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Dáil Éireann debate -
Friday, 3 Dec 2021

Vol. 1015 No. 3

Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2021: Second Stage [Private Members]

I move: "That the Bill be now read a Second Time."

I acknowledge the work done by former Deputy John Brassil in preparing this Bill. The Bill essentially replicates the good work John undertook in the previous Dáil. I am eternally grateful to him for his efforts in this regard and his support over recent months. I also acknowledge the good work of the Minister for Health, Deputy Stephen Donnelly. I am aware that he has to go to a Cabinet sub-committee meeting now, but since he has assumed office he has provided the largest drugs budget in the history of the State, adding €50 million to the overall budget this year. This investment needs to be acknowledged as it eclipses any previous drugs budget the State has had.

That said, there is still a significant problem regarding the classification of rare diseases and the processes that surround the approval and reimbursement of orphan drugs. Under existing legislation, there is no differentiation between orphan medicinal drugs and mainstream drugs. It is abundantly clear that the orphan drugs used to treat rare diseases continuously struggle to secure reimbursement in Ireland. Juxtapose that with the position in other European states. The disheartening thing for me when I started my research on this was that Ireland is considered a laggard in European terms. That is the unfortunate situation we find ourselves in. Many countries in the eastern part of Europe are light years ahead of us in the provision of orphan drugs.

The reimbursement process is often protracted, public and pits patients against the State. Patients often have to take to the streets, lobby their public representatives or campaign for the approval of drugs. The purpose of this Bill is to amend the Health (Pricing and Supply of Medical Goods) Act 2013 to put in place a structure for the HSE to appropriately assess orphan medicinal drugs when making a relevant decision regarding an item for reimbursement. It will provide clear guidance to the HSE on appropriate use of the health technology assessment, HTA, guidelines when assessing an orphan drug, as well as establish new criteria for the HSE to consider when making a decision.

The provisions of the Bill are set out in four sections. This is a technical Bill. I do not want to get too technical, but section 1 sets out to define what an orphan drug is. Section 2 inserts the definition into the principal Act. Section 3 amends section 19 of the principal Act, which deals with the HSE decision-making process in adding medical items for reimbursement. It deals with issues around cost-effectiveness, the validity of the quality-adjusted life year, QALY, model and the role of the HTA. Section 4 establishes a new set of criteria for consideration of orphan medicinal drugs, mitigating against low quantitative data and guaranteeing the HSE would consider qualitative data presented on an orphan medicinal product for patients. Section 4 also deals with the concept of the State and pharma engaging in risk-sharing and obliges the State to be cognisant of what our European counterparts are engaged in.

I ask the Minister of State, Deputy Butler, to imagine what it feels like to be diagnosed with a rare disease and possibly be told there is no cure, treatment or way of curtailing the pain. I can only imagine the toll it would take on anyone in that position. Imagine being told there is a new drug or treatment that may help manage this pain, suppress symptoms or improve quality of life. Imagine the hope that availing of such a drug would bring to somebody diagnosed with a debilitating disease. Then - bang - the unfortunate reality hits that in Ireland it takes more than 1,000 days, on average, to potentially get access to that drug.

In response to recent parliamentary questions he tabled, my colleague, Deputy Michael Moynihan, received an outline of all drugs that have gone through the process and been signed off on by the HSE. Comparing these with public data available from the European Medicines Agency, EMA, and HSE websites, we see that for June 2020 to 2021, on average, orphan medicinal products took 1,361 days from securing EMA authorisation to being authorised in Ireland. The breakdown of that 1,361 wait is 426 days to make an application, followed by 429 days undergoing assessment under the so-called rapid review. At 429 days, I question its rapidity. A further 289 days are spent negotiating between the State and the pharma companies. Once a drug is approved, there are a further 217 days before it is implemented. In total, that is 1,361 days or 3.7 years. For somebody with a debilitating disease who could be experiencing great pain, that must be an excruciating wait.

Imagine how deflating and soul-destroying it must be for any human being to have a potentially life-changing drug at his or her fingertips, only to be most likely told he or she cannot be reimbursed for it because there is not enough data to support its use, it is in excess of the restrictive €45,000 QALY threshold or fails some other aspect of the health technology assessment. That is the scenario many people who suffer from rare diseases face. It is unconscionable that the State, which has responsibility for the health and well-being of its citizens, would block, refuse or defer any decision on approving a drug because of the arbitrary €45,000 figure. That is the archaic system presided over in this country.

Let us look at what our EU counterparts are doing. I will not compare us with France or Germany because they have far superior resources to us. I will make comparisons with countries like Sweden and Scotland. In Scotland, an ultra-orphan pathway was established, providing a distinct process for assessment for these rare conditions where qualitative data can be used in the absence of significant quantitative data. The Scots also provided for interim acceptance of some drugs. That acceptance and the subsequent reimbursement are conditional on the provision of further data and evidence over a specific period up to three years. Reimbursement is then provided on the basis that the drug in question clearly demonstrates its efficacy. Any drug that is not proved effective would not be the subject of reimbursement. This idea of risk-sharing between the state and the pharma industry means patients get quick access to potentially life-changing medications. If they do not work or something better comes along, and inevitability in this sphere many drugs come along to replace others, the state does not bear the burden of the cost. It is a clear example of the state and industry working together to provide for patients and improve health outcomes. This State, however, continues to apply the most rigid HTA criteria, which gives orphan drugs little or no chance of being approved if they exceed the arbitrary €45,000 threshold, as attested to by Professor Michael Barry, head of the National Centre for Pharmacoeconomics, in front of an Oireachtas health committee in 2019.

Since 2019, 31 drug indications have been approved by the EMA. Scotland has made 19 of these available; we have only made four of them available. The average time to approve those drugs in Scotland was 13 months; in Ireland, 21 months. These are significant differences in timelines considering the serious and often life-threatening diseases we are speaking about.

The assessment for reimbursement in Sweden is based on three core principals. One, everybody has a right to healthcare. Two, priority is given to patients with the greatest need and those most vulnerable. Third is cost-effectiveness. The big difference between Ireland and Sweden is that, while both use the QALY threshold and the Irish model employs the €45,000 threshold, the Swedes employ no such arbitrary figure. All drugs are assessed on their medical efficacy and the outcomes they deliver for patients, while still being cognisant of cost-effectivenes. An arbitrary figure is not used as a bazooka to disapprove a drug regardless of its level of efficacy.

Some critics of this Bill suggest that to remove the rigid QALY threshold we have in this State would somehow weaken our hand in any negotiations with big pharma. This is a fallacy that was created to detract from this Bill when brought forward previously by former Deputy, John Brassil. No doubt it is a myth that will be circulated again at some stage of this debate. It is stated implicitly in Part 4 that any drug must be assessed as to its potential or actual budget impact in any question being analysed. There is no magic money tree out there to approve all drugs all of the time, but it is possible to streamline legislation and guidelines on this issue in a way that gives patients hope but, more important, often gives them life-changing and life-saving treatments.

In regard to the first section, I undertook an awful lot of research over the past few months into this topic. I refer to the drugs Spinraza, Orkambi, Translarna, Zolgensma and to Onpattro, about which I raised an issue most recently and which the Minister of State has spoken about in previous Dáil debates on this matter. The bottom line is that is no way to conduct an assessment for any type of drug. Deputies and public representatives should not be hauled in here pleading and begging with the Minister of the day to approve one drug over another. There should be a clear transparent process with specific timelines. That evidently is currently not the case in light of the 1,300 plus days I referenced earlier in the reply to Deputy Michael Moynihan's parliamentary questions. That is a life sentence and, in many cases, a death sentence for people.

We need transparency in this regard and this Bill provides it. I am open to discussion and amendments of any description so long as the current system is overhauled and improved because clearly it is not working. Many people in the industry will say that such is the high threshold and, often, failure rates in Ireland in terms of this question in regard to orphan drugs Ireland is one of the least attractive places for any pharmaceutical company to come and negotiate.

Ireland has entered into an arrangement with other small nations in the Beneluxa agreement. The logic behind that was that together a group of countries could compete and negotiate with big pharma companies in the same way as bigger nations such as France and Germany do. The group came together with the ambition of negotiating better prices for the State and, ultimately, getting drugs for patients. The Beneluxa group has been in place for two years and only one drug has been approved in that two years through that arrangement. To say that we will work with other EU partners is great, I am all about co-operation and working together and improving the case for the State and the case for patients, but the Beneluxa arrangement has implicitly failed in the past two years in that, to my knowledge, it got only one drug approved.

I acknowledge that the Minister of State is standing in for the Minister, Deputy Donnelly, as he has to attend the Cabinet sub-committee. In the past, there has been near universal agreement on this matter. It is something I am willing to work with other parties on. I am glad to see some of them represented here today. I believe we need to do more for patients. I would welcome the Minister of State's statement on the matter.

I thank the Deputy for tabling this Bill which seeks to address an issue of great importance to us all. I would like put on the record the apologies of the Minister, Deputy Donnelly, who, as the Deputy mentioned, is attending the Cabinet sub-committee, but I understand the Minister engaged privately with the Deputy on the Bill in advance of this debate. I recognise his sincere interest in patients who are in need of orphan medicines. It is a cause he has championed for quite some time. We hear about it regularly at our parliamentary party meetings. I acknowledge the work of former Deputy, John Brassil, on this issue. He, too, was very passionate about it.

The Minister supports the intent behind the Bill. He also wants to secure faster access to medicines for the most vulnerable of our patients. I can safely say this is something we are all aligned on. As public representatives, we are all moved by the tragic circumstances faced by many people suffering from rare diseases. The pipeline of new innovative medicines coming to market remains strong and it is vital that Irish patients continue to benefit from these medical advances. I understand the Minister is open to any ideas or proposals that might improve our existing pricing and reimbursement assessment processes, with a special focus on orphan products. For that reason, he is not opposing the Bill. I do, however, want to use this opportunity to set out the current process and to outline recent reforms that have been undertaken.

The Health (Pricing and Supply of Medical Goods) Act 2013 is the statutory framework under which all new medicinal products must be assessed by the HSE. The HSE is the statutory decision-maker for the reimbursement of such products and the Act ensures that these decisions are made on an objective and scientific basis. In making decisions, the HSE carefully considers a number of criteria that are contained in the Act, such as clinical evidence, efficacy and the available resources. It is only fair to acknowledge that these are difficult decisions and those involved must balance the availability of finite Exchequer resources with ever-competing demands.

