Messori A, Trippoli S, Becagli P, Zaccara G
Drug Information Centre, Pharmaceutical Service, Azienda Ospedaliera Careggi, Florence, Italy.
Pharmacoeconomics. 1999 Aug;16(2):153-63. doi: 10.2165/00019053-199916020-00004.
In patients with amyotrophic lateral sclerosis, long term treatment with riluzole has been reported to improve survival or tracheostomy-free survival in comparison with placebo. We conducted a pharmacoeconomic analysis for estimating the cost per life-year gained using this drug.
This study was an incremental cost-effectiveness lifetime analysis.
The clinical material was derived from 2 placebo-controlled randomised controlled trials comparing riluzole versus usual care without riluzole, which were identified through a literature search based on the IOWA and the Medline systems.
The study included 633 patients with amyotrophic lateral sclerosis. Patient-level information was retrieved from 313 patients treated with riluzole and 320 patients assigned to placebo. Survival after randomisation was compared between the 2 groups using standard statistics (log-rank test and Cox analysis), whereas the lifetime survival gain was estimated using Gompertz extrapolation. Cost data relative to the expenditure for healthcare resources were obtained from published information (using the US average wholesale price for the acquisition cost of riluzole). Sensitivity testing assessed the impact of different cost-of-illness assumptions for treated and untreated patients.
Our primary analysis showed that treatment with riluzole significantly prolonged survival [death risk = 0.77; 95% confidence interval (CI): 0.62 to 0.96; p = 0.022]. The lifetime survival gain (including 3% annual discounting) was, on average, 2.3 months per patient, while the incremental cost was around $US12,000 per patient. Hence, the cost-effectiveness ratio of riluzole versus usual care without riluzole was $US62,609 per life-year gained (discounted dollars per discounted years; 95% CI: $US13,458 to $US205,714). The sensitivity analysis, considering different values of national cost for riluzole, suggested an interval for this parameter ranging from $US45,048 to $US62,609.
Our study indicates that in patients with amyotrophic lateral sclerosis, riluzole has an unfavourable cost-effectiveness ratio or, at best, a borderline pharmacoeconomic profile.
据报道，与安慰剂相比，肌萎缩侧索硬化症患者长期使用利鲁唑可提高生存率或无气管切开生存率。我们进行了一项药物经济学分析，以估算使用该药物每获得一个生命年的成本。
本研究为增量成本效益终身分析。
临床资料来源于两项安慰剂对照随机对照试验，比较了利鲁唑与不使用利鲁唑的常规治疗，这些试验是通过基于爱荷华州和医学文献数据库系统的文献检索确定的。
该研究纳入了633例肌萎缩侧索硬化症患者。从313例接受利鲁唑治疗的患者和320例分配至安慰剂组的患者中获取患者层面的信息。使用标准统计学方法（对数秩检验和Cox分析）比较两组随机分组后的生存率，而使用Gompertz外推法估算终身生存获益。与医疗资源支出相关的成本数据来自已发表的信息（使用利鲁唑获取成本的美国平均批发价格）。敏感性测试评估了不同疾病成本假设对治疗和未治疗患者的影响。
我们的初步分析表明，利鲁唑治疗可显著延长生存期[死亡风险=0.77；95%置信区间（CI）：0.62至0.96；p=0.022]。每位患者的终身生存获益（包括3%的年度贴现）平均为2.3个月，而增量成本约为每位患者12,000美元。因此，与不使用利鲁唑的常规治疗相比，利鲁唑的成本效益比为每获得一个生命年62,609美元（贴现美元/贴现年；95%CI：13,458美元至205,714美元）。敏感性分析考虑了利鲁唑的不同国家成本值，表明该参数的区间为45,048美元至62,609美元。
我们的研究表明，在肌萎缩侧索硬化症患者中，利鲁唑的成本效益比不佳，或至多处于药物经济学临界状态。
