1. 1

2. 2 Treaty establishing the European Community (EU-6) Signing Ceremony in Rome, Campidoglio - March 25, 1957 Welcome

3. 3 AIFA – The Italian Medicines Agency Governanance and maintenance of the economic ceiling Marketing Authorisation Pharmacovigilance Clinical trials Independent research Drug Communication Pricing & Reimbursemnt GMP-GCP Inspections

4. 4 The key (common) issues The assessment process: how safe and effective a medicine is Pricing & Reimbursement: which patients will benefit most The transferability of the b/r assessment into in the real life Public funding of drug research for a better decision making process: an impossible task for National Agencies?

5. 5 Problems coming from the European MA Process Source: J. Avorn, Circulation 2006 Place in therapy of newly licensed drugs ATV (Added Therapeutic Value) assessment for new drugs

6. 6 Innovation and cost-effectiveness The case of new anticancer drugs Poor predictivity of responders Patients with disease progression failures) identifiable only at follow-up Reimbursement of innovative drugs as a function of their real effectiveness Sharing Innovation – Risks - Costs

7. National Registry of new anticancer drugs

8. 8 Drug Female N. Male N. Total (F+M) N. Total (F+M) % AVASTIN779917611.1 ELOXATIN22129051132.2 EMEND77140.9 ERBITUX14920335222.2 FASLODEX4340 27.4 FOSCAN0220.1 GLIADEL69150.9 TARCEVA2437613.8 ZEVALIN129211.3 Total9306561586100.0 Distribution of patients treated by drug and gender N. of Patients: 1586

9. 9 http://oss-sper-clin.agenziafarmaco.it/

10. 10 8* 61* 12 10 24 18 14 10 30 13 1* 4 18 14 26 4 5 35 4 2* OSSERVATORIO NAZIONALE Ministero della Salute Ethics Committes in Italy: Ethics Committes in Italy: 309

11. 11 Clinical Trials per Phase Phase Nr. CTs % Phase III1.89953.8 Phase II1.19834.0 Phase IV3068.7 Bioeq / Biod702.0 Phase I54 1.5 Totale3.158 100

12. 12 CTs per Year and Phase %%% Nr. CTs Phase III Phase II Phase IV Bioeq / Bioav Phase I 200056061,627,57,92,10,9 200161053,833,58,83,10,8 200256552,436,472,32 200356553,735,47,71,32 200460753,235,18,121,5 200562049.536.09,81,12.3 Total3.15854,533,98,12,11,5 Year

13. 13 Clinical Trials per Therapeutic Classification

14. 14 The Italian Clinical Trials Registry The general sense and direction of research Which diseases are investigated by which drug(s) and strategy The degree of innovative research in real life The key issues

15. 15 Independent Research AREAs Orphan drugs and rare desease Head to head clinical trials Pharmacovigilance and appropriateness Promoted and funded by AIFA

16. 16 Aims of the conference To discuss The best way to implement evidence in regulatory decisions To consider Licensing, pricing, reimbursement and impact on population health To share Experiences and points of view

17. 17

18. 18

19. 19

20. 20