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Basket Trials: Review of Current Practice and Innovations for Future Trials
Brian P Hobbs1, Roberto Carmagnani Pestana2, Emily C Zabor3
1Dell Medical School, The University of Texas at Austin, Austin, TX.
Abstract:
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.
Insights
Basket trials evaluate targeted cancer therapies across various tumor types. This review discusses their statistical design and interpretation challenges in oncology research.
Area of Science:
- Oncology
- Immunology
- Genetics
Background:
- Advances in cancer biology and immunology reveal shared genetic and immune traits across distinct cancer histologies.
- Pharmacologic innovations enable targeted therapies for multiple tumor types based on identified molecular alterations.
Purpose of the Study:
- To review the implementation of basket trials in oncology.
- To discuss the statistical design and analysis challenges associated with basket trials.
Main Methods:
- Review of oncology studies utilizing basket trial designs.
- Discussion of statistical considerations for phase II basket trials.
Main Results:
- Basket trials are a key tool for evaluating biomarker-targeted therapies across multiple histologies.
- Challenges exist in the statistical design and interpretation of results from basket trials.
Conclusions:
- Basket trials offer a novel approach for testing targeted agents in diverse cancer types.
- Addressing statistical complexities is crucial for the effective use and interpretation of basket trial data.
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