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invaluable in shaping research priorities, informing policymaking, and driving resources towards improving
[8]
the lives of PLWRD .
In 2009, EURORDIS and NORD inspired the European Commission (EC) and National Institute of Health
(NIH) to join forces with other public funders to promote research in rare diseases at the global level
through the establishment of the International Rare Diseases Research Consortium (IRDiRC) in 2011 .
[27]
IRDiRC is a global consortium aimed at advancing rare disease research by working together to build a
multi-stakeholder collaborative approach to research funding and priorities, and to achieve targeted goals .
[28]

In 2013, Ågrenska Foundation and EURORDIS, together, thanks to their status as Non-Governmental
Organisations (NGOs) and working relationship with the United Nations (UN), created the NGO
[29]
Committee for Rare Diseases at the UN ECOSOC Committee of NGOs . EURORDIS also supported the
creation of Rare Diseases International (RDI), the global alliance of rare disease patient organisations
worldwide. EURORDIS mobilised patient groups from around the world to support the initiative and
provided financial and in-kind resources until 2023. Now, RDI is a full, independent, legitimate, and
credible international NGO. EURORDIS-RDI’s major joint accomplishments have been the inclusion of
rare diseases in the UN political declaration of universal health coverage in 2019, which was reinforced in
2023. Additionally, the Resolution on Addressing the Challenges of Persons Living with a Rare Disease and
[30]
their Families was adopted in 2021 .
REPOSITIONING EURORDIS FOR THE FUTURE: SCIENTIFIC ADVANCEMENTS,
GROWING KNOWLEDGE AND TECHNOLOGIES
EURORDIS’ more than 25 years of experience have positioned the organisation to lead the way for the
future of the rare disease community, aligning with the UN Sustainable Development Goals Agenda 2030,
One Sustainable Health Approach, and the future European Health Union. These initiatives promote an
innovative, transformative European model of care that will be driven by the needs of PLWRD and a
collective responsibility to prioritise solidarity and equity . The Foresight Rare 2030 study enabled a multi-
[31]
stakeholder consensus analysis of the main challenges and trends for the next 15 years. This study has
confirmed the positioning of EURORDIS as a network leverager within an increasing and complex
ecosystem and produced a set of recommendations . Since these recommendations were published,
[32]
EURORDIS and its members have been calling for a European Action Plan for Rare Diseases which would
bridge the gaps between all actions and initiatives on rare diseases at the national and European levels .
[33]

Besides EURORDIS’ long-term strategy, based on three complementary pillars - empowerment, partnering,
and advocating - three strategic objectives have been defined. The first one is a new European policy
framework to achieve measurable goals to prolong and improve the lives of PLWRD by 2030. The second
goal is to deliver on six priority areas [Table 1]. The last one is to "leave no one behind" through the
inclusion of all rare diseases with specific targeted actions (e.g., auto-immune rare diseases, rare infectious
diseases, rare health hazards, ultra-rare diseases, the one in a million) in all regions of Europe, combining
policy frameworks (e.g., Universal Health Coverage, UN Resolution, WHO Resolution, and WHO Work
Programmes) with bottom-up empowerment of patient groups and clinicians, and local dialogue on
national strategies .
[31]

PROMOTING AND SUPPORTING NEW FLAGSHIPS INITIATIVES: HOW THIS WILL BE
TRANSLATED IN THE ECOSYSTEM?
With the upcoming changes to the General Pharmaceutical Legislation and other forthcoming initiatives,