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
2.
3.
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”
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!
20.
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
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?