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September 15, 2017

FDA clears orphan drug designation backlog, eyes policy changes

Daily Briefing

    FDA Commissioner Scott Gottlieb in a blog post Tuesday announced the agency has successfully cleared a backlog of orphan drug designation requests several weeks ahead of schedule, and unveiled plans to reform the review process for such drugs amid concerns of abuse.

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    Background on the orphan drug program

    The Orphan Drug Act (ODA), signed into law in 1983, created incentives for drugmakers to develop drugs for rare, or "orphan," diseases. Under the program, a drugmaker receives seven years of exclusive rights to the marketplace for a treatment for an orphan disease, meaning FDA will not approve another version of the drug during that seven-year period, even if the company's patent expires. The exclusivity period is intended to compensate drugmakers for producing a medication expected to serve only a small population.

    However, a January Kaiser Health News investigation found that the number of orphan drug approvals grew considerably in 2015 and 2016, and about one-third of approvals under the program have been for mass market drugs repurposed to treat orphan diseases or for products that already had received several orphan approvals.

    FDA earlier this year also announced that the program had a backlog of about 200 orphan drug designation requests that were older than 120 days, and in June launched a strategic plan, called the Orphan Drug Modernization Plan, to clear the backlog by Sept. 21.

    FDA clears backlog, moves to close loopholes

    Gottlieb on Tuesday announced FDA cleared the orphan drug designation requests backlog on Aug. 28—nearly a month ahead of the agency's Sept. 21 deadline. Gottlieb said FDA accomplished the goal early thanks to "the dedicated efforts of the orphan drug designation team who oversee the Orphan Drug Designation Program."

    Gottlieb also announced that FDA will implement new policies to improve the orphan drug program's efficiency and close regulatory loopholes within the program. 

    Specifically, Gottlieb said FDA will:

    • Advance guidance and other policies aimed at closing regulatory loopholes, which allow drugmakers to avoid obligations such as studying drugs in pediatric populations;
    • Design a new process map for the orphan drug program based on the assessment of delays, redundancies, and other metrics;
    • Examine how FDA grants orphan drug designations;
    • Hold a public meeting on the appropriate application of orphan incentives;
    • Review the incentives FDA awards to drugmakers under ODA to ensure the law is implemented as intended; and
    • Reorganize the agency's review staff to better leverage staff expertise.

    Gottlieb said, "For all the success of the ODA, there's been criticism that some sponsors are using designations as a way to sidestep other important public health goals set out by Congress." Gottlieb continued, "We need to make sure our policies take notice of all of these new challenges and opportunities" (Tribble, Kaiser Health News, 9/13; Bell, BioPharma Dive, 9/12; Gottlieb, "FDA Voice," FDA, 9/12).

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