Off-Label Drug Use in Pediatrics

Specific off-label drugs can be prescribed for children under certain circumstances and conditions. Off-label medicines are used when there is an inconsistency or lack of pediatric information in Summary of Product Characteristics (SmPC) (Lee et al., 2018). For example, there is no safe and effective therapeutic information in SmPC for biliary atresia, a condition common in infants (Lee et al., 2018). As a result, Rifampicin is used to induce enzymes among infants with biliary atresia due to the absence of therapeutic options for the condition (Allen et al., 2018). Another circumstance under which children can be prescribed off-label drugs is convincing evidence that such medicines are safe and effective in particular situations. For example, the use of Sildenafil has proved to be effective and safe in managing pulmonary hypertension in children (Schrier et al., 2020). Physicians know their patients’ conditions well and have the right to prescribe off-label medicines when they have convincing evidence on the effectiveness of prescriptions for better patient care.

One of the strategies to use off-label drugs safely for children is to administer medication to inpatients only and not outpatients for close monitoring and examinations of adverse reactions. Adverse medication reactions are reported as per the physicians’ professional code for documentation to be used as a reference in subsequent cases (Schrier et al., 2020). Another strategy is to apply an electronic health system in the prescription to improve its quality by integrating diagnosis parameters, including body weight, medical history, age, etc., for complex dosage calculations (De Zen et al., 2018). Tramadol is an off-label drug and an excellent opioid analgesic for relieving pain. Cidofovir is also an off-label drug used in pediatrics and a nucleoside analog used as antiviral agents. Another off-label drug is clozapine that treats schizophrenia as an antipsychotic agent.


Allen, H.C., Garbe, M.C., Lees, J., Aziz, N., Chaaban, H., Miller, J.L., Johnson, P., & DeLeon, S. (2018). Off-label medication use in children, more common than we think: A systematic review of the literature. The Journal of the Oklahoma State Medical Association, 111(8), 776.

De Zen, L., Marchetti, F., Barbi, E., & Benini, F. (2018). Off-label drugs use in pediatric palliative care. Italian Journal of Pediatrics, 44(1), 1-6.

Lee, J. H., Byon, H. J., Choi, S., Jang, Y. E., Kim, E. H., Kim, J. T., & Kim, H. S. (2018). Safety and efficacy of off-label and unlicensed medicines in children. Journal of Korean Medical Science, 33(37), e227.

Schrier, L., Hadjipanayis, A., Stiris, T., Ross-Russell, R.I., Valiulis, A., Turner, M.A., Zhao, W., De Cock, P., de Wildt, S.N., Allegaert, K., & van den Anker, J. (2020). Off-label use of medicines in neonates, infants, children, and adolescents: A joint policy statement by the European Academy of Paediatrics and the European Society for Developmental Perinatal and Pediatric Pharmacology. European Journal of Pediatrics, 179(5), 839-847.

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