ERDERA has received funding from the European Union’s Horizon Europe research and innovation programme under grant agreement N°101156595.
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© 2025 | ERDERA — European Rare Diseases Research Alliance
ERDERA is coordinated by Insem
A conference entitled “Towards an EU Action Plan on Rare Diseases” will be held on 10 April 2025 at the Medical University of Warsaw, Poland, under the auspices of the Polish Presidency of the EU Council. Organised by the Polish Ministry of Health, the European Economic and Social Committee (EESC), and the Medical University of Warsaw, the event will mark an important step in shaping future European policy on rare diseases. This high-level meeting will bring together policymakers, researchers, clinicians, and patient representatives to discuss key priorities for a European action plan. With national strategies evolving, the focus now shifts READ MORE
On 27–28 March, ERDERA will participate in the 5th International Conference on Rare Diseases, held in Athens, Greece. ERDERA’s Scientific Coordinator, Daria Julkowska, will be among the esteemed speakers at this event, organised by Rare Diseases Greece (RDG), EURORDIS-Rare Diseases Europe, and Boussias Events, with the support of ERDERA. This conference will focus on the theme: “Sustainability in Action: Rethinking the Rare Disease Paradigm from Prevention to HTA and Access.”
ERDERA coordination participated in the Chan Zuckerberg Initiative’s “Building a Roadmap to Systematically Characterize Rare Diseases” workshop, taking place March 24–26 at CZI Headquarters in Redwood City, California.
The RE(ACT) Congress & IRDiRC Conference 2025, an international platform for knowledge sharing on rare and orphan diseases, has been a catalyst for scientific collaboration and advancement since its inception in 2012.
The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) have jointly issued a clear warning about unregulated Advanced Therapy Medicinal Products (ATMPs). While these therapies—based on genes, tissues or cells—offer genuine promise, its limited regulation procedures raise serious health and safety concerns. Authorities urge patients to confirm that any ATMP is approved through official channels to avoid potential risks.
The European Medicines Agency (EMA), together with the European Medicines Regulatory Network, has issued a renewed call for registrations in their Real-World Data (RWD) Catalogues, marking one year since the initiative’s launch. This publicly accessible online resource replaced the previous ENCePP Resources Database and the EU PAS Register, consolidating metadata on RWD sources and studies READ MORE
The 8th RE(ACT) Congress and the 6th IRDiRC (International Rare Diseases Research Consortium) Conference concluded today, marking the end of a three-day summit where scientists, patient advocates, funders, and policymakers tackled the rare disease field’s most pressing challenges. READ MORE
As Rare Disease Day 2025 comes to a close, its impact continues to resonate across Europe and beyond. Held annually on the last day of February, this year’s event drew critical attention to the 30 million people in Europe living with a rare disease, underscoring the urgent need for faster diagnoses, increased research, improved treatments, READ MORE
On 28 February, coinciding with the 2025 Rare Disease Day, Parliament’s public health committee, SANT, launched an online survey to gather insights on rare diseases. The initiative aims to build a detailed understanding of the challenges faced by individuals affected by rare diseases, as well as to collect views from stakeholders—including patients, caregivers, industry representatives, READ MORE
Imagine having a disease so rare that doctors struggle to diagnose it, researchers have little data to study it, and treatments are scarce. For the 30 million people in Europe living with a rare disease, this is a daily reality. But change is happening. READ MORE
The landscape of clinical research and genomic data governance is evolving rapidly, with two significant developments shaping the future of global health research and regulatory frameworks: the ongoing revision of the ICH E6 (R3) Good Clinical Practice (GCP) guideline and the recent release of WHO guidance on the ethical collection, access, use, and sharing of READ MORE
We are pleased to announce that the platform for submitting project applications for the Joint Transnational Call for Proposals (JTC) 2025 is now officially live. This highly anticipated call invites research teams across Europe and internationally to collaborate on cutting-edge projects aimed at “Pre-clinical therapy studies for rare diseases using small molecules and biologicals – READ MORE
For decades, rare disease patients and their families have endured a painful “diagnostic odyssey,” bouncing between specialists and often going years without answers. Rare diseases, by definition, affect fewer than 5 in 10,000 people in the European Union, but with over 7,000 such conditions cataloged, their collective impact is profound. A staggering 70% of these READ MORE
