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

Abstract

Study objective We evaluated US Food and Drug Administration labels for drugs approved under the accelerated approval pathway and whether these labels contained in sufficient information regarding their accelerated approval. Design Retrospective, observational, cohort study. Data source Label information for drugs with an accelerated approved indication were ascertained from two online platforms: Drugs@FDA and FDA Drug Label Repository. Intervention Drugs with indications receiving accelerated approval after January 1, 1992, but had not received full approval by December 31, 2020. Measurements Outcomes include whether the drug label indicated the use of the accelerated approval pathway, identified the specific surrogate marker(s) that supported it, or described the clinical outcomes being evaluated in post‐approval commitment trials. Results 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. Conclusion 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.

Characterization of accelerated approval status, trial endpoints and results, and recommendations in guidelines for oncology drug treatments from the National Comprehensive Cancer Network: cross sectional study

“…There have been concerns that physicians might overestimate the evidence of new drug treatments' efficacy and, therefore, might not be fully aware of residual uncertainty that results from approvals based on clinical trials using surrogate markers as primary endpoints. 11 26 While the NCCN guidelines name the specific surrogate endpoints used in pivotal and postapproval confirmatory trials, opportunities exist for NCCN guidelines to consistently and more comprehensively inform physician and payer decisions by explicitly stating the FDA approval pathway (ie, traditional or accelerated), whether confirmation of clinical benefit is still pending, and the type of endpoints (ie, clinical, surrogate, or composite) used in pivotal and postapproval confirmatory trials in a more standardized format.…”

“… 10 Therefore, there is often uncertainty about the true clinical benefit of drug treatments receiving accelerated approval even after the completion of postapproval confirmatory trials, and concerns have been raised that key evidentiary gaps might not be consistently communicated to patients, physicians, and healthcare payers. 11 12 …”