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Choosing Appropriate Estimands in Clinical Trials.

Abstract：

:Lack of adherence to study protocol and missing data are often unavoidable in clinical trials, and both increase the need to differentiate between the ideal treatment effect if the medication is taken as directed and the treatment effect in presence of the actual adherence pattern. In this regard, estimands have become the focus of attention. An estimand is simply that which is being estimated. In the context of treatment benefit, an estimand may address either efficacy or effectiveness aspects. Defining the estimand of interest is an essential step to take before deciding on trial design and primary analysis. The choice of estimand has consequences for various other factors to be considered during any clinical trial's planning phase. This study presents a process chart including all aspects to consider during planning. After deciding on the primary estimand, the trial design should be specified, followed by the primary analysis. Both should appropriately address the chosen estimand. Finally, sensitivity analyses should be taken into account. Provided are suggestions for all the planning steps involved, especially on choosing between efficacy and effectiveness, and relevant examples from clinical practice to illustrate them. It is recommended that one bear in mind the process chart during planning of any clinical trial and give reasonable justification for each decision in the study protocol.

journal_name

Ther Innov Regul Sci

journal_title

Therapeutic innovation & regulatory science

authors

Leuchs AK,Zinserling J,Brandt A,Wirtz D,Benda N

doi

10.1177/2168479014567317

subject

Has Abstract

pub_date

2015-07-01 00:00:00

pages

584-592

issue

4

eissn

2168-4790

issn

2168-4804

journal_volume

49

pub_type

杂志文章

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