Understanding Off-Label and Unlicensed Medications in Children: How Home Medication Reviews Can Help Ensure Safe Use

As parents and caregivers, we often trust healthcare professionals to provide the best treatment for our children. However, when it comes to medication, there’s a challenge that many families may not be aware of: off-label and unlicensed medications. These are drugs that are used outside of the official guidelines set by regulatory authorities, such as Australia’s Therapeutic Goods Administration (TGA). While this practice is not inherently unsafe, it requires careful consideration and monitoring to ensure your child’s safety.

If you’re worried about the medications your child is using, a Home Medication Review (HMR) could be a valuable resource in managing the risks associated with these practices. Here's what you need to know about off-label and unlicensed medicines in children and how an HMR can help ensure that your child’s treatment plan is both safe and effective.

What Are Off-Label and Unlicensed Medications?

Off-label medications are drugs prescribed for uses that are outside their official approval. For example, a medication approved for treating adults may be prescribed for a child, even if it hasn’t been formally tested in younger populations. It could also involve adjusting the dose or changing how the medication is given (e.g., using a pill form when a liquid form is needed).

Unlicensed medications are those that have not been approved by regulatory authorities for any specific use. This can happen when a doctor or pharmacist alters a medication (e.g., crushing a tablet to make it easier for a child to swallow), or when a medication is used in a way that hasn’t been officially evaluated or endorsed by authorities.

While off-label and unlicensed medicines are not automatically unsafe, they come with added risks, especially for children, whose bodies can react very differently to medications than adults. That’s why careful management and monitoring are crucial.

Why Are Off-Label and Unlicensed Medications Common in Children?

Children are not just "small adults" — their bodies develop in complex and distinct ways that affect how they metabolize medications. For example, newborns, toddlers, and adolescents all experience different stages of growth that can influence how they process drugs. As a result, many medications are not tested in children before they are marketed, leaving a gap in information about how safe and effective they are in younger populations.

The absence of age-specific data on many medicines means that healthcare providers may need to prescribe medications off-label to treat conditions in children. These could include common issues such as cough and cold symptoms, gastroesophageal reflux disease (GERD), or even mental health conditions like ADHD and anxiety.

In fact, studies show that off-label prescribing is quite common in children, with some estimates suggesting that between 36-93% of hospital prescriptions for children are off-label. This trend is even higher in vulnerable populations, like those in neonatal intensive care units.

The Risks of Off-Label and Unlicensed Medications for Children

While off-label and unlicensed drugs may be necessary for treating your child’s condition, they carry several potential risks:

1. Increased Risk of Side Effects: Children may experience unexpected side effects because their bodies may react differently to medications, and there is often limited data on how a drug works in younger populations.

2. Dosing Challenges: Pediatric doses must be carefully adjusted based on a child’s weight or age. Off-label prescribing often lacks clear dosing guidelines, increasing the risk of underdosing or overdosing, which can be dangerous, especially for infants.

3. Lack of Efficacy Data: Many off-label medications have not been rigorously tested in children, so their effectiveness for specific conditions in younger patients may be unclear.

4. Ethical and Legal Concerns: When medications are prescribed off-label, healthcare providers must ensure they properly communicate the risks and benefits to parents and caregivers. Consent must be informed, and in some cases, there may be legal implications if the risks are not adequately explained.

5. Formulation Issues: Medications often come in forms (such as tablets or capsules) that aren’t suitable for children, which can complicate dosing. For example, tablets may need to be crushed, or liquid formulations may need to be compounded — and these practices can introduce additional risks.

How Home Medication Reviews (HMRs) Can Help

A Home Medication Review (HMR) is a service where a qualified pharmacist reviews all the medications you and your family are using. This review can be especially helpful for parents of children using off-label or unlicensed medications, as it helps ensure that the medications are being used correctly and safely.

Here’s how an HMR can support you:

1. Ensuring Proper Dosing: The pharmacist can check that your child’s medications are being administered at the correct doses for their age, weight, and condition. This is critical when it comes to off-label medicines, where dosing guidelines may be unclear.

2. Identifying Medication Interactions: An HMR can help identify any potential interactions between the various medications your child is taking, including prescription, over-the-counter, and complementary medicines. This is especially important in paediatric patients, who may be more vulnerable to the side effects of drug interactions.

3. Assessing Safety: The pharmacist will review whether the medications are safe for your child based on their medical history, current condition, and any other treatments they may be undergoing. This is key for managing off-label and unlicensed drugs, which might have unforeseen side effects.

4. Clarifying the Use of Off-Label Medications: If your child is prescribed an off-label or unlicensed medication, the pharmacist can help explain the reasons behind the choice, answer any questions you may have, and ensure that you understand the risks and benefits of the medication.

5. Monitoring Effectiveness and Side Effects: After the review, the pharmacist can help you set up a system for monitoring how well the medication is working and whether there are any signs of adverse effects. Regular follow-ups can help you identify any issues early.

Key Takeaways

• Off-label and unlicensed medications are often necessary for treating children, but they come with added risks due to the lack of specific testing in younger populations.

• Dosing, safety, and effectiveness can vary significantly for children, especially when medications are used off-label.

• Home Medication Reviews (HMRs) provide an opportunity for parents to ensure that medications are being used correctly and safely, with the support of a qualified pharmacist who can answer questions, monitor side effects, and provide expert guidance on the proper use of medications.

By taking an active role in your child's medication management, you can help ensure that they receive the best possible care — with all the safety checks in place to support their health and well-being.

If you’re unsure about any medications your child is taking, or if you’d like to discuss the possibility of a Home Medication Review, speak to your pharmacist or healthcare provider today. They are there to help you navigate the complexities of pediatric medication safely and effectively.

Have questions about off-label medications or how to get a Home Medication Review? Let us know in the comments below!