从美国角度评估雷珠单抗和阿柏西普治疗糖尿病性黄斑水肿的成本效果：Protocol T 研究两年数据分析。

Cost-effectiveness of ranibizumab and aflibercept to treat diabetic macular edema from a US perspective: analysis of 2-year Protocol T data.

机构信息

Pepose Vision Institute, Chesterfield, MO, USA.

Veritas Health Economics Consulting, Inc., Carlsbad, CA, USA.

出版信息

J Med Econ. 2020 Mar;23(3):287-296. doi: 10.1080/13696998.2019.1666855. Epub 2019 Nov 4.

DOI:10.1080/13696998.2019.1666855

PMID:31502893

Abstract

Protocol T (NCT01627249) was a head-to-head study conducted by the Diabetic Retinopathy Clinical Research Network that compared intravitreal aflibercept, bevacizumab, and ranibizumab for the treatment of diabetic macular edema (DME). A cost-effectiveness analysis accompanying the 1-year data of Protocol T revealed that aflibercept was not cost-effective vs ranibizumab for all patients, but could have been cost-effective in certain patient sub-groups if the 1-year results were extrapolated out to 10 years. The present study evaluated the cost-effectiveness of US Food and Drug Administration-approved anti-vascular endothelial growth factor agents (ranibizumab, aflibercept) for treatment of DME using the 2-year data from Protocol T. Costs of aflibercept 2.0 mg or ranibizumab 0.3 mg, visual acuity (VA)-related medical costs, and quality-adjusted life-years (QALYs) were simulated for eight VA health states. Treatment, adverse event management, and VA-related healthcare resource costs (2016 US dollars) were based on Medicare reimbursement and published literature. VA-related health utilities were determined using a published algorithm. Patients were stratified by baseline VA: 20/40 or better; 20/50 or worse. Total 2-year costs were higher, and QALYs similar, for aflibercept vs ranibizumab in the full cohort ($44,423 vs $34,529; 1.476 vs 1.466), 20/40 or better VA sub-group ($40,854 vs $31,897; 1.517 vs 1.519), and 20/50 or worse VA sub-group ($48,214 vs $37,246; 1.433 vs 1.412), respectively. Incremental cost-effectiveness ratios in the full cohort and 20/50 or worse VA sub-group were $986,159/QALY and $523,377/QALY, respectively. These decreased to $711,301 and $246,978 when analyses were extrapolated to 10 years. Key potential limitations include the fact that VA was the only QALY parameter analyzed and the uncertainty surrounding the role of better- and worse-seeing eye VA in overall functional impairment. This analysis suggests that aflibercept is not cost-effective vs ranibizumab for patients with DME, regardless of baseline vision.

摘要

方案 T（NCT01627249）是由糖尿病视网膜病变临床研究网络进行的一项头对头研究，比较了玻璃体内注射阿柏西普、贝伐单抗和雷珠单抗治疗糖尿病黄斑水肿（DME）的效果。伴随方案 T 的 1 年数据的一项成本效益分析显示，对于所有患者，阿柏西普不如雷珠单抗具有成本效益，但如果将 1 年的结果外推至 10 年，那么它可能在某些特定患者亚组中具有成本效益。本研究使用方案 T 的 2 年数据，评估了美国食品和药物管理局批准的抗血管内皮生长因子药物（雷珠单抗、阿柏西普）治疗 DME 的成本效益。阿柏西普 2.0mg 或雷珠单抗 0.3mg 的治疗费用、视力相关医疗费用和质量调整生命年（QALY），针对 8 种视力健康状态进行了模拟。治疗、不良事件管理以及与视力相关的医疗资源成本（2016 年美元），基于医疗保险报销和已发表的文献。与视力相关的健康效用，使用已发表的算法确定。根据基线视力，患者分为以下两组：20/40 或以上；20/50 或以下。在全队列中，阿柏西普的 2 年总费用更高，而 QALY 与雷珠单抗相似（$44423 比 $34529；1.476 比 1.466），20/40 或以上视力亚组（$40854 比 $31897；1.517 比 1.519）和 20/50 或以下视力亚组（$48214 比 $37246；1.433 比 1.412）。在全队列和 20/50 或以下视力亚组中，增量成本效益比分别为每 QALY $986159 和每 QALY $523377。当分析外推至 10 年时，这些数字分别降至每 QALY $711301 和每 QALY $246978。主要潜在限制因素包括，VA 是唯一分析的 QALY 参数，以及更好和更差视力眼 VA 在整体功能障碍中的作用存在不确定性。本分析表明，对于 DME 患者，阿柏西普不如雷珠单抗具有成本效益，无论基线视力如何。