‘If it was a very common condition, would the government pay for this or would they let everyone wither away and die?’

BY EIMEAR O'DWYER

These are the words of Emma O’Shea from Kealkill, appealing to the government to provide a life-changing drug that could significantly slow the progression of her rare, life-limiting Friedreich’s Ataxia (FA).

FA is a genetic neurological disease that affects mobility, speech, coordination and heart health.

Emma was nine when she was diagnosed with FA but did not become symptomatic until her early teens, when her condition began to accelerate.

Sadly, her older brother Timmy passed away from FA in 2016 at the age of 29.

‘He was 13 years older than me and he was diagnosed when he was ten,’ said Emma.

‘Timmy developed Friedrich’s symptoms very early on, very quickly. Timmy died from heart failure. Growing up, I could see Timmy suffering and ultimately dying. I was 16 when he died so I was fully aware of what was happening.’

Emma experienced more serious symptoms in her late teens.

‘I found walking very difficult. I wouldn’t be able to walk far and then I’d have to sit down. And then when I was 20 I started using one of Timmy’s old wheelchairs, just temporarily to go long distances.’

By age 23 Emma began to use a wheelchair for shorter distances and to get around her home.

She has spoken to people from other countries who have been on the drug Skyclarys who say it has had a profoundly positive impact on their lives.

‘I spoke to a boy who has issues with coordination, much the same as me. When he lifted his hand, it would shake; he couldn’t point it straight or keep it steady. He says that since being on the drug, he has noticed his coordination has gotten so much better.’

A three-year test study of the drug showed a 55% slower progression of symptoms in patients who took Skyclarys, in contrast to those who took a placebo. In 2024, the drug was approved for all 27 EU member states by the European Medicines Agency but only 12 EU countries have reimbursed the drug so far.

Biogen, which produces Skyclarys, has been trying since 2024 to get permission to sell the drug in Ireland, which has the highest number of people with FA per capita in the EU.

However, due to legislation on the supply and pricing of medicines, the cost of the drug is causing barriers to its implementation.

‘I understand it is a huge cost, like it’s only 200 people [in Ireland with FA],’ said Emma.

‘But if it was you and your next-door neighbour and then someone you went to college with and someone you work with. If it was a very common condition, would the government pay for this or would they let everyone wither away and die? 200 lives at €280,000 a year.’

Since early 2024, Emma’s neurologist has organised incremental supports for her, including occupational therapy and speech and language therapy.

Emma believes the medication would save government spending on care for patients with FA as it would slow the progression of their symptoms.

Ironically, the medication is currently being produced in Cork and shipped out to the countries that reimburse it.

In the last eight or nine months, Emma says her coordination has deteriorated, and she finds it difficult to grasp the wheels on her wheelchair when they are moving. ‘I do rely on people to help me push the wheelchair, which is very annoying – I can’t go anywhere myself.’

Emma said her hopes that Skyclarys would be reimbursed has dwindled. ‘I suppose to be honest with you, like, I have no other choice but to put up with it. I know what’s coming. I have seen it happening.’

Many people with FA have taken their campaign to the Dáil and set up petitions. Last week, the HSE Drugs Group met to consider the pricing and reimbursement of medicines, but Skyclarys was not on the agenda.

Emma’s case in particular was raised by Independent Ireland leader Michael Collins TD in the Dáil and by Senator Noel O’Donovan (FG) in the Seanad.

Taoiseach Michéal Martin said in the Dáil that Skyclarys will be discussed at their next meeting in July.

Emma says this is just ‘another unacceptable delay.’

To sign a petition to appeal for the reimbursement of Skyclarys follow this link https://c.org/v8QyqNxphF.