World Orphan Drug Congress USA 2026

America's Largest Orphan Drug & Rare Disease Event. The World Orphan Drug Congress USA brings together leading pharmaceutical and biotechnology companies, government and regulators, patient advocates, payers, investors, and solution providers to brainstorm ways to advance Orphan Drug development and improve access to life-saving therapies for rare disease patients. Over 3 days, attendees will hear from 300+ speakers across 16 themes of content, engage in networking opportunities with over 2,000+ rare disease attendees, and meet with over 100+ exhibitors. The event focuses on breaking barriers and fostering collaboration within the rare disease community, exploring the industry’s latest innovations and technologies, and providing keynote-driven content. Key themes include patient data, next generation therapies, payers, clinical development & regulatory, commercial strategies, rare disease advocacy, global market & patient access, pricing & reimbursement, pitch & partner opportunities, advanced therapies manufacturing, clinical development and commercialization, rare oncology, digital health & artificial intelligence, diagnosis, and discovery. The event also addresses challenges such as access to treatments, regulatory complexities, lack of biomarkers and natural history data, and new legislation impacting orphan drugs.