Eurordis - Rare Disease Europe

A look at #EMM2025: Maximising our impact

The 2025 EURORDIS Membership Meeting will take place in Riga, Latvia, on 22-24 May. Under the theme “Maximising our impact: Strengthening the voice of the rare disease community in decision-making,” the meeting will bring together rare disease advocates, patient leaders, and stakeholders from across Europe to strengthen our collective impact. 

Across Europe, decision-makers are shaping the future of healthcare, innovation, and support systems. To ensure that the needs of people living with rare diseases are at the heart of those decisions, our collective voice must be stronger, more informed, and more strategic than ever.

That’s why this year’s EMM workshops are designed to equip you – our member organisations and advocates – with the tools, knowledge, and confidence to make your voice heard where it matters most.  

Through interactive, hands-on sessions led by experienced speakers and fellow community members, we’ll explore essential topics including mental health, advocacy strategies, patient engagement in the healthcare system, and how to use data effectively to influence policy. These sessions go beyond discussion; they’re practical, empowering, and rooted in the real experiences of our community. 

Mental Wellbeing

The path to diagnosis is long – an average of five years – and filled with uncertainty. Many people face chronic uncertainty, isolation, and stigma, due to the lack of awareness and understanding that is the hallmark of rare diseases. It’s no surprise that people living with a rare condition are three times more likely to experience depression than the general population. [1]

At the European level, mental health is finally gaining recognition, but people living with rare diseases remain overlooked. Rare conditions are not formally recognised as a vulnerable group in EU mental health strategies. National plans often exclude mental health, leaving care patchy, underfunded, or blocked by practical barriers like geography, language, or complex procedures. Psychological support is rarely built into care pathways.

What’s next? 

We must integrate mental wellbeing into every aspect of rare disease care. This means funding dedicated programmes, developing psychosocial services, and making sure mental health is treated as a right and not a luxury. The wellbeing of millions depends on it. 

About the Workshop 

This workshop is designed to build the capacity of patient group leaders to confidently navigate conversations around mental health, advocate for the right support within their communities, and foster safe, open spaces where individuals living with rare conditions can share their experiences without fear or stigma. 

This workshop will empower you to learn and use tools and approaches that have worked for other communities, with a focus on practical, peer-led learning. Through hands-on exercises and open discussion, you’ll learn how to: 

• Gain skills to promote discussion around increasing the importance of quality of life and taking care of wellbeing in your communities. 

• Learn practical, peer-tested tools and advocacy strategies to strengthen your community’s resilience and emotional wellbeing 

• Recognise and prevent burnout and strike a healthy work-life balance as a patient advocate 

Supporting others starts with protecting your own mental health. This space is about equipping you to do both. 

Advocacy at National and EU Levels 

Advocacy has long been the heartbeat of progress in the rare disease movement. Over the years, our communities have helped shape policies, influence research agendas, and secure concrete improvements in access to healthcare, treatments and social inclusion. Yet, as we look across the European and national landscapes, it’s clear that while much has been achieved, we’re only partway up the mountain. 

At the European level, the rare disease community has secured key wins: 

• The Orphan Medicinal Products Regulation has fostered innovation for rare diseases since 2000. 
• The Rare2030 Foresight Study created a strategic vision and a set of recommendations – backed by broad community input – for future rare disease policies in Europe. 
• The prioritisation of rare conditions in the EU Pharmaceutical Strategy shows that policymakers are listening – when we speak clearly and collectively. 

But policy change at the national level, where health systems are managed and implemented, is just as vital. Here, we face more uneven progress: 

• Some countries have developed National Plans for Rare Diseases that improve diagnosis, care coordination, and access to treatment. Others are still in the early stages or have plans that remain underfunded or inactive. 
• Health inequalities persist, especially in countries with limited rare disease expertise or resources. 

So, what’s next?

People living with a rare disease in Europe continue to call for a world where they can have longer and better lives and achieve their full potential, in a society that values their well-being and leaves no one behind. A lot remains to be done, at the local, national, European and international levels. 

We must ensure that people living with rare diseases are not only represented but are actively shaping policies that affect their lives. That means being ready – with the right skills, tools, and messages. 

About the Workshop 

The Engaging with Policymakers workshop will give participants a hands-on introduction to advocacy: what it looks like in practice, how to start or scale your efforts, and how to build relationships with national and EU-level decision-makers. You’ll work with a new Advocacy Toolkit, developed by EURORDIS, which walks you through every step: from identifying opportunities for influence, to creating impactful campaigns and presenting the right evidence. 

This is your space to: 

• Learn by doing, not just listening 
• Build confidence in how you advocate 
• Connect with others doing similar work in their countries 
• Get inspired by the wins we’ve already achieved – and plan the next ones 

Patient Engagement 

For people living with a rare disease, regulatory decisions, access to treatments and clinical practice are never abstract. They’re urgent, personal, and often life-defining. That’s why patient partnerships are essential – not optional. When patient representatives help shape care services, treatment development and access, the results can be more relevant, more sustainable and more impactful. 

Patient involvement has gained ground in recent years. More organisations now open their doors to patient voices – including the European Medicines Agency (EMA), European Reference Networks (ERNs), and Health Technology Assessment (HTA) bodies. 

These efforts are starting to deliver real results, especially in therapeutic development through the involvement of patient representatives in the EMA. Over the past 25 years, patient representatives have played a critical role in reinforcing trust in the regulatory system, making sure that medicines reflect the real-world needs of the people they are intended to serve, and contributing valuable insights into the safety, efficacy, and impact of treatments.    

Yet serious gaps remain. Engagement can at times feel tokenistic and patient representatives often go unsupported. Volunteers are facing an increased number of requests to contribute and provide input from different organisations. And, while the patient community has made significant strides in gaining a voice in decision-making spaces, ongoing efforts are needed to ensure that representation remains strong and meaningful.

About the Workshop

Volunteer engagement across the medicine’s lifecycle and healthcare is a two-part workshop that will guide you through how patient representatives can partner meaningfully in regulatory decision-making, access to treatments and care. You’ll learn what involvement really looks like — and where your voice can make a difference. 

Part 1, moderated by Maria Cavaller and Ines Hernando, will explore how patient representatives contribute to the work of the EMA and ERNs. You’ll get practical details on time commitments, skills needed, and how to navigate different engagement policies. Through short presentations and by engaging with experienced volunteers who will answer your questions you’ll leave with a clear view of what’s possible – and how to get started. 

Part 2, led by Julien Delaye and François Hoüyez, will take you deeper into Health Technology Assessment cooperation at the EU level, and what it means for national reimbursement decisions. You’ll hear from patient advocates active in EURORDIS taskforces and Community Advisory Boards (CABs), who will share case studies and insights into clinical trial design, digital tools, and why these platforms matter. 

This is your space to: 

• Discover how patient organisations and patient representatives can have real impact when partnering with other stakeholders – ERNs, EMA, HTA bodies and industry 
• Learn how to engage with key stakeholders. 
• Understand how EURORDIS collaborates with and supports patient representatives who partner with health professionals, regulators, HTA bodies and industry. 
• Understand the structure and goals of Community Advisory Boards Connect with a network of peers shaping the future of rare disease clinical practice, drug development and access to treatments.  

Data-Driven Advocacy with Rare Barometer

Clinical knowledge alone doesn’t capture the full picture of life with a rare disease. Real progress depends on understanding what matters the most to patients and families. That’s where the Rare Barometer programme plays a key role. By turning thousands of personal experiences into reliable, community-led data, it gives people with a rare disease the power to influence policy and drive change.  

Since 2015, Rare Barometer has gathered the views of the rare diseases community across Europe and beyond. The findings fuel reports, factsheets, and dashboards used to support advocacy on healthcare access, research funding, diagnosis, and social inclusion. But not every patient organisation has the tools, time, or confidence to use this data effectively – especially smaller or volunteer-run groups. 

About the Workshop 

Using Rare Barometer Results for Advocacy is a hands-on workshop designed to help patient representatives turn data into action. Led by Jessie Dubief and a panel of experienced advocates, you’ll learn how to find the right insights, understand key statistics, and use Rare Barometer results in practical, impactful ways. 

You’ll work on three real-world exercises using Rare Barometer data: 

• Creating a clear and compelling scientific poster.

• Preparing a presentation for policymakers.

• Developing an advocacy strategy for your organisation.

This is your space to: 

• Learn how to communicate evidence with clarity.

• Build confidence in using data to support your message. 

• Share what works with peers from across Europe. 

• Make your advocacy efforts stronger, smarter, and more persuasive. 

Good data makes a difference when it’s in the hands of people ready to use it. 

[1] EURORDIS, “Juggling care and daily life: The balancing act of the rare disease community – report“, May 2017 

If you’re ready to grow your impact, connect with others, and gain practical tools to support your advocacy, don’t miss #EMM2025 in Riga, 22–24 May. These workshops are built for you – patient representatives, advocates, and leaders – to strengthen your voice and shape the future of rare disease policy together.  

Claudia Gugliuzzo, External Communications Intern