Abstract：
OBJECTIVE: This study aimed to evaluate the cost-effectiveness of anti-vascular endothelial growth factor drugs (anti-VEGFs) compared with panretinal photocoagulation (PRP) for treating proliferative diabetic retinopathy (PDR) in the United Kingdom.
METHODS: A discrete event simulation model was developed, informed by individual participant data meta-analysis. The model captures treatment effects on best corrected visual acuity in both eyes, and the occurrence of diabetic macular edema and vitreous hemorrhage. The model also estimates the value of undertaking further research to resolve decision uncertainty.
RESULTS: Anti-VEGFs are unlikely to generate clinically meaningful benefits over PRP. The model predicted anti-VEGFs be more costly and similarly effective as PRP, generating 0.029 fewer quality-adjusted life-years at an additional cost of £3688, with a net health benefit of -0.214 at a £20 000 willingness-to-pay threshold. Scenario analysis results suggest that only under very select conditions may anti-VEGFs offer potential for cost-effective treatment of PDR. The consequences of loss to follow-up were an important driver of model outcomes.
CONCLUSIONS: Anti-VEGFs are unlikely to be a cost-effective treatment for early PDR compared with PRP. Anti-VEGFs are generally associated with higher costs and similar health outcomes across various scenarios. Although anti-VEGFs were associated with lower diabetic macular edema rates, the number of cases avoided is insufficient to offset the additional treatment costs. Key uncertainties relate to the long-term comparative effectiveness of anti-VEGFs, particularly considering the real-world rates and consequences of treatment nonadherence. Further research on long-term visual acuity and rates of vision-threatening complications may be beneficial in resolving uncertainties.
METHODS: A discrete event simulation model was developed, informed by individual participant data meta-analysis. The model captures treatment effects on best corrected visual acuity in both eyes, and the occurrence of diabetic macular edema and vitreous hemorrhage. The model also estimates the value of undertaking further research to resolve decision uncertainty.
RESULTS: Anti-VEGFs are unlikely to generate clinically meaningful benefits over PRP. The model predicted anti-VEGFs be more costly and similarly effective as PRP, generating 0.029 fewer quality-adjusted life-years at an additional cost of £3688, with a net health benefit of -0.214 at a £20 000 willingness-to-pay threshold. Scenario analysis results suggest that only under very select conditions may anti-VEGFs offer potential for cost-effective treatment of PDR. The consequences of loss to follow-up were an important driver of model outcomes.
CONCLUSIONS: Anti-VEGFs are unlikely to be a cost-effective treatment for early PDR compared with PRP. Anti-VEGFs are generally associated with higher costs and similar health outcomes across various scenarios. Although anti-VEGFs were associated with lower diabetic macular edema rates, the number of cases avoided is insufficient to offset the additional treatment costs. Key uncertainties relate to the long-term comparative effectiveness of anti-VEGFs, particularly considering the real-world rates and consequences of treatment nonadherence. Further research on long-term visual acuity and rates of vision-threatening complications may be beneficial in resolving uncertainties.
摘要：
目的：评估抗血管内皮生长因子药物（抗VEGF）与全视网膜光凝（PRP）治疗英国增生性糖尿病视网膜病变（PDR）的成本-效果。
方法：开发了离散事件仿真模型，由个体患者数据荟萃分析提供信息。该模型捕获了对双眼最佳矫正视力的治疗效果，以及糖尿病性黄斑水肿（DMO）和玻璃体出血的发生。该模型还估计了进行进一步研究以解决决策不确定性的价值。
结果：抗VEGF不太可能比PRP产生有临床意义的益处。该模型预测的抗VEGF成本更高，与PRP相似，减少0.029个QALY，额外费用为3,688英镑，在20,000英镑的支付意愿阈值下，净健康福利为-0.214。情景分析结果表明，只有在非常选择的条件下，抗VEGFs才可能为PDR的经济有效治疗提供潜力。失去随访的后果是模型结果的重要驱动因素。
结论：与PRP相比，抗VEGF不太可能是早期PDR的经济有效治疗方法。抗VEGFs通常在各种情况下与更高的成本和相似的健康结果相关。虽然抗VEGF与较低的DMO率相关，避免的病例数量不足以抵消额外的治疗费用。关键的不确定性与抗VEGFs的长期比较效果有关，特别是考虑到不遵守治疗的实际发生率和后果。对长期视力的进一步研究，和视力威胁并发症的发生率可能有助于解决不确定性。
方法：开发了离散事件仿真模型，由个体患者数据荟萃分析提供信息。该模型捕获了对双眼最佳矫正视力的治疗效果，以及糖尿病性黄斑水肿（DMO）和玻璃体出血的发生。该模型还估计了进行进一步研究以解决决策不确定性的价值。
结果：抗VEGF不太可能比PRP产生有临床意义的益处。该模型预测的抗VEGF成本更高，与PRP相似，减少0.029个QALY，额外费用为3,688英镑，在20,000英镑的支付意愿阈值下，净健康福利为-0.214。情景分析结果表明，只有在非常选择的条件下，抗VEGFs才可能为PDR的经济有效治疗提供潜力。失去随访的后果是模型结果的重要驱动因素。
结论：与PRP相比，抗VEGF不太可能是早期PDR的经济有效治疗方法。抗VEGFs通常在各种情况下与更高的成本和相似的健康结果相关。虽然抗VEGF与较低的DMO率相关，避免的病例数量不足以抵消额外的治疗费用。关键的不确定性与抗VEGFs的长期比较效果有关，特别是考虑到不遵守治疗的实际发生率和后果。对长期视力的进一步研究，和视力威胁并发症的发生率可能有助于解决不确定性。
