Seona and Hugo are parents of Chloe and Matilda, or Tilly. Tilly is a beautiful young girl, four years old, as she told me.
And she was diagnosed very early on with spinal muscular atrophy.
Spinal muscular atrophy is a very difficult disease. It’s a motor neuron condition for young children, and it is the largest genetic condition in terms of lives lost for under-twos.
It’s hard to imagine something which is more confronting.
Tilly has been on Spinraza since, I think, just before she was one. And you’ll see her running around, you’ll see her laughing.
This is what life is about, this is what medicine’s about.
Today I am delighted to announce that the Australian government will list Spinraza for presymptomatic children with spinal muscular atrophy from the first of December.
What does that mean? It means that instead of having to wait until symptoms have developed, the medicine will be available earlier.
There’s been a lot of science and a lot of research which has been put into this. The Pharmaceutical Benefits Advisory Committee has approved it and we’ve listed it.
It’s a medicine that would otherwise have cost $367,000 a year and would be beyond the reach of virtually every Australian family.
It’s only a small number of children that are likely to be affected and in need, thankfully, about 18.
But for those 18 children, for those 18 families a year, this can be an absolute lifesaver, life-changer, and to be able to see the fruits of medical research, of medicines being brought onto the Pharmaceutical Benefits Scheme in beautiful children such as Tilly is to see something that is immensely powerful and important.
Already on the conditions which have been listed to date about 160 children have had this potentially lifesaving or life-changing medicine.
It won’t work in every case but it will work in a lot of cases. And in cases like Tilly’s, this beautiful girl will grow up and lead a rich, full life.
Every month there’s new science and new developments. John as the chief regulator has been very involved in the passage of this medicine.
At the same time as the enormous progress on the listing of new medicines, we’re also seeing progress on the listing of vaccines for COVID-19.
John has been representing Australia and I can announce that Australia will be part of a new international regulatory partnership for COVID-19 vaccines and therapeutics.
It’s about the sharing of research, the sharing of information.
Australia, the United Kingdom, Canada, Switzerland, Singapore will all be part of this and they’ll work with other like-minded and similar countries such as the United States.
But it’s a five nations regulatory agreement. That will mean earlier information, faster information and a much larger pool.
Instead of 25 million, it’s over 145 million people that will be part of the broad catchment for information and the sharing of knowledge as it comes through.
All of that will help make sure that we have better safety data and earlier safety and effectiveness data.
So these are very important developments on both fronts today.
We know that the last few days for which there’s been full data have shown the two highest days worldwide in terms of new cases and the two highest days, agonisingly, in terms of lives lost.
In Australia, by comparison, both days were zero national days. Six out of the last seven days have been zero cases of community transmission Australia-wide.
We’re not out of the woods. We have to continue our containment measures, but at the same time we’re building our capacity. And the news on vaccines continues to be positive.
We’re cautious, but that’s why we’ve struck this international regulatory partnership.
I might ask John to speak about that and then, I think, Seona is speaking on behalf of the family, and then we’ll take any questions. John.
Well, thank you Minister, and as the head of Australia’s regulator, it’s a real lesson in why we get out of bed each morning and do our job when you see children like Tilly succeeding with Spinraza.
Spinraza is a new therapy that we paid a lot of particular attention to because it actually turns on a gene, and this gene that’s been turned on in people who receive it is the reason why children like Tilly can walk and play along with other children her age.
It’s one of the products that’s at the forefront of new therapies.
Yes, chemical medicines will be important for years and years to come. But these medicines that can turn genes on and off, especially those associated with inherited disorders, are very much the future of medicine.
On the collaboration with five countries, it’s called the ACCESS Consortium. It’ll provide greater access to new medicines.
We’ve been working with four of the countries on other medicines.
But the new announcement is the joining of the United Kingdom with their very large and sophisticated research and regulatory system. And of course we know that a number of the COVID vaccines have come out of the UK as well.
Whether it’s the well-known Oxford-AstraZeneca one or another vaccine coming along from Imperial College that’s also showing promise in its early results.
So the ability to work with like-minded countries with like-minded health systems, the ability to gather safety data from a larger number of countries and people, will actually give us greater reassurance as these products are rolled out. Thanks very much.
Are you happy to tell us about Tilly’s journey?
We’re very excited today for the SMA community that Spinraza has been listed for presymptomatic conditions.
Clearly, every day without Spinraza is a day lost, and a day lost because the motor neurons are dying for these individuals.
So it’s an awesome, awesome thing and we do thank you very much for that.
Matilda’s journey has been quite a difficult one. We were told at 11 months old that she had spinal muscular atrophy and that her condition was life-limiting.
When we asked the question: what was life-limiting? Two years old life expectancy is pretty horrid for me and us as parents. Life-limiting two years old is just horrid.
But we were very lucky. Five days later Matilda started on Spinraza and the treatment was just amazing.
It’s phenomenal. As the honorable Greg Hunt suggested, it’s life-changing and in Matilda’s case it was life-saving.
