Erasmus School of Health Policy & Management, Erasmus University Rotterdam, , Rotterdam, The Netherlands
Department of Biostatistics, Erasmus MC, Rotterdam, The Netherlands.
BMJ Open. 2022 Apr 8;12(4):e058279. doi: 10.1136/bmjopen-2021-058279.
To evaluate the incremental value of new drugs across disease areas receiving favourable coverage decisions by the UK's National Institute for Health and Care Excellence (NICE) over the past decade.
DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional study assessed favourable appraisal decisions of drugs between 1 January 2010 and 31 December 2020. Estimates of incremental benefit were extracted from NICE's evidence review groups reports.
Incremental benefit of novel drugs relative to the best alternative therapeutic option, expressed in quality-adjusted life-years (QALYs).
184 appraisals of 129 drugs provided QALYs. The median incremental value was 0.27 QALY (IQR: 0.07-0.73). Benefits varied across drug-indication pairs (range: -0.49 to 5.22 QALY). The highest median benefits were found in haematology (0.70, IQR: 0.55-1.22) and oncology (0.46, IQR: 0.20-0.88), the lowest in ophthalmology (0.09, IQR: 0.04-0.22) and endocrinology (0.02, IQR: 0.01-0.06). Eight appraisals (4.3%) found contributions of more than two QALYs, but one in four (50/184) drug-indication pairs provided less than the equivalent of 1 month in perfect health compared to existing treatments.
In our review period, the median incremental value of novel drugs approved for use within the English National Health System, relative to the best alternative therapeutic option, was equivalent to 3-4 months of life in perfect health, but data were heterogeneous. Objective evaluations of therapeutic value helps patients and physicians to develop reasonable expectations of drugs and delivers insights into disease areas where medicinal therapeutic progress has had the most and least impact.
评估过去十年英国国家卫生与保健优化研究所（NICE）对各疾病领域内获得有利覆盖决策的新药的增量价值。
设计、设置和参与者：本横断面研究评估了 2010 年 1 月 1 日至 2020 年 12 月 31 日期间药物的有利评估决策。从 NICE 的证据审查小组报告中提取增量获益估计值。
新型药物相对于最佳替代治疗选择的增量获益，用质量调整生命年（QALY）表示。
184 项药物评估涉及 129 种药物，提供了 QALY 数据。中位增量值为 0.27 QALY（IQR：0.07-0.73）。药物-适应证对之间的获益存在差异（范围：-0.49 至 5.22 QALY）。最高的中位获益见于血液学（0.70，IQR：0.55-1.22）和肿瘤学（0.46，IQR：0.20-0.88），最低的见于眼科（0.09，IQR：0.04-0.22）和内分泌学（0.02，IQR：0.01-0.06）。8 项评估（4.3%）发现增量贡献超过 2 QALY，但四分之一（50/184）的药物-适应证对提供的获益相对于现有治疗方法，与完全健康状态下的 1 个月相当。
在我们的审查期间，相对于最佳替代治疗选择，在英国国家卫生系统内批准使用的新型药物的中位增量价值，相当于完全健康状态下 3-4 个月的生命，但数据存在异质性。对治疗价值的客观评估有助于患者和医生对药物产生合理的期望，并深入了解药物治疗最有成效和最无成效的疾病领域。
