Rehabilitation Research: We Should Care and We Should Act

Unique Focus of PM&R 

The focus of PM&R is human function and participation. In contrast with research that focuses on a tissue or organ (eg, a drug to improve heart function), it may not be possible to blind subjects to rehabilitation interventions, and there is no single measurement that can be used to assess human function and participation. Therefore, the breadth and complexity of rehabilitation, small sample sizes because of the need to individualize treatment, an inability to blind subjects or use a placebo control (eg, rehabilitation program or medical device), and the difficulty assigning an ethical and practical control group (ie, who not to provide with rehabilitation) have all been barriers to generating high-quality evidence.

Grading of Evidence 

These challenges, in the context of the evidence standards and methods used in many systematic reviews, have contributed to reviews of evidence in PM&R in which the authors conclude that very little or no Level 1 evidence exists [2]. Such results may reflect a dearth of well-controlled rehabilitation studies rather than a lack of intervention effectiveness. The double-blinded RCT is the most rigorous research design for assessing efficacy because of the researcher's ability to determine cause and effect while avoiding spurious causality and bias that is inherent in other research designs. A multitude of RCTs have been conducted to evaluate community and behavioral interventions in the fields of psychology, nursing, public health, and other fields, indicating that this design is possible to apply more frequently than it has been in rehabilitation research. Therefore, in testing rehabilitation interventions, efforts should be made to conduct RCTs whenever possible.

However, some research on adaptive equipment or behavioral interventions may not be feasible to either randomize or blind. There are accepted methods of controlling for differences between experimental and control groups other than an RCT (eg, mixed methods with multivariable adjustment). Therefore, in assessing rehabilitation research questions for which an RCT may not be appropriate, grading systems may need to be modified to incorporate those methods in determining levels of evidence.

Selection of Outcome Measures 

In addition to addressing selection and grading of research design, rehabilitation researchers face a challenge in selecting outcome measures that accurately reflect the genuine needs, views, and desires of people with impairments, functional limitations, or disabilities. This challenge has been discussed very thoughtfully by the Task Force on Standards of Evidence and Methods, established by the National Center for the Dissemination of Disability Research [8]. To develop better outcome measures, there is a need to examine current outcome measures and then collaborate with experienced researchers within and outside of PM&R to develop outcome measures that will better characterize aspects of function and participation of greatest interest to people with disability.

Funding 

Another challenge is a relative lack of funding for rehabilitation research, and such lower levels of funding often are inadequate for rigorous scientific inquiries that involve large or multisite studies. One reason for the lower levels of funding is the difficulty in obtaining industry sponsorship. This difficulty is attributable to the relatively low fraction of rehabilitation treatments that can be captured as intellectual property; many treatments are physical rehabilitation or behavioral therapies delivered by human beings rather than by pharmaceuticals. In addition to the decreased reliance on pharmaceuticals for rehabilitation, many of the technology companies that provide products to people with disabilities have small research budgets in comparison with pharmaceutical companies. Therefore, rehabilitation research has a greater need for public funding in comparison with other specialty areas that may be able to obtain corporate funding more easily.

Further, the difficulty obtaining funding is exacerbated by the cost of conducting certain types of rehabilitation research. Many rehabilitation therapies are personnel-intensive, making the cost of the intervention on a grant much larger than in most pharmaceutical studies in which the cost of providing a drug is minor. For example, many research grants would be insufficient to cover an additional 3 weeks of inpatient rehabilitation to assess whether a longer length of stay leads to a better outcome.

Finally, in addition to lower levels of corporate and federal funding available and the higher costs of studying rehabilitation interventions, the challenge in obtaining sufficient funding also relates to insufficient infrastructure, including trained researchers within the specialty. As discussed in this perspective, rehabilitation research infrastructure is being addressed, and there are additional opportunities to further enhance the pace of scientific development.

Research Development 

Service providers and policymakers are now joining people with disabilities and their advocates who are asking more and more frequently for objective evidence of “which therapy works?” Thus, there is a compelling need for clinical guidelines determined by evidence derived across a range of related research studies. Research should be directed to enhance the evidence base supporting best practices in clinical service delivery and health-promotion programs for persons with disabilities. The specific foci for research were well outlined in responses to the NIDRR Long-Range Plan For Fiscal Years 2010 through 2014: “Specifically, the health and function agenda at the individual level should fund research that supports the development and evaluation of new interventions, products, devices, and environmental adaptations aimed at improving the health status and functional abilities of people with a wide range of disabling conditions. These interventions should address the needs of people who are aging with disability, with particular emphasis on minimizing secondary health conditions and disabilities. These interventions should also address the unique needs of infants, children and adolescents with disabilities.” The responses further called for “research that leads to the development of the next generation of valid and reliable measures of health and functional status among people with disabilities” [11].

