European regulatory use and impact of subgroup evaluation in marketing authorisation applications. Issue 12 (December 2017)

• Record Type: Journal Article
• Authors: Tanniou, Julien; Teerenstra, Steven; Hassan, Sagal; Elferink, Andre; van der Tweel, Ingeborg; Gispen-de Wied, Christine; Roes, Kit C.B.
• Is Part Of: Drug discovery today. Volume 22:Issue 12(2017)
• Page Start: 1760
• Page End: 1764
• Publication Date: 2017-12
• Subjects: Drugs -- Design -- Periodicals; Drugs -- Research -- Periodicals; 615.1
• DOI: 10.1016/j.drudis.2017.09.012
• Languages: English
• ISSNs: 1359-6446

Abstract

Highlights: Subgroups — prominent role in marketing authorisation applications (MAAs). Regulators — how and for what purpose(s) are assessments made? Assessments — mainly related to consistency or heterogeneity of treatment effect. Main role — choice of the final indication. Abstract : Marketing authorisation application dossiers relating to medicinal products containing new active substances and evaluated by the European Medicines Agency (EMA) over the period 2012–2015 were examined. Major objections and other concerns relating to efficacy and safety of the day 80 assessment reports were reviewed. Overall, approved products have more subgroup concerns than nonapproved products, which seems to be a consistent pattern. Subgroup analyses are mainly assessed to have the insurance that subgroups of patients that might lack a positive benefit: risk ratio will not be wrongly included in the approved treatment indication.