PSY6 - Orphan Drugs Versus Ultra-Orphan Drugs: Price Comparison in England

Abstract

Ultra-orphan drug (UOD) is a specific classification in England, encompassing drugs with a prevalence of less than 1/50,000. In May 2013, England enhanced specificity of UODs through a highly specialized technology (HST) process. This process confirms a focus on the overall magnitude of benefits to patients and society, rather than the cost-effectiveness threshold usually used for technology assessment. The aim of this study was to compare the prices of orphan drugs (ODs) and UODs in England. ODs approved in Europe up until 01/01/2018 were identified from the European Medicines Agency website. Based on the prevalence of the targeted diseases in England, ODs were divided between UODs (prevalence<1/50,000) and ODs (prevalence>1/50,000). Ex-factory prices available in England were extracted from IHS POLI database and annual treatment prices were calculated based on the posology described in the summary of products characteristics (SmPC). Median and mean annual prices were calculated in each group. One-hundred-two ODs approved in Europe were identified. Overall, 75 drugs had prices available in England: 12 UODs and 63 ODs. ODs’ annual prices varied widely between 610€ and 875,590€, while UODs annual prices varied between 6,868€ and 819,244€. UODs’ median annual prices was 2-fold the median prices of ODs (78,722€ versus 38,508€, respectively). Coherently, the mean annual prices reached almost the double of ODs’ mean annual prices; 193,248€ versus 108,790€ respectively. UODs benefit in England from a special decision analysis process and pricing pathway. This process incentivizes manufacturers to develop UODs, but may have a reverse effect for ODs. However, this may have led to very high prices, and, therefore, UODs are currently amongst the most expensive medicines on a yearly cost-per-patient basis. Still, there are important questions surrounding the actual use and access of such expensive drugs in England.