我国抗肿瘤药物超说明书用药价值评价模型的探索性实证研究

An exploratory empirical study on the value assessment model for off-label use of antineoplastic agents in China

目的:评价我国抗肿瘤药物超说明书用药价值评价模型(以下简称"中国模型")的科学性、实用性和可行性。方法:以卵巢癌为例,涵盖目前我院备案使用的5个药物,10个超说明书用药治疗方案。每位小组成员使用中国模型、美国临床肿瘤学会价值框架(American Society of Clinical Oncology Value Framework,ASCO-VF)、欧洲肿瘤内科学会临床获益量表(European Society for Medical Oncology Magnitude of Clinical Benefit Scale,ESMO-MCBS)对其进行评价。计算各药物治疗方案的平均得分和标准差,肯德尔和谐系数(Kendall’s coefficient of concordance,Kendall’s W)和组内相关系数(intraclass correlation efficient,ICC)评估聚合效度和评分者间信度。使用SPSS 25.0软件进行分析。结果:盐酸多柔比星脂质体联合卡铂用于卵巢癌一线和维持治疗的价值评价结果得分最高。3种药物价值评价工具的Kendall’s W为0.706,P=0.048,两两比较时,中国模型与ESMO-MCBS组最高,中国模型与ASCO-VF组最低。3种工具中的临床获益Kendall’s W(0.778,P=0.030)最高,不良反应最低(0.302,P=0.490)。中国模型、ASCO-VF、ESMO-MCBS的ICC和95%CIs分别为0.633(0.410,0.860)、0.918(0.806,0.983)、0.827(0.634,0.960)。结论:我国抗肿瘤药物超说明书用药价值评价模型在预测抗肿瘤药物超说明书用药价值方面具有较高的可靠性和可行性,值得推广应用。

OBJECTIVE To evaluate the scientificity,practicality and feasibility of the value assessment model for off-label use of antineoplastic agents in China(“China model”for short).METHODS Taking ovarian cancer as an example,we selected 5 drugs registered and used in our hospital,including 10 therapeutic regimens of off-label uses.Each panelist used three value assessment tools including China model,American Society of Clinical Oncology Value Framework(ASCO-VF)and European Society for Medical Oncology Magnitude of Clinical Benefit Scale(ESMO-MCBS)to assess the same therapeutic regimens.We counted the average score and standard deviation of each therapeutic regimen and evaluated the convergent validity and interrater reliability through Kendall’s coefficient of concordance(Kendall’s W)and intraclass correlation efficient(ICC).SPSS 25.0 software was used for analysis.RESULTS The overall value score of doxorubicin hydrochloride liposome combined with carboplatin for first-line and maintenance treatment of ovarian cancer was the highest for each tool.Kendall’s W was 0.706,P=0.048 across three tools,highest for China model vs.ESMO-MCBS and lowest for China model vs.ASCO-VF for pairwise.Kendall’s W was highest for clinical benefit(0.778,P=0.030)and lowest for adverse reactions(0.302,P=0.490)for each tool.ICC and 95%CIs of panelists’assessments were 0.633(0.410,0.860)for China model,0.918(0.806,0.983)for ASCO-VF,and 0.827(0.634,0.960)for ESMO-MCBS respectively.CONCLUSION The value assessment model for off-label use of antineoplastic agents in China has high reliability and operability in predicting the value of off-label uses for antineoplastic agents,which is worthy of popularization and application.