Tony Zappia (Makin, Australian Labor Party, Shadow Parliamentary Secretary for Manufacturing) Share this | Hansard source

I welcome the opportunity to speak on the motion of the member for Mayo. Cystic fibrosis is an insidious disease that starts at birth and causes life-threatening damage to the lungs, airways and digestive system. It affects more than 3,000 Australians, whose average life expectancy is just 38 years. It affects one in every 2,500 babies and is the most common serious genetic condition in Australia. Children with CF have chest infections and breathing problems and, as they get older, their lungs can become permanently damaged. Because the pancreas doesn't produce the usual enzymes and secretions needed for digestion, sufferers can become malnourished, some will have liver problems and 40 per cent will develop diabetes.

Finally, it seems that a solution may be available for many sufferers but, sadly, they can't access that solution. The Pharmaceutical Benefits Advisory Committee has, for the third time, refused to recommend that the potentially life-saving drug Orkambi be put on the PBS so it can be accessed by those who need it. My understanding is that, because the government and the drug company Vertex can't negotiate a price, the drug is not available. Vertex says the government is demanding a 90 per cent discount, which Vertex says is untenable.

Health minister Greg Hunt's response has been to ask Vertex to resubmit its application and, while that is being done, provide further clinical trials to give more patients access to the drug. That is the government's plan for providing access to Orkambi: ask the drug company, with which it can't negotiate a price, to give away free samples. So the government has confidence in Orkambi if it is freely given away by Vertex, but it will not list it on the PBS.

The chief executive of Cystic Fibrosis Federation Australia, Nettie Burke, says the CF community is 'dramatically disillusioned'—those are her words—that the government and the drug company can't come to an agreement to deliver a life-extending drug to thousands of Australians. Ms Burke, whose very job is to know the suffering this disease brings, says:

It's almost unimaginable that children and adults with a debilitating disease like cystic fibrosis can be denied a drug that could quite literally help save their lives because of cost … it is difficult to understand any sense behind this quarrel over dollars.

To highlight how important Orkambi can be for those with CF, consider this: most people with CF will take up to 40 enzyme replacement tablets every single day. They will also be required to take added vitamins and salt in order to properly digest their food. Many have to inhale medicines to open up their airways. Others require intensive daily chest physiotherapy to break up the build-up of mucus in the lungs, when they could take just two Orkambi tablets a day for a result vastly superior to those provided by any and all of those current methods. If Orkambi were approved for the PBS, more than 1,000 Australians would immediately have a chance to lead a far better and longer life.

There is already research from highly respected sources to support the use of Orkambi. The results of positive trials were published in the British medical journal The Lancet in 2014. In 2015, TheNew England Journal of Medicine reported that two clinical trials found Orkambi, a mixture of the ivacaftor and lumacaftor drugs, produced significant improvements in lung function and vital weight gain. The data also showed a 30 per cent reduction in the number of infections and need for hospitalisation experienced by those who were tested with the drug. I understand that Orkambi has been found to correct mutated genes in people with CF and, rather than deal with the symptoms, treats the root cause. It's been approved by the US Food and Drug Administration, and it's already achieving impressive results in the US.

It's not a catch-all for every strain of CF, but it's a major improvement. Perhaps it could be limited to those people who have the particular strain that it has been found to be effective on. It has been argued that Orkambi is only directed at a specific mutation of CF and that there are 1,800 rare mutations, but it still addresses the needs of about 50 per cent of sufferers today, and it's those 50 per cent that the listing should be focusing on. Continued research is needed, but in Orkambi there is at least relief that, for half of those who suffer from cystic fibrosis, there is a possible solution. I urge the PBAC to move on their review of the application so that Orkambi can be available to those with cystic fibrosis sooner rather than later, because for some it may ultimately be too late.

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