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

L. A. Renfro, D. J. Sargent

Research output: Contribution to journalArticle

84 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 languageEnglish (US)
Pages (from-to)34-43
Number of pages10
JournalAnnals of Oncology
Volume28
Issue number1
DOIs
StatePublished - Jan 1 2017

Keywords

  • Basket trialbiomarker-based trialmaster protocolplatform trialprecision medicineumbrella trial

ASJC Scopus subject areas

  • Hematology
  • Oncology

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