OBJECTIVES: Funding orphan drugs (OD) is a sensitive and challenging endeavour. Payers deal with uncertainty surrounding product benefits while attempting to address high unmet medical needs. At the same time, the industry aims to secure returns on substantial upfront investments while targeting orphan diseases with small and fragmented patient populations. This complex trade-off may lead to differences in OD prices globally. Our objective was to explore the difference in OD prices between European countries (EU) and Japan.
METHODS: ODs approved, in both Japan and EU, up to 01 March 2018 were identified from the Pharmaceuticals and Medical Devices Agency (PMDA) and the European Medicines Agency (EMA) websites, respectively. The annual OD price per patient was then calculated based on the posology recommended in the summary of product characteristics and the ex-factory price in Japan, France, Germany, and the UK. The Japanese price was compared to the mean price of the 3 European countries.
RESULTS: Overall, 31 ODs approved in both Japan and EU were identified. The mean price of all ODs was higher in Japan (€189,974) than in the EU (€170,900). However, when considering each OD separately, prices were often higher in EU (21 drugs out of 31) than in Japan. Only 3 ODs had similar prices in EU and Japan with a price ratio close to 1 and only 5 ODs had an EU price lower than 75% of the Japanese price. Using median price shows similar trends while median being higher than mean prices.
CONCLUSIONS: Our results showed that OD prices are heterogeneous between European countries and Japan. This may be explained by the heterogeneous pricing processes used in the different countries. OD prices are not predictable. Further studies to identify price drivers are warranted.