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Johns Hopkins University | PH.140.689

Adaptive Enrichment Designs for Confirmatory Randomized Trials: Methods and Software

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Provides an overview of the strengths and limitations of randomized trial designs that adaptively change enrollment criteria during a trial (adaptive enrichment designs) and have the potential to provide improved information about which subpopulations benefit from new treatments. Explains recent advances in statistical methods for these designs, and presents adaptive design software planning tools. Discusses FDA guidance documents on adaptive designs. Examines methods for improving precision of estimators of the average treatment effect, by leveraging information in baseline variables; these methods can be used in adaptive designs as well as standard (non-adaptive) trial designs.

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