Online Program

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Wednesday, September 25
Wed, Sep 25, 2:45 PM - 4:00 PM
Thurgood Marshall East
Statistical Considerations and Methods: Precision Medicine Clinical Trials for Therapy Agnostic to Cancer Site

A Novel Statistical Method for Precision Medicine Agnostic to Cancer Sites (301030)

*Meijuan Li, FDA/CDRH 

The transition to precision medicine approaches in cancer has sparked a much-needed shift in the design and implementation of clinical trials. Recently, a new promising approach in precision medicine is developing therapy for patients with specific molecular characteristic which are agnostic to cancer site. In this approach, rather than requiring separate drug and device programs for each type of cancer, it will be based on biomarkers irrespective of organ site or histology. For example, the Food and Drug Administration (FDA) approved pembrolizumab, a programmed death 1 (PD-1) inhibitor, for the treatment of adult and pediatric patients with unresectable or metastatic, microsatellite-instability–high (MSI-H) or mismatch-repair–deficient (dMMR) solid tumors, regardless of tumor site or histology. There are many statistical issues for this new precision medicine approach from both device and therapy perspectives; yet the issues are less known to the society. In this section, we will discuss a novel statistical method in clinical trial designs and data analysis for this new emerging precision medicine approach.