The 2013 Act does not include a specific rule set for the assessment of orphan drugs. However, the HSE and National Centre for Pharmacoeconomics, NCPE, have assured the Minister that in making their decisions they have particular regard for the unique circumstances surrounding orphan drugs. These criteria include not only cost-effectiveness and budget impact, but aspects such as clinical need and added therapeutic benefit against existing standards of treatment. The HSE can, and does, take a nuanced approach with respect to the particular challenges posed by rare diseases, such as small patient populations and unmet clinical need. This capacity for nuance is reflected in a number of reforms carried out to date under the existing Act. The rare disease technology review committee is fully operational. It carries out reviews of reimbursement applications for new orphan medicines in cases where the drugs group requires further guidance. This committee gives voice to rare disease patients and to clinicians with expertise in the specific illnesses in question. The committee played a key role in the HSE’s approval in July of Patisiran, a new medicine for the treatment of the rare, inherited life-limiting condition called hATTR Amyloidosis. Using the guidance from the committee, the HSE was able to take account of the challenges faced by patients in terms of unmet need and the potential impact of the drug, alongside considerations of cost effectiveness.

The HSE drugs group has been expanded to also include representation from the National Patient Forum and further clinical expertise in the area of rare diseases. The NCPE’s patient organisation submission process encourages the lived experience of patients, carers and their families, as well as their views on the difference a new medicine may make. This information is then used as part of the health technology assessment of the medicine. Since implementation of this process in 2018, there has been a doubling in the number of patient organisation submissions received and it is expected that this number will continue to increase. The challenge of accessing innovative medicines at affordable prices is one shared by our international partners. It is estimated that 100 new indications will receive European Medicines Agency, EMA, market authorisation in Europe each year over the next five years. The Minister, therefore, fully endorses the State's participation in international collaboration and co-operation in efforts to secure affordable and timely access to new medicines for rare diseases. Our involvement in the Beneluxa international initiative with other EU countries demonstrated its potential to have a positive impact in relation to orphan medicines.

As announced previously, in October the HSE approved the gene therapy drug, Zolgensma, for reimbursement for the treatment of spinal muscular atrophy. This followed the first joint agreement on the price of a drug between Belgium, Ireland and the Netherlands, carried out through the Beneluxa initiative. This Bill presents one such opportunity. However the Minister is also keen to maintain an open mind and be receptive to non-legislative avenues. He wants to look forensically at the current system and identify further options for reform. Increased transparency and communication would make for a more responsive system. The Minister is committed to seeing where this could be achieved across the NCPE, the HSE and rare diseases technology review committee.

Turning to the provisions of the Bill, I wish to outline the relevance of quality-adjusted life year and incremental cost-effectiveness ratio measurements. The quality-adjusted life year metric is the economic evaluation system used by the NCPE to determine the cost-effectiveness of a given medicine. It is a measure of the state of health of a person or group through which the benefits in terms of length of life are adjusted to reflect the quality of life. One quality-adjusted life year is equal to one year of life in perfect health. The incremental cost-effectiveness ratio expresses the value of health outcomes in terms of the cost per quality-adjusted life year. Our experts believe the quality-adjusted life year metric is a valuable tool in the decision-making process. The HSE and the NCPE consider it to be the best method we have to assess the cost-effectiveness of medicines in a systematic and scientific way.

If the HSE were unable to consider the cost per quality-adjusted life year thresholds for orphan medicines, it would have no guidance on their cost-effectiveness to support commercial price negotiations. The executive would, therefore, enter such negotiations in an uninformed and disadvantageous position. The Minister is assured by the NCPE and the HSE that the current system accommodates the approval and reimbursement of expensive orphan medicines. Fifteen of the 43 new medicines approved by the HSE in 2021, up to 12 November, were orphan medicines for the treatment of various conditions, including, but not limited to, rare cancers and spinal muscular atrophy. Together, they represent more than one third of approvals this year. In many cases, these drugs significantly exceeded the guideline cost-effectiveness threshold of €45,000 per quality-adjusted life year. That metric, therefore, does not pose any barrier to the reimbursement of orphan medicines. Indeed, the HSE has expressed concern that without having carried out such cost-effectiveness assessments, it would end up paying significantly more for orphan medicines than would otherwise be the case. The advice provided is that abandoning the methodology would potentially have added an additional €250 million cost over the next five years in respect of the orphan medicines already approved for reimbursement in 2021.

It is clear, too, from an analysis of the trends over recent years that the overriding factor in medicines making it through the process to reimbursement is the availability of Exchequer resources. The 15 orphan drug approvals to date in 2021 were enabled by an allocation in budget 2021 of €50 million for new drugs. By contrast, when no such funding was included in budget 2020, only eight orphan drugs were approved and, moreover, they were approved only when funding was made available through industry price realignments. For this reason, budget 2022 has allocated a further €30 million for the approval of new drugs. This will provide the HSE with the resources necessary to continue providing access for Irish patients to new innovations in the area of rare diseases. We cannot overlook that, in the context of finite resources, paying more for orphan medicines would have a very real impact on the health service's capacity to fund even more new medicines, including subsequent innovations in the area of rare diseases.