To generate strong evidence in these individual research areas, high-quality studies should be designed to minimize bias (threats to internal and external validity), have sufficient sample size and effect size, and assess for consistency among studies. Bias is minimized through appropriate research methodology, selection of subjects, measurement of outcomes, and interpretation of results. There are numerous sets of criteria for quality for various types of research (eg, Consolidated Standards of Reporting Trials, [CONSORT], Meta-analysis Of Observational Studies in Epidemiology [MOOSE], Quality of Reporting of Meta-analyses [QUOROM], Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA), Transparent Reporting of Evaluations with Nonrandomized Designs [TREND], Standards for Reporting of Diagnostic Accuracy [STARD], National Research Council, etc.). However, a single high-quality study is generally insufficient for determining evidence for or against a treatment and translation into practice. Therefore, there is a need to follow-up initial studies with larger studies or studies with additional populations to assess for consistency through systematic review [8]. Systematic reviews can be useful and efficient because their resulting evidence base can provide valid, reliable information regarding “what works” in a particular context. This information enables care providers to evaluate the effectiveness of procedures and practices as well as to demonstrate to payers that rehabilitation therapies are of value.

Systematic reviews minimize the time and expertise necessary to evaluate evidence in comparison with that needed to locate, appraise, and synthesize individual studies and therefore may be of particular importance. Systematic reviews require an exhaustive examination of the literature and are strengthened by the presence of studies with similar design and data collection. A registry of systematic reviews that are relevant to disability and rehabilitation research and that have been published since January 2003 can be accessed through the National Center for the Dissemination of Disability Research (http://www.ncddr.org), funded by NIDRR. Individual rehabilitation studies can be accessed through a source such as the Center for International Rehabilitation Research Information and Exchange, funded by NIDRR 1999-2009, which compiled international rehabilitation research references. All abstracts are in English regardless of the original language of publication and the search engine interface was upgraded in 2009 (http://cirrie.buffalo.edu/search/).

Although traditional research studies as well as a compilation of their findings into systematic reviews are needed to assess whether, on average, interventions result in improvement, a new paradigm is also needed. Best practice will not necessarily be achieved through care aimed at the average patient, but rather from individualized treatment based on what is best for individual patients. Such care will be most efficient in achieving positive outcomes for patients as well as in indicating to payers that they are purchasing value in rehabilitation care. More individualized evidence will require studies large enough to enable substratification.

Also, comparative effectiveness research is needed. Whether or not efficacy was sufficiently assessed before a therapy was used, there is a need to assess its effectiveness and cost in comparison with alternatives once it is part of clinical practice. Comparative effectiveness research is a subset of outcomes research. For the same diagnosis, some physicians may prescribe modalities, whereas others may prescribe medications, surgery, or monitoring. In many circumstances this disparity exists because, it is not known which is most effective. In most published studies authors research new therapies, comparing them with placebo, rather than comparing them with a current therapy. Therefore, it is unclear which therapy is most effective.

Most new drug studies are funded by the pharmaceutical industry and are undertaken to compare outcomes with those for placebo, but very few of these include a formal cost-effectiveness analysis [12]. In contrast, the majority of comparative effectiveness studies are funded by nonindustry sources. Therefore, the $1.1 billion of the American Recovery and Reinvestment Act that Congress appropriated to promote comparative effectiveness research is of great value [13]. Unless rehabilitation researchers propose to answer some of the fundamental questions facing us—which type, intensity, duration, and setting is most effective in reducing disablement—a great opportunity to advance understanding of effectiveness of treatment could be missed.

Both the Joint Commission and CMS strongly support public disclosure of health-care outcomes to enable patients to be better informed and serve as an incentive for providers to practice evidenced-based care. In 2007, CMS began to publish detailed comparative medical and surgical outcomes for each Medicare-certified hospital on the web (http://www.hospitalcompare.hhs.gov/Hospital). This was followed by the Tax Relief and Health Care Act of 2006 [14], which authorized the Physician Quality Reporting Initiative, a CMS physician-level quality report. The measures to be reported are to be “submitted by a physician specialty.” The current proposal for federal reporting of provider and institution-level clinical quality measures for 2012 would require direct report to CMS through electronic means or through the creation of databases of patient-level data stored at the state or regional level [15].

This trend will have an increasingly greater impact on rehabilitation care, and therefore it is insufficient to merely develop guidelines on the basis of expert opinion. To be held to outcome measures that recognize the special focus of rehabilitation specialists on human performance and participation, rather than measures designed by specialists with a different focus, there is a need to conduct PM&R outcomes research that will support quality rehabilitation care and programs. Deferring to others to generate the research critical to the survival of PM&R as a distinct specialty may lose sight of the unique challenges facing rehabilitation research as it relates to function and social integration. Similarly, to confine rehabilitation research to the realm of organ system- and diagnosis-driven questions may not be in the best interests of the field or our patients and may be detrimental to the future of our specialty.

Knowledge Translation 

To have a positive effect on the lives of our patients, it is not enough to stop after generating evidence from individual studies and systematic reviews. These need to be reviewed to formulate clinical practice guidelines, and those guidelines need to be translated into clinical practice, direction of systems related to disability and rehabilitation, and policy improvements. In other words, knowledge translation is intended to close the gap between the production of rehabilitation research and its use by service providers, policymakers, and people with disabilities. Resolving this gap would enable realization of the benefits from high-quality studies through improving the health, quality of life, and productivity of our patients. Therefore, beyond producing research, the second main need is to develop, implement, evaluate, and disseminate strategies to translate the results of research into rehabilitation practice.

Capacity Building (Infrastructure) 

Finally, the third main need is to increase the capacity of researchers, institutions, and the specialty as a whole to conduct high-quality disability and rehabilitation research. This will require research training programs as well as interdisciplinary collaboration to improve research networks and greater material support for rehabilitation research. This topic has been thoughtfully discussed in reports that emerged from the 2005 “Rehabilitation Medicine Summit: Building Research Capacity” [16]. In addition to these efforts, collaboration with nonphysician researchers will also be key in building rehabilitation research capacity.

Establishing a Rehabilitation Research Coordinating Center 

In addition to having a research council to advise Academy members on issues related to research, to meet the aforementioned needs, the Academy should consider sponsoring the establishment of a Rehabilitation Research Coordinating Center, a center for research training and facilitation, with capabilities to manage and analyze a central rehabilitation outcomes database and report to members. This center would efficiently meet the needs for research development, translation, and capacity building while providing all members with the means to demonstrate outcomes across provider groups, clinical settings, diagnoses, and treatments. This center would promote a critical need for rehabilitation as a specialty, the development of interdisciplinary research teams to address the needs of our patients.

To enable systematic collection of data that can support analyses of comparative effectiveness as well as other rehabilitation research while recognizing the relatively limited resources within the field of rehabilitation, one potential solution would be a data coordination and analysis center. This center would be designed to provide rehabilitation researchers with access to the biostatisticians, computer programmers, and other information technology professionals needed to conduct state-of-the-art research. For some clinical practices, this step may involve interfacing with an electronic or paper record-keeping system, whereas for other practices this could involve assistance in answering specific clinical questions. This resource would allow practitioners without time or training to participate in research that would advance their practice and our specialty.

Collection of outcomes data would only be feasible if collected as a part of the process of care, not if conducted as a process in parallel to patient care. The coordinating center would assist in this integration, which would allow the provider to focus on the objectives of care during initial work-up as well as subsequent evaluation of outcomes. This integration of data collection into clinical practice would also enhance eventual translation of findings into clinical practice. For patients with problems that may not necessitate a follow-up visit, structured phone or web-based tools could be designed to assess outcomes after appropriate management, thereby extending the ability of providers to assess their treatment outcomes without requiring additional clinic time.

There are already working models of such a coordinating center for measuring and enhancing clinical outcomes. In response to hospitals' need to facilitate the measurement and improvement of cardiovascular quality measures, the American Heart Association and the American Stroke Association established the “Get With The Guidelines” program (http://www.americanheart.org/presenter.jhtml?identifier=3045578). This program is an example of how a coordinating center can conduct research by the use of rigorous protocols, generate performance measures on the basis of that evidence, translate those findings into clinical care improvements, and provide either retrospective or concurrent quality outcomes data back to providers for submission to CMS, or comparison with other providers with similar case-mix profiles.

