OBJECTIVES: Orphan drugs (OD) have in common a unique pricing framework where payers may accept high prices despite uncertainties surrounding product benefits. Decisions are often based on the lack of approved treatment options, pressing clinical unmet needs and low disease prevalence. Our objective was to explore the difference in OD prices by disease area 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. Finally, ODs were grouped by disease area and the mean price for each disease area was calculated. The Japanese price was compared to the mean price of the 3 European countries.

RESULTS: Overall, 4 disease areas with more than 1 OD approved both in Japan and EU were identified. ODs were grouped into oncology, metabolism disorders, cardiovascular disease and neurology with 17, 6, 3 and 2 products, respectively. Japanese prices were higher in all disease areas (cardiovascular disease: 33,931 vs. 23,640; neurology: €287,461 vs. €212,149; oncology: €68,200 vs. €35,249) except for metabolism disorders (€772,820 in EU vs. €514,541 in Japan).

CONCLUSIONS: Comparison of ODs prices by disease area support higher price in Japan vs Europe. Oncology in Japan enjoy the highest ratio with about 2-fold the EU price, while the metabolic endocrine products enjoy the highest absolute margin mean difference of € 250,000 per patient year treatment. It is unclear if differences are explained by policy difference or differential value perception of different conditions.