‘She’s never had to think about what she’ll do in the future’: Patients hail listing of CF drug

Doug Hendrie

21/08/2018 4:33:47 PM

News that a long-sought cystic fibrosis drug will be listed on the PBS has given many Australians a sense of hope.

Sonia Marshall and her daughter Evie, who is ‘completely over the moon’ with news of the new PBS listing.
Sonia Marshall and her daughter Evie, who is ‘completely over the moon’ with news of the new PBS listing.

Early Tuesday morning, Evie Marshall turned to her mother and asked a simple question.
‘Does this mean I can go to work and get a job, like other people?’
Her mother, Sonia, just nodded.
Evie, a 12-year-old from the Sunshine Coast, has cystic fibrosis, a cruel disease that dramatically shortens lifespan to an average of 37.5.  
The Pharmaceutical Benefits Advisory Committee (PBAC) last week recommended that a ground-breaking combination therapy, Orkambi, be placed on the Pharmaceutical Benefits Scheme (PBS) after years of negotiation with drug company Vertex over the cost.  
At present, the drug – a combination of lumacaftor and ivacaftor – costs a prohibitive $250,000 a year.
The drug is not a cure, but it may hold the promise of a significant boost to lifespan of up to 20 years for the 1000 people who make up the largest group with cystic fibrosis – those who have two copies of the F508del CFTR mutation.   
‘Evie’s still in shock,’ Sonia Marshall told newsGP.
‘She’s never had to think about what she’ll do in the future before. She knew she might not have one. 
‘Evie has been a kid with a life expectancy hanging over her head. She understands this very well, and she’s seen other kids with the same condition pass away.
‘Now she’s completely over the moon. It’s completely life-changing.’
In clinical trials in the US, the drug therapy has been shown to reduce pulmonary exacerbations, which are linked to mortality, by around 40%.
Last month, the Pharmaceutical Benefits Advisory Committee (PBAC) announced there was a ‘clinical place for Orkambi at a price commensurate with its clinical benefits … [h]owever, the evidence presented at that time did not support the clinical claims made by [Vertex] that Orkambi slows the rate of decline in lung function’.
The PBAC subsequently accepted a new submission from Vertex.
However, experts caution that the long-term benefits of the drug remain unclear.
‘Short-term outcomes show promise, particularly as it reduces the rate of pulmonary exacerbations – that is, the acute worsening of symptoms – because this is a predictor of survival,’ Dr Siobhain Mulrennan, director of the Adult Cystic Fibrosis unit at Perth's Sir Charles Gairdner Hospital, told the ABC.
‘But longer-term data and analysis will be required if we are to determine if Orkambi can modify the course of the disease.’
Many people with cystic fibrosis have already gained access to the drug through a compassionate trial offered by Vertex.
When approved for PBS listing, Orkambi will be available to people over the age of six with cystic fibrosis. The Department of Health will monitor the drug’s impact closely and collect data on its long-term effects.  
Cystic Fibrosis Australia CEO Nettie Burke told newsGP that people in the community are ‘walking on air’.
‘It’s incredible. I’ve spoken to people who will benefit personally, and to parents who can now see their child live a normal life expectancy and do what everybody should be allowed to do,’ she said.
‘Most of the current treatments treat the symptoms, but this is treating the disease – and cystic fibrosis is a really dreadful disease.
‘We’re absolutely thrilled that they saw fit to give us this drug. The cystic fibrosis community was an inspiration in advocating for it – we all got together with one voice, very loud, and people had to listen.’

cystic fibrosis Orkambi PBS

newsGP weekly poll How do you use PBS authorities?

newsGP weekly poll How do you use PBS authorities?



Login to comment