Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both

Abstract

High-quality evidence is what we use to guide medical practice. The standard approach to generating this evidence — a series of clinical trials, each investigating one or two interventions in a single disease — has become ever more expensive and challenging to execute. As a result, important clinical questions go unanswered. The conduct of “precision medicine” trials to evaluate targeted therapies creates challenges in recruiting patients with rare genetic subtypes of a disease. There is also increasing interest in performing mechanism-based trials in which eligibility is based on criteria other than traditional disease definitions. The common denominator is a need to answer more questions more efficiently and in less time.