Department of Pharmaceutical and Health Economics, School of Pharmacy, University of Southern California, 635 Downey Way, Los Angeles, CA, 90089-3333, USA.
Kaiser Permanente Northwest, Portland, OR, USA.
BMC Urol. 2022 May 13;22(1):76. doi: 10.1186/s12894-022-01025-4.
To assess the price range in which fexapotide triflutate (FT), a novel injectable, is cost-effective relative to current oral pharmacotherapy (5 α-reductase inhibitor, α-blocker, 5 α-reductase inhibitor and α-blocker combination therapy) as initial therapy followed by surgery for moderate-to-severe benign prostate hyperplasia patients with lower urinary tract symptoms (BPH-LUTS).
We developed a microsimulation decision-analytic model to track the progression of BPH-LUTS and associated costs and quality-adjusted life years in the target population. The cost-effectiveness analysis was performed from Medicare's perspective with a time horizon of 4 years using 2019 US dollars for all costs. The microsimulation model considered treatment patterns associated with nonadherence to oral medication and progression to surgery. Model parameters were estimated from large randomized controlled trials, literature and expert opinion. For each initial treatment option, simulations were performed with 1000 iterations, with 1000 patients per iteration.
Three upfront oral pharmacotherapy options are close in cost-effectiveness, with combination therapy being the most cost-effective option. Relative to upfront oral pharmacotherapy options, FT slightly increases quality-adjusted life years (QALY) per patient (1.870 (95% CI, 1.868 to 1.872) vs. 1.957 (95% CI, 1.955 to 1.959) QALYs). Under the willingness-to-pay (WTP) threshold of $150,000 per QALY, at price per injection of $14,000, FT is about as cost-effective as upfront oral pharmacotherapy options with net monetary benefit (NMB) $279,168.54. Under the WTP threshold of $50,000 per QALY, at price per injection of $5,000, FT is about as cost-effective as upfront oral pharmacotherapy options with NMB $92,135.18. In an alternative 10-year time horizon scenario, FT price per injection at $11,000 and $4,500 makes FT as cost-effective as oral pharmacotherapies. One-way sensitivity analysis showed this result is most sensitive to upfront therapy prices, FT efficacy and initial IPSS. At price per injections of $5,000, $10,000 and $15,000, the probability that FT is either cost-effective or dominant compared to upfront oral pharmacotherapy options using a WTP threshold of $150,000 per QALY is 100%, 93% and 40%, respectively.
Compared to upfront oral pharmacotherapy options, FT would be cost-effective at a price per injection below $14,000, assuming a WTP threshold of $150,000 per QALY.
评估特夫比妥三氟酯（FT）的价格范围，FT 是一种新型注射用药物，与当前口服药物疗法（5α-还原酶抑制剂、α-阻滞剂、5α-还原酶抑制剂和α-阻滞剂联合治疗）相比，对于中重度良性前列腺增生伴有下尿路症状（BPH-LUTS）的患者，作为初始治疗后再进行手术的治疗方案是否具有成本效益。
我们开发了一个微观模拟决策分析模型，以追踪目标人群中 BPH-LUTS 的进展情况以及相关成本和质量调整生命年（QALY）。成本效益分析从医疗保险的角度出发，使用 2019 年美元作为所有成本的时间范围为 4 年。微观模拟模型考虑了与口服药物治疗不依从性和手术进展相关的治疗模式。模型参数是从大型随机对照试验、文献和专家意见中估计得出的。对于每种初始治疗方案，在 1000 次迭代中进行了模拟，每次迭代有 1000 名患者。
三种初始口服药物治疗方案的成本效益相近，联合治疗方案是最具成本效益的选择。与初始口服药物治疗方案相比，FT 略微增加了每位患者的 QALY（1.870（95%CI，1.868 至 1.872）与 1.957（95%CI，1.955 至 1.959）QALYs）。在支付意愿（WTP）阈值为每 QALY 15 万美元的情况下，注射价格为 14000 美元时，FT 与初始口服药物治疗方案的成本效益相当，净货币收益（NMB）为 279168.54 美元。在支付意愿阈值为每 QALY 5 万美元的情况下，注射价格为 5000 美元时，FT 与初始口服药物治疗方案的成本效益相当，NMB 为 92135.18 美元。在另一个 10 年时间范围内，FT 的注射价格为 11000 美元和 4500 美元时，FT 与口服药物治疗方案的成本效益相当。单因素敏感性分析表明，这一结果对初始治疗价格、FT 疗效和初始国际前列腺症状评分（IPSS）最为敏感。在注射价格为 5000 美元、10000 美元和 15000 美元时，FT 相对于初始口服药物治疗方案在支付意愿阈值为每 QALY 15 万美元的情况下具有成本效益或优势的概率分别为 100%、93%和 40%。
与初始口服药物治疗方案相比，假设支付意愿阈值为每 QALY 15 万美元，FT 的注射价格低于 14000 美元时具有成本效益。
