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Retinal Physician

Article | 2026 Retina World Congress

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
Retinal Physician May 14, 2026

Clinical Scorecard: FDA Updates: Retina Trials and Regulatory Pathways

At a Glance

CategoryDetail
ConditionRetinal diseases
Key MechanismsEvolving regulatory pathways and trial designs in ophthalmology
Target PopulationPatients with retinal diseases, including rare and ultra-rare conditions
Care SettingOphthalmology 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.

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