Power/sample size calculations for assessing correlates of risk in clinical efficacy trials.

ABSTRACT: In a randomized controlled clinical trial that assesses treatment efficacy, a common objective is to assess the association of a measured biomarker response endpoint with the primary study endpoint in the active treatment group, using a case-cohort, case-control, or two-phase sampling design. Methods for power and sample size calculations for such biomarker association analyses typically do not account for the level of treatment efficacy, precluding interpretation of the biomarker association results in terms of biomarker effect modification of treatment efficacy, with detriment that the power calculations may tacitly and inadvertently assume that the treatment harms some study participants. We develop power and sample size methods accounting for this issue, and the methods also account fo

SUBMITTER: Gilbert PB 

PROVIDER: S-EPMC4965346 | biostudies-literature | 2016 Sep

REPOSITORIES: biostudies-literature