Oireachtas Joint and Select Committees
Wednesday, 15 October 2025
Joint Oireachtas Committee on Health
Treatment of Rare Diseases: Discussion
2:00 am
Pádraig Rice (Cork South-Central, Social Democrats)
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Apologies have been received from Deputy Cullinane and Senator Costello. I express my sympathy to Senator Costello on the passing of her father. Deputy Buckley will substitute for Deputy Cullinane and Senator Rabbitte will substitute for Senator Costello.
I remind members of the constitutional requirement that members must be physically present within the confines of Leinster House complex in order to participate in public meetings. I will not permit members to participate if they are not adhering to this constitutional requirement. Therefore, a member who attempts to participate from outside the precincts will be asked to leave the meeting. In this regard, I ask members participating via Microsoft Teams that prior to making their contributions to the meeting, they confirm they are on the grounds of the Leinster House complex.
Are the minutes of the meetings of 7 and 8 October agreed? Agreed.
Today, the committee will consider issues relating to the treatment of rare diseases, including access to and refunds for medicines. To commence the committee's consideration of these matters, I welcome from the National Centre for Pharmacoeconomics, Professor Michael Barry and Dr. Lesley Tilson, and from Rare Diseases Ireland, Ms Vicky McGrath. Joining us on Teams a little later will be Professor Postma from the University of Groningen. He will join us at 10 a.m.
The Irish Pharmaceutical Healthcare Association was also invited to participate but its respondents stated that because of other commitments, it is unable to attend. Additionally, written submissions have been received from Muscular Dystrophy Ireland and the Irish Platform for Patients' Organisations, Science and Industry, which were circulated yesterday by the committee secretariat.
Members and witnesses are reminded of the long-standing parliamentary practice to the effect that they should not criticise or make charges against any person or entity by name or in such a way as to make him, her or it identifiable, or otherwise engage in speech that might be regarded as damaging to the good name of the person or entity. Therefore, if their statements are potentially defamatory in relation to an identifiable person or entity, I will direct them to discontinue their remarks. It is imperative that they comply with any such direction.
To commence consideration of the matter, I invite Professor Barry to make opening remarks on the behalf of the National Centre for Pharmacoeconomics.
Professor Michael Barry:
I thank the committee for inviting us. I will go through the opening statement as follows. At the start of our submission, we highlighted the fact that the term "orphan drug" refers to medicines that are used to treat conditions that affect less than one in 2,000 citizens. While individual orphan conditions may be rare, collectively they are not. The National Rare Disease Strategy 2025-2030 publication highlights that an estimated 300,000 people in Ireland are living with a rare disease. Orphan drugs account for approximately 22% of rapid reviews and 30% of full health technology assessments, and approximately 27% of all medicines reimbursed over the past five years.
To give the committee a flavour of what is happening right now, I will present some statistics for the first nine months, January to September, inclusive, of 2025. Some 36 drugs for 38 therapeutic indications have been considered for reimbursement by the HSE drugs group. Our reports go to the HSE corporate pharmaceutical unit, which then goes to the drugs group. The drugs group makes recommendation for reimbursement for the cost of medicines. The HSE leadership decides whether the cost of a drug will be reimbursed or not. I should say that is not something we decide but we feed into the process. Reimbursement was recommended for 29 of the therapeutic indications reviewed, which equates to 76, and rejected for six, or 15.8%. Orphan drugs accounted for 17 of the 38 indications, or nearly 45%, reviewed by the HSE drugs group and 12 of the 17 orphan drugs were recommended for reimbursement.
Table 1 presents the orphan drugs that have been looked at this year up to 30 September. It starts with artesunate, tafasitamab, iptacopan down to talquetamab, along with the indications. There is a wide variety of indications but many are cancer. Many orphan drugs are for the treatment of cancer as well. Ten of these are cancer drugs. There is an overlap. The reimbursement recommendation is also shown. Just two of the drugs were not recommended for reimbursement, vosoritide for achondroplasia and carfilzomib for multiple myeloma. All of the others were approved for reimbursement. Three are awaiting a decision. This process is ongoing. There may be further price offers and the HSE would consider same.
It can be seen that the majority, 70.6%, of orphan drugs considered were recommended for reimbursement despite the fact that just one of the drugs, imlifidase, in the area of renal transplantation, satisfied the cost-effectiveness threshold for this country, which is €45,000 per quality-adjusted life year. Not all drugs will be subject to a full health technology assessment. Some get through at the rapid review phase. For those that were, ten had an incremental cost effectiveness greater than €100,000 per quality-adjusted life year and four exceeded €200,000 per quality-adjusted life year. Essentially, they were not cost effective. The failure to prove cost effective is likely to be related to the fact that only three of the 17 orphan drugs improved overall survival rates and just two were shown to significantly improve quality of life. For seven of the 17 drugs reviewed, the clinical evidence was based on single-arm clinical trials, meaning there is no comparator. It is very hard to define the relative effectiveness of a drug if there is no comparator in the clinical trial. We see this not just in orphan drugs but also in cancer drugs.
The NCPE assessment process and subsequent price negotiations facilitated the reimbursement of drugs initially deemed not to be cost effective. Just because a drug is not cost effective does not mean it will not be reimbursed. The health technology assessment indicates to the HSE at what price the drug might present value for money and the HSE negotiates the price. Orphan drugs are very expensive. For example, iptacopan for paroxysmal nocturnal haemoglobinuria costs over €400,000 per patient per year at the list price including VAT. Carfilzomib plus daratumumab plus dexamethasone for multiple myeloma costs over €424,000 per treatment course and ciltacabtagene for the treatment of multiple myeloma costs €478,800 per treatment. That is a CAR T-cell therapy. A number of the drugs on the list are CAR T-cell therapies, which are an exciting new innovation but they come at a cost. I also mention in the table the percentage of these drugs that cost over €100,000, €200,000, etc. They are expensive drugs. The five-year gross budget impact for the 17 orphan drugs considered so far in 2025 amounts to €381 million. They are high-cost drugs with a very high budget impact. The HTA process and subsequent negotiations have to date reduced that figure by €142 million, which is now available for something else in the health service. That is the key.
Pádraig Rice (Cork South-Central, Social Democrats)
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We are over time so we might just wrap up.
Professor Michael Barry:
Apologies. Members can see the timelines on the list from EMA marketing authorisation to when the drug reimbursement recommendation is made. The drug is reimbursed about a month after that. We will have that information at the end of the year. We account for 222 days. I wish it was less; we are working hard to make sure it is less. We are not responsible for the vast majority of the delays. Table 3 shows the number of orphan drugs reimbursed over the past five years.
I apologise for going over time.
Pádraig Rice (Cork South-Central, Social Democrats)
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I thank Professor Barry. I now invite Ms McGrath to make her opening remarks.
Ms Vicky McGrath:
Good Morning. I am the chief executive of Rare Diseases Ireland. We are the national alliance for rare disease patient organisations in Ireland working across all rare diseases, including oncology, to improve the lives of the estimated 300,000 people living with rare diseases in Ireland. I thank the Chair and the committee for affording me the opportunity to address them today. Seven years ago, almost to the day, I sat in front of this committee and answered questions on the same topic of access to medicines for rare diseases. I spoke then of a reimbursement system that continues to fail the patients it is supposed to serve. I would love to say that seven years later things have changed but sadly I cannot. It was November 2018 when I last addressed the committee on this topic. It is worth noting that 2018 was also the year in which the health committee published the Evaluating Orphan Drugs report, which called for reform of the reimbursement process specifically for orphan medicinal products, OMPs. As a quick reminder, OMPs, or orphan drugs, are defined as medicines that prevent or treat rare diseases, which affect less than one in 2,000 people. There are an estimated 6,000 to 8,000 rare diseases According to Orphanet, the European standard database for all things rare diseases, approximately 150 OMPs from 87 pharmaceutical companies were authorised for sale in Europe as of March 2025. Therefore, less than 5% of rare diseases benefit from an orphan product.
Marketing authorisation from the European Medicines Agency is the trigger for permitting a new OMP to be made available. Specifically, marketing authorisation means a pharmaceutical company has received approval to sell a medicine in all EU countries based on a thorough scientific assessment of the medicine's quality, safety and efficacy. In the five-year period following the 2018 meeting and the publication of the health committee report, the EMA together with the Commission granted marketing authorisation to 76 orphan medicinal products. Eight were subsequently withdrawn from the market, leaving 68 OMPs authorised by the EMA to be placed on the market. That is 68 new medicines specifically targeting rare diseases. However, only 19 have been approved for reimbursement by the HSE. That means that just 28% of orphan products authorised in the period between January 2019 and December 2023 are available to Irish patients today. The average time from authorisation to HSE reimbursement for these 19 products is 1,024 days, approximately two years and nine months.
When so few innovative medicines are available to public patients in Ireland, no one can deny the system is broken. I would like the committee to note I am specifically referring to public patients. Those with access to private healthcare get access to some of these non-HSE-reimbursed OMPs privately. The Government talks of Sláintecare, a universal single-tier service where access is based on need and not ability to pay. The reality is very different on the ground for people living with rare diseases, in particular rare cancers. Members may hear the HSE and industry say delays in reimbursement decisions are largely the fault of the other party. It is certainly not the fault of patients seeking access to these OMPs, which will slow disease progression, stabilise health, improve quality of life and are sometimes potentially even curative. They are OMPs that their friends and family members get access to in Northern Ireland or across the water in Great Britain and mainland Europe. Even more galling, their friends and family may access them through private healthcare here in Ireland. It is not their fault.
What of the 49 OMPs that have received EMA market authorisation but are not reimbursed by the HSE? For 27 of them, an application for reimbursement has never even been filed with the HSE. Who is the gatekeeper when it comes to making clinical decisions on the best care for a patient? It is not the healthcare expert or even the HSE. Control has been ceded to the manufacturer in these circumstances. We had such an example from CHI over the last few weeks where a clinician had sought access to a therapy but was denied. The drug in question received authorisation over four years ago. Where is the equity in this? Correspondence from the Minister’s office to the affected family dated August of this year simply stated that no pricing or reimbursement application had been received by the HSE.
That is it; there is nothing more. The manufacturer has not submitted an application and, therefore, the drug is not available. There is nothing we can do. Again I ask, who is responsible for this? Who will take leadership and make certain that no more families are left to the whims of individual manufacturers?
Twenty-seven OMPs with no application for reimbursement means that the manufacturer has either determined that they will not seek reimbursement for the Irish market or that they are delaying seeking reimbursement. We must ask why this is. We are a wealthy country. We are the home to many pharmaceutical companies. We are a leader of life sciences innovation in Europe. Yet, no application for reimbursement is even being made. What type of reputation does Ireland have among small and innovative drug companies that they will not even make a submission for reimbursement? We are seeking to attract these innovators to our shores, but we cannot even entice them to sell their novel products into our public healthcare system. Applications for reimbursement have been submitted for the remaining 22 orphan drugs but no final decision has been made. Nineteen of the 22 have been in the reimbursement system for over two years and eight of them have been there for over three years. In fact, as of the start of this week, one product has been in the system for 2,190 days – that is six years.
As an example of the real-world impact these delays have on people living with rare diseases, there is another drug that was approved by the EMA in 2021. The application for reimbursement was submitted by the manufacturer over three years ago, in mid-2022. The product is designed to target inoperable tumours on nerves in children aged three and over. The drug slows tumour growth and helps with symptoms like pain and weakness. While we wait for a decision to be made, tumours grow and the affected children’s physical and psychological health is allowed to deteriorate. Why do we allow this to happen? Why must children suffer while the grown-ups gather around tables to negotiate? These individuals should spend a day in the shoes of these children’s parents to see the real pain and absolute urgency of their decisions.
Pádraig Rice (Cork South-Central, Social Democrats)
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Ms McGrath might move to a conclusion. I am conscious of time.
Ms Vicky McGrath:
Okay. A positive reimbursement decision is not the end of the road. There is often a managed access program, which also adds delays, and there is not much capacity for novel therapies coming along, which adds further delays to the submission process.
Last year, in the run-up to the election, we sought a Government commitment to make OMPs available in Ireland within 12 months of EMA authorisation. I use this opportunity to reiterate this ask. We absolutely must strive to make all OMPs available to Irish patients within 12 months of authorisation over the lifetime of this Government. That does not necessarily mean that a drug is reimbursed, but we need early access programmes to be implemented as soon as possible.
Pádraig Rice (Cork South-Central, Social Democrats)
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I thank Ms McGrath. Professor Postma will be joining us after 10 o'clock, so I propose we take the first speaker and then the statement from Professor Postma, if that is agreeable. The first slot is for Fianna Fáil.
Martin Daly (Roscommon-Galway, Fianna Fail)
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I thank the witnesses for coming in this morning. This is a difficult subject. We are trying to balance the cost-effectiveness of drugs against true and real-life human suffering, which is extremely depressing. At the end of the day, what we want is a fair system that is timely and that really needs an overview to see how we can improve access to expensive drugs for rare conditions.
That is the key to it. There is a sense that this takes an inordinate length of time from submission to reimbursement. There are a number of issues I would like the witnesses to comment on. One would be the lack of early access programmes in this country. That would be number one and I would like the witnesses' views on that. Second, we pride ourselves on being a wealthy and sophisticated country with regard to health matters but we are a laggard when it comes to clinical trials. We have a very poor clinical trial environment in this country. That is one way, internationally, where people who require access to experimental drugs or even drugs that are in the process of being evaluated nationally do so - through clinical trials.
There are some drugs I will ask the witnesses about specifically. We have had presentations from Muscular Dystrophy Ireland with regard to givinostat. There is also another issue and I cannot understand how this works. I know of a 54-year-old man with severe advanced progressive osteoporosis. His life has changed in the last four years. He has had multiple fractures. He has gone from being a high-performing executive to someone who is disabled and he cannot get access to denosumab, or Prolia, because it is licensed in this country for females only and osteoporosis. I cannot get an answer, after six months, as to why a woman his age can get access to Prolia - that is the trade name for denosumab - and he cannot. His condition is progressive and it is severely disabling.
I would like to ask specifically about a young woman, a mother of three, who developed a desmoid tumour during Covid. There was a little bit of a delay in diagnosis because of Covid but no one was at fault for that. Nirogacestat has been approved by the EMA and is available to people in France on compassionate grounds. She is a young mother and it is inoperable. She has tried chemotherapeutic options. With three young children, does the cost-benefit analysis include not just her life but the lives of her children and their quality of life because they are likely to lose their mother in the next few years if she does not get access? It is a non-operable tumour. She would lose her whole hind quarter and she would simply not survive surgery.
Professor Michael Barry:
There were a number of points there. With regard to the submission of applications and why we are lagging behind on applications, the reality is that for up to 40% of our rare disease drugs, there is no application for pricing and reimbursement. That has to come down to, in large part, finance. Companies will launch and apply for reimbursement in larger countries - Germany with 80 million, France with almost 70 million, Italy with 60 million and Spain with 50 million in population. It is about money, to be fair about it. It is not all of it but it is a large part of it.
The Deputy mentioned early access. That is an interesting one. We have, through the office of the chief clinical officer, started to look at and are in discussions with regard to early access. I agree that there is a role for early access. It is a question of the devil being in the detail with regard to a lot of this. What drugs would go into an early access scheme? Essentially, we are looking at cancer initially. Who pays? How would you identify the drugs and who would identify them? The devil is in the detail, as I said, but I agree with the Deputy. Early access definitely needs to be looked at. It is a fair point and we are looking at it.
Regarding clinical trials, we have had problems with clinical trials over many years. The Deputy will know himself. We tried to do clinical trials in the past and they proved difficult to do. It is getting better but it is a very important area of access.
On givinostat, the drug for Duchenne muscular dystrophy, we were asked to review that initially. It received a marketing authorisation on 6 June. We were asked to look at it in August. We completed our initial review in nine days. We have met with the company since and we await its assessment of the full dossier. We will make sure that is done quickly.
Martin Daly (Roscommon-Galway, Fianna Fail)
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I thank Professor Barry.
Professor Michael Barry:
I would say that with regard to, for example, fenfluramine, which is for Dravet syndrome, as the Deputy will know, we did that HTA in 54 days, so it can be done quickly and we would strive to do that.
On denosumab, I do not know the answer to that. I am sure it is probably because of the application that was put in to the HSE but we can check that out for the Deputy.
Martin Daly (Roscommon-Galway, Fianna Fail)
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I thank Professor Barry.
Martin Daly (Roscommon-Galway, Fianna Fail)
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Nirogacestat for desmoid tumours.
Martin Daly (Roscommon-Galway, Fianna Fail)
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Okay. That is fine. I thank Professor Barry. I have a minute left. This balancing exercise between cost-effectiveness and benefit, and the balance with the cost taken out of the rest of the health service, is a huge challenge.
Professor Michael Barry:
It is, and that is exactly why we do this stuff, the so-called opportunity cost, because if you are going to spend millions of euro, which is what we are talking about, in one area, then it is not available for somewhere else. The Deputy knows the challenges as well as I do in relation to many aspects of the health service. What we are trying to do is make the best use we can of all the resources. For example, the €142 million that will not be paid for orphan drugs this year is available for somewhere else. It is not for me to decide where it should go, but the Deputy is absolutely right about the concept of opportunity cost. We could, if we wanted to, pay the asking price upfront, straight away, but there is a price to be paid for that.
Martin Daly (Roscommon-Galway, Fianna Fail)
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Does Professor Barry think that some of the drug manufacturers have the public purse by the throat in some way?
Pádraig Rice (Cork South-Central, Social Democrats)
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Speakers should be brief as we are running out of time.
Pádraig Rice (Cork South-Central, Social Democrats)
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I thank Professor Barry very much. We will move next to the Sinn Féin slot and Deputy Clarke.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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I thank the witnesses for coming in this morning. They are very welcome. This is an issue that comes up for me on a regular basis, even though we are talking about rare diseases. I think most other committee members would have the same experience.
The question I want to put is on that relationship that exists between the health technology assessment, the legislation around price and supply, the HSE, the medical professionals and the patient. There is a very specific reason I ask this question. Earlier this summer in my constituency, a little boy, Toby, was born at the end of May. By the June bank holiday weekend, when he was a few days old, Toby was intubated. He was in ICU in Temple Street hospital. Toby's mum, Lucy, is a nurse and she knew exactly what was happening. His dad was, for the majority of the earlier time of Toby's illness, looking after their toddler who was at home. Toby has propionic acidemia, which is a rare metabolic condition whereby he cannot break down proteins, which leads to a build-up of acid that could damage his liver and brain and lead to coma. The application for his medication was refused on the basis of economic and budgetary impact. This was despite the fact that Temple Street and the wonderful staff there - Lucy and Niall sing their praises consistently and constantly - sent Toby home with a ten-day supply. After four weeks of being in hospital, this ten-day supply of medication allowed Toby to return home and to have a normal life back with his mum, dad, sister and family. This is lifesaving medication.
Why do TDs have to get involved? Why were we in a position of sending in representations on behalf of this family? I am not a doctor. Please do not come to me looking for help with your car, because I am also not a mechanic. I know what I can and cannot do, but I should not be going to a Minister or anybody seeking medication for an incredibly sick little boy when the doctors have provided it to him to allow him to come home. I want to know how that relationship works.
To be frank, when Ms McGrath said "gatekeeper", I had it written down. I want to know why that situation happened, because that should never happen. Where is the lack of connectivity between the structures that we have that result in TDs making representations for medication?
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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But it was not available for Toby at home.
Professor Michael Barry:
There is a process, outside of the standard process, where somebody can apply for exceptional funding. That is where cases like this would be considered. In other words, there is an opportunity to make the case and say we need this to be funded simply because of what the Deputy says, that it makes sense to be out of hospital and for the family to be at home.
I was not involved in the decision-making. All I can say is that is what I know of this case. Our role is very simple. If we are asked to say what the value for money of a drug is, that is fine, but we were not asked to look at that drug. I apologise, but I cannot tell the Deputy the ins and outs of it. I know of it and I know of the situation. There is an exceptional funding mechanism where people do not have to go into the process. That is the one I would use. Very often, people may not be able to negotiate around the process or even know about it.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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Toby is very fortunate.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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The medication was supplied.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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It was supplied at the end of July.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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It was made available to him and he is doing very well.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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He has a path ahead of him - there is no doubt about that - but he is doing very well. How frequently do decisions like this happen, where a family or patient is told they cannot get access to medication, only for it then to turn out after a little bit of political pressure - kicking, screaming, roaring and shouting - that a decision is made in their favour?
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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Why? It should not happen.
Professor Michael Barry:
That may be argued, but it is the situation in which we find ourselves. It does not happen that often. I certainly do not find myself being brought into a discussion like this too often, but occasionally we will. Generally, we look at the situation like the Deputy described, and from my point of view, I have no problem in supporting something like that. It makes sense to support it.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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This indicates to me that there is a fundamental flaw. The systems in place simply are not working. You should be able to go to your doctor and for the doctor to say you need a certain medication, and it is kept out of the political sphere or spectrum where the decisions are made by those who have the greatest access to information and knowledge of the condition and the medication. This says to me that there is a structural flaw in the reimbursement system.
Professor Michael Barry:
There is a process whereby you can get exceptional funding for medicines. I would not be able to count the number of times the political arena has made contact with me about various medications, although obviously not as serious as that situation. That is the way things work in this country, as I see it.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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Does Professor Barry agree with it?
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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Does it not undermine the trust of the medical professionals?
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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If this is the way things work in Ireland and politicians need to resort to emails to get a patient a medication, are we not undermining trust in the professional doctors in that scenario?
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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I have no hesitation in saying I do not agree with Professor Barry.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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This should be a medical decision. Trust should be put in the doctors who are treating the patients, as opposed to overruling them. Our job is legislation. That is what we do. We are legislators.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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Access to a politician or political entity should not result in one person receiving medication above somebody else who may not have that access.
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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Okay. I will very quickly ask another question. There is a commitment in the programme for Government to a review of the drug reimbursement process. Has anybody had any contact on this issue? Has it started? Is there any update?
Sorca Clarke (Longford-Westmeath, Sinn Fein)
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There are question marks over the Mazars report because the scope did not address those structural flaws.
Pádraig Rice (Cork South-Central, Social Democrats)
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The Deputy's time is up.
Pádraig Rice (Cork South-Central, Social Democrats)
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I am sorry to interrupt Professor Barry but the time is up for this slot. I would appreciate it if people could please adhere to the time.
Pádraig Rice (Cork South-Central, Social Democrats)
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I thank Professor Barry very much.
We are joined by an international guest from the Netherlands, Professor Postma, who has joined the meeting online on Teams and can hopefully hear us. If so, I welcome him to the meeting and ask him to provide a brief opening statement for five minutes.
Professor Maarten Postma:
I thank the committee for this opportunity. What I am going to say links to the previous discussion. My address is on health technology assessment, HTA, and its role in rare diseases. Health technology assessment differs between countries in Europe, although we now have the joint clinical assessment which tries to bring them together a little. The Netherlands and Ireland work together. Zolgensma is an example of a drug for spinal muscular atrophy that was assessed between the Netherlands and Ireland jointly in the Beneluxa Initiative. The Netherlands is strict on health technology assessment so I hope we did not force that on Ireland too much in the Beneluxa Initiative. For example, I have a list of drugs, including tecartus, carvykti and casgevy, which were all denied access. They are for rare diseases. There are more on my list but I will not bother the committee too much. These are innovative drugs. It illustrates the difficult time that drugs have in getting through the health technology assessment and getting reimbursed in the Netherlands. The Netherlands is a country with very strict negotiations and we tend to achieve relatively low prices and big discounts for orphan drugs, which means that if there are supply shortages globally, they often soon emerge in the Netherlands because other countries seem to pay higher prices.
In health technology, as Members of Parliament may have heard, it is often about the cost per quality. There is a value on human life and a maximum of what we pay for the life years that we gain from orphan drugs. We take a set quality of life into account. That comes in the end to the metric of cost per quality-adjusted-life-year, QALY. The cost per QALY maximum in the UK is £30,000. It is €50,000 in my country. The maximum for severe disease is €80,000. Many rare diseases qualify as severe disease, unfortunately for the patients. Drugs for rare diseases unfortunately cannot make the threshold of €80,000. Whether we are in a country which relies heavily on economic assessments or less on economic assessments, such as Germany, all over Europe, you will see that drugs for rare diseases have problems getting through HTA and cost-effectiveness is just one of them.
Together with Professor Michael Barry, in the International Society for Pharmacoeconomics and Outcomes Research, we have-----
Pádraig Rice (Cork South-Central, Social Democrats)
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We seem to have lost Professor Postma due to technical issues. We will move to our next speaker and hopefully Professor Postma can come back.
Colm Burke (Cork North-Central, Fine Gael)
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I thank the witnesses for coming here this morning. I thank them for their work over the past years. I go back to the 2018 report. I think I am the only member of the health committee who is still here. I think Ms McGrath was there in 2018 when that report was drafted. Clear proposals were set out in that report about trying to expedite the process. There has been very little change in those seven years with regard to dealing with the issues. It has led to a whole lot of frustration. We had a presentation here last week about the drug being made available for the first 180 days. The pharmaceutical company carries the cost of that and negotiations try to deal with issues after that. We have not been innovative in dealing with cases where there is clear evidence. All these drugs are already approved by the European Medicines Agency. Why has there not been change in those seven years even though there is a report here from a committee which was carefully researched, with many presentations on it?
Professor Michael Barry:
I do not think I was around for the 2018 meeting. The Deputy makes a good point. This issue of EMA approval is important, particularly when looking at cancer drugs and drugs for rare diseases. Not all drugs approved by the European Medicines Agency, especially cancer drugs, will prolong life and not all of them will improve the quality of life. We see this time and time again. Only a third of cancer drugs that are approved this year, and many of them are drugs for rare diseases too, will prolong life. Less than that, 25%, will improve quality of life. The concept of EMA approval and then reimbursing, in my view, is not quite as simple.
Colm Burke (Cork North-Central, Fine Gael)
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If you take Germany, for example, it brings a drug onto the market, and while I know we are talking about larger volumes of patients, it negotiates the price over the following 12 months and the drug is available to patients at a very early stage. I know it can afford to do that because it has larger numbers. We might have a drug that is only suitable for 50 patients here while it might be 5,000 patients in Germany and I fully understand that. Surely we should look at trying to progress the current situation? Professor Barry has outlined the process in Ireland. We are now behind most other European countries regarding the timeframe from the time a company applies for a drug to be approved to actually getting it approved.
Professor Michael Barry:
Yes. I suppose there are a number of aspects there. The timelines are impacted significantly by the industry in two ways. One is that it does not submit an application here. I cannot do anything about that if it does not. The other is that when it is asked to submit the evidence for a technology assessment, there are delays in that. The Deputy will see in table 2 that there is a delay with price negotiation. That is an important fact, when 27% of the timeframe for orphan drugs is lost in negotiations. You can make all those things better. To come back to the Deputy's point on whether we can do things better, I mentioned earlier that we are looking at early access and what an early access scheme would look like. In other words, you would allow a drug onto the market, continue to do the assessment, and put a timeframe on it of maybe a year and a half or two years, when you would know what the value for money is, and then negotiate on price again. That can be done. The Deputy is right. It is a fair point and it is being looked at right now.
Colm Burke (Cork North-Central, Fine Gael)
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On assessments, there is the whole issue of, for argument's sake, a medication that is only suitable for 50 people a year. Can we join with other countries in sharing and processing the information so that rather than Ireland looking at ten cases with 50 patients in each case, we would share information with other countries? Ireland would take two of those and another country would take another two, so we are not replicating what has been done. Where are we with that?
Dr. Lesley Tilson:
That is happening.
Since that meeting in 2018, there has been a European health technology assessment, HTA, regulation, whereby all European countries are doing joint comparative clinical assessments. That is the next step following the European Medicines Agency licence, where there is a comparative assessment of the drug versus the current standard of care in a particular country. That is starting to happen. The NCPE is playing a leading role in that. We are currently taking part in two of these joint clinical assessments, which are Europe-wide.
Colm Burke (Cork North-Central, Fine Gael)
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Going back to the 2018 report, I was very involved in the drafting of that report. There was a draft report done first. I was involved in redrafting it and making sure we set out a clear programme of action. However, nothing has happened on that. I am concerned that we are here talking seven years later. Will the same conversation be held in seven years' time? I am concerned about the delays that have occurred in progressing and expediting the processing and manufacture of medication. The other issue arising now is that, where something is approved by the NCPE, there is not a buy-in by the HSE. The medical insurers are now saying to patients with private health insurance that they are able to access a drug, but a public patient is not able to get it. There is even a difference among insurance companies, where one will cover the cost of a drug and another will not. We are creating huge division in the country as a result.
Professor Michael Barry:
To address the point about private insurers, we have reviewed 22 cancer drugs this year. The HSE has reimbursed most of those cancer drugs this year. I know for a fact that private health insurers only cover five of those drugs. There is a new innovation in cancer called CAR T-cell therapy. We received four applications for CAR T-cell therapy this year and we approved three. Do members know how many CAR T-cell therapies are approved by private insurers? The answer is none. We can talk about drugs for rare diseases or drugs for cystic fibrosis. How many of those are covered by private insurers? None. This concept out there that the private health insurers are paying for everything and the public system pays for nothing is not right.
Colm Burke (Cork North-Central, Fine Gael)
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I am not saying they are paying for everything.
Colm Burke (Cork North-Central, Fine Gael)
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I fully accept that, but it is creating a division and it is causing a problem.
Professor Michael Barry:
Yes, for some drugs. However, I would turn that around and ask what about CAR T-cell therapy. That is delivered in our own hospital, St. James's Hospital. How many private insurers pay for that? That is one of the greatest innovations in cancer care. Do private insurers pay for it? Of course, they do not because it is too expensive. The public system is left paying for it.
Colm Burke (Cork North-Central, Fine Gael)
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Overall, does Professor Barry accept that we need to improve the system we have?
Pádraig Rice (Cork South-Central, Social Democrats)
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I welcome Professor Postma back to the meeting. We lost connection. I hope he can hear us again.
Pádraig Rice (Cork South-Central, Social Democrats)
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Would Professor Postma like to conclude his remarks?
Pádraig Rice (Cork South-Central, Social Democrats)
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That is perfect. We will continue with questions. Members may have questions for Professor Postma as well.
Pádraig Rice (Cork South-Central, Social Democrats)
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We will continue with questions. I have a number of questions around newborn screening and the heel prick test. I understand there are nine conditions currently screened for newborn babies using the heel prick test and that, in 2023, the Government approved two more, namely, spinal muscular atrophy, SMA, and severe combined immunodeficiency, SCID. Funding was approved in 2024 but it is still not provided. There was an implementation team set up by officials in the HSE and staff in Temple Street Children's Hospital, but it still has not happened. Has Ms McGrath heard from the Department or the HSE on whether there is an updated timeline for the expansion of the screening programme? I see Professor Barry wishes to comment.
Professor Michael Barry:
This is an interesting one. We reimburse most of the drugs that are available for spinal muscular atrophy, including Zolgensma, the gene therapy. From our perspective, the reimbursement process is set up and ready. If patients are diagnosed, they get treated. Drugs like nusinersen, or Spinraza, as well as Zolgensma and Risdiplam, are all reimbursed by the HSE. I have nothing to do with the timelines around screening, but I do know it is active. We have been contacted in relation to it. I am just highlighting that this is under way. I do not know the timelines unfortunately. Does Dr. Tilson?
Pádraig Rice (Cork South-Central, Social Democrats)
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Has Ms McGrath been engaged with in relation to the expansion of the screening programme?
Ms Vicky McGrath:
Yes. The approval process, or the approval provided by the national screening advisory committee, NSAC, and the Minister, was 2-plus years ago and then there was the allocation of budgets, etc. It should be up soon, but I am not 100% sure. I have not heard recently about when those should be implemented. It is a travesty that we are still waiting. It needs to be activated far earlier than it has been in this country.
