1. An agency of the European Union Agnes Saint Raymond, Human Medicines Special Areas Human Medicines Development and Evaluation Unit How effective is the Paediatric Regulation? Should we change it? Informal CHMP-PDCO Warsaw 2011

2. Measures of effectiveness Is the Regulation producing -More medicines for children (incl. forms/formulations) -More information on medicines for children -Better (ethical) and more clinical trials with children? -Are the resources well used for the results? 2 © EMA

3. 3 For the MAJORITY of medicines used in children (especially in neonates) up to 1997-2000: Medicines had not been studied, assessed and approved for use There were no/few age appropriate formulations, no dose recommendations, no safety information Data came from very few paediatric clinical trials (2-<10% of adult trials in Member states) Where do we come from? © EMA 2011

4. Experience with the centralised procedure 4 289 active substances, until Jan 2006 ~10 paed indications per year © EMA

5. 5 CHMP opinions: January to July 2011 29 New Products 20 Extension of Indications 14 paed indications in 7 months © EMA 2011

6. 6 Paediatric Clinical Trials in EU (and outside) © EMA 2011 EudraCT data: 2,508/29,625 total (8.5%) June 2011

7. 7 Some trials not included in the database Trials not performed for several reasons not mutually exclusive: - Trials are too complex (Industry and PIP/PDCO issue) - Trials are not worth the cost (and no penalties) - Parents don’t include children in trials - Paediatricians not convinced of generating evidence Are the trials not performed? © EMA 2011

8. 8 Article 46 at CHMP in 2010 60 products with 9 SmPC changes Article 46 at CMD(h) in 2010 19 products with 6 SmPC changes Publication of assessment reports (art 45) ongoing More information on paediatric use? © EMA 2011

9. 9 SPC extension and other Rewards? 8 medicinal products with SPC extension at end of 2010 EFPIA data: 18 final compliance – 7 SPC extensions Maximum of 13 MS in which SPC extension granted (plus extensions pending) In some MS, no SPC or SPC refused One PUMA approved with data exclusivity No orphan extension so far © EMA 2011

10. © EMA Paediatric Committee Workload 200820092010Total Applications (indications) 271 (395) 273 (395) 326 (403) 1003 (1420) Opinions on PIP/Waivers 133202260649 Modifications851107199

11. 11 No simple answer to a complex problem Are all these requirements worth the efforts and costs? YES Major achievements Not enough paediatric trials Too complex PIPs? Some aspects can clearly be improved –Some without a change in Regulation –Some would require a change in the Regulation © EMA

12. 12 Should we change the Regulation? In absolute terms YES, but risk to lose more than gain? Keep the obligation of paediatric development Transform the ‘collaboration’ with other committees into a consensus building process Simplification (art 8 authorised products) Progressive PIP Clarification on scope (indication vs condition) Educate paediatricians and families Reward more efficient © EMA

13. Do we really need to change?

14. Making the system more efficient? - example of a NCE Clinical Trials Authorisation PIPSc. Advice Orphan MA Pharmacovigilance Pricing Member States’ responsibility Sc. Advice Pharmacovigilance MA Biologicals, Cancer, Diabetes, Neurodeg. dis, Viral dis, HIV, Immuno, Orphan Generics, non-mandatory scope EMA’s responsibility