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Access to Orphan Drugs

What Are Orphan Drugs?

An orphan drug is defined as a medicine, vaccine or in vivo diagnostic agent that is:

  • intended to treat, prevent or diagnose a rare disease; or
  • not commercially viable to supply to treat, prevent or diagnose another disease or condition.


Medicines need to be designated as orphan drugs by the TGA before an application to register an orphan drug on the Australian Register of Therapeutic Goods (ARTG) will be accepted.

The full definition of an orphan drug can be found in the Therapeutic Goods Regulations 1990, section 16H.


Designated Orphan Drugs

Before an application to register an orphan drug can be made, a sponsor must first seek orphan drug designation. Information on applications for orphan drug status can be found in the Australian Regulatory Guidelines on Prescription Medicines.

The quality, efficacy and safety of orphan drugs are assessed at the same standard as for other registered medicines.
Drugs designated as orphan drugs by the TGA are shown in the table that can be seen by clicking the link below (PM Orphan Drugs). Only those with a date in the 'Date registered in Australia' column can be supplied in Australia.

Information source: PM Orphan Drugs

Date: January 2013


In Australia, the mechanism by which drugs/treatments for rare conditions are made available is through the Federal Health & Ageing Department’s Life Saving Drugs Program (LSDP).

Through the LSDP, the Australian Government provides subsidised access for eligible patients, to expensive life saving drugs for very rare life-threatening conditions.

Before a drug is made available on the LSDP, it must generally be accepted by the Pharmaceutical Benefits Advisory Committee as clinically effective, but not recommended for inclusion on the Pharmaceutical Benefits Scheme due to unacceptable cost-effectiveness.

Access to the LSDP

Subsidised access through the LSDP is granted in accordance with specified eligibility criteria and subject to certain conditions: Life Saving Drugs Program Criteria and Conditions
Further information go to the Australian Dept of Health & Ageing website link: http://www.health.gov.au/lsdp