What is the research question? Estimands explained

European Journal of Endocrinology, vol. 192, pp. E5–E7

Abstract

Although many papers of medical research report on a treatment effect, it is not always clear what is exactly meant by that effect. An estimand is a precise definition of a treatment effect and includes 5 attributes: population, treatment, (outcome) variable, intercurrent events, and summary measure. In this paper, we discuss how the estimand framework helps to align different phases of studies of medical treatments, from research objectives to design, conduct, analysis, and reporting of results.

Authors 3

1. Rolf H. H. Groenwold corresponding Aachen Department of Biomedical Data Sciences Department of Clinical Epidemiology

   Leiden University Medical Center

   Affiliation as printed

   Department of Biomedical Data Sciences, Leiden University Medical Center , 2333ZA Leiden ,

   Department of Clinical Epidemiology, Leiden University Medical Center , 2333ZA Leiden ,

   Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, the Netherlands

   Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands

2. Saskia le Cessie Aachen Department of Biomedical Data Sciences Department of Clinical Epidemiology

   Leiden University Medical Center

   Affiliation as printed

   Department of Biomedical Data Sciences, Leiden University Medical Center , 2333ZA Leiden ,

   Department of Clinical Epidemiology, Leiden University Medical Center , 2333ZA Leiden ,

   Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, the Netherlands

   Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands

3. Olaf M. Dekkers Aachen Department of Clinical Epidemiology Department of Endocrinology, Leiden University Medical Center

   Aarhus University · Leiden University Medical Center · Aarhus University Hospital

   Affiliation as printed

   Department of Clinical Epidemiology, Aarhus University and Aarhus University Hospital , 8200 Aarhus ,

   Department of Clinical Epidemiology, Leiden University Medical Center , 2333ZA Leiden ,

   Department of Endocrinology, Leiden University Medical Center , 2333ZA Leiden ,

   Department of Clinical Epidemiology, Aarhus University and Aarhus University Hospital, Aarhus, Denmark

   Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, the Netherlands

   Department of Endocrinology, Leiden University Medical Center, Leiden, the Netherlands