Adherence-adjustment in placebo-controlled randomized trials: An application to the candesartan in heart failure randomized trial.

ABSTRACT:

Background

The per-protocol effect provides important information in randomized trials with incomplete adherence. Yet, because valid estimation typically requires adjustment for prognostic factors that predict adherence, per-protocol effect estimates are often met with skepticism. In placebo-controlled trials, however, the validity of adjustment can be indirectly verified by demonstrating no association between adherence and the outcome among the placebo arm. Here, we describe a two-stage procedure in which we first adjust for time-varying adherence in the placebo arm and then use a similar procedure to estimate the per-protocol effect.

Methods

We use the Candesartan in Heart Failure: Assessment of Reduction in Mortality and Morbidity (CHARM) randomized trial. First, we comp

SUBMITTER: Murray EJ 

PROVIDER: S-EPMC12107675 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature