ARCS Conferences

The Orphan Drug Program in Australia; experiences to date and where to next (T7)

Synopsis

Australia’s Orphan Drug Program was introduced in 1998. The aim of the program is to encourage sponsors to develop and to market medicines for the treatment, prevention and diagnosis of rare diseases. Orphan drug designation is dependent on the disease being rare, defined by criteria of prevalence (not more than 2000 persons), or commercial non-viability. A major benefit of the Australian program is the waiver of application and evaluation fees for the initial registration. Marketing approval of an orphan drug product in Australiainvolves two stages: an initial application for the product to be designated as an orphan, followed by an application for registration of the product. Almost 200 drug products have now been designated as orphans in Australia, and approximately 30% of these have since been registered for the specified rare disease. The most common therapeutic areas are oncology, haematology and infectious diseases.

This session will comprise an outline of the current procedures for orphan drug designation and registration of orphan drug products, a review of the drugs and indications that have been designated as orphans, the types of epidemiological data required to support an orphan drug designation, and a case study to illustrate the clinical and regulatory challenges faced by developers of orphan drugs.

Learning Objectives:

• Understanding current procedures for orphan drug designation and registration inAustralia
• Understanding data requirements for orphan drug designation
• Awareness of challenges faced by developers of orphan drugs

 

Level of Experience:

Intermediate/Advanced

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