Conference 1 - EURORDIS’ recommendations for National Plans, Yann le Cam, EURORDIS


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Conference 1 - EURORDIS’ recommendations for National Plans, Yann le Cam, EURORDIS

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Conference 1 - EURORDIS’ recommendations for National Plans, Yann le Cam, EURORDIS

  2. 2. RECOMMENDATIONS FORRARE DISEASE NATIONAL PLANS Yann LE CAM Chief Executive Officer, EURORDIS Vice Chair, EU Committee Experts on Rare Diseases (EUCERD)
  3. 3. TABLE OF CONTENT• Introduction Strategic messages• Part 1: Key priorities to be covered• Part 2: Key elements of methodology• Part 3: EUCERD and National Plans• Conclusions Key success factors3
  4. 4. OUR COMMON GOAL To build a unique EU integrated, comprehensive and sustainable strategy to address rare disease patients’ needs everywhere in Europe4
  5. 5. OUR COMMON LONG TERM VISION • Rare diseases is a public health priority which is complex and long term: 30 million patients are or will be affected in the course of their life by one of 5000 to 8000 rare diseases. It will take several generations to address these challenges • Hence, the only possible public policy approach = common strategy across rare diseases, collaboration between all interested parties, EU integrated approach, sharing of intrastructure tools and data so to optimize the European community added value with an International perspective. Act local and short term but always with global and long term view. => 2008-2013 = 2nd EU Public Health Programme + 7th EU Research Framework Programme + Commission Communication + Council Recommendations + National Plans in EU 27 MSs => 2014-2020 = 3rd EU Public Health Programme + 8th Research Framework Programme + EUCERD Recommendations + 2nd generation of National Plans in EU 28 MSs and beyond in Europe5
  6. 6. INTEGRATED • Commission Communication + Council Recommendation + EUROPLAN project’s outcomes + National Strategies & Action Plans = a unique opportunity for an integrated strategy between the European and national levels coordinated by the EU Committee of Experts on Rare Diseases (EUCERD) • The Council Recommendation is not an isolated piece of policy = it provides essential elements for national strategies on rare diseases which are articulated with other EU Programmes such as Research and Public Health, other EU Legislations such as the EU Regulation on Orphan Drugs and the EU Directive on Cross Border HealthCare, or other EU recommendations such as the EU Pharmaceutical Forum => Each main objective of national strategies and action plans is to be articulated with these EU Programmes and EU Legislations6
  7. 7. COMPREHENSIVE • Commission Communication + Council Recommendation + EUROPLAN project’s outcomes + National Strategies & Action Plans = a unique opportunity to elaborate comprehensive research and healthcare pathways for rare disease patients and families from diagnosis, care, information, support, research and treatments • The aim of national strategies and action plans is to address the needs of patients and families. Each main priority area supports the other: information is linked to access to diagnosis which is directly linked to access to experts or centres of expertise which in turn are supporting research and therapy development which are correlated to the existence of both patient registries and patient organisations. => National strategies and action plans must be comprehensive and cover all 5 key priority areas7
  8. 8. SUSTAINABILITY • National plans to be adopted before December 2013 are only a start. • We already need to think about the next plans, the 2nd generation of plans for 2015-2020 • We need to take a long term approach with clear strategies, consistent over time, with adequate resources, to achieve measurable results. • It’s important to build in the first national plans, the indicators on measures being implemented, the studies to collect data about patients & families experience and needs, and the financial mechanism for sustainable actions => 2010-2015 = patients need building blocks for future national and EU strategies ; Policy and financial sustainability is a must at EU & national levels8
  9. 9. Part 1 Key priorities to be covered in national strategies and action plans for rare diseases9
  10. 10. Research Identify ongoing research projects and existing research resources + Identify needs and priorities for basic, clinical, translational and social research Link these activities with centres of expertise Encourage the participation of researchers in EU funded project Create new additional financial resources for research Allocate resources both to transversal infrastructure across rare diseases and to disease specific research projects Cover biomedical, public health and social research => Our objective at European level is to obtain the same approach for EU Research Framework Programmes, to encourage collaboration between researchers and patient groups, to promote public-private partnership when relevant, to put in place new funding mechanisms for the long term sustainability of research infrastructures such as biobanks, databases, registries, clinical research infrastructure… because of the nature of rarity10
  11. 11. Centres of Expertise and European Reference Networks for Rare Diseases • Identify Centres of Expertise or Network of Experts or Experts at national level • Adopt a multidisciplinary approach + comprehensive medical & social care + coordination between hospital & community & home care • Put in place long-term public funding mechanism for continued improvement of quality of care • Support patient mobility across EU (transposition of the EU Directive on Cross Border Health Care) => Our objective at European level is to integrate Centres of Expertise, Networks of Experts and Experts with Reference Diagnostic Laboratories and Patient Registries, involving collaboration with Patient European Networks11
  12. 12. Information and Patient Services • Use the OrphaCode and the future ICD 11 classification • Contribute to the inventory of rare diseases • Support national and regional specific disease information networks, registries and databases using common approach across EU • Raise public awareness: take part in the Rare Disease Day • Support national rare disease information helplines with a free number –toward a unique number in EU • Support web-based information tools such as local Orphanet services and patient groups web services • Develop respite care services and therapeutic recreative programmes for patients and families => Our objective is to progressively create European Networks of Rare Disease Help Lines, Information Centres, Respite Care Services, Therapeutic Recreative Programmes, and, to integrate rare diseases into national social policies12
