Module232025

09/05/2025

Simulated PD summary plot (with SD):

The Organisation for Professionals in Regulatory Affairs

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NCE for treatment of a rare disease in adult and paediatric patients aged ≥2 to <18 years. Paediatric development (via PIP agreement) mandatory for a new chemical entity (NCE) with potential benefit in children Indication targeted by PIP:Treatment and prevention of in paediatric patients with ≥2 to <18 years of age. Same condition and pathophysiology as in adults PK matching exposure and PK/PD metrics above target to support extrapolation Use popPK modelling to select study dose levels targeting the adult exposures in the various paediatric populations 2. Confirm the adequacy of these dose levels in pediatric studies with PK as a primary measurement 3. Assess safety in pediatric studies with safety as a secondary measurement 4. Extrapolate efficacy from adults while verifying the extrapolation concept through inclusion of exploratory efficacy measurements in pediatric studies Agreed PIP General clinical strategy for paediatric development: 1.

The Organisation for Professionals in Regulatory Affairs

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