Results
Title

Master Protocols for Precision Medicine in Oncology: Overcoming Methodology of Randomized Clinical Trials.

Authors

Di Liello, Raimondo; Piccirillo, Maria Carmela; Arenare, Laura; Gargiulo, Piera; Schettino, Clorinda; Gravina, Adriano; Perrone, Francesco

Abstract

Randomized clinical trials are considered the milestones of clinical research in oncology, and guided the development and approval of new compounds so far. In the last few years, however, molecular and genomic profiling led to a change of paradigm in therapeutic algorithms of many cancer types, with the spread of different biomarker-driven therapies (or targeted therapies). This scenario of "personalized medicine" revolutionized therapeutic strategies and the methodology of the supporting clinical research. New clinical trial designs are emerging to answer to the unmet clinical needs related to the development of these targeted therapies, overcoming the "classical" structure of randomized studies. Innovative trial designs able to evaluate more than one treatment in the same group of patients or many groups of patients with the same treatment (or both) are emerging as a possible future standard in clinical trial methodology. These are identified as "master protocols", and include umbrella, basket and platform trials. In this review, we described the main characteristics of these new trial designs, focusing on the opportunities and limitations of their use in the era of personalized medicine.

Subjects

CLINICAL trials; MEDICAL research; MEDICAL protocols; INDIVIDUALIZED medicine; NEW trials; CANCER prognosis

Publication

Life (2075-1729), 2021, Vol 11, Issue 11, p1253

ISSN

2075-1729

Publication type

Academic Journal

DOI

10.3390/life11111253