Systematic analysis of off-label and off-guideline cancer therapy usage in a real-world cohort of 165,912 US patients.

ABSTRACT: Patients with cancer may be given treatments that are not officially approved (off-label) or recommended by guidelines (off-guideline). Here we present a data science framework to systematically characterize off-label and off-guideline usages using real-world data from de-identified electronic health records (EHR). We analyze treatment patterns in 165,912 US patients with 14 common cancer types. We find that 18.6% and 4.4% of patients have received at least one line of off-label and off-guideline cancer drugs, respectively. Patients with worse performance status, in later lines, or treated at academic hospitals are significantly more likely to receive off-label and off-guideline drugs. To quantify how predictable off-guideline usage is, we developed machine learning models to predict which

SUBMITTER: Liu R 

PROVIDER: S-EPMC10983036 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature