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Estimands: discussion points from the PSI estimands and sensitivity expert group
Author(s) -
Phillips Alan,
AbellanAndres Juan,
Soren Andersen,
Bretz Frank,
Fletcher Chrissie,
France Lesley,
Garrett Andrew,
Harris Raymond,
Kjaer Magnus,
Keene Oliver,
Morgan David,
O'Kelly Michael,
Roger James
Publication year - 2016
Publication title -
pharmaceutical statistics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.421
H-Index - 38
eISSN - 1539-1612
pISSN - 1539-1604
DOI - 10.1002/pst.1745
Subject(s) - addendum , sensitivity (control systems) , computer science , set (abstract data type) , clinical trial , management science , medical education , psychology , actuarial science , econometrics , medicine , political science , mathematics , economics , engineering , electronic engineering , law , programming language , pathology
ICH E9 Statistical Principles for Clinical Trials was issued in 1998. In October 2014, an addendum to ICH E9 was proposed relating to estimands and sensitivity analyses. In preparation for the release of the addendum, Statisticians in the Pharmaceutical Industry held a 1‐day expert group meeting in February 2015. Topics debated included definition, development, implementation, education and communication challenges associated with estimands and sensitivity analyses. The topic of estimands is an important and relatively new one in clinical development. A clear message from the meeting was that estimands bridge the gap between study objectives and statistical methods. When defining estimands, an iterative process linking trial objectives, estimands, trial design, statistical and sensitivity analysis needs to be established. Each objective should have at least one distinct estimand, supported by sensitivity analyses. Because clinical trials are multi‐faceted and expensive, it is unrealistic to restrict a study to a single objective and associated estimand. The actual set of estimands and sensitivity analyses for a study will depend on the study objectives, the disease setting and the needs of the various stakeholders. Copyright © 2016 John Wiley & Sons, Ltd.