Definitions and statistical properties of master protocols for personalized medicine in oncology. Issue 2 (4th March 2018)

• Record Type: Journal Article
• Authors: Renfro, Lindsay A.; Mandrekar, Sumithra J.
• Is Part Of: Journal of biopharmaceutical statistics. Volume 28:Issue 2(2018)
• Page Start: 217
• Page End: 228
• Publication Date: 2018-03-04
• Subjects: Basket trials -- master protocols -- oncology -- platform trials -- personalized medicine -- umbrella trials; Pharmacy -- Statistical methods -- Periodicals; Drugs -- Testing -- Statistical methods -- Periodicals; Biometry -- Periodicals; Biopharmaceutics -- Periodicals; Pharmacokinetics -- Periodicals; 615.19
• DOI: 10.1080/10543406.2017.1372778
• Languages: English
• ISSNs: 1054-3406

Abstract

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.