Simon Harris (Wicklow, Fine Gael) | Oireachtas source

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.

See this speech in context