Emerging Opportunities for Utilizing Real-World Evidence to Impact Drug Development and Regulatory Decision-Making

• Activity Number: 24 - Emerging Opportunities for Utilizing Real-World Evidence to Impact Drug Development and Regulatory Decision-Making
• Type: Topic Contributed
• Date/Time: Sunday, July 28, 2019 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Weili He* and Jessica M Franklin* and David Martin * and David Benkeser*
• Companies: AbbVie and Brigham and Women’s Hospital and Harvard Medical School and Emory and FDA
• Keywords: Real world data; Real world evidence; Regulatory decision

Abstract

Real-world data (RWD) and RW world evidence (RWE), which have been increasingly used within the healthcare system, previously had limited utility in drug development as data and evidence from traditional randomized trials are deemed to be gold standard to determine the drug efficacy. However, under the 21st Century Cures Act signed into law in Dec, 2016, the FDA is directed to develop a program to evaluate how RWE can potentially be used to support approval of new indications for approved drugs or to support/satisfy post-approval study requirements. This brings new opportunities to utilize statistical innovations and advances to address data quality as well as to establish causal inference based on RWD for regulatory decision-making. Our fits well with the JSM 2019 theme “Statistics: Making an Impact” as it demonstrates how the era of big data is shifting the statistical framework for regulatory science and the critical role of statistics in making valid regulatory decisions which impact on real patient’s lives. The diverse panel includes David Martin, FDA CDER, Weili He, AbbVie, Mark van der Laan, UC Berkeley, and Jessica Franklin, Harvard Medical School