rehabilitation. The NINDS funded NIH StrokeNet in January 2014 to enlist 25 regional centers across the United States in supporting first-rate trials in stroke rehabilitation. One of the first trials is a study of telerehabilitation developed by Dr. Steven Cramer with support of both the NCMRR and the NINDS. In the past, NINDS and NCMRR have co-funded major multisite stroke trials, including Locomotor Experience Applied Post-Stroke (LEAPS) and Extremity Constraint-Induced Therapy Evaluation (EXCITE).
Dr. Nitkin discussed a recent meeting at the Brookings Forum on biomedical innovation. He said attendees focused on issues of how to promote medical device innovation in the United States, specifically how to assist the medical device industry in developing new technologies. The Forum developed actionable strategies to enable government industry and other entities to create an environment conducive to innovation, and that would help underserved populations. Dr. Nitkin also highlighted the Trans-NIH Medical Devices Research Interest Group, which meets regularly to discuss innovation in medical devices, marketing strategies, and other research and translational issues.
Dr. Nitkin described an upcoming collaboration with the President's Council on Fitness, Sports, and Nutrition to sponsor a one and one-half day workshop in October 2014 to promote activity, nutrition, and health for children with disabilities. The first day would highlight the mentor-based program called I Can Do It, You Can Do It! The second day would take on more of a research focus, with specific sessions on physiological impairments and needs, nutritional support, environmental and psychosocial issues, and outcome assessment to identify research needs and opportunities to support health and fitness for children with disabilities.
Dr. Nitkin indicated that despite the constraints on NIH travel and approval, NCMRR staff attended a number of meetings across the rehabilitation research spectrum and continues to seek creative ways of engaging the research community.
Dr. Nitkin discussed the NCMRR's continued work to enact the recommendations of the Blue Ribbon Panel on Rehabilitation Research, especially with respect to increasing coordination across the NIH. With a fixed budget to support rehabilitation research, the NCMRR was working with other NIH Institutes to promote co-funding and other cooperative funding strategies to support meritorious research grants. NICHD Director Dr. Alan Guttmacher added that this reiterative process would continue as the NCMRR worked to coordinate activities across the NIH. He said that fiscal year (FY) 2014 was a transition year, and that funding strategies continued to evolve. The aim was to improve quality of rehabilitation research, reduce overlap, and ensure that no key research needs fell between respective Institute priorities. Dr. Guttmacher indicated that for FY2014, NCMRR grants would have an expandedpayline compared with grants in other NICHD programs to provide additional incentives for other Institutes and Centers (ICs) to work with the NCMRR. Dr. Nitkin indicated that the extended paylines and discretionary funding translated into more flexibility to work with other ICs, and that there was significant enthusiasm for program staff in these other ICs.
Dr. Nitkin indicated that during the current FY, the NCMRR already committed more than $2 million to co-funding activities with other ICs, with additional commitment to the out-years support of those grants. During the current year, NCMRR was cofunding 16 grants across eight other NIH ICs. In addition, the NCMRR participated in activities with agencies beyond the NIH, including the National Robotics Initiative, which connects the NCMRR, National Institute of Biomedical Imaging and Bioengineering (NIBIB), and the National Institute of Nursing Research (NINR) with the National Science Foundation.
Dr. Nitkin discussed various strategies for using discretionary funding, including additional support for training grants and career development grants (Ks) to enhance the pipeline of new rehabilitation researchers. The Center also considered targeting R01 grants in novel or underfunded areas or providing support for new investigators at a crucial stage in their research careers. The Center must also consider the balance between R03-pilot and R21-innovative research grants versus the larger R01 grants. Small business and technology transfer (SBIR/STTR) grants could be used to promote the innovative use of technologies and their translation into clinical practice with some balance between lower cost Phase I and higher cost Phase II grants.
Dr. Nitkin discussed other strategies for improving the coordination of rehabilitation research across the NIH:
- Reinvigorating the Trans-NIH Rehabilitation Coordinating Committee
- Strategic planning, especially through this trans-NIH committee
- Development of specific research initiatives across IC boundaries to address common areas of interest and potential gaps
- Cofunding and supplemental funding of current rehabilitation research activities (but without reassignment of current grants)
- Increasing investments in the Research Infrastructure Network, especially rehabilitation research opportunities that have trans-IC appeal
- NIH ICs coming together to develop workshops and symposia at appropriate national meetings and at NIH
Dr. Nitkin provided some follow-up to the previously approved initiative for pediatric orthotics to support the special needs of growing children. The proposal received concept clearance approval from the Board, and a Request for Application was issued in February 2013. Several applications were submitted by the SBIR and STTR community and five awards were being processed.
In response to previous Board discussions, Dr. Nitkin provided some data on how NCMRR rehabilitation research applications performed in NIH peer review. In FY 2014, 70 percent of NCMRR applications were assigned to six study sections in particular, five in the Center for Scientific Review and one within the NICHD. Analysis of the peer-review scores from these panels indicated that the NCMRR applications did not appear to be disadvantaged; the scores of NCMRR R01, R21, and R03 applications were spread in a manner that was similar to that of applications from related scientific disciplines. Thus, throughout these very competitive times at the NIH, NCMRR research applications performed as well as those assigned to other programs across the NIH.
