1. Interactions between CHMP and PDCO; reflections from a CHMP perspective Tomas Salmonson vice-chair, CHMP

2. 5 years of the Paediatric regulation whose objectives are: –to increase the availability of medicines intended for children –to make information on those medicines widely available –to stimulate high quality paediatric research.

3. Early concerns from CHMP Delay in drug development Timing of studies in children PIP vs SA Too "heavy" procedure (for industry and regulators)

4. Early concerns from CHMP Delay in drug development Timing of studies in children PIP vs SA Too "heavy" procedure (for industry and regulators) Relax – I will not try to be the judge

5. CHMP – PDCO interactions PDCO-members (bridge to CHMP)

6. CHMP – PDCO interactions PDCO-members (bridge to CHMP) –has not worked - I still believe my proposal would have been a better one

7. CHMP – PDCO interactions PDCO-members (bridge to CHMP) SAWP

8. CHMP – PDCO interactions PDCO-members (bridge to CHMP) SAWP –250 PIPs received EMA scientific advice or protocol assistance before or after PIP opinion –PDCO members / EMA paed staff have been involved in 205 SAWP procedures – PDCO delegates are automatically involved in paediatric SA/PA –an empty "PDCO-chair " at SAWP –SAWP not consulted in PIP-discussions

9. CHMP – PDCO interactions PDCO-members (bridge to CHMP) SAWP Guidelines

10. CHMP – PDCO interactions PDCO-members (bridge to CHMP) SAWP Guidelines –PDCO involvement in guidelines drafting (e.g.: adopted this year Paediatric addendum for PAH, Clinical investigation of recombinant and human plasma-derived factor VIII products and factor IX)

11. CHMP – PDCO interactions PDCO-members (bridge to CHMP) SAWP Some examples (MAAs)

12. Examples Enbrel; RoActemra (extrapolation of efficacy) –Enbrel: to extend the lower age range in polyarticular juvenile idiopathic arthritis (JIA) from four to two years.

13. Examples Enbrel/RoActemra (extrapolation of efficacy) Buccolam (PUMA)

14. European Medicines Agency gives first positive opinion for paediatric-use marketing authorisation Buccolam recommended for treatment of prolonged, acute, convulsive seizures in paediatric patients from 3 months to 18 years The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given its first positive opinion for a paediatric-use marketing authorisation (PUMA) for Buccolam (midazolam), from ViroPharma SPRL, intended for the treatment of prolonged, acute, convulsive seizures in paediatric patients from the age of 3 months to 18 years. June 2011 (PIP approved August 2009)

15. Dr Agnès Saint-Raymond said: “We welcome this first opinion for a PUMA. Since the coming into force of the Paediatric Regulation in 2007 companies have given too little attention to the incentives for paediatric-use marketing authorisations.”

16. Examples Enbrel/RoActemra (extrapolation of efficacy) Buccolam (PUMA) Schizofrenia (joint SAG, Invega)

18. In summary… Lots of PIPs

19. In summary… Lots of PIPs Many SA related to paediatrics

20. In summary… Lots of PIPs Many SA related to paediatrics Significant interactions between SAWP and PDCO (still room for improvement)

21. In summary… Lots of PIPs Many SA related to paediatrics Significant interactions between SAWP and PDCO (still room for improvement) Few direct interactions between PDCO and CHMP (big room for improvement)

22. In summary… Lots of PIPs Many SA related to paediatrics Significant interactions between SAWP and PDCO (still room for improvement) Few direct interactions between PDCO and CHMP (big room for improvement) Few applications – too early to judge?? –eg: 26 applications for PIPs for PUMAs have been received and 7 opinions have been given by the PDCO. (June 2011)

23. The future…… (?) PRAC - PASS CAT – conditional approvals CHMP – PAES, HTA PDCO – PIP

24. The future…… (?) PRAC - PASS CAT - conditional approvals CHMP – PAES, HTA PDCO – PIP We (CHMP) need a system to initially provide an outline of a follow-up measure and later a more detailed advice (through SAWP). This may be a model that can be explored for other committees