The project aims to create a transparent ecosystem to foster decision-making in paediatric and orphan drugs development by integrating innovative methodologies, such as modelling and simulation (M&S) methods and related data types, within regulatory procedures. With three pillars: a data repository, the development of high-quality standards for data and analytical methods, and an Artificial Intelligence-based platform, it aims to establish the credibility of M&S methods and related results as sources of evidence within regulatory procedures.

ROLE OF THE FOUNDATION

The Foundation is involved in constructing the integrated model for the application of innovative methodologies in paediatric and orphan drug development, while also assessing their regulatory acceptability. Furthermore, it is involved in applying the model to three specific case studies of paediatric and rare diseases. Lastly, the Foundation collaborates to the preparation and application of the qualification procedure to the European Medicines Agency (EMA) to validate the project methodology tools.

FUNDED BY: Horizon Europe–Health, GA 101137141 2024-27 (www.cordis.europa.eu/project/id/101137141).

DURATION: Jan 2024 - Dec 2027

PARTNERS:

• Fondazione per la Ricerca Farmacologica Gianni Benzi - ETS, Italy
• University of Namur, Belgium
• Systems Resource Lab AS (SRLAB), Norway
• Federal Agency for Medicines and Health Products, Belgium
• University of Valencia, Spain
• Consorzio per Valutazioni Biologiche e Farmacologiche, Italy
• University Medical Center Groningen, the Netherlands
• University of Catania, Italy
• National Agency for the Safety of Medicines and Health Products, France
• Agentschap College Ter Beoordeling Van Geneesmiddelen, the Netherlands
• European Business Summit Network, Belgium
• Spanish Agency of Medicines and Pharmaceutical Products, Spain
• Certara UK Limited, United Kingdom
• The University of Warwick, United Kingdom
• University College London, United Kingdom
• Aridhia Informatics Limited, United Kingdom