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

Project Information

ERAMET

Grant agreement ID: 101137141

DOI

10.3030/101137141

EC signature date 17 November 2023

Start date 1 January 2024

End date 31 December 2027

Funded under

• Health

Total cost

€ 3 865 507,06

EU contribution

€ 3 865 043,13

Coordinated by

UNIVERSITE DE NAMUR
Belgium

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Publications

Peer reviewed articles (4)

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

Author(s): Mariam A. Ahmed, Bilal AbuAsal, Jeffrey S. Barrett, Karim Azer, Yuen Yi Hon, Salwa Albusaysi, Elizabeth Shang, Meng Wang, Maria Burian, Noha Rayad
Published in: Clinical and Translational Science, Issue 18, 2025, ISSN 1752-8054
Publisher: Wiley
DOI: 10.1111/CTS.70215

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

Author(s): Rajesh Krishna, Satyendra Suryawanshi, Juliane Rascher, Sonja Hartmann, Bernard Sebastien, Jeffrey S. Barrett
Published in: Clinical Pharmacology & Therapeutics, 2025, ISSN 0009-9236
Publisher: Wiley
DOI: 10.1002/CPT.3683

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

Author(s): 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
Published in: Clinical and Translational Science, Issue 18, 2025, ISSN 1752-8054
Publisher: 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

Author(s): 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
Published in: British Journal of Clinical Pharmacology, Issue 91, 2025, ISSN 0306-5251
Publisher: 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

Author(s): Grace Shalom Govere, Jean-Michel Dogne, Flora Musuamba
Published in: 2025
Publisher: 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

Author(s): Flora Musuamba Tshinanu, Ine Rusten, Helene Haguet, Lisa Hanquet, Marco Dionisi, Shalom Govere, Happy Djokoto, Camille Massaux, Jean-Michel Dogné
Published in: 2025
Publisher: 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

Author(s): Orestis Papatryfonos, Linda Wanika, Amy Cheung, Michael Chappell
Published in: 2025
Publisher: 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

Author(s): Linda Wanika, Ine Skottheim Rusten, James Kermode, Amy Cheung, Mike Chappell
Published in: 2024
Publisher: PKUK

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

Author(s): 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
Published in: 2025
Publisher: 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

Author(s): Charlotte Simons, Loes Maton, Maaike van Dartel, Michiel van den Heuvel, Loes den Otter, Carolien Versantvoort, Pieter Colin, Jeroen Koomen
Published in: 2025
Publisher: 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

Author(s): Marco Dionisi, Jean-Michel Dogné, Flora Musuamba
Published in: 2025
Publisher: Population Approach Group in Europe (PAGE)

Constructing the ERAMET Digital Research Environment (DRE)

Author(s): Jeff Barrett, Flora Musuamba Tshinanu, Ine Skottheim Rusten, Scott Russell, Kara Lasater, Alicia Gibson, David Sibbald
Published in: 2024
Publisher: Population Approach Group in Europe (PAGE)

Uncertainty quantification in Mixed effects modelling

Author(s): Linda Wanika, Ine Skottheim Rusten, James Kermode, Mike Chappell
Published in: 2025, ISSN 1871-6032
Publisher: Population Approach Group in Europe (PAGE)