Since Flexner's 1910 report, the engagement of U.S. medical schools and teaching hospitals in biomedical research has transformed not only our understanding of human ailments but also our power to prevent and intervene to reduce suffering and dramatically enhance the length and quality of life. A century later, the historic Patient Protection and Affordable Care Act (HR 3590), signed into law March 23, 2010 by President Barack Obama, has brought into sharp focus the need and opportunity for an expanded continuum of biomedical research. This new generation of biomedical research must build on basic science and classical clinical investigation to place a more deliberate emphasis on patient- and population-outcome-oriented science. It must also apply science to help transform our current inefficient and expensive health care system into a more evidence-based system of effective, coordinated, safe, and patient-centered health care.
To that end, the legislation authorizes the establishment of a Patient-Centered Outcomes Research Institute (PCORI), charged with prioritizing and promoting research that emphasizes the generation of relevant, high-quality evidence to help physicians and patients make informed decisions about the patient's health care; to assist health care delivery systems in providing evidence-based, efficient, and safe health care; to ensure that evidence is obtained from and extends to ethnically diverse and geographically dispersed communities; and to develop broad prevention and screening strategies that promote health across diverse populations. The legislation identifies the National Institutes of Health (NIH) and Agency for Healthcare Research and Quality (AHRQ) as preferential partners for contract awards and as members of the governing board of the PCORI, and directs AHRQ, in consultation with NIH, to develop programs to train researchers in appropriate research and to disseminate research findings. Indeed, “science to support health care reform” has been designated as one of five NIH priorities.1,2
Much reporting about the PCORI research agenda has focused on the term “comparative effectiveness research” (CER). Although the intellectual underpinning of effectiveness research has existed for over half a century, the legislation lays out a far more expansive and inclusive role for CER. In addition to comparing therapeutic treatments, CER is intended as the rigorous application of science to evaluate and compare health outcomes, clinical effectiveness, and risks and benefits of a much broader set of interventions—from new health care delivery methods, to medical procedures and devices, to drugs and biologicals, to integrative health practice strategies, to prevention strategies. Moreover, the legislation directs that CER must include broad participation across the nation to create a representative evidence base that could help identify and address health disparities and inform decisions by patients in special population groups. The research settings for CER are also important. Unlike some traditional research, CER should extend beyond the walls of academic health centers (AHCs) and teaching hospitals to include research in partnership with community-based care settings and practice-based networks, as well as what has become known as community-engaged research, which brings into play skills in developing and maintaining long-term engagement with communities as partners in research.3
To realize the hopes for CER, the continuum of scientific inquiry must involve rigorous science to ensure the uptake and use of knowledge gained from research. The gap between the findings of clinical investigations and changes in everyday clinical practice and health decision making is not new, as noted by the former NIH National Heart, Lung, and Blood Institute director, Claude Lenfant, in 2003.4 However, the health care reform legislation and public expectations have placed a new emphasis on closing the implementation gap. The scientific domain for closing the gap (i.e., from knowledge to practice) has been variably described as dissemination, health services research, knowledge translation or transfer, implementation science, and implementation research.5–8
Irrespective of nomenclature, the “science” of implementing research findings, whether the findings emanate from CER or other health-related research, is multifaceted, multi- and interdisciplinary in the extreme, and touches on numerous variables. “Closing the gap” involves navigating human and organizational behaviors, prior training and ongoing continuing education of the relevant health professionals, the practice setting, the health care financing environment, patient and individual preferences, and the culture and quality framework. In essence, the science supporting health care reform embraces rigorous empiricism to determine what works best for whom, in what setting, under what circumstances, and how the evidence can best be translated into changes in practice and behavior, whether in individuals, populations, or health care delivery systems.
The extended continuum of scientific inquiry in the era of health care reform does not and should not abandon basic science as the foundation of new knowledge. Rather, within this continuum, a strategy is laid out to develop a rich research agenda that also embraces and breaks new ground in comparative effectiveness studies, community and population health research, and a more science-based system of health care delivery—all linked to expectations for improved health outcomes. Transitioning to a robust research environment to support this expanded agenda within AHCs will not be easy, but it will be impossible unless the research community uses as an anchor the qualities that brought us to research originally: a passion for and commitment to improving the health of patients and society through discovery. If academic medicine is to play a leading role in this 21st-century transformation of health care through research, as it did in the 20th century, we must think strategically about what we need to do to be part of the solution for transforming the nation's health care delivery systems and prevention strategies, and the changes in institutional, organizational, and individual behaviors and values required to get there. Not all institutions will engage in science called for in health care reform, but for those institutions with the interest, capacity, and resources to move forward, what is needed?
