Practical Aspects of Predicting RCT Findings Using Healthcare Databases
Department of Epidemiology Seminar Series
Speaker:
Sebastian Schneeweiss, M.D., Sc.D.
Professor of Medicine at Harvard Medical School and Chief of the Division of Pharmacoepidemiology, Department of Medicine, Brigham and Women’s Hospital in Boston
Department of Epidemiology of the Harvard T.H. Chan School of Public Health.
Professor of Epidemiology, Harvard T.H. Chan School of Public Health
Abstract: Healthcare databases have been used for decades to understand the safety of prescription drugs and are increasingly considered to study their intended treatment effects. In an FDA-funded demonstration project, we identified 30 RCTs designed for regulatory submissions, emulated their design and measurements as much as possible in claims data, and estimated and compared effect estimates. We also predicted the findings of 7 ongoing trials. Going through a defined process incl. pre-registration of study protocols, several challenges to emulating trials conducted for regulatory purposes emerged. Yet among those trials that could be emulated well in their design and measurement, we were able to predict findings reasonably well (r=0.94). I will discuss some implications for pharmacoepidemiology.
Bio: His research focuses on assessing the effectiveness and safety of biopharmaceuticals in clinical practice. He has developed and applied analytic methods to improve the accuracy of estimating causal treatment effects of newly-marketed drugs using complex digital healthcare databases. His work is published in >500 articles and is used for regulatory and coverage decision making around the globe. He is funded by NIH, PCORI, Burroughs Wellcome, and the FDA where he is also a voting consultant. He is Principal Investigator of the FDA Sentinel Innovation Center and co-leads the RCT-DUPLICATE initiative and ENCORE to understand when and how real-world evidence studies can reach robust conclusions to support regulatory and HTA decisions.
He is Past President of the International Society for Pharmacoepidemiology and is a Fellow of the American College of Epidemiology, the American College of Clinical Pharmacology, and the International Society for Pharmacoepidemiology. In 2024, he is Visiting Fellow of the Center for Advanced Studies, Ludwig-Maximilians-Universität, Munich.