Joint Committee on Health debate - Wednesday, 21 Nov 2018

Evaluating Orphan Drugs: Discussion (Resumed)

Chairman

The purpose of this morning's meeting is to consider the implementation of the recommendations in the committee's report on evaluating orphan drugs, which was published last February. We will hold two sessions on the topic. The first session will involve Rare Diseases Ireland, which is the umbrella body for patients with rare diseases and will present to the committee first. The second session will be with officials from the Department of Health and representatives from the HSE and the National Centre for Pharmacoeconomics.

On behalf of the committee, I welcome Mr. Philip Watt, chair of the Rare Disease Taskforce and CEO of Cystic Fibrosis Ireland; Ms Vicky McGrath, interim CEO of Rare Diseases Ireland; and Dr. Derick Mitchell, CEO of the Irish Platform for Patients, Organisations, Science and Industry.

I draw the attention of witnesses to the fact that by virtue of section 17(2)(l) of the Defamation Act 2009, witnesses are protected by absolute privilege in respect of their evidence to the committee. However, if they are directed by the committee to cease giving evidence on a particular matter and they continue to so do, they are entitled thereafter only to a qualified privilege in respect of their evidence. They are directed that only evidence connected with the subject matter of these proceedings is to be given and they are asked to respect the parliamentary practice to the effect that, where possible, they should not criticise or make charges against any person, persons or entity by name or in such a way as to make him, her or it identifiable. I advise witnesses that any opening statements made to the committee may be published on the committee website after the meeting.

Members are reminded of the long-standing parliamentary practice to the effect that they should not comment on, criticise or make charges against a person outside the House or an official either by name or in such a way as to make him or her identifiable. I invite Ms McGrath to make her opening statement.

Ms Vicky McGrath

My name is Vicky McGrath and I am interim CEO of Rare Diseases Ireland. We are the national alliance for voluntary patient-led organisations for people affected or at risk of developing rare diseases in Ireland. We are committed to the identification, treatment and cure of rare diseases. Today, I am joined by Mr. Philip Watt, chair of the Rare Disease Taskforce and the Medical Research Charities Group and CEO of Cystic Fibrosis and Dr. Derick Mitchell, CEO of the Irish Platform for Patients, Organisations, Science and Industry.

Orphan drugs are defined as medicines that prevent or treat rare diseases. There are an estimated 6,000 to 8,000 rare diseases that we know of with more being described. Collectively, rare diseases may affect up to 8% of the population. It is estimated that rare diseases affect 300,000 people in Ireland during their lifetime. Rare diseases have a significant impact on our citizens and an associated economic impact on our society. A recent survey across Europe revealed that more than 70% of people living with rare diseases have difficulties with daily activities, motor and sensorial functions and social interaction. A total of 30% of carers spend more than six hours per day on illness-related tasks. Over 95% of primary carers are family members with the vast majority being women. A total of 70% of patients and carers had to reduce or stop their professional activity due to the disease. There is a significant mental health burden with rare disease patients and carers being three times more likely to feel depressed compared to the general population.

Orphan drugs provide an opportunity to improve lives considerably or even cure disease. During 2017, the members of this committee met with two Irish rare disease patient organisations - the Alpha One Foundation and Muscular Dystrophy Ireland. During these meetings, members heard about the challenges facing these organisations' patient communities around access to novel and life-saving orphan drugs. These medicines have been found by regulators to be both safe and effective and have been approved for reimbursement by many of our peers across Europe yet they are still not available in this country. Over the intervening 12 months, the committee has, no doubt, encountered many other patients and patient groups with similar stories. Access to life-saving and curative therapies in this country is lagging behind that of our neighbours. This situation must change. The reimbursement system in Ireland continues to fail the patients it is supposed to serve. We must take an innovative approach to reimbursement and design a system that is fit for purpose - a system that is not only suited to so-called "common" diseases but also addresses the needs of rare disease patients. Such a system will ensure that all members of our society can contribute maximally.

There are no comparative therapies for most rare diseases. In most instances, the patient will not transition from one therapy to a better one. Orphan drugs constitute the only game in town and provide the best opportunity to slow disease progression and improved quality of life. As with cancer patients, we are seeking to avail of the best therapies today in the hope of being in a position to avail of new and better therapies in the future. Many rare disease patients are not being afforded this chance at a better and longer life. We must build a reimbursement system that provides that bridge to the next therapy. We must have a system that is robust enough to address challenging decisions. We know curative gene therapies are on their way. These transformative therapies will be expensive.

We all want value for money but how does one define value when one considers the ability of an individual to walk unaided rather than being wheelchair bound for decades or the child who will have have his or her vision restored and can thus integrate normally into classroom life? What type of society do we wish to have ten years hence? Today we are on the cusp of a step change when it comes to rare disease patient care. Access to the European reference networks as a result of the EU's cross-border directive will allow rare disease patients in Ireland access to the best clinical minds across Europe. Our care will be on a par with that of rare disease patients throughout Europe. Our care pathways will be best in class and we will be provided with opportunities to enrol in clinical trials. Innovation will be at the heart of our care. However, if the reimbursement system in Ireland does not change we will fail at the final hurdle and continue to be denied the therapies we need.

We acknowledge that progress has been made over the past 12 months, but it is very slow and just too late for many patients. In recent weeks the rare disease technology review committee began its work. Patients finally have the opportunity to provide real world experience of living with a condition and the impact that a particular drug may have on quality of life for both the patient and his or her carers. We are not there to give a sob story but as experts on our diseases. More significantly, patients are often members of the committee and we finally have a seat at the table as equals. We are no longer viewed simply as stakeholders. We are decision makers within the committee and have an equal say in the recommendations that the committee will provide to the HSE leadership, where the ultimate decision around reimbursement lies.

We understand that these are very early days for the technology review committee and we caution that more time is required to bed down the processes and learn from experience. Nonetheless it has finally been recognised that patients have a valuable contribution to make to the discourse and decision making process. To build on this we seek a seat at all stages of the process, be it horizon scanning, the pre-submission consultation or the HSE's decision making meetings. We have a growing cohort of educated patients who understand the process and the decisions involved. If we do not have a seat at the table, are we just on the menu? We have no interest in recommending treatments that do not work. We do not wish to take medicines that will not work and we do not want to waste money. We believe that innovative approaches to reimbursement decisions are required. Payments based upon defined clinical outcomes, so-called pay for performance, or guaranteed access to future improved therapies should be the norm, not the outlier.

Another development in recent months is Ireland joining the BeNeLuxA initiative, which aims to ensure timely access to, and affordability of, innovative medicines. Disappointingly, since the Minister for Health signed the agreement five months ago there has been no evidence that we have made any progress.

Finally, orphan drugs are at the cutting edge of medicine. We are moving into the realm of personalised or precision medicine. Orphan drugs should only be provided to those who will benefit from them. They are not designed for the wider community or even for every patient who might have a particular rare disease. A genetic diagnosis will be required for many orphan drugs that will come onto the market. Genetic services, by which I mean consultant geneticists, genetic counsellors and genetic testing facilities, fall far short of what we need in Ireland. Waiting lists are growing by the day. Current services are under-resourced and overwhelmed. We must address this basic building block of medicine in the 21st century if we are to have any hope in the future. Genetic diagnosis coupled with patient registries will ensure that only those who will benefit from the drug will be put on the drug. Registries will also allow for post-marketing data collection, enabling the pay for performance reimbursement model to be employed.

In summary, we have three messages to deliver today. First, we must develop an all-inclusive, transparent and accountable reimbursement system that will withstand the challenges that lie ahead. We must think creatively and innovatively. We must build a system that ensures that the needs of every patient in the country are addressed. Just because somebody is the only one with a particular rare disease does not mean we are indifferent to that person's needs.

Second, we must build genetic services capacity that meets the needs of not just the rare disease community today but those of the nation. Personalised or precision medicine is not just a dream. It is knocking on our door and the only way we can take part in this revolution is by ensuring that every patient has the opportunity to know more of his or her genetic composition.

Third, we are not just a voice or even stakeholders, we are decision makers and must be allowed to take on this role in the development of our health service. Of all the interested parties, patients have the most risk. It is our health that is at stake. There is a perception among patients that the health authorities fear our input. We are not to be feared. We are the experts on our diseases and we must be involved in all decisions involving our treatment. Revelations within the health service in Ireland over the last 12 months have shown that patients must be included in all decisions - nothing about us without us. I thank the committee for its time.

Chairman

We will open the discussion to members. I call Deputy Donnelly.

Deputy Stephen S. Donnelly

I thank the witnesses for their time today and, more broadly, for all the work they do. The advocacy work and the pressure and technical expertise they bring to the table are incredibly valuable. I realise they can feel they are ploughing a lonely furrow for much of the time so I wish to acknowledge all the work they do on behalf of many men, women and children throughout the country. They should keep it up and we will support them in any way we can.

I wish to focus on some of the possible solutions. I keep hearing that Ireland is lagging behind. We signed up to the BeNeLuxA initiative. It agrees on a certain orphan drug yet Ireland does not introduce it. I saw data recently that showed we are the last or one of the last countries in the EU to introduce drugs that have been approved across the EU. It is probably not always the case but that is what I hear from the parents of sick children, experts such as the witnesses, clinicians and the pharmaceutical companies. Everybody seems to be saying that Ireland is last or close to last with regard to some of the incredible drugs that are coming through. The witnesses referred to that several times in their statements. Why do the witnesses think we appear to be slower than most of the rest of Europe in giving people access to these drugs? What can we do in real terms to speed up the process?

