Dr. Pincus is professor and vice chair, Department of Psychiatry, and codirector, Irving Institute for Clinical and Translational Research, Columbia University, New York, New York, director of quality and outcomes research, New York–Presbyterian Hospital, New York, New York, and senior scientist, RAND Corporation, Santa Monica, California.
The author is grateful for the support of the Irving Institute for Clinical and Translational Research at Columbia University (UL1 RR024156), which is funded by the National Center for Research Resources (a component of the National Institutes of Health) and the Mental Health Center for Education and Research in Therapeutics at Rutgers, the State University of New Jersey, through a subcontract to Columbia University, which is funded by the Agency for Healthcare Research and Quality (5 U18 HS016097).
In many ways, the term comparative effectiveness research (CER) is like a projective test. Individuals (and organizations) have presumptions about the connotations and “baggage” that accompany the term. To some, it implies studies that compare Drug A against Drug B (so-called “Coke versus Pepsi” studies). For others, as noted by Rich et al1 in this issue, the term raises the specter of government interfering with medical practice, dictating specific treatment procedures. To others, it conjures up the “R” word (rationing) and images of death panels. The battles surrounding health care reform attached so much baggage that a new term for CER was created in the Patient Protection and Affordable Care Act—patient-centered outcomes research. In fact, CER has a specific definition spelled out in an Institute of Medicine report,2 built around the simple concept of providing more and better information to patients, providers, and policy makers (see the box in VanLare et al3 in this issue). Although some of the language surrounding CER is clearly overwrought, the controversy underscores the fact that the development and application of CER affect individuals and organizations and, ultimately, involve the weighing of values and interests. And, as the articles in this issue of Academic Medicine make clear, there are diverse approaches that academic health centers (AHCs) can apply to get involved in CER.
It may be helpful, conceptually, to dissect the principal activities that are related to CER to avoid (or at least narrow) the attachment of “excess baggage” and to better understand the particular roles and responsibilities of AHCs. These activities can be placed into seven buckets, each having a variable level of political baggage, and each suggesting particular roles for AHCs as primary or major contributors to or developers of CER, as organizations that implement the results of CER, as participants contributing to the policy and political process, and as stakeholders affected by CER. These buckets, described below, are similar to but a bit more fine-grained than the four dimensions discussed by VanLare et al.
Virtually all of the CER-related articles in this issue note the critical need for investigators with the necessary skills and experience to conduct CER and the central role of AHCs in developing these investigators. Importantly, the disciplines that need to be brought together are extensive and diverse, extending well past schools of health sciences. Iribarne et al4 describe innovative training approaches that cut across clinical research, economics, and decision making through the Columbia Clinical and Translational Science Award. As Rich et al remind us, the workforce needs are not limited to investigators but also extend to training for those health care providers whose decision making will require a detailed understanding of CER. Moreover, they point out the cultural challenges AHCs face as they address these research training and clinical education needs.
Conducting CER is, for the most part, not a “Lone Ranger” enterprise that can be carried out in isolation. It requires a variety of supports, as VanLare et al point out, including practice-based research networks, extensive databases, and longitudinal registries that have rich clinical detail and are connected through interoperable health information technology. This infrastructure also includes a base of biostatistical expertise that can develop and provide necessary tools for analysis of multiple types of CER research designs. AHCs clearly have a central role in building this infrastructure but will need to expand their bridges to local health providers and community organizations as well as consortia of other health systems. Several articles in this issue, as well as a white paper published by members of the national CTSA consortium,5 note that Clinical and Translational Science Awards funded by the National Institutes of Health are remarkably well suited to serve as a nidus for CER infrastructure development within an AHC. Marantz et al6 provide an illustration of a CTSA-based model at Albert Einstein College of Medicine of Yeshiva University.
Conducting research is obviously a major domain of AHC activity, but accommodating and expanding CER will require some adjustment. VanLare et al point to the need to expand efforts in “real-world observational research” as well as in behavioral, surgical, and other nonmedical interventions. MaGaghie et al7 remind us that evaluation of medical education interventions is also a part of CER, and Rask et al8 demonstrate that CER also extends to the area of predictive health and personalized medicine. Importantly, AHCs are not just generators of this research; they are organizations (and include individuals) for whom the outcomes of CER can have significant impact. As such, irrespective of the topic or focus of CER, the research must be conducted in a scrupulous and transparent manner that carefully adheres to rules of ethics and management of potential conflicts of interest.
Analysis and synthesis.
To have impact, the data from CER must be subjected to sophisticated analysis, and the results of multiple studies must be synthesized in a manner that balances the strengths of the evidence with the limitations of the methods employed. This process is itself a science and requires sophisticated methodologies, including methods for economic evaluation, as underscored by Iribarne et al. It is also critical that syntheses frame the findings in a way that is relevant to and usable by different groups: clinicians, patients and families, and policy makers (public and private, national and local). Zerzan et al9 describe a highly innovative partnership between AHCs and state Medicaid agencies, ongoing since 2003, as well as the lessons learned from that experience.
