Twenty-Four Years After the Launch of the EU Orphan Regulation: Analyzing Dutch Price Dynamics, Biosimilars, and Generics for Orphan Medicinal Products

Objectives: The Orphan Regulation, introduced in 2000, offers a 10-year exclusivity to recoup investments on orphan medicinal products (OMPs). Despite expectations of price drops, many OMPs seem to lack competition after exclusivity. This study examines OMP price trends and the entry of generics/biosimilars in The Netherlands. Methods: OMPs authorized before January 1, 2010, were included unless they had active substance patents or were withdrawn before the end of market exclusivity. Data on European generic/biosimilar authorization were gathered from the European Medicines Agency and Dutch Medicines Evaluation Board. List price data were collected from the Dutch national database. Results: Twenty-eight small molecules and 9 biologicals were included in the primary analysis, all authorized between 14.0 to 22.3 years. The median of their most recent prices in The Netherlands was 81% of the initial price (range 11%-104%). Eleven small molecule (39%) and 8 biological (89%) OMPs did not have a generic/biosimilar registered. If generic/biosimilar entry did occur, this was after a median of 14.2 years (range 11.5-20.3 years). Median prices of OMPs with generic/biosimilar competition were lower than those without (to 66% vs 88% of the initial price, respectively, P < .01). Conclusions: Postexclusivity competition and subsequent price decrease is limited for OMPs. First, generics/biosimilars for OMPs enter later than non-OMPs. Second, for some OMPs (especially biologicals), there is no competition at all. Third, prices of OMPs with generic/biosimilar competition decreased only minimally compared with non-OMPs. New policies are needed to correct this apparent market failure.