Adjusting for treatment selection in phase II/III clinical trials with time to event data.

ABSTRACT: Phase II/III clinical trials are efficient two-stage designs that test multiple experimental treatments. In stage 1, patients are allocated to the control and all experimental treatments, with the data collected from them used to select experimental treatments to continue to stage 2. Patients recruited in stage 2 are allocated to the selected treatments and the control. Combined data of stage 1 and stage 2 are used for a confirmatory phase III analysis. Appropriate analysis needs to adjust for selection bias of the stage 1 data. Point estimators exist for normally distributed outcome data. Extending these estimators to time to event data is not straightforward because treatment selection is based on correlated treatment effects and stage 1 patients who do not get events in stage 1 are foll

SUBMITTER: Khan JN 

PROVIDER: S-EPMC10098876 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature