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A recap of the conference in Budapest: ‘For an EU Commitment to Tackling Rare Diseases’

January 2025

On Friday 29 November 2024, stakeholders from across Europe gathered in Budapest for the European Economic and Social Committee’s (EESC) conference, For an EU Commitment to Tackling Rare Diseases.’

Held under the auspices of the Hungarian Presidency of the Council of the EU, the event united policymakers, civil society representatives, patient advocates, and healthcare experts to chart the future of rare disease policies in Europe.

At the conclusion of the conference, EURORDIS issued a media statement acknowledging the vital contributions made throughout the event. Alongside its member organisations and the rare disease community, EURORDIS brought essential perspectives to discussions, marking a significant milestone in the push for a European Action Plan for Rare Diseases by 2030 and reinvigorating advocacy efforts.

The conference conclusions and recommendations called for urgent action, emphasising the need for a comprehensive European Action Plan by 2030, backed by political commitment and a steering group that includes patient representatives. Key priorities in the recommendations include addressing healthcare inequities, integrating care services, advancing research, and adopting innovative diagnostics and therapies. Collaboration, targeted funding, and incentives were also highlighted as crucial to success.

Civil society and patient organisations were recognised as central to raising awareness, shaping policy, and tailoring support. Priorities include strengthening psychosocial support, improving access to information, and fostering public-private partnerships. A unified European approach is essential to align health, social care, research, and data policies.

The conference recommendations will be shared with EU institutions and Member States to guide policymaking and ensure equitable, effective care for rare disease patients. This article recaps the event, showcasing key contributions and the shared vision for progress.


The Opening Session

Moderated by Claude Rolin, EESC member, the opening session struck a tone of urgency and a central theme that resonated throughout the day: the need for immediate action and the reassurance that a European Action Plan for Rare Diseases can build on existing progress rather than starting from scratch. Speakers emphasised the foundations already in place, calling for efforts to consolidate and expand these frameworks to achieve transformative progress.

Baiba Miltoviča, President of the Section for Transport, Energy, Infrastructure, and the Information Society at the EESC, called for urgent action, stating, “It is crucial to have patients directly represented when discussing any topics that directly affect their lives.” Highlighting the EU’s unique ability to bring “real added value” through cross-border strategies, she advocated for an Action Plan to ensure access to diagnosis within one year and lifelong care for patients wherever they live in the EU.

Péter Takács, Hungary’s State Secretary for Health, echoed the importance of international collaboration in rare disease diagnostics and research. He highlighted Hungary’s active participation in ERNs, with five national centres dedicated to rare diseases, and underscored the dual health and social challenges faced by families of patients. “Most rare disease patients, unfortunately, die before the age of five,” he said, stressing the moral duty to provide support without delay.

Péter Takács speaks at the conference in Budapest.

In his video address, Antonio Parenti, Director at DG SANTE, celebrated ERNs as “a flagship activity with clear EU added value” and praised the EU’s frameworks, such as the Cross-Border Healthcare legislation, which enable millions to access care abroad. He concluded, “Access to the best diagnostics, treatment, and healthcare should be ensured for all.”

MEP Tomislav Sokol (EPP, Croatia) delivered a video message calling for a unified Action Plan to address the needs of over 30 million people affected by rare diseases. “A European Action Plan will foster collaboration, moving beyond isolated efforts to build a networked approach that efficiently addresses the rare disease pathway,” he said, urging for a framework that provides dignity, support, and quality of life for those often unheard.

Together, these contributions from the Opening Session had set a tone of urgency for the conference.


Session One: How to Achieve a Comprehensive European Action Plan by 2030?

Moderated by Donata Meroni, Head of Unit at DG SANTE, this session explored the steps needed to deliver an Action Plan and began with reflections from key advocates.

Ágnes Cser, EESC rapporteur, presented the exploratory opinion Leaving No One Behind: European Commitment to Tackling Rare Diseases. Whilst reiterating the call for a European plan for rare diseases – the leitmotiv of her Opinion – she called for a European coordination body to centralise resources and ensure equitable care. “All people suffering from rare diseases should receive a diagnosis before one year,” she stressed, adding, “Rare disease families need support and information in their mother tongue, which is often not provided today. They should not have to travel thousands of kilometres just to understand their children’s health problems.”

Speaking on behalf of Europe’s rare disease community, Dr Virginie Bros-Facer, Chief Executive Officer of EURORDIS, delivered a compelling speech on the urgent need for a comprehensive Action Plan to address fragmented and inconsistent efforts across the EU. She praised the EESC for its advocacy and acknowledged existing resources, such as the European Reference Networks (ERNs) and the Rare 2030 recommendations. “An Action Plan is not about reinventing the wheel,” she said. “However, what is urgently needed is the ‘glue’ to bring these elements and stakeholders together in a cohesive, organised, and coordinated manner.”

