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Lookup NU author(s): Professor Quentin AnsteeORCiD
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
© 2022 The Author(s)Non-alcoholic fatty liver disease is a condition that affects 25% of the population. Non-alcoholic steatohepatitis (NASH) is a progressive form of the disease that can lead to severe complications such as cirrhosis and hepatocellular carcinoma. Despite its high prevalence, no drugs are currently approved for the treatment of NASH. The drug development pipeline in NASH is very active, yet most assets do not progress to phase III trials and those that do reach phase III often fail to achieve the endpoints necessary for approval by regulatory agencies. Amongst other reasons, the methodological and operational features of traditional clinical trials in NASH might impede optimal drug development. In this regard, platform trials might be an attractive complement or alternative to conventional clinical trials. Platform trials use a master protocol which enables evaluation of multiple investigational medicinal products concurrently or sequentially with a single, shared control arm. Through Bayesian interim analyses, these trials allow for early exit of drugs from the trial based on success or futility, while providing participants better chances of receiving active compounds through adaptive randomisation. Overall, platform trials represent an alternative for patients, pharmaceutical companies, and clinicians in the quest to accelerate the approval of pharmacologic treatments for NASH.
Author(s): Pericas JM, Tacke F, Anstee QM, Di Prospero NA, Kjaer MS, Mesenbrink P, Koenig F, Genesca J, Ratziu V
Publication type: Review
Publication status: Published
Journal: Journal of Hepatology
Year: 2023
Volume: 78
Issue: 2
Pages: 442-447
Print publication date: 01/02/2023
Online publication date: 07/10/2022
Acceptance date: 20/09/2022
ISSN (print): 0168-8278
ISSN (electronic): 1600-0641
Publisher: Elsevier B.V.
URL: https://doi.org/10.1016/j.jhep.2022.09.021
DOI: 10.1016/j.jhep.2022.09.021
PubMed id: 36216134
Data Access Statement: Supplementary data to this article can be found online at https://doi.org/10.1016/ j.jhep.2022.09.021.
