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Abstract:
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There seems to be growing interest in patient subpopulation enrichment whether pursued at study baseline or in an ongoing clinical trial for its potential to achieve the goal of personalized medicine. Adaptive enrichment has been proposed in multiple disease areas in drug development programs. According to the FDA draft guidance on enrichment strategies for drugs and biologics, in principle, enrichment can help to reduce patient heterogeneity, select a high risk patient subset or identify a subgroup of patients who are more likely to respond to treatment. In this presentation, we give a brief overview on adaptive enrichment and the methodologies that are growing in statistical literature. An approved neurologic drug development case example, which was planned to adapt two design elements, i.e., patient population adaptation and statistical information adaptation, will be shared. We will articulate the challenges in the implementation of a confirmatory adaptive enrichment trial and what population the treatment effect applies. We will also assess the consistency of treatment effect before and after statistical information adaptation.
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