Joe Flaherty (Longford-Westmeath, Fianna Fail) | Oireachtas source

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.

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