Journal Article

An Overview of Precision Oncology Basket and Umbrella Trials for Clinicians

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

2020

Overview

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, over-arching 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 com-mon 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 num-bers of basket and umbrella trials, but they are still poorly understood. This article re-views common characteristics of basket and umbrella trials, key trials and recent US Food and Drug Administration approvals for precision oncology, and important con-siderations for clinical readers when critically evaluating future publications on bas-ket trials and umbrella trials and for researchers when designing these clinical trials.

• Publisher: Wiley Subscription Services, Inc
• Subject: Biomarkers / Clinical trials / Design / Oncology / Patients / Precision medicine / Risk factors