Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2018: Second Stage [Private Members]

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

(Interruptions).

Ministers, some of us have to stay here until the end of business.

Let everybody have their private meetings. I am in no rush.

I can appreciate that.

Under the current Act, there is no differentiation between orphan medicinal products and other products. In recent years, it has become clear that there is an issue in Ireland whereby orphan drugs continually struggle to secure reimbursement here, often despite their wide availability elsewhere in Europe. This reimbursement process is protracted, unnecessarily public and often antagonistic, with patients continually forced to protest publicly or engage with politicians to seek fairness and equity over the course of a two-year campaign from initial application in Ireland to its final decision.

The issue of low availability of orphan drugs or treatment specifically designated for rare diseases is a complex one, with several factors contributing to the problem ranging from the appropriateness of the assessment mechanism, the current framework agreement between the State and the pharmaceutical industry, industry pricing and prioritisation of Ireland.

This Bill is not a panacea to this complex issue but looks to address one of the clear barriers to access in Ireland in how we apply a standardised assessment mechanism to medicines that are far from standard. Currently, the same health technology assessment is carried out by the National Centre for Pharmacoeconomics, NCPE, for new medicines, irrespective of their medicine type. This is a quality-adjusted life year, QALY, based health technology assessment whereby quantitative data on the medicine and the disease it treats are used to assess its cost effectiveness against an incremental cost effectiveness ratio, ICER. In Ireland's case, this threshold is set at €45,000 per quality-adjusted life year.

This mechanism presents several issues for orphan drugs, which often possess limited quantitative data due to low clinical data or the low epidemiology around a rare disease, given such a small patient population. As such, a QALY-based health technology assessment will result in great uncertainty around orphan drugs. Last month before the Joint Committee on Health, Professor Michael Barry, head of the NCPE, stated that none of the drugs for rare diseases will reach the €45,000 per quality-adjusted life year.

The consequence of using this form of health technology assessment is that commercial negotiations between the State and industry commence in a poor position and the information available to the HSE is of limited value. This is directly contributing to the two-year process of protracted negotiations as both parties seek to reach a fair and accurate agreement of the true value of the medicine. From a broader perspective, this two-year delay does not serve patients who need orphan drugs because, as per the European Medicines Agency licensing requirement, they have life-threatening or chronically debilitating diseases. The delay can swiftly exceed two years as companies deprioritise the Irish market in favour of European access. This is in light of Ireland's growing reputation for having a slow and challenging access environment in respect of drugs used to treat rare diseases.

I will now comment on the Bill. Responsibility for addressing this matter has fallen between two stools. The Minister for Health has stated at every opportunity that statutory responsibility for medicine pricing and reimbursement decisions lies with the HSE in accordance with the Health (Pricing and Supply of Medical Goods) Act 2013. For its part, the HSE informed the Joint Committee on Health that the 2013 Act does not make separate provision for orphan drugs. As a result, the processes and procedures do not make separate provision for distinct criteria for the assessment of orphan drugs. When challenged on whether the HSE had any flexibility under the Act to allow for a tailored approach to assessing orphan drugs, the executive's representatives informed the joint committee that it has pushed the legislation as far as it can. The Bill seeks to resolve this issue by clarifying for the HSE the application of the health technology assessment for evaluating orphan drugs, as well as establishing set criteria to be considered when making a relevant decision on reimbursement in respect of an orphan drug. It clarifies that the HSE should carry out health technology assessments in a manner that does not rely on an incremental cost-effective ratio threshold. Instead, it should conduct assessments that will provide meaningful and credible evaluations of orphan drugs.

This is not to suggest that we should not carefully consider orphan drugs. Rather, we should look to EU member states such as Sweden, which has adapted its health technology assessment process in such a manner. In addition, the Bill places on a statutory footing some important considerations for the HSE to assess before making a final decision on an orphan drug. These include the availability of the drug elsewhere in Europe, guaranteed input of patients and the level of certainty that can be provided by industry through risk-sharing agreements. The Bill also acknowledges the limitations of traditional quantitative data. Importantly, the Bill does not seek to undermine the ability of the HSE to seek an affordable price from industry. Important criteria, such as the potential budget impact of the drug and the resources available to the HSE, are maintained as statutory considerations. As such, I see no specific budgetary implications. This Bill simply aims to speed up the process and ensure that it starts on the right footing.

It should be acknowledged that there are ongoing reforms in the HSE with regard to orphan drugs, driven in part by the work carried out by the members of the Joint Committee on Health during the past two years. Reforms include the creation of a rare disease technology review committee. This is welcome, as is the Minister's engagement with other EU member states via the BeNeLuxA and Valetta initiatives. It has been acknowledged by several Members that European co-operation is a logical but long-term solution that is unlikely to materially resolve the orphan drug issue, which is immediate and urgent. The technology review committee will ultimately consider a flawed health technology assessment and, as such, this Bill remains a necessary reform that must proceed.

A collaborative approach is required with the input of the Minister for Health and his officials in conjunction with the Joint Committee on Health. On 27 September last year in a debate on the availability of the drug Ocrevus, the Minister stated:

If people feel that the current system is broken, I would like to hear their views on what should replace it. I think it is broken too. Coming up with a better model is the conundrum.

I am providing the Minister with an answer to that conundrum. I am asking the Minister to work with me and to support the Bill to stop the endless marching and protesting that seems to be required for every drug that comes before us. I urge the Minister to support this measure and I urge every Member likewise.

I welcome the opportunity to contribute to this debate. I commend my colleague, Deputy Brassil, who has done considerable work on the basis of professional experience and qualification in this area. He has sought to find an alternative solution to the situation in which we find ourselves.

We know the current system is not working. In recent years, drug after drug has been debated at length in the House. We have had Orkambi and Respreeza and now we have Spinraza. More and more orphan drugs for rare diseases will continue to surface. Every month and every year they will come down the tracks. We need to find a better way of dealing with these drugs when they come online. We need to find a better way of assessing these drugs in a fair and transparent manner, one that at least gives some opportunity for drugs that work to make it through the system.

As it stands, the process will never allow a drug such as Spinraza to get through. Spinraza will never meet the cost ratio analysis because it is quite expensive and a very small number of children and adults are affected by the disease that it seeks to treat. That should not preclude a drug that we know to work and that save lives. It considerably increases the quality of life for children in this country. We should give such drugs a fair chance of being approved for funding for those families and children who need it.

In the context of the Bill, we are not suggesting that every drug for rare diseases will make it through. That is not sustainable or affordable. Of course we have a finite budget in the health service. We need to balance the budget and try to afford all of these things. As matters stands, these drugs will never get through. What we are saying is that some drugs should have at least some chance of making it through the system, especially in circumstances where we know they work to save lives and improve the lives of children and adults.

I am keen to focus on Spinraza because I have been working on this issue in recent months. Parents and children are forced to go through a terrible journey. Parents have contacted local Deputies throughout the country. We have organised a march outside Leinster House. There have been several questions posed to the Minister, the Taoiseach and the Tánaiste in the Chamber. There have been parliamentary questions. The list is endless. These are the avenues taken by parents who are desperate to get these drugs for their children. No family should be placed in this type of situation whereby the only avenue open to them is intense lobbying to create intense political pressure to try to get a decision that is politically made because approval does not work inside the system. We know this and we do not want to subject families and children in future to march outside the gates of Leinster House.

We heard from a young girl, Grace, who is not even ten years old. She presented to an audience of adults in the audiovisual room. She told us why she needs this drug to live. We should not have that situation. There needs to be a fair process whereby these drugs are properly assessed. If the system is set up so that these drugs automatically fail, then it is not fair.

The Bill would go some way towards redressing the balance. It would at least give a chance to drugs that we know to be efficient and work. Families and children are openly saying that all they want is a chance. If a given drug does not work for them, then they do not expect the State to pay for it in the long term. However, they want the opportunity to get a drug that could make a major difference to their lives in terms of surviving and living longer. In such instances, I hope to see Government and Opposition support for this Bill.

I commend Deputy Brassil on bringing forward this important Bill on behalf of Fianna Fáil. The Bill will help to reform the current HSE reimbursement process and provide a better situation for assessing orphan drugs.

Approximately one person in every 12 will develop a rare disease.

Orphan drugs are currently assessed by the same mechanism as all pharmaceutical products where the odds are completely stacked against them because the numbers affected are so small, the medicines are usually very costly and the prices often exceeds the criteria set out by the National Centre for Pharmacoeconomics, NCPE, and the HSE. While these drugs are very costly to develop and the low patient population and low success rate in bringing the drugs to market can make it very difficult to assess based on clinical data, effectively these drugs are often set up to fail under the current system. These include Kuvan, Translarna, Respreeza, Orkambi, Spinraza and an ever-growing list of drugs. These families and their children want hope and a fair chance.

I will focus on Translarna for Duchenne muscular dystrophy which affects approximately 12 people in the country. The drug available for its treatment, Translarna, can only be used for children of a certain age and only five children fall into that category. Those families have been outside the gates of Leinster House and have presented in the audiovisual room several times. Muscular dystrophy is a debilitating genetic disease that will unfortunately lead to these children dying. Their muscles eventually waste away and they eventually lose the capacity to breathe and ultimately their heart gives out. Translarna is the first licensed therapy to address the underlying causes of this disease. It is available in 22 countries across Europe but not here. It works, it saves lives and there are no alternatives. Those distraught families and their sons and daughters are up against the clock. They know that if they do no use this drug by a particular age it is too late.

I know that many of the pharmaceutical companies effectively operate a system of blackmail, and pit countries against one another. Nevertheless, we can put better processes in place in this country to effectively provide a proper system so that these orphan drugs can be fairly assessed. That is what these families find most distressing. They see a very opaque system which has no transparency, there are no dates for meetings and there is no information about what is happening and how some drugs are, or are not, being approved. They need hope and they desperately need these drugs.

We do not have time to more than touch on the waiting list for genetics services to assess children and have them diagnosed. We are talking about genetic diseases, there is nothing that the children can do about it.

Even if we look at cancer drugs, some 20% of new cancer drugs launched internationally in 2015 and 2016 were made available in Ireland, compared with 76% in Germany, 71% in Austria and 67% in Denmark. Patients' groups are saying that Ireland is an outlier. I ask the Minister to support the Bill tabled by Deputy Brassil and Fianna Fáil. It is a very good Bill which sets up a way for a proper and fair reimbursement process to help give these families hope for their children.

This Bill is a response to the medicines crisis faced by Irish patients suffering with rare diseases and I compliment Deputy Brassil on his work on it. The Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2018 seeks to reform the HSE's current reimbursement process by establishing for the first time a unique process for assessing an orphan drug for reimbursement in Ireland. The Bill brings a long overdue and credible solution to the ongoing crisis surrounding access to medicine for patients with rare diseases in Ireland. Orphan drugs treat, diagnose or prevent life-threatening or chronically-debilitating diseases which affect fewer than five people in every 10,000. They are currently assessed under the same guidelines and procedures as standard drugs despite the clear differences between the two. Rare, high-tech drugs will naturally treat an extremely low patient population. To measure their effectiveness using the same criteria as common conditions will naturally cause difficulty. Other EU countries have adapted the process for this reason and we believe it is time we did likewise.

In recent years, I have become aware of two orphan drugs. The first such drug is Respreeza which I first learned of at a presentation in the audiovisual room where I met a gentleman, Johnny Hannon, who was on a trial through a compassionate access programme. Respreeza is used for maintenance treatment to slow the progress of emphysema in adults. The drug has had a game changing effect on his life. However, that day I met another lady in the room who did not have access to the drug on the compassionate access programme. Every breath she took was a struggle. She told me how basic things such as getting up, washing and going into town to do some shopping was a struggle. It is wrong that people have to take to the streets to fight for access to a drug that can change their life. It is not acceptable that citizens suffering from severe illnesses should be forced to do that or to go public in the media to make their voices heard.

Orkambi is another drug that people are familiar with. Jillian McNulty who had benefitted from the drug which, as she said, transformed her life ran a high profile campaign. An estimated 60% of the cystic fibrosis population in Ireland have begun Orkambi treatment, which is very welcome. Gerry McElvaney, professor of medicine at the RCSI said, "In general the results from Orkambi were better than I expected and I think it has given significant relief to a significant number of patients". He has also said that we have a duty and obligation to ensure that patients with rare diseases receive the same treatment and care as any other patient in Ireland.

Access to drugs such as these can result in less time spent in hospital, fewer acute beds being used and can allow patients to spend more quality time at home which means cost savings to the State. This Bill is a timely and logical step and I hope the Minister will support it and work with Deputy Brassil for it to be enacted as soon as possible. I cannot imagine what it is like for a parent of a child with a rare disease. All one would want to do would be to give the child every chance at life. If there is a drug that would give children such as these a fighting chance then the onus is on us as legislators to ensure those children are given every chance.

I also thank Deputy Brassil for tabling this Bill. I recognise his particular interest in this, his commitment to the issue, his professional background and for not giving out about the problem but bringing forward ideas and proposals. I appreciate the opportunity for discussion during Private Members' time of what is a very important issue for us all.

I certainly do not argue with the spirit of the Bill. It is one which has been tabled with the intention of ensuring faster access for patients to orphan medicines which, I assure the House, is also a goal of mine. No one could help but be moved by the tragic and difficult cases that we all confront, particularly children with rare diseases. Many of the names which have been raised by Members opposite are also known to me, as we come across people as we do our work. I also want patients to have timely access to new orphan drugs and I will work with patients, industry, the HSE and other agencies to make that happen. I will never say that our system is perfect because it certainly is not. That is why we are constantly looking at ways in which we can do things better.

I will start by describing the system we have, what we have done and are doing to improve it. I acknowledge that Deputy Brassil was kind enough to acknowledge some of this work. In 2013 the Oireachtas put in place a robust legal framework in the Health (Pricing and Supply of Medical Goods) Act 2013. This gives full statutory powers to the HSE to assess and make decisions on the reimbursement of medicines, taking account of a range of objective factors and expert opinion, as appropriate. There is no doubt but that this legislation made our medicine reimbursement process fairer. It has provided access to a significant number of new treatments since its introduction. The Act specifies the criteria to be applied in making reimbursement decisions. These include the clinical and the cost effectiveness of the product, the opportunity costs and the impact on resources available. These are very difficult decisions and ones which we would prefer not to have to make, but we must all operate within certain resources to spend on all our services. We, as legislators, battle every day with having to make choices and ensuring that resources are used in the most effective way possible.

Increasingly new drugs target very rare conditions and they often come with very high list prices. The HSE must, under the current Act, have regard to funding these drugs and the budget impact of the drug in question. It also draws on the expertise of the National Centre for Pharmacoeconomics before making its decisions.

