Dr. Maggie De Block:

I thank the Chairman, Senators and Deputies very much for inviting me to this session regarding a national action plan for patient access to innovative medicines. In the tight time slot allocated, I would like to explain how Belgium under my tenure as Minister of Social Affairs and Public Health can serve as an example of good practice.

First, I will explain the pharmaceutical pact although I will not enter into detail. I will highlight the most important aspects to demonstrate our case. Next, I will explain in a nutshell the BeNeLuxA initiative. We felt efforts to increase international co-operation is to the benefit of patients with rare diseases. Ireland was the fifth country to enter into the agreement, for which I am very grateful. I will emphasise budgetary aspects and procedural characteristics and how to influence the time of access to a reimbursed product for the patient.

In light of the elections of 2013, I thought it was important to consider a tailored pharmaceutical policy and there were several good reasons to do so. Together with representatives of the board and the generic and brand name drug companies, we decided on a multiannual agreement that emphasised four main topics for the benefit of the patient. The first was to guarantee better access and lower the out-of-pocket cost for the patient. Next, the pharmaceutical industry in Belgium is an undeniable asset of our national economy. Often, public opinion is very critical regarding the pharmaceutical industry and thus regulating the transparency and ethical aspects was the third pillar of the pact. Finally, we had to agree on a budgetary pathway that would allow growth and access to innovation but at the same time keep the budget under control. This meant that structural savings were necessary for some aspects and an agreement at an early stage would increase mutual understanding. One of the spillovers of this policy was the reduction of the time to access, although the pharma pact did not include structural reforms of the procedures that lead to the reimbursement itself.

Several factors influence the time delay in which a therapy will be accessible under reimbursement for the patient. One aspect depends on the strategy of the company itself, for instance, preferences for bigger markets. It is hard for national governments to influence these strategical choices. The second aspect that contributes to the time lapse in which a reimbursed drug is available is public investment and creating a favourable ecosystem. The last aspect is the administrative procedure to apply for reimbursement and how long this procedure takes, at which milestone a company can file for reimbursement, how much room there is for negotiation, what the conditions are regarding data and the evidence to obtain reimbursement for a certain medicine.

An important aspect that steered the time to access to reimbursed drugs was the budgetary framework. As we can see from the budgetary evolution, the pact scheduled structural budget cuts but at the same time left a margin for controlled growth.

On an annual basis, 0.5% growth was foreseen. There was negative growth in the previous years. In reality, the average growth was 1.4%. One of the reasons for this was an underestimation of the amount in the pipeline that would enter the market. For some medication, like immunological medication, we thought that the train was still far away but it was in front of our noses. For some pathologies, only poor national epidemiologic data was available and it was difficult to estimate the price. Approximately €1.4 billion of new measures and reimbursements in pharmacy were allowed.

Nevertheless, all the savings and expenditure cuts foreseen in the pact were implemented. Among others were the patent cliff, the clawbacks and encouragement to prescribe the lowest priced drugs. In 2018, the pharmaceutical industry paid a total product tax within the public healthcare insurance budget of more than €729 million. The budgetary framework had the perspective of four years because the elections were in 2018, so the pact was in place from 2015 to the end of 2018. As of 2019, the budgetary measures were taken without a multiannual perspective and, as can be seen, this resulted in an immediate rise in the budget.

In my opinion, to accelerate access and reduce waiting time, a sound budgetary perspective is equally as important as the administrative procedure itself. This brings us to timelines of procedures and reimbursement of other pharmaceutical products. Once the European Commission has endorsed the European Medicines Agency, EMA, market authorisation, pharmaceutical companies can apply for reimbursement in the Commission for reimbursement for medicines. That is stated in the administration of the ministry. A classic procedure takes a minimum of 180 days and due to possibilities to stop the clock for the pharmaceutical firms - for instance, to prepare answers to questions for the committee, the time-lapse might increase. If no solution is found in this Commission, a managed entry agreement can then be negotiated by law, covered by the confidential clause in a contract that defines the rights and the obligations of both of the parties. The operationalisation of the decision to reimburse a medicine sometimes took up to two months after notification. We used technical intervention to shorten this timeline.

Taking market authorisation as a starting point, the minimum procedure time is 180 days plus 120 days so that results in 300 days. However, when we look at the statistics on the average delay to availability, it is much longer at 440 days. If we wanted to further reduce this time-lapse, structural reforms of the procedures would be necessary. The fast track is an example of such a reform. We created a fast track for new drugs with new indications. The old procedure still remains. However, if some drugs prove their quality and robustness of outcome, in terms of quality of life or other indications, then the procedure is shortened for the new indications. This procedure of EMA is then one plus one month. Learned conditions in data to support the demand are directly linked to the new indications.

This was mostly done for immunotherapy which was very new in the treatment of cancer, for example. Since the introduction of the fast track in 2017, 20 indications for immunotherapy have been reimbursed and the impact of these procedures in terms of an adjusted quality of life and years gained is enormous. This was a big achievement and great progress has been made in treating patients for diseases for which nothing was on the market previously.

This system needs to answer so-called "compassionate use" for unmet medical needs and applications can be submitted even before market authorisations are licensed. The procedure can lead to early access for the patient two years earlier than in the regular procedure. Most of the innovative drugs are reimbursed under a managed entry agreement. Specifically with regard to orphan drugs, in the period between 2016 and 2019, 29 new products became available in Belgium. In Ireland, it was only 12. Between 2014 and 2020, we signed 33 new orphan drug products for 36 indications for reimbursement. Previously, it was only a few a year.

With regard to expenditure for managed entry agreements, if a company does not comply to contractual objectives, then refund levies are charged. Managed entry agreements, MEAs, now have a share of 11% in the total refunds for medicines budget exceed levies. That is compared to 1% in 2014. We can see that the share has been growing.

We will now come to the BeNeLuXA initiative. It was first Belgium, the Netherlands and Luxembourg. Austria and Ireland then joined but BeNeLuXair sounds more like an airline so we decided to keep the name because it was so well-known, but we were very glad that Ireland joined us. The BeNeLuXA initiative involved international co-operation. We started on horizon scanning, health technology assessment and shared policy expertise and practices. Towards the end, we also added in price negotiations which were not simple because all countries had different administrations and different systems of reimbursement. You might ask why we did it? I mentioned earlier that bigger markets are more interesting to the pharmaceutical companies, and possibly lead to lower prices for the patients. This was one of the main motives for considering co-operation between smaller countries because it resulted in added value for the patients. It is clear it is not only about reimbursement. The horizon scanning taught us much and helped us understand what could be expected to enter the market in coming years. Through mutual recognition of each other's health technology assessments, reimbursement procedures run more efficiently and effectively. Information sharing is always good to improve the market position as are joint negotiations for pricing. For rare diseases, the prices are very high. We also wanted more transparency on pricing between the collaborating countries. The first practice case was Spinrasa. Last year, with the BeNeLuXA countries, Ireland also reached an agreement for the reimbursement of Zolgensma.

To conclude I will outline, in my opinion, the most important lessons learned from the pharma pact and BeNeLuxA. The multi-annual framework for the budget allowed for the budget allowed for better guidance of the pharmaceutical policies. Having several procedures to come to reimbursed medicines increases access. Within these procedures there is also room to deploy several instruments. For example, it is possible to have fees and taxes and to establish different entry moments to apply for reimbursement, reducing delays in the decision process. A sustainable reimbursement ecosystem creates spillover effects that promote accelerated accessibility for the patient. In the mid term, BeNeLuxA should ideally evolve to enhanced co-operation.

I thank Senators and Deputies for their attention. Of course, I am willing to answer questions and members may also contact me via the address given to the committee. I will also share everything I said today with the committee.

Colm Burke (Cork North Central, Fine Gael)
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I thank Dr. De Block for giving her time this morning because I know she is very busy. I was looking at the figures and time periods for the BeNeLuxA agreement and there seems to be much variation in even within BeNeLuxA countries around the availability of drugs. For example, in the Netherlands, its average period is 213 days, whereas the period in Belgium is 440 days and in Ireland it is 477 days. There is also the question of price negotiation. Can more work be done in getting price negotiation between the BeNeLuxA countries and the pharmaceutical companies expedited so all countries could have drugs available in a timely manner? Will Dr. De Block explain why the Netherlands, which is part of the BeNeLuxA agreement, has drugs available on average within 213 days whereas the period for Belgium is 440 days and Ireland is 477 days?

Dr. Maggie De Block:

The system in Netherlands is very different because here we have national insurance administration procedures. In the Netherlands it is very different and there are private organisations and the Zorgverzekeringswet. It has several organisations or insurance companies and they take the decision. It is not down to the administration in the country or the minister taking the decision. They negotiate between them and give the information to the minister about how much they are prepared to pay for the drug in question. It is very different here and we have a national administration, the RIZIV-INAMI, and it has private partners.

Colm Burke (Cork North Central, Fine Gael)
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If countries looked at the system in the Netherlands or Germany, where the period is 120 days, would we get a better result and drugs available at an earlier date than with current processes?

Dr. Maggie De Block:

In Germany they have another system. When there is EMA approval, it is reimbursed immediately after a month, I believe. After a year, Germany re-evaluates if the drug will be reimbursed and under what price that would happen if it continues. That is one way. The current minister is also looking for a shorter way and we are also looking at systems in other countries. We cannot do what they do in Netherlands because we do not have the private partners that decide what to pay for the drug. We are looking at the German example, and that would shorten by a lot the period for reimbursement. Everyone is searching for the miracle procedure.

David Cullinane (Waterford, Sinn Fein)
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I welcome Dr. De Block to the meeting and she is very welcome. I thank her for the presentation. Nobody here believes there is a miracle solution or a magic wand but we are looking at how to improve the reimbursement system in Ireland. Looking at all the data, Ireland lags far behind the EU's top performers in the reimbursement of orphan medicines that are EMA-approved. That is consistent when we look at tables over the past ten years in particular. There is clearly a problem with our system.

My questions relate to the Irish system and how it compares with systems in the EU. Has Dr. De Block studied the Irish system itself? How does she see the reimbursement system in Ireland comparing to systems in the European Union generally? How does it compare with the reimbursement system in Belgium?

Dr. Maggie De Block:

I did not have the time to study the Irish system. I thought I would explain ours and how we improved it. I am sorry but I can and will do it before sending a written response to the question.

We must take into account all the stages. This includes procedure and how negotiations are done. We must, of course, shorten the periods because people with rare diseases are waiting and some have no time. Spinraza is an example. People who are treated on diagnosis have a much better quality of life than people who have for years not had the right therapy for their symptoms. I would like to help the committee in that regard but to do so I would need information on the necessary data indicating where the process is taking too long, for example.

David Cullinane (Waterford, Sinn Fein)
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I thank the witness.

Pádraig O'Sullivan (Cork North Central, Fianna Fail)
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I thank the committee for facilitating me. I am substituting for Deputy John Lahart this morning. I welcome Dr. De Block to the meeting and it is interesting to see our system compared with the Belgian system.

Dr. De Block's presentation indicates we must ensure we have a sustainable reimbursement system not just in Belgium or Ireland but right across the EU. Will she expand on what she means by that? In Ireland, as speakers have said, our waiting times are quite lengthy. We have had answers to parliamentary questions indicating waiting times ranging from 800 to 1,200 days, depending on criteria. Is that reflective of a sustainable reimbursement system?

In Ireland we do not have a specific pathway to assess orphan drugs and it is part of the mainstream assessment. In Belgium is there a specific assessment pathway for orphan drugs, or is it like the Irish system, where every drug is assessed under the same threshold?

I have a question on the number of drugs in Belgium approved over the past few years. Will Dr. De Block give a breakdown of the past two or three years? Until recently in Ireland the number has been quite low but there has been an injection of funding in the past couple of years that helped us to approve a certain amount of drugs. How do we compare with Belgium in the list of drugs approved?

The last question is in regard to patient groups and their involvement in the process. Do patient groups in Belgium feel there is open and transparent analysis of the drug approval process and the reimbursement system or do they feel outside of the loop, so to speak, as they do here? I know that is a lot of questions.

Dr. Maggie De Block:

That is okay. First, the Deputy asked if there is a different procedure for reimbursement for orphan drugs or other drugs. We have the same but the issue is having enough data. That is why we need the contracts because, sometimes, the orphan drug does not have enough patients treated before it enters reimbursement. Of course, it is difficult to decide based on small patient numbers. These are rare diseases and there might only be five patients in a country and, as Belgium is a small country, we might only have two patients. That is why we are working together with other countries.

On the numbers that we tried to find for the committee, we found them for the orphan drugs but not for all drugs. Because of coronavirus, our administrations have been overwhelmed with work, in particular the health department, and there has been a delay in giving statistics. When we have them, we can send them to the committee. The administration did everything to continue other care because there were not just patients with coronavirus but also those with other needs.

Concerning the patient organisations for orphan drugs, they are well organised in Belgium. They are not around the table when the negotiations take place, as they would like to be, but there is transparency and they have the information. There are so many organisations and they cannot all be around the table. Everyone will ask for himself and because everything in health is important, whether it is one disease or another, the moment people are involved on behalf of their family or themselves, it becomes very emotional. That is why we choose transparency but not to put them around the table of the commission which decides. Of course, it would be good if we could harmonise the criteria in the Benelux countries, which would also give more transparency to the organisations. It would also allow the data and information to be valid in the five countries, which would give them more information.

The faster we can achieve our commitments, the more favourable the reimbursement agreements and the better they will be. We know the prices of medicines are rising, certainly for orphan drugs. It is a big concern for our administration that the budget is always higher every year. We can save lives and we can have better quality of life for certain patients, but we know that, at the end, everyone has to pay the bill, which is a big concern. For that, we need a new pharmaceutical pact but, up to now, the current Minister has not arrived at the end of the negotiations.

I hope my collaborator noted the Deputy’s questions and that I have answered most of them.

Pádraig O'Sullivan (Cork North Central, Fianna Fail)
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I thank Dr. De Block. I have one supplementary question. Belgium provides a fast-track, early temporary reimbursement model. As Deputy Colm Burke said earlier, Ireland's waiting times are significantly longer than most of our EU counterparts. Will Ms De Block give us her analysis of what that fast-track model has done in terms of better outcomes for patients? Quite often in Ireland, it is thrown at us that the HSE and Department of Health through tough negotiations save the State hundreds of millions but, in that analysis, there is very little reference to the benefits of earlier interventions through early access to medicine. Has Belgium done any analysis on how patients’ lives have improved and how health outcomes have improved, be it an economic assessment or a qualitative assessment of better health outcomes?

Dr. Maggie De Block:

Yes, there have been calculations not only by us, but also by patient organisations and universities. With the fast-track model, patients receive the medicine they need much earlier. I can send the committee a longer answer but I will give a resumé now. We saw that there was a calculation of how many life years we saved. I am not sure of the figure.

Mr. Benoit Mores:

For immunotherapy, it was 9,000.

Dr. Maggie De Block:

A university calculated that when we had the immunotherapy reimbursed much earlier, at a certain moment a few years ago we had already saved 9,000 life years for patients - it could be one year for one patient and ten years for another patient. There were different cancers to treat. For example, for melanoma, where people were already in stage 4 with metastatic symptoms, some have been in remission for years and they look like they have regained their life and also their quality of life. In life years, it was 9,000 after a year and a half.

Of course, once the immunotherapy was there, the early track was also for treating lung cancers and old cell cancers which previously could not be cured, as well as ovarian cancer. Once it was reimbursed, it was used as a third-line therapy for many cancers and then came the data that it helped and was okay, and, as I said, the train was there for many patients to be helped with that. I have much more information on that and I will send the answers to the committee.

Colm Burke (Cork North Central, Fine Gael)
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I want to come back in on the issue of the price negotiation with the Benelux countries. Germany is able to make drugs available within 120 days, although I know it is a different process.

Obviously, Germany is a bigger country with more patients with a rare disease. In Ireland there may be as few as 20 people who need a particular treatment whereas in Germany there might be 2,000 such people. If the Benelux countries worked together at price negotiations then the system could progress better and negotiations with pharmaceutical companies could get up and running at an early stage so that drugs are available faster and at a competitive price, like the way Germany does it. Is this being examined at all from the point of view of the six countries that are part of the BeNeLuXA initiative?

Dr. Maggie De Block:

As I said, we asked Germany and France to also co-operate but the bigger countries are stronger in negotiation because, as the Deputy has said, the numbers of patients are much higher and there would be more interest for the companies to have reimbursement in Germany or France than in Ireland or in Belgium, for example. The bigger countries do not need us and they do not want to join us, even though this is still within the European Union and it is a problem that bigger countries have other prices. Of course, if this is due to their economic situation, for countries such as Hungary and Romania for example, I would understand this because there are other problems there with regard to getting access to medication such as antibiotics. I understand that this is because of economic reasons. For Germany and France, however, one cannot say that. This is how it has always been. I do not agree with it. I believe that a German patient is a patient who needs treatment but an Irish patient is the same patient who has the same needs. For me this is the key. There should be much more co-operation for patient care in the European Union. As the committee is aware, it is not for nothing that in the EU member states, there is no agreement to take care of the patient care together. This was also one of the problems when we had the Covid situation. The Commission had to negotiate for the first time for the price of the vaccines. We can see that it was not a success at first. It took them all so much time to negotiate with the firms.

Yes, one could also do a shared risk, if one begins to negotiate in the earlier phase such as phase 3 or phase 4. Then one could to do risk sharing also with the pharmaceutical firms. However, in the negotiations for the BeNeLuXA initiative we have always said it must be safety first. We must be sure that we are helping the patients and not giving medication that could have secondary effects or which may make their condition worse. It is safety first, but if it is safe and they can give us the safety ticket, then we should do more efforts with countries together.

It is always the same, however, that the big countries say "We do not need you". I believe that it could benefit their patients too, and certainly when it comes to rare diseases. This is why we also created the rare diseases network. It is a network of co-operation of our academics and researchers. It did not get us too far but we did create a big network concerning rare diseases. Indeed, in the smaller countries we only have a few of the patients there.

Bernard Durkan (Kildare North, Fine Gael)
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I thank our witnesses and I congratulate Dr. De Block. It is undoubtedly great to hear her speak on the subject, on which I have campaigned on for years. I believe, as Dr. Block obviously does, that we are all members of the European Union, we have all an equal entitlement to equal treatment and at an equal price as everybody else, regardless of which country we are from. The time has come to do something about this. I believe in early diagnosis, early treatment and early payment with no nonsense. The patient is waiting. The patient is a prisoner while waiting and while the rest of us talk about it, equivocate about it, and put forward nice proposals and so on and so forth.

It is time that we brought European Union countries together in the whole area of sourcing the orphan drugs, pricing the orphan drugs, and not allowing ourselves to be held to ransom by the drug companies. This is what is happening. Obviously they want to do deals with the bigger countries because it suits them. How does Dr. De Block feel about us smaller countries, those of us who are vocal on the subject, intervening at EU Commission level to try to ascertain the basis on which it is intended to treat all countries within the European Union in the same fashion? They can do this, they should do this and are bound to do so under the Internal Market and the Single European Act. That is the question. How does Dr. De Block feel about us going that way?

Dr. Maggie De Block:

I totally agree with the Deputy and that is why I created the BeNeLuXA initiative in the first place. It is because the patient is waiting and it is also for the future impacts. It was Paul Janssen of Johnson & Johnson who said that we must work at it and find more therapies because the patient is waiting. We should be able to make a statement to the European Commission but we need to do this together. We must work together with as many countries as possible. We could also mobilise the patient organisations on this issue because they also have representation at European Union level. This is also what we did to create the network for the academics for the rare diseases. The more we are, the better it will be heard. This is what we could do now when, I hope, we come out of Covid times. This is the moment to make that statement: that we see we are not an island. This is also how we can break the tradition that the pharmaceutical firms always go to the bigger countries. They are the reference for the pricing and the little countries are not attractive in that regard. I will certainly inform our liberal friends in the European Union. We have the ALDE group which is the Alliance of Liberals and Democrats for Europe. It would be good if it could come from all sides of the European Parliament hemicycle that they should join. People may have their political colour and conviction but when it comes to patients, we are all the same. Inside we are all the same so we should not make any difference.

Bernard Durkan (Kildare North, Fine Gael)
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Dr. De Block has clearly indicated and laid out a policy line for the European Union in general. It is something we should support in every possible way to link up with her and with other like-minded countries throughout the European Union with a view to achieving the level of equilibrium that is required to give the best value for money, the best treatment and the earliest treatment to the patients who are waiting, even as we speak. I will not delay any further other than to say I would strongly support Dr. De Block if she was to contact the European Commission on this subject as a matter of urgency.

It would be a huge leap forward for the patients who are awaiting treatment, testing and so on, individually, across Europe. It is a totally artificial way of dealing with the situation. There is no necessity for that at all. It can all be done in one fell swoop and with application right across the EU.

Bernard Durkan (Kildare North, Fine Gael)
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Absolutely.

Dr. Maggie De Block:

Yes, we had some cases like that where pharmaceutical firms contacted families and made a whole campaign in the journals, on television, did declarations and so on to put on pressure for reimbursement of their medications. That is of course very sad for the families they are using and it is a big pressure, and we saw it also in the Netherlands for the minister. We should not let the firms decide for us but we should co-operate with them. That is what we did with the pharmaceutical pact by being together every month. Every time, we saw how the implementation of the pact went because all we put in it was done and in full transparency.

The problem with the negotiations for the vaccines was, first of all, there were negotiations and there were pipelines but there was little evidence it was the right vaccine at that moment. It was also very new for the Commission to do that work. Now that it has done it, it is the right moment to say it must also look to put the countries together and to speed up the procedures that are possible, because then the pharmaceutical firms have only one partner to negotiate with. Then all the patients will have access at the same time. Currently, it takes perhaps two years longer in one country than in another, which is the worst case, and other countries are not able to pay for it. There are patients who have to move from their country to another, which is of course almost impossible for sick patients. I do not think it is the moment to ring the bell in the European Commission. I am not a member of the European Parliament but I could mobilise the ones I know from my political group, the Alliance of Liberals and Democrats for Europe, ALDE. I also have had good relations with the European Commissioner, Ms Kyriakides, of Cyprus. I think she would be sensitive to that too.

Dr. Maggie De Block:

No, but we had more tests introduced to the heel test. We put other disease tests in there so now it is two bloods. How many diseases are we screening for now?

Mr. Benoit Mores:

It is 13.

Dr. Maggie De Block:

It is now 13 and it used to be nine. We made progress in that.

Dr. Maggie De Block:

I thank the Chairman. I would have liked to come to Dublin because I was there with the European Presidency, but unfortunately it is not the right time for me as there is too much work to do here.

Dr. Maggie De Block:

I thank the Chairman very much.

Mr. Liam Woods:

I thank the Chair for introducing my colleagues. With the support of the Department of Health, patients and their families, the national rare diseases office, NRDO, was established by the HSE in 2015. This was a key recommendation of the first National Rare Disease Plan for Ireland 2014-2018. The HSE and Department of Health each have a role in this plan for patients with rare diseases.

Since establishment of the NRDO, information scientists have provided a free information helpline service for members of the public, patients and healthcare staff. Information provided includes current and reliable evidence-based information about rare diseases and informs patients where in Ireland the centres of expertise are located. It also gives information about support groups for people living with rare diseases. The NRDO maintains and updates a database on rare diseases called Orphanet Ireland. The work of the NRDO, in collaboration with multi-disciplinary clinical staff and rare disease patient representatives, in relation to care pathway development, reduces waiting times to see a rare disease specialist and shortens the time to a diagnosis, which we know can be prolonged for people living with rare diseases.

The European Commission cross-border directive supports strengthening co-operation between highly specialised healthcare providers across the European Union by the establishment of a system of European reference networks, ERN. The NRDO is the co-ordination hub for Ireland's ERNs. A total of 18 collaborative groups from more than 40 centres of expertise in Ireland, led by five major academic teaching hospitals, have received full ERN membership to date. ERN membership facilitates sharing of information and access to expertise across Europe. It provides Irish patients and healthcare professionals access to the latest research and education opportunities. ERNs are predicted to greatly improve the access to and quality of care for Irish rare diseases patients, including access to participation in clinical trials, where Ireland may have limited capacity due to our small population. Funding will be sought in the Estimates process to support these five hospital sites to set up their rare disease registries and to support clinicians to participate in research.

The NRDO is currently in the process of designing care pathways, including paths to diagnosis and genetic testing, across the 18 ERNs. These pathways set out a multidisciplinary approach to care for the more common rare diseases which would address current service gaps, particularly in psychological and social care support and timely access to genetic testing and counselling.

The NRDO has led on and ensured that Ireland is a participant in a number of key EU grants related to rare diseases. There is now an opportunity to participate in an upcoming European Commission ERN 2023 integration grant, with further grant opportunities thereafter.

The HSE intends to work closely with the Department of Health on developing an updated plan for rare diseases. As with the National Rare Diseases Plan for Ireland 2014-2018, the involvement of patients and their families living with rare diseases will be fundamental to the new plan. With regard to the issue of genetic testing of newborn babies for rare diseases, the purpose of population screening is to reduce death and illness and improve quality of life through early detection and treatment of disease. In Ireland, the national newborn bloodspot screening programme is responsible for the heel prick test for babies when they are three to five days old. The test identifies treatable metabolic disorders and other inherited or congenital disorders in infants and allows for timely intervention and treatment. The programme currently screens for nine disorders. In 2019, a national screening advisory committee, NSAC, was established. This is an independent advisory committee which advises the Minister and Department of Health on all new proposals for population-based screening programmes and revisions to existing programmes. The committee plays a significant strategic role in the development and consideration of population-based screening programmes in Ireland.

In November 2021, the NSAC had a public call for submissions which was open to patients, public and health professionals and open to proposals for a new screening programme or changes to one of the existing programmes. All areas of national screening were considered, including newborn screening.

In addition, in recent weeks, the HSE has established an expert steering group, including patient and patient advocacy representation, to develop a national genetics and genomics strategy for Ireland. This group will develop an agreed strategy and implementation plan for genomics in Ireland. This work will improve access for patients to genetic expertise, which can impact timely diagnosis and treatment.

Bernard Durkan (Kildare North, Fine Gael)
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I welcome the statement. I refer to the three important issues, which are speed and access to early diagnosis, access to treatment, and reimbursement. There is a uniform system right across the European Union. All countries might not be able to aspire to that but at least all countries could try. I believe it is essential that we move in that direction as quickly as possible, for all the reasons already said and repeated many times. Some 500 million people live in the European Union. That has to mean something when it comes to purchasing medicines and providing treatments across the European Union. All members of the European Union are entitled to this. How does Mr. Woods envisage, in these early days, the setting up such a process that encompasses all the issues that I referred to?

Mr. Liam Woods:

We should acknowledge that we heard Dr. Maggie De Block and the discussion about this earlier. The Deputy already heard the presentation on the Benelux process and approach and some of the limiting factors relating to differences between systems that would need to be overcome. A policy matters underlies what the Deputy is asking us. I will ask Mr. Flanagan to address progress in the BeNeLuXA discussions.

Bernard Durkan (Kildare North, Fine Gael)
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The important thing is to recognise that anything that is done-----

Mr. Shaun Flanagan:

Ireland joined the BeNeLuxA initiative several years ago. Last year was the first time there was a successful medicine taken from the start of the assessment process through to reimbursement. The medicine in question is Zolgensma, for spinal muscular atrophy. In that assessment, Ireland took the lead on the health technology assessment side. The National Centre for Pharmacoeconomics, led by Michael Barry, did the assessment for the three countries. Arising from that assessment, the three countries agreed to go into a joint negotiation process. That process went reasonably well. Within it, there were several matters that had to be ironed out. When one gets into these things, it is amazing how different systems can be. For example, the Irish negotiators had to deal with the fact that the HSE would have to pay 23% VAT on Zolgensma. It can be matters as simple as that. One of the other countries does not pay VAT on medicines. Within the negotiations, that had an impact on how the deal had to be configured so that it worked for everybody. One of the great learnings from the process was that when one is in those joint negotiations, one has the assurance that at least two other countries are getting the same deal as the country one is representing. One's country is doing no worse and no better than those three countries. As the Deputy stated, there is the additional population benefit of negotiating for well in excess of 20 million people - probably 30 million people - across the three countries, rather than just 5 million people, or the small number of people within the population of 5 million who will benefit.

From the perspective of the HSE, we are committed to the BeNeLuxA process. This week, several of the team are engaged in meetings with BeNeLuxA colleagues to try to make progress on that. As part of the BeNeLuxA process, we are very close to the first major output from it in the area of horizon scanning. A new horizon scanning process will be in place that will enable all the countries involved to benefit from shared information in respect of what is coming down the road in terms of the medicines and health technology assessments for which we have to plan and the expertise we have to get in place to be able to do those health technology assessments. Obviously, there is the benefit now that where there is agreement, we can rely on the expertise within three countries.

Bernard Durkan (Kildare North, Fine Gael)
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To go back to the core issue, the population of the European Union is in the region of 500 million people. Those 500 million people are not heard in these processes. Only a limited number of people are listened to and have influence. My argument is that, as a member of the European Union, we are obliged to abide by its rules in most situations, and we do so in most situations, but in this particular situation it seems that everybody is equal but some are more equal than others. That is untenable. We cannot operate on that basis. As time goes by, it will become increasingly important to do the things that we want to do, and to do them quickly. Waiting for 160, 180 or 200 days and so on is crazy, particularly as we have access, in every sense of the word, to the European Union, and it to us. We are all part of the same Union. The same regime should apply and the same negotiation. The team should negotiate on behalf of everybody; not on behalf of the most powerful. I believe it is necessary to set the wheels in motion, and to do so soon. When a pandemic occurs, it is too late to do those kinds of things. It concentrates the mind but it is too late. If we have done our work beforehand, we should be able to avail of the benefits of economies of scale. We are not doing so at the moment.

Mr. Liam Woods:

We are not disagreeing with the Deputy but we cannot act on that because it is clear some of those matters are political and for discussions at European level. I get his point on economies of scale across a wider population.

David Cullinane (Waterford, Sinn Fein)
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I welcome Mr. Woods, Mr. Flanagan and Dr. Martin. My first question relates to the reimbursement process. As Mr. Woods is aware, that issue was discussed earlier. In 2018, the committee recommended that there be a review into Ireland's reimbursement process. My understanding is that Mazars conducted that review but it has not been published. It is important that the report be published. Is there a timescale for its publication? It is important for all of us, including those in opposition and members of the committee, to be informed as to the recommendations that were made, what the thinking is, how the system can be improved and so on. It is frustrating that it has not yet been published. I assume it is a matter for ministerial decision. Can our guests provide a sense of when that will happen?

Mr. Liam Woods:

It is a departmental report. We cannot comment on its publication. It is really a question for the Department and potentially the Minister. As regards the wider comment made earlier in respect of reimbursement and timescales, multi-annual budgeting and so on, we obviously find some sympathy with that.

David Cullinane (Waterford, Sinn Fein)
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Perhaps the committee can write to the Department of Health seeking publication of the report. It arose from one of the recommendations of a report of the committee.

David Cullinane (Waterford, Sinn Fein)
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Okay. We will try again.

In the context of the national newborn bloodspot screening programme, the witnesses may be aware of a Bill brought forward by Senator Norris some years ago that was working its way through the Seanad. It seems that Bill has fallen off a cliff. It would have required the national screening advisory committee, NSAC, to prepare a report and make recommendations to the Minister for Health to expand the range of conditions tested by NSAC within six months. We need legislation in this area. We need to improve transparency and accountability in respect of NSAC. Is it true that the committee has made only one recommendation so far to expand the screening programme?

Mr. Liam Woods:

I thank the Deputy. I ask Dr. Martin to respond on the NSAC.

Dr. Ciara Martin:

The committee was set up relatively recently, in 2019. At that stage, there was a new recommendation going through, which came to the bloodspot screening in May this year. We now screen for nine conditions on that bloodspot screening. The committee has been set up. As we stated, it has gone to the public and to clinical professionals to ask for nominations to be considered for screening. To my knowledge, it has received more than 50 recommendations and will be going through that procedure. The committee is independent and will be following international guidelines. It will be making recommendations in respect of policy to the Department of Health and in respect of operation to us. It is hoped that we will expand the national newborn screening to include more inherited and other conditions.

David Cullinane (Waterford, Sinn Fein)
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I understand it is a process and has to be somewhat independent. My point, however, is that we should be looking at international comparisons in terms of what other countries are doing and what screening is being done in other EU countries that is not being done here. It is like the reimbursement process we discussed earlier. The process can be cumbersome and there is a need for reform to make it quicker and more efficient. I appreciate the NSAC is only up and running since 2019 but the legislation proposed by Senator Norris would introduce more accountability in this regard and more urgency in terms of how it reports to the Minister for Health. That is probably not an issue for our guests; it is more for the Minister and the Department of Health to consider it. I appreciate that the NSAC does its work, and I assume it does good work. The Government put a 12-month stay on the Bill, which is the kiss of death for any Opposition Bill as far as I can see. The Bill seemed to have value when I looked at it again but it appears to have fallen off a cliff.

I recommend that we come back and look at the substance of that Bill.

I will also touch on a number of things Mr. Woods said earlier. He indicated that the HSE intends to work closely with the Department of Health on developing an updated plan for rare diseases. Is it correct that the national plan for rare diseases was published in 2014?

Mr. Liam Woods:

Yes.

David Cullinane (Waterford, Sinn Fein)
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Has it been updated since then?

Mr. Liam Woods:

No.

David Cullinane (Waterford, Sinn Fein)
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There has not been an update in eight years. It is important that the HSE leads on any update of the plan. We should not be waiting for the Department of Health to appoint somebody to lead it. If that is required, the HSE needs to be injecting some urgency into the matter. From talking to patient organisations, I know that they are ready to roll up their sleeves and play their part in updating the plan. Is there are any timeframe for when that will happen? Who will lead on the update?

Mr. Liam Woods:

I might ask Dr. Martin to respond clinically to that question. There is a rare diseases office within the HSE.

Dr. Ciara Martin:

The rare diseases office has been established and we have our clinical lead for rare diseases. However, we need to do more and work together across the four areas with our patients, families and those with lived experience. The HSE, the Department of Health and the rare diseases office must work together.

David Cullinane (Waterford, Sinn Fein)
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We have a national plan that has not been updated for eight years. There is a commitment to update it. Perhaps a note could be sent to the committee on precisely how that will happen, if there are indicative timeframes, who will lead, what role there will be for patient organisations and so on. The committee can then consider how we can help in that process.

Mr. Woods stated that funding will be sought in the Estimates process to support the five hospital sites involved in the European reference networks to set up their rare disease registries and to support clinicians to participate in research. That is good and welcome. We need to consider the development of care pathways to allow for patients to access expert care in other EU countries. Funding would have to be provided for that. Could that also be considered? Will that be sought in the same Estimates process?

Mr. Liam Woods:

It will be sought in the Estimates process. Work is already under way on quite a number of care pathways. We will see that progressing and leading to funding requirements. As stated earlier, we are at a stage of developing a national strategy which we will then resource. It will include care pathways.

Gino Kenny (Dublin Mid West, People Before Profit Alliance)
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I thank our guests. I am under a time constraint because I have to speak in the Dáil Chamber so I have only one question. My question intends to get to the heart of the issue around orphan drugs and access to them. There is a perception, and I think there is a lot of truth in it, that big pharma companies have governments and patients over a barrel in respect of the price they charge for drugs. Some of the prices are jaw-dropping. People who are in need of these drugs, which can be hugely beneficial, and their families have a right to get them but those companies, some of which are based in Ireland, have whole states over a barrel when it comes to the prices they charge.

Another perception is that the assessment of these drugs is quite arbitrary. It can be very cumbersome and protracted, to say the least. I could name a number of drugs whose assessment has gone on for years. That is particularly true in recent years. It leaves families unable to get those drugs. It can be extremely stressful and family members have died because they cannot get those drugs because of the price. That is unethical and immoral. Is that perception of big pharma companies and their economics correct or incorrect? Do they have states and patients over a barrel in the context of the prices they charge?

Mr. Liam Woods:

We have processes in place to negotiate price and to assess the clinical effectiveness of drugs for the benefit of patients. I will ask Mr. Flanagan to refer to those briefly. The processes we enter into have delivered significant savings and have brought new drugs into use. I will ask Mr. Flanagan to comment.

Mr. Shaun Flanagan:

There is no doubt but that this area is challenging. Pharmaceutical companies are private corporations and have responsibilities to their shareholders and so on. It is important for a state to have robust processes. We have a health technology assessment which is carried out by the National Centre for Pharmacoeconomics, NCPE. We also have a commercial negotiation process and an independent drugs group, which reviews the outputs of the assessments and the negotiation processes. Any unmet need is considered. The clinical evidence, cost effectiveness and budget impact are considered. The final decision maker in the HSE is the executive management team. There is now board oversight of that. It is one of the areas at which the board looks.

We would like to get more efficient in getting through drugs quicker but we feel we do address some of the excesses that are tabled in respect of price. I will give the committee a feel for some of that. In 2021, the HSE approved 29 new medicines and 21 new uses for existing medicines. The commercial negotiations and health technology assessment process took away €398 million in costs over the next five years for those medicines. The difference between what the pharmaceutical companies originally tabled for those 29 new medicines, 21 new uses for existing medicines and two extensions and what was finally agreed was €400 million over five years. That is one part that may give the committee some assurance in that regard.

Our corporate pharmaceutical unit, the part of the HSE that leads on commercial negotiations, has gone back and conducted a review of the 134 drug group recommendations which were received between July 2016 and May 2021. Of those 134 recommendations, 46 of the drugs involved had orphan designation status at the time of the drugs group review. The original cost effectiveness estimate, which is driven on the one side by cost and by benefits on the other side, was that 78% of those orphan-designated drugs received a drugs group recommendation. Of those, 31 were subject to a full health technology assessment. Of those 31 drugs, 21 had an incremental cost-effectiveness ratio, ICER, which is a cost per quality-adjusted life year, in excess of 100,000 before the negotiations took place. In the final assessment after the negotiations were completed, only nine of those drugs had an ICER of more than 100,000. That shows that even in the space of orphan drugs, the negotiation process makes a significant difference in respect of the price that the State is paying.

Under the standard cost-effectiveness measure, something is regarded as cost effective if it satisfies a cost-effectiveness ratio of 45,000 per quality-adjusted life year. For the orphan drugs, four of the 31 with positive recommendations satisfied those criteria before negotiations and 18 of them could be deemed cost effective after the negotiations. The process makes a difference and saves the taxpayers a substantial amount of money. More importantly, that enables the HSE to stretch the money it has as far as possible to provide access to as many medicines as possible. I hope that gives the Deputy some context.

Gino Kenny (Dublin Mid West, People Before Profit Alliance)
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Most people understand that states have to get value for money on drugs that can be beneficial to their citizens. Do our guests acknowledge, at the same time, that pharmaceutical companies are making vast amounts of money? They are beholden to their shareholders rather than the people their drugs are meant to treat.

Some of the profits that these companies make are absolutely incredible. Obviously, they will argue that they put all the research and development in and the final product, the drug itself, was produced because of the money they put in. That is fair enough, but there is something immoral about the vast amount of money they price these drugs at. There have been instances where literally entire states do not get certain drugs because of price. I know individuals who simply have gone without because they cannot get it because of price. To me and to most people listening, that is not right. Mr. Woods must acknowledge that these companies make a vast amount of money but do not have an obligation to states and patients in relation to access.

Mr. Liam Woods:

Some of the drugs we are speaking of and that Mr. Flanagan referred to, we would acknowledge, are expensive. The processes we engage in can reduce the cost and allow us to provide more care to a wider number of people. The motivations of the companies is not a matter for us to comment on. We certainly experience the high cost of new drugs and new technologies. As the Deputy rightly said, I am sure the companies themselves would reflect back on the research, other requirements and, indeed, the products that never come to market. However, it is not for us to comment on their motivations. We experience high cost, but also, in some cases, high benefit, it has to be said.

Colm Burke (Cork North Central, Fine Gael)
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I thank our guests for making themselves available and for the presentation. I just want to go back to the national rare diseases plan. My understanding is that there is not any one person in the Department of Health who is in charge of national rare diseases at present. Is this causing its own problems for the HSE in regard to progressing the review of the national rare diseases plan?

Mr. Liam Woods:

It will be, obviously, a question for the Department. We can and have worked with the Department collaboratively on this issue and will-----

Colm Burke (Cork North Central, Fine Gael)
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I am asking Mr. Woods if there is not a problem if someone has not been specifically designated to be in charge of the review of this national rare diseases plan. Is this then causing frustration within the HSE?

Mr. Liam Woods:

I am not aware of the frustration. There has been previous engagement and in the previous plan there was leadership within the Department around this.

Colm Burke (Cork North Central, Fine Gael)
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However, there is no timeframe.

Mr. Liam Woods:

I hear that. However, I just hear a question about frustration-----

Colm Burke (Cork North Central, Fine Gael)
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Would it not be helpful from the HSE’s point of view to now engage with the Department, first about trying to have a specific person who the HSE could engage with on a regular basis, and second, that there is a clear timeframe put in place about the review of the national plan.

Mr. Liam Woods:

Yes, and in response to the earlier question from Deputy Cullinane, we will revert with the timescale and process for that, worked out jointly with the Department.

Colm Burke (Cork North Central, Fine Gael)
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It might be helpful for the committee if we could get a breakdown of the number over the past five years – Mr. Woods referred to the approximately 29 new orphan drugs approved in 2021 – of new drugs that were brought into the equation in respect of reimbursement and the cost over that time. I understand there were negotiations with the pharmaceutical industry on reducing the cost of drugs that are used on a day-to-day basis. What were the cost savings as a result of those negotiations? In other words, the idea is that you would have cost savings in one area that you could then use up to make sure that we have new drugs coming on the market available at an earlier date. It would be helpful to the committee if we could have that information provided to us.

To continue, my colleague who was speaking previously was giving out about the private sector. I understand that, in fact, the time period for a drug being made available in the Netherlands is shorter compared to other countries. We had the former Minister of Social Affairs and Public Health from Belgium in earlier. In the Netherlands, it is 213 days whereas in Ireland it is 477 days in relation to a drug being made available. My understanding is that they have an engagement with the private sector in the Netherlands compared to here. Have we at any stage looked at that to see how we can make medication available in an earlier timeframe?

Mr. Liam Woods:

I heard the contribution and saw the graph actually. I think we were about 477 days versus the Netherlands figure the Deputy just quoted. Belgium I think was at 447 or so. A differentiation the speaker made was that there are seven or eight insurance funds operating in the Netherlands, which are in kind of a private capacity, whereas in Belgium with the social insurance, there is an administration, as she referred to it. There are different models, and I think she was pointing to that. The question on what the model needs and what effect the model has on the timing of the introduction perhaps is slightly different. On the question of whether we can and do look at the reimbursement process and how could that be speeded up, as Deputy Cullinane referred to, we can of course within our current model of delivery. The Netherlands and, indeed, Germany, as the speaker identified, introduce immediately on approval by the European Medicines Agency, EMA, as I understand it.

Is there anything Mr. Flanagan wanted add about the reimbursement process?

Mr. Shaun Flanagan:

Again, on the reimbursement process, we have been going back over the past number of years trying to find ways to speed up things. One of the things that rang true for me was the sustainable reimbursement model that Dr. De Block talked about and the importance of certainly and surety around the amount of funding that will be available in future years. At times, because of the economic situation, we have obviously been challenged around the budget for new drugs that has been in place, or not been in place, in particular years. That can have an impact on the ability of the HSE to fund products. Again, echoing what the Deputy said, the importance of things such as the 2022 Irish Pharmaceutical Healthcare Association, IPHA, agreement in terms of making headroom is critical. It is critical to be able to do that.

One of the benefits, strangely enough, of the pandemic has been that previously on our negotiations, we were face to face, whereas obviously, with the pandemic, we have had to get negotiations more virtual and had MS Teams-type scenarios. The negotiating team has continued that model now that we are hopefully – we thought anyway at least – exiting Covid-19. That has given us additional benefits in terms of just being able to schedule. Again, like many parts of the HSE, there is a view going on at the moment around whether we need additional resources to accelerate our ability to carry on more, if you want to put it that way, negotiations on at the same time because, obviously, at the moment, we have a limit in terms of the team being fairly put to the pin of its collar. That is something again that may very well feature in the national service plan in terms of an ask for additional resource.

Colm Burke (Cork North Central, Fine Gael)
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I wish to just touch on two other issues. One is the cystic fibrosis drug available for children. I think there are about 35 children in the country who require this particular medication and it is not being made available. What is the timeframe for coming to a decision on that particular drug?

Second, a steering group for the development of a national genetics and genomics strategy was set up. We are way behind other countries in relation to the whole issue around genomics and genetics. Even compared to Northern Ireland, the number of people working in that area in the State is only about 16, whereas in Northern Ireland it is far more. What is the timeframe in relation to that review? I always worry about committees being set up without any clear timeframe put in place. We need to employ many more people in that area if we want to make sure that, for instance, the family members of a person with a particular medical condition that may be genetic also can be tested for that particular medical problem. We appear to be way behind other countries on that issue.

Mr. Liam Woods:

I will take the last part first. The timing on the national strategy is quarter 3 or early quarter 4 this year. We hope that will be subject to consideration for the 2023 Estimates. I entirely agree with the broad premise of the Deputy's statement that we need to build up our resource in terms of capacity. The national doctors training programme, NDTP, did a review in 2019 of the availability of doctors in this area and the likely need. At that time it was clear we would need to grow by a factor of four. That is before referencing counselling, therapies and other supports. We expect the strategy will bring forward conclusions in that area before the end of this year. There is a group in place, as the Deputy referenced, to drive that.

On the particulars of the CF drug, we will respond in writing, unless one of my colleagues can make a comment on that.

Colm Burke (Cork North Central, Fine Gael)
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My understanding is there are 35 families in the country. They are writing to us and lobbying us very much on this drug being made available and it is not being made available. I ask that it be reviewed at the earliest possible date.

Mr. Liam Woods:

To be helpful to the committee, we will respond on where it is at right now. We will come back quickly on that.

Colm Burke (Cork North Central, Fine Gael)
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I appreciate that.

Mr. Shaun Flanagan:

I will correct one thing concerning the numbers I gave - 29, 21 and two. In case anyone assumed they were rare diseases, that was all drugs. The figures I gave on the HTA and the ICERs were the orphan drugs.

Cathal Crowe (Clare, Fianna Fail)
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I welcome our guests. I have been following some of the meeting. There are a few meetings coinciding this morning so forgive me if some of my questions repeat what others have asked.

I will take up where my colleague, Deputy Colm Burke, left off on cystic fibrosis and Kaftrio. These 35 children, last month, in the age-six-to-11 category were due to receive this drug and the floor has collapsed from under them. They have had their blood test and were anticipating this. Given the age category these children belong to, most had a full awareness of what was ahead of them and of the new lease of life this miraculous drug would have given them. I have met with many of the families and they are devastated that there is a delay and procrastination.

I think there is a bit of detail the witnesses could give us today. They have said they will respond in writing but can they account for the delay and what the Department is doing?

Mr. Liam Woods:

I thank the Deputy. I am not sure if we can say anything right now.

Mr. Shaun Flanagan:

I was the lead in the negotiation in 2017 which led to the putting in place of the portfolio deal with Vertex. In 2017, the HSE entered those negotiations clearly in the position it wanted access to all the Vertex portfolio within the resources available to the HSE. We sought to include every drug in the portfolio deal. Vertex was not willing to do so. At the time of the negotiations an enormous amount of money had to be approved to provide access to those drugs. As we exited the negotiation, we made clear to Vertex that it would be almost impossible for us to find additional resources for access to additional drugs from the Vertex portfolio.

Since 2017 the HSE has spent more than €300 million. I cannot give the exact figure because it is commercially confidential. It has provided Vertex with more than €300 million for access to the cystic fibrosis drugs. The HSE is committed in the next five years to providing well in excess of another €300 million to Vertex for access to the new drugs.

Between 2017 and 2021 the HSE was dealing with the same negotiating team, in the main. Three amendments were made to the deal. On each occasion Vertex acknowledged the position the HSE had taken in 2017 and provided access to additional patients. This year Vertex did not acknowledge what was said in 2017 and did not accept that was the HSE position clearly enunciated to Vertex. It has taken the view it is entitled to additional funding for those drugs. That is the position at the moment. The HSE is working as closely as it can with Vertex and has met its representatives eight times. I cannot give a timeline on outcome or a guarantee of outcome.

Cathal Crowe (Clare, Fianna Fail)
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Mr. Flanagan has explained that very well. I met with the Minister, Deputy Stephen Donnelly, and Mr. Flanagan has given the extra layers of detail he is privy to. I know this is sensitive, that negotiations are ongoing and that Mr. Flanagan is mindful of all these children waiting in the wings for the news. Can he give any indication of when these negotiations might wrap up? We do not need to know the details of the negotiations but is there any indicative timeframe within which the HSE and Government hope to wrap all this up?