Matilda had stopped rolling around 10 months but within two months of receiving treatment she was able to roll again.
Very shortly after, she could sing Twinkle, Twinkle Little Star with her hands above her head.
At the age of two-and-a-half she took her first unsupported steps using parallel bars. And then in August last year she walked unsupported.
We were told she would never walk. So the fact that she’s going to die at two, she’ll never walk, it’s quite amazing.
And here she is, a bit of a monster, a four-year-old monster, into everything. So it’s very, very exciting.
But we continue also to advocate to the Minister for pre-listing of- sorry, I’ll start that all again. Sorry about that. We continue to advocate the SMA community to make sure that SMA is also added to the newborn screening.
This makes early detection and diagnosis, which means that children can start on Spinraza as early as possible. Thank you.
Thank you. Happy to take any questions.
The Medical Services Advisory Committee recommended listing for genetic screening for SMA as well as cystic fibrosis and (inaudible). When is that going to be added to the PBS?
So, we’ve received that and we’ll be proceeding with it. So we’re now working with the Medical Services Advisory Committee.
But the next steps for this condition are, firstly, we’ve had some states already do the newborn screening.
And I’ve referred that to the Health Ministers’ Committee to make sure that every state and territory’s doing that.
Secondly, we’re looking at an early listing date for the Medical Services Advisory Committee.
I’m really thrilled that they’ve done that. It fits with what we’ve done with what’s called Mackenzie’s Mission.
So we’re already providing that newborn screening through Mackenzie’s Mission, $20 million for 10,000 couples to have screening and I think it’s about 1540 couples that have commenced.
And so that service is underway, but what we want to do now is to add it to Medicare as quickly as possible as well.
So as soon as all the preparations are done then we’ll do that, but Mackenzie’s Mission has actually got ahead of it and provided that service to over 1500 couples.
Minister, Coalition Senators were pretty vocal in their opposition to a prescription only model for e-cigarettes at the vaping inquiry on Friday. Are you committed to that prescription only model?
Well, there are two different questions. There’s the customs approach and there’s the independent medical approach, and the independent medical approach, which is actually carried out by the TGA is just that.
As we’ve always said, I won’t pre-empt the inquiry, but where there’s medical advice we’ll continue to follow the medical advice.
Minister, the national vaccine rollout plan released on Friday indicated that the Federal Government to have a no jab no entry situation to Australia.
How likely do you think that is to be implemented as a way of encouraging Australians to take that up, and can you see any other situations where there may be a need to have a proof of vaccination, to be able to go about your daily life?
Sure, so the vaccine policy, as you say, has left open those options. It’s certainly going to be voluntary. We, at this stage, haven’t made any decisions.
What we’ve seen is that Australians are extraordinary vaccinators.
One of the big surprises of this year is that even through the lockdowns, whether it was through the first wave or through the Victorian lockdown, we’ve had an increase in five-year-old vaccination rates to record levels, and I would not have expected that.
We went to 94.8 per cent in the June quarter and then to 94.9 per cent in the June quarter for national five-year-olds, and to 97 per cent for Indigenous five-year-olds.
And so that’s coupled with the fact that we’ve gone from 8, to 11 to 13 to 17 million flu vaccines.
So we’re expecting very wide take-up. There are always options which have been reserved, but at this stage I’m not anticipating that they’ll be drawn upon.
What sort of situation will be required for the Government to have to consider such a measure?
So, in relation to vaccination practices and policies, we’ll continue to be guided by the medical experts.
I think that’s seen the hallmark of what we’ve done in Australia over the last year.
We’ve been fortunate to have very, very significant success by global standards but with, of course, losses and tragedy along the way, which can never be minimised.
But we followed the medical advice and we’ll continue to follow the medical advice.
The Australian Technical Advisory Group on Immunisation leads that process in guiding the terms and the circumstances for vaccination, and where they believe that additional actions are required then historically we’ve always followed that.
Minister since August, the number of Australians waiting to come has almost double to around 35,000. Effectively the PM is aiming to get as many Australians, all Australians home by Christmas.
That effectively means that we need to have those people home in the next four weeks if they’re going to be out of quarantine by Christmas. Is it realistic?
I think what’s happened is that we have met and exceeded all of those that were on the list in terms of the total numbers.
The total numbers of Australians that have come home have surpassed the PM’s target.
Additional people are now choosing to do that, and that will be forever in terms of Australians seeking to come home, that Australians leave, Australians seek to come home.
There is a flow around the world to and from Australia, and what we have seen is an increase in hotel quarantine capacity.
We’ve seen the Howard Springs facility come on board. We are exploring with the Northern Territory, and I want to thank the Northern Territory, they’ve provided some very strong guidance that the capacity in Howard Springs could be lifted, and we have the green lane with New Zealand.
There’s the potential for other green lanes with countries where the medical advice is that they are considered overwhelmingly safe for bringing people to Australia without quarantine.
But that’s something that will be used cautiously and sparingly.
