Originally published at: https://www.eurordis.org/role-in-erdera-and-reshaping-rare-disease-research/
EURORDIS-Rare Diseases Europe and our community of advocates have long called for a more coordinated research environment – one that connects countries, reduces duplication and gives every family the fairest chance at diagnosis and treatment.
The European Rare Diseases Research Alliance (ERDERA), launched in September 2024, was built on earlier joint programmes such as the European Joint Programme on Rare Diseases (EJP-RD) and E-Rare, and represents the first attempt to create a framework for rare disease research at this scale.
It brings together 178 organisations from 37 countries to develop shared tools, capacity and clinical research pathways that no single country could build alone. The European Union contributes around €150 million to this co-funded seven-year partnership through Horizon Europe, while the remainder comes from Member States, countries associated with Horizon Europe, and cash and in-kind contributions from public and private partners.
EURORDIS supported the partnership from its earliest conception and continues to ensure that patient experience and unmet needs shape ERDERA’s governance, research design and national outreach. That role matters now more than ever: rapid advances in genetic diagnostics, functional genomics and advanced therapies are changing what is possible for rare diseases. Across the partnership, tangible progress is emerging – from deeper national alignment to growing ATMP readiness and an expanding training offer for patient advocates.
This article explains how EURORDIS is contributing to ERDERA and how those contributions are shaping the partnership’s development.
National Mirror Groups: strengthening alignment
One of the key developments within ERDERA is the continued expansion of National Mirror Groups (NMGs), initiated during the EJP-RD. NMGs provide a platform that fosters rare disease research (in particular) at the national level by coordinating relevant actors and initiatives, as well as aligning with and fostering increased international efforts. These actors include authorities, researchers, ERN experts, funders, in some cases industry, and Rare Disease National Alliances who provide the patients’ perspective.
The work of NMGs should increasingly support national rare disease policies and plans while offering valuable insight into the latest scientific and technological developments, with the aim to integrate them into healthcare.
Eighteen NMGs are now in place, with the goal of establishing one in each of the 37 ERDERA partner countries.
Established NMGs are developing their three-year plan to implement agreed activities, emphasising cross-country collaboration. Regional clusters based on shared needs are planned, and NMGs are expected to participate across ERDERA initiatives, such as the train-the-trainer programme.
Fostering engagement of underrepresented countries in rare disease research
ERDERA is committed to supporting the participation of countries that remain under-represented in rare disease research calls, consortia, and other research-related initiatives. The recent online workshop, “Networking of Underrepresented Countries for the Enhancement of Rare Disease Research” (20–21 November), attracted 157 participants from 25 underrepresented countries, including Widening and Associated Countries.
This event was an important milestone, gathering numerous valuable insights that will help shape ERDERA’s forthcoming activities, strengthen the visibility and inclusiveness of underrepresented countries, and support a more connected and balanced rare disease research landscape.
“EURORDIS is actively engaged and works closely with Rare Disease National Alliances to support the development, expansion, and consolidation of NMGs. It is also fully committed to promoting fairer inclusion of underrepresented countries in rare disease research by leveraging its network of rare disease patient organisations in these countries”.
– Ariane Weinman, Public Affairs Senior Manager, EURORDIS
Advancing the future of therapies: EURORDIS’ role in shaping ATMP development
Advanced Therapy Medicinal Products (ATMPs) – including gene, cell and RNA-based therapies – are rapidly changing what is possible in the rare disease therapeutic landscape. ATMPs hold a lot of promise for rare and ultra-rare diseases, not only for their life-transformative potential but also because they are particularly amenable to genetic conditions regardless of their prevalence. In fact, rare diseases have a track record of being an effective hub for ATMP-related innovation.
From a strategic perspective, as Europe is striving to ‘pick-up the Biotech Competitiveness’ pace’, ATMPs, and especially ATMP-based technology platforms, can be game-changers in achieving this ambitious, but necessary goal. Because many rare conditions are genetic, ultra-rare or lack any therapeutic pathway, preparing Europe for responsible ATMP development has been a strategic priority within ERDERA from the outset.
EURORDIS has taken a leading role in this area through its co-development of an ATMP prioritisation framework. The framework is designed to help identify which rare diseases are most suitable for ATMP development and will guide ERDERA’s Technology Accelerator as it matches platform technologies with specific conditions. Crucially, the framework is being built so it can be used well beyond ERDERA – by biotech and pharmaceutical developers, academic teams and patient organisations – thanks to its rigorous, evidence-based and participatory design.
