Sinn Féin leader Mary Lou McDonald has written to Taoiseach Micheál Martin asking him to intervene after a HSE group recommended against funding a drug for the rare disease Friedreich’s ataxia
In the letter, she urged the Taoiseach to “use your office to ensure that patients and families are heard, and that every possible means of securing access to this treatment is pursued without further delay”
McDonald’s letter comes after a majority of Fianna Fáil’s parliamentary party called on the HSE to reconsider its refusal to fund the drug, omaveloxolone, branded as Skyclarys
Friedreich’s ataxia causes progressive damage to the nervous system. Individuals with the condition have a shorter-than-average life expectancy. About 200 people have the condition in Ireland
In February 2023, Skyclarys was approved in the United States for the treatment of the disease. The EU followed suit 12 months later. 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
Last December, the National Centre for Pharmacoeconomics (NCPE) recommended that the drug not be considered for reimbursement by the HSE as it would cost about €280,000 per patient annually
The HSE Drugs Group upheld the NCPE recommendation, stating there were “limitations and uncertainties associated with the available clinical efficacy data”
The recommendation will now go to a meeting of the HSE senior management team on August 25th for a final decision
In her letter to the Taoiseach, McDonald wrote that there was “deep distress among patients and their families”
“It is particularly difficult to understand given 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.”
[ Father of boy with Friedreich’s ataxia devastated as HSE recommends against drug fundingOpens in new window ]
She said “time matters” for people living with Friedreich’s ataxia, “a progressive and debilitating condition”
The Sinn Féin leader added that patients who had campaigned for access to this treatment for the past two years “are continuing to lose ability while they wait”
She wrote: “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 asked Martin “to meet urgently with people living with Friedreich’s ataxia and their families, together with the relevant HSE officials, so that they can hear directly the position of the HSE and have their concerns properly heard”
McDonald also urged the Taoiseach to ensure that a scheduled August 25th meeting of HSE senior management was brought forward
She wrote: “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.”
The HSE said the current price of Skyclarys was “substantially above the level typically regarded as cost-effective in Ireland having regard to the limited efficacy of the drug”
On Thursday, a large group of Fianna Fáil politicians called on the HSE to reconsider its decision. In a letter sent to the Taoiseach, Tánaiste Simon Harris, Minister for Health Jennifer Carroll-MacNeill, the Independent Minister of State Seán Canney and HSE chief executive Anne O’Connor, the group rejected the HSE stance
The signatories included the Minister of State Catherine Ardagh, TDs, Senators and three of Fianna Fáil’s four MEPs, Cynthia Ní Mhurchú, Barry Cowen and Billy Kelleher
[ More than 60 Fianna Fáil politicians oppose HSE refusal to fund Friedreich’s ataxia drugOpens in new window ]
The letter, seen by The Irish Times, states that seven other European countries have approved the treatment for reimbursement
The Fianna Fáil group accused the NCPE and the HSE of not giving patients with this “rare and progressive condition a fair hearing within a reasonable time frame”
They added: “We do not believe this outcome reflects what the public or the Government wants for people living with rare diseases in Ireland . . . We are asking you to use whatever powers are available to you to ensure that all necessary reFriedreich’s ataxia.”
A HSE statement said that its drugs group “carefully considered” Skyclarys “and tried to balance the potential impact for patients against the wider health needs of the population”
The statement said that “final decisions” are made by the HSE senior management team in accordance with 2013 legislation on the pricing and supply of medical goods “taking account of patient need, clinical effectiveness, safety, value for money, budget impact and the re
