Vertex Pharmaceuticals‘ Ivacaftor (Kalydeco), a breakthrough medication for the treatment of patients suffering from a specific gene mutation of cystic fibrosis, has been approved to be added to the list of the Pharmaceutical Benefits Scheme (PBS) in Australia, and will become available on December 1st, as announced by Australian Health Minister Pete Dutton.
Addition to the PBS means that the costs of Kalydeco will be subsidized by the Australian government, as part of its National Medicines Policy. The purpose of the decision is to provide medication to patients suffering from the rare and life-threatening disease in order to improve health outcomes and economic means.
In Australia, more than 250 people suffer from the cystic fibrosis G551D gene mutation, and Kalydeco is currently the one medication that specifically addresses the underlying causes of the disease, instead of just treating the symptoms. “The PBS subsidy of this medicine, which would otherwise cost approximately $300,000 a year per patient, will bring great relief to the patients and the families of people affected by this life threatening condition,” said the Health Minister.
“With this new treatment many patients can experience an improved quality of life with reductions in respiratory and gastrointestinal complications, improved lung function and fewer hospitalizations,” he added, announcing that the Australian government is granting $174.5 million to support the supply of Kalydeco on the PBS over the next four years.
“Consistent with the advice of the Pharmaceutical Benefits Advisory Committee (PBAC) that this medicine could be listed on the PBS based on a pay-for-performance basis, all cystic fibrosis patients six years and older who have a G551D mutation in the CFTR gene will be treated with ivacaftor for as long as needed,” he said. “I’m pleased to announce that the sponsor of ivacaftor, Vertex Pharmaceuticals, has agreed to proceed with the PBS listing as recommended by the PBAC,”
In addition to Kalydeco, ten other new medications are also going to be added to the PBS list in December, including therapies for prostate cancer, hepatitis C, and for atypical Haemolytic Uraemic Syndrome (aHUS), another rare and life-threatening disease that affects the vital organs of adults and children. “I’m pleased that patients will now have affordable access to this medication which will help improve their quality of life,” Dutton said. The lists may be subject to final arrangements, to be made with the medications’ suppliers.