A narrative review of estimands in drug development and regulatory evaluation: old wine in new barrels?

ABSTRACT:

Background

An estimand defines the target of estimation for a clinical trial through specification of the treatment, target population, variable, population-level summary and of the strategies for intercurrent events. A carefully defined estimand aligns the clinical trial design and analysis with the scientific question of interest and adequately accounts for so-called intercurrent events. The ICH E9(R1) addendum suggests five estimand strategies. We evaluated to what extent current practice in drug development and regulatory assessment fits in the estimand framework.

Methods

We systematically evaluated what estimands, especially what strategies for intercurrent events are advised in European Medicines Agency disease guidelines, used in sponsors' trials and additionally requ

SUBMITTER: Mitroiu M 

PROVIDER: S-EPMC7376663 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature