1

2

>70300

˜100

3

>2 000

>380 000

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

11

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 PDCO

13

Early

14

PIP applications

EMEA received after 12 months of activities (by submission deadline 20.12.2008)

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

Applications by therapeutic area

19%

17%

13%5%

2%

2%

3%

5%

5%

6%

5%

8%

5%

2%

3%

Oncology

Endocrinology,gynecology, m etabolismCardiovascular

Neurology

Pain

Im m unology

Haem atology

Vaccines

Other

Infectious diseases

Pneum ology/Allergy

Uro/Nephrology

Psychiatry

Ophtalm ology

Diagnos is

Facilitate Research

16

RapporteurRapporteur

17

Peer ReviewerPeer Reviewer

18

www.emea.europa.eu

19

Thank you