Adaptive Phase II Trials

Abstract

Phase II trials are designed to obtain preliminary efficacy information about a new therapy in order to assess whether the new therapy should be tested in definitive (phase III) trials. Adaptive trial designs allow the design of a trial to be changed during its conduct, possibly using accruing outcome data. Adaptations to phase II trials considered in this chapter include formal interim monitoring, phase II/III trial designs, adaptations related to biomarker subgroups, sample size reassessment, outcome-adaptive randomization, and adaptive pooling of outcome results across patient subgroups. Adaptive phase II trials allow for the possibility of trials reaching their conclusions earlier, with more patients being treated with therapies that have activity for them.