Module232025

12/05/2025

Existing Clinical data – paving the way

• Existing clinical data

◦ Pharmacokinetic properties ◦ Pharmacodynamic properties

◦ Summary of efficacy data ◦ Exposure-response analyses ◦ Summary of Safety data • Rationale for paediatric dose to be used in clinical studies ◦ Modelling and simulation analyses supporting paediatric development ◦ Description of the M&S analysis proposed and role in the development ◦ Population (PK/PD) Model building methodology and model evaluation. ◦ Physiologically based pharmacokinetic models

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Presentation title

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Extrapolation plan in the PIP Opinion

See Q&A 1.7: How do I discuss use of extrapolation in my PIP - Paediatric investigation plans: questions and answers | European Medicines Agency (EMA)

Essentially all M&S, clinical studies and other studies proposed to be conducted as part of the PIP based on the uncertainties identified during the PIP assessment, based on the information provided in the scientific document

Following approaches as discussed in ICH E11A, that is: • Exposure matching approach • PK/PD (biomarker) approach • Efficacy studies including: ◦ Single-arm studies

◦ Externally controlled studies ◦ Concurrent controlled studies

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https://www.ema.europa.eu/en/ich-guideline-e11a-pediatric-extrapolation-scientific-guideline https://www.ema.europa.eu/en/human-regulatory-overview/research-development/paediatric-medicines-research-development/paediatric-investigation-plans/paediatric-investigation-plans-questions-answers

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