LAST UPDATE | 25 Aug

THE HSE HAS approved funding for a drug for patients suffering from Friedreich’s ataxia.

In a statement today, the HSE’s senior management team said it will fund the provision of a drug called Skyclarys, which has been shown to help alleviate the effects of the neuromuscular disease. 

In recent weeks, a HSE drug group recommended against funding the treatment, which costs €288,000 per patient per year.

It is manufactured by US biotechnology company Biogen.

The HSE said its senior management team “took account of a substantially improved financial offer made in recent days by the company Biogen to the HSE arising from intensive discussions”.

The HSE added that “Biogen has moved substantially from its previous position” and that this resulted in a “very different commercial offering to that which the HSE Drugs Group considered”.

“On balance the SMT decided to approve reimbursement for Skyclarys based on the new commercial offering,” said the HSE.

A spokesperson added that had the HSE 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.

Meanwhile, the HSE said its senior management team also considered the “limited efficacy that has been shown for the drug, and the high cost that remains despite the improved financial offer”.

However, it also agreed with the observations of the Drugs Group in relation to the “substantial unmet need of patients suffering from what is a devastating disease and the absence of any other medicines to treat it”. 

Engagements are ongoing with Biogen to finalise the administrative arrangements which will underpin reimbursement.

The HSE said it will “progress these engagements quickly, and understands that Biogen are also committed to finalising details at an early stage”.

‘Government will fully support this’

The issue had become a political one in recent weeks, with opposition politicians calling for the HSE to approve the funding of the drug.

Taoiseach Micheál Martin met families affected by the rare genetic disorder last week.

He welcomed today’s decision and said that the government “will fully support this”.

“I wish to pay tribute to all those living with Friedreich’s Ataxia, and their families, who have endured so much,” said Martin.

Martin added that he “understands fully their concerns and anxieties in relation to gaining access to this drug, the first treatment of its kind for this condition”.

He noted that it has been a “very challenging period for the families”.

“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,” said Martin.

He added: “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.”

Martin said Minister for Health Jennifer Carroll MacNeill and the government will “now work to support implementation of the HSE’s recommendation as quickly as possible”.

‘Stick to the rule of law’

In a statement MacNeill said that the “statutory process through which these decisions are made is complex” and that it is “important that politics and politicians allow the space for science to be the decision maker”.

“Ireland will continue to stick to science and to the rule of law,” said MacNeill.

“Simply put, these processes must be independent and led by experts.”

She also praised the “commercial team involved in the negotiations for their continued and active engagement to reach today’s decision”.

“I am grateful to the patients who have so carefully articulated their lived experiences,” said MacNeill.

“The HSE will now engage with the company to put in place the process by which patients can gain access to the drug,” she added.

“I have asked that this work commence immediately.”

‘No further delays’

Elsewhere, Sinn Féin leader Mary Lou McDonald remarked that “those living with Friedreich’s Ataxia have fought a two-year campaign to get access to a treatment that is available in many other countries.”

She said they “should never have had to battle so hard” and added that “it is critical that there are no further delays”.

“Every delay has had real consequences,” said McDonald.

“The government needs to ensure that there are no further delays and today’s decision is actioned quickly.”

Labour’s Marie Sherlock TD said that Skyclarys “has the potential to slow the progression of this debilitating condition and offer patients greater independence and a better quality of life”. 

“For the small number of people in Ireland living with Friedreich’s ataxia, access to this treatment will bring real hope,” she said. 

Independent Ireland leader Michael Collins meanwhile remarked that “common sense has finally prevailed” but added that “it should never have come to this”.

He paid tribute to the campaigners for their “relentless courage and absolute refusal to give up”,

“Their determination has brought this issue to the forefront, and they deserve enormous credit for refusing to be silenced,” said Collins.

“Your determination made it impossible for the political system to ignore this issue.”

-With additional reporting from Diarmuid Pepper