Rare diseases and disorders / Projects

Second Programme of Community action in the Field of Health 2008-2013
Dissemination and Implementation of the Standards of Care for Duchenne Muscular Dystrophy in Europe (including Eastern countries) [CARE-NMD]
CARE-NMD (3 years; M€1.6; 6513 person/days) aims to optimize the treatment of Duchenne muscular dystrophy (DMD)in participating countries and by that to improve the quality of life for patients.
Spe...
CARE-NMD (3 years; M€1.6; 6513 person/days) aims to optimize the treatment of Duchenne muscular dystrophy (DMD)in participating countries and by that to improve the quality of life for patients.
Specific objectives are:
-> Identify the status of DMD treatment approaches in targeted countries by (online)- questionnaires by month 24, response rate will be the measure.
-> According to the results of questionnaires obtained, training sessions will be organised at national levels with healthcare providers to support compliance of healthcare practices with the recommended and established SoC over the full 36month. Measured by number of participants and number of workshops organised in each targeted country.
-> Popularize the CARE-NMD project, measured by CARE-NMD Website visits and registration rate in national patient registries and care and trial Site registry
-> Increase awareness and knowledge about treatment recommendations, measured by amount of input online discussion forum and amount of national multidisciplinary training workshops.



Start date: 01/10/2009 - End date: 01/10/2012

Call: Promote Health (Hp-2009)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
4th Eastern European Conference for Rare Diseases and Orphan Drugs "TOGETHER FOR INTEGRATIVE APPROACH TO RARE DISEASES" [4th EEC RDOD]
The general objective of the conference is to present rare diseases as a common issue of
all policies at national/EU level requiring integrated approach by all stakeholders.
Specific objectives:
1....
The general objective of the conference is to present rare diseases as a common issue of
all policies at national/EU level requiring integrated approach by all stakeholders.
Specific objectives:
1. To invite participants from new member states (MS) and other Eastern European
countries (EEC) working in different areas of medicine, industry, finance, social policy
etc., concerning rare diseases;
2. To invite speakers sharing best practises on integrative approach to rare diseases;
3. To increase public awareness on rare diseases in new MS and EEC and add value to
the public health knowledge.
The theme of the 4th EEC RDOD correlates with the second priority area of the Health
programme 2008-2013.
Rare diseases are a common issue of all policies at national/EO level and cover variety of
aspects: science, medicine, industry, finance, health care policy, social policy. Different
groups of people are involved and good coordination and cooperation between those
groups are the guarantee for successful practice in the area of rare diseases which is a
challenge of the contemporary United Europe.
Rare diseases are complex and complicated problem requmng timing information,
relevant quality of treatment, adequate social policy. An integrative approach toward rare
diseases will increase the quality of life of people affected by rare disease, will prolong
the healthy life years across their life cycle.
The 4th EEC RDOD is a continued action in the field of rare diseases aimed to improve
knowledge, facilitate the access of the EEC to information on these diseases and add
value to the public health knowledge. The conference will become an Eastern European
unified stage where all stakeholders involved in the area of rare disease will meet and
manifest their good will to integrative approach toward the objective of rare diseases.

Start date: 13/06/2009 - End date: 13/01/2010

Call: Promote Health (Hp-2008)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
European network and registry for homocysinurias and methylation defects [E-HOD]
The overall aim of the network and registry for homocystinurias and methylation defects (E-HOD) is to promote health for children, adolescents and adults affected with these rare and severe diseases....
The overall aim of the network and registry for homocystinurias and methylation defects (E-HOD) is to promote health for children, adolescents and adults affected with these rare and severe diseases. It is our aim to reduce variation between countries and allow patients, wherever they live, to access the necessary expertise and services. E-HOD has three specific objecives:
1) improving knowledge on homocystinurias (HCU) and methylation defects (MD) through the collection of clinical data into a registry,
2) developing diagnosis and clinical care recommendations,
3) evaluating the newborn screening (NBS) programme with recommendations.


Start date: 15/02/2013 - End date: 15/05/2016

Call: Promote Health (Hp-2012)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
Building Consensus and Synergies for the EU Registration of Rare Disease Patients [EPIRARE]
The general objective of this initiative is to build consensus and synergies to address regulatory, ethical and technical issues associated with the registration of RD patients and to elaborate possi...
The general objective of this initiative is to build consensus and synergies to address regulatory, ethical and technical issues associated with the registration of RD patients and to elaborate possible policy scenarios. Specific attention will be given to the scenario of the creation of a EU platform for the collection of data on RD patients and their communication among qualified users, based on a feasibility study. To this aim, the project will define the options for the preparation of a legal basis, the possible scopes to achieve most effective synergies, the corresponding governance framework and possible options for sustainability. The feasibility of registration of a minimum data set common to all rare diseases, designed to inform policy-making, the conditions to admit research-driven disease or treatment-specific modules and the ways to ensure a sustainable data flow will be assessed.
Start date: 16/04/2011 - End date: 16/04/2014

Call: Promote Health (Hp-2010)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
National policies and plans for rare diseases in Europe : shared experiences and recommandations [national rare diseases plans ]
The overall objective of the Conference is to promote the concept, rationale, benefits, scope of National Plans on Rare Diseases in -EU member countries.
Start date: 18/11/2008 - End date: 18/05/2009

Call: Promote Health (Hp-2008)
Topic: Rare diseases and disorders