Estimand in benefit-risk assessment

ABSTRACTICH E9(R1) introduces the estimand framework to strengthen dialogues between sponsors and regulators during drug development. A well-structured benefit-risk assessment (BRA) framework also intends to facilitate communication among stakeholders. However, the estimand in ICH E9(R1) is written mainly from the perspective of a single measure of treatment effect in clinical trials. There is lack of systematic discussion on estimand in the context of BRA. This paper initiates the BRA discussion under the estimand framework. By identifying two types of BRA approaches, we summarize and discuss completed clinical trials, using the estimand language for BRA. Benefits and challenges of using estimand for BRA are also discussed.KEYWORDS: Estimandbenefit-risk evaluationpopulation-level BRApatient-level BRA AcknowledgementsBrian Waterhouse, Lianzhe Xu, Shahrul Mt-Isa.Disclosure statementNo potential conflict of interest was reported by the authors.Additional informationFundingThe author(s) reported there is no funding associated with the work featured in this article.