Rich, Eugene C. MD; Bonham, Ann C. PhD; Kirch, Darrell G. MD
In 2007, when the Congressional Budget Office (CBO) released an ominous report on the growth of health care costs,1 then-CBO-director Peter Orszag termed this the nation's greatest fiscal challenge. He noted that central to this cost growth problem was that “relatively little rigorous evidence is available about which treatments work best for which patients.”2 Dr. Orszag proposed comparative effectiveness research (CER) as providing the leading opportunity “to constrain health care costs without incurring adverse health consequences.” A broad range of stakeholders joined in calling for expanded public investment in CER, hoping that more evidence-based clinician and patient decisions, guided by CER findings, could allow the United States to provide universal access to high-quality health care while simultaneously controlling unsustainable cost growth.3–8 With Dr. Orszag as the first director of the Obama Administration's Office of Management and Budget, CER became an element of the White House health reform agenda.
The last four years have seen a variety of legislative and administration actions relevant to expanding support for CER, culminating in the provisions contained within the landmark Patient Protection and Affordable Care Act (PPACA) of 2010.9 Although health care reform has many elements that resonate with the multifaceted social mission of medical schools and teaching hospitals, this new emphasis on the use of research evidence to help transform the health care system perhaps most directly engages their intellectual core. Thus, CER offers a great opportunity, albeit with some considerable challenges, for academic medicine to play a central role in health care reform. In this article, we will summarize these issues and reflect on their implications for both academic medicine and CER.
Past Federal Support for CER
Until 2009, explicit federal research support for CER had been quite modest.10–13 The American Recovery and Reinvestment Act of 2009 (ARRA) dramatically increased funding to federal research agencies for CER.14 The ARRA directed $1.1 billion primarily through the National Institutes of Health (NIH) and the Agency for Healthcare Research and Quality (AHRQ) but also through other federal agencies and offices to build the nation's capacity for this research.
The ARRA's CER initiative illustrated both the challenges as well as the possibilities of CER for academic medicine. It was during the ARRA debate that 1990s concerns over federal investments in CER reemerged. Some argued that the cost-control imperative on executive branch leadership would be so great that expensive interventions would not be “cost-effective,” thereby leading to denial of access to important treatments.15–17 Some politicians and pundits likened the ARRA's Federal Coordinating Council for Comparative Effectiveness Research (FCCCER) to a “rationing board,”18 the first evocation of “death panels” that became such a prominent part of the 2009 health care reform debate. Others were concerned that population-based studies focusing on “average effects” would fail to detect benefits for special populations and would stifle the emerging promise of “personalized medicine.”19–22 Similarly, there were concerns that simplistic demands for comparative effectiveness information could raise the cost of bringing promising new innovations to market, with negative consequences on both U.S. health care and economic growth.17,23–26 And some were concerned that a narrow focus of CER on “which treatments work best for which patients” might be misguided and that large opportunities for improved efficiency and quality in the health care system lay in evidence-based delivery system reform, not individual clinical decisions informed by CER.27,28 Perhaps reflecting this tension, when the ARRA-funded Institute of Medicine CER Priorities Committee identified 100 top national priority topics for CER, 50 of these involved “health delivery” as a primary or secondary research area.28
CER Funding in Health Care Reform
As important as the ARRA “down payment” for CER has been, the more lasting impact will be through the substantial and sustained CER funding provided through the PPACA. This legislation establishes the Patient-Centered Outcomes Research Institute (PCORI) with a trust fund derived from an assessment on public and private health insurance.9 This fund will grow to yield at least $500 million per year for CER by 2013.
