Methods for observational post-licensure medical product safety surveillance. (April 2015)

• Record Type: Journal Article
• Authors: Nelson, Jennifer C; Cook, Andrea J; Yu, Onchee; Zhao, Shanshan; Jackson, Lisa A; Psaty, Bruce M
• Is Part Of: Statistical methods in medical research. Volume 24:Number 2(2015:Apr.)
• Page Start: 177
• Page End: 193
• Publication Date: 2015-04
• Subjects: data mining -- observational study -- post-licensure safety -- sequential testing; Medicine -- Research -- Statistical methods -- Periodicals; Research -- Periodicals; Review Literature -- Periodicals; Statistics -- methods -- Periodicals; Médecine -- Recherche -- Méthodes statistiques -- Périodiques; 610.727
• DOI: 10.1177/0962280211413452
• Languages: English
• ISSNs: 0962-2802

Abstract

Post-licensure medical product safety surveillance is important for detecting adverse events potentially not identified pre-licensure. Historically, post-licensure safety monitoring has been accomplished using passive reporting systems and by conducting formal Phase IV randomized trials or large epidemiological studies, also known as safety surveillance or pharmacovigilance studies. However, crucial gaps in the safety evidence base provided by these approaches have led to high profile product withdrawals and growing public concern about unknown health risks associated with licensed products. To address the limitations of existing surveillance systems and to facilitate more accurate and rapid detection of safety problems, new systems involving active surveillance of large, population-based cohorts using observational health care databases are being developed. In this article, we review common statistical methods that have been employed previously for post-licensure safety monitoring, including data mining and sequential hypothesis testing, and assess which methods may be promising for potential use within this newly proposed prospective observational cohort monitoring framework. We discuss gaps in existing approaches and identify areas where methodological development is needed to improve the success of safety surveillance efforts in this setting.