1. IRCCS San Raffaele, Rome
From bench to bedside: making the right question getting the right answers
Carlo Tomino Pharm.D.
IRCCS San Raffaele, Rome

2. 2

3. CLINICAL Research: how we can get there?3

4. 4

5. We need (for the IMP):
QUALITY data and
PRE-CLINICAL data 5

14. Than, we can move to the CLINICAL TRIAL14

15. PRE- MARKETING 15

18. 18

19. POST-MARKETING 19

20. Effectiveness (in the “real world” condition)
After Marketing authorization, we need to study more in depth the new product
Safety problems
Cross reaction
Effectiveness (in the “real world” condition) 20

21. Post- marketing studies
Post Authorization Safety study (PASS)
Post-Authorization Efficacy Study (PAES)
Observational study
Real world data (public registries) 21

22. The Register of post-marketing use
Since the second half of 2005, AIFA (the Italian Medicines Agency) authorised for the marketing new drugs highly innovative (and very expensive). For their use, case report forms must be filled in, in an appropriate web-based Monitoring Registers. 22

23. 23

24. increasing the patient’s protection by promoting
The Register of «post-marketing use» role:
increasing the patient’s protection by promoting
drugs’ appropriate use 24

25. verifying the real efficacy and tolerability of drugs registered by shortened processes25

26. A cultural revolution…26

27. each project’s participant must assume his own responsibilities
a new concept:
‘Sharing the Risk’
each project’s participant must assume his own responsibilities 27

28. Cost Sharing, Risk Sharing e Payment by Results
Three different procedures for the accountability of the involved actors. 28

29. First therapy cycle for eligible patients
Treatment continues - entirely paid by the National Health Service (SSN)
Responders
First therapy cycle for eligible patients
Response’ evaluation
Drug
Treatment stops
Non Responders
Agreeements with Companies in order to define percentage to be paid by the National Health Service (SSN) 29

30. PRE & POST 30

31. DISCOVERY AND PRE-CLINICAL31

32. PTs. EC PI BR CRO Gov Sponsor
For doing this, great collaboration among all actors (and Basic Researchers!!!)
… IS MANDATORY !!!
PTs. EC PI BR
CRO
Sponsor
Gov 32 32

33. Thank you 33