Rebekha Sharkie (Mayo, Nick Xenophon Team) Share this | Link to this | Hansard source

I move:

That this House:

(1) notes that:

(a) Cystic Fibrosis is a condition that causes impairment of the lungs, airways and digestive system and leaves sufferers with an average life expectancy of 37 years;

(b) over 3,000 Australians live with Cystic Fibrosis and every four days an Australian child is born with the condition;

(c) over one million Australians are carriers of the gene that causes Cystic Fibrosis;

(d) there is currently an application before the Pharmaceutical Benefits Schedule Advisory Committee (PBSAC) for the drug known as Orkambi which is used to treat the most common mutation of Cystic Fibrosis; and

(e) if approved, Orkambi will be available to over 1,000 Australians aged 12 and over who are currently suffering from this life shortening condition; and

(2) calls on the Government to:

(a) continue to support research into Cystic Fibrosis and its possible cure; and

(b) expedite the PBSAC review of the application to have Orkambi listed on the Pharmaceutical Benefits Scheme so that over 1,000 Australians can have access to a potentially lifesaving drug.

I rise today to speak about a disease that affects over 3,000 Australian men, women and children: cystic fibrosis. Cystic fibrosis is a life-threatening disease for which there is no cure, and it is the most common life-threatening disorder among Caucasians across the world. Over one million Australians carry the gene that causes cystic fibrosis. When a person is diagnosed with cystic fibrosis, they experience a life of frequent hospital stays and ongoing physiotherapy. This is a disease that affects young people: the average life span of patients is just 37 years. Cystic fibrosis causes glands to secrete large amounts of mucus, which obstructs the lungs. Bacteria then get trapped in the lungs, leading to repeated infections and blockages and causing irreversible lung damage. Lung transplants are a treatment measure, but they are not the cure. Cystic fibrosis exists in communities across Australia.

I want to share with you the story of a nine-year-old girl living in my electorate. Her name is Ellis. This week she has been in hospital, being treated for cystic fibrosis and related respiratory infections. Earlier this year, I was contacted by Ellis's grandfather, pleading for help for his granddaughter. Ellis has to undertake an hour and a half of physiotherapy every day and must take enzyme capsules with everything she eats. She takes antibiotics every day and spends around four weeks per year in the Adelaide Women's and Children's Hospital.

The drug known as Orkambi is a new treatment that is proven to improve lung function. Clinical trials have shown a reduction in lung damage by at least 40 per cent more than normal in sufferers of cystic fibrosis. It reduces incidents of chest infections and hospital stays. It treats symptoms of the disease and tackles the disease itself.

For a sufferer of cystic fibrosis, this drug can literally change their life. When I initially lodged this motion, the application to have Orkambi listed on the Pharmaceutical Benefits Scheme was still being assessed. On Friday 18 August the PBS advisory committee handed down its recommendation that this treatment not be added to the PBS. I am not necessarily here today to criticise the PBS advisory committee. I believe that the correct and proper oversight for which medicines are subsidised by the Commonwealth government is fundamental to our country's health system. However, it is my understanding that the efficacy and safety of Orkambi have long been established and that the primary reason for the rejection of the latest application was due to the cost of the drug being too high.

I have read various media reports that place the blame at the foot of the manufacturer and others that place the blame on the government. We should not play the blame game with children across the country while they continue to battle this terrible disease and while we can do something meaningful to assist that suffering—and we can do it quite quickly. Orkambi has recently been approved in Canada for children aged between six and 11 years, and it has also been approved in the United States for children aged 12 and over. Patients in Ireland, France, Germany, Greece and Italy all have access to the drug. I am hopeful that Australia will eventually join these countries in allowing patients affordable access to the drug, but I am frustrated that it is taking so long.

