The World Orphan Drug Congress (WODC) Europe returns to Amsterdam from 27 to 29 October 2025 at the RAI Congress Centre. Now less than a month away, the event is recognised as one of the most significant international meetings for orphan drugs and rare diseases, offering a platform for critical discussion, exchange of knowledge and cross-sector collaboration.
On Monday 27 October at 10:00, ERDERA, RealiseD and INVENTS will co-host an interactive workshop entitled “Innovative methodological approaches accelerating rare disease drug development: bridging data, innovative design, and decision-making.” The session will address current methodological and operational challenges in rare and ultra-rare disease research, from limited patient populations and disease variability to the identification of clinically meaningful endpoints.
The workshop will explore how collaborative initiatives across Europe are working to optimise trial design for rare conditions, use diverse data sources including Real World Data, streamline evidence generation, and ensure alignment with regulatory and HTA expectations. The discussion will also highlight approaches to ensuring patient perspectives remain central.
The session will be chaired by Daria Julkowska, who will guide participants through case examples and methodological insights demonstrating how joint efforts by ERDERA, RealiseD and INVENTS are shaping new standards in rare disease research. Conference delegates will require a valid pass to attend.
The Congress, which brings together leading voices from research, industry, policy and patient advocacy, continues to serve as a point of reference for advancing therapeutic development for rare and ultra-rare diseases. The full agenda is available via the World Orphan Drug Congress Europe 2025 programme.
Those research and policy-making representatives registering may wish to note that a discount option has been made available through the code ERDERA20, while patient group organisations, ERNs, charities, public health officials, and individuals working at selected pharmaceuticals/biotechs may apply for a complimentary guest pass here.
