Guidance on orphan drug designation has been published by the TGA as part of an ongoing initiative to provide stakeholders with a clear understanding of Australia's regulatory requirements for therapeutic goods.

'Orphan drugs' are often developed to treat small and very specific patient populations who suffer from rare diseases and conditions.

The application and evaluation fees for orphan drugs can be waived to:

• Help reduce their development costs
• Facilitate their access to the Australian marketplace

However, a medicine needs to be:

• Designated by us as an orphan drug before an application can be accepted to register it on the Australian Register of Therapeutic Goods (ARTG)
• Evaluated for quality, safety and efficacy in the same way as other registered medicines

For more details, go to: http://www.tga.gov.au/industry/pm-orphan-drug-applications.htm#.VBDhq0t63fM