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Cavaller-Bellaubi et al. Rare Dis Orphan Drugs J 2024;3:23 Rare Disease and
DOI: 10.20517/rdodj.2023.58
Orphan Drugs Journal

Perspective Open Access

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

Maria Cavaller-Bellaubi , Virginie Hivert, Roseline Favresse, Yann Le Cam

EURORDIS - Rare Diseases Europe, Paris 75014, France.
Correspondence to: Maria Cavaller-Bellaubi, Sant Antoni Maria Claret 167, Barcelona 08025, Spain. E-mail:
maria.cavaller@eurordis.org

How to cite this article: Cavaller-Bellaubi M, Hivert V, Favresse R, Le Cam Y. Framing the European Rare Diseases field through a
structured movement of patient organisations. Rare Dis Orphan Drugs J 2024;3:23. https://dx.doi.org/10.20517/rdodj.2023.58

Received: 16 Dec 2023 First Decision: 10 Apr 2024 Revised: 28 Jun 2024 Accepted: 26 Jul 2024 Published: 31 Jul 2024

Academic Editors: Jacques S. Beckmann, Anneliene Jonker Academic Editor: Fangling Lan Production Editor: Fangling Lan

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

© The Author(s) 2024. Open Access This article is licensed under a Creative Commons Attribution 4.0
International License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, sharing,
adaptation, distribution and reproduction in any medium or format, for any purpose, even commercially, as
long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and
indicate if changes were made.

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