GAO raises questions on FDA's orphan drug designation process

The Government Accountability Office (GAO) said it has discovered that FDA does not always ensure all information is consistently recorded and evaluated when making orphan drug designation determinations, suggesting that in some cases FDA reviewers do not fully understand the context of a drug's intended use for a particular rare disease.

The Government Accountability Office (GAO) said it has discovered that FDA does not always ensure all information is consistently recorded and evaluated when making orphan drug designation determinations, suggesting that in some cases FDA reviewers do not fully understand the context of a drug's intended use for a particular rare disease. "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 report follows an inquiry from lawmakers on potential abuses of the Orphan Drug Act. The analysis did not address such abuses, however, and focused more on the processes by which FDA evaluates orphan designation requests. Reviewers must record certain background information in a standard review template to inform their evaluation. Yet GAO's analysis of 148 designation review templates found that FDA reviewers did not include background data on the drug's U.S. marketing history in 48 of the templates and did not include the results of independent sources to verify the population estimate in 23 of them. HHS said the recommendation would be considered as part of FDA's efforts to improve the designation review template and to train reviewers.