Ecosystem for rapid adoption of modelling and simulation METhods to address regulatory needs in the development of orphan and paediatric medicines 

Informations projet

ERAMET

N° de convention de subvention: 101137141

DOI

10.3030/101137141

Date de signature de la CE 17 Novembre 2023

Date de début 1 Janvier 2024

Date de fin 31 Decembre 2027

Financé au titre de

• Health

Coût total

€ 3 865 507,06

Contribution de l’UE

€ 3 865 043,13

Coordonné par

UNIVERSITE DE NAMUR
Belgium

CORDIS fournit des liens vers les livrables publics et les publications des projets HORIZON.

Les liens vers les livrables et les publications des projets du 7e PC, ainsi que les liens vers certains types de résultats spécifiques tels que les jeux de données et les logiciels, sont récupérés dynamiquement sur OpenAIRE .

Livrables

Documents, reports (8)

DEC plan 1

Plan for dissemination and exploitation including communication activities & update

Project Risk and Quality Management Plan

Procedures for risk monitoring and evaluation throughout the project, including a risk register

DPM model for BPD

Mature DPM model is validated and ready for use in BPD

PMX models for paediatric extrapolation

Mature PMX and hybrid models are validated and ready for paediatric extrapolation

Repository

Repository of key drug development questions, related data and methods created

PMX and hybrid models for SCD and thalassemia

Mature PMX and hybrid models are validated and ready for use in SCD and thalassemia

UISS model for ataxia

Mature UISS model is validated and ready for use in AT

DEC plan 2

Plan for dissemination and exploitation including communication activities & update

Websites, patent fillings, videos etc. (2)

Website Creation

A websitewill be created to dissiminate the result of the project

AI-based platform DRE

AI-based tools are developed to identify relevant data sources (automation of data searching, structuring and formatting) and data analysis

Publications

Peer reviewed articles (5)

Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists

Auteurs: Mariam A. Ahmed, Bilal AbuAsal, Jeffrey S. Barrett, Karim Azer, Yuen Yi Hon, Salwa Albusaysi, Elizabeth Shang, Meng Wang, Maria Burian, Noha Rayad
Publié dans: Clinical and Translational Science, Numéro 18, 2025, ISSN 1752-8054
Éditeur: Wiley
DOI: 10.1111/CTS.70215

Advancing the frontier of rare disease modeling: a critical appraisal of in silico technologies

Auteurs: Francesca Pistollato, Fabia Furtmann, Lindsay J. Marshall, Surat Parvatam, Jan Turner, Flora Tshinanu Musuamba, Giulia Russo, Francesco Pappalardo
Publié dans: npj Digital Medicine, Numéro 8, 2025, ISSN 2398-6352
Éditeur: Springer Science and Business Media LLC
DOI: 10.1038/S41746-025-02068-1

Innovations and Best Practices for Therapeutic Development in Pediatric Rare Diseases: A Model‐Informed Drug Development Perspective

Auteurs: Rajesh Krishna, Satyendra Suryawanshi, Juliane Rascher, Sonja Hartmann, Bernard Sebastien, Jeffrey S. Barrett
Publié dans: Clinical Pharmacology & Therapeutics, 2025, ISSN 0009-9236
Éditeur: Wiley
DOI: 10.1002/CPT.3683

AI‐Driven Applications in Clinical Pharmacology and Translational Science: Insights From the ASCPT 2024 AI Preconference

Auteurs: Mohamed H. Shahin, Prashant Desai, Nadia Terranova, Yuanfang Guan, Tomáš Helikar, Sebastian Lobentanzer, Qi Liu, James Lu, Subha Madhavan, Gary Mo, Flora T. Musuamba, Jagdeep T. Podichetty, Jie Shen, Lei Xie, Mathew Wiens, Cynthia J. Musante
Publié dans: Clinical and Translational Science, Numéro 18, 2025, ISSN 1752-8054
Éditeur: Wiley
DOI: 10.1111/CTS.70203

A review on the role of extrapolation as basis for paediatric marketing authorization applications of medicines in the EU

Auteurs: Charlotte W. M. Simons, Loes C. H. Maton, Maaike van Dartel, Michiel van den Heuvel, Loes den Otter, Carolien Versantvoort, Pieter J. Colin, Jeroen V. Koomen
Publié dans: British Journal of Clinical Pharmacology, Numéro 91, 2025, ISSN 0306-5251
Éditeur: Wiley
DOI: 10.1111/BCP.16395

Conference proceedings (12)

The use of modeling and simulation to inform Haemoglobinopathies drug development– a review of Scientific Advices from January 2014 to December 2024

Auteurs: Grace Shalom Govere, Jean-Michel Dogne, Flora Musuamba
Publié dans: 2025
Éditeur: Population Approach Group in Europe (PAGE)

Credibility assessment of model informed drug development approaches in paediatric and rare diseases: a review of EMA submissions and assessment reports from 2014 to 2024

Auteurs: Flora Musuamba Tshinanu, Ine Rusten, Helene Haguet, Lisa Hanquet, Marco Dionisi, Shalom Govere, Happy Djokoto, Camille Massaux, Jean-Michel Dogné
Publié dans: 2025
Éditeur: Population Approach Group in Europe (PAGE)

Use of modeling and simulation to inform the development of monoclonal antibodies to treat moderate-to-severe asthma: a review of EMA centralized procedure from 2014 to 2024.

Auteurs: Helene Haguet, Jean-Michel Dogne, Flora Musuamba
Éditeur: Population Approach Group in Europe (PAGE)

Development of a Population PK-PD Model for Tapentadol to Assess Postoperative Pain in Children Aged 6 to <12 using Nonlinear Mixed-Effects Modelling

Auteurs: Orestis Papatryfonos, Linda Wanika, Amy Cheung, Michael Chappell
Publié dans: 2025
Éditeur: Population Approach Group in Europe (PAGE)

Operationalizing the ERAMET Digital Research Environment (DRE) – Building the Ecosystem

Auteurs: Ine Skottheim Rusten, Flora Musuamba, Michael Chappell, Amy Cheung, Alicia Gibson, Jeff Barrett
Éditeur: Population Approach Group in Europe (PAGE)

Paediatric dosing regimen of tetrabenazine: A case study on the credibility and evaluation of paediatric and rare disease models

Auteurs: Linda Wanika, Ine Skottheim Rusten, James Kermode, Amy Cheung, Mike Chappell
Publié dans: 2024
Éditeur: PKUK

Towards Developing a Harmonizing Framework for PK and/or PKPD Scaling for Pain Medications in the Pediatric Population

Auteurs: Soumya Perinparajah, Sophie Rhodes, Yin Edwards, Anna Largajolli, Li Qin, Elke H.J. Krekels, Yu-Wei Lin, Justin Hay, Piet van der Graaf, S.Y. Amy Cheung
Publié dans: 2025
Éditeur: Population Approach Group in Europe (PAGE)

A review on the role of extrapolation as basis for paediatric marketing authorization applications of medicines in the EU

Auteurs: Charlotte Simons, Loes Maton, Maaike van Dartel, Michiel van den Heuvel, Loes den Otter, Carolien Versantvoort, Pieter Colin, Jeroen Koomen
Publié dans: 2025
Éditeur: Population Approach Group in Europe (PAGE)

Model informed drug development and CAR-T cells : A review of their use and credibility assessment in the context of EMA centralized procedures

Auteurs: Lisa Hanquet, Jean-Michel Dogne, Flora Musuamba
Éditeur: Population Approach Group in Europe (PAGE)

Credibility assessment of model informed drug development (MIDD) in pediatric pain: a review of pediatric investigation plans from 2008 to 2024

Auteurs: Marco Dionisi, Jean-Michel Dogné, Flora Musuamba
Publié dans: 2025
Éditeur: Population Approach Group in Europe (PAGE)

Constructing the ERAMET Digital Research Environment (DRE)

Auteurs: Jeff Barrett, Flora Musuamba Tshinanu, Ine Skottheim Rusten, Scott Russell, Kara Lasater, Alicia Gibson, David Sibbald
Publié dans: 2024
Éditeur: Population Approach Group in Europe (PAGE)

Uncertainty quantification in Mixed effects modelling

Auteurs: Linda Wanika, Ine Skottheim Rusten, James Kermode, Mike Chappell
Publié dans: 2025, ISSN 1871-6032
Éditeur: Population Approach Group in Europe (PAGE)