Design analysis indicates Potential overestimation of treatment effects in randomized controlled trials supporting Food and Drug Administration cancer drug approvals.

ABSTRACT:

Objective

Statistical significance drives interpretation of randomized controlled trials (RCTs). We examined the type S error risk-claiming a new drug is falsely beneficial-and exaggeration ratio-how estimated effects differ from true effects-to re-emphasize direction and magnitude of treatment effects.

Study design and setting

We systematically reviewed RCTs supporting Food and Drug Administration (FDA) approval of cancer drugs between 2007 and 2016. We extracted data for overall survival (OS), progression-free survival (PFS), and response outcomes from FDA reviews. We estimated type S error risks and exaggeration ratios by considering replicated RCTs of equal size and a range of true effects.

Results

We analyzed 42 RCTs for 39 approved drugs. Across 38 RCTs reportin

SUBMITTER: Lord EM 

PROVIDER: S-EPMC8978539 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature