Unlicensed medicines

What is a UK medicine licence?

Before a company can market a medicine in the UK it must obtain a Product Licence (also called Marketing Authorisation) from the Medicines and Healthcare products Regulatory Agency (MHRA).

The licence specifies:

• The illness(es) it is approved to treat.
• The dose and form (tablet, liquid, injection, eye drop etc).
• The age group and route of administration.
• Evidence that benefits outweigh risks and that it is made to high quality standards.

What do “off label” and unlicensed mean?

"Off-label”

“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 that the medicine works very well for this illness or condition. This use may be supported by expert groups, but the drug company has not applied for a licence to treat this illness or condition.
• 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

There are several reasons why a specific medicine (or form of medicine) may not have a licence:

• The medicine has a licence in other countries, but not the UK. It must be imported because it is not available in the UK.
• The medicine needs to be made up as a special product because it is not readily available. This is often the case for liquid formulations of medicines which only have a licence for the tablet form.
• The medicine that has no licence at all: These are often medicines used for treating rare illnesses. 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.

Why might my Moorfields clinician choose one of these options?

• Most effective (or cost effective) treatment: High quality studies or long clinical experience show the medicine works well for your eye condition even though it is not licensed for it.
• No suitable licensed alternative: There may be no licensed eye preparation, or the licensed product is temporarily unavailable.
• Special formulation needed: Young children, people with swallowing difficulties or allergies to preservatives may need a different strength or preservative free version.
• Practicality of licensing: Clinical trials and licence applications are expensive and time consuming. For rare eye diseases or very small patient groups, a manufacturer may not be able to justify the cost of seeking a full UK licence.
• Awaiting UK authorisation: The medicine is licensed abroad and undergoing the UK approval process.

How do we make sure it is safe?

Evidence review: Our ophthalmologists and specialist pharmacists review published research, national guidance and local audit data.

Quality assured sourcing: Imported or specially prepared (“special”) medicines come from pharmacies that follow strict MHRA standards.

Benefit–risk balance: The expected benefit to your vision is weighed against any known risks or side effects.

Ongoing monitoring: Your treatment is regularly reviewed and may switch to a licensed alternative if one becomes available.

Special considerations for children

Many medicines are originally licensed only for adults. Moorfields’ paediatric team follows national paediatric ophthalmology guidelines and internal governance checks before prescribing off label or unlicensed medicines to babies, children or young people.

Why does the manufacturer’s leaflet look incorrect or not relevant to me?

The patient information leaflet in the pack is written for the licensed use, so it may not mention your eye condition, dose or age group. That does not mean the medicine is unsafe – it simply reflects the limits of the licence. Always follow the instructions given by your Moorfields clinician or pharmacist.

If the medicine comes from another country and the packaging is not in English, we might have the same information in English. Please contact us if you do not have the information in English.

Side effects, worries or choosing not to take the medicine

• Talk to us first: Phone the number on your clinic letter or speak to your pharmacist before stopping or altering the dose.
• Report side effects: Tell us straight away. We may submit a Yellow Card report to the MHRA to help improve national safety data.
• Other options: If you decide against an off label or unlicensed medicine, we will discuss licensed alternatives, though they may be less effective or carry different risks.

Further information and support

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We can provide large print, Braille, audio, easy to read or translated versions on request. Please phone PALS on 020 7566 2324 or ask a member of staff.

Your right to timely treatment

Under the NHS Constitution you have the right to start consultant led treatment within 18 weeks of your GP’s referral. Moorfields is committed to meeting this standard. Contact PALS if you think we have missed it.