MINISTER FOR HEALTH AND AGED CARE MARK BUTLER: Thanks so much for coming out for this really, really exciting occasion for the listing of a terrific new treatment – a world leading new hope for life for hundreds and hundreds of families here in Australia but around the world as well. Truly nation leading work, and world leading work undertaken here over the last 10 years in Australia.
Today, I'm delighted to announce that Voxzogo will be listed on the PBS, effective from the 1st of May. This is a terrific, exciting new treatment for achondroplasia, which is the most common form of dwarfism all around the world. This is a very serious condition that particularly impacts people when they're at their younger ages. Patients with this condition are 50 times more likely to die before their fifth birthday than the national average. And if they survive that long, there are a range of very invasive major pieces of surgery, they are likely to have to undertake, as well.
Now, here in Australia, led by the Murdoch Children’s Research Institute, particularly Professor Ravi who will say a few words, there have been clinical trials underway with the company BioMarin, for a new treatment that effectively attacks the genetic causes of this condition and allows the bone growth to resume in a much more standard way. Those clinical trials over the last 10 years have shown terrific effects for these young children, and has now been approved by the Pharmaceutical Benefits Advisory Committee and listed on the PBS. We think there are about 140 young Australians who will benefit from this listing every single year and being listed on the PBS, as people know, makes it affordable for families. Without a PBS listing, this treatment would cost as much as $330,000 for families every single year. These treatments will start very young in life, Dr Ravi will talk about that, but very, very young and endure right through until the growth plates close, which is around 16 or 18 years of age. So, $330,000 a year for maybe 15 to 16 years of life is obviously beyond the means of any family, which is why the PBS is such a terrific medicine system here in Australia. Instead of paying $330,000, this treatment will be available at PBS prices – $30 a script or even $7 for concessional patients.
I want to pay amazing tribute to the families who've been part of these clinical trials, the researchers from the Murdoch Children’s Research Institute that have led the clinical trials work around the world here for 10 years. As I've said, amazing work now pays off for the recognition of this treatment, and a listing on the PBS and obviously also the pharmaceutical company that has led this work BioMarin. Without further ado, I'll ask Professor Ravi to say a few words and then after a couple of more contributions, I'm happy to take questions.
PROFESSOR RAVI SAVAIRAYAN: Thank you very much, Minister. It's an absolute pleasure to be here and today marks a very special occasion. I'd like to, on behalf of all the families, thank the Minister and the Australian Government for making the very foresighted decision to list this new medication, Voxzogo, on the PBS scheme, which means that for every child in Australia, and for every parent in Australia who wishes to use this medication as a lifesaving and definitely lifechanging medication in their child, they have the opportunity to do so. That's an important piece in terms of equity. I think that's an incredible outcome. Specifically, I'd like to thank the real heroes of today, represented by Oscar, Ari and Kira because these are the children and their brave families who have put their trust in our hands and the hope that rigorous science will bring around life changing benefits for their children.
Achondroplasia, as the Minister has correctly said, it's not just a condition that affects height. It's a lifelong and serious condition. And really, for many years, I was left in embarrassed silence when patients would ask me, well, what can you do about my child's problem? Because there'd been no real advances and treatment despite the fact the gene from achondroplasia was found about 30 years ago. I guess the first clue that there might be an answer came in 2011, when I happened to be convening an international bone meeting in Palm Cove, and I found out that a company called BioMarin had a new molecule that looked very promising and in achondroplasia animal models had helped improve bone growth. We invited the company to come to our meeting in Palm Cove in 2011 and they presented some very compelling data. But of course, we needed to trial this in young children with achondroplasia and I spoke to the Chief Medical Officer at the time, and he said, we're looking at starting a small trial of 35 patients, how many can Australia take? And I said, 'we'll take them all'. That was the beginning of a great journey.
In the past 10 years, we've led those trials from initial proof of concept to trials in children. Currently, we have over 250 children worldwide in trials of vosoritide, continuing to show its effectiveness over time, and also its safety profile. It's important to note that this medication is not just to increase height, because children with achondroplasia experience a variety of health complications. We are trying to increase health and lifelong health for these children so that they can have increased independence, better functionality, decreased complications, and hopefully will put me out of a job sometime because they don't need to come and seek doctors like myself.
It's a fantastic day. As the global lead investigator, we're really proud of the Murdoch, that has led all these trials from the beginning to the end. And I hope Minister, that this can provide a great template for all Australians living with rare genetic diseases, because we want to have cures for our children, and we want them to live better lives and healthier lives. Today's a great day. It's great to be celebrating this amazing occasion in our nation's capital. And again, I'd like to thank my profound gratitude to the Health Minister and the Australian Government for making this decision and the children who have been on this journey with us, thank you very much.