I would like to acknowledge the work of the federal Minister for Health in supporting the process that saw the drug known as Kalydeco being included on the PBS earlier this year. Like Orkambi, Kalydeco is a lifesaving drug for young children suffering from cystic fibrosis. At that time, Minister Hunt was quoted as saying:

… we've been able to deliver the right outcome with Kalydeco, and I am very hopeful that given time, … that we can make real progress on Orkambi.

I would encourage the minister to reflect on these words and on the lives of children like Ellis.

Cystic fibrosis sufferers in Australia are calling for the minister to intervene and to begin negotiations with the pharmaceutical manufacturer, and today I lend my support to that call. I will continue to advocate on behalf of families across my electorate and across the country who live with this disease, and I urge our federal government to keep these children and young people at the forefront of their thoughts when making decisions so that a thousand Australians can have affordable access to this lifesaving medication. Thank you.

Maria Vamvakinou (Calwell, Australian Labor Party) Share this | Link to this | Hansard source

Is the motion seconded?

Milton Dick (Oxley, Australian Labor Party) Share this | Link to this | Hansard source

I second the motion.

George Christensen (Dawson, National Party) Share this | Link to this | Hansard source

In recent days, a little six-year-old girl from Mackay called Maddie said, 'Mummy, when I grow up I want to have 100,000 baby girls.' Now, that's an innocent remark—the aspiration of a child—but painful for her mother to hear, because Maddie has cystic fibrosis. Maddie may not be able to fall pregnant and, depending on the conditions of her lungs at the time, she may not be able to carry a child. The sad reality is that Maddie may not live long enough even to have a family of her own.

The guarantee undertaken by Maddie's parents, Gary and Penne Kaddatz, to keep their daughter healthy and functioning is one that we can scarcely comprehend. Another Mackay family, the Brazils, have a 12-year-old daughter, Ashlin, who battles the same debilitating genetic disorder. It affects the lungs and the digestive and reproductive systems. The Brazils have been through a very rough couple of months. Ashlin contracted an infection that significantly reduced her lung function and she spent a month in hospital.

For both of these families, the possible listing of the drug treatment Orkambi on the Pharmaceutical Benefits Scheme represents a hope that they are too scared to believe in because it represents the possibility of relief from the daily battle and ongoing pain they endure to keep their girls alive and well. I rise today to give a voice to these two mothers—Penne Kaddatz and Michelle Brazil—and to their families to explain their struggles so that others can understand how important it is for them to be able to access an effective medication.

Six-year-old Maddie's daily treatment consists of the following, when she is healthy: 20-minute nebuliser sessions, morning and night, with different medications to loosen mucous; Ventolin twice daily to prevent irritation and inflammation; and vibration vest sessions for 20 minutes a day. She must take enzymes, probiotics, vitamins and salt replacement tablets, and have extra fat added to every meal because maintaining a healthy weight is a constant battle. Every day, Maddie's parents weigh her to see if she is on track. Every cough or sniff they hear from Maddie makes them fear that there might be a lung infection. Every cough from another person makes them want to run a mile to protect their child. They live with what seems to be a ticking time bomb which leaves them in a constant state of fear and panic. They are grateful that their daughter has fared well so far with the condition that she has, but the progressive reality of living with cystic fibrosis is that every infection leaves their child weakened, and sufferers have a life expectancy of 37 years.

They need only to look to the experience of their good friends, the Brazils, to see what life might be like for Maddie in a few years. Ashlin has experienced her worst year to date with cystic fibrosis. Recently, instead of enjoying school holidays, she spent a month in hospital in Mackay and then had to go down to Brisbane battling the condition. Ashlin undertakes airway clearance treatments for up to two hours every single day, and she takes 40 pills just to keep her system functioning. Think of it—40 different pills every day. Despite all she goes through, her mother, Michelle, said her daughter faces every day with pluck and resilience. She said, of children with cystic fibrosis: 'They laugh louder. They hug harder. They play longer. They fight more than they should ever have to.' Both parents hope for the day that a cure is found for cystic fibrosis. Until then, though, they must battle every day against infections which could leave their children with significant and irreversible lung damage. Most deaths from cystic fibrosis are from lung complications or lung failure.

If it were listed on the Pharmaceutical Benefits Scheme, the drug Orkambi would be made available to 1,000 Australians aged 12 and over, and Ashlin is one of them. It has the potential to reduce the amount of medication she needs to take, and it could slow the deterioration of her lungs, which I am sad to say is already occurring. Maddie is too young to be included right now, but it would provide her parents with hope for the future. Even at her young age, her parents can see lung deterioration on the CT scans that they get for their daughter. It can improve the quality and quantity of the lives of these young girls. It could make the difference between life and death.

The current state of play is that the independent Pharmaceutical Benefits Advisory Committee has asked for more information or further evidence of the effectiveness of Orkambi. I hope the company concerned can fulfil any and every requirement that the advisory committee has as soon as possible, because this government has a solid record of funding all drugs recommended by that body, and I call for parties involved to expedite the review. (Time expired)

Milton Dick (Oxley, Australian Labor Party) Share this | Link to this | Hansard source

I rise to support the member for Mayo and, importantly, the need to continue research into cystic fibrosis and its possible cure. Cystic fibrosis affects thousands of Australians every day, and currently there is no cure. The average life expectancy of someone living with cystic fibrosis is just 37 years. It is a debilitating condition that affects many parts of the body, including the lungs, liver, pancreas, reproductive organs and sweat glands. A person born with cystic fibrosis undergoes constant medical treatment and physiotherapy for their entire life. Management and treatment of cystic fibrosis is lifelong, ongoing and relentless. A person with cystic fibrosis may consume up to 40 capsules daily to help digest food, and they may need to do up to three hours of airway clearance each day.

In Australia, one in 2,500 babies are born with cystic fibrosis—that is one every four days. It is the most common life-threatening, regressive, genetic condition affecting Australian children. On average, one in 25 people carry the cystic fibrosis gene—most of whom are unaware they are carriers, with no symptoms apparent. Unfortunately, cases of cystic fibrosis are on the rise, with a 45 per cent increase of cases in Queenslanders over the past decade. In my own electorate of Oxley, there are currently 100 people who live every day with cystic fibrosis.

Unfortunately, figures revealed by the National Health and Medical Research Council showed that funding of research for cystic fibrosis is on the decline: from a peak of $4.6 million in 2014 down to just $2.4 million last year. We must do more to combat this growing disease. Today, I call on the health minister to increase funding so that research can continue, as it must. It is unacceptable that, whilst the rate of Australians living with cystic fibrosis continues to rise, the level of funding into research to find a cure is on the decline. We must ensure we are at least matching the pace at which cystic fibrosis is growing with funding to see that a cure is found.

As the member for Mayo outlined in her speech a short time ago, there is something else that we can do to help the thousands of people living with cystic fibrosis. Many sufferers and families have been calling for the drug Orkambi to be placed on the PBS. This is used to treat the most common mutation of cystic fibrosis and, if approved, Orkambi would be available to over 1,000 Australians aged 12 and over who are currently suffering from this life-shortening, cruel condition. It will principally treat those who are homozygous and have two copies of the mutation in the CFTR gene. This is the most common mutation in people with cystic fibrosis, with around half of cystic fibrosis patients in Australia being homozygous. The Australian Therapeutic Goods Administration observed in 2016 that patients who are homozygous 'have a high unmet medical need and none of the currently approved treatments for this population treat the underlying cause of cystic fibrosis'.

