Workshop 2 - Centres of expertise & Networks of experts "Presentation of the concept, definitions and terminology" Samantha Parker, E-IMD, OrphanEurope

1. Centres of expertise (CoE) and European Reference Networkes (ERN) Samantha Parker, Orphan Europe (Recordati Group) Presentation of the concepts, definitions and terminology and Orphanet analysis of designated centres

4. CoE and ERN Concept 5.1. Improving universal access to high-quality healthcare for rare diseases, in particular through development of national/regional centres of expertise [also called centres of reference or excellence) and establishing EU reference networks Recommends that MS contribute to the identification of their expert centres and support them financially Article 15 of the cross-border directive provides for the development of ERN to be facilitated by Member States

9. The 10 pilot EU reference networks (DG Sanco) There are many more European Reference Networks founded before the concept of ERN’s or funded by DG research, or existing with reduced funds and activities

10. Linking ERN’s and CoE’s: EuroWilson Patient organisation Centre of expertise Paris Lyon Toulouse Marseille Besançon Bordeaux Rennes Lille

14. Sharing expertise: information in patients’ own language

16. Sharing expertise: best practice guidelines Collaboration with other networks/groups i.e. USA

17. Sharing experience: quality assurance Partner with network for external QA i.e. EMQN Set up internal QA i.e. Porphyrias Long-term sustainability through subscription fees

19. Sharing experience: registries (E-IMD) Share core data sets SOP EU registry Industry registry EU Industry registry US US Longitudinal study