Clancy, Carolyn M. MD
Through their extensive research output, U.S. academic health centers (AHCs) have ensured that advances in biomedical and clinical science constitute the backbone of medical education. Those same advances—the envy of the world—have shaped a clinical landscape in which patients and clinicians must frequently choose among multiple options for diagnosis, prevention, treatment, and palliation. Routine practice often relies, by default, more on best guesses and trial-and-error medicine. What has been missing is valid, easily accessible information to inform daily clinical decisions. The promise of patient-centered outcomes research (PCOR) is reliable scientific information that will enhance clinical precision at the point of care. Such evidence-based information is an essential component of the personalized, patient-centered care that all Americans expect and deserve.
PCOR, also known as comparative effectiveness research, examines which precise medications, treatments, devices, or delivery systems work best for which patients under which circumstances. This type of research is unique in that it (1) incorporates outcomes that matter to and are reported by patients and their families (e.g., improvement in symptoms, costs and side effects of a treatment, impact on ability to work or perform usual activities), (2) entails longitudinal follow-up of patients, and (3) engages diverse stakeholders, from insurers to policy makers, in the entire research process.
In short, the evidence-based comparative information that results from PCOR cannot only close the gap between clinicians' aspirations for providing the best possible clinical care and the daily reality of dealing with clinical uncertainty but can also reconcile the outputs of traditional biomedical and clinical science and the evidence needed to identify the best option for a specific clinical situation. Indeed, the definition of comparative effectiveness research used by the Federal Coordinating Council for Comparative Effectiveness Research (FCCCER) emphasizes the importance of research that is “(directly) responsive to the needs of clinicians and patients.”1
Although this type of research is not new, several recent federal laws have reinvigorated PCOR. These laws provide support for, and help establish as an essential component of universal access to high-quality care, what might be labeled “precision science”—that is, information to guide decisions regarding which approach is the best fit for an individual's needs, preferences, and values. The Medicare Modernization Act of 2003 provided new authority to the Agency for Healthcare Research and Quality (AHRQ) to support research and syntheses focused on topics of highest priority to populations served by the Medicare, Medicaid, and Children's Health Insurance Programs. That statute directed a broad, transparent approach to setting priorities for this research and included clear language regarding the importance of making the research findings available and accessible to multiple audiences, including clinicians, patients, and policy makers. The American Recovery and Reinvestment Act of 2009 (ARRA) provided $1.1 billion to the AHRQ, the National Institutes of Health, and the Department of Health and Human Services for comparative effectiveness research and similarly encouraged transparent, collaborative priority setting and rapid, effective dissemination and translation.
Opportunities and Challenges of PCOR
In this issue, several articles describe the opportunities for AHCs to engage in this research, and some detail the important challenges that PCOR entails. The article by Rich and colleagues2 highlights the achievements of AHCs in biomedical and clinical innovations while also articulating the imperative to establish effective multidisciplinary collaborations and vibrant partnerships with communities in order to advance comparative effectiveness research. Moreover, these authors note the need for PCOR to ameliorate the stubborn persistence of clinical practice variations, and they strike a note of humility with respect to the ongoing challenge of incorporating evidence-based practice, a tenet of PCOR, into medical education.2
VanLare and colleagues3 reinforce similar themes. Organized around the framework for comparative effectiveness research employed by the FCCCER, their observations address the specific dimensions of PCOR: data infrastructure, human capital, research and translation, and dissemination. They use specific examples from two centers—Mount Sinai (New York, New York) and Cincinnati (Ohio) Children's—to reinforce their premise that this new opportunity can be adapted to the unique assets of individual AHCs.3 Zerzan and colleagues4 focus on the potential relevance of PCOR to state Medicaid programs, and they describe recent efforts to apply scientific research to common challenges confronting policy makers in an environment of constrained fiscal resources. The salience of timely, relevant information for policy makers and the implications for academic researchers, especially for dissemination, receive special attention.4
Iribarne and colleagues'5 article describes clinical economic evaluation as an inseparable discipline vital to comparative effectiveness, and the authors note the potential foundation for comparative effectiveness research in those AHCs that enjoy ongoing Clinical and Translational Science Awards programs. Finally, McGaghie and colleagues6 emphasize the importance of a competent health professional workforce to the application of comparative effectiveness research for achieving quality, patient-centered care. These authors also present the results of a novel meta-analysis; they found that simulation-based education programs with deliberate practice yield better results than traditional medical education programs in terms of fostering the requisite competencies to achieve precision in the application of scientific knowledge to patient care.6
PCOR Differs From Biomedical and Clinical Research
The overall tenor of these articles reflects considerable optimism regarding a new enterprise; the authors devote less discussion to specific dimensions of this new research and to the attendant challenges it poses for the research enterprise of AHCs. Both the adventures and challenges of PCOR stem from the way it differs from biomedical and clinical research in governance, methods, funding mechanisms, and impact on academic culture.
A substantial and growing proportion of the public fully expect to be engaged in decisions about their care. Individuals bringing online printouts to medical encounters is far from a novel phenomenon. Similarly, the media attention devoted to research advances has inspired considerable public interest in the research enterprise itself. As part of ARRA funding, both the Institute of Medicine (IOM) and the FCCCER have engaged stakeholders in articulating priorities and specific topics for research. Since 2005, AHRQ's program has benefited enormously from a panel of diverse stakeholders, including patient advocates, physicians, nurses, payers, and policy makers, who provide input at every step of the process—from setting priorities and clarifying key questions to reviewing drafts of reports and being available for public comment. Transparency and stakeholder governance are essential components of the new Patient-Centered Outcomes Research Institute, established in the legislation of the Patient Protection and Affordable Care Act. This broad participation and openness are in stark contrast to the traditional, single-investigator-driven approach to research at many AHCs.
