HSE agrees to fund Skyclarys for patients with Friedreich’s Ataxia

Michael Bolton

The HSE’s Senior Management Team (SMT) has approved the reimbursement of Skyclarys for patients with Friedreich’s Ataxia.

The SMT took account of a substantially improved financial offer made in recent days by the company Biogen to the HSE arising from intensive discussions.

Biogen has moved substantially from its previous position, and the HSE expressed its appreciation of the flexible and constructive approach shown by the company.

The HSE's Drugs Group earlier this month recommended the drug not be reimbursed, over cost-effectiveness concerns.

Around 200 people in Ireland are living with Friedreich's Ataxia, and Skyclarys is the first treatment for the condition.

Campaigners say access is now urgent, with the treatment already available in several European countries.

Skyclarys is not a cure, but patients and advocates say it has the ability to slow the progression of Friedreich’s ataxia by up to 55 per cent.

It is a rare, inherited neurodegenerative disorder that affects the nervous system and can lead to severe movement and coordination issues, often beginning in childhood.

In a statement on Tuesday, the HSE said: "On balance, the SMT decided to approve reimbursement for Skyclarys based on the new commercial offering. It concluded that had it been asked to consider the same terms the HSE Drugs Group considered, it would have come to the same position as the Drugs Group and would not have proposed reimbursement.

"The SMT also considered the limited efficacy that has been shown for the drug, and the high cost that remains despite the improved financial offer.

"It considered some of the potential weaknesses in the clinical evidence and other relevant matters the Drugs Group considered under the Health (Pricing and Supply of Medical Goods) Act 2013."

In a statement, Taoiseach Micheál Martin said: "I welcome today’s decision of the HSE Senior Management Team to recommend the use of Skyclarys, and the Government will fully support this decision.

"I wish to pay tribute to all those living with Friedreich's Ataxia, and their families, who have endured so much. I thank them for their engagement and their advocacy.

"I understand fully their concerns and anxieties in relation to gaining access to this drug, the first treatment of its kind for this condition.

"It has been a very challenging period for the families, and I wish to acknowledge that.

"We appreciate the fact that this decision came on the back of substantive engagement between the HSE and the company Biogen over the past number of days and weeks.

"The outcome will result in strong engagement with Biogen over the coming years in respect of the treatment of Friedreich’s Ataxia.

"It was important that the statutory process for approving the drug was brought to its conclusion, and we welcome this decision, first and foremost for those who need access to Skyclarys.

"The Minister for Health and the Government will now work to support implementation of the HSE’s recommendation as quickly as possible.

Tánaiste Simon Harris said it has been an “extraordinarily difficult and anxious” time for patients, their families and communities.

Speaking before its approval, Harris said the process to approve a drug and make it available to patients in Ireland has to be “independent”.

“It is important that the process is robust, and it’s important because this is the drug today, and I say this with full empathy, full respect and with hope for clarity today,” he added.

“This is the drug today and, of course, there’ll be other patients and other new and innovative drugs, and there’ll be need for that, too, so having a robust process in place that gets the best outcome possible, to ensure as many of our citizens as possible can have access to innovative medicine is important.

“I think there’s a broader conversation to be had, not for today, but about we’re living through a time, which is a really positive thing, of lot of new innovations, lots of new drugs, lots of new medicines, perhaps at a scale we’ve never seen before.

“I think a broader conversation about Ireland and how the European Union, perhaps, funds that and interacts with that, is worth having.”