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Activity Number: 493 - Leveraging Historical Data and Real World Evidence in Drug Development Program Evaluation
Type: Topic Contributed
Date/Time: Wednesday, July 31, 2019 : 10:30 AM to 12:20 PM
Sponsor: Health Policy Statistics Section
Abstract #306826
Title: Real World Evidence Use in CBER
Author(s): Jennifer Kirk*
Companies: FDA, Center for Biologics Evaluation and Research (CBER)
Keywords: Real World Evidence; Regulatory Statistics; Real World Data

Since the passage of PDUFA VI, there has been significantly increased interest in the use of real-world data and evidence (RWE) in the regulatory setting. In this talk, I present some examples of RWE use in CBER, such as BEST, SENTINEL, and post-marketing studies. I will also discuss issues that have arisen in these uses that can inform the current discussion. Topics covered include: How can we describe or quantify the strength of evidence provided by a study design? What is the role of existing studies in the literature that might be relevant? How can we mitigate the risk that a trial fails because of lack of power or other design problems that emerge during analysis? How can we handle the submission and use of extremely large data sets?

Authors who are presenting talks have a * after their name.

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