Mr. Philip Watt

That is a fantastic question. My sense, and the committee will probably hear this from the representatives of the National Centre for Pharmacoeconomics and the HSE later, is that some positive things have begun and we welcome those. They include the BeNeLuxA initiative and the rare disease technology review committee. They are moves in the right direction. Professor Michael Barry would probably say that we need to tweak the system rather than reform it. We are saying we need to go a little further than that. What we are asking the Government to do is set out an overall policy position which would cover everything, not just the minute detail of appraising a particular drug, representation on one or two committees and so forth. It would look at the entire process and, indeed, the cost, because we see it from the committee's perspective as well. These drugs are a horrendous price. We would all say that we will put on our Irish jerseys and support the Government to get the best and fairest price it can for these drugs. However, that entails us working together in a more partnership way than has been the case up to now. Certainly, the last thing we want to do is what Cystic Fibrosis Ireland had to do which was protest outside the Oireachtas. We would far prefer to see the system being reformed. It is going in the right direction but it must be far more comprehensive than it has been to date.

Dr. Derick Mitchell

I agree with Mr. Watt. An additional element we would like is probably more transparency in the process. It has been very frustrating to have to go to multiple different sources to get the facts about the delays. A number of studies have been performed but the system does not seem to release information readily. From a patient perspective to know where a particular drug is in the process at any stage would be one thing, but also to know which stakeholder has been holding up the process would be very beneficial as well.

Deputy Stephen S. Donnelly

I have a question on that. I was going through the process on this last week and the view I got was that the clinical evaluation is quite open and moves at a decent clip. The National Centre for Pharmacoeconomics, whether one agrees or disagrees with what it decides, arrives at a recommendation quite quickly. It has its €45,000 per year measure, with which we can agree or disagree, but it is there and the centre moves through that quite quickly.

Their recommendation goes into the HSE or the Department and at that point it becomes completely opaque. Does that sound about right to the officials or would they describe it differently?

Mr. Philip Watt

That is part of the problem but we were advised that virtually no rare disease drug will get through the system as it stands because of the way the drug is measured. It is often done through the health technology assessment process which looks at one key outcome for a drug, but science has moved on. These new drugs are treating the underlying cause of the disease, not just the symptoms. The drug must be measured by several outcomes. There must be reform. I expect that Professor Barry would also acknowledge that these discussions are taking place at a European and international level.

The Deputy is absolutely correct about transparency around the second stage of the process, which involves senior people in the HSE and politicians, but in addition, the whole system needs to be looked at in a holistic way to see what reforms need to be introduced.

Deputy Stephen S. Donnelly

Specifically on evaluation, one of the issues in passing these rare drugs is that they do not meet this magic number of €45,000 per quality-adjusted life year, QALY, or a disability adjusted life year, that rare drugs are not meeting. This is for several reasons, including the lack of comparator therapies to use, because they are very expensive due to the small numbers of people to whom they apply and partly, we are told, because the State does not have a system of payment by performance. Are the officials in favour of a ring-fenced budget that would say that a certain amount of millions of euro was designated for orphan drugs and the €45,000 would be moved to something else and bring in extra issues?

Professor Barry has repeatedly made the point that in the case of orphan drugs, there must be payment by results. One reason it is so unaffordable is because there could be a phenomenal drug that works on one in 20 people suffering from a rare disease, but because one must pay for all 20, it becomes uneconomical for the one person. I have put that directly to the pharmaceutical companies, including some of those making rare drugs, and they have said that they would be very happy to move to a payment by results or outcomes system, however, the IT systems are not in place. They said that it is nothing to do with the pharmaceutical companies but relates to the inability to cope by IT systems in Irish hospitals. They have cited places such as Scotland and England where they have been introduced. Does that sound correct? Have the pharmaceutical companies told the HSE and the Department that they are willing to move to payment by outcomes?

Ms Vicky McGrath

The general view is that would be a desirable avenue to take. The systems are not currently in place, and we have some hope that they will become available but it has been a very slow process. We need to ramp up investment in the area to ensure that it can happen in the next two to five years, or else we will always lag behind and will never be in a position to identify that one patient who is getting good outcomes, and we will always struggle to reimburse.

A ring-fenced fund is a good idea in theory and it is something that we would like to see in the short to medium term. However, we do not want the ring-fencing to be a limit or barrier where we have a discrete pot of money and we end up arguing among ourselves over which patients we will spend the pot on. There are other areas where the health service can be streamlined so that funds could be taken from one pot and put into another so that it does not become the case that there is only one block of money available.

The Deputy spoke of a cost-benefit, the QALY and so on. Mr. Watt has alluded to re-jigging the entire heath technology assessment, HTA, system. We must take account of the burden borne by people in general and by society generally. It is not only the Department of Health budget or the Government, but it is about all the work we do and how better can we invest our money and time into other areas if these therapies were being made available.

Mr. Philip Watt

We also have to deal with a particular mindset. When we are trying to fight for rare disease drugs we are told that they are so expensive that we are denying others services. It is not only that patients are having to live with their disease but they are also being blamed from taking resources from somewhere else. We would counter that it is not a zero-sum game. The Government has been criticised for years for spending far too much on drugs which are much cheaper in other European countries, and that we do not have enough investment into biosimilar and generic drugs which would come at a fraction of the cost of many of these drugs. We still have a long way to go to reducing our drugs cost and using at least some of that money on innovative and new technologies such as those for rare diseases. There is also a lack of recognition that the cost of developing a rare disease drug is recognised internationally as far greater than one for a more common disease. I am not defending drug companies trying to exploit countries but that is an economic reality. If there is only a small number of patients from which a company can make back its money, and it costs $1 billion to develop a drug, there is no doubt that the drug will be expensive.

Deputy Alan Kelly

The witnesses are very welcome and I thank them for their contributions. The committee has written its report and several of us have argued and fought for various different organisations. I and Deputy Brassil, and others, have worked closely with the Alpha-1 foundation in its fight. In the last week or so, we secured a guarantee on the 19 remaining patients that were on clinical trials. I had a sad situation of my own where someone I knew well, Marion Kelly from Nenagh, who is no relation, unfortunately passed away after being taken off Respreeza. Her family had some satisfaction last week.

Responsibility in clinical trials is a real issue. There are issues around honouring commitments, whereby pharmaceutical companies sign up to these trials but then can withdraw from them. We need protocols around clinical trials in Ireland, but my question is how we do that. It is a very important area and I do not want to see something like this happen again. As far as I am concerned, two people lost their lives after coming off a trial because of how it would be administered. One of those people had been in my office only two weeks earlier.

I am trying not to overlap with the good questions Deputy Donnelly asked. I refer to reimbursement and the whole process surrounding it. There is an issue around jumping between the Department of Health, HSE and the National Contact Points, NCP. Unlike Britain, the NCP here is not aligned with the budget. Therefore, unlike other jurisdictions, those who decide on drugs do not have control over the budget, which is a real issue. How can we change this? We have seen so much jumping around on decision making. The committee found a farcical situation where there were letters going between the Department of Health, the HSE and the NCP; it was insane and it is infuriating. Will the officials give their views on how we can have budgets and decision-making in the one place?

Will the officials comment on the rare disease committee being decision makers and how that might work into the future? I can see that the witnesses are passionate about this but how do they anticipate it will happen?

How do the witnesses envisage they will form part of the team that makes decisions? I ask that because I do not want to see a situation where the witnesses are not part of the team.

Some of us here have had a lot of discussions on biosimilars. How can we move quickly in this area? We must move a lot quicker in this area because it will solve some of the issues. The witnesses referred to the issue, on which I have strong views.

My last question is on genetic services. It is a topical issue because of the revelation about a transcription error in Crumlin. I understand that Ireland is quite behind in terms of genetic services. The HSE has tried to recruit senior people with expertise in this area in order to upgrade services, which hinges on the fact that personalised drugs are the future of medicine. I do not think they can match until genetic services reach the required level from a quality and quantity analysis perspective. In other words, we need a service that is able to review many people pretty quickly. We are nowhere near that stage in Ireland. I would like the witnesses to give their thoughts on that matter.

Dr. Derick Mitchell

The Deputy has asked comprehensive questions and I will start with his first question. In terms of compassionate access to innovative drugs that have not received marketing approval or been approved by the European Medicines Agency, that has been a grey area for us. The Deputy is right that protocols should be put in place. It is something we have included as part of our joint work on our Drug Iceberg reports where we feel that any patient who is on trial, as part of a compassionate programme, should have access to that drug for their life. When we talked to other stakeholders in the area there seems to be a reluctance to adhere to that aim for a number of different reasons. Let us consider the environment for clinical trials. A new clinical EU directive is coming down the tracks that will detail how clinical trials will, potentially, be harmonised. The Department of Health is doing a lot of work into research ethics in terms of creating a single committee. The Helsinki Protocol governs how patients should gain access to clinical trials. The area of compassionate access has not been structured in a satisfactory way for patients. Recently the European rare diseases organisation, EURODIS, has compiled a report on trying to get a consistent approach to compassionate access and made a number of recommendations. We could consider those recommendations in order to move the issue forward in Ireland. If there is a potential that Irish patients would get compassionate access, then we should ensure that the company that allows a drug to be available for free adheres to a set of guidelines.

On whether the same body should be responsible for assessing and considering the price of drugs, that happens by default. There should be a scientific analysis of the particular drugs using a hospital treatment insight, HTI, system that we can all agree with, and then that assessment is done. It is slightly odd to see the person responsible for making those judgments talking on RTÉ the next day arguing about the price. I greatly admire Professor Michael Barry. To me, it is a conflict of interest when he, as the Chief Medical Officer decides whether a drug is good enough, goes on radio the next day and says that the drug is not worth the price that has been offered. Naturally, when one is on the airwaves one will use any argument to support one's reason to deny a drug and one quickly starts talking spin whether one likes it or not. The NCP should be given the task of making judgments and somebody from the corporate pharmaceutical unit, CPU, of the HSE should be the person discussing the price on the airwaves. The squeezing of the margins is part of the problem.