The methodology underpinning the framework has been extensive. It began with one-to-one interviews with patient advocates, clinicians, academic researchers and biotech and pharma experts, exploring which criteria should guide prioritisation. These insights were tested and refined through an online questionnaire, before being brought together in a hybrid multi-stakeholder workshop that included ethics experts. This process has ensured the framework reflects not only scientific and infrastructure readiness, but also the ethical, psychosocial and societal dimensions of living with a rare disease. Factors such as quality of life, caregiver burden and barriers to participation are treated as core elements, not secondary considerations.
European Reference Networks and ePAG advocates will be more deeply involved in the next phase of disease-area rating. Throughout the process, EURORDIS has ensured that patient perspectives remain central and that underserved communities are not overlooked. In doing so, it is helping shape an ATMP development model that is ambitious, evidence-driven and grounded in lived experience.
“What makes this ATMP framework unique is its holistic and multi-stakeholder take, particularly how deeply it reflects patient realities. By capturing scientific, ethical and psychosocial dimensions together, it gives Europe a clearer path toward developing advanced therapies where they are needed most”.
Rita Francisco, Patient Engagement Manager (ATMPs & Clinical Research), EURORDIS
Training and empowerment: building a research-ready rare disease movement
A resilient research ecosystem depends on advocates who can engage confidently with scientists, regulators and policymakers. ERDERA’s partnership with the EURORDIS Open Academy strengthens this foundation through expanded, high-quality training designed to prepare patient representatives to participate across the partnership.
The 2025 Open Academy Schools illustrate this momentum. Across the Medicines Research & Development School and the Scientific Innovation & Translational Research School, more than 170 people applied and over 70 advocates and early-career researchers from more than 25 countries received training. Participants praised the expert-led content, the practical focus and the value of connecting with peers across Europe.
Specialised training has also grown. The first edition of the Data, Ethics and AI programme, developed in collaboration with the World Duchenne Organisation and the Duchenne Data Foundation ran from January to June. It combined eleven webinars with eLearning modules and brought together 31 participants from 17 countries. Feedback highlighted the clarity of the learning materials and the strength of the cross-country discussions. The next edition will adopt a hybrid format to deepen engagement.
Young people are also being supported. The Young Patients Training held in Athens and coordinated by TEDDY, the Institut Imagine, and Conect4Children, introduced youth advisory groups to clinical research, ethics and patient rights, helping prepare a future generation of informed advocates.
Beyond structured teaching, the Open Academy launched this year the ERDERA Mentorship Programme for alumni. Over four months, participants work in small groups with expert mentors on skills relevant to real-world advocacy, including community advisory boards, registries, centre accreditation, partnerships in clinical networks and mental health within organisations. This ongoing support strengthens advocates’ ability to contribute within ERDERA and beyond.
Together, these initiatives reflect a shared conviction: that scientific progress must be matched by empowered patients. ERDERA’s ambitions can only succeed if the community is equipped to shape them – and the Open Academy is central to that effort.
“Progress happens when people have the tools to help shape it. Through the Open Academy, we’re giving advocates the knowledge and confidence to become architects of the research that will change their future”.
Marta Campabadal, Open Academy Senior Manager
Looking ahead: a shared commitment to long-term transformation
ERDERA’s work is progressing with a clear pipeline of key action.
The 2026 Joint Transnational Call, launching on 10 December, will fund multinational teams to resolve unsolved rare genetic and non-genetic diseases. A one-off Clinical Trials Call — supported by €30 million from the European Commission — and the Public-Private Collaboration Accelerator, due to open in early 2026, will strengthen collaboration between academia, industry and patient communities. Work will continue across diagnostics, real-world data, clinical research readiness, methodological guidance and international alignment.
EURORDIS will remain fully engaged: strengthening patient engagement across the partnership, driving ATMP prioritisation and innovation, and expanding training and mentoring so advocates can contribute meaningfully at every stage. ERDERA embodies a patient-centred approach to innovation — placing patient needs at the start of the research and development process — and EURORDIS will keep ensuring those needs shape decisions and outcomes.
Looking ahead, ambition must be matched by resources. The next Multiannual Financial Framework (2028–2034), together with the EU Life Sciences Strategy and the Biotech Act, are pivotal moments to secure predictable, long-term support for ERDERA.
EURORDIS is actively calling for adequate EU investment and the policy frameworks needed to sustain the partnership, because predictable funding is essential if the scientific, social and economic returns of rare disease research are to be realised for families across Europe.
Written by Julien Poulain, Communications & Policy Liaison Senior Manager, EURORDIS.
