Research Centre for Pharmaceutical Care and Pharmaco-economics, Faculty of Pharmaceutical Sciences, Katholieke Universiteit Leuven, Leuven, Belgium.
Appl Health Econ Health Policy. 2011 Jul 1;9(4):275-9. doi: 10.2165/11590170-000000000-00000.
The literature indicates that the expenditure on orphan drugs will be increasing over the coming years. The market for orphan drugs has inherent market characteristics that sometimes result in high prices. The aim of this study was to analyse whether awarding orphan designation status has an influence on the price setting of drugs for rare disease indications. To this effect, prices of designated orphan drugs were compared with other non-designated drugs for rare disease indications. We identified 28 designated orphan drugs and 16 comparable non-designated drugs for rare disease indications for which we collected official hospital prices (per defined daily dose) in Belgium in 2010. Orphan-designated drugs had a higher median price (138.56 Euros [interquartile range; IQR 406.57 Euros]) than non-designated drugs (16.55 Euros [IQR 28.05 Euros]) for rare disease indications (p < 0.01). In conclusion, our results suggest that awarding orphan designation status in itself is associated with higher prices for drugs for rare disease indications. In order to gain full insight into orphan drug pricing mechanisms, future research should focus on collecting information about the different factors influencing orphan drug pricing.
文献表明，未来几年孤儿药的支出将会增加。孤儿药市场具有内在的市场特征，这有时会导致价格高昂。本研究旨在分析授予孤儿药认定地位是否会对罕见病适应证药物的定价产生影响。为此，我们比较了有认定地位的孤儿药和其他无认定地位的罕见病适应证药物的价格。我们确定了 28 种有认定地位的孤儿药和 16 种有认定地位的罕见病适应证药物，收集了 2010 年比利时官方医院（每规定日剂量）的价格。有认定地位的孤儿药（中位数 138.56 欧元[四分位间距：406.57 欧元]）比无认定地位的孤儿药（中位数 16.55 欧元[四分位间距：28.05 欧元]）价格更高（p<0.01）。总之，我们的结果表明，孤儿药认定地位的授予本身与罕见病适应证药物的高价有关。为了全面了解孤儿药定价机制，未来的研究应集中收集影响孤儿药定价的各种因素的信息。
