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ERDERA General Assembly sets priorities for an integrated and delivery-focused third year

ERDERA’s third General Assembly brought partners together in Coimbra to review its second year, strengthen connections across the partnership and align priorities for a more integrated and delivery-focused third year.
ERDERA 2026 Open Session

From individual cases to new treatment pathways: ERDERA Open Session focuses on ultra-rare diseases

ERDERA marked the close of its second year with an Open Session on ultra-rare diseases, bringing together European, regulatory and scientific perspectives on how innovation can reach patients.
Training online

Information Webinar – ERDERA Joint Transnational Call 2027

December 14 @ 3:00 pm - 4:00 pm
This information webinar on the 2027 Joint Transnational Call for Proposals is scheduled for 14 December 2026.
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ERDERA launches Ethics & Regulatory webinar series with introductory webinar on ethics across the research continuum

ERDERA’s ethics and regulatory experts are launching a dedicated webinar series to help the rare disease research community navigate two fast-moving areas of biomedical research: Advanced Therapy Medicinal Products (ATMPs) and Artificial Intelligence.
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EMA launches consultation on proof-of-concept data in paediatric oncology

This initiative is also highly relevant from a rare diseases perspective, as paediatric cancers are rare conditions where small patient populations make robust non‑clinical proof‑of‑concept data essential for responsible and ethical trial initiation.
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Towards animal-free testing: virtual control groups gain regulatory acceptance

For rare diseases, where research often relies on limited animal models and small development teams, the regulatory acceptance of virtual control groups could help streamline early non‑clinical studies while maintaining a strong focus on patient safety.
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Advancement of Treatments for Rare Diseases

June 16 - June 17
The two-day event will focus on translating research and policy initiatives into tangible benefits for Rare Disease patients through better prevention, diagnosis, and treatment.
Publicaciones (1)

EU rare disease action plan- European Added Value Assessment

This study investigates possible measures that could be taken at EU level to address these challenges. It finds significant European added value in harmonising coordination and access across the 27 Member States, mainly in terms of improved diagnostic tools and availability of medical treatment, better health outcomes, particularly lower infant mortality, and improved well-being of family members and caregivers.
Medicines

EMA starts review of Tavneos for rare autoimmune diseases GPA and MPA

The European Medicines Agency has begun a formal review of Tavneos (avacopan) after emerging information raised questions about the integrity of key clinical trial data supporting its EU authorisation, with potential implications for adults living with rare autoimmune vasculitis.
Young Patients Training

ERDERA opens call for young rare disease advocates to join training in Paris

Taking place on 23–25 April at Institut Imagine in Paris
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