Table Of Content. Policy Action and Information for Rare Diseases in Europe... 2 Summary... 3 Coordinator, Leader contact and partners...

Transcription

1 Table Of Content Policy Action and Information for Rare Diseases in Europe... 2 Summary... 3 Coordinator, Leader contact and partners... 6 Kontaktudvalget for Mindre Sygdoms-og handicapforeninger (KMS)... 6 Ågrenska Atkiebolag... 6 ALAN - Association luxembourgeoise d'aide pour les personnes atteintes de maladies neuromusculaires Asbl... 6 Federación Española de Enfermedades Raras... 6 University of Ulster... 6 ALLIANCE MALADIES RARES... 6 Outputs... 8 Executive summary of the Interim report... 8 Interim report... 8 Original Full Information (Dec 2008)... 8 Page 1/8

2 Policy Action and Information for Rare Diseases in Europe JA GPSD [705038] START DATE: 01/11/2004 END DATE: 01/12/2005 DURATION: 13 month(s) CURRENT STATUS: Finalised PROGRAMME TITLE: First Programme of Community action in the field of public health ( ) PROGRAMME PRIORITY: - CALL: Health Information (Hi 2004) TOPIC: Improving information and knowledge for the development of public health EC CONTRIBUTION: EUR KEYWORDS: PORTFOLIO: Rare diseases Page 2/8

3 SUMMARY General objectives - To share rare disease information and knowledge in an Enlarged Europe in order to reduce inequalities and provide the basis on which to develop an integrated EU approach for rare diseases - To demonstrate the importance of EU actions on rare diseases and review progress made so far through a European Rare Disease Conference. This conference will therefore serve as a spearhead for the involvement of all interested parties and a broad dissemination of information and awareness on rare diseases. Strategic relevance and contribution to the public health programme Health information strand. Improving information and knowledge for the development of public health. Operating the health information and knowledge system. The aim of this project is to share rare disease information and knowledge in an Enlarged Europe in order to reduce inequalities and provide the basis on which to develop an integrated EU approach for rare diseases. It consists in a platform to review the difficulties faced by the rare disease community and the actions undertaken by all parties involved. Methods and means The strategy is to bring together, in a common effort, patient groups, researchers, health professionals, public policy makers and other interested parties, across different diseases and across EU Member States, to share information and issues, exchange views and debate on results achieved to raise awareness, build consensus and facilitate future actions. The specific mechanisms used are a European Conference during the EU presidency by Luxembourg, followed by the dissemination of the proceedings. For the conference programme, some representatives of the associate beneficiaries, health professionals and members of the Task Force on Rare Diseases participated to the programme committee of the conference in charge with the selection of the abstracts for poster sessions. Other members of the Task Force who are not in the programme committee to be informed throughout the project. Leaders of projects on rare diseases supported by the EC to be invited to submit an abstract (both DG Research and DG Health programmes). More than 100 research projects on rare diseases are completed or being executed (Cordis), while 24 projects supported by DG Health (from 2000 to 2002) are completed. Preparatory documents that describe rare diseases in Europe (epidemiological fact sheet and patients` testimonies) to be updated and presented to all participants. A document summarising available epidemiological information on rare diseases published in scientific literature, and a process to longitudinally collect epidemiological information from selected sentinel rare diseases to be developed under the supervision of Orphanet, with the participation of the Alliance Maladies Rares, the Institut National Page 3/8

4 de Veille Sanitaire (INVS), and probably the Institut de Recherches sur les Maladies Rares (GIS Maladies Rares). The same materials to serve for the media and for the conference awareness campaign (press package). Preparatory materials, presentations, articles, written reports from the sessions of the Conference to be included in the conference proceedings to be largely disseminated in the next months that follow the conference. In parallel to the organisation of the conference, and fully benefiting from its dynamic, interested stakeholders to prepare their contribution through various methods, e.g. an EMEA workshop with patients` organisations and health care professionals in March 2005, e.g. the EMEA/COMP report to the European Commission on the implementation of the orphan drug regulation five years after its adoption (report to be validated during the Luxembourg presidency during an informal EMEA/COMP meeting 4-5 May 2005), e.g. a DG Research workshop with the participation of leaders of projects on rare diseases, e.g. an analysis of national policies on rare diseases by Orphanet and its partners in the frame of the Task Force on Rare Diseases. Expected outcomes period Work package n 1 - Project Management and Coordination This work package is pivotal to all our objectives as its aim is to establish the management structure which will be the steering engine of the whole project. It will also include the day-to-day management and the quality supervision of the project as well as reporting to the European Commission. The main expected result is to smooth coordination of the different steps of the project so they are realised on time within the budget limits and according to the predefined objectives. Another expected result is also the coordination with partners so they are properly involved and regularly updated on the implementation of the project. Work package n 2 - European Rare Disease Conference in Luxembourg The workpackage is key to demonstrate the importance of EU action on rare diseases and review the progress made so far. It will share information and knowledge in an Enlarged Europe. It will encourage exchange between leaders of EU funded projects. It will also launch a process to identify best practice and to propose recommendations. In June 2005, under a Luxembourg EU Presidency, the European Rare Disease Conference will build on current achievements and enhance rare disease awareness. Most participants will only know about few rare disorders, while all rare diseases? 28 to 35 million patients in EU affected by RD - are sharing very similar problems: access to information, access to diagnosis, access to care and treatment, access to research programmes, clinical trials and drugs. The Conference will foster the rare disease EU community of policy makers, scientists and patients` organisations. Work package n 3 - Study on Epidemiological Surveillance of Rare Diseases RDTF scientific secretariat Expected results: - Provision of scientific support to EC services Provision of scientific support to EC services - Wider dissemination of available information to all stakeholders in order to encourage continuity of work and transnational cooperation - Wider dissemination of the information produced by funded projects (DG Sanco and Research, EMEA) - Support prioritisation and harmonisation of data collection in order to: - better monitor morbidity and mortality better monitor morbidity and mortality - Produce Produce transnational transnational comparisons comparisons - Establish a common EU framework Establish a common EU framework Methods: - Ad Hoc meetings with EC services + national experts + international organisations - Conference calls with RDTF members and meetings - Progress reports - Electronic newsletter - Web site for site Page 4/8

5 for the Community - Web board for the TF members board and EC services Work package n 4 - Dissemination and Outreach This work package is crucial to the successful transfer of best practices to all Member States as well as ensuring consistency of work and ongoing cooperation at EU level. Specifically a wide dissemination of the information gathered during the project is key to actively involving new Members States in mechanisms of exchange of information and reducing knowledge gaps and inequalities concerning rare diseases. Page 5/8

6 COORDINATOR, LEADER CONTACT AND PARTNERS COORDINATOR EURORDIS - European Organisation for Rare Diseases - Plateforme Maladies Rares (EURORDIS - European Organisation for Rare Diseases - Plateforme Maladies Rares) 102 rue Didot Paris France PARTNERS Kontaktudvalget for Mindre Sygdoms-og handicapforeninger (KMS) Country: Denmark Ågrenska Atkiebolag Country: Sweden ALAN - Association luxembourgeoise d'aide pour les personnes atteintes de maladies neuromusculaires Asbl Country: Luxembourg Federación Española de Enfermedades Raras Page 6/8

7 Country: Spain University of Ulster Country: United Kingdom ALLIANCE MALADIES RARES Country: France Page 7/8

8 Powered by TCPDF ( OUTPUTS Executive summary of the Interim report EURORDIS - European Organisation for Rare Diseases - Plateforme Maladies Rares Policy Action and Information for Rare Diseases in Europe (PARACELSUS) Published on: 01/11/ Interim report EURORDIS - European Organisation for Rare Diseases - Plateforme Maladies Rares Policy Action and Information for Rare Diseases in Europe (PARACELSUS) Published on: 01/11/ Original Full Information (Dec 2008) EURORDIS - European Organisation for Rare Diseases - Plateforme Maladies Rares Policy Action and Information for Rare Diseases in Europe (PARACELSUS) Published on: 01/11/ Page 8/8