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Park JJH, Hsu G, Siden EG, et al. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin. 2020;70(2):125-137doi: 10.3322/caac.21600.
Park, J. J. H., Hsu, G., Siden, E. G., Thorlund, K., & Mills, E. J. (2020). An overview of precision oncology basket and umbrella trials for clinicians. CA: a cancer journal for clinicians, 70(2), 125-137. https://doi.org/10.3322/caac.21600
Park, Jay J H, et al. "An overview of precision oncology basket and umbrella trials for clinicians." CA: a cancer journal for clinicians vol. 70,2 (2020): 125-137. doi: https://doi.org/10.3322/caac.21600
Park JJH, Hsu G, Siden EG, Thorlund K, Mills EJ. An overview of precision oncology basket and umbrella trials for clinicians. CA Cancer J Clin. 2020 Mar;70(2):125-137. doi: 10.3322/caac.21600. Epub 2020 Feb 07. PMID: 32031692; PMCID: PMC7187272.
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CA Cancer J Clin. 2020 Mar;70(2):125-137. doi: 10.3322/caac.21600. Epub 2020 Feb 07.

An overview of precision oncology basket and umbrella trials for clinicians.

CA: a cancer journal for clinicians

Jay J H Park, Grace Hsu, Ellie G Siden, Kristian Thorlund, Edward J Mills

Affiliations

  1. Experimental Medicine, Department of Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
  2. Department of Health Research Methodology, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.
  3. Cytel Inc, Vancouver, British Columbia, Canada.

PMID: 32031692 PMCID: PMC7187272 DOI: 10.3322/caac.21600

Abstract

With advancements in biomarkers and momentum in precision medicine, biomarker-guided trials such as basket trials and umbrella trials have been developed under the master protocol framework. A master protocol refers to a single, overarching design developed to evaluate multiple hypotheses with the general goal of improving the efficiency of trial evaluation. One type of master protocol is the basket trial, in which a targeted therapy is evaluated for multiple diseases that share common molecular alterations or risk factors that may help predict whether the patients will respond to the given therapy. Another variant of a master protocol is the umbrella trial, in which multiple targeted therapies are evaluated for a single disease that is stratified into multiple subgroups based on different molecular or other predictive risk factors. Both designs follow the core principle of precision medicine-to tailor intervention strategies based on the patient's risk factor(s) that can help predict whether they will respond to a specific treatment. There have been increasing numbers of basket and umbrella trials, but they are still poorly understood. This article reviews common characteristics of basket and umbrella trials, key trials and recent US Food and Drug Administration approvals for precision oncology, and important considerations for clinical readers when critically evaluating future publications on basket trials and umbrella trials and for researchers when designing these clinical trials.

© 2020 The Authors. CA: A Cancer Journal for Clinicians published by Wiley Periodicals, Inc. on behalf of American Cancer Society.

Keywords: basket trials; master protocols; precision medicine; precision oncology; umbrella trials

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