Maximising our impact: Reflections from EMM 2025 in Riga
May 2025This week, the EURORDIS Membership Meeting (EMM) brought together leading rare disease advocates from across Europe. For the first time, the biennial event was held in Riga – a city whose history of resilience and civic action offered a fitting backdrop for discussions on strengthening the voice of the rare disease movement.
Opening the event, Avril Daly, President of EURORDIS-Rare Diseases Europe, offered a clear challenge: “We are stronger when we speak as one – and when we back our words with the tools, evidence, and ambition to build the future our community needs.”
Across two days of workshops, strategy sessions, and shared learning, participants took up that challenge. EMM 2025 focused not only on raising voices, but on strengthening them – with knowledge, clarity, and purpose.
We take a look at the topics and skillsets covered throughout this year’s meeting, and hear from many of the passionate and energetic advocates who spoke to us at the event.
Empowering advocacy at the European level
The European policy landscape is evolving fast. The European Health Data Space and Health Technology Assessment (HTA) Regulation are now entering implementation. The Critical Medicines Act has been proposed, and the revision of the EU pharmaceutical legislation is approaching trilogue negotiations.
In this context, the EMM 2025 workshop on political advocacy equipped participants to engage with these developments not just in theory, but in practice. Sessions looked ahead to key political moments – including the next EU budget – and offered guidance on aligning national advocacy efforts with broader Europe-wide opportunities.
“What brought me into this work was seeing my best friend’s daughter go through a long and difficult diagnostic journey with neurofibromatosis,” said Vera Lipkovskaya, Public Policy & Project Officer at NF Patients United, who attended the event’s workshop on advocacy.
“There was so little information, even among healthcare professionals, and no clear support. It quickly became clear that unless someone pushes at the system level, nothing improves for the next families facing the same condition – still waiting for a diagnosis or for care to move forward.”
“Doctors can help individual patients, but if you want an innovative treatment to actually become available, or a condition to be properly recognised and researched, you need policy change,” explained Vera. “And today, that change is increasingly being shaped at the European level – through legislation, budgets, and strategic direction.”
“If your disease isn’t visible there, it’s invisible in practice. That’s why patient organisations need a seat at the EU table – not just to be heard, but to ensure rare diseases are treated as a priority, with the funding and research they deserve. What we need now is something that brings these efforts together: a comprehensive European Action Plan on Rare Diseases. Such a plan will not only improve patient outcomes but also drive cost efficiencies, delivering long-term savings for taxpayers and strengthening the sustainability of our healthcare systems.”
Strengthening advocacy at the national level
While European frameworks offer valuable alignment, most decisions that shape the lives of people with a rare disease – from diagnosis and care coordination to treatment access and social support – are made at national level. Yet across Europe, national rare disease strategies remain inconsistent: some are renewed and resourced, others are outdated or incomplete.
At EMM 2025, the advocacy workshop had also addressed this challenge head-on. It focused on what it takes to build momentum for national action – identifying political windows, crafting messages that resonate with domestic audiences, building cross-sector alliances, and sustaining pressure where needed. The emphasis was not just on what to ask for, but on how to ask effectively.
In Portugal, these lessons resonate clearly. Joaquim Brites, President of the Associação Portuguesa de Neuromusculares (APN, the Portuguese Neuromuscular Association), described recent developments in his country’s national policies: “Portugal has made positive progress in updating its national rare disease strategy, most notably by launching a public consultation on the draft Action for Rare Diseases 2025–2030.”
At the same time, he warned that critical gaps remain. “At present, it appears that the plan will not include the dedicated funding necessary to achieve its goals. This is an area where further improvement is needed,” Joaquim said.
“For the APN, and for our friends at other Portuguese rare disease organisations, ongoing advocacy remains crucial – not only to help shape the strategy itself, but also to push for the financial commitments that will ultimately determine its success.”
Engaging with EMA bodies
One of the most urgent issues discussed in Riga was the future of patient involvement in the European Medicines Agency (EMA). As part of the EU pharmaceutical legislation revision, there’s concern that patient representatives on key EMA committees – the CHMP and PRAC – may not be guaranteed voting rights.
In a workshop on regulatory engagement, participants explored what it means to be part of EMA structures and why it matters that patient voices are not only included, but empowered. This workshop took place against the backdrop of a recent joint statement having been published by EURORDIS and the European Patients’ Forum (EPF) calling on EU Member States to protect these rights – not as a gesture, but as a safeguard for credible, inclusive decision-making relating to the lifecycle of medicines.
Tomasz Grybek from Poland, a EURORDIS Board member, father of a child with metachromatic leukodystrophy, and patient representative on the EMA’s Paediatric Committee (PDCO), spoke from direct experience. “Patients bring something essential to EMA Committee discussions – the lived experience,” he explained.
