Journal
STATISTICS IN BIOPHARMACEUTICAL RESEARCH
Volume 2, Issue 1, Pages 72-83Publisher
AMER STATISTICAL ASSOC
DOI: 10.1198/sbr.2010.08039
Keywords
Clinical trials; Enriched clinical trials; Sensitive population; Type I error rate control; Weighted power
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This article deals with clinical trials with a sensitive subpopulation of patients, that is, a subgroup that is more likely to benefit from the treatment than the overall population. Given a sensitive subgroup defined by a prespecified classifier, for example, a clinical marker or pharma-cogenomic marker, the trial's outcome is declared positive if the treatment effect is established in the overall population or in the subgroup. We provide a summary of key considerations in clinical trials with a sensitive subgroup, including multiplicity and enrichment adjustments as well as optimality considerations in the analysis strategy. The methodology proposed in this article is illustrated using a neuroscience clinical trial and its operating characteristics are assessed via a simulation study.
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