Mr. Shaun Flanagan:

I cannot give a timeline and it would be unfair to give one. At all points, the HSE is trying to provide access to as many drugs as it possibly can. Particularly in this portfolio and in dealing with this company, we have tried to provide access for every patient we can within the resources available to us. We are committed to continuing to do that and to engaging with Vertex for as long as it takes but I cannot give a guarantee on outcome because there are two parties to the negotiations.

Cathal Crowe (Clare, Fianna Fail)
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I appreciate that.

Mr. Shaun Flanagan:

I also have to be clear that if additional funding is required, there is a process we have to go through. That normally involves an assessment process. We have tried to put the negotiation in front of that assessment process to expedite this and get to a solution as quickly as possible.

I should have acknowledged at the start that I understand absolutely that people would be very upset that they do not have access to the drug,. That is not where any of us would want to be. We would want to have been in a position where we could have provided access quickly, as we did for all the other licence amendments as they came along.

Cathal Crowe (Clare, Fianna Fail)
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It is important that whatever is negotiated is all-encompassing. We are now talking six-to-11-year-olds but those under six are also waiting with bated breath to see what happens their age cohort. Please factor that into all of this.

Mr. Shaun Flanagan:

We find it ironic that people 12 and over have access to this drug. That is an important point. I will not say any more than that.

Cathal Crowe (Clare, Fianna Fail)
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It is devastating. I know Mr. Flanagan is doing his best. I spoke to one dad whose son has this and misses out by a number of months. That boy watched his cousin die during Covid from cystic fibrosis complications. It is a life and death matter. I do not wish to overstate that.

The date, 1 January, is the cut-off date for kids qualifying for age categories in the GAA. That is the age cut-off most kids home in on. Most 11- and 12-year-olds think about whether they are playing under-13s or under-14s hurling next year. I do not wish to trivialise it but weeks and days are qualifying or disqualifying these kids for a life-saving drug. It is so important to state and restate that.

I turn to the economics of it. This is not directed at the witnesses, but the HSE and all Government institutions operate as silos, in a way. This will be examined through an economic lens but other arms of the public health service would see this in a different light. I had cause yesterday to be in the Children's Ark ward of University Hospital Limerick, where children are currently hospitalised due to CF complications. The economics determine a drug becoming publicly available in the Irish market with Government backing and funding, but on the other end of that spectrum are kids on drips and laid up in hospital beds when they should be enjoying the last week or two of the school year.

I thank the witnesses for what they are doing. I know they cannot go into further detail. We will hopefully come back to it as a committee.

Mr. Shaun Flanagan:

Just to be clear, it is not always just economics. Unmet need, the magnitude of the clinical evidence and all those things are factored into the drugs group decision. We have significant evidence, as I flagged before, that we often reimburse medicines, even when they do not satisfy conventional cost-effectiveness thresholds, as you might call them. Economics is not the only thing considered.

Cathal Crowe (Clare, Fianna Fail)
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I appreciate that. The Pembro drug is being championed by Vicky Phelan. Can the witnesses offer any commentary on the prevalence or uptake of that in Ireland? Is there any headway they can flag?

Mr. Shaun Flanagan:

There are a number of immunotherapy drugs. Pembrolizumab, Nivolumab, Atezolizumab and Avelumab are all very similar drugs. Each has multiple uses, or "indications" as they are called in the wording of the European Medicines Agency. Pembrolizumab is reimbursed for a wide range of indications.

On the indication for ovarian or gynaecological cancers, to the best of my knowledge there is no European market authorisation for that yet. I am open to correction and will double-check, and as a consequence it would not be going through the process yet. When it gets a market authorisation in Europe for that -----

Cathal Crowe (Clare, Fianna Fail)
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Yes, EMA clearance.

Mr. Shaun Flanagan:

Yes, exactly. I understand there are special arrangements for the 221+ cohort around access to the drug but I need to clarify that and will come back with a note to be sure.

Cathal Crowe (Clare, Fianna Fail)
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I thank Mr. Flanagan for his responses.

Frances Black (Independent)
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I thank the witnesses for their very precise presentations. I was going to touch on some of what Deputy Crowe spoke about relating to young people. The question was answered very clearly so I thank the witnesses for that. As legislators, we see people with rare diseases. It is devastating to see that their families and supporters have to lobby and protest to get access to certain drugs. It creates a highly adversarial relationship between the patients and the institutions responsible for their care and places a great burden on people who are struggling. As Deputy Crowe said, it is a life-and-death situation. Is there anything we can do to relieve them of this burden or how can we do that?

Mr. Liam Woods:

Overall, we should acknowledge that we are typically talking about new therapies that are beneficial and helpful. The Senator is right in saying that in the early stages of introduction it can appear problematic because we are in dialogue about how to bring those into quick and effective use. In some responses to parliamentary questions recently, we have flagged dialogue with patients and families, and improved and extensive dialogue with patients and families is very important. That is included in our strategic thinking as well as around individual drugs and what assessments might be undertaken in the NCPE.

Dr. Ciara Martin:

Aside from the drugs, the question is how we support families and people who are living with rare diseases. With the national rare disease plan there has been a lot of progress made in supporting families. With the setting up of the National Rare Diseases Office there are supports for families. By reaching out to the European networks, we are able to offer advice and counselling to people who need it. We are able to put them in touch with the right specialists at the right time so that people know where to go for the right information. The setting up of the 18 ERNs means we will have centres of excellence in Ireland where we can cohort databases, get established with research and be able to offer advice on treatment protocols. That is very significant for families who need to be able to reach out and know that, although their family member has something rare, they are not alone and that someone knows about it and can give some information on the medication. We are all used to going on various platforms like Google just to know what is out there, and for someone with a rare disease to go out and not find anything is very difficult. The National Rare Diseases Office has done a lot of work in that respect. It was part of the plan. We acknowledge there are some parts of the plan that need to be developed past 2018, but the feedback on the initial part is very positive.

Frances Black (Independent)
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That is very good to hear. I look forward to seeing how it expands on an ongoing basis. Will the officials elaborate on the sorts of illnesses that can be detected by population screening? How can that be expanded?

Mr. Liam Woods:

Mr. Flanagan has a comment on the Senator's previous question and then I will ask Dr. Martin to comment on screening.

Mr. Shaun Flanagan:

The HSE, the Department of Public Expenditure and Reform, and the Department of Health were engaged in negotiations around a new framework agreement with the pharmaceutical industry in 2022. I should acknowledge that part of the discussions on that were how both sides could improve the throughput of medicines through the process and how we could find ways to shorten the timelines. We are committed to trying to address some of the issues that came up there. We see somewhat of a change from some companies in how they submit their pricing to us in that they tend to get to the end quicker, as it were, where before we would have had fairly extended negotiations that might have gone on longer than they should have. We get a feeling that at least some companies are getting to the end point quicker, which is helpful for all of us.

Frances Black (Independent)
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That is very good to hear.

Dr. Ciara Martin:

Can I clarify if the Senator's question relates to general population screening or particularly newborns screening?

Frances Black (Independent)
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General population screening.

Dr. Ciara Martin:

We have national population screening for breast, cervical and bowel. There are different areas within that.

Frances Black (Independent)
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Is Dr. Martin's area more in newborns?

Dr. Ciara Martin:

I suppose from my paediatric background, yes. On the invitation we were-----

Frances Black (Independent)
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Would Dr. Martin like to say a little about that?

Dr. Ciara Martin:

Newborn screening is with a dried blood spot - the heel prick. We look at all babies who are born within Ireland when they are aged three to five days and we take a heel prick from them. We look for nine conditions. We acknowledge that puts us somewhere in the middle Europe-wide of what is looked at and we would like to expand that. The national screening advisory board is considering that. We acknowledged earlier that we need agility to improve our time from acknowledging conditions should be on that dried blood spot screening to actually getting conditions in place on it, mindful that everything we screen for needs a good pathway, to have evidence behind it and to have treatment for it. I would confident we have the right people on the advisory side who can advocate for children and all the groups that are there. I hope we continue to improve as we go by working with the advisory board and the Department.

Frances Black (Independent)
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That is fantastic. I thank Dr. Martin. The progress on the European research networks sounds quite positive, especially the psychosocial support. Have there been any studies or reports of patients' experiences engaging with this system? Will the officials speak about any personal insights they might have?

Mr. Liam Woods:

I am not sure that we have that information with us. We can come back to the Senator with some references on that, if it is helpful.

Frances Black (Independent)
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I thank everyone for the great work they are doing. Keep it up.

Mr. Liam Woods:

I will ask Dr. Martin to address that question, please.

Dr. Ciara Martin:

The Cathaoirleach is right. The Italians brought in their 40 tests on their dried blood spot screening. Around that time, in 2019, the EU Parliament called for a European-wide approach and for all of us to look at that Italian model. As yet, there is no harmonised system across all of Europe. When one looks at what one can test for on those heel pricks, one sees that 35 to 40 conditions can be done with the biochemical test.

As part of the process which surrounds that, one has to ensure that each of those tests has good quality assurance. That is the most important thing in order that we know we are not finding more positives and, on that basis, treating people who do not need to be treated for diseases. Many criteria go into having a good screening model. Across Europe, everybody is looking at it to try to increase it as best they can. It varies greatly. France checks for one, we check for nine, the UK checks for nine and Italy checks for 40. Everywhere else is somewhere in the middle. I read recently that we need to have the breadth of the Italian experience but we also need the rigour which our own process puts these tests through. This is also the case in the NHS in the UK and in Sweden. We must ensure that we have a test we can stand over and that we are not testing people and telling them they are positive or negative when they are not. We must also ensure that we have a good treatment for every single one of those diseases and that we can stand over it. We are in that process since 2019 with the standing up of the committee. It is slow but I think we are on track. We are not alone across Europe in making this journey.

Dr. Ciara Martin:

The national screening committee will make the recommendation to the Department of Health. It will then be operationalised. The national screening committee hopes that the Department will be recommending more than the nine diseases, that we will speed up the process, and that it will be possible to test for more diseases.

Dr. Ciara Martin:

No, I do not have that but I would certainly try to get that for the committee.

Bernard Durkan (Kildare North, Fine Gael)
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I thank the Chairman. International co-operation within the EU is of prime importance in dealing with rare diseases, with the drugs to meet the challenge of these diseases, and with co-ordinated general action across the EU. I am strongly of the opinion that co-ordinated action across 500 million people gives a much better chance to the patients than any other action that can be taken on an isolated basis across the EU. Our witnesses might comment on that.

Mr. Liam Woods:

I am aware that the earlier dialogue was probably about pricing but the research networks are doing precisely that. Dr. Martin might want to say little more about that.

Bernard Durkan (Kildare North, Fine Gael)
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Yes.

Dr. Ciara Martin:

When we have a patient with a rare disease in Ireland, the research networks will reach out across Europe to other areas where patients with similar conditions will be presenting in order to combine their research, their clinical trials and their knowledge. From a clinical point of view and from the point of view of being able to support families and caregivers, that is going on now across the clinical sphere. In fact, a conference on that issue is happening at the moment.

Bernard Durkan (Kildare North, Fine Gael)
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Yes.

Mr. Shaun Flanagan:

Two committees are involved in the process. The drugs group is the standard committee which reviews all new medicines that are coming to the HSE for reimbursement. Then there is the rare diseases TRC, which was in effect a recommendation from the previous report of the Joint Committee on Health for more patient engagement. One of the challenges around the rare diseases TRC is that because of its special nature, you have to constitute new expert members each time a drug goes to it. Basically, you need the clinicians who are the experts in that area to populate that committee.

The drugs group is a more general committee. Usually, it is not possible to make a quorum in August but other than that it meets monthly. In an effort to try to make the process as efficient as possible, the drugs group looks at a medicine first. If it is an orphan drug and the drugs group is minded to say "Yes", it does not send the drug to the rare diseases TRC because the HSE will have said "Yes" already. If the drugs group has some concerns or is unable to say "Yes" on the basis of the assessment in front of it, it then engages with the rare diseases TRC to try to acquire additional information to enable the drug to get over the line. A judgment has been made that this the most efficient way to utilise the expertise of the rare diseases TRC. Rather than asking it to look at every orphan drug, we ask it to focus on the orphan drugs that we are having difficulties around. That is why it seems to meet infrequently. When it has a medicine in front of it, my understanding is that it meets frequently and works hard.

Mr. Shaun Flanagan:

The biggest challenge around the availability of new drugs relates to the funding of them, regardless of whether they are orphan or non-orphan drugs. I tried to give information earlier which made it clear that even though in many instances there is an assumption that the only matter and factor that the process considers is cost-effectiveness, that is not the case. I used to be the head of the negotiating teams in the HSE. I have moved from that role and I am now a member of the drugs group. I see, along with my colleagues on the drug group, that we look at everything. We look at unmet need, clinical evidence, cost-effectiveness and economic evidence such as the budget impact. We look at the representations we receive and at the lived experience of the patients as they are submitted in the National Centre for Pharmacoeconomics, NCPE, documents. People have an opportunity to provide information as part of the NCPE process around their experience of what they believe the drug will do for them. All of that information is looked at by the drugs group. There is ample evidence, some of which we can provide afterwards to the committee, that drugs are approved even when they are not cost-effective in a conventional sense.

It should be a matter of recognising unmet need, recognising that the drug works well and that it will make a substantial difference to an individual patient's life. Then it will get approved.

The reality is that for rare diseases, orphan drugs are often very expensive. There can be difficult negotiations. The same can be true in the case of non-orphan drugs. There can be difficult negotiations in trying to get the budget impact of a drug to a level that the State feels is reasonable. As I say, there are the industry engagements. I refer here to the 2022 IPHA agreement. A significant feature of the deliberations on the latter relates to sharing the information on what would work better on both sides. One of the issues on which we all agreed was that it would help if we could in some way get to the end of the negotiation quicker. That would involve the companies being in a position to get to their final offer. Sometimes that can be challenging.

Mr. Liam Woods:

The 2013 Act has not proved an impediment to our work as we have experienced it. Obviously, the resources available and the timeframes under consideration can be significant factors.However, the legislation per sehas not impeded us from doing our work. In fact, we are clearer about our duties.

Mr. Liam Woods:

I might ask Dr. Martin to make a comment on that.

Dr. Ciara Martin:

Once our patients are identified, if clinical trials are ongoing across Europe or internationally, selected patients may be entered into those clinical trials as part of our research project. That will help to inform, for example, a particular medication or treatment option that a patient would receive. It is hoped that, with the ERNs, we will be able to co-ordinate that across Europe and that we will be able to give our own families and patients access to participation, if appropriate, in those clinical trials. That would then be a matter for the consultant and clinician who is looking after the patient, as well as the patient, to work out. The NRDO would help to co-ordinate that too. Does that answer the Chairman's question?

Dr. Ciara Martin:

Rare diseases, by their nature, are rare. That makes it difficult. We are making the world smaller by being able to search online for experts who are working in areas. Families are coming to clinicians or doctors with information on diseases that they have obtained from the Internet. Then, by means of the NRDO, etc., we are able to look at people who are specifically looking at a rare disease. This may be a disease that, for example, one in 100,000 people will have. If you have that, you may be the only person in Ireland who has it. In that case, you will want to link up with people across Europe or across the world who have a similar disease. You will want experts looking after you to have the required knowledge. If there are enough people and there is a company that is willing to put a drug forward for trial, then a number of people will be able to co-ordinate in a community and participate in a trial. Ideally, if it is a drug trial, there will be a number of people on the drug and a number on a placebo. They will not be aware whether or not they are receiving the drug. Then you need all the rigour that goes with the clinical trial, such as close attention to side effects and whether the medication is delivering what it says it will.

For every drug that comes forward as an orphan drug and gets licensed, there are many others that will not. Unfortunately, families will go through clinical trial and it will not deliver the results that were expected. It is a difficult area to be involved in. It requires much rigour on the part of the families, the clinicians and the doctors and nurses who are looking after their patients on the trial. However, it is worth it when it delivers something like a cure. It requires careful co-ordination with the right people to make sure that you are getting the right information at the right time. That is where our ERNs are worthwhile.

Mr. Liam Woods:

It is important to distinguish between a specialist interest and rare conditions, because I understand that in the generic sense a doctor may have a specialist interest in something. However, rare diseases are, as their name implies, truly rare. Therefore, the connectivity across Europe and the research networks are important. That integration fund, as I understand it, is to support interaction across Europe for clinicians and institutions with common interests in rare conditions, of which there are more than 8,000. Work is ongoing to address the main rare conditions that are impacting in our environment, as was set out earlier. From our point of view, the networks are important. They are important for patients and families, as well as for clinicians to share evidence and experience and to hopefully develop new ways of treating. From our point of view, they are useful. To address the point the Chair is raising, they provide a forum whereby specialists with common interests can interact, if a specialist in Ireland may not have a sufficient case volume to have sufficient experience in isolation.

Dr. Ciara Martin:

That is where our NRDO has its strength. That was initially set out in the 2014 to 2018 plan to support families when they been faced with a rare disease diagnosis. That is the resource that we provide for them through the NRDO. That is where to go for official support. They will guide you to where you can get support. They will put you in contact with patient advocacy groups across the world that are legitimate. They will put you in touch with or they will give you the details of what specialists legitimately looking into that area of rare disease. They will support you through that journey. That is one of the key functions of our NRDO. It has been set up together with patients, carers, families and clinicians.

Colm Burke (Cork North Central, Fine Gael)
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I thank the Chair. Mr. Woods referred to the genomics issue stating that the committee is set up and so on. I understand we have one specialist in that area per 700,000 of population in the Republic whereas in Northern Ireland it is one per 285,000. This has a significant benefit for the health sector. If we are at that stage here, how long before we could get to a ratio of one to 285,000 the same as Northern Ireland? Obviously, a plan has to be put in place. We have to get the people trained and with the experience, but I presume that will take time. Has any indication been given as to when we can get to the same ratio?

The second issue I want to touch on is the Benelux agreement with the six countries together working with the pharmaceutical industry to try to come to an agreement on various issues about a drug being made available. What is the medical connectivity between the six member states? Is there any work being done on that? To a large extent Ireland has been very much attached to the UK when it comes medical development. With the UK out of the European Union, is there a need to focus on working with those other five countries from a European point of view as regards the European Medicines Agency, Europe going forward, trying to get the best price for drugs and getting drugs them at an earlier date?

Mr. Liam Woods:

On genomics and posts, particularly consultant posts, if the Deputy has not seen it, we can send him a copy of the National Doctors Training and Planning, NDTP, report, which is a 2019 report. It states the numbers. There were four people in post, a complement of seven and a requirement for 15. There has been a locum appointment approved in this area but I am not sure it has been filled since. Further posts would be subject to estimates and the completion of that plan. In timescale terms, it is reasonable to assume it would take 12 months to fill such a post, and maybe longer, because it is a relatively new area. It would take some time. I am happy to copy that report.

On the Deputy's comment on the BeneluxA, I might ask Mr. Flanagan to contribute. I think it is a wider point the Deputy is making around the European Union and co-operation internationally. Clearly, the ERNs in this area are very much that. In fact, in the area of orphan drugs, we had a close look at what was happening in Scotland because they had some very interesting work completed in this area. Is there anything clinical specific to the Benelux process on which Mr. Flanagan would want to comment?

Mr. Shaun Flanagan:

No. My experience of the BeneluxA is on the price and reimbursement assessment side of things. That is the part of it with which I am familiar. I am not aware of any other engagements on a medical community-----

Colm Burke (Cork North Central, Fine Gael)
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Would the witnesses accept that if we are trying to get a drug approved or we want to make it available, there has to be some connectivity between the medical people here and the medical people in those Benelux countries so that there is co-operation?

