Many medications are prescribed for conditions they haven’t been tested for, here’s what you need to know

All prescription drugs need a license from a regulator to treat a specific condition. But licensed drugs can be prescribed for conditions for which they have not been tested in a clinical trial. This is known as off-label prescribing and is very common.

The UK has no current figures on overall off-label prescribing, but a US study found that one in five prescriptions was off-label.

It is important to note that off-label drug use is not the same as experimental use. Legally, once a drug has been licensed, it can be used for any condition in any patient. A license is proof that a medicine is safe, effective and of good quality.

So why is off-label prescribing so common? This practice is more common in children, the elderly, pregnant women and psychiatric patients, usually with changes in dosage and formulations. It is unusual for drugs to have been tested in these patient groups.

Obtaining a new license for a drug (to treat a different disease) is time-consuming, often taking up to eight years to obtain approval. It is also very expensive, as new revenue from additional uses is often not enough to offset the cost of conducting new clinical trials, marketing and relabeling the drug.

In determining whether a drug can and should be prescribed off-label, a prescriber may consider several factors. There may be common features in two similar conditions that ensure that there is some degree of cross-efficacy. For example, a licensed medication for anxiety could reasonably be used to treat post-traumatic stress disorder. And a drug approved to treat one type of cancer could reasonably be expected to target other types of cancer.

Some off-label uses have been found accidentally. Beta-blockers were licensed in the 1960s to treat heart disease. People who were prescribed these medications and suffered from migraines found that their migraine symptoms improved after they started taking the medication. Today, these medications are commonly prescribed for migraine.

Sometimes, the use of off-label medications can become the primary treatment option for a given condition. Amitriptyline, an older type of antidepressant, is often used to treat nerve pain.

This older-generation tricyclic antidepressant is often used as a first-line treatment for nerve pain.
Paul Mogford / Alamy Stock Photo

Typically, a prescriber will have considered all other medical options for a patient and reviewed the evidence to support any off-label prescribing. This includes evidence summaries provided by the National Institute for Health and Care Excellence. These are available to GPs to support safe decision making.

The summaries examine current guidelines and reviews, as well as research studies, such as clinical trials and observational studies, and expert opinions. Although the guidelines focus primarily on licensed medications, they also discuss off-label uses that are common in practice.

Some non-listed medicines may also be listed in the British National Formulary, a reference book containing prescribing information about medicines available in the UK.

Great responsibility

Whether there is a large body of evidence or not, there is a greater responsibility for the prescriber when prescribing drugs off-label.

One of the risks is the absence of information from the patient leaflet. The patient leaflet for metformin, for example, talks about its use in diabetes, but there is no information about its alternative use to treat polycystic ovary syndrome, which can confuse patients.

A greater risk is the incidence of side effects, which increase when off-label prescribing is not based on sound medical evidence. Alternatively, the drug may not work at all.

Although strongly advocated by some doctors and politicians at the time, a 2021 review debunked the use of hydroxychloroquine to treat COVID and found an increased risk of death with its use.



Read more: Coronavirus: Scientists promoting chloroquine and remdesivir act like sports rivals


Recently, Ozempic (semaglutide) caused a storm, with celebrities touting its miraculous weight loss effects. This off-label prescribing has led to continued drug shortages affecting people with diabetes. But also to the development of Wegovy for weight loss.

No legal duty

Although there is no legal obligation to inform a patient that they have been prescribed an off-label medication, prescribers follow professional guidelines. They say it’s good practice to tell patients if a drug is being prescribed off-label.

Patients should be given information about the drug, including possible side effects and reasons why they have been prescribed an off-label drug versus an authorized drug.

A patient who has been told or suspects that their medication is off-label should ask why they have been prescribed this medication and what the risks and benefits are. They might wonder how likely this medicine is to work and what might happen if they don’t have the medicine.

Off-label drugs do not have the same medical evidence as licensed drugs, and improper use could lead to more side effects. However, for some people, off-label medications may be the best or only option, and they need to receive honest and accurate information about their treatment.

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