Adaptive seamless clinical trials using early outcomes for treatment or subgroup selection: Methods, simulation model and their implementation in R.

ABSTRACT: Adaptive seamless designs combine confirmatory testing, a domain of phase III trials, with features such as treatment or subgroup selection, typically associated with phase II trials. They promise to increase the efficiency of development programmes of new drugs, for example, in terms of sample size and/or development time. It is well acknowledged that adaptive designs are more involved from a logistical perspective and require more upfront planning, often in the form of extensive simulation studies, than conventional approaches. Here, we present a framework for adaptive treatment and subgroup selection using the same notation, which links the somewhat disparate literature on treatment selection on one side and on subgroup selection on the other. Furthermore, we introduce a flexible and ef

SUBMITTER: Friede T 

PROVIDER: S-EPMC8614126 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature