Market Insight - The "ultra-orphan" finds a home at Shire
This article was originally published in Scrip
As discussed in an earlier Market Insight article, Orphan drugs, multiple benefits (Scrip No 3374), the groundbreaking 1983 US Orphan Drug Act provided drugmakers with financial incentives to offset the costs associated with conducting R&D in small patient populations (fewer than 200,000). But what about patients suffering from even rarer diseases? Most recently, some have begun to refer to illnesses affecting fewer than 10,000 US patients as "ultra-orphan" and, of course, these diseases come with their own unique set of challenges.
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