A lot of medication in trans healthcare is prescribed for unlicensed use. This sounds scary, but it’s actually extremely common.
Unlicensed medication, also referred to as off-label or off-licence, is medication that is prescribed for a use other than that listed in the terms of its licence, within the country of use.
The licence says what conditions the medication can be used for, what age patients it can be given to, how much can be given and how to give it. However, there are many medications that are used either outside of the terms of their licences or without a licence at all, when it is in the patient’s best interests.
One common example is the group of medicines called oral contraceptives. These are licensed for use as contraceptives, but some of them have been effective in a much wider range of applications, including the treatment of period pain and endometriosis. In fact, unlicensed medication is used by a wide variety of people. It is particularly common in paediatric (young people) and geriatric (old people) care.
The British Association of Psychopharmacology (BAP) has stated: ‘The reasons for and implications of off-label prescribing, including the potential clinical benefits/risks and medico-legal implications, are often poorly understood by both patients and prescribers.’
So what are the reasons? To put it simply, drug manufacturers have to pay to license their medications, and they tend to do so for those groups of people who are most likely to use them, as this is where they make the most money.
Unless a drug has been developed specifically for a younger or older market, there tends to be less use within these groups and so, while the drug may well be effective, it remains unlicensed for use in that group.
The same thing happens in transgender healthcare – because patient numbers are small, it is not profitable for drug manufacturers to license medications specifically for their use. This is why a great deal of medication in transgender healthcare is prescribed off-licence.
It is also why understanding and prescribing off-label medication is so important, particularly in the care of gender diverse youth, as outlined in the statement on the subject issued by the BAP: ‘The off-label use of medicines for children and adolescents remains a common and important issue for prescribing practice across child and adolescent psychiatry, paediatrics and primary care.’
This means that being able to safely prescribe off-licence is a common and important part of good medical practice, within and beyond transgender healthcare. In its statement on the subject of prescribing off-license medication, the British Association of Psychopharmacology warned that: ‘An important unintended consequence of the uncertainties and confusion surrounding the status of off-label prescribing for children and adolescents may be that effective drug treatments are being withheld or underused.’
Different organisations are responsible for licensing medication in different countries. In the UK it is the role of the Medicine and Healthcare product Regulatory Agency (MHRA) and the European Medicines Agency (EMA). Because transgender healthcare is often not a priority for drug manufacturers, few medications are licensed specifically for this use. As a result, off-licence medication use is considered best medical practice for transgender healthcare and recommended by both the General Medical Council (GMC) and the General Pharmaceutical Council (GPhC).
The GMC and GPhC both offer guidance for the use of unlicensed medication in the UK. The GMC guidelines state that, ‘You should usually prescribe licensed medicines in accordance with the terms of their licence. However, you may prescribe unlicensed medicines where, on the basis of an assessment of the individual patient, you conclude, for medical reasons, that it is necessary to do so to meet the specific needs of the patient.’
Because the legal responsibility lies with the clinician who signs the prescription, it is important for GPs and other prescribers to ensure that the patient can give fully informed consent to their treatment. Professionals who are prescribing off-label should always inform their patients that this is the case and explain what it is being prescribed for, why it is being prescribed off-licence, and any risks or side-effects that could result. Patients should be advised of any alternatives that exist and have all their questions answered fully before they can consent.
If, as is the case with transgender healthcare, there are established guidelines for best practice with the use of unlicensed medication, then these should be followed. Off-licence medication use is both safe and effective when it is used in accordance with best medical practice. Unlicensed, off-licence and off-label refer only to the use of the medication beyond the terms of its licence, and do not refer to improper, illegal, unsafe or experimental use, as noted by the American Academy of Pediatrics.
At GenderGP we prescribe in line with the Endocrine Society Guidelines for the Care of Transgender Patients and the UCSF Guidelines for the Primary and Gender-Affirming Care of Transgender and Gender Nonbinary People, which offers guidance for the off-label use of medications specifically in the field of trans healthcare, these include puberty blockers and gender-affirming hormone therapy. Our prescribing protocol and a list of prescribed medications can be found here.