Marc L Berger1, Harold Sox2, Richard J Willke3, Diana L Brixner4, Hans-Georg Eichler5, Wim Goettsch6, David Madigan7, Amr Makady6, Sebastian Schneeweiss8, Rosanna Tarricone9, Shirley V Wang8, John Watkins10, C Daniel Mullins11. 1. New York City, NY, USA. Electronic address: mlberger301@gmail.com. 2. Patient-Centered Outcomes Research Institute, Washington, DC, USA. 3. International Society for Pharmacoeconomics and Outcomes Research, Lawrenceville, NJ, USA. 4. University of Utah, Salt Lake City, UT, USA. 5. European Medicines Agency, London, UK. 6. Zorginstituut Nederland and University of Utrecht, Utrecht, The Netherlands. 7. Columbia University, New York City, NY, USA. 8. Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA. 9. Bocconi University, Milan, Italy. 10. Premera Blue Cross, Mountlake Terrace, WA, USA. 11. University of Maryland, Baltimore, MD, USA.
Abstract
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.
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.
Authors: Luca Cicchitti; Alessandra Di Lelio; Gina Barlafante; Vincenzo Cozzolino; Susanna Di Valerio; Paola Fusilli; Giuseppe Lucisano; Cinzia Renzetti; Marco Verzella; Maria Chiara Rossi Journal: Med Sci (Basel) Date: 2020-06-24
Authors: Shirley V Wang; Olga V Patterson; Joshua J Gagne; Jeffrey S Brown; Robert Ball; Pall Jonsson; Adam Wright; Li Zhou; Wim Goettsch; Andrew Bate Journal: Drug Saf Date: 2019-11 Impact factor: 5.606