Online Program

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Wednesday, September 25
Wed, Sep 25, 2:45 PM - 4:00 PM
Estimands for Safety and Benefit-Risk Evaluation

Creating a Benefit-Risk Estimand from My Drug Program’s Efficacy and Safety Estimands (300982)

*Susan P Duke, FDA/CDER/OB 

Keywords: estimands, safety, benefit risk

The practice of prespecifying estimands has been encouraged to more fully characterize population-level parameters being targeted by analyses of the primary efficacy endpoint with respect to population under study, handling of intercurrent events, and the primary summary measure. This structured thinking benefits industry, as well as regulatory statisticians and clinicians, by being more precise and transparent about the primary and secondary goals of the study. Estimands are also being defined for safety. This talk will use an example indication, beginning with a benefit-risk value tree. We will then discuss how a benefit-risk estimand may be created from the efficacy and safety estimands. The structured thinking of value trees and estimands will demonstrate how benefit-risk assessment may be considered in the planning phase of a clinical program, based on the scientific questions that require elucidation in this example clinical program.