A global community of clinicians, patient leaders, regulators and researchers met in Heidelberg (Germany) and online for the second International Conference on Clinical Research Networks (CRNs), organised by the European Rare Diseases Research Alliance (ERDERA), Rare Diseases International (RDI) and the International Rare Diseases Research Consortium (IRDiRC). CRNs are structured collaborations of expert sites, laboratories, […]
Read MoreIRDiRC is pleased to announce that COMBINEDBrain, represented by Terry Jo Bichell (CEO), has joined the IRDiRC Patient Advocacy Constituent Committee (PACC). The Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders is a non-profit organization devoted to speeding the path to clinical treatments for people with rare genetic neurodevelopmental disorders by pooling efforts, studies, and data. […]
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Want to stay informed about the latest breakthroughs, global initiatives, and collaborative efforts shaping the future of rare disease research? Join the International Rare Diseases Research Consortium (IRDiRC) community by subscribing to our newsletter. Why Subscribe? By signing up, you’ll receive: Who Is It For? Researchers, clinicians, industry partners, patient advocates, regulators, policymakers, and anyone committed to […]
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A new analysis of orphan drug approvals across six global regulatory regions highlights significant delays in access to innovative therapies for people living with rare diseases. The study, conducted by the International Rare Diseases Research Consortium (IRDiRC) Regulatory Science Committee (RSC), examined all new non-oncology orphan medicines approved between 2021 and 2022 and found substantial […]
Read MoreThe International Rare Diseases Research Consortium (IRDiRC) is pleased to announce the appointment of Shekhar Natarajan (Vice President, Head of EU and International Regulatory Affairs at Dyne Therapeutics, USA), as Chair, and Diana Kwast-Hoekstra (Executive Director at (Un)limited Forward!, Netherlands), as Vice Chair of the Therapies Scientific Committee (TSC). The TSC unites a diverse group of stakeholders from across the global […]
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Regulatory sandboxes are emerging as a powerful tool to accelerate innovation in healthcare, and their potential in the rare disease space is especially promising as they offer flexible, structured environments where novel approaches to regulation can be safely tested and refined. This topic was part of the special session at the IRDiRC Consortium Assembly and […]
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IRDiRC is pleased to share a new publication “What Matters Ethically About How the UDN Has Changed Since Its Inception”, recently published in AMA Journal of Ethics. For individuals living with undiagnosed rare diseases, the search for a diagnosis can be long, uncertain, and emotionally burdensome. A delayed diagnosis, or none at all, can significantly add […]
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IRDiRC announces publication of a new manuscript, developed as part of the International Rare Disease Research Consortium (IRDiRC) N-of-1 Task Force: “Applying the IRDiRC N-of-1 Therapy Task Force Eligibility Criteria for Individualised Therapies – Use Case: Duchenne Muscular Dystrophy“. Nucleic acid-based therapies, including antisense oligonucleotides (ASOs), are opening new doors for the treatment of rare diseases. With over […]
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The 2nd International Conference on Clinical Research Networks for Rare Diseases will take place on 9–10 December 2025 in Heidelberg, Germany, with full hybrid access for participants worldwide. This edition features a distinctly hands-on format, placing delivery at the centre. Each session —plenary and workshops alike— will close with agreed action points that will be captured live […]
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The International Rare Diseases Research Consortium (IRDiRC) is pleased to participate in this year’s World Orphan Drug Congress Europe, taking place from 27–29 October 2025 in Amsterdam, Netherlands. We warmly invite all attendees to visit the IRDiRC booth (10.510) to learn more about our mission, collaborative initiatives, and how we are accelerating the development of diagnostics and therapies for people living with […]
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