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 […]
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The 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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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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IRDiRC Chair and members, Dr. David Pearce, Dr. Gareth Baynam, Helene Cederroth and Mikk Cederroth have co-authored a new publication on the importance of the Undiagnosed Hackathon in finding new ways to diagnose individuals living with a longstanding medical conditions without a diagnosis. 350 million people live currently with an undiagnosed disease worldwide. While genome […]
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Cerebrum DAO is a community-powered network organization dedicated to sourcing, funding, and translating research that advances solutions to extend healthy brain lifespan. Their current research priorities include: Cerebrum DAO offers project funding of up to $200.000, including overhead. To learn more or apply for funding, visit cerebrumdao.com/researchers or contact Michele Gallia at michele@cerebrumdao.com.
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IRDiRC has four Scientific Committees: Diagnostics, Therapies, Interdisciplinary, Regulatory. The Interdisciplinary Scientific Committee (ISC) is a multi-stakeholder, multi-disciplinary group of experts working on rare disease (RD) data sharing, ontologies, natural history, biobanks, registries, ethical and societal aspects of rare diseases. ISC would like to broaden the committee membership, to represent the worldwide rare diseases community in its […]
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