Do payers value rarity? An analysis of the relationship between disease rarity and orphan drug prices in Europe

Journal of Market Access & Health Policy

Katherine Eve Young¹  Micheline Wille²  Goran Medic²  Michiel Hemels²  Daria Korchagina³  Mondher Toumi⁴  Maarten Jacobus Postma⁵ 

[1] Creativ-Ceutical;Horizon Pharma Europe B.V., Market Access EMEA;Mental Health and Public Health, University Paris-Sud, Maison de Solenn;Public Health Department, Research Unit, Aix-Marseille University;University of Groningen;

关键词: Rare diseases;    orphan drugs;    pricing;    treatment cost;    Europe;   

DOI  :  10.1080/20016689.2017.1299665

来源: DOAJ

【 摘 要 】

Background and Objective: Orphan drugs have been a highlight of discussions due to their higher prices than non-orphan drugs. There is currently no European consensus on the method of value assessment for orphan drugs. This study assessed the relationship between the prevalence of rare diseases and the annual treatment cost of orphan drugs in France, Germany, Italy, Norway, Spain, Sweden, and UK. Methods: Approved orphan drugs and prevalence data were extracted from the European Medicines Agency website. Annual treatment costs were calculated using ex-factory price. Simple regression was used to analyse the relationship between costs and prevalence. A specific bivariate analysis was performed for the rarest diseases (≤1 per 10,000). Results: 120 drugs were analysed. Prevalence ranged from 0.001 to 5 per 10,000 (mean 1.24, median 1). Annual treatment costs per patient ranged from €755 to €1,051,956 (mean €100,000, median €39,303). Results show a statistically significant inverse correlation between annual treatment cost and disease prevalence in all countries (France: r = −0.370, p = 0.002; Germany: r = −0.365, p = 0.002; Italy: r = −0.340, p = 0.002; Spain: r = −0.316, p = 0.041; UK: r = −0.358, p = 0.0004; Sweden: r = −0.414, p = 0.014; Norway: r = −0.367, p = 0.002). When analysis was focused on the rarest diseases, a stronger correlation exists in all countries (France: r = −0.525, Germany: r = −0.482, Italy: r = −0.497, Spain: r = −0.531, UK: r = −0.436, Sweden: r = −0.455, Norway: r = −0.466; all p < 0.05 except Sweden p = 0.077). Conclusions: This study shows an inverse correlation between annual treatment cost and prevalence with high statistical significance in the studied countries. Although pricing is a complex process where different attributes are assessed, this study supports the idea that payers value rarity in pricing decisions.

【 授权许可】

Unknown