Clinical and systems implementation.
As major organizational providers of care, AHCs have a responsibility to use the best information in providing clinical care at the bedside and in the clinic. Moreover, AHCs have larger responsibilities—to provide leadership in clinical care and systems improvement for their communities, their region, and the nation. Developing models for integrating these activities is expected to be encouraged through health care reform initiatives such as Healthcare Innovation Zones. These zones and several organizational models for implementing CER are described in this issue of the journal.
Not everyone will agree on which issues or questions should be tackled first and with what level of resources. Although methodologies for systematically setting priorities have been proposed, and an Institute of Medicine committee2 has developed an initial set of priorities, it remains to be seen how the new Patient-Centered Outcomes Research Institute will function in this regard. Whatever process is established, individuals and organizations from academic medicine must play active roles. Also, at a local level, AHCs should engage their communities in an ongoing process to assess their needs and priorities.
Ultimately, the rubber hits the road in CER when it comes to areas such as Food and Drug Administration rule making, Medicare/Medicaid and private health plan coverage decisions, determination of quality, safety, and efficiency, measures and expectations in evidence-based purchasing, and others. VanLare et al speak to the need for AHCs to develop formalized institutional policy expertise in order to shape the policy debate. It is also important for AHCs and their associated professional societies to understand and engage in the political arena to ensure that their voices as stakeholders are heard.
Each of the CER-related articles in this issue demonstrates different strategies, projects, and foci that AHCs are addressing to engage in CER activities. Ultimately, the approaches that AHCs take with CER are not that different from those taken in other fields of academic endeavor and are rooted in the basics of the AHC's mission10:
* Develop the data, tools, and methods to answer key questions across the translational spectrum, from bench to bedside to community.
* Conduct research to answer these questions.
* Train future leaders in research and clinical care.
* Develop collaborations across multiple disciplines and with practicing clinicians and the community.
* Deliver the highest-quality health care, serving as a model for innovation.
This is not to say that CER is “old wine in new bottles.” Although the basic processes are the same, they are being extended in new directions with novel tools, expanded partnerships, and more complex structures. It is still “wine,” but these new blends will require training a new generation of vintners, new methods for cultivation, and a more effective distribution system. Purity of the product must be ensured through oversight and transparency. Equally important, AHCs will need to engage creatively in the policy process and wade assertively into the swamps of political controversy to provide their unique perspectives as stakeholders to shape the further national debates that are certain to ensue.
Harold Alan Pincus, MD
1Rich EC, Bonham AC, Kirch DG. The implications of comparative effectiveness research for academic medicine. Acad Med. 2011;86:684–688.
2Committee on Comparative Effectiveness Research Prioritization, Institute of Medicine. Initial National Priorities for Comparative Effectiveness Research. Washington, DC: National Academies Press; 2009.
3VanLare JM, Conway PH, Rowe JW. Building academic health centers' capacity to shape and respond to comparative effectiveness research policy. Acad Med. 2011;86:689–694.
4Iribarne A, Easterwood R, Wang YC. Integrating economic evaluation methods into Clinical and Translational Science Award consortium comparative effectiveness educational goals. Acad Med. 2011;86:701–705.
5Selker H, Strom B, Ford D. White paper on CTSA consortium role in facilitating comparative effectiveness research. Clin Transl Sci. 2009;3:29–37.
6Marantz PR, Strelnick AH, Currie B, et al. Developing a multidisciplinary model of comparative effectiveness research within a CTSA. Acad Med. 2011;86:712–717.
7McGaghie WC, Issenberg SB, Cohen ER, Barsuk JH, Wayne DB. Does simulation-based medical education with deliberate practice yield better results than traditional clinical education? A meta-analytic comparative review of the evidence. Acad Med. 2011;86:706–711.
8Rask KJ, Brigham KL, Johns MME. Integrating comparative effectiveness research programs into predictive health: A unique role for academic health centers. Acad Med. 2011;86:718–723.
9Zerzan JT, Gibson M, Libby AM. Improving state Medicaid policies with comparative effectiveness research: A key role for academic health centers. Acad Med. 2011;86:695–700.
Editor's Note: In this issue of the journal you will find a timely group of articles that examine comparative effectiveness research (CER) and its growing effect on academic health centers. Harold Pincus, MD, coordinated this collection and ensured that these articles convey both the current status of CER in medical schools and teaching hospitals and the challenges that lie ahead. I thank Harold for his good work and for writing the Guest Editorial to introduce the articles. A special thanks goes to Academic Medicine staff member Jennifer Campi, who worked tirelessly to develop and organize the articles so that they would most effectively reveal key lessons for advancing CER in academic health centers.
—Steven L. Kanter, MD