The legislation reflects compromises to address a variety of aspects of the 2009 debate over federal involvement in CER. To allay concerns that federal officials will be tempted to manage research findings to reduce access to expensive care, Section 6301 of the PPACA states that the PCORI is a nonprofit corporation “which is neither an agency nor establishment of the United States Government” (for details, see http://www.gao.gov/about/hcac/pcor_sec_6301.pdf). The concept of CER is both narrowed, by inserting the word “clinical” into “comparative clinical effectiveness research,” but also broadened. In the PPACA, CER is defined as
research evaluating and comparing health outcomes and the clinical effectiveness, risks, and benefits of 2 or more medical treatments, services, and other items... health care interventions, protocols for treatment, care management, and delivery, procedures, medical devices, diagnostic tools, pharmaceuticals (including drugs and biologicals), integrative health practices, and any other strategies or items being used in the treatment, management, and diagnosis of, or prevention of illness or injury in, individuals.9
There are also constraints on the use of cost-effectiveness analysis in PCORI-funded CER. The PCORI
shall not develop or employ a dollars-per-quality adjusted life year (or similar measure that discounts the value of a life because of an individual's disability) as a threshold to establish what type of health care is cost-effective or recommended.9
The PCORI will allocate trust fund dollars through contracts with a “preference” to allocating these funds through AHRQ or the NIH. Management of potential conflicts of interest is emphasized in the CER legislation. The legislation also notes the PCORI's responsibility to ensure regular reevaluation of previous CER findings in light of new discoveries. The details of these processes will await final decisions by the 21-member PCORI board appointed by the comptroller general in September 2010.
The Relevance of CER to the Missions of Academic Medicine
At a time of growing constraints on government spending, these new investments in CER are being made in a concerted attempt by policy makers to bring the power of science to bear on fixing the problems of the U.S. health care system. This commitment of resources reflects faith in the capacity of evidence and human reason to guide clinicians and honors the long tradition of medical schools' and teaching hospitals' use of science to inform medical care.
After the Flexner Report, academic medicine led the remaking of U.S. medical practice, replacing nostrums and 19th-century empiricism with pathophysiologically informed disease classifications as well as true “wonder drugs” and “miracle cures.”29 Medical schools and teaching hospitals have educated five generations of clinicians in the application of scientific principles to the diagnosis and treatment of disease. They have also trained, and often employed as faculty, those who have discovered biomedical and clinical innovations that have rendered curable numerous previously fatal diseases and provided years of quality life for millions afflicted with chronic diseases. Regionally and nationally, lay leaders look to medical school faculty as the interpreters and translators of research discoveries to guide health care improvements.
Thus, academic medicine has the opportunity to be an important force in this effort to use CER to improve U.S. health and health care. As with biomedical science discoveries, not every CER insight will emerge from the laboratories of academic medicine. Nonetheless, scientists conducting CER will acquire their skills in the training programs of academic health centers (AHCs). And the clinicians who are interpreting the medical literature and applying the insights from CER to the unique problems of individual patients will need to learn this evidence-based, patient-centered care from the educators, mentors, and role models at U.S. health science schools and teaching hospitals. These are weighty leadership responsibilities for academic medicine to fulfill, but they are important to shoulder if our society is to successfully use science to guide our way to an affordable and equitable health care system.
Research Development Challenges for Medical Schools and Teaching Hospitals
Although U.S. academic medicine has developed a robust and vibrant infrastructure for biomedical and clinical science, building specific capacities relevant to the conduct of CER will be a new endeavor. The first challenge will be to further develop the scientific disciplines and intellectual rigor demanded of CER. The “high stakes” nature of this work has been emphasized elsewhere.5,13,30,31 Indeed, it is because of the intense interest by a broad range of stakeholders (from investors to patient representatives, advocacy groups, manufacturers, health care facilities, and various clinicians) that CER has had such a difficult past. The unique funding and governance of CER in the PPACA, unprecedented in the history of U.S. research investment, were established because policy makers see CER as a uniquely challenging area of science.13 Thus, academic medicine will need to take most seriously the responsibility to manage the potential for investigator conflict of interest and to develop and promulgate rigorous scientific expertise in the conduct and dissemination of CER findings.
No single scientific discipline can be responsible for all CER competencies, which embrace a broad range of methodological skills needed to answer different CER questions. These skills will include those used in traditional clinical trials, pragmatic studies in practice-based research networks, analyses of electronic health records, observations from clinical registries, and sophisticated computer simulations.5,11,13,21,22,28,32 Outcomes measurement will be another methodological emphasis of CER. For this research to inform patient-centered clinical decisions, outcomes must be measured in terms immediately salient to the point of care. Thus, CER will demand a mix of research skills and expertise, from clinical epidemiology and health economics to sociology, psychology, and implementation science.33
Prior to the ARRA there had been quite limited federal funding in the disciplines and resources relevant to CER.10,34 Therefore, current capacities are limited in size, geographic distribution, and representation among health sciences faculties. Furthermore, for CER to transform the evidence base that guides clinical practice, this expertise cannot be isolated to just a few schools and teaching hospitals. The principles and practice of CER will be essential elements wherever clinical investigators are trained and wherever research to inform clinical practice is conducted. Many new investigators will be needed,10,11 and academic medicine will need to expand and deepen the expertise and educational capacity for training investigators in CER.
To conduct this training as well as to engage in this research, AHCs will need to apply all that has been learned in recent years regarding successful collaboration across disciplines, schools, and institutions, including effective team organization, infrastructure, and alignment of incentives.35 Insights can be gained from the experiences of the recipients of NIH Clinical and Translational Science Awards, which face similar challenges related to building transcampus multidisciplinary science programs with robust basic science, clinical research, research training, and methodological expertise.32 Other relevant teams of scholars have been developed at AHCs (often teamed with community organization partners) through such AHRQ initiatives as the Evidence-Based Practice Centers, the Centers for Education and Research on Therapeutics, and the Developing Evidence to Inform Decisions about Effectiveness Network.
To conduct successful CER, these new research teams will need to establish effective partnerships far outside the walls of the traditional academic setting. The FCCCER's definition of CER emphasized “comparing the benefits and harms of different interventions and strategies to prevent, diagnose, treat and monitor health conditions in ‘real world’ settings.”11,34 Health policy makers and program administrators are sobered by the frequency with which promising new innovations achieve impressive results in highly controlled trials only to fail when implemented in the routine practice of typically trained clinicians, multitasking support personnel, and patients with diverse clinical conditions, values, and life concerns. Thus, CER must conduct trials in, or acquire data from, diverse clinical settings organizationally, geographically, and often culturally remote from the clinical practices most closely affiliated with medical schools and teaching hospitals.
Perhaps the greatest challenge CER brings to traditional academic research derives from the new understanding of the consumers of the research products. As the FCCCER noted in its definition, CER's purpose is to provide “evidence-based information to patients, clinicians, and other decision-makers, responding to their expressed needs.”11,34 This is in marked contrast to the long-established intellectual traditions of academic medicine where the best research is investigator-initiated work, testing hypotheses to advance the understanding of the investigator's scientific peers. In this well-established paradigm, the critical audience for the investigator is the community of scholars who, as study section members, manuscript reviewers, and journal editors, assess the worthiness of the investigator's specific aims, the elegance of the methods, and the importance of the results. Stunning advances in our understanding of human illness have been the result of this tradition of scientific inquiry. But health policy experts like Sean Tunis36 have noted that the current approach to clinical inquiry yields large numbers of published clinical studies each year but relatively few “point of care” answers, found through systematic reviews of these same publications. For CER to be successful, CER researchers will need to rigorously focus on answering the questions relevant to typical clinician and patient decision makers.
Cultural Challenges for Medical Schools and Teaching Hospitals
The above criterion for success means that incorporating CER into the intellectual culture of academic medicine will be another challenge, perhaps as daunting as the challenges of formalizing the methods, expanding the training programs, and developing the research capacity. Academic medicine will need to build on its developing skills in interdisciplinary and cross-institutional research. It will also need to incorporate the new types of scholarship and intellectual expertise relevant to successful and socially beneficial CER. As discussed above, these will include new types of research design and outcomes measurement as well as analytic and data management expertise and scholarship in evidence adoption and dissemination.33 Although these CER-related areas of expertise may differ in emphasis from those prevalent today in academic medicine, accommodating them should not be a substantive challenge. Medical schools have effectively incorporated many new research disciplines over the decades as advances in biomedical research have provided new focused areas of study.
