Treatment for Friedreich's ataxia should not be funded by HSE, drugs group recommends

THE HSE DRUGS Group has recommended that a drug to treat the rare disease Friedreich’s ataxia should not be funded by the health service at this time. 

The group met today to consider the application for the reimbursement of Skyclarys – reimbursement meaning it will be covered for patients who have medical cards or who are covered by another state scheme. 

The recommendation will now go to a meeting of the HSE senior management team on 25 August, where a final decision will be made.  

Friedreich’s ataxia is a progressive, neuromuscular disease that mainly affects the nervous system and the heart. It is a hereditary disease caused by a gene mutation.

While there is no cure for the disease, Skyclarys has been found to slow the disease progression and improve neurological function.

The treatment was approved by the European Medicines Agency in February 2024 and has been made available to patients in other European countries.

Around 200 people have Friedreich’s ataxia in Ireland, and a campaign has been underway to make the drug available by the HSE. 

In a statement, the HSE said that when considering any drug for reimbursement, the drugs group looks at the benefits the drug would bring patients, the clinical evidence available for the drug and the impact that reimbursing the drug would have on overall resources available to the health service. 

Advertisement

“Everyone involved in this process is acutely aware of the impact their decisions have on the lives of patients and their families,” the statement said.

It said the HSE Drugs Group examined submissions and data from Friedreich’s ataxia patients, their doctors and advocacy groups and discussed “the profound and devastating impact” that the disease has on quality of life and life expectancy.

The group considered input from the Rare Diseases Technology Review Committee (RDTRC), which was positive about the impact of the drug on the experience of patients and highlighted the substantial unmet need of those suffering from the disease. 

It also considered an assessment from the National Centre for Pharmacoeconomics, published in December last year, which identified limitations and uncertainties associated with the available clinical data.

It concluded that the drug would cost approximately €280,000 per patient per year, which equates to around €130 million over five years. 

“Based on all the evidence considered by it today, the Drugs Group has decided not to recommend this drug for reimbursement at this time,” the HSE statement concluded. 

Sinn Féin leader Mary Lou McDonald has described the decision as “devastating” for those living with Friedreich’s ataxia and called for “full transparency” from the HSE and the Minister for Health on how this decision was reached.

“We must also see an immediate and drastic improvement in the standard of communication with the disease,” McDonald said.

“It is wrong that engagement with patients and their families ranges from radio silence to drip feeding from the government and the HSE. It is an unacceptable way to treat people deteriorating from a debilitating disease, people who are terrified.”