Drugs for exceptionally rare diseases: a commentary on Hughes et al

Recently in this journal, Hughes and colleagues discussed special funding status to ultra-orphan drugs. They concluded that there should be a uniform policy for the provision of orphan drugs across Europe; that complete restriction was impractical, and that UK policy should aspire to the values of the EU directive on orphan drugs. We critically assess these arguments, demonstrating that they failed to justify special status for treatments for rare diseases.