A report by the US GAO on the Trump Administration's handling of the Orphan Drug Act.

The U.S. Government Accountability Office (@USGAO) recently issued a report on orphan drugs outlining some of the deficiencies in the current Administration's handling of the Orphan Drug Act.

The report found that 3,690 orphan drug applications were received from 2008 to 2017 - 3,491 with a designated therapeutic area from the Office of Orphan Products Development. In total, there were 351 marketing approvals for 252 unique drugs across 27 therapeutic areas - 7 of those areas already having 10 or more approved orphan drugs.

Approvals were concentrated in 2 areas during the period : hematology (10.8%) and oncology (42.5%).

In its review, the GAO found that the @US_FDA does not consistently ensure that all information - such as other orphan designations of a product or for the disease state it is intended to treat - is appropriately recorded and used in the regulatory decision-making process | @heatinformatics 

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