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Activity Number: 656 - Subgroup Evaluations in Advancing Drug Development
Type: Topic Contributed
Date/Time: Thursday, August 2, 2018 : 10:30 AM to 12:20 PM
Sponsor: Biopharmaceutical Section
Abstract #329231
Title: Evaluation of Treatment Effect: Beyond Traditional Subgroups
Author(s): Yeh-Fong Chen*
Companies: US FDA
Keywords: subgroups; two-stage designs; composite endpoints; SPCD

Treatment effects in related subpopulations are frequently investigated in clinical trials in order to increase efficiency and feasibility. Analysis of subpopulations may influence the interpretation of the overall study results. For example, in trials that include both adolescents and adults the number of adolescents may be so low as to hinder approval beyond the adult population. Another example is two-stage designs that combine information from both stages to make inference but it is important to analyze each stage separately. Sequential parallel comparison designs (SPCD) is an example where we look at the effect for the entire population not only placebo non-responders. Moving our focus from study populations to study endpoints, trials may use composite endpoints when the expected incidence rate of one or more component events is too low to allow a study of reasonable size to have adequate power. However, analyses of the components can influence interpretation of the overall study results. We will discuss examining components of a composite endpoint to ensure it is not driven by a single component and the difficulties that can arise when determining if an issue exists or not.

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

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