The World Orphan Drug Congress 2025 brought together leading voices in the rare disease community for 3 days of insight, collaboration, and forward-looking discussion. Representatives from the International Rare Diseases Research Consortium (IRDiRC) shared key perspectives on advancing research and improving access to therapies for people living with rare conditions.
A highlight of the congress, which took place in Amsterdam (Netherlands) last week, was the workshop on “Public–Private Partnerships in Rare Disease: Ideas for Action”, organized by IRDiRC Companies Constituent Committee member Sanofi, represented by Vinciane Pirard. The session showcased successful cross-sector collaborations, practical tools for sustainable partnerships, and innovative initiatives transforming the rare disease ecosystem. By promoting open dialogue and shared expertise, participants worked to translate ideas into tangible strategies that improve outcomes for patients.
The morning of the first day began with a keynote panel exploring “What the future holds for Europe’s Life Science Strategy and what it means for rare diseases” where Christina Kyriakopoulou, Policy Officer at the European Commission and member of the IRDiRC Funders Constituent Committee, highlighted the role of policy alignment and strategic investment in driving rare disease innovation.
In the afternoon, Stefano Benvenuti of Fondazione Telethon (member of FCC) presented a not-for-profit model demonstrating how research can be effectively translated into therapies for ultra-rare conditions. Samantha Parker, Patient Engagement Lead at Italfarmaco (member of CCC) and IRDiRC Vice Chair, discussed the challenges and probabilities of success in orphan drug development, emphasizing the importance of patient involvement in shaping research priorities.
Daniel O’Connor, Director of Regulatory Policy & Early Access at the ABPI and Chair of IRDiRC’s Therapies Scientific Committee (TSC), led a session on the growing focus on prevention, outlining opportunities and barriers in shifting from treatment to early intervention. Later, Anneliene Jonker, Vice Chair of TSC, and Director at Duchenne Parent Project, encouraged greater engagement of young researchers in rare disease science, presenting a roadmap for future action.
The second day of the World Orphan Drug Congress 2025 focused on the evolving landscape of rare disease innovation, opening with the plenary session “New treatments on the block: 2025’s rare disease pipeline.” Experts examined the latest advances in gene and cell therapies, the policy frameworks needed to support new approvals, and strategies to ensure the long-term sustainability of rare disease drug development. IRDiRC members Violeta Stoyanova-Beninska (Chair of Regulatory Scientific Committee, European Medicines Agency) and Daniel O’Connor (ABPI) shared their insights on regulatory science and access, underscoring IRDiRC’s active role in shaping global approaches to rare disease research and innovation.
Visitors were invited to stop by IRDiRC’s Booth to learn more about the Consortium’s initiatives and mission to accelerate progress in rare disease research.
The congress concluded with a clear message: through collaboration, innovation, and collective commitment, the global rare disease community continues to build momentum toward a healthier future for all.