Jiang Qian, Feng Mei, Li Youping, Lang Jinyi, Wei Hua, Yu Ting
Sichuan Cancer Hospital and Institute, Sichuan Cancer Centre, School of Medicine, University of Electronic Science and Technology of China, Chengdu, China.
China Chinese Evidence-Based Medicine Centre, West China Hospital, Sichuan University, Chengdu, China.
Front Pharmacol. 2020 Dec 18;11:574511. doi: 10.3389/fphar.2020.574511. eCollection 2020.
Influx of innovative therapies and dramatic rise in prices have been prompting value-driven decision-making. Both the American Society of Clinical Oncology (ASCO) and the European Society for Medical Oncology (ESMO) have independently proposed value assessment frameworks. To comprehensively examine the value of nivolumab and pembrolizumab by two value assessment frameworks with a cohort of published randomized controlled trials and offer insight into the association between these two frameworks. Trials were identified with a cutoff date of Nov 30th, 2019. Receiver operating characteristic curves were generated to establish the predictive value of ASCO-VF score to meet ESMO-MCBS grade and discriminate the agreement of these two value assessment tools. Spearman correlation was used to assess the association between monthly cost and ASCO-VF score/ESMO-MCBS grade. 19 randomized controlled trials were eligible. seven (36.8%) trials were of treatment included nivolumab while 12 (63.2%) pembrolizumab. 8 (42.1%) of the trials were of treatments for non-small-cell lung cancer, 5 (26.3%) for melanoma, 2 (10.5%) were for head and neck squamous cell carcinoma, 2 (10.5%) for gastric or gastro-oesophageal junction cancer and 1 (5.3%) for urothelial cancer and renal-cell carcinoma respectively. ASCO scores ranged from 7 to 94.7 with median 40.90. 11 (57.9%) trials met the ESMO criteria for meaningful value achieved. Of 14 trials not meeting the ASCO cutoff score, only 8 did not meet the meaningful ESMO criteria. Agreement between these two frameworks thresholds was only fair ( = 0.412, ＜0.05). A negative correlation was noted between increment monthly cost and value assessment results. There is only fair correlation between ASCO and ESMO value assessment frameworks. Not all treatment with nivolumab and pembrolizumab meet valuable thresholds.
创新疗法的涌入和价格的急剧上涨促使基于价值的决策制定。美国临床肿瘤学会（ASCO）和欧洲医学肿瘤学会（ESMO）都独立提出了价值评估框架。通过两个价值评估框架，利用一组已发表的随机对照试验全面检验纳武利尤单抗和帕博利珠单抗的价值，并深入了解这两个框架之间的关联。试验的截止日期为2019年11月30日。生成受试者操作特征曲线以确定ASCO-VF评分对达到ESMO-MCBS等级的预测价值，并区分这两种价值评估工具的一致性。采用Spearman相关性分析评估每月成本与ASCO-VF评分/ESMO-MCBS等级之间的关联。19项随机对照试验符合条件。7项（36.8%）试验的治疗药物为纳武利尤单抗，12项（63.2%）为帕博利珠单抗。8项（42.1%）试验用于治疗非小细胞肺癌，5项（26.3%）用于治疗黑色素瘤，2项（10.5%）用于治疗头颈部鳞状细胞癌，2项（10.5%）用于治疗胃或胃食管交界癌，1项（5.3%）分别用于治疗尿路上皮癌和肾细胞癌。ASCO评分范围为7至94.7，中位数为40.90。11项（57.9%）试验达到了ESMO关于实现有意义价值的标准。在14项未达到ASCO截止评分的试验中，只有8项未达到有意义的ESMO标准。这两个框架阈值之间的一致性仅为中等（ = 0.412，＜0.05）。每月成本增加与价值评估结果之间存在负相关。ASCO和ESMO价值评估框架之间的相关性仅为中等。并非所有使用纳武利尤单抗和帕博利珠单抗的治疗都达到了有价值的阈值。
