"My Medication Is Being Used Off-Label – Should I Be Worried?" Understanding Off-Label Prescribing

One of the phrases that can understandably make people nervous when discussing medication is "off-label".

A psychiatrist recommends a medication and then explains that it is being prescribed off-label. The patient goes home, opens the information leaflet and discovers that their particular condition may not even be mentioned.

The obvious question is: "If this medication isn't licensed for my condition, why am I being prescribed it?"

It is a very reasonable question. However, off-label prescribing does not mean that a medication is experimental, illegal or that your doctor has simply decided to ignore the rules. It means something much more specific.

What Does "Off-Label" Actually Mean?

Medicines in the UK are licensed for particular uses. The licence can specify things such as the conditions the medicine is approved to treat, the ages in which it can be used, the recommended doses and how the medicine is given.

Off-label prescribing occurs when a licensed medicine is used outside one or more of those specific terms.

For example, a medicine might be licensed for one condition but have evidence supporting its use for another. A medicine might be licensed for adults but sometimes be appropriately prescribed to children. In other circumstances, the dose or way in which a medicine is used may fall outside the exact terms of its licence.

Importantly, the medication itself may still be a familiar, established medicine that has been prescribed for many years.

Why Doesn't the Licence Cover Every Use?

It is easy to assume that a medication's licence represents a complete list of everything doctors know the medication can do.

It doesn't.

Obtaining and changing a medicine's licence is a regulatory process. The licence reflects the particular uses for which the manufacturer has obtained regulatory approval. Clinical knowledge and practice can sometimes extend beyond those specific circumstances.

This is particularly relevant in areas such as psychiatry and children's medicine, where clinicians may sometimes have good evidence or considerable clinical experience supporting a treatment even though that particular use is not included within the product licence.

That does not mean that anything can simply be prescribed for anything. There still needs to be a sound clinical reason for using the medication.

Does Off-Label Mean Unsafe?

No. The words "off-label" do not themselves tell you whether a treatment is safe or appropriate.

The important questions are why the medication is being recommended, what evidence or clinical experience supports its use, what the potential benefits and risks are, and whether there are more appropriate alternatives.

All medicines have potential risks, including medicines prescribed entirely within their licence. Equally, an off-label medicine may sometimes represent a sensible treatment option for a particular person.

The decision needs to be based on the individual rather than simply the wording of the licence.

Why Might a Psychiatrist Recommend an Off-Label Medication?

There are several possible reasons.

Sometimes established treatments have not provided sufficient benefit or have caused unacceptable side effects. Sometimes a person's symptoms or circumstances mean that another medication may offer a better balance of benefits and disadvantages.

There may also be situations where clinical guidelines or established specialist practice support a particular use despite it falling outside the formal licence.

Psychiatry is not simply a process of matching a diagnosis with one approved tablet. Two people with the same diagnosis may have very different symptoms, physical health considerations, previous medication experiences and treatment priorities.

Prescribing therefore requires individual clinical judgement.

Why Does the Information Leaflet Say Something Different?

This can be particularly confusing.

You may have had a detailed conversation with your psychiatrist about why a medication could help, only to read the manufacturer's leaflet and find that your condition isn't mentioned.

Sometimes the leaflet may even appear inconsistent with the way you have been told to take the medication.

The reason is that the manufacturer's information generally reflects the medicine's licensed uses.

It does not necessarily describe every clinically accepted way in which that medicine may be prescribed.

If something in the leaflet worries you, don't assume either that the leaflet is wrong or that your prescription is wrong. Ask your prescriber or pharmacist to explain the difference.

Should My Doctor Tell Me?

You should be given enough information about proposed treatment to make an informed decision.

If a medicine is being used in a way that is unusual or where there are licensed alternatives available, understanding why your clinician is recommending the off-label option can be particularly important.

A useful conversation might include what the medication is intended to help with, why it has been chosen, what evidence supports the decision, what the important side effects are and how the treatment will be monitored.

You should also have an opportunity to ask questions.

The purpose of explaining off-label prescribing is not to frighten somebody with regulatory terminology. It is to make sure that they understand the treatment being proposed.

How Will We Know Whether It Is Working?

Off-label medication should still have a clear treatment goal.

Before starting, it is worth being specific about what you are hoping will change. Are we trying to reduce a particular symptom? Improve functioning? Reduce the frequency of episodes? Help somebody tolerate something that previous treatments have not addressed?

The medication can then be reviewed against those goals.

If it is helping significantly and side effects are acceptable, continuing may be reasonable. If it is providing little benefit or causing significant problems, the treatment should be reconsidered.

Being off-label does not remove the need for ordinary good prescribing practice. If anything, having a clear rationale and review plan becomes particularly important.

What Should I Ask If I'm Unsure?

If you are offered an off-label medication, you do not need to understand the technical details of pharmaceutical licensing before making a decision.

There are some much simpler questions that matter more.

Why are you recommending this medication for me? What are we hoping it will improve? What are the main risks and side effects? Are there alternatives? How will we know whether it is working? What monitoring will I need?

A good prescribing conversation should be able to answer those questions regardless of whether the medication is being used on-label or off-label.

A Final Thought

The phrase "off-label" can sound much more alarming than it actually is.

It does not mean "untested". It does not automatically mean "unsafe". And it certainly does not mean that normal prescribing safeguards no longer apply.

It simply tells us that the particular way in which a licensed medication is being used falls outside the specific terms of its licence.

The more important question is whether there is a good clinical reason for using it that way.

Whenever I discuss medication with a patient, I think the aim should be to move beyond simply asking, "Is this medication licensed for this?"

We also need to ask whether there is good evidence or clinical experience supporting its use, whether it makes sense for this particular person, what alternatives exist, what the potential benefits and risks are, and how we are going to monitor what happens next.

Those are the questions that turn prescribing from simply giving somebody a medication into making a treatment decision together.

Dr James Glass
MBChB MRCPsych
Medical Director, WMI Psychiatry