Emerging healthcare interventions: Patient-Centered Outcomes Research Institute's programmatic initiative.

The Patient-Centered Outcomes Research Institute (PCORI) is a nonprofit, nongovernmental organization established by the U.S. Congress to fund comparative clinical effectiveness research focusing on patient-centered outcomes through the engagement of stakeholders. Evaluation of emerging healthcare innovations is one of PCORI's five National Priorities for Health. One such initiative is PCORI's Emerging Technologies and Therapeutics Reports program, established to provide timely overviews of evidence on new drugs and other healthcare technologies. This article provides an overview of completed and ongoing Emerging Technologies and Therapeutics Reports including lessons learned to date. In addition to systematic searches, systematic selection of studies, and transparent reporting of the available evidence, informed by a select number of stakeholders (i.e., key informants), these reports focus on contextual factors shaping the diffusion of emerging technologies that are often not reported in the medical literature. This article also compares processes and methodologies of health technology assessments (HTAs) from a selected number of national and international publicly funded agencies with a goal toward potential future enhancement of PCORI's Emerging Technologies and Therapeutics Reports program. HTAs vary considerably in terms of funding, types of assessments, the role of manufacturers, stakeholder engagement, timeline to complete from the start to the finish of a draft report publication, and communication of uncertainty for informed decision making. Future Emerging Technologies and Therapeutics Reports may focus on rapid reports to support a more expedient development of evidence. Future research could explore the role of contextual factors identified in these reports on targeted evidence generation.

Patterns of public participation.

Purpose - The paper summarizes data from 12 countries, chosen to exhibit wide variation, on the role and place of public participation in the setting of priorities. The purpose of this paper is to exhibit cross-national patterns in respect of public participation, linking those differences to institutional features of the countries concerned. Design/methodology/approach - The approach is an example of case-orientated qualitative assessment of participation practices. It derives its data from the presentation of country case studies by experts on each system. The country cases are located within the historical development of democracy in each country. Findings - Patterns of participation are widely variable. Participation that is effective through routinized institutional processes appears to be inversely related to contestatory participation that uses political mobilization to challenge the legitimacy of the priority setting process. No system has resolved the conceptual ambiguities that are implicit in the idea of public participation. Originality/value - The paper draws on a unique collection of country case studies in participatory practice in prioritization, supplementing existing published sources. In showing that contestatory participation plays an important role in a sub-set of these countries it makes an important contribution to the field because it broadens the debate about public participation in priority setting beyond the use of minipublics and the observation of public representatives on decision-making bodies.

An overview and discussion of the Patient-Centered Outcomes Research Institute's decision aid portfolio.

Decision aids (DAs) help patients make informed healthcare decisions in a manner consistent with their values and preferences. Despite their promise, DAs developed with public research dollars are not being implemented and adopted in real-world patient care settings at a rate consistent with which they are being developed. To appraise the sum of the parts of the portfolio and create a strategic imperative surrounding future funding, the Patient-Centered Outcomes Research Institute (PCORI) tasked the Duke Evidence Synthesis Group with evaluating its DA portfolio. This paper describes PCORI's portfolio of DAs according to the Duke Evidence Synthesis Group's analysis in the context of PCORI's mission and the field of decision science. The results revealed a diversity within PCORI's portfolio of funded DA projects. Findings support the movement toward more rigorous DA development, assessment and maintenance. PCORI's funding priorities related to DAs are clarified and comparative questions of interest are posed.

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.

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.

Building sustainable multi-functional prospective electronic clinical data systems.

A better alignment in the goals of the biomedical research enterprise and the health care delivery system can help fill the large gaps in our knowledge of the impact of clinical interventions on patient outcomes in the real world. There are several initiatives underway to align the research priorities of patients, providers, researchers, and policy makers. These include Agency for Healthcare Research and Quality (AHRQ)-supported projects to build flexible prospective clinical electronic data infrastructure that meet the needs of these diverse users. AHRQ has previously supported the creation of 2 distributed research networks as a new approach to conduct comparative effectiveness research (CER) while protecting a patient's confidential information and the proprietary needs of a clinical organization. It has applied its experience in building these networks in directing the American Recovery and Reinvestment Act funds for CER to support new clinical electronic infrastructure projects that can be used for several purposes including CER, quality improvement, clinical decision support, and disease surveillance. In addition, AHRQ has funded a new Electronic Data Methods forum to advance the methods in clinical informatics, research analytics, and governance by actively engaging investigators from the American Recovery and Reinvestment Act-funded projects and external stakeholders.

Debunking myths of protocol registration.

Developing and registering protocols may seem like an added burden to systematic review investigators. This paper discusses benefits of protocol registration and debunks common misperceptions on the barriers of protocol registration. Protocol registration is easy to do, reduces duplication of effort and benefits the review team by preventing later confusion.

Comparative effectiveness research priorities at federal agencies: the view from the Department of Veterans Affairs, National Institute on Aging, and Agency for Healthcare Research and Quality.

In the last year, attention has been focused on translating federally sponsored health research into better health for Americans. Since the passage of the American Recovery and Reinvestment Act (ARRA) on February 17, 2009, ARRA funds to support Comparative Effectiveness Research (CER) have increased this focus. A large proportion of topical areas of interest in CER affects the older segment of the population. The Department of Veterans Affairs (VA), the National Institute on Aging (NIA), and the Agency for Healthcare Research and Quality (AHRQ) have supported robust research portfolios focused on aging populations that meet the varying definitions of CER. This short article briefly describes the research missions of the AHRQ, NIA, and VA. The various definitions of CER as the Congressional Budget Office, the Institute of Medicine, and the ARRA-established Federal Coordinating Council have put forward, as well as important topics for which CER is particularly needed, are then reviewed. Finally, approaches in which the three agencies support CER involving the aging population are set forth and opportunities for future CER research outlined.

AHRQ series paper 1: comparing medical interventions: AHRQ and the effective health-care program.

In 2005, the Agency for Healthcare Research and Quality established the Effective Health Care (EHC) Program. The EHC Program aims to provide understandable and actionable information for patients, clinicians, and policy makers. The Evidence-based Practice Centers are one of the cornerstones of the EHC Program. Three key elements guide the EHC Program and thus, the conduct of Comparative Effectiveness Reviews by the EPC Program. Comparative Effectiveness Reviews introduce several specific challenges in addition to the familiar issues raised in a systematic review or meta-analysis of a single intervention. The articles in this series together form the current Methods Guide for Comparative Effectiveness Reviews of the EHC Program.

Funding opportunities in knowledge translation: review of the AHRQ's "Translating Research into Practice" initiatives, competing funding agencies, and strategies for success.

The Agency for Healthcare Research and Quality actively funds and conducts research to improve health care for all Americans. This article is intended to provide a brief overview of Agency for Healthcare Research and Quality activities in knowledge translation and to accompany the presentation given on May 15, 2007, to the Academic Emergency Medicine Consensus Conference, "Knowledge Translation in Emergency Medicine: Establishing a Research Agenda and Guide Map for Evidence Uptake."