Rare diseases and disorders / Projects

First Programme of Community action in the field of public health (2003-2008)
Patients' Consensus on Preferred Policy Scenarii for Rare Diseases [POLKA]
The project aims at developing strategies and mechanisms for exchange of information among people affected by rare diseases (RD), as well as at supporting for European Networks of Reference for RD in ...
The project aims at developing strategies and mechanisms for exchange of information among people affected by rare diseases (RD), as well as at supporting for European Networks of Reference for RD in an effort to establish guidelines for best practice on treatment, and to share knowledge on RD, together with evaluation of performance. This objective will be pursued through the collection, analysis and presentation through EU conferences of relevant experiences and informed opinions on health policy themes from RD patients, with the support of health care professionals, at both national and EU levels.
Start date: 01/09/2008 - End date: 01/09/2011

Call: Health Information (Hi 2007)
Topic: Rare diseases and disorders
First Programme of Community action in the field of public health (2003-2008)
A reference network for Langerhans cell histiocytosis and associated syndrome in EU [EURO-HISTIO-NET 2008]
The EURO HISTIO NET 2008 project aims to set up a network for reference centres organising care and clinical research for Langerhans cell histiocytosis (LCH) and associated syndromes in EU Member Stat...
The EURO HISTIO NET 2008 project aims to set up a network for reference centres organising care and clinical research for Langerhans cell histiocytosis (LCH) and associated syndromes in EU Member States.
Start date: 01/09/2008 - End date: 01/09/2011

Call: Health Information (Hi 2007)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
Single Hub and Access point for paediatric Rheumatology in Europe [SHARE]
The objective of SHARE is to improve the quality of care of patients suffering from PRD (i.e. JIA, JDM, SLE, APS, vasculitis and scleroderma) by means of:
(I) Resolving the barriers between: (a) ne...
The objective of SHARE is to improve the quality of care of patients suffering from PRD (i.e. JIA, JDM, SLE, APS, vasculitis and scleroderma) by means of:
(I) Resolving the barriers between: (a) networks and projects, (b) PRD research teams, and (c) all actors involved in the itinerary from the conception of an idea or lead to the development of a new medical technology or drug and the treatment of patients;
(II) Improving a patients access to relevant data/information about his/her disease;
(III) Facilitating the exchange of ideas, results, data and best practices, and;
(IV) Facilitating the implementation of results in training programs of health care professionals.
Building on established networks (PReS/PRINTO), national registers and European projects, SHARE will have a considerable impact on the treatment and management of PRD by catalyzing progress in research, by harmonizing treatment, by improving the foothold of patients and by supporting regulatory and policy decisions.
Start date: 01/09/2012 - End date: 01/01/2016

Call: Promote Health (Hp-2010)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
European Surgical Registry for Rare Endocrine Tumours [EUROCRINE]
General objective:
The EUROCRINE project aims to decrease the morbidity and mortality of rare endocrine tumours, by means of a pan-European database. By recording and accessing data, best practice whi...
General objective:
The EUROCRINE project aims to decrease the morbidity and mortality of rare endocrine tumours, by means of a pan-European database. By recording and accessing data, best practice which, due to the extreme rarity of these tumours cannot be identified on a local or national level, is only possible to evaluate on a European level.
Dissemination of results will raise clinical standards and reduce differences in practice between member states, and thereby decrease morbidity and mortality. Comparison of practice and outcomes will be especially helpful for centres with a standard of care below that which is currently considered best practice.
Strategic relevance and contribution to the programme:
The proposed project satisfies the objective of supporting a European rare disease information network, by establishing the EUROCRINE registry of rare endocrine tumours with an incidence of 0.015 - 1.0/1 000 000 population. Information on diagnosis and outcome based on pooled data, is currently virtually non-existent. EUROCRINE will be a key means by which the medical profession, researchers. health policy makers and patients will benefit from an increase in knowledge related to the diagnosis, treatment and future clinical research in the field of rare endocrine tumours. EUROCRINE will produce theoretical and practical benefits from the collection of data on rare diseases in addition to the opportunity to disseminate results from a specific medical field with a low level evidence base. The analysis could also show whether some diseases could be best managed in the European context, i.e., a European Referral Centre, and identify objective variables for this. Such analysis could be of major interest for other non-related health care issues.
Methods and Means:
A Project Board with representation from all partners is to convene four times a year. The Board will take strategic decisions of relevance to the project and elaborate a plan for economic sustainability of the database.
A project coordinator will be employed. Monitoring of the project at the local level will be performed by partners` project leader. The project coordinator will travel regularly to partners. Education will be provided by the main partner for registry secretaries. Information material will be disseminated at partners` conferences, through membership lists and advertisement in the medical press. A dedicated web-site will be published for information and registration.
The design, content and process of implementation of European Registries, are of utmost importance. Therefore, the project leader will perform in-depth interviews with key staff of the European Registry of Quality Outcome for Cataract and refractory Surgery (EUREQUO)as well as dialogue with existing registries for rare diseases to draw on the experience of creating a European registry.
To review the variables to be captured by the database and to elaborate the design of the database, a workshop is planned for all stakeholders.
The database will be "stress tested" in three countries. Thereafter it is planned to successively roll-out the registry in Europe. After 18 months of full operation, evaluation of the standard of care for the tumour groups will be performed.
Expected Outcome:
The database is expected to be up and running with all partners by the end of the project. Analysis of data will yield information on diagnosis, treatment and outcome from a European perspective with comparative data on the national level. The outcomes will be discussed at a dedicated Conference for stakeholders; health professionals, patient organizations, health policy makers, researchers and published as a positional statement. EUROCRINE is expected to continue on a sound financial basis. Links with other cancer networks are foreseen to sustain the project.
Start date: 01/09/2013 - End date: 01/04/2017

Call: Promote Health (Hp-2012)
Topic: Rare diseases and disorders
First Programme of Community action in the field of public health (2003-2008)
European Haemophilia Safety Surveillance System [EUHASS]
The European Haemophilia Safety Surveillance (EUHASS) project aims to establish a pharmacovigilance program to monitor the safety of treatments for people with haemophilia. In addition it will develop...
The European Haemophilia Safety Surveillance (EUHASS) project aims to establish a pharmacovigilance program to monitor the safety of treatments for people with haemophilia. In addition it will develop and maintain a database of all the Haemophilia centres in Europe and establish a Rapid Alert System for immediate Europe-wide notification in case of unexpected or serious adverse events.
Start date: 01/10/2008 - End date: 01/10/2011

Call: Health Information (Hi 2007)
Topic: Rare diseases and disorders