Reference has been made to managed access programmes. We have done that with Ramicin and Respreeza. I want to acknowledge the family of the late Marion Kelly and the work they did in her memory, as well as Mr. Johnny Hannan and others who Deputy Butler referenced. I would like to see this happen with other drugs like Spinraza, from which a small number of affected people could see a significant benefit. We will do it for to pembrolizumab for women with cervical cancer. There is now a second application before the HSE concerning Translarna, which Deputy Browne referenced. Deputy Butler referenced Orkambi. I am very proud that we are providing Orkambi in this country. It is still not provided north of the Border. We are managing to get drugs over the line, but we must always look at new and better ways of improving our process. The challenge of accessing innovative medicines at an affordable price is one shared by most if not all developed countries.

About 45 new molecules are due to receive market authorisation in Europe in each of the next five years. These are really good things. They represent more new and innovative drugs. The challenge is in making sure that we can access them. It is in this high-tech space, which includes orphan drugs, that the greatest challenges will arise in the years ahead. Expenditure on the high-tech arrangement through which most new high-cost medicines, including orphan medicines, are funded has increased from about €400 million in 2012 to almost €700 million this year. That level of growth is a major challenge for the sustainability of our health service. That is why I have been not only advocating but working for international collaboration and pursuing an active international agenda to build consensus on this issue at EU level. A significant development happened just last June when the BeNeLuxA initiative accepted my application for Ireland to join. This is a new initiative on pharmaceutical policy, involving working together with other EU nations to look at jointly procuring drugs and sharing information and ways of working together. This collaboration will support the Government's objective of co-operating with other European countries to identify workable solutions in an increasingly challenging environment and to secure timely access for patients to new medicines, including orphan medicines, in an affordable and sustainable way.

Our active participation in international fora has not distracted us from domestic policy, however. The 2016 framework agreement with industry is a cornerstone in our national pharmaceutical policy. It is central to the State's medicines policy and I am pleased to report that it is working. The agreement helps to support sustainable access to medicines for patients. The savings provided are essential in creating headroom for access to new medicines, including orphan drugs. We must remain vigilant about continuing to identify efficiencies in the existing system. A priority for 2019 is for the HSE to actively engage in a strategic programme of creating efficiencies through the national drugs management scheme, the medicines management programme and other initiatives.

The Joint Committee on Health has done a great deal of work on this issue, producing its report on the evaluation of orphan drugs in February 2018. The incremental cost-effective ratio was one matter raised by the committee, and it has been raised again in Deputy Brassil's Bill. The quality-adjusted life year, QALY, is the economic evaluation used to set a threshold of cost-effectiveness when assessing medicines in Ireland. It measures the state of health of a person or group by adjusting the benefits in terms of length of life to reflect the quality of life. One QALY is equal to one year of life in perfect health. QALYs are calculated by estimating the years of life remaining for a patient following a particular treatment or intervention and weighting each year with a quality-of-life score. It is often measured in terms of a person's ability to carry out daily activities and his or her freedom from pain and disturbance. The QALY is currently the best metric we have in health economics to express the value of health outcomes. Its greatest value is that it allows medicines to be assessed systematically on a scientific basis. Removing the QALY would have serious financial implications and currently there is no comprehensive, reliable or validated methodology with which to replace it. The National Centre for Pharmacoeconomics, NCPE, is adamant in its advice to me that until a comprehensive, reliable and scientifically validated alternative to the QALY can be found we should continue to use it to express health outcome benefits from the medicines we assess.

The aim of Deputy's Brassil's Bill is honourable and I will continue to work with him on these issues. However, the proposed changes to section 19 of the 2013 Act would fundamentally change the statutory reimbursement criteria for orphan medicines. The Bill seeks to maintain the concept of cost-effectiveness but perhaps unintentionally rules out any structured mathematical system to assess it. The criteria proposed for insertion into Schedule 3 would introduce entirely new reimbursement criteria for orphan drugs. The clause linking reimbursement decisions in Ireland to availability in other European countries is particularly challenging. It would be entirely inappropriate to compare vastly different reimbursement processes. Perhaps that is one of the challenges on which we need greater international collaboration. Ultimately a lighter-touch regulatory approach will not solve the challenges we face with access to orphan drugs. The main challenge with almost all new medicines is their price and affordability in a budget-limited health service.

Deputy Brassil has tabled a Bill which seeks to amend the current Act to establish specific criteria to be used by the HSE in reaching decisions on the reimbursement of orphan medicines. The premise of this Bill is that the statutory process is not fit for purpose where orphan drugs are concerned. The proposed amendments to the Act would establish a different rule set for assessing orphan drugs. I acknowledge that we need to do more on this, and in fairness Deputy Brassil acknowledged the work that is being done around rare diseases. Paradoxically, however, this Bill would retain the concept of cost-effectiveness but as I have said it would rule out a structured, methodical and mathematical system for assessing drugs. This Bill essentially proposes a less rigorous rule set for orphan drugs and would do away with accepted clinical and economic evaluation systems for assessing medicines.

The 2013 Act has put in place a clear and robust statutory framework, but we must continue to look for ways to do better within that context. I believe there is an awful lot more we can do. While the 2013 Act does not include a specific rule set for orphan drugs, the HSE seeks as far as possible to take into account issues such as small patient numbers and the nature of the condition to be treated when evaluating these medicines. We can point to examples where I am making progress in this regard. The evaluation criteria allow sufficient scope to take on board the specific circumstances of orphan drugs.

I am very confident that the current Act is serving us well, but there is much more we need to do to make sure that the system is fit for purpose within the context of the Act. That is why we have established the technology review committee for rare diseases, which the Deputy acknowledged. That committee provides a mechanism for issues such as unmet clinical need, clinical effectiveness and alternative therapies to be considered in the assessment process. That committee will give a platform for patients and their families, in addition to clinicians, to give an input to HSE leadership on reimbursement decisions. In addition, the HSE drugs group is being expanded to include two representatives from the national patient forum and more clinical expertise on rare diseases, something I know everyone in this House will welcome. Both measures are intended to bring greater balance and transparency to the assessment process.

We have a lot more to do. We will continue to do more in the managed access space, where we can consider cases where a small number of patients would significantly benefit from a drug. That needs to be looked at and can be looked at in the context of the existing legislation. For these reasons, I am not in a position to accept this Bill but I look forward to continuing to work with Deputy Brassil, who I acknowledge has done huge work in this area. I thank him not just for bringing forward a Bill listing all of the challenges, but for genuinely trying to bring forward proposals. I hope I have outlined to the House the reasons I am not in a position to accept it.

Deputy Louise O'Reilly has 15 minutes.

I will not need all that time. I thank Deputy Brassil for bringing this Bill forward. Deputy Brassil and I are members of the Joint Committee on Health. We have had occasion to attend many presentations and hearings on the issue of access to orphan medicines. We have also been approached, as every person in this House probably has, by patients, their families and clinicians on this very issue. In recent years it has become increasingly apparent that when it comes to approving drugs for rare diseases, the system is simply not fit for purpose. While this Bill is not a panacea for every issue surrounding access to orphan drugs and the approval process, it does go some way towards addressing a fundamental problem. It allows for the establishment of specific criteria applicable to orphan medicinal products for the purposes of the Health Service Executive's decision on adding an item to the reimbursement list.

The HSE estimates that 300,000 people in the State will develop a rare disease at some stage in their lives. There are between 6,000 and 8,000 known rare diseases. Only 5% of these have a licensed treatment option and there is a high level of unmet need. It is that unmet need that we meet at the gates of this institution when we are going in and out. It is that unmet need that causes people to get out of their sickbed, or people to come here on behalf of their children, to protest outside the Dáil. That unmet need that must be addressed. It puts sometimes unfair pressure on members of the Government, but people feel that they have no option. The pressure is put on all of us. Deputies encounter people waving pieces of paper asking what price is a life. That is no way to do business. That is no way for sick people to have to behave, but they are not doing it because it is their first option. They are doing that because they feel they have been left out of the process. Which of us would not do that? If it was our children, our loved ones, someone in our family or our neighbour, which of us would not do it? Of course we would if we thought it might work. Every time we see a protest outside here, it is evidence that the system for assessment is not fit for purpose.

The metrics used by the Health Service Executive to evaluate the effectiveness of and approve orphan drugs are unfit for purpose. They do not take into account the distinct differences between drugs for rare diseases and drugs for non-rare diseases. Therefore, we see it as imperative that the HSE changes the way it evaluates medicines to treat these very rare conditions. We believe that when it comes to the approval of orphan drugs, the process needs to be more transparent as well as giving patient groups and clinicians a stronger voice in the National Centre for Pharmacoeconomics.

We are also aware that the pricing strategy adopted by some pharmaceutical companies in recent years presents significant challenges and this is leading to serious affordability problems for the health service in Ireland and internationally. It is exactly that to which I was referring. It is that pricing strategy adopted by some of the pharmaceutical companies that puts pressure on the campaign groups, which, in turn, put pressure on politicians who, in turn, find themselves asking the Minister what is the price of a life. That is no way for this to be run. None of us wants to be in that position. That is why I welcome the Bill. I would urge the Minister and his colleagues to reconsider support for this. I am sure Deputy Brassil would be open to working with them, if the belief is that amendments are needed. We all have acknowledged that a change is needed and that is what we should be working for.

The national rare disease plan, the generic policy framework for rare diseases compiled by the Department of Health, recommended the bringing forward of appropriate decision criteria for the reimbursement of orphan rare medicines and technologies. However, to date, no dedicated decision-making criteria have been forthcoming. Instead orphan drugs have to be assessed in this narrow confine where matters of cost almost immediately rule out their approval for any patient.

The HSE has traditionally been an early adopter of new medicines where clear evidence of clinical benefits to patients can be demonstrated and value for money assured. This remains the case when it comes to non-orphan drugs. However, when it comes to approval and adoption of new orphan medicines for rare diseases, it is failing. It is important that the Bill seeks to establish a new set of criteria for consideration of orphan medicinal products mitigating against low qualitative data and guaranteeing that the HSE will consider qualitative data presenting on an orphan medicinal product for patients, clinicians and others.

We are all aware that a fundamental problem with the current approval process is that there are no dedicated criteria for the approval of orphan drugs. Unfortunately, unlike many other health systems across Europe, there are no specific protocols and processes to evaluate orphan medicines, meaning that orphan medicines are assessed under the same methodology intended to be applicable to all other medicines. Essentially, a drug that treats a patient with muscular dystrophy is appraised using the same cost-effectiveness process that is used to assess and approve an aspirin to treat headaches. We agree with experts, including the Irish Pharmaceutical Healthcare Association, that the quality system and the threshold are fair in assessing the value for money of innovative non-rare disease medicines brought forward by companies, but not for rare disease medicines.

Furthermore, the current decisions regarding the cost-effectiveness of a new drug for a rare disease do not take into account the wide-ranging savings that could be made from a drugs approval. For instance, it does not factor in savings from a patient not needing to return to hospital or require a more expensive treatment down the line, or, indeed, time taken off work or time committed by a family carer.

We need to change the way we evaluate medicines to treat very rare conditions. As well as the passing of this Bill, we also need to see a new assessment system and approval process which guarantees input from patients and clinicians and gives them a stronger voice in the HSE decision-making. Such a system works extremely well in Scotland where they have a patient and clinician engagement assessment process. If we had such a process here, it would give patient groups and clinicians a stronger voice in the HSE decision-making and the assessment process. It would also allow the HSE to gather detailed information which would allow a discussion on the benefits of a medicine, including how it can impact on the quality of a patient's life. This information is not currently captured within the present assessment process and is why patient groups tell us that they feel left out of the decision-making process. As I stated, that manifests itself at the gates. That is where that ends up. People do not feel that they have been part of the process. They feel that they have been shut down, that the answer is "No" before anyone ever looks at the assessment and, more importantly, that nobody is taking their child, their loved one, the person they represent, into account. I am not suggesting that is how it is. I am telling the Minister that is how they feel. That is how they end up outside. They feel that they are locked out of the process or that the process is not fit for purpose and cannot adapt to assess properly what the needs are for their loved one.

The drug approval process I have referred to works incredibly well in Scotland. It ensures that all those who are affected by decisions, such as approval for reimbursement, feel that their opinion and evidence, most especially the patient's, have been heard.

The current situation we have regarding orphan medicines is clearly not working. That is why Deputy Brassil has brought forward this Bill and why, I believe, there will be cross-party support for it. I commend this Bill which Sinn Féin will support. We hope that it is the start of a significant movement on this issue.

We all have listed off the cases. We know the names of the drugs. We usually know the first names of those who are campaigning because we meet them but we should not have to meet them and they should not have to feel that they have to come here. The truth is the pressure they apply often works. When they get the initial "No", sometimes when sufficient pressure is applied it works. That should not be the criterion. It should not be the case that the sickest people who travel the furthest and who stand outside the longest are the ones who can get the medicines. It has to be the case that there is a process that is fit for purpose, that does not always give every person exactly what he or she believes it should, that is based on clinical evidence and that involves those patients. I would urge the Minister to reconsider and join with us, Sinn Féin, and the other parties in ensuring that the Bill is passed.

I very much support this Bill which I feel strongly about. I congratulate Deputy Brassil on all his work, which I have seen first hand in committee. I have worked closely with Deputy Brassil on some aspects of how we need to proceed with orphan drugs. I would go as far as to say, as I stated to Deputy Brassil previously, that if he was not able to get this Bill through I would even look at providing some Labour Party time, or co-signing it to help him if needs be, because I feel that strongly about it.

This is absolutely necessary. We have to change quickly what we are doing in regard to orphan drugs. If we do not, we will let down people all over the country. My party was the only one that put forward a separate ring-fenced budget for orphan drugs in our proposals for the previous budget and a requirement for extra funding and for it to be distinguished and separate.

The 2013 Act, while progressive at the time, is limited. That is because this issue is moving so quickly. It is moving all the time. We are getting to the stage where drugs will be customised for the individual patient. Later, I will get to other issues relating to that, specifically to do with genetics. That is where we are going. The legislation and protocols need to change rapidly to reflect this dynamic situation.

On the process of getting through a health technology assessment, HTA, as currently identified, Professor Michael Barry, in fairness to him, admitted straight-up that a drug that can affect fewer than 100 patients would never meet the thresholds. That goes for a range of potential drugs. We obviously have a serious problem in that this needs to be changed.

We need to get to a position where we find out whether drugs are worth it from a medical point of view for the patient rather than merely from a cost or return point of view. In many cases, we cannot get to that because there is not enough capacity to generate data to analyse because we are not getting them through in the first place.

It is a chicken and egg scenario and that is a big problem. There are major delays in orphan drugs being approved in Ireland compared with other countries and issues in terms of how they are reimbursed. The process in Ireland is far slower than those of most other European countries. The health technology assessment process is not just about cost-effectiveness or affordability as other issues such as equity of access, prioritisation, severity of the condition and the demographics of those affected must be taken into account in the decision-making process.

I acknowledge the decision of the Government to create a national rare disease plan for Ireland and the positives that have come from that plan. I also acknowledge the decision to joint the BeNeLuxA initiative although I have not seen much progress in that regard and it has had little impact. However, both are positive developments. There is such rapid change in this area that we must act far more quickly. Multi-annual budgets for orphan drugs should be brought in to allow us to plan ahead in terms of the approval process. As other Members have stated, funding must be ring-fenced.

The Oireachtas Joint Committee on Health carried out a significant amount of work in preparing a report on orphan drugs. I was not impressed by the contributions of representatives of the Department and the HSE with regard to our report because I was unsure that the impact of the committee's comments or its emphasis on areas of particular concern were fully taken on board.

There are issues in regard to clinical trials and how they are managed. The Minister made reference to my neighbour, Marion Kelly, one of two Alpha-1 antitrypsin deficiency patients who died as a result of a clinical trial which was stopped. There are again concerns regarding the provision of Respreeza. However, that situation should not have happened. Until very recently, the communication in regard to that matter was disastrous, as Deputy Brassil and others in the Chamber are aware. Families must be kept informed on such issues and the HSE must decide on how drugs are to administered and people looked after. Communication with the families and support groups was extremely poor.

Immunotherapy will be critical in terms of the future of drug provision. Obviously, there will be more orphan drugs and they will provide greater benefits. However, I have a serious concern with our ability to provide such drugs and make decisions in that regard if we do not build up our capacity for genetic diagnosis. The recent issue in Crumlin hospital should be a warning to all Members that our capacity in terms of genetic analysis is far from where it ought to be. We need more genetic services, consultant geneticists, genetic counsellors and genetic testing facilities to be able to take advantage of drugs that will come onto the market and analyse patients for suitability. Until those services are put in place, waiting lists will grow, decisions on drugs will be deferred and we will not be able to do the best for those who need it. I ask the Minster of State, Deputy Stanton, to relay to the Minister, Deputy Harris, my concerns which regard to both the protocols and budget provision for orphan drugs and the process by which we will customise our future decision making and how that can be done without the required genetic services.

It is obvious that we cannot continue with the current situation with regard to the provision of the drugs referred to by Members and the continuous lobbying for access to such drugs. I do not blame anyone for such lobbying. People are entirely right to lobby for them. They are passionate about the issue. Family members or friends of patients consistently hold briefings in the audiovisual room or protest outside the gates of Leinster House to lobby for access to certain drugs. It is a myth that the decision on access to drugs is solely in the remit of the HSE and the NCPE. I explored this issue at the health committee. The Minister has the power to bypass that process, as was done for Orkambi. It can be done for any other drug, as evidenced by the making available of pembrolizumab to 221 women affected by the CervicalCheck scandal. An article by Professor Michael Barry in last weekend’s The Sunday Business Post addressed political interference in this area. The majority of political interference in terms of circumventing the process for assigning drugs in this country is at the hands of the Government, as was the case for Orkambi and pembrolizumab. Members argue in favour of certain drugs. However, the Government has decided to circumvent the process in certain circumstances instead of it being progressed exclusively by the HSE and the NCPE. That power is referenced in the agreement with the Irish Pharmaceutical Healthcare Association.

With the support of other Deputies, I have raised the critically important issue of access to pembrolizumab on numerous occasions. The Government decided to assign pembrolizumab for Vicky Phelan. We cannot continue with the situation whereby Áine Morgan and Tracey Brennan, who are watching these proceedings, are not being allowed to access pembrolizumab but Vicky Phelan is getting it for free. The State cannot treat women differently. On three occasions, the Minister has publicly committed to sorting out this issue. This is the Christmas present those women want. I urge and implore the Minister of State, Deputy Stanton, to ensure the Minister grants them their Christmas wish. They deserve it, as do other women with stage 4 cervical cancer. Those women must not be treated differently from Vicky Phelan, who has campaigned for equal access to the drug. I speak to her regularly on this issue. This badly needs to be done. The current situation is very unfair and must end. The women must be given immediate access to the drug free of charge. Legally, two sets of women must not be treated in that way in this country.

I welcome the opportunity to contribute on orphan drugs. There debate on the issue has been ongoing since I was elected to the Dáil in February 2016. The Bill will go some way to providing equity for those who live with rare conditions in Ireland. The campaign by those affected by cystic fibrosis for access to Orkambi probably brought this issue to the fore. Since then, parents and families those with rare diseases have had to consistently protest and highlight their situation.

I refer to Kuvan and Spinraza. Kuvan is used in the treatment of phenylketonuria, PKU. It is readily available in mainland Europe but, bizarrely, is not available to those who have PKU in Ireland. Ironically, the drug is made in Ireland, as are many orphan drugs that are not available to Irish citizens. It is incredible that parents are forced to fundraise, beg or go public to get access to these drugs.

Some pharmaceutical companies get large grants from the IDA and benefit from low levels of corporation tax. Big pharma has governments and patients over a barrel. The companies make vast profits. Pharmaceutical companies across the world have grown exponentially in the past 15 or 20 years because there are significant money and profits to be made from drugs.

It is probably no exaggeration to state that the public perceives the current evaluation of orphan drugs process to be unfair. I read the Oireachtas Joint Committee on Health report on evaluating orphan drugs published in February of this year and it is a really good document.

Some of the documents published in the Oireachtas probably never see the light of day. I encourage Members to read this one, however. It is a really good piece of work. Some of the recommendations are bang on the money. They include changing the evaluation process, more patient participation and a review of the 2013 Act, which is what we are speaking about today. Also included are recommendations on reimbursement and pricing and compassionate use. It is recommended that the National Centre for Pharmacoeconomics QALY processes be revised and replaced. This is at the heart of the debate. It is the committee's view that unless the State has some involvement in the research and development element of orphan drugs, we will constantly be on the back foot with regard to funding. This is really important work by the joint committee. The committee recommended that the State have a role in drugs manufacturing, research and so forth.

This is about life and death. Nobody has a monopoly on compassion, regardless of what side of the House one is on. Everybody wants to see the best done for those we engage with, who need this medicine. It is absurd to force families to campaign to get medication. When their lives are at risk, they cannot get it because it comes down to money. To me and most people, that is absurd. Medicine has made astronomical strides for humankind but when it comes to profit it has made strides backwards. It is absurd in this day and age that one cannot gain access to medicine. Therefore, we welcome this Bill.

Some 300,000 Irish people are affected by rare diseases. They comprise one of the largest health groupings in the country. I welcome the fact that the Bill is attempting to deal with access to drugs, which is a major issue for those people. As has been mentioned, we have seen people having to campaign for Orkambi and Spinraza and now from pembro. It is unfortunate that the Minister is not present. He has to commit tonight that women will have access this week to pembro on an equal basis before Christmas. Women will die if it is left until the new year. We all know it will be several weeks later if it is left until the new year. Lives are literally at risk. We passed emergency laws here one night for the banks. This can be done for the affected women. We have heard the Taoiseach saying it will happen but there is a time impediment. A date must be set. I commend Vicky Phelan on giving of her time for the other women affected.

It would be wrong for us not to bring the drug companies into the equation. What an indictment of capitalism and the system that people suffer and die because vital drugs and treatment are out of their reach because private drug companies profit and thrive on rarity and need. The CEOs of drug companies were once doctors but now they are businessmen. They are paid seven-figure sums. The CEO of Merck, one of the top ten global pharmaceutical companies and the company that produces pembro, is paid $25 million. The company's revenue is $35 billion. The global pharmaceutical market is worth $1.4 trillion. This is a massive business. The onus is on all the companies to satisfy the shareholders, not help humanity.

