'We are failing these children spectacularly': Row over drug with 'exorbitant' cost rumbles on

Negotiations, and clashes, over the price of Spinraza are ongoing behind the scenes.

IMG_1282 Kayla, Sam and Sarah Bailey Fiona Bailey Fiona Bailey

PARENTS OF CHILDREN with a rare genetic condition have called for negotiations between the HSE and a pharmaceutical company over access to a drug to be sped up.

Last month, the HSE confirmed that it would not reimburse Spinraza (also known as Nusinersen) at its current price. 

The organisation said the medication would cost more than €20 million over a five-year period – €600,000 per patient for the first year and €380,000 per patient per year thereafter. 

Biogen, the company which makes Spinraza, said these figures are out of date but, when asked by to elaborate, said it “cannot comment on pricing”. 

Twenty-six children in Ireland who have Spinal Muscular Atrophy (SMA), a rare and life-limiting muscle-wasting condition, could benefit from Spinraza. 

Fiona Bailey, whose son Sam has SMA, said it is “heartbreaking” and “frustrating” for parents to watch protracted negotiations while their children become weaker.

Twenty-five of the 28 countries in the European Union have granted access to the medication, except Ireland, the UK (bar Scotland) and Estonia.

SMA Ireland has called on the HSE to reverse its decision; and held a protest outside Leinster House on 28 February.

Negotiations, and clashes, over pricing are ongoing behind the scenes. Biogen has hit out at the process used by the National Centre for Pharmacoeconomics (NCPE) to assess the cost-effectiveness of medication.

In turn, the NCPE’s Director, Professor Michael Barry, sharply criticised the “exorbitant” prices charged by pharmaceutical companies for certain medication, such as Spinraza.

In December 2017, the NCPE did not recommend the reimbursement of Spinraza as it was not deemed “cost-effective”. Its estimated the cost at that time was €37.88 million over five years.

As well as the cost, the HSE also cited the “limitations of the current evidence on clinical effectiveness” of Spinraza when announcing its decision not to reimburse the drug last month.

‘Urgent reform needed’  

Biogen has criticised the process used by the NCPE in assessing medication. A spokesperson said: “In SMA there are challenges associated with producing the type of data that the NCPE need to see when reviewing Nusinersen in a general health technology assessment process to determine cost-effectiveness.

“For example, measuring quality of life in a young paediatric population is very difficult, and yet it is a major determining factor of cost-effectiveness in the NCPE process.”

The spokesperson said other challenges “include the uncertainty around long-term health outcomes that inevitably come with a significant therapeutic advancement”.

While the data we have so far is extremely compelling, there are only so many years of data available and limited comparators, which makes determining cost-effectiveness very difficult.

“In more specialised reimbursement methodologies, such as what is now available in Scotland, these kinds of factors are considered.”

Biogen has repeatedly called on the HSE to “urgently reform the appraisal process for orphan medicines like Nusinersen – to allow a greater degree of flexibility for the assessment of such treatment options”.

‘An easy target’ 

When asked about Biogen’s comments, Professor Barry defended the process used by the NCPE, saying the company’s statement is “typical of what we have come to expect from the pharmaceutical industry here in Ireland”.

He told the assessment process is “an easy target for the industry” and “an excuse for not dealing with the fundamental problem of exorbitant pricing of medicines that frequently add little in terms of health outcomes”.

“The failure to reimburse these extremely high-cost drugs is not due to the assessment process, it is due to a funding issue. One of the main purposes of the assessment process is to support the HSE in its discussions and negotiations with the pharmaceutical companies.

I would argue that it is not the assessment process that should be “urgently reformed” but the unrealistic pricing strategies being practiced by the pharmaceutical industry. There is a very clear way to get drugs reimbursed in Ireland and that is to price them fairly.

Barry said we often hear the industry talk about pricing but “rarely about value”.

The reality is that many of the new expensive medicines offer little in terms of health outcomes. In many cases we would be far better off placing our scarce healthcare resources into other areas of the health service like looking after our elderly patients, investing in mental health and disabilities not to mention investing in general practice.

The National Institute for Health and Care Excellence (NICE) in the UK met again last week to discuss reimbursing Spinraza. A decision is expected next month. 

shutterstock_1225164733 File photo Shutterstock / AngleStudio Shutterstock / AngleStudio / AngleStudio

Barry added that highlighting what is going on in other countries is “also of limited value”.

