An Overview of Cancer Drugs Approved by the US Food and Drug Administration Based on the Surrogate End Point of Response Rate.

ABSTRACT:

Importance

Approximately one-third of cancer drugs are approved based on response rate (RR)-the percentage of patients whose tumors shrink beyond an arbitrary threshold-typically assessed in a single-arm study.

Objective

To characterize RR end points used by the US Food and Drug Administration (FDA) for cancer drug approval.

Design, setting, and participants

A retrospective review of FDA-approved drug indications in oncology from 2006 to 2018.

Exposures

Data related to cancer type, line of therapy (first-line, second-line, or third-or-later-line treatment for advanced/metastatic disease), type of FDA approval pathway, trial design, sample size, and level of innovation were extracted.

Main outcomes and measures

The primary outcome was the RR used as the b

SUBMITTER: Chen EY 

PROVIDER: S-EPMC6547222 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature