Benchmarking Single-Arm Studies Against Historical Controls From Non-Small Cell Lung Cancer Trials ? An Empirical Analysis Of Bias

Background: Recent trials of novel agents in ?rare? molecular subtypes of non-small cell lung cancer (NSCLC) have used single-arm trial designs and benchmarked outcomes against historical controls. We assessed the consistency of historical control outcomes using docetaxel data from published NSCLC randomized controlled trials (RCTs). Material and methods: Advanced NSCLC RCTs including a docetaxel monotherapy arm were included. Heterogeneity in tumor objective response rates (ORRs), progression-free survival (PFS) and overall survival (OS), and correlations between outcomes and year of trial commencement were assessed. Results: Among 63 trials (N?=?10,633) conducted between 2000 and 2017, ORR ranged from 0% to 26% (I-2?=?76.1%, p(heterogeneity)?更多