In terms of genetics, Professor Smyth compiled a report on the future of genetic services in Ireland five years ago. Virtually nothing has been done but the HSE is recruiting a couple of people to work in this area, which is positive. I was not surprised by what was said earlier concerning genetic services because they are completely understaffed. The staff are fantastic, do their best and provide great support to patients. The current situation is a national outcome of a decade where clinical genetic services have been underfunded.

We considered the HSE's service plan from two years ago. We noted that clinical genetic services were not mentioned and not even the word "genetics". The HSE is playing catch up. Genetics and the human genome are the way forward in terms of personalised medicine. It is only now that we are getting our heads around that aspect.

That is why there must be an overall Government strategy that recognises all of these new developments.

Deputy Alan Kelly

This is an area to which we might return. The issue of genetics is very interesting.

Chairman

I thank Deputy Kelly. Deputy O'Reilly is next.

I did not mention any company but it was high-profile-----

Mr. Philip Watt

I certainly think that if we have a system that everybody can trust it would reduce the amount of advocacy. Fundamentally, what we are saying is the system needs to be more than tweaked, which is the standard response from the National Centre for Pharmacoeconomics and the Government. The change needs to be more fundamental with regard to the health technology assessment process and afterwards. This is the point Deputy Donnelly raised with regard to transparency after the process. When we drew up our documents we tried to find a flowchart of the drug approval system in Ireland but we could not find one. There is none on the HSE website. We had to make it up based on what we guessed.

Deputy Louise O'Reilly

It looked like a map of an underground from somewhere.

Mr. Philip Watt

Exactly.

Deputy Louise O'Reilly

It was very convoluted.

Mr. Philip Watt

Chairman

At a previous hearing, a recommendation or suggestion was made that orphan drugs need to be dealt with differently to standard drugs but the legislation does not allow for a distinction between a standard or common drug and an orphan drug. A recommendation was made that the legislation would be reviewed to see whether amendments to the legislation were necessary to allow a different process to be undertaken for orphan drugs. Do the witnesses believe that the legislation in its present form can accommodate orphan drugs or does it need to be amended?

Dr. Derick Mitchell

To a certain extent changes can be incorporated under the Act but it is very clear the process is not sufficiently flexible to incorporate the specific needs of orphan drugs.

Deputy John Brassil

When Mr. Shaun Flanagan came before the committee he was asked that question.

His response was that the HSE has pushed the legislation as far as it can. He was basically saying that what is in place and what it is doing with it is the reason for the decisions it is making. As the witnesses know, I have tabled an amendment to the Bill, which I am trying to get to Second Stage; I will give a copy the witnesses a copy of it. It lists nine criteria but I have extended that to 12 to include three small additions, which the witnesses spoke about previously, taking into account patient advocacy groups and giving orphan drugs a specific criteria, namely, if they are not quantitatively assessed that they would be so assessed.

Chairman

I will allow the Deputy back in to expand on that in a few minutes. I thank the witnesses for those responses.

Senator Colm Burke

I thank the witnesses for their presentation. Regarding orphan drugs, Ireland is at a disadvantage because of the size of the country and our population. A country with a large population like Germany is able to make progress at a faster rate. I refer to recommendation 13 in our report. We had done an agreement in the past five months and the witnesses are saying there is no evidence that working closer with other countries has come into effect. Ireland's approach seems to be to deal with countries with smaller populations. Would we be better off aligning with a country with a bigger population in that we would be in a better position both in terms of assessment and trying to deal with the price issue? I am aware of a drug coming onto the market which would have benefited only ten people in the Thirty-two Counties. Of the ten, six would have had a real benefit from it, which is a very small core group of people. However, if we were dealing with a country with a larger population, more people would benefit from a particular drug. Should we examine that in a more comprehensive manner?

Regarding assessment, we seem to be going through a long process of assessment when the European Medicines Agency may have approved the drug already. There was a recommendation in our report on working with other countries in doing the assessment rather than having a team here devoting all its time to doing it when three or four countries are doing the exact same assessment. There is a duplication in that regard. I recall speaking to a pharmaceutical company that had presented more than 300 pages of documentation on its research and development of a drug here. The process seems to be drawn out. We are in the European Union. The idea was that we would work together to ensure there would be benefits for everyone thereby removing all the duplication.

My next question is on rare diseases. I dealt with a particular case of someone with epidermolysis bullosa, EB. Debra Ireland raised a simple point with me five years ago, namely, that even if patients with EB had medical cards, the cost of bandages was not covered by the medical card. I arranged a meeting with the medical card service to ensure that bandages could be made available to those on the medical card scheme. I am sure there are small areas that are being ignored because that is the regulation and we cannot bend that rule. Could we be doing much to assist patients with rare diseases that do not fit into the criteria provided in regulation or legislation because we do not have a system within the health service to help them get access to an improved level of care? It is one of the problems that tend to arise for people with rare diseases.

My final point is on cross-Border healthcare. As someone who was involved in that issue in Brussels in 2008 and 2009, the directive has now been transposed. Could we work with other countries on cross-Border healthcare and remove some of the hurdles here to getting access to care? We are doing that in many other areas. Why can we not do it much more in healthcare? We seem to be unable to do that.

Mr. Phillip Watt

Those were excellent questions. To start with the one on cross-Border healthcare, we work with the rare diseases group in the North. Members can tell from my accent that I am from Belfast. That group feels utterly abandoned. There is no administration, and they are dealing with Brexit as well. They are very concerned about the cross-Border services that will be affected by Brexit. They are distraught; that is the only word I can use. We need to extend a hand to them much more often than we do currently in terms of making sure they are included.

We still have backs to the Border. Everybody in the South looks at Dublin and everybody in the North looks at Belfast. It is far too small an island to do that. We need to pool all our resources, and there are some fantastic services in the North. They are developing genetic services of which we can avail. Some retina operations are done in the North. Likewise, patients with heart conditions are treated in the South. As the chief medical officer in Northern Ireland stated, we just have to get on with it and try to ignore Brexit and the fact that there is no Administration in the North and make sure those services work for our patients. I believe we should not stop that but enhance those services. I take that point fully on board.

Ms Vicky McGrath

I will go back to the Senator's first question and then move on to the cross-Border directive. In terms of the BeNeLuxA initiative, it is five months in. We need to give them a little time to do some work but it comes down to transparency. Have they made any progress? What have they done? None of us seems to know. The initiative has been signed. That is great. We had all the fanfare but we do not know whether anything has happened in the intervening period. I would not throw it out as being a case of wanting to go with a larger country. We need to let it bed down.

That rolls very well into the cross-Border directive and what we refer to as the European reference networks. The Commission has decreed that Europe needs to work as one when it comes to rare diseases. There is no longer an Irish rare disease healthcare system; it is a European rare disease healthcare system. In so doing, I would expect the likes of BeNeLuxA to work with the German and the French authorities. They will start to work together and then to do reimbursement for drugs across Europe. The chances are that some of these therapies will be made available, as in administered, in only one or two countries because the diseases are so rare. There are so few patients they will end up having to travel, and that is how we will get access to it. We will get access to it because Ireland is signed up to these reference networks. We are still in the very early stages. There are 24 networks. Some countries have representations in all 24. Currently, we have representations in two, but we understand at least 21 applications are due to be submitted by the next deadline. The deadline has not been set, so it is not that we are behind the ball in that regard. By the middle of next year, we expect to have the applications in and, hopefully, they will be processed by the end of the year to ensure Ireland is well represented in the European realm in terms of these reference networks. They will provide many solutions. The will provide some of the infrastructure we want around the registries and ensuring we have the best care pathways. It is a major burden to put on the European reference networks, but the pharmaceutical sector is calling for it.

The challenge we face is that the pharmaceutical industry will stop coming to Ireland seeking to get drugs reimbursed because our population is too small. If the industry is dealing with Europe, there is some hope that it will end up coming across. It will be a challenge for our health system to equate to Germany's, but we have to address that in the years ahead. There are positives, but in the intervening period, we need to readdress the existing system and make sure that we do not fall behind. We must also consider the other drugs and diseases that are not termed "rare".

Dr. Derick Mitchell

To complement that, and to emphasise the point Ms McGrath is making, there is a certain perception that the European Reference Networks are just about service delivery. The vision for the European Reference Networks is also about data collection. It is about harnessing the power of Europe to work for patients in individual countries. In Ireland in particular, part of the development of a new national rare disease plan would be more emphasis and much more supports for the clinical leaders. However, patients are playing leading roles in several of the European Reference Networks, and such a plan should also allow for greater Irish leadership in those reference networks. We believe they will become one of the major solutions for things like pricing, reimbursement and ultimately better assessment processes.

I refer also to the joint clinical assessment initiatives that are going through the European Legislature at the moment. The Commission's proposal for joint clinical assessments is something we have been looking at and of which we are highly supportive. While there are differences in opinion in various member states as to how it affects their assessment processes, joint clinical assessments applicable across jurisdictions certainly make sense from a patient perspective and with regard to duplication. Some 14 patient umbrella groups at European level recently released a statement about the consensus on patient involvement in that proposal. Patient involvement needs to be a greater consideration in the overall co-ordination of efforts in joint clinical assessments.

Senator Colm Burke

I wish to make a final point about the supports that should be made available to patients with rare diseases. They have no one to turn to in many cases. Could there be a better structure for supporting people who could be provided with care but who do not know how to access it? Does more need to be done to provide information to people who are identified as having rare diseases?

