Session (CTI)

Rare disease products face distinct hurdles in the evolving landscape of EU Joint Clinical Assessment (JCA). From limited data sets to complex endpoints, navigating these challenges requires a tailored and strategic approach. In this focused 30-minute session, our expert panel will break down the journey into three actionable segments: Rare Disease-Specific Challenges Understand the unique barriers orphan drug developers encounter in the JCA process, including evidence limitations and stakeholder expectations. Five Methodological Steps to Success Explore a proven framework for overcoming these challenges—from PICO prediction to submission development—designed to bring clarity and structure to your JCA strategy. Key Learnings from the Field Gain insights from real-world case studies that illustrate how these steps have been successfully applied to rare disease products across the EU. Whether you're preparing for your first JCA submission or refining your current strategy, this session will equip you with practical tools and expert guidance to move forward with confidence.

Sponsored by RTI Health Solutions