Li, Yimei, Nelson, Robert, Izem, Rima, Broglio, Kristine, Mundayat, Rajiv, Gamalo, Margaret, Wen, Yansong, Pan, Haitao, Sun, Hengrui and Ye, Jingjing (2024) Unlocking the Potential: A systematic review of Master Protocol in Pediatrics. Therapeutic Innovation and Regulatory Science.

Abstract

Importance: The use of master protocols allows for innovative approaches to clinical trial designs, potentially enabling new approaches to operations and analytics and creating value for patients and drug developers.
Objective: Pediatric research has been conducted for many decades, but the use of novel designs such as master protocols are in pediatric research is unclear. We aimed to conduct a systematic review to evaluate the utilization of master protocols in pediatric drug development.
Evidence Review: A systematic search was performed in September 2022 using two data sources (PubMed and ClinicalTrials.gov) and included studies conducted in the last 10 years. General study information was extracted such as study type, study status, therapeutic area, clinical trial phase. Study characteristics that are specific to pediatric studies were also collected such as the minimum and maximum age of the study participants, whether the study is in the same adult master protocol, whether the pediatric drug dosing is the same as adult. Lastly, important study design elements of the master protocol were collected such as number of test drug arms and whether randomization and/or concurrent control was used.
Findings: Thirty-eight studies were included in the final analysis, and 16 (42%) are platform trials, 15 (39%) are basket trials and 7 (18%) are umbrella studies. Oncology (58%) is the largest therapeutic area for the included studies, followed by infectious diseases treating COVID (18%) and HIV (8%). The majority of the studies are early phase trials (60%). The use of master protocol in pediatrics started in 1997 and suddenly increased in 2020, mostly in Oncology disease area. Twenty-six (68%) of the 38 pediatric studies are included in the same adult master protocol, and 12 (32%) studies used the same dosing as adult. Overall, 17 studies (45%) used randomization, among which the majority (13 out of 17, 76%) used concurrent control.
Conclusions and Relevance: Master protocols are starting to be adopted in pediatric clinical research, but on a small scale and could be substantially expanded. Work is still required to further understand the barriers in implementing pediatric master protocols, from setting up infrastructure to interpreting study findings.

Item Type:	Article
Date Deposited:	29 Aug 2024 00:45
Last Modified:	29 Aug 2024 00:45
URI:	https://oak.novartis.com/id/eprint/52761