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Multiple type I error concepts for clinical trials with overlapping populations

2026/07/31 by Remi Luschei, Werner Brannath
Mathematics · #stat.ME

paper · pdf

arxiv created 2026/07/31 · arxiv updated 2026/08/03

Abstract

The population-wise error rate (PWER) was introduced as a more liberal alternative to the family-wise error rate (FWER) for clinical trials with multiple, overlapping patient populations. These trials are particularly relevant in personalized medicine, which aims to find therapies tailored to specific patient subgroups. By controlling an average multiple type I error probability over all population strata, the PWER can substantially improve statistical power. However, one disadvantage of this concept is that the error probability for a given population can strongly depend on the presence or absence of other populations included in the analysis. To address this issue, we propose two modifications of the PWER that enforce individual error control either for all target populations, or for all possible unions of target populations. We call these approaches the PWER over the populations (PWER-P) and the PWER over population unions (PWER-U). We investigate the properties of these new error rates and compare them with the PWER and FWER in terms of type I error control and power.

Citations