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LAST UPDATE | 25 Feb 2019
FAMILIES OF CHILDREN with a rare genetic condition have criticised the HSE’s decision not to reimburse a drug that could improve their quality of life.
About 26 children in Ireland have Spinal Muscular Atrophy (SMA) – a life-threatening and debilitating disease that causes progressive muscle weakness and loss of movement due to muscle wasting – and could benefit from Spinraza (also known as Nusinersen).
The medication is an orphan drug, meaning it is commercially underdeveloped and therefore very expensive. About one in 11,000 babies are affected by SMA. It is the number one genetic cause of death of infants and is in the same family as motor neurone disease.
More than 20 countries in Europe have reimbursed Spinraza. Access to the medication has been approved in all but 3 EU countries – Ireland, Estonia and the UK (bar Scotland). Two young girls in Ireland with were granted access to the drug in January 2017 as part of an emergency programme.
The HSE said it does not deem the drug to be cost-effective, as it would cost over €20 million over five years. Parents, who have been campaigning for access to the drug for years, said they are very disappointed by the decision.
SMA Ireland is planning to demonstrate outside Leinster House at 11am on Thursday, which is World Rare Disease Day. Members will present a petition calling on the government to grant access to Spinraza; it has almost 90,000 signatures to date.
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. Type 1 is the most severe form of SMA and accounts for between 50–70% of cases of childhood onset SMA. About 95% of children with Type 1 who are untreated die before the age of two.
Spinraza is delivered through the lower back via a lumbar puncture. Children who have been granted access to the medication have generally responded well, with some gaining strength and motors skills and reaching milestones previously thought impossible.
Cost and clinical trials
In a statement the HSE said it “regrets” that it has to date been unable to come to an agreement with the drug’s manufacturer, Biogen. The statement notes “the limitations of the current evidence on clinical effectiveness, and the high price being charged by the pharmaceutical company”.
The HSE said when assessing Spinraza, the organisation’s Drugs Group focused on two areas: reviewing the evidence of the clinical effectiveness of drug (the benefits for patients undergoing clinical trials) and the cost-effectiveness of the drug.
The HSE said the medication would cost €600,000 per patient for the first year and €380,000 per patient per year thereafter, and so would have an estimated budget impact in excess of €20 million over a five-year period.
A spokesperson said, in comparison, the HSE’s total expenditure for complex paediatric homecare packages in 2018 was €23.8 million.
While the HSE is anxious to provide all possible support to this very vulnerable group of patients and those who care for them, the decision reached by the Leadership Team concurs with the recommendation of the Drugs Group, which is that reimbursement could not be approved at the price currently being charged by the pharmaceutical company.
The spokesperson added that the HSE wrote to Biogen last Thursday informing it that the medication would not be reimbursed at the current price.
Under the requirements of the Health (Pricing and Supply of Medical Goods) Act 2013, the company has 28 days to respond or make representations to the HSE’s proposed decision.
‘Figures out of date’
Biogen said it is “genuinely surprised” by the HSE’s statement. “The figures referenced are at least six months out of date. In terms of efficacy, the effectiveness of nusinersen in type II/III SMA patients is supported by a randomised clinical trial in the same way that efficacy has been demonstrated in type I SMA patients,” a statement notes.
It adds that both trials were stopped early on the advice of the US Food and Drug Administration and the European Medicines Agency “given the strength of the interim results”.
The statement also notes that Spinraza has been approved for reimbursement in 25 other European countries, saying the price being paid in those countries is in line with what was offered to the HSE after negotiations.
Biogen recognises that this decision marks a deeply concerning day for SMA patients and their families, and we share that concern: Ireland is now significantly behind many parts of the world in making nusinersen available.
Biogen asked the HSE to re-enter discussions “as a matter of utmost priority”.
Fiona Bailey, whose son Sam has SMA Type 2, this evening called on the HSE to reverse its decision urgently, noting the “debilitating and degenerative” nature of the condition.
“Our son Sam, who’s [ninth] birthday is next week, needs Spinraza now, it is the only drug to treat Spinal Muscular Atrophy,” she said.
Fiona previously told TheJournal.ie she understands that Spinraza is expensive but 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.”
Cost of National Children’s Hospital
Elaine McDonnell, CEO of Muscular Dystrophy Ireland, said it is “unconscionable that new therapies which we know would make a huge difference to the lives of people with SMA are being denied to them”.
And as one of the last countries to hold out on approval for these therapies, it is clear that Ireland is now at odds with regulators, health authorities and experts across Europe.
“Our children deserve to be treated no less than their fellow citizens in other European countries and deserve the health benefits and life experiences that can be afforded by Spinraza, that otherwise may not be attainable.
“There is a tragic irony here in that our government and the HSE have found every penny possible to build a National Children’s Hospital, but are denying the funds necessary to deliver the treatments to the patients for whom the hospital is being built,” McDonnell said, calling on Health Minister Simon Harris to intervene.
‘A price has now been put on the value of life’
Sinn Féin TD Caoimhghín Ó Caoláin is among the TDs to raise Spinraza in the Dáil. Speaking today, he said parents’ hopes “have now been dashed after months of relentless campaigning”.
A price has now been put on the value of life and wellbeing. This decision is morally wrong and has again trapped very ill patients in the middle of a bidding war between Government and the pharmaceutical industry.
Fianna Fáil TD John Brassil also criticised the decision and urged the government to ensure that a Bill which would improve the availability of orphan drugs for rare diseases in Ireland “progresses through the Oireachtas without delay”.
“Spinraza has been one of the more high-profile drugs which has caught the public’s attention, mainly because the families of children suffering with rare neuromuscular disorders have been campaigning so effectively.
“But they should not have to do that. They, and many others, suffering from rare conditions should be able to access the medication they need,” Brassil said.
More information about SMA and Spinraza can be read on SMA Ireland’s website.
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20 million not cost effective vs 1 billion wasted on building the new hospital.
20 million ovet 5 years. How much do our idiot politicians pay themselves in salary, expenses and pensions in 1 month. I’d say its a couple of million. How much are the compensation packages sitting there for the lot of them for when they all leave politics. It wouldn’t be far off 20 million.
@Adrian:
What if the cost increases to €40 million?
Or €60 million?
Or €200 million?
Because that is what drug companies will charge if they think that a Government will pay it.
What you’re advocating is that the State provide absolutely free drugs medications and treatment regardless of cost?
And then you criticize the HSE for wasting money?
Can you see any link between blindly advocating a “pay whatever” policy and spiraling costs?
Genuine question.
@Brinster: How would you feel if it was your child going through this?
Would your opinion change if you were affected?
Genuine questions
@Brinster: how much has the price been hiked since 20 other European countries started paying for it? Or is this the same pro establishment spin that says having an incompetent running a crucial government department is just fine and dandy?
@Pixie McMullen: I’d be disappointed that the drugs weren’t cheaper. Would I expect the taxpayer to fork out €600,000 per year or them and €380,000 per year thereafter? No, I wouldn’t.
@Mike: the figures don’t add up though, based upon €600k in year 1, €380k for years 2-5 and 25 patients equates to €53 million over the 5 years, not €20 million as per the article. I also read last week that due to all the other expensive drugs approved for other groups the budget for these new and rare drugs had already been exhausted.
@Pixie McMullen:
Fair question Pixie.
I’d be doing everything I could.
I assume you would too?
But I like to think I’d have enough common sense to recognize that a State that agrees to pay absolutely any cost for medication in every circumstance is unsustainable.
Maybe I wouldn’t.
Getting back to reality – do you think we should always pay whatever drug companies charge?
Genuine question.
@Mike: If it was your child you wouldn’t care less what it costs the “taxpayer”.
@Brinster: Why is Ireland on the list as one of three countries who won’t pay? Why? FG loves to brag about how well the country is doing. Is it as people suspect the doing well only applies to one small section of society? Why does Ireland have such a penny pinching attitude to cost where people’s welfare in health matters are concerned.It’s worse when you think in this case it’s children who are involved.
@Dave Doyle: It’s all spin … Leo spent more time working with spin doctor’s than he has done with real doctors. remember in 2011, one of the first cuts that FG implemented was to take medical cards of terminally ill children.FG have no interest in helping these people.
@Dave Doyle:
As far as I know Dave, we have several experimental and expensive drugs prioritized.
Orcambi comes to mind.
Ireland sadly has a much higher propensity for spina bifida I believe, as well as some other very rare conditions.
So we are already spending large amounts on very expensive drugs for small numbers.
I think that could be a contributing factor.
The question remains though – do you think the State should blindly pay whatever it costs?
If not, then by and large we agree.
@Dave Doyle:
Over €2 million on a drug for one child over five years could be the equivalent of other life saving treatments for 50 children over the same period.
The HSE are obviously still in negotiation with the manufacturer, and are holding out for a better deal than they are being offered now. The price they are being asked to pay will be completely different than what Germany, France, Italy etc will be asked to pay.
A lot of the drugs companies see Ireland as an easy touch because of muppets like yourself and Pixie thinking we should pay whatever they ask.
