Criteria for Centres of Expertise for Rare Diseases in the EU following EUCERD Recommendations

1. Criteria for Centres of Expertise for Rare Diseases in the EU following EUCERD Recommendations RARECARENet Project: Consensus meeting on centres of expertise Leiden, 15th January 2013 Teresinha Evangelista

2. Centres of expertise (CEs) for rare diseases THE CONTEXT

3. The need for CEs for rare diseases • There are over 6’000 rare diseases • The challenge of rarity : • Patients are rare • Experts are rare • Gathering experience to improve knowledge, care, diagnostic and treatments

4. CEs in national plans/strategies for RD • EU Member States (MS) are elaborating national plans/strategies for rare diseases to be concluded by 2013 • CEs are key components of these RD plans/strategies • CE will be the building blocks of future European Reference Networks foreseen in the Cross-Border Healthcare Directive

5. Heterogeneity in the establishment of CEs • In form • Reflecting heterogeneity of national health care systems • Depending on budget allocation • In focus • Specialised centres vs general centres • Clinical only vs clinical research • Focus on technology / expert intervention • In designation process • Specific policy regarding RD or not • National vs regional approach

6. Aim of EUCERD recommendations: “Help MS in their reflections/policy developments concerning national plans and strategies for RD”

7. Scope of the Recommendations 45 recommendations 4 main areas: • Mission and scope of CEs • Criteria for designation • Process for designation • European dimension

8. Recommendations:Mission and scope • Definition • Coverage • Patient focus • Core competencies • Role in spreading information and education • Role in research

9. Mission and scope Definition of what is a CE: • Infrastructures for the management and care of RD patients on regional, national or international level

10. Mission and scope Coverage of CEs: • CEs should cover all RD patients’ needs • CEs liaise with other CEs at National or European level • A national directory of designated CEs should be compiled and made public

11. Mission and scopePatient focus of CEs • CEs collaborate with patient organisations • CEs respond to the needs of patients from different cultures and ethnic groups (ie have cultural sensitivity) • CEs ensure respect of non-discrimination and non-stigmatisation of RD patientsacross Europe

12. Mission and scopeCompetencies of CEs • CEs bring together or co-ordinate multidisciplinary competencies/skills (including paramedical skills, social services, rehabilitation and palliative care…) • CEs contribute to building healthcare pathways from primary care • CEs have links with specialized laboratories and other facilities

13. Recommendations: Criteria for designation of CEs • Leadership and credibility • Multidisciplinarity and inclusiveness • Capacity • Links and collaborations • Mechanisms for measuring performance/ evaluation

14. Criteria for designation of CEsLeadership and credibility • High level of expertise and experience documented for example by: annual volume of referrals and second opinions, peer reviewed publications, grants, number of experts, teaching and training activities • Contribution to state of the art in research

15. Criteria for designation of CEsMultidisciplinarity and inclusiveness • Multidisciplinary approach, integrating medical, paramedical, psychological and social needs • Appropriate capacity to manage RD patients and provide appropriate advice • Organisation of collaborations to ensure continuity of care • Between childhood, adolescence and adulthood • Between all stages of the disease

16. Criteria for designation of CEsCapacity of CEs • Improve the delivery of care and shorten the time to diagnosis • Produce and adhere to good practice guidelines • Propose quality of care indicators and implement outcome measures • Participate in data collection for clinical research and public health purposes • Capacity to participate in clinical trials • Capacity to develop and participate on E-Health solutions

17. Criteria for designation of CEsLinks and collaborations • With patient organisations where they exist • With other CE at national, european or international level

18. Recommendations: Process for designation of CEs • Core principles of designation • Designation criteria • Duration of designation

19. Process for designation of CEsCore principles • MS are responsible for: *Establishing transparent designation criteria *Designation of CEs *Maintain a ongoing evaluation process *Facilitate access of citizens • The designation process at MS level must ensure that the designated CEs have the capacity and the resources to fulfill the obligations of designation

20. Process for designation of CEsDesignation criteria • CEs may not fulfill some of the designation criteria defined by the MS so long as: * Absence of fulfillment of criteria does not impact on the quality of care * CEs have a strategy in place to attain designation criteria in a defined time period

21. Networking • Networking of CEs is a key element, to ensure: * expertise travels rather than patients; * exchange of data, biological samples, radiological images, other diagnostic materials * promotion of e-tools for tele-expertise “Designated CEs at MS level are the key elements of the future ERNs”

22. Thank you for your attention! The text of the recommendation can be found on the EUCERD’s website www.eucerd.eu