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Off-Label Drug Use in Pediatrics


In pediatrics, off-label use is common due to multiple reasons. The primary factor that determines whether a clinician chooses to use unapproved drugs is the availability of medications that provide instructions for dosage for pediatric patients (Bazzano, Mangione-Smith, Schonlau, Suttorp, & Brook, 2009). In many cases, these guidelines are unavailable, and healthcare workers make a decision to use medicine that would help the patient’s specific situation.

While prescribing off-label use, medical professionals should rely on the latest and most reliable evidence to ensure that the child is safe. Moreover, such actions should be preceded by calculations that acknowledge the patient’s age and specific pharmacokinetic differences. Medications for cardiovascular conditions, antidepressants, antibacterials, and anti-inflammatory drugs can be considered as examples of being frequently prescribed off-label (Czaja, Reiter, Schultz, & Valuck, 2015; Palmaro et al., 2015). To care for pediatric patients, health care providers should utilize the most recent and trustworthy evidence as well as pharmacokinetic specificities of children when prescribing off-label use.

Prescription Requirements

There are many situations in which clinicians choose to prescribe children off-label drugs. The decision to utilize unapproved medications does not mean that they are harmful or dangerous but that the official research for pediatric patients is absent or lacking. However, as a wide variety of medicine does not contain any information for children, health care providers are limited in their ability to use only approved medicines.

For example, children with complex chronic conditions often have symptoms that cannot be alleviated or managed by the available medications. Other cases include children with organ failure or dysfunction, rare diseases, the need for emergency care, and young age (less than 5 years old) (Czaja et al., 2015). According to Neville et al. (2014), health care professionals should prescribe drugs off-label to achieve the primary goal of treatment – caring for the patient’s well-being and health-related needs. Clinicians have many reasons to prescribe off-label drug use, but they should remember to do it safely.

Strategies to Make Off-Label Use Safe

The main strategy of making off-label use as safe as possible lies in the use of reliable evidence. Neville et al. (2014) state that label indications are not always updated enough to represent all possible situations that a clinician may encounter. Therefore, the gold standard of evidence expressed through available studies and examples should be used by health care providers if the particular situation calls for it.

Furthermore, the increased attention to research and approval of medicines for children should be outlined as a necessity for future considerations of organizations such as the FDA. However, if a specialist has to make a decision at the present time, he or she should use the available knowledge and information. It should be noted that children of various ages respond to drugs differently. For example, the pharmacokinetics of adolescents is similar to that of adults, but infants have a low rate of medication absorption and elimination (Arcangelo, Peterson, Wilbur, & Reinhold, 2017). Thus, the dosage should be calculated with the acknowledgment of these factors.

Some specific examples of medications that should be treated with caution include neurological and cardiovascular drugs. Czaja et al. (2015) reveal that many high-priority drugs such as ketamine, hydromorphone, lorazepam, and methadone are used off-label, although the FDA does not approve them for children. Czaja et al. (2015) also provide such examples of high alert drugs as bupivacaine, fentanyl, insulin aspart, and others. Many of them have a high-risk status, which means that they have harmful side effects or the potential to endanger patients if misused.


Off-label use is inescapable in pediatric care at the present moment because clinicians often have a limited choice of approved medications for children. Young patients with chronic conditions, organ failure, and acute problems require off-label drugs more often than others. To make such prescriptions safer, health care providers should use reliable evidence and remember the differences in children’s pharmacokinetics. Professionals should also pay increased attention to high-risk drugs.


Arcangelo, V. P., Peterson, A. M., Wilbur, V., & Reinhold, J. A. (Eds.). (2017). Pharmacotherapeutics for advanced practice: A practical approach (4th ed.). Ambler, PA: Lippincott Williams & Wilkins.

Bazzano, A. T., Mangione-Smith, R., Schonlau, M., Suttorp, M. J., & Brook, R. H. (2009). Off-label prescribing to children in the United States outpatient setting. Academic Pediatrics, 9(2), 81-88.

Czaja, A. S., Reiter, P. D., Schultz, M. L., & Valuck, R. J. (2015). Patterns of off-label prescribing in the pediatric intensive care unit and prioritizing future research. The Journal of Pediatric Pharmacology and Therapeutics, 20(3), 186-196.

Neville, K. A., Frattarelli, D. A., Galinkin, J. L., Green, T. P., Johnson, T. D., Paul, I. M., & Van Den Anker, J. N. (2014). Off-label use of drugs in children. Pediatrics, 133(3), 563-567.

Palmaro, A., Bissuel, R., Renaud, N., Durrieu, G., Escourrou, B., Oustric, S.,… Lapeyre-Mestre, M. (2015). Off-label prescribing in pediatric outpatients. Pediatrics, 135(1), 49-58.

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"Off-Label Drug Use in Pediatrics." StudyKraken, 20 Sept. 2021,

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StudyKraken. "Off-Label Drug Use in Pediatrics." September 20, 2021.


StudyKraken. 2021. "Off-Label Drug Use in Pediatrics." September 20, 2021.


StudyKraken. (2021) 'Off-Label Drug Use in Pediatrics'. 20 September.

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