Advancing evidence generation for rare diseases and orphan drug development
27–29 October 2026 | RAI Amsterdam, The Netherlands
OXON Epidemiology will be attending World Orphan Drug Congress Europe 2026, a leading international event bringing together pharmaceutical and biotechnology companies, researchers, patient organisations and other stakeholders to advance rare disease research and orphan drug development.
Rare disease research presents unique challenges, including small patient populations, limited natural history data and difficulties establishing appropriate comparators. Robust Real-World Evidence (RWE) and innovative study designs are essential to address these challenges and support regulatory, health technology assessment (HTA) and market access decisions.
Supporting Rare Disease Research with Real-World Evidence
At OXON, we work with pharmaceutical and biotechnology companies to design and deliver scientifically rigorous studies that address the complex evidence needs of rare diseases and orphan therapies.
Our expertise encompasses natural history studies, patient registries, external control arms, primary and secondary observational studies, and advanced biostatistical methodologies, supported by integrated epidemiology, data management and clinical operations capabilities.
By combining clinical trial expertise with real-world data, we help generate reliable evidence to support orphan drug development, regulatory submissions and market access strategies.
Poster presentation at WODC Europe 2026
Nawab Qizilbash, OXON’s Chief Medical Officer, will present “Beyond Approval: Designing External Controls for Orphan Drug Development and Market Access.” The poster explores how early external comparator planning can strengthen evidence generation for orphan drug approval and market access, drawing on three real-world examples.
Poster presentation sessions:
- 27 October | 13:40–14:10
- 28 October | 10:30–11:15 and 13:15–13:45
Let’s Meet at WODC Europe 2026
Our team will be available at booth #319 throughout the congress to discuss your rare disease research objectives, evidence generation strategies and opportunities for collaboration. Whether you are planning a natural history study, exploring external comparators or looking for an experienced CRO partner, we would be delighted to connect.
