DUBLIN, Ireland: Taoiseach Micheál Martin has been urged to step in and ensure there is "full transparency" about a recommendation to refuse public funding for a drug used to treat Friedreich's Ataxia, a rare and progressive degenerative disease.

Sinn Féin leader Mary Lou McDonald said the HSE Drugs Group's decision not to recommend Skyclarys for reimbursement had caused serious concern and "deep distress among patients and their families."

Friedreich's Ataxia is a rare neuromuscular condition that gradually worsens over time. Around 200 people in Ireland are believed to be living with the disease.

Earlier this week, the HSE Drugs Group issued its recommendation against reimbursing Skyclarys. The group raised concerns about the drug's effectiveness, pointing to "limitations and uncertainties" in the available evidence. It also highlighted the treatment's current price as a concern.

The HSE is expected to make a final decision on whether to fund the drug later in August.

However, McDonald has now written to Taoiseach Micheál Martin and called on him to intervene.

She pointed out that Skyclarys has already been approved for use throughout the European Union since February 2024. She also said the treatment is publicly funded or otherwise available to patients in several European countries.

McDonald said she wanted the Taoiseach to ensure that the assessment process was fully open and clear. "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."

She said patients and their families had a right to understand how the decision had been made. She said they should be told what scientific evidence had been examined, which factors had influenced the recommendation and, most importantly, what choices were still available to them.

McDonald also called on Martin to hold an "urgent" meeting with young people living with Friedreich's Ataxia and their families.

She also asked for the HSE meeting at which the final decision on Skyclarys is due to be brought forward.

The Sinn Féin leader said all possible ways of giving patients access to the drug should now be considered without delay. She also said Ireland should examine how other European countries had made Skyclarys available and consider whether those arrangements could provide useful options.

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."

McDonald warned that the issue should not be allowed to remain unresolved because of further administrative delays. She said patients and their families had already been waiting for two years for access to the treatment.

She said, "This matter cannot be allowed to drift through further administrative delay. These patients and their families have already waited for two years."

In a statement to RTÉ earlier this week, the HSE explained the reasoning behind the Drugs Group's recommendation.

The HSE said its Drugs Group had reviewed an assessment prepared by the National Center for Pharmacoeconomics. According to the HSE, that assessment found some evidence that Skyclarys could help slow the progression of Friedreich's Ataxia.

However, the assessment also identified weaknesses and uncertainties in the available clinical evidence on how effective the drug is.

The HSE said the Drugs Group had also considered the cost of the treatment. It said the group concluded that the current price of Skyclarys was substantially higher than the level normally considered cost-effective in Ireland, particularly given the limited evidence of the drug's effectiveness.