Australia: Orphan Drugs And Rare Diseases
In Australia, the administrative authority involved in orphan drug regulations, is the Therapeutic Goods Administration (TGA). TGA is part of the Australian government’s Department of Health & Ageing. The following are a few features of orphan drugs in Australia (per the OrphaNet portal) :
• Orphan Drug Policy set up in 1998
• Orphan designation is intended for drugs for rare diseases with prevalence of < 2,000 patients in the total population of 18 million OR approximately 1-2/10,000
• Waiver of application and evaluation and no annual registration fees
• 5-year exclusivity (similar to other drugs)
• All costs of the orphan drug designation process is covered by TGA
• R&D not supported by grants or tax incentives.
The alphabetical list of designated orphan drugs in Australia from the TGA is updated as of February 5, 2015.
The Pharmaceutical Benefits Advisory Committee (PBAC) makes national funding decisions for the Australian public healthcare system. The PBAC assesses orphan drugs separately from non-orphan drugs using very strict criteria.
Since 1995, the Life Saving Drugs Program (LSDP) in Australia provides subsidized access for life saving drugs meeting specific criteria for rare diseases. Reimbursement is provided through a separate budget. The LSDP is important to Australians with rare diseases because it ensures that patients have the same access to treatments as those suffering from common diseases. Ten medicines are currently available for eligible patients for the treatment of the following 7 conditions:
• Gaucher Type 1 Disease: Imiglucerase (Cerezyme), Velaglucerase (VPRIV), & Miglustat (Zavesca)
• Fabry Disease: Agalsidase alfa (Replagal) & Agalsidase beta (Fabrazyme)
• Mucopolysaccharidosis Type I (MPS I): Laronidase (Aldurazyme)
• Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome): Idursulfase (Elaprase)
• Mucopolysaccharidosis Type VI (MPS VI or Maroteaux-Lamy Syndrome): Galsulfase (Naglazyme)
• Infantile-onset & Juvenile Late-onset Pompe Disease: Alglucosidase alfa (Myozyme)
• Paroxysmal Nocturnal Haemoglobinuria (PNH): Eculizumab (Soliris).
Rare Voices Australia (RVA), is a national, not-for-profit organization created in 2012 with a vision “to be the unified voice for ALL Australians living with a rare disease.” RVA is Australia’s national alliance advocating for those who live with a rare disease. Learn about RVA supported activities for Rare Disease Day 2015.
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