Definitions and statistical properties of master protocols for personalized medicine in oncology

Lindsay A. Renfro, Sumithra J. Mandrekar

Research output: Contribution to journalReview article

9 Scopus citations

Abstract

Within the field of cancer research, discovery of biomarkers and genetic mutations that are potentially predictive of treatment benefit is motivating a paradigm shift in how cancer clinical trials are conducted. In this review, we provide an overview of the class of trials known as “master protocols,” including basket trials, umbrella trials, and platform trials. For each, we describe standardized terminology, provide a motivating example with modeling details and decision rules, and discuss statistical advantages and limitations. We conclude with a discussion of general statistical considerations and challenges encountered across these types of trials.

Original languageEnglish (US)
Pages (from-to)217-228
Number of pages12
JournalJournal of Biopharmaceutical Statistics
Volume28
Issue number2
DOIs
StatePublished - Mar 4 2018

Keywords

  • Basket trials
  • master protocols
  • oncology
  • personalized medicine
  • platform trials
  • umbrella trials

ASJC Scopus subject areas

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

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