With the identification of novel targets, the number of interventional clinical trials in ophthalmology has increased. Visual acuity has for a long time been considered the gold standard endpoint for clinical trials, but in the recent years it became evident that other endpoints are required for many indications including geographic atrophy and inherited retinal disease. In glaucoma the currently available drugs were approved based on their IOP lowering capacity. Some recent findings do, however, indicate that at the same level of IOP reduction, not all drugs have the same effect on visual field progression. For neuroprotection trials in glaucoma, novel surrogate endpoints are required, which may either include functional or structural parameters or a combination of both. A number of potential surrogate endpoints for ophthalmology clinical trials have been identified, but their validation is complicated and requires solid scientific evidence. In this article we summarize candidates for clinical endpoints in ophthalmology with a focus on retinal disease and glaucoma. Functional and structural biomarkers, as well as quality of life measures are discussed, and their potential to serve as endpoints in pivotal trials is critically evaluated.

随着新靶点的确定，眼科介入临床试验的数量不断增加。长期以来，视力一直被认为是临床试验的金标准终点，但近年来，很明显，包括地图样萎缩和遗传性视网膜疾病在内的许多适应症都需要其他终点。对于青光眼，目前可用的药物是根据其降低眼压的能力而获得批准的。然而，最近的一些研究结果确实表明，在相同的眼压降低水平下，并非所有药物对视野进展都有相同的影响。对于青光眼的神经保护试验，需要新的替代终点，其中可能包括功能或结构参数或两者的组合。眼科临床试验的许多潜在替代终点已经确定，但其验证很复杂，需要坚实的科学证据。在本文中，我们总结了眼科临床终点的候选者，重点关注视网膜疾病和青光眼。讨论了功能和结构生物标志物以及生活质量测量，并严格评估了它们作为关键试验终点的潜力。