Unlicensed medicines in pediatrics is so common. In order for a manufacturer to sell its drug on the market, it will have to receive a valid license from the US Food and Drug Administration (FDA), or the equivalent review board in countries outside the US. This license will specify exactly how the medicine should be used and what patients are allowed to use it.

However, when it comes to prescribing medicines for children, it is common to find that the only option available is an unlicensed medicine. Not surprisingly, the use of the word ‘unlicensed’ can scare parents or caregivers. However, there are a number of reasons why using unlicensed medication to treat children is particularly common. And, although it may sound scary, it is important to remember that a physician will only suggest using an unlicensed drug if they think it is the best thing for the child.

Licensed for adults

Some of the most common medications on the market are licensed for use in adults but not in children. This is because these drugs have been tested in clinical trials with adults only. It is much harder to get children to enroll in trials, and often times, drug manufacturers cannot find enough participants to justify conducting a separate trial for children. This means the safety and efficacy data from the trials can only apply to adult patients. Such drugs would, therefore, have to be prescribed to pediatric patients on an unlicensed basis.

Licensed for a particular dosage

Even if a drug is licensed for use in children, a physician or healthcare provider does not have free-reign to use that drug however they see fit. The license will clearly specify what dosage the drug should be taken in and how often it should be taken. However, as is common in pediatrics, a child can have a rare form of a disease, or a unique reaction to the medication, in which case the dosage specifications may not be affective, or could be harmful to the child. If a healthcare provider decides that a stronger or weaker dosage, or a different schedule of usage, would be more appropriate for a child, that would be an unlicensed use of the drug.

Licensed in the wrong form

Sometimes a drug that is licensed for use in children can be provided in a form that is unsuitable for a particular child. Maybe the drug only comes in pill form, and a child suffers from a condition that causes difficulty swallowing. Or, a drug might need to be administered intravenously by a physician, and a child lives too far from the closest hospital or treating center. In situations like these, a child’s physician can decide to prescribe the child a different drug, that is either not licensed for use children, or not licensed to treat that child’s particular condition, but which comes in a more suitable form for the patient.

Licensed in other countries  

It is also a possibility that a drug will be licensed in certain countries but not in others. Maybe there are not enough children willing or able to participate in a clinical trial in a certain country, or, the drug has been taken off the market due to adverse reactions. Whatever the reason, sometimes a healthcare provider will hear about a drug that is licensed and being used successfully to treat children elsewhere in the world, and, after exhausting all other viable treatment options, will want to use that medication to treat their patient. The drug will then be provided to the healthcare provider, and given to the child, as an unlicensed medication.

As has been shown, although using an unlicensed medication is not appropriate for every child, it can be the best treatment option available in certain cases.