Rare Diseases and Orphan Drugs

Biography

Biography: Fizza Batool

Abstract

The word orphan comes from the Greek word orphanos, a child who has lost one parent or both, or an adult who has lost a child. It goes back to the putative Indo-European root ORBH, bereft, as in the Latin word orbus. The obsolete English words orbation and orbity meant orphanhood or childlessness..In modern English the word orphan is most commonly used in its original Greek sense, but metaphorical meanings have also emerged. An orphan vehicle, for example, is a discontinued model, and an orphan is a line of type that begins a new paragraph at the bottom of a column or page. In the last 20 years efforts have been made to encourage companies to develop orphan drugs. The Orphan Drug Act in the USA (1983) was succeeded by similar legislation in Japan (1985), Australia (1997), and the European Community (2000) [9]. The encouragement takes three forms: tax credits and research aids, simplification of marketing authorization procedures, and extended market exclusivity [9, 13]. In Europe only the last is available.In this issue of the Journal, Joppi et al. show how slow the development of orphan drugs in Europe was during the four years after the introduction of legislation that allowed a drug to be designated an orphan on the basis of rarity of the disease, a plausible mechanism of action, and likelihood of benefit Of 255 applications to EMEA, only 18 were approved for marketing, and in many cases the supporting studies were poorly designed. This contrasts with reported experience from the USA, where between 1983 and 2002 nearly 1100 drugs and biological products were designated orphan products and 231 were approved. This difference may be due to lack of incentives in Europe compared with the USA, although without directly comparing the two sets of dossiers we cannot know whether there were other reasons; such a comparison would be justified.