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Page 2 of 8 Cavaller-Bellaubi et al. Rare Dis Orphan Drugs J 2024;3:23 https://dx.doi.org/10.20517/rdodj.2023.58

INTRODUCTION
EURORDIS is a non-governmental patient-driven alliance of 1,042 patient organisations in over
74 countries, covering more than 4,000 diseases. EURORDIS is a purpose- and value-driven organisation,
multinational and multilingual by nature, with an impact in Europe and beyond. EURORDIS is an
[1]
independent organisation with strong governance and balanced revenue sources .

Since its establishment in 1997, EURORDIS has been at the forefront of advocating for the rights and well-
being of people living with rare diseases (PLWRD) globally. Building upon a powerful community by
uniting PLWRD, their families, and support groups, EURORDIS has influenced research, healthcare,
medicines development and access, and has simultaneously shaped policies at national, European, and
global levels for more than two decades. EURORDIS has done so by promoting awareness, disseminating
information, enabling networking, and sharing experiences to empower PLWRD, support groups and
[2]
patient organisations . EURORDIS, as a civil society organisation, brings together key stakeholders to
engage with policy makers and competent authorities.

This perspective paper explores some of the main achievements of EURORDIS over the past two decades
and examines how the organisation has contributed to impacting the lives of the 30 million people in
Europe facing the challenges of living with rare diseases.

ADVOCACY - INFLUENCE FOR CHANGE
EURORDIS was created in 1997 by four funding members: the AFM-Telethon, AIDES, Vaincre La
Mucoviscidose, and La Ligue Nationale Contre le Cancer with the initial goal to advocate for European
Union (EU) laws to bring medicines to PLWRD .
[2]

Since then, EURORDIS has been a strong advocate for PLWRD at the EU level and beyond. Through its
policy, advocacy efforts, and innovative solutions, the organisation has enabled change, advocating for rare
diseases (RDs) to be recognised as a public health priority at the EU level, improving access to healthcare
services, research funding, and development of orphan drugs. By creating a trustful and collaborative
environment and dialogue with policymakers, regulators, researchers, developers, clinicians, payers, and
other stakeholders, EURORDIS has successfully influenced the development of policies, regulations, and
frameworks to address the challenges faced by PLWRD, improving the quality of life.

The founding members and EURORDIS have played a crucial role in shaping the EU Regulation on Orphan
Medicinal Products (2000) from contributing to reports, working closely with the National Organisation
[3]
for Rare Disorders (NORD), the Food and Drug Administration (US FDA), leading patient advocates,
industry associations in Europe, and the French EU Presidency and Minister of Social Affairs, Health and
Cities, Simone Veil, in 1995, to promoting the key features and positive impact of the US Orphan Drug Act
through media and conferences (French Senate, October 1996). The organisation went on to work with the
European Commission on the legislative proposal (1997-1998) and with the European Parliament
Rapporteur, Françoise Grossetête (1998-1999), on the amendments and eventual adoption.

As a direct result of EURORDIS’ efforts, in 2008, the Commission issued a communication to promote RDs
as a priority in the Member States through national plans and strategies. Together with the Council
recommendation on addressing the challenges of PLWRD (2009), this led to new opportunities for policy
development and investment in rare diseases. The organisation has also played a key role in shaping other
legislation such as the Paediatric Regulation (2006) , the Cross-Border Healthcare Directive (2011) , and
[5]
[4]
more recently, the Health Technology Assessment Regulation (2022) and the European Health Data Space
[6]