Campaigners advocating for those affected by Friedreich’s Ataxia have again appealed to the Health Service Executive (HSE) to approve the Skyclarys treatment ahead of a final decision next week.
Friedreich’s Ataxia is a rare progressive neuromuscular condition affecting around 200 people in Ireland.
Speaking after a meeting in Dublin, 31-year-old Jessie Abbey called on the HSE to approve the drug.
“I want to live. I have everything to live for. I want to see my little girl grow up.
“I can’t even believe that I’m doing this, I am a very private person and having to go out publicly and say I’m vulnerable is very upsetting.
Jessie Abbey said that the future looks uncertain
“Three years ago, I could walk, I’m now in a wheelchair, what’s the next three years going to look like?
“I need this drug, I want to live, I love my little girl.”
Earlier this month, the HSE Drugs Group recommended that Skyclarys should not be covered by the HSE.
A meeting of HSE senior management team will make a final decision on 25 August.
Shanice O’Reilly who also has Friedreich’s Ataxia appealed to the HSE.
Shanice O’Reilly said that there will be disappointment if the Skyclarys treatment is not approved
“They need to give us a chance. I know a few people in America who take the drug and they are all doing so well.
“If it doesn’t go ahead, there will be a lot of disappointment.”
Isabella Travers was diagnosed when she was 12.
“When I was first diagnosed, I had problems with my balance but by the time I was 16, I was a full time wheelchair user.
“It’s so hard, I don’t understand how they don’t think that our lives are worth the money.”
19-year-old Isabella Travers has been using a wheelchair for the last three years
Several opposition parties have called for the approval of Skyclarys including Sinn Féin, Independent Ireland and Labour.
There is also huge support within the Fianna Fáil and Fine Gael parties.
The HSE has said that the drugs group considered an assessment from the National Centre for Pharmacoeconomics which found that while there is evidence that Skyclarys may slow disease progression in Friedreich’s Ataxia, that there remain limitations and uncertainties associated with the available clinical efficacy data.
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It also concluded that the current price was substantially above the level typically regarded as cost effective in Ireland having regard to the limited efficacy of the drug.
At the current listed price, Skyclarys would cost around €280,000 per patient per year, with a five-year budget impact of around €130m.
Posters on display at the public meeting on Friedreich’s Ataxia
The drug is made by Biogen which has said it continues to engage with the HSE.
Skyclarys has been approved by the European Medicines Agency and the Food and Drug Administration in the US.
Campaigners have organised a demonstration at Dublin’s Garden of Remembrance this Sunday to call for Skyclarys reimbursement.