EURORDIS 2024: Strategic Pathways and Collaborative Endeavours

By Yann Le Cam , Chief Executive Officer, EURORDIS-Rare Diseases Europe

As 2024 unfolds, EURORDIS stands at a critical juncture of transformative change. This year, against the backdrop of the EU elections, is pivotal in steering our path towards the ambitious objectives set for 2030. Our commitment deepens as we aim to thoroughly integrate the narrative of rare diseases into the wider context of healthcare, economic, and social considerations on national, European, and international levels. My transition to focus on international collaboration reflects this expanded horizon in rare disease advocacy on a global scale.

Strategic Focus in Europe

The year 2024 in Europe is earmarked for intensified policy advocacy, addressing the unique challenges and opportunities presented by rare diseases. With the EU elections in sight, our focus is on shaping an all-encompassing European action plan for rare diseases, with our strengthened Brussels public affairs team leading the charge.

Influencing the New EU Parliament and Commission: At the heart of our agenda is advocating for a European action plan, integrating rare diseases into the broader healthcare, economic, and social fabric. Our dual focus includes shaping new comprehensive policies, such as the European Health Data Space, and actively engaging in the review of existing legislation, such as the Orphan Medicinal Products Regulation.

The ‘Championing the Rare’ Campaign (#ActRare2024): This campaign, commencing on Rare Disease Day at the EU Parliament and continuing through to the EU elections, is designed to rally EU candidates around our cause, amplifying the voice of the rare disease community in pivotal political spheres.

Strengthening Networks for Greater Impact: 2024 is a year when we reinforce our support for member organisations, particularly focusing on regions requiring our utmost attention, including South-East and Eastern Europe. Additionally, we will concentrate on countries such as Spain, Italy, Greece, Luxembourg, Ireland, Sweden, and Finland, where emerging national plans for rare diseases necessitate a harmonised approach at both national and European levels.

Advancing Healthcare and Research: A foundational element of our strategy is to minimise diagnostic delays and improve access to specialised care, especially for ultra-rare conditions. Pioneering initiatives like the European Rare Disease Research Alliance (ERDERA) and the Rare Disease Moonshot are pivotal in propelling our understanding of rare diseases through cutting-edge research and clinical networks.

Global Reach and Collaboration

In 2024, EURORDIS will elevate rare diseases as a global health priority, engaging with international stakeholders to make them a central part of the global health narrative.

Advocacy at the United Nations: A key objective is to position rare diseases prominently on the global agenda. Leveraging the United Nations system, we aim to build upon our past achievements, including the Political Declaration on UHC in 2019 and the UN Resolution on Persons Living with a Rare Disease in 2021. Our focus is on advocating for the inclusion of rare diseases in a technical WHO resolution, the Convention on the Rights of Persons with Disabilities, and the Human Rights Council.

Fostering International Alliances and Partnerships: Central to our global strategy is building and nurturing international networks and collaborations on research initiatives. By sharing experiences and learning from global partners, we aim to address the challenges faced by the rare disease community on an international scale.

Global Healthcare Pathways: In advocating for comprehensive healthcare frameworks globally, our aim is to be at the forefront of developing tools for early diagnosis, and access to expertise, preparing the global healthcare system for the upcoming wave of diagnostic and therapeutic innovations.

2024 is a year of unprecedented opportunity to forge a future where rare diseases are not just recognised but are integral to the European and global agenda. Our mission is clear: to ensure that no one with a rare disease is left behind in our collective pursuit of a healthier, more inclusive world.