Fiona O'Loughlin (Fianna Fail)
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The issue I want to raise before the House, and it is not the first time that I have spoken about it here, is the area of hereditary amyloidosis. Like many Members, I would not have heard about this very debilitating and difficult disease if it had not affected somebody that I know very well. Pat Tinsley is a well known and well loved member of the Newbridge community whose life has been absolutely destroyed by this degenerative disease. This debilitating and fatal illness attacks the heart and nervous system of patients.As it progresses, it causes loss of sensation, limb weakness and pain. The drug patisiran, which helps alleviate and manage symptoms, is available in the UK and the US but is not available here.

Pat is running out of time and desperately needs this drug, as do approximately 30 other people in Ireland. Thankfully, the Department secured a significantly enhanced budget with an extra €50 million for new medicines in 2021 but I have been informed that this budget allocation will be insufficient to cover the costs of all new medicines in 2021. I know that the HSE is committed to providing access to as many new medicines as possible from the resources provided and I urge it and the Minister to include patisiran in that list.

Some 4,000 of my constituents signed a petition calling for patisiran to be made available to Pat and the other sufferers of this disease and I am speaking on their behalf. I have submitted that petition to the Minister for Health, the chief executive of the HSE and the Chair of the Oireachtas Committee on Health. According to the HSE, the required National Centre for Pharmacoeconomics, NCPE, health technology assessment report was received by the HSE on 24 February 2020. At that point, under a different Government and regime, it was recommended that patisiran not be recommended for reimbursement unless cost effectiveness could be improved relative to existing treatments. Patisiran was then considered at a meeting of the drugs group in July 2020 and, unfortunately, no recommendation was made then.

The agenda of the December 2020 drugs group meeting was full and patisiran was added to the list of items for the next available meeting. Patisiran currently sits as the third item for consideration on the draft January drugs group agenda. The drugs group is expected to provide a recommendation to the HSE executive management team on the basis of all of the available evidence at that time.

I understand that the HSE executive management team is required to consider a range of criteria which include unmet need, clinical evidence, economic evidence, budget impact and the resources available. I urge the executive to remember that behind these statistics and criteria lie real people who desperately need this drug. Those with amyloidosis understand that other people are also waiting for drugs but there is a huge sense of urgency for sufferers of hereditary amyloidosis due to the rapid progression of the disease. I am pleading with the Minister of State and the HSE on their behalf. They absolutely need to have this drug and if they do not get it, the quality of their lives is at risk, and even their lives are at risk.

Frank Feighan (Sligo-Leitrim, Fine Gael)
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I thank Senator O'Loughlin for raising this important issue and for giving me the opportunity, on behalf of the Minister, Deputy Stephen Donnelly, to speak on the reimbursement of patisiran for hATTR amyloidosis. I fully understand that access to potentially beneficial drug treatments is an extremely important issue for people with this condition.

As the Senator is aware, the HSE has statutory responsibility for medicine pricing and reimbursement decisions under the Health (Pricing and Supply of Medical Goods) Act 2013. The Act specifies certain criteria for decisions on the reimbursement of medicines which include unmet need, clinical evidence, the resources available to the HSE when making reimbursement decisions and 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 NCPE. It uses a decision framework to systematically assess whether a drug is cost effective as a health intervention.

The Minister is advised by the HSE that it received an application in December 2018 for the reimbursement of patisiran for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. In January 2019, a full health technology assessment, HTA, was commissioned by the HSE. In February 2020, following the HTA, the NCPE recommended that patisiran not be considered for reimbursement unless cost effectiveness could be improved relative to existing treatments.

In July 2020, the HTA report, the patient group submission received during the HTA process, and the outputs of commercial negotiations were reviewed by the HSE drugs group. The drugs group was unable to recommend in favour of patisiran at that time. The drugs group therefore sought additional patient and clinician engagement input via the rare diseases technology review committee, RDTRC, to assist the group in making its recommendation to the HSE executive management team.

In September 2020, the rare diseases technology review committee reviewed the clinical data previously submitted as part of the established pricing and reimbursement process and heard from consultants involved in the specialist management of patients with this illness. In November 2020, the committee convened again and continued its review of patisiran, which included patient input on the impact of this illness. Following these meetings, the committee finalised a statement summarising the clinician and patient engagement on patisiran, which was forwarded to the HSE drugs group for consideration.

The HSE has advised that patisiran currently sits as the third item for consideration on the January drugs group draft agenda. The statement from the rare diseases technology review committee will be considered by the drugs group along with the health technology assessment and the outcomes from commercial negotiations.

As the Senator will be aware, additional funding of €50 million was provided for new medicines in budget 2021. This funding will allow the HSE to approve medicines which have been recommended by the HSE drugs group on the basis of efficacy and value for money in line with the 2013 Health Act. The application for patisiran remains under consideration with the HSE and the reimbursement of this medicine will be considered in this context.

Fiona O'Loughlin (Fianna Fail)
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I have listened closely to what the Minister of State has said and I accept that last February and July, a decision was made in the negative on patisiran but things have changed rapidly. The fact that an extra €50 million was included in the budget in order to treat rare diseases is important and an absolute game changer. I accept, of course, that there has to be criteria for the spending of public money on medicines. However, the 30 people who we are speaking about are lucky that we know that there is a drug that can absolutely help to support them with their difficult symptoms. That is hugely important. I accept that the whole reality of trying to negotiate with commercial pharmaceutical firms is something that we need to do. We need to have that negotiation. I hope that the Minister of State will intervene to ensure that those 30 people, including my friend Pat, will have the opportunity to access patisiran in early 2021.

Frank Feighan (Sligo-Leitrim, Fine Gael)
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The Minister, Deputy Stephen Donnelly, has received a number of representations on this matter from patients and concerned family members. I fully appreciate that this is a worrying time for patients, families and carers, and I hope the Senator's friend Pat and any other people who want this drug will get a good result. The Minister and I hope that this process comes to a satisfactory conclusion for all concerned. It is important to note that the HSE executive management team is the decision-making body for the reimbursement of medicines under the Health (Pricing and Supply of Medical Goods) Act 2013. It will, on receipt of the outcome of the drugs group's deliberation, make the decision as to whether patisiran will be reimbursed.The Senator is absolutely right that the additional funding for new medicines provided in budget 2021 will allow the HSE to provide access to evidence-based, cost-effective medicines, and ultimately to provide more medicines to Irish citizens. This item is third for consideration on the draft January drugs group meeting agenda, and hopefully there will be a positive outcome. The decision on this issue is made by the HSE executive management team, on receipt of the outcome of the meeting of the drugs group.