Dr. Nitkin updated the Board on a recent NIH decision to relax the resubmission policy. Over the years, the NIH policy for revision of unsuccessful research applications evolved as a balance between providing researchers with sufficient opportunity to revise a given proposal and not unduly burdening the peer-review system in increasing stringent funding times. Recently, the NIH focused down to allowing only one chance to revise an unsuccessful submission (even if it represented the competitive renewal of a funded study) before that application had to be significantly retooled and resubmitted as a "new" application. However, a recent policy change would allow applicants additional opportunity (starting with the May 2014 receipt date) to revise unsuccessful applications.
Dr. Nitkin expressed thanks to Dr. Yvonne Maddox, who was retiring as Deputy Director of the Institute, for her service to NICHD. He noted that at one time she served as acting director for the NCMRR. Although retired from the NICHD, Dr. Maddox was taking on the role of acting director for the National Institute on Minority Health and Health Disparities (NIMHD) at the NIH.
Dr. Nitkin announced five new nominees for the Board, who would begin their terms in December of 2014:
- Randolph J. Nudo, Ph.D., professor in the Department of Molecular and Integrative Physiology and director of the Landon Center on Aging at the University of Kansas Medical Center
- Kevin M. Means, M.D., acting chair of Physical Medicine and Rehabilitation at the University of Arkansas for Medical Sciences
- Amy J. Bastian, Ph.D., P.T., director of the Motion Analysis Lab at Kennedy Krieger Institute and professor of neuroscience and neurology at Johns Hopkins University
- Joe Hidler, Ph.D., Chief Executive Officer of Aretech, Rehabilitation Robotics
- Michelle Camicia, M.S.N., CRRN, director of Rehabilitation Operations at Kaiser Permanente in Valllejo, California
Dr. LaRocca said he was impressed by how much happened in the last year and noted that it was one of the busiest years for NCMRR. Several other Board colleagues agreed that they were impressed by the NCMRR's progress in the past year.
NIDRR: Model Systems: A Resource for Outcomes Research
Dr. Nitkin introduced Ruth Brannon, director of the research sciences division at NIDRR, and Dr. Cate Miller, rehabilitation program specialist, to discuss the NIDRR's model systems, which he described as a great resource for researchers.
Ms. Brannon provided a brief overview of the three NIDRR Model Systems centers programs, which are focused on spinal cord injury (SCI), TBI, and burns. She said that the NIDRR has three model systems and three model systems databases, all of which track the natural history and natural outcomes for people with these conditions across the lifespan. The databases focused on long-term outcomes, including employment and other measures, which provide the NIDRR with the opportunity to contribute to evidence-based rehabilitation practices.
Ms. Brannon said the SCI Model System (SCIMS) originated first, in 1970. Some people in this system received follow-up 40 years post-injury; it now captured approximately 13 percent of all SCIs occurring in the United States. The TBI Model System (TBIMS) was established in 1987 with five centers, before increasing to 17 centers in 1988. The Burn Model System (BMS) started in 1994 with four centers. The current enrollment of the systems was almost 30,000 SCI, 12,000 TBI, and 5,000 burn individuals.
Ms. Brannon provided an overview of the requirements for Model System Centers, which must provide multidisciplinary clinical care and support center-specific and multicenter research studies. All model systems contributed to the Model Systems Knowledge Translation Center to enhance the relevance and visibility of model systems research. The national databases were designed to track the demographic characteristics of populations, types of treatment and services, cost of treatment, and other factors.
Dr. Miller described the BMS as a newer and smaller system than the other two model systems developed by NIDRR. She said BMS played a pioneering role in the promotion of the rehabilitation model within the burn injury community and also led to research on depression and return to work post-burn. The NIDRR was funding four centers and provided funding to former data centers to follow-up with patients so they can keep collecting data. Patients are followed at 1 year, 2 years, and 5 years post-injury, and then every 5 years until death.
Dr. Miller indicated that the TBIMS has the largest TBI database in the world, with connections to 16 current centers and three follow-up centers. The Centers were conducting a variety of trials, including drug trials and other interventions, relating to such issues as fatigue, headaches, and sleep. Dr. Miller added that the TBIMS National Database (NDB) had a relatively low attrition rate, with good success collecting data on enrollees over time. However, some limitations include the lack of control or comparison groups, lack of uniformity in treatment across centers, attrition in follow-up, and inability to track systematically post-acute service utilization. Dr. Miller said that at least 110 studies cite the TBIMS NDB, which has helped illuminate the epidemiology of moderate to severe TBI and the natural history of TBI outcomes and comorbidities. In partnership with the Centers for Disease Control and Prevention (CDC), the TBIMS NDB is being used to study many issues related to a moderate to severe TBI diagnosis, including unemployment, alcohol use, predictors of deterioration and improvement 5 years post-TBI, and other outcomes. The NIDRR is also partnering with the Department of Veterans Affairs in researching and tracking TBI. But perhaps the two most important missing items were a treatment registry and a medication registry.
