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

Projektinformationen

ERAMET

ID Finanzhilfevereinbarung: 101137141

DOI

10.3030/101137141

EK-Unterschriftsdatum 17 November 2023

Startdatum 1 Januar 2024

Enddatum 31 Dezember 2027

Finanziert unter

• Health

Gesamtkosten

€ 3 865 507,06

EU-Beitrag

€ 3 865 043,13

Koordiniert durch

UNIVERSITE DE NAMUR
Belgium

CORDIS bietet Links zu öffentlichen Ergebnissen und Veröffentlichungen von HORIZONT-Projekten.

Links zu Ergebnissen und Veröffentlichungen von RP7-Projekten sowie Links zu einigen Typen spezifischer Ergebnisse wie Datensätzen und Software werden dynamisch von OpenAIRE abgerufen.

Leistungen

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

Veröffentlichungen

Peer reviewed articles (5)

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

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

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

Autoren: Francesca Pistollato, Fabia Furtmann, Lindsay J. Marshall, Surat Parvatam, Jan Turner, Flora Tshinanu Musuamba, Giulia Russo, Francesco Pappalardo
Veröffentlicht in: npj Digital Medicine, Ausgabe 8, 2025, ISSN 2398-6352
Herausgeber: 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

Autoren: Rajesh Krishna, Satyendra Suryawanshi, Juliane Rascher, Sonja Hartmann, Bernard Sebastien, Jeffrey S. Barrett
Veröffentlicht in: Clinical Pharmacology & Therapeutics, 2025, ISSN 0009-9236
Herausgeber: Wiley
DOI: 10.1002/CPT.3683

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

Autoren: 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
Veröffentlicht in: Clinical and Translational Science, Ausgabe 18, 2025, ISSN 1752-8054
Herausgeber: 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

Autoren: 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
Veröffentlicht in: British Journal of Clinical Pharmacology, Ausgabe 91, 2025, ISSN 0306-5251
Herausgeber: 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

Autoren: Grace Shalom Govere, Jean-Michel Dogne, Flora Musuamba
Veröffentlicht in: 2025
Herausgeber: 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

Autoren: Flora Musuamba Tshinanu, Ine Rusten, Helene Haguet, Lisa Hanquet, Marco Dionisi, Shalom Govere, Happy Djokoto, Camille Massaux, Jean-Michel Dogné
Veröffentlicht in: 2025
Herausgeber: 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.

Autoren: Helene Haguet, Jean-Michel Dogne, Flora Musuamba
Herausgeber: 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

Autoren: Orestis Papatryfonos, Linda Wanika, Amy Cheung, Michael Chappell
Veröffentlicht in: 2025
Herausgeber: Population Approach Group in Europe (PAGE)

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

Autoren: Ine Skottheim Rusten, Flora Musuamba, Michael Chappell, Amy Cheung, Alicia Gibson, Jeff Barrett
Herausgeber: 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

Autoren: Linda Wanika, Ine Skottheim Rusten, James Kermode, Amy Cheung, Mike Chappell
Veröffentlicht in: 2024
Herausgeber: PKUK

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

Autoren: 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
Veröffentlicht in: 2025
Herausgeber: 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

Autoren: Charlotte Simons, Loes Maton, Maaike van Dartel, Michiel van den Heuvel, Loes den Otter, Carolien Versantvoort, Pieter Colin, Jeroen Koomen
Veröffentlicht in: 2025
Herausgeber: 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

Autoren: Lisa Hanquet, Jean-Michel Dogne, Flora Musuamba
Herausgeber: 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

Autoren: Marco Dionisi, Jean-Michel Dogné, Flora Musuamba
Veröffentlicht in: 2025
Herausgeber: Population Approach Group in Europe (PAGE)

Constructing the ERAMET Digital Research Environment (DRE)

Autoren: Jeff Barrett, Flora Musuamba Tshinanu, Ine Skottheim Rusten, Scott Russell, Kara Lasater, Alicia Gibson, David Sibbald
Veröffentlicht in: 2024
Herausgeber: Population Approach Group in Europe (PAGE)

Uncertainty quantification in Mixed effects modelling

Autoren: Linda Wanika, Ine Skottheim Rusten, James Kermode, Mike Chappell
Veröffentlicht in: 2025, ISSN 1871-6032
Herausgeber: Population Approach Group in Europe (PAGE)