RT Journal Article
SR Electronic
T1 Estimands: bringing clarity and focus to research questions in clinical trials
JF BMJ Open
JO BMJ Open
FD British Medical Journal Publishing Group
SP e052953
DO 10.1136/bmjopen-2021-052953
VO 12
IS 1
A1 Timothy Peter Clark
A1 Brennan C Kahan
A1 Alan Phillips
A1 Ian White
A1 James R Carpenter
YR 2022
UL http://bmjopen.bmj.com/content/12/1/e052953.abstract
AB Precise specification of the research question and associated treatment effect of interest is essential in clinical research, yet recent work shows that they are often incompletely specified. The ICH E9 (R1) Addendum on Estimands and Sensitivity Analysis in Clinical Trials introduces a framework that supports researchers in precisely and transparently specifying the treatment effect they aim to estimate in their clinical trial. In this paper, we present practical examples to demonstrate to all researchers involved in clinical trials how estimands can help them to specify the research question, lead to a better understanding of the treatment effect to be estimated and hence increase the probability of success of the trial.