Online Program

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Wednesday, September 25
Wed, Sep 25, 10:45 AM - 12:00 PM
Currently Used Statistical Models in Phase II Basket Trial Designs

Basket Trials in Development of Tumor-Agnostic, Biomarker-Based Indications (301027)

*Shenghui Tang, FDA 

Keywords: Master Protocols, Basket Trial Designs, Tumor-Agnostic, Biomarker-Based Indications

A tumor-agnostic treatment is used to treat patients with a very specific molecular alteration regardless of tumor type. Recently, pembrolizumab was approved for the treatment of patients with a molecular alteration called microsatellite instability-high (MSI-H) or DNA mismatch repair deficiency (dMMR); larotrectinib was approved for the treatment of patients with a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation. To develop tumor-agnostic, biomarker-based indications, novel trial designs with information borrowing across different tumor types have been proposed. They have allowed us to pool patients for a regulatory endpoint. This talk will discuss our review experience with basket trial designs using examples. It will also review FDA’s draft guidance on master protocols (Master Protocols: Efficient Clinical Trial Design Strategies To Expedite Development of Oncology Drugs and Biologics).