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

[7]
(EHDS) Regulation (2024) , among others.

EURORDIS’ way of working to promote and shape legislation, including national plans, is through
consultation with its members. This includes: surveys of PLWRDs, structured multi-stakeholder dialogues
[9]
such as EU-funded projects , the biennial European Conference on Rare Diseases (ECRD) , and the
[8]
EURORDIS Round Table of Companies (ERTC) ; EURORDIS Position Papers and numerous
[10]
[11]
contributions to the Commission’s fact-finding studies and public consultations. Upon the commencement
of a new piece of legislation, EURORDIS engages in active dialogue with the Commission services drafting
the legislative proposals, the most relevant Member States, and the members of the European Parliament
who are rapporteurs for the legislation. After its adoption, EURORDIS plays a key role in the
implementation of legislation, thanks to its European network of national alliances.

Most recently, with the revision of the General Pharmaceutical Legislation , EURORDIS aims to ensure
[12]
that incentives are provided to keep enhancing research towards effective treatments for PLWRD.

The establishment of the 24 European Reference Networks (ERNs) in 2017 marked another significant
milestone in EURORDIS’ contributions to the RD field. These networks connect specialised healthcare
centres across Europe, facilitating collaboration, knowledge, and expertise sharing. EURORDIS pushed and
defined rules and guidelines for the creation, development, and functioning of the ERNs, while ensuring
that they address the specific needs of PLWRD through cross-border healthcare .
[13]
EURORDIS advocacy style has evolved and matured over the years. Early on, EURORDIS initiated
[14]
scientific publications to support its evidence-based advocacy . In 2015, EURORDIS equipped itself to
perform independent surveys through the Rare Barometer Programme. Rare Barometer runs surveys in 24
different languages through a panel of more than 20,000 PLWRD and/or their carers across the globe and
[15]
conducts qualitative and quantitative studies . The results, generated directly from the experiences of
PLWRD, enable EURORDIS to deploy an evidence-based advocacy strategy geared towards person-centric
policies [Figure 1].

EURORDIS places the empowerment of patients and capacity building of patients’ organisations at the core
of its activities. Through training programmes and educational resources, the EURORDIS Open Academy
has equipped hundreds of patient advocates and researchers from more than 50 countries with the essential
skills and knowledge to become effective patient representatives and change makers, as well as to act as full
partners in research . By strengthening the collective voice of PLWRD, EURORDIS enables greater patient
[16]
engagement in research projects , Research & Development (R&D) , and participation in decision-
[17]
[18]
making processes . Some concrete examples include identifying patients, mentoring and supporting them
[19]
to navigate and effectively contribute to regulatory processes within the European Medicines Agency
(EMA) and Health Technology Assessment bodies, as well as in early dialogues with developers and
[20]
payers through the Mechanism of Coordinated Access (MoCA) .
[21]
EURORDIS also acts as a network leverager connecting advocates and stakeholders. The strategic approach
of EURORDIS creates a critical mass voicing the common challenges across all RDs and European countries
for the 30 million PLWRD. Over the years, EURORDIS has developed a set of activities involving the (at
[23]
present) 36 European National Alliances and the 80 European or International Federations to foster
[22]
cross-fertilisation amongst countries, member organisations, and disease areas, resulting in a pan-European
movement of over 2,000 rare disease patient organisations.