Children: Legal considerations

Last updated: Thursday, July 09, 2015

An oral syringe      © Crown copyright 2017


Many children receive unlicensed or off-label medicines. The Human Medicines Regulations 2012 enables such use and it follows that you will sometimes be asked to give advice in these situations when efficacy and safety data may be limited.


If you are asked about the choice of drug in a paediatric patient, always consider if there is an appropriate, licensed drug first. If you can't use a licensed medicine, you should take extra steps to ensure that the use of the medicine is justified in terms of evidence, necessity, and safety. A lack of information may sometimes make it harder to check other parameters like dose and frequency, suitability of formulation, precautions, interactions, and monitoring parameters.



Practical Tips

When advising on the use of unlicensed/off-label medicines in children it is important to protect the patient, yourself, and other healthcare professionals. These steps will help you do this:

  • Try to gather as much evidence as is available, preferably from higher quality sources (although this is often challenging). Be thorough.

  • Always discuss with your peers (especially when you feel rushed to give a quick answer, the intervention is critical to the patient’s immediate health, or when on-call in the middle of the night).

  • Satisfy yourself that your advice follows that of a respectable, responsible body of professional opinion or if not, that your advice can be justified from the data you have collected and the information provided by the enquirer. It is good practice to document your reasoning carefully explaining why you gave this advice (and why other options were not suitable).

  • Make it explicit to the enquirer that the use of the medicine is unlicensed (more accurately we should say that it is outside the terms of a ‘marketing authorisation’), and highlight any limitations in the advice/ information you provide.