1. European Clinical Trial Directive (Directive 2001/20/EC) dr. Cees Smit (NPCF/EGAN) EPF Annual Meeting May 19, Brussels

2. European Clinical Trial Directive 1998/1999 Concerns expressed by European Patient Organisations Recommendation: one European research/registration file 2004 – 2006: Implementation at MS level 2007 (Evaluation EU/EMEA October 3, 2007, London) More paper work, bureaucracy 1 Directive, 27 Member States Implementations Less research, less academic research Higher prices, higher insurance premiums, Less research money/grants Patients not represented in Research Ethic Committees

3. The current situation Numerous articles and case-studies have postulated that Directive 2001/20/EC, the ‘Clinical Trial Directive’ has failed to promote efficient clinical research in Europe and to better protect the study participants However, there are more causes for the decreasing clinical research activity in Europe (if so!) than the legal framework alone Consultation EC end 2009, response EPF

4. Response EPF January 19, 2010 (1) Questionnaire EC highly technical The effectiveness of clinical trials throughout the EU is of fundamental importance for EPF and its members. EPF and its members very active in this area: So input drawn on evidence from Value+, PatientPartner (VSOP, EGAN, EFGCP), Involve, Respect, EMA/EPF, Europa Donna, IDF-EU

5. Response EPF January 19, 2010 (2) Meaningful patients’ involvement in clinical trials processes (Eurordis charter OD) Access to quality information at all stages Informed consent in an accessible language Clinical trials for paediatric patients Transparancy regarding clinical trials across EU incl. learning from trials that have failed Access to treatment after the trial

6. Response EPF January 19, 2010 (3) Finally, EPF welcomes the Commission’s initiative to consult the public on the Directive EPF is committed to work with the Commission and other relevant stakeholders in translating the vision and the core issues outlined in their response into more effective, patient-centred EU legislation on clinical trials

7. Commissioner John Dalli Recently told the Parliament that the Clinical Trials Directive is going to be reopened, but it is not clear yet whether it will be a light revision of the text or a full revision of the text. A first draft is expected by Winter 2011

8. Drug development process 12345678910111213141516171819 20 fundamental pre-clinical research Ph. 1 Phase 2 Phase 3 Phase 4 Registration clinical research post marketing research effective patent-period Patent request launch product End of patent Years Ref: Jan Raaijmakers (2005, UU) JR 2005

9. The theory: levels of patient participation in clinical research ref: www.patientpartner-europe.eu 12/09/2015 9 Research subject Information provider Advisor Reviewer Co-researcher Driving force

10. 12/09/2015 10 Outcome PatientPartner survey Co-researcher Advisor Information Provider Research subject Driving Force Reviewer

12. Role of Patient Groups in Research and their Priorities for the Future Fabrizia Bignami Eurordis Therapeutic Development Director Rare Disease Day Workshop Brussels, 1 March 2010 www.eurordis.org

13. 37% of POs financially support research Multisyst Musculosk Neuromusc Haemato Neurology Ophthalmo Oncology Others Dermato Metabolic Cardiovasc Umbrella >75% 60-75% 45-60% 30-45% 15-30% <15% Distribution by disease type Distribution by country

14. 81 % Basic research 57% Therapeutics 56% Diagnosis 46% Human and Social Science 19% Research infrastructures 54% Epidemiology / Natural history of the disease 24% Assistance technologies / Daily life What kind of research do patients fund?

15. Development costs of a new drug

16. The ‘productivity’ gap Input: $ 50 – 80 miljard R&D spending/year Output: 5 – 6 ‘first in class’ drugs/year Payers/HTA adding C + E criteria further reduce the number of products that will reach the patient (Hans-Georg Eichler, TI Pharma, April 28, 2010)

17. For further information smit.visch@wolmail.nl