Unapproved medicines

In Aotearoa New Zealand most medicines you're prescribed are approved medicines. This means they've been through a process by Medsafe to ensure that the quality of the product meets the standard and that the product can be considered safe and effective for the uses listed in that medicine’s data sheet.

What is an unapproved medicine?

An unapproved medicine is a medicine that has not been approved by Medsafe for use in Aotearoa New Zealand. This means Medsafe has not assessed that medicine’s quality, safety, and effectiveness, and there is no approved New Zealand datasheet for it.

What is a data sheet?

A data sheet is a document that's written by the medicines manufacturer for healthcare professionals. It has detailed information about the medicine, including the approved uses, doses, age ranges and routes (ways that a medicine is given). 

Most manufacturers also provide consumer medicine information (CMI) written for patients. Data sheets and CMI for medicines are available on the Medsafe website(external link). Medsafe is the New Zealand Medicines and Medical Devices Safety Authority and is a part of Health New Zealand | Te Whatu Ora. It's responsible for the regulation of medicines.

Before a medicine is approved

Before a medicine is approved, the manufacturer must submit clinical and scientific data to Medsafe for review. The data must show that the medicine is of acceptable quality and is safe and effective for its intended uses. 

‘Safe’ doesn't mean that the medicine has no side effects. It means Medsafe considered the benefits of using the medicine for a particular purpose outweigh the potential risks. Because this balance can vary from person to person, your healthcare provider will carefully consider which medicines are most suitable for you.

When a medicine is approved

When a medicine is approved, that approval is specific to the uses, doses, age ranges and routes (ways that a medicine is given) that have met Medsafe’s standards. These are published on the Medsafe website and in the medicine’s datasheet.

If changes are needed, such as a new use or dose, the manufacturer must apply for these changes to be reviewed and approved by Medsafe.

In some situations, your healthcare provider may recommend an unapproved medicine, for example when there are limited or no suitable approved alternatives. This must always be discussed with the patient, and informed consent must be obtained before treatment is started.

What is informed consent?

Informed consent means the patient is given clear information about the medicine, why it's being recommended, the potential benefits and risks, and any alternative options, and then agrees to the treatment.

When an approved medicine is prescribed for a different use or condition, patient age, dose or route, this is considered off-label use. This means that the use, dose, age range or route has not been assessed and approved for use in New Zealand.

It's legal for prescribers to use medicines off label, but they must discuss this with you and get your informed consent first.

It’s not legal for drug companies to market (advertise or promote) their medicines for off-label uses.

There are many reasons why your healthcare provider may prescribe an unapproved medicine or the off-label use of an approved one.

Prescribing medicines for children

Many medicines given to children are used off label. This is because clinical trials have not usually been done in children or the manufacturer has not applied for use to be approved for this age group.

Dose or route not approved in Aotearoa New Zealand

Sometimes a medicine is used off label because a different dose or route (way of giving the medicine) is recommended or used in other countries, even though it hasn't been approved for that dose or route in Aotearoa New Zealand.

Supply issues (shortages of approved medicines)

If an approved funded medicine is in short supply in Aotearoa New Zealand, your healthcare provider may prescribe an unapproved alternative funded by Pharmac to ensure treatment continues. Your healthcare provider will discuss the benefits and risks of the alternative medicine and obtain your informed consent before prescribing. Read more about unapproved medicines for supply issues(external link).

Giving informed consent ensures that you understand why the medicine is being prescribed, how it may help you, and the potential risks.

Before you're prescribed an unapproved medicine, you should be given detailed information, including:

• that the medicine prescribed is unapproved in Aotearoa New Zealand
• the possible benefits of treatment and any known risks and side effects
• the possibility of unknown risks and side effects
• any other available treatments using approved medicines.

Off-label use: If you're prescribed a medicine that is approved, but is being prescribed for a use or dose or route that is unapproved, then your healthcare provider should also discuss this with you.

Questions you may want to consider asking your healthcare provider

If your healthcare provider is thinking about using an unapproved medicine or approved medicine for an unapproved use, you may want to ask them questions such as:

• What evidence supports the off-label use of this medicine to treat my medical condition?
• Is this unapproved medicine likely to work better than an approved medicine?
• Are there other medicines or therapies that are approved to treat my medical condition?
• What are the potential benefits?
• What are the risks of harm in my situation?

In Aotearoa New Zealand, the Medicines Act 1981 allows an authorised prescriber (such as your GP) to prescribe, administer or arrange for the administration of medicines for the treatment of a patient in their care. The medicine and its use may or may not be approved. However, only medical doctors can prescribe unapproved medicines. 

Section 25 of the Medicines Act allows an authorised prescriber to “procure the sale or supply of any medicine” for a patient in their care. This means that prescribers may obtain for supply any medicine for a patient under their care (within their scope of practice), regardless of whether it's approved or unapproved in Aotearoa New Zealand. 

However, the prescriber must always provide a professional and ethical standard of care, which includes having informed consent from the patient for use of that medicine.

Section 29 of the Medicines Act allows the sale or supply of unapproved medicines. The person or company who supplies the medicine must notify the Director-General of Health of the supply (via Medsafe), and record the prescriber, the patient and the name and place of supply. Only medical doctors can prescribe an unapproved medicine.

Approved medicines that are prescribed for an unapproved use, dose or route can be supplied or prescribed by any authorised prescriber. There are no reporting requirements associated with such supply.

Note: Section 29A allows the supply of a Pharmac-funded unapproved medicine as an alternative when an approved funded medicine is in short supply in Aotearoa New Zealand.

This means your healthcare provider can prescribe a Pharmac-funded unapproved medicine to replace the usual medicine so that your treatment can continue. Your healthcare provider will explain the benefits and risks and obtain your informed consent before prescribing.