OBJECTIVES: Orphan Drugs (ODs) are intended for the diagnosis, prevention, or treatment of life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the European Union. Despite the small target population, many ODs became blockbusters, and oncology ODs are often claimed to enjoy the highest prices. The aim of this study was to investigate whether the therapeutic area was a determinant of OD prices in European countries.

METHODS: The ODs approved in Europe until 01/03/2018 were identified from the official website of European Medicines Agency (EMA). ODs were categorized based on targeted disease areas: cardiovascular disease, endocrinology, gastroenterology, haematology, infectious diseases, metabolism disorders, neurology, oncology, pulmonology, ophthalmology, dermatology, and musculoskeletal disease. The median value of OD annual prices per disease area for 29 European countries (26 countries in European Union(except for Malta and Cyprus), in addition to Norway, Switzerland and Russia) were calculated using ex-factory price extracted from IHS POLI database.

RESULTS: Prices were available for 96 ODs out of 102 approved ODs by EMA. Oncological disease had the largest number of approved ODs (N=37), followed by metabolism disorder (N=14), and hematological disease (N=10). Prices of ODs in each country varied widely between different disease areas. Musculoskeletal disease had the highest annual price (Median=838,956€), followed by metabolism disorders (Median=214,166€), gastroenterological diseases (Median=170,820€), hematological diseases (Median=62,372€), ophthalmological diseases (Median=54,600€) and oncological disease (Median=43,693€). Neurological diseases showed the lowest annual price with a Median of 6,160€. The ranking is consistent at specific country level.

CONCLUSIONS: Setting prices for ODs is a process involving multiple determinants such as disease prevalence, severity, and availability of alternative treatments. Disease area seems to be one of the important driver for OD prices in Europe, while oncology products do not happen to be the most expensive ODs.