Instituto Nacional de Cardiologia, NATS, Rio de Janeiro, Brazil; Universidade Federal do Estado do Rio de Janeiro, Rio de Janeiro, Brazil.
Universidade de Brasília, Brasília, Brazil.
Value Health Reg Issues. 2022 Sep;31:47-52. doi: 10.1016/j.vhri.2022.02.006. Epub 2022 Apr 13.
Several trials have demonstrated the benefit of the CDK 4/6 inhibitors for postmenopausal women with luminal advanced breast cancer. This research aims to compare the cost-utility of the CDK 4/6 inhibitors in patients with no history of resistance to endocrine therapy.
A Markov model was constructed to estimate the incremental cost per quality-adjusted life-years (QALYs) of treatments from the Brazilian public health system perspective over a lifetime horizon (30 years) with 5% annual discount rate for both benefits and costs. Efficacy parameters were extracted from the pivotal studies. Costs were based on open data from the Brazilian Ministry of Health. The utilities were calculated according to the overall population preferences from a British study. Deterministic and probabilistic sensitivity analyses evaluated the robustness of the results.
The most cost-effective drug was ribociclib (US$50 748/QALY), followed by abemaciclib (US$64 052/QALY) and palbociclib (US$65 289/QALY). The univariate analysis showed that the incremental cost-utility ratio (ICUR) was mainly sensitive to the overall survival hazard ratio. The one thousand-probabilistic simulation showed that all ICUR values were above classical thresholds such as 1 to 3 gross domestic product (GDP) per capita per QALY.
Even though there is no established willingness to pay threshold in Brazil, the estimated ICUR for CDK 4/6 inhibitors is >6 times the Brazilian GDP per capita (GDP per capita = US$5694.73), which might be a barrier to their inclusion in the Brazilian public health system.
多项试验已证明 CDK4/6 抑制剂对激素受体阳性、绝经后晚期乳腺癌患者有益。本研究旨在比较 CDK4/6 抑制剂在无内分泌治疗耐药史患者中的成本效用。
构建马尔可夫模型，从巴西公共卫生系统的角度，在 30 年的时间范围内（终生），采用 5%的年贴现率对治疗方案进行成本效用分析，贴现率分别用于计算效益和成本。从关键性研究中提取疗效参数。成本基于巴西卫生部的公开数据。效用值根据来自英国的一项研究中的总体人群偏好进行计算。确定性和概率敏感性分析评估了结果的稳健性。
最具成本效益的药物是瑞博西利（每 QALY 50748 美元），其次是阿贝西利（每 QALY 64052 美元）和哌柏西利（每 QALY 65289 美元）。单变量分析表明，增量成本效用比（ICUR）主要对总生存风险比敏感。1000 次概率模拟表明，所有 ICUR 值均高于经典阈值，如每 QALY 1 至 3 个国内生产总值（GDP）。
尽管巴西尚未确定支付意愿的阈值，但 CDK4/6 抑制剂的估计 ICUR 是巴西人均 GDP 的 6 倍以上（人均 GDP=5694.73 美元），这可能成为其纳入巴西公共卫生系统的障碍。
