In 2010, PPACA authorized the establishment of PCORI as a nonprofit  corporation aimed at advancing the quality and relevance of evidence  through CER to help patients, clinicians, purchasers, and policy-makers in  making informed health care decisions. PCORI Has Established Priorities and Processes for Funding Comparative Clinical Effectiveness Research and Is Developing Dissemination Plans, Consistent with Its Legislative Requirements

PCORI has developed five broad research priorities and developed a  research agenda to identify how each priority will be addressed. The  institute has established a multi-step merit review process to score and  identify applications for funding and a process for monitoring contractors. PCORI Has Established Research Priorities and a Research Agenda

In 2012, PCORI established five broad research priorities: (1) assessment  of prevention, diagnosis, and treatment options; (2) improving health care  systems; (3) communication and dissemination research; (4) addressing  disparities; and (5) accelerating patient-centered outcomes research and  methodological research. PCORI also developed a research agenda to  identify how each priority would be addressed. PCORI established its research priorities and agenda consistent with  PPACA requirements. PCORI has established a multi-step research funding process,  which includes merit review, that is designed to select high quality  research that has the best potential to improve patient outcomes,  according to PCORI officials. Once funded, PCORI contractors are monitored by PCORI staff. PCORI officials stated that they are  currently developing a peer review assessment process, as required by  law, to review final reports submitted by contractors at the conclusion of a  project. PCORI Is Developing Dissemination Plans and Coordinating Dissemination Activities with AHRQ

PCORI is currently developing a plan for the dissemination of the  research it funds, as required by PPACA, and expects to begin  disseminating research results as early as 2015. PCORI Has Started Awarding Contracts for Research, and Plans to Award Additional Contracts through 2019

As of October 2014, PCORI has awarded 360 contracts to fund research  projects across 12 funding areas. PCORI made a total of $670.8 million in  commitments to fund these contracts. Overall, PCORI expects to  receive an estimated total of $3.5 billion through fiscal year 2019 from the  PCORTF to fund its work.$2.6 billion of its $3.5 billion on contracts for research and research  infrastructure, with the remaining amounts spent on research support  activities and administrative expenses. According to PCORI officials, approximately $106 million in commitments  to date are for the PCORnet data research network, the aim of which is to  improve the capacity for and speed of conducting CER. PCORI officials  stated that they expect to spend a total of $271 million on PCORnet  through 2019. Officials stated that limited amounts  of data will be available through PCORnet for queries by researchers  after September 2015, with the amount of available data increasing over  time. PCORI Has Established an Evaluation Plan and Anticipates Developing Efforts to Measure Outcomes

PCORI’s evaluation group—a body composed of members from its Board  of Governors, methodology committee, advisory panel on patient  engagement, external experts, and PCORI staff —has developed initial  plans for evaluating PCORI’s efforts against its three strategic goals,  which are to increase information, speed implementation, and influence  research. To do so, PCORI identified primary outcome measures for each  of its strategic goals. In its strategic plan, PCORI notes that these are  meant to be long-term measures because research typically requires  several years to complete and additional years for the results to be  disseminated and implemented. Therefore, since 2013, PCORI has been  using early and intermediate process and output measures as a way to  monitor its progress toward its strategic goals. PCORI anticipates having  some early results related to its primary outcome measures starting in  2017 after the first CER studies are completed and their findings  released, although full evaluation of the results of these outcome  measures will not be possible until around 2020, after a large number of  CER studies have been completed and a few years have elapsed,  allowing time for study results to be taken up. PCORI provided technical comments, which we incorporated as  appropriate.
Why GAO Did This Study

In 2010, PPACA authorized the establishment of PCORI as a federally funded, nonprofit corporation to improve the quality and relevance of CER. PCORI, which began operation in 2010, is required to identify research priorities, establish a research project agenda, fund research consistent with its research agenda, and disseminate research results, among other things. To fund PCORI, PPACA established the Patient-Centered Outcomes Research Trust Fund, through which the institute is expected to receive an estimated $3.5 billion from fiscal years 2010 through 2019.
PPACA mandated that GAO review PCORI's activities by 2015 and 2018. This report examines (1) the extent to which PCORI established priorities and processes for funding and disseminating comparative clinical effectiveness research consistent with its legislative requirements; (2) the status of PCORI's efforts to fund comparative clinical effectiveness research; and (3) PCORI's plans, if any, to evaluate the effectiveness of its work. GAO reviewed relevant legislative requirements and PCORI documentation, including funding data, and interviewed PCORI officials. GAO also interviewed relevant stakeholders, including health policy experts and PCORI contractors. PCORI provided technical comments, which GAO incorporated as appropriate.

What GAO Found

The Patient-Centered Outcomes Research Institute (PCORI) has established priorities and processes for funding comparative clinical effectiveness research (CER)—which is research that evaluates and compares health outcomes and the clinical effectiveness, risks, and benefits of two or more medical treatments, services, or items such as health care interventions—and is developing dissemination plans, consistent with the legislative requirements of the Patient Protection and Affordable Care Act (PPACA). In 2012, PCORI established five broad research priorities: (1) assessment of prevention, diagnosis, and treatment options; (2) improving health care systems; (3) researching communication and dissemination strategies; (4) comparing interventions to reduce health disparities; and (5) accelerating patient-centered outcomes research and methodological research. PCORI also developed a research agenda to identify how each priority would be addressed. PCORI has established a multi-step research funding process designed to assess and select contract applications for funding. Funded contracts are monitored by PCORI staff. Per legislative requirement, PCORI is developing a peer review assessment process to review final reports submitted by contract awardees and is in the process of developing a plan for the dissemination of funded research potentially beginning in 2015, in coordination with the Agency for Healthcare Research and Quality.
PCORI has started awarding contracts for research and plans to award additional contracts through 2019. As of October 2014, PCORI has awarded 360 contracts to fund research projects, committing a total of $670.8 million to them. PCORI expects to commit about $2.6 billion to research contracts, out of $3.5 billion in total estimated spending. Approximately $106 million in commitments to date are for PCORnet, a data research network aimed at improving the capacity for and speed of conducting CER. PCORI officials stated that they expect to spend a total of $271 million on PCORnet through fiscal year 2019. PCORI officials stated that limited amounts of data will be available through PCORnet for researchers to use after September 2015 with the amount of available data increasing over time.
PCORI has established an evaluation plan and is developing efforts to measure outcomes. PCORI has developed initial plans for evaluating the institute's efforts against its three strategic goals, which are to increase information, speed implementation, and influence research. To do so, PCORI has developed primary outcome measures for assessing PCORI's progress related to these strategic goals. In its strategic plan, PCORI notes that these are meant to be long-term measures because research typically requires several years to complete and additional years for the results to be disseminated and implemented. Therefore, since 2013, PCORI has been using early and intermediate process and output measures—such as the number of people accessing or referencing PCORI information—as a way to monitor its progress toward its strategic goals. PCORI anticipates having some early results related to its primary outcome measures starting in 2017 after the first CER studies are completed and their findings released, although full evaluation of the results of these outcome measures will not be possible until around 2020.