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Orphan drugs may serve small groups of patients, but they generate big numbers at the Food and Drug Administration.

Last year, the agency issued a record-breaking 91 orphan drug approvals, up from 81 approvals in 2017 and well above levels seen in previous years. We should point out, though, that approvals include not only new drugs, but also applications for new orphan uses of previously approved medicines, otherwise known as repurposed drugs.


Meanwhile, orphan designations dropped to 337 in 2018 from 477 the year before, while the number of designation requests made were 507 last year, compared with 526 in 2017 and 582 the year before that. A designation means the FDA has decided a drug qualified for orphan status and occurs before a drug is approved.

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