Tools to help patients and other stakeholders' input into choice of estimand and intercurrent event strategy in randomised trials

TL;DR

This paper introduces three practical tools—a video, infographic, and editable slides—to facilitate stakeholder involvement in selecting estimands and intercurrent event strategies in randomized trials, aiming to improve communication and decision-making.

Contribution

It provides accessible resources to help researchers, patients, and stakeholders collaboratively understand and choose estimands and intercurrent event strategies in clinical trials.

Findings

Tools improve stakeholder understanding of estimand concepts.

Resources facilitate better communication in trial design discussions.

Enhanced stakeholder involvement may lead to more relevant trial outcomes.

Abstract

Estimands can help to clarify the research questions being addressed in randomised trials. Because the choice of estimand can affect how relevant trial results are to patients and other stakeholders, such as clinicians or policymakers, it is important for them to be involved in these decisions. However, there are barriers to having these conversations. For instance, discussions around how intercurrent events should be addressed in the estimand definition typically involve complex concepts as well as technical language. We three tools to facilitate conversations between researchers and patients and other stakeholders about the choice of estimand and intercurrent event strategy: (i) a video explaining the concept of an estimand and the five different ways that intercurrent events can be incorporated into the estimand definition; (ii) an infographic outlining these five strategies; and (iii) an editable PowerPoint slide which can be completed with trial-specific details to facilitate conversations around choice of estimand for a particular trial. These resources can help to start conversations between the trial team and patients and other stakeholders about the best choice of estimand and intercurrent event strategies for a randomised trial.