Framing the European Rare Diseases field through a structured movement of patient organisations

Abstract

EURORDIS-Rare Diseases Europe is an alliance of patient organisations, working across countries, empowering patients with trainings, and generating evidence through surveys. It advocates for the needs of people living with rare diseases in Europe, influencing policy, legislation, research, healthcare services, social inclusion, medicines development and access. Since its creation, EURORDIS has maintained a global perspective. By nature of the rarity of these conditions, knowledge and expertise are scattered, so collaboration on a global level is required to have an understanding of disease progression. Thus, every rare disease patient group must be equipped to collaborate beyond its own borders. EURORDIS impacts global activities with a focus on raising awareness; empowering a global alliance; supporting international, national and regional initiatives; and recognising rare diseases within the international community while fostering collaboration among stakeholders. For these reasons, the organisation’s impact extends beyond Europe, as demonstrated by its international partnerships. This perspective paper explores EURORDIS’ achievements over two decades, examining its role in shaping policies and regulations, to demonstrate the vital importance of global collaboration in the rare disease field. Looking ahead, EURORDIS’ Strategic Goals, to be achieved by 2030, align with the United Nations Sustainable Development Goals Agenda 2030 and advocate for a European Action Plan for Rare Diseases. This illustrates EURORDIS’ continued commitment to transforming policies into tangible outcomes. With its ongoing dedication, collaborative efforts, and enduring impact on the rare disease community, EURORDIS remains a driving force for positive change in the lives of people living with rare diseases.

Keywords

Rare diseases / patient organisations / ecosystem / policy / framework / empowerment / advocacy / partnership

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