Background: All medicines prescribed in the UK should have a ‘licence’ granted by MHRA, i.e. medicine is effective, clinical safety has been assessed and manufacturing process produces a quality product. When licensed medicines are used outside of this marketing authorisation this would be ‘off-label’.
Objectives: To determine the context for recommending off-label use of medicines in NICE guidelines.
Methods: Clinical guidelines (2012 onwards) were examined for review questions including off-label use of medicines.
Results: 43 guidelines were identified and we classified recommendations as:
-off-label indications for adults or adults and children/young people, in 24 guidelines
-off label indications in children/young people only, in 13
-class of medicines where only some members are licensed for specific indication in 5
-off-label doses in 2
-combinations of medicines not included in licence in 3
-unlicensed medicine in 1
-also, nutraceuticals or herbal medicines in 3
Particular issues included where an off-label medicine was recommended over a licensed medicine based on evidence of higher effectiveness, or where no published evidence for licensed product was available whereas evidence was available for a medicine used off-label. Also differences in licensing among members of a class of medicines and also between branded and generic medicines; in some questions evidence for all medicines was reviewed but recommendations applied to only licensed medicines. Some questions included off-label medicines where no licensed medicine was available.
Conclusions: The NICE guideline manual provides advice on reviewing off-label medicines. And any recommendations must inform the prescriber that use is off-label as the prescriber must take full responsibility for prescribing medicines outside the terms of their licence; good practice wording is agreed in collaboration with MHRA. It is important to identify off-label use of medicines early in the guideline-development process and consider the risks in terms of effectiveness, safety and availability and quality of product and the resultant implications for prescribers and practice.