You’re okay Dave, you won’t have to pay anything.
@Brinster: The real question is these drug companies make billions so when it comes to these rare drugs why don’t they take a bit of a hit? In the long run it won’t affect much of their profits and would also be a great public relations for drug companies.
@Dave Doyle: I know I wouldn’t, Dave. I wouldn’t give a damn about the taxpayer. But that doesn’t mean I’d expect to get anything!
@Brinster: The way i see it is, in an economy the quality of life of a sick child becomes an exercise in cost accounting. In a properly functioning society the quality of life of a sick child is never an exercise in cost accounting.
@Dave Doyle:
Is that the case anywhere though?
NHS is brilliant but even they don’t find every drug.
My understanding is that per capita our health spend is fourth in the world.
Obviously no value for money but the point a being made by many commenters that we’re wasting money whilst simultaneously berating the HSE for not finding every drug is contradictory.
Why are we still buying from pharmaceuticals as an isolated small nation, why is there not an EU body to procure these drugs at a group discount. These pharma companies depend on these stories of hopelessness to blackmail Governments into paying ridiculous money.
@The long walk home☘️: probably would have made most sense to buddy up with the uk groups but that is hardly likely to happen now.
Cost effective? And they throwing money at a hole in the ground and more suits than medical staff. Plus a government giving away free water and encouraging unemployment. Gas.
@Don Juan: Don’t know what country you live in, but here in Ireland we pay for our water. The rest of your point is well made.
@The Risen: No we don’t
@Roland D Hay: You will when you leave school/college and start paying your way in the world.
@The Risen:
I don’t know about Roland, but I have left college a long time, and I am working and paying tax for over 30 years. We don’t pay for private use of water. That is an inarguable fact.
We pay taxes, (well I do, I don’t know about you), which funds a central exchequer. But we don’t pay for water.
@CrabaRev: So by that argument, taxes go into central exchequer and don’t fund any infrastructure/service? LOL! The mental gymnastics leos fanbois have to do!!
Civil servant her with 20+ years experience, I know how things work.
” We don’t pay for private use of water. That is an inarguable fact.”
Nope. Water has been paid for from central exchequer for the entire history of the state, which includes income from taxation. Now supplemented by 66% of motor tax going to keep Irish waters doors open and their fleet of Audi A^s on the road.
If you’re going to use the word ‘fact’, try doing your homework first.
@CrabaRev: Check out the 1997 Local Government (Financial Provisions) Legislation. The facts of how water is paid for are there.
@The Risen: well, it’s paid for by only the 65 odd % of people that actually pay tax in this country.
@The Risen: I pay for electricity. The more I use the more I pay. I pay for food. The more I eat the more I pay. I pay for petrol. The more I use the more I pay. I pay for clothes. The more I get the more I pay. I pay for water. The more I use the more…………..oh wait.
@sean o’dhubhghaill: Do you pay for any of those twice?
@The Risen: Water, actually. Running and maintaining my own well AND that portion of taxation that you keep going on aboit
@The Risen:
When you “pay for something” you hand out a certain amount of money and you get a certain amount of a product and service in return. That is what paying for something means.
You quite clearly don’t understand what it is to “pay” for something:
“Water has been paid for from central exchequer for the entire history of the state”
That statement is totally incorrect. The reality is that the central exchequer funds water services. That does not equate to anyone “paying” for water. We do not pay for water. That is an inarguable fact. you tired to argue it and failed.
I recollect from some post of yours years ago you saying you were a public servant. If that is the case, Do I pay for you? If so can I get a refund.
“A price has now been put on the value of life”
What an im-becile comment from a politician. What a demagogue. Of course there is a price in the value of life.
Health care is full of continuous financial choices, the health managers are always choosing one service or treatment or recruitment or preventive campaign over another. Be sure that when they choose to treat something, someone somewhere is going to suffer.
So, if you choose to treat a rare expensive disease, it might affect the start of a new diabetes clinic in your area or no appointment of a new respiratory consultant in your hospital, etc. That’s life.
There is nothing free and the money pot is limited. Politicians and managers must make hard choices and live with it. Some populist politicians just live to make p-athetic comments.
The cost over run on the Childrens hospital would pay for more than a hundred and fifty years of this drug for those needing it
The government can splash money around for landlords, allow vultures and banks their profits tax free, think nothing of the billion, so far, in cost overrun of a hospital, yet when kids need an expensive drug to improve their quality of life, it’s deemed not “cost effective”.
There’s a disgusting meanness of mind deep in the heart of FG when it comes to ordinary people.
