Friedmann Carlotta, Levy Pierre, Hensel Paul, Hiligsmann Mickaël
a Department of Health Services Research, Care and Public Health Research Institute (CAPHRI) , Maastricht University , Maastricht , The Netherlands.
b Université Paris-Dauphine , Laboratoire d'Économie et de Gestion des Organisations de Santé (LEDa-LEGOS) , Paris , France.
Expert Rev Pharmacoecon Outcomes Res. 2018 Apr;18(2):135-146. doi: 10.1080/14737167.2018.1414603. Epub 2017 Dec 20.
Multi-criteria decision analysis (MCDA) could potentially solve current methodological difficulties in the appraisal of orphan drugs.
We provide an overview of the existing evidence regarding the use of MCDA in the appraisal of orphan drugs worldwide. Three databases (Pubmed, Embase, Web of Science) were searched for English, French and German literature published between January 2000 and April 2017. Full-text articles were supplemented with conference abstracts. A total of seven articles and six abstracts were identified.
The literature suggests that MCDA is increasingly being used in the context of appraising orphan drugs. It has shown itself to be a flexible approach with the potential to assist in decision-making regarding reimbursement for orphan drugs. However, further research regarding its application must be conducted.
多标准决策分析（MCDA）有可能解决当前罕见病药物评估中存在的方法学难题。
我们概述了全球范围内有关MCDA用于罕见病药物评估的现有证据。检索了三个数据库（PubMed、Embase、科学网），以查找2000年1月至2017年4月期间发表的英文、法文和德文文献。全文文章辅以会议摘要。共识别出7篇文章和6篇摘要。
文献表明，MCDA在罕见病药物评估中的应用越来越多。它已证明自身是一种灵活的方法，有潜力协助进行罕见病药物报销决策。然而，必须对其应用开展进一步研究。
