1 Modeling and Simulation, Evidera, Waltham, Massachusetts.
2 Modeling and Simulation, Evidera, London, United Kingdom.
J Manag Care Spec Pharm. 2018 Jul;24(7):608-616. doi: 10.18553/jmcp.2018.24.7.608.
Age-related macular degeneration (AMD) is the leading cause of vision loss in the United States. The most severe vision loss occurs in patients with neovascular AMD, known as wet AMD (wAMD). The most commonly used antivascular endothelial growth factor (VEGF) therapies approved by the FDA to treat patients with wAMD are ranibizumab, 0.5 mg administered by intravitreal injection once a month (approximately every 28 days), and intravitreal aflibercept injection (IAI), 2 mg every 4 weeks (monthly) for the first 12 weeks (3 months), followed by IAI 2 mg once every 8 weeks (2 months). Given the similar efficacy and safety profiles between IAI and ranibizumab, their associated costs and comparative cost-effectiveness are key factors in determining which one represents a more rational investment of scarce health care resources to help address the increasing cost of prescription drugs in the United States, a source of concern for patients, prescribers, payers, and policymakers.
To assess the cost-effectiveness of intravitreal aflibercept injection 2 mg every 8 weeks after 3 initial monthly doses (IAI 2q8) versus ranibizumab 0.5 mg monthly (Rq4) and pro re nata (PRN) in the treatment of patients with wAMD from a U.S. payer perspective.
A Markov cohort model was developed to estimate the lifetime quality-adjusted life-years (QALYs) and costs of treating patients with wAMD with IAI 2q8, Rq4, and ranibizumab PRN. The model considered changes in best-corrected visual acuity in the affected and fellow eyes over time, and the effect of blindness on mortality. Efficacy for IAI 2q8 and Rq4 was from VIEW 1 and VIEW 2 studies and from the Comparison of AMD Treatments Trials for ranibizumab PRN. Utilities and costs (in 2016 U.S. dollars) were from published literature. Health outcomes and costs were discounted at an annual rate of 3%.
Over a lifetime, IAI 2q8 provided equal health benefits with Rq4 (5.44 QALYs) at a lower total cost ($33,745 vs. $48,031) as a result of fewer injections. IAI 2q8 yielded slightly greater QALYs versus ranibizumab PRN (5.44 vs. 5.40) at a slightly higher cost ($33,745 vs. $33,652), with an incremental cost per QALY gained of $2,583. Results were sensitive to variations in drug acquisition costs and number of injections of both drugs and the baseline age of the cohort.
IAI 2q8 can be cost saving and cost-effective compared with Rq4 and ranibizumab PRN for the treatment of wAMD in the United States.
This study was funded by Regeneron Pharmaceuticals, the manufacturer of aflibercept. Hernandez, Lanitis, Cele, and Toro-Diaz are employed by Evidera, which received funding from Regeneron Pharmaceuticals to conduct this study. Gibson and Kuznik are employed by and own stock in Regeneron Pharmaceuticals.
年龄相关性黄斑变性(AMD)是美国导致视力丧失的主要原因。最严重的视力丧失发生在新生血管性 AMD 患者中,称为湿性 AMD(wAMD)。美国食品和药物管理局(FDA)批准的最常用于治疗 wAMD 患者的抗血管内皮生长因子(VEGF)治疗药物是雷珠单抗,0.5mg 玻璃体腔内注射,每月一次(约每 28 天一次),以及玻璃体腔内阿柏西普注射(IAI),前 12 周(3 个月)每 4 周(每月)注射 2mg,然后每 8 周(2 个月)注射一次 2mg。鉴于 IAI 和雷珠单抗的疗效和安全性相似,其相关成本和比较成本效益是决定哪种药物更能合理利用有限的医疗资源的关键因素,这也是解决美国处方药日益上涨的成本问题的关键,这也是患者、处方者、支付者和政策制定者关注的问题。
从美国支付者的角度评估玻璃体内注射阿柏西普 2mg 每 8 周(IAI 2q8)与雷珠单抗 0.5mg 每月(Rq4)和按需治疗湿性年龄相关性黄斑变性(wAMD)患者的成本效益。
开发了一个马尔可夫队列模型,以估计接受 IAI 2q8、Rq4 和雷珠单抗 PRN 治疗的 wAMD 患者的终生质量调整生命年(QALYs)和成本。该模型考虑了受影响眼和对侧眼的最佳矫正视力随时间的变化,以及失明对死亡率的影响。IAI 2q8 和 Rq4 的疗效来自 VIEW 1 和 VIEW 2 研究以及 ranibizumab PRN 的 AMD 治疗试验比较。效用和成本(2016 年美元)来自已发表的文献。健康结果和成本按每年 3%的贴现率贴现。
在一生中,由于注射次数较少,IAI 2q8 与 Rq4(5.44QALYs)提供相同的健康益处,总成本更低(33745 美元与 48031 美元)。IAI 2q8 与雷珠单抗 PRN 相比(5.44 与 5.40)获得了略多的 QALYs,成本略高(33745 美元与 33652 美元),每获得一个 QALY 的增量成本为 2583 美元。结果对药物获得成本和两种药物注射次数以及队列的基线年龄的变化敏感。
与 Rq4 和雷珠单抗 PRN 相比,IAI 2q8 可节省成本并具有成本效益,可用于治疗美国的 wAMD。
这项研究由 aflibercept 的制造商再生元制药公司资助。Hernandez、Lanitis、Cele 和 Toro-Diaz 受雇于 Evidera,该公司从再生元制药公司获得资金进行这项研究。Gibson 和 Kuznik 受雇于再生元制药公司并拥有该公司的股票。