Empowering Patients And Tackling Orphan Drug Development Challenges

Part of Novotech’s “Navigating Regulatory Changes & Market Dynamics” Series

In this episode, industry experts from Novotech, Avidity Biosciences, Leap Cure, and the patient advocacy community examine how collaborative, patient-focused approaches are redefining orphan drug development.

Moderated by Novotech’s Scott Schliebner, VP, Drug Development Consulting, and featuring Michal Meller, Program Management Director, the discussion explores:

• The real-world burden of rare disease trial participation
• Strategies to embed the patient voice into protocol design
• The importance of site readiness and patient concierge models
• Balancing regulatory rigor with day-to-day feasibility
• The value of early and consistent engagement with patients, investigators, and regulators