Picavet Eline, Morel Thomas, Cassiman David, Simoens Steven
KU Leuven Department of Pharmaceutical and Pharmacological Sciences, Herestraat 49, PO box 521, Leuven 3000, Belgium.
Orphanet J Rare Dis. 2014 Apr 27;9:62. doi: 10.1186/1750-1172-9-62.
The pricing mechanism of orphan drugs appears arbitrary and has been referred to as a "black box". Therefore, the aim of this study is to investigate how drug- and disease-specific variables relate to orphan drug prices. Additionally, we aim to explore if certain country-specific pricing and reimbursement policies affect the price level of orphan drugs.
Annual treatment costs per indication per patient were calculated for 59 orphan drugs with a publicly available price in Belgium, the Netherlands, Czech Republic, France, Italy and the United Kingdom. A multiple linear regression model was built with 14 drug- and disease-specific variables. A Mann-Whitney U test was used to explore whether there is a correlation between annual treatment costs of orphan drugs across countries with different pricing and reimbursement policies.
Repurposed orphan drugs, orally administered orphan drugs or orphan drugs for which an alternative treatment is available are associated with lower annual treatment costs. Orphan drugs with multiple orphan indications, for chronic treatments or for which an improvement in overall survival or quality-of-life has been demonstrated, are associated with higher annual treatment costs. No association was found between annual treatments cost of orphan drugs across countries and the different pricing and reimbursement systems.
This study has shown that prices of orphan drugs are influenced by factors such as the availability of an alternative drug treatment, repurposing, etc. Current debate about the affordability of orphan drugs highlights the need for more transparency in orphan drug price setting.
罕见病药物的定价机制似乎具有随意性，被称为“黑匣子”。因此，本研究的目的是调查药物和疾病特定变量与罕见病药物价格之间的关系。此外，我们旨在探讨某些特定国家的定价和报销政策是否会影响罕见病药物的价格水平。
计算了比利时、荷兰、捷克共和国、法国、意大利和英国59种有公开价格的罕见病药物每种适应症每位患者的年度治疗成本。构建了一个包含14个药物和疾病特定变量的多元线性回归模型。使用曼-惠特尼U检验来探讨不同定价和报销政策国家的罕见病药物年度治疗成本之间是否存在相关性。
重新利用的罕见病药物、口服给药的罕见病药物或有替代治疗方法的罕见病药物，其年度治疗成本较低。具有多种罕见病适应症、用于慢性治疗或已证明可提高总体生存率或生活质量的罕见病药物，其年度治疗成本较高。未发现不同国家的罕见病药物年度治疗成本与不同的定价和报销系统之间存在关联。
本研究表明，罕见病药物价格受替代药物治疗的可用性、重新利用等因素影响。当前关于罕见病药物可负担性的争论凸显了在罕见病药物定价方面提高透明度的必要性。
