Sandmann Frank G, Mostardt Sarah, Lhachimi Stefan K, Gerber-Grote Andreas
a Faculty of Epidemiology and Population Health , London School of Hygiene & Tropical Medicine , London , UK.
b Institute for Quality and Efficiency in Health Care (IQWiG) , Cologne , Germany.
Expert Rev Pharmacoecon Outcomes Res. 2018 Oct;18(5):475-486. doi: 10.1080/14737167.2018.1497976. Epub 2018 Jul 18.
The efficiency-frontier approach (EFA) to health economic evaluation aims to benchmark the relative efficiency of new drugs with the incremental cost-effectiveness ratios (ICERs) of non-dominated comparators. By explicitly considering any differences in health outcomes and costs, it enhances the internal reference pricing (IRP) policy that was officially endorsed by Germany as the first country worldwide in 1989. However, the EFA has been repeatedly criticized since its official endorsement in 2009. Areas covered: This perspective aims to stimulate the debate by discussing whether the main objections to the EFA are technically valid, irrespective of national contextual factors in Germany with reservations towards using cost-per-quality-adjusted life year (QALY) thresholds. Moreover, we comparatively assessed whether the objections are truly unique to the EFA or apply equally to IRP and cost-effectiveness thresholds. Expert commentary: The plethora of objections to the EFA (n = 20) has obscured that many objections are neither technically valid nor unique to the EFA. Compared with cost-effectiveness thresholds, only two objections apply uniquely to the EFA and concern intended key properties: (1) no external thresholds are needed and (2) the EFA is sensitive to price changes of comparators. Combining these policies and developing them further are under-utilized research areas.
健康经济评估中的效率前沿方法(EFA)旨在通过非劣效性对照药物的增量成本-效果比(ICER)来衡量新药的相对效率。通过明确考虑健康结果和成本的任何差异,它改进了内部参考定价(IRP)政策,该政策于1989年被德国作为全球首个国家正式批准。然而,自2009年正式批准以来,EFA一直受到反复批评。涵盖领域:本观点旨在通过讨论对EFA的主要反对意见在技术上是否有效来激发辩论,而不考虑德国在使用质量调整生命年(QALY)阈值方面持保留态度的国家背景因素。此外,我们比较评估了这些反对意见是否真的是EFA所独有的,还是同样适用于IRP和成本-效果阈值。专家评论:对EFA的大量反对意见(n = 20)掩盖了许多反对意见在技术上既无效也不是EFA所独有的这一事实。与成本-效果阈值相比,只有两个反对意见是EFA所独有的,并且涉及预期的关键特性:(1)不需要外部阈值;(2)EFA对对照药物的价格变化敏感。将这些政策结合起来并进一步发展是未得到充分利用的研究领域。