Shaw James W, Bennett Bryan, Trigg Andrew, DeRosa Michael, Taylor Fiona, Kiff Christopher, Ntais Dionysios, Noon Katie, King Madeleine T, Cocks Kim
Worldwide Health Economics and Outcomes Research, Bristol Myers Squibb, Princeton, NJ, USA.
Bristol Myers Squibb Pharmaceuticals Ltd, Uxbridge, England, UK.
Value Health. 2021 Nov;24(11):1651-1659. doi: 10.1016/j.jval.2021.05.022. Epub 2021 Aug 26.
There is growing interest in condition-specific preference measures, including the European Organisation for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (QLU-C10D). This research assessed the implications of using utility indices on the basis of the EQ-5D-3L, a mapping of EQ-5D-3L to the EQ-5D-5L, and the QLU-C10D, and compared their psychometric properties.
Data were taken from 8 phase 3 randomized controlled trials of nivolumab with or without ipilimumab for the treatment of solid tumors. Utilities for progression-related states were calculated using the UK and English value sets and incremental quality-adjusted life-years (QALYs) derived from established UK cost-effectiveness models. The psychometric properties of the utility indices were assessed using pooled trial data.
Compared with the EQ-5D-3L index, the mapped EQ-5D-5L index yielded an average of 6% more and the QLU-C10D index an average of 2% fewer incremental QALYs for nivolumab versus comparators. All indices could differentiate between groups defined by performance status, cancer stage, or self-reported health status at baseline and detect meaningful changes in performance status, tumor response, health status, and quality of life over approximately 12 weeks of treatment.
The lower QALY yield of the QLU-C10D was balanced by evidence of greater validity and responsiveness. Benefits gained from using the QLU-C10D may be apparent when treatments affect targeted symptoms and functional aspects, including sleep, bowel function, appetite, nausea, and fatigue. The observed differences in QALYs may not be sufficiently large to affect health technology assessment decisions.
针对特定疾病的偏好测量方法,包括欧洲癌症研究与治疗组织生活质量效用测量核心10维度量表(QLU-C10D),人们的兴趣日益浓厚。本研究评估了基于EQ-5D-3L、EQ-5D-3L到EQ-5D-5L的映射以及QLU-C10D使用效用指数的影响,并比较了它们的心理测量特性。
数据取自8项关于纳武单抗联合或不联合伊匹木单抗治疗实体瘤的3期随机对照试验。使用英国和英语价值集以及从已建立的英国成本效益模型得出的增量质量调整生命年(QALY)计算与疾病进展相关状态的效用。使用汇总的试验数据评估效用指数的心理测量特性。
与EQ-5D-3L指数相比,映射后的EQ-5D-5L指数得出的纳武单抗相对于对照的增量QALY平均多6%,而QLU-C10D指数平均少2%。所有指数都能区分由基线时的体能状态、癌症分期或自我报告的健康状态所定义的组,并能检测出在大约12周的治疗期间体能状态、肿瘤反应、健康状态和生活质量的有意义变化。
QLU-C10D较低的QALY产出被更强的有效性和反应性证据所平衡。当治疗影响包括睡眠、肠道功能、食欲、恶心和疲劳等靶向症状和功能方面时,使用QLU-C10D所获得的益处可能会很明显。观察到的QALY差异可能不够大,不足以影响卫生技术评估决策。