Unlicensed and “off-label” medicines

Medicines are products used to prevent or treat a medical condition. They can come as tablets, capsules, liquids, injections, inhalers, creams, eye drops/ointments, suppositories, and patches.

A drug company must have a product licence to advertise and sell a medicine. The licence will state which illness or condition the medicine can be used for; the age of patients it can be given to; how much to give and how to give it. The licence is provided by a government organisation called the MHRA (Medicines and Healthcare products Regulatory Agency).

To get a licence, the drug company must prove that the drug works for the illness or condition to be treated and is safe. They do this by trying it first in clinical trials, usually in adults aged 18-65 years. Information from the clinical trials is then given to the MHRA when the drug company applies for a licence. The drug company cannot advertise or make any recommendations about using a medicine outside the terms of its licence. The licensing process and clinical trials are very expensive. This means that once a drug is on the market, the company may decide not to try getting the original licence extended if it is found to treat other symptoms.

“Off-label” use means that the medicine is being used in a way that is different to that described in the licence.

Some examples of “off-label” uses are:

• Using a medicine for a different illness to that stated in the licence. Doctors may have found thatthe medicine works very well for this illness or condition. This use may be supported by expert groups, but the drug manufacturer has not extended the licence.
• Using a medicine in an age group outside the licensed range (usually children or the elderly).
• Using a medicine at a higher dose than stated in the licence.

“Unlicensed use” means:

• A medicine has a licence in other countries, but not the UK. It has to be imported because it is not available in the UK.
• A medicine has a licence but needs to be made up to be taken as an unlicensed formulation. This may be as a specially prepared liquid for someone who has difficulty swallowing the licensed tablets.
• A medicine that has no licence at all. These are often medicines used for treating rare illnesses.
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The manufacturer may have decided that it was too expensive to carry out the clinical trials or it would be difficult to find enough patients for the clinical trials needed to get a licence.