背景:贝伐单抗已被证明在治疗脑放射性坏死(CRN)方面具有优异的疗效,但它的高成本可能会加剧疾病负担。本研究旨在从美国付款人的角度评估贝伐单抗与皮质类固醇治疗CRN的成本效益。
方法:构建决策树模型来模拟贝伐单抗和皮质类固醇在CRN短期和长期治疗中的过程。关键的临床数据来自NCT01621880试验。成本和效用值来自美国官方网站和已发表的文献。主要结果是总成本,质量调整寿命年(QALYs),和增量成本效益比(ICER)。进行单向和概率敏感性分析以评估模型的稳健性。
结果:在短期和长期模型中,与皮质类固醇治疗相比,贝伐单抗增加了0.11(0.46vs0.35)和0.16(0.54vs0.38)的QALYs,相应的增量成本分别为12,351美元和23,253美元。由此产生的ICER短期和长期治疗为$112,987/QALY和$150,245/QALY,分别。单向敏感性分析表明,无复发状态的效用值,体重和每周期贝伐单抗的价格是两个模型中ICER的最大影响因素.在美国15万美元/QALY的支付意愿门槛下,贝伐单抗在CRN短期和长期治疗中具有成本效益的概率分别为63.9%和49%,分别。
结论:与皮质类固醇相比,从美国付款人的角度来看,贝伐单抗是CRN短期治疗的一种经济替代方案,而长期治疗却得出了相反的结论。
BACKGROUND: Bevacizumab has been demonstrated to have superior efficacy in the treatment of cerebral radiation necrosis (CRN), but its\' high cost may exacerbate the disease burden. This study aimed to assess the cost-effectiveness of bevacizumab in comparison with corticosteroids for treating CRN from the US payers\' perspective.
METHODS: Decision tree models were constructed to simulate the process of bevacizumab and corticosteroids in CRN short-term and long-term therapy. Critical clinical data were derived from the NCT01621880 trial. Costs and utility values were obtained from the US official websites and published literatures. The main outcomes were total costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). One-way and probabilistic sensitivity analyses were performed to assess the robustness of models.
RESULTS: In the short-term and long-term models, bevacizumab added 0.11 (0.46 vs 0.35) and 0.16 (0.54 vs 0.38) QALYs compared with corticosteroids therapy, with corresponding incremental costs of $12,351 and $23,253, respectively. The resultant ICERs were $112,987/QALY and $150,245/QALY for short-term and long-term treatment, respectively. The one-way sensitivity analysis indicated that utility value of non-recurrence status, body weight and bevacizumab price per cycle were the most influential factors for ICER of both models. At the willingness-to-pay threshold of $150,000/QALY in the US, the probabilities of bevacizumab being cost-effective for CRN short and long-term treatment were 63.9% and 49%, respectively.
CONCLUSIONS: Compared with corticosteroids, bevacizumab is an economical alternative for CRN short-term treatment from the US payers\' perspective, while long-term therapy draws an opposite conclusion.