Thomas Jefferson University, Philadelphia, Pennsylvania 19107, USA.
J Gastroenterol Hepatol. 2010 Nov;25(11):1739-46. doi: 10.1111/j.1440-1746.2010.06404.x.
A double-blind, randomized phase III trial of sorafenib in advanced hepatocellular carcinoma demonstrated that sorafenib significantly prolonged overall survival compared to placebo (median overall survival = 10.7 months vs 7.9 months, P < 0.001). Sorafenib is the first and only systemic agent demonstrating survival benefit in these patients. The aim of this study was to assess the cost-effectiveness of sorafenib versus best supportive care in the treatment of advanced hepatocellular carcinoma in the USA.
A Markov model was developed following time-to-progression and survival using phase III trial data. Health effects are expressed as life-years gained. Resource utilization included drugs, physician visits, laboratory tests, scans, and hospitalizations. Unit costs, expressed in 2007 $US, came from diagnosis-related groupings, fee schedules, and the Red Book. Costs and effects were evaluated over a patient's lifetime and discounted at 3%.
Results are presented as incremental cost/life-year gained. Deterministic and probabilistic sensitivity analyses were conducted. Life-years gained were increased for sorafenib compared to best supportive care (mean ± standard deviation: 1.58 ± 0.17 vs 1.05 ± 0.10 life-years gained/sorafenib patient and best supportive care, respectively). Lifetime total costs were $US40,639 ± $US3052 for sorafenib and $US7, 804 ± $US1349 for best supportive care. The incremental cost-effectiveness ratio was $US62,473/life-year gained.
The economic evaluation indicates that sorafenib is cost-effective compared to best supportive care, with a cost-effectiveness ratio within the established threshold that US society is willing to pay (i.e. $US50,000-$US100,000) and significantly lower than alternative thresholds suggested in recent years ($US183,000-$US264,000/life-year gained, or $US300,000/quality-adjusted life-year) in oncology.
索拉非尼治疗晚期肝细胞癌的双盲、随机 III 期临床试验表明，与安慰剂相比，索拉非尼显著延长了总生存期（中位总生存期=10.7 个月 vs 7.9 个月，P＜0.001）。索拉非尼是第一个也是唯一一个在这些患者中显示生存获益的系统治疗药物。本研究旨在评估索拉非尼与最佳支持治疗在治疗美国晚期肝细胞癌中的成本效益。
根据 III 期试验数据，采用时间进展和生存情况建立了一个 Markov 模型。健康效果以获得的生命年数表示。资源利用包括药物、医生就诊、实验室检查、扫描和住院。单位成本以 2007 年美元表示，来自诊断相关分组、收费表和 Red Book。成本和效果在患者的一生中进行评估，并贴现 3%。
结果以增量成本/获得的生命年数表示。进行了确定性和概率敏感性分析。与最佳支持治疗相比，索拉非尼获得的生命年数增加（索拉非尼患者和最佳支持治疗分别为 1.58±0.17 和 1.05±0.10 个生命年数）。索拉非尼的终身总费用为 40639 美元±3052 美元，最佳支持治疗的费用为 7804 美元±1349 美元。增量成本效益比为 62473 美元/生命年数。
经济评估表明，与最佳支持治疗相比，索拉非尼具有成本效益，其成本效益比在被美国社会愿意支付的既定阈值内（即 50000 美元至 100000 美元），并且明显低于近年来提出的替代阈值（即 183000 美元至 264000 美元/生命年数或 300000 美元/质量调整生命年数）。