Mr. Philip Watt

The conditions are so diverse. The Senator's point is correct. In some cases, more visibility is needed. To get back to the point about epidermolysis bullosa, EB, it is ridiculous that people have to pay for bandages when that is a core part of their medical treatment. There is a kind of grey area between what is a drug or medication and what is not which definitely needs to be resolved. Moreover, without a universal healthcare system, we just have the long-term illness scheme which was introduced in 1971. That names about 43 conditions for which there is basically a universal healthcare system. We are very lucky that cystic fibrosis, CF, is one of them. However, many other conditions, including many rare diseases, are not named in the scheme but should be equivalents. I do not advocate getting rid of the scheme but it should be expanded to cover many more conditions until we have a universal healthcare system. That is crucial as well.

Deputy John Brassil

I thank the witnesses for their presentation. I refer where we are as a country with regard to orphan drugs. The European Medicines Agency has designated 148 medicines as orphan drugs for rare diseases. Currently we reimburse 53 of those or 35%. The UK reimburses 68, Spain reimburses 75, Italy reimburses 84, France reimburses 116 and Germany reimburses 133. We are at 35%. Germany is best at 90%. Spain, a country whose economy is not going as well as ours, is at 50%. We are at the lower end for reimbursement for orphan drugs and drugs for rare diseases. The NCPE is aware of this. We have been fighting individual battles, over Respreeza for alpha-1 proteinase inhibitor deficiency, as Deputy Kelly mentioned, and Orkambi for CF. It seems that if the lobby is strong enough, or given the unfortunate and tragic deaths of two patients, the HSE takes notice.

As a result of my work as spokesperson on primary care I have tried to modify the Bill. This committee has stated that it needs to be modified. It is being resisted at all ends. That is the context of our dealings with the NCPE and the HSE in trying to establish a better model to reimburse more drugs and to give patients with rare diseases, who are as deserving of proper care as anyone else, some chance of either dealing with their symptoms or in some cases dramatically changing their lives.

The rare disease technical review committee was referenced. That is a welcome addition. The key point, which the witnesses have recognised, is that this group is reviewing the decision, not making it. Because it is reviewing a decision based on a flawed process, it will find it difficult to come up with a different decision. The 2013 Act needs to be amended. That is my focus and it should be that of the witnesses as well. Unless we amend the process by which these drugs are reviewed, we will be fighting an uphill battle.

Dr. Mitchell made reference to Sweden. Sweden's model is a based on quality-adjusted life year, QALYs, but it does not have an upper limit. The Swedish model allows for situations where maybe half a dozen patients are suffering from one condition, where one could not possibly get the type of data that is needed and meet the threshold of 45,000. Interestingly, in 2015, the proportion of health expenditure on medical goods was greater in Ireland than it was in Sweden.

Deputy Donnelly raised the issue of biosimilars. The IPU has identified a potential saving of €307 million on biosimilars alone over three years. I have met children suffering from spinal muscular atrophy. A drug called Spinraza has revolutionised treatment. Its total cost to the system would be approximately €9 million. Multiples of that cost are available.

We need to amend the Act to give some hope of orphan drugs being reviewed. If we do not do so, the companies will just stop applying, as Ms McGrath pointed out. They know the answer will be "No", so why would they waste time and resources applying for something when they are going to be refused? That will have a knock-on effect.

I met officials from Vertex, the company that makes Orkambi, recently. After all the battling, arguments, protests, marches and so on, that drug was reimbursed, including future iterations. I believe the next Orkambi, whatever it is called, is coming on-stream shortly without any debate. Vertex told me it is looking for other countries to consider reimbursement in the way the agreement was made in Ireland.

Mr. Philip Watt

That has happened. Sweden and Denmark copied Ireland.

Deputy John Brassil

It can be done. Orkambi was not reimbursed because the NCPE suddenly saw the light, however. Massive political pressure and patient-centred demonstration and focus made officials realise they could no longer ignore it or make the decisions they were making without being challenged. When they were challenged, they made the decision. I do not know if I had any question there; it was more of a statement. I would appreciate if the witnesses' organisations could look at the Bill I am-----

Mr. Philip Watt

We need to use that power responsibly. I give an assurance that my organisation would never campaign for a drug we did not believe in or see the scientific evidence for. Our point was that the system was flawed so that no orphan drug would get past. That was fundamentally why we were protesting. It is great to see some reforms being put in place, partly as a legacy of that but also as a result of the work of all the patient groups. Our key point is that the reforms need to be broader. A broader strategy needs to be put in place which considers issues such as cost and where the money is going to come from, rather than simply tweaking things by putting a couple of patient representatives here and there. All that is welcome and great but it must be part of a broader change. There is also an issue in respect of how patient representatives are picked. We want to set down a marker that we should elect our own representatives and not have them hand-picked by the HSE and the Department of Health. There was an election for the rare diseases committee, which was welcome, but other processes are happening daily whereby the HSE simply picks a patient or an organisation. Those days are gone and the structures need to be much fairer and more transparent.

Deputy John Brassil

On the budget for orphan drugs, I have consistently sought a figure but cannot get it. Deputy Donnelly raised this earlier as well. Unless we have a specific budget it is difficult to make comparisons, decide how much we should spend or know how much of those savings will be reinvested in orphan drugs. That is something else we should insist on. Although I know they have the data, the companies keep coming back saying they do not. In the application process for any company, one of the questions is whether there are orphan drugs ratifications. It is in their applications so they know what is an orphan drug and what is not and, therefore, they can tell us how much they are spending on orphan drugs. They just do not want to do so.

Dr. Derick Mitchell

I reiterate that there is a specific recommendation in the national plan for a budget. It does not specify the total amount and certainly is not viewed as a target but as a way in which we can effectively measure the spend. It has not been touched as part of the current plan, however. We certainly hope that as the new plan is being developed, it will be re-examined in that context.

Deputy Bernard J. Durkan

I am sympathetic to the issue of orphan drugs, particularly where it has been shown that they work and that a patient cohort will benefit from them. The problem, which the committee has discussed many times, is where the determination as to their benefit and safety comes from. Is it made at EU level? There is a vetting system at EU level but, as we found out in this committee a year or so ago, we have an internal system that vets them again. I do not see the purpose of that exercise at all. We appear to have, and to contribute to, two systems, as the vetting system we have here is also replicated at European level. When it comes down to it, the EU is in the best position to command a price. If someone is negotiating on behalf of 500 million people, the case is an awful lot stronger than on behalf of three or four. Despite the good cause, it comes down to the power of the market but we do not use that argument in this country at all. An average of 14 member states are involved in these processes. The Bill referred to by Deputy Brassil is a classic example. Why could it not be the entire EU 27? It could have been 28 member states up to recently but could still be 27 from here on in. Whoever is vetting and dictating the going rate to individual states, no individual state can have the same power, except perhaps the larger states. We should get to that point as early as possible. I spoke about this issue 20 or 30 years ago in another capacity. It has not changed over the years. We are still engaged in procurement standing alone on our own two feet and it is not working. It does not, and cannot, work. Having a duplicate system where we have one system operating at EU level and another one superimposed on it here is daft and stupid in the extreme.

I acknowledge the benefit of patient representative groups but I would also balance that benefit against the points raised by Deputy O'Reilly. We could find ourselves in an awkward position. We have the telling tales of the patients and lobby groups on the one hand, while, on the other, we have this amorphous system, which we do not know how to deal with and cannot manage. Everything comes down to cost in the final analysis. Deputy Brassil makes a useful point about the 2013 Act. It was framed and passed in the midst of the worst economic crisis this country or any other European country ever had the misfortune to experience. It might be a good idea to examine it again in a different light. However, as a note of caution, before the economic crash we believed we were one of the wealthiest countries in the world but that was a figment of our imaginations. We were not and we are not. There is no use pretending we are. Wealth is made up of series of factors of which housing, goods, services and trade are a part. They tend to float with the market and can leave us high and dry when the tide goes out. We need to be cautious that we do not walk ourselves into trouble, not necessarily in respect of pharmaceuticals and health only but in other respects as well.

Diagnosis is an important issue, particularly for rare diseases. For example, Guillain-Barré syndrome is a rare disease. I know five people who have been unfortunate enough to wake up and find themselves paralysed. There is no answer to it at all and they spent considerable time in hospital while their cases were examined before diagnosis eventually happened, sometimes on a haphazard basis. That needs to be examined. Modern communication should make it much easier to compare cases across the country and the continent. This condition is curable. The same people were walking around six months after being diagnosed. They had to go a long route to learn to walk again and needed rehabilitation but it happens. Early diagnosis is of considerable economic benefit in that area.

I would be one of those strongly in favour of utilising it as much as possible.

I have already referred to my final point. We need a single system of adjudication. It is either in Europe or it is here. We do it one way or the other; there is no use doing it twice because there is a cost involved in that. From a safety point of view, utilising new drugs on the market is of paramount importance. Years ago, there was considerable anxiety and questions were raised about the manner and methodology used to test the drugs and those on whom the tests were carried out. Considerable controversy arose at the time and people remain concerned. It must be one of the issues we consider. We need to move on. We need to remove the emotional situation that arises on a daily basis where it requires a significant amount of publicity or attention to be drawn to a particular condition before there is a response but that response must be tempered by the ability of the State. We return to the question of how much a life costs, a point raised by Deputy O'Reilly.

Chairman

I am conscious of the fact that we have the HSE and the NCPE outside. Was there a specific question in that?

Deputy Bernard J. Durkan

It was more of a comment.

Mr. Philip Watt

The EMA makes the decision and it goes to individual countries. The Deputy is right. There is considerable inefficiency in that process. It is about whether the Government is willing to put its trust in the EMA. The question is whether it becomes a economic burden the Government is unwilling to shoulder. It makes so much more sense. The EMA makes the decision on a drug and all European countries can negotiate at once with the drug companies so they have far better leverage when it comes to getting a better price. I do not think the HSE is ready for that but it is a principle worth working towards.

