Abstract
Differential effectiveness of treatments across subgroups defined by pretreatment variables are of increasing interest, particularly in the expanding research field of pharmacogenomics. When the pretreatment variable is difficult to obtain or expensive to measure, but can be assessed at the end of the study using stored samples, nested case-control and case-cohort methods can be used to reduce costs in large efficacy trials with rare outcomes. Case-only methods are even more efficient, and reliable under a range of circumstances.
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