World Orphan Drug Congress USA 2026

Join America’s leading rare disease and orphan drug event in Boston!

The World Orphan Drug Congress is an award-winning series of conferences and exhibitions that have grown to become the largest and most established rare disease meetings of its kind across the globe. It offers a unique opportunity to explore cutting-edge developments, forge partnerships, and drive progress in rare disease therapy innovation.

The 2026 USA Congress will host 2,000+ attendees, 280+ speakers, and 100+ exhibitors bringing together global leaders, scientists, regulators, and patient advocates across the entire orphan drug and rare disease landscape.

With key tracks focused on next-generation therapies (Cell & Gene), clinical development for small patient populations, regulatory pathways, global market access & pricing, patient-centricity, and Real-World Evidence (RWE), this event covers the full spectrum of global orphan drug efforts across the whole rare disease value chain. Attendees will dive deep into topics including strategies to shorten the diagnostic odyssey, breakthroughs in innovative gene therapies, and ensuring equitable access for rare disease patients globally.

The event features expert speakers, interactive networking opportunities, an exhibition showcasing the latest innovations, and workshops offering hands-on learning experiences. Whether you’re looking to stay at the forefront of rare disease innovation or build meaningful connections, this is the must-attend event for professionals in the field.

Don’t miss out on this opportunity to be part of the rare disease revolution!

Check if you are eligible for a free ticket on our registration page, otherwise use the discount code below.

*Use discount code MLSC20 for 20% off your ticket.*