2nd European Congress on Rare Diseases and Orphan Drugs

The Rare Diseases Forum 2026 will take place in Boston, Massachusetts, bringing together professionals involved in the development, regulation, commercialization, and delivery of therapies for rare diseases. The programme explores the evolving orphan drug landscape, with discussions covering regulatory strategy, clinical development, patient recruitment, real-world evidence, market access, pricing and reimbursement, and patient engagement throughout the product lifecycle.

Sessions examine the challenges of bringing rare disease therapies from research and clinical development to commercialization and patient access. Additional topics include advanced therapies, cell and gene therapies, evidence generation, healthcare policy, value assessment, payer perspectives, and strategies for addressing unmet medical needs in small patient populations. Through case studies, panel discussions, and industry-led presentations, the forum highlights current trends and practical approaches across the rare disease ecosystem.