Orphan drug development across Europe: bottlenecks and opportunities.
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.
Heemstra
Harald E
HE
Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for Pharmaceutical Sciences, Faculty of Science, Utrecht University, PO Box 80.082, 3508 TB Utrecht, The Netherlands.
de Vrueh
Remco L A
RL
van Weely
Sonja
S
Büller
Hans A
HA
Leufkens
Hubert G M
HG
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
D004364:Pharmaceutical Preparations