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

Informacje na temat projektu

ERAMET

Identyfikator umowy o grant: 101137141

DOI

10.3030/101137141

Data podpisania przez KE 17 Listopada 2023

Data rozpoczęcia 1 Stycznia 2024

Data zakończenia 31 Grudnia 2027

Finansowanie w ramach

• Health

Koszt całkowity

€ 3 865 507,06

Wkład UE

€ 3 865 043,13

Koordynowany przez

UNIVERSITE DE NAMUR
Belgium

CORDIS oferuje możliwość skorzystania z odnośników do publicznie dostępnych publikacji i rezultatów projektów realizowanych w ramach programów ramowych HORYZONT.

Odnośniki do rezultatów i publikacji związanych z poszczególnymi projektami 7PR, a także odnośniki do niektórych konkretnych kategorii wyników, takich jak zbiory danych i oprogramowanie, są dynamicznie pobierane z systemu OpenAIRE .

Rezultaty

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

Publikacje

Peer reviewed articles (5)

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

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

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

Autorzy: Francesca Pistollato, Fabia Furtmann, Lindsay J. Marshall, Surat Parvatam, Jan Turner, Flora Tshinanu Musuamba, Giulia Russo, Francesco Pappalardo
Opublikowane w: npj Digital Medicine, Numer 8, 2025, ISSN 2398-6352
Wydawca: 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

Autorzy: Rajesh Krishna, Satyendra Suryawanshi, Juliane Rascher, Sonja Hartmann, Bernard Sebastien, Jeffrey S. Barrett
Opublikowane w: Clinical Pharmacology & Therapeutics, 2025, ISSN 0009-9236
Wydawca: Wiley
DOI: 10.1002/CPT.3683

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

Autorzy: 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
Opublikowane w: Clinical and Translational Science, Numer 18, 2025, ISSN 1752-8054
Wydawca: 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

Autorzy: 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
Opublikowane w: British Journal of Clinical Pharmacology, Numer 91, 2025, ISSN 0306-5251
Wydawca: 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

Autorzy: Grace Shalom Govere, Jean-Michel Dogne, Flora Musuamba
Opublikowane w: 2025
Wydawca: 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

Autorzy: Flora Musuamba Tshinanu, Ine Rusten, Helene Haguet, Lisa Hanquet, Marco Dionisi, Shalom Govere, Happy Djokoto, Camille Massaux, Jean-Michel Dogné
Opublikowane w: 2025
Wydawca: 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.

Autorzy: Helene Haguet, Jean-Michel Dogne, Flora Musuamba
Wydawca: 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

Autorzy: Orestis Papatryfonos, Linda Wanika, Amy Cheung, Michael Chappell
Opublikowane w: 2025
Wydawca: Population Approach Group in Europe (PAGE)

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

Autorzy: Ine Skottheim Rusten, Flora Musuamba, Michael Chappell, Amy Cheung, Alicia Gibson, Jeff Barrett
Wydawca: 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

Autorzy: Linda Wanika, Ine Skottheim Rusten, James Kermode, Amy Cheung, Mike Chappell
Opublikowane w: 2024
Wydawca: PKUK

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

Autorzy: 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
Opublikowane w: 2025
Wydawca: 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

Autorzy: Charlotte Simons, Loes Maton, Maaike van Dartel, Michiel van den Heuvel, Loes den Otter, Carolien Versantvoort, Pieter Colin, Jeroen Koomen
Opublikowane w: 2025
Wydawca: 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

Autorzy: Lisa Hanquet, Jean-Michel Dogne, Flora Musuamba
Wydawca: 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

Autorzy: Marco Dionisi, Jean-Michel Dogné, Flora Musuamba
Opublikowane w: 2025
Wydawca: Population Approach Group in Europe (PAGE)

Constructing the ERAMET Digital Research Environment (DRE)

Autorzy: Jeff Barrett, Flora Musuamba Tshinanu, Ine Skottheim Rusten, Scott Russell, Kara Lasater, Alicia Gibson, David Sibbald
Opublikowane w: 2024
Wydawca: Population Approach Group in Europe (PAGE)

Uncertainty quantification in Mixed effects modelling

Autorzy: Linda Wanika, Ine Skottheim Rusten, James Kermode, Mike Chappell
Opublikowane w: 2025, ISSN 1871-6032
Wydawca: Population Approach Group in Europe (PAGE)