Basket Trials and the MD Anderson Precision Medicine Clinical Trials Platform

Abstract Precision medicine incorporates information regarding tumor biology involved in patients' carcinogenesis and individualized treatment of patients using drugs that inhibit the molecular basis of their disease. Implementation of precision medicine accelerated the drug approval process, translating discoveries in basic science and biotechnology into patient care. Clinical trials with innovative or adaptive design including “basket” and “umbrella” trials explore personalized therapies against in selected tumor types and/or across tumor types. In 2007, we started the Initiative for Molecular Profiling and Advanced Cancer Therapy, the first precision medicine program across tumor types. We demonstrated that therapy matched to patients' tumor molecular profiling is associated with improved rates of response, progression-free survival, and overall survival compared with nonmatched targeted therapy. We have now entered a new era of precision medicine that includes comprehensive tumor testing and multiple innovative modalities hoping to overcome the complexity of tumor biology to improve patient outcomes.