Interim analysis incorporating short- and long-term binary endpoints.

ABSTRACT: Designs incorporating more than one endpoint have become popular in drug development. One of such designs allows for incorporation of short-term information in an interim analysis if the long-term primary endpoint has not been yet observed for some of the patients. At first we consider a two-stage design with binary endpoints allowing for futility stopping only based on conditional power under both fixed and observed effects. Design characteristics of three estimators: using primary long-term endpoint only, short-term endpoint only, and combining data from both are compared. For each approach, equivalent cut-off point values for fixed and observed effect conditional power calculations can be derived resulting in the same overall power. While in trials stopping for futility the type I error

SUBMITTER: Niewczas J 

PROVIDER: S-EPMC6590444 | biostudies-literature | 2019 May

REPOSITORIES: biostudies-literature