Pádraig Rice (Cork South-Central, Social Democrats)
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My understanding is that in other countries far more conditions are covered by this newborn screening and that our screening is quite limited. It would be great to see that expanded, particularly given that the funding is already there.
Ms Vicky McGrath:
Yes, absolutely. We are way behind other countries in terms of the amount of screening we conduct here. I believe there are a number of products currently being assessed but, given the track record, it could be four, five or six years before they get implemented, presuming they get approved.
Pádraig Rice (Cork South-Central, Social Democrats)
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What is causing the delay in expanding the screening?
Ms Vicky McGrath:
The biggest delay is probably the fact that we are conducting assessments in Ireland when assessments have already been conducted at a European stage. It is a little bit like us saying we will throw EMA out the window and we are all going to do our own assessments of whether a drug is safe and effective. We are in that stage now with the newborn screening, where we just do not collaborate more across Europe and say, "Here is a European decision; let us implement it here." There is definitely lab work that has to be done locally, on the ground in Ireland. We cannot deny that, but it is the precursor to initiating that lab work. It could help if we could initiate the lab work at the same time as the NSAC is doing its reviews. There is reluctance to do that, however, because NSAC could potentially say "No", but it seems incredibly unlikely. All the clinicians are requesting this happen. We are not doing anything unusual. There is nothing particularly unique about the Irish system.
Pádraig Rice (Cork South-Central, Social Democrats)
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I heard about a case of an Irish person who gave birth abroad and had newborn screening done there. That screening picked up a case of SMA, but if the child been born here, that would not have been picked up. The person was then able to get earlier intervention. It is unfortunate we are seeing that.
Pádraig Rice (Cork South-Central, Social Democrats)
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Perhaps that is one we can follow up on with the HSE and the Minister.
I understand the HSE has approved three treatments for SMA that can halt the condition's progression and significantly improve quality of life. However, an arbitrary age limit of 18 years was imposed at the time of reimbursement. The treatments were made available for those living with SMA and under the age of 18 at the time of approval and this cohort continues to receive treatment. Meanwhile, those over the age of 18 at the time of approval were denied access and continue to be excluded from the treatment. I understand there are approximately 20 adults in Ireland currently affected by this policy. According to Muscular Dystrophy Ireland, which provided the committee with a written statement, the original reimbursement submission for two of the treatments included both adult and paediatric populations, yet reimbursement was restricted to those under the age of 18. Countries such as the UK, Germany, Italy, Spain, Portugal and Hungary have no age-based restrictions on that. What was the basis for the arbitrary age limit for access to SMA treatments?
Professor Michael Barry:
The basis for that was the clinical data. It was appreciated that the clinical efficacy of these drugs was far greater in the younger patients. The younger the patients were, the better they did. That was the basis of the decision. Whether it is right or wrong is another day's work, but that was the basis of it. It was based on the clinical data.
Pádraig Rice (Cork South-Central, Social Democrats)
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Has there been any review of the current age-based restrictions for these treatments?
Professor Michael Barry:
We have not been asked to look at it recently. It is something that could be looked at. As I said, however, that was the position taken with the information available to us at the time. The Cathaoirleach highlighted a number of people who were not on treatment. Maybe that should be revisited, but certainly at the time it was based on clinical data.
Pádraig Rice (Cork South-Central, Social Democrats)
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The stakeholders who have been in touch with us are calling for a clinical reassessment framework allowing adults with SMA to access therapy based on specialist clinical assessment and not chronological age. What is Professor Barry's view of that kind of proposal? From whom would direction be needed to act on that?
Professor Michael Barry:
A case could be made that it should not be based just on age but on a clinical presentation. We see this across many therapeutic areas. It could be argued that the criteria we follow should be different but that was what it was based on at the time. If it merits re-examination, that should not be too difficult.
Ms Vicky McGrath:
I was at an event on this very product and challenge in the audiovisual room here before the summer. Something needs to happen. We cannot just dice and slice the population up in any old way. We have to be far more collective, so that everybody gets access to therapies based on the clinical expertise of their clinicians.
Pádraig Rice (Cork South-Central, Social Democrats)
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I have one quick question for Professor Postma. In the Netherlands, 27 conditions are screened for in newborns, compared with Ireland, which does just nine. Has any research been done into the cost-benefit analysis of expanding newborns screening?
Marie Sherlock (Dublin Central, Labour)
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I very much thank the National Centre for Pharmacoeconomics, Rare Diseases Ireland and Professor Postma for attending. I pay tribute to the work of Rare Diseases Ireland. I know many other groups have been campaigning in this space for many years. It is absolutely heartbreaking when people come to me to tell me they may have to travel to the North, Britain or further afield to access medicines they cannot access here. There are obviously a multitude of issues with regard to the application process and processing time. The blatantly obvious thing is Ireland is negotiating as a small population of 5 million people, when the negotiations should happen at a European level.
I will direct my questions to Professor Barry. I want to be clear on a number of things he said with regard to those suffering with SMA. Has he been asked by the Department to examine the age-based restriction to access to the two drugs that have been approved for those aged under 18 but not for those over 18?
Marie Sherlock (Dublin Central, Labour)
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Again, to be clear, am I hearing Professor Barry correctly when he said that he rejected that there is an inequality between access to oncology drugs via health insurers and what is available in the public system? Did he say that inequality does not exist at the moment?
Professor Michael Barry:
I am not saying that. I am just saying that what is being portrayed in not entirely accurate. Many therapies are being paid for by the public purse that are not being paid for by private insurers. I am sure if you looked hard enough, you would find drugs that are not being paid for by the HSE that are being paid for by private insurers. I will say one thing, however. I went through this very carefully, not for this meeting but another meeting we are having tomorrow, in relation to the 22 cancer drugs that were assessed this year, the vast majority of which were reimbursed. Private health insurers cover five of them. I am saying it is portrayed as if there is this huge inequality, but I do not agree with that. CAR-T cell therapy is one major area, which I mentioned.
Marie Sherlock (Dublin Central, Labour)
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I know. I hear Professor Barry on that, but does he accept some drugs are available through private insurers in Ireland, including oncology drugs, that are not available through the public system?
Marie Sherlock (Dublin Central, Labour)
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Of course. With regard to processing time, there is a figure of 222 days. We have seen longer processing time over the last number of years. Staffing was talked about. Will Professor Barry tell me what the current staffing level is, what it was and where he hopes it to go?
Dr. Lesley Tilson:
We were granted additional funding through the Mazars process. We got approval to recruit more staff in the summer of 2024. We had 20.5 whole-time equivalents until that time and we have been recruiting over the last 12 months. We now have 36.5 whole-time equivalents. It is a major change for us.
Marie Sherlock (Dublin Central, Labour)
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Where does the NCPE want to go to?
Marie Sherlock (Dublin Central, Labour)
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Does Dr. Tilson expect there will be a further improvement in the processing time or is this it?
Dr. Lesley Tilson:
We expect a further improvement from the NCPE perspective. There was a big capacity deficit. We are now going through the training process with all these new staff, so we expect improvements from the NCPE. We have looked at those timelines. We are only one proportion of those timelines. We are not the longest time in the process.
Marie Sherlock (Dublin Central, Labour)
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I appreciate that but there is frustration about the particular NCPE part of the process. What is its target with regard to the processing time?
Marie Sherlock (Dublin Central, Labour)
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Okay, but I am not hearing the target. Dr. Tilson said there is a delay of six months at the moment-----
Marie Sherlock (Dublin Central, Labour)
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So the NCPE can start immediately once it comes in.
Professor Michael Barry:
I will make one point to give some encouragement. For example, the total number of assessments we did in 2024 was 77. To the end of September 2025, we have done 79. I am hoping that shows things are changing and getting better. The Deputy is absolutely right. We want it down to be able to start the HTA as soon as it comes in.
Marie Sherlock (Dublin Central, Labour)
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On the QALY thresholds, I understand Ireland is at €45,000.
Marie Sherlock (Dublin Central, Labour)
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We heard from Professor Postma that it is €80,000 in the Netherlands. When was the last time that threshold was set? I want to understand how out of date it is at this stage.
Professor Michael Barry:
About that. I will say one thing in relation to it and a point I made earlier. Only one drug satisfied the cost-effectiveness threshold of the orphan drugs we looked at this year, but we have reimbursed 12. If you are negotiating on very expensive drugs, the question is where you are negotiating to. What is the price? The assessment indicates to the HSE what price you would like to get it at. That is what the negotiations do. Even though one satisfied the criteria, 12 were reimbursed. Do you know what I mean? It does not-----
Marie Sherlock (Dublin Central, Labour)
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What is the point of the €45,000 then? Does it have any relevance at all?
Professor Michael Barry:
I think it does. Some people have looked at these thresholds to see whether they are correct or whether they should be higher or lower, but it gives us something to aim for in terms of value. We then negotiate on that.
The drugs reimbursed by the HSE this year will not all reach the €45,000 threshold. Some will be below it and some will be above it, but it is an indicative target.
Marie Sherlock (Dublin Central, Labour)
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Has updating that figure not been considered?
Marie Sherlock (Dublin Central, Labour)
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It is the Department that-----
Marie Sherlock (Dublin Central, Labour)
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Has the NCPE made an application to the Department to review the threshold?
Professor Michael Barry:
It could be reimbursed. I have not made any application to change it, but it could be done. The interesting thing in relation to drugs for rare diseases is that even if we took the figure Professor Postma spoke about – €80,000 per QALY – it would not make a huge difference because most are above that anyway. Does the Deputy know what I mean?
Marie Sherlock (Dublin Central, Labour)
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Yes.
It is wonderful to hear early access is being looked into but I just want to understand more precisely what that means. Is the NCPE having active conversations with the Department of Health on this at the moment? Could he set out in detail what has been considered?
Professor Michael Barry:
The discussions are with a number of groups, through the office of the chief clinical officer. We are having discussions with the Irish Pharmaceutical Healthcare Association and the industry. It is envisaged that once some sort of format is agreed, it will then be taken into the healthcare setting. I am very encouraged by the discussions to date.
Tom Clonan (Independent)
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I thank the witnesses. I have a young man at home who has a rare disease, and unfortunately for him I do not think there is any treatment available for him at the moment. We live the life of the family witnessing a person’s quality of life deteriorating, with life-limiting and life-altering implications for him.
For how long has Professor Barry been in his post?
Tom Clonan (Independent)
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Twenty-seven years. Looking at Professor Barry's senior management team and review group, I imagine they are a veritable treasure trove of Trinity voters. They are mostly Trinity graduates.
Tom Clonan (Independent)
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Professor Barry can vote for me in the new constituency that is being established. I have not seen the details of everyone in the team, but the vast majority are pharmacists and statisticians. Professor Barry will have to forgive me because I was at the meeting of the disability committee and may have missed something. Apart from the experts from a natural sciences background, which is a quantitative discipline, does the NCPE have people with an ethics background?
Professor Michael Barry:
No. We do not have an ethicist on our board. I was going to say, and I do not know if the Senator was present to hear this, that we do liaise on every single assessment we do with our clinical colleagues, whether in oncology, rheumatology or other disciplines, but not in ethics.
Tom Clonan (Independent)
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Have families a formal mechanism for feeding into the assessment?
Tom Clonan (Independent)
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Does the National Centre for Pharmacoeconomics deal directly with patients?
Tom Clonan (Independent)
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Do patients get to feed directly into the assessment process?
Tom Clonan (Independent)
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We have a similar concern in the disability sector because, usually, service providers or representative groups are not disabled persons’ organisations. The centre does not deal directly with people with the qualitative experience in question.
Professor Michael Barry:
There is another mechanism: a rare disease technology review committee. I actually chaired that for a few years. I found it really useful because we invited people to come in and present their stories. We did it for spinal muscular atrophy very effectively, and that really informed the process. The information was fed back to the drugs group. This is happening not only at NCPE level but also through the rare diseases technology review group. Both, but particularly the rare diseases technology review group, give the patient a voice. That is very important.
Tom Clonan (Independent)
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Something struck me from what I heard, and I apologise again as I was at the disability matters committee meeting. I suppose it goes with the title “pharmacoeconomic review”, but Professor Barry talked about a cost–benefit analysis. Does he believe that is an ethical way to assess the funding, approval or otherwise of drugs?
Professor Michael Barry:
I do, and I will tell the Senator why. I would argue we advocate for everybody, not just the person in front of us and not just in respect of the drug in front of us. There is a finite healthcare budget. The point I would make, which I made earlier, is that if we spend all of it in one area, we are not going to be able to spend in another. Somebody always loses out, and that is the person who does not have a voice. For example-----
Tom Clonan (Independent)
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That is Professor Barry’s subjective view.
Tom Clonan (Independent)
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Is it informed by any formal ethical framework? If it is, I would be very interested in hearing which one.
Professor Michael Barry:
I suppose you have to look at where this all comes from. We are talking about development over 25 to 27 years. It is grounded mainly in health economics and clinical medicine. Should there be an ethical framework around it? Yes. That is in the definition of “health technology assessment”-----
Tom Clonan (Independent)
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What experts from the human sciences does Professor Barry have on his review boards and assessment panels who can speak to the qualitative and ethically grounded aspects of the centre’s decision-making?
Tom Clonan (Independent)
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Those are all quantitative measures.
Tom Clonan (Independent)
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I understand that. The witnesses mentioned several times in their presentation-----
Tom Clonan (Independent)
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-----what the data tells them, but how are they informed about the human dimension? I would put it to them that having a deterministic, mechanical assessment process, as they have set out, is inherently unethical by definition.
Dr. Lesley Tilson:
We have a patient organisation submission process and there is a template. The organisations submit their representations via the NCPE and they are presented to the drugs group. There is a public representative on the drugs group, and they consider the wider range of criteria in addition to the NCPE evidence. Some of this refers to beyond the NCPE.
Tom Clonan (Independent)
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On the basis of the outcomes and international comparators, we seem to be behind our peers in other parts of the EU. I have serious concerns that the NCPE does not have an ethicist and does not have enough interface with the human sciences and expertise in this area. That is something the centre ought to consider. Its decision-making process, if it entails a simple cost–benefit analysis, is inherently unethical.
Professor Michael Barry:
We do not make decisions. The reimbursement recommendation is by the HSE drugs group, which has many stakeholders, including laypeople. It is not our job to make decisions. We provide information in relation to what we are asked, and we are asked a very simple question: is this value for money or is it not? That is what we provide. We have never made a decision. I have never made a decision on a drug in my life.
Tom Clonan (Independent)
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As an ethical defence, that is highly relativistic. It is actually quite a controversial ethical defence to put forward.
Tom Clonan (Independent)
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Professor Barry has been 27 years in his post. It is quite an onerous position in terms of self-care and professional development. Is it healthy for an organisation to have somebody at the helm for 27 years?
Tom Clonan (Independent)
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I mean personally. In terms of self-care but also in terms of organisational development, it strikes me as being quite unusual.
Tom Clonan (Independent)
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I am just talking about heterogeneity.
Pádraig Rice (Cork South-Central, Social Democrats)
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The Senator's time is up.
Pádraig Rice (Cork South-Central, Social Democrats)
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The time is up. I wish Professor Barry well with his final time in post.
I suggest we take a quick comfort break and resume in five minutes. Is that agreed? Agreed.
Pádraig Rice (Cork South-Central, Social Democrats)
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We will now continue our consideration of rare diseases. Our next slot is for Fianna Fáil. I call Deputy O’Sullivan.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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I welcome all the witnesses. I appreciate the debate up until now. I know Professor Barry has a very difficult job. I have no doubt he has seen a lot of parliamentary questions from the likes of Deputy Burke and me over the years. We understand he has a difficult role and it is a role he tries his best to perform diligently. It has been interesting listening to the past half hour of the debate and the discussion of question of ethics versus economics. As I said, I understand tough decisions have to be made.
In table 2 of the NCPE’s submission, it shows a timeline of 1,165 days from European Medicines Agency, EMA, marketing authorisation to the HSE drugs group review. Delays with the manufacturer account for 52.7% of that timeline. The NCPE’s own time of 222 days, combined with the 18 days at the Health Service Executive’s corporate pharmaceutical unit, CPU, total 240 days, or approximately eight months. Under the health Act, as I understand it, there is a requirement for this process to be completed within 180 days. To state the obvious, we are acknowledging that requirement is not being met, albeit it is not a massive disparity with what is required. It is still not being met at the same time.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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Section 18(2) of the Health (Pricing and Supply of Medical Goods) Act 2013 mandates decisions within 180 days, but it is my understanding that the manufacturer slot, that is, the period from EMA approval to application stage, is not included in that requirement under legislation.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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It is not a requirement under the legislation.
Professor Michael Barry:
The other important point is that if the HSE asks for a full health technology assessment or more information, the clock can stop as well. I have included all the timelines in the table.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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For the benefit of people listening in, that is not a requirement under the health Act.
I will ask Professor Postma a question because his line is coming and going.
Under the WAIT report of 2024, Ireland approved 13 OMPs. Can I ask Professor Postma what the comparative figure was in Netherlands?
Professor Maarten Postma:
Is the Deputy referring to the health technology assessment? I do not know the exact number but what I do take from the discussion so far is that the situation in the Netherlands is certainly not as good as it is in your country. We have denied reimbursement for various rare diseases drugs. We also now have a lock for expensive drugs, which means another delay of one year. There is a shortage of staff and drugs await assessment. I apologise that I do not have the exact numbers. The situation with us is actually worse. Your country is better off in general here, I would say. I hope that helps.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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Following on from Deputy Clarke's comments earlier and part of the submission from Professor Postma, there is a debate about having a clinically orientated or an economically orientated model. Where would our guests say the Irish model falls if they were to define it one way or the other?
Professor Michael Barry:
I would say it is a combination of them. There is no denying that there is an economic component to it and that it is a robust assessment of the clinical evidence and how it relates to the economics of it. For all the assessments, particularly for cancer drugs and drugs for rare diseases, we do seek the opinion of experts. We are very grateful to them. They feed into virtually every assessment we do. For example, with the chimeric antigen receptor, CAR T-cell therapy, we have very good local contacts. They have been fantastic in guiding us through the clinical aspects of that. At the end of the day, we are looking at all the healthcare benefits and all the costs, and trying to marry all of those together.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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I am not trying to pigeonhole the professor one way or the other, but on a scale where 1 is the economic orientation of the model and 10 is the clinical orientation, where is it skewed?
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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Close enough to the middle, I suppose. I read Ms McGrath's presentation. She said that in an ideal world, we would have access to OMPs in Ireland within 12 months of EMA authorisation. Is that realistic?
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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How can it be done? What needs to change?
Ms Vicky McGrath:
It would involve some type of early access programme. I am happy to hear that the chief clinical officer has initiated discussions but I am a bit surprised to hear that the patient organisations involved are not getting a seat at those discussions. It is somewhat typical sometimes of the way the system is set up. It is not unreasonable. The products are being manufactured in this country and we are not getting access to them. We need to pivot this. I think 4 is very generous on that scale of 1 to 10.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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I would agree with you.
Ms Vicky McGrath:
I would love just to refer to one particular drug, EMA approval and what it said. During the study period, patients maintained similar progress to healthy subjects. This is in relation to a life-limiting condition. There was evidence of continued benefit on follow-up up to eight years. As it is a rare disease, the studies are necessarily small and the amount of data available on side effects is limited. Given the seriousness of the condition and the lack of existing treatments, the EMA decided that the drug's benefits are greater than its risks and that it can be authorised. By contrast, the NCPE's summary states that after reviewing the data, it concluded that it is not clear that the medicine works as well or better than other ways to manage the condition. We are talking about a life-limiting condition. "Other ways" means palliative care. The NCPE stated that the price of the medicine is too high compared with other ways to manage its condition and that it believes the medicine is very poor value for money. That says it all. As a patient, as somebody living with a rare disease, as a family member, you are looking at this and asking what is going on. It is very difficult for families to see that.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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From what Ms McGrath has reported there, I would reassess my number and go closer to a 1 on the scale.
Pádraig O'Sullivan (Cork North-Central, Fianna Fail)
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What is Professor Barry's view on an early access scheme? Patients want it. Is it something we can deliver? Under the programme for Government, the Minister has commenced an investigation into it. Is it something that can be done here and what would it look like?
Professor Michael Barry:
I think it can be done. There are a number of important points. The concept is that it would start with cancer drugs but that raises the ethical issue as to whether it should only be for cancer drugs or for everything. My own feeling is that if we are going to do it for one area, we are probably obliged to do it for other areas as well. Do all drugs go into an early access scheme or just some? Of course, it would not be mandatory for the industry. If not all, how would we decide? For example, if a cancer drug improves overall survival, regardless of how much overall survival it gives, would we include that? There are many other questions. We are earnestly looking into that and discussing with stakeholders and it will be widened out.
Pádraig Rice (Cork South-Central, Social Democrats)
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We might tease that out in further questioning later. Next is Senator Nicole Ryan of Sinn Féin.
Nicole Ryan (Sinn Fein)
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It has been quite interesting listening to the debate this morning. I do not think anybody chooses to have a rare disease. When we hear people come in like the SMA group who are baring their souls in the AV room, who are crying, they are looking for something that will improve the quality of their life. It is not going to save their lives. The officials are here saying the case was not made for adults, therefore they did not do it for adults. What people who are living with these rare diseases are hearing is a question about whether their lives are worth value for money. How do we put a value on somebody's life? It is really infuriating to hear this. People are hearing a question about whether their life is even worth it. They are wondering how much their life is worth that they cannot get this medicine for themselves.
In Professor Barry's opening statement, he said that 70% of the orphan drugs reviewed this year were recommended for reimbursement despite failing the €45,000 QALY threshold. What factors are being used to justify those positive recommendations?
Professor Michael Barry:
These are recommendations made by the drugs group. The drugs group will consider the economic evidence but also a whole range of other aspects, for example unmet clinical need, and whether there are any alternative treatments available. These are the factors they will consider. The input from patient groups will also be available to the drugs group. That is for the drugs group to decide. It is not for us. We simply give our part of the information. Incidentally, we do not actually put a value on people's lives. We put a value on the benefit of a drug above and beyond what we have available to us. That is what we do. There is a slight difference. I would say the decision is made by the HSE drugs group not just on economic grounds but on a lot of other aspects as well. That is why we get positive recommendations even when the cost-effectiveness threshold is not met, which is a good thing.
Nicole Ryan (Sinn Fein)
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I will go back to exactly what Ms McGrath just referred to previously, a recommendation on a drug and the improvement it had made. We had it right here when Deputy O'Sullivan was asking a question. You guys had made a recommendation to say that it did not improve and that they should-----
Nicole Ryan (Sinn Fein)
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So you are telling me that you are not making recommendations.
Dr. Lesley Tilson:
There are increasing challenges of early availability of medicines and more limited evidence coming from clinical trials. It is the uncertainty around that. The ideal gold standard would be to have a randomised control trial but we are not always receiving that at the time we are being asked to assess these drugs.
There is a trend of making these drugs available to patients earlier, but then we have less robust evidence and that is a challenge. It is also a concern for patients because we have to look at the safety of the drugs, how long they have been studied for and the quality of the evidence. That is what is being addressed in the additional stages following EMA licensing.
Nicole Ryan (Sinn Fein)
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What are Ms McGrath's thoughts on this?
Ms Vicky McGrath:
Randomised controlled trials are not really an option for rare diseases because there are not enough patients to generate that type of data. It is, therefore, a little unethical for that to be the bar these conditions are being held to. The EMA is well regarded. It is making assessments and recognising there are risks with these assessments, as not everything passes muster a year or two later. When the EMA approves something it puts its best foot forward. The patient community and clinicians are putting their best foot forward. It strikes me that there has to be a way to say we will make a product available even though we do not have the robust data and if it does not work, there could be some kind of comeback on what has been paid to industry, or something like that. It is not beyond the wit of man to work out this kind of stuff. The problem at the moment is that patients are waiting for many years post approval before they get access to these medicines.
Deputy Pádraig O'Sullivan mentioned the number of days for approval. They are the days for the medicines that have been approved. What about the tens of others that have been sitting in the system for well over 600 days? If we approved all of those today, tomorrow the average wait would be up to 1,000 or 1,200 days. That is the real impact. We are looking at the subset that makes it through, but it is about all of those that do not make it through and those that are not even being submitted for reimbursement in this country. We need to turn the system on its head and put the patient first.
Nicole Ryan (Sinn Fein)
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I will move on because we are all trying to be professional and there is no need for the arrogance.
My next question is for Ms McGrath. We have seen repeatedly that Government strategies have stated that access will be immproved. In her ideal world, what kind of accountability mechanisms would be needed to make it happen, instead of just having promises and saying again and again that we will do this? As we saw, the report was published eight years ago and we are still here having almost the same discussions.
Ms Vicky McGrath:
Oh my lord, accountability mechanisms. I was not expecting a question like that. From the patient perspective, we need to move away from the votes and the people on the street and patients having to bare their souls in a public forum. It is very undignified that that is what is happening a lot of the time for people to get access to medicines. The Senator mentioned the spinal muscular atrophy briefing in the audiovisual room, which was heartbreaking. Grown men and women were in tears. We really have to question what is happening there. As a country, we will be held accountable by industry leaving. That is the real risk we run. We have seen what has happened in the last few months, with investments being pulled from the UK in part due to access to medicines and pricing. I fear the same thing will happen in this country. We are desperately trying to attract more innovation into the country to support the healthcare system and provide the tax euro to support it. It is counterproductive that we cannot provide the medicines to people.
Pádraig Rice (Cork South-Central, Social Democrats)
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The next slot is for Fine Gael with Deputy Roche. We will then go back at the top of the list, with Senator Rabbitte followed by Deputy Buckley and Senator Boyle.
Peter Roche (Galway East, Fine Gael)
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The witnesses are welcome. Like other speakers, I have a huge interest in the conversation that has taken place thus far. One of the things that strikes me about delays in providing a drug to people, whether they have been recently diagnosed or have been diagnosed for quite a while, is that they might then discover that somewhere, sitting on a shelf in a stock room there is a drug that could potentially change their lives. There are bureaucratic and convoluted processes and procedures and obstacles that are manufactured and there is a lack of will and sometimes of investment. There is absolutely no denying that economics should not come into it when it is about quality of life. Every family has had an experience or knows someone who is waiting for a life-changing drug. Imagine the torment and soul-destroying feeling people would have when they think there is potentially a drug, but they are looking at two or three years before they can get access to it. That is what I find difficult to understand.
I appreciate and respect the job Professor Barry has to do, but we are all aware of what happened when the pandemic came. Mountains were moved to get access to a drug which might have saved millions of lives around the world. It was done because there was a human or medical emergency. Every diagnosis is a medical emergency that deserves the same level of empathy, compassion and support in providing the drug that will give the patient quality of life. Quality of life is paramount in all this. Thankfully, we have pharmaceutical companies that are able to devise drugs in response to a diagnoses. They do so in the hope it may be the life-changing intervention patients require, yet access is an anomaly.
I would like to think we are all in this together because it is about people's well-being and lives and about early intervention and access. What does Professor Barry believe needs to change to have a better process under which people will have access?
Professor Michael Barry:
First, I believe in the concept of getting value for money. When we follow this process we reduce expenditure on medication, which makes money available in other areas of the health service.
I agree with the Deputy that we would all like to see a faster, more efficient process. That is what we are moving towards. In recent years, we have seen investment in that, and quite rightly. We take approximately 222 days for orphan drugs and 219 days for cancer drugs. I want to see that time reduced significantly because I get what the Deputy is saying, completely. We are only one part of the delays, as the Deputy will see. However, we will focus on ourselves for the moment. I want to see the process get better and I believe we can do better in it. On the issue about early access, as we mentioned already, it is something we are beginning to open up.
I am hopeful that will also be brought to bear and that we will make drugs available and do the assessments. Then you can decide later what you are going to pay for the drug. It should not surprise us in the area of therapeutics, but with cancer drugs, for instance, not all of them will prolong your life. Not all cancer drugs will improve your quality of life. In fact, fewer than half will. That is the reality. I have no issues with paying top dollar for drugs that work very well. It is when they do not that is the issue. Then the question is whether we would be better putting those millions of euro into somewhere else, like home help, disabilities or acute hospital care. Those are the challenges, as members well know. We can do better and we can do it faster. We should assess these drugs.
Peter Roche (Galway East, Fine Gael)
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The value for money part of it is something I have a concern about. I will park that for a second because Professor Barry said twice that we could do better. In what regard, or what is it he believes the NCPE can do to make it better or to expedite the process? Where are the deficits and how can that gap be bridged?
Professor Michael Barry:
It was a resource issue. There is no question about that. The more resources we have, the better and the faster we do it. For example, I said last year that we did 75 or 76 assessments. This year, we have already done 79 and we are beginning to see the impact of additional resources and new people. I want to get the time down as low as we possibly can and we will keep doing it. I want to get drugs to people as well. That is our aim. The only thing I would say is we advocate for everybody - not just one sector but across the therapeutic areas. I want to see it done faster and that is what we will do. We give our guarantee we will keep doing that as best we can.
Peter Roche (Galway East, Fine Gael)
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In the 30 seconds I have left, I will say that I would welcome something along the lines of a progress report some time in the future. This is a very critical journey we are all on together in terms of access.
Anne Rabbitte (Fianna Fail)
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I thank the Chair for facilitating a substitution. I am here on behalf of Senator Costello. She sent me a statement I want to read in and then I have a rake of questions.
I would like to apologise for my absence today as this is an issue that is close to my heart. I would also like to thank the witnesses for coming before the committee today. As some of you may know I have been working with families affected by Duchenne muscular dystrophy and access to the drug givinostat. Until a few months ago I had never even heard of DMD. Then a young boy from Tallaght named Archie Ennis was diagnosed with the disease. I saw not only a family but the entire community heartbroken and desperate and I knew something had to be done. As someone who had never heard of this disease six months ago it is now never far from my mind. Since then I have met with other families affected by the disease and have been working closely to push for access to the drug. I organised demonstrations outside Leinster House earlier this year where hundreds of people gathered to raise awareness of DMD and demand urgent action. On the same day I hosted a briefing for TDs and Senators to educate Members of the Oireachtas about the disease and the urgent need for treatment. This has become a personal plight of mine. I have met the families, I have seen the love, the pain and the determination. I stood with them outside Leinster House and I will continue to stand with them until children get access to the treatment.
Senator Costello has two questions. The first is on how many orphan medicine applications have met the 180-day reimbursement decision timeline in the past five years.
Anne Rabbitte (Fianna Fail)
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Very few. That is okay. Senator Costello also wants to know what factors lead to it taking over 700 days on average for Irish patients to access orphan medicines approved by the EMA.
Anne Rabbitte (Fianna Fail)
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Perfect. I thank Professor Barry.
As my colleague, Deputy Daly, said earlier, this is a very difficult subject and what we all want is a fair, timely system. I pay tribute to Rare Diseases Ireland and the "I am Number 17" campaign. I also acknowledge that extra funding has been got and Professor Barry's team has grown. This is not easy for anyone, but it is important that the team is growing and more people leading to more outcomes is what we hope for. I have a question I want to leave until last but I am going to tell the witnesses what it is so that they can think about it. I do not want them to answer until the end. My colleague, Deputy O'Sullivan, who is beside me, has a Bill on orphan drugs going through the Dáil. I want to know whether the witnesses are aware of it and if they have an opinion on it. I will leave that with them until the end.
Do the witnesses think the current system is doing a good job? That is a yes-no question.
Anne Rabbitte (Fianna Fail)
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All right. Does Professor Barry think the early access programme is a good idea for drugs? I am going to go further on early access, so what is his opinion on it?
Anne Rabbitte (Fianna Fail)
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I really welcome Professor Barry's answer on that. I want to go another step. If we do not change the way we are doing, it the assessment process for patients will continue to face delays in getting potentially life-changing treatments. Is it possible to establish an early access programme for rare disease treatments to allow patients with little or no options to avail of treatments early while still assessing them for reimbursement? I am thinking of selumetinib.
Anne Rabbitte (Fianna Fail)
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They are already in that whole process but they cannot get early access. The compassionate grounds were stopped. It is on that element I am asking the question. There are so few patients in the rare disease category that they cannot compete with cancer patients or heart and stroke patients. The drug I have mentioned pertains to two a year. You cannot compete. Other countries have separate assessment pathway criteria for orphan medicines and early access programmes exist in other countries. Have Professor Barry and his team looked around to see what is happening elsewhere?
Professor Michael Barry:
Yes. I am very familiar with Professor Postma and what is happening in the Netherlands. As he said, we actually compare very well. In broader terms in relation to early access, I made the point earlier that if you are willing to look at early access for cancer drugs, you have to look beyond that. It would be hard to defend not looking at early access for orphan drugs, and more widely.
Anne Rabbitte (Fianna Fail)
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Does Professor Barry agree on separate pathways, though?
Anne Rabbitte (Fianna Fail)
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Early access is one thing but it is the pathways that determine the access.
Professor Michael Barry:
The Senator is right that there are certain differences between the drugs and differences in the evidence and so on. I do not see why we would not look at that area, but it is only fair to say the discussion on early access in relation to cancer is just recent. The Senator might argue it should have been long before now, but that is where we are at.
Anne Rabbitte (Fianna Fail)
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What about index linking the pricing? Currently, we do not do that. We have a set threshold. Did Professor Barry say that was €45,000?
Anne Rabbitte (Fianna Fail)
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What would it take for the NCPE and HIQA to review the threshold and take similar action?
Professor Michael Barry:
HIQA produces the guidelines in relation to health technology assessment and we work to those guidelines. Obviously, if the guidelines were that we had to work to a different threshold, so be it, we would work to a different threshold. I do not have any philosophical problems with that.
Anne Rabbitte (Fianna Fail)
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I was a little struck earlier by Professor Barry's over-and-back with Senator Clonan. The reason I was a little struck is that families play a huge role in this. They are the principal caregivers, and for decisions to be made without their having a role in the decision process is a little disingenuous. It goes back to the rare diseases technical review committee and families, but who invites them in? The review committee invites them in, but in what way does that process work? I do not understand it.
Pádraig Rice (Cork South-Central, Social Democrats)
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Just a brief answer.
Anne Rabbitte (Fianna Fail)
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And does Professor Barry support Pádraig O'Sullivan's Bill?
Anne Rabbitte (Fianna Fail)
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So Professor Barry does not agree on having-----
Anne Rabbitte (Fianna Fail)
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He has said to me he agrees on assessments but he does not agree with me on-----
Pádraig Rice (Cork South-Central, Social Democrats)
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Just a brief answer to the question, please.
Professor Michael Barry:
I do not agree with the Bill because, essentially, its implications are enormous for the drugs budget. Already this year, if we go to September for the orphan drugs and if we imagine that Bill were here, whereby there is no assessment, it would cost €140 million. It will probably cost €200 million a year. That is what it will cost. If people want to vote for that and want to pay that much money, so be it - I will not take it personally - but, from a financial aspect for the HSE, I would say no. I am very clear on that.
Pádraig Rice (Cork South-Central, Social Democrats)
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We move to the next slot, which is a Sinn Féin one. Deputy Cullinane has been substituted by Deputy Buckley.
Pat Buckley (Cork East, Sinn Fein)
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I thank all the witnesses. There is a big advantage when you come in near the end of the full conversation in that you have nearly all the answers and you can break them down. In our job, specifically with the HSE and so on, we will ask a question and we will get a response, and then we go back and ask a question about the response we got. It is about deciphering stuff down.
I have made a few notes on this. This is about patients, human beings and families. I can understand where both sides are coming from here. Ms McGrath mentioned at the start of her contribution that she was here seven years ago and she is here again, and access to this, to anything we are looking for, has not changed in seven years. There is no progression. I was lucky with one or two other Members here to have sat on the committee that dealt with the Sláintecare report, which was about equality, putting patients first and the idea that everything should be based on your needs and not your means, that is, money. I will probably get into trouble for saying this but I do not care. Trouble is my middle name. When I listen to the witnesses saying they have to fight for proper prices for these orphan drugs and so on, it keeps coming into my head that pharmaceutical companies do not do cures, they do customers. Professor Barry himself said earlier that some of these drugs, even those for cancer, do not even work, so how do we find a balance?
The reason I ask these questions is that a very good friend of mine has a daughter who has Fraser syndrome, the incidence of which is one in 1.5 million or something. There is very little that can be done unless it is operations removing organs as she gets older or whatever. I look at her and think, "How do we support this family and give them better quality of life?" There are no guarantees. Any of us can walk out the door here and get knocked down going across the road or whatever. I listened to the witnesses speak about the Dutch model and the way the Dutch seem to have a stronger power in negotiating. When I hear "negotiating", it is price versus patient. On one side there are the cogwheels of how the system is working or not working, and on the other side the witnesses are like the oilers of the cogwheel, but sometimes they do not have enough oil so the cogs do not move and the whole thing is siloed whereby nothing is resourced properly. It is like being on a football team: if you do not have a full team, your chances of winning the match lessen.
We have pharma here that is funded by taxpayers' money. We put in the funding, they produce the drug, they get the pat on the back and they come back and say, "Thanks very much for that. We appreciate it. Now we have it and you are not getting it unless you pay extortionate money." They do not give one damn about the patient. They do not even call them people; they are patients. Patients equals money. The system is very much swayed towards pharma. It is as simple as that. It is swayed towards the producer. I have had the privilege of having sat on a lot of different committees here, between autism, disability, Sláintecare and mental health. These are all the knock-on effects boxed off into one for each and every one of these patients. When you are told you have a rare disease, we are normally, as citizens, as human beings, extremely reactive and very slow to be proactive. Put yourself in one of these people's position. They are told, "You have a very rare condition. There could be a drug in about three or four years' time. It might not work but you are going to have to wait another three or four years to find out anyway."
That is not a system that is working. It seems that in a lot of organisations across all the services I mentioned a while ago it is all silos, and silos slow down things. As Ms McGrath mentioned, we are in the European Union. God, they are very fast here to push the European army on us, but we cannot bring on a European cost-benefit analysis of human beings' health and welfare if we can pool our resources and get things out to people faster. Is that not a better outcome than saying to the pharma companies, "You beat us 3-0 last week in the match but we are playing you again in two weeks' time, and hopefully we will have a better result"? It is a football match or a hurling match - whatever you want to call it - but at the end of the day the winners are the producers of the drug. The guinea pigs are the people who have the disease. I do not want to sound derogatory, and people will take that out of context, but there are other words I would use that would lapse more into profane language, which I will not use. We, as legislators, are all human beings. We might not all have the same politics but we go into certain committees here, and the Sláintecare one had probably the best result. We all agreed to sign off on that for the betterment of our whole society, and it has been slow. As a Legislature, could we do something within the system that streamlines everything, including the brass tack supports? Ministers have statutory instruments to change things straight away. Why can we not use stuff like that?
If there were just one line, if the witnesses could get up in the morning and say, "Right, I need to streamline everything and fix it. I need this to happen", I would like to hear from both sides on that. Thank you, Chair, for your patience.
Pádraig Rice (Cork South-Central, Social Democrats)
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We will take a response from each of the witnesses.
Pat Buckley (Cork East, Sinn Fein)
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The witnesses should understand my Cork accent very easily.
Professor Michael Barry:
It is. Fifteen years ago, we thought statin therapy was expensive at €500 per patient per year. We are now talking about millions.
I have always said there is a way to get your drug reimbursed quickly in this country, and that is to price it fairly. Drugs do not have to go into a full health technology assessment. We have a rapid review system which is done in 30 days. In fact, we did givinostat in nine days. If they come in and price it appropriately at that point in time, we say yes. That is all I will say.
Pádraig Rice (Cork South-Central, Social Democrats)
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We might just get a response from Ms McGrath. There are only 30 seconds left in this slot.
Ms Vicky McGrath:
Discussions about pricing are not the remit of patients. We are looking for the best healthcare available to other citizens of the EU, other citizens of Europe, and what can be done to make those available in Ireland. I have heard a lot of "stuff is available here and not there".
We are way behind our comparators in Europe and we have to do something to turn this on its head. I think an early access programme is the start of that. Let all of those negotiations happen in parallel. Argue over pricing, fine, but keep us out of it and make sure that we have access to these medications.
Pádraig Rice (Cork South-Central, Social Democrats)
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The next slot belongs to Fine Gael and I call Senator Boyle.
Manus Boyle (Fine Gael)
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I thank the witnesses for coming in today. To be honest, this is totally new to me and makes hard listening. My fellow Senator who is not here today, Senator Teresa Costello, drew my attention to the situation with givinostat. On one of the first days I came here, Senator Costello had people in the audiovisual room where I met two lovely young fellas from Donegal from my own area. I learned that one brother gets givinostat across the Border while the other brother does not. I cannot understand the reason givinostat, which is something that was applied and went through all the mechanisms in England and cross-Border, cannot be fast-tracked here to help the other brother. Perhaps I look at things differently because I look at this from a personal point of view. That mother and father stores medication in their fridge for one of their boys but not for the other. To me, that situation is totally wrong. If a medication was approved in England a few years ago then why can we not fast-track it through here?
Givinostat has been mentioned a couple of times. When will givinostat be made available here? When can I say to the Langan family in Donegal that their second son can get givinostat? Will it be a month, two months or when? The current situation is soul destroying for families. I have met the family and understand how they feel. The child is getting worse. If I had the money myself I would buy the medication. People must consider the fact that families are personally affected by this situation. It has been proved that givinostat works for one of the siblings, so why is there a delay in rolling out provision? I seek a definitive answer as to when givinostat will be made available in Ireland.
Professor Michael Barry:
I will answer that question. I will say that it does not just go one way. For example, the drug Orkambi, for cystic fibrosis, was available here for nearly two years before it was available in the United Kingdom. So it does not always go one way.
In relation to this drug, as I mentioned to Senator Rabbitte we assess this in the rapid review system as fast as we possibly could, and literally within nine days. At the moment we are waiting for the manufacturer to give us the evidence and I am told that this is going to be very quick. All I can say is that we will do that assessment as quickly as we possibly can.
When will it be available? That is a challenging question. The answer is I do not know. What I can tell the committee is that I would hope that if we get the information that we require from the manufacturer that we will have our opinion on this, hopefully, by the end of the year or the start of 2026. We will have that done very quickly, I can assure the Senator. Then it would be the decision of the Health Service Executive to decide whether it was going to pay for the drug or not. We will not be found wanting with this. We understand the issues here. The Senator laid them out very well and I understand it completely.
Manus Boyle (Fine Gael)
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As my fellow members of this committee said earlier, it should not be down to politicians to come in here to push for a drug for a child. I believe that the system is wrong. We should have a system whereby if a doctor recommends a person gets a drug that politicians are taken out of the equation. I know no more about givinostat than the professor would know about fishing or anything like that so the current position is totally wrong.
Manus Boyle (Fine Gael)
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When a medical person like Professor Postma or a consultant recommends that a child or adult should get a drug, then the political end should be removed. It seems to me that it is all down to political pressure and the more political pressure applied, the greater chance - perhaps - of living.
Professor Michael Barry:
I will say one thing in general about that. Over the past 27 years, even though it has been suggested that I should probably retire, I have never been exposed to political pressure, to be fair to everybody. We have always been left to get on with our work and we have done it as earnestly as we possibly can. I would like to thank the political system for supporting us. We have never felt under pressure, and we have always done the work as fast and as well as we possibly can. That will continue for as long as I am around. I thank all of you for supporting us.
Manus Boyle (Fine Gael)
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Earlier we talked about the early access scheme. We, as a committee, should push really hard for an early access scheme. I say that because of the experience of my cousin, who has now passed on. He was in America and the medical profession did everything they could for him when he was ill. He told me himself that he participated in clinical trials. He received this, that and the other, and he said he was proud of himself for doing that. So we must try and see what is going to work here too.
Manus Boyle (Fine Gael)
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We, as a committee, to help the organisations present, should work on securing an early access scheme too. Nobody wants to suffer in pain so the easier we can make anybody's life then the better and that is what we are all here for.
Pádraig Rice (Cork South-Central, Social Democrats)
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Next to speak is Senator Maria Byrne.
Maria Byrne (Fine Gael)
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I, too, want to raise the same issue. I have had dealings with a family whose grandchild lives in Wexford. The child is six years of age and is badly in need of givinostat. It is a fact that in April 2025 the European Medicines Agency granted conditional approval and on 6 June the European Commission granted conditional approval.
Maria Byrne (Fine Gael)
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It is now mid-October but we are no further on. Professor Barry has said his organisation is having engagement. How long will that engagement take because many children are waiting to receive this drug?
Maria Byrne (Fine Gael)
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I heard Senator Boyle talk about the fact that some people over the Border have access to the drug, and how a family he knows where two children are affected but one child is in receipt of givinostat while the other child is not.
Professor Michael Barry:
The Senator is talking about the timeline between 6 June and 5 August when the relevant company put in a pricing and reimbursement application. It has nothing to do with me. I have no hand, act or part in that. All I can do is take ownership of what we have done. When we were asked to look at it on 5 August, we finished on 14 August. Where is that now? It is a very high-cost drug and has a significant budget impact. There is no question about that. So we asked for more information from the company that is very happy to provide it and I understand that the information will be with us in a month or so. When we receive the information I can assure the Senator that it will receive our full attention and will be done as quickly as possible. It will be done properly but it will be done quickly.
Maria Byrne (Fine Gael)
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I have a question about what happens once the information is received.
Maria Byrne (Fine Gael)
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If the National Centre for Pharmacoeconomics is happy with the information, does that mean the drug will become readily available and what are the next steps?
Professor Michael Barry:
First, we make our report public. It is on the website so anybody can look at it and the timelines are clearly outlined. Next, we send that report to the HSE and it goes before the drugs group, which make the reimbursement recommendation. The senior leadership, usually very quickly, respond to that. Once we have done our bit, we send it immediately to the drugs group, and then a recommendation is made and it is usually fairly quickly.
Maria Byrne (Fine Gael)
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Is Professor Barry hopeful that the NCP will hear within the next month from the company?
Maria Byrne (Fine Gael)
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I want to discuss rare diseases and orphan drugs. Why is there a long waiting time of 710 days compared with other countries that seem to have a faster process? Is the process fit for purpose? Does it need a review or changes to be made to fast-track the process?
Professor Michael Barry:
You can always do better. We can always do better. We feel we can do better but, as I have said, we are responsible for 19% or 20% of the timelines. There are other areas that can do better as well. We can all do better, but from our point, we will certainly try to do better than the 220 days that it takes us, and I think we are getting there. As I was saying to Senator Rabbitte earlier on, we have done more assessments this year by September then we did in the entirety of last year. This is reflective of the support we have got, in fairness, from the Department and with all the members' support. We are very grateful for that and we will do it as quickly as we can.
Maria Byrne (Fine Gael)
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Professor Barry referred to the whole transparency side and decision-making. What steps should be taken or are there more steps that should be taken to improve that whole area?
Professor Michael Barry:
Yes. This is very important. We made a decision many years ago to put timelines on our website so people know exactly where it is and what is going on until the time our assessment is over. The HSE has now introduced a system where you can track the drug throughout the process. I very much welcome that. It is up and running now so we can see it. I get it. A lot of people would have come to me and asked where it was in the system, that they could not see it, but now they can, and that is a good development. Things are changing and they are getting better. There are massive challenges but we will deal with them as best we can.
Maria Byrne (Fine Gael)
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Great. I have one other question to do with the oncology and the non-oncology orphan medicines that are not available in Ireland. What proportion have seen an application made to the reimbursement assessment process by the marketing authorised holders?
Professor Michael Barry:
This is a big issue. For up to 40% of orphan drugs we do not have an application for pricing reimbursement. I cannot do anything about that; I honestly cannot. It is probably to do with finances, as Deputy Buckley was saying earlier. Why do they not launch here? They launch in big markets where they get more resources, essentially. That is an issue. I get Ms McGrath's frustration with this but we cannot assess a drug if there is no reimbursement application, unfortunately.
Maria Byrne (Fine Gael)
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On the orphan drugs that fail the HTA due to restrictive cost-effectiveness, is the Department considering a different valuation framework or just a threshold for these medicines?
Professor Michael Barry:
A point I was making earlier was that there is only one drug that actually satisfied the criteria but we reimbursed 12. The drugs group in its decision-making is not just taking into account what we say. It is taking into account lots of other parameters like unmet need, no other available therapies, and so on. That should be reassuring for the committee in the sense that it is not purely down to economics. If it was, we would have reimbursed only one orphan drug this year whereas we have reimbursed 12 so far.
Maria Byrne (Fine Gael)
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How are patient organisations and clinicians meaningfully involved in the HSE reimbursement process for orphan drugs?
Dr. Lesley Tilson:
For the NCPE process we engage with clinicians for all of the assessments that we undertake. Then similarly for patient organisations, we proactively reach out to patient organisations to invite submissions for all of the HTAs . For the wider reimbursement process there are clinicians on the drugs group and there is a public representative on the drugs group. There is also the rare diseases therapy review committee. Professor Barry may know the composition of that.
Professor Michael Barry:
I used to be the chair of it. Essentially, it was people who had expertise in the area of paediatrics and adult medicine. We would invite patients and patient groups to come in and present to us. I found it really useful, particularly around the time of spinal muscular atrophy, SMA. I thought the testimonies from patients and parents were really moving.
Pádraig Rice (Cork South-Central, Social Democrats)
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That is all the questions from our members and substitute attendees. Deputy Aird has also joined us and he has just one question to ask.
William Aird (Laois, Fine Gael)
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I thank the Chairman. I want to ask about the SMA drugs for people who were born before 1994. Could I have an update on that please?
Professor Michael Barry:
The pricing reimbursement assessment at the time looked at this. The drug was quite expensive, with Zolgensma costing €2 million. Essentially, it was based on the clinical evidence. One may agree or disagree with that but it was based on the fact that the clinical evidence suggested that, the younger a person was, the better they would respond to the drug. The 18-year timeframe was introduced at that stage. As I mentioned earlier, that can be reviewed if necessary but that is where the decision came from. It was based on the clinical trial data at that particular point in time. I have not looked at it recently in the sense of what that timeline should be but that is how it came about. It was based on the clinical trial data at the time that the reimbursement recommendation was made.
William Aird (Laois, Fine Gael)
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I thank Professor Barry for this reply. I have heard more from him than anyone and I have been researching this since I was elected. How do I go back and tell two first cousins in my own hometown in Portlaoise that this is about money at this stage? Surely to God the date you were born should not make a difference. These two people happened to be born just before 1994. If they had been born after 1994 they would be on the drug. Surely that is not fair, is it?
Professor Michael Barry:
I know where the Deputy is coming from on this. We do know that drug therapy, not just in this area but in other areas, can be impacted by your age. You can have drugs that work better in younger people as compared with older people, and vice versa there are drugs that work very well in elderly people that would not be administering to younger people. You can have variations in drug response but whether a drug should be made available or not based on age is a difficult decision. I know it is not one that was made lightly but, as the Deputy has said, maybe it needs to be looked at.
William Aird (Laois, Fine Gael)
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Who made that decision? Is the professor trying to tell me, or is anybody trying to tell me, that a person born in 1995 should be treated differently from a person who was born in 1994? That does not stack up in all fairness. How could anybody come up with that?
William Aird (Laois, Fine Gael)
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Tell me who came up with it and how do we change it?
Professor Michael Barry:
I will tell the Deputy how decisions are made. Decisions are made by the drugs group, which is a wide group of people from the health service, including lay people. They make recommendations, not me. I do not make recommendations. They make recommendations to the HSE senior leadership team and the HSE senior leadership team ultimately makes the decision on reimbursement based on what they have been informed. That is how it is done. If one wants to look at it again then, essentially, it has to be looked at through the HSE leadership team.
Pádraig Rice (Cork South-Central, Social Democrats)
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That brings us to towards the end of our considerations on this. I will offer my own reflections on it. As Deputy Buckley said, at one end we have patients who are really desperate, really want access to medicines and really need them, and on the other end we have those who want to make a lot of money off it, with a lot of political pressure on both sides and a difficult process in the middle. A lot of political pressure is put on people around this and there is a lot of keen interest from members on it. It is a difficult one to tease out and to figure out exactly how to do it right. I do not envy the position that people are in who are trying to get the best model with both of those things and a lot of pressure around it. No doubt it is a matter we will come back to again. Many of the Members in the wider Oireachtas area are quite interested in this area. Maybe it is one we will have future engagement on.
I thank the representatives from the NCPE and Rare Diseases Ireland, and Professor Postma, for their engagement with the committee today, for their time preparing statements, and for their frank answers and engagement. I really appreciate that. The meeting will now adjourn until 3 p.m. next Tuesday, 21 October when we will meet in private session.