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

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.

KEYWORDS:

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

Declaration of interest

The authors have declared no conflicts of interest.

Funding

This publication (or project) was supported by CTSA (Grant Number KL2 TR000136) from the National Center for Advancing Translational Science (NCATS). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the NIH.

Additional information

Funding

This publication (or project) was supported by CTSA (Grant Number KL2 TR000136) from the National Center for Advancing Translational Science (NCATS). Its contents are solely the responsibility of the authors and do not necessarily represent the official views of the NIH.