Challenges Facing the US Patient-Centered Outcomes Research Institute

Realizing the promise of comparative effectiveness research and achieving health care practice change require not only identifying research priorities, but also determining how to conduct the studies and implement the results. The new Patient-Centered Outcomes Research Institute (PCORI) is a nonprofit entity charged with prioritizing and supporting studies and improvement in the methods of comparative effectiveness research.¹ By 2014, the PCORI budget may increase to more than $500 million. It is funded by Medicare, private health insurers, and self-insured plans.

Even though the PCORI is a new entity, it is rooted in recommendations made a decade ago by members of the Clinical Research Roundtable (CRR) at the Institute of Medicine of the National Academies.² The CRR focused on the critical challenges facing the nation's clinical research enterprise and how they might be resolved.

One challenge was the translation of new research findings into clinical practice. In a frequently cited article,³ the CRR identified 2 translational blocks: bench to bedside and bedside to clinical practice, each requiring specific attention. Notably, the CRR, with regard to bedside to clinical practice, argued for increased efforts focused on effectiveness research.^{4 - 7}

The CRR expressed concern that the research enterprise did not focus enough on effectiveness. Recent analyses of the level of evidence used by organizations to develop guidelines for infectious diseases showed that the recommendations in the guidelines largely relied upon expert opinion and consensus statements.⁸ Members of the CRR concluded that new mechanisms were needed to enhance the output of effectiveness research, and that all public and private stakeholders should be involved in the planning, conduct, and dissemination of the research.

Members of the CRR proposed and discussed several alternative mechanisms and focused on a recommendation to create a public-private cooperative for health care improvement research.⁵ The model identified by the CRR is similar to the long-standing cooperative research programs of the National Academies' Transportation Research Board.^{5 ,9} These programs focus on applied research, development of research priorities, and sponsorship and monitoring of research studies. The research priorities are developed through public information requests and a committee of public and private stakeholders. Specific requests for proposals are then developed by the National Academies of Science in collaboration with expert review panels. Peer review by scientific panels and monitoring of contract mechanisms ensure that specific goals are met. The PCORI model draws from these recommendations; however, the mechanism for implementing its research priorities is left unclear. For instance, how will the PCORI review and fund research? Will it use existing public-sector mechanisms or will it develop new approaches?

Clinical implementation of research findings can be hindered by issues such as experimental design and perceived generalizability of the study approach. Perceptions that create doubt are likely to hinder acceptability of implementing approaches to change clinical practice. With these issues in mind, the PCORI should place an emphasis on ensuring clear and thorough review of study methods and research protocols. Other issues such as the timing of the research and the need for implementation studies are also important and should be addressed by the PCORI. Assessment of the feasibility of proposed studies is of critical importance. If patients or clinicians do not perceive clinical equipoise, participation in the study will be hampered. Careful consideration of patient and clinician perception regarding equipoise should be considered during the review of proposed research. In addition, methods to improve patient and clinician awareness and engagement in studies should be emphasized. Novel approaches for study design as well as patient recruitment also should be encouraged.

Lacking a substantial effort focused on implementation, the published results of comparative effectiveness research are unlikely to change medical practice on their own. An example of the type of implementation effort needed is provided by the dissemination study of the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack (ALLHAT) trial, a large comparative effectiveness research study.¹⁰ The ALLHAT results indicated that treatment with a thiazide-type diuretic was superior to a calcium channel blocker, an angiotensin-converting enzyme inhibitor, or an α-blocker in preventing major cardiovascular disease events. When the ALLHAT trial results were published, concerns regarding study design and generalizability were raised. These concerns may have negatively affected the dissemination efforts. A dissemination study provided academic detailing efforts (1698 presentations to 18 524 physicians) in 41 states over 2.5 years. However, only modest changes were observed in the group with the highest level of detailing. In the postintervention study of select practices,¹⁰ 46.5% of patients with hypertension were prescribed thiazides compared with 39.4% of patients in the comparison group. The cost of the dissemination project, including the evaluation, was $3.7 million.

Resource materials provide a basis for an educational campaign, but on their own will not produce desired changes. Face-to-face interaction, involvement of opinion leaders, clinical audit and feedback, and reminders are needed to produce changes in behavior in the health care system. Clinical judgment will still be required; however, comparative effectiveness research studies will provide a guidepost to aide clinicians and patients in their judgment concerning treatment choices. To facilitate change, the PCORI should establish a subcommittee focused on implementation and should also participate, as appropriate, in the development of clinical practice guidelines. Another barrier to implementation is the potential impact of practice change to clinical practice revenue in which appropriate incentives should be considered to motivate changes in deep-rooted clinical practices. In addition to these considerations, significant efforts will be needed to bring comparative effectiveness research information with appropriate decision support to both patients and clinicians at the point of care.