Mühlbacher Axel C, Sadler Andrew
Gesundheitsökonomie und Medizinmanagement, Hochschule Neubrandenburg, Neubrandenburg.
Gesundheitswesen. 2019 Jan;81(1):e21-e32. doi: 10.1055/s-0043-107235. Epub 2017 Jun 19.
The German Institute for Quality and Efficiency in Health Care (IQWiG) recommends the concept of the efficiency frontier to assess health care interventions. The efficiency frontier supports regulatory decisions on reimbursement prices for the appropriate allocation of health care resources. Until today this cost-benefit assessment framework has only been applied on the basis of individual patient-relevant endpoints. This contradicts the reality of a multi-dimensional patient benefit.
The objective of this study was to illustrate the operationalization of multi-dimensional benefit considering the uncertainty in clinical effects and preference data in order to calculate the efficiency of different treatment options for hepatitis C (HCV). This case study shows how methodological challenges could be overcome in order to use the efficiency frontier for economic analysis and health care decision-making.
The operationalization of patient benefit was carried out on several patient-relevant endpoints. Preference data from a discrete choice experiment (DCE) study and clinical data based on clinical trials, which reflected the patient and the clinical perspective, respectively, were used for the aggregation of an overall benefit score. A probabilistic efficiency frontier was constructed in a Monte Carlo simulation with 10000 random draws. Patient-relevant endpoints were modeled with a beta distribution and preference data with a normal distribution. The assessment of overall benefit and costs provided information about the adequacy of the treatment prices. The parameter uncertainty was illustrated by the price-acceptability-curve and the net monetary benefit.
Based on the clinical and preference data in Germany, the interferon-free treatment options proved to be efficient for the current price level. The interferon-free therapies of the latest generation achieved a positive net cost-benefit. Within the decision model, these therapies showed a maximum overall benefit. Due to their high additional benefit and approved prices, the therapies lie above of the extrapolated efficiency frontier, which suggests that these options have efficient reimbursement prices. Considering uncertainty, even a higher price would have resulted in a positive cost-benefit ratio.
IQWiG's efficiency frontier was used to assess the value of different treatment options in HCV. This study demonstrates that the probabilistic efficiency frontier, price-acceptability-curve and the net monetary benefit can contribute essential information to reimbursement decisions and price negotiations.
德国医疗质量与效率研究所（IQWiG）推荐使用效率前沿概念来评估医疗保健干预措施。效率前沿为医疗资源合理分配的报销价格监管决策提供支持。迄今为止，这种成本效益评估框架仅基于与个体患者相关的终点进行应用。这与多维患者获益的现实情况相矛盾。
本研究的目的是说明考虑临床效果和偏好数据中的不确定性来实现多维获益的操作方法，以便计算丙型肝炎（HCV）不同治疗方案的效率。本案例研究展示了如何克服方法学挑战，以便将效率前沿用于经济分析和医疗保健决策。
在多个与患者相关的终点上实现患者获益的操作化。分别来自离散选择实验（DCE）研究的偏好数据和基于临床试验的临床数据，反映了患者和临床视角，用于汇总总体获益得分。在蒙特卡罗模拟中进行10000次随机抽样构建概率效率前沿。与患者相关的终点用贝塔分布建模，偏好数据用正态分布建模。总体获益和成本的评估提供了有关治疗价格合理性的信息。通过价格可接受性曲线和净货币获益说明了参数不确定性。
基于德国的临床和偏好数据，目前价格水平下无干扰素治疗方案被证明是有效的。最新一代的无干扰素疗法实现了正的净成本效益。在决策模型中，这些疗法显示出最大的总体获益。由于其高额外获益和获批价格，这些疗法位于外推效率前沿之上，这表明这些方案具有有效的报销价格。考虑到不确定性，即使更高的价格也会导致正的成本效益比。
IQWiG的效率前沿用于评估HCV不同治疗方案的价值。本研究表明，概率效率前沿、价格可接受性曲线和净货币获益可为报销决策和价格谈判提供重要信息。
