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

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

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

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

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 …

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

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

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

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

Linking ERN’s and CoE’s: EuroWilson

Paris

Lyon

Toulouse Marseille

Besançon

Bordeaux

Rennes

Lille

Patient organisationCentre of expertise

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

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

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

Sharing expertise: information in patients’ own language

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

Sharing expertise: best practice guidelines

Collaboration with other networks/groups i.e. USA

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

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)

Share core data sets

SOP

Sharing experience: registries (E-IMD)

EU registry

Industry registry EU

Industry registry US

USLongitudinal study

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