Reply to M.L. Lanthier et al

Abstract

We appreciate comments by Lanthier et al 1 of the US Food and Drug Administration regarding whether accelerated approval (AA) has expedited approval of new molecular oncology entities. Based on nonpublicly available data, they report median development times of 6.3 years for AA versus 7.2 years for regular approval (RA). However, the US Food and Drug Administration reported to the General Accounting Office 2 that AA resulted in early approval for some cancer drugs “years before the drug would have been available under regular approval procedures” suggesting that the success metric should be years—not months. Ourcontentionisthatdrugsforrarecancershavedifferentchallenges than drugs approved for common cancers. The goals and mechanisms for marketing approval should reflect the particular circumstances. For drugs for common cancers, development times are similar for RA versus AA. The longer biologic licensing agreement development time for AA versus RA (median, 9.2 v 6.7 years) is explainedbyLanthieretalasbeingduetosmallnumbers.However,it is novel biologic agents for rare cancers that are the expected primary beneficiaries of AA. Lanthier et al 1 suggest that sponsors prefer RA versus AA. The reality is that over time, AA appears to be an increasingly difficult hurdle. Before 2003, AA supported 78% of new molecular oncology entities versus 33% since then. Another concern is lack of data transparency at US Food and Drug Administration. Despite extensive searching of public sources, we identified development times for only 75% of new molecular oncology entities. There have been several calls for improved data transparency and completeness including the General Accounting Office’s review of AA and of the US Food and Drug Administration’s nonpublicly available documents, Congressman Ed Markey’s review of the AA program, and a recent policy piece. 2-4 Some of these documents also point out that the US Food and Drug Administration has