Examining the role of funders in ensuring value and reducing waste in research: An organizational case-study of the Patient-Centered Outcomes Research Institute.

International experts have recommended actions that funders can take to improve the value of research investments. They state that self-assessment and public sharing are the basis for accountability and improvement. We examined our policies and practice to determine the extent to which the Patient-Centered Outcomes Research Institute's (PCORI) policies and practices as a research funder align with international best practice recommendations. A self-audit of current policies and practice against 17 recommendations and 35 sub-recommendations representing five major stages of research production, based on adapted methods used for self-assessment by another funder, was performed.  Fit of existing PCORI policies and practices with 35 sub-recommendations, qualitative assessment of adequacy (area of strength; area of partial strength; area of growth; not applicable) for 17 recommendations for five stages of research production was assessed. Of the 17 recommendations, 15 were applicable to PCORI's research mission and focus.  PCORI has policies and practices in place for all elements of six recommendations ("area of strength") and policies that address each element but with some still in active development for three ("area of partial strength"). PCORI is partially addressing six of the 15 relevant recommendations ("area of growth"). Areas for growth include making study protocols publicly available, improving policies on data sharing, and enhancing collaboration with other funders to reduce redundant funding. A voluntary consortium of international funders is underway to encourage further progress, including additional self-assessment and public sharing for accountability. These findings indicate PCORI has undertaken efforts to align its funding practices with international recommendations to ensure the value of public dollars invested in research.  Further efforts will likely require additional coordination and collaboration between funders and stakeholders.

Primary Care Research in the Patient-Centered Outcomes Research Institute's Portfolio.

In their article in this issue, Mazur and colleagues analyze the characteristics of early recipients of funding from the Patient-Centered Outcomes Research Institute (PCORI). Mazur and colleagues note correctly that PCORI has a unique purpose and mission and suggest that it should therefore have a distinct portfolio of researchers and departments when compared with other funders such as the National Institutes of Health (NIH). Responding on behalf of PCORI, the authors of this Commentary agree with the characterization of PCORI's mission as distinct from that of NIH and others. They agree too that data found on PCORI's Web site demonstrate that PCORI's portfolio of researchers and departments is more diverse and more heavily populated with clinician researchers, as would be expected. The authors take issue with Mazur and colleagues' suggestion that because half of clinical visits occur within primary care settings, half of PCORI's funded research should be based in primary care departments. PCORI's portfolio reflects what patients and others tell PCORI are the critical questions. Many of these do, in fact, occur with more complex conditions in specialty care. The authors question whether the research of primary care departments is too narrowly focused and whether it sufficiently considers study of these complex conditions. Research on more complex conditions including heart failure, coronary artery disease, and multiple comorbid conditions could be highly valuable when approached from the primary care perspective, where many of the comparative effectiveness questions first arise.

Patient-powered research networks aim to improve patient care and health research.

The era of big data, loosely defined as the development and analysis of large or complex data sets, brings new opportunities to empower patients and their families to generate, collect, and use their health information for both clinical and research purposes. In 2013 the Patient-Centered Outcomes Research Institute launched a large national research network, PCORnet, that includes both clinical and patient-powered research networks. This article describes these networks, their potential uses, and the challenges they face. The networks are engaging patients, family members, and caregivers in four key ways: contributing data securely, with privacy protected; including diverse and representative groups of patients in research; prioritizing research questions, participating in research, and disseminating results; and participating in the leadership and governance of patient-powered research networks. If technical, regulatory, and organizational challenges can be overcome, PCORnet will allow research to be conducted more efficiently and cost-effectively and results to be disseminated quickly back to patients, clinicians, and delivery systems to improve patient health.

Launching PCORnet, a national patient-centered clinical research network.

The Patient-Centered Outcomes Research Institute (PCORI) has launched PCORnet, a major initiative to support an effective, sustainable national research infrastructure that will advance the use of electronic health data in comparative effectiveness research (CER) and other types of research. In December 2013, PCORI's board of governors funded 11 clinical data research networks (CDRNs) and 18 patient-powered research networks (PPRNs) for a period of 18 months. CDRNs are based on the electronic health records and other electronic sources of very large populations receiving healthcare within integrated or networked delivery systems. PPRNs are built primarily by communities of motivated patients, forming partnerships with researchers. These patients intend to participate in clinical research, by generating questions, sharing data, volunteering for interventional trials, and interpreting and disseminating results. Rapidly building a new national resource to facilitate a large-scale, patient-centered CER is associated with a number of technical, regulatory, and organizational challenges, which are described here.

Patient-Centered Outcomes Research Institute seeks to find out what works best by involving 'end-users' from the beginning.

Joe V Selby: A family physician, clinical epidemiologist and health services researcher, Dr Joe V Selby has more than 35 years of experience in patient care, research and administration. He is responsible for identifying strategic issues and opportunities for the Patient Centered Outcomes Research Institute (PCORI) and implementing and administering programs authorized by the PCORI Board of Governors. Dr Selby joined PCORI from Kaiser Permanente (CA, USA) where he was Director of the Division of Research for 13 years and oversaw a department of more than 50 investigators and 500 research staff members working on more than 250 ongoing studies. He was with Kaiser Permanente for 27 years. An accomplished researcher, Dr Selby has authored more than 200 peer-reviewed articles and continues to conduct research, primarily in the areas of diabetes outcomes and quality improvement. His publications cover a spectrum of topics, including effectiveness studies of colorectal cancer screening strategies; treatment effectiveness, population management and disparities in diabetes mellitus; primary care delivery; and quality measurement. Dr Selby was elected to membership in the Institute of Medicine in 2009 and was a member of the Agency for Healthcare Research and Quality study section for Health Care Quality and Effectiveness from 1999 to 2003. A native of Fulton (MI, USA), Dr Selby received his medical degree from Northwestern University (IL, USA) and his master's in public health from the University of California (CA, USA). He was a commissioned officer in the Public Health Service Corps from 1976 to 1983 and received the Commissioned Officer's Award in 1981.

Comparative effectiveness of bariatric surgery vs. nonsurgical treatment of type 2 diabetes among severely obese adults.

Although all weight-loss approaches may improve insulin sensitivity in type 2 diabetes, bariatric surgery is believed to be the only reliable means of achieving diabetes remission. We conducted a retrospective cohort study to compare rates of diabetes remission, relapse and all-cause mortality among severely obese individuals with diabetes who underwent bariatric surgery vs. nonsurgically treated individuals. Severely obese adults with uncontrolled or medication-controlled diabetes who underwent bariatric surgery or received usual medical care from 2005 to 2008 in three health care delivery systems in the United States were eligible. Diabetes status was identified using pharmacy, laboratory, and diagnosis information from electronic medical records. A propensity approach and exclusion criteria identified 1395 adults with diabetes who had bariatric surgery and 62,322 who did not. Most procedures were Roux-en-Y gastric bypass (72.0% laparoscopic; 8.2% open); 4.4% were gastric banding, 2.4% sleeve gastrectomy, and 13.2% were other procedures. At two years, bariatric subjects experienced significantly higher diabetes remission rates [73.7% (95% CI: 70.6, 76.5)] compared to nonsurgical subjects [6.9% (95%CI: 6.9, 7.1)]. Age, site, duration of diabetes, hemoglobin A1c level, and intensity of diabetes medication treatment were significantly associated with remission. Bariatric subjects also experienced lower relapse rates than nonsurgical subjects (adjusted HR: 0.19; 95% CI: 0.15-0.23) with no higher risk of death (adjusted HR: 0.54; 95% CI: 0.22-1.30). We conclude that bariatric surgery can effectively induce remission of diabetes among most severely obese adults, and this treatment approach appears to be superior to nonsurgical treatment in inducing diabetes remission.

How the Patient-Centered Outcomes Research Institute is engaging patients and others in shaping its research agenda.

Clinical research has been driven traditionally by investigators, from generating research questions and outcomes through analysis and release of study results. Building on the work of others, the Patient-Centered Outcomes Research Institute (PCORI) is tapping into its broad-based stakeholder community--especially patients, caregivers, and their clinicians--to generate topics for research, help the institute prioritize those topics, select topics for funding, and ensure patients' involvement in the design of research projects. This article describes PCORI's approach, which is emblematic of the organization's mandate under the Affordable Care Act to seek meaningful ways to integrate the patient's voice into the research process, and describes how it is being used in selection of research that PCORI will fund. We also describe challenges facing our approach, including a lack of common language and training on the part of patients and resistance on the part of researchers to questions that are not researcher generated. Faced with the reality that PCORI will not be able to fund all research questions posed to it, there will also be difficult decisions to make when selecting those that have the highest priority for funding.

Reviewing hypothetical migraine studies using funding criteria from the Patient-Centered Outcomes Research Institute.

What role can rigorous observational comparative effectiveness studies play in guiding clinical decision making? What criteria should be used in determining whether the results of such studies should be communicated to clinicians and to patients? We address these questions by considering two hypothetical observational studies in patients with migraine against the backdrop of the review criteria drawn up by the Patient-Centered Outcomes Research Institute (PCORI). These criteria emphasize that patient-centered comparative effectiveness research should exhibit relevance to patients, have great potential to affect practice and improve outcomes, and be conducted using rigorous analytic methods. We conclude that these hypothetical studies would be unlikely to have been funded or communicated by PCORI, and we offer suggestions for improving their relevance and analytic approaches. We also conclude that high-quality observational studies can effectively complement findings from randomized trials, and that communicating their results to patients and clinicians is warranted.