The Biostatistical Landscape of Causal Inference Frameworks of Clinical Studies using RWD/RWE

• Activity Number: 525 - Translating Real-World Data into Real-World Evidence for Regulatory Decisions and Medical Practice: Opportunities and Challenges
• Type: Invited
• Date/Time: Thursday, August 6, 2020 : 1:00 PM to 2:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Martin Ho*
• Companies: FDA-CBER
• Keywords: Real-World Evidence; FDA ; Real-World Data; Clinical Trial Design; causal methods; medical products

Abstract

Surging number of available real-world data (RWD) sources (e.g., EHRs, claims data, registries) have attracted many experts from multiple disciplines to actively exploring and developing ways to translate RWD into Real-World Evidence (RWE) to bridge the current gaps in clinical trial enterprise. However, the statistical community can participate more fully in this novel research area. Therefore, the ASA BIOP Section RWE Scientific Working Group (SWG) was chartered in 2018, aiming to use statistics generate RWE designed to inform regulatory decisions of medical products. The SWG has two work streams and they have been focusing on using RWE: (a) to modify existing labels of medical products and (b) to inform better clinical study designs. In this presentation, early study results of the SWG will be presented. This presentation will first discuss the current statistical landscape on using RWD and other external data, followed by some examples, and then concluded with some identified topics where the SWG can make most substantial impacts and future research agendas.