Joe Flaherty (Longford-Westmeath, Fianna Fail)
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I want to discuss a rare and uniquely Irish disease. Hereditary ATTR amyloidosis is a genetic condition. The disease was brought to my attention by a lady who lives in County Longford. There are several manifestations of this amyloidosis worldwide, but it includes a specific Irish variant that originates in north-west County Donegal. The condition is known locally as Donegal Amy. While my constituent's mother is from County Leitrim it shows the condition is not necessarily confined to the Donegal areas. Amyloidosis became know as Donegal Amy due to its prevalence along a 15 mile stretch of coastline between Carrigart and Burtonport in County Donegal. Its origin has been linked back to Conall Gulban, an Irish king and ancestor of the Cenéi Conaill, who founded the kingdom of Tír Chonaill, which we now know as Donegal. Until recent times the disease was aggressive and fatal. Innovative new treatments such as gene silencing are slowly becoming available. My constituent and her siblings inherited the condition from their late mother who sadly passed away in 1995 at the young age of 71 years. When my constituent's mother was diagnosed there was no treatment available. Moving to the present day my constituent and her siblings are now lucky that there is much greater awareness among medical professionals about this disease and there are also treatments available. There was a lifesaving breakthrough in 2021 when the HSE approved the use of a new treatment for hATTR. That drug is known as Onpattro, or patisiran, and it is supplied to them by the HSE through a managed access programme. The patients receive the infusions in their home, but the cost of receiving this medication for each patient is in the region of €400,000. It is estimated that 1% of the population of Donegal has the hereditary condition. The late mother of Sinn Féin's Martin McGuinness hailed from the Inishowen peninsula and she died as a result of the disease.

Many of the patients availing of the current treatment are aware of a newer version from the manufacturers of patisiran. This new drug is called vutrisiran, with the proprietary name Amvuttra. Both drugs are manufactured by the same company, but crucially the new drug is administered to patients on a three-monthly basis. That is four times annually as against the 17 administrations for the current drug. This would be more cost-effective for the HSE, but also less restrictive for patients and their families. However, patients were dealt a terrible blow in August last year when the National Centre for Pharmacoeconomics completed a rapid review of the new drug, with the recommendation that the drug should not be considered for reimbursement by the HSE. The Minister of State will also be aware that patisiran became available in Northern Ireland in 2019, a full two years before it became available in the South. It regrettably appears we are seeing a similar scenario play out with the new drug, which was approved in the UK in January last year. We are playing a waiting game here again.

I hope the Minister of State will engage with the necessary authorities and that we will see progress on that. We do not want patients to play another waiting game.

Mary Butler (Waterford, Fianna Fail)
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I thank Deputy Flaherty for raising this issue and for his comprehensive explanation. It is not a condition I am overly familiar with. I will first provide some background for our colleagues and then address his question. This medicine is used to treat polyneuropathy - nerve damage - caused by hATTR. The European Medicines Agency, EMA, granted marketing authorisation for this product in September 2022. As hATTR amyloidosis is a rare disease, the EMA has also designated this an orphan medicine. The company marketing this medicine made an application for addition to the HSE’s reimbursement list in July 2023.

The HSE has statutory responsibility for medicine pricing and reimbursement decisions under the Health (Pricing and Supply of Medical Goods) Act 2013. 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. There are formal processes, which govern applications for the pricing and reimbursement of medicines, and new uses of existing medicines to be funded or reimbursed. In August 2023 the NCPE completed a rapid review of vutrisiran. Factors evaluated during rapid review include the cost of the drug relative to potential comparators, uncertainty relating to comparative clinical effectiveness, uncertainty relating to cost effectiveness and the potential drug budget impact. On this occasion, the NCPE did not recommend a full health technology assessment. Their recommendation was that this medicine not be considered for reimbursement at the price proposed by the company marketing it.

The NCPE’s recommendation is importantly only one component considered by the HSE in making a decision on the reimbursement of a medicine. The Health (Pricing and Supply of Medical Goods) Act 2013 requires that nine criteria be considered in reaching a decision. These include the health needs of the public and the clinical need for the medicine. Following engagement with the manufacturer of this drug, the HSE has decided to approve reimbursement subject to a managed access protocol. These protocols identify patients who would most benefit from the specific treatment being reimbursed. The processes necessary to implement this are currently being developed by the HSE. I reiterate that the HSE has decided to approve reimbursement subject to a managed access protocol.

The State acknowledges the importance of access to medicines and has made considerable investments in new medicines in recent years. Last year, a record of more than €3 billion of public funding was spent on medicines for patients in Ireland. This represents almost €1 of almost every €8 spent by the State on healthcare. This level of investment is unprecedented in supporting patients through the availability of the latest and wide-ranging medicines. The last three budgets have included dedicated funding for new medicines of €98 million. This has enabled the HSE to approve reimbursement for almost 150 medicines, including 61 for treating cancer and 39 for treating rare diseases. For 2024, funding of €20 million has been allocated to enable access to new medicines. The HSE has, in addition, been instructed to identify and realise a further €10 million of efficiencies. When achieved, reinvestment in new drugs will be a possibility. I am delighted that the Deputy will be able to go back and tell his constituent that the HSE has decided to approve the drug for reimbursement.

Joe Flaherty (Longford-Westmeath, Fianna Fail)
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I think we will all agree that it was well worth waiting until 10.15 p.m. tonight for that good news. After that good news I almost feel I should move constituency to Donegal. There is at least 1% of the Donegal population who will probably view that as better news than Jim McGuinness returning as team manager. It is excellent news and I applaud the Minister of State for her work on this to date. The HSE has obviously decided to allow the drug based on a managed access protocol. I know when they started using the current drug it was limited to nine people and phased in over a similar length of time.

If we can see the same progress, initiative and conviction behind the roll-out of this drug, it will be very positive. It is probably unique that we have a rare disease like this. Ironically, this particular variant of it was only first discovered in Ireland in about 1986. Affected patients have a 50% chance of passing on the genetic susceptibility to their children. Until recent times, many people with amyloidosis were misdiagnosed as having heart disease due to other more common symptoms such as high blood pressure. It is getting late in the evening, but I will finish with a little bit of history. A 1995 study of the County Donegal cases found that nearly all those affected were direct descendants of a man called Conall Gulban. His descendants subsequently founded what we now know as Tír Chonaill or Donegal. It is timely as we come up to St. Patrick's Day that we should note that Conall Gulban was, in fact, a son of Niall of the Nine Hostages, who became the first nobleman to convert to Christianity. With a mix of good news for the patients affected and a solemn burst of history, I will finish up on that. I thank the Minister of State for that good news.

Mary Butler (Waterford, Fianna Fail)
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I thank the Deputy very much. I will bring his comments to the attention of the Minister for Health. As I said, the State spent more than €3 billion on medicines last year, which is a new record. This is in the context of a total health budget of €22.5 billion, which is another record. At the same time, however, it is a person who needs that orphan drug, for example, and it will make a difference to that person's life. The Deputy said that in August 2023, the NCPE completed a rapid review of Vutrisiran, the factors of which are evaluated during rapid review. On this occasion, it decided not to recommend it. Importantly, that is only one component of nine. The decision was taken by the HSE. At the same time, 1% of the population in Donegal is a large number of people, so I am delighted to be able to confirm again that the HSE has decided to approve reimbursement subject to a managed access protocol. Hopefully, that will happen very quickly. I thank the Deputy again for raising this really important issue.