Mendelian randomization: A novel approach for the prediction of adverse drug events and drug repurposing opportunities

Venexia M. Walker*, George Davey Smith, Neil M. Davies, Richard M. Martin

*Corresponding author for this work

Research output: Contribution to journalArticle (Academic Journal)peer-review

89 Citations (Scopus)
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Identification of unintended drug effects, specifically drug repurposing opportunities and adverse drug events, maximizes the benefit of a drug and protects the health of patients. However, current observational research methods are subject to several biases. These include confounding by indication, reverse causality and missing data. We propose that Mendelian randomization (MR) offers a novel approach for the prediction of unintended drug effects. In particular, we advocate the synthesis of evidence from this method and other approaches, in the spirit of triangulation, to improve causal inferences concerning drug effects. MR addresses some of the limitations associated with the existing methods in this field. Furthermore, it can be applied either before or after approval of the drug, and could therefore prevent the potentially harmful exposure of patients in clinical trials and beyond. The potential of MR as a pharmacovigilance and drug repurposing tool is yet to be realized, and could both help prevent adverse drug events and identify novel indications for existing drugs in the future.

Original languageEnglish
Pages (from-to)2078-2089
Number of pages12
JournalInternational Journal of Epidemiology
Issue number6
Early online date11 Oct 2017
Publication statusPublished - 1 Dec 2017


  • Adverse drug events
  • Drug repurposing
  • Mendelian randomization
  • Pharmacovigilance


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