The availability of Orkambi® (containing lumacaftor and ivacaftor) through the Pharmaceutical Benefits Scheme for patients with cystic fibrosis

Page last updated: 05 September 2018

The Australian Government will invest hundreds of millions over the next four years to subsidise a potentially life-changing medicine which treats Cystic Fibrosis, cutting the cost to patients by hundreds of thousands of dollars.

From 1 October 2018 Orkambi® (lumacaftor with ivacaftor) will be available on the Pharmaceutical Benefits Scheme (PBS) for the treatment of all patients six years of age and over who have two copies of the f508del mutation in the CFTR gene.

Patients would otherwise pay more than $240,000 a year for the medicine. This listing will now mean they only pay a maximum of $39.50 per script in 2018, with concessional patients paying just $6.40.

Cystic fibrosis is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time. In people with Cystic Fibrosis, a defective gene causes a thick, sticky build- up of mucus in the lungs, pancreas, and other organs. Lung failure is the major cause of death for someone with Cystic Fibrosis. There is no cure.

Each year around 80 babies with Cystic Fibrosis are born in Australia. About half have two copies of the f508del mutation, making this the most common form of Cystic Fibrosis in Australia.

Orkambi has been shown to slow the rate of decline in lung function and reduce the number of infections in people with Cystic Fibrosis for periods of up to two years. If these improvements can be shown to continue longer term, this could be a significant health improvement for patients with this disease.

The Minister for Health has thanked Vertex for immediately opening up compassionate access to Orkambi from 1 September 2018 ahead of the 1 October 2018 listing on the PBS.

The independent Pharmaceutical Benefits Advisory Committee (PBAC) recommended the listing of Orkambi. The PBAC considered it would be appropriate for the listing to occur under a Managed Access Program (MAP). The PBAC is independent of Government by law and in practice. By law the Australian Government cannot list a new medicine on the PBS without a positive recommendation from PBAC.

The MAP recommended by the PBAC provides for a subsidy to be paid at the Vertex asking price for an extended period to allow Vertex to provide further data to the PBAC on the long term benefits of treatment. If Vertex cannot do that, the subsidy will continue, but at a price consistent with the evidence provided to the PBAC.

It is important to analyse the performance of expensive medicines over time to ensure that the promised benefits become reality and that people’s health and quality of life are indeed better.

The Australian Government has helped improve the health of Australians by subsidising more than $9 billion worth of new medicines and is committed to subsidise all drugs recommended by the PBAC.

PBS listings are published on the Schedule of Pharmaceutical Benefits, which is available through the PBS website.