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Putting a price on a child’s future

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‘INCREDIBLY TOUGH SENTENCE’ Ann Marie Walsh-Harte and her son Lewis (5) at their home in Castlebar.

Mayo family face ‘incredibly tough sentence’ after HSE drug funding refusal

Cory Kilbane

A Castlebar family have been devastated by the HSE’s refusal to fund a new medicine for children with Duchenne muscular dystrophy.
Ann Marie Walsh-Harte and her husband, Pádraig, were eagerly awaiting a decision on whether the drug, Translarna, would be approved for HSE funding. Their five-year-old son, Lewis, (pictured)  suffers from the life-limiting condition, and they believe the new treatment could add an extra seven to ten years to his life. When the news broke on Tuesday morning last that Translarna had not been approved, they were left in a state of shock and disbelief.
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy characterised by progressive muscle degeneration and weakness. Typically, muscle loss starts in the lower body followed by the arms, soon affecting the ability to stand up. Most affected children are unable to walk by the age of 12.
The disease primarily affects boys, but in rare cases it can affect girls. The average life expectancy is mid to late 20s; however, it is thought that with excellent care, some sufferers may live well into their 30s or 40s.

Torture
Lewis has been suffering from DMD since birth but was only diagnosed with the terminal disease at the age of two. For Ann Marie, it has been a long wait for the Translarna decision, which was itself an appeal on an earlier appeal against a previous refusal, and the outcome has left her heartbroken.  
“It’s been torture for us, waiting, hanging on to see where our fate lies,” Ann Marie Walsh-Harte told The Mayo News last week.
“The biggest thing for me is the sheer devastation. For them to say no, so coldly, to something so important, is just ridiculous.”
The HSE released a statement to Muscular Dystrophy Ireland last Thursday, stating why it  decided not to fund Translarna: “The decision not to reimburse the drug was made following an intensive, transparent and detailed statutory review process over the past 18 months … These are challenging decisions to make but it is important to understand that the HSE Drugs Group who reviewed the effectiveness of the drugs did not consider the evidence for the clinical benefit of Ataluren (Translarna) to be sufficiently strong in the context of the proposed cost and budget.”
The statement also said that the HSE is aware that the decision would upset patients and their families, as well as the clinicians who support patients suffering from the illness.

Battling alone
Reacting to the HSE’s statement, Ann Marie Walsh-Harte said that she ‘can’t understand what more data is needed to prove that the drug is needed in this country’.
“What evidence is missing that they need?” she asked. “Twenty-two other countries can see that this drug is needed, so why can’t we?
“I feel like I am fighting this battle alone; there’s been little said by anyone. I just honestly do not get it. I feel like I am going around in a circle and going nowhere,” she added.
Commenting on Lewis’s plight, Senator Rose Conway-Walsh said that it ‘highlighted the human consequences that arise from the failure to secure reasonably priced treatment’, pointing out that Translarna could extend Lewis’s ability to walk ‘by many years’.
“[Last week’s] decision was a response to an appeal of [the HSE’s] decision back in April 2016. Between April 2016 and Tuesday, we have been waiting, and to get the answer then that the overall outcome is that they are not going to give it, is devastating. It was a huge amount of time to be waiting,” she explained.
Lewis’s family are not in a position to launch another appeal. “We haven’t got another two years to start this process all over again,” said Ann Marie. “Lewis will be seven by then, and his situation will have deteriorated at that stage. We have already wasted eight months.”

Sentence
“The drug can extend patients’ ability to walk by seven to ten years. Without it, Lewis faces an incredibly tough sentence. He will be wheelchair bound in just two years, and from there, his situation will deteriorate,” Ann Marie explained.
“[With DMD sufferers,] the legs are the first to go, the walk … then when they go into a wheelchair you’re looking at their hands, and then everything starts to fail. Their heart, their lungs, their breathing, swallowing, everything.
“It’s terminal. It’s usually heart failure that kills them in the end. It’s a truly devastating disease.”

 

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