Orphan drugs distribution

An orphan drug is one that is intended for use in rare diseases and thus for which there is not a sufficient market to justify the investment needed to demonstrate safety and effectiveness in order to obtain approval of an NDA. Por more than 20 years PDA had permitted orphan drugs to be distributed through a permanent IND, with little or no thought that it would ever progress to an approved NDA. In 1983, Congress enacted the Orphan Drug Act to provide... 

S.2.3.3 Treatment of Trypanosomiasis The difluoromethylornithine (DFMO), eflomithine is a mechanism-based inhibitor of ornithine decarboxylase— a pyridoxal-dependent key enzyme of the polyamine s biosynthesis from ornithine. Fluorine atoms are essential for the inhibition process (cf. Chapter 7). Eflornithine was first clinically developed for cancer, but its development has been abandoned for this indication. The activity of eflornithine on trypanosomes was then discovered. Now, despite its very low bioavailability, eflornithine is the best therapy for sleeeping sickness (trypanosomiasis)—in particular, at the cerebral stage—due to Trypanosoma brucei gambiense parasite. Eflornithine is registered with orphan drug status and is distributed by the WHO. 

DISCOVERY, DEVELOPMENT, MARKETING AND DISTRIBUTION OF ORPHAN DRUGS... 

Discovery, development, marketing and distribution of orphan drugs... 

Distribution methods differ more between orphans and non-orphans more than the other categories discussed in this section. Conventional drugs are generally sold through wholesalers and directly to institutions. Orphan drugs more often use alternative distribution techniques —these include mail order pharmacies and direct sales to patients, physicians, and institutions. 

---