GAO Finds FDA Granted Orphan Drug Designation to Some Applications Missing Required Information

December 4, 2018

Rare Daily Staff

The Government Accountability Office in a new report said that the U.S. Food and Drug Administration granted orphan drug designation in a number of cases where applicants failed to supply required information and that the agency could improve the consistency of its designation review.

The report came in response to questions raised by Republican Senators in March 2017 in response to a controversy over the pricing of an orphan drug and potential abuses of the Orphan Drug Act.

Approximately 7,000 rare diseases affect an estimated 30 million people in the United States, and only 5 percent of rare diseases have FDA-approved treatments. To encourage development of therapies for rare conditions, Congress passed the Orphan Drug Act, which provides incentives, including tax credits and exclusive marketing rights, for manufacturers to develop drugs to treat rare diseases, which are typically defined as affecting fewer than 200,000 people in the United States.

The GAO was asked to examine FDA’s orphan drug processes. It examined the actions FDA has taken to address the growing demand for orphan designations, the extent to which FDA has used consistent criteria and complete information in reviewing orphan designation applications, and steps FDA has taken to address rare disease drug development challenges.

For its study, the GAO analyzed FDA documents and data, as well as all designation review templates FDA completed as of March 2018 for applications received from October to December 2017. GAO interviewed agency officials, as well as stakeholders, including drug manufacturers, industry experts, and patient advocacy groups.

The GAO said FDA reviewers generally apply two consistent criteria in evaluating designation applications—the size of the rare disease population and the 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 U.S. marketing history. FDA officials told the GAO this information provides important context, such as whether FDA has experience with a little-known disease, critical to ensuring a complete designation application review.

But the 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 U.S. marketing history. “As such, FDA cannot be sure that reviewers are conducting complete evaluations that include all critical information needed for assessing its criteria,” the GAO said

The GAO recommended that FDA should ensure that all required information for reviews of orphan designation applications is consistently recorded and evaluated. The report said the agency agreed with the recommendations.

December 3, 2018

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