In recent years, pharmacogenomic research has begun to integrate genetics, gene expression and pharmacological phenotypes. MicroRNAs (miRNAs), 21- to 25-nucleotide, non-coding RNAs that are present in almost all metazoan genomes, are a class of gene regulators that downregulate gene expression at the post-transcriptional level. Experimental evidence for the role of miRNAs in regulating pharmacology-related genes and drug responses is increasing. Given the universal roles of miRNAs in various diseases, including cancer, miRNAs (eg, chemotherapy) are anticipated to have potential therapeutic effects in various diseases. The incorporation of miRNAs into pharmacogenomic research could provide improved insight into drug responses. However, more studies are necessary to evaluate the effects of these molecules in patients.