ERAMET stands for “Ecosystem for rapid adoption of modelling and simulation METhods to address regulatory needs in the development of orphan and pediatric medicines”. The project is managed by a multidisciplinary consortium made up of 17 partners located in Belgium, Norway, UK, Italy, Spain, France and the Netherlands. The ERAMET project is a collaborative initiative aimed at enhancing model-informed drug development (MIDD) for pediatric and rare diseases
ERAMET’s overall objective is to provide and implement a robust framework for the development and validation of mature modelling and simulation methods, specifically tailored to address regulatory needs in the development and assessment of orphan and pediatric medicines. It will establish a transparent ecosystem for drug development and assessment, facilitating the adoption of modelling and simulation (M&S) methods along with various data types, including real world data such as registries and electronic healthcare data.
The ecosystem is based on three pillars:
(1) A repository that serves as a hub connecting questions, data and methods.
(2) The establishment and validation of rigorous standards for data and analytical methods. These standards will cover modelling and simulation, digital twins, AI, hybrid approaches, standard statistics, and pharmacometrics, ensuring robust analysis and encompassing alternative data types and sources such as real-world data, eHealth data, registries, historical regulatory submissions, and scientific and non-clinical trials.
(3) An AI-based platform designed to automate and optimise data collection, formatting, modeling, simulation analysis, and credibility assessment processes.
The ecosystem will be applied to five distinct use cases, including pediatric extrapolation and the characterisation of drug benefit/risk across four groups of rare diseases: ataxia, transfusion-dependent hemoglobinopathies, bronchopulmonary dysplasia, and degenerative neuromuscular disorders. Each use case will be strategically designed to result in the submission and regulatory approval of at least one validated M&S tool through the EMA qualification procedure. Additionally, training sessions will be offered to familiarise regulatory assessors, drug developers, and clinical researchers with this innovative approach.
