OBJECTIVES: This study assessed orphan drugs (ODs) price differences between France, Germany, and United Kingdom (UK) by comparing annual treatment prices of available OD in the 3 countries.

METHODS: Authorized ODs in Europe up to 03/03/2018 were identified from the European Medicines Agency website. Annual treatment prices per OD in each country were calculated using ex-factory prices extracted from IHS POLI database based on the posology described in the summary of products characteristics (SmPC). Prices in Germany were used as reference and price ratios were analyzed

RESULTS: Seventy-six ODs had available prices in the three countries out of 102 authorized in Europe. The annual prices were greatly heterogeneous between the 3 countries. In the majority of cases (87% of products), Germany had higher prices than France and UK with a mean ratio of 0.83 and 0.78 respectively. Nine OD (18%) had higher prices in France than Germany whereas 13 (27%) had prices in France lower than 75% of the German price . The ratio compared to Germany varied in France between 0.27 and 1.21. Moreover, UK had the lowest prices ; where only 5 OD had prices higher than German price and 33 products (44%) had prices lower than 75% of the German price. The ratio compared to Germany varied between 0.31 and 1.52.

CONCLUSIONS: ODs prices may vary widely across European countries. This heterogeneity in pricing may be due to multiple factors among which the complex pricing process specific to each country, the unmet needs, the severity of disease, the availability of alternative treatment and many other factors that disproportionally impact prices in each country. Significant vagueness still surrounds the orphan drug pricing mechanisms. Further studies to identify the determinants of orphan drug prices in Europe are needed.