Dr. Miller and Ms. Brannon then responded to several questions and comments from the Board. The databases' paucity of treatment data made it difficult to make statements on the comparative effectiveness of treatments. The NIDRR pushed for easier access to the model systems' data and was trying to promote for increased access to researchers and the public at large. The decision to exclude initially children younger than age 15 stemmed from the history of tracking SCIs, but the NIDRR was making a special effort to add children to the mandate of current centers. The NIDRR databases could be an excellent resource for looking at health policy issues because they include information about who has been admitted to inpatient and non-inpatient rehabilitation. The NIDRR was interested in the Affordable Care Act and its effects on rehabilitation, including how people are improving or not improving over time. The NIDRR had an evolving approach to managing databases by instituting benchmarks and trainings, creating a list of operating procedures, and refining data quality and dictionary standards and was particularly interested in considering the incorporation of additional relevant treatment variables.
The FDA Approval Process with a Focus on Medical Devices
Dr. Nitkin introduced the next presentation by FDA, which would cover regulatory pathways for medical devices. Dr. Nitkin said that it was important researchers were aware of the guidelines necessary to get medical devices approved. Dr. Michael Hoffman presented an overview of the FDA, regulatory pathways, and a specific focus on approval of medical devices. Drs. Vivek Pinto and Eugene Civillico followed up with additional information.
Dr. Hoffman explained that FDA's Center for Devices and Radiological Health (CDRH) performs medical device regulation. The CDRH employed many types of professionals to perform these assessments. A medical device is defined as an instrument or apparatus used to diagnose, cure, treat, mitigate, or prevent a condition, but that does not achieve its primary purpose through chemical means or being metabolized. Small businesses or manufacturers make most medical devices and some even hire regulatory consultants. Dr. Hoffman said that the point at which a consumer product becomes a medical device depends on how it is used and, at that point, different standards must be met. FDA's comprehensive classification system ranged from lowest risk (Class I) to highest risk (Class III). Most Class I medical devices, such as wheelchairs, only needed a registration and listing. For Class II devices, simple controls were not sufficient to provide safety, so developers need to provide a submission to FDA comparing their device to something that is already on the market before legally marketing their device. Class III devices required premarket approval (PMA) and may require clinical trials to demonstrate safety. This classification often applied to life-sustaining devices like pacemakers.
Dr. Pinto discussed the regulatory pathway and review for medical devices. A lead reviewer assembles a team that includes the diverse expertise and skills required. When FDA does not have the required expertise in house, they work with their advisory committee to recruit from additional reviewers from academia and industry. FDA aims to get all necessary input when reviewing submissions, with the goal of getting products to markets.
Dr. Pinto described premarket milestones within the review process. The FDA would like to see submissions when a company is getting ready to bench-test a device. Dr. Pinto highly encouraged sponsors to ask questions before the testing phase to save time and money. He described Q-submissions, or the three main ways sponsors and principal investigators (PIs) can obtain early advice:
- Presubmission—the sponsor/investigator request FDA's feedback
- Study Risk Determination—determining whether a study requires an Investigational Device Exemption for a marketing application
- Formal Early Collaboration Meetings
Dr. Pinto indicated that the FDA can begin discussion not just with a PI but also with an investor. Feasibility studies and pivotal studies are the two main ways sponsors and PIs study a device.
Dr. Pinto described the different regulatory pathways within FDA that devices follow when a sponsor is ready to market a submission. The three pathways are:
- 510(k) premarket notification—typically Class II devices, when a company thinks its device is similar to an already marketed device
- De Novo—new technology or device, new questions, but a low- to moderate-risk device
- PMA—generally for Class III devices, where risk is high
Dr. Pinto discussed how FDA reviews these submissions. For 510(k) devices, the sponsor has to establish equivalence to a legally marketed device and address indications for use and the safety and effectiveness of their device. The De Novo and PMA class devices have a longer regulatory pathway than 510(k) devices, and FDA reviews whether the sponsor has submitted evidence demonstrating the safety and effectiveness of the device. Dr. Pinto recommended sponsors review FDA guidance documents and engage in early communication with FDA.
Dr. Civillico discussed regulatory science research at FDA. Dr. Civillico works in the lab wing of the Center for Devices, which conducts intramural research and is how the agency prepares for the future and for new challenges in medical devices. FDA also redistributes money to high-priority research on medical devices through funding grants.
Dr. Civillico discussed how NIH can help medical device innovation. He said that many of the important things that need to happen are not incentivized; investigators needed career incentives that related to the translation process of science, not only to producing publications, as well as for data sharing. Dr. Civillico discussed new research frontiers at FDA, which include motion capture and physiology as well as things that begin life as a consumer platform and then become medical devices, such as Kinect and smartphones.