  13. 13. Gathering of expert opinions • It is essential that each EU 27 MS adopts measures in their national plans to engage into European gathering of expert opinions and to refer to common recommendations or common assessment reports • Main ones: - EUCERD recommendations: Centres of Expertise, European Reference Networks, Population Screening, Diagnostic Tests, Registries, Training of Social Service Providers, Methodology on Social Guidelines - EU Assessment Reports on the Clinical Added Value of Orphan Drugs (CAVOD) => Toward more equal access to high quality care, medicines and to social rights13
  14. 14. Empowerment of patient organisations • Patients and families are not only « end users » of national strategies and action plans = they are carers, health and social actors, managing complex day to day care all along their life span • Patient support & advocacy groups are the best allies to take an active role in shaping research, heathcare and social national and regional policies for rare diseases => Ensure that patients and patients’ representatives are involved at each step of the policy and decision-making processes in the field of rare diseases => Ask support for patient groups and rare diseases national alliances: awareness-raising, capacity building & training, exchange of information, networking and outreach14
  15. 15. Part 2 Elements of methodology for the development, management and evaluation of national strategies and action plans on rare diseases15
  16. 16. GOVERNANCE OF THE PLAN • Steering Committee • All interested parties, including patient organisations, industry, national authorithy on health budget • To develop the strategy and action plan • To coordinate and manage its implementation16
  17. 17. CONTENT OF THE PLAN • A clear written strategy – referring to: The Commission Communication « introduction », « issue » and « objectives » The Council Recommendation « whereas » The specific national context • Clear objectives covering each key priority area of actions • Well identified measures or deliverables + easy to understand + assigned to specific competent authorities or interested parties + possible deadlines • Measurable activities or results17
  18. 18. BUDGET • Each priority or objective should have a well identified budget • When possible, each measure or deliverable should have a well identified budget • Each budget line should have well identified funding source18
  19. 19. ACCOUNTABILITY & TRANSPARENCY • The national strategy and action plans should be made public • Regular meetings of the national steering committees • Annual reporting (a) priority areas by priority areas (b) on each measure or deliverable (c ) on each budget line • Adjustments of the measures, deliverables, assignments, deadlines, budgets • Summary of Steering Committee meetings and Annual reporting should be made public19
  20. 20. EVALUATION & INDICATORS • Common indicators should be shared across Member States • Evaluation of activities and results should be based on the adopted strategy and action plans • Evaluation should include a collection of opinion from all stakeholders20
  21. 21. PART 3 The EU Committee of Experts on Rare Diseases (EUCERD) will help shape the future of rare disease policies at the EU level impacting on national policies21
  22. 22. EUCERD • EU MS/ EEA Countries + candidate countries • EC/EMA + ECDC + pharma indus. + reps of EC funded RD research and public health projects • 8 rare disease patients’ representatives • Sharing of national experiences, promotion of national best practices for research, healthcare pathways and social services (eg: EUCERD Website, EUCERD Workshops, European Conferences on Rare Diseases & Orphan Products, Reports “ State-of-the-Art of Rare Diseases in Europe”) • In the coming years, EUCERD will develop “recommendations” & “guidelines” & “reports” on different key issues to support and guide national plans for rare diseases (e.g. centres of expertise, registries, genetic testing, specialised social services…)22
  23. 23. National Plans in EU: an incremental process EC Commission on Rare Diseases EU Council Recommendation on Rare Diseases EUROPLAN 2008-2011 National EUROPLAN 2012-2014 Plans on & Reports « State of Recommendations + Rare Monitoring Indicators the Art of Rare Diseases Diseases in Europe » EUCERD Recommendations & Guidelines23
  24. 24. Present / Future: Integration & Cooperation at Country AND Thematic Level Commission Communication € / CZK / £ … Centresof Expertise EUCERD Registries € Diagnosis Treatment Council Recommendation
  25. 25. 1.What is the EUCERD?
  26. 26. CONCLUSION : KEY SUCCESS FACTORS Focus on essential building blocks Keep sight of the integrated, comprehensive and long term strategy Follow up (get involved) and implement the EUCERD Recommendations Collect your national indicators Build-in the sustainability and think on which base you will promote your 2nd national plan26
  27. 27. CONCLUSION: KEY SUCCESS FACTORS • National plans and strategies can only address part of the needs of 30 million patients affected by all rare diseases for which there are no cure today! • By joining our forces and going in the same direction based on common strategies, we can already improve the lives of million of people living with rare diseases in the next five years and much more within next ten years27
  28. 28. EURORDIS Concrete tools & policy • Where can I find information about national plans from all Member states (regularly updated)? - Section « Rare Disease Policy » / « National Policy » - => - Section « Rare Disease Policy » / « EU Policy » - Information in English • Where can I find practical tools to support your advocacy work? - => - Policy Fact Sheets - Ex: Research, Centres of Expertise & European Network, Registries, Help Lines, Orphanet, Respite care Services, Access to Orphan Drugs… • Where to exchange information and experiences to build my capacities as a patient advocates for national plans? - Eurordis Membership Meeting 2011 Amsterdam 13-14 May (&2013)28
  29. 29. Thank you!29
  30. 30. 30