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在美国的卫生技术评估建议中,增量成本效益、背景因素考量和其他获益是如何被看待的?

How Are Incremental Cost-Effectiveness, Contextual Considerations, and Other Benefits Viewed in Health Technology Assessment Recommendations in the United States?

机构信息

Division of Health Policy and Management, Department of Public Sciences, University of California, Davis, Davis, CA, USA; Center for Healthcare Policy and Research, University of California, Davis, Davis, CA, USA.

Institute for Clinical and Economic Review, Boston, MA, USA.

出版信息

Value Health. 2020 May;23(5):576-584. doi: 10.1016/j.jval.2020.01.011. Epub 2020 Apr 1.

DOI:10.1016/j.jval.2020.01.011
PMID:32389223
Abstract

OBJECTIVES

To review assessments from the Institute for Clinical and Economic Review (ICER) and describe how cost-effectiveness, other benefits or disadvantages, and contextual considerations affect Council members' assessments of value.

METHODS

Assessments published by the ICER between December 2014 and April 2019 were reviewed. Data on the assessment, intervention, results from cost-effectiveness analyses, and Council members' votes were extracted. Voting data were examined using bar charts and radar plots. Spearman's correlations between the number of votes for other benefits and contextual considerations were estimated. Two case studies (tisagenlecleucel and voretigene neparvovec) explored the relationship between different aspects of value and the vote.

RESULTS

Thirty-one ICER assessments were reviewed, which included 51 value votes and 17 votes on other benefits and contextual considerations. On average, interventions with lower cost-effectiveness ratios received a higher proportion of high and intermediate value votes; however, there was heterogeneity across assessments. Of other benefits or disadvantages, having a novel mechanism of action received the most votes (n = 138), and reducing health disparities received the fewest (n = 24). Of contextual considerations, treating a condition that has a severe impact on length and quality of life received the most votes (n = 164). There was a strong positive correlation between votes for reduced caregiver/family burden and improving return to work/productivity (ρ = 0.88, P < .05). Two case studies highlighted that factors beyond cost-effectiveness can lead to lower (tisagenlecleucel) or higher (voretigene neparvovec) assessments of value.

CONCLUSION

Council members' judgments about the value of interventions are influenced by other benefits or disadvantages and contextual considerations but anchored by cost-effectiveness.

摘要

目的

回顾临床与经济评价研究所(ICER)的评估,并描述成本效益、其他获益或劣势以及背景因素如何影响委员会成员对价值的评估。

方法

回顾了 2014 年 12 月至 2019 年 4 月间发表的 ICER 评估。提取了评估、干预措施、成本效益分析结果以及委员会成员投票的数据。使用柱状图和雷达图检查投票数据。估计了其他获益和背景因素的投票数量之间的 Spearman 相关系数。通过两个案例研究(tisagenlecleucel 和 voretigene neparvovec)探讨了不同方面的价值与投票之间的关系。

结果

共审查了 31 项 ICER 评估,其中包括 51 项价值投票、17 项关于其他获益和背景因素的投票。一般来说,成本效益比低的干预措施获得高和中等价值投票的比例更高;然而,评估之间存在异质性。在其他获益或劣势方面,具有新颖作用机制的干预措施获得的投票最多(n=138),而减少健康差距的干预措施获得的投票最少(n=24)。在背景因素方面,治疗对生活质量和寿命有严重影响的疾病获得的投票最多(n=164)。减少照顾者/家庭负担和提高重返工作/生产力的投票之间存在很强的正相关(ρ=0.88,P<0.05)。两个案例研究强调,成本效益之外的因素可能导致价值评估降低(tisagenlecleucel)或提高(voretigene neparvovec)。

结论

委员会成员对干预措施价值的判断受到其他获益或劣势以及背景因素的影响,但以成本效益为基础。

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