Beyond "Intent-to-treat" and "Per protocol": Improving assessment of treatment effects in clinical trials through the specification of an estimand
Publication year
2020Source
British Journal of Clinical Pharmacology, 86, 7, (2020), pp. 1235-1239ISSN
Publication type
Article / Letter to editor

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Organization
Health Evidence
IQ Healthcare
Journal title
British Journal of Clinical Pharmacology
Volume
vol. 86
Issue
iss. 7
Page start
p. 1235
Page end
p. 1239
Subject
Radboudumc 18: Healthcare improvement science RIHS: Radboud Institute for Health SciencesAbstract
There is a key problem in randomised clinical trials as outcomes can be distorted due to informative post-randomisation events. This is inadequately addressed by the use of traditional intention-to-treat or per protocol analysis sets and often either ignored or wrongly labelled as missing data. As a consequence, the treatment effects of interest in a clinical trial are not well defined and their estimates might be misinterpreted. The estimand framework should help all those planning, conducting and analysing clinical trials as well as those interpreting the results to better define, estimate and understand the treatment effects of interest. This framework is described in the addendum to ICH E9 and addresses precisely this problem. It is relevant for regulatory drug trials and academic-run trials, as well as for trials of nonpharmacological interventions.
This item appears in the following Collection(s)
- Academic publications [204968]
- Electronic publications [103219]
- Faculty of Medical Sciences [81049]
- Open Access publications [71773]
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