Accelerated approval drug labels often lack information for clinical decision making.

Abstract

The accelerated approval pathway allows for drug approval based on surrogate outcome measures "reasonably likely" to predict clinical benefit. FDA guidance states that, when an indication is based on accelerated approval and hasn't yet received full approval, the drug label must describe the accelerated approval and should specify the specific surrogate marker(s) that supported it, as well as the clinical outcomes being evaluated in post-approval commitment trials. We reviewed the labels of all drugs with indications receiving accelerated approval after January 1, 1992 that had not received full approval by December 31, 2020. 253 clinical indications corresponding to 146 drugs received accelerated approval. We identified a total of 110 accelerated approval indications across 62 drugs that had not received full approval by December 31, 2020. A total of 13% of labels for accelerated approved indications lacked sufficient information that approval was via the accelerated approval or based on surrogate outcome measures: 7% did not mention accelerated approval but described surrogate markers, 4% did not mention accelerated approval nor describe surrogate markers, and 2% mentioned accelerated approval but did not describe surrogate markers. No label described the clinical outcomes being evaluated in post-approval commitment trials. Labels for accelerated approved clinical indications that do not yet have full approval should be revised to include the information required in the FDA guidance to help guide clinical decision making.