Statistical controversies in clinical research: Basket trials, umbrella trials, and other master protocols: A review and examples

L. A. Renfro; D. J. Sargent

doi:10.1093/annonc/mdw413

Statistical controversies in clinical research: Basket trials, umbrella trials, and other master protocols: A review and examples

L. A. Renfro, D. J. Sargent

Quantitative Health Sciences

Research output: Contribution to journal › Article › peer-review

112 Scopus citations

Abstract

In recent years, cancers once viewed as relatively homogeneous in terms of organ location and treatment strategy are now better understood to be increasingly heterogeneous across biomarker and genetically defined patient subgroups. This has produced a shift toward development of biomarker-targeted agents during a time when funding for cancer research has been limited; as a result, the need for improved operational efficiency in studying many agent-and-target combinations in parallel has emerged. Platform trials, basket trials, and umbrella trials are new approaches to clinical research driven by this need for enhanced efficiency in the modern era of increasingly specific cancer subpopulations and decreased resources to study treatments for individual cancer subtypes in a traditional way. In this review, we provide an overview of these new types of clinical trial designs, including discussions ofmotivation for their use, recommended terminology, examples, and challenges encountered in their application.

Original language	English (US)
Pages (from-to)	34-43
Number of pages	10
Journal	Annals of Oncology
Volume	28
Issue number	1
DOIs	https://doi.org/10.1093/annonc/mdw413
State	Published - Jan 1 2017

Keywords

Basket trialbiomarker-based trialmaster protocolplatform trialprecision medicineumbrella trial

ASJC Scopus subject areas

Hematology
Oncology

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10.1093/annonc/mdw413

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title = "Statistical controversies in clinical research: Basket trials, umbrella trials, and other master protocols: A review and examples",

abstract = "In recent years, cancers once viewed as relatively homogeneous in terms of organ location and treatment strategy are now better understood to be increasingly heterogeneous across biomarker and genetically defined patient subgroups. This has produced a shift toward development of biomarker-targeted agents during a time when funding for cancer research has been limited; as a result, the need for improved operational efficiency in studying many agent-and-target combinations in parallel has emerged. Platform trials, basket trials, and umbrella trials are new approaches to clinical research driven by this need for enhanced efficiency in the modern era of increasingly specific cancer subpopulations and decreased resources to study treatments for individual cancer subtypes in a traditional way. In this review, we provide an overview of these new types of clinical trial designs, including discussions ofmotivation for their use, recommended terminology, examples, and challenges encountered in their application.",

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note = "Funding Information: This work was supported by the National Institutes of Health [CTSA Grant Number KL2 TR000136 from the National Center for Advancing Translational Science (NCATS) to LAR]; and the National Cancer Institute at the National Institutes of Health (R01 CA174779 to DJS). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the NIH. Publisher Copyright: {\textcopyright} The Author 2016. Published by Oxford University Press on behalf of the European Society for Medical Oncology. All rights reserved.",

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Statistical controversies in clinical research: Basket trials, umbrella trials, and other master protocols: A review and examples

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