Survival-Inferred Fragility of Statistical Significance in Phase III Oncology Trials.

ABSTRACT:

Background

Statistical significance currently defines superiority in phase III oncology trials. However, this practice is increasingly questioned. Here, we estimated the fragility of phase III oncology trials.

Methods

Using Kaplan-Meier curves for the primary endpoints of 230 two-arm superiority phase III oncology trials, we reconstructed data for individual patients. We estimated the survival-inferred fragility index (SIFI) by iteratively flipping the best responder from the experimental arm to the control arm (SIFI_B) until the interpretation was changed according to the significance threshold of each trial. Severe fragility was defined by SIFI ≤1%.

Results

This study included 230 trials enrolling 184,752 patients. The median number of patients required to

SUBMITTER: Sherry AD 

PROVIDER: S-EPMC11759605 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature