Module232025

12/05/2025

Role of regulators

• Regulators have a defined mandate (based on [paediatric] regulation(s))

• A (PIP) life-cycle approach to evidence underpins decisions making – stepwise PIP

• Acknowledgement that choices must be made within the drug development ecosystem - taking into account that not every development/ PIP will be successful

• Driver for regulators is to facilitate and support these choices as part of this ecosystem

https://www.tandfonline.com/doi/full/10.1080/21678707.2018.1491304 https://ascpt.onlinelibrary.wiley.com/doi/10.1002/cpt.1939 https://www.sciencedirect.com/science/article/pii/S0959804922007651?via%3Dihub

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PIP life cycle

PIP agreed in certain disease of unmet need

Marketing Authorisation Application

1. Dose finding; PK, PD; activity

Clinical proof of concept

2. Pivotal evidence generation

Pre specified criteria go/ no go decision

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