Cost-effective clinical trial design: Application of a Bayesian sequential model to the ProFHER pragmatic trial.

ABSTRACT:

Background/aims

There is growing interest in the use of adaptive designs to improve the efficiency of clinical trials. We apply a Bayesian decision-theoretic model of a sequential experiment using cost and outcome data from the ProFHER pragmatic trial. We assess the model's potential for delivering value-based research.

Methods

Using parameter values estimated from the ProFHER pragmatic trial, including the costs of carrying out the trial, we establish when the trial could have stopped, had the model's value-based stopping rule been used. We use a bootstrap analysis and simulation study to assess a range of operating characteristics, which we compare with a fixed sample size design which does not allow for early stopping.

Results

We estimate that application of the mo

SUBMITTER: Forster M 

PROVIDER: S-EPMC8592107 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature