Basket Trials: Review of Current Practice and Innovations for Future Trials
Cet article passe en revue les essais cliniques de type panier évaluant des thérapies ciblées en cancérologie puis examine les problèmes liés à leur modèle statistique et à l'interprétation des résultats
Advances in biology and immunology have elucidated genetic and immunologic origins of cancer. Innovations in sequencing technologies revealed that distinct cancer histologies shared common genetic and immune phenotypic traits. Pharmacologic developments made it possible to target these alterations, yielding novel classes of targeted agents whose therapeutic potential span multiple tumor types. Basket trials, one type of master protocol, emerged as a tool for evaluating biomarker-targeted therapies among multiple tumor histologies. Conventionally conducted within the phase II setting and designed to estimate high and durable objective responses, basket trials pose challenges to statistical design and interpretation of results. This article reviews basket trials implemented in oncology studies and discusses issues related to their statistical design and analysis.
Journal of Clinical Oncology , résumé, 2021