Three-outcome designs for external pilot trials with progression criteria.

ABSTRACT:

Background

Whether or not to progress from a pilot study to a definitive trial is often guided by pre-specified quantitative progression criteria with three possible outcomes. Although the choice of these progression criteria will help to determine the statistical properties of the pilot trial, there is a lack of research examining how they, or the pilot sample size, should be determined.

Methods

We review three-outcome trial designs originally proposed in the phase II oncology setting and extend these to the case of external pilots, proposing a unified framework based on univariate hypothesis tests and the control of frequentist error rates. We apply this framework to an example and compare against a simple two-outcome alternative.

Results

We find that three-outcome

SUBMITTER: Wilson DT 

PROVIDER: S-EPMC11446087 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature