‘Orphan drugs’ are ascendant. What will that mean for people with rare diseases?
6 min readMay 29, 2018
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For most drugs, the path from the Petri dish to pharmacy shelves is paved with uncertainty. It’s a decade-long journey, with billions in investment dollars at stake, seemingly countless administrative hoops to jump through, and the risk of complete failure lurking at every bend. Ultimately, studies show that only a meager 10 percent of drugs in…