Determining sample size in a personalized randomized controlled (PRACTical) trial.

ABSTRACT: In clinical settings with no commonly accepted standard-of-care, multiple treatment regimens are potentially useful, but some treatments may not be appropriate for some patients. A personalized randomized controlled trial (PRACTical) design has been proposed for this setting. For a network of treatments, each patient is randomized only among treatments which are appropriate for them. The aim is to produce treatment rankings that can inform clinical decisions about treatment choices for individual patients. Here we propose methods for determining sample size in a PRACTical design, since standard power-based methods are not applicable. We derive a sample size by evaluating information gained from trials of varying sizes. For a binary outcome, we quantify how many adverse outcomes would be pr

SUBMITTER: Turner RM 

PROVIDER: S-EPMC7616655 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature