Off label ... off limits?

Published in MedicinesTalk

Date published: About this date

Health and medicines information in this article may have changed since the date published. This information does not replace advice from a health professional.

You’ve just been given a new medicine, and you’ve read its consumer medicine information (CMI) leaflet. But, your condition is not mentioned in the list of conditions the medicine is used for. Does that matter? Should you talk to your doctor?

What conditions is the medicine used for?

When a pharmaceutical company applies for approval to sell a medicine in Australia, it provides detailed information to the Government. This includes information about the safety of the medicine, the conditions it is intended to treat, and its effectiveness in treating those conditions.

The Government, with the help of experts, reviews the information and decides whether or not the medicine should be approved.

Once approved, the conditions that the medicine is intended to treat are referred to as ‘approved indications’.

The information provided by the pharmaceutical company forms the basis of the information written for patients in the consumer medicine information (CMI) leaflet. The medicine’s approved indications are listed in the ‘What [the medicine name] is used for’ section of the leaflet.

Can the medicine be used for other conditions?

On some occasions, doctors may prescribe a medicine for a condition that has not been approved. Prescribing a medicine for an unapproved condition is sometimes referred to as ‘off-label prescribing’.

Off-label prescribing is not illegal. However, it is not usually encouraged by the Government and medical authorities.

Situations where off-label prescribing may occur

Medicine is safe and effective for the condition

The first situation where off-label prescribing may occur is where good quality research shows that the medicine works and is safe when used for the unapproved condition. This situation may arise if the medicine’s effectiveness for the condition was discovered after the medicine was first approved, and the company that makes the medicine has not yet applied to have the additional condition approved by the Government.

Clinical trials

The second situation where off-label prescribing may occur is where the medicine is being used as part of an official clinical trial to determine whether it is indeed safe and effective for the condition.

People with exceptional circumstances

The third situation where off-label prescribing occurs is to treat people with serious or rare medical conditions whose exceptional circumstances mean that there are no approved medicines that can be prescribed for them.

Is the medicine suitable for children?

Many medicines are tested only in adults. If so, the medicine may be approved only for use with adults.

Because children’s bodies handle medicines differently to adults, doctors cannot be sure that a medicine tested only in adults will work the same way for children. Therefore, some medicines tested only in adults are not approved for use by children.

The children most likely to be prescribed medicines off-label are those being treated in hospital for serious conditions for which there are no medicines approved for use with children.

The medicine may cost more

In Australia, the cost of most prescription medicines is subsidised by the Government under the Pharmaceutical Benefits Scheme (PBS). However, the Scheme only covers medicines prescribed for approved uses. If you have been prescribed a medicine off-label, you may have to pay the full price of the medicine.

Talk to your doctor

Doctors are strongly encouraged by medical authorities to tell you if they are prescribing a medicine off-label, and to highlight any uncertainties about its potential side effects and benefits for you and your condition. They are also encouraged to obtain your written consent.

If your doctor prescribes a medicine for you off-label, ask them any questions you may have. In particular, ask why they think it would be useful for you, and if there are any uncertainties about its potential benefits and side effects. Similarly, ask these questions if your condition does not appear in the consumer medicine information (CMI) leaflet for the medicine.

Asking these questions does not mean that you’re being rude or disrespectful. Rather, it means that you’re taking an active role in your healthcare and getting the information you need to be well informed about your treatment.