Estimands in Clinical Trials and HEOR: Applications and Considerations

PURPOSE: This workshop will focus on the application of the estimand framework to different analyses in trials. Workshop participants will become familiar with using and interpreting estimands to address different post-randomisation events in trials, including treatment switching and attrition due to mortality or disease progression. Participants will also learn about how commonly used statistical methods in trials, such as time-to-event models, can be applied within the estimand framework.

DESCRIPTION: The ICH addendum on estimands in clinical trials was introduced in 2019 and has since been implemented by regulatory agencies around the world. The goal of this estimand framework is to better link trial design, objectives and analysis. Using estimands can improve communication across trial stakeholders and facilitate clearer interpretation of treatment effects. Applying and interpreting estimands is essential for health economics and outcomes researchers working with trials or trial data. This workshop will cover the application, dissemination and use of estimands for statistical and health economic analyses.

Dr. Roydhouse will open the session with a discussion of the estimand framework and examples of estimand use in practice (10m). Dr Floden will discuss the development of estimands for time-to-event endpoints in oncology settings, where attrition due to mortality, progression and toxicity are frequent (10m). Prof Latimer will discuss handling treatment changes when estimating long-term survival for health economic analyses (10m). Dr Regnault will discuss the analysis of patient-reported outcome data with estimands (10m).

The workshop will use real-time polling and discussion with the audience to engage participants and work through questions about the application and use of estimands. The workshop will be beneficial for health economics and outcomes researchers and industry stakeholders working with clinical trials or trial data.