Review of Phase II Basket Trials for Precision Medicine

The advancement of molecular biology and genomic technologies has made paradigm shift from conventional paradigm with cytotoxic agents to precision medicine using molecularly targeted agents. The premise of precision medicine is that only a subset of patients is sensitive to and benefits from a given treatment. Under this new paradigm for clinical development, studies for drug activity require to consider inter-patient heterogeneity and intra-patient heterogeneity. Basket trials evaluate the effect of the targeted therapy with a certain genetic mutation across cancer types. The basket study is increasingly becoming recognized as an attractive approach for drug development under the precision medicine paradigm. It leads to novel statistical methodologies development and conducting clinical trials based on the basket study. In this review paper, we describe the basket trials with statistical methods and examples.

Keywords: Basket trials; Master protocols; Phase II trials; Precision medicine