Module232025

07/05/2025

Developmental differences: children not small adults The KEF: Include buffers and use precise wording – to minimise future PIP modifications. Keep close contact with EMA coordinator – make sure you interpret the summary report as intended. Clinical strategy: The paediatric trials must be of a standard to allow for a future paediatric indication. Meaning that demonstration of efficacy is generally expected as a primary endpoint (unless substantial extrapolation for efficacy possible). Clinical trial design: Include sufficient details and justification for each element in the PIP scientific document. Less details in key elements form. M&S and extrapolation can help reducing sample size consistently – remember that extrapolation is an effort requiring development of extrapolation concept and plan Study endpoints: clinically relevant, validated

The Organisation for Professionals in Regulatory Affairs

59

Good luck!

mt@pipadviser.com

The Organisation for Professionals in Regulatory Affairs

60