Medical devices for rare diseases are becoming increasingly important as healthcare innovation expands beyond orphan drugs and biologics. Emerging technologies such as digital remote monitoring systems are creating new opportunities for prevention, diagnosis, treatment, and long-term disease management. Despite these advances, only a small percentage of rare diseases currently have approved orphan devices. This growing […]
Read More
We are pleased to share a new publication produced by the IRDiRC Functional Analysis Task Force, highlighting the growing impact of multi-omics in advancing rare disease research and precision medicine. Multi-omics, combined with advanced computational methodologies, integrates diverse omics datasets to deliver deeper insights into molecular interactions. This powerful approach has transformative potential for unraveling […]
Read More
The third Hope for Rare Science Conference will be held in Shanghai, China from June 25th to 27th, 2026. The conference is hosted by Hope for Rare Foundation, with National Children’s Medical Center/ Children’s Hospital of Fudan University and Chinese Organization for Rare Disorders (CORD) as co-hosts. The conference will last for 3 days, featuring […]
Read More
IRDiRC is delighted to be represented by multiple members at the Undiagnosed Day 2026 taking place in Gdansk (Poland) and online. Bringing together specialist clinicians, clinical geneticists, and invited experts, this event aims to support phenotype-led diagnosis through live case discussions, shared clinical reasoning, and practical exchange. Inspired by the collaborative spirit of international Undiagnosed […]
Read More
On Rare Disease Day 2026, International Rare Diseases Research Consortium reaffirms its unwavering commitment to advancing research for the 300 million people and families living with rare diseases worldwide. With more than 65 member organizations — including research funders, companies, patient advocacy groups, and scientific leaders — IRDiRC continues to catalyse global progress through collaboration, innovation, and […]
Read More
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 […]
Read More
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 […]
Read MoreRegulatory 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 […]
Read More
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 […]
Read More