Regulatory model-informed drug discovery and development in EU – News flash and examples
Kristin E. Karlsson(1),(3) and Flora Musuamba Tshinanu(2),(3)
(1) Swedish Medical Products Agency, Uppsala, Sweden, (2) Federal Agency for Medicines and Health Products, Brussels, Belgium, (3) EMA Modelling and Simulation Working Party
Objectives: To provide an update on modelling and simulation involvement in the EU regulatory activities and procedures and highlight the latest news regarding MIDD in EU regulatory framework and to present examples of procedures where model-based approaches were essential for decision making.
Overview/Description of presentation: The presentation will provide some key statistics on M&S related regulatory activities and highlight some regulatory documents where model-based approaches are described and promoted. Examples of documents that have been published within the last year are Extrapolation Reflection Paper[1], Modelling and Simulation Working Party Paediatric Q&A[2], and Guideline on the reporting of physiologically based pharmacokinetic modelling and simulation[3]. Furthermore, there is ongoing work within various aspects of paediatric drug development, and the strategy document EMA Regulatory Science to 2025.
The second part of the presentation will focus on EU regulatory examples where model-based approaches played a vital role in the development and authorization of medicinal products. There are numerous examples of where model-based approaches have proven to be pivotal for the benefit/risk assessment in a market authorization application. Other regulatory interactions with increasing visibility of model-based drug development are central scientific advices (through the Scientific Advice Working Party) and paediatric investigation plans (through the Paediatric Committee) will be highlighted.
References:
[1] https://www.ema.europa.eu/en/documents/scientific-guideline/adopted-reflection-paper-use-extrapolation-development-medicines-paediatrics-revision-1_en.pdf
[2] https://www.ema.europa.eu/en/human-regulatory/research-development/scientific-guidelines/clinical-pharmacology-pharmacokinetics/modelling-simulation-questions-answers
[3] https://www.ema.europa.eu/en/about-us/how-we-work/regulatory-science-2025
