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2 > ˜100

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4 Paediatric regulation

5 Key objectives of the new EU Paediatric Regulation –To improve the health of the children of Europe by: increasing high quality research for medicinal products for children promoting the development and authorisation of such medicines at the EU level improving the information on medicines designed for children –While avoiding unnecessary studies in children and not delaying the authorisation of medicines for adults

6 EU Paediatric Regulation 3 Pilars Paediatric Investigation Plan (PIP) Reward (incentives) for studies conducted Paediatric Committee (PDCO) at the EMEA

7 PIP Includes timing of planned studies for all the paediatric population subsets applicable to 1.Pharmaceutical development (paediatric forms) 2.Pre-clinical studies (such as juvenile toxicity) 3.Clinical studies in children Subsets of population to be considered (neonates, infants, children, adolescents)

8 EU Paediatric Regulation Key elements Mandatory paediatric development for new products according to a PIP agreed upon by the PDCO (possible deferrals or waivers) Mandatory submission of paediatric data when filing new applications unless waiver or deferral approved by the PDCO New Marketing Authorisation Procedure for off-patent products (PUMA)

9 Incentives Incentives for new medicinal products and line extensions of patented medicinal products: Reward for studies conducted: 6-month extension of the patent or the supplementary protection certificate (SPC) Covers new indications, new pharmaceutical forms and new route of administration For orphan medicines, 2-year additional market exclusivity (10+2)

10 Incentives Incentives for off-patent medicines specifically developed for children New type of Marketing Authorisation (PUMA) Paediatric Use Marketing Authorisation Covers only the paediatric indication(s) and formulation(s) Enabling 10-year (8+2) data protection

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12 COMPOSITION: 5 CHMP members + 1 members per Member State not yet represented + 6 members from families & HCP associations Each member has an alternate PDCO

13 Early

14 PIP applications EMEA received after 12 months of activities (by submission deadline ) PIP applications* 356 corresponding to Number of indications covered in the requests for PIPs or waivers: 596 including full waivers for the paediatric development for the whole indication 58 * ‘Application’ covers one or more indication(s)

15 Facilitate Research

16 Rapporteur

17 Peer Reviewer

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19 Thank you