Clinical Report: The Limitations of AMD Trials
Overview
A systematic review evaluates the use of vision-related patient-reported outcome measures (PROMs) in randomized controlled trials (RCTs) for age-related macular degeneration (AMD). The findings highlight inconsistencies in PROM reporting.
Background
Age-related macular degeneration (AMD) is a leading cause of irreversible vision loss, affecting millions globally. Traditional measures of visual acuity may not fully capture the patient experience, which includes challenges in reading, mobility, and psychosocial well-being.
Data Highlights
| Study Characteristics | Findings |
|---|---|
| Number of RCTs Included | 38 |
| Years Spanned | 2001 to 2024 |
| Dominant PROM Instrument | NEI VFQ-25 |
| NEI VFQ-25 Scores for Early/Intermediate AMD | 75.34 to 89.13 |
| NEI VFQ-25 Scores for Neovascular AMD | 59.1 to 77.9 |
Key Findings
- 11 distinct vision-related PROM instruments were identified across the trials.
- The NEI VFQ family was used in 30 of the 38 studies, with NEI VFQ-25 being the most common.
- Baseline PROM reporting was inconsistent, with many trials presenting only composite scores.
- Only 13% of studies reported both better- and worse-seeing eye acuity.
- Baseline PROM results were not stratified by demographic factors, limiting interpretability.
Clinical Implications
Clinicians and researchers should prioritize comprehensive reporting of patient-reported outcomes in AMD trials to enhance understanding of patient experiences. Improved PROM reporting can complement visual acuity measures, providing a fuller picture of treatment impact.
Conclusion
The review discusses the reporting of PROMs in AMD trials.
Related Resources & Content
- Bodmer NS, Guezelguen M, Huber L, et al., Ophthalmology Science, 2023 -- Vision-Related Patient-Reported Outcomes in Randomized Controlled Trials of Age-Related Macular Degeneration: A Systematic Review
- Retinal Physician — Will the AMD Therapies Now in Clinical Trials Deliver What They Promise?
- Retinal Physician — Using Genetics to Guide AMD Therapy: Are We There Yet?
- Retinal Physician — Genetic Testing for AMD
- retinal physician — Ophthotech AMD Combo Fails in Phase 3
- Age-Related Macular Degeneration Preferred Practice Pattern® - Ophthalmology
- Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments | FDA
- European Medicines Regulatory Network (EMRN) workshop on geographic atrophy endpoints | European Medicines Agency (EMA)
- Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials - PubMed
- Avacincaptad Pegol for Geographic Atrophy Secondary to Age-Related Macular Degeneration - Ophthalmology
- Drug Approval for the Treatment of Geographic Atrophy: How We Got Here and Where We Need to Go - PMC
- Ranibizumab for Neovascular Age-Related Macular Degeneration | New England Journal of Medicine
- Functional endpoints in patients with geographic atrophy: What to consider when designing a clinical trial - PubMed
- A comprehensive review of structure-function correlations in age-related macular degeneration: Contributions of microperimetry - ScienceDirect
- Vision-Related Patient-Reported Outcomes in Randomized Controlled Trials of Age-Related Macular Degeneration: A Systematic Review - ScienceDirect
- Minimum set of outcome measures for non-advanced age-related macular degeneration: a Delphi consensus statement by the TRACER (intermediate age-related macular degeneration) study group | British Journal of Ophthalmology
Based on findings from:
The Limitations of AMD Trials
The Ophthalmologist, 2026.
https://theophthalmologist.com/issues/2026/articles/august/the-limitations-of-amd-trials/
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