“It’s hope. It’s going to add 30 years to his life. It gives him a life.”
Minutes after the announcement that a cystic fibrosis drug costing $250,000 a year will soon cost something more like $40, these were a Kootingal mum’s words.
Orkambi, which has the potential to double the life expectancy of some people with the disease, has been recommended this afternoon for listing on the Pharmaceutical Benefits Scheme.
Joanne Jones said that, although little Jesse was still too young to access the drug, it gave her and husband Lad something to work towards – and it gave their son a future.
“I think we’ll have a peaceful sleep tonight knowing that Jesse’s life has been changed for the better,” she said.
The decision today was considered crucial, as it was drug manufacturer Vertex’s fourth submission to the Pharmaceutical Benefits Advisory Committee to list the treatment for patients aged 12 years and over.
It was also its first bid to list Orkambi for children aged six to 11 years.
Vertex had indicated this would be its final attempt to negotiate.
But now the committee has recommended the PBS listing for patients with the relevant type of the disease.
Mrs Jones said the aim now was to keep 10-month-old Jesse “as healthy as possible, so he doesn’t have any prior [lung] damage before he starts the drug”.
People with CF develop an abnormal amount of excessively thick and sticky mucus in the lungs, airways and digestive system.
Those with the disease have an expected life span of 37 years.
Daily treatment can include hours of physiotherapy – which for children includes active play but also an adult pounding on their upper body to loosen mucus; nebuliser sessions; extra intake of salt on food and in capsules; and taking enzyme supplements.
Tamworth radiographer Tara Bartlett said it was “perfect timing” for her daughter Alexis, who is four-and-a-half and could be eligible to start the drug in less than 18 months.
“We’re fortunate Alexis has been very healthy … but obviously we want to protect that health as much as possible,” she said.
Mrs Bartlett said she was “ecstatic” at the news and what it meant for Alexis, whom she described as fiercely independent, very compassionate and a big Elsa-from-Frozen fan.
“She’s very good with taking her medication and has been since day dot; she learnt to swallow tablets from about two,” Mrs Bartlett said.
“The permanence of it, she doesn’t understand: she constantly says, ‘Mummy, can you take it away today? I don’t want it anymore’.
“It just means hopefully less hospital admissions and being able to participate in everyday life as a normal kid ... That’s what everyone wants for their kid: to do what they want to do without being hindered by their health …
“She might be able to have a family and all that stuff that we never thought would be possible when we first heard about the diagnosis ... it is amazing.”
People in the CF community gathered in person and on a web videoconference to support each other in the lead-up to what would be emotional news either way in the 5pm announcement.
In the hour before the decision came down, a mother shared her hopes for her child, who turns 1 tomorrow; a woman with a 25-year-old newly engaged daughter with CF said if the news was good she might even be able to welcome some grandchildren; and a dad hugged his 13-year-old daughter as he introduced her to the community while they waited for the life-changing news.
One woman said it was like having a gun to the head and waiting to see whether the trigger would be pulled.
Cystic Fibrosis Australia CEO Nettie Burke said before the announcement that the support and advocacy organisation would either be “going into celebration mode or attack dog mode”.
She said she expected the drug could be available immediately through compassionate access while the listing process was completed.
About cystic fibrosis:
- Cystic fibrosis (CF) primarily affects the lungs and digestive system
- People with CF develop an abnormal amount of excessively thick and sticky mucus in the lungs, airways and digestive system
- This causes impairment of the digestive functions of the pancreas and traps bacteria in the lungs resulting in recurrent infections, leading to irreversible damage
- Lung failure is the major cause of death
- There is currently no cure
- In Australia, one in 2500 babies is born with the disease – one every four days
- Orkambi has been available in the US for two years
- It is suitable for people with CF who have two copies of the F508del gene mutation
- This is Vertex’s fourth submission to the PBAC to list the treatment for patients aged 12 years and over, and its first submission to list the drug for those aged six to 11 years
Source: Cystic Fibrosis Australia