OBJECTIVES: To compare additional benefit rating during health technology assessment (HTA) and drug prices for orphan drugs (ODs) in France and Germany, and to assess the relationship between HTA score and annual drug costs.

METHODS: ODs authorized in Europe between January 2011 and November 2017 were identified using the European Medicines Agency’s database, including ODs which orphan designation has expired or been withdrawn. For drugs with multiple indications, only the first one was considered for assessment of annual cost. Ex-factory unit prices were extracted from AMELI and Thériaque databases for France, and Orphan POLI Global for Germany and served to calculate annual drug costs. A regression analysis was performed to evaluate the relationship between HTA additional benefit score and annual cost in France and Germany.

RESULTS: Nineteen ODs for which prices were available in both France and Germany were identified. A significant correlation between HTA additional benefit score and annual costs was observed in France, but not in Germany. Annual costs in France and Germany were strongly correlated (correlation coefficient: 0.99, regression equation: y=0.96x + 10900). Because annual costs in France and Germany were very similar, the involvement of external reference pricing was considered and the history of price setting dates for the two countries was investigated. Of 19 ODs, 95% first entered the market in Germany, where they were freely priced, suggesting that many OD prices in France were influenced by the free price in Germany.

CONCLUSIONS: Annual costs of ODs in France and Germany were found to be similar. Almost all the ODs first entered the market in Germany, suggesting that OD prices in France were influenced by the free prices in Germany.