Rare diseases and disorders / Projects

Second Programme of Community action in the Field of Health 2008-2013
European Organisation for Rare Diseases [EURORDIS_FY2012]
EURORDIS is a patient-driven alliance of Patient Organisations(POs) and individuals active in the field of rare diseases (RD). It represents the voice of an estimated 29 million citizens in the EU.

E...
EURORDIS is a patient-driven alliance of Patient Organisations(POs) and individuals active in the field of rare diseases (RD). It represents the voice of an estimated 29 million citizens in the EU.

EURORDIS mission is to build a strong pan-European community of POs and People Living With RD (PLWRD), to be their voice at the European level and to fight against the impact of RD on their lives.

To this end, EURORDIS undertakes activities on behalf of its members:

- Empowering RD patient groups
- Advocating RD as a public health priority
- Raising public awareness on RD
- Improving access to information, treatment, care and support for PLWRD
- Improving quality of life
- Encouraging good practices in relation to these issues
- Promoting scientific and clinical research on RD
- Promoting the development of treatments and drugs for people with RD

EURORDIS plays a pivotal role in the implementation of the EU strategy on RD (EC Communication and Council Recommendations), in the EU Committee of Experts on RD and in the elaboration of National Plans or Strategies on RD.
Start date: 01/01/2012 - End date: 01/01/2013

Call: Promote Health (Hp-2010)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
European Porphyria Network: providing better healthcare for patients and their families [APHP-EPNET_FY2012]
Our mission is to improve the lives of porphyria patients by improving the diagnosis and treatment of these rare conditions. EPNET has been funded by the EU Commission (DG SANCO, PHEA programme)since ...
Our mission is to improve the lives of porphyria patients by improving the diagnosis and treatment of these rare conditions. EPNET has been funded by the EU Commission (DG SANCO, PHEA programme)since 2007. The objective is to establish an effective network of specialist porphyria centres throughout the EU. Currently, EPNET consists of 28 EU specialist centres from 17 European countries that work together to develop an up-to-date approach to the management of patients and families with porphyria that conforms to uniform standards. We also have members from Australia, New Zealand, South Africa and the USA.
Evidence from the last years shows that this network and its activities have a positive effect on the quality of diagnosis, number of patients diagnosed and treatment choices (shown through the collection of data, enquiries to the drug database and external quality scheme). However, porphyria centres and/or clinical experts are still not present in all EU countries; therefore the care is not equal in all member states. An objective for 2012 will be to work with national plans, the laboratory network and the cross boundary directive to find a method to reach out to patients in countries currently outside EPNET e.g. Bulgaria, Greece, Portugal, Croatia and others.
EPNET focuses on: provision of information to patients (in their own languages) and healthcare professionals (HCPs); use of external quality assessment to develop quality standards for diagnosis and clinical advice; a web-based registry to collect data about the porphyrias to inform clinical practice and healthcare planning; dissemination of information on safety of drugs. Progress is communicated to partners through regular meetings and reports. Information on the porphyrias and drug selection is made available to patients, public health authorities and HCP`s at www.porphyria-europe.org; www.drugs-porphyria.org.
Start date: 01/01/2012 - End date: 01/01/2013

Call: Promote Health (Hp-2010)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
The European Wilson's Disease Network [AP-HP-WILS_FY2012]
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...
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).
Start date: 01/01/2012 - End date: 01/01/2013

Call: Promote Health (Hp-2010)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
UNEW_FY2013 [UNEW_FY2013]
TREAT-NMD is an international network for rare inherited neuromuscular disorders (NMDs). Its mission is to provide an infrastructure to increase collaboration between clinicians, scientists, and patie...
TREAT-NMD is an international network for rare inherited neuromuscular disorders (NMDs). Its mission is to provide an infrastructure to increase collaboration between clinicians, scientists, and patients, accelerate therapy development for these incurable conditions, improve patient care through publication and dissemination of best-practice consensus guidelines, and provide reliable information for patients and professionals. TREAT-NMD was established in 2007, and until the end of 2011, was funded through FP6 to bring together stakeholders representing all NMDs (>200 diseases). Although NMDs are individually rare, TREAT-NMD activities address ~200,000 patients in the EU, and ~3 million worldwide.
Since 2007 TREAT-NMD has increased the collaborating capacity of the field, raised the profile of NMDs within the wider rare disease (RD) field, become a model for RD collaboration, and increased the profile of European NMD research and healthcare globally. A public consultation in 2010 strongly endorsed the continuation and further development of TREAT-NMD`s core activities.
The TREAT-NMD Alliance has extended beyond the original network members to integrate new participants from around the world. The original number of ~350 scientists and clinicians that were integrated into the network through the 22 partners has now more than doubled and constitutes a critical mass for innovation and harmonisation in NMD care and research globally.
TREAT-NMD outputs:
-highly developed communication/dissemination infrastructure with extensive website and newsletter
-network of over 270 NMD care and trial sites
-global patient registries for several NMDs with >20,000 patients
-range of preclinical resources
-tools supporting translational & clinical research
TREAT-NMD vision:
-enhance European excellence in the NMD field
-increase visibility & recognition of NMDs in national health policy
-sustain core resources and added value of networking developing tools that cannot be maintained by individual research projects
-extend networking activities and resources to new experts worldwide
Start date: 01/01/2013 - End date: 01/01/2014

Call: Generate And Disseminate Health Information And Knowledge (Hi-2012)
Topic: Rare diseases and disorders
Second Programme of Community action in the Field of Health 2008-2013
EMSP_FY2013 [EMSP_FY2013]
Founded in 1989, EMSP is the umbrella organisation representing 38 national MS societies from 34 European countries. EMSP represents their interests at European level and works collectively with its m...
Founded in 1989, EMSP is the umbrella organisation representing 38 national MS societies from 34 European countries. EMSP represents their interests at European level and works collectively with its members to achieve our goals of high quality equitable treatment and support for Persons with Multiple Sclerosis (PwMS) throughout Europe.

EMSP vision is to move from high quality treatment and support for all PwMS in Europe to a world without MS!

EMSP`s mission is to ensure the development and the implementation of high quality standards of treatment and support for PwMS, their families and their carers in Europe, to allow them to lead their life independently and to recognise them as equal members of the society.

EMSP work priorities to achieve this mission are to:
- influence EU institutions and other decision-making bodies to improve their quality of life for PwMS;
- network with other European organisations which share of vision of truly patient centred approach;
- provide MS member organisations with information on relevant EU draft legislation and how we can influence it, and evidence - based projects that will contribute to their work;
- develop and promote recommendations for improved medical treatment and rehabilitation to be implemented in the EU member states and other countries;
- encourage coordinated research aiming to benefit of PwMS;
- initiate and implement cross-border projects to improve the living conditions of PwMS (e.g. in the field of therapies, employment, social security, infrastructure, etc.);
- act as an information platform by exchanging knowledge and best practices relevant to the work of the national MS member organisations;
- enhance a general awareness on MS by informing and educating external audiences;
- and to support national MS Societies, both recent and long-established, in Europe.
Start date: 01/01/2013 - End date: 01/01/2014

Call: Generate And Disseminate Health Information And Knowledge (Hi-2012)
Topic: Rare diseases and disorders