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The challenges of delivering pharmacogenomics into clinical pediatrics

The Pharmacogenomics Journal volume 2, pages 141–143 (2002)Cite this article

Progress in two major areas over the past 10 years has contributed to increasing optimism for improved understanding of pediatric disease and its pharmacologic management both now and in the future. First, a series of legislative changes targeted improved drug therapy in children of all ages. In 1994, the Food and Drug Administration (FDA) Pediatric Rule essentially provided an avenue for manufacturers to insert information pertaining to the clinical pharmacology and therapeutic use of drugs in pediatric patients into approved product labeling. The FDA Modernization Act (FDAMA) of 1997 provided an incentive for the pharmaceutical industry to complete pediatric studies of marketed drug products with patent life remaining while the FDA Final Rule of 1998 (effective April 1999) currently requires pharmaceutical companies to perform pediatric studies prior to filing of a new drug application (NDA) on new drugs that could benefit pediatric patients and/or are anticipated to be used in a substantial number (ie >50000) of pediatric patients. These legislative changes produced a dramatic increase in the number (ie >400) of pediatric clinical trials conducted in the US (mostly pharmacokinetic), involving more than 50000 children ranging in age from birth to adolescence and resulted in expanded pediatric labeling for more than 30 drug products.1

The Human Genome Project, initiated in 1990 with the goal of sequencing the entire complement of human genes by the year 2005, is the second development that is expected to impact the diagnosis and treatment of pediatric diseases. This three billion dollar public investment is expected to lead to decreased morbidity and mortality through the development of more effective strategies to diagnose, treat and prevent human disease.2 Society has every right to expect that adults and children will be able to benefit equally from the promise of the genome era. In pediatrics, the newly acquired information has considerable potential to improve our understanding of: (1) genetic determinants of age-specific disease processes; (2) genetic and developmental factors that contribute to the increased susceptibility of children to certain drug-related adverse events; and (3) genetic factors that influence interindividual variability in drug disposition and response across the entire pediatric age spectrum. Nevertheless, there are several factors unique to pediatrics that must be addressed before pharmacogenetic and pharmacogenomic strategies can be fully implemented in the context of pediatric disease. We will provide some perspectives on these issues as well as current ethical and methodological concerns that impact the translation of these strategies to pediatric clinical medicine.

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  1. Division of Pediatric Pharmacology and Medical Toxicology, Section of Developmental Pharmacology and Experimental Therapeutics, Children's Mercy Hospital and Clinics, Kansas City, MO, USA

    J S Leeder & G L Kearns

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  1. J S Leeder
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  2. G L Kearns
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Correspondence to J S Leeder.

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Leeder, J., Kearns, G. The challenges of delivering pharmacogenomics into clinical pediatrics. Pharmacogenomics J 2, 141–143 (2002). https://doi.org/10.1038/sj.tpj.6500104

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