Virginie Bros-Facer speaks at the conference in Budapest.

Dr Bros-Facer outlined essential components for an effective Action Plan, including establishing SMART (Specific, Measurable, Achievable, Relevant, Time-bound) goals, developing robust governance structures, and securing sustainable funding. “The European Union cannot fail our community. We believe that by working together, we can create a European Health Union where no one with a rare disease is left behind,” she concluded.


Session Two: Civil Society and Patient Organisations at the Heart of Rare Disease Policy

Moderated by Alain Coheur, EESC Rapporteur on Ensuring Strong European Solidarity for Rare Disease Patients, Session Two explored the critical role of civil society and patient organisations in addressing the multifaceted challenges faced by people living with rare diseases. Coheur underscored the importance of keeping rare diseases high on the EU agenda while addressing the struggles families face, lamenting, “We need to take into account families who are struggling with financial problems. Their means are limited, treatments are expensive, and treatments take a long time. We don’t talk about this enough.”

Erika Borszékiné Cserháti, representing the Érintettek Parents Association in Hungary, shifted the focus from diagnostics and treatments to two often-overlooked needs: empathy and information. “The first need is empathy – I’m convinced that this is the most important: to feel that we are not alone and to feel that we are not left behind,” she said. “The second is information. It’s crucial to prevent searching on the internet, where there is a lot of misleading and false information.”

Daniel Theisen of ALAN, Luxembourg, highlighted the transformative impact of his country’s 2018 national plan for rare diseases, emphasising the pivotal role his national alliance has played in implementing Luxembourg’s psychosocial care model. He stressed the importance of a European framework to build on these achievements, stating that Luxembourg’s effective plan would “still work best within a European approach.”

Lene Jensen, Chief Executive Officer of Rare Diseases Denmark, stressed the importance of recognising civil society’s contributions in delivering national strategies. She called for patient organisations to be formally supported and financed to provide critical services like helplines. “We have to make our case day after day, week after week, year after year – civil society is a resource and driver for change,” she said.

Jean-Philippe Plançon speaks at the conference in Budapest.

The session also included inputs from Gábor Pogány, President of RIROSZ (the Hungarian National Association of People with Rare and Congenital Disorders), Annalisa Scopinaro, President of UNIAMO Italy, and Jean-Philippe Plançon, Vice-Chairman of Alliance Maladies Rares France, who emphasised the need for coordination between health and other services, appropriate legislation and political engagement at a European and global level respectively.


Session Three: Unlocking the Potential of the European Health Data Space

The final session of the day focused on the transformative potential of the European Health Data Space (EHDS). Speakers explored how digital innovation can support rare disease research and improve healthcare delivery.

Hélène Dollfus, Coordinator of ERN-EYE, described the ERNs as “a perfect operational use case for the European Health Data Space (EHDS)” and highlighted their pioneering role in leveraging health data for primary and secondary uses.

Ana Rath, Director of Orphanet, underscored the critical need for rare diseases to be visible in health information systems, citing gaps in coding standards like ICD-10 and ICD-11. She advocated for the integration of OrphaCodes, a domain-specific terminology, to improve interoperability and public health insights within the EHDS.

Stefan Živković speaks at the conference in Budapest.

Stefan Živković, from the National Organisation of Rare Diseases of Serbia, called for ethical governance and patient empowerment to maximise the EHDS’s potential. “The EHDS is a unique opportunity that can revolutionise healthcare, especially for rare disease patients,” he said. “But ethical governance must ensure trust and empower patients, so they know how their data is shared and for what purposes.”


Closing Session: Charting the Path Forward

The conference concluded with a call to action, reaffirming the commitment of European institutions, civil society, and patient advocates to advancing rare disease policy.

Judit Bidló, Deputy State Secretary for the Professional Management of Health in Hungary, called for coordinated action to ensure equitable access to care across Europe.

Mónica García Gómez, Spain’s Minister for Health, reiterated her country’s dedication to reducing diagnostic delays and supporting families affected by rare diseases, stating, “Our challenge is to reduce the average time to diagnosis to under one year and to promote research into new treatments.”

Conference speakers cheerfully hold and raise hands in solidarity with the rare disease community at Budapest conference.

The conference reaffirmed the EU’s shared responsibility to develop a European Action Plan for Rare Diseases by 2030 and set a determined course for its delivery.

EURORDIS looks forward to advancing these efforts at the rare diseases conference proposed by Poland, provisionally scheduled for April 2025.


Julien Poulain, Communications Manager


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