OBJECTIVES : To understand recent trends in orphan drug pricing between the US and Europe (France, Germany and the UK) and to assess whether launch pricing differentials between markets are narrowing with time.

METHODS : 10 orphan drugs, including hospital and non-hospital medicines spanning a variety of indications, were chosen. Prevalence rates for indications in the US and Europe ranged from <500 people to ~230,000 people. FDA and EMA approval dates of each drug ranged from 2014–2018. Launch pricing data was gathered from a number of publicly available sources for each market and included ex-factory prices, hospital prices and pharmacy purchase prices. Prices were adjusted to USD using historic month-specific exchange rates and annual treatment costs were calculated using the defined daily dose. FDA, EMA and WHO websites and other publicly available sources were used to source launch dates, prevalence estimates and defined daily dose information (used to calculate annual treatment costs).

RESULTS : Annual treatment costs at launch were higher in the US than in Europe in 8 out of 10 cases. On average, annual treatment costs were 22% higher for those treatments more expensive in the US, and 47% higher for those treatments (asfotase alfa and cerliponase alfa) more expensive in Europe. While pricing differentials showed no time-dependent correlation between markets, launch prices were more strongly correlated with disease prevalence in Europe (R²=0.2336) than in the US (R²=0.1722).

CONCLUSIONS : In general, orphan drugs are priced higher in the US than in Europe. While we found no trend suggesting a narrowing in orphan drug pricing between markets since 2014, launch prices do appear more strongly correlated with disease prevalence in Europe than in the US.