Sinn Féin leader urges Taoiseach to intervene after HSE refusal to fund drug for rare disease

Sinn Féin leader Mary Lou McDonald has written to Taoiseach Micheál Martin asking him to intervene after a refusal by the HSE to fund a drug for the rare disease Friedreich’s ataxia.In her 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.READ MOREIn 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”.In her letter to the Taoiseach, McDonald wrote that the decision not to fund the drug in the State had “caused 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”. She said patients who had campaigned for access to this treatment for the past two years “are continuing to lose ability while they wait.”McDonald 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 group of 48 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 resources and mechanisms are made available to families affected by Friedreich’s ataxia.”