All Times EDT
Keywords: Tumor-agnostic indications; basket trials; type I error control
Recent years have witnessed a few successful drug approvals with tumor agnostic pathway. Meanwhile, the limited numbers of approvals in this pathway may indicate tremendous challenges from translational, regulatory and statistical perspectives. In this presentation, ASA BIOP Oncology Methods SWG Master Protocol Sub-team and DIA Innovative Design Scientific Working Group (IDSWG) oncology sub-team will provide a deep dive into the cases leading to successful approval and a few case studies with investigational drug yet approved and share some implications and lessons learned from the working group. Statistical considerations such as controlling type I error, addressing homogeneous efficacy in certain tumor types with small sample sizes, and defining/handling potential negative data post accelerated approval will be discussed. Regulatory challenges will also be reviewed and shared with case examples.