Investigation of subgroups in confirmatory clinical trials - Scientific guideline

This document provides guidance for assessors in European regulatory agencies on assessment of subgroup analyses in confirmatory clinical trials. It should also be useful to clinical trial sponsors and to assessors engaged in providing scientific advice.

Keywords: Subgroup analysis, confirmatory clinical trials, randomised controlled trials, internal consistency, heterogeneity, biostatistics, assessment of clinical trials, analysis plan, exploratory analysis, benefit/risk assessment

Current version

Guideline on the investigation of subgroups in confirmatory clinical trials

AdoptedLegal effective date: 01/08/2019Reference Number: EMA/CHMP/539146/2013

Document history

Guideline on the investigation of subgroups in confirmatory clinical trials

AdoptedLegal effective date: 01/08/2019Reference Number: EMA/CHMP/539146/2013

Draft guideline on the investigation of subgroups in confirmatory clinical trials

Consultation dates: 03/02/2014 to 31/07/2014Draft: consultation closedReference Number: EMA/CHMP/539146/2013Summary:

Investigation into the effects of treatment in well-defined subsets of the trial population is an integral part of clinical trial planning, analysis and inference that follows the inspection of the primary outcome of the trial. The guideline should assist in the planning and presentation of these investigations and in the understanding of factors to be discussed when considering the credibility of findings.

Concept paper on the need for a guideline on the use of subgroup analyses in randomised controlled trials

Consultation dates: 22/04/2010 to 31/07/2010Draft: consultation closedReference Number: EMA/CHMP/EWP/117211/2010