Off-Label Medicines in the UK: What Does Off-Label Use Mean?

Medicines are normally prescribed for the conditions, doses, age groups, and methods of use included in their UK licence. However, doctors and other authorised prescribers may sometimes use a medicine off-label when they believe it is appropriate for an individual patient.

Off-label use does not necessarily mean that a medicine is unsafe or experimental. It means that the medicine is being used in a way that is outside the specific terms of its licence, such as for a different condition, dose, age group, or route of administration. The MHRA recognises that there are clinical situations where off-label use may be appropriate based on available evidence and the patient’s needs.

What Does Off-Label Mean?

Off-label use means using a licensed medicine outside one or more conditions stated in its UK marketing authorisation.

For example, a medicine may be:

• Licensed for adults but prescribed to a child.
• Licensed for one medical condition but used for another condition.
• Prescribed at a different dose from the licensed dose.
• Used through a different route of administration.
• Used in a patient group that is not specifically covered by its licence.

The medicine itself can still be licensed. It is the particular use of the medicine that is outside the licence.

Is Off-Label Prescribing Legal in the UK?

Off-label prescribing can be lawful in the United Kingdom when an appropriately qualified prescriber makes an appropriate clinical decision for an individual patient.

The General Medical Council (GMC) states that doctors should normally prescribe licensed medicines according to their licence. However, medicines may be used outside their licensed terms when this is medically appropriate for the patient’s individual needs.

The decision should be based on the patient’s circumstances, available evidence, and relevant clinical guidance.

Why Are Medicines Used Off-Label?

There are several reasons why a healthcare professional may consider off-label treatment.

1. No suitable licensed treatment

Sometimes there may not be a licensed medicine that adequately meets a patient’s specific needs. In these situations, an alternative use of an existing medicine may be considered.

2. Children may require different treatment

Off-label prescribing is relatively common in some areas of paediatric medicine because medicines may not have been specifically licensed for every age group or formulation. The MHRA notes that off-label use is particularly common in areas such as paediatrics.

3. Clinical evidence may support another use

Research and clinical experience can sometimes support the use of a medicine for a purpose that is not included in its licence.

A prescriber must still consider whether there is sufficient evidence or experience to support the medicine’s safety and effectiveness for the proposed use.

4. Individual patient needs

Patients do not always respond to medicines in the same way. A prescriber may consider an off-label option when it is judged to better meet an individual’s clinical needs.

Off-Label vs Unlicensed Medicines

Off-Label vs Unlicensed Medicines

These terms are often confused, but they are not the same.

Who Can Prescribe

Off-label prescribing may be carried out by appropriately authorised prescribers within their professional competence and scope of practice.

The MHRA states that certain independent prescribers, including doctors and other authorised prescribing professionals, may prescribe licensed medicines off-label. Prescribers remain responsible for considering their competence, relevant professional requirements, and the patient’s needs.

Is an Off-Label Medicine Safe?

An off-label medicine is not automatically unsafe simply because the proposed use is outside its licence.

However, the evidence supporting the specific off-label use may differ from the evidence supporting the medicine’s licensed indication. This is why healthcare professionals need to consider the available evidence, potential risks, alternatives, and the patient’s circumstances.

The GMC says prescribers should be satisfied that there is sufficient evidence or experience to demonstrate safety and efficacy when proposing an unlicensed medicine or an off-label use.

What Should Patients Know About Off-Label Treatment?

Patients should be given enough information to make an informed decision about their treatment.

When an off-label use is not routine, or when a suitable licensed alternative is available, the General Medical Council (GMC) says the prescriber should explain that the medicine is being used outside its licence and give the relevant reasons.

Patients can ask their healthcare professional:

• Why is this medicine being used off-label?
• What evidence supports this use?
• Are there licensed alternatives?
• What are the possible benefits and risks?
• What dose should I take?
• How long will I need treatment?
• What side effects should I watch for?
• Will I need monitoring or follow-up?

Does Off-Label Mean the Medicine Is Experimental?

Not necessarily.

Some off-label uses are supported by substantial clinical evidence and may be established in medical practice, even though the medicine’s formal licence has not been extended to cover that particular use.

At the same time, an off-label use should not automatically be assumed to have the same level of evidence as a licensed indication. The strength of evidence depends on the specific medicine and its proposed use.

Monitoring During Off-Label Treatment

Monitoring may be particularly important when using a medicine outside its licensed terms.

The GMC recommends appropriate arrangements for monitoring, follow-up and review of medicines, taking into account the patient’s needs and potential risks.

Depending on the medicine and condition, monitoring could include follow-up appointments, symptom assessment, blood tests, blood pressure checks, or other clinical assessments.

Can You Buy an Off-Label Medicine Online in the UK?

Off-label prescribing does not mean that a medicine can be legally purchased without a prescription.

If a medicine is prescription-only, the normal prescription requirements still apply. Patients should obtain medicines through legitimate healthcare and pharmacy channels and should not self-medicate based solely on information about an off-label use.

The GMC also states that doctors should not participate in unlawful advertising of prescription-only or unlicensed medicines to the public through websites that breach advertising regulations.

Final Thoughts

Off-label prescribing is a recognised part of medical practice in the UK. It means that a licensed medicine is being used outside the specific terms of its licence, rather than necessarily meaning that the medicine itself is unlicensed.

References

Off-label or unlicensed use of medicines – gov.uk

Deciding if it is safe to propose – gmc-uk.org