ISSUE: Regulatory agencies have accepted external controls with RWD in certain cases when evaluating drug approvals and indication expansions. However, with focus on RWE, will external control arms become more acceptable? Questions frequently arise around the use RWD and analytic methods to develop sound external controls in lieu of a randomized-controlled trials (RCTs).

OVERVIEW: Moderator and panelists representing the pharmaceutical industry, academic institutions, regulatory policy organizations, and RWE analytics providers will discuss data and methods considerations for developing external control arms. The moderator will begin by reviewing and defining key terms - e.g., single arm trials, external controls with RWD. Panelists then will discuss recent examples where the EMA, FDA, and other regulators have accepted submissions using external controls, and where there are similarities or differences to note across these examples. Industry and academic panelist(s) will discuss some successes and challenges they have experienced with RWD vs. RCT data, and what their organizations are doing to advance the approach of RWD control arms. We also will provide a deeper dive into key analytical and statistical methods that can be used to effectively match patients for external control analyses, and will discuss regulatory and policy methods and feedback. Finally, panelists will discuss ways to ensure analysis quality when using external controls. Moderator will facilitate questions from the audience and panel discussion.