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THE PARENTS OF a young boy with a rare genetic condition have pleaded to be granted access to medication that could greatly improve his quality of life.
Sam Bailey has Spinal Muscular Atrophy (SMA) – a life-threatening and debilitating disease that causes progressive muscle weakness and loss of movement due to muscle wasting.
The most common form of the disease is 5q SMA, which has four different types: 1, 2, 3 and 4 – based on age of onset and the highest physical milestone achieved.
Sam, who will turn nine in March, is from Rathcoole in Co Dublin. He was diagnosed with SMA Type 2 when he was 13 months old.
Symptoms of SMA Types 1, 2 and 3 begin at different ages in childhood. Symptoms of SMA Type 4 begin in adulthood.
About one in 11,000 babies are affected by SMA. It is the number one genetic cause of death of infants. Type 1 is the most severe form of SMA and accounts for between 50–70% of cases of childhood onset SMA. It is in the same family as motor neurone disease.
SMA is passed from parents to their children through their SMN1 genes. When two people who are carriers have a child together, their child may inherit two faulty SMN1 genes, one from each parent. If this happens, their child will have SMA.
In December 2016, the Food and Drug Administration in the US gave the green light to Spinraza (Nusinersen), the first drug approved to treat children and adults with SMA.
More than 20 countries in Europe have since approved the drug’s use. Two young girls in Ireland with SMA Type 1 were granted access to the drug in January 2017 as part of an emergency programme. Spinraza is delivered through the lower back via a lumbar puncture.
Children who have been granted access to the medication have responded well, gaining strength and motors skills and reaching milestones previously thought impossible.
There are about 25 children in Ireland who could benefit from the medication, and their families are campaigning for access to it. Spinraza is an orphan drug – it is commercially underdeveloped and therefore very expensive, although its price has significantly reduced.
Providing the drug would cost the government around €20 million over a five-year period, about half a previous estimate following on from various negotiations.
In December 2017, the National Centre for Pharmacoeconomics (NCPE) did not recommend the reimbursement of Spinraza as it was not deemed “cost-effective”. Its estimated cost at that time was €37.88 million over five years.
The HSE is currently assessing whether or not to approve the drug. Parents of children with SMA believed a decision on Spinraza would be made before Christmas but this did not happen. A decision may be reached later this month.
More than 85,000 people have signed a petition calling for access to the drug to be granted.
‘You wouldn’t tell a child they couldn’t have chemo’
Sam’s mother Fiona said she understands that Spinraza is expensive but, given how much the cost has reduced, doesn’t believe the price should be the main focus of the discussion.
It’s a lot of money, but if a child has cancer would someone say they can’t have chemotherapy because it’s too dear? Of course not.
“We’re basically being told they can’t have the medication they need, full stop. I understand it’s dear but, as a doctor said to me, a night in ICU is dear, a week in ICU is dear, two weeks in ICU is dear.
“We can expect at least two more stints in ICU in 2019, if we’re lucky. Sam will get to come with us again, if we’re lucky.”
The symptoms of SMA worsen over time and Fiona said it’s “infuriating” to watch Sam’s mobility and strength reduce while prolonged negotiations go on behind closed doors.
“Every moment without Spinraza is a moment lost, the earlier they get it the better. The waiting is torture.
It’s just torturous, there’s no other word for it. Every day SMA is like a ticking time bomb, a cold or chest infection could be fatal. It’s like a shadow following us around, we don’t know when it’s going to pounce.
“When he’s well, he’s super – clever, witty, charming, just gorgeous.”
Sam is fed through a tube and Fiona said one of his biggest wishes is to be able to feed himself. Another is to have the ability to hug his sisters – Kayla (six) and Sarah (two).
Sam uses a wheelchair that he is able to operate himself but his hand function has decreased in recent months.
“Spinraza could make his life so much better, he could regain some of his strength, be able to wrap his arms around his sisters.
If he could throw his arms around his sisters and be well day to day, that would be huge for us.
Sam is in second class in Scoil Chrónáin in Rathcoole but often misses school due to his condition.
He missed most of last October – spending two weeks in the intensive care unit of Our Lady’s Children’s Hospital, Crumlin, and then recuperating at home. He missed over three months of the school year when he was four years old.
Fiona said members of staff at the school have been very supportive.
“Sam said to me the other day, ‘Do you know what mommy, I just love school.’ For an eight-year-old to say that, that’s massive, they spend so much of their day there.”
Sam is passionate about football, West Ham in particular, and plays wheelchair football when he is feeling well enough.
