FDA Updates: Retina Trials and Regulatory Pathways
William M. Boyd, MD, director of the FDA’s Division of Ophthalmology, discussed the agency’s evolving approaches to clinical evidence, trial design, and expedited regulatory pathways at the 2026 Retina World Congress meeting.
By
Matthew Solan, senior writer
Matthew Solan, senior writer
Matthew Solan, senior writer
May 14, 2026
Clinical Scorecard: FDA Updates: Retina Trials and Regulatory Pathways
At a Glance
Category Detail
Condition Retinal diseases Key Mechanisms Evolving regulatory pathways and trial designs in ophthalmology Target Population Patients with retinal diseases, including rare and ultra-rare conditions Care Setting Ophthalmology clinical trials and drug development
Key Highlights
FDA encourages strategies to minimize bias in trials using sham injections. Real-Time Clinical Trials program allows near real-time access to clinical data. Commissioner’s National Priority Voucher Pilot Program aims to reduce review timelines. Single pivotal trials must be large and statistically robust for approval. New initiatives focus on rare and ultra-rare diseases in retinal medicine. Guideline-Based Recommendations
Diagnosis
Engage with the FDA early to discuss trial designs for drug approval. Management
Consider multiple dosing levels and trial design strategies to minimize bias. Monitoring & Follow-up
Utilize the Real-Time Clinical Trials program for ongoing data access. Risks
Sham injections may introduce bias in clinical trials. Patient & Prescribing Data
Individuals with retinal diseases, including those with rare conditions.
Therapies should demonstrate improvement in clinical outcomes or biomarkers.
Clinical Best Practices
Utilize the FDA Rare Disease Innovation Hub for guidance on rare diseases. Engage in discussions with the FDA for clarity on trial endpoints and definitions. Related Resources & Content
