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Needs assessment for creation of a platform trial network in metabolic-dysfunction associated steatohepatitis

Lookup NU author(s): Professor Quentin AnsteeORCiD

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This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).

Abstract

© The Author(s) 2024.Background: The EU Patient-cEntric clinicAl tRial pLatforms (EU-PEARL) project (IMI2-853966) aimed to develop tools to establish integrated research platforms (IRP) for conducting adaptive-design trials in various diseases, including metabolic-dysfunction associated steatohepatitis (MASH). One essential component of a successful MASH IRP is a robust and reliable Clinical Research Network (CRN). Herein, we outline the required elements and anticipated steps to set-up such a CRN. Methods: We identified European clinical research sites that could potentially serve as the foundation for MASH IRP and a CRN. A survey was sent to sites to assess their interest in joining a CRN, their familiarity with platform trials, and their capacity to participate in a future MASH IRP. Results: A total of 141 investigators were invited to participate in the survey, and 40% responded. More than half of the answers (52%) identify MASH with advanced fibrosis (F3-4) as the subpopulation with the greatest unmet need. Regarding the difficulty in identifying candidates for trials, 65% find it is moderately difficult and 30% very difficult. Most respondents (94%) believe that a platform trial could offer substantial benefits to patients. Nearly all researchers express interest in participating in a platform trial (78%), with 22% indicating their interest would be contingent on initial industry funding. Conclusion: While preliminary, our findings on responding sites are encouraging for the potential establishment of a CRN for a MASH IRP. However, funding schemes and sustainability strategies to provide proof-of-platform in MASH seem key in the short-term scenario.

Publication metadata

Author(s): Sena E, Tacke F, Anstee QM, Di Prospero N, Skalshoi- Kjaer M, Munoz-Martinez S, Rivera-Esteban J, Jimenez-Masip A, Banales JM, Martinez-Gomez M, Koenig F, Genesca J, Ratziu V, Pericas JM

Publication type: Article

Publication status: Published

Journal: Communications Medicine

Year: 2024

Volume: 4

Issue: 1

Online publication date: 16/07/2024

Acceptance date: 25/06/2024

Date deposited: 24/09/2024

ISSN (electronic): 2730-664X

Publisher: Springer Nature

URL: https://doi.org/10.1038/s43856-024-00560-5

DOI: 10.1038/s43856-024-00560-5

Data Access Statement: Anonymized individual responses to the survey are available upon request. Source data for Fig. 3 are available as Supplementary Data 3

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