奥拉帕利单药维持治疗新诊断晚期BRCA基因突变上皮性卵巢癌、输卵管癌或原发性腹膜癌患者的药物经济学评价

Pharmacoeconomic evaluation of olaparib monotherapy in patients with newly diagnosed advanced BRCA mutation epithelial ovarian cancer,fallopian tube cancer, or primary peritoneal cancer

目的本研究基于医保支付方角度,评估奥拉帕利用于BRCA基因突变的晚期上皮性卵巢癌、输卵管癌或原发性腹膜癌初治成人患者在接受了一线含铂化疗后达到完全缓解或部分缓解的维持治疗的经济性。方法在Excel中构建分区生存模型,代入SOLO1研究亚洲人群的临床数据、效用数据和中国本土成本数据,评估患者接受了一线含铂化疗,达到完全缓解或部分缓解后,使用奥拉帕利单药维持治疗,观察等待的成本和健康产出,从而进一步计算增量成本效益比(incremental cost-effectiveness ratio,ICER)。并通过单因素敏感性分析和概率敏感性分析评估模型的稳健性。结果在基础分析中,奥拉帕利组平均治疗成本为386 134.80元,健康产出7.62质量调整生命年(quality-adjusted life years,QALYs);观察等待组平均治疗成本为204 971.53元,健康产出为5.56 QALYs,ICER为88 090.28元/QALYs,约为1.24倍人均GDP。单因素敏感性分析结果显示,在纳入分析的因素中,效果贴现率、总生存时间估计效应值、BRCA基因突变患者的额外死亡率对结果影响较大,其他因素对结果影响有限。概率敏感性分析结果显示,当阈值为3倍人均GDP时,相对观察等待,奥拉帕利具有经济性的概率为89.3%。敏感性分析结果显示模型较稳健。结论 BRCA基因突变的晚期上皮性卵巢癌、输卵管癌或原发性腹膜癌初治成人患者在接受一线含铂化疗,达到完全缓解或部分缓解后,使用奥拉帕利单药维持治疗相比观察等待具有经济性。

Objective To evaluate the cost-effectiveness of olaparib maintenance therapy in adult patients with BRCA-mutated advanced epithelial ovarian cancer,fallopian tube cancer or primary peritoneal cancer after complete or partial response with first-line platinum-based chemotherapy from a health insurance payment perspective.Methods Partitioned survival model was constructed in MS excel.Substituting SOLO1 into the model,the clinical data,utility data and local cost data of Asian population were studied.The incremental cost-effectiveness ratio(ICER) were calculated by evaluating the cost and health outcomes of olaparib-monotherapy maintenance versus watch-and-wait in patients who had achieved complete or partial response to first-line platinum-based chemotherapy.The robustness of the model was evaluated by deterministic sensitivity analysis(DSA) and probabilistic sensitivity analysis(PSA).Results In the basic analysis,the average treatment cost of olaparib group was 386134.80 yuan,and the average health outcome was 7.62 quality-adjusted life years(QALYs).The average treatment cost of watch-and-wait group was 204 871.53 yuan,health outcome was 5.56 QALYs,ICER was 88 090.28 yuan/QALYs,which was about 1.24 times GDP per capita.The results of DSA showed that among the factors included in the analysis,the discount rate of effect,the estimated effect value of OS,and the additional mortality of patients with BRCA mutations had a greater impact on the results,while other factors had a limited impact on the results.The results of PSA showed that compared with watch-and-wait,the probability of olaparib being cost-effective at a 3 times GDP per capita per QALYs threshold was 89.3%.The sensitivity analysis results showed that the model was robust.Conclusion Compared with watch-and-wait,maintenance therapy with olaparib monotherapy is a more cost-effective option for adult patients with BRCA-mutated advanced epithelial ovarian cancer,fallopian tube cancer or primary peritoneal cancer who have achieved complete or partial response to first-line platinum-based chemotherapy.