Flexible Spline Models for Blinded Sample Size Reestimation in Event-Driven Clinical Trials.

ABSTRACT: In event-driven trials, the target power under a certain treatment effect is maintained as long as the required number of events is obtained. The misspecification of the survival function in the planning phase does not result in a loss of power. However, the trial might take longer than planned if the event rate is lower than assumed. Blinded sample size reestimation (BSSR) uses non-comparative interim data to adjust the sample size if some planning assumptions are wrong. In the setting of an event-driven trial, the sample size may be adjusted to maintain the chances to obtain the required number of events within the planned time frame. For the purpose of BSSR, the survival function is estimated based on the interim data and often needs to be extrapolated. The current practice is to fit st

SUBMITTER: Mori T 

PROVIDER: S-EPMC11893375 | biostudies-literature | 2025 Mar-Apr

REPOSITORIES: biostudies-literature