An adaptive design for updating the threshold value of a continuous biomarker.

ABSTRACT: Potential predictive biomarkers are often measured on a continuous scale, but in practice, a threshold value to divide the patient population into biomarker 'positive' and 'negative' is desirable. Early phase clinical trials are increasingly using biomarkers for patient selection, but at this stage, it is likely that little will be known about the relationship between the biomarker and the treatment outcome. We describe a single-arm trial design with adaptive enrichment, which can increase power to demonstrate efficacy within a patient subpopulation, the parameters of which are also estimated. Our design enables us to learn about the biomarker and optimally adjust the threshold during the study, using a combination of generalised linear modelling and Bayesian prediction. At the final analy

SUBMITTER: Spencer AV 

PROVIDER: S-EPMC5378309 | biostudies-literature | 2016 Nov

REPOSITORIES: biostudies-literature