Schey C, Krabbe P F M, Postma M J, Connolly M P
Unit of PharmacoEpidemiology & PharmacoEconomics, Department of Pharmacy, University of Groningen, Groningen, The Netherlands.
Global Market Access Solutions (GMAS), St-Prex, Switzerland.
Orphanet J Rare Dis. 2017 Jan 17;12(1):10. doi: 10.1186/s13023-016-0555-3.
Since the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs. The National Institute for Health and Clinical Excellence indicated that several criteria other than cost and efficacy could be considered in reimbursement decisions for orphan drugs. This study sought to explore the multi-criteria decision analysis (MCDA) framework proposed by (Orphanet J Rare Dis 7:74, 2012) to a range of orphan drugs, with a view to comparing the aggregate scores to the average annual cost per patient for each product, and thus establishing the merit of MCDA as a tool for assessing the value of orphan drugs in relation to their pricings.
An MCDA framework was developed using the nine criteria proposed by (Orphanet J Rare Dis 7:74, 2012) for the evaluation of orphan drugs, using the suggested numerical scoring system on a scale of 1 to 3 for each criterion. Correlations between the average annual cost of the drugs and aggregate MCDA scores were tested and plotted graphically. Different weightings for each of the attributes were also tested. A further analysis was conducted to test the impact of including the drug cost as an attribute in the aggregate index scores.
In the drugs studied, the R , that statistically measures how close the data are to the fitted regression line was 0.79 suggesting a strong correlation between the drug scores and the average annual cost per patient.
Despite several limitations of the proposed model, this quantitative study provided insight into using MCDA and its relationship to the average annual costs of the products.
自欧洲出台孤儿药立法以来，有人提出，评估药物报销的一般方法不一定适用于孤儿药。英国国家卫生与临床优化研究所指出，在孤儿药报销决策中，除成本和疗效外，还可考虑其他几个标准。本研究旨在探讨《孤儿病杂志》（2012年第7卷，第74页）提出的多标准决策分析（MCDA）框架在一系列孤儿药中的应用，以便将综合得分与每种产品的患者年均成本进行比较，从而确定MCDA作为评估孤儿药价值与其定价关系的工具的价值。
采用《孤儿病杂志》（2012年第7卷，第74页）提出的九个标准开发了一个MCDA框架，用于评估孤儿药，对每个标准使用建议的1至3分制数字评分系统。测试了药物年均成本与MCDA综合得分之间的相关性，并以图形方式绘制。还测试了每个属性的不同权重。进行了进一步分析，以测试将药物成本作为综合指数得分中的一个属性的影响。
在所研究的药物中，统计学上衡量数据与拟合回归线接近程度的R值为0.79，表明药物得分与患者年均成本之间存在强相关性。
尽管所提出的模型存在一些局限性，但这项定量研究为使用MCDA及其与产品年均成本的关系提供了见解。
