Tutorial- Tuesday 9:00 AM EDT
Title: Randomized Pragmatic Clinical Trials Utilizing RWD: Myths & Realities
Presenters: Frank Rockhold, Duke University

Abstract:
The Substantial Evidence Amendment to FDA Regulations was updated in 2019 to consider RWD as an adjunct to Randomized Controlled Clinical Trials (RCTs) “to consider such data and evidence to constitute substantial evidence”. There is general agreement among experts that the use of these existing healthcare data holds promise for making randomized trials more efficient and generalizable. Regulators and the academic community have outlined a framework for evaluating the use of real-world data (RWD) to support regulatory decision-making using pragmatic clinical trials (PCTs), which are randomized trials designed to inform clinical or policy decisions by assessing the real-world effectiveness of an intervention. These benefits of relying on real-world data sources must be balanced against a clear understanding of the data provenance and characteristics of the electronic health record (EHR) and claims data used in PCTs. Data collected primarily for the health care of the patient can limit their fitness for research purposes. This talk will examine the role of EHR data in PCT’s and review some of the issues involved.

Bio:
Frank Rockhold is currently Professor of Biostatistics and Bioinformatics at the Duke University Medical Center, Affiliate Professor of Biostatistics at Virginia Commonwealth University, and Managing Partner of HunterRockhold, Inc. He has diverse research interests and consulting experience including trial design, data monitoring, benefit/risk, and pharmacovigilance and has been a leader in the scientific community in promoting data disclosure and transparency in clinical research. His career includes numerous senior management positions in industry, most recently as Chief Safety Officer and senior vice president for pharmacovigilance at GSK. Dr. Rockhold has served as Chairman of the board for CDISC and president of the Society for Clinical Trials. He also served on the inaugural PCORI Clinical Trials Advisory Board and is currently Chairman of the board of the Frontier Science Research Foundation and an advisor to EMA. Frank serves on numerous Independent Data Monitoring Committees.

Dr. Rockhold holds a BA in Statistics from The University of Connecticut, an ScM in Biostatistics from The Johns Hopkins University, and a PhD in Biostatistics from the Medical College of Virginia at Virginia Commonwealth University. He is an Elected Fellow of both the American Statistical Association, the Society for Clinical Trials, and the Royal Statistical Society. He is an Accredited Professional Statistician, PStat®, and a Chartered Statistician, CStat.