What Is An Off-Label Medication?

Medicines that are supplied to a home pharmacy almost invariably undergo a rigorous approval system before they can legally be prescribed to patients.

This has been a legal requirement since 1968 when the use of thalidomide to help treat morning sickness led to thousands of birth defects and necessitated a far more rigorous medical approval system, once that has been modified and adapted throughout the years.

However, given that for a medicine to be approved it needs to go through three clinical trial phases, it can potentially take over 15 years for a medication to be approved, and that can be a major barrier for people with rare diseases or conditions to receive the best possible treatment.

Doctors, as well as some other healthcare professionals who have the authority to write prescriptions, have two potential options in these cases; they can attempt to use a medicine that is not licensed for use in the UK or use a medicine approved to treat another condition instead.

The latter is known as prescribing “off-label”, referring to the specific terms of use found on the label of prescription medication, and can be done if a physician is satisfied that there is enough evidence or experience of the medicine working, or sometimes as part of approved research.

A common example of off-label use would be prescribing for a child a medication only approved for adults, which can happen when a child has a condition that is more common in adults than children or is rare in its entirety.

It can also happen in cases where a different dosage is prescribed than one that is licensed, or in a different form than the one it is licensed for.

There are other cases where a medicine meant for a completely different treatment is used off-label to treat a different condition.

An example of this would be how quetiapine (often prescribed under the brand name Seroquel) is primarily used for bipolar disorder or schizophrenia but it is sometimes also used to treat insomnia.

Sometimes, a treatment can be so effective in this new role that it eventually receives approval to be used in this way. This is known as drug repositioning and the most famous examples of these include the use of thalidomide to treat leprosy and sildenafil to treat erectile dysfunction.

In the NHS, there is a spectrum of off-label use based on the level of risk, from “near-label” usage where the risks of prescription are low to higher-risk off-label prescriptions that should only be used in the absence of any alternative treatment.

By contrast, using an unlicensed medicine entirely (often known as a “special”) is considered a higher risk than even an off-label medication, only being used when a patient has unique needs that cannot be met by a licensed product, or where there is a special issue such as allergies to a commonly sued ingredient.

There are a few notable examples of this, with one of the most highly-publicised examples being the case of Alfie, a child with a rare and especially violent form of epilepsy that led to up to 150 seizures a day.

After a campaign to legally allow prescriptions of medical cannabis oil, his seizures stopped for two years, allowing him to attend school.