JOURNALIST: Professor, would you mind just explaining in layman's terms, what the drug actually does and how it works?
PROFESSOR SAVAIRAYAN: Sure, so achondroplasia is like trying to grow your bones, it's like trying to drive a car with a handbrake on, you can't reach average speeds. This medication comes in by precise mechanism, and basically takes that handbrake off, so that average bone growth can resume. It really is like a lock and key therapy. It's one a great example of personalised and precision medicine.
JOURNALIST: And how effective has it been so far? And what do you expect from the hundreds of kids that are going to be able to access it now?
PROFESSOR SAVAIRAYAN: We've already seen that growth is resuming to more average levels. That's translating into better function and better independence and in the very youngest children, we have some data that's going to be published very soon, showing that it may actually increase the diameter of the spinal cord and the base of the skull, which will hopefully impact those really, really serious things like spinal cord compression and sudden death and that increased incidence of sudden death as, as the Minister has already alluded to. So really life changing, lifesaving. It’s much more than height, it's really about continuing health.
JOURNALIST: How widely has the drug being used so far?
PROFESSOR SAVAIRAYAN: It has now been approved in over 37 countries and every week, we're getting more and more approvals across the world. We believe that there's over 400,000 people worldwide impacted. I guess it was really nice to get emails from around the world saying, how do we move to Melbourne? That's what we want. We want people coming to Australia for the best innovation and science so that we can we lead the world in that way.
KATHRYN EVANS, MANAGING DIRECTOR BIOMARIN PHARMACY AUSTRALIA: Thank you, my name is Kathryn Evans. I'm the Managing Director for BioMarin here in Australia and it’s my pleasure to be here today. I want to thank the Minister and the Australian Government for listing Voxzogo on the PBS from the 1st of May, for children and adolescents with achondroplasia whose growth plates are not closed. This is a real milestone to have this option available for all Australian families who would choose to take it up after a diagnosis. I want to acknowledge Professor Kathryn North AC, the Director of the Murdoch Children's Research Institute, and in particular, the families that that Ravi acknowledged before because without them, we wouldn't have got to this point.
I really want to reiterate Ravi’s comments that this has been a really great partnership. We don't do these things in isolation and having the ability to partner with research organisations and investigators like Ravi enables us to bring these products through from those early stages of understanding what these molecules do in the mouse model right through to putting them to market and bringing them to patients because they is at the heart of what we're doing.
BioMarin was founded in Northern California just 25 years ago. Many of you will be familiar with Silicon Valley Tech Hub, which is about an hour south of San Francisco city. But what you might not know is that San Francisco Bay area is also home to a big biotechnology hub. BioMarin is one of those companies based less than an hour north of San Francisco city over the Golden Gate Bridge and we have a very singular purpose, which is transforming lives through genetic discovery. What that means is that we've been able to bring new innovative therapies to patients with rare diseases. We have 8 marketed products and today's announcement brings the sixth one here to Australian patients.
Last year was our 10th anniversary here in Australia. I'm so proud and so thrilled to have been partnering with, with people like Professor Ravi through that journey because it is really a partnership. We need to bring all of these people together and no more so than the families who, as Ravi said, who have put their trust in all of us to get to this point. This is a real milestone, we're so thrilled to be committed to the achondroplasia community here in Australia. Australia really is one of the leaders in the world in this area. And we're looking forward to bringing more important therapies like this to Australian patients.
PROFESSOR KATHRYN NORTH, AC, DIRECTOR MURDOCH CHILDREN’S RESEARCH INSTITUTE: Thank you. Hi, everyone, I'm Katherine North. I'm the director of the Murdoch Children's Research Institute. And just a few points that I'd like to make after what you've heard about this momentous moment. First of all, I'd like to thank Kathryn Evans from BioMarin. And what an amazing partnership it has been, from research into clinical care, into partnerships with industry – which is the only way that we can really make these sorts of steps forward happen at scale.
I've been talking about this being a moment in history. Because a gene for achondroplasia was discovered 30 years ago. 30 years ago, I, like Ravi, was – actually Ravi is much younger than me – was training as a clinical geneticist. And it was a very different world. And we were able to give a diagnosis to less than 1 in 10 kids with rare genetic disorders that affect around 1 in 12 of our community. And there were no treatments. So when a parent has a child that's developing in a different way, what they want the answers to: why is this happening? What is the diagnosis? What can I expect for the future for my child? What are the risks to my other kids or my other family members? And most importantly, what can you do about it? How can you treat to it? And as a clinician, it's been so frustrating way back then to not be able to answer any of those questions.
The promise of genetic medicine and now as we go into what's known as the genomic era, where we have much better technologies to look at genes, is that to make a diagnosis is fundamental so that we can understand what the mechanism of disease is – as Ravi has described. And then target therapies to that change, that the genetic abnormality has caused.
So, 30 years ago, as I said, the achondroplasia gene was discovered. And to me, this is a moment in history, because today, Ravi and all of his colleagues around the world can answer all of those questions that a parent would say to them when the diagnosis is made. And we are able to offer effective therapies. The second thing that I'm incredibly proud of is that we in Australia have led these international trials that are changing the lives of children around the world. And so what now has given Australia the edge in this area? I think that we have a culture of excellence and innovation and collaboration in our health and medical research sector. And I think that was very obvious during the COVID pandemic. We have outstanding people, outstanding researchers, and it was Ravi's passion and enthusiasm, but also the scientific rigor and excellence that's necessary to do the studies to take a drug like this from a mouse model into the patient and into market. Those first trials that Ravi talked about with those 35 patients back in 2013, were the first trials in the world for children with dwarfism and that happened right here in Australia. In Australia, we've also embraced the power of genomics and genomic research.
And we're now leading in a number of areas where we're implementing this new technology, really a disruptive technology into clinical practice. We have very strong global collaborations, whether it's in discovery research, or in clinical trials, so that we bring the very best in international practice and make it happen here in Australia. No longer do you need to go overseas to get the treatment or the best care. Our strong national networks also allow us to function as a single cohort of 26 million people, which is very important for us to be competitive on the world stage. Combined with our quality, our speed our people, I think that's what's given us the competitive advantage to underpin our wonderful partnership with BioMarin. The final piece of the puzzle is the great support that we've had from the Australian Government. We've made a major investment in genomics research over the years, and it's led to a supportive environment where we can bring the very best ideas to fruition very quickly, and drive them into clinical practice to benefit patients and their families. And this investment in effective therapies will ensure equity of access, which is so important to life changing medications. So I'd like to finish by thanking you, Minister Butler and your Government for what I think is quite visionary in terms of investing in these life-changing treatments that are going to change lives. Thank you so much.
JOURNALIST: There are warnings that Cairns is about to lose private maternity services, which would follow a string of similar closures. Is the federal government concerned about women in regional Australia missing out on these essential services and will it step in to help keep them open?
BUTLER: We’re obviously very deeply concerned about equity of access across regional Australia, including particularly for women's health right through the reproductive spectrum. So we will be looking at those reports very carefully. At a more systemic level, Ged Kearney the Assistant Minister for Health with responsibility for women's health, is steering a process to examine all of these challenges across Australia, particularly in regional Australia. Many of them are driven by workforce shortages that have been really exacerbated by the COVID pandemic. So we take very seriously reports like the one you've mentioned, and we want to engage with private sector stakeholders, and obviously, the Queensland State Government to see what we can do to alleviate those pressures and ensure there is real equity of access to services in regional Australia.
JOURNALIST: Minister, the Indian Government is calling for more alternative practices to be added to Medicare. Is this something that the government is considering. And what would that process involve?
BUTLER: We're very interested to welcome the expansion of traditional Indian medicine services here in Australia, as we have for traditional Chinese medicine over years, as well. These are obviously therapies that have a long history and are very deeply embedded in Indian culture. And that's such a big part of Australia now, a very fast-growing part of Australia. As I've said over the last few days, we welcome applications from medical practitioners or sponsors for therapies, including from traditional medicine areas like India and China. They obviously need to go through the usual processes, and that may be an assessment by the Medical Services Advisory Committee. But we're also going to examine, obviously, the place of practitioners of traditional Indian medicine as we have in the past of traditional Chinese medicine.
JOURNALIST: Minister, the government recently put about $10 million towards Indigenous mental health in the Budget ahead of the referendum. And we've seen the ASIO boss yesterday say there's the possibility of spontaneous violence. Do you have any concerns about the tone of public debate heading into the vote?
BUTLER: We know through often the bitter experience of the plebiscite around marriage equality that these things do have an impact on levels of mental distress on parts of the community that are subject to an Australian vote. And in that case, it was gay and lesbian Australians, and in this case will be First Nations people. So we have set aside money to support the mental health of Indigenous Australians through this process because we know through that bitter experience that sometimes, at the edges, the debate can become bitter, it can become personal, it can become distressing and hurtful. Now we will engage with the Indigenous health sector through NACCHO – the Community Controlled Health Organisation sector about the deployment of those funds. And we're obviously going to monitor the mental health of Indigenous Australians through this process very closely.
JOURNALIST: But also just in regards to what ASIO said yesterday. Do you have any concerns generally about the tone of debate?
BUTLER: My job is as Minister for Health. Obviously, I've noticed the remarks made by the Director General of ASIO. I don't have direct responsibility for those issues, you know, threats of violence, for example. My job is to look after the health, including the mental health, of all Australians. And through this referendum process, obviously, First Nations people will be the subject of a vigorous national debate. And unfortunately, at the edges, that debate sometimes becomes deeply hurtful, deeply personal. As to the possibility of violence, I'll leave that for other relevant ministers to talk to.
JOURNALIST: Last night at Senate Estimates the Age Discrimination Commissioner raised concerns about the elderly woman who was tasered in the aged care home. The Commissioner suggested that maybe police shouldn't be the first responders in situations like these, sort of noting that residents in aged care homes might be becoming older, more distressed as the ageing population. Can I ask your concern on that? I wouldn’t ask you to weigh in on that specific case. But generally, should police be the first responders in aged care homes, or should there be some other rather specialised response for residents in distress like this case?
BUTLER: The first thing to say is how deeply distressed I think all of us have been by these reports, and the impact it's had on this elderly lady and her family. And other reports that are surfacing, in this context. Deeply, deeply distressing. Obviously, as you know, Josh, this is the subject of a police investigation, as I understand it, at a state level. We also have the Aged Care Quality and Safety Commission looking at this from an aged care regulation perspective. And I know the Minister for Aged Care is monitoring this very closely. And we will be very open about what we learned from this case and other cases that have been ventilated in the context. Obviously, these matters, the response to some of these calls by aged care facilities, are directly a matter of state government responsibility. But I do hope that in time we are able to have a mature, informed conversation about the way in which people with dementia, as we've had about people experiencing mental illness, are responded to. You know, often sometimes these are these are calls that are made by aged care facilities. But we need to make sure that the response is sensitive to the conditions that so many residents of aged care live in now, which is substantial cognitive impairment through dementia. So I'm not going to pre-empt what might come out of this. Obviously, everyone's thoughts right now are with that woman and her family. But over time, I suspect there will be the need for that sort of a conversation.
JOURNALIST: Minister, a question on telehealth. A lot of private companies have entered this space, and are both prescribing and supplying drugs to Australians, without a video consult without a phone call without even fact checking information? Do you think that this is good medical practice? And do you think that the Medical Board of Australia needs to tighten its regulation in this space?
BUTLER: I think everyone understands that we made an enormous leap in technology in the health sector through the COVID pandemic, through necessity, and much of that has been terrific for patients and terrific for providers, particularly doctors and nurses. But we need to make sure that that happens in a way that aligns with good clinical practice. And I have been concerned at some of the reports about models – or business models in particular – that have developed to fill what is clearly a shortage of access to traditional doctors’ services that I've talked about to a great degree over the last couple of years. We've got the Medical Board having a look at some of the business models that you've talked about. Again, I don't want to pre-empt what comes from that. But I'm determined to see that, as we harness the benefits of modern technology - and frankly, the health sector has been pretty slow to do that compared to other parts of the economy – but as we do harness the benefits of that technology, we continue to adhere to good clinical practice. And I think there is a question mark over some of these business models that are being developed at the fringe.
JOURNALIST: We've heard from the head of the ADF yesterday that around 20% to 25% of illicit drugs passing through Australia's borders are being detected. What does that mean for your vape ban?
BUTLER: We've got a discussion underway within the Commonwealth between government agencies about the way in which the clear government policy will be implemented and enforced. And that clear government policy is to return to the original intent of vapes or e-cigarettes, which was sold to governments around the world as a therapeutic product to help long term smokers kick their habit. It was not sold as a recreational product particularly targeted at the youngest Australians. That's what it's become. And as a result, it is one of the very serious public health menaces that we have in this country, particularly for young Australians. I mean it has become a pathway into cigarettes for young Australians. Which means it's no surprise, really, that the only cohort in the community where smoking rates are actually rising, are youngest Australians. So we are determined to adopt this as a policy. Now, obviously, implementation requires work within the Commonwealth with agencies like Border Force and the Therapeutic Goods Administration, in partnership with state and territory governments. I said when I announced this policy, it is supported by all Health Ministers – Labor and Liberal alike – across the country. And we will have to engage with state and territories to ensure we have a comprehensive enforcement framework. We are committed to making sure that Commonwealth agencies are properly resourced to do that. We've made that clear to Border Force, we've made that clear to the TGA. But the precise shape of those resources will depend on the enforcement mechanism that we agree with states and territories, we will start that work very quickly.
