The International Rare Diseases Research Consortium (IRDiRC) participated in the 2026 edition of World Orphan Drug Congress (WODC) USA in Boston (MA, USA), contributing to discussions focused on accelerating innovation and improving outcomes for people living with rare diseases.
Across multiple sessions, IRDiRC constituent and task force members highlighted progress and emerging strategies in advanced therapies, prevention, sustainability, and integrated care pathways.
The fireside chat on “Where is N-of-1 Going?“, was moderated by Dr. David Pearce, Chair of IRDiRC, and explored the evolving landscape of N-of-1 therapies for ultra-rare diseases, together with Tim Yu (Boston Children’s Hospital) and Sarah Glass (n-Lorem Foundation). The session examined opportunities and challenges in delivering personalised therapies at scale, with discussion centred on scientific progress, implementation pathways, and the future potential of tailored treatments for patients with ultra-rare conditions. Jon Brudvig (Amicus Therapeutics), presented the IRDiRC Task Force: Facilitating the Development of Preventative Medicines for Rare Diseases, highlighting how clinical development strategies and regulatory innovation can help accelerate access to preventative therapies, while enabling earlier intervention and improved long-term outcomes. Dr. David Pearce also moderated the panel discussion Beyond N-of-1: Sustainability of Genetic Therapies for Rare Diseases, with speakers P.J. Brooks (NIH/NCATS), Alexander Natz (EUCOPE – European Confederation of Pharmaceutical Entrepreneurs) and Karen Kahn (ForeBatten Foundation). Lisa Emrick (Baylor College of Medicine), presented the IRDiRC Task Force: Bridging Diagnostics to Care for Rare Diseases, emphasising the importance of integrated pathways that move beyond diagnosis to ensure access to treatment, coordinated services, and comprehensive patient support across the care journey.
IRDiRC’s participation at WODC USA 2026 reflected the consortium’s continued commitment to fostering international collaboration and advancing solutions that accelerate diagnosis, improve treatment options, and enhance outcomes for people living with rare diseases worldwide.