To reiterate, the existing assessment criteria in the 2013 Act do not pose an impediment to the approval of orphan drugs. However, our system is not perfect. No one familiar with the challenges faced by citizens with rare diseases would make that argument. The Minister has asked his officials to consider the available policy options in this area. In particular, he is hopeful that opportunities exist to improve the transparency of the assessment process and communication with its stakeholders, including patients and industry. Notwithstanding the concerns I have outlined today, I reiterate that this Bill is honourable in its intent and must serve as an occasion to explore the policy options available to us. For this reason, the Minister does not intend to oppose it, as we continue to strive for an assessment process for orphan medicines that will provide some of our most vulnerable patients with access to the innovative treatments they deserve. I congratulate Deputy Pádraig O'Sullivan on all his work in this area and his determination to bring the Bill to the floor of the Dáil.

I commend Deputy Pádraig O'Sullivan on bringing forward this excellent Bill, which relates to an area in which I have an interest. The previous health committee looked at this issue and made recommendations, many of which are included in the Bill. I thank an Teachta O'Sullivan for his interest in this matter and for continually raising all these important points, as well as his predecessor, John Brassil, and others, from all parties, who have championed this issue for so long.

The first point I want to make is to acknowledge the amazing work that is being done in the area of innovation in medicines and healthcare. We are seeing a lot of groundbreaking treatments, all of which are to be welcomed. Many new medicines have come on stream, including a large number of high-tech drugs, especially in the area of cancer, as well as huge advances in technology in regard to precision treatment and gene therapies. Many of these treatments require high-tech drugs, including orphan drugs, which are very costly but also very necessary if we are to save lives. This ties in closely with drug trials and the need for more funding in that area. When we look at all this innovation, it is noteworthy that many of the high-tech drugs that are coming forward, several of which an Teachta O'Sullivan named, are produced in Ireland by pharmaceutical companies and their workers. Much of the research, development and innovation that is leading to the development of these drugs, which are saving lives across the world, is happening here on our island and in this State.

I am somebody who believes we should be leaders in research and development, encourage innovation and support and reward it when it happens. I have met many people in the industry and they are at a loss when they look at the league tables of countries that can avail of many of the drugs produced on this island through reimbursement processes similar to our own. We languish somewhere near the bottom when it comes to how quickly public patients can access those drugs in this State. Even though we produce the drugs and support the research and development, because of our complicated, convoluted, bureaucratic system to enable drugs to get on the reimbursement list, patients on this island are not able to avail of them as quickly as patients in other countries. That needs to change.

We all appreciate and understand why the Minister, Deputy Donnelly, is not here to take this debate. The Minister of State, Deputy Butler, said in her opening statement, "We cannot overlook that, in the context of finite resources, paying more for orphan medicines would have a very real impact on the health service's capacity". There are many areas of the health service where capacity is needed but has not been delivered by the Government. We have all asked for more capacity in healthcare. In the case of new medicines and drugs, which we are talking about in this debate, the action needed is costly, yes, but it saves lives. People simply cannot afford the huge cost that would be associated with any of these drugs being administered. No individual would be able to fund it. There are times when the State has to step up and give funding because it simply cannot be done by individuals.

The Minister of State is saying we have finite resources and cannot put in place the type of funding that is necessary in this area because we must look at all the other areas of healthcare, which we do. That statement is juxtaposed with what was done in regard to Covid. Let us take a step back and look at how much money was spent on dealing with the pandemic over the past two years. It comes not to tens of millions of euro but billions. The reason we spent billions on Covid was, above all, that we had to protect people and save lives, which involved spending money on testing, tracing and vaccines. It was the pharmaceutical industry that stepped forward in rapid time to create a vaccine from which we are all benefiting. The booster jab is now being rolled out, which is giving us a very high level of protection from Covid. That would not have been possible were it not for the innovation, research and development that is happening in the sector and politicians trusting the science which by and large most of them in this State did.

When Covid-19 came, we responded quickly and we put in the funding. We spent billions of euro to save people's lives by putting and infrastructures for testing, tracing and vaccine roll-out and so on. Yet, for whatever reason, we do not seem to want to fund orphan drugs to the same extent, or even at a fraction of it. In budget 2020 - and I welcomed this - the Minister made €50 million available for new drugs. In our alternative budget 2021, we provided for an additional €50 million, but truth be known this still is not enough. I agree that you cannot throw endless amounts of money at it and that there cannot a blank cheque for any area of healthcare. Demand will always outstrip supply in healthcare because of the decades of neglect we have seen in investment into our public service.

However, we need to put a much stronger emphasis on the areas of drug pricing and the supply of new drugs. I have made my points about funding, about the need for the State to do more and my rationale as to why the State can be the only driver for this. My understanding is some of these drugs cost hundreds of thousands of euro to administer, in some cases maybe more than a million euro. Very few citizens who could afford that, and certainly not many I know. If we want patients and people to avail of the groundbreaking drugs that can keep people alive, then the State simply has to do more. That is on the funding side of it.

There are also problems, to which the mover of the motion has already referred, about the reimbursement process. Again, I have spoken to industry. I accept fully, and I put my cards on the table here, that we have to strike a balance between what the pharmaceutical sector wants and protecting the taxpayer. Nobody is saying and I am not saying there should be a blank cheque. There has to be a process. I assume the proposer of the motion supports this. Of course we have to have a process. However, the process is too convoluted and it takes too long. As an Teachta Pádraig O’Sullivan said, it can take years for many of these drugs to find themselves on the reimbursement list. I looked at the layers and layers involved in the process and all of the hoops that have to be gone through. When you compare that, as the mover of the motion did, to the situation in other countries, they have much quicker processes, yet they can still find that balance that protects the taxpayer and they have a process that enables transparency and accountability. Although we have that process, other countries can get much quicker decision-making so that drugs can appear on the reimbursement list more quickly.

Those are the two issues here, if we are to cut to the chase. We have to streamline the reimbursement process. We have to make sure the drugs can be approved as quickly as possible and find themselves on the reimbursement list. Critically, we also have to provide the additional funding. This is because we can approve what we like, but if the funding is then not there then the drugs cannot be made available to patients.

I talk to many people in healthcare. On the course of my travels over recent months, I met many people in the area of cancer care, who provide excellent work. It is mind-blowing what these specialists do in our hospitals throughout the State. We sometimes talk about the inefficiency of our healthcare system. However, we cannot overlook the work that is being done by so many people. When we see the precision technology, robotic technology, the advancements in cancer care, the gene therapy treatments, the trials and the research being done in our hospitals with linkages to universities, it is mind-blowing. We also see the treatments that come with that work, and all of these new drugs that are coming on board. People working in healthcare are telling me they can only do so much. They can only do their jobs as consultants. Their job is to save people's lives.

Imagine being a consultant who is treating a very sick patient and knowing that a particular drug can save that person's life, but it is not on the reimbursement list so he or she cannot get it. Imagine knowing, as a consultant, that this drug would make a difference but you are not in a position to prescribe that drug because it has not found itself on the reimbursement list. That is what consultants and specialists are dealing with day-in-day-out. I am sure they understand everything the Minister of State said about finite resources. However, when we talk to them about balance, funding and other capacity issues, they say they have to make decisions every day that save people’s lives. They are not in a position, at times, to be able to administer, provide or prescribe drugs, which they would like to be able to do, because the funding is not available. I have heard one of our former colleagues who was a Member of the Upper House, Professor John Crown, talk about this issue several times. As we know, he is a specialist in this area and has been critical of the slow pace of getting many of these oncology high-end drugs onto the reimbursement process.

I appeal to the Government to do more to listen to the experts and the consultants, to make sure we put patients first and to work with Deputies, such as an Teachta Pádraig O’Sullivan and others, who have taken a keen interest in this issue.

I thank my colleague from Cork North-Central for introducing this Bill, which follows on from the Bill that had been originally published by our former colleague John Brassil. Deputy Pádraig O’Sullivan has outlined the extensive work he has done in this area. It is important we give recognition to that and that we acknowledge the commitment he has made. I also thank the Minister of State for her response.

This whole area has been in the health committee for well over ten years now, since I first served on the committee in 2011. We published a document in 2013, which was on recommendations of the national clinical programme on rare diseases. One of its recommendations was to set up an office to deal with support for parents of children with rare diseases. We must remember there are more than 6,000 rare diseases. I remember coming across one particular rare disease where only 12 children in the entire island of Ireland had it. That is an indicator of how rare some of these diseases are. Therefore, when other countries come to deal with the pharmaceutical companies, they are dealing with far bigger numbers. For instance, Germany starts using the medication immediately after it becomes available and then it negotiates the price of that medication over the following 12 months. Obviously, that is because Germany has larger numbers than Ireland. We might have ten people who have a particular rare disease here in Ireland, whereas Germany might have 1,000. They are, therefore, in a far stronger position to negotiate.

I always wonder about us teaming up with smaller countries to negotiate as a group. Would we have been better-off teaming up with a bigger country in negotiating down the price of drugs? I acknowledge what Deputy Cullinane said about the pharmaceutical companies. A huge amount of research in this area is done in this country, yet when it is completed and the drug is produced, the drug might still not be available to people living in this country four or five years later. We need to look at that. In my own county alone, for instance, we have nine of the ten top pharmaceutical companies in the world. They are based in Cork and are within a 50-mile radius of Cork city. The work and research are being done and the end product is being produced, yet there are parents who have to fight to get access to the drug. We need to look at the timescale.

This even relates to ordinary drugs. I remember being involved in the health committee and dealing with an issue where it took on average 23 months for any drug to be approved by the agency, even though the drug is available at a far earlier stage. Remember that any drug that becomes before the assessment process here in Ireland has already been approved by the European Medicines Agency, EMA. Even though the drug is approved by the European Medicines Agency, we are still taking another two years to come to a decision on whether we will include the drug in the reimbursement system. My colleague has already outlined the situation in Scotland and we need to look other jurisdictions to see how they fast-track the assessment process. We need to engage with them to see if we can replicate what they are doing. This is not about cost in real terms. This is about people’s lives. It is about families whose entire lives are surrounded by taking on the State to try to get access to a drug that they know will be of assistance to a member of their family.

During my short time in the European Parliament, I had to go abroad for medical treatment. One of the things I fought for when I was in the European Parliament was about the right to access medical treatment in another member state if you cannot get it in your own state or if there is undue delay.

We started working on that in 2008. It was eventually signed off on in 2009 and approved by the Commission, the Parliament and the Council in 2011. It was then transposed into Irish law a number of years later. That to which I refer gives people the right to access medical treatment in other member states if they cannot get it in their own state. We should also be making sure there is joined-up thinking across Europe as regards access to medication, especially on orphan drugs. We can come to a far better arrangement from a European point of view by working together with all member states of the European Union on access to a particular drug rather than only working with a number of countries. It is something we need to look at. We have done it with medical treatment so why can we not do it with medication, medical products and orphan drugs?

This Bill is welcome. My former colleague in the House, John Brassil, who was on the health committee, did work on this matter previously. We should not be putting this off when there are families who know there is a drug available and two to three years after coming on the market at European level, they are still not able to get access to it. Families have moved to other countries in order to get access to the drugs they required. We need to look at why it takes, on average, 23 months for drugs to be assessed. That is for all drugs, not just orphan drugs. We need to review this now. We did the review of the clinical programme for rare diseases back in 2013. It is now time to review that clinical programme again. It is nearly ten years on. What part of that programme has been implemented? What needs to be implemented? What needs to be delivered? In fairness to the Department, there are a lot of issues relating to Covid that it has to deal with, and I fully understand that people are under pressure, but we need to look at this as well. We should not say we will come back and look at this in two years. Now is the time to start the review and see what has been implemented, what needs to be implemented and how we can make sure that children or individuals who need a particular treatment can get access to it. I support this Bill and I hope it will go on to the next Stage and that we can progress it. At the same time, we need to bring about improvements in order that people can access the care and treatment they require.

I commend my constituency colleague, Deputy Pádraig O'Sullivan, on bringing forward this Bill. It is vitally important that we make medicines as accessible and affordable as possible. We need to remove barriers to medicine and this will be a positive step in the right direction, albeit not the final step, in achieving this. Given my portfolio, the epidemic of crack cocaine comes to mind when talking about orphan drugs and new medicines. A great deal of work is being done at the minute to find a pharmaceutical treatment for crack cocaine addiction. This is an epidemic that is destroying communities and the lives of people and families. Unlike heroin, there is no pharmaceutical substitute for crack cocaine and this is proving a huge barrier to people accessing treatment. I would hate to think that, if and when we are at a stage where we can provide this vital treatment to those in the throes of addiction, cost will be a barrier to accessing that.

There are serious issues with the process of approving new drugs. There are also many issues around rare diseases and people with rare diseases need to be treated much better. Last year, I was contacted by a lady suffering from very severe morning sickness. She needed a drug called Cariban, which is used to treat severe morning sickness. Her doctor agreed that she needed it but unfortunately, this drug is not available on the medical card and she had to pay for it out of her own pocket until such time as she could no longer afford to do so. Unfortunately, when she had to stop taking Cariban, because she did not have the money, she ended up being hospitalised due to dehydration. This goes to the core of this Bill. It is about affordability and making drugs, and orphan drugs in particular, accessible under the reimbursement scheme. This is only one case but it highlights the impact unaffordable medicines can have on people.

It is disappointing that despite the Joint Committee on Health producing a report on these new medicines in 2018, it has taken a Private Members' Bill to progress this issue. I suppose inaction on health from this Government is not surprising. The fact that health policy is being driven by members of the Government from the backbenches says a lot. The reluctance to properly resource the health service is matched by a reluctance to make healthcare affordable. After months of pleading with the Government to consider antigen tests, we finally got there but instead of making these tests affordable, the Government has decided to leave this vital aspect of our fight against Covid-19 to Lidl. That is where we are now. Instead of properly resourcing healthcare we are relying on the free market to make healthcare affordable. The Minister of State might ask those trapped in the housing crisis how that approach has gone. As Deputy Pádraig O'Sullivan said, it has taken almost four years to get a new, life-changing drug included in the reimbursement scheme. That is a shocking figure and an indictment of our healthcare service. People with rare diseases need to be supported.

The Minister of State made a number of points. She stated that the Minister for Health wants to look forensically at the current system and identify options for reform, and that he wants to increase transparency and communication to make a more responsive system. I respect that the Minister of State is being honest. She is not opposing this Bill today, but the question is whether she, and the Minister, will support it. Will they support this Bill that is trying to make a difference to people with rare diseases? Deputy Colm Burke spoke about the ten-year history of discussions in health about this issue, in 2011 and 2013.

We did take action on rare diseases.

Deputy Pádraig O'Sullivan bringing forward this Bill today shows that action was not delivered. I hope the Minister for Health will finally deliver for people. I know people who have been waiting years to get the drugs they need on the reimbursement scheme. I congratulate my constituency colleague on bringing forward this Bill.

I feel like I have walked into a Cork North-Central public meeting.

Only two of them are bringing forward the Bill. Did the Deputy notice that?

I thank Deputy Pádraig O'Sullivan for bringing forward this Bill and I thank the Minister of State for being here. I also acknowledge that the Minister of State, Deputy Rabbitte, has entered the Chamber.

Today is International Day of Persons with Disabilities. While that is not the subject matter of this Bill, we are talking broadly about people having access to healthcare, whatever that may be, so it is important to recognise this day.

I hope that we will have more focused debates on the matter in the coming weeks.

I thank Deputy Pádraig O'Sullivan for introducing this Bill and for speaking to me about it earlier this week. The area of orphan drugs is not one I am very familiar with, being relatively new to this brief. What is not new to me or any of us as public representatives is individuals coming to our clinics and ringing our offices who have rare diseases but are unable to access medicines that would help them and make their lives somewhat better or could cure them. They are unable to access these medicines because of the medicines' exclusion from the reimbursement scheme.

In researching this matter and being briefed on it, Ireland was described to me as a medications paradox. We are a massive producer of pharmaceutical products but because of the system that is set up, some of these medications cannot get to the people who need them. That must change. Deputy Pádraig O'Sullivan mentioned former Deputy John Brassil and his work on this matter. I understand that Mr. Brassil is a pharmacist by trade, so he is someone with expertise in this area and who would have seen the weaknesses in the system on the front line. That is why I am happy to see this Bill before us. I hope that it makes some progress, given that there are weaknesses in the system. When there are drugs that will help or cure people but that we cannot provide to the 280,000 people in this country living with rare diseases, then there is a weakness in the system. We have to work together. Being present for this debate and its contributions from every side of the House, I can see that everyone wants to get to that point.

I thank Deputy Pádraig O'Sullivan for introducing this Bill. There is much more work to do. I certainly have to do much more work in terms of my understanding of this area, but from the brief research I have done in the past couple of days, I can see that this Bill is needed. I hope to see it progress.

I thank the Ceann Comhairle for affording me the time to speak.

It is great to have two Ministers of State present. Since we have some time left, I will first call the Minister of State, Deputy Rabbitte, and then the Minister of State, Deputy Butler.

I thank the Minister of State for giving me a little of her time. I also thank the Ceann Comhairle for giving his time yesterday evening to mark International Day of Persons with Disabilities, and I thank the House for turning Leinster House purple.

I thank Deputy Pádraig O'Sullivan for introducing this Bill. This matter is something about which I am passionate. On the Opposition benches, I saw the great work we did with Orkambi and Kalydeco in respect of cystic fibrosis and the difference that work made to people's lives. It was a battle no different than the one Deputy Colm Burke mentioned, given the length of time it took us and all the hoops we had to jump through. All the while, we could see people's lives disintegrating because they did not have access to those drugs.

It is important that the Minister, Deputy Stephen Donnelly, has given his support to the Bill and to us understanding the process and why it takes so long to turn drugs around when we know that we have fantastic drugs in the system. I am here to lend my support and I see the value this Bill can have. The House should support it collectively.

I thank the Minister of State for her comments and for acknowledging that today is International Day of Persons with Disabilities. I thank the Deputies who contributed on this discussion, in particular the Bill's sponsor, Deputy Pádraig O'Sullivan. I commend the goal of the Bill, which seeks to provide faster access for people to new medicines for the treatment of rare diseases.

I was particularly struck by Deputy Gould's comments. He was amazed that a backbencher could introduce a health Bill. Does that mean that Deputy Gould is not allowed to introduce health Bills? I compliment the two of his colleagues who are present - I am working with Deputy Colm Burke on a Bill on standards for home care and I am happy to work with Deputy Pádraig O'Sullivan on this Bill on orphan drugs. In government, we welcome all opinions. We all have individual minds, thoughts, strengths and ideas. I encourage anyone who wants to introduce a Bill. Members should not feel like they cannot. It is important that we hear from everyone across the House. That goes for all colleagues.

I have outlined some reforms of the existing assessment process carried out to date by the HSE and the National Centre for Pharmacoeconomics. I wish to take the opportunity to acknowledge their work in that regard. These reforms are ensuring that many expensive orphan drugs are getting reimbursed. I also wish to recognise the contribution made in recent years by the Joint Committee on Health. As Deputy Colm Burke mentioned, the committee has been considering this issue for the ten years he has been a member of it. It has held a number of productive discussions on the subject of rare diseases. Since the committee published its report in 2018, there has been substantial engagement between the Department of Health, the HSE and the National Centre for Pharmacoeconomics on each of the recommendations. In the previous Dáil, we all campaigned for the inclusion of Spinraza in the scheme on behalf of many kids who were moving into adulthood. We are all aware of how that drug has made such a difference to those families. It was an emotive issue at the time. There was only a small number of children involved, but it was great to see provision being made for the drug and those children being able to get on with their lives.

The substantial engagement I mentioned has resulted in a number of positive changes in the assessment process for orphan medicines. This includes the setting up of the rare diseases technology review committee, which facilitates patient and clinician input and gives these expensive medicines a fighting chance of getting through the system and becoming available to patients.

Irrespective of the costs involved, we must not forget the human element of this debate. We saw that with Spinraza and the effect it had on the children and their families. People with rare diseases are among our most vulnerable and they are often faced with a large level of unmet clinical need. Due to small patient populations, the development of new medicines for the treatment of rare diseases has historically been hampered by reduced commercial incentives. However, the pipeline of new medicines for rare diseases has become stronger in recent years thanks in part to the European Parliament's adoption of measures to encourage innovation in this area.

Today's debate offers an opportunity to progress our conversation around rare diseases. We must continue to question and improve our systems with a view to providing faster access to new innovations. Recent investment in research of treatments in this area is encouraging. However, more could be done to support access to treatments for rare diseases. The tabling of this Bill represents an opportunity to look comprehensively at the current system and consider how issues such as transparency and communication can be improved. I understand that the Minister has asked his officials to do that. In the circumstances, he does not propose to oppose the Bill.

I thank everyone who has contributed on this debate. It is great to see that the Bill has universal support and I look forward to it going to Committee Stage. Deputy Colm Burke and our own Deputy Lahart are on the Committee on Health. They are passionate about this topic.

I will make a couple of comments in summary. There are a few inescapable truths. One is that these drugs are expensive. Of that, there is no doubt. The second inescapable truth is that we as a country make people wait much longer than they probably should, certainly compared to our European neighbours.

