Centre for Health Economics, Monash Business School, Monash University, Clayton, VIC, 3800, Australia.
Centre for the Health Economy, Macquarie University, North Ryde, NSW, 2109, Australia.
Pharmacoeconomics. 2018 Mar;36(3):323-340. doi: 10.1007/s40273-017-0586-1.
There is much interest from stakeholders in understanding how health technology assessment (HTA) committees make national funding decisions for health technologies. A growing literature has analysed past decisions by committees (revealed preference, RP studies) and hypothetical decisions by committee members (stated preference, SP studies) to identify factors influencing decisions and assess their importance.
A systematic review of the literature was undertaken to provide insight into committee preferences for these factors (after controlling for other factors) and the methods used to elicit them.
Ovid Medline, Embase, Econlit and Web of Science were searched from inception to 11 May 2017. Included studies had to have investigated factors considered by HTA committees and to have conducted multivariate analysis to identify the effect of each factor on funding decisions. Factors were classified as being important based on statistical significance, and their impact on decisions was compared using marginal effects.
Twenty-three RP and four SP studies (containing 42 analyses) of 14 HTA committees met the inclusion criteria. Although factors were defined differently, the SP literature generally found clinical efficacy, cost-effectiveness and equity factors (such as disease severity) were each important to the Pharmaceutical Benefits Advisory Committee (PBAC), the National Institute for Health and Care Excellence (NICE) and the All Wales Medicines Strategy Group. These findings were supported by the RP studies of the PBAC, but not the other committees, which found funding decisions by these and other committees were mostly influenced by the acceptance of the clinical evidence and, where applicable, cost-effectiveness. Trust in the evidence was very important for decision makers, equivalent to reducing the incremental cost-effectiveness ratio (cost per quality-adjusted life-year) by A$38,000 (Australian dollars) for the PBAC and £15,000 for NICE.
This review found trust in the clinical evidence and, where applicable, cost-effectiveness were important for decision makers. Many methodological differences likely contributed to the diversity in some of the other findings across studies of the same committee. Further work is needed to better understand how competing factors are valued by different HTA committees.
利益相关者非常关注了解卫生技术评估（HTA）委员会如何为卫生技术做出国家资金决策。越来越多的文献分析了委员会过去的决策（显示性偏好，RP 研究）和委员会成员的假设决策（陈述性偏好，SP 研究），以确定影响决策的因素，并评估其重要性。
对文献进行系统回顾，深入了解委员会对这些因素的偏好（在控制其他因素后）以及得出这些因素的方法。
从 Ovid Medline、Embase、Econlit 和 Web of Science 数据库中检索从建库至 2017 年 5 月 11 日的文献。纳入的研究必须调查 HTA 委员会考虑的因素，并进行多变量分析以确定每个因素对资金决策的影响。根据统计学意义将因素归类为重要因素，并使用边际效应比较它们对决策的影响。
14 个 HTA 委员会中有 23 项 RP 研究和 4 项 SP 研究（包含 42 项分析）符合纳入标准。尽管因素的定义不同，但 SP 文献普遍发现临床疗效、成本效益和公平性因素（如疾病严重程度）对药品福利咨询委员会（PBAC）、英国国家卫生与临床优化研究所（NICE）和全威尔士药品策略小组均很重要。这些发现得到了 PBAC 的 RP 研究的支持，但其他委员会的研究则没有，这些委员会发现 PBAC 和其他委员会的资金决策主要受临床证据的接受程度以及在适用情况下的成本效益的影响。决策者对证据的信任非常重要，相当于将增量成本效益比（每质量调整生命年的成本）降低 38000 澳元（澳大利亚元），对于 PBAC 降低 15000 英镑（英国英镑），对于 NICE。
本综述发现决策者信任临床证据，在适用情况下，信任成本效益。对同一委员会的研究中，许多方法学差异可能导致一些其他发现的多样性。需要进一步研究，以更好地了解不同 HTA 委员会如何重视竞争因素。
