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'He’s still walking but he’s struggling': Parents fight for access to 'life-changing' drug

The families of two boys are fighting for access to Translarna, a drug that could help delay the effects of a rare muscle-wasting condition.
Sep 7th 2017, 6:00 AM 10,354 7

20170813_113946 Lewis Source: Padraig Walsh

THE FAMILIES OF two young boys are fighting for access to an expensive drug that could help delay the effects of a rare muscle-wasting condition.

The HSE recently confirmed it would not be funding Translarna (also known as ataluren) as it was not deemed cost-effective.

Muscular Dystrophy Ireland (MDI) says it’s only aware of five boys in Ireland with nonsense mutation Duchenne muscular dystrophy (nmDMD). Two of these boys are over five years of age and therefore eligible for Translarna.

The remaining three boys may become eligible for the drug if they are still able to walk at the age of five. Most boys with the condition start to lose muscle power at the age of six, with the majority unable to walk by age 12. The average life expectancy for people with the condition is mid-20s.

In 2016, the National Centre for Pharmacoeconomics (NCPE), which conducts the health technology assessment of pharmaceutical products for the HSE, did not to give the green light to Translarna. At the time, it said the price would be over €400,000 per child per year. It’s understood a lower price tag was offered to the HSE following negotiations. has been following the story of Lewis Harte, a five-year-old boy from Castlebar in Co Mayo who has nmDMD, since last year.

His mother Ann Marie Harte Walsh told us she’s fighting to overturn the “heartbreaking” decision. A number of TDs are supporting her case and she hopes to attend Leinster House to highlight her situation in the near future.

Translarna is available in 22 European countries including Northern Ireland and the UK.

family The Harte Walsh family Source: Padraig Walsh

Ann Marie says she and her husband Padraig Walsh are considering leaving the country to try to gain access to the drug, but want to explore every option here first.

If we have to leave Ireland to get access to the medication, at least I can go knowing I did everything I could to try to get access to it here. There’s no way I’m leaving him not getting it.

Ann Marie says she knows of boys in England and elsewhere who are doing very well since getting access to the drug. She says it wouldn’t be easy to move but she’ll do it if she has to.

“My daughter is doing her Leaving Cert, me and my husband have jobs here, we have loans, our family is here, our lives are here. I don’t even have the money to uproot and go somewhere but I’ll do anything I can.”

Richard Lodge, Chief Executive Officer of MDI, says the process has been “disappointing and frustrating for the families involved”.

“Since 2014, over 400 children in 22 European countries have been receiving this treatment. That means that 80% of eligible children are now receiving the drug and this leaves Ireland as one of the last remaining countries to facilitate treatment.

It would appear that Ireland’s decision not to reimburse for Translarna is at odds with regulators, health authorities and experts across Europe. Would this imply that the European Medicines Agency and the 22 other European Countries have all gotten it wrong? Or is it just that we are behind the curve?

Lodge admits Translarna is expensive, but says paying for the medication would likely be cheaper than paying for 24-7 care for the few children affected by nmDMD once they can no longer walk. He says the medication would also allow them to live a normal life for much longer than they would otherwise – hopefully allowing them to finish school and reach other milestones.

Needing a wheelchair

Ann Marie says she’s aware Translarna is very expensive, but thinks any parent would fight for access to the medication if it was their child’s health at stake.

“The medication could stop him going into a wheelchair – the way things are now we have maybe two years, tops, before that happens.”

As well as muscle wasting, the condition also leads to other serious health issues, such as heart and lung problems.

20170906_171745 Ann Marie and Lewis Source: Padraig Walsh

Ann Marie says Lewis “really loves school” and is “very intelligent” but struggles with things other children his age find easy.

He’s still walking but he’s struggling. He can still move [his hands] but, say for catching a ball, that’s very difficult for him. He can’t run and he can’t jump, he can’t kick a ball, he can’t play sports.

“If he got the medication today, it could help him remain at the same level he is now for seven to 10 years, maybe even longer.”

Lewis has been on steroids – four tablets a day – for almost two years.

MDI is still in talks with PTC Therapeutics, the company which makes Translarna, to see if anything can be done to make it available to the two boys in Ireland who could benefit from it.

Not cost-effective 

In the letter the HSE sent to parents on 31 July informing them it would not be funding Translarna, the executive said it had reached the decision “following an intensive, transparent and detailed statutory review process over the past 18 months”.

The HSE said the decision was “challenging” but it “did not consider the evidence for the clinical benefit of [Translarna] to be sufficiently strong in the context of the proposed cost and budget impact”.

In the letter, seen by, the HSE’s Primary Care National Director John Hennessy said: “I know this is a very difficult time for patients with duchenne muscular dystrophy, but for the HSE affordability of new medicines is a real challenge due to the extraordinary prices that are increasingly being proposed by drug manufacturers.

The HSE must have regard to its obligations to the wider 4.7 million population and needs to maintain the full range of health services to all of the other patient groups within the finite resources at its disposal.

When asked for a comment about the situation, a HSE spokesperson told “The HSE Leadership team has carefully considered the issues in relation to the reimbursement of Translarna. The HSE is not in a position to fund this medicine on the basis of the application submitted by PTC Therapeutics.”

Today is World Duchenne Awareness Day, more details about it can be found here. More information about DMD can be read on MDI’s website.

Read: Two boys fighting for access to drug that could help them keep walking

Read: ‘In danger of snatching defeat from the jaws of victory’: How tension built ahead of Orkambi deal

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