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Documents necessary for the design and elaboration of a national plan/ strategy for rare diseases
The European Recommendations and policies listed have been adopted by consensus by EU Member States and stakeholders (academia, industry, patients).
The EURORDIS position papers and recommendations have been adopted following an extensive consultation with member rare disease patient organisations, and sometimes with additional expert stakeholders.
These reference documents are organised per strategic area, constituting the national plan/strategy:
1. Governance
- Core indicators to monitor rare disease national plans and strategies (2013)
- European Recommendations and Indicators for Governance (EU Council_EUCERD_ EUROPLAN)
2. Definition, codification, inventorying of rare diseases and patient registries
- European Commission Group of RD Experts Recommendations on RDs Codification
- Toolset for implementation of ORPHAcodes into Health Information Systems
- European Commission Group of RD Experts Recommendations on RD patient registration and data collection
- EURORDIS-NORD-CORD Joint Declaration of 10 Key Principles for Rare Disease Patient Registries
- European Recommendations and Indicators for codification, inventorying of Rare Diseases and registries (EU Council EUCERD EUROPLAN)
3. Research (including development and access to orphan drugs, innovative treatments)
- EURORDIS Position on Rare Disease Research
- Rare Barometer Survey Rare disease patients’ participation in research
- European Commission Group of RD Experts Cross border genetic testing of rare diseases in the European Union
- International Rare Diseases Consortium Recommendations Clinical Trials in Small Populations
- Patients' full and equitable access to rare disease therapies in Europe
- Early access to medicines in Europe: Compassionate use to become a reality
- European Recommendations and Indicators for Research Orphan Drugs (EU Council EUCERD EUROPLAN)
4. Centres of Expertise and European Reference Networks (ERNs)
- European Commission Group of RD Experts Quality Criteria for Centres of Expertise for Rare Diseases
- EU Directive on 9 March 2011 on the application of patients’ rights in cross-border healthcare (article 12 on ERNs)
- European Commission Group of RD Experts Recommendations on European Reference Networks on Rare Diseases
- European Commission Group of RD Experts Recommendations Addendum ERN Grouping and patients’ involvement
- About the 24 European Reference Networks for Rare Diseases and Rare Cancers
- EURORDIS Recommendations on the Integration of European Reference Networks into National Health Systems
- European Recommendations and Indicators for Centres of Expertise and ERNs (EU Council EUCERD EUROPLAN)
5. Social services and programmes adapted to rare diseases
- Position Paper: Achieving Holistic Person-CentredCare to Leave No One Behind - A contribution to improve the everyday livesof people living with a rare disease and their families (May 2019)
- European Commission Group of RD Experts Support the Incorporation of Rare Diseases Into Social Services and Policies
- Juggling care and daily life: The balancing act of the rare disease community
- Results of the INNOVCare Project (Innovative Patient-Centred Approach for Social Care Provision to Complex Conditions)
- European Recommendations and Indicators for social services and programmes adapted to rare diseases (EU Council EUCERD EUROPLAN)
6. Empowerment of patient organisations
- Policy Brief on Patient Empowerment
- European Recommendations and Indicators for Patient Empowerment (EU Council EUCERD EUROPLAN)
7. Sustainability
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The voice of rare disease patients in Europe
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative