You are currently viewing PRESS RELEASE: First-Year Achievements in Advancing Paediatric and Rare Disease Research

PRESS RELEASE: First-Year Achievements in Advancing Paediatric and Rare Disease Research

Brussels, 29 April 2025 – The ERAMET consortium is proud to announce the publication of a new article summarising the major achievements from its first year, marking a significant step forward in the use of model-informed drug development (MIDD) for paediatric and rare diseases. This milestone article outlines the foundations of the ERAMET ecosystem, focusing on regulatory innovation, computational modelling, and the development of a secure research infrastructure to support cross-disciplinary collaboration.

Key highlights from the past year include the creation of a regulatory question repository by the University of Namur, the development of model credibility frameworks by the University of Warwick and Fondazione Gianni Benzi, and pioneering work on paediatric extrapolation use-cases led by Certara. Notably, the University of Valencia has advanced cohort selection strategies using simulation scenarios, while Aridhia has deployed a cloud-based secure platform for research teams.

The article also presents early outcomes from exemplar disease models—including predictive tools for Juvenile Huntington’s Disease and bronchopulmonary dysplasia—as well as novel methodologies for assessing uncertainty in model parameters. These innovations are helping to reshape how drugs for vulnerable populations are developed, tested, and approved.

This publication marks a strong foundation for ERAMET’s future efforts, as the consortium continues to support regulatory science, enhance data-driven methodologies, and improve health outcomes for children with rare conditions.

Read the full article here

 

Leave a Reply