The Minister of State, Deputy Butler, has taken this debate on behalf of the Minister. In my opening speech, I referred to the fallacy of budgetary constraints, the concept that these drugs would be costly and that we would be opening a can of worms. That is precisely where I want this debate to move away from, as it is a fallacy. There is no truth to it and it is a myth. The Minister of State said: "The advice provided is that abandoning the methodology would potentially have added an additional €250 million cost over the next five years". I would love to see how that figured was arrived at. I imagine it assumes that all orphan drugs that went for reimbursement got approval. I am not asking for that, though. I know the process inside out at this stage. I know that not all orphan drugs get approval. That €250 million figure is not a true reflection and does not stand up to any scrutiny.

I agree with Deputy Cullinane on one point he made, in that this sector is moving at a fast pace.

My honest view is that this Bill is probably outdated already, insofar as it was initially put forward in 2018. I essentially replicated it, but in the meantime we have made significant progress in the area of genetics and gene therapy, for example. The Bill does not have those types of therapies included, and that is probably something that might emerge as a further amendment in the health committee. That is something I would welcome. Deputy Cullinane is correct in saying that this sector is moving fast. I hope the Bill will become legislation in a few years. There is no doubt that by the end of the debate, further provisions will be required.

I wish to put to bed the myth that if we abandon the €45,000 QALY threshold, costs are going to spiral out of control. We can look at a dozen countries across the EU that do not have that QALY threshold. I mentioned Sweden and Scotland, and Denmark is very similar to us in terms of population and size, but none of them operates a specific QALY threshold, be it for €45,000 or €100,000. They assess a drug on its efficacy and then they do a cost-benefit analysis in the process. All drugs get a fair chance. The fundamental issue here is that orphan drugs are not assessed as orphan drugs, which means if 1 million patients in a country need a new drug, there is a specific pathway for them, but as Deputy Colm Burke alluded to earlier, if there are ten or 20 patients needing a drug, obviously the cost will go up and there is no specific pathway or process for the drug to be analysed. It is analysed in the same way as a drug that is destined for 1 million patients, which is complete madness.

There should be a specific pathway, criteria and timelines because the reality, as per Deputy Michael Moynihan's parliamentary question, is that it takes 1,361 days on average for a drug to come into someone's possession. A total of 426 days are required for the initial application. When we drill down into the 426 days, any of the companies that are applying for reimbursement for orphan drugs know the answer to the application process already. They are going to be over the €45,000 threshold. It makes no sense for them to be assessed in the same way as a drug destined for 1 million patients. It is completely bonkers. In that respect, what we are trying to do here is to provide a statutory footing for orphan drugs. They do not have that at the moment. The intention is to provide hope for patients in the future that the drugs they require will be assessed on their individual merit and not with mainstream drugs or illnesses. That is the crux of the matter.

Other speakers have referred to the importance of pharmaceutical companies to us as a country. Deputy Colm Burke mentioned that nine of the ten largest pharmaceutical companies in the world are based here. It is unfortunate that drugs being made in Ringaskiddy, Little Island or wherever it might be are potentially unavailable here, but we are exporting them all over the Continent.

The Bill gives people a definite pathway and it also gives them hope and opportunity. I had only been a Deputy for two months when I had the hardest conversation I ever had around a coffee table outside Cork city. I was told that a person was one of a couple of dozen people in the country that suffers from amyloidosis. The Minister of State, Deputy Butler, mentioned it earlier. This person had already had a relative die of that disease. In the meantime, the person knew that for every year they did not have the drug, it was taking years off their life. Thankfully, after four years the system approved the drug, patisiran, to specifically treat amyloidosis. I befriended a number of people who suffer from the condition, which is especially prevalent in Donegal of all places. It is connected with a specific gene and a number of related families suffer from this very specific illness. There is relief for them now that they have a life and access to a drug.

As Deputy Gould mentioned, many people cannot afford these drugs. That is the simple reality. I know the detractors of the Bill will say that it will open a can of worms, as has been said previously, and that a bottomless pit of money will be required that we cannot provide. There is a specific provision in the Bill stating that it must operate within budgetary constraints. Nobody is suggesting for a moment that it would be otherwise. If a drug is effective and, following intense negotiations with a company, the State can get it at a reasonable price, it should be made available. That is what this Bill provides for.

Ireland is probably three to five years behind most of its leading counterparts in this regard. We are playing catch-up. Realistically, all the quantitative and qualitative data are already done in European countries. Those pharmaceutical companies will use the same data when they are selling these products to us. The reality is that because we are so far behind, we know the drugs work. If we were ahead of the game, we would be taking a greater risk, but we are not. We are years behind. In that respect, we are playing catch-up. We know that many of the drugs that will be sought for reimbursement work because they are being used across the Continent. The Bill enshrines in legislation that orphan drugs will have a specific pathway. That is all we can give to people. We cannot expect the Department to give drugs if we are dubious about their efficacy, but because we are so far behind, we know the drugs work elsewhere and are enhancing and improving people's lives all across the Continent.

I thank everybody for their support. I thank the Ministers of State, Deputies Butler and Rabbitte, as well. I met the Minister, Deputy Stephen Donnelly, last week on a Zoom call and I am grateful for his support of the Bill. I look forward to the Committee Stage debate on the Bill.

Question put and agreed to.
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