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The European Wilson's Disease Network [AP-HP-WILS_FY2012] [20113203] - Operating Grant
Mission and vision of the operating grant holder

EuroWilsoN`s (EW) mission is to improve quality of care and access to multi-disciplinary expertise for EU patients with Wilson`s disease (WD). EW was founded in 2004 through the FP6 programme(EW (LSHM CT2004 503430), and is currently funded by the European Commission Directorate General for Health and Consumers operating grant 2010. It has achieved:

- A European multidisciplinary network for sharing expertise in patient management. A multidisciplinary approach is particularly necessary in WD which has variable clinical manifestations
- A network composed of all stakeholders concerned by WD: clinicians, research scientists, patients, patient organisations, regulatory authorities and industry
- A WD patient registry collecting longitudinal data
- External molecular quality network
- Contribution to the development of recent guidelines
- A structure for patient groups to network and share experience

EW`s goals include maintaining and enhancing this network; the French centre of expertise for WD serves as a network model. The network includes all stakeholders involved in patient care: medical, scientific para-medical, EU authorities and patients` associations. EW will:

- Promote exchange between participants
- Provide up-to-date disease information on its website (eurowilson.org) and Orphanet
- Develop and publish evidence based clinical guidelines
- Develop telemedicine within the network
- Enhance the registry to provide epidemiological data, outcome indicators, country comparisons and to facilitate collaborative research and public health projects
- Support patients and patient associations in the EU
- continue to serve as a research and knowledge centre for WD

Members of Eurowilson are actively participating in European and international activities with the aim to transfer knowledge from this network to other networks, patient regsitries and to national authorities and policy makers. We serve the European Committee of Experts in Rare Disease (EUCERD) and the International Rare Disease Research Consortium (IRDiRC).

Strategic objectives and specific activities

The general objective of EW is to develop the EU WD multi-disciplinary network and its registry and ultimately to provide EU patients and their families with up-to-date information, optimised management and equal access to expert advice, diagnosis and care.

Objective 1: improve information and services to patients
- Development and dissemination of online up-to-date information and educational documents for patients and families in their own language
- Help set up EU patient meetings, on average once every year with the aim to transfer experience between different national associations. Torben Gronnebaek and Eurordis will have a strong role in this activity. The first meeting will be held in June 2011.


Objective 2: Increase knowledge and exchange of experience of WD
- Continue information sharing between different WD expertise. Since its beginning, EW has successfully produced a neurology scoring system, a hepatic scoring system, a laboratory external quality assessment programme, several publications and information for children, parents and adult patients in patients own language
- Continue the annual network meetings
- Continue to enter newly diagnosed patients in countries that have resources to do so and follow-up into the registry, analysis of indicators to show evolution of care and impact of rare disease plans. The feasibility of this has been determined by the success of the French contribution to the EW database which currently has a cohort of 395 patients with an average follow-up of 15 years.
- Continue a trimestrial "bibliographic journal watch" containing summaries of recent progress and research in WD
- Continue to develop European guidelines on the management of WD
- Work with the MS national plans or strategies for rare disease with the objective to work with one database on WD which provides policy makers with information necessary in resource decisions. Long-term sustainability is one of our aims within these national plans or through the cross-boundary directive (Article 12 lays out the structure for European Reference Networks)

Objective 3: Improve access and quality of biochemical and genetic testing
- Encourage new labs to participate to the EMQN mutation quality assay; we currently have 33 partipants (12 EU countries and 5 non EU). About 15 of our members send samples to non regsitered labs.
- Evaluate current status of the availability of WD diagnostic tests in Europe

Expected outcomes

Objective 1: improve information and services to patients; The lead of these activities is T.Gronnebaek, patient rep & director of Eurordis
- In 2011 a survey to patients, in their own language, will be performed in France, UK, Germany and Denmark in order to determine the needs and expectations of patients. The 2012 strategy will build on the results of this survey. We will also extend the survey to other countries.
- The 2011 several members of WD groups in Europe will meet to exchange experience; EW will provide the platform to extend beyond the current groups
Objective 2: continue to collect data in the registry and increase knowledge
- The EW database will be maintained and enhanced by inclusion of new cases and collection of follow-up data
- The interface between the EU database and the French CNR has been successful; its applicability will be continue to be explored in other countries i.e. developing multi-disciplinary teams working with the database
- Registry data will be produced on: treatments being used and different clinical scenarios; doses used; changes of Rx and reasons for change; outcomes will provide data on drug efficacy, side effects and compliance
- Quality issues to be examined are: completeness of family screening; indicators of quality diagnosis (symptom-diagnosis interval), treatment (adherence to guidelines) and outcome.
Objective 3: improve knowledge sharing in WD and high-quality care
- A yearly network meeting to improve awareness, knowledge and quality of care
- Recruitment of clinicians from under-represented countries, particularly focusing on countries with or developing a RD plan
- Review of difficult cases eg by transmission of MRI images, liver biopsy slides, or neurological video and development of telemedicine
- Develop and disseminate educational documents adapted to the needs of psychologists, social workers, physiotherapist, speech therapists and nurses.
- In 2011 Eurowilson members developed guidelines;these will be disseminated, gaps will be identified and measures taken to complete missing inforamtion.
- Continue the Journal Watch which is a review of recent publications relevant to WD
Objective 4: improve biochemical and molecular testing for WD
- encourage participation to the EMQN scheme
- suggest additional quality schemes for the network to consider.

EB Members lead the different tasks. A registry manager will validate new cases in the registry, give feed-back, analyse data and produce reports. The programme manager based in Lariboisiere will have responsibility for day-to-day running in collaboration with the CNR Wilson for writing the reports
Expected outcomes: Knowledge will be improved concerning current care protocols, services provided and outcomes. Recommendations will be given to optimise patient care. Patients and HCP`s will get access to up-to-date information on WD in their own language. Qol of patients and families should be improved as they have more efficient diagnosis, care and information.
Target groups: Experts will be able to exchange information, have access to a registry for patient follow-up, patients will benefit from access to improved care, HCP`s will have access to information and guidelines and could share their experiences. Health authorities will be aware of the availability of the access to diagnostic test and to expert clinical care in Europe.
The registry has ethical approval. Network members are involved with their national patient organisations. EW will apply with the Eurordis guidelines for patient collaboration with ERN`s

Details
Start date: 01/01/2012
End date: 01/01/2013
Duration: 12 month(s)
Current status: Finalised
Programme title: Second Programme of Community action in the Field of Health 2008-2013
EC Contribution: € 159 150,00