Orkambi aims to treat the underlying cause in these patients by increasing the amount and function of the defective CFTR protein. However, despite three attempts, most recently on 18 August this year, Orkambi is yet to receive approval and be placed on the PBS, due largely to the exorbitant asking price of the manufacturer and pharmaceutical company, Vertex. I note that the Labor shadow minister for health and member for Ballarat has already stated how extremely frustrating this is. Indeed, Labor have been encouraging the government to undertake work with the manufacturer to increase the likelihood of a positive PBAC recommendation and wrote to the government offering our full support on this. Even the chair of the PBAC, Professor Andrew Wilson, conceded this month that large drug companies are not coming to the table with fair offers to begin with. Earlier reports have suggested that the company is seeking nearly three-quarters of a billion dollars to list Orkambi on the PBS for around 1,000 patients. The fact is that, without listing on the PBS, Orkambi will cost sufferers $250,000 a year, or more than $20,000 a month, to access, putting it out of the reach of most of the sickest Australians. We must do all we can to assist people suffering from cystic fibrosis, but this does not mean succumbing to the outlandish demands of multinational drug companies with billion-dollar revenues. I call on the company and the government to continue negotiations and find a resolution as quickly as possible. (Time expired)

Andrew Laming (Bowman, Liberal Party) Share this | Link to this | Hansard source

This is an important debate which throws into the spotlight exactly how the PBAC system works. Cystic fibrosis obviously is the most common life-limiting autosomal recessive disease, certainly for those of European heritage. For a long time there has been very little help available apart from supportive care. One hundred years ago, it was almost certainly a life sentence in the first few years of life, and now, thanks to medical advances, we have life-lengthening treatments like the ones we are debating today. We have a very proud PBAC system, which the rest of the world looks at with envy. We were the first developed economy to put a cost-benefit calculation on every new drug, comparing it explicitly not only to the nearest comparator but also to the dollar benefit achieved for each investment of the PBAC. This is a very broad but utterly fair intersectoral way of comparing new pharmaceuticals and their entry into the Australian system, which, while relatively small on a size basis, allows very rapid penetration and accessibility to the patient cohort once a drug is approved.

Whenever the PBAC considers a drug, it looks at all the evidence provided by the manufacturer and then makes a decision on the benefit to our Australian population as a result. As the previous speaker, the member for Oxley, said, we are increasingly seeing large-ambit, non-cost-benefit-related price claims being made by manufacturers. This causes a great deal of frustration for the general population, who think it is government being reluctant to fund as opposed to there being two parties, only one of whom is using an evidentiary base for deciding the price that we pay per patient—and that of course is the PBAC itself. There is nothing stopping a proponent from coming with a price per patient of one, two or any multiple of what has been decided on a cost-benefit analysis. But increasingly where a drug achieves a life-extending or life-saving ability, it does become more complicated because we are not considering a QALY, a quality-adjusted life year, but trying to measure life itself, and that is increasingly hard.

Up until now we have had million-dollar orphan drugs that were used in such rare circumstances, or biospecific drugs that were only used in particular patients, and we could contemplate these on a case-by-case arrangement. But increasingly we have moved from the 2000s and the era of the blockbuster drug, where you could euphemistically say that adding it to the water would benefit the population. It was almost impossible to measure the tiny gains in cardiovascular survival by giving a drug that appeared to help virtually everyone over the age of 40. Those drugs have come and gone and are in the system. We are seeing the new biospecific drugs that can help a very tiny proportion of the population but have absolutely transformative impacts, and this is one that we are discussing today.

There is no doubt we would love to see a resolution, but we can't move away from the system that until now has served us extremely well. To suggest the minister intervene may be wishful thinking but, ultimately, the experts in the field are commissioned to do this work and to come to an agreement. I do hate to say it—and I say it without the other protagonists in the room: if an agreement can't be struck, it is simply because typically there is no agreement on the specific cohort to be treated, or the amount sought is larger than every agreement we have ever drawn up for patients with different conditions. We have to be a little bit dispassionate here. If they are going to strike a deal with other countries and keep that price confidential, it will be hard for Australia to be blamed when we do not see the rapid approval of a drug here.

I go back to why cystic fibrosis is so important. This is a condition that starts early in life with the colonisation of small airways in particular, and other parts of the body, with infections such as haemophilus and strep. As one moves past the teenage years, pseudomonas and other more complicated infections can have an incredible impact on life. While many of those suffering cystic fibrosis may be infertile, they are not sterile. And there is now the potential to have children through assisted technology. So now, increasingly, we need to be able to screen for this recessive condition and give parents information. It is very expensive to do the screening. So, typically, we do one parent and, if they are positive with this recessive gene, a second parent is offered that screening. While it is tempting to have other parties, including the minister, intervene, this system has stood very well. We need to be careful that, if we do intervene, we do not end up with a deal whereby every other current agreement will then need to be renegotiated.

Tony Zappia (Makin, Australian Labor Party, Shadow Parliamentary Secretary for Manufacturing) Share this | Link to 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.

Trevor Evans (Brisbane, Liberal Party) Share this | Link to this | Hansard source

I am proud to be speaking in this parliament again in support of those living with cystic fibrosis. I will always take every opportunity I can to raise awareness of this disease. Last year, as a very new MP, I went to meet with the team at Cystic Fibrosis Queensland to learn more about their vital work, and I learned that here in Australia we now have more adults than children living with CF due to our improving ability to manage the effects of this incurable disease. Many of them live in Queensland, I should add, because of the weather. As it is a respiratory disease, sufferers tend to move to warmer climes for relief, and so Queensland has a disproportionately high number of people living with CF, and that increases the importance of and reliance on the hard work and resources of Cystic Fibrosis Queensland. It's possibly why so many speakers from Queensland have been joining with me to speak on this motion here today.

The current life expectancy, as was previously said, is only 38 years for those with cystic fibrosis. I turned 36 years old just last week, so that number really resonates with me. Last time I spoke in this parliament about cystic fibrosis, I spoke about my pride that this government had just expanded access to the life-expanding drug Kalydeco by listing it on the PBS for those aged two to five. That announcement by the Minister for Health, Greg Hunt, meant that 30 young children around Australia now have the help they need to stave off the lung disease that ultimately denies them their life expectancy.

Naturally, as I predicted then and as I welcomed at that time, the cystic fibrosis community has now turned its focus to drugs like Orkambi. I note the PBAC has made this recent decision not to list Orkambi on the PBS at this time, and without that recommendation by the PBAC a government can't subsidise the cost of the medicine under the National Health Act, yet it does seem clear that this drug with further clinical testing and trials will have a tangible benefit to the cystic fibrosis community. My understanding is that the PBAC's offer would have seen Vertex, the owner of the drug Orkambi, receive more than $100 million in public funding, but that was declined in those negotiations.

So I and many of my colleagues across this parliament will continue to push for an agreement that will see further testing and future access to the medicine. I note, for instance, the very thoughtful contribution of the member for Bowman just before on how the PBAC works and how sometimes the stakeholders in this global world do not necessarily make it easy for countries like Australia. What I do know is that the Minister for Health, Greg Hunt, has been working tirelessly to see as many life-saving medicines as possible listed on the PBS. The decision to list and fund Kalydeco was one of many decisions that this government's made as part of its investments in new medicines.

It is a solid track record there. This government has so far provided $7 billion of additional subsidies for new medicines. Over 1,000 new medicines have been listed and are being funded. This government has a 100 per cent track record of funding all of the new drugs and medicines that do get recommended by the PBAC. It has a perfect track record of finding the money to deliver those subsidies. I just want to note in passing that that kind of track record doesn't just happen automatically. This is one more example of why it matters to everyone to have strong national economic management. Everyone hoping for new medicines to be listed and then subsidised will rely on a government that can manage the economy and the budget effectively to find the money to pay for them.

My understanding is that there is now a proposal on the table for a phase 4 clinical trial, which would allow patients access to the drug whilst getting the data that we need on its effectiveness. The findings of a trial like that could be used to help get the medicine listed on the PBAC in the future. It seems I join with many of my colleagues across the parliament to call on Vertex to resubmit an application and to consider running further clinical trials to give more patients access to the drug. It's vital that both PBAC and Vertex continue negotiations on the best way forward for Orkambi.

Australia's PBS is one of the foundations of our universal healthcare system, which is the envy of most countries around the world and which we must continue to work hard to defend and to refine over time. It's in this environment—in this policy setting—that I hope for and look forward to Orkambi's eventual listing on the PBS. Achieving quality of life and longer life expectancies for the cystic fibrosis community is paramount, and I look forward to continuing to work hard with Cystic Fibrosis Queensland, with cystic fibrosis sufferers around the country and with other relevant stakeholders to ensure that their advocacy is strong and that they have a voice here in Canberra and in government.

Mike Freelander (Macarthur, Australian Labor Party) Share this | Link to this | Hansard source

This is a personal thing for me. I've looked after people with cystic fibrosis since the 1970s. I've cared for many children who, unfortunately, haven't survived. I've seen the slow but steady improvements in the care of people with cystic fibrosis, and now life expectancy has gone from the late teens to the late 30s. So really there have been some significant changes.

I've seen children with cystic fibrosis present in many different ways, from a condition called meconium ileus, which is a type of bowel obstruction in the neonatal period, through to hyponatremia, low salt levels in the blood and seizures; failure to thrive; malnutrition; and recurrent pneumonia—all different presentations of cystic fibrosis.

Like with many paediatric disorders, Australia has actually led the world in management of cystic fibrosis, and we were one of the first countries in the world to introduce the newborn-screening test for cystic fibrosis. This led to early diagnosis, early treatment and better outcomes. In the days before the 1980s, the diagnosis was made on clinical suspicion, and many children had much-delayed diagnoses and were quite sick and malnourished by the time they were diagnosed.

The underlying defect in cystic fibrosis is a genetic defect in salt transport across the cell membrane. Children with cystic fibrosis produce very sticky mucus, which leads to problems with pancreatic function, malnutrition, recurrent chest infections and chronic cough. Other complications include something called biliary cirrhosis, where the bile is sticky and blocks the bile ducts, and chronic liver disease follows and all the complications of that.

The primary defect really depends on the genetic mutation. There are a whole list of genetic mutations that cause cystic fibrosis. It's what's called autosomal recessive, so you need to get an abnormal gene from each parent. If they are both carriers, their children have a one-in-four chance of having cystic fibrosis. The commonest genetic defect is a gene deletion called delta F508. That's the one that is being targeted in the use of Orkambi. Orkambi is actually a combination of drugs: it's a combination of the drug ivacaftor, Kalydeco, which is used for a couple of specific genetic mutations, and lumacaftor, which is a different sort of drug that targets the two mechanisms that are involved in delta F508 homozygous people with cystic fibrosis, whereas Kalydeco is a life-changing, game-changing medication for people with cystic fibrosis with what's called the G551D mutation. Orkambi is not as game-changing for the 50 per cent of people who have the two doses of the delta F508 mutation. We know from studies that Orkambi does make some difference in lung function, and some improvements in weight gain and in nutrition, but it is not as life-changing and game-changing as Kalydeco.

The government and the minister are to be congratulated for the approval of the use of Kalydeco in the five per cent of people with cystic fibrosis for which it works. That is a wonderful, life-changing drug for those people. Orkambi is slightly different, in that the studies have not shown as dramatic improvements in the delta F508 homozygous people, which is about 50 per cent of the people with cystic fibrosis. Nevertheless, it has shown some improvements. We know that there are better drugs on the horizon, so I think it would be reasonable to have the Orkambi until better drugs are available. I do think we need to spend much more time, effort and resources on better treatments for people with cystic fibrosis. That means better home nursing, better home physiotherapy, better research et cetera, and we can fund the specific clinics for this.

Maria Vamvakinou (Calwell, Australian Labor Party) Share this | Link to this | Hansard source

The time allotted to this debate has expired. The debate is adjourned, and the resumption of the debate will be made an order of the day for the next sitting.