September 26, 2024

   

15:00 CET

Empowering Patients & Challenges in Orphan Drug Development

Join us for an insightful webinar on orphan drug development, where industry experts will share practical examples and key strategies for overcoming the unique challenges in rare disease research. This session will delve into patient-centered clinical trials, emphasizing the importance of reducing patient burden and enhancing engagement through innovative trial designs.

The discussion will center around these key elements:

  • Orphan drug market: Illustrate the unique challenges of developing a drug for rare diseases.
  • Patient-focused drug development: Designing patient-centered clinical trials.
  • Operational Considerations: Endpoint selection; reducing the burden of participation; cost; sites; patient concierge services.
  • Collaboration: Focus on the need for collaboration in this space: sponsors, CRO (Clinical Research Organizations), patients, patient advocacy groups, investigators, vendors.

The event will be moderated by Scott Schliebner (Vice President - Drug Development Consulting - Novotech)

Panelists:

Afshawn Chakamian – (Director of Program Management at Avidity Biosciences)

Lauren Dahl (Patient Success at Leap Cure)

Michal Meller (PM Director at Novotech)

Ella Balasa (patient, consultant at Patient Advocacy)