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:
- Exploring early disease signs and endpoint selection strategies to optimize regulatory timelines for gene therapy trials
- Examining microperimetry and FST endpoint validation challenges to address real-world implementation barriers in inherited retinal diseases
- Comparing FDA and EMA regulatory perspectives on functional endpoints to navigate divergent approval requirements effectively
- Discussing novel endpoint acceptance and clinical meaningfulness criteria to advance ophthalmology-specific gene therapy development pathways
