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

Informazioni relative al progetto

ERAMET

ID dell’accordo di sovvenzione: 101137141

DOI

10.3030/101137141

Data della firma CE 17 Novembre 2023

Data di avvio 1 Gennaio 2024

Data di completamento 31 Dicembre 2027

Finanziato da

• Health

Costo totale

€ 3 865 507,06

Contributo UE

€ 3 865 043,13

Coordinato da

UNIVERSITE DE NAMUR
Belgium

CORDIS fornisce collegamenti ai risultati finali pubblici e alle pubblicazioni dei progetti ORIZZONTE.

I link ai risultati e alle pubblicazioni dei progetti del 7° PQ, così come i link ad alcuni tipi di risultati specifici come dataset e software, sono recuperati dinamicamente da .OpenAIRE .

Risultati finali

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

Pubblicazioni

Peer reviewed articles (5)

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

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

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

Autori: Francesca Pistollato, Fabia Furtmann, Lindsay J. Marshall, Surat Parvatam, Jan Turner, Flora Tshinanu Musuamba, Giulia Russo, Francesco Pappalardo
Pubblicato in: npj Digital Medicine, Numero 8, 2025, ISSN 2398-6352
Editore: 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

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

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

Autori: 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
Pubblicato in: Clinical and Translational Science, Numero 18, 2025, ISSN 1752-8054
Editore: 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

Autori: 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
Pubblicato in: British Journal of Clinical Pharmacology, Numero 91, 2025, ISSN 0306-5251
Editore: 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

Autori: Grace Shalom Govere, Jean-Michel Dogne, Flora Musuamba
Pubblicato in: 2025
Editore: 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

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

Autori: Helene Haguet, Jean-Michel Dogne, Flora Musuamba
Editore: 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

Autori: Orestis Papatryfonos, Linda Wanika, Amy Cheung, Michael Chappell
Pubblicato in: 2025
Editore: Population Approach Group in Europe (PAGE)

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

Autori: Ine Skottheim Rusten, Flora Musuamba, Michael Chappell, Amy Cheung, Alicia Gibson, Jeff Barrett
Editore: 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

Autori: Linda Wanika, Ine Skottheim Rusten, James Kermode, Amy Cheung, Mike Chappell
Pubblicato in: 2024
Editore: PKUK

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

Autori: 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
Pubblicato in: 2025
Editore: 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

Autori: Charlotte Simons, Loes Maton, Maaike van Dartel, Michiel van den Heuvel, Loes den Otter, Carolien Versantvoort, Pieter Colin, Jeroen Koomen
Pubblicato in: 2025
Editore: 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

Autori: Lisa Hanquet, Jean-Michel Dogne, Flora Musuamba
Editore: 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

Autori: Marco Dionisi, Jean-Michel Dogné, Flora Musuamba
Pubblicato in: 2025
Editore: Population Approach Group in Europe (PAGE)

Constructing the ERAMET Digital Research Environment (DRE)

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

Uncertainty quantification in Mixed effects modelling

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