EURORDIS-Rare Diseases Europe

is just around the corner! 🚨

Join Session 2 of the Preparing Reimbursement Decisions track to explore how the EU Health Technology Assessment Regulation (HTAR) can better support people living with rare and ultra-rare diseases.

Bringing together stakeholders from across the rare disease community, the session will examine the practical consequences of HTAR implementation and perspectives on its future development.

Building on experiences with oncology products and advanced therapy medicinal products (ATMPs), discussions will explore how European joint assessments are being used at national level and whether they are supporting more effective dialogue and decision-making. The session will also address patient involvement, the use of real-world data and evidence, and preparations for the inclusion of orphan diseases and medical devices within the HTAR scope in 2028.

📆 Thursday 4 June 2026
💻 Online participation remains available until the conference begins, learn more about it now: https://go.eurordis.org/ECRDprogramme

That’s a wrap on the 2026 EURORDIS Open Academy x Erdera Schools’ in-person training week! 🎬

After six months of learning, collaboration, and exchange, more than 70 patient advocates, early-career researchers, and community leaders from across Europe came together in Barcelona this week for the culmination of their journey with the EURORDIS Open Academy.

👏 Congratulations to all participants on completing their trainings. We look forward to welcoming you to our active alumni community and to seeing the impact you will continue to make going forward!

Whether you are completely new to the Open Academy or are already an alumni of our schools, we have a wealth of opportunities to help you progress in your rare disease advocacy journey. 👉 Learn more about the Open Academy and how you can get involved: https://openacademy.eurordis.org

⏳ is less than one week away! As the EU Health Technology Assessment Regulation (HTAR) begins reshaping how medicines are assessed across Europe, one key question is becoming increasingly urgent: how can the system effectively respond to the realities of rare diseases?

At , session one of the Preparing Reimbursement Decisions track will provide an overview of the HTAR and reflect on the first 18 months of implementation.

The discussion will explore experiences with joint clinical assessments (JCAs), joint scientific consultations (JSCs), and European reports, while examining both the progress achieved and the challenges that remain, particularly for orphan and advanced therapy medicinal products (ATMPs).

📆 Thursday 4 June 2026 | 09:00–10:30

⏰Time is running out to join the discussions online! Register now: https://go.eurordis.org/ECRDprogramme

Rare disease progress cannot stop at diagnosis and treatment.

Because living with a rare disease is also about mental health.
About education and employment.
About social participation, disability recognition, and access to coordinated support.

Yet across Europe, too many people and families are still left navigating fragmented systems that fail to address the full reality of living with a rare condition.

👉 As approaches, these challenges will take centre stage in Prague on 3–4 June through dedicated discussions on:
🔹 Evidence-Based Holistic Care
🔹 Mental Health
🔹 Specialised Healthcare Access

Drawing on the latest EURORDIS Rare Barometer findings, our new article explores why Europe must move towards holistic, evidence-based care that goes beyond the medical dimension alone.

These are exactly the conversations that will bring to the forefront.

📰 Read the full article: https://go.eurordis.org/beyondcare

Lift-off for the EURORDIS Open Academy x ERDERA Schools' in-person training week! 🚀

This week, we are bringing together 70+ patient advocates, researchers, and community leaders from across Europe for the culmination of their six-month learning journey with the EURORDIS Open Academy.

Through shared experiences, capacity building, and meaningful discussions, participants will deepen their knowledge and strengthen the skills needed to engage as empowered and equal partners in rare disease medicines development and scientific research.

Thank you to our Erdera partners involved in making this training week happen and to all participants bringing their energy and commitment to the table!

👀 Stay tuned to learn more about what our trainees will be getting up to throughout the week, including inspiring site visits to see theory put into action, ethical debates, and advocacy simulation exercises!

👉 Learn more about the Open Academy and how you can get involved: https://openacademy.eurordis.org

Behind every delayed diagnosis, long journey to a specialist centre, or unmet healthcare need, there is a person living with a rare disease trying to access the care they need at the time that they need it.

At , the second session of the Specialised Healthcare track will explore how health systems can better measure access to specialised healthcare for people living with a rare or complex condition.

It will examine which indicators and metrics can help policymakers understand where gaps remain, assess the scale of unmet needs, and evaluate the impact of care pathways, Centres of Expertise, and cross-border healthcare services.

By linking structural indicators with performance, outcomes, and patient experiences, the session aims to support more actionable evidence and equitable access to specialised care for people living with rare diseases.

📆 Thursday 4 June 2026 | 14:00 – 15:30

🔗 Registration to attend online is still open! Don’t miss the opportunity to join over 700 people already signed up: https://go.eurordis.org/ecrd-fb

Over the course of the first edition of our Open Academy x ERDERA Mentoring Programme 20 Open Academy alumni spent four months in small mentoring groups being guided by experienced mentors, working through real world challenges together, strengthening their leadership skills, and turning ideas into concrete action.

In this new article, Marta Campabadal, our Open Academy Senior Manager, shares key lessons from the pilot edition and her thoughts on why structured, peer-based support is not only valuable, but a vital necessity in the rare disease community.

👉 Read now: https://go.eurordis.org/blogstaff

Looking ahead, the 2026-2027 edition of the programme will expand to six months, allowing for deeper engagement and more tailored mentor-mentee connections.

The call for mentors is now open to professionals and experienced patient advocates looking to share their experience!

📌 Become a mentor: https://go.eurordis.org/mentorcall

Today is Clinical Trials Day! 💊

For people living with rare diseases, participating in a clinical trial can still mean facing major obstacles: limited trial availability, complex enrolment processes, fragmented data, and systems not built for small patient populations.

💉On this , it is important to recognise that accelerating innovation also means transforming the clinical trial ecosystem itself.

In her latest blog article, Rita Francisco, our Patient Engagement Manager, explores how the RealiseD project is working to reshape rare disease clinical trials through innovative trial designs, improved enrolment strategies, patient engagement, and multi-stakeholder collaboration.

Read the full article and learn more about it: https://go.eurordis.org/yIi4Mw