Module232025

09/05/2025

M&S in paediatric drug development

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M&S allows to – Predict exposure in different paediatric ages departing from PK in adults or older children – popPK methods – Allometric scaling – Maturation-based semi-physiologic scaling – PBPK This allows to optimize paediatric studies with regard to: – Dose finding – PK sampling schedule – Sample size

The Organisation for Professionals in Regulatory Affairs

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M&S in paediatric drug development M&S also provides the framework for extrapolation of efficacy ● Bridging with PK/PD/Efficacy data available in adults ● Rationalize gap filling studies ● ensure an adequate representation of data from the target population – Otherwise bias towards the parameters of the reference population Possible approaches: ● Exposure matching ● Probability of Target attainment ● Biomarker bridging ● estimate the quantitative relationships between biomarkers and clinical efficacy

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The Organisation for Professionals in Regulatory Affairs

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