Revising the mechanisms of investigator support, reward, and recognition in academic medicine may prove a greater challenge to building the cadre of CER researchers. Securing support for CER through mechanisms like the PCORI will require that investigators adapt to some new types of research funding.37 Although the academic community should advocate continued opportunities for investigator-initiated CER relevant to methods and measurement development, it must also recognize CER's fundamental differences from much of clinical research. Focusing the research questions on the clinical decision-maker perspective introduces an emphasis on additional forms of scientific expertise and scholarship. For example, the successful development of major CER initiatives will rely on skillfully soliciting the insights of patients and clinicians in the field. Furthermore, a sophisticated understanding of the diversity of communities, cultures, patient perspectives, practice settings, and clinical contexts will be required to recruit and retain representative research participants and settings into relevant study networks.38,39 Academic medicine will need to develop effective incentives for CER investigators and ways to recognize new roles for CER scholarship such as research network development, patient and community engagement, and synthesis and translation of research findings.33
Accommodating these new areas of scholarly expertise and building partnerships across the health sciences (including nursing, public health, and allied health professions) will facilitate academic medicine's role in the development and use of CER. These new skills and partnerships will also enhance the other intellectual and social missions of academic medicine. Indeed, as early reports from the Clinical and Translational Science Award programs suggest, engaging the clinical practice, public health, nursing, and patient communities can provide important insights and speed the development of clinically important applications of basic science discoveries.
CER and the Education of Physicians
We have focused much of our preceding discussion on the implications of CER for evidence creation and health care science. However, for CER to truly improve the health of the nation, this evidence must be regularly used in individual clinical decisions in the broad range of practice settings.2,22,40 Moreover, the value of CER to the health of the nation will depend on a workforce prepared to interpret and implement evidence from CER at the point of care. Medical students and residents have long been exposed to the principles of medical science and evidence-based clinical decision making during training. Nonetheless, widespread practice variations, with underuse of effective therapies41 and the all-too-frequent overuse of unproven diagnostic or therapeutic approaches,42,43 suggest limited success of this aspect of medical education. The new societal emphasis on CER challenges the medical education community to be more successful but also provides the rationale and resources to teach evidence-based practice (16% of the PCORI Trust Fund is for CER dissemination and training through the AHRQ). As with research development, new investments in faculty, curriculum, and educational methods will likely be required to better prepare the clinical workforce for careers of using CER. Certainly, undergraduate and graduate medical education programs will need to provide effective training for assessing the medical literature and applying it in evidence-based practice. To prepare physicians for compassionate, culturally appropriate, and personalized use of CER at the point of care, educators will also need to demonstrate and facilitate patient-centered use of CER findings in the clinical practice of learners. CER investments will, however, give educators access to new resources and the motivation to try new approaches to fulfill their long-held aspiration that medical practice be guided by an artful melding of science with patient values.
For most practicing clinicians, the educational role of medical schools and teaching hospitals is, and will likely remain, somewhat indirect regarding the implementation of CER. Nonetheless, academic medicine provides intellectual leadership for the medical community and has an important role to play in facilitating the uptake of CER in community care. To do so, program leaders and faculty must be clear and consistent in their teaching of evidence-based clinical practice and in their role modeling of the appropriate use of CER findings in patient-centered care. This is particularly challenging, given the concurrent responsibility of academic medicine to provide leadership in evidence-based clinical innovation. It is, of course, essential that medical schools and teaching hospitals remain centers for innovation. To conduct research on their promising discoveries, these institutions and their faculty must make these innovations visible and recruit patients for further study. Nonetheless, these same institutions must take care that advocacy for further research on promising innovations not seem to encourage dissemination of these approaches without sufficient CER. By successfully fulfilling this dual responsibility to be advocates for both evidence-based practice and also research on promising innovations, academic medicine can play a central role in both the development and the use of science to advance health care.
Policy makers have high hopes for CER. The greatly expanded public emphasis on the development and use of clinical evidence challenges both the research and practice communities to demonstrate how clinicians can provide high-quality care for all Americans without compromising the nation's fiscal stability. Medical schools and teaching hospitals have the opportunity to play an important role in this effort. This will require investment in research infrastructure, adaptations of institutional culture, development of new disciplines and research methods, establishment of new collaborations, training of new faculty, and the expansion and refocusing of educational capacity. This is an exciting, albeit daunting, charge. By successfully responding to this challenge, academic medicine can further strengthen its long-standing commitment to the scientific practice of medicine and the use of evidence in patient-centered, personalized care.
The views expressed are solely those of the authors and do not necessarily represent the views of the Association of American Medical Colleges.
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