Potential Statistical Issues Between Designers and Regulators in Confirmatory Basket, Umbrella, and Platform Trials

Abstract

Platform and basket trial have the potential to alter the landscape of drug development in many disease areas. Although these trials create many efficiencies compared with simple trials, they also create new statistical challenges for regulators. I highlight some of these new issues and discuss the regulatory challenges emerging from these trials. I highlight some critical points made by Collignonet al.,(1)while providing some conflicting views on these statistical challenges.