Skip to main content
Premium Trial:

Request an Annual Quote

Not Always Complete

A new report from the US Government Accountability Office criticizes the Food and Drug Administration's orphan drug program, according to NPR.

The program was established with the aim of encouraging drug companies to invest in developing therapeutics for conditions that affect fewer than 200,000 people, but as NPR reports, critics argue that the program has been misused. Last year, Kaiser Health News reported that drug makers are taking advantage of the program and that about a third of orphan drug approvals were either repurposed mass-market drugs or drugs that garnered approvals for multiple orphan conditions.

This prompted three lawmakers to call on the GAO to investigate the program, NPR adds. In its report issued Friday, the GAO found that the FDA's review of orphan status for drugs was sometimes incomplete. For instance, it reviewed 148 orphan drug applications to find that 102 were missing data on the drug's US regulatory history. NPR notes that 26 applications with incomplete information were still given orphan status.

"[W]e found that FDA does not always ensure that all information is consistently recorded in its review templates and evaluated when making designation determinations, which are critical steps needed to understand the full context of a drug's intended use in the rare disease," the report says.