Precision Medicine in Children

A Review of the Literature

Marcia L. Buck, PharmD, FCCP, FPPAG, BCPPS


Pediatr Pharm. 2017;23(2) 

In This Article

Abstract and Introduction


Precision medicine, also referred to as personalized medicine, incorporates the patient's individual characteristics into treatment, rather than relying on population means. Over the past several years, it has become a significant focus for research. Developments in pharmacogenomics, the study of genomic variations that influence response to drugs, has accounted for much of the recent work in this area.[1,2] As of November 2016, the prescribing information for more than 150 drugs included guidance on using pharmacogenomics information in drug selection or dosing.[3] Understanding and incorporating genomic variation, however, is just one component of providing precision medicine. Age, gender, environment, and comorbidities are only a few of the potential factors to be considered in providing personalized pharmacotherapy.

Research in pediatric precision medicine, including pharmacogenomics, is growing at a rapid rate. A 2015 review of the Food and Drug Administration's pharmacogenomics biomarker labeling data identified 38 drugs with information pertinent to use of the drug in pediatric patients.[4] The authors also identified 137 pediatric pharmacogenomic studies listed on Information generated from studies in adults often can be of value in treating children, such as the identification of the correlation of human leukocyte antigen B variant allele HLA-B*1502 and carbamazepine-induced Stevens Johnson Syndrome or toxic epidermal necrolysis and the role of thiopurine methyltransferase polymorphisms in the development of thiopurine-induced bone marrow suppression.[5–7] The following studies represent a sample of the recent literature in this new field.