Navigating Novel Ophthalmic Endpoint Development & Regulatory Validation to Accelerate Disease Therapeutic Approvals
Time: 10:00 am
day: Pre-Conference Workshop Day
Details:
Novel endpoint development and validation are major challenges in ophthalmic gene therapy, with regulatory acceptance crucial for approval. Traditional vision measures often fall short in rare, slow-progressing diseases, while patient-reported outcomes add variability. Join us in this workshop to identify effective endpoints and regulatory strategies to increase regulatory approval.
Key Discussion Points:
- Navigating FDA’s RDEA pilot program and building compelling cases for novel endpoint acceptance through strategic regulatory engagement
- Implementing long-term secondary endpoint collection strategies in early-phase studies to establish pivotal trial readiness over 3-4-year timelines
- Eliminating confounding variables in vision testing by distinguishing true visual function from alertness, fatigue, and other behavioral factors that can compromise trial validity
- Advancing objective biomarkers and AI-driven assessment methods to subjective endpoints
