The Patient-Centered Outcomes Research Institute (PCORI) National Priorities for Research and Initial Research Agenda

Abstract

THE PATIENT PROTECTION AND AFFORDABLE CARE ACT of 2010 created the Patient-Centered Outcomes Research Institute (PCORI) to fund and promote comparative clinical effectiveness research (CER) that will “assist patients, clinicians, purchasers, and policymakers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis.” CER is not a new concept, but appreciation of its potential for providing patients and their clinicians with uniquely valuable information on what works, tailored to the clinical situation and to patient priorities, has increased rapidly in recent years. The research institute founded by this legislation was named to emphasize the critical importance of a patientcentered perspective in conducting this research. The PCORI Board of Governors determined early on that taking this name seriously, placing patients at the center of CER and actively engaging clinicians and other stakeholders was an ideal strategy to ensure that the PCORI research agenda stays focused on practical questions, relevant outcomes and study populations, and the possibility that treatment effects may differ across patient populations. The institute developed a definition of patient-centered outcomes research that emphasizes the voice of the patient in assessing health care options. The PCORI mission statement commits to producing and promoting high-integrity research that is “guided by patients, caregivers, and the broader healthcare community.” The institute’s first funding announcement solicited projects focused on methods for engaging patients and other stakeholders in all aspects of the research process. Merit review of more than 800 responses to this announcement is being conducted by the Center for Scientific Review of the National Institutes of Health (NIH). PCORI has modified the process in 2 ways. First, stakeholders, including patients, caregivers, and clinicians, were invited via the PCORI website to sign up if they were interested in participating in the merit reviews. A total of 48 individuals were subsequently recruited, trained by the NIH, and included as voting members of the 16 study sections (3 per section). Second, proposed scoring criteria included an additional criterion—extent of patient engagement. The founding legislation required PCORI to develop national priorities for research and a research agenda, and to post both for a 45to 60-day public comment period before major funding for research could begin. The development process extended from July to December 2011. It included examination of other recent prioritization efforts and consideration of 9 criteria cited in the statute (BOX), of the PCORI definition of patient-centered outcomes research, and of input received through discussions with stakeholder groups, including patients and their caregivers, clinicians, hospitals and health care systems, payers, the life sciences industry, and the research community. The process produced 5 national priorities (Box) and the research agenda, which are available on the PCORI website. The national priorities encompass many research areas cited by earlier priority-setting groups, but add a patientcentered perspective. Helping patients make informed health care choices will not be achieved solely by producing more CER evidence on prevention, diagnostic, or treatment strategies. New evidence is unlikely to be rapidly adopted unless the systems in which patients and clinicians make decisions are improved, removing current barriers to acting on the new information. Better methods for making CER results available and for communication about those results between patients and their clinicians are needed. Similar to studies evaluating health disparities, CER seeks evidence on possible differences in the effectiveness of treatments, in preferences for various outcomes, and in information needs across various populations. CER evidence can be further purposed to reduce or eliminate health disparities among vulnerable populations. Another priority addresses the need to build and sustain a national research infrastructure that facilitates learning from clinical experience. It calls for building clinical research databases, improving analytic methods for conducting this research, and train-