What’s Inside?

To acknowledge Rare Disease Day and the struggles facing patients with rare diseases, the World Orphan Drug Congress hosted a webinar titled, “How can AI impact industry?” The session, which explores the role of real-world evidence (RWE) to improve orphan drug development and access, brought together leaders in their field with deep knowledge of rare diseases and the importance of RWE in helping to identify the right solutions for the right patient.

• Rare Disease Differences and Commonalities
• Building a Development Plan with RWE at the Centre
• The Role of the Regulator
• Engagement, consent, and privacy
• Creating a shared purpose
• Overcoming data sharing issues
• The promise of AI from a patient perspective