Workshop 2 - "Presentation of the concept, definitions and terminology"


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

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  • Can be a huge burden on the pharmaceutical industry
  • Can be a huge burden on the pharmaceutical industry
  • Originally a small group of people that work well toghether: inserm funding Expanded through contacts made: industry, meetings etc Joined other networks i.e. drug database Defined membership criterea Now expanding to develop new centres through diagnostic labs
  • Workshop 2 - "Presentation of the concept, definitions and terminology"

    1. 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
    2. 2. Presentation objectives <ul><li>Understand the concept and definitions of European Reference Networks (ERN) and Centres of Expertise (CoE) </li></ul><ul><li>Appreciate Orphanet analysis of designated centres and key areas of action; experience from EPNET, EUROWILSON, EUNEFRON and E-IMD </li></ul>
    3. 3. <ul><li>Working for Orphan Europe (Recordati group) for 11 years </li></ul><ul><li>Member of EUCERD </li></ul><ul><li>Orphan Europe has 8 Designated Orphan Drugs and 4 with Market Autorisations </li></ul><ul><li>Mostly very rare diseases i.e. NAGS deficiency </li></ul><ul><li>Philosophy of collaboration with the rare disease community of stakeholders to build sustainable health-care systems: value to patients, healthcare professionals, payors </li></ul>Personal background
    4. 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
    5. 5. CoE and ERN Concept <ul><li>IV. CENTRES OF EXPERTISE AND EUROPEAN REFERENCE NETWORKS FOR RARE DISEASES </li></ul><ul><li>Identify appropriate CoE … by the end of 2013 and consider supporting their creation </li></ul><ul><li>Foster the participation of centres of expertise in ERN… </li></ul><ul><li>Organise healthcare pathways …through the …cooperation with relevant experts and exchange of …expertise within the country or from abroad … </li></ul><ul><li>Support the use of information and communication technologies i.e. telemedicine </li></ul><ul><li>[support] the diffusion and mobility of expertise and knowledge in order to facilitate the treatment of patients in their proximity </li></ul><ul><li>Encourage COE to be based on a multidiscplinary approach … </li></ul>
    6. 6. Criterea for the designation of CoE & ERN <ul><li>Criterea for CoE according to: </li></ul><ul><li>Europlan recommendations </li></ul><ul><li>High level group on Health services and Medical care </li></ul>Additional criterea for ERN
    7. 7. <ul><li>MS are encouraged to faciliatate the development of the ERN </li></ul><ul><ul><li>By connecting appropriate HCP and centres of expertise throughout their national territory and ensuring the dissemination of information towards appropriate HCP and centres of expertise throughout their national territory </li></ul></ul><ul><ul><li>By fostering the participation of healthcare providers and centres of expertise in the ERN </li></ul></ul><ul><li>The networks shall be based on voluntary participation by its members </li></ul>Cross border directive, Article 12, ERN’s
    8. 8. <ul><li>France: “Centres de référence labellisés et centres de compétences désignés pour la prise en charge d’une maladie rare ou d’un groupe de maladies rares” e.g. Wilson’s disease </li></ul><ul><li>UK: Metabolic pathways networks of care (inborn errors of metabolism): </li></ul><ul><ul><li>4 large metabolic centres in England and 1 in Scotland (>2 consultants). Additionally there are 6-7 additional smaller centres which are single handed, and they are supposed network with one of the larger centres </li></ul></ul>Examples of Centres of Expertise Paris Lyon Toulouse Marseille Besançon Bordeaux Rennes Lille
    9. 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. 10. Linking ERN’s and CoE’s: EuroWilson Patient organisation Centre of expertise Paris Lyon Toulouse Marseille Besançon Bordeaux Rennes Lille
    11. 11. Four general areas of action (EUCERD report) <ul><li>Identifying expertise/networking </li></ul><ul><li>Sharing expertise for patient management </li></ul><ul><li>Building standards of care </li></ul><ul><li>Improving clinical Research </li></ul>Reference: EUCERD report « preliminary analysis of the outcomes and experiences of pilot European Reference Networks for rare diseases, February 2011
    12. 12. Identifying expertise <ul><li>Small group with primarily a research vision </li></ul><ul><li>Interest from other centres to join </li></ul><ul><li>Establishment of membership criterea </li></ul><ul><li>Joining with other networks and patient groups </li></ul><ul><li>Information network for patients and training for healthcare professionals </li></ul>
    13. 13. Networking: coordination Associate partners Collaborating partners <ul><li>Multi-stakeholder: Academics, patient groups, regulators, ethics, industry </li></ul><ul><li>Clear structure and consortium agreement </li></ul><ul><li>Monitoring committment to the network </li></ul>Disease specific patient representatives supported by Eurordis
    14. 14. Sharing expertise: information in patients’ own language
    15. 15. <ul><li>Systematically developed statements that assist practitioner and patient decisions in appropriate healthcare for specific clinical circumstances </li></ul><ul><li>Provide recommendations for effective practice in clinical situations where variations in practice are known to occur and where effective care may not be delivered uniformly </li></ul>Sharing expertise: best practice guidelines definition <ul><li>Expert opinion </li></ul><ul><li>Data from the registry i.e. incidence of liver cancer in AIP </li></ul><ul><li>Consensus protocoles i.e. SIGN methodology </li></ul>
    16. 16. Sharing expertise: best practice guidelines Collaboration with other networks/groups i.e. USA
    17. 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
    18. 18. Sharing experience: registries (EuroWilson) <ul><li>Geographical distribution of all patients followed in France by the Reference Centres </li></ul><ul><li>Geographical distribution of patients diagnosed from 01/05 in the EW database </li></ul>
    19. 19. Sharing experience: registries (E-IMD) Share core data sets SOP EU registry Industry registry EU Industry registry US US Longitudinal study
    20. 20. Conclusions <ul><li>A CoE is a national centre of expertise in a specific disease or group of diseases supported by MS </li></ul><ul><li>An ERN is a disease specific European network of CoE’s </li></ul><ul><li>The cross border directive provides recommendations for collaboration between CoE ’s and ERN’s: sharing registries, guidelines, telemedicine etc. </li></ul><ul><li>Role of patient groups, alliances and Eurordis needs to be optimised </li></ul><ul><li>Think beyond the borders of Europe </li></ul>