Rare Disease Innovation: Key Insights from the World Orphan Drug Congress USA 2026 Agenda

The World Orphan Drug Congress USA convenes June 9–11, 2026, at the Thomas M. Menino Convention & Exhibition Center in Boston, Massachusetts. This hybrid-format gathering centers on rare disease innovation.

Key themes on June 10 include integrating patient perspectives into policy and development, aligning global regulatory approaches, and exploring artificial intelligence for diagnostics and trial optimization.

Organizers aim to foster cross-sector collaboration, address access barriers, and accelerate individualized treatment pathways through workshops, keynotes, track sessions, and interactive working groups.

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Diverse Programming Across Policy, Technology, and Patient Engagement

Morning keynotes examine patient-centered policy frameworks and feature a sponsored panel on global regulatory alignment, real-world data, and next-generation trial methodologies.

Afternoon tracks cover these specialized areas:

• Expanded Access Programs (EAP)
• Advanced therapies
• Rare advocacy
• AI and digital health
• Clinical development and regulatory affairs
• Commercialization
• Innovation showcases

Sessions address telehealth in rare disease care, clinical trial recruitment, commercial insurer coverage policies, N-of-1 treatments, caregiver perspectives, and direct-to-patient models.

Multiple innovation showcases will present candidates including DDC-02 for neurodevelopmental disorders and leriglitazone for X-linked adrenoleukodystrophy.

Interactive working groups will explore genetic counseling integration, authentic patient storytelling in AI-driven media, claims data reliability, and evidence generation for individualized medicines.

Leadership Voices Shaping Rare Disease Discourse

Prominent participants on June 10 include:

• Pamela Gavin, CEO of the National Organization for Rare Disorders (NORD)
• Wendy Erler, Sarepta Therapeutics
• Annie Kennedy, EveryLife Foundation for Rare Diseases
• Amy Comstock Rick, FDA’s Rare Disease Innovation Hub

Additional speakers and moderators include Rachel Smith of Parexel, Dave Pearce of IRDiRC, and representatives from patient advocacy organizations, academic centers, and biotech firms such as n-Lorem Foundation and Boston Children’s Hospital.

Expertise spans regulatory science, patient affairs, clinical operations, and digital health, blending advocacy leadership with technical and commercial perspectives.

Charting Pathways for Sustainable Rare Disease Progress

The day emphasizes co-creating policy frameworks, harmonizing international standards, and deploying AI-enabled tools for patient identification and trial design.

Discussions on expanded access, caregiver integration, and commercialization models suggest sustained progress depends on strengthened multistakeholder partnerships and adaptive regulatory navigation.

By linking real-world patient experiences with evolving trial methodologies and evidence strategies, the agenda equips participants to drive actionable improvements in rare disease innovation and access.