Module232025

12/05/2025

A PIP life-cycle approach to evidence underpins decisions making – stepwise PIP pilot

Number of requests: 28 (no information on therapeutic area for 2 requests)

Submitted sPIPs: 15

Refused during PDCO discussion: 1

Opinions adopted: 7 (and 1 in January 2025)

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Reasons for applying

• Data on activity against paediatric cancers need to be generated

• Challenges with development in ultra-rare conditions for dose finding and endpoints

• Lack of relevant animal model , no established PD markers or endpoints

• Study in infants first (most affected), then in older children and adolescents

• Collection of natural history data first, before defining the PIP

• Condition in children different to adults , no proof of concept in humans yet

• Rare, life-threatening disease, recently identified in children , no approved medicine no regulatory guidance and no validated endpoints

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