No medicine can be placed on the market without a Marketing Authorisation (formerly known as a Product Licence) granted by the Medicines and Healthcare Products Regulatory Authority (MHRA). This Marketing Authorisation (MA) signifies that the medicine concerned meets the appropriate quality standards and is safe and efficacious for its designated use. However, fully licensed products will not always meet the clinical needs of an individual patient in every situation. Therefore, the legislation provides an exemption to allow the manufacture, supply and administration of unlicensed medicines (i.e. medicines without a MA) when necessary. In addition, provision is also made for licensed medicines (i.e. medicines with full MA) to be prescribed for unlicensed indications, in unlicensed dosages or in unlicensed formulations i.e. "off-label".
The following issues must be considered before prescribing an unlicensed / off-label medicine:
- Unlicensed medicines and off-label medicines should only be prescribed if their use can be clearly justified from a clinical / pharmaceutical perspective.
- Products with the appropriate MA should be used to treat patients in preference to unlicensed medicines or off-label use whenever possible. However, use of unlicensed / off-label medicines may be necessary in order to provide the optimum treatment for patients.
- The decision to start treatment with an unlicensed medicine or an off-label medicine should be made by the clinican in charge of the patient's care, usually this is the patient's consultant.
- Any clinician prescribing an unlicensed medicine or a licensed medicine for an unlicensed indication must take responsibility for their actions. The prescriber carries the burden of the patient's welfare and in the event of adverse reactions may be called upon to justify the decisions that they have taken.
- Appropriate documentation must be completed by the responsible consultant before an unlicensed medicine or a high risk off-label medicine is prescribed as per NHSGGC Unlicensed Medicines (ULM) Policy. Pharmacists can advise further on the process and when forms need to be completed.
- Suitable patient information should be provided and informed consent obtained for patients prescribed unlicensed medicines.
- The completed ULM medicine request form, including the decision record, should be stored in the electronic patient record under 'correspondance'.
- Appropriate arrangements must be made for continuity of supply when a patient continues to take an unlicensed medicine after their discharge from hospital or specialist clinics (see Policy for more details).
- Comprehensive information about the rationale for use should be supplied to prescribers who may be asked to continue prescribing unlicensed or off-label medicines.
The full policy can be found on the GGC Medicines website, document 5.7. The ULM request form is available on the GGC Medicines website here.
Advanced Therapy Medicinal Products (ATMPs)
As ATMPs are medicines they are subject to the same requirements as for other medicinal products. Most current usage is in clinical trials (Advanced Therapy Investigational Medicinal Products (ATIMPs), but ATMPs are also available as licensed and unlicensed medicines.
Authorisation of unlicensed ATMPs in NHSGGC needs to be sought using the ULM request form, available here.
Guideline reviewed: November 2022
Page last updated: December 2022