Endpoints for clinical trials in ophthalmology

Area of Science:

• Ophthalmology and Clinical Trial Endpoints
• Retinal Diseases and Glaucoma Research

Background:

• The number of interventional ophthalmology clinical trials is rising due to novel target identification.
• Traditional endpoints like visual acuity are insufficient for many conditions, including geographic atrophy and inherited retinal diseases.
• Current glaucoma drug approvals rely on intraocular pressure (IOP) lowering, but effects on visual field progression vary.

Purpose of the Study:

• To identify and evaluate potential surrogate endpoints for ophthalmology clinical trials.
• To critically assess functional and structural biomarkers, and quality of life measures for pivotal trials.
• To focus on endpoints relevant to retinal diseases and glaucoma.

Main Methods:

• Review and summarization of candidate clinical endpoints in ophthalmology.
• Discussion of functional and structural biomarkers.
• Evaluation of quality of life measures as potential endpoints.

Main Results:

• Existing endpoints like visual acuity are inadequate for certain ophthalmic conditions.
• Novel surrogate endpoints, including functional and structural parameters, are necessary for neuroprotection trials in glaucoma.
• Validation of identified endpoints requires robust scientific evidence.

Conclusions:

• There is a critical need for validated surrogate endpoints in ophthalmology clinical trials.
• Functional and structural biomarkers, alongside quality of life measures, show promise but require further validation.
• Developing appropriate endpoints is essential for advancing treatment and understanding of retinal diseases and glaucoma.