AUSTRALIANS battling a killer blood disease will be granted immediate access to life-saving and prolonging treatment after a drug company ended its gridlock with the federal government over funding.
FEWER than 30 Australians are known to be living with atypical Haemolytic Uraemic Syndrome (aHUS) – an extremely rare blood disease that attacks the body’s vital organs, including the kidneys, heart and brain.
Untreated, one in 10 will die within the first year of diagnosis, and one in three will die within four years. Without the life-saving Soliris medication, which comes with a price tag of $600,000 per adult per year, patients are attached to machines until they die. The drug has been approved by the Therapeutic Goods Administration (TGA) since October 2012, but the manufacturer Alexion and the federal government have been struggling to negotiate a funding deal to make it affordable under the Pharmaceutical Benefits Scheme (PBS). The TGA approved Soliris on the basis that it would be taken for life, but the government only wants it made available under the PBS for the first year. On Friday, the drug’s manufacturer announced it would make the drug available even if it is disappointed with the government’s conditions. “While the government’s treatment funding conditions are not ideal, we will continue to fight to influence this criteria as more clinical data becomes available,” president of the aHUS Patient Support Group Australia (aPSGA) Kerri Grey said in a statement. “In the interim, Australians in desperate need of Soliris will finally be granted access to this life-saving treatment, and no further lives will be unnecessarily lost.” Since March 2013 four Australians have died from aHUS, which the treatment might have prevented, an aPSGA spokeswoman said. Ten patients have been granted compassionate access to the drug since TGA approval.