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What is an Orphan Drug?

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.

Read the Fact Sheet "What is an Orphan Drug?"

These drugs are called “orphan” because under normal market conditions, the pharmaceutical industry has little interest in developing and marketing products intended for only a small number of patients.

For drug companies, the extremely high cost of bringing a medicinal product to market would not be recovered by the expected sales of the product.

As a result, the potential market for new drug treatment is also small and the drug companies industry would actually incur a financial loss.