The FDA presenters then responded to questions and comments from the Board. It was noted that a developer does not necessarily need separate applications for hardware and software applications if it is one product. However, the FDA does receive applications in different stages and if a developer comes up with a new use for a device, it would be a different submission. Therapists were already using smartphones for therapy without receiving any regulatory advice, but the FDA did have guidance documents on apps. Presenters noted that there is a lot of confusion on that topic, and that area has really exploded. The FDA is fielding a lot more questions on where the line is as to which apps require FDA approval.
If a device consists of standard pieces of already-approved equipment, along with software that knits it together, and that device gets approved, the developer can make changes to a device without needing approval. There is information available about when someone does need to get approval; when the changes get more significant and could affect the safety of the device, then it would be important to discuss it with the FDA. However the FDA does understand that components change as a normal part of a product's life. Whether approval is necessary depends on the scope of change. Even if something that is purely software, such as Web-based cognitive remediation, the FDA does consider that software can be a medical device.
There are incentives to increase the development of devices targeted for children, including a lower fee associated with those devices. And Dr. Nitkin indicated that a recent Brookings Forum focused on how to incentivize the development of medical devices for underserved populations. The timeframe for receiving approvals is measured based on the number of days after FDA receives the application. For 510(k), FDA has a total of 90 days to review. PMA application timeframes usually go up to 180 days. However, the FDA does not have funds to target the development of devices for a specific problem and the FDA does not participate in the development of particular devices.
The FDA does consider data security especially in the context of devices that collect data as they work. At the Brookings conference, the issue of innovation in medical devices came up, and FDA was considered a problem; but recently FDA came out with an announcement regarding how it is trying to improve the image and the process of facilitating these applications. There was also a process that FDA was exploring for diseases and issues where there is an unmet device need and the need is not small enough to fit under the humanitarian-use pathway. FDA was looking for commentary and ideas on addressing this type of unmet need.
A CMS representative at the Board meeting indicated that the CMS does not accept FDA standards for safety and effectiveness. Unfortunately, these regulatory differences also discouraged innovation. The FDA has a group that is trying to find early interaction in device development flow to get all three parties on the same page, so that when a researcher is developing a clinical trial, they could address both standards. The presenters indicated that FDA does understand that that is an issue. As far as enforcement, the FDA has an office of compliance, which oversees manufacturing and labeling issues. On occasion, a new risk developed on a formerly safe product. Once the FDA receives notice of an adverse effect, it undertakes a process to determine how big the issue is.
There was a question about whether the FDA could provide more information about anticipatory research done in expectation of innovation and how the FDA targets its research activities. Presenters explained that this is an organic process that maybe could use a little more coordination. The FDA is evaluating research priorities; currently it relies on staff scientists going to hearings, reading papers, and proposing research activities, which are evaluated by an oversight committee. There had been a transition to being more proactive than reactive, which is starting to produce benefits. The challenge is that there are a lot of unmet needs, and the FDA has to support incentives to spur development.
Nominations for Next Chair-Elect
This meeting was Dr. LaRocca's last meeting as chair of NABMRR, with Dr. Rich Shields stepping into the position for the next term. Thus, the next chair-elect would have to be a Board member with at least 2 more years left in his or her term. Various nominations were considered, but ultimately Dr. Tony Delitto was selected as the new chair-elect.
Dr. Anne Krey, scientific review officer for the medical rehabilitation subcommittee, led the concept clearance discussion. The NCMRR was presenting an RFA on robotics and requested input from the Board. Robotics is becoming increasingly important in rehabilitation diagnoses and treatment and this RFA was to encourage research on co-robotics to enhance life, health, and the management of disability. The term "co-robot" is used for robots that have close proximity to an individual or to humans in general. Robotics is an emerging market, but this RFA would not focus on already active fields, such as exoskeletons. This RFA was designed for the small business community with a particular focus on the most translational applications. A key part of this RFA was affordable and accessible technologies. Over the last 10 years, the length of stay in rehabilitation has been dramatically cut and applications that interface with health policy would be particularly timely. Participants noted a need to bring both the FDA and CMS into this discussion; if the CMS would not reimburse for a particular intervention and/or if the FDA would not approve it, the application would be severely limited. Researchers need to make a serious effort to document outcomes for assistive and therapeutic robots. In conclusion, the Board approved support of this robotics proposal.
CMS: Understanding Boundary Conditions
Dr. Nitkin indicated that the CMS is an important player in the rehabilitation field, which is why NCMRR invited the agency to make a presentation about the issues it faces in regards to reimbursement of rehabilitation therapies. Mr. John Kane, a skilled nursing facility (SNF) program analyst at CMS made a presentation on Understanding Medicare Payments for Inpatient Rehabilitation and Skilled Nursing Facilities. His presentation covered the following four items:
- How does Medicare pay for inpatient rehabilitation facility (IRF) and SNF services?
- What types of beneficiaries typically receive these services?
- What is the connection between payments and therapy service delivery? How do CMS's payment policies for these services translate into what services facilities provide?
- What can be done to ensure providers give beneficiaries the highest quality of care?
