Shi Guohai, Park Sang Hee, Ren Hongye, Xue Mei, Lu Xiaolin, Dong Peng, Gao Xin
a Department of Urology , Fudan University Shanghai Cancer Center , Shanghai , PR China.
b Department of Oncology , Shanghai Medical College, Fudan University , Shanghai , PR China.
J Med Econ. 2018 Dec;21(12):1150-1158. doi: 10.1080/13696998.2018.1515769. Epub 2018 Sep 10.
Targeted therapies, including sunitinib, sorafenib, axitinib, and everolimus, have recently become the mainstay for the treatment of metastatic renal cell carcinoma (mRCC). The objective of this study was to estimate the costs of sequential treatment regimens for mRCC and associated adverse events (AEs) from the Chinese payers' perspective.
Key inputs included in the calculation were patient population, dosing information, incidence rates and associated costs of Grade 3/4 AEs, treatment costs (including drug discount programs), and patients' progression-free survival (PFS) as a proxy for length of treatment. To calculate PFS, this study identified pivotal clinical trials and generated a reconstructed individual patient data set from the published Kaplan-Meier survival curves. The median PFS from the pooled estimates were used in the calculation. In the base-case scenario, sunitinib was used as first line and the other three therapies were used as second line. Sensitivity analyses were conducted where (1) sorafenib was used as first line, or (2) a third-line therapy was added to the base-case scenario.
In the base case, the cost per patient per treatment month (PPPM) cost was the lowest for sunitinib + axitinib among all sequential regimens (¥14,898) and was the highest for sunitinib + sorafenib (¥20,103). If sorafenib is used as first line, everolimus had lower per patient per months (PPPM) cost than axitinib (¥17,046 vs ¥23,337), but also had shorter PFS (13.5 months vs 15 months). Second sensitivity analysis with an additional third-line therapy showed consistent results with the base-case scenario; axitinib as second line was the least costly.
This study demonstrates that, for mRCC sequential treatment, sunitinib followed by axitinib generates the highest cost savings from the Chinese payers' perspective. Future studies are warranted to examine the cost-effectiveness of various mRCC treatment regimens in Chinese populations.
包括舒尼替尼、索拉非尼、阿昔替尼和依维莫司在内的靶向治疗最近已成为转移性肾细胞癌（mRCC）治疗的主要手段。本研究的目的是从中国支付方的角度估算mRCC序贯治疗方案及相关不良事件（AE）的成本。
计算中纳入的关键输入数据包括患者群体、给药信息、3/4级AE的发生率及相关成本、治疗成本（包括药物折扣计划），以及作为治疗时长替代指标的患者无进展生存期（PFS）。为计算PFS，本研究确定了关键临床试验，并根据已发表的Kaplan-Meier生存曲线生成了一个重建的个体患者数据集。计算中使用汇总估计值的中位PFS。在基础病例情景中，舒尼替尼用作一线治疗，其他三种疗法用作二线治疗。进行了敏感性分析，其中（1）索拉非尼用作一线治疗，或（2）在基础病例情景中增加三线治疗。
在基础病例中，所有序贯治疗方案中，舒尼替尼+阿昔替尼的每位患者每月治疗成本（PPPM）最低（14,898元），舒尼替尼+索拉非尼的成本最高（20,103元）。如果索拉非尼用作一线治疗，依维莫司的每位患者每月成本（PPPM）低于阿昔替尼（17,046元对23,337元），但PFS也较短（13.5个月对15个月）。增加三线治疗的第二项敏感性分析结果与基础病例情景一致；阿昔替尼作为二线治疗成本最低。
本研究表明，对于mRCC序贯治疗，从中国支付方的角度来看，舒尼替尼后接阿昔替尼可实现最高成本节约。未来有必要开展研究，以考察各种mRCC治疗方案在中国人群中的成本效益。
