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'Look in my child's eyes and tell him his life is not worth saving': Parents call for access to medication

About 26 children with SMA, a rare muscle-wasting condition, could benefit from Spinraza.

PARENTS OF CHILDREN with a rare genetic condition have said it is heartbreaking that they have not been granted access to a drug that could improve their quality of life.

About 26 children in Ireland who have Spinal Muscular Atrophy (SMA) – a life-threatening and debilitating disease that causes progressive muscle weakness and loss of movement due to muscle wasting – could benefit from Spinraza (also known as Nusinersen).

On Monday, it was confirmed that the HSE would not reimburse the drug at its current price – more than €20 million over five years.

SMA Ireland held a rally outside Leinster House today and presented a petition calling on the government to grant access to Spinraza; it has almost 90,000 signatures to date. 

Twenty five of the 28 countries in the EU have granted access to the medication, all bar Ireland, the UK (bar Scotland) and Estonia.

Speaking at the rally Fiona Bailey, whose son Sam has SMA, said Spinraza “would be life-changing for Sam” and many other children with the condition.

sam1 Sam and Fiona Bailey Source: Órla Ryan

“Sam has had to fight all his life to stay well, to get well, and now Sam and all the other SMA sufferers in Ireland have to fight to live, just because they live in Ireland.”

Fiona, from Rathcoole in Dublin, said children in Europe who have gained access to the treatment are getting “stronger and healthier”, while children in Ireland get weaker, asking: “How is that just and how is that fair?”

She said protesters are calling on the government to “reassess the value it places on a human life and make Spinraza available now”.

SMA-types-e1536103644514 Source: SMA Ireland

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.

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.

Sam, who turns nine next week, has Type 2. Fiona said: “[My husband] Paul and I have such mixed emotions around his birthday.

We feel so grateful that he is still with us and feel so blessed with the wonderful young man he is becoming, but it fills us with dread too because each passing year is like a ticking time bomb.

Ahead of the rally, five parents of children with SMA met cross-party TDs inside Leinster House. One of the mothers, Rebecca Bulman, told TheJournal.ie the meeting was “really emotional”.

Her five-year-old son Harry also has Type 2 SMA. She said it’s “heartbreaking” that a drug which could help her son is being denied. 

Look Harry in the eyes and tell him that his life isn’t worth saving or his life has a cost on it – because it doesn’t.

“We’re not going to give up without a fight,” she said. Rebecca and her family travelled from Youghal in Cork to attend the rally.

Harry’s father, Ian Ryan, added: ”Have a look at your own child, what price would you put on your own child’s life? You can’t put a price on child’s life.”

Cost and clinical trials

In a statement issued on Monday 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 noted “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.

IMG_20190228_114841 Harry Bulman with his parents Rebecca and Ian and other relatives outside Leinster House. Source: Órla Ryan

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 was “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 from the company noted.

IMG_20190228_113422 The rally outside Leinster House. Source: Órla Ryan

It added 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”.

The issue was raised during Leaders’ Questions today, with Fianna Fáil’s Dara Calleary telling Tánaiste Simon Coveney that the figures he quoted in the Dáil were out-of-date and not accurate. 

The denying of this medication to patients is a “death sentence” to some, said Calleary, who called on Coveney to directly intervene. 

The Tánaiste said the matter is a priority for the government, and encouraged both sides to come together to reach a deal. 

Sinn Féin’s Caoimhghín Ó Caoláin said TDs had tears in their eyes listening to those with SMA outside the gates of Leinster House today. 

Today is International Rare Diseases Day, he noted, adding that it should have been a day of celebration.

Ó Caoláin said the figures Coveney is relying on are not correct, having met with Biogen himself. He said not finding the full facts makes the Tánaiste complicit in this failure, adding that “shirking responsibility” is no longer acceptable.

Coveney said he would speak directly with Health Minister Simon Harris and Taoiseach Leo Varadkar on the matter. 

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

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Órla Ryan

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