Mr. Kane described the basic payment structure for IRFs. The CMS pays a per-discharge payment rate for patients at IRFs, which is adjusted based on the primary reason the person is in the IRF. For a facility to be classified as an IRF by CMS, 60 percent of all cases that are admitted must be for one or more of 13 selected conditions, which include spinal cord conditions, burns, arthritis, strokes, and other conditions. Other factors such as facility location affect the CMS payment rate.
Mr. Kane added that the average IRF patient:
- Can benefit from inpatient stay and an interdisciplinary team approach.
- Requires and can benefit from an intensive rehab therapy program, which is usually 15 hours per week.
- Is typically admitted for stroke, spinal cord, congenital deformity, amputation, brain injury, and several other conditions.
Mr. Kane described the basic payment structure for SNFs as well as the typical SNF patient. Medicare Part A provides an "extended care" benefit, when the beneficiary is admitted to the SNF after no less than a 3-day medically necessary hospital stay. This coverage is limited to a maximum of 100 days per benefit period, and the patient must receive skilled care on a daily basis. CMS makes a global per diem payment that includes all costs of covered SNF services per patient, whereas CMS pays INFs per discharge.
Mr. Kane explained that the payment rate to an SNF depends on resident classification into one of 66 Resource Utilization Groups (RUGs); there are 23 rehab RUGs. The rehab RUGs fall into one of five categories, primarily based on the number of therapy minutes, therapy disciplines, and days a patient receives therapy. Those 23 rehab RUGs fall into ultra-high, very high, high, medium, and low minute classifications; to get into the ultra-high group, a patient must receive at least 720 therapy minutes. Each therapy minute threshold serves as a hard marker for which therapy category applies to a particular patient in terms of payment rate. The average SNF resident needs short-term skilled care on an inpatient basis following a hospital stay. Mr. Kane said SNF residents tend to be an older population; the average age is 79 years and typically patients are admitted following joint replacement, septicemia, kidney infection or urinary tract infection, or pneumonia.
Mr. Kane noted a correlation between the design of the payment system and the type/amount of services delivered to beneficiaries by facilities. The differences between the SNF payment system (per diem, resource-based) and the IRF payment system (per discharge, characteristic-based) may partially explain differences in care delivery. In SNF, if a facility provides more therapy, it receives a greater payment from CMS. Mr. Kane showed a chart that illustrated the number of therapy minutes received by patients. Often, a facility provided the minimum therapy minutes for each payment threshold, but no more. He said the majority of SNF residents land in the "very high" or "ultra high" rehab categories. Mr. Kane described how some CMS payment policy changes, like reimbursement based on therapist-to-patient ratio, have also corresponded with changes in facility practice.
Given the connection between payment system and service delivery, Mr. Kane discussed what CMS could do to ensure providers give beneficiaries the highest quality of care. He said that CMS is building quality measures and reporting mechanisms for IRF and SNF payment systems. Additionally, he said that quality measures must focus on functional status, in addition to clinical metrics. In addition to quality measurements that CMS uses like number of infections, CMS is working to measure functional outcomes as a result of therapy in IRF and SNF settings. Future quality measures in development include functional outcomes, falls, and readmissions.
Mr. Kane said that CMS is starting to make progress in developing these measures, but measuring functional outcomes is a difficult task. He provided an overview of website resources about CMS. He also provided information on CMS's Innovation Center, which includes a site where users can share ideas. The Innovation Center is working to improve beneficiaries' lives without additional costs, and is considering a whole host of different payment models.
Mr. Kane then responded to various questions from the Board. Functional outcomes depend on the setting; for IRFs, CMS considers measures such as a patient's ability to walk certain distances, transfer to the toilet, or dress him or herself. However, the CMS would like to try to develop measures that are homogenized so it can track people's functional status across settings. The CMS does consider cognitive function, including the FIM™ measurement tool, which includes cognitive domains. However, the Board indicated that in practice it can still be very difficult to get support for cognitive therapies, especially for younger patients who are dealing with TBI or stroke.
Because private insurance often follows CMS rules, CMS decisions even affect patients who are not eligible for Medicare and Medicaid. The Board noted that the government is spending millions of dollars on acute care, but is not creating the systems for the younger populations who need such services. While there is a need for systems of care, people need different services at different times. A lot of the reimbursement policy is being driven by Congress; however, the CMS does want to harmonize metrics, especially as people move from one setting to another. Mr. Kane indicated that the CMS would be very open to suggestions on how this could be accomplished. The Board indicated that there is still not strong support for functional measures nor the application of certain measures that might ensure quality services. Researchers do not have good data on outcomes related to the intensity of physical therapy, speech therapy, and other therapies, but there is really strong evidence for clinical improvement. However, practitioners are not able to provide therapy, even if it is supported by good evidence, because no one will pay for it. There has been particular interest in the impact of short-term, very intense rehabilitation.
