Shared responsibility

The pharmacy team should work closely with the prescriber, and the patient where appropriate, to identify the most appropriate product if a prescriber decides that an unlicensed medicine is required to establish optimal treatment. This will ensure an informed decision is taken in the context of the options available.

The pharmacy team should be involved in monitoring the treatment and the response to it. Safety and efficacy of the unlicensed medicine is not assured to the same degree as for a licensed medicine.  The patient’s condition and their special clinical need may change with time and the care environment. Therefore, the choice of medicine should be regularly reviewed to ensure that it remains appropriate and that continued prescribing of an unlicensed medicine remains justified.

Reason for using unlicensed medicines

Most medical treatment is given by prescribing licensed medicines, which provides a high level of assurance of quality, safety and efficacy. Unlicensed medicines should be prescribed and supplied only if the prescriber is satisfied that the patient has a special clinical need which cannot be met by any available licensed medicine.

Options to explore before deciding to use an unlicensed medicine are:

• using an alternative licensed presentation of the same drug molecule
• using an alternative licensed medicine
• using a licensed medicine off-label

In many cases, an unlicensed medicine is already established in use for patient groups with the same clinical need. Nevertheless, those involved should ensure they are satisfied that the unlicensed medicine is still appropriate.

Understanding patient needs

The first step is to understand the special clinical needs of the individual patient. This will involve identifying the active substance required and dose, and then defining the characteristics of the medicine that will meet the patient’s clinical needs.The following factors may need to be taken into consideration:

• route of administration
• strength or concentration range to minimize potential for dosing errors
• excipients
• flavour, smell or appearance
• number of tablets or volume of liquid for each dose
• release characteristics and bioavailability
• number of units required
• any specific labelling or information needs

This information will form an outline specification so that the process of sourcing a medicine can begin.

Note that the end user might be a parent, carer or healthcare professional, as well as the patient. Therefore, all the key stakeholders should be consulted as part of the process.

The patient needs may vary with time and the ability to meet those needs may also vary depending upon the availability of a product and the care environment. For example, it may be necessary and practical for nursing staff to crush a tablet or use an injection orally for the first few doses before a more suitable alternative product can be sourced to support ongoing care at home.

Our Purchasing Specials page provides more information.

Establishing optimal treatment for the patient

Once the patient’s requirements have been established, there may be a number of different options for providing treatment.

Identifying appropriate products

In general, the options for choosing the most appropriate product can be listed as a broad hierarchy:

• using a licensed medicine off-label
• using an imported medicine licensed in another suitable country
• using a UK manufactured Special
• using a medicine that is unlicensed but made in a suitable country
• using a medicine prepared under Section 10 exemption
• manipulating a licensed dose form or using a licensed dose form via an unlicensed route

Sometimes a medicine from lower down this hierarchy might provide the optimal balance of quality, safety and efficacy to meet a patient’s needs, even when a medicine higher up the hierarchy may be available. It is therefore essential to review the risks and benefits associated with each option before making a decision.

Understanding unlicensed medicines provides additional information about types of unlicensed medicines.

Choosing a suitable product

Once the patient’s needs are understood, it is confirmed that no appropriate licensed medicine is available and all potentially suitable unlicensed medicines have been identified, an option appraisal will identify the optimal treatment for the patient at that time. The prescriber and the pharmacist should make a joint decision to ensure the medicine:

• meets the needs of the patient
• is of the appropriate quality
• is available at the time when it is required and will continue to be available

The prescriber and the pharmacist will also need to ensure that appropriate information is available:

• for the patient as unlicensed medicines are not always supplied with information leaflets and there may be specific risks that need to be explained and discussed
• for the healthcare professional who will administer the medicine and for those who will be responsible for continuing treatment.

In rare cases it may be decided that no suitable medicine can be made available, and the most appropriate decision is to withhold treatment.

Where possible, the decision should be a shared decision with the patient and/or carer and reflect the patient’s needs, values and preferences.

Monitoring and reviewing

The optimal treatment for the patient may change over time as circumstances change, for example the patient’s needs, the care setting and the availability of alternative or newly licensed medicines.

Unlicensed medicines do not undergo any evaluation by the MHRA as part of a licensing process and ongoing pharmacovigilance. Therefore the quality, safety and efficacy of unlicensed medicines should be continually monitored and reviewed by the clinicians involved in the care of the patient and the purchasing of the medicine.

Safety and efficacy

The strategy for monitoring and review of safety and efficacy will take into account:

•  toxicity and therapeutic index of the medicine involved
•  presentation and route of administration
•  patient or patient group. For example, stroke, intensive care or palliative care patients and children may have difficulty in communicating adverse events or other issues
• care setting
• the outcome of the quality assessment of the medicines, for example taking into account how the medicine was manufactured or prepared

In addition to monitoring the patient and their treatment, outcomes and patient feedback should be recorded in the patient record to inform future prescribing. Treatments should be regularly reviewed, and in the absence of evidence of clinical value or in case of concerns about safety or toxicity, they should be discontinued.

Quality

The quality of unlicensed medicines should be kept under review.

Our Purchasing Specials and Making unlicensed medicines pages provide more information about assurance of quality of unlicensed medicines.

Report adverse drug reactions or defective medicines via the MHRA’s Yellow Card system.

Managing defective medicines contains further details.

Further information

Information on legal mechanisms for supply and administration is available on the following links:

• Unlicensed medicines and use of PGDs
• Off-label or unlicensed product use under HMR 2012 exemptions

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