World Orphan Drug Congress USA, Boston
The World Orphan Drug Congress USA has been a premier annual gathering for the rare disease and orphan drug sector, bringing together pharmaceutical leaders, biotech innovators, patient advocates, and regulators to accelerate access to life-saving therapies.
Key 2026 Event Details
The most recent edition of the congress was held from June 9–11, 2026, at the Thomas M. Menino Convention and Exhibition Center in Boston. As America’s largest event dedicated to orphan drugs, it convened stakeholders to “break barriers, foster collaboration, and improve access to life-saving therapies.”
Major Themes and Sessions
The 2026 agenda centered on three critical pillars: market access, patient partnership, and evidence generation.
- Access and Pricing Strategy: Sessions addressed the complex global pricing environment. A workshop explored how Most Favoured Nation drug pricing and International Reference Pricing could reshape European launch sequencing and influence US commercial strategy. Additional panels tackled “Coverage That Keeps Up With Innovation,” “Barriers to Access,” and coverage patterns for rare disease therapies in US commercial insurance.
- Patient Partnership: The congress went beyond patient centricity toward comprehensive partnership. Sessions explored integrating patient perspectives into regulatory engagement, clinical trial design, and evidence generation. A dedicated Patient Zone served as a “Rare Community Hub,” featuring organizations including CheckRare, RTW Foundation, and Pathways to Trust, providing spaces for advocacy groups, patients, and industry to connect.
- Global Perspectives: Panels examined HTA and market-driven pricing across Europe and the US, with sessions on the UK, Ireland, and EU access pathways, featuring experts from NICE and the Amyloidosis Research Consortium.
Event Status for 2027
The 2027 edition of the World Orphan Drug Congress USA was initially scheduled for May 10–12, 2027, again in Boston. However, organizers have since announced that the World Orphan Drug Congress USA will no longer be running in 2027. This decision, described as difficult, comes after over a decade of the event serving as a central force for the rare disease ecosystem.
In its place, a new event, the World Evidence Pricing & Access Congress USA, is scheduled for September 14–15, 2027, in Boston for those with existing passes.