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

Research output: Contribution to journalArticle

Original languageEnglish
JournalInternational Journal of Epidemiology
StateAccepted/In press - 28 Aug 2017


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 pre- or post-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.

    Research areas

  • Mendelian randomization, pharmacovigilance, drug repurposing, adverse drug events



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