Fiona said Sam will be nine on 6 March but added: “That’s a lifetime away if we don’t get access to Spinraza.”
‘Lives are more important’
Sinn Féin TD Caoimhghín Ó Caoláin is among the TDs to raise Spinraza in the Dáil in recent weeks.
Last month he said it was clear the current process for approving orphan drugs is “not fit for purpose”.
We have seen this situation with Spinraza develop in far too many times for persons with rare diseases which, in many cases, are life-limiting.
Ó Caoláin said patients seeking access to expensive medication often have to protest outside the gates of Leinster House, as have people from SMA Ireland, in order for progress to be made.
“The HSE argue that the cost ratio balance must be right. Of course cost is a factor. But it cannot and must not be the primary consideration. Lives are more important.”
When asked for comment, a spokesperson for the HSE said: “An application for Nusinersen (Spinraza) is currently undergoing assessment.
“The statutory assessment process involves a Health Technology Assessment followed by detailed consideration by the HSE expert groups on new drug therapies, including the Technology Review Group for Rare Diseases and the Drugs Committee.
The assessment focus is twofold and particularly centred on reviewing the evidence of the clinical effectiveness of this new drug therapy i.e. the benefits for patients undergoing clinical trials; and on the cost-effectiveness of the product in view of the prices being charged for the drug, which has an estimated budget impact in excess of €20 million over a five-year period.
They added that the application is “currently going through the final stages of assessment, and the various reports are due to be discussed at the February HSE Leadership Team meeting”, after which a final decision will be made.
A spokesperson for the Department of Health it is “the HSE alone who will make the final decision whether Spinraza will be reimbursed taking into consideration the statutory criteria as specified under the Health Act 2013″.
More information about SMA and Spinraza can be read on SMA Ireland’s website.
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If you don’t have kids you get very little.
@Alan OConnor: Minimum wage set at 13.50 an hour. If you’ve no kids you don’t need it. I remember getting 11, with no kids and having money to spend. Cost of leaving hasn’t gone up that much since.
@Vincent Wallace: oujuhh1q1e asmifcrrmid
@Alan OConnor: That’s because without children one needs very little.
@Vincent Wallace: your talking rubbish son. 600 a week b4 tax isn’t livable even without kids .
Do I need to type a list of reasons why ?
@kevin rock: You have got to be joking. My take home pay is 625 a week. We’re a single income family with 2 kids and a mortgage and while I haven’t got loads of money to throw around I get by OK. I genuinely can’t understand how a single person could not live on 600 a week
@Alan OConnor: It’s worth it.
@Alan OConnor: very true government is a joke should be gone
@Shane O Mac: Typo or grand mal while typing?
@kevin rock: There is a difference between living the way you want, and living comfortably. It’s more than possible to live comfortably on that, you won’t be going to Vegas but you’ll get out.
@Vincent Wallace: I think Shane had a Trumpian moment covfefe :)
@Alan OConnor: apart from increase in tax allowances, reduction in USC, increase in tax bands and energy credits etc
@Alan OConnor: if you work you get very little.
@Vincent Wallace: minimum wage workers shouldn’t have money to spend?
@Alan OConnor: Look on the bright side, the upside of not having kids is not being driven up the wall
@Vincent Wallace: Were you living at home with your parents ? The minimum wage condemns people to shared housing and substandard rentals . No help up the housing ladder which kids provide ( and is often the reason people have them in the first place)
@Shane Harrington: social life.
@Alan OConnor: absolute nonsense, maybe you’re too lazy or too politically motivated to actually read it.
@IMHO: No I was renting with other professionals.
I think there should be a non-childrens allowance for us planet loving people who don’t want to add to it’s destruction!
@Alan Kennedy: Amen!
@Fiona Wyse: A society which pays for not having children. Right!
@Alan Kennedy: Well, why save the planet if there are no children to inherit the planet? You’re not very smart god bless you. I’ll burn some oil in your honour.
@Alan Kennedy: I’m with you on that. Never mind mickey money there should be zip up your mickey money
@Eoghan Augusta: where did he say that no one should have children? Also overpopulation is one of the reasons why the planet is struggling.
@smatrix mantra: it’s very simple, we need people to have children but, we also need people to not have children because we can’t have too many children because that causes issues too!
@Fiona Wyse: who going to look after you when you can’t and bury or burn you….ahhh a robot.
@Alan Kennedy: no children noone to look after you when too old take care yourself
@Fiona Wyse: I agree but it makes this country sound like an economy rather than a society.
@martin finnegan: Neices and nephews have my back!
@Alan Kennedy: Aren’t you a smart cookie! Your siblings will bring up children for your benefit, while you claim you are superior because you are looking out for the planet.
@smatrix mantra: china
@Eoghan Augusta: 2 kids is replacement. More than that and you’re contributing to overpopulation.
The photo of the two politicians giving a thumbs up is Photoshopped. The real one is them giving us a middle finger.
Great country for the dole scroungers!
@Mary M: Is it dole or are all people on social payments all scroungers!
For example , pensioners, people with disabilities or long term illness.
Should we just be culled instead!
If it is so great, try living on an income below the poverty line.
If the journals budget calculator is accurate, I’m up just over €1200. I’ll take that. Thanks, FFFGG!
@honey badger: That’s before tax
@Robert Clifford: eh, no, it’s after tax. After all, it is a tax calculator
@Kevin Kerr: It was a joke Kevin. Lighten up.
@Robert Clifford: Fair enough, tho difficult to tell in type
@Kevin Kerr: Calm down Jeffrey Dahmer
@honey badger: Im no better off when you include fuel tax increase. Maybe 3-10 euro a week.
@honey badger: Don’t worry. The carbon tax will look after that
@honey badger: I’m getting back €1109 that I was been overcharged, but carbon tax increase alone will wipe that out multiple times..
@T Paul Kelly: your getting an extra 12€ on the dole T Paul Kelly.
Double payments two times……. Jesus wept. What’s the point.
@Stephen Mark: Those double payments are also for disabled people and pensioners not just people on the dole
@Stephen Mark: Red the article, it is for the disabled and carers and the old age pensions!
12euro more now to fill a petrol tank, so now everything will go up because so much in life is related to fuel, particulary everything we buy, so more chance of recession and creating greater poverty! Well done government!
@derek: recession is coming definitely. More people are struggling than ever as cost living so high.
I’m 63 – an extra €K a year? I paid, (and still paying) tax all my life, bar two years, and this is what I get in a “windfall” budget? I have no problem with extra finance going to housing (they should have increased that spend by an extra €2b) but why arent they doing something about Bin charges, Water rates etc with the extra finance they are not adding to my income?
@CWrex: you’re so called government don’t care about you or me or anyone day will have food on the table and clothes on there backs and planty of cash in the wallet
@CWrex: households don’t pay water charges
@Alan Moloney: Aw you poor Al? Always someone else’s fault, isn’t it? Whinger.
@Pat Hazzard: Thank you Joan Collins TD!
It’s an EU Directive more than a budget, Nothing for the ordinary working man/woman.
Chambers is hilarious a minister for finance and didn’t even know what he’s reading. Paschal jumped because he would be asked about his bike shed. And Harris went onto Tubridy podcast to pretend he cried about Jean McConville. That’s a man with no backbone and you can tell the way he’s hunched over so far.
@Eoghan Augusta: stupid comment Chambers didn’t know what he was reading ?Wise up
Children s allowance should be means tested people getting it that dont need it.
That donoghue excuse for a human is so slimy he has people convinced he’s great.
@Fintan Stack:
Can’t stand him! Looks and acts like the know it all goody goody kid in school!
@Alan Kennedy: SO EVERYONE STOP HAVING KIDS AND WHEN YOUR A PENSIONER THERE WILL BE NOBODY TO PAY TAX FOR YOU TO LIVE OR TAKE CARE OF PEOPLE IN NURSING HOMES HOSPITALS POLICE AND ANYWHERE ELSE
@Paul Dennis: jeez Paul, no need to shout
@Paul Dennis: I am a pensioner who pay taxes and PRSI for over 52 years. Later on when the usc was introduced, I paid that as well. So the pension I’m getting I’ve payed for over all those years I worked.
@Paul Dennis: You need to fix your phone, your keyboard seems to be broken
I am the average PAYE worker and great full to have employment. I’m going to put the poor mouth on now. I have just calculated that i will be just about €7.50 per week better of, which I suppose is better than nothing, but I will be still paying income tax/prsi/usc on my weekly wages. Those on JS allowance/benefit will be €12 better off per week. My little win in this Budget is well of the €1000 that the average worker will be better of as advertise by the powers that be last week.
So people with kids and social welfare recievers well done. Never anything substantial for other citizens. Petrol 12e extra per tank! Everything we rely on needs petrol to get to us so more hikes on everything, less money in pockets, bigger risk of recession. Well done!!
What about single people!!!
@Moha Bli: plenty for them throughout this budget, maybe read it !
@Moha Bli: They’ll find love too one day, some flowers just bloom later than others.
€380 more… time to spend it on hiked prices!
@Kieran Menon: Before the carbon tax increase are applied.
There’s someone in Ireland that has a load of kids, earns 43k per year, just set up a company and is about to do some research and development in their new company while caring for a loved one who’s just today gave up smoking and has also just started 3rd level education who’s really happy today.
A slap in the face to working families, the Exchequer flush with cash & anything they gave out is minimal & costing more now to travel to work. If you sit on your hole, your first bonus payment is this month either a double payment at Christmas. Nothing done to rein in profiteering by Energy companies, Insurance, Fuel, Groceries, all still a rip off. No point getting up early to go to work every day, work hard, be honest & pay your tax, it doesn’t pay to be honest & hard working in Ireland anymore. This country is set up to punish workers & enrich the Golden Circle. Better off not working & sitting on your hole or coming in & identifying as Ukrainian. Sickening watching the smug arrogance of Jack Chambers & Paschal Donohue, who have abandoned the working Irish people.
@SV3tN8M4: go on and try it living on the dole.
Then come back and say you’re living the high life.
@SV3tN8M4: Try living under the poverty line and see how it feels.
5 days funding for a seven day week!
Carbon tax is a joke. No sense to it at all, as there’s no alternative for lots of people
When are they going to scrap the emergency usc tax seeing as we are not bankrupt anymore
I’m reducing my active contribution to the economy. They’re a bunch of tight fisted criminals. Taxpayer screwed again.
The 400 additional staff announced for the International Protection Department Dept just goes to underline what a joke the whole system is. The stated projected numbers to be processed could be interpreted as an additional 11000 pa for the 400 new staff – a little over 1 person every two weeks on average. Or is it a total of 11000, spread over the new figure of 900 staff, which is even worse.
In the eternal words of Father Fintan Stack “A pair of W……..s”
Enjoy your crumbs. Maybe even save up a few to buy the latest gadget or treat for yourself. But remember your children’s future struggle as they will try put a roof over their head… all because those same crumbs went back to the multinational companies that seek to flood your country with cheap labour to fund their greedy expansion.
9 euros a week better off and social welfare get 12 mmm
@Mark Dawson: And are still under the poverty line!
Just so insulting to be thrown a bone of a few euros by these clowns who have destroyed any chance at a normal life for so many in this country.
Any financial gain from this budget will go straight into the pocket of my landlord. Who puts the rent up every single year, despite dangerously damp and moldy conditions that I have no choice to put up with. Maybe I will have a couple extra quid for a plane ticket if I’m lucky. I can visit friends who were forced to leave to have any chance at having kids.
And kildare county council announce a increase in the property tax!!!! Couldn’t make it up…
@6gRrQtLn: Donegal Co Co decided to keep their 15% increase too they have it a few years now
Just outright doubled the price of ejuice. W@nkers
I would like to have seen Fair City get a special allowance. It is quite brilliant lately. Wha?
What’s the Artists income? Increased, right?
Might it have been prudent to finish the children’s hospital before encouraging half the population to procreate by offering financial reward?
Buttons
Worked all my life paid tax prsi and usc, now in receipt of contributory OAP .
Got the same €12 in the budget as someone who has never worked, never will and contributes nothing, where is the respect and maybe a little thanks?
Why does JC have a limp thumb in the photo?
Does anyone know how the University fees will work this year, i.e. the €1000 reduction in fees if you’ve already paid some or all?
@Dara Cummins: Don’t know Dara but I’d say it’ll only kick in next year.
@Pat Barry: thank you. I did some research and last year’s budget had a similar reduction and it mentioned anyone who paid would be due a refund. I’ll be contacting the citizens information and/or the college directly.
A fine picture of two people who never had a real job, but pretend they know what is going on…can we have their level of tax free exspenses annually – I dont think so.
Why isn’t anyone talking about the national debt we are in big trouble economic wise
We
Pascal and his young apprentice jack won the lotto.they have 3 brothers and 3 sisters.they decide to buy 3 houses and give to 1 brother and 2 sisters. Now pascal and Jack have big houses so they don’t have to worry.the two brothers and 1 sister ask why there not getting houses.the answer was they need to hold onto the money in case they and the two sisters and 1 brother might need the rest of the money in the future and anyway they can rent and save a deposit and get the help to buy scheme money.but the builders increase the price because they know the help to buy scheme is there.
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