Online Program

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Tuesday, September 24
Tue, Sep 24, 2:45 PM - 4:00 PM
Thurgood Marshall East
Statistical Considerations in the Evaluation of Vaccine Efficacy and Effectiveness

Real-World Evidence Use in CBER (300934)

*Jennifer L Kirk, FDA/CBER 

Keywords: vaccines, real world evidence, real world data, regulatory statistics

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, with a focus on issues that are relevant to vaccines. 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 datasets?