CVBF has achieved a major milestone by securing a significant role in the Horizon Europe-funded ERAMET project. Guided by Prof Adriana Ceci, the Lead Scientist of ERAMET for FGB-CVBF, this project is set to transform paediatric and orphan drug development. CVBF contribution is important as we will provide the expertise to enable the adoption of Modelling & Simulation (M&S) techniques gained with DEEP (DEferiprone Evaluation in Paediatrics), which aimed to study the efficacy and safety of deferiprone in children affected by β-thalassemia and other transfusion dependent haemoglobinopathies.
ERAMET will focus on paediatric and orphan drugs, enhancing transparency for drug development and assessment, and promoting the use of Modelling & Simulation (M&S) methods and various data types, including real-world data from registries and electronic healthcare records. The core goal of ERAMET is to establish a framework to boost the credibility of M&S methods and their outcomes within regulatory procedures. This will be achieved through three key pillars:
- Repository for Questions, Data, and Methods
ERAMET will introduce a central repository that connects drug development questions, the necessary data, and the methods for data analysis and simulation.
- Development and Validation of High-Quality Standards
ERAMET will develop and validate high-quality standards for data and analytical methods, covering computational M&S, digital twins, AI, hybrid approaches, standard statistics, pharmacometrics, and alternative data sources.
- AI-Based Automation Platform
ERAMET will implement an AI-based platform designed to automate data collection, formatting, modelling, and simulation analysis, while ensuring the credibility of the results.
This ecosystem will be applied in five distinct use-cases, focusing on paediatric extrapolation, and characterizing the benefit/risk of drugs in different rare disease groups. Each use-case is expected to lead to the submission and regulatory approval of at least one validated M&S tool through the EMA qualification procedure.