United States Regulatory Approval of Topical Treatments for Dry Eye
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Published on: April 1, 2019
David Cui1, Ian J Saldanha2, Gavin Li3
1From the Ocular Surface Disease Clinic, The Wilmer Eye Institute, Johns Hopkins University School of Medicine (D.C., G.L., P.M.M., M.X.L., E.K.A.), Baltimore, Maryland; The Krieger Eye Institute, Sinai Hospital of Baltimore (D.C.), Baltimore, Maryland.
Purpose:
To report the heterogeneity in methodology of clinical trials submitted to the US Food and Drug Administration (FDA) for approval of topical dry eye treatments.
Design:
Comparative analysis of clinical trials' methods.
Methods:
We reviewed the online, publicly available FDA database, application review files, ClinicalTrials.gov registry records, and journal articles for each FDA-approved topical dry eye treatment. For each trial, we extracted information about the study, patient demographics, treatment names and doses, sample size in each arm, and the measurement instrument in a systematic fashion.
Results:
Fourteen trials were included that assessed 5 topical treatments for dry eye (cyclosporine 0.05%, cyclosporine 0.09%, lifitegrast 5%, and loteprednol 0.25% eye drops and varenicline 0.03-mg nasal spray). Median treatment duration was 12 weeks (range, 2-24 weeks). In all trials, treatments, including varying concentrations of the same treatment, were compared with vehicle. Twelve trials (85.7%) evaluated a primary clinician-measured clinical sign, and 10 trials (71.4%) evaluated a primary patient-reported symptom. Corneal staining was the most frequently evaluated clinical sign primary outcome, reported in half (6 of 12) of the trials, and was graded using 4 different scoring systems. Conjunctival staining, conjunctival hyperemia, and tear production were each measured using 2 different scoring systems. Ocular discomfort, the only patient-reported symptom primary outcome, was measured using 5 different instruments.
Conclusion:
A variety of outcome measures were used in these clinical trials. Clinically meaningful dry eye outcome measures and standardized measurements can optimize the assessment of and comparison of therapeutic benefits.
Insights
Clinical trials for dry eye treatments show varied methods. Standardizing outcome measures for corneal staining and ocular discomfort is crucial for comparing therapeutic benefits effectively.
Area of Science:
- Ophthalmology
- Clinical Research Methodology
- Drug Development
Background:
- Dry eye disease is a prevalent condition affecting millions worldwide.
- Topical treatments are a cornerstone of dry eye management.
- Evaluating the efficacy of these treatments relies on robust clinical trial data.
Approach:
- A systematic review of FDA-approved topical dry eye treatments was conducted.
- Data from clinical trials, including methodology, patient demographics, and outcome measures, were extracted.
- Publicly available FDA databases, ClinicalTrials.gov, and journal articles were utilized.
Key Points:
- Five topical treatments (cyclosporine, lifitegrast, loteprednol, varenicline) were assessed across fourteen trials.
- Treatment durations varied, with a median of 12 weeks.
- Both clinician-measured signs (e.g., corneal staining) and patient-reported symptoms (e.g., ocular discomfort) were primary outcomes.
- Significant heterogeneity was observed in measurement instruments and scoring systems for both signs and symptoms.
- Corneal staining was assessed using four different grading systems.
- Ocular discomfort was measured using five distinct instruments.
Conclusions:
- Clinical trials for topical dry eye treatments exhibit considerable methodological diversity.
- The use of varied outcome measures complicates the direct comparison of therapeutic benefits.
- Standardized, clinically meaningful outcome measures are essential for optimizing the assessment and comparison of treatment efficacy.
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