USA – GAO Raises Questions on FDA’s Orphan Drug Designation Process

The Government Accountability Office (GAO) announced Friday that it has found that the US Food and Drug Administration (FDA) does not always ensure that all information is consistently recorded and evaluated when making orphan drug designation determinations.

The missing of these critical steps by the agency in some cases means that FDA reviewers do not fully understand the context of a drug’s intended use for the rare disease, GAO said.

“The Commissioner of FDA should ensure that information from orphan drug designation applications is consistently recorded in OOPD [Office of Orphan Products Development] review templates and evaluated by OOPD reviewers when making an orphan designation decision,” GAO recommended.

The recommendation follows an inquiry from Sens. Orrin Hatch (R-UT), Chuck Grassley (R-IA) and Tom Cotton (R-AR) on potential abuses of the Orphan Drug Act. Grassley also has said he is looking into abuses of the act that allow such incentives, meant to encourage drug development for rare diseases, to be bestowed on a drug that actually reaches a larger population than what it won the orphan designation for, or companies that conduct limited research to win approval for such a drug.

However, the GAO report did not address such abuses and delved more into the processes by which FDA evaluates orphan designation requests.


FDA generally evaluates orphan designation requests by looking at the size of the rare disease population and the company’s scientific rationale that the drug may effectively treat the disease. To inform their evaluation, reviewers must record certain background information in a standard review template, such as the drug’s marketing history. Officials told GAO that this information provides important context, such as whether FDA has experience with a rare disease, which is critical to ensuring a complete designation application review.

However, GAO’s analysis of 148 designation review templates found that FDA does not consistently record or evaluate background information when making designation decisions. “For example, 48 of 148 review templates GAO analyzed were missing information on the drug’s US marketing history. As such, FDA cannot be sure that reviewers are conducting complete evaluations that include all critical information needed for assessing its criteria,” GAO said…