Submission to the PBAC in support of Orkambi being listed on the PBS.
The clinical evidence and lived experience as a result of trial and/or compassionate access to Orkambi is unequivocal. Many people experience significant improvement as a result of a decrease in exacerbations, fewer hospitalisations, a reduction in lung damage and an improved quality of life.
Without providing universal access for every eligible person there is no way of knowing who will benefit from Orkambi. An equitable pay-for-performance trial period of 12-18 months would provide the Government, Vertex and clinicians with the patient specific evidence needed to make evidenced based decisions about who should have ongoing, subsidised access.
Globally the investment in cystic fibrosis research is providing new hope and treatments for people who until recently had very little. Every month that their access to Orkambi is delayed is another month of irreversible lung damage while they wait for other, perhaps more affordable, treatments to come onto the market.
I implore the Government to establish a supply arrangement that will ensure all Australians have the opportunity for improved health and wellbeing.
CEO, Cystic Fibrosis NSW