B0659
Title: Biomarker adaptive two-stage design for Phase II targeted therapy
Authors: Zheyu Wang - Johns Hopkins University (United States) [presenting]
Gary Rosner - Johns Hopkins University (United States)
Chenguang Wang - Regeneron Pharmaceuticals (United States)
Abstract: The success of drug development in targeted therapy critically relies on the accurate selection of a sensitive patient population, primarily based on biomarker levels. However, during the planning stage of a Phase II study, the lack of a defined threshold value for identifying the sensitive patient population poses a significant challenge for adopting existing biomarker-guided designs. To tackle this issue, a two-stage design is presented that enables the simultaneous selection of biomarker thresholds and evaluation of treatment efficacy, while also accommodating scenarios where the drug exhibits efficacy across the entire patient population. This approach addresses the need for adaptive trial designs that integrate biomarker information, ensuring optimal patient selection and maximizing treatment outcomes. Real-world case studies are discussed, and the implementation and interpretation of this biomarker adaptive two-stage design is illustrated.
