ENCE Kick off Meeting - Frankfurt - 28 April 2009
Centres of Expertise for Rare Diseases and European Reference Networks
Recommendations from the RDTF and the High Level Group on
Health Services and Medical Care
Ségolène AyméTask Force on Rare Diseases
European CommissionDG Public Health
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Rare Diseases Task Force (RDTF)
Established in 2004Sept 2005: Issue of a first report on CR in Europe
Ad hoc expert group 2 workshops (June and September 05)
Dec 2005: RDTF meeting: Update the 2005 reportMarch 2006: report to HLG on plans
Sept 2006: Workshop of the expert groupDec 2006: Publication of the second report
March 2008: Workshop of the expert group
July 2008: Publication of the third report
ENCE Kick off Meeting - Frankfurt - 28 April 2009
I- Centres of Expertise / Reference
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Expert Centres have always existed in practice…
Hierarchy of knowledgeable experts– Teachers / Authors of articles– Leaders of opinion
Technical platforms– Rich institutions with developped services– Research institution with innovative services
BUT
The information was restricted to a few professionals
No objective criteria
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Reasons to move to a more formal approach…..
Budget constraint in teaching hospitals– Annual budget to serve the area population– Strict budget for research activities– Analytical budget
Regulation of clinical research– More administrative constraints– Higher costs
Accessibility of the information through Internet– Second opinion requests by phone, fax, e-mail– Mobility of patients / free choice
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Expert Centres for RD seem to be needed….
Patients are rare but experts are rare too….– Need to identify them
Expertise may be found only at International level– Impossible for most countries to offer appropriate services to
all patients
Clinical research is badly needed– Requires cohorts large enough– Systematic collection of data– Production of clinical recommendations
ENCE Kick off Meeting - Frankfurt - 28 April 2009
But patients should not have to travel….
Financial constraints– Cost of travel + accomodation
Communication constraints– 23 languages in Europe + cultural differences
Logistical contraints– Expert departments have a limited number of beds
and of clinics
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Centres of Reference
Official CR for Rare Diseases:– Denmark, France, Italy, Sweden– In preparation: Spain….
Official networks of CR:– France, Germany, Spain
Official CR but not for Rare Diseases specifically:– Austria, Belgium, Finland, Greece,
Ireland, Netherlands, Spain, UK Non official CR / established by
reputation: all countries
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Statements consensus reached
European added value of expert centres Need for disseminating the information Definition of what is an expert centre
– High level of expertise / high volume of activity / capacity to provide expert advice / multidisciplinary approach / links with other centres and patient organisations
Priority for networks of centres Rejection of hierarchy between European
centres and national/regional centres
ENCE Kick off Meeting - Frankfurt - 28 April 2009
The definition of what is a CE/CR differs from one country to another…
Definition of rare disease differs
Size of the population to serve differs
Definition of the task differs
Number of diseases to be covered differs
Process to identify CR differs
Financial support differs
Purpose for establishing CR differs
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Topics with disagreement (1) Balance benefice / malefice
– Positive• Improving access for EU citizens• Maximising cost /effectiveness• Helping to share knowledge / training
– Negative• Distant referral / long waiting list• Psychological burden: language + isolation• Loss of trust in local services• Loss of interest by other professionals
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Topics with disagreement (2)
Scope of activity– Disease centred
• By disease• By medical specialty
– Technology centred• Surgery / investigation / highly specialised
treatment
– Social support centred• Polyhandicap / familial dimension /
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Topics with disagreement (3)
Geographical reach of centres– European centres– National centres– Regional centres
VS
Level of designation / funding– EU– Member states– Regions
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Topics with disagreement (4)
Centres of reference– National centres– Regional centres– where patients may be referred
VS
Centres of excellence / of expertise– Characterised by communication– Production of guidelines– Shared expertise for patients
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Topics with disagreement (5)
From the concepts to the reality– Diffusion of the concepts– Publication of experiences– Follow-up of national initiatives– Definition of responsibilities
• European networks: – European Commission
• Centres at national or regional level– bilateral contracts between countries
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Conclusion
Read the RDTF reports
www.rdtf.org
Read the French National Plan for Rare Diseases and the document estblishing the policy around « centres of reference »
www.orpha.net
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Conclusions from the third report on Centres of expertise
MS having a policy agree as much as possible on an operational definition of what is a CE and on how to designate them
MS with established CE share their experience and their outcome measures
MS without CE either establish CE or contract with Existing CE
MS identifies their CE and provide them with necessary funding
EC promote the identification of CE and diffuse the information
ENCE Kick off Meeting - Frankfurt - 28 April 2009
II- European Reference Networks
ENCE Kick off Meeting - Frankfurt - 28 April 2009
ERN definition and scope
« Physical or virtual networking of knowledge and expertise »
Types of networks– Research networks
• Share data through systematic collection• Establish repositories of biological samples• Share expertise for research puroposes
– Public Health networks• Share clinical experience to sort out difficult cases• Produce guidelines based on shared clinical experience• Produce information for all stakeholders
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Conclusions from the third report on European Reference Networks
Questions: How can the ERN of highest quality be
selected ? How are selected members of ERN ? How to avoid limiting new initiatives ? How to ensure long-term funding and
competition ? Question of liability of virtual clinics
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Conclusions from the third report on European Reference Networks
Recommendations: Provide funding to pilot projects until their
evaluation Establish methods to assess the added-value
of ERN Encourage electronic tools Encourage production of guidelines to
protect patients
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Dissemination of Information on expert clinics and European networks
Orphanet services in 5 languages: English, French, German,
Italian, Spanish
ENCE Kick off Meeting - Frankfurt - 28 April 2009
ENCE Kick off Meeting - Frankfurt - 28 April 2009
ENCE Kick off Meeting - Frankfurt - 28 April 2009
ENCE Kick off Meeting - Frankfurt - 28 April 2009
Conclusion
Read the RDTF reports
www.rdtf.org
Read the French National Plan for Rare Diseases and the document establishing the policy around
« centres of reference »
www.orpha.net
ENCE Kick off Meeting - Frankfurt - 28 April 2009
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