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  • 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. 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!
  • 3. 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)
  • 4. A result of collaborative action European Commission Rare Disease Community Member Member Member Member Member Member State State State State State State
  • 5. 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?
  • 6. 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
  • 7. 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!
  • 8. 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
  • 9. 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
  • 10. 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
  • 11. 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
  • 12. 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)
  • 13. 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
  • 14. 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”
  • 15. Eurordis & Nord working together
  • 16. 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!
  • 17. 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
  • 18. 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
  • 19. 10th Cooperative Policy-Making Forum = EPPOSI = Patients + Science + Industry www.epposi.org
  • 20. 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?
  • 21. THANK YOU! wills.hughes-wilson@genzyme.com +32 2 714 1743