This resource is for people with a rare disease who may benefit from a treatment that is hard to access in Australia because it is:

• Not approved for use in Australia.
• Approved in Australia only for some conditions.
• Not subsidised in Australia.
• Subsidised in Australia only for some conditions.

A treatment is unapproved in Australia if it is:

• Still being tested in a clinical trial.
• Awaiting overseas approval.
• Approved for use overseas (for example, by the Food and Drug Administration in the United States or European Medicines Agency in Europe) but not yet approved by Australia’s regulator, the Therapeutic Goods Administration (TGA). This may be because the company that makes the treatment has not yet applied, or its application is still being assessed.

You can check a treatment’s approval status and approved uses on the Australian Register of Therapeutic Goods (ARTG). If you can’t find a treatment on ARTG, or a treatment is listed as unapproved, it means it hasn’t been assessed for safety, quality or effectiveness in Australia.

A treatment is unsubsidised if it is not funded through the Pharmaceutical Benefits Scheme (PBS), Medicare Benefits Schedule (MBS), Medical Devices and Human Tissue Advisory Committee (MDHTAC), or the National Blood Authority (NBA). For a treatment to be subsidised, a sponsor (usually a pharmaceutical company) must apply for funding.

Different pathways may be available depending on the reason you cannot access the treatment.

You can look for clinical trials by:

• Asking your health care team.
• Searching the Australia New Zealand Clinical Trials Registry for local trials.
• Searching ClinicalTrials.gov for international trials.

These registries provide the criteria for participating and the number of participants researchers are looking for. You should check this information carefully to see if the trial is suitable.

People can stay informed about clinical trials by:

• Telling their clinicians they are interested in clinical trials.
• Monitoring clinical trial registries.
• In some cases, participating in rare disease registries. Some rare disease registries help people connect with relevant research. Before joining any registry, make sure you understand how your information will be used.

Always seek advice from your medical team about clinical trials. This resource about participating in rare disease health and medical research may help if you are looking into clinical trials.

The TGA has pathways that may help people access unapproved therapies. Each pathway has different requirements, so it’s important to check the criteria carefully. Your specialist usually needs to apply for these programs, so speak with your medical team.

• Personal Importation Scheme: an approved person can import a supply of up to three months for certain medicines. This is not applicable to vaping products, controlled substances or any prohibited substances. You may require a valid Australian prescription. Buying medicines online can be risky. Check the TGA’s advice before doing this.
• Special Access Scheme: your doctor may be able to prescribe a medicine that is not approved in Australia. This does not mean the cost of the treatment will be covered so you will need to consider this (see more below).
• Medical Treatment Overseas Program: some people with life-threatening conditions may be eligible for support to access treatment overseas.

Many rare disease treatments are very expensive and not accessible to individuals and families without subsidy. If a treatment is approved but not subsidised for your condition, you could try the following:

1. Ask a patient support group to contact the sponsor and advocate for them to apply for a subsidy through the PBS or provide compassionate access. If there is no condition-specific group, try a broader organisation related to your condition. For example, Epilepsy Australia or Liver Foundation.
2. If there is no patient organisation, contact the sponsor directly or ask the Health Technology Assessment Consumer Evidence and Engagement Unit to contact them for you.
3. If the sponsor will not seek subsidy and you are treated in a public hospital, ask your clinical team whether the hospital can fund the treatment.

Getting access to therapies can be challenging. If these options do not work, talk to your doctor about other treatments or upcoming clinical trials. Patient Voice Initiative’s factsheet has more information on these pathways.

This resource has been co-designed by Rare Voices Australia (RVA) Ambassadors who live with a rare disease, and RVA’s education and advocacy team. Learn more about the team on RVA’s website.