School of Health and Related Research, University of Sheffield, Sheffield S1 4DA, UK.
Pharmacoeconomics. 2013 Jun;31(6):479-88. doi: 10.1007/s40273-013-0050-9.
The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of cabazitaxel (Jevtana®, sanofi-aventis, UK) to submit evidence of its clinical and cost effectiveness for the second-line treatment of metastatic hormone-refractory prostate cancer (mHRPC). The School of Health and Related Research Technology Appraisal Group (ScHARR-TAG) at the University of Sheffield was commissioned to act as the independent Evidence Review Group (ERG). The ERG produced a critical review of the evidence for the clinical and cost effectiveness of the technology based upon the manufacturer's submission to NICE. Clinical evidence was derived from a multinational randomized open-label phase III trial of cabazitaxel plus prednisone or prednisolone in men with mHRPC that had progressed during or following treatment with docetaxel. The comparator was mitoxantrone plus prednisone or prednisolone. Use of cabazitaxel was associated with a statistically significant improvement in overall survival, median progression-free survival and time to tumour progression. However, it was also associated with an increased incidence of adverse events such as neutropenia. Utility data were based on interim results from the early access programme for cabazitaxel. Data were only available for a small number of patients with stable disease, resulting in great uncertainty as to the effect of cabazitaxel on quality of life. For their economic evaluation, the manufacturer estimated that the use of cabazitaxel was associated with an incremental cost of £74,908 per QALY gained. However, the ERG disagreed with the manufacturer over two key methodological points. The first concerned modelling and extrapolating survival; the second point was concerned with the choice of patient population. The ERG altered the manufacturer's evaluation to take into account these two points of disagreement. The resulting cost per QALY gained was £82,950. The NICE Appraisal Committee believed the analysis presented by the ERG to be more plausible, and likely to be an underestimate of the cost per QALY. They concluded that whilst the clinical effectiveness of cabazitaxel had been proven, it was not likely to represent a cost-effective use of NHS resources and so its use could not be recommended.
英国国家卫生与临床优化研究所（NICE）邀请卡巴他赛（商品名：Jevtana®，赛诺菲-安万特，英国）的制造商提交二线治疗转移性去势抵抗性前列腺癌（mHRPC）的临床和成本效益证据。谢菲尔德大学健康与相关研究技术评估小组（ScHARR-TAG）受委托担任独立证据审查小组（ERG）。该 ERG 根据制造商向 NICE 提交的内容，对该技术的临床和成本效益证据进行了批判性评价。临床证据源自一项多中心、开放性、III 期试验，纳入了既往接受多西他赛治疗后进展的 mHRPC 男性患者，比较卡巴他赛联合泼尼松或强的松与米托蒽醌联合泼尼松或强的松的疗效。卡巴他赛治疗与总生存期、无进展生存期和肿瘤进展时间的统计学显著改善相关。然而，它也与不良反应发生率增加相关，如中性粒细胞减少。效用数据基于卡巴他赛早期准入计划的中期结果。只有少数稳定疾病患者的数据可用，导致对卡巴他赛对生活质量影响的极大不确定性。在其经济评估中，制造商估计卡巴他赛的使用与每获得一个质量调整生命年（QALY）增加 74908 英镑的增量成本相关。然而，ERG 在两个关键方法学问题上与制造商存在分歧。第一个问题涉及生存模型和外推；第二个问题涉及患者人群的选择。ERG 改变了制造商的评估，以纳入这两个分歧点。由此产生的每获得一个 QALY 的成本为 82950 英镑。NICE 评估委员会认为，ERG 提出的分析更合理，可能低估了每 QALY 的成本。他们的结论是，尽管卡巴他赛的临床疗效已得到证实，但它不太可能成为 NHS 资源的成本效益使用，因此不能推荐其使用。
