Module232025

07/05/2025

The Scientific advice working party does not address questions under PDCOs remit, e.g.:

1. Can we have a waiver/do we need a paediatric formulation, juvenile animal studies, paediatric studies (instead of e.g. an extrapolation approach)? 2. Here is our paediatric development: does this suffice for a paediatric indication? 3. Can we replace X PIP study by this new Y study or can we be exempted altogether from doing X PIP study? PDCO is responsible for the whole plan, whereas Scientific Advice may be requested on details of the plan

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Scientific advice Tight cooperation PDCO/Scientific advice working party: • PDCO is represented in Scientific Advice Working Party • Ad-hoc involvement of scientific advice representatives for relevant PIP procedures E.g. joint PDCO/Scientific advice evaluation of innovative study designs.

Also consider: • Parallel FDA-EMA paediatric advice

• Biomarker qualification advice – e.g. gene therapy • HTA/EMA advice – highly relevant for orphan medicines • Parallel FDA-EMA scientific advice

The Organisation for Professionals in Regulatory Affairs

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