Medication safety research by observational study design

Int J Clin Pharm. 2016 Jun;38(3):676-84. doi: 10.1007/s11096-016-0285-6. Epub 2016 Mar 22.


Observational studies have been recognised to be essential for investigating the safety profile of medications. Numerous observational studies have been conducted on the platform of large population databases, which provide adequate sample size and follow-up length to detect infrequent and/or delayed clinical outcomes. Cohort and case-control are well-accepted traditional methodologies for hypothesis testing, while within-individual study designs are developing and evolving, addressing previous known methodological limitations to reduce confounding and bias. Respective examples of observational studies of different study designs using medical databases are shown. Methodology characteristics, study assumptions, strengths and weaknesses of each method are discussed in this review.

Keywords: Methodology; Observational study; Pharmacoepidemiology.

Publication types

  • Review

MeSH terms

  • Databases, Pharmaceutical*
  • Humans
  • Observational Studies as Topic / methods*
  • Pharmacoepidemiology / methods*
  • Research Design*