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Orphan drugs may target small patient populations, but they are racking up big numbers at the Food and Drug Administration.

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, 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.


The reasons for the uptick are not surprising.

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. And 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.

In fact, a survey of 32 managed care administrators found that orphan drugs “remain a safe haven from payer pressure,” according to Leerink analysts who track biopharma stocks. Spending on these drugs is “relatively low on the payer radar,” they wrote in an investor note. And they pointed out that only 6 percent of the respondents plan to increase “active management” of orphan drug spending.


As a result, “FDA’s orphan drug program continued to break new ground,” wrote Kurt Karst on the FDA Law Blog, which tallied the industry and regulatory actions. He works at Hyman, Phelps & McNamara, a law firm that specializes in regulatory matters and represents drug makers.

Since 1983, when the Orphan Drug Act went into effect, the FDA has received more than 5,100 requests for orphan designation, issued more than 3,600 designations in response, and approved 552 orphan drugs.

However, Karst also noted that the “avalanche” of requests may be straining FDA resources. He pointed out that the agency received requests for about 1.9 designations per day last year, based on 251 working days in 2015. Meanwhile, the OOPD staff has remained at about 25 employees for many years, while OOPD funding has remained relatively flat over the past few years, he wrote.

“We’re already starting to see some of the strains of the increasing workload on OOPD,” he continued. Usually, the OOPD sets an internal target date for responding to designation requests at about 60 to 90 days from receipt, but that has grown to around 90 to 120 days.

We asked the FDA for comment and will update you accordingly.

Not everyone may be pleased to see the uptick in agency actions, though. In a recent paper in the American Journal of Clinical Oncology, a team of researchers argued that drug makers are exploiting loopholes that allow them to widen the market for such drugs and distorting the original purpose of the law.

They maintained that drug makers sometimes seek orphan drug approval but actually have a long-term plan to ensure the medicines are used more broadly than if they were designed only for rare disease patients. In their view, companies try to game the system and cash in on a taxpayer-funded benefit.

Meanwhile, a spokesman for the pharmaceutical industry trade group wrote us to say the Orphan Drug Act has been “a great success,” since more than 500 orphan drugs were approved since its passage, including 250 in the past decade. By contrast, the FDA approved fewer than 10 orphan drugs in all of the 1970’s before the law was passed.