B1233
Title: Leveraging real world data and real world evidence in clinical trial design and analysis and its causal implications
Authors: Chenguang Wang - Regeneron Pharmaceuticals (United States) [presenting]
Abstract: Incorporating real-world data (RWD) in regulatory decision-making demands more than mixing RWD with investigational clinical trial data. The RWD must undergo appropriate analysis for deriving the right real-world evidence (RWE). Moreover, such analysis should be integrated with the design and analysis of the investigational study for regulatory decision-making. The standard clinical trial toolbox does not offer ready solutions for such tasks. Therefore, there is an unmet need for sound clinical trial design and analysis for leveraging RWE in clinical evaluations in the context of regulatory decision-making. Recently, methods leveraging external RWD in clinical trial design and analysis in the context of regulatory decision-making are proposed. The methods use propensity score or entropy balancing to pre-select a subset of RWD patients that are similar to those in the investigational study. The methods are reviewed, and the underlying causal assumptions that the methods require are discussed.