Mr. Shaun Flanagan:

When I say that, I assumed the Deputy was talking about a pan-approach. If it involves specific medicines, obviously, each of the countries will be engaging with its medical communities. In that way, they are linked if there is specific medication going through the process.

I was answering the question in the converse. I thought the Deputy was asking, under BeneluxA, is there an agreement around some sort of engagement between our professional bodies. There is not. It is a price and reimbursement assessment engagement.

Mr. Liam Woods:

The BeneluxA process is really around pricing. I think the Deputy's wider point is around opportunity for co-operation clinically, especially in light of Brexit and where that leaves us.

Colm Burke (Cork North Central, Fine Gael)
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It is also about cross-border healthcare in the sense this is a small country and we will not necessarily have all of the expertise here. If we working on pricing with five other countries, could we also be working with them on the provision of expertise?

Mr. Liam Woods:

The clinical evidence base clearly is international and it is internationally available. The information that would drive some of the considerations around particular drugs is also available. Through research and, particularly in this area, through the ERNs, that kind of information would be available as a backdrop against which decisions around pricing and access to market could be considered.

To the Deputy's wider point, there is co-operation already. The European Union-funded research programmes, including the one to which the Chair referred, are available for us and other European partners to bid into, as it were. It is probably a matter of some regret that colleagues in the nearest jurisdiction may not have quite the same access at this stage despite, as the Deputy says, the historical connections.

Colm Burke (Cork North Central, Fine Gael)
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Going back to the genomics issue whereby there is one expert per 285,000 people in Northern Ireland, has there been engagement with Northern Ireland on sharing expertise so that expertise is available that might not otherwise be, and is there is a quid pro quofor Northern Ireland? has there been any engagement on working together with those in that area in Northern Ireland?

Mr. Liam Woods:

Interestingly, I am not aware of it being an all-island co-operation model. Clinically, I would be fairly sure there is a strong connection. It is something we could come back to the Deputy on. In terms of detail, I do not have it available here. I would think clinically there would be and we would be interested in building a connection.

Colm Burke (Cork North Central, Fine Gael)
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There is an advantage. For instance, we already have people coming from Northern Ireland to Dublin for particular medical treatments. Likewise, we have people from the South going to Northern Ireland for treatment. In this area, could there be more co-operation and, as a result, there would be a benefit for the entire island?

Mr. Liam Woods:

The answer to that is, broadly, "Yes." As the Deputy says, there are already areas of co-operation. Crumlin hospital is doing significant work for people coming from the North of Ireland, and citizens of the Republic can still travel to Northern Ireland under the cross-border directive and the treatment abroad scheme. There is already activity.

The INTERREG programme in Europe supports funding for cross-border initiatives and there is a whole programme of activity in that area administered through an organisation called CAWT, which is an abbreviation. I hope the Deputy does not ask me what the abbreviation is for.

Mr. Ray Mitchell:

It is Co-operation and Working Together.

Mr. Liam Woods:

I thank Mr. Mitchell. I can come back with information on the activities of CAWT. That is administering funds under a European programme for cross-border activity in clinical areas and it has quite significant resources available to it in the current year, as I understand it.

Mr. Liam Woods:

Not that I am aware of, although I am stepping beyond my competence. We are talking truly rare as in very small numbers of cases. As for the definition, to the extent there is one, the Chairman has caught us on the hop there. We are talking very small numbers of cases. Long Covid certainly would not fit into that.

Mr. Shaun Flanagan:

Orphan drugs are defined in the European regulations. I do not want to put a number on it because I am not in the drugs space day to day anymore, but my memory is one in 50,000 people. I would have to double-check that. It is defined, and there is an orphanet. There is an EU reporting mechanism around how a drug is designated an orphan drug. I believe it is a prevalence rate of less than one in 50,000, but I would have to double-check that. There are other aspects around the rules in that companies can make commercial decisions to de-designate an orphan drug for whatever commercial reason a company might have. It is not so simple.

Mr. Liam Woods:

It is about networking to strengthen the overall approach, and the ERNS help with that, but also investment in our environment in clinicians with strong specialist interest. Overlapping with academic appointments is very important in supporting research, education and training. That is the base from which our clinicians are coming. Dr. Martin might want to say more about that.

Dr. Ciara Martin:

The definition of a rare disease can vary across different countries because something that is rare in Ireland may not be rare somewhere else. A rare disease is a very specific disease that affects a very small population and is so rare that it is very difficult, as was said, to get easy diagnosis and support. It is a difficult disease to get enough people to research. As I said earlier, if we are talking about doing a trial for which we need 200 people, we may not have 200 people with this disease across the world. It is something very rare and that is why it needs special recommendations around it.

The definition of a rare disease is very broad, but it can feel very isolating for a family or patient with it because they are faced with something they do not have a community or network around. They do not have a group of people who can meet Deputies or even lobby for what they need. That is why it takes us, the Department and the rare disease organisations to say to people that while they may have a rare disease, we understand what that means and we will work with the family and anybody who has an interest in this, whether it is in Ireland or another country in Europe, to try to get the best care and advice they need and, if there is any treatment out there that is being proposed, we will work with other communities to get research. Does that help?

Mr. Liam Woods:

For clarity, and I am not sure any of these would be a rare disease, but there are conditions that require us to grow our capacity to provide service or to enhance specialism in particular areas. That happens from time to time, which also requires a clinical presence that will take a specialist interest, and investment behind that, to address specific conditions. I am thinking of conditions such as hepatitis C or, as the Deputy mentioned, Lyme disease, where we need to invest in clinical capacity and also consider treatment pathways that were referenced earlier.

Bernard Durkan (Kildare North, Fine Gael)
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Is it feasible to have a centre of excellence in Europe, in this country or somewhere similarly located, with which the medical profession and patients, if necessary, could make contact with a view to identification of a disease, diagnosis, early diagnosis and treatment? Where can we get information on orphan drugs? Is it possible to get it from a central location in such a way that it would be of benefit to the medical profession?

Mr. Liam Woods:

In our environment, that is, in effect, the rare diseases office and the service provided through it. Could that happen at a wider European level? That is about the connectivity between clinicians. From our point of view, the work the office does here can support patients in identifying expertise elsewhere so some of that is in place already. I do not really have anything further to say on that.

Bernard Durkan (Kildare North, Fine Gael)
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What I am trying to get at is that I recently had occasion to meet a patient with a particular form of arthritis, which was horrifying to observe. It was obvious the patient was in very serious pain. In that kind of situation, it must be possible, with all the expertise that is available around the world, to come to grips with that particular type of arthritis in a way that would make life a little more meaningful for the patient and at least give some kind of quality of life to that patient. How do we deal with that? Can we deal with that?

Mr. Liam Woods:

I get that. Normally, the connection for us, through the treatment abroad scheme, is the treating clinician or clinical colleagues here referring to an expert in a subspecialism internationally. The treatment abroad scheme is geared to resource and facilitate those kinds of connections. There is an advisory in specialties, and clinical leads, that fulfil that role in assessing appropriate locations, supporting patients in going to them and creating the connections required to facilitate that, if there is a treatment available somewhere else that is not available here. The treatment abroad scheme facilitates that on an ongoing basis. As the Deputy rightly said, it is not just about rare diseases. It could be any condition for which there is a treatment that we may not have a service in at present.

Mr. Liam Woods:

As much predictability as is feasible around the available moneys to invest in this area in the future is of assistance. Otherwise, it can be a little jerky. We can have stop-start dialogues because there are financial constraints within a time period. As the Chairman is aware, we are funded on an annual basis through a Vote of the Oireachtas. The estimates process is annual for us. We have indications there is a figure of €30 million for new drugs in the current service plan. In the previous 2021 budget the figure was €50 million. Those figures have become known to us through that process. I heard the former Minister's observations. One of her take-home messages was that a multi-annual resourcing framework would be of assistance. Mr. Flanagan would probably find that helpful, as he has had previous experience in the area.

Mr. Shaun Flanagan:

I would not make a recommendation about multi-annual, but certainty around-----

Mr. Shaun Flanagan:

At the end of the day, and I do not mean this to be in any way offensive, processes are more efficient when they are continuous and are flowing seamlessly. Having certainty around funding at the end of the process makes it easier to get things over the line. It reduces logjams. The past couple of years, in particular, have been very helpful in that way. As I said, we had 52 medicines in 2021 and we are at approximately 26. That is, in effect, almost one a week being approved. The way the system works they are approved in pulses but it helps when there is certainty around funding. It particularly helps the decision makers. Mr. Woods is closer to the decision-making then I am. I have worked on the negotiation and on the drugs group, which has the luxury of making recommendations but does not have to make the actual final decisions. It is people on the HSE's executive management team nearer to the board that have to make the difficult decisions.

Mr. Liam Woods:

We are being careful about not going off base, but I noticed a previous contributor did not reference a timescale. It seems to me a window of three years is more than reasonable to have predictability. It is not that we are impeded in making decisions.

It is a bit like asking if more money would help. The answer from me is always "of course". However, from our point of view, we have to recognise that we work within the voted limits of the Oireachtas through the Department of Health Vote.

Mr. Shaun Flanagan:

On the timeline, it is important to flag that horizon scanning in the pharmaceutical field is a challenging enough predictive process. I would not call it a science. It can be hard to figure out what medicine is going to be on the market in two or three years' time. For example, it was expected that a medicine used in the treatment of Alzheimer's disease would be close to market at this stage, but it has had setbacks in the US. It does not look like it will get to the European market in the next year. This time last year, we expected that the medicine would be close to market now. Things like that happen, so it can be difficult to predict. The BeNeLuxA initiative is spending a significant amount of time and resources on trying to make the horizon scanning process more robust, so that we can better predict what our demands or needs are, and what our ask of the Oireachtas will be in terms of what funding we need.