The Orphan Drug Act (ODA) offers multiple incentives to manufacturers that bring a drug to market with an orphan designation. A recently released U.S. Government Accountability Office (GAO) report revealed that while the number of applications both received and granted for this designation has grown, FDA reviewers were not consistently recording or evaluating required information that is mandated to consider granting this designation, AIS Health reported.

The report, titled Orphan Drugs: FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue (GAO-19-83), shows that from 2008 to 2017, both orphan designation applications received as well as orphan designations granted rose.

The report, titled Orphan Drugs: FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue (GAO-19-83), shows that from 2008 to 2017, both orphan designation applications received as well as orphan designations granted rose.

Of the 3,690 orphan drug designation applications that the FDA received from 2008 to 2017, about 71% were granted that designation by April 2018; 21% were deemed pending, 5% were denied, and 2% were withdrawn.

In June 2017, the FDA issued the Orphan Drug Modernization Plan, intended to eliminate within 90 days the backlog of 138 applications for the designation that had been pending for more than 120 days. In addition, it sought to ensure that future applications would be reviewed within 90 days of the agency’s receiving them. The FDA achieved the first goal, and, as of July 20, 2018, had “overall met its 90-day timeliness goal for reviewing designation applications since mid-September 2017 and has completed most application reviews within 60 days of receipt,” according to the GAO report.

Researchers also assessed whether FDA reviewers were using consistent criteria to evaluate applications. On this, however, reviewers’ performance left a bit to be desired.

From October to December 2017, after the implementation of the modernization plan, the agency analyzed 148 review templates. According to the GAO report, “of the five review template sections where reviewers are required to record information, we found that OOPD [i.e., the Office of Orphan Products Development] does not ensure that all required information is consistently recorded in the background information section and evaluated when making designation decisions.”

Of the 148 templates, the FDA granted orphan designation to 26 applications that were missing required information. GAO recommends that the “FDA should ensure that all required information for reviews of orphan designation applications is consistently recorded and evaluated. The agency concurred with our recommendation.”