Using short-term endpoints to improve interim decision making and trial duration in two-stage phase II trials with nested binary endpoints.

ABSTRACT: In oncology, phase II clinical trials are often planned as single-arm two-stage designs with a binary endpoint, for example, progression-free survival after 12 months, and the option to stop for futility after the first stage. Simon's two-stage design is a very popular approach but depending on the follow-up time required to measure the patients' outcomes the trial may have to be paused undesirably long. To shorten this forced interruption, it was proposed to use a short-term endpoint for the interim decision, such as progression-free survival after 3 months. We show that if the assumptions for the short-term endpoint are misspecified, the decision-making in the interim can be misleading, resulting in a great loss of statistical power. For the setting of a binary endpoint with nested measu

SUBMITTER: Zocholl D 

PROVIDER: S-EPMC10540486 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature