No slowing down: Orphan drug designations skyrocket in 2017

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Designations for orphan drugs skyrocketed in 2017, with the Food and Drug Administration receiving 1.4 requests per day and granting those requests at a record rate of 1.3 per day.


The FDA granted 77 orphan drug approvals last year and granted 476 orphan drug designation requests, according to law firm Hyman, Phelps & McNamara, which focuses on food, drug, medical device and cosmetic law. For context on the size of the increase from years past, 2014 saw only 49 orphan drug approvals; in 2015, only 355 orphan drug designation requests were made.


The increase has been largely attributed to the FDA's Orphan Drug Designation Modernization Plan, which was predicated on eliminating the backlog of pending designation requests, in addition to implementing a 90-day timetable for processing designation requests. The agency also established the Orphan Products Council to better handle the scientific and regulatory challenges of orphan products.


Asked why drugmakers sought more orphan drug designations than in years past, Hyman, Phelps & McNamara director Anne Walsh said that companies are catching on to the economic and financial benefits that such designations provide.


“The whole point is that it's been a success, that it is indeed working and people are trying to develop products in areas that would otherwise not be developed,” she said.


Since 1983, the FDA has granted more than 650 orphan drug approvals, nearly 4,500 orphan drug designations, and received more than 6,300 drug designation requests. Some experts believe 2018 may see more designations and requests than ever before, thanks to a pilot program with components that include streamlined requests via a fillable form, an online tutorial to guide sponsors through the admissions process, and inter-center consult process that will standardize the agency's communications process.


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