Examples of Applying Causal-Inference Roadmap to RWE Clinical Studies

• Activity Number: 78 - Key Steps in Deriving Real-World Evidence from Analysis of Real-World Data
• Type: Topic-Contributed
• Date/Time: Monday, August 9, 2021 : 10:00 AM to 11:50 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Martin Ho* and Yixin Fang
• Companies: Google and AbbVie
• Keywords: Causal inference; Real-World Evidence; Clinical studies; Observational studies; External control; Informative censoring

Abstract

Built upon the published RWE Causal Inference Roadmap (DOI: 10.1080/19466315.2021.1883475), the ASA Biopharmaceutical Section Real-world Evidence Scientific Working Group will use illustrative examples to demonstrate how to apply each step of the Roadmap in design, analysis, and interpretation of RWE clinical studies. Additional values of these steps to the conventional statistical practices will be discussed. In these examples, single-arm clinical studies are compared against external controls in various scenarios and datasets are simulated for illustration purpose only. The presentation will begin with a brief review of the Roadmap and motivation for its application. Then, it will introduce a simple case of outcome at a fixed timepoint with a single treatment at baseline. Next, it will demonstrate how to apply the roadmap to a longitudinal case with multiple periods and informative censoring.