How is it not “cost effective” when childrens lives have benefited?! You can bet your life that a politicians child would recieve it without delay!!!
The amount of money which has been wasted on the new kids hospital would have bought a sh#t load of drugs for an awful lot of very sick people.
Hypocrisy of the highest order.
More ridiculous tripe from the HSE.
Bank Guarantee, Write downs by NAMA, Childrens Hospital overrun, Irish Water debacle, cost of tribunals and reports, HSE with their agency staff, pensions for disgraced Gardai, politicians and bankers, AIB not paying tax for 20 years, vulture funds avoiding tax… etc…etc.
The money is always there whether it be wasted or just not collected but it’s always there. This is just a pen pushers decision to try save €20 million which will inevitably be used to plug the hole in the Hospital overrun.
Look the drug itself at 600k per person for 25 people is not cost effective. It provides zero value to the HSE or the taxpayer and is unsustainable. Is it ethical to withhold it, no it is certainly not. We live in a capitalist society where these choices need to be made, the new children’s hospital is an investment which will EVENTUALLY yield a profit and is seen as same which allows the state to borrow and spend the money to build it. The Irish people have overwhelming and consistantly voted FF / FG into government since the foundation of the state and therefore are in full support of the government making these cost decisions. 25 people do not constitute any form of crisis, it is unfortunate but that is capitalism, either vote differently or put up with it.
I just knew the crazies would be out in this story. Politicians expenses, Bankers, NCH overruns and even water charges.
@davey boy: The truth hurts Davey?
The HSE isn’t at fault here it is the pharmaceutical industry. Someone make the drug at a reasonable cost.
Do we have to go to Mars to get some of the ingredients or what? Pharma industry needs to be regulated so companies don’t hold people to ransom like this. Capitalism at it’s worst
@Marcas Ivarrson: if only it was that easy. It costs the pharmaceutical companies billions to get a drug to market & can be over 12 years before it is released to market.
@Marcas Ivarrson: also this drug is most likely still within its patent period which means no one else can manufacture it. Not that any other companies will due to the fact its an orphan drug
@Marcas Ivarrson: Wrong dude this is capitalism at its best. The pharma company that produced the drug owns the patent, the system this allows them a number of years to make as much money as possible before said patent expires. It’s called the profit motive, they don’t give a damn about human lives…their sole motivation is to drive growth in the business and the money accumulated will go into reaserching the long term treatment of another disease an extracting more profit….rinse and repeat
Ah sure no worries we shall leave the common folk to suffer and we all can have a nice payrise before the Dail summer break sure who needs medication
Just to let you know that Orkambi CF drug is still not available in the NHS in the UK or health providers in Switzerland. They are holding though until the price is considerably reduced.
Sad.
Greedy drug companies – how can they justify charging 20Million per patient on a drug they are not interested in making more wildly available.
Sounds to be just blackmail.
@Anthony Clark: because it costs them billions to develop the drug & as its for a very rare disease they actually dont sell much of it.
Disgusting and atrocious behavior but hardly surprising
The drug is unethically expensive and of questionable efficacy except in a very small subset of the population. This isn’t like Kalydeco, PTC124 or Okambri which has proven and extreme benefits in certain cohorts. It’s appalling that the manufacturers are charging that much but also unrealistic to expect the government to pay for it.
The government spends over 100 million a year on the asylum system… so 20 million over 5 years for sick children in this country seems like the decent thing to do
@John Power: what a stupid comment?
It will be unethical for govt to give quality of life (questionable for the drug works for very small subset of already small set of patients) of 25 children a higher priority over 1000+ asylum seekers.
In ideal world, no one has to make these hard choices but govt has to. Its an uncomfortable truth.
The whole system of drug negotiations is flawed in my opinion.
For e.g. BIOGEN makes quite a few drugs which are very much cost effective and using by vast popultion. We should be wiling to pay a bit more of these drugs if this drug is cheaper. Paying 25million for a drug (used a majority of population) which otherwise would cost 22million will not break the HSE and at same time give more money to Biogen overall.
People here playing right into drug campanies hands. Handwringing will only keep the price high. People don’t get it
it’s not cost effective but over spending 2billion on a children hospital that being developed in the wrong location is?? how can you place a value on life? what if minister Harris own child needed the medication? cost effective then?
So the HSE is playing God again. No surprise there. I wonder if one of their members children has this condition what would they do? Of course, how stupid of me, with their salary it wouldn’t be a problem. It’s only us mortals and our children who have to suffer
Those poor children, cant imagine how the parents feel..
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