“I have not heard anyone discuss the Orkambi situation in England, where reimbursement is still not approved almost two years after the drug was reimbursed in Ireland.

In addition, the concept that drugs should be reimbursed in Ireland just because they are reimbursed elsewhere is a flawed argument as it does not consider value of the drug or the differences in healthcare systems across Europe, the differing abilities to pay and, crucially, the complete absence of transparency of the various ‘deals’ being done in the different countries.

Barry said the problem is “a direct result of pharma pricing strategies”, adding: “The answer is in their own hands.”

Last month Barry told the Sunday Business Post the HSE had already “almost exhausted” the available funding for new drugs for the whole of 2019.

‘The weakest children in Ireland’ 

About one in 11,000 babies are affected by SMA – a debilitating disease that causes progressive muscle weakness and loss of movement due to muscle wasting. 

SMA is the number one genetic cause of death of infants and is in the same family as motor neurone disease.

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. 

download SMA Ireland SMA Ireland

Children who have been granted access to Spinraza,  have generally responded well, with some gaining strength and motors skills and reaching milestones previously thought impossible. The medication is delivered through the lower back via a lumbar puncture. 

Sam Bailey is one of the 26 children who could benefit from the drug; he has SMA Type 2. His mother Fiona is among the parents who have been campaigning for access to Spinraza. She gave an impassioned speech at the recent rally outside Leinster House.

Speaking to, Fiona said waiting for a decision while negotiations go on behind the scenes is “heartbreaking”.

“Twenty-five other countries [in the EU] granted access to Spinraza, some as long as a year ago.

“The details are up to Biogen and the NCPE to hammer out. Our lives are full with trying to contain this disease and keep out children pain-free.”

Fiona said she’d “move over [to Scotland] in the morning if Sam could get the drug” but that it isn’t available to people from other countries.

We’re part of the EU, how can we be any different? To me it’s nonsensical, our kids are not getting same treatment as kids in other countries … Our country is failing these children spectacularly.

Parents in Ireland are keeping a close eye on Spinraza negotiations in the UK. Two babies and a teenager have died since the start of the campaign there. “Is that what’s going to happen in Ireland? It’s just terrifying for parents,” Fiona said.

Sam and the other 25 kids are the most vulnerable kids in the country – they can’t do anything for themselves, they need 24-hour care.

She said families “will be back outside the gates of the Dáil” if they need to be but she hopes the situation will be resolved soon.

“It’s terrible to have to put our children through that – children in wheelchairs outside Leinster House, literally begging for their lives.”

‘It’s a miracle he’s still alive’

Sam celebrated his ninth birthday last Wednesday. Fiona said her son is “the same as any other kid” and “was delighted with himself” on the day. However, he is aware of what is going on.

Sam will not see his teenage years if he doesn’t get Spinraza, the fact he’s here today is actually a miracle.

“He was borderline Type 1 at diagnosis, those children don’t normally see their second birthday.

“One chest infection could put Sam in hospital, could put him in ICU. If he gets sick, his life hangs in the balance – he’s one of the most severe sufferers of Type 2 in the country.”

IMG_1727 The Bailey family at Sam's ninth birthday party during the week. Fiona Bailey Fiona Bailey

Fiona said she is obviously aware that Spinraza is an expensive drug but pointed out that so is a spell in ICU or full-time care, which would need to be provided if “God forbid, anything happened me or Paul (Sam’s father)”.

She said getting access to Spinraza would not only be life-changing for Sam but for the whole family. “Sarah and Kayla (his sisters, who are about to turn three and seven) are watching him get weaker, they’re seeking reassurances too,” she said.

“He is losing strength every single month, every single week.”

‘Very vulnerable group’ 

When asked about the ongoing negotiations, a spokesperson for the HSE said “it would be inappropriate to comment” beyond its recent statement until the process is complete.

In its statement last month the HSE said it “regrets” that it has to date been unable to come to an agreement with Biogen. 

IMG_1548 Sarah, Sam and Kayla Bailey at Dublin Zoo Fiona Bailey Fiona Bailey

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. 

“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 statement said. 

Under the requirements of the Health (Pricing and Supply of Medical Goods) Act 2013, Biogen has 28 days from the date of notification to respond or make representations to the HSE’s proposed decision.

More information about SMA and Spinraza can be read on SMA Ireland’s website.

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