The Institute for Clinical and Economic Review, Boston, MA, USA.
Department of Clinical Pharmacy, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.
Value Health. 2021 Jun;24(6):839-845. doi: 10.1016/j.jval.2021.02.008. Epub 2021 May 10.
To evaluate alternative methods to calculate and/or attribute economic surplus in the cost-effectiveness analysis of single or short-term therapies.
We performed a systematic literature review of articles describing alternative methods for cost-effectiveness analysis of potentially curative therapies whose assessment using traditional methods may suggest unaffordable valuations owing to the magnitude of estimated long-term quality-adjusted life-year (QALY) gains or cost offsets. Through internal deliberation and discussion with staff at the Health Technology Assessment bodies in England and Canada, we developed the following 3 alternative methods for further evaluation: (1) capping annual costs in the comparator arm at $150 000 per year; (2) "sharing" the economic surplus with the health sector by apportioning only 50% of cost offsets or 50% of cost offsets and QALY gains to the value of the therapy; and (3) crediting the therapy with only 12 years of the average annual cost offsets or cost offsets and QALY gains over the lifetime horizon. The impact of each alternative method was evaluated by applying it in an economic model of 3 hypothetical condition-treatment scenarios meant to reflect a diversity of chronicity and background healthcare costs.
The alternative with greatest impact on threshold price for the fatal pediatric condition spinal muscular atrophy type 1 was the 12-year cutoff scenario. For a hypothetical one-time treatment for hemophilia A, capping cost offsets at $150 000 per year had the greatest impact. For chimeric antigen receptor T-cell treatment of non-Hodgkin's lymphoma, capping cost offsets or using 12-year threshold had little impact, whereas 50% sharing of surplus including QALY gains and cost offsets greatly reduced threshold pricing.
Health Technology Assessment bodies and policy makers will wrestle with how to evaluate single or short-term potentially curative therapies and establish pricing and payment mechanisms to ensure sustainability. Scenario analyses using alternative methods for calculating and apportioning economic surplus can provide starkly different assessment results. These methods may stimulate important societal dialogue on fair pricing for these novel treatments.
评估用于单种或短期疗法成本效益分析的替代方法，以计算和/或归因经济剩余。
我们对描述潜在治愈疗法成本效益分析替代方法的文献进行了系统综述，这些方法采用传统方法评估时，由于长期估计质量调整生命年（QALY）收益或成本抵消的幅度，可能导致难以承受的估值。通过内部审议并与英国和加拿大卫生技术评估机构的工作人员进行讨论，我们开发了以下 3 种替代方法进行进一步评估：（1）将比较组的年度成本上限设定为每年 150000 美元；（2）通过仅将成本抵消的 50%或成本抵消和 QALY 收益的 50%分配给疗法的价值，与卫生部门“共享”经济剩余；（3）将疗法仅归功于平均每年成本抵消或终生成本抵消和 QALY 收益的 12 年。通过将每种替代方法应用于 3 种假设病情-治疗方案的经济模型来评估每种替代方法的影响，这些方案旨在反映出慢性程度和背景医疗保健成本的多样性。
对致命儿科疾病 1 型脊髓性肌萎缩症的阈值价格影响最大的替代方法是 12 年截止情景。对于假设的血友病 A 一次性治疗，将成本抵消上限设定为每年 150000 美元的影响最大。对于嵌合抗原受体 T 细胞治疗非霍奇金淋巴瘤，成本抵消上限或使用 12 年阈值的影响很小，而包括 QALY 收益和成本抵消在内的 50%剩余共享则大大降低了阈值定价。
卫生技术评估机构和决策者将努力探讨如何评估单种或短期潜在治愈疗法，并建立定价和支付机制以确保可持续性。使用替代方法计算和分配经济剩余的情景分析可以提供截然不同的评估结果。这些方法可能会激发关于这些新型疗法公平定价的重要社会对话。
