5 burning questions for an orphan drug CEO
This article was originally published in Scrip
Legislation introduced since the 1980s and 1990s to incentivise the development of new treatments for rare diseases is paying off (about a third of new drug approvals are now for orphan medicines), but there is also growing concern among payers that some orphan drug prices may be unsustainable. The recent pricing of BioMarin Pharmaceutical's Vimizim at an estimated annual cost per patient of $380k drew attention to the issue once again (scripintelligence.com, 18 February 2014).
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