Assignment Pediatrics Week 11

Assignment Pediatrics Week 11

Off-Label Drug Use in Children

Off-label use refers to the use of a drug for an unapproved indication, age group, dosage, or route of administration (Yackey et al., 2019). Off-label drug use is a relatively common practice in the United States. It is legal and has an estimated prevalence of 25% (Yackey et al., 2019). The US Food and Drug Administration (FDA) necessitates that all pharmacologic agents are tested for safety and efficacy at a particular dosage prior to approval for clinical use in a specific population.

However, children pose ethical, clinical, scientific, and logistical concerns that limit this process. Consequently, a vast majority of medication use in the pediatric age group is off-label as there is limited comprehension of safety, efficacy, and appropriate dosage (Yackey et al., 2019). In the subsequent paragraphs, circumstances under which children should be prescribed drugs for off-label use, strategies to ensure safer off-label use, and dosage of drugs from infancy to adolescents and off-label drugs that require extra care and attention when used in pediatrics shall be discussed.

Circumstances for Off-label Drug Use in Children

Pediatric patients may be prescribed off-label drugs for various reasons. Pediatric patients who have exhausted approved options may resort to off-label drugs (Mei et al., 2019). For instance, nortriptyline for ADHD after a failed response to standard therapy with stimulants and atomoxetine. Similarly, unavailability of the standard treatment related to the cost, desired strength, and formulation may lead to off-label prescription in children (Schrier et al., 2020).

For instance, the lack of appropriate strength of fluoxetine, an FDA-approved drug, may lead to the use of sertraline in the treatment of depression in children. Off-label drugs may also be used when there is overwhelming scientific evidence regarding their effectiveness in the management of various pediatric conditions, and the benefits outweigh the risks (van der Zanden et al., 2021).

For instance, lithium is the only FDA-approved mood stabilizer for use in children over 12 years. However, due to its benefits, lamotrigine, an anticonvulsant, has been prescribed as a mood stabilizer in children. Off-label drugs are also prescribed when the FDA-approved drugs are not feasible due to drug-drug interactions and devastating side effects. Finally, rare diseases with no FDA-approved medication may warrant off-label drugs.

Strategies for Safer Off-Label Drug Use

The off-label drug is not always safe. Subsequently, strategies have to be developed and implemented to enhance the efficacy of this practice as well as the patient’s safety. One such strategy is ensuring that prescribers use off-label drugs backed by substantial scientific evidence (Schrier et al., 2020). Likewise, physicians should only prescribe off-label drugs when the benefits vividly outweigh the risks (Rusz et al., 2021).

Furthermore, following prescription, the healthcare providers should closely monitor their patients for the onset of side effects and response to treatment. Additionally, patients and their caregivers should be enlightened on the distinct medications they are taking, including their use, drug interactions, and side effects. Finally, prescribers should precisely calculate the dosage for age and weight (Rusz et al., 2021).

Off-label drug use in the pediatric age group is a safety threat due to disparities in pharmacokinetics and pharmacodynamics (Schrier et al., 2020). Drugs that require extra care include SSRIs due to increased risk of suicidal ideation, particularly in adolescents. Likewise, lithium, valproic acid, tricyclic antidepressants, lamotrigine, atypical antidepressants, and carbamazepine have to be used cautiously in children as their safety has not been established.


Off-label drug use is common and legal in the United States, provided it is backed by scientific research. Off-label drug use is not always safe. Consequently, a risk-benefit analysis must be undertaken before the off-label prescription of drugs. Children are commonly prescribed off-label drugs due to limited scientific research and hence limited information regarding the safety and efficacy of drugs in this age group.


Mei, M., Xu, H., Wang, L., Huang, G., Gui, Y., & Zhang, X. (2019). Current practice and awareness of pediatric off-label drug use in Shanghai, China -a questionnaire-based study. BMC Pediatrics19(1), 281.

Rusz, C.-M., Ősz, B.-E., Jîtcă, G., Miklos, A., Bătrînu, M.-G., & Imre, S. (2021). Off-label medication: From a simple concept to complex practical aspects. International Journal of Environmental Research and Public Health18(19), 10447.

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 Pediatrics179(5), 839–847.

van der Zanden, T. M., Mooij, M. G., Vet, N. J., Neubert, A., Rascher, W., Lagler, F. B., Male, C., Grytli, H., Halvorsen, T., de Hoog, M., & de Wildt, S. N. (2021). Benefit-Risk Assessment of off-label drug use in children: The BRAvO framework. Clinical Pharmacology and Therapeutics110(4), 952–965.

Yackey, K., Stukus, K., Cohen, D., Kline, D., Zhao, S., & Stanley, R. (2019). Off-label medication prescribing patterns in pediatrics: An update. Hospital Pediatrics9(3), 186–193.

Assignment Pediatrics Week 11 Instructions

In the United States, the FDA approves a drug for a certain set of uses after the drug has been put through rigorous studies. Once approved, the FDA and the drug manufacturer create the drug’s “label” which is an information document that details the drug’s specific approved uses. However, researchers may later discover that the drug can be used to treat other conditions. In this case, doctors may legally prescribe a drug to treat a condition not listed on the drug’s label. Thus, “off-label drug use” means to use a drug for a purpose not listed on the drug’s label. Typically, an ethical doctor will only prescribe a drug for off-label purposes when the benefits of use outweigh the risks. Credible medical evidence such as clinical trials and medical literature should also support the doctor’s decision.

Examples of off-label drugs

Drug      (Brand  name)                                                                                                                                                                                                                   Common              on-label                use                                                                                                                                                                                                                         Common              off-label                use bupropion (Wellbutrin)            depression                          bipolar

diphenhydramine (Benadryl)       allergy sx, insomnia                 N/V related to pregnancy

escitalopram (Lexapro)            depression, anxiety                 bipolar

fluoxetine (Prozac)                depression, OCD, bipolar            autism spectrum disorders montelukast (Singulair)            asthma                             COPD quetiapine (Seroquel)              schizophrenia, bipolar               insomnia



Write     a              1+           page      paper    in            APA       format  that        addresses            the         following:

  • Explain the circumstances   under    which    children               should  be           prescribed          drugs    for                off-label                use.        Be           specific and        provide examples.


  • Describe strategies to            make     the         off-label                use         and        dosage  of            drugs    safer                for          children               from      infancy to            adolescence.       Include descriptions       and                names   of            off-label                drugs    that        require extra     care       and        attention             when                used      in            pediatrics.