Life Saving Drugs Program (LSDP)

Through the LSDP, the Australian Government provides subsidised access, for eligible patients, to expensive lifesaving drugs for very rare life-threatening conditions.There are currently thirteen medicines available to eligible patients for the treatment of nine conditions. To view the Guidelines and application forms, visit:

Treatment of Gaucher disease (Type 1): Imiglucerase (Cerezyme®), Velaglucerase (VPRIV®), Taliglucerase (Elelyso®) and Miglustat (Zavesca®)

Treatment of Fabry disease: Agalsidase alfa (Replagal®) and Agalsidase beta (Fabrazyme®)

Treatment of Mucopolysaccharidosis Type I (MPS I): Laronidase (Aldurazyme®)

Treatment of Mucopolysaccharidosis Type II (MPS II): Idursulfase (Elaprase®)

Treatment of Mucopolysaccharidosis Type IVA (MPSIVA): Elosulfase alfa (Vimizim®)

Treatment of Mucopolysaccharidosis Type VI (MPS VI): Galsulfase (Naglazyme®)

Treatment of Infantile-onset, Juvenile Late-onset or Adult Late-onset Pompe disease: Alglucosidase alfa (Myozyme®)

Treatment of Paroxysmal Nocturnal Haemoglobinuria (PNH): Eculizumab (Soliris®)

Treatment of Hereditary Tyrosinaemia Type I (HTI): Nitisinone (Orfadin®)


‘Right to Try’ Legislation

Right to Try laws are commonplace in the US, already law in 37 states and under consideration in 13 more. They were created to enable terminally ill patients to try experimental therapies that have completed phase 1 testing but not yet been approved by the Food and Drug Administration, so not yet on pharmacy shelves.  Right to Try expands access to potentially life-saving treatments years before patients would normally be able to access them. If you have a terminal illness and live in a right to try state, you and your doctor should discuss best treatment options for your condition. If those options include a qualifying investigational drug your doctor believes is your best hope, he/she can initiate contact with that drug manufacturer’s compassionate use program director to discuss your options for access. View a sample letter to a drug company.

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