1. European Policy Actions
Cooperation in the field of Rare Diseases
“Share an inspired vision in rare diseases”
Wills Hughes-Wilson
Health Policy Europe, Genzyme
Cluj, 26 June 2009
Balkans Congress for Rare Diseases

4. EU Orphan drug policy – an ongoing process
 1999 – EU’s Orphan Regulation adopted
 2001 – First Orphan product authorised in EU
 2003 – Eurordis survey: “issues remain”…
 2005 – 2nd Eurordis survey: “issues still remain!”
 2005 – Eurordis calls for action to address
 2007 – First draft of an EU policy document appears
 2008 – Public consultations
 2009 – EU Communication & Recommendation!

5. Results
Recommendation & Commission Communication
National Rare Disease EU proposals on access,
Plans in all EU countries NBS, Compassionate Use…
8-9 June 2009(?) 2009 onwards…
Political commitment Mechanisms & machinery
at country level at EU level
ACTION: ACTION:
Needed at government Needed at both
level in each country (1) EU / Commission level &
(2) Coordinated country action(s)

6. A result of collaborative action
European Commission
Rare Disease Community
Member Member Member Member Member Member
State State State State State State

7. Why is this important?
 Issues of key importance to the whole rare disease
community
 Proposed fundamental changes to EU approach to
diagnosis, treatment and care for rare diseases
 Work done – internal & external
 Where are we now? And where next?

8. What did we
already do together to get here?
 Internal engagement
• Internal conversations
• Messaging, process & engagement – countries
• Training & workshops
• Programme of meetings
 Outputs
• Internal positioning – deciding what is key?
• External positioning
• Delivering the messages
 External engagement
• Other stakeholders
• Decision-makers: EU Commission, EMEA & Member States

9. EU Orphan drug policy – an ongoing process
 1999 – EU’s Orphan Regulation adopted
 2001 – First Orphan product authorised in EU
 2003 – Eurordis survey: “issues remain”…
 2005 – 2nd Eurordis survey: “issues still remain!”
 2005 – Eurordis calls for action to address
 2007 – First draft of an EU policy document appears
 2008 – Public consultations
 2009 – EU Communication & Recommendation!

10. What remains to be done?
 Stimulate / engage in lobbying at country level on
National Plans
=> 8-9 June 2009: ACTION
 Engage with European Commission
• Conditional reimbursement
• Other actions: NBS, Registries, etc.
 What is happening? When? Opportunities to
engage?
 Identify goals / objectives in engagement
• Focus & priorities
• Roles & responsibilities

11. National Plans for Rare Diseases – 2009 onwards
Recommendation
National Rare Disease
Plans in all EU countries
8-9 June 2009(?)
Political commitment
at country level
ACTION:
Needed at government
level in each country

12. National Plans for Rare Diseases 2009-2014
 What could be in an ideal National Plan?
 Multi-stakeholder committee – tailor the plans
 Toolkit – “what’s happening in other countries?”
 Lobbying essential at country level
 Collaboration at country level
 National Rare Disease Days 2010

13. Cooperation – National Plans
Council Recommendation
(& Commission Communication)
Adopted June 2009
“NATIONAL RARE DISEASE PLANS” – 2011
Cooperative efforts, e.g., “Europlan”
Member Member Member Member Member Member
State State State State State State

14. Results – 2009 and next 5 years…
Commission Communication
EU proposals on access,
NBS, Compassionate Use…
2009 onwards…
Mechanisms & machinery
at EU level
ACTION:
Needed at both
(1) EU / Commission level &
(2) Coordinated country action(s)

15. European Commission Communication
on “Rare Diseases – Europe’s Challenges”
1. (Introduction / background)
2. Improve political recognition
3. Recognition & visibility of RD
4. European Cooperation & Improve Access to Quality
Healthcare
5. International Cooperation – ICORD, NIH, WHO
6. Governance & Monitoring

16. Facilitating Access / Conditional Reimbursement
Council Commission
Recommendation Communication
Rare Disease National Plans EU proposals
Art. 5(1)(c) Art. 5(3)
“Member States to “European Commission
cooperate on to set up a Working Party
Common Assessment Reports with Member States &
Clinical / Therapeutic EU authorities on the
Added Value of orphan drugs” scientific assessments of
clinical added value
of orphan drugs”

18. Eurordis & Nord working together

19. International Cooperation
 Regions cooperating to share
 But also learning from experience
 International forum:
• World Health Organisation
• ICH – International (regulatory) harmonisation efforts
• ICORD – International Conference on Rare Diseases
• What is in an ideal national plan?
• What is the minimum a government needs to do?
 Bringing ideas, experience – do not all need to start
from the beginning any more!

21. 2009 – a year of change at European level
 Commission & Parliament will change
• Some dossiers have been postponed
• But not rare diseases!
 Re-appointment of European Commission
• New work plans & focus?
• Engagement now & friends!
 European Parliament elections
• New dossiers, new responsibilities
• Build new contacts

22. What remains to be done?
 Stimulate / engage in lobbying at country level on
National Plans
 Engage with European authorities & stakeholders
 What is happening? When? Opportunities to
engage?
 Identify goals / objectives in engagement
• Focus & priorities
AND
• Roles & responsibilities

23. 10th Cooperative Policy-Making Forum
= EPPOSI = Patients + Science + Industry
www.epposi.org

24. Conclusions?
• There is a lot happening. And a lot of opportunities to
directly affect the outcome!
• Cooperation is essential. We cannot be successful without
cooperation
• Objective = to achieve the best outcome for the rare disease
community
• We have achieved a lot together already!
THE FUTURE?
Where do we go from
here?
How can we work
together to be
successful?

25. THANK YOU!
wills.hughes-wilson@genzyme.com
+32 2 714 1743