The use of external control data for predictions and futility interim analyses in clinical trials.

ABSTRACT:

Background

External control (EC) data from completed clinical trials and electronic health records can be valuable for the design and analysis of future clinical trials. We discuss the use of EC data for early stopping decisions in randomized clinical trials (RCTs).

Methods

We specify interim analyses (IAs) approaches for RCTs, which allow investigators to integrate external data into early futility stopping decisions. IAs utilize predictions based on early data from the RCT, possibly combined with external data. These predictions at IAs express the probability that the trial will generate significant evidence of positive treatment effects. The trial is discontinued if this predictive probability becomes smaller than a prespecified threshold. We quantify efficiency gains and

SUBMITTER: Ventz S 

PROVIDER: S-EPMC8804894 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature