Estimating treatment effects from a randomized controlled trial with mid-trial design changes.

ABSTRACT: BackgroundIn randomized controlled trials (RCTs), unplanned design modifications due to unexpected circumstances are seldom reported. Naively lumping data from pre- and post-design changes to estimate the size of the treatment effect, as planned in the original study, can introduce systematic bias and limit interpretability of the trial findings. There has been limited discussion on how to estimate the treatment effect when an RCT undergoes major design changes during the trial. Using our recently completed RCT, which underwent multiple design changes, as an example, we examined the statistical implications of design changes on the treatment effect estimates.MethodsOur example RCT aimed to test an advance care planning intervention targeting dementia patients and their surrogate decision-m

SUBMITTER: Paul S 

PROVIDER: S-EPMC11996067 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature