Life-changing cystic fibrosis medicines on the PBS

Patients with the most common cystic fibrosis mutation (F508del) aged 12 years and older will be able to access the ‘life changing’ combination medication tezacaftor with ivacaftor (Symdeko) from the Pharmaceutical Benefits Scheme (PBS) from 1 December. Lumacaftor and ivacaftor combination therapy (Orkambi) will also be expanded to include patients aged 2–5 years.

Patients can gain access to Symdeko and Orkambi immediately, with
Vertex Pharmaceuticals agreeing to the Government’s request to provide early access, until the medicine is listed on the PBS on 1 December.

Cystic Fibrosis Australia Chief Executive Nettie Burke described the new listing as an ‘amazing milestone’ and said it would add an estimated 20 years to recipients’ lives.

‘We believe that every Australian has the right to have a healthy life and a regular life and a long life, and drugs like Orkambi and Symdeko do just that,’ Ms Burke said.

‘You imagine, you’ve got a young child who might be starting school shortly. If they don’t have these types of drugs they can’t, because they will get sick, they’ll end up in hospital and their life will be shorter.

‘For adults, they can finish their education, they can get a job and work full time, they can get married, they can have babies, they can have sex, you know. It’s great, this is what these drugs give.’

The average life expectancy for Australians with cystic fibrosis is 38, still less than half that of the average Australian.

The listing of Symdeko will save each patient up to $250,000 per year, with each script now $40.30 or $.6.50 for concession holders.

Federal Health Minister Greg Hunt said the listing is expected to provide new or improved treatment options for up to 1,400 patients. ‘This is immensely important news for families. It is literally a breath of life for families and for patients,’ he said.

‘That means real hope, quality of life, extended life and an extraordinary outcome for wonderful cystic fibrosis patients and their families.’

However, Shadow Health Minister Chris Bowen has criticised the length of time it has taken to list Symdeko on the PBS, given it was recommended by the Pharmaceutical Benefits Advisory Council (PBAC) in March 2019. The majority of PBS listing delays relate to price negotiations between manufacturers and the Government.

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