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As more drug makers race to win orphan designations, a new report finds the Food and Drug Administration fails to consistently record and evaluate needed information, which suggests the agency may not always include critical data for granting the coveted orphan status.

Specifically, the FDA granted the designation to 26 applications that were missing required information, such as verified estimated patient populations, and 102 of 148 so-called review templates were missing background information. Moreover, FDA guidance was not always clear in instructing agency staff on how to use the recorded information, according to the U.S. Government Accountability Office.


The report follows sustained scrutiny of this lucrative corner of the pharmaceutical market. Under the Orphan Drug Act, which was passed in 1983, the FDA began approving medicines to treat rare diseases that affect fewer than 200,000 people. The incentives to fill these unmet medical needs include tax credits and seven years of exclusive marketing rights.

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