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Accessing unlicensed drugs for brain tumours

Various schemes in Europe and the US allow some patients to access promising unlicensed drugs outside clinical trials to treat brain tumours.

Short Summary

Some people diagnosed with a brain tumour want to know what other (non-standard) treatments they can try. This is particularly the case if their current treatment no longer seems to be working. For example, unlicensed drugs that are showing promise.

What are unlicensed drugs? These could be drugs that are still being used in clinical trials, drugs that are licensed for other conditions but not brain tumours, or possibly drugs are licensed for brain tumours but are not used in the UK.

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About unlicensed drugs

For people with a brain tumour in the UK, drugs are unlicensed in a variety of situations:

  • drugs under investigation that are not yet licensed at all (in clinical trials)
  • drugs that are licensed in the UK for other conditions, but not brain tumours
  • drugs that are licensed for brain tumours, but are either not for use in the UK or not offered routinely by the NHS.

A drug can only be used for a particular purpose or health condition in the UK, after it‘s been authorised (licensed) by The Medicines and Healthcare Products Regulatory Agency (MHRA) and the European Medicines Agency (EMA).

Before they can be licensed and approved, the medicines are tested in people with the particular disease or condition, in a clinical trial. These unlicensed medicines are usually only available to people who are taking part in a clinical trial.

Accessing unlicensed drugs through clinical trials

The main way of accessing unlicensed drugs for brain tumours is by taking part in a clinical trial

A clinical trial is a research study that involves patients in a new way of managing a condition. This often involves testing new drugs to find out if they’re better than the existing ones. It can also include testing existing drugs in other illnesses that they’re not licensed for.

It can take as long as 10-15 years for a promising drug to go through this process and become licensed.

If you’ve been treated as part of a clinical trial that has finished, you may be able to continue treatment as part of an Expanded Access Programme.

Unfortunately, not everyone can get onto a clinical trial and access promising drugs this way. 

Each trial has a limited number of spaces and there are strict criteria you need to meet before you can take part. There may not be any clinical trials for your brain tumour type that are recruiting. Furthermore, suitable trials may be too far from your home.

Unmet medical needs

There are various schemes in Europe and the US, which allow some patients to access unlicensed drugs outside clinical trials.

To access these schemes you must have what’s known as an unmet medical need. This means that:

  • you must have tried and exhausted all the standard treatments for your tumour type.
  • there must be no other comparable or suitable therapies available to you.
  • there must be no appropriate clinical trial available to you (or you’re unable to get on one).

If you believe you have an unmet medical need and are interested in accessing promising unlicensed drugs, you should speak to your healthcare team about any available options.

According to the GMC (General Medical Council), when prescribing an unlicensed medicine, doctors must:

  • be satisfied that there’s sufficient evidence or experience of using the medicine to demonstrate its safety and effectiveness
  • take responsibility for prescribing the medicine and overseeing the patient’s care, monitoring, and any follow-up treatment, Alternatively, they must make sure that arrangements are made for another suitable doctor to do this
  • make a clear, accurate and legible record of all medicines prescribed and, where they’re not following common practice, their reasons for prescribing an unlicensed medicine
  • make sure you understand all the possible risks of taking the unlicensed drug being prescribed.

Prescribing unlicensed medicines is only possible when there’s no suitably licensed medicine that will meet the patient’s need. The codes and ethics of an NHS trust, or the prescribing policies of their employers, may also prevent many doctors from being able to prescribe unlicensed medicines.

The MHRA sets priorities when choosing which medicinal products to prescribe. They are in order of priority and assume that the earlier choices aren’t available:

  • Use a licensed product within the terms of its licence (i.e. the label)
  • Use a licensed product off-label
  • Use an imported product that has a licence elsewhere
  • Use a product that’s not licensed anywhere, but which has been made in the UK as a special.
The following schemes are all ways of accessing promising, unlicensed drugs, but it’s important to note that:
  • they may not be available or suitable for you or your tumour type
  • they’re often via difficult and complex routes
  • your doctor may not be able to refer you via these alternative ways because the codes, ethics or prescribing policies of their NHS trust, or their private employer, may prevent this
  • the medicine won’t yet have been cleared as safe and effective (although initial tests will have been done)
  • the medicine may or may not be effective
  • the medicine may cause unexpected and potentially serious side-effects.

Accessing unlicensed drugs through compassionate use

What is compassionate use?

Compassionate use is a way of making a promising medicine, which has not yet been licensed for a particular condition, available to patients with an unmet medical need. Often, drug companies set up a compassionate use programme to allow people to access drugs they’re developing.

This special programme can be set up to for people who meet certain conditions that are very strictly defined. There are only a limited number of compassionate use programs in the UK and most of these won’t be for brain tumours.

The medicine must also be undergoing clinical trials or the pharmaceutical company must have started the application to market the medicine. While early studies will generally have been completed, the safety and correct dosages of the drug may not be fully established.

In the US, this may also be called an Expanded Access Program. A company may choose to run an Expanded Access program to let some patients get early access to a promising medicine outside clinical trials.

Which medicines can be made available for compassionate use?

Compassionate use programmes can only be put in place for medicines that are expected to help seriously ill patients with life-threatening, long-lasting or seriously disabling illnesses.

Generally, studies will have been completed to see if the medicine is safe (non-toxic) to humans. And initial studies, looking at how the medicine is handled by the body, will have been completed.

However, there may still be some uncertainties about:

  • the best way to give the medicine to patients, for example, the exact dose to use or how often the drug should be taken
  • the medicine’s safety profile, which includes the side-effects it can cause and how serious they’re likely to be.

How can I take part in a compassionate use programme?

To take part in a compassionate use programme, you must speak to your healthcare team.

They will first check if there’s a suitable clinical trial in the UK.

If not, and the NHS Trust’s or private employer’s regulations allow it, your consultant can speak to the MHRA to find out if a suitable compassionate use programme is available. If there is, your healthcare team can tell you how the compassionate use programme will work.

Your doctor, or their healthcare trust, will often be required to sign a formal agreement with the drug company.

The MHRA will keep a register of the patients treated with the medicine under the compassionate use programme. They will also record any side-effects reported by the patients or their doctors.

Accessing unlicensed drugs through the Early Access to Medicines Scheme

The Early Access to Medicines Scheme (EAMS) is designed to give people with life-threatening or seriously disabling conditions access to medicines (drugs) before the drugs have completed the whole licensing process. 

As with all other means of accessing unlicensed drugs, the person must have a clear unmet medical need. EAMS will only be available in a small number of cases.

Which medicines can be made available for EAMS?

Medicines must have already been classed as a Promising Innovative Medicine (PIM) by the MHRA, based on early clinical research. The MHRA must also have given an “early access scientific opinion” on the medicine. This describes the balance between the risks and benefits of the medicine.

There may still be uncertainties about:

  • how safe the drug is
  • how well it works
  • what side-effects it may cause.

This opinion, which lasts for 1 year, can be used by your doctor and you to help decide whether you should use the medicine before it’s licensed. Normally it would take several years from this point for a medicine to be licensed and available for use.

EAMS can also be used to give doctors information about whether to prescribe drugs that are ‘off-label’. These are drugs that are licensed, but for a different illness or condition, or only for use in other countries.

Named-patient basis

Named-patient basis is when doctors are able to get a promising medicine for a specific, named patient by requesting a supply of a medicine directly from the manufacturer.

It’s similar to a compassionate use programme, but only provides access for the specific, named patient, not a group of patients.

The doctor will have to follow the rules of their healthcare trust and the named patient must be under the requesting doctor’s direct responsibility. Patients must also have given their consent.

Manufacturers of the drug do record what they supply. However, there’s no central register of the group of patients that are being treated in this way. Also, any side-effects they may have are not recorded.