A year or so ago, Martin Shkreli told the Financial Times his only obligation was to shareholders. He infamously increased the price of the HIV and transplant drug Daraprim from $13.50 per dose to $750, representing a 5,000% increase. That is what these people can do. Nobody has a monopoly on compassion but some have a very strict attachment to an ideology that allows this to happen. Parasites are running these pharmaceutical companies. EpiPens were increased in price by 400%. As has been said, many of these are being produced in this country.

What is the solution? This Bill is making an effort to improve the current situation, which is outrageous given the length of time it takes for people to get a drug trial, a licence and so on. These drug companies should be nationalised and brought under public control. Their research should be shared. They should be forced to open their books to show us what it actually costs to produce the drugs. An antidepressant that Martin Shkreli, who put up the price of the HIV drug, was taking was 17 cent per pill, yet these parasites are allowed to profiteer beyond belief.

When we debated water charges here about three years ago, nobody in the Dáil wanted to privatise water. How could one privatise drug companies? The drugs are life-saving medications that are produced because of the ingenuity of scientists, most of whom want to benefit humanity. We should now consider forcing the companies to share their research. We should examine patent laws. There is an example from South Africa concerning the HIV drug. The companies should be taken under public control and run in the public interest. I do not see why it would be any different from water or anything else. Health should not be a commodity.

The Minister really has to make a commitment. It has been put out on social media that the women will get equal treatment but that is not the case. There is a fear, as expressed by Vicky Phelan, that this will not happen before Christmas. That is untenable for the women involved.

I thank Deputy Brassil for introducing this Bill and applying to it his expertise in this area. It involves an amendment to the Health (Pricing and Supply of Medical Goods) Act 2013. Deputy Brassil is proposing to establish specific criteria that would be applicable to orphan medical products in regard to the HSE making decisions on reimbursement and prescription by doctors.

The health committee has had several meetings on orphan drugs, most recently last month. One of the recommendations of the committee was that there should be a review of the legislation. Recommendation No. 2 states: "The Committee recommends that a review of the 2013 Act be commenced by the Department of Health, to present a report to the Minister for Health within 6 months of the publication of this report, identifying potential legislative barriers to the reimbursement of orphan drugs and corresponding legislative amendments, as appropriate." It was identified by witnesses who appeared before the committee that there was a legislative barrier to the consideration of orphan drugs. It was stated by representatives from the HSE and the Department that the 2013 legislation limited the examination of orphan drugs. It was stated that since it did not specify specific criteria for orphan drugs, the examination of orphan drugs was limited. It was said that if there were to be change, there would have to be a change to the legislation. That was one of the recommendations of the committee.

When the representatives of the HSE and Department were before the committee last month, however, the representatives of the Department said the Minister was satisfied there is no legal barrier to the assessment of orphan drugs because there is sufficient scope for the HSE to have regard to the special circumstances that apply to them. The legislation was introduced in 2013. It is now almost six years later and the issue of orphan drugs is far more complex than the legislation envisaged. That is why Deputy Brassil's Bill is timely. It is introducing new, specific criteria to apply to orphan drugs. The Minister has determined and said to the committee that he sees no legal barriers but there are legal barriers because orphan drugs are different from regular drugs.

The committee made 15 recommendations. I will not go through them all. There was a recommendation that specific evaluation of orphan drugs take into account that the drugs are for rare and very rare diseases, with perhaps 5,000 and 10,000 people affected.

The numbers in Ireland are small and that is an issue for patients and their advocacy groups. There was a recommendation that because of the small numbers we should look internationally at the evaluation process for orphan drugs in Europe and tap into it and avail of the qualitative information available throughout Europe. As the numbers in Ireland are small, we have to have a collaborative approach throughout Europe and Ireland has started to engage with other European countries on health technology assessment and, perhaps, on negotiating a price from the pharmaceutical companies. Orphan drugs are confined by existing legislation and what Deputy Brassil has proposed is that the quality assessment threshold of 45,000 should not apply to orphan drugs because they are a unique niche-type of drug that apply to a very small number of people but have a huge effect on them if they are seen to be effective. The assessment process needs to be more transparent. The companies, patients and support groups feel they are left out of the assessment process. There is also a recommendation to appoint an independent expert to review the assessment process. I do not believe this has been achieved yet.

The proof of the pudding is that the number of orphan drugs available in Ireland is low compared to other European countries. This points to a deficiency in the assessment process. Ireland has approved only 22 orphan drugs whereas many European countries have approved two to three times this number. We must bring it back to the patient because there is a detachment between the assessment process and the patient. There are opportunities to have managed access programmes to negotiate a price only for those who respond to treatment, because not everybody responds to treatment, and to pay only for the responders.

This is a very important Bill. The amendment to the Health (Pricing and Supply of Medical Goods) Act 2013 will establish specific grounds with regard to getting some orphan products assigned to the reimbursements list. Unfortunately, we are seeing more and more rare conditions in Ireland and it is vital those receiving medication for rare conditions are entitled to get it reimbursed through the State. Some of these medications are extremely expensive and most people would not be able to afford these life-changing medicines.

At present, the HSE does not differentiate orphan medicinal products from other medicines in assessing their cost effectiveness when making a decision in respect of whether a medicine should be reimbursed. The problem with this is the HSE is looking at medicines that affect the highest number of the population and taking this into consideration when deciding whether a medicine will be reimbursed. Orphan medicinal products are for a minority of people. If this is the HSE's method of deciding then people with a serious condition who are in the minority have no chance of getting reimbursed even though their conditions are so serious and the orphan medicine can be life-saving for them. The HSE still bases its decisions on the percentage of the population affected and does not take into account how life saving a particular medicine could be to a person. The HSE's method of making decisions on reimbursement needs to be addressed as a matter of urgency.

I cannot stand up here tonight when we are speaking about rare conditions and not mention the hardship Vera Twomey and other families in Ireland have had to endure. Deputy Gino Kenny has been a champion for these causes down through the years. It is a disgrace these families have had to go to the lengths they did to get medicinal cannabis for their children. Earlier this month, a Cork based mother travelled to Barcelona to access medicinal cannabis for her son, Michael, who was born with a rare disease that causes him to suffer seizures. This lady was stopped at the airport on the way home and the medicinal cannabis was confiscated from her. I have pleaded that medicinal cannabis should be made available in Ireland for cases such as that of Vera Twomey's daughter, Ava. We could introduce medicinal cannabis safely into our country, whereby it would be available only through our pharmacies and under strict regulations. Medicinal cannabis has been legalised in more than ten European countries, Canada, Australia and 30 US states. We need to step up to the plate and stop innocent children who have rare conditions from suffering any more than they already do.

Another massive letdown is the increase in the VAT rate to 23% on food supplements, vitamins and minerals. A total of 80% of people use health food supplements, including vitamins, minerals, omega 3, probiotics and super foods. The Government will increase the VAT rate on these products from 1 January. A total of 84% of the population disagrees with this increase. When will the Minister for Health listen to the people who elected him? I ask him to reverse the VAT increase. It is his duty as Minister for Health to do all he can to encourage the people of Ireland to live as healthily as they can but instead he is putting financial obstacles in the way of the people when it comes to their health.

Another example of this is the FreeStyle Libre device for people with diabetes to replace the current method by which diabetics check their blood levels. The device has a small sensor that automatically measures and continuously stores glucose readings day and night. The device is particularly useful for children and the elderly and can be used while the person sleeps. The device costs €120 to buy and an additional €120 a month for 16 patches. Since 3 April, the FreeStyle Libre system has been reimbursed for some patients between the ages of four and 21. I very much welcome this decision but many of our elderly diabetic patients struggle with the cumbersome old method of using lancets to draw blood. As a diabetic I fully understand the constraints this chronic condition places on a person's life. The risk of diabetes increases with age and it is estimated that 15,600 people over the age of 80 suffer from diabetes.

I thank my colleague, Deputy John Brassil, for bringing forward the Bill and I welcome the opportunity to speak on it. I do not profess to be a member of a health committee but a couple of weeks ago I sat in the audiovisual room where Deputy Alan Kelly hosted Vicky Phelan and two other ladies, Áine Morgan and Tracey Brennan. To my dismay I could not believe what I was hearing, which was that Vicky was in receipt of pembro but the other two ladies were not. They are at advanced stages of cervical cancer. Their only ask on the night was to have access to this orphan drug. This is something for which we should press really hard, as was done successfully with Orkambi and Kalydeco. They brought immense relief to all the families involved. For those ladies, who are sitting at home at this moment in time, their ask is with regard to pembro. On that evening, a consultant was with them. Fewer than 50 people are looking for access to it. The Bill is a response to a medicine crisis faced by Irish patients suffering from a rare disease.

The Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2018 seeks to reform the HSE's current reimbursement process by establishing for the first time a unique process for assessing an orphan drug for reimbursement in Ireland. The Bill brings a long overdue and credible solution to the ongoing crisis surrounding access to medicine. I compliment Deputy Brassil. When he spoke earlier he said that on 27 September 2017 he listened to a response from the Minister and has returned with a solution. I hope the Government will support him.

I commend Deputy Brassil on introducing the Bill to reform the HSE's reimbursement process by establishing a unique process for assessing orphan drug for reimbursement in Ireland. There are around 7,000 rare or orphan diseases for which no authorised or satisfactory method of treatment exists. Orphan drugs treat, diagnose or prevent diseases that affect fewer than five in 10,000 people. Many are life-threatening or debilitating, and they particularly affect the very young, who often do not survive beyond adolescence. Without treatment, the quality of life of these patients is seriously affected and their lives may be shortened. This represents the grim reality they and their families are facing.

There have been great advances in developing orphan drugs but at present they are assessed under the same guidelines and procedures as standard drugs despite the clear differences between the two.

Other EU countries have adapted their processes for this very reason. It is time we did so.

The purpose of the Bill is to put in place a structure for the HSE to appropriately assess orphan drugs when making a relevant decision regarding adding an item to the reimbursement list. The Joint Committee on Health recommended considerable change to the evaluation process of orphan drugs. It agrees that the current assessment is not effective and that this process neglects to ensure the best outcome for patients. Reforming the process for assessing the impact and benefit of orphan and rare drugs would be a fairer way of ensuring people get the treatment they need and deserve. Much-needed orphan drugs would reach and benefit patients with rare fatal genetic diseases sooner. It is not acceptable that citizens suffering from severe illnesses are forced to take to the streets or go public in the media to have their voices heard.

We read daily about or meet patients who suffer from severe, often life-threatening, rare diseases who cannot secure access to medicine. Some notable examples of orphan drugs, on which we have all made representations previously, include Orkambi for cystic fibrosis, CF, Vimizim, which overcomes the deficiency causing mucopolysaccharidosis type IV, or Morquio syndrome, and Respreeza, the life-changing drug for emphysema caused by Alpha-1. Orkambi is a game-changer because it is the first drug to treat the underlying cause of CF in over half the CF population in Ireland. The successful funding of Orkambi will result in more research and development into rare disease drugs, not just for those with CF but also for those with other rare diseases.

In short, the funding of Orkambi will save more lives and not only within the CF community. Vimizim is by no means a cure for the rare Morquio disease but it has increasingly enhanced the quality of lives and ensured people have more energy and improved health. The disease causes lifelong problems including heart disease, skeletal abnormalities, vision and hearing loss and respiratory issues. Vimizim is the only medicine available to treat the condition. It replaces the enzyme that the body is missing. Finally, Respreeza is the only therapy clearly shown to slow the progression of emphysema caused by Alpha-1, resulting in a reduction in the frequency and severity of chest infections and associated hospital admissions, and, therefore, increased life expectancy. This Bill brings a long overdue and credible solution to the ongoing crisis surrounding access to medicine for patients with rare diseases in Ireland. I again commend Deputy Brassil on his work in respect of this Bill.

The Minister is unable to respond because he can only speak once in the debate. That is unusual and it happens on Tuesdays and Wednesdays. He can speak twice on a Thursday. It is bizarre but that is the way it is. He has listened carefully to what has been said. He has informed me that he will take note of this debate. It has been sincere and important. I commend my colleagues on the quality of the debate, to which I have listened. I thank Deputies on all sides for their contributions to this important debate on the Health (Pricing and Supply of Medical Goods) (Amendment) Bill 2018. I welcome the opportunity to contribute.

I appreciate that this Bill represents a well-meant endeavour on the part of Deputy Brassil to allow patients timely access to new and innovative orphan medicinal products. I acknowledge the considerable work which Deputy Brassil and members of the Joint Committee on Health have done in recent months in respect of orphan drugs. In February, the joint committee published a report - cited on a number of occasions this evening - on evaluating orphan drugs. The Minister and the HSE have given detailed consideration to the recommendations contained in that report. As a result, a number of recent changes have been introduced to the assessment and reimbursement process within the confines of the Health (Pricing and Supply of Medical Goods) Act 2013. This provides a greater balance and transparency to the assessment process where orphan drugs are concerned.

As every Deputy is aware, a rare disease diagnosis poses particular difficulties for patients who are affected and for the people who care for them. Living with a rare disease is a constant daily challenge which places enormous pressure on patients and their families. Deputy O'Reilly mentioned people outside of the gates of Leinster house and we can understand why they are there. It is vital that these patients receive the best evidence-based, cost-effective treatments our health service can provide. I completely understand that access to potentially beneficial medicines for the treatment of rare diseases is a crucial issue for these patients and their families. I join Deputy Gino Kenny in stating that nobody has a monopoly on compassion. The Deputy is correct and we feel that on all sides of the House.

The Minister is aware of campaigns by parents and advocacy groups for access to orphan medicinal products and he wishes to assure them that he, his Department and the HSE continuously work to address issues around access for patients to new and innovative medicines. The Government will be opposing this Bill on the basis that the statutory process for assessing medicines as contained in the Health (Pricing and Supply of Medical Goods) Act 2013 is fit for purpose where orphan drugs are concerned. In 2013, following on from substantial deliberations, Members of the Oireachtas put in place a thorough legal framework in the 2013 Act. The latter accords full statutory powers to the HSE to assess and make decisions on the reimbursement of medicines, taking account of a range of objective factors and expert opinion as appropriate. The Act specifies the criteria to be applied, including the clinical effectiveness and cost-effectiveness of the product, the opportunity cost and the impact on resources that are available to the HSE. While the Health (Pricing and Supply of Medical Goods) Act 2013 did not establish specific criteria to be used by the HSE in reaching decisions on the reimbursement of orphan medicinal products, the HSE and the NCPE consider issues such as the small patient numbers and the rarity of the condition to be treated when evaluating these medicines. There is an acute awareness of the vulnerability of these patients and the chronically debilitating or life threatening nature of many of these rare diseases. It is the Government's view that the criteria that apply to the evaluation process allow ample scope for the HSE to consider the particular circumstances that pertain to orphan drugs including that oftentimes there is a high level of unmet need for these rare disease conditions.