JOURNALIST: Minister, just on the Senate inquiry into reproductive health access. That report is obviously coming out tomorrow. And you've said in the past, this is something that's going to inform your decision making in the area. We didn't see the Budget touched on it much. How soon will you be reading it? And when will you and Ged Kearney meet to discuss it? What's the timeline of that?
BUTLER: Obviously, I haven't seen the report and won't see it until it's published. But I look forward to the report, I very strongly supported the inquiry that was put in place by the Senate Committee. There was a very strong response across the country, which goes back to one of the early questions that I had, about equity of access, particularly in regional Australia. And I'm looking forward to having a look at the report when it's published. And we'll have more to say in due course about it.
JOURNALIST: Do you know when we can expect a response from that inquiry?
BUTLER: We'll have more to say about that in due course, we want to have a look at the scope of the recommendations, the degree to which some recommendations might be better, you know, relatively easy to implement, if we accept them. Others of them may I suspect, require discussion with state and territory governments, which will obviously take longer to implement. I'm not going to pre-empt that. I'll have more to say about it in due course.
JOURNALIST: On the vapes, given the Border Force, even with its kind of full resourcing is only able to stop or detect a quarter of narcotics coming into the country. Despite an illicit tobacco taskforce, the illicit tobacco market has grown. Is this task, potentially impossible? And are you worried about a black market continuing nonetheless? And if there will be full resources, how much money are we thinking?
BUTLER: We're not going to raise the white flag on what is a very serious public health menace for young Australians. After all of the work done in Australia and around the world to drive down smoking rates, the idea that we would raise the white flag on a product that is deliberately designed to create a new generation of nicotine addicts to provide a gateway to cigarettes is simply unacceptable for us. Now, we don't pretend this is going to be easy, as I think I've said before, these things don't come into the country in big shipping containers labelled 'vapes'. So they are going to require a fair bit of work, but they require work in partnership with state governments to enforce the sale of these products as well. Four out of 5 young people have said in research, that these are easy to get and you get them at the convenience stores. Sometimes vape shops deliberately set up down the road from schools because they know that is their target market. So we're confident we can make very serious inroads into this market and respond to very clear calls by parents and school communities and health groups that we need to act to stamp out this public health menace.
JOURNALIST: On aged care and just regarding the closure of aged care facilities. What is your response that the government is moving too fast with this reform rollout, and would the Government consider expanding exemptions for the 24/7 nursing requirements if metropolitan facilities seem to be the ones that are struggling particularly with filling them, at the moment?
BUTLER: What we have moved fast to do is to increase the resourcing of aged care facilities. Even after cutting $2.5 billion dollars out of aged care just before COVID, and the added pressures of COVID that we saw particularly hit the aged care sector – in their last Budget, the former government indexed last year aged care funding per bed by only 1.7%. In spite of all of the pressures that everyone knew about in that sector. Now compare that to our Budget we delivered 2 weeks ago, the per bed increase in funding was not 1.7% but 17%. I mean, this is a huge increase, which has been welcomed by the sector – regarded as a game changer by providers to their financial viability.
Now, we're not going to apologise for our commitment to put nurses back into nursing homes, to increase the time to care that carers have, strictly in line with the recommendations of the Royal Commission. Now, there have been closures over the last little while, as there were in substantial numbers in the final period of the Morison Government. But those closures by and large reflect the financial pressure that had been building on the aged care sector since the $2.5 billion cut to aged care funding in 2016 and 2017. What you see now, are occupancy levels in residential aged care facilities lower than at any time in the 30 years that I've been working with this sector: occupancy levels in the mid 80%, which does make it very hard for an aged care facility to 'wash its face', to be viable. So you are seeing consolidation among big providers that seek to reduce the number of facilities that they have and increase the occupancy level and improve their financial viability. But I'll come back to the point: a 1.7% increase per bed in the last Morrison Government Budget compared to a 17% increase in the Budget we handed down 2 weeks ago. This is a game changer for aged care viability, at the same time that we're lifting standards.
JOURNALIST: Obstetricians would say that it would help a lot if health funds offered women fairer pregnancy cover, including making all policies include pregnancy. Is this something the federal government is willing to take up with insurance companies?
BUTLER: As you know, I think this was a matter among many others ventilated during the Senate inquiry that you asked about a bit earlier. And I imagine that that will be dealt with in the report. I've deliberately allowed this inquiry to run its course. There's been a terrific response by women's health groups, groups like the College that you refer to, and many others besides. And I want to see how that washes out in a series of recommendations that that committee will make to government. Thanks, everyone.