Comparative Effectiveness Research: HHS Should Evaluate Its Performance of Related Activities

What GAO Found

Comparative clinical effectiveness research evaluates and compares the health outcomes of two or more medical treatments, services, or items. In 2010, Congress authorized the establishment of the Patient-Centered Outcomes Research Institute (PCORI) to conduct this research and improve its quality and relevance, and directed the Department of Health and Human Services (HHS) to publicly disseminate and help incorporate these research findings into clinical practice. PCORI and HHS obligated a total of $3.1 billion for fiscal years 2019 through 2024.

PCORI Award Obligations and HHS Obligations for Comparative Clinical Effectiveness Research Activities, Fiscal Years 2019 Through 2024

Note: PCORI's award obligations and HHS's obligations may be expended in years subsequent to the year they were made. PCORI's award obligations do not include funds used by PCORI for its operations.

From 2016 to March 2025, HHS disseminated findings from 16 PCORI-funded studies through a newsletter and social media, among other methods, according to officials. HHS also helped to implement the findings of one PCORI-funded study through one of its dissemination and implementation programs.

GAO found that PCORI uses key performance management practices—identifying long- and near-term goals and associated performance measures—to assess the performance of its dissemination and implementation efforts.

Similarly, HHS does this for its three largest dissemination and implementation programs. In 2020, GAO reported HHS had plans to evaluate its dissemination and implementation portfolio and, according to officials, this evaluation would develop near-term goals and performance measures. However, it is unclear whether the evaluation will be conducted as planned. HHS has not yet requested proposals for the evaluation, and officials attributed recent delays to leadership changes in August 2024 and March 2025. Further, in March 2025, HHS announced staff reductions and a departmental reorganization, which had not been implemented as of April 2025. Establishing near-term goals and performance measures as part of the planned evaluation will help enable HHS to regularly assess performance and determine whether the dissemination and implementation portfolio efforts as a whole are achieving the intended aim of promoting evidence-based, patient-centered care to improve health outcomes.

Why GAO Did This Study

Federal law includes provisions for GAO to regularly review PCORI and HHS comparative clinical effectiveness research activities. PCORI, a federally funded nonprofit corporation, makes awards for research studies that focus on health issues facing people in the U.S., such as mental and behavioral health, cardiovascular disease, and cancer. Disseminating findings from this research can provide important information on effective treatments to improve health care decision-making for stakeholders, such as providers and patients. Also, implementing these findings into clinical practice may further result in improved health outcomes.

This report describes HHS efforts to disseminate and help implement PCORI-funded research findings and examines the extent to which PCORI and HHS have used key performance management practices to assess their respective efforts, among other issues.

GAO reviewed PCORI and HHS documentation and data for fiscal years 2019 through 2024. GAO also interviewed PCORI representatives, HHS officials, and representatives from a nongeneralizable sample of nine stakeholder groups, such as provider and patient organizations.