“These are highly educated panels, but that real-world perspective is often the missing piece of the puzzle. Skilled patient advocates have become true partners in the process of medicines’ evaluation, helping to complete the picture. And if our community is counting on more treatments becoming available, then we need to be ready to contribute meaningfully at every stage.”
Participating in the assessment of health technologies
You might not hear about Health Technology Assessment (HTA) every day, but its impact is felt everywhere in healthcare. HTA helps decide which treatments are reimbursed, when, and for whom – shaping access to care across Europe.
The workshop on HTA focused on the new EU HTA Regulation, which has introduced Joint Clinical Assessments (JCAs) at European level. These assessments – which will gradually expand to cover orphan medicines – are intended to streamline national decisions and reduce duplication.
Participants explored how patient advocates can influence and meaningfully inform this process: by interpreting clinical data through the lens of real-world needs, by helping define what value means for their communities, and by joining structured efforts like EURORDIS’ HTA Taskforce and Community Advisory Boards (CABs).
Among those reflecting on this shift was Gaëtan Duport, representative of the European Haemophilia Consortium in the EU HTA Stakeholder Network. “When I first entered HTA discussions, I wasn’t always confident I belonged – I didn’t have a PhD in economics, and it took time to understand how my voice could carry weight,” he said.
“But I came to realise that what I bring, as a patient and as someone grounded in real-world experience, is precisely what these systems often lack. Health economists are important, but they can’t fully capture what matters most to patients on their own. That’s why meaningful patient engagement in health technology assessment is so indispensable.”
Participating in European Reference Networks
The European Reference Networks (ERNs) are a success story – but like many success stories, their future depends on what happens next. Built to connect expert centres across borders, the ERNs have grown into a key part of Europe’s rare disease infrastructure. And patients have played a role in that from the beginning.
The workshop series on volunteer engagement also offered a grounded look at how that involvement can deepen. Participants heard from experienced advocates and came away with a clearer sense of how to contribute – and where gaps still exist.
As Flavia Galletti, patient advocate in ERN ERKNet, shared: “Being part of ERN ERKNet as a patient advocate has been one of the most rewarding experiences I’ve had. You gain real skills, grow through collaboration, and work alongside people who share your purpose.
“Scientists in ERNs are like expert archers – focused, precise, with a clear target in sight. Patients, by contrast, are like alpinists, climbing with both eyes wide open – we see the broader landscape, the challenges and opportunities that shape everyday life with a rare disease. In an ERN, it’s this combination that makes the work truly impactful. Strong patient representation not only improves the network’s output – it inspires new lines of research and helps push good ideas further: to decision-makers, and ultimately, into people’s lives.”
Deploying valuable data from Rare Barometer
While other workshops focused on who and how to engage, the workshop on using EURORDIS Rare Barometer findings addressed a different question: what data can be brought by patient advocates to the conversation? Participants explored how EURORDIS’ Rare Barometer surveys – which regularly capture the experiences and priorities of thousands across Europe – can make advocacy more grounded, more credible, and harder for policymakers and wider society to ignore.
Whether pressing for national strategies, HTA reform, or stronger ERNs, Rare Barometer provides a foundation of evidence. It transforms personal stories into collective insight – giving advocates what decision-makers need most: clarity, credibility, and data-backed urgency.
“The Rare Barometer surveys offer critical insights into the realities faced by individuals living with rare diseases, including those affected by rare lipid conditions such as Homozygous Familial Hypercholesterolaemia and Familial Chylomicronaemia Syndrome,” said Maja Bartoszewicz-Moritz, Project Manager for Rare Diseases at the FH Europe Foundation. “These findings serve as a valuable tool for understanding the multifaceted challenges encountered by the rare disease community and are instrumental in informing more effective advocacy and policy work across Europe.”
“One in three people with a rare disease are not recognised as having a disability. This reflects a wider issue among individuals with invisible or misunderstood conditions. The consequences of such misrecognition are significant – including exclusion from essential services to inadequate policy responses. At FH Europe, such data is used to underpin efforts to drive forward inclusive health and social policies, ensuring that the needs of people with rare diseases are acknowledged and addressed within appropriate frameworks and national health strategies.”
A community ready to make an impact
EMM 2025 was not just a meeting of minds – it was a moment of skill-building, message-testing, and shared direction.
Across regulatory systems, national governments, and EU institutions, the rare disease community has a role to play. The workshops in Riga helped make that role clearer. What comes next depends not only on ambition, but on preparation – and on continuing to speak not just loudly, but effectively.
Julien Poulain, Communications Manager