Last year, the agency received a record 472 requests from companies to have their medicines designated as orphan drugs. And the FDA agreed to award 354 designations, which was a 22 percent increase over 2014.
As for approvals, Silverman reported that the agency endorsed 41 orphan medicines, just seven fewer than the previous year, according to the FDA Office of Orphan Products Development. A designation, by the way, means the FDA has decided a drug qualifies for orphan status and takes place before a drug is approved.
Orphan drugs allow drug makers to benefit from seven years of marketing exclusivity awarded by Congress. The FDA faces increasing pressure to approve these medicines, which are treatments for rare diseases that afflict small but often vocal patient populations.
In addition, payers are willing to absorb high prices rather than endure negative publicity that can stem from denying coverage or forcing patients to absorb much of the costs.
For Silverman’s full STAT story, click here.