Relevance to diverse populations
As noted, several articles in this issue of Academic Medicine note the importance of collaborative partnerships with communities to ensure that any research undertaken addresses the needs of patient populations who are not traditionally well represented in research, including members of racial and ethnic minorities, individuals with multiple comorbidities, the elderly, children, and those who may be geographically isolated. The list of successful examples of this type of collaboration is growing (a good example is the ongoing partnership between Columbia University [New York, New York] and the nearby Washington Heights neighborhood), but it is not yet clear that there is sufficient experience to scale these partnerships rapidly. In addition, building the trust required for sustainable collaborations can be labor-intensive and time-consuming; some may perceive the time required to build and sustain these relationships as interfering with the scholarly productivity required for promotion and tenure. Moreover, many communities will understandably expect to be co-owners as well as coproducers of subsequent research findings. They will want—and they deserve—a voice in publications, a right to dissemination, and possibly a share in the intellectual property rights and other assets derived from the research.
Recent reports—as well as some of the articles in this issue—clearly acknowledge the need for a clinical effectiveness research workforce. What is less evident is how researchers now in training will succeed in the face of traditional academic incentives for promotion and tenure. For example, contracts—rather than grants—may support some portion of PCOR, even though contracts are sometimes thought to be less attractive to academic institutions. In addition, the core processes of transparency and stakeholder engagement, which bring vital insights to this enterprise, may require alternative metrics for scholarly contributions. Also, effective dissemination and translation will likely require close collaboration with colleagues working in clinical medicine, quality improvement, and clinical operations, so the path to academic advancement for these individuals may need to be redefined.
Dissemination and translation
In 2007, Lee and colleagues7 published a short article noting that the practice of prescribing beta-blockers to eligible patients who have recently had a heart attack has become so routine that this measure is no longer counted in how health plans are scored by the National Committee for Quality Assurance. This superb example of universal excellence was not observed until 25 years after the landmark clinical trials that demonstrated reductions in subsequent mortality.7 Ten years previously, in 1997, Soumerai and colleagues8 had applied epidemiological methods (i.e., Cox proportional hazards modeling) to demonstrate that heart attack patients older than those enrolled in the original trials who received beta-blockers enjoyed the same mortality reduction. Their study is a clear example of observational and other methods that investigators can apply to complementing and extending findings to unique populations such as the elderly.8
To accelerate the delivery of high-quality, patient-centered care, researchers who engage in PCOR must rapidly disseminate and implement their findings—tasks often delegated vaguely to the clinical enterprise. In this issue, VanLare and colleagues3 note the potential to link the research output with clinical operations, an exciting concept. Indeed, previous research has shown that a common theme among academic institutions with the best performance is integration of academic and clinical leadership at the highest levels of the institution.9 External demands for demonstrated performance in quality and safety should motivate more—rather than less—integration, but the downstream effects of these demands on academic culture have received little attention. What might the career paths of investigators who apply their results to clinical care improvement look like? Metrics for promotion and tenure and explicit strategies for mentoring and enhancing success are clearly needed. Kirch's10 concept of health innovation zones offers another means for AHCs to take leadership positions in health care. Both operational and cultural implications warrant urgent attention.
The sphere of dissemination and translation itself offers enormous opportunities. Quantifying the time from publication of research results to routine practice is conceptually straightforward (though obtaining timely information regarding the state of practice remains challenging). Creating accelerated paths forward, however, is a virtual frontier. Most professional and lay communities now take full advantage of electronic media, through such means as Web postings, but, in general, the traditional approaches to the dissemination of health research (e.g., conduct study, publish report in peer-reviewed journal, issue press release, hope that knowledge alone impacts practice directly) are relatively weak. Researchers should explore strategies that build on advances in and broader adoption of health information and communication technologies, including social networking, in order to accelerate customized information delivery at the point of decision making. Such strategies will require the efforts of communication scholars and cognitive scientists as well as journalists and bloggers. Those involved in traditional academic processes, such as peer review and analytic methodology, must adapt to the swift pace of new, digital knowledge-communication modes, learning to rapidly apply the lessons that they discover as a by-product of delivering care. In short, the delivery system can serve both as a platform for discovery and as a means for rapid translation of research into practice. It is not remotely premature to anticipate this future.
The New Landscape
The boundaries of PCOR are evolving: Is the ultimate goal better health care or better health—or both? Both the IOM and the FCCCER concluded independently that the focus of research should include strategies and approaches for improving care delivery and, at least by implication, for also improving community-based efforts that address the root causes of disease and illness. The burden of suffering and the costs attributable to chronic illnesses, along with the incremental progress to date of improving care and outcomes, suggest that effective partnerships between care delivery organizations and community partners (including but not limited to public health entities) should be a high priority for AHCs.
As PCOR matures, there are and will continue to be occasions for sharing opportunities—as well as challenges—with the well-established areas of clinical, health services, and public health research. PCOR, which uniquely includes longitudinal follow-up of patients, explicit incorporation of patient-reported outcomes, and dynamic interactions between researchers and all stakeholders, offers distinctive practice paradigms that complement those of traditional models.
The opportunities to close the gaps between what physicians and medical scientists do and what they know, to support clinicians and patients who are working together in order to rapidly identify the best option for a unique individual, and to lead the way in addressing barriers to achieving personalized, patient-centered care should engage key members of AHCs in comparative effectiveness research, so that these institutions become a vital link connecting biomedical innovation and its precision application in diverse populations.
The author gratefully acknowledges the counsel of Jean Slutsky, Director, Center for Outcomes and Evidence, Agency for Healthcare Research and Quality.
The opinions expressed here are the author's and do not represent official policy of the Department of Health and Human Services or the Agency for Healthcare Research and Quality.