Off-label prescribing of drugs is the use of a medication for an indication other than what it has been approved for by the Food and Drug Administration (FDA). Off-label prescribing is often used to treat children, especially when there are no FDA approved medications or therapies available to treat their condition. While off-label prescribing is not uncommon in clinical practice, it presents unique ethical and legal concerns that must be taken into account when it comes to treating children.

First and foremost, any decision to prescribe drugs for off-label use should always be based on sound medical evidence. The provider should consider potential risks as well as benefits before making such a recommendation. Researching published peer-reviewed studies, consulting experts in the field, and considering clinical experience can help guide this process. Some conditions may benefit from an off-label drug without significant risk; however, there are also times when off-label prescribing can lead to serious adverse effects or even death if not done with caution. Thus, providers must assess individual cases carefully before deciding whether an off-label prescription is appropriate or beneficial for a child’s health care needs.