Confident identification of subgroups from SNP testing in RCTs with binary outcomes.

ABSTRACT: In modern drug development, genotype information becomes more frequently collected in randomized controlled trials (RCTs) for individualized risk prediction and personalized medicine development. Finding single nucleotide polymorphisms (SNPs) that are predictive of differential treatment efficacy, measured by a clinical outcome, is fundamentally different and more challenging than the traditional association test for a quantitative trait. With the objective to confidently identify and infer genetic subgroups with enhanced treatment efficacy from a large RCT for an eye disease, age-related macular degeneration (AMD), where the clinical endpoint is binary (progressed or not), we propose a novel SNP-testing procedure for binary clinical outcomes. Specifically, we formulate four contrasts to s

SUBMITTER: Wei Y 

PROVIDER: S-EPMC11927680 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature