title: Estimands: discussion points from the PSI estimands and sensitivity expert group.
abstract: 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.
authors.affiliation: Biostatistics, ICON, Marlow, UK.
authors.forename: Alan
authors.initials: A
authors.lastname: Phillips
authors.affiliation: Data Sciences, Grunenthal, Aachen, Germany.
authors.forename: Juan
authors.initials: J
authors.lastname: Abellan-Andres
authors.affiliation: Biostatistics, Novo Nordisk AS, Bagsvaerd, Denmark.
authors.forename: Andersen
authors.initials: A
authors.lastname: Soren
authors.affiliation: Novartis, Basel, Switzerland.
authors.forename: Frank
authors.initials: F
authors.lastname: Bretz
authors.affiliation: Biostatistics, Amgen Ltd, Cambridge, UK.
authors.forename: Chrissie
authors.initials: C
authors.lastname: Fletcher
authors.affiliation: Biometrics and Information Sciences, AstraZeneca, Cheshire, UK.
authors.forename: Lesley
authors.initials: L
authors.lastname: France
authors.affiliation: Quintiles, Bracknell, UK.
authors.forename: Andrew
authors.initials: A
authors.lastname: Garrett
authors.affiliation: Biostatistics, Eisai Limited, Hatfield, UK.
authors.forename: Raymond
authors.initials: R
authors.lastname: Harris
authors.affiliation: ECD, AstraZeneca Gothenburg, Sweden.
authors.forename: Magnus
authors.initials: M
authors.lastname: Kjaer
authors.affiliation: GlaxoSmithKline, Uxbridge, UK.
authors.forename: Oliver
authors.initials: O
authors.lastname: Keene
authors.affiliation: Independent Consultant, Wokingham, UK.|Department of Pharmaceutical Medicine, Kings College London.
authors.forename: David
authors.initials: D
authors.lastname: Morgan
authors.affiliation: Advisory Services Analytics, Quintiles, Dublin, Ireland.
authors.forename: Michael
authors.initials: M
authors.lastname: O'Kelly
authors.affiliation: Livedata (UK) Ltd, Chinnor, UK.|London School of Hygiene and Tropical Medicine, London, UK.
authors.forename: James
authors.initials: J
authors.lastname: Roger
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D003627:Data Interpretation, Statistical; D015195:Drug Design; D004345:Drug Industry / Q000379:methods*; D006801:Humans; D015233:Models, Statistical; D012107:Research Design
keywords: ICH E9; estimands; sensitivity analyses