Off-label prescribing
Off-label prescribing means a doctor prescribes a licensed medicine outside the terms of its UK licence, for example for a different condition, dose or route, when they judge on the available evidence that it is in the patient's best interest.
Full definition
Every licensed medicine in the UK has a marketing authorisation that sets out its terms of use in its Summary of Product Characteristics: what it treats, at what dose and by what route. The MHRA explains that a licensed medicine has been assessed for efficacy, safety and quality, and that it is within those terms that its benefits have been judged to outweigh its risks. Prescribing outside those terms (outside the stated indications, doses or routes, or contrary to listed warnings) is called "off-label". The MHRA recognises that a prescriber may judge off-label use to be in a patient's best interest on the basis of the available evidence, and that it is common in some areas of medicine. It also says the prescriber's responsibility may be greater than when prescribing within the licence. The BNF puts it similarly: prescribing outside the marketing authorisation alters, and probably increases, the prescriber's professional responsibility and potential liability, so the prescriber should be able to justify it, feel competent to do it, and tell the patient. Doctors must also follow the GMC's guidance on prescribing. Off-label is different from "unlicensed", which describes a medicine with no UK licence at all, such as a compounded unlicensed special. In hair loss, dutasteride is licensed for an enlarged prostate and is prescribed off-label for hair loss (is dutasteride licensed for hair loss?). Oral minoxidil tablets are licensed for severe high blood pressure (Loniten SmPC), and low-dose oral minoxidil for hair loss is off-label. One practical consequence, which the MHRA flags, is that the leaflet in the pack may describe a different condition or dose from the one you were prescribed it for.
What it does not mean
Off-label does not mean illegal, unregulated or experimental, and it does not mean the medicine is unsafe. It also does not mean the MHRA has approved the medicine for that use: it has not, which is exactly why the use must be called off-label. A prescriber using a medicine off-label should tell you so, explain why, and be able to justify the choice.
Examples in use
Honest use
"Dutasteride is licensed in the UK for an enlarged prostate. Prescribing it for hair loss is off-label, and your doctor will explain what that means before you decide."
Common misuse
"Our MHRA-approved dutasteride hair loss treatment." (Dutasteride holds no UK licence for hair loss, so this implies an approval that does not exist.)
UK regulatory source
MHRA Drug Safety Update, Off-label or unlicensed use of medicines: prescribers' responsibilities (article date April 2009); BNF, Guidance on prescribing (prescribing unlicensed medicines); Loniten 2.5 mg SmPC section 4.1; GMC, Good practice in proposing, prescribing, providing and managing medicines and devices