Statistical Consideration for Fit-For-Use Real-World Data to Support Regulatory Decision Making in Drug Development

• Activity Number: 411 - Addressing Key Issues to Promote the Uptake of Using RWE/D for Regulatory Decisions
• Type: Topic Contributed
• Date/Time: Wednesday, August 10, 2022 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Weili He*
• Companies: AbbVie
• Keywords: Real-world evidence; Real-world data; fit-for-purpose; data-drive approach

Abstract

A Real-World Evidence (RWE) scientific working group of the American Statistical Association Biopharmaceutical Section has been reviewing the statistical considerations for the generation of real-world evidence to support regulatory decision making. As part of the effort, the working group is addressing the fitness-for-use of real-world data (RWD). RWD may be used in a variety of ways and study designs including in randomized studies, externally controlled studies, and purely observational studies. The use of RWD poses unique issues surrounding study integrity, transparency, and reproducibility. The working group proposes a data-driven approach framework for determining the fit-for-use of RWD.