Cristino Joaquim, Finek Jíndřich, Jandova Petra, Kolek Martin, Pásztor Bálint, Giannopoulou Christina, Qian Yi, Brezina Tomas, Lothgren Mickael
a Amgen (Europe) GmbH , Zug , Switzerland.
b Pilsen Faculty Hospital, Clinic of Oncology and Radiotherapy (FN v Plzni) , Pilsen , Czech Republic.
J Med Econ. 2017 Aug;20(8):799-812. doi: 10.1080/13696998.2017.1328423. Epub 2017 Jun 7.
This study assessed the cost-effectiveness of the subcutaneous RANKL inhibitor, denosumab, vs the intravenous bisphosphonate, zoledronic acid, for the prevention of skeletal-related events (SREs) in patients with prostate cancer, breast cancer, and other solid tumors (OST) in the Czech Republic.
A lifetime Markov model was developed to compare the effects of denosumab and zoledronic acid on costs (including drug costs and administration, patient management, SREs, and adverse events), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios from a national payer perspective. Different discount rates, time horizons, SRE rates, distributions, and nature (asymptomatic vs all SREs), and the inclusion of treatment discontinuation were considered in scenario analyses. The robustness of the model was tested using deterministic and probabilistic sensitivity analyses.
Across tumor types, denosumab was associated with fewer SREs, improved QALYs, and higher total costs over a lifetime. The incremental cost per QALY gained for denosumab vs zoledronic acid was 382,673 CZK for prostate cancer, 408,450 CZK for breast cancer, and 608,133 CZK for OST. Incremental costs per SRE avoided for the same tumor type were 54,007 CZK, 51,765 CZK, and 94,426 CZK, respectively. In scenario analyses, the results remained similar to baseline, when different discount rates and time horizons were considered. At a non-official willingness-to-pay threshold of 1.2 million CZK, the probabilities of denosumab being cost-effective vs zoledronic acid were 0.64, 0.67, and 0.49 for prostate cancer, breast cancer, and OST, respectively.
The SRE rates used were obtained from clinical trials; studies suggest rates may be higher in clinical practice. Additional evidence on real-world SRE rates could further improve the accuracy of the modeling.
Compared with zoledronic acid, denosumab provides a cost-effective treatment option for the prevention of SREs in patients with prostate cancer, breast cancer, and OST in the Czech Republic.
本研究评估皮下注射RANKL抑制剂地诺单抗与静脉注射双膦酸盐唑来膦酸相比，在捷克共和国预防前列腺癌、乳腺癌和其他实体瘤（OST）患者骨骼相关事件（SRE）方面的成本效益。
建立终身马尔可夫模型，从国家支付方的角度比较地诺单抗和唑来膦酸对成本（包括药物成本、给药、患者管理、SRE和不良事件）、质量调整生命年（QALY）以及增量成本效益比的影响。在情景分析中考虑了不同的贴现率、时间范围、SRE发生率、分布和性质（无症状与所有SRE）以及治疗中断情况。使用确定性和概率敏感性分析测试模型的稳健性。
在所有肿瘤类型中，地诺单抗在一生中与更少的SRE、改善的QALY和更高的总成本相关。地诺单抗相对于唑来膦酸每获得一个QALY的增量成本，前列腺癌为382,673捷克克朗，乳腺癌为408,450捷克克朗，OST为608,133捷克克朗。相同肿瘤类型下每避免一次SRE的增量成本分别为54,007捷克克朗、51,765捷克克朗和94,426捷克克朗。在情景分析中，当考虑不同的贴现率和时间范围时，结果与基线相似。在非官方支付意愿阈值为120万捷克克朗时，地诺单抗相对于唑来膦酸具有成本效益的概率，前列腺癌为0.64，乳腺癌为0.67，OST为0.49。
所使用的SRE发生率来自临床试验；研究表明临床实践中的发生率可能更高。关于实际SRE发生率的更多证据可进一步提高模型的准确性。
与唑来膦酸相比，地诺单抗为捷克共和国前列腺癌、乳腺癌和OST患者预防SRE提供了一种具有成本效益的治疗选择。
