Garcia de Albeniz Martinez X, Chirila C, Hernan MA, Leaver DRM. Ready or not, here come estimands! The emerging attention to causal estimands in real-world evidence studies. Presented at the 39th ICPE Annual Conference; August 25, 2023. Halifax, Canada.

BACKGROUND: An estimand is a quantity that we would like to estimate using data. The so-called “estimand framework” was proposed in 2019 by the International Council of Harmonisation (ICH) to describe causal estimands other than the intention-to-treat effect in randomized trials. The goal is to better align a trial’s objective, design, analysis, and interpretation. This framework is endorsed by the European Medicines Agency and by the US Food and Drug Administration. Target trial emulation was developed to improve causal inference in observational analyses of real-world data (RWD). Because this framework starts by specifying the causal estimands in the (randomized) target trial, it has played a crucial role in reconciling results of randomized trials and observational studies. The causal estimands considered in target trials are different than those described by the ICH framework. Therefore, investigators who work with both randomized trials and observational studies find themselves having to master both frameworks. This situation is exacerbated by the emergent use of external comparators in randomized trials. This symposium is aimed to researchers who work with RWD, particularly in studies used for drug development and regulatory purposes such as external control arms.

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