Adaptive enrichment trial designs using joint modelling of longitudinal and time-to-event data.

ABSTRACT: Adaptive enrichment allows for pre-defined patient subgroups of interest to be investigated throughout the course of a clinical trial. These designs have gained attention in recent years because of their potential to shorten the trial's duration and identify effective therapies tailored to specific patient groups. We describe enrichment trials which consider long-term time-to-event outcomes but also incorporate additional short-term information from routinely collected longitudinal biomarkers. These methods are suitable for use in the setting where the trajectory of the biomarker may differ between subgroups and it is believed that the long-term endpoint is influenced by treatment, subgroup and biomarker. Methods are most promising when the majority of patients have biomarker measurements

SUBMITTER: Burdon AJ 

PROVIDER: S-EPMC11577695 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature