Vincent P Martin (Green Party)
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The Minister of State, Deputy Chambers, is welcome to Seanad Éireann. Friedreich's ataxia is a rare, progressive, life-limiting neuromuscular condition. This disease was first recognised in 1863, and after many decades of research, the first and only treatment called omaveloxolone has recently been discovered. It can slow progression of this illness by 50%. I commend the groundbreaking work of Reata Pharmaceuticals, Texas, on delivering this cutting-edge medication.In the US where it has been approved it gives a precious lifeline to those suffering from Friedreich's ataxia, but Ireland is sadly a different story. The Irish Pharmaceutical Healthcare Association said last month that Ireland is one of the slowest in Europe to reimburse and make available new approved innovative medicines. I ask the Minister of State to assure me that the Government will give this medication to those who urgently needed without one day's or one hour's unnecessary delay.

When, as is anticipated, the EU approves this drug, based on verified track records, it is expected that the new drug will be readily available within weeks in France and Germany, yet Irish patients fear they will be facing a potential wait of up to two years. We have seen this before. In the past we have seen the Government's initial refusal to fund Orkambi, a vital intervention for suffers of cystic fibrosis. The late Vicky Phelan had to persist and plead for pembrolizumab, a life-saving cervical cancer treatment, to be made available. This was eventually done in exceptional circumstances without EU approval showing that where there is a will there is a way.

A Sallins native Niamh Ní Hoireabhaird, her brother and persons living in my home county in Maynooth and Newbridge are some of the 200 people in Ireland suffering with this rare disease. Niamh Ní Hoireabhaird is clearly a courageous, resilient warrior who wants to speak up for these 200 people. In a recent letter to me she stated:

Dear Senator Martin,

I was diagnosed with Friedreich's ataxia when I was 13, I am now 26 and have been progressively losing my mobility and my independence. A decade ago, I was able to walk without assistance but now I'm a full-time wheelchair-user. I often wonder where I'll be ten years from now.

When this drug was approved in the US, I initially felt hopeful for the future in a way that I have rarely felt since my diagnosis. But unfortunately Senator, that hope quickly faded when I remembered Ireland's atrocious track record for approving and funding rare disease treatments. I'm worried I will have to spend years of my already-limited life campaigning for funding of this drug.

I have constantly been told by officials about the cost of these medicines. What about the cost of a life lived dependent on services of the state? What about the cost of the years we will have to spend pressuring the government to do the right thing.

Let me be clear: This issue Senator, goes beyond this drug. We need a proper and equitable strategy for rare disease treatments. As a member of the rare disease community, I have little faith in this country's ability to care for me, my brother and other Friedreich's ataxia patients. Can you blame me for that? Senator, Please help.

Jack Chambers (Dublin West, Fianna Fail)
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I thank Senator Martin for raising this issue which I am taking behalf Minister for Health, Deputy Stephen Donnelly. The Department of Health has informed me that Friedreich's ataxia is a rare disease affecting fewer than 200 people in Ireland. The condition involves progressive neuromuscular degeneration leading patients to have difficulty with things such as walking, speech and co-ordination. Unfortunately, the progression of the disease can shorten patients' life expectancy and severely impact on their quality of life. There is currently no approved treatment for Friedreich's ataxia in Europe. A new medicine omaveloxolone has recently been approved by the Food and Drug Administration, FDA, in the United States for this condition, as the Senator mentioned.

The European Medicines Agency, EMA, is a centralised agency of the European Union responsible for the scientific evaluation, supervision and safety monitoring of medicines in the EU. The EMA plays integral role in the authorisation of medicines in the EU. The company marketing this treatment has now applied for marketing authorisation from the EMA and its evaluation commenced on 28 December last year. In Ireland the Health Service Executive has statutory responsibility for decisions on pricing and reimbursement of medicines and medical items in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013.In line with the Act and the national framework agreed with industry, a company must submit an application to the HSE to have a new medicine added to the formal reimbursement list. Reimbursement is for licensed indications that have been granted market authorisation by the European Medicines Agency, EMA, or the Health Products Regulatory Authority. In making a relevant reimbursement decision, the HSE is required under the Act to have regard to a number of criteria, including efficacy, the health needs of the public, cost-effectiveness, and potential or actual budget impact.

HSE decisions on which medicines are reimbursed by the taxpayer are made on objective scientific and economic grounds on the advice of the National Centre for Pharmacoeconomics. As Omaveloxolone has not yet been granted marketing authorisation by the EMA, an application for pricing and reimbursement cannot be made to the HSE. If marketing authorisation is granted and the company chooses to apply, the HSE will give fair consideration to the application with consideration of the criteria detailed in the 2013 Act. Omevaloxolone has been designated as an orphan drug by the EMA. This designation is given to substances for treating, preventing, or diagnosing rare and serious conditions.

The Government is committed to making new medicines available to patients in Ireland, with almost €100 million of funding dedicated to new drugs over the past three budgets. Budget 2023 allocated €18 million of dedicated funding for new drugs. As of the end of March, 15 new drugs have been approved, five of which were orphan drugs. Budget 2022 allocated €30 million for new drugs. This enabled approval for 60 drugs,16 of which were orphan drugs. Budget 2021 allocated €50 million for new drugs; 52 new medicines were approved from this funding, including 18 new orphan drugs.

Vincent P Martin (Green Party)
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I thank the Minister of State. Are we at the ready? Will things change? Sufferers want assurances. We have a track record of being in the Vauxhall non-league conference in terms of speed, when it comes to approving and funding such drugs. What is stopping Ireland being in the premier division? Then again, should we not all be equal in the context of the EU, as we are equal members of that family? Perhaps, there is a need for a fundamental, global and societal response, which has equity at its core. These sufferers want assurances. Is the footdragging track record of delay in Ireland caused by incompetence, lack of resources or something else? Does Ireland jump in the queue only when it has already formed? The only certainty is we will always get a place at the back of the queue, but if we got up a little earlier we might be near the front of it.

The truth is sickness is sickness and pain is pain but there should be no queues when it comes to a person's wellbeing and care. It should not be about the haves and have-nots. The wait for the roll-out of this drug is near unbearable. I ask and implore the Minister of State to take pre-emptive action and ensure, this time, that things are done in the most time efficient and fair way. Time is of the absolute essence for these sufferers.

Jack Chambers (Dublin West, Fianna Fail)
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I thank the Senator for his statement and feedback and, in particular, the feedback from many of the people he engaged with who are seeking progress relating to this orphan drug, which has made progress in the United States. I will reflect that feedback to the Minister for Health, Deputy Stephen Donnelly. As well as dedicated funding for new medicines, the Minister plans to develop a revised national rare disease plan. Initial work for the new plan has begun. This plan will be a key step in progressing an improved overall service for individuals living with a rare disease. The Minister and officials in his Department will monitor the progress of Omaveloxolone with interest. As I said, decisions on approval or reimbursement will be then the statutory responsibility of the HSE.

I will, however, fully reflect what the Senator said back to the Minister, in addition to the feedback from various people in Sallins and elsewhere, to whom this matter is of particular interest. I appreciate him raising it.