Orphanet: About orphan drugs

Orphan drugs in Australia

The Australian orphan drugs policy was set up in 1997.

This orphan drugs programme aims to ensure the availability of a greater range of treatments for rare diseases and allows the Australian Therapeutic Goods Administration (TGA) to use information from the US Food and Drug Administration (FDA) Orphan Drugs Program as part of the Australian evaluation process.

The Australian Orphan Drugs Program helps manufacturers to overcome the high cost of marketing drugs which have proved to be commercially not viable because of small patient population.

Orphan designation is intended for drugs which aim to treat diseases with a prevalence of 2000 patients/subjects or less in the Australian population (18 million). Another alternative criterion which leads to orphan designation consists in combining the fact that the drug is not commercially viable, when used in the patient population it is indicated for, and an acceptable rationale for the drug and its indication.

Limit of prevalence for a rare disease : 1,2/10 000

Once orphan designation is granted, the TGA waives the evaluation fees, thus removing a major impediment to making these crucial drugs available. A distinct evaluation pathway for processing orphan drugs is also set up.

One of the programme's important purposes is the possibility of making drugs available to treat leprosy and trachoma which affect the aboriginal population.

The main characteristic of the Australian Orphan Drugs Program is that it is based upon a close collaboration of the TGA with the US FDA. The Australian programme takes into account the FDA's orphan drugs evaluations. Additional criteria are also established for identifying and evaluating orphan drugs in Australia which have not been evaluated in the USA or do not meet the US criteria.

The main characteristics of the orphan drug policy in Australia are :

Regarding the funding of orphan drugs, the TGA covers all the costs of the orphan drug designation process, and then balances its expenditures with other components of the health care system overall budget.

The health-care financing system in Australia may be an issue in the delivery of orphan drugs to patients. In fact, the cost of orphan drugs may prevent some patients using them. Australia has a Pharmaceutical Benefits Scheme, which provides subsidies to make some drugs affordable. The place of orphan drugs in such a scheme is under discussion by decision-makers in the Australian HealthCare Authorities.

In Australia, R&D is not supported by grants or tax incentives.

There is no specific law concerning intellectual property for orphan drugs. The legal status is applied to orphan drugs as for any other drug registered for supply in Australia.

On the other hand, registration fees are covered by the Therapeutic Goods Administration.

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