Estimands in published protocols of randomised trials: urgent improvement needed.

ABSTRACT:

Background

An estimand is a precise description of the treatment effect to be estimated from a trial (the question) and is distinct from the methods of statistical analysis (how the question is to be answered). The potential use of estimands to improve trial research and reporting has been underpinned by the recent publication of the ICH E9(R1) Addendum on the use of estimands in clinical trials in 2019. We set out to assess how well estimands are described in published trial protocols.

Methods

We reviewed 50 trial protocols published in October 2020 in Trials and BMJ Open. For each protocol, we determined whether the estimand for the primary outcome was explicitly stated, not stated but inferable (i.e. could be constructed from the information given), or not inferable.

R

SUBMITTER: Kahan BC 

PROVIDER: S-EPMC8500821 | biostudies-literature | 2021 Oct

REPOSITORIES: biostudies-literature