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

• Published in: Pharmacoepidemiology and drug safety 2017-09, Vol.26 (9), p.1033-1039
• Main Authors: Berger, Marc L., Sox, Harold, Willke, Richard J., Brixner, Diana L., Eichler, Hans‐Georg, Goettsch, Wim, Madigan, David, Makady, Amr, Schneeweiss, Sebastian, Tarricone, Rosanna, Wang, Shirley V., Watkins, John, Daniel Mullins, C.
• Format: Article
• Language: English
• Subjects: Advisory Committees - standards; comparative effectiveness; Decision Making; Delivery of Health Care - methods; Delivery of Health Care - standards; Economics; Economics, Pharmaceutical - standards; guidelines; Health care industry; Humans; Internationality; Original Report; Original Reports; pharmacoepidemiology; Pharmacoepidemiology - standards; Pharmacology; Pragmatic Clinical Trials as Topic - methods; Pragmatic Clinical Trials as Topic - standards; Prospective Studies; real‐world data; Retrospective Studies; Societies, Scientific - standards; Statistics as Topic - methods; Statistics as Topic - standards; Studies; Task forces; treatment effectiveness; Treatment Outcome
• ISSN: 1053-8569; 1099-1557
• DOI: 10.1002/pds.4297

Purpose 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. Methods 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. Results 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. Conclusion 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.