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概率效率前沿:德国成本效益分析框架在丙型肝炎治疗方案中的实际应用

The Probabilistic Efficiency Frontier: A Framework for Cost-Effectiveness Analysis in Germany Put into Practice for Hepatitis C Treatment Options.

作者信息

Mühlbacher Axel C, Sadler Andrew

机构信息

Gesundheitsökonomie und Medizinmanagement, Hochschule Neubrandenburg, Neubrandenburg, Germany.

Gesundheitsökonomie und Medizinmanagement, Hochschule Neubrandenburg, Neubrandenburg, Germany.

出版信息

Value Health. 2017 Feb;20(2):266-272. doi: 10.1016/j.jval.2016.12.015. Epub 2017 Feb 16.

DOI:10.1016/j.jval.2016.12.015
PMID:28237207
Abstract

BACKGROUND

The German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen) adapted the efficiency frontier (EF) approach to conform to statutory provisions on cost-effectiveness analysis of health technologies. EF serves as a framework for evaluating cost-effectiveness and indirectly for pricing and reimbursement decisions.

OBJECTIVES

To calculate an EF on the basis of single multidimensional benefit by taking patient preferences and uncertainty into account; to evaluate whether EF is useful to inform decision makers about cost-effectiveness of new therapies; and to find whether a treatment is efficient at given prices demonstrated through a case study on chronic hepatitis C.

METHODS

A single multidimensional benefit was calculated by linear additive aggregation of multiple patient-relevant end points. End points were identified and weighted by patients in a previous discrete-choice experiment (DCE). Aggregation of overall benefit was ascertained using preferences and clinical data. Monte-Carlo simulation was applied. Uncertainty was addressed by price acceptability curve (PAC) and net monetary benefit (NMB).

RESULTS

The case study illustrates that progress in benefit and efficiency of hepatitis C virus treatments could be depicted very well with the EF. On the basis of cost, effect, and preference data, the latest generations of interferon-free treatments are shown to yield a positive NMB and be efficient at current prices.

CONCLUSIONS

EF was implemented taking uncertainty into account. For the first time, a DCE was used with the EF. The study shows how DCEs in combination with EF, PAC, and NMB can contribute important information in the course of reimbursement and pricing decisions.

摘要

背景

德国医疗质量与效率研究所(Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen)调整了效率前沿(EF)方法,以符合关于卫生技术成本效益分析的法定规定。EF作为评估成本效益的框架,并间接用于定价和报销决策。

目的

通过考虑患者偏好和不确定性,基于单一多维效益计算EF;评估EF是否有助于告知决策者新疗法的成本效益;通过慢性丙型肝炎的案例研究,确定在给定价格下一种治疗是否有效。

方法

通过对多个与患者相关的终点进行线性加总来计算单一多维效益。终点在之前的离散选择实验(DCE)中由患者确定并加权。使用偏好和临床数据确定总体效益的加总。应用蒙特卡洛模拟。通过价格可接受性曲线(PAC)和净货币效益(NMB)解决不确定性问题。

结果

案例研究表明,EF能够很好地描述丙型肝炎病毒治疗在效益和效率方面的进展。基于成本、效果和偏好数据,最新一代的无干扰素治疗显示出正的NMB,并且在当前价格下是有效的。

结论

实施EF时考虑了不确定性。首次将DCE与EF结合使用。该研究表明,DCE与EF、PAC和NMB相结合如何能够在报销和定价决策过程中提供重要信息。

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