The Board suggested that the fundamental problem is that the CMS is looking at quantity of therapy and not the quality of therapy. An analysis based only on rehabilitation therapy minutes, will miss the nuances of speech, physical, and occupational therapy to target the patient's needs. The Board asked if there was a major trial to show a particular therapy is effective, would the CMS work with researchers who want to evaluate a therapy and compare it to therapies that are already in use? Mr. Kane indicated that the CMS's Innovation Center is probably the best proxy for working with on-the-ground researchers and trials. A new therapy has to improve quality without increasing cost. If something could improve quality without increasing cost, the Innovation Center could work with the new approach. The Innovation Center is the nexus in terms of testing new payment methods. The Innovation Center will test certain models, sometimes with states (waivers have to go through certain states). On the Medicare side, the CMS is bound by statute in some aspects, but the Innovation Center has some flexibility. It can test new approaches and particular patient populations. In addition, Pioneer Accountable Care Organizations (ACOs) are popping up more frequently around the country and have certain waivers. But the shift from investigation to policy depends on the outcomes. CMS does not want to ramp up prematurely following preliminary positives, nor does it want to get stuck in the investigation phase.
The Board appreciated the opportunity to discuss these issues with the CMS representative. A presentation earlier in the morning highlighted how policy changes could be directly correlated with changes in rehabilitation outcomes, so perhaps the CMS should review how policy changes across decades may have impacted outcomes. Mr. Kane indicated that the CMS tends to have a forward focus, but that there may be opportunity for retrospective analyses in reviewing certain design metrics.
NCMRR Research Investments: New Model for Coordination
Dr. Nitkin indicated that it was a year in transition for the NCMRR, especially as it took on the Blue Ribbon Panel challenge to increase coordination and support for rehabilitation research across the NIH. Through discussions with other ICs, the NCMRR explored various opportunities and encouraged the Board to discuss the following:
- Reinvigorating the Trans-NIH Rehabilitation Coordinating Committee
- Strategic planning, especially through this Trans-NIH committee
- Development of specific research initiatives that cross IC boundaries to address areas of common interest as well as potential gaps
- Co-funding and supplemental funding of rehab research proposals (but without reassignment of current grants away from the NICHD)
- Increasing investments in the rehabilitation research infrastructure network, especially opportunities that have trans-IC appeal (The current network of seven centers has been very effective and valued by the research community.)
- NIH ICs collaborating to develop workshops and symposia at appropriate national meetings and at the NIH, or to have yearly symposia at the NIH and archived webinars, which could highlight key topics in rehabilitation research
Dr. Nitkin asked whether these approaches were a good use of resources.
The Board asked how the NCMRR would actually lead these coordination efforts. It can be very difficult to get NIH ICs to change—budgets are getting smaller, and every one of those research activities require a huge amount of time invested. This is a critical issue, and people are committed to it, but there has to be some money invested in a specific position within the NCMRR to take care of coordination. Dr. Guttmacher responded that part of the issue is that the search for a permanent director for the NCMRR has not been completed, and the NICHD would like the new director to figure out how to do staffing. He indicated that the new director would likely lead the coordinating role.
The Board highlighted the importance of strategic planning; this would drive a lot of the agenda. Dr. Nitkin suggested that one model envisioned for strategic planning is to have the Trans-NIH Rehabilitation Research Committee do its best to develop a plan and then invite comment from the rehabilitation community. Another possible model would be to have formal workshops and engage the research and advocacy communities in the development of a plan. Dr. Guttmacher assured the Board that any plan would come back them for comment. Dr. Nitkin added that through discussions with other ICs, the NCMRR could better understand needs are in the field; not just a survey of the rehabilitation research field, but where the gaps are, how the NIH can really help, and where the NCMRR should invest. Good applications are going to be submitted regardless, but broader discussions would help NCMRR exert the best judgment over what research is underway and what some research priorities should be.
One member of the Board indicated that with the NICHD strategy of having rehabilitation researchers seek primary assignment to other ICs so that the NCMRR could maximize co-funding opportunities (as discussed in the previous Board meeting), a few applicants were having trouble finding an IC interested in their application. However, Dr. Nitkin was quick to indicate that the NICHD had shifted back to a policy where the NCMRR would take primary assignment on appropriate rehabilitation research applications. The NCMRR was encouraged to consider a dividing-and-conquering approach by bringing together researchers in certain fields, such as neurology, to help refine how program officers support these topics. The Board also encouraged the NCMRR to work with the Trans-NIH Rehabilitation Research Committee to enhance partnerships and getting buy-in for these activities over the long haul. Dr. Guttmacher indicated that the Committee consisted of representatives from specific ICs, and that it is now being given substantive work and coordination tasks, which is reengaging the group. Dr. Nitkin added that reinvigorating the Trans-NIH Committee and NCMRR having its own budget could have significant impact on the field. Dr. Guttmacher agreed that in developing strategic planning, it was important to get real buy-in from different ICs but also to pay attention to the areas that seem to fall between ICs. The Board questioned whether the Trans-NIH Committee is the right group to identify the areas that are missing. Dr. Nitkin admitted that they might not be the people to identify the gap, perhaps it would be more appropriate for the NCMRR and its advisors to do that, but that the Trans-NIH Committee could then help develop strategies and incentivize it.
In reference to the renewal of the rehabilitation research networks, the Board asked whether there were funds set aside for these networks. Dr. Nitkin said that the NCMRR viewed them as an important investment, adding that the networks were receiving more interest from other ICs, and that the NINDS and NIBIB would continue to contributed funding. But ultimately, funding would depend on the quality of applications that come in and what research domains they cover.
On another note, the Board indicated that the length of stay is getting shorter and shorter in rehabilitation facilities, and that rehabilitation facilities used to take in sicker patients. Now acute rehabilitation facilities are getting patients with more moderate issues. With CMS providing rehabilitation for less acute patients in environments that are not good at collecting data, outcomes were not going to come up as positive. There was also discussion on whether there would be appropriate data collected on the quality of care. With pressures to optimize the delivery of care, it is not clear that already-burdened therapists would have the neither time nor incentives to help support data collection even if it had to potential to improve outcomes. The Board also asked whether there was sufficient focus on the "plasticity" of a patient and providing access to services that could better serve their goals and needs. Dr. Nitkin indicated that one of the NCMRR-funded research infrastructure centers focuses on analysis of large data sets and health services research, but that the NCMRR would continue to focus on outcomes that impact people's lives.
The Board also encouraged the NCMRR to work with other ICs to support research symposia and collaborations with national scientific committees to highlight research opportunities. Dr. Nitkin agreed. The Board also indicated the need to encourage advocacy organizations to focus on rehabilitation. Patients need access to effective services in order to improve their lives.
Report of the NICHD Director
Dr. Alan Guttmacher thanked Board members for their service on the Advisory Board and stated that NICHD does consider their advice seriously.
Dr. Guttmacher provided an update on personnel changes across the NIH. Dr. Philip E. Bourne, formerly of the University of California, San Diego, has joined the NIH as the first Associate Director for Data Science, which would help NIH address issues regarding big data across the agency. NIH hired Dr. Hannah Valantine as the first Chief Officer for Scientific Workforce Diversity, which is within the Office of the NIH Director. Dr. Valantine is charged with helping the NIH do a better job supporting diversity in the United States scientific workforce and especially here at the NIH. Dr. Guttmacher also indicated that Dr. Yvonne Maddox has moved from the NICHD to serve as Acting Director at the National Institute on Minority Health and Health Disparities.
Dr. Guttmacher noted that the Dalai Lama visited the NIH to give the annual J. Edward Rall Cultural Lecture. He visited an NIH rehabilitation research facility, where he demonstrated a natural aptitude for interacting with sick children and their siblings. In addition, the Dalai Lama has a particular interest in brain research.
Dr. Guttmacher highlighted some changes in the NIH biosketch format to better evaluate a research applicant's actual contributions versus their academic and research pedigree. One proposal was to replace the list of 15 peer-reviewed publications with a narrative of the applicant's five most significant contributions. A trial of the new format is underway, and later this fall, the final biosketch format will be extended to all incoming applications. Dr. Guttmacher also indicated that the NIH is working to maximize the use of core research facilities, including workshops on how cores can be shared more effectively and improved coordination of core activities supported by NIH Institutes.
Dr. Guttmacher addressed recent discussions about the failure to reproduce research findings. He mentioned the recent piece in Nature by Drs. Francis S. Collins and Lawrence A. Tabak about NIH plans to enhance reproducibility. He said the feedback on the commentary has been mostly supportive, and that there is a need for NIH to support replication studies and incentivize scientists to perform such work. The NIH has held a number of meetings on transparency and reproducibility, and is considering workshops with journal editors, professional societies, and funders, along with separate workshops envisioned for industry. NIH is also working to develop training resources on experimental design.
Dr. Guttmacher provided an update on news from within the Institute. The NICHD is conducting a national search for a Deputy Director and hopes to have the position filled by early fall. The NCMRR Director position search is underway; Board members are encouraged to identify people in the rehabilitation community who might be appropriate for the position.
Dr. Guttmacher highlighted two recent rehabilitation-related NICHD meetings, the Pediatric Trauma and Critical Illness Branch Strategic Planning Workshop and the Unintentional Injury Prevention: Research Needs meeting, which focused on sports-related head injuries and household injuries. Dr. Guttmacher indicated that the NICHD would be relocating in a year to 6710 Rockledge Drive in Bethesda, Maryland, which is an accessible building.
Dr. Guttmacher provided an overview of legislative updates. In January, the FY 2014 the NIH budget was established: an increase over FY 2013, but less than pre-sequestration FY 2012 budget. The NICHD received about $1.28 billion or about 2.9% more than FY 2013. The December 2013 budget agreement set funding ceilings through FY 2015, and there is no threat of sequester through FY 2015. Dr. Guttmacher said that on March 13, the U.S. House of Representatives Subcommittee on Commerce, Manufacturing, and Trade held a hearing on sports-related concussions. He also described the Gabriella Miller Kids First Research Act, which uses money from the presidential "check-off funds" on pediatric research. This Act also authorizes, but does not appropriate, funds for a 10-year pediatric research initiative fund.
