Discussions and Challenges in Multi-Arm Multi-Stage (MAMS) Designs

Music City Center 

Multi-Arm Multi-Stage (MAMS) clinical trial designs are seamless Phase 2/3 designs that have multiple treatment arms and concurrent control arm. These designs allow treatment selection at early stage and uses these early stage patients' data in making confirmatory decision at the later stage; thus, saving time and resources. While the designs have been around for some time, they are not popular because of two major challenges. The first challenge is controlling the overall Type I error which is more complicated than traditional two-arm design due to various multiplicity issues. The second challenge is the choice of appropriate endpoint for treatment selection and how that choice may impact the confirmatory outcome. In this presentation, we will discuss these challenges and provide insights on the solutions that one may use to overcome these challenges. Simulation studies will be presented to show benefits of the proposed solutions