The vast majority of non-ministerial members of the Fianna Fáil parliamentary party have signed a letter raising "serious concerns" about a HSE group recommendation to refuse funding for Skyclarys, a drug used to treat the degenerative disease Friedreich's Ataxia.
The letter, signed by 48 TDs, senators, and MEPs - including one Minister of State - is addressed to the HSE leadership and states that Ireland’s system for approving funding for new drugs, known as reimbursement, is "broken and not fit for purpose".
There are 53 members of the Fianna Fáil parliamentary party who are not a minister or minister of state, and 71 in total.
Minister of State at the Department of Justice, Home Affairs and Migration Catherine Ardagh is the only signatory with a portfolio.
The letter was also sent to Taoiseach and Fianna Fáil leader Micheál Martin, Tánaiste and Fine Gael leader Simon Harris, and Minister for Health and Fine Gael TD Jennifer Carroll MacNeill.
It calls on them to use whatever powers are available to ensure that all available resources and mechanisms are available to families affected by the disease.
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Opposition parties Sinn Féin and Aontú have joined Fianna Fail backbenchers by writing to the Taoiseach to intervene and fund the Skyclarys drug for Friedreich's Ataxia.
In a letter sent yesterday morning, Sinn Féin leader Mary Lou McDonald repeated her party's previous call for the move to take place, while in a statement this afternoon Aontú leader Peadar Tóibín also demanded action.
In her letter, Ms McDonald called on the Taoiseach to give the matter his "urgent attention" and to ensure that patients and families are heard.
"This matter cannot be allowed to drift through further administrative delay," she said.
Meanwhile, Mr Tóibín said that the drug "must be reimbursed now for sufferers in desperate need".
Friedreich’s Ataxia is a rare progressive neuromuscular condition affecting around 200 people in Ireland.
Skyclarys (Omaveloxolone) is the only approved treatment, and has been shown to slow the progression of symptoms by up to 50% in some patients.
Treatment at the current proposed fee following negotiations with the drug manufacturer, Biogen, is estimated to cost in the region of €280,000 per patient per year. That results in a projected cost to the State of more than €130 million over five years.
The National Centre for Pharmacoeconomics (NCPE), which assesses the value for money of new medicines, recommended last year that Skyclarys not be reimbursed unless the price of the drug is significantly reduced.
Last month, the HSE Drugs Group considered that assessment but postponed making a recommendation, referring the matter to the Rare Diseases Technology Review Committee (RDTRC) for additional input.
The RDTRC is made up of clinicians and patient advocates and is advisory in nature. It does not make recommendations on the basis of cost or budgetary impact.
The RDTRC report, seen by RTÉ's Prime Time, was positive on the benefits of Skyclarys and strongly supportive of the drug being made routinely available in Ireland.
However, on Tuesday, the HSE Drugs Group reconvened to consider the report and again recommended that Skyclarys not be recommended for reimbursement.
In a statement to RTÉ News, the HSE said: "The group also 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.
"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."
A decision on accepting or rejecting the recommendation to not reimburse Skyclarys is expected to be made by HSE leadership on 25 August.
Speaking on RTÉ’s Prime Time last night, Emily Felix, a 28-year-old trainee solicitor who was diagnosed with Friedreich’s Ataxia at the age of 12, said that she was "shocked and devastated" by the HSE Drugs Group recommendation, particularly in light of the "overwhelmingly positive" report by the RDTRC.
Watch: Emily Felix speaks to Fran McNulty on Prime Time
Ms Felix also said that the Drugs Group recommendation is purely advisory and that the leadership team has the opportunity to disregard it, adding that they have been repeatedly told by the Taoiseach, Tánaiste and Minister for Health that the reimbursement process is science led.
"The cost needs to be at the bottom of the list of criteria. We are more than a cost figure on a budget. We deserve much more. We want a future. We want to live," Ms Felix said.
The letter signed by Fianna Fáil parliamentary party members states that "the NCPE and Drugs Group process was never designed to give patients with rare and progressive conditions a fair hearing within a reasonable timeframe, and this case is further proof of that.
"We do not believe this outcome reflects what the public or the Government wants for people living with rare diseases in Ireland."
It adds that "at least seven other EU countries... have already approved this drug for reimbursement".
The letter also mentions a contribution provided to a previous parliamentary party meeting given by Cork man Craig Coady, whose two sons Paudie and Rory were diagnosed with Freidreich’s Ataxia. Rory died from the condition just over a year ago at the age of 13.
"Craig sat before us and pleaded for this drug to be reimbursed, he told us that Paudie was 'all he had left'. Craig's words have stayed with us ever since."
Additional reporting Fiachra Ó Cionnaith
