GAO mostly okays FDA’s approval process for rare disease designations

More-complete information is desirable on some applications


The Government Accountability Office (GAO) has evaluated FDA’s process for reviewing applications for orphan-drug designation, a status that entitles the applicant (depending on the application) to tax credits, accelerated review for approval, extended market exclusivity and, sometimes, financial support. In 2017, there were 527 such applications, up substantially from past years and, since 2008 351 such applications have been approved.

In its report, overall, GAO found little to be critical of; its main conclusion is that “FDA should ensure that all required information for reviews of orphan designation applications is consistently recorded and evaluated.” The FDA response to this finding is that “the agency concurred with our recommendation.”

Orphan drugs, intended for rare diseases, came into being with passage of the Orphan Drug Act in 1983. In the US, a rare disease is defined as one affecting fewer than 200,000 patients in a given year. That marker is one of the shortcomings that GAO found; some applications have relatively little information on the potential patient population. “Without ensuring that its reviewers conduct and record the results of independent verification of population estimates, [the Office of Orphan Products Development] cannot be assured that quality information is consistently informing its designation determinations,” say the GAO examiners. (Documented populations, says GAO, range from 0 to—someone had a play-the-game-by-the-rules attitude here—199,966.)

Another area of designation-review weakness is knowledge of prior or related applications, especially in cases where the same has been offered for multiple rare diseases. (The record holders here are Genentech’s Avastin [bevacizumab] and Pharmacyclics/Janssen’s Imbruvica Imbruvica [ibrutinib], both blockbuster drugs that have 9 orphan designations each.)

FDA Commissioner Scott Gottlieb initiated a “modernization” program in June 2019 of OOPD; that effort resulted in reducing the backlog of ODA applications and clarified issues around the approval process. For the past few years, orphan drug approvals have been a significant portion of the count of newly approved drugs issued by FDA annually.