EUROPEAN ORGANISATION FOR RARE DISEASES
Budget
€400 — €0
EP Access
3
accredited persons
Staff
8
4 FTE
EU Grants
€906,168
Mission & Goals
EURORDIS-Rare Diseases Europe is a patient-driven alliance of over 1130 patient organisations (POs) active in the field of rare diseases (RD) from 77 countries. It represents the voice of an estimated 30 million citizens in the EU. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies and patient services. EURORDIS vision is to enable better lives and cures for people living with a rare disease and its mission is to work across borders and diseases to improve the lives of people living with a rare disease. To this end, EURORDIS-Rare Diseases Europe undertakes activities on behalf of its members: • Raising public awareness on RD • Empowering PLWRD and RD patient groups and strengthening the capacity of rare disease patients’ representatives • Advocating for RD as a public health priority and for national and EU-level
EU Legislative Interests
All areas of the Commission Communication on Rare Diseases: Europe's challenges and Council Recommendations on an action in the field of rare diseases (2009/C 151/02) Implementation of the new Directive 2023/0132 on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC and Directive 2009/35/EC; and the new Regulation 2023/0131 laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006 Regulation on standards of quality and safety for substances of human origin intended for human application and repealing Directives 2002/98/EC and 2004/23/EC Implementation of the European Health Data Space Regulation Implementation of the HTA Regulation (Reg. EC n° 2022/2282) Implementation of the Cross-border Healthcare Directive and European Reference Networks Proposal for a Regulation on establishing a framework of measures for strengthening Union’s biotechnology and biomanufacturing sectors particularly in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act) Proposal for a Regulation amending Regulations (EU) 2017/745 and (EU) 2017/746 as regards simplifying and reducing the burden of the rules on medical devices and in vitro diagnostic medical devices, and amending Regulation (EU) 2022/123 as regards the support of the European Medicines Agency for the expert panels on medical devices and Regulation (EU) 2024/1689 as regards the list of Union harmonisation legislation referred to in its Annex I Europe’s Beating Cancer Plan EU Strategy on Mental Health European Pillar of Social Rights and its Action Plan EU Disability Strategy Proposed next long-term EU Budget for 2028-2024 (Multiannual Financial Framework, MFF), including the proposal for a Regulation establishing a European Competitiveness Fund The EU4Health Programme in general, with specific focus on issues relevant to the EU strategy on Rare Diseases. The EU Research Programme 2021-2027: “Horizon Europe” in general, with particular focus on projects and EU collaboration relevant to the rare diseases field. ESF+ and the EASI Programme by DG Employment and Social Affairs, with particular focus on social care and innovation.
Communication Activities
All areas for implementation of the above-mentioned initiatives, policies and legislation represent areas of interest for EURORDIS-Rare Diseases Europe and its members, and therefore EURORDIS-Rare Diseases Europe is potentially called to act upon all these relevant files together with other stakeholders involved in the rare diseases fields, such as EU decision-makers, national authorities, private sector, healthcare professionals, HTA bodies, payers and regulators at both EU and national levels. EURORDIS set up and co-manages the Stakeholder Network on Rare Diseases hosted by the EU HPP (Health Policy Platform). EURORDIS-Rare Diseases Europe is the main organiser of the biannual European Conference on Rare Diseases and Orphan Medicinal Products. EURORDIS-Rare Diseases runs the secretariat of the EP Intergroup on Cancers and Rare Diseases with two other organisations (ECO and SIOP-E)This involves co-establishing the work programme, implementing the activities laid out therein, including organising public hearings and EP events; communication via th website; and other initiatives. Also, EP events are organised by EURORDIS, such as an event on the "Impact of Rare Diseases: More than you can imagine" (March 2025, co-hosted by SANT Chair and Vice-Chair Adam Jarubas MEP and Stine Bosse MEP), and the “Public Hearing on the Pharmaceutical Legislation in Europe” (July 2025, co-chaired by MEP Tomislav Sokol (EPP, HR), MEP Vlad Voiculescu (RE, RO), MEP Tilly Metz (Greens/EFA, LU), MEP Stine Bosse (RE, DK), and MEP Alessandra Moretti (S&D, IT). EURORDIS leads the global awareness raising campaign on Rare Disease Day (https://www.rarediseaseday.org/), celebrated every year on 29 (28) February with hundreds of events across over 100 countries. EURORDIS publishes position papers and other policy documents based on extensive consultation with members and, where relevant, other stakeholders. A list can be found here: https://www.eurordis.org/resources/ EURORDIS also leads the “Rare Barometer Voices” initiative, a survey programme based on a database of thousands of people living with a rare disease. The results of its studies are published here: https://www.eurordis.org/voices EURORDIS is part of the “EU4Health Civil Society Alliance” campaign (https://eu4health.eu/) gathering a number of European health organisations to promote a stronger role for the EU in the field of health promotion.