At this point, Dr. Guttmacher engaged the Board in discussion. The Board asked about discussion of potential legislation on rehabilitation and disabilities issues that that authorizes activities or programs without additional appropriations. Dr. Guttmacher responded that many programs or activities may be authorized, without appropriations. However, the legislative activities do provide some indication of interest in women and gender health issues as well as disabilities. Dr. Guttmacher acknowledged the important role of NIH staff in enabling the agency to continue to manage and cope with tighter and more restricted funding . Dr. Guttmacher encouraged suggestions about how to reward and recognize the contributions of people who come to work at NIH. Dr. Guttmacher also encouraged the Board to continue to give guidance to the NCMRR on how the Center functions and the overall needs of the field.
Acknowledgement of Retiring Board Member and Comments
Dr. Nitkin noted that four NABMRR members had finished their term with the Board as of this meeting: David Good, Carol Espy-Wilson, Gary Smith, and Marilyn Hamilton.
Summary of Monday's Discussions with NIDRR, FDA, and CMS
The second day of the Board meeting began with a recap of discussions from the previous day by NIDDR, FDA, and CMS.
The NIDDR is an agency within the Department of Education that supports model systems in areas such as SCI, TBI, and burns to conduct longitudinal studies and contribute to improving care of patients through evidence-based medicine. These systems catalogue patient demographics, cause and severity of injury, diagnosis, services, costs, and other aspects. The model systems have evolved over time and tried to address some of their limitations.
The FDA Center for Devices and Radiological Health targets the regulation of medical devices; their mission is to bring safe and effective devices to the market. Most manufacturers are small business and they have to register with FDA. Developers need incentives to address underserved populations. The FDA is trying to accelerate the regulatory process, however the CMS has different standards concerning medical devices with more of a focus on efficacy. A major challenge for the FDA is anticipating intramural research frontiers. The FDA presentation was a great start for improved relationships with NIH research activities. Perhaps, the NCMRR could help to catalyze these discussions and even coordinate regulatory issues with CMS to improve access to assistive technologies especially in the home environment.
The CMS provides support for inpatient rehabilitation and skilled nursing facilities. Service patterns tend to follow the payment design. Providers have to work with the system to provide access to the highest quality care. They need to implement quality reporting and measuring, and develop quality metrics based on functional outcomes. In recent years, there has been discussion of evidence-based funding. This sounds like a good idea, however lack of research is being used to cut off reimbursement for certain services, which may be beneficial for the patients. The Board should try to understand more about the CMS Innovation Center, which drives adoption of new approaches, and understand the role of payment systems that better account for quality of services. The field is stuck with legacy instruments, such as the FIM™, which need to be updated and optimized; however, the integration of newer outcome measures such as Patient-Reported Outcomes Measurement Information System (PROMIS) could help. There may also be a need to create new ways to aggregate different instruments and measures.
Orchestrating the Brain in Health and Disease: Waking and Sleep
Dr. Edgar Garcia-Rill offered a research presentation titled Brain Music: Waking and Sleep in Health and Disease. He is the director of the Center for Translational Neuroscience at the University of Arkansas for Medical Sciences. Dr. Garcia-Rill's presentation helped to address the question of whether someone is cognizant based on their stage of sleep or being awake. He explained that the brain works more like an orchestra, through waveforms, and that music is akin to the machine language that drives the brain. His research focused on the intrinsic properties of cells and how they are generated.
Through funding from NINDS and National Institute of General Medical Sciences, his lab examined the brain's workings during the three states of the brain: awake, REM sleep, and non-REM sleep. He discussed the amplitude and frequencies of brain waves, as reflected through electroencephalogram patterns, during each of these states. Two important elements in the large-scale interactions of neurons are coherence and frequency. This type of analysis also provides information about how the brain works when it is affected by TBI, Alzheimer's disease, and mental illness like schizophrenia. Dr. Garcia-Rill identified several clinical implications that resulted from this research.
New Business and Agenda for Next Meeting
At this point, chair-elect Dr. Richard Shields took over from Dr. LaRocca. He noted that by the next Board meeting, there would be probably be a new NCMRR director. The Board discussed the following issues as items to consider for the December meeting or for future meetings:
- Create some Board subcommittees to focus on issues such as advocacy perspectives and research opportunities.
- Invite the Trans-NIH Rehabilitation Coordinating Committee to present to NABMRR, including about their work on a strategic plan.
- Have NCMRR present on NCMRR and NIH's rehab-related portfolios.
- Develop more proactive coordination to help influence ICs' rehabilitation research and enhance work across ICs.
- Invite the CMS Innovation Center to present to NABMRR.
- Invite FDA to walk NABMRR through a hypothetical medical device regulatory process, from the start to the end of approval.
- Consider NCMRR coordination of another rehab research summit.
- Work with NIDRR to identify ways to collaborate on model systems data research or to help make data sets more accessible to researchers.
- Invite CDC to present on their vital sessions newsletter, which recently focused on adults with disabilities, or about exercise and disability.
- Send notes to FDA, CMS, and NIDRR thanking them for their presentations.
- Receive an update on the NCMRR-funded research networks and their standing.
The meeting adjourned at 11:45 a.m.