Reaplicating clinical trial data using RWD

Background: As part of the FDA's efforts to lay the groundwork for using RWE on product effectiveness and safety to support regulatory approval for new or expanded indications, several demonstration projects were initiated to assess the feasibility, utility and methodological challenges of replicating clinical trial results using real‐world data (RWD) stemming from claims and electronic health records. The idea is to first demonstrate the validity of the use of RWD analysis techniques by replicating clinical trial results that were already known; then the evidence on the utility of these techniques can be expanded for RWD usage to predict clinical trial results or to serve as an external control arm for single arm trials. In addition to the FDA project, several demonstration projects by other sponsors also have been presented and showed promising results. This symposium is intended to have focused presentations and panel discussion with panel members who have been contributing in the various initiatives in this area to provide updates on their projects and insights gained from the work so far. In addition, the regulatory implications of these findings and insights will be discussed.

Objectives: 1. To provide an overview of completed and ongoing projects replicating clinical trial results using RWD a. To discuss the methodologies that were used in the various projects b. To identify important considerations for control of bias and confounding when conducting such analyses 2. To provide insights on the potential applications of the methodologies and learnings from the various projects for regulatory submissions using RWE a. From regulator's perspective b. From researcher's perspective.

Description: The symposium will start with a brief introduction of the background and objectives of the symposium (Solomon Iyasu, 5 minutes). An overview of the landscape, updates of the demonstration projects and regulatory applications will be provided (David Martin, 20 minutes). The findings of the demonstration projects will be discussed (Jessica Franklin, 20 minutes). Methodologic and study design considerations will be discussed (Miriam Sturkenboom, 20 minutes). Panel discussion (all, 20 minutes) and a brief conclusion will then follow (Solomon Iyasu and Kai‐Li Liaw, 5 minutes).