Interests Represented
Promotes their own interests or the collective interests of their members
Member Of
External networks: - EUROPEAN MEDICINES AGENCY (COMP, PDCO, EnprEMA, PRAC, PCWP, SAWP, CAT, EU Clinical Trial Information system, Topic Group on Patient Experience Data, Advisory Group on Real World Evidence, Network Data Steering Group, ACT-EU) - EUROPEAN COMMISSION: EU Disability Platform, EU Health Policy Platform - EUROPEAN PARLIAMENT: Intergroup on Cancers and Rare Diseases - EU Cooperation on Health Technology Assessment (HTA): HTA Stakeholder Network - Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) - European Reference Networks (ERNs) via ePAGs (European Patient Action Groups) Member of European Not-for-Profit Organisations & Initiatives: - EUROPEAN PATIENTS’ FORUM (EPF) - EU4Health Civil Society Alliance - EDF: European Disability Forum - EFGCP: European Forum for Good Clinical Practice - SOCIAL PLATFORM – European Platform of European Social NGOs - European Network of Rare Diseases Helplines (ENRDHL) - Mental Health Europe - WECAN: informal network of leaders of cancer patient umbrella organizations active in Europe - FRIENDS OF EUROPE - Get-Real Institute - Rare Disease Platform in Paris - 4th French Rare Disease National Plan Steering Committee - ECO PAC: European Cancer Organisation Patient Advisory Committee - Italian Rare Diseases Plan Steering Committee - RareResourceNet – European Network of Resource Centres (Board member) - Zebra Diagnosis Coalition - TRANSFORM Alliance Partnering with European Not-for-Profit Organisations & Initiatives: - EFPIA: European Federation of Pharmaceutical Industries and Associations - EUPATI - EUROPABIO Patients Advisory Group - European Children's Hospitals Organisation (ECHO) - European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) - European Expert Group on Orphan Drug Incentives (OD Expert Group) - RARE DISEASE MOONSHOT – scaling up public private partnerships to accelerate research in rare diseases - TEDDY Network International Institutions, Not-for-Profit Organisations & Initiatives: - NGO Committee for Rare Diseases - IRDiRC – International Rare Diseases Research Consortium - Rare Diseases International (RDI) - ORPHANET - PFMD - Patient Focused Medicines Development Initiative - UDNI: Undiagnosed Diseases Network International (UDNI) - WHO – Europe – Novel Medicines Platform - iCONS: The International Consortium on Newborn Sequencing European networks (mostly EU co-funded projects): - BBMRI Stakeholders Forum - DeCODe – Medical devices - Supporting the development of paediatric and orphan devices - ERDERA – European Rare Diseases Research Alliance and Partnership - EuroCAB programme - FACILITATE – Framework for Clinical Trial Participants’ Data Reutilization for a Fully Transparent and Ethical Ecosystem - HealthData@EU Pilot project – External Advisory Board - JARDIN – Joint Action on ERN integration into National Healthcare Systems - JOIN4ATMP – Map, join and drive European activities for Advanced Therapy Medicinal Product Development and implementation for the benefits of patients and society - MoreEuropa – More Effectively Using Registries to support Patient-centered Regulatory and HTA decision-making - PERIFORMANCE – Public Engagement in Research Infrastructures for Mission Cancer: Managing Complexity of Emerging Technologies - RealiseD – Comprehensive methodological and operational approach to clinical trials in ultra-rare diseases - REMEDI4ALL – The European Platform for Medicines Repurposing - Screen4Care – Shortening the path to RD diagno Partnership with Learned Societies: - European Hospital & Healthcare Federation (HOPE) - European Society of Human Genetics (ESHG) - European Connected Health Alliance – ECHAlliance - European Alliance for Personalised Medicine - Rare Cancers Working Group of the European Society for Medical Oncology (ESMO RCWG)
Organisation Members
https://download2.eurordis.org/membership/Eurordis_Members_Jan_2026.pdf
Commissioner Meetings
No recorded meetings with EU commissioners.