The European Reference Network for rare Hereditary Metabolic Diseases (MetabERN) aims to facilitate access to the best available care and address the needs across European borders of all patients affected by rare inherited metabolic diseases (IMDs) and their families. It is unprecedented that a pan-EU network covers expertise encompassing all IMDs (over 700) regardless of their incidence and prevalence, severity, availability of treatment and characteristics of patient population. MetabERN is driven by the principle of patient-centeredness for the provision of its services aiming at improving the quality of life of patients and families. In this spirit, family associations (FAs) play a crucial role into the planning and decision-making system related to patient care and management as well as policy activities impacting on the EU Rare Disease Policy Agenda. World-known experts will work hand-in-hand with FAs to develop guidelines for accurate diagnosis and optimal management of IMDs in order to strengthen referral systems across the border. MetabERN will facilitate access to treatment by channeling expertise through virtual and web-based platforms to minimize patient mobility. MetabERN will serve as a hub of knowledge and information by connecting the most relevant scientific associations and EU established networks in this area. A structured collaboration is activated with other European Reference Networks with overlapping disease areas to ensure that patients receive the best available care. Knowledge of IMDs will increase among target groups, mainly Healthcare Professionals, Patients and Researchers, thanks to the tailored training activities that will be developed after a thorough check of their needs. Early uptake of medical advances will be bolstered by developing recommendations to overcome the barriers to translational research. Our focus on safety and cost-effective treatments will drive the future IMD research agenda for the next 10 years.
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The European Organisation for Rare Diseases – EURORDIS – represents the voice of an estimated 30 million people living with a rare disease in the EU, and their families. The proposal submitted for Operating Grant FPA 2015-2017 is structured around four main objectives of utmost importance for the European rare disease patients’ community:
1. To consolidate the Rare Disease patients’ community and strengthen the RDs patients’ voice;
2. To actively engage rare disease patients’ representatives into the effective implementation and monitoring of relevant EU legislation / strategies and support their integration at national level;
3. To build capacities and empower rare disease patients advocates, members and volunteers;
4. To sustain human, financial and organisational resources.

EURORDIS achieves these objectives through the following activities: collecting data as well as gathering, disseminating and sharing information; support capacity-building to empower patients’ advocates; providing patients, families, carers, decision-makers and other stakeholders with relevant expertise to inform policy-making; generating EU added-value and support Member States efforts; promoting translational research towards therapeutic intervention for patients; serving as a relay of two-ways information flow between policy makers and rare disease patient groups; promoting access to high quality and safe healthcare services for rare disease patients throughout the EU.

Through these activities, EURORDIS will contribute to the implementation of the priorities of the Third Public Health Programme which are to complement, add value and support national policies and strategies implemented by Member States to improve health, reduce health inequalities, support health capacity-building, generate and disseminate health information, contribute to innovative, efficient and sustainable health systems, support cross border healthcare in order to facilitate access to better and safer healthcare.
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The Association of European Cancer Leagues (ECL) provides a unique platform in Europe as the only organisation with the mission of uniting, at the European level, national and regional cancer leagues to achieve a cancer-free Europe. Active since 1980, ECL currently has 26 members from 23 countries in the European Region, covering 19 EU member states.
The objectives of this proposal are to:
- inform policy to ensure cancer control remains high on the political agenda;
- promote the European Code Against Cancer (ECAC) as the essential tool for cancer prevention in Europe;
- support leagues in the implementation of the Council Recommendation on Cancer Screening;
- unite leagues to advocate for equal access to treatment, support, and rehabilitation for cancer patients;
- advocate for the European Guide for quality improvement in comprehensive cancer control to be mainstreamed in national cancer control plans.
These objectives will be achieved through ECL's role as the secretariat of the MEPs against Cancer group; continuing our extensive work in tobacco control, including organising the triannual European Conference on Tobacco or Health (ECToH); developing training workshops and study exchanges for leagues; networking leagues in the ECL Patient Support Working Group, and Task Force on Access to Medicines; facilitating multi-stakeholder policy dialogues at national and European levels; and supporting targeted dissemination of ECAC to young people, including making use of the EU Solidarity Corps.
The activities of this proposal are particularity relevant to the work programme of the Third EU Health Programme as they directly address the core priorities of cancer, health promotion and prevention of chronic diseases, access to healthcare, and patient empowerment. These actions deliver high EU added-value by disseminating key outcomes from the EU level at the national level, and enabling the efficient input from cancer leagues to the EU policy dialogue.
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