Keywords: Real world data; estimand; real world evidence
“Real world data” (RWD) from clinical practice and medical product utilization – outside of clinical trials – offers an increasingly promising source of data for use in a range of study designs to provide evidence for the benefits, risks, and use of medical products. The use of these data holds great potential to increase efficiency and improve clinical development and life cycle management of medical products. However, many challenges remain on how RWD could be translated into robust evidence, real-world evidence (RWE), to support regulatory decisions. The ASA Biopharmaceutical Section RWE SWG WS1 was charged to investigate statistical aspects of the use of RWD and RWE for label expansion. Under this scope, we reviewed and explored key issues on the appropriate use of RWD and RWE, including: regulatory, scientific, and ethical aspects; data sources and data quality; outcome, exposure, and covariate ascertainment and validation; estimands and missing data; and control of confounding. The output from WS1 will potentially have significant impact in the field and move the dialogue on RWD/RWE usage forward. In this presentation, we will present the progress to date on the WS1 work and any recommendations from the working group, if available.