Published Sep 2017
Berger ML, Sox H, Willke RJ. 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. Value Health 2017; (8): 1003-1008.
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.
Keywords: comparative effectiveness, decision making, guidelines, pharmacoepidemiology, real-world data, treatment effectiveness.
Copyright © 2017, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc.