Good practices for real‐world data studies of treatment and/or comparative effectiveness: Recommendations from the joint <scp>ISPOR‐ISPE</scp> Special Task Force on real‐world evidence in health care decision making

TLDR

Real‑world evidence, derived from observational studies and registries, offers insights beyond randomized trials and is increasingly used to inform health‑care decisions. The task force aims to provide recommendations on procedural best practices to strengthen confidence in evidence from real‑world data studies. The recommendations were developed through an iterative consensus process involving peer review by ISPOR/ISPE members, covering seven key topics such as study registration, replicability, and stakeholder involvement. The resulting recommendations establish a trustworthy foundation for expanding the use of real‑world evidence in health‑care decision making, emphasizing procedural best practices for hypothesis‑driven studies in defined populations.

Abstract

Real-world evidence (RWE) includes data from retrospective or prospective observational studies and observational registries and provides insights beyond those addressed by randomized controlled trials. RWE studies aim to improve health care decision making.The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the International Society for Pharmacoepidemiology (ISPE) created a task force to make recommendations regarding good procedural practices that would enhance decision makers' confidence in evidence derived from RWD studies. Peer review by ISPOR/ISPE members and task force participants provided a consensus-building iterative process for the topics and framing of recommendations.The ISPOR/ISPE Task Force recommendations cover seven topics such as study registration, replicability, and stakeholder involvement in RWE studies. These recommendations, in concert with earlier recommendations about study methodology, provide a trustworthy foundation for the expanded use of RWE in health care decision making.The focus of these recommendations is good procedural practices for studies that test a specific hypothesis in a specific population. We recognize that some of the recommendations in this report may not be widely adopted without appropriate incentives from decision makers, journal editors, and other key stakeholders.