Robustness of testing procedures for confirmatory subpopulation analyses based on a continuous biomarker.

ABSTRACT: With the advent of personalized medicine, clinical trials studying treatment effects in subpopulations are receiving increasing attention. The objectives of such studies are, besides demonstrating a treatment effect in the overall population, to identify subpopulations, based on biomarkers, where the treatment has a beneficial effect. Continuous biomarkers are often dichotomized using a threshold to define two subpopulations with low and high biomarker levels. If there is insufficient information on the dependence structure of the outcome on the biomarker, several thresholds may be investigated. The nested structure of such subpopulations is similar to the structure in group sequential trials. Therefore, it has been proposed to use the corresponding critical boundaries to test such nested

SUBMITTER: Graf AC 

PROVIDER: S-EPMC6566459 | biostudies-literature | 2019 Jun

REPOSITORIES: biostudies-literature