Why are drugs accessed on a named-patient basis?

Accessing drugs on a named-patient basis can be done for several reasons:

  • a licence has not been granted in the UK yet, due to ongoing clinical trials
  • a drug has not been licensed or has been discontinued in the UK, but is licensed and available in another country
  • there are drug shortages or temporary supply problems in the UK, but it’s still readily available in another country
  • the specific, named patient has particular needs (these medicines are known as specials).

In named-patient programmes, the doctor takes responsibility for any harm to the patient due to side-effects or adverse reactions.

Your doctor will need to have a formal agreement with the manufacturers. If the drug is coming from abroad, there’s a lot of checking to be done, such as, is the manufacturer reputable. There’s also a lot of paperwork to import the drug. This can be complex and time-consuming.

The prescription and dispensing of such unlicensed medicines normally takes place in the hospital. If a patient is discharged from hospital, your GP will become responsible for giving you repeat prescriptions, You will get the medicine from a regular retail (community) pharmacist.

Continuing access to unlicensed drugs after discharge from hospital

GP’s aren’t obliged to continue prescribing unlicensed medication if they don’t feel they have enough experience with its use. 

If this happens, usually arrangements can be made for the hospital to continue prescribing the drug and supplying it from the hospital pharmacy.

The community pharmacist is responsible for making sure of the suitability and quality of the unlicensed medicine. 

As such they’re not obliged to use the same source (manufacturer) for the medicine. If they change sources, this could take time, so arrangements would need to be made for the hospital pharmacy to continue to supply the drug.


Specials are unlicensed drugs that are specially made or imported for the treatment of a specific, named patient. They can be made from scratch or can be based on a licensed drug, which needs to be changed before it can be used for the named patient. 

A common example is if a patient is allergic to one of the ingredients of the licensed product. Any change to the drug to prevent the allergic reaction means that the drug no longer fits the licencing requirements. Therefore it is classed as unlicensed.

It’s important that the drug is consistently made to the same formula, so that each batch is as similar as possible. For this reason, if the drug is made in the UK, the manufacturer must hold a specials licence, issued by the MHRA. If it’s imported from outside the UK, the manufacturer must hold other special licences.

A special can’t be supplied if an equivalent licensed product is available that could meet the patient’s needs. Essential records must be kept and serious adverse drug reactions must also be reported to the MHRA.

Off-label use

When a drug is licensed, the ways in which it can be used are strictly set out. For example:

  • the illnesses it can be used for (called indications)
  • how much can be used (called the recommended dose)
  • when it shouldn’t be used (called contraindications)
  • any special warnings or precautions.

These restrictions are based on all the trials that have been done to test how well it works (known as its efficacy), its safety and its quality. They’re used to make sure that when the drug is used, the benefits are judged to outweigh the potential risks.

What is off-label use?

However, sometimes a doctor may feel that a drug not licensed for use in brain tumours, works in a way that could be useful in treating brain tumours in a particular patient despite the possible risks. 

This is known as off-label use because the drug is being used in a way that differs from how the drug ‘label’ shows it should be used. This could be:

  • a drug licensed for use in another condition
  • a licensed drug used outside the recommended dosage
  • a drug licensed for a specific type of patient (for example, medicine normally meant for adults that may meet the needs of a child)
  • a licensed drug used in circumstances the “label” specifically warns against (for example, if the patient has another condition).

If a doctor feels that prescribing a medicine off-label would benefit the patient, the doctor has to take on responsibility for any harm to the patient due to side-effects or harmful reactions. The ‘advantage’ of off-label use is that, because the drug is licensed, its safety has already been fully tested for use in people.

However, many doctors may not be able to prescribe off-label, as the codes and ethics of their NHS trust, or the prescribing policies of their employers, may prevent this.

If you have further questions, need to clarify any of the information on this page, or want to find out more about research and clinical trials, please contact our team:
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