Sample Size for Enriched Biomarker Designs With Measurement Error for Time-to-Event Outcomes.

ABSTRACT: A major emphasis in personalized medicine is to optimally treat subgroups of patients who may benefit from certain therapeutic agents. One relevant study design is the targeted design, in which patients have consented for their specimens to be obtained at baseline and the specimens are sent to a laboratory for assessing the biomarker status prior to randomization. Here, only biomarker-positive patients will be randomized to either an experimental or the standard of care arms. Many biomarkers, however, are derived from patient tissue specimens, which are heterogeneous leading to variability in the biomarker levels and status. This heterogeneity would have an adverse impact on the power of an enriched biomarker clinical trial. In this article, we show the adverse effect of using the uncorrec

SUBMITTER: Guo S 

PROVIDER: S-EPMC12683776 | biostudies-literature | 2025 Sep-Oct

REPOSITORIES: biostudies-literature