title: Orphan drug development across Europe: bottlenecks and opportunities.
abstract: With the assignment of the 500th European Union orphan drug designation in 2007, the Regulation on Orphan Medicinal Products truly begins to show its potential for delivering new medicines to patients with rare diseases. Here, we analysed European orphan drug development at a national level and unveil a strong relationship between orphan drug development and pharmaceutical innovation performance in Europe. Moreover, we identify gaps in transition from science into orphan drug development as important bottlenecks that exist in several European countries. Our findings underline the importance of innovation-based policies to enhance the development of orphan drugs in Europe.
authors.affiliation: Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, PO Box 80.082, 3508 TB Utrecht, The Netherlands.
authors.forename: Harald E
authors.initials: HE
authors.lastname: Heemstra
authors.forename: Remco L A
authors.initials: RL
authors.lastname: de Vrueh
authors.forename: Sonja
authors.initials: S
authors.lastname: van Weely
authors.forename: Hans A
authors.initials: HA
authors.lastname: Büller
authors.forename: Hubert G M
authors.initials: HG
authors.lastname: Leufkens
mesh_terms: D017277:Drug Approval / Q000191:economics; D005060:Europe; D005062:European Union; D033161:Government Regulation; D006801:Humans; D007880:Legislation, Drug; D009965:Orphan Drug Production / Q000191:economics; D004364:Pharmaceutical Preparations / Q000191:economics
chemical_list: D004364:Pharmaceutical Preparations