I went to the annual conference concerning children with Duchenne muscular dystrophy. They have a window of two or three years and if they do not get the medication, they will go into a wheelchair straight away. They will not be able to walk. Those medications are broadly available in the rest of Europe. Cost seems to be the reason why they are not being brought-----

Senator Rose Conway-Walsh

With regard to the impact of delays in the BeNeLuxA countries, we know the situation regarding Spinraza in all of the BeNeLuxA countries. Surely we can use that as a lever.

The children are being admitted to ICU. Even taking out the inhumane aspect and the distress caused, the additional costs are building up in trying to-----

Ms Vicky McGrath

It comes down to whether we are willing to even pay the price BeNeLuxA has negotiated. I do not know. In the very short term, as patients and patient organisations, the big thing we would like to see happen is an opening up of the relationship between pharma and the HSE. Things go into the black box - I think "opaque" is a nice word to use - but there is no visibility and one, two or three years could pass by. We hear anecdotally from the industry which is far more willing to discuss this issue than not that it has had a meeting, or not as the case may be, or a phone call, or that an email was sent in the intervening period. That there is no visibility is one of the biggest challenges we face in the short term and it would be easy to address, to at least say in broad terms that there was communication, or that a meeting was held and that people promised to come back in three months' time. That would help us a lot in the short term. We would then look at rebuilding the systems and stuff like that.

Senator Rose Conway-Walsh

It is a shame that the clinicians are not involved in the decision-making. We are lucky, in a country of this size, that we have well qualified clinicians, yet their opinions are just dismissed. There is protectionism within the system that certainly does not serve either the clinicians or the families and those who need the solutions and treatment.

Mr. Philip Watt

There is also cynicism. Recently I talked to people involved in pharmacoeconomics across Europe and they all seemed to have the sense that somehow patient groups were in the pockets of pharma companies. To be honest, it is really insulting. As patient groups, we would be betraying patients if we ever advocated for use of a drug which was not effective. That would be disgusting and we need to be more robust in showing that is the case. Clinicians are accused of the same because they are involved in clinical trials and so on. It is so simplistic and wrong and needs to be challenged.

Senator Rose Conway-Walsh

The Minister needs to challenge it.

Chairman

On behalf of the committee, I thank Mr. Watt, Ms McGrath and Mr. Mitchell for coming to give their evidence. Of course, they can wait and observe the next session.

Sitting suspended at 11.13 a.m. and resumed at 11.17 a.m.

Chairman

The committee will now continue its consideration of the implementation of the recommendations contained in the report on the evaluation of orphan drugs which was published last February. On its behalf, from the HSE I welcome Professor Michael Barry, clinical director of the National Centre for Pharmacoeconomics; Mr. John Hennessy, national director of acute strategy and planning; and Mr. Shaun Flanagan, chief pharmacist in the corporate pharmaceutical unit. From the Department of Health I welcome Mr. Finian Judge, principal officer in the community pharmacy, dental oral and aural policy unit.

By virtue of section 17(2)(l) of the Defamation Act 2009, witnesses are protected by absolute privilege in respect of the evidence they are to give to the committee. If, however, they are directed by it to cease giving evidence on a particular matter and continue to so do, they are entitled thereafter only to qualified privilege in respect of their evidence. They are directed that only evidence connected with the subject matter of these proceedings is to be given and asked to respect the parliamentary practice to the effect that, where possible, 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. I advise that any submission or opening statement made to the committee may be published on its website after the meeting.

Members are reminded of the long-standing parliamentary practice to the effect that they should not comment on, criticise or make charges against a person outside the Houses or an official, either by name or in such a way as to make him or her identifiable.

I ask Mr. Judge to make his opening statement.

Mr. Finian Judge

I thank the Chairman and committee members for the invitation to discuss the report on evaluating orphan drugs. I am accompanied by Mr. John Hennessy, national director of acute strategy and planning; Mr. Shaun Flanagan, chief pharmacist in the corporate pharmaceutical unit; and Professor Michael Barry, clinical director of the National Centre for Pharmacoeconomics.

In March the Department of Health received the committee's report on evaluating orphan drugs. The report included a number of significant recommendations related to the assessment and reimbursement process for orphan drugs. Some of the recommendations concerned the policy and statutory framework underpinning the process, with the remainder focused on the evaluation and reimbursement process, including the clinical criteria deployed in that evaluation.

I note that the 2013 Act is very comprehensive and provides for a range of important matters, including the criteria to which the HSE must have regard when making reimbursement decisions on new medicines. The Act includes provisions on interchangeable products, rules for the establishment and maintenance of reimbursement lists and provisions on reference pricing. In its report the committee focused on that part of the Act which provided for the evaluation process for new medicines proposed for inclusion in the reimbursement list.

Since the publication of the committee's report, the Department has been engaging with the HSE and the NCPE to address the recommendations contained in it on the framework and process for assessing orphan drugs. In this contribution I will address those recommendations directed to the Minister for Health or the Department. My colleagues from the HSE and the NCPE will address the recommendations on the evaluation and reimbursement process, including the clinical criteria pertaining to evaluation. Under statute that function is vested in the HSE.

The Minister is satisfied that the Oireachtas has put in place a robust legal framework in the Health (Pricing and Supply of Medical Goods) Act 2013 for the assessment and reimbursement of medical goods. The Act provides the HSE with full statutory powers to assess and make decisions on the reimbursement of all medicines and includes detailed criteria to which the HSE is required to have regard when making those decisions. The Act puts in place a clear statutory framework by which new products must be assessed by the HSE and ensures decisions are made on an objective and scientific basis. It provides for a fair, transparent and rigorous process for the assessment of all drugs and has delivered tangible results for patients. The Minister is satisfied that there are no legislative barriers to the reimbursement of orphan drugs, as distinct from any other drug, and is further satisfied that the rules pertaining to the assessment of all drugs do not discriminate against orphan drugs. In fact, the criteria which apply to the evaluation process contained in Schedule 3 to the 2013 Act provide sufficient scope for the HSE to take on board the particular circumstances of orphan drugs. My colleagues from the HSE and the NCPE can elaborate further on that matter.

In its report the committee, rightly, identified that drugs were increasingly being developed to target very rare conditions and that these drugs often came with a very high list price. Under the Act, the HSE is required to have regard to the funding challenges these drugs represent. It does so by drawing on the criteria contained in the Act, including the potential or actual budget impact of the drug in question and the cost-effectiveness of meeting health needs by supplying a particular item, rather than by providing other health services. These are difficult decisions, but they recognise the core challenge presented by the availability of finite resources in the face of ever-competing demands.

The committee recommended the appointment of an independent person to conduct a review of the current process and its role in orphan drug availability. The Department is preparing to undertake a review of the governance arrangements applying to the internal HSE process. The review will examine the systems, structures and resources which support the decision-making process for reimbursement.

In its report the committee also expressed support for collaboration with other EU member states. The Minister is very much in agreement with that notion. In fact, the challenge of accessing innovative medicines at an affordable price is one shared by most, if not all, developed countries. Demographic change will have to be managed, with a continually growing pipeline of new and innovative medicines, many of which will come with asking prices that risk making them unaffordable or unsustainable. It is estimated that approximately 45 new molecules are due to receive market authorisation in Europe in each of the next five years. It is in this high technology space which includes orphan drugs that the greatest challenges will arise in the years ahead.

Already, expenditure under the high technology arrangements through which the majority of new high cost medicines, including orphan drugs, are funded has increased, from approximately €400 million in 2012 to close to €700 million this year. That level of growth is a major challenge for the Government and the HSE and a reason Ireland is working with other countries in Europe to develop common solutions to a universal problem. A significant development since the publication of the committee's report has been Ireland joining the BeNeLuxa initiative on pharmaceutical policy in June. This collaboration will support the Minister's objective of co-operation with other EU member states to identify workable solutions in an increasingly challenging environment. As the committee acknowledged in its report, this is a long-term strategy. However, it is one we expect will complement existing domestic policy in this area.

While the Department has not undertaken a formal review of the Act in its entirety, it has, in conjunction with the HSE and the NCPE, considered in detail the committee's report and the recommendations contained therein. There has been substantial engagement between the Department, the HSE and the NCPE on each of the recommendations. I am pleased to note, as well as the policy developments I have outlined, that a number of significant changes have been made within the confines of the Act at operational level in the assessment and reimbursement process to address a number of the recommendations made in the report.

I will hand over to my colleagues from the HSE and the NCPE to elaborate on the operational developments.

Mr. John Hennessy

I thank the joint committee for the invitation to discuss orphan drugs. As Mr. Judge said, I am joined by my colleagues, Professor Michael Barry, Mr. Shaun Flanagan and Mr. Ray Mitchell from our parliamentary affairs unit. Our preparations focused on the committee's report of February 2018 and the measures taken or under way to address the recommendations contained in it. Mr. Judge has covered the main policy and legislative elements. I will focus on the operational aspects.

On recommendation No. 4, I confirm that discussions have commenced with the pharmaceutical sector to provide clarity on pricing and measures to speed up the negotiating process. The discussions have also explored the issues of risk sharing and post-approval review to ensure ongoing expenditure is justified by evidence of clinical effectiveness.

On recommendations Nos. 6 and 7, there is already a high degree of communication between the HSE and the pharmaceutical sector, as well as, of course, with patient advocacy groups. However, steps have been taken to strengthen this engagement through the provision of additional resources at both NCPE level and within the corporate pharmaceutical unit. This is intended to enable representations to be considered at both the HTA and negotiation stages. There is also routine engagement with the pharmaceutical industry, while the composition of the drugs group has been expanded to include patient representatives and more clinical expertise in the area of rare diseases. Enhancements have also been made regarding medicines for older persons and medical ethics.

The HSE has written to hospital groups setting out the importance of having a clear understanding and informed consent arrangements in place prior to the commencement of clinical trials and compassionate access programmes. The establishment of a specific budget for high technology and orphan drugs is also being examined. This was also a recommendation made in the report. To a large extent, it is already something we have in place in that high technology and orphan drugs are, by and large, funded centrally, rather than from individual hospital budgets.

The measures now under way to strengthen the representation of rare diseases in the assessment process and to involve patient representation and medical ethics should also refine the process of assessing medicines in the manner intended and recommended by the committee. The issues highlighted in recommendation No. 10 are being pursued further with the HRB, which appears to be the most obvious route to influence research in collaboration with the university sector. With regard to recommendation No. 12, the HSE has established a national rare disease technology review committee. The role of the committee is to enable clinicians and other stakeholders to input into the assessment process and in the post-health technology assessment phase to review proposals, prescribing guidelines etc. for consideration by the drugs group. This is designed to operate similarly to the National Cancer Control Programme therapeutic review committee, which has been operating successfully. Such guidelines will, where appropriate, include recommendations relating to the prescribers who should be enabled to access orphan medicines. I will ask my colleague, Professor Barry, to elaborate on the work of this group. A model of care has also been prepared by the HSE’s clinical programme for rare diseases, which aims to improve access to care for patients with rare conditions and improve value and quality in this area.

As the process for evaluating applications was covered in detail at our meetings with the committee in 2017, I will not repeat this other than to emphasise again that the criteria for assessment are as detailed by the legislation, the Health (Pricing and Supply of Medical Goods) Act 2013, and as set out specifically in section 19(4) of the Act. The important point to bear in mind is that the legislation requires the HSE to have regard to the cost-effectiveness and the clinical benefits of the product being applied for. The particular features that relate to orphan conditions are obviously considered during the assessment process and factors such as clinical effectiveness and the number of potential patients affected are clearly set out in the reports compiled. As members are aware, the HSE currently spends in excess of €2 billion per year on drugs and medicines, and on a per capita basis the Irish health service continues to be one of the highest spenders on medicines in the OECD area, with expenditure still increasing annually. I will now hand over to Professor Barry for further detail on the rare diseases technology review group.

Professor Michael Barry

Professor Michael Barry

No, it was a few days ago. I just wanted to give the committee a flavour of what is going on. There were five meetings in 24 days and it is testimony to the significant work done by committee members, who do such work on their own time and in an unpaid capacity.

The committee has focused on two medicines to date, which are sapropterin or Kuvan for phenylketonuria, and nusinersen or Spinraza for the treatment of spinal muscular atrophy, SMA. As an example, the committee recommended to the HSE drugs group that access to sapropterin be considered under a managed access programme. In other words it would occur where clinical criteria were laid down for starting and stopping the medication if it was not working. It was taken by the HSE drugs group, which made a positive recommendation with regard to Kuvan, which is good news. The funding issue must be decided on by leadership. Currently we are preparing a rare diseases technology review committee recommendation for nusinersen for spinal muscular atrophy and we hope to submit it to the drugs group shortly. That is a flavour of how the new committee is working.

Yes, but to clarify, that is if there is a budget impact. If money is being spent, it is the director general, the directorate and the HSE senior leadership team that must sign off on it. There are some medicines, however, for which the net cost is not additional, and we accelerate the process for them.

Deputy Stephen S. Donnelly

I turn now to the process. Professor Barry made a point, on which I fully agree with him, that the State should pay on an outcomes basis for orphan drugs. If there is a drug, be it Spinraza or something else, which works on, say, one in five people, and if it is not cost effective to provide ongoing treatments for all five, we can identify and pay for the one for whom it works while stopping the treatment for those for whom it does not work, although that is not always easy and it can be politically and administratively difficult but it must be done. I have put Professor Barry's point to several of the bigger pharmaceutical companies which provide some of these fantastic drugs and they were positive.

They said they want to do it as well and they gave me examples of other countries in which they are doing this. The impression I got was that pharmacoeconomics here is not up for it. I am not suggesting that is what I was told, rather it is what I had assumed. However, what they said is that the problem in Ireland is that we do not have the necessary IT systems in place to track the patients and, therefore, be able to do this. Is that the witnesses's understanding? Is the barrier to the payment by outcomes, which hopefully would allow for more of these wonderful drugs, that we do not have in place the IT systems to do it?

Professor Michael Barry

What the Deputy is referring to is what we call managed access programmes with specific starting and finishing criteria, or stopping criteria if the system is not working. I do not think there are IT barriers to it. For example, in the case of Nusinersen we are not talking about a lot of patients so it should be relatively easy to track patients and their outcomes. The focus on managed access programmes came on stream with the rare diseases technology review committee, the focus of which is not on cost effectiveness or money but clinical outcomes from the point of view of patients and patient representatives. It is a question of the system focusing on managed access programmes. I think they can be delivered but I take the Deputy's point that for the conditions in respect of which the patient numbers are larger there would be a challenge. For the conditions about which we are speaking, the patient numbers are not large and it should be eminently possible to do it. The idea of a managed access programme is that we know from the trials that, say, 50% of patients may not respond to the drug. I do not think anybody wants to be funding expensive drugs like Spinraza at a cost of €500,000 in the first year and €250,000 per patient in the second year. It is about trying to do the best we can and to ensure that people who respond to the drugs get them.

Deputy Stephen S. Donnelly

I would like to discuss the alleged delay in Ireland in getting some of these drugs onto the market. The witnesses may disagree but it has been put to me by patients, patient advocacy groups, clinicians and the pharmaceutical companies that relative to other EU countries Ireland is very slow in getting the drugs to market. For example, in the case of Translarna, 22 other countries-----

Mr. Shaun Flanagan

My apologies for cutting across the Deputy but I would question the figure of 22.

Deputy Stephen S. Donnelly

That is fine. It has been put to me by a number of people that Ireland is slower than other countries at getting drugs to the market, for whatever reason. What is being put to me, fairly or unfairly, is that the NCPE moves pretty quickly and is transparent in what it does - whether one agrees or not with the full process that is what happens - and that the EMA at a European level is pretty good and is tipping along but when it comes to the Department of Health and the HSE the process slows. I am not making that allegation. It has been represented to me by numerous groups that at Department of Health and HSE level the process becomes opaque and there is not the level of interaction that people are looking for. Is it fair or accurate that the process slows once it reaches Ireland and, if so, what three actions can we take to make it more transparent and quicker?

Mr. Finian Judge

The Deputy mentioned the HSE and the Department in terms of where the bottlenecks may occur. As I said in my opening statement, statutory responsibility for decision making in terms of reimbursement rests solely with the HSE. The Department is out of the loop in terms of the decision making process.

On the substantive issue of the perceived delay in Ireland, there are various lists of where Ireland sits in regard to other European countries. We have no idea how these lists are put together and what is regarded as reimbursement in one country in comparison with another. Over the last number of years a lot of medicines have been reimbursed. Practically none has been refused.

Deputy Stephen S. Donnelly

It is the timing of the process that is in question.

Mr. Finian Judge

I will ask Mr. Flanagan to respond on the timing issue because it is a matter for the HSE but the timing issue cannot be looked at separately from the pricing and funding issues.

Mr. Shaun Flanagan

I cannot remember a year of my career when the HSE was under spend on drugs. There is clearly a funding challenge for every country around the globe. Many countries are trying to get into multilateral type arrangements, whether BeNeLuxA or Valletta, such that Ireland is not the only country struggling in that regard. I would not argue that we are slower than other countries. In my view, the challenge is around the availability of sufficient resources and trying to match the resources we have with the demand.

Deputy Stephen S. Donnelly

On the issue of resources, is Mr. Flanagan speaking about drug budget resources or human resources?

Mr. Shaun Flanagan

It is drug budget resources. The NCPE and the pharmaceutical unit did have a challenge around human resources but that is no longer an excuse as over the last 18 months the HSE has given us substantial resources. I will give a practical example, which might better explain the delay. Commercial negotiations can take a long time. It can be hard to get a company to the point of an offer that will be acceptable to the State. The system is broken internationally from a pricing point of view in that we have a system whereby companies come with lists prices of, say, €100, and they get involved with one country in non-transparent negotiations and reduce the price to, say, €90; they then move on to the next country for which they reduce the price to €80 and then for a third country they reduce it to €70. If Ireland is the first country it pays €100. It is unfortunate but true that the further down the line a country is, the better the offer it gets. It is not possible for me to put particular information into the public domain but I will try to illustrate what happens in the case of a non-orphan drug. The drug Pablociclib - Ibrance - is used to treat breast cancer. It was approved by the EMA in 2016. At the time of the pricing and reimbursement application the company in question applied for a price of €3,800 per pack. This would have resulted in a budget impact of €66 million. This was in commercial negotiation for over a year. Eventually, a pricing decision was made in a different country. The price offering given to us at that point was €2,190 per pack. Within three months the HSE had completed all processes and had reimbursed it. The net difference is €28 million over five years. If we go early, we pay more which means we can reimburse fewer drugs. The challenge is getting the balance right in terms of the right amount to be spent at a premium over other prices versus fighting as hard as we can for reduced prices.

Deputy Louise O'Reilly

I thank the witnesses for being here. We respect and appreciate the job the witnesses do and we acknowledge that very often they are portrayed as the bad guys but sometimes they do not help their own cause. Patients come to us because they want to know the status of their approvals. They should not have to contact their local public representatives to get that information. It should be readily available to them but clearly it is not because if it was patients would not be making representations to us. Patients are awaiting the approval of drugs that they believe will be effective for them but are unable to find out the status of the approval of those drugs.

What I am hearing from the Department of Health is that it is out of the loop with regard to the process. I understand there are various meetings and so on. Is it possible to bring transparency to this while respecting commercial sensitivity? I fully appreciate that the Department must uphold commercial sensitivity. Is it possible to put in place a system that keeps the families informed? Everyone seems to be waiting. The clock is ticking and people are told another meeting will take place. Yet, the Department does not know if the matter will get onto the agenda of the meeting. If it does, the Department does not know what is to happen at the next meeting. It seems that no matter who I talk to, that person is never the person who is responsible. It seems it is always a matter for another person.

I found it curious for Mr. Judge to say that the Department was out of the loop with regard to the process. I would have thought the Department would have been central to or involved more in the process. Do the officials have any suggestions on how we might be able to improve transparency?

I will bank the questions. It will be easier in terms of time and I know others wish to come in. Has the rare disease technology review group published a work programme? If so, I have not seen it. If there is a work programme, perhaps it can be shared with us. If there is none I would be a little concerned.

The question of reviews and expected timelines is relevant. We know that we are not dealing with things that necessarily move in a straight line but the possibility of expected timelines and sharing that information is important.

The patient and clinical engagement system in Scotland is a relevant model. Scotland has a similar population size, profile and so on in terms of health. It seems there are far fewer problems in Scotland and the system it has appears to work. Has consideration been given to replicating the best aspects of what they are doing in Scotland? It appears to be working. Central to the system there is the involvement of the patients. That is important.

Will the officials give us an update on or idea of what progress, if any, has been made on the BeNeLuxA group? I apologise if the question was already asked - I may have missed it.

There are problems at the moment relating to clinician and patient input into any new assessment programme. Will this take place before or after the health technology assessment will be completed? I am referring to the opportunity for clinicians and patients to have input. I am trying to understand how it works with the various committees. The officials will know this better than I do but the problem with the HTA and orphan drugs is the low level of information available. Often families say to us that they cannot satisfy the necessary numbers – they simply do not have them. Some assessments may set the bar higher in terms of numbers but if they are not there, they are simply not there. Often that leads people to believe they are excluded from the process and it seems to them as if they are not being listened to. That is why they come back through their local representatives.

Both clinicians and patients are involved in the health technology assessment. Is there an opportunity at that point for patients to have an input and influence over the process so that they will be there and will be able to discuss their experience? Often we are not dealing with big groups and we do not have a large population from which to gather the information. It seems sometimes that the information is not gathered from the people who are there because there are not enough of them.

Mr. John Hennessy

The issue of transparency was central to the committee report in February 2018. We have been attempting to address it in several ways. I believe we have made progress but if there is more to be done we would be happy to engage with members on how we can strengthen the transparency factor.

I wish to remind members that the National Centre for Pharmacoeconomics reports are published on the website once they are concluded. The deliberations of the drugs group are notified to the particular companies involved. There can be commercial sensitivities around what goes into the public domain. However, we are particularly keen to be as transparent as possible and to report publicly the outcome of the drugs group deliberations.

Deputy Louise O'Reilly

My question was about moving along through the process.

Mr. John Hennessy

I am getting there. The recommendations of the drugs group come to the leadership team. The deliberations of the HSE as a public body are available as well. The items on the agenda are public knowledge and the decisions taken at those meetings are publicly available and reported on the HSE website. If there are ways to put more transparency around it, we would be happy to explore those and deliberate on them.

Deputy O'Reilly's point goes to the issue of delay or the perception of delay. There is no deliberate slowing down or delay factor coming through the process at our end. What we see and hear about is access in different jurisdictions but, as Mr. Flanagan mentioned, access can mean different things from one jurisdiction to another and it is not always easily comparable.

We aim for a two-fold agenda. We want to ensure access to medicines for Irish patients and to ensure as much value as we can get throughout the process. We have been exploring ways to speed up the processing with the pharmaceutical sector. Those discussions are productive and proactive in fairness to the industry. We have ongoing discussions on the matter. We have put additional resourcing into the process so that we can handle the additional numbers of applications coming through now for reimbursement. It is a two-way process. Getting to the final price is one of the issues we tend to struggle with in dealing with the pharmaceutical sector. We are trying to resolve that as well. Mr. Flanagan mentioned that the list price and the final price can often differ substantially. It is important we get access to drugs. It is also important that we can get value and demonstrate that as well.

Deputy O'Reilly asked about the rare diseases review group and publishing the work programme. I will ask Professor Barry to speak to that point. Deputy O'Reilly also asked about the Scottish experience. We have been examining that closely and we see opportunities there. Some of the steps that have been taken so far this year in response to the committee report are modelled on the Scottish model of transparency and patient involvement. We hope to go further on that as well. Mr. Judge will talk about the BeNeLuxA question and I will bring in Professor Barry on the health technology assessment and patient involvement aspect.

Mr. Finian Judge

A couple of questions were directed to the Department. Deputy O'Reilly referred to the Department being out of the loop. That was a decision taken by the Oireachtas when it passed the 2013 Act. The idea was that responsibility would be solely vested in the HSE. The Minister has no role in the final decision-making process. That is contained in statute in the 2013 Act.

There was a question about BeNeLuxA. As committee members probably know, the whole concept of regional collaboration is in its infancy. There are a few regional collaboration groups across Europe. We signed up to BeNeLuxA in June of this year. The BeNeLuxA grouping envisages collaboration in four domains. The idea is to collaborate around information exchange, HTA, horizon scanning and joint price negotiations. At this point our involvement is in its early stages, as committee members will appreciate. The win so far for us is really around information exchange. In fact, some of my colleagues today are at a meeting in Vienna on BeNeLuxA. We are merely getting into the process at this stage.

That is fine. I thank Mr. Flanagan.

Deputy Alan Kelly

It is obvious that we need an extensive national medicines policy to be driven at departmental level. It is to be hoped that will happen in the next Government. Mr. Judge stated that the Minister cannot get involved in drug reimbursement decisions. If that is so, what happened in the case of Orkambi? Did the Minister become involved contrary to the Act referred to by Mr. Judge or did he have no involvement? Is there a clause in the deal with the Irish Pharmaceutical Healthcare Association, IPHA, whereby drug cases may be referred to the Department in certain instances?

There was a case in Crumlin last week, and we heard earlier from the witnesses that genetic services in this country, which are intertwined with decision-making, are simply not up to the standard required. Is this accurate? I have spoken to people in the HSE who have said they have been trying to employ people for a number of years. The case last week has caused alarm. Is it the case that genetic services are not up to the standard required? That is my second question to Mr. Judge.

My next questions are probably a mix for the other three witnesses. The first concerns affordability, which is an issue in the case of orphan drugs. What is the HSE's position on biosimilars and biosimilar penetration? What are the expected savings in this regard for 2019? I presume that with the budget and projections, the HSE must put in something.

Fourth, what is the typical weighting price for tenders on drugs? How do we compare with other countries, such as Germany and the UK? I understand that in many cases the weighting they have on price in their tenders could be up to 80% while we are generally very low at 25%. Are we not losing money as a result of this?

My last question concerns pembrolizumab, the drug Vicky Phelan is on. I do not expect any of the four witnesses to criticise Government policy, but has any of them ever seen a similar case in which a decision was made to give a drug such as this, which is off licence, free of charge to one cohort of women or men outside of the normal Health Products Regulatory Authority, HPRA, procedure, which is to go two years? Would this be in any way normal? How can it be that Áine Morgan in Loughrea - this is all in the public domain, by the way - is fundraising to go on pembrolizumab? Ms Morgan has cervical cancer but is not one of the 221 women, yet Vicky Phelan, to whom I have spoken in depth about this and who is one of the 221 women, is getting the drug for free from the State. One cohort is getting it for free. Women in another cohort must get a clinician to say they need it, but they do not get it for free. The drug costs €8,500 every three weeks.

I put the following questions to each and every one of the witnesses. Do they believe this is equitable and fair or discriminatory? Do they believe it is legal? I certainly have issues with it. I cannot in any way justify how one can distinguish between two women who have cervical cancer such that one gets a treatment free of charge because of a decision made at Government level and another does not. I would like to hear the witnesses' views on this. I am aware this drug is used to treat other conditions and other cancers but I want to know specifically how within our drug administration policy this is equitable or fair. Have the witnesses ever seen this done before? I have spoken to many of their colleagues from different walks of life and there is deep unease in the HSE and other parts of the health service about this. Something will have to be done about it.

I want to put on record that I disagree with Mr. Judge's opening statement and the Minister quite clearly disagrees as well. He says there are no legislative barriers to the reimbursement of orphan drugs. I ask why then would the Minister make the statement he thinks the system is broken too? They are two directly opposing views from the same Department.

Moving on to trying to get the best value, Mr. Flanagan gave a very good example of getting in there and batting and waiting for a particular drug that went from €3,800 per unit to €2,100 per unit. I have pointed out, on numerous occasions since I was elected, that there are potential savings of multiple millions in biosimilar products. The IPU has done a report which outlines savings of €320 million over three years. The HSE is anxious to hold out for the best price, and I agree with that approach on orphan drugs. Why is the HSE not banging down the door for the savings it can achieve in the context of biosimilars?

I agree with what Mr. Flanagan is saying, that it is down to prescribers as well, but we will be issuing that guidance very early in 2019. I agree that there are lots of potential savings. It is about creating space in order that we can afford the other drugs that we are discussing.

Deputy John Brassil

I welcome that. I would love to see a budget for orphan drugs. I have asked, again on several occasions, for a specific breakdown and I keep getting the reply that there is nothing in the Act to differentiate orphan drugs from any other drug. That needs to be changed. That is why I have submitted a Bill to change the legislative position in order to allow for orphan drugs to be treated separately. If they were treated separately and there was a separate budget for them, then the savings that were made on one side could be looked at towards providing on the other.

With my last contribution, I want the witnesses to look at the Swedish model. They, ironically, have the same proportion of health expenditure and medical goods as we do, yet they use a qualitative analysis in their orphan drugs reimbursement but do not set a threshold. They take the types of things we have been talking about - patient advocacy, human value and cost-effectiveness - over a very small patient group.

When one is looking for data, particularly for an ultra-rare disease, it is almost impossible to get because there might only be half a dozen patients and the type of assessment that is being undertaken will not facilitate this.

Professor Michael Barry

I reiterate what Professor Barry said. The €45,000 per QALY is not a binary decision. If something costs less than €45,000 per QALY, the HSE will always say "Yes". No cost-effective medicine has ever been denied. As the cost per QALY increases, the probability reduces a little but that is offset by the magnitude of the unmet need and the magnitude of the clinical efficacy. The drugs groups try all the time to arrive at a judgemnt on that. It is honorifically challenging stuff for them, for which I do not envy them. The €45,000 per QALY threshold is not a binary "Yes" or "No".

Deputy Kate O'Connell

Mr. Hennessy referred to the possibility of legislation for biosimilars similar to that for generics 15 years ago. That needs to be examined further than at a meeting of a health committee. That is not substitutable and I cannot imagine a situation where we would introduce legislation to force a consultant dealing with a complex condition to change medication based on a direction by the Oireachtas.

In his opening statement, Mr. Judge said there would be a governance review. Has this been sparked by anything?

I am very much on the same side as the witnesses. I understand the challenge of a pharmacist or a medic trying to balance budgetary constraints.

Following Deputy Brassil's contribution, it is of great concern, particularly in the case of Orkambi, that when there is a well-organised group, it can result in a situation where he who shouts the loudest gets what he wants. That makes me uncomfortable, both as a legislator and a healthcare professional. In light of Deputy Kelly's comments, while acknowledging the autonomy of prescribers to make decisions as they always have, it is not fair to say that a decision to jump over certain regulations can be equated to the use of exempt medicines in the normal scheme of things. There is much talk about conscience these days, but some of us might want to examine our consciences if we were answering those who shout loudest over those with no voice.

When pharma and economics blend, some of the language might come across as crass. However, ultimately, if €500,000 is being spent annually on a patient, someone else is not receiving something and that must be remembered. If we have given something to a particular group, does this committee suggest that we should give it to everybody or we take it away from those we have given it to? It is easy for members to come here and throw out loose statements but, ultimately, it is a question of whether we take a medication away from somebody or whether we have a free for all.

I must leave now. I am in favour of there always being a level playing field, where if there must be a decision that goes outside the parameters, it should not be made in these Houses or by a Minister for Health but by a professional at the top of his or her game, namely a consultant operating in his or her specialism. Members who make representations have a responsibility when throwing out terms such as "life-saving" and "people are being denied a chance". Leaving aside QALY and health technology assessment, the reason many of these drugs are not mainstream is because the evidence base is not there.

People in these rooms and elsewhere talk about money for this and money for that, but there is a limited pot. This is difficult for the families affected and they are entitled to fight for themselves and what they want, but we have a responsibility and we must be reminded of it every so often.

I must leave as I have an engagement at 1 p.m. but I will read the replies if the witnesses wish to comment. I apologise for leaving.

Chairman

Perhaps the witnesses will bank those questions while Senator Colm Burke makes his contribution.

Senator Colm Burke

Recommendation No. 13 in the committee's report of February 2018 was to work with other countries on the assessment process. It recommended that the HSE works with other European countries on dealing with assessments to expedite that process. Has anything been done on using the assessment process in other countries to help people arrive at a decision? Has there been co-operation with other countries in that area? There is a great deal of duplication in assessments. If something is passed by European Medicines Agency then each country examines it individually. The idea behind co-operation is that to speed up the process in dealing with that. Has anything been done on that area?

We hear every year that the cost of pharmaceuticals is approximately €2 billion but we do not get a breakdown of that. Are the figures available? The healthcare budget for the coming year is more than €17 billion. Is there a breakdown of what amounts are being spent on generic drugs, patent drugs and orphan drugs?

The other issue that arises is whether there is variation in the use of generic drugs from one part of the country to another. If we can save money in one area, it means more money could be available for another area. Perhaps there is an analysis of that but I have not seen it. We must examine that issue of value for money as well as value for money in the HSE. It is fine to say more money is being poured into healthcare but unless there are efficiencies or value for money, there will not be a benefit.

Mr. John Hennessy

I will comment briefly on Deputy O'Connell's points first, many of which I agree with. The biosimilar and substitution point is fair enough but we have also seen dramatic levels of biosimilar penetration across different jurisdictions and states. While in Ireland it is quite low at present, other countries appear to have moved ahead on substitution. That is where we aim to go as well.

Mr. Judge covered the governance point. The trigger for that governance review is probably the volume of applications coming through, particularly in the cancer and rare diseases space. It has dramatically increased on what it was five and ten years ago.

On the point about who shouts loudest, we cannot control that. At all points we try to ensure that our systems and processes are fair, transparent and equitable and, in particular, that they are evidence-based so decisions are taken on the basis of evidence rather than who is shouting loudest at any point in time.

On Senator Colm Burke's question about cost breakdown, we have a large volume of information on cost and the breakdown of what is happening in pharmaceuticals. By and large, it reveals stabilisation and even cost reduction in the generic and high volume spaces. For example, expenditure on the drugs payment and the long-term illness schemes is currently running at levels similar to those in 2000 on a cost per item basis. We are experiencing a significant increase in the high tech, cancer, orphan drugs and rare disease areas. There are two graph lines. One is stable while the other is increasing. We will be happy to share that data with the committee. I will need to check further about local variation and uptake as I am not sure if we can get that information easily, but we will certainly look for it.

On the Senator's final point on whether we can do more, I believe we can do more to drive value. It is related to the points about biosimilars and substitution. However, it is useful to remember as well that it is difficult to move patients off a drug or product they have been on for some time and sometimes the claims of products are not necessarily that they are curative but that they prevent or reduce deterioration. That is difficult to measure objectively in a manner that would result in the withdrawal of a drug from a patient.

I appreciate the difficulties prescribers have in that regard.

Senator Colm Burke

Can we have a breakdown of the cost of pharmaceuticals on a year-on-year basis by the different areas, whether they are patent, generic or orphan drugs, and if there is variation in different parts of the country? It may not be possible.

Mr. Shaun Flanagan

Deputy Bernard J. Durkan

It is unlimited. This issue was mentioned in passing at a meeting I attended in Vienna yesterday. I am aware it is coming up today in another context. There are two things I want to deal with. First is the emotional element and the life-or-death situation. As Deputy O'Connell said, it is down to whoever can exert the most influence or shout loudest. The problem is that individual patients who find themselves confronted with a particular condition see no way out if they cannot access treatment. They find it particularly upsetting if the treatment is available selectively. It is also unconstitutional. That is the reality of life. It is unconstitutional to prescribe something for a group of people, assuming there are no other barriers, clinical or otherwise, and not to approve it for another. It is as simple as that. I hope it is being borne in mind because not only is it unfair, it is subject to there being no other medical reason as to why they should not avail of it or have it made available to them. They are entitled to it under the laws of the land. It should be borne in mind.

The job this group does is a very difficult one. I have no doubt about it. It is torn in all directions. There is one thing missing. I have spoken about it many times. Everybody else has spoken about it today. If I were asked to find out what impact the cost of a particular drug will be on the budget, which is an issue, I would like to find out from the people who know. I would presume I could press a button and find out where the drugs were being used throughout Europe and what the cost was in each case.

I do not agree with the principle of lying downwind in the negotiating process until other countries have gone ahead. That is not the way to do it at all. The market of 500 million people in the European Union should be used. It should negotiate a first and last point. We should use the weight of the 500 million people the European Union represents. The European Union is there to back that up. I cannot understand why it is not the first port of call. It should be the first reference. It should not be to the country that has negotiated successfully individually. There is an institution for doing it so why does it not happen? Why does it not happen in a way that is most beneficial to the patients and the cost of the delivery of the services? The only way it can happen is by reference to the biggest single market of which we are but one. We are only 4 million out of 500 million. We discussed this 25 and 30 years ago and arrived at this conclusion before the European Union was what it is now. To what extent do we now use that reference when coming to a conclusion on the suitability of the drug for all patients, the cost of the drug and recognising there may be variations in patient reactions?

I would like some clarification about biosimilar and generic drugs. I was around at the time when this discussion initially took place. Many patients reported that generic drugs did not have the same impact, but the clinical experts told them that they were wrong and that it was just their imagination. Does the Chairman remember that? As legislators, we were in a difficult position. We did not know who was telling us what they thought was the truth and what was accurate. Legislators can only work based on the information they are given. As such, I would like a clear indication of the extent to which those two thought processes have been merged to give us some idea as to what we should be doing in future while also keeping costs in mind.

I cannot believe that we are still using 14 reference countries. It is all nonsense. It gives an average, but we know how averages are determined, so we will not go into that. For God's sake, can someone somewhere initiate a method whereby we can have a reference to the best possible people in the drug-buying business? The drug companies will, of course, give drugs to some people for free to prove they are effective and to make a bargaining position when dealing with people like the witnesses. That is what they do and that is the commercial world in which we live. There is no use in pretending that it does not exist when it does. It is up to the HSE to set up a structure whereby it can determine which drug is the safest and most effective and appropriate and what is the best value at which it can be purchased so that patients in Ireland pay at least the same as they would in other European countries.

The joint committee adjourned at 1.40 p.m. until 9 a.m. on Wednesday, 28 November 2018.