So the answer is that there will always be people seeking to come home, and I think that’s a wonderful thing. But in terms of the goals that the Prime Minister has set, we’ve achieved the numbers but we’re now expanding those numbers.
Just back on (inaudible) now have flow on effects to other parts of the economy; Education Minister Dan Tehan today made clear that he wouldn’t allow international students to even come back on pilot programs, separate to existing quarantine caps.
Ultimately if this is going to continue there’s always going to be people trying to come home as long as our borders are closed. Is that going to have a broader impact?
No, I think the pathway here is that we’ll be able to achieve both things. But our priority for now is those Australians who are vulnerable and seeking to come home.
But as the capacity is expanded, whether it’s through hotel quarantine or alternative quarantine arrangements such as Howard Springs, or the third line of support here such as the green lanes, all of those increase the capacity of the country.
And I know that the university and education sector is working, not just with Dan Tehan, but with the Chief Medical Officer and Home Affairs on the potential for expanding those.
What we don’t want to do is displace Australians from coming home. So that’s our principle.
If we can add to the capacity and the states and territories are willing to assist in adding to the capacity, then that gives us the ability to bring not just Australians home, but additional people on top of that.
But the Prime Minister has been absolutely clear that he would not detract from the ability to bring Australians home in order to bring other people in.
I think that provides an important incentive for the states, to say: if we can bring our Australians home and you can provide space for students then that will work for everybody. That’s the pathway.
Victoria’s now (inaudible) two weeks (inaudible) considering where they were. But with temperatures now in the 30s and 40s in regional Victoria, would you be hoping to see the Victorian Government potentially relax (inaudible) around masks?
This is one area where I’ve been very respectful of the state Chief Health Officers’ roles. I think it is appropriate.
As you know I was an early if not entirely effective adopter of mask wearing and provided an example to the country of: we should be wearing masks in appropriate circumstances. And my wife provided the example of how we should be wearing the masks.
But that’s one that we are leaving to the state authorities.
We’ve supported states using masks, and if I’m on a plane I always wear a mask and where we’ve been in parliamentary sittings we’ve continued to wear the masks.
What if you’re in an empty paddock though?
So look, I’ll leave it to the Victorian Government to outline their reasoning. But we have not sought to apply any pressure to the states on that.
What we do want to see is the borders open overwhelmingly by Christmas. We’d like to see all of them open.
We know that WA has a little bit further to go, but there is no advice from the Commonwealth Chief Medical Officer that any state or territory should be closed to any other state or territory.
Minister, to clarify, is there any advice on the table now regards to the coronavirus vaccine, that something like no jab no pay, which has been effective in increasing childhood rates, would be effective in increasing the rollout of the coronavirus vaccine in Australia?
I haven’t received or seen any.
Would you be open to that idea?
It’s not part of our plans at the moment. We’ve been very careful to focus on the voluntary nature of this. And so it’s not a plan.
I’m not going to suddenly rule things out. But what I expect on the basis of the way that Australians have adopted the flu vaccine, the range of National Immunisation Program vaccines is that they’ll probably be vaccinated in record numbers on an entirely voluntary basis.
Minister, when you’re talking about the discussions between Australia and other countries (inaudible) opening international borders, we’ve got the situation with New Zealand, and others that deem to be low risk. You pointed earlier to the records we’re seeing around the world in terms of new infections and deaths.
Does that broader context slow that process down when you see (inaudible) spiralling out of control?
The assessments are done on a country-by-country basis. And so the Chief Medical Officer of Australia, Professor Paul Kelly, leads that process.
It’s obviously deeply focused on how much flow in and out of second countries there are. And it looks at the risk to Australia, so it’s what’s called a risk stratification process.
It assesses each country individually, it looks at their overall case numbers but also their own border control situations, and it’s only when they believe that the situation in that country does not pose a risk to Australia that we’d be willing to consider a country.
But we’ll continue to build those up.
We know that outside of Australia at the moment it is a very grim situation for much of the world and our international borders have sadly been necessary to protect Australians, but they’ll stay in place whilst those protections are required.
Can I just clarify on the quarantine numbers, so obviously the Prime Minister said no international students until Australians are home. We’ve seen over the past months certain business people, actors, (inaudible) film productions coming to Australia, who obviously would have effectively displaced Australians trying to home in terms of the (inaudible) quarantine spots available. When the PM says no international students to replace Australians, Australians first, how broadly does that extend? Does it include business travellers, does it include actors, does it include people who aren’t Australians?
No, where it’s something that an individual is critical to activity, and there are many reasons in terms of national interest. We would like to see students coming, but because there are large numbers that could displace a very large number of Australians, we would like to see those students come when there is additional capacity over and above what’s required to bring home Australians.
I think that’s sensible and I think Australians would find it odd if we were to suddenly say large numbers of Australians should wait. Our goal is to bring our people home.
I’m going to finish now if that’s all right. Matilda and Chloe have been model citizens. If you could talk to my children I’d be very happy with that.
Anyway, thank you very much, take care.