Thus, a rehabilitation research coordinating center would strengthen the base of evidence for clinical practice, enable development of performance measures in clinical areas most critical to PM&R, and would develop means of collecting key metrics that will encourage broad participation of physiatrists in contributing to rehabilitation outcomes databases. The outcomes research that would become possible would demonstrate the level of effectiveness of rehabilitation services in comparison with alternatives as well as advance understanding of the effects of dose, duration, and combinations of therapy.

In addition to informed data management and analysis, this center would have 2 additional integrated components—lending research expertise and resources to rehabilitation providers without the time or training to conduct research and providing rigorous research training to expand the number rehabilitation scientists. The coordinating center support team would perform a needs assessment and prospectively plan for analysis and data coordination needs at the inception of rehabilitation research projects. In this way, the center would provide expertise to ensure high-quality research. Second, the center would contain a training program for rehabilitation researchers, statisticians, computer scientists, and project coordinators. Training would be specific to the learners' needs to provide hands-on training in the practical aspects of conducting research, including study design, database creation, data management, web applications, statistical programming, project management, and team collaboration.

Establishment of such a center of expertise may be the most efficient means of collecting and analyzing the data necessary to support future coverage for treatment of rehabilitation patients. This is in line with the Institute of Medicine recommendation that those “engaged in disability research should coordinate their activities in order to reduce wasteful duplication of effort and identify neglected research issues… that will help people maintain their independence and ability to function in community life” [7].

Use of the center would also increase the productivity of physiatric research through experts enhancing the quality of studies. The training program would increase the base of rehabilitation researchers. In addition, the core resources of the center would enable unbiased data collection and analysis infrastructure that would attract additional grant funding by sources interested in supporting cost-effective, evidence-based neuromuscular and musculoskeletal care. Through pooling resources to provide required practice outcomes data, train researchers, and develop infrastructure that can attract grant funding, such a center would provide considerable savings to rehabilitation practitioners and researchers.

Building on Existing Initiatives 

There are a number of centers that already demonstrate the type of research development that can serve as examples. Effectiveness of care on specialized rehabilitation units versus general medical units has been studied for amputees [17], and a similar study of functional recovery and home discharge after stroke rehabilitation in these settings is underway. This study will include data for more than 15,000 stroke patients by the use of statistical methods to minimize bias to assess differences in the care of patients on the basis of whether acute rehabilitation was provided, in which setting, and what elements in that setting were responsible for differences (AHRQ health services grant #HS018540). However, even a smaller study of comparative effectiveness of exercise programs for low back pain can serve as a base for future research development [18].

In addition, there are several programs that have demonstrated success in expanding research capacity, particularly with regard to developing a cadre of rehabilitation researchers. The Rehabilitation Research Experience for Medical Students program enables first year medical students to complete 8-week research externships within PM&R departments and has shown early signs of success. This program began as an extension of the most successful rehabilitation research capacity building program, the Rehabilitation Medicine Scientist Training Program. Through research training, mentorship, and career development to residents and early-career physiatrists committed to developing productive careers in academic medicine and research, the Rehabilitation Medicine Scientist Training Program has produced generations of rigorously trained, extramurally competitive, and scientifically productive rehabilitation scientists.

There are published data that demonstrate that identification of medical students and residents with research interest and potential and the administration of support in faculty appointment and early-career mentorship and infrastructure is effective in expanding the number of physiatrists with successful rehabilitation research careers [19]. In follow up to the 2005 Research Summit, the Foundation for PM&R established a Research Capacity Building Initiative and a task force that recommended compilation of a database of physiatric research resources (http://www.foundationforpmr.org/research/index.aspx). This tool describes models of successful research infrastructure and makes involvement in rehabilitation research achievable at any stage of a medical career with the goal of further expanding the cadre of rehabilitation researchers as well as enhancing the quality of studies and dissemination of findings.

Finally, a considerable amount of thought has been invested in determining priorities for rehabilitation research, development of valid and reliable long-term outcome measures that adjust for case-mix, development of outcome measures for cognition and psychological function, and design of rehabilitation research [2, 3, 8, 16, 20]. In addressing the needs and overcoming the barriers described previously, rehabilitation researchers should build upon the strength of work that has already been completed. We know the needs; it is now time to roll up our sleeves and address them.