Interim futility analysis with intermediate endpoints.

Publication Type:

Journal Article


Clinical trials (London, England), Volume 5, Issue 1, p.14-22 (2008)


2008, Clinical Trials, Phase III as Topic, Computer Simulation, Decision Making, Endpoint Determination, Humans, Medical Futility, Neoplasms, Public Health Sciences Division, Research Design, Survival Analysis


Interim analysis of Phase III trials typically includes testing for both efficacy and futility. Futility testing is commonly performed on the primary outcome at very low levels (e.g., one-sided alpha=0.0025) at one or two times before final analysis. When overall survival is the primary outcome and events accrue slowly, and if a suitable intermediate endpoint is available, then using this endpoint for interim futility testing may yield a higher probability of stopping early for futility in the absence of any treatment effect.