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 language | English (US) |
|---|---|
| Pages (from-to) | 217-228 |
| Number of pages | 12 |
| Journal | Journal of Biopharmaceutical Statistics |
| Volume | 28 |
| Issue number | 2 |
| DOIs | |
| State | Published - 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)