Off-label Drug Use in Pediatrics

Off-label drug use refers to the use of approved pharmacological agents for unapproved age groups, through unapproved routes of administration or at unapproved dosages. Off-label drug use can be dangerous because physicians usually prescribe them by making adjustments to match the weight of the patient. Such modifications seem reasonable, but it is worth noting that other factors affect the pharmacodynamics and pharmacokinetics of the drugs. In children, drug metabolism rates, distribution, absorption, and excretion are all different from those of adults. Medical professionals can administer drugs for off-label use when they deem it necessary, but it is vital that healthcare stakeholders adopt measures to ensure the safety of these drugs.

Circumstances Under Which Doctors Should Prescribe Off-label Drugs for Children

Even though there is inadequate research on various drugs for use in pediatric patients, doctors still need to prescribe medications for off-label use in the management of such patients. In the United States, the law allows medical professionals to use their professional knowledge to prescribe a drug safely and effectively based on their professional judgment, even though the indication is outside the indications listed by the Food and Drug Administration (Kesselheim, 2019). In pediatrics, doctors prescribe off-label drugs when there are no approved drugs to treat the condition of the patient. Children rarely participate in clinical trials, so data on the efficacy of such treatments for this age group are unavailable. For instance, there are limited options for the treatment of pediatric rhabdomyosarcoma (Slotkin, 2016). For this reason, some physicians use bevacizumab, unapproved for such use in children. In asthma management, doctors often prescribe albuterol to infants, even though the FDA only approves use in children over two years old (Yackey et al., 2019). Physicians can also prescribe drugs for off-label use when approved drugs are ineffective. When children have multidrug-resistant tuberculosis, physicians may resort to off-label use of bedaquiline with varying success (Achar et al., 2017). Physicians often resort to off-label drugs to manage pediatric patients for whom approved drugs do not exist, or approved treatments are ineffective.

Strategies to Make Off-label Use Safer for Children

When selecting a drug for off label use in pediatric patients, doctors should ensure that they base their decisions on their professional knowledge and additionally consult published studies. The doctor should start with the lowest reasonable dose based on the child’s age and the severity of their condition (Gore et al., 2017). They should then continuously monitor the child for adverse reactions and response to therapy. Such care is necessary for the treatment of pediatric patients with furosemide for cardiovascular disorders (Yackey et al., 2019). Before administering the drug, the physician should consider other approved treatment modalities and weigh the risks against the benefits. Since dosage guidelines are not available for this drug, the doctor should consult published studies as a starting point to determine the safe and effective dose for the child’s age and weight (Yackey et al., 2019). After administering the drug, the doctor should monitor the patient’s progress and make adjustments to accommodate response and adverse reactions. A more systematic approach is required to standardize treatment for neonates, infants, toddlers, children, and adolescents. Governments and pharmaceutical companies should engage in more research to establish clear guidelines (Kesselheim et al., 2019). Doctors should prescribe drugs diligently, and governments should ensure that more research considers drug use in children.

In conclusion, off-label drug use in pediatric patients is an essential facet of clinical practice, but doctors should be careful to ensure that such use is safe for the patient. It is the responsibility of governments and pharmaceutical companies to ensure that they make data on drug use in children available to physicians by carrying out more research. Such measures ensure that patients do not suffer from adverse effects unnecessarily.

• References

Achar, J., Hewison, C., Cavalheiro, A. P., Skrahina, A., Cajazeiro, J., Nargiza, P., … & Seddon, J. A. (2017). Off-Label Use of Bedaquiline In Children and Adolescents with Multidrug-Resistant Tuberculosis. Emerging Infectious Diseases, 23(10), 1711.

Gore, R., K Chugh, P., D Tripathi, C., Lhamo, Y., & Gautam, S. (2017). Pediatric Off-Label and Unlicensed Drug Use and Its Implications. Current Clinical Pharmacology, 12(1), 18-25.

Kesselheim, A. S., Woloshin, S., Lu, Z., Tessema, F. A., Ross, K. M., & Schwartz, L. M. (2019). Physicians’ Perspectives on FDA Approval Standards and Off-Label Drug Marketing. JAMA Internal Medicine, 179(5), 707-709.

Slotkin, E. K., Magnan, H. D., Meyers, P. A., Chou, A. J., Ambati, S. R., & Wexler, L. H. (2016). Off-Label Use of Bevacizumab in Relapsed and Refractory Pediatric Sarcoma Patients: The Memorial Sloan Kettering Cancer Center Experience.

Yackey, K., Stukus, K., Cohen, D., Kline, D., Zhao, S., & Stanley, R. (2019). Off-Label Medication Prescribing Patterns in Pediatrics: An Update. Hospital Pediatrics, 9(3), 186-193.