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 terminology" Samantha Parker, E-IMD, OrphanEuropeTRANSCRIPT
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Centres of expertise (CoE) and European Reference Networkes (ERN)
Samantha Parker, Orphan Europe (Recordati Group)
Presentation of the concepts, definitions and terminologyand
Orphanet analysis of designated centres
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Presentation objectives
Understand the concept and definitions of European Reference Networks (ERN) and Centres of Expertise (CoE)
Appreciate Orphanet analysis of designated centres and key areas of action; experience from EPNET, EUROWILSON, EUNEFRON and E-IMD
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Working for Orphan Europe (Recordati group) for 11 years
Member of EUCERD Orphan Europe has 8 Designated Orphan Drugs
and 4 with Market Autorisations Mostly very rare diseases i.e. NAGS deficiency Philosophy of collaboration with the rare disease
community of stakeholders to build sustainable health-care systems: value to patients, healthcare professionals, payors
Personal background
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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
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CoE and ERN Concept
IV. CENTRES OF EXPERTISE AND EUROPEAN REFERENCE NETWORKS FOR
RARE DISEASES
11.Identify appropriate CoE … by the end of 2013 and consider supporting their
creation
12.Foster the participation of centres of expertise in ERN…
13.Organise healthcare pathways …through the …cooperation with relevant experts
and exchange of …expertise within the country or from abroad …
14.Support the use of information and communication technologies i.e. telemedicine
15.[support] the diffusion and mobility of expertise and knowledge in order to facilitate
the treatment of patients in their proximity
16.Encourage COE to be based on a multidiscplinary approach …
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Criterea for the designation of CoE & ERN
Criterea for CoE
according to:
•Europlan
recommendations
•High level group on
Health services and
Medical care
Additional criterea for
ERN
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MS are encouraged to faciliatate the development of the ERN 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
By fostering the participation of healthcare providers and centres of expertise in the ERN
The networks shall be based on voluntary participation by its members
Cross border directive, Article 12, ERN’s
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1. 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
2. UK: Metabolic pathways networks of care (inborn errors of metabolism): 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
Examples of Centres of Expertise
Paris
Lyon
Toulouse Marseille
Besançon
Bordeaux
Rennes
Lille
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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
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Linking ERN’s and CoE’s: EuroWilson
Paris
Lyon
Toulouse Marseille
Besançon
Bordeaux
Rennes
Lille
Patient organisationCentre of expertise
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Four general areas of action (EUCERD report)
1. Identifying expertise/networking2. Sharing expertise for patient management3. Building standards of care4. Improving clinical Research
Reference: EUCERD report « preliminary analysis of the outcomes and experiences of pilot European Reference Networks for rare diseases, February 2011
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Identifying expertise
1. Small group with primarily a research vision
2. Interest from other centres to join
3. Establishment of membership criterea
4. Joining with other networks and patient groups
5. Information network for patients and training for healthcare professionals
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Networking: coordination
Associate partners
Collaborating partners
• Multi-stakeholder: Academics, patient groups, regulators, ethics, industry
• Clear structure and consortium agreement• Monitoring committment to the network
Disease specific patient representatives supported by Eurordis
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Sharing expertise: information in patients’ own language
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Systematically developed statements that assist practitioner and patient decisions in appropriate healthcare for specific clinical circumstances
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
Sharing expertise: best practice guidelines
definition
1. Expert opinion2. Data from the registry i.e. incidence of liver cancer in AIP 3. Consensus protocoles i.e. SIGN methodology
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Sharing expertise: best practice guidelines
Collaboration with other networks/groups i.e. USA
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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
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Geographical distribution of all patients followed in France by the
Reference Centres
Geographical distribution of patients diagnosed from 01/05 in the EW database
Sharing experience: registries (EuroWilson)
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Share core data sets
SOP
Sharing experience: registries (E-IMD)
EU registry
Industry registry EU
Industry registry US
USLongitudinal study
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Conclusions
A CoE is a national centre of expertise in a specific
disease or group of diseases supported by MS
An ERN is a disease specific European network of CoE’s
The cross border directive provides recommendations for
collaboration between CoE ’s and ERN’s: sharing
registries, guidelines, telemedicine etc.
Role of patient groups, alliances and Eurordis needs to be
optimised
Think beyond the borders of Europe