First, AHC leaders need to engage all stakeholders to look closely at the entire spectrum of their research activities, from basic science to CER to implementation science. They need to make disciplined decisions about the size, scope, focus, and alignment of their research missions with their clinical and education missions and with their goals, values, resources, and expertise.
Second, this expanded research agenda requires AHCs to forge new partnerships, including partnerships with economic, behavioral, anthropological, and managerial sciences, building not only on the skills of clinical epidemiologists and health economists but also those of decision scientists, health care educators, operations researchers, systems engineers, information technologists, behavioral psychologists, health services researchers, and other scholars. Similarly, implementation of this agenda requires stronger partnerships with clinical colleagues in nursing, pharmacy, social work, and other health professions as well as with other institutional, administrative, and community partners, and with patients themselves.
Third, institutional leaders need to demonstrate that their institutions value and reward faculty engagement in the new research agenda by publicly recognizing faculty accomplishments and by revising the merits and promotions process to align incentives with expressed values and recognize the collaborative and interdisciplinary nature of research. In addition, the president and Congress need to assure AHCs and teaching hospitals of predictable and sustained funding. The $1.1 billion distributed through the CER provisions of the Recovery Act as well as the designated appropriations authorized by the health care reform law are promising down payments, but leaders in academic medicine are understandably reluctant to leverage their own scarce resources in this area without clear, reliable sources of funding or models for sustainability.
Fourth, new scholarship requires core support, including new data resources, advanced analytical methods, information technology, and other infrastructure. The physical facilities and capital equipment requirements for such research may be modest relative to investments in biomedical science and technology development. Nonetheless, given the economic realities of resource constraints, investment in these new areas of science must be not only cost-effective but well thought out as part of a long-term strategy. While the federal government is focusing more intently on the need to develop health information technology systems that can better support a reformed health care system, the research needs of electronic medical records and other electronic information systems must also be considered as a priority.
Finally, new funding and programmatic approaches to training are needed to generate a workforce fully prepared to engage in the developing areas of effectiveness and implementation research. As a case in point, the legislation has directed AHRQ to develop training programs in CER and dissemination methodologies. Institutions need resources to develop coherent, interdisciplinary training programs that will provide a theoretical underpinning and practice-based features that encourage students to move between or among fields as appropriate; that inculcate collaborative and team-oriented skills; and that provide flexibility in accommodating diverse students with a variety of career goals.
Efforts in undertaking CER can benefit from experiences from other programs begun over recent years. Insights can be gained from the experiences of the first recipients of the NIH Clinical and Translational Science Awards, who faced some similar challenges in building cross-campus, multidisciplinary programs with robust basic science, clinical research, research training, and methodological expertise, as well as establishing meaningful community engagement. Other relevant teams of scholars have been developed at AHCs through such NIH initiatives as the HMO Research Network and AHRQ initiatives such as Evidence-Based Practice Centers, the Centers for Education & Research on Therapeutics, and the DEcIDE (Developing Evidence to Inform Decisions about Effectiveness) Network. These initiatives can serve as resources and models as leaders in academic medicine prepare to accommodate this substantial new role in the science enabling health care reform.
Sufficient investment in the full continuum for research, and in an expanded research agenda centered on patient and population outcomes, may help realize the potential of health care reform—defined both by the recent legislation and the much broader national movement envisioned by the administration and others—to generate evidence for rationally guiding decisions by physicians and patients for more efficient, high-quality, and safe health care, to extend the benefits of health to diverse and geographically dispersed communities, and to develop broad prevention and screening strategies that promote health for all Americans. Rigorous science will put decision makers in a position to choose the best interventions to transform the health care system and to use evidence to guide complex and expensive health care reform activities that otherwise might be misapplied or even doomed to fail. Once these ongoing investments have engaged AHCs in the type of sustained intellectual growth and rigorous empiricism that powered biomedical research after the Flexner Report, the nation may have another achievement equally momentous to commemorate in the next century.