Predictors of withdrawal of anticancer drug indications granted accelerated approval: a retrospective cohort study.

ABSTRACT:

Background

The accelerated approval pathway allows the FDA to approve drugs for serious conditions based on promising surrogate measures, with confirmatory studies required later. If subsequent testing shows an unfavorable benefit-risk profile, the indication may be withdrawn. This study aimed to identify factors associated with the withdrawal of drug indications following accelerated approval.

Methods

In this retrospective cohort study, we identified FDA-approved drugs for solid and hematologic cancers from 1992 to 2022 and extracted factors present at the time of accelerated approval, including pivotal trial characteristics, outcomes, and confirmatory study initiation timing from drug labels and published reports. Clinical benefit was assessed using the European Society of

SUBMITTER: Tibau A 

PROVIDER: S-EPMC12273735 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature