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Page 6 of 8 Cavaller-Bellaubi et al. Rare Dis Orphan Drugs J 2024;3:23 https://dx.doi.org/10.20517/rdodj.2023.58

Table 1. EURORDIS priority areas of work
Six priority areas:
1. Earlier, faster and more accurate diagnosis within 6 months
2. High-quality national and European healthcare pathways, including cross-border healthcare. The goal is to improve survival by 3 years on
average over 10 years and to reduce the mortality of children under 5 years of age by one-third
3. Integrated medical and social care with a holistic life-long approach and inclusion in society by reducing the social, psychological, and
economic burdens
4. Research and knowledge development that is innovative and led by the needs of people living with a rare disease
5. Optimised data and health digital technologies for the benefit of people living with a rare disease and society at large
6. Development and availability, accessibility, and affordability of treatments, particularly transformative or curative therapies

EURORDIS’ next important milestone is to transform all the frameworks and current policies into
opportunities of care and treatments for patients.

One of the latest initiatives is the Rare Diseases Moonshot, launched in 2022, aimed at accelerating research
in “white spot” areas where no treatment is available, and where no research is being undertaken. This
coalition of stakeholders to which EURORDIS contributes is a natural follow-up of previous joint
endeavours aimed at building a trustful and collaborative research environment to speed up public-private
partnerships for the ultimate benefit of patients .
[34]

EURORDIS is a full partner of the European Joint Programme on Rare Diseases (EJP RD programme). In
2019, EURORDIS constituted a group of patient representatives, funding agencies, and researchers in order
to discuss patient partnerships in research and to develop a guide with best practices on how to co-create
research, from the initial stages of biomedical research, together with patients and researchers. Those
guidelines were the first guidelines in the biomedical rare diseases research area at the EU level and have
helped address challenges in developing patient partnerships in research by providing information and
resources to applicants trying to navigate the application process. Building upon the experience of the EJP
RD , where the EU and Member States jointly co-fund activities, the European Rare Diseases Research
[35]
Alliance (ERDERA) shall coordinate national, local and European research and innovation programmes
from September 2024 onwards. ERDERA goes deeper in research cooperation, encompassing clinical
research networks, a concept that EURORDIS has strongly pushed for many years, along with coordinating
training activities and bringing patients’ voices to research programmes, as has been done within EJPRD
since 2019.

EURORDIS is also very active in scaling up the training opportunities provided to patients through the
EURORDIS Open Academy and advocating for further development and progress in healthcare settings.
For instance, since 2014, EURORDIS has been instrumental in developing the terms of reference for the
ERNs, followed by the development of dedicated European Patient Advisory Groups (ePAGs) . To further
[36]
support ERNs’ development and turn them into concrete opportunities for patients to receive treatment
[37]
and/or to enrol in clinical research studies, EURORDIS is supporting the Together4RD initiative which
aims to alleviate the barriers to ERN-industry collaboration .
[38]
CONCLUSION
EURORDIS’ achievements and contributions to the rare disease community are vast and far-reaching.
EURORDIS has constantly redefined its unique role in the rare disease ecosystem. From its initiation as a
network leverager engaging patient organisations, to its active involvement in shaping policies, fostering
research, empowering patient organisations and individuals to be involved in decision making, EURORDIS