Progress in the areas of genomics, disease pathways, and drug discovery has advanced into clinical and translational cancer research. The latest innovations in clinical trials have followed with master protocols, which are defined by inclusive eligibility criteria and devised to interrogate multiple therapies for a given tumor histology and/or multiple histologies for a given therapy under one protocol. The use of master protocols for oncology has become more common with the desire to improve the efficiency of clinical research and accelerate overall drug development. Basket trials have been devised to ascertain the extent to which a treatment strategy offers benefit to various patient subpopulations defined by a common molecular target. Conventionally conducted within the phase II setting, basket designs have become popular as drug developers seek to effectively evaluate and identify preliminary efficacy signals among clinical indications identified as promising in preclinical study. This article reviews basket trial designs in oncology settings and discusses several issues that arise with their design and analysis.
Bibliographical noteFunding Information:
J.S.K. is supported in part by the National Institute on Drug Abuse (R01 DA046320). M.J.K. was supported in part by the Yale Comprehensive Cancer Center (P30 CA016359). B.P.H. was supported in part by the Case Comprehensive Cancer Center (P30 CA043703).
© 2019 by American Society of Clinical Oncology