Pal Sumanta K, Jonasch Eric, Signorovitch James E, Reichmann William M, Li Nanxin, Liu Zhimei, Perez Jose Ricardo, Vogelzang Nicholas J
a a City of Hope Comprehensive Cancer Center , Duarte , CA , USA.
b b MD Anderson Cancer Center , Houston , TX , USA.
J Med Econ. 2016;19(5):462-8. doi: 10.3111/13696998.2015.1131705. Epub 2016 Jan 11.
To describe dosing patterns and to compare the drug costs per month spent in progression-free survival (PFS) among patients with advanced renal cell carcinoma (aRCC) treated with everolimus or axitinib following a first tyrosine kinase inhibitor (TKI).
A medical record retrospective review was conducted among medical oncologists and hematologists/oncologists in the US. Patient eligibility criteria included: (1) age ≥18 years; (2) discontinuation of first TKI (sunitinib, sorafenib, or pazopanib) for medical reasons; (3) initiation of axitinib or everolimus as a second targeted therapy during February 2012-January 2013. Real-world dosing patterns were summarized. Dose-specific drug costs (as of October 2014) were based on wholesale acquisition costs from RED BOOK Online. PFS was compared between everolimus and axitinib using a multivariable Cox proportion hazards model. Everolimus and axitinib drug costs per month of PFS were compared using multivariable gamma regression models.
A total of 325 patients received everolimus and 127 patients received axitinib as second targeted therapy. Higher proportions of patients treated with axitinib vs everolimus started on a higher than label-recommended starting dose (14% vs 2%) or experienced dose escalation (11% vs 1%) on second targeted therapy. The PFS did not differ significantly between patients receiving everolimus or axitinib (adjusted hazard ratio (HR) = 1.16; 95% confidence interval [CI] = 0.73-1.82). After baseline characteristics adjustment, axitinib was associated with 17% ($1830) higher drug costs per month of PFS compared to everolimus ($12,467 vs $10,637; p < 0.001).
Retrospective observational study design and only drug acquisition costs considered in drug costs estimates.
Patients with aRCC receiving axitinib as second targeted therapy were more likely to initiate at a higher than label-recommended dose and were more likely to dose escalate than patients receiving everolimus. With similar observed durations of PFS, drug costs were significantly higher-by 17% per month of PFS-with axitinib than with everolimus.
描述给药模式，并比较在一线酪氨酸激酶抑制剂（TKI）治疗后接受依维莫司或阿昔替尼治疗的晚期肾细胞癌（aRCC）患者无进展生存期（PFS）期间每月的药物成本。
在美国的医学肿瘤学家和血液肿瘤学家中进行了一项病历回顾性研究。患者入选标准包括：（1）年龄≥18岁；（2）因医学原因停用一线TKI（舒尼替尼、索拉非尼或帕唑帕尼）；（3）在2012年2月至2013年1月期间开始使用阿昔替尼或依维莫司作为二线靶向治疗。总结了真实世界的给药模式。特定剂量的药物成本（截至2014年10月）基于《RED BOOK Online》的批发采购成本。使用多变量Cox比例风险模型比较依维莫司和阿昔替尼的PFS。使用多变量伽马回归模型比较依维莫司和阿昔替尼PFS每月的药物成本。
共有325例患者接受依维莫司作为二线靶向治疗，127例患者接受阿昔替尼作为二线靶向治疗。与接受依维莫司治疗的患者相比，接受阿昔替尼治疗的患者中，更高比例的患者开始使用高于标签推荐的起始剂量（14%对2%）或在二线靶向治疗中出现剂量增加（11%对1%）。接受依维莫司或阿昔替尼治疗的患者的PFS无显著差异（调整后风险比（HR）=1.16；95%置信区间[CI]=0.73-1.82）。在对基线特征进行调整后，与依维莫司相比，阿昔替尼PFS每月的药物成本高17%（1830美元）（12467美元对10637美元；p<0.001）。
回顾性观察性研究设计，且药物成本估计仅考虑了药物采购成本。
与接受依维莫司治疗的患者相比，接受阿昔替尼作为二线靶向治疗的aRCC患者更有可能以高于标签推荐的剂量开始治疗，且更有可能增加剂量。在观察到的PFS持续时间相似的情况下，阿昔替尼的药物成本比依维莫司显著更高——PFS每月高17%。
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