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

Información del proyecto

ERAMET

Identificador del acuerdo de subvención: 101137141

DOI

10.3030/101137141

Fecha de la firma de la CE 17 Noviembre 2023

Fecha de inicio 1 Enero 2024

Fecha de finalización 31 Diciembre 2027

Financiado con arreglo a

• Health

Coste total

€ 3 865 507,06

Aportación de la UE

€ 3 865 043,13

Coordinado por

UNIVERSITE DE NAMUR
Belgium

CORDIS proporciona enlaces a los documentos públicos y las publicaciones de los proyectos de los programas marco HORIZONTE.

Los enlaces a los documentos y las publicaciones de los proyectos del Séptimo Programa Marco, así como los enlaces a algunos tipos de resultados específicos, como conjuntos de datos y «software», se obtienen dinámicamente de OpenAIRE .

Resultado final

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

Publicaciones

Peer reviewed articles (5)

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

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

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

Autores: Francesca Pistollato, Fabia Furtmann, Lindsay J. Marshall, Surat Parvatam, Jan Turner, Flora Tshinanu Musuamba, Giulia Russo, Francesco Pappalardo
Publicado en: npj Digital Medicine, Edición 8, 2025, ISSN 2398-6352
Editor: 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

Autores: Rajesh Krishna, Satyendra Suryawanshi, Juliane Rascher, Sonja Hartmann, Bernard Sebastien, Jeffrey S. Barrett
Publicado en: Clinical Pharmacology & Therapeutics, 2025, ISSN 0009-9236
Editor: Wiley
DOI: 10.1002/CPT.3683

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

Autores: 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
Publicado en: Clinical and Translational Science, Edición 18, 2025, ISSN 1752-8054
Editor: 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

Autores: 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
Publicado en: British Journal of Clinical Pharmacology, Edición 91, 2025, ISSN 0306-5251
Editor: Wiley
DOI: 10.1111/BCP.16395

Conference proceedings (9)

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

Autores: Grace Shalom Govere, Jean-Michel Dogne, Flora Musuamba
Publicado en: 2025
Editor: 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

Autores: Flora Musuamba Tshinanu, Ine Rusten, Helene Haguet, Lisa Hanquet, Marco Dionisi, Shalom Govere, Happy Djokoto, Camille Massaux, Jean-Michel Dogné
Publicado en: 2025
Editor: 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

Autores: Orestis Papatryfonos, Linda Wanika, Amy Cheung, Michael Chappell
Publicado en: 2025
Editor: 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

Autores: Linda Wanika, Ine Skottheim Rusten, James Kermode, Amy Cheung, Mike Chappell
Publicado en: 2024
Editor: PKUK

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

Autores: 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
Publicado en: 2025
Editor: 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

Autores: Charlotte Simons, Loes Maton, Maaike van Dartel, Michiel van den Heuvel, Loes den Otter, Carolien Versantvoort, Pieter Colin, Jeroen Koomen
Publicado en: 2025
Editor: 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

Autores: Marco Dionisi, Jean-Michel Dogné, Flora Musuamba
Publicado en: 2025
Editor: Population Approach Group in Europe (PAGE)

Constructing the ERAMET Digital Research Environment (DRE)

Autores: Jeff Barrett, Flora Musuamba Tshinanu, Ine Skottheim Rusten, Scott Russell, Kara Lasater, Alicia Gibson, David Sibbald
Publicado en: 2024
Editor: Population Approach Group in Europe (PAGE)

Uncertainty quantification in Mixed effects modelling

Autores: Linda Wanika, Ine Skottheim Rusten, James Kermode, Mike Chappell
Publicado en: 2025, ISSN 1871-6032
Editor: Population Approach Group in Europe (PAGE)