The use of off-label and unlicensed medications is common and necessary in pediatrics. However, it poses potential risks due to lower levels of evidence on the pharmacokinetic (PK) profile, drug efficacy, and safety profile (i.e., pharmacodynamics [PD]), and potential genetic modifiers of PK and PD. In general, the PK variation is not only significant along the developmental trajectory but also within specific age and disease categories. Accordingly, when expected clinical effect is not realized or when adverse events are observed, the lack of evidence-based specific information on established drug doses and achieved drug concentrations results in challenges for the optimal clinical care for children.

Various international initiatives (e.g., Pediatric Trials Network) as well as policies from regulatory agencies (e.g., Food and Drug Administration, European Medicines Agency) aim to close the information gap for drugs that are already used as well as those that are currently in development, respectively. While these initiatives are an important first step to improve pharmaceutical care for children, a gap remains in achieving the goals of precision medicine for the individual child.

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