Taoiseach urged to intervene over funding for rare neuromuscular condition drug

By Jonathan McCambridge, Press Association

Taoiseach Micheál Martin has been urged to intervene to ensure there is “full transparency” around a recommendation to refuse funding for a drug used to treat the degenerative disease Friedreich’s Ataxia.

Sinn Féin leader Mary Lou McDonald said the decision of the HSE Drugs Group not to recommend Skyclarys for reimbursement had caused “deep distress among patients and their families”.

Friedreich’s Ataxia is a rare progressive neuromuscular condition affecting around 200 people in Ireland.

Earlier this week, the HSE Drugs Group made its recommendation, raising concerns about “limitations and uncertainties” associated with the efficacy of the drug and its current price.

A final decision on the drug is expected to be made by the HSE later in August.

But Ms McDonald has written to the Taoiseach on the issue.

She said that Skyclarys “has been approved for use across the European Union since February 2024 and is now publicly funded or otherwise accessible in a number of European countries”.

She said: “I am asking you, as Taoiseach, to intervene to ensure that there is full transparency around the HSE assessment, the findings of the Drugs Group and the basis upon which this decision was reached.

“Patients and their families are entitled to know what evidence was considered, what factors determined the outcome and, crucially, what options remain open to them.”

She also asked Mr Martin to meet “urgently” with young people with Friedreich’s Ataxia and their families.

This matter cannot be allowed to drift through further administrative delay

Ms McDonald also asked for the HSE meeting to make a final decision on the drug to be brought forward.

She said: “Every available avenue for providing access to Skyclarys should now be examined urgently, including what can be learned from the arrangements already in place in other European countries.

“This matter cannot be allowed to drift through further administrative delay.

“These patients and their families have already waited for two years.

“They deserve transparency, meaningful engagement and a clear pathway forward.”

In a statement to RTÉ earlier this week, the HSE said its Drugs Group had “considered an assessment from the National Centre for Pharmacoeconomics which found that while there is some evidence that this drug may slow disease progression in Friedreich’s Ataxia, that there remain limitations and uncertainties associated with the available clinical efficacy data”.

It said: “They 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.”