The enactment of the Best Pharmaceuticals for Children Act has led to improvements in prescriptions for children. However, the use of off-label drugs is still a vital public health concern for infants, children, as well as adolescents. The reason for this concern is because a significant number of drugs still do not have labeling information for pediatric use. Off-labeling use aims to benefit an individual patient, and so health practitioners often use their professional judgment to determine when to use off-label drugs (Arcangelo et al., 2017). Off-label drugs refer to the use of drugs that do not have FDA approved labeling for the drug. However, the term off-labeling does not mean that the drug is illegal, improper, or contraindicated. In the US, the FDA needs substantive evidence for safety and efficacy, commonly through well-controlled clinical trials before a drug is approved. It does not, however, mean that off-label medications are unsafe for use (Arcangelo et al., 2017). A doctor should make decisions to use off-label drugs based on the best expert judgment, scientific evidence, and or peer-reviewed literature.
There are several circumstances where medical practitioners use off-label drugs in children. One such situation is in the case of asthma. Many doctors prescribe the use of albuterol to treat asthma in children all ages. However, the FDA only approves this drug for children who are above two years (Magalhães et al., 2015). The FDA only approves other asthma inhalers such as Advair and Dulera for use in children over the age of twelve. However, many medics prescribe these drugs for children even younger than twelve years. The reason for this is because, in asthma cases, there are no alternative medications that can be used and these drugs have been used in many instances and proved to be safe through research (Magalhães et al., 2015). Therefore, doctors opt to use these drugs to treat asthma instead of not preventing or treating a child’s asthma symptoms.
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Another circumstance where doctors prescribe off-label drugs to children is in cancer treatment. The reason for this is because some of the cancer drugs have been found to work to counter the effects of many different types of tumors. Similarly, chemo treatment usually uses a combination of drugs, and these combinations may include some drugs that may not have the FDA approval for the type of cancer being treated (Gore et al., 2017). Furthermore, treatment for cancer is rapidly changing and improving and therefore doctors often use a combination of drugs; some of which may not be FDA approved to try and find the best treatment options for cancer in children. Also, patients and oncologists are often more willing to try out off-label drugs as opposed to other medical specialties.
Another significant reason why doctors prescribe off-label drugs is due to the unavailability or lack of standards, effective, safe, and licensed therapeutic medications for specific diseases or conditions in children. Because of the lack of these drugs, doctors often use their best judgment and evidence from previous uses in adults to prescribe the drugs to treat conditions in children. Other reasons include lack of alternative therapy forms for specific age groups, absence of clinical trials in certain age groups such as infants, neonates, and adolescents (Gore et al., 2017).
Several recommendations have been made to make the use of off-label drugs safer for children. For example, decisions about using off-label drugs should at all times be guided by the best evidence and benefits for an individual patient. Additionally, standard clinical trials for certain drugs are many times unavailable, and therefore medical professionals must use conclusive information from experts who have dealt with similar age groups or use definitive evidence from a different target population to guide the decision to use off-label drugs (Bennett, 2018). Practitioners should also apply the use of evidence-based medicine to help assess the effectiveness of off-label drugs before prescription to patients. This evidence can be found in peer-reviewed studies, the American Academy of Pediatrics practice guidelines and policy statements, handbooks, consensus statements, and other databases such as Cochrane, Harriet Lane, and Lexicomp. Sometimes, there may be little or no available published information to help guide decision making, especially in cases of treating rare diseases or populations which are sparse such as neonates. In this case, a doctor can play a vital role in publishing their own experience using off-label drugs (Arcangelo et al., 2017). Such publications can be a basis for more formal safety and efficacy research and can serve as essential resources for other physicians. Lastly, a physician should take the responsibility to report any adverse effects to the FDA in case of the use of off-label drugs.
The table below shows a list of some of the off-label drugs that require special attention and care when used in pediatrics.
Table 1.
Example of Off-Lable drugs
| Drug | Prescription |
| High doses of amoxicillin | Used to treat otitis media in children (Ward et al., 2018) |
| Sildenafil | Treatment of Pulmonary hypertension in children |
| Atenolol | Treatment of hypertension in children |
| Morphine | Treatment of pain in children |
| Tobramycin | Used once daily in neonates (Ward et al., 2018) |
| Intranasal Desmopressin | Treatment of Nocturnal enuresis in children |
| Rifampicin | Used for enzyme induction in infants with biliary atresia |
| Beclomethasone | Used in infants under 12 months (Gore et al., 2017). |
References
Arcangelo et al., (Eds.). (2017). Pharmacotherapeutics for advanced practice: A practical approach . Lippincott Williams & Wilkins. Retrieved from https://www.academia.edu/17991062/Pharmacotherapeutics_for_Advanced_Practice
Bennett, M. (2018). Best practice in prescribing off-label medication for children. Nurse Prescribing , 16 (5), 234-237. DOI:10.12968/npre.2018.16.5.234.
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. DOI: 10.2174/1574884712666170317161935.
Magalhães, J., Rodrigues, A. T., Roque, F., Figueiras, A., Falcão, A., & Herdeiro, M. T. (2015). Use of off-label and unlicenced drugs in hospitalised paediatric patients: A systematic review. European Journal of clinical pharmacology , 71 (1), 1-13. DOI: 10.1007/s00228-014-1768-9. Epub
Ward, R. M., Benjamin, D. K., Davis, J. M., Gorman, R. L., Kauffman, R., Kearns, G. L. ... & Sherwin, C. M. (2018). The need for pediatric drug development. The Journal of pediatrics , 192 , 13-21. DOI: 10.1016/j.jpeds.2017.08.011.
