Episode Description
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.