Post by Parexel

One theme emerging across discussions at World Orphan Drug Congress this week: innovation alone isn’t enough. Bringing therapies to patients with rare diseases increasingly requires global alignment across regulatory pathways, evidence strategies, patient engagement and operational execution. During today's panel discussion on 𝘎𝘭𝘰𝘣𝘢𝘭 𝘈𝘭𝘪𝘨𝘯𝘮𝘦𝘯𝘵 𝘪𝘯 𝘙𝘢𝘳𝘦 𝘋𝘪𝘴𝘦𝘢𝘴𝘦 𝘋𝘳𝘶𝘨 𝘋𝘦𝘷𝘦𝘭𝘰𝘱𝘮𝘦𝘯𝘵, Rachel Smith, Vice President, Therapeutic Area Lead, Rare and Genetic Diseases, and fellow industry leaders discussed how greater alignment across regulators, sponsors, sites, and patient communities can accelerate development for small and geographically dispersed populations. The panel explored how sponsors can navigate evolving regulatory expectations, regional differences and development strategies while accelerating patient access. As rare disease research advances, the challenge is no longer whether innovation is possible, but how to translate scientific breakthroughs into development programs that efficiently and equitably reach patients worldwide. Thank you to everyone who joined the discussion. If you're attending WODC USA, visit us at booth #213 to continue the conversation. #WODC #WODC2026 #RareDisease #OrphanDrugDevelopment