Deputies have correctly identified that increasingly orphan medicinal products are being developed to target very rare conditions. In view of the small number of patients affected by rare diseases, the pharmaceutical industry has been reluctant to invest in the research and development of medicinal products to treat them. In 2000, the EU introduced new legislation with the aim of providing incentives for the development of medicines for rare diseases. This intervention has proven very successful and has stimulated the development of many orphan drugs. The Government is fully aware and appreciative of the potential which these new orphan medical products can have in creating better health outcomes for Irish patients living with a rare disease diagnosis. We, of course, want all of our citizens to have access to the newest, most innovative medicines out there and as quickly as possible. Increasingly, the orphan medicinal products being developed to target very rare conditions are coming with extremely high list prices, prices which in many cases go well beyond what our health service can afford.

Health financial systems throughout the developed world are grappling with the challenges which these new and very often expensive orphan medicinal products pose. In many cases, the main driver of the price is an estimate of the maximum amount that health care systems can pay and is not connected to the health benefit of the treatment. Deputy Kelly is correct. It is expected that the trend will be towards more applications for orphan drugs in the years ahead. It is estimated that in the region of 45 new molecules, many of which will be orphan drugs, are due to receive market authorisation in Europe each year over the next five years. There is, therefore, no end-point whereby the pressure on the system to fund new products will abate. That is why it is essential that Ireland retains a threshold incremental cost-effective ratio so as to ensure that decisions in relation to the reimbursement of medicines are made on an objective and scientific basis, recognising the health needs of the entire population and other factors.

Affordability and sustainability remain major challenges for the health service in the context of our budget-limited health service. A less rigorous set of rules for orphan products will not change this. The HSE is required under the 2013 Act to consider the funding challenges that orphan drugs represent. It does this by drawing on the criteria contained in the Health Act including the potential or actual budget impact of the drug in question and the cost-effectiveness of meeting health needs by supplying a particular item rather than providing other health services. These are difficult decisions that must be made in the context of the availability of finite resources in the face of ever-competing demands. Since the signing of the four-year framework agreement on the supply and pricing of medicines in 2016, a significant number of orphan drugs have been reimbursed by the State.

In terms of the availability of orphan medicinal products in Ireland, the 2013 Act is delivering and patients with rare diseases are receiving access to new and innovative medicines. The Government is opposing this Bill because it is satisfied that the current criteria contained in Act are fit for purpose when assessing orphan drugs. There is sufficient flexibility contained within the current legislation to allow ample scope for the HSE to consider all relevant evidence that pertains to orphan drugs.

The Government is of the view that the 2013 Health Act is delivering on its objectives to provide new and innovative medicines to Irish patients, including those with a rare disease diagnosis.

The Bill as drafted would disregard any threshold incremental cost-effective ratio in the assessment of orphan medicines, thereby removing an accepted, validated economic evaluation system for assessing medicines. In doing so, it would dilute considerations around affordability and sustainability. The health service must look to care for the entire population. Accordingly, every decision, including the reimbursement of orphan medicinal products, must be based on scientific assumptions, clinical evidence and cost-effective analysis as provided for by the 2013 Health Act.

The Minister for Health is aware of the human side of this entire process. Patients who are living with a rare disease diagnosis face enormous challenges in their day-to-day life. Such a diagnosis has significant repercussions for the whole family. These diseases can be acute or chronic in nature. While there has been remarkable progress in advancing innovation for rare diseases, there still remain significant challenges in that there are many rare conditions which still do not have a treatment option.

It is right that Deputies assess and question the way that we deliver health services to our citizens. The Minister for Health and I appreciate the conviction and sentiment behind the Bill. The Minister for Health looks forward to further positive engagement with Deputies in seeking solutions to address the challenges which we all face around securing more timely access to new and innovative medicines for all our citizens in an affordable and sustainable manner. I will bring the Deputies’ comments this evening to the Minister’s attention. This is an important and sincere debate. It is one which will add to our thinking and progress in the future. I thank Deputy Brassil for the work he has done on this matter. The Minister is looking forward to continuing to engage on this important issue.

I must remind the Minister for Health of his statement on 27 September which can be summarised as, “I think it is broken too.” His comment then was accurate. However, his reply tonight denies the fact that the current system is not sufficient to allow for access to orphan drugs on reimbursement programmes.

The Minister of State and his officials have heard the debate and support for the Bill is evident. I am acting in good faith and request the Minister of State to work with me with a view to improving the process whereby orphan drugs can be made available in a more timely manner and which will ensure value for money for the HSE. Speaking on value for money, I have pointed out many times that savings of up to €300 million can be made on biosimilar products available to the HSE if it goes after it. At the Oireachtas health committee last month, the point was made that biosimilar products are not interchangeable. However, as the Minister of State knows as well as I do, if physicians are directed to prescribe biosimilar products, then the original brand will match the biosimilar price in order to maintain market share. That is simple economics. I am asking that part of the savings made with biosimilar products be ring-fenced for new orphan drugs every year. This I believe is practical, cost-effective and doable.

The history in this area is not good. Respreeza for Alpha-1 took the death of two people before it was sorted. Orkambi would never have been approved were it not for the protests and public outcry over it. Pembro, which did not even go through the National Centre for Pharmacoeconomics, NCPE, process, would not be available if it were not for Vicky Phelan. It is obvious the current system is not fit for purpose. Unless we amend the Act to give further direction and clarification to the HSE and NCPE, the current cycle will go on.

The PKU Association of Ireland has been exemplary in its approach in getting reimbursement for Kuvan. Last month we were told at the Oireachtas health committee that Kuvan was approved on a limited access basis. To my knowledge, it is still delayed and will only work among a small subgroup of patients. However, a newer product, Palynziq, which has FDA approval, should work for up to 85% of Irish sufferers. Are we to spend another two years waiting for this drug to go through the same process as we did for Kuvan? Similarly, for Spinraza for spinal muscular atrophy, this drug is needed now and has been proved to work specifically for type 1 sufferers.

I want to work with the Minister and the NCPE to improve the Act. My Bill will in no way oblige the HSE to reimburse any medicine which it does not believe represents good value for the State. Section 4, detailing the criteria which the HSE must consider when making a decision on reimbursing the drug, would ensure it weighs up the potential budget impact of the proposed medicine, as well as the resources available to the HSE at that time. It will place on a statutory footing the key criteria which should be assessed in reaching a timely decision on orphan drugs, including clinical and cost effectiveness, unmet need, the relevant views of patient organisations and the availability of the medicine in other European countries.

The HSE is doing good work in securing value for the State. However, the time it takes to reach such agreements for orphan drugs is unacceptable. Very often these medicines are available across Europe but not in Ireland. If patients are fortunate to be given access to such a medicine, it can take up to two years which in many cases is often too late. We need a system whereby value is secured in a timely manner, that is not undermined and, at its very foundation, affected by a flawed assessment process. Increasing the availability of orphan drugs would undeniably result in more cost. However, it is important to remember that rare diseases only affect a small patient population, between 5,000 to 10,000 people. Often one will hear about expensive medications per patient. However for rare diseases, the actual budget impact, namely, the cost of the drug multiplied by the total number of applicable patients, is typically small. Licensed orphan drugs only exist for a fraction of rare diseases. Any cost incurred will be viewed in the context of Ireland catching up with European countries where these medicines are already available. Every country in the world is faced with the decision to not purchase medicines and save money in the short term. However, this approach results in a widening gap in patient outcomes in Ireland versus the rest of Europe. In my view, this stance is morally wrong.

Will the Minister of State retract his opposition to this Bill and work with me through Committee Stage? The Bill will get the support of the rest of the House. I am trying to improve access and not break the HSE. I am aware of the constraints but there is a workable solution. I am asking the Minister to work with me on this.

Question put.

In accordance with Standing Order 70(2), the division is postponed until the weekly division time on Thursday, 17 January 2019.