Bullement Ash, Nathan Paul, Willis Anna, Amin Amerah, Lilley Cameron, Stapelkamp Ceilidh, Hatswell Anthony, Pescott Chris, Bharmal Murtuza
BresMed, Sheffield, UK.
Delta Hat, Nottingham, UK.
Pharmacoecon Open. 2019 Sep;3(3):377-390. doi: 10.1007/s41669-018-0115-y.
Metastatic Merkel cell carcinoma (mMCC) is a rare and aggressive skin cancer. Until recently, there were no licensed treatment options for patients with mMCC, and prognosis was poor. A cost-effectiveness analysis was conducted for avelumab, a newly available treatment option for mMCC, versus standard care (SC), from a UK National Health Service perspective.
A partitioned survival model was developed to assess the lifetime costs and effects of avelumab versus SC. Data from the JAVELIN Merkel 200 trial (NCT02155647) were used to inform estimates of quality-adjusted life-years (QALYs). Unit costs and associated frequencies of use were informed by published literature and clinical expert opinion. Results were presented as incremental cost-effectiveness ratios (ICERs, i.e. the cost per QALY gained) for treatment-experienced (TE) and treatment-naïve (TN) patients. Uncertainty was explored through a range of sensitivity analyses.
Discounting costs and QALYs at 3.5% per annum, avelumab was associated with ICERs of £35,274 (TE)/£39,178 (TN) per QALY gained. Probabilistic sensitivity analysis results demonstrated that avelumab was associated with an 88.3% (TE)/69.3% (TN) probability of being cost effective at a willingness-to-pay threshold for end-of-life treatments of £50,000 per QALY gained. Results were most sensitive to alternative survival extrapolations and dosing assumptions.
The analysis results suggest that avelumab is likely to be a cost-effective treatment option for UK mMCC patients. The results for TN patients are subject to some uncertainty, and a confirmatory analysis will be conducted with more mature data.
转移性默克尔细胞癌（mMCC）是一种罕见且侵袭性强的皮肤癌。直到最近，对于mMCC患者还没有获批的治疗方案，且预后较差。从英国国家医疗服务体系的角度，对阿维鲁单抗（一种新的mMCC治疗选择）与标准治疗（SC）进行了成本效益分析。
建立了一个分区生存模型，以评估阿维鲁单抗与SC的终身成本和效果。来自JAVELIN Merkel 200试验（NCT02155647）的数据用于估计质量调整生命年（QALY）。单位成本和相关使用频率由已发表的文献和临床专家意见提供。结果以治疗经验丰富（TE）和初治（TN）患者的增量成本效益比（ICER，即每获得一个QALY的成本）呈现。通过一系列敏感性分析探讨不确定性。
按每年3.5%对成本和QALY进行贴现，阿维鲁单抗每获得一个QALY的ICER为35,274英镑（TE）/39,178英镑（TN）。概率敏感性分析结果表明，在每获得一个QALY的临终治疗支付意愿阈值为50,000英镑时，阿维鲁单抗具有成本效益的概率为88.3%（TE）/69.3%（TN）。结果对替代生存外推法和给药假设最为敏感。
分析结果表明，阿维鲁单抗可能是英国mMCC患者具有成本效益的治疗选择。TN患者的结果存在一定不确定性，将用更成熟的数据进行验证性分析。
