Assessing the impact of efficacy stopping rules on the error rates under the multi-arm multi-stage framework.

ABSTRACT:

Background

The multi-arm multi-stage framework uses intermediate outcomes to assess lack-of-benefit of research arms at interim stages in randomised trials with time-to-event outcomes. However, the design lacks formal methods to evaluate early evidence of overwhelming efficacy on the definitive outcome measure. We explore the operating characteristics of this extension to the multi-arm multi-stage design and how to control the pairwise and familywise type I error rate. Using real examples and the updated nstage program, we demonstrate how such a design can be developed in practice.

Methods

We used the Dunnett approach for assessing treatment arms when conducting comprehensive simulation studies to evaluate the familywise error rate, with and without interim efficacy looks on

SUBMITTER: Blenkinsop A 

PROVIDER: S-EPMC6442021 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature