I move: "That the Bill be now read a Second Time."
I acknowledge the work done by former Deputy John Brassil in preparing this Bill. The Bill essentially replicates the good work John undertook in the previous Dáil. I am eternally grateful to him for his efforts in this regard and his support over recent months. I also acknowledge the good work of the Minister for Health, Deputy Stephen Donnelly. I am aware that he has to go to a Cabinet sub-committee meeting now, but since he has assumed office he has provided the largest drugs budget in the history of the State, adding €50 million to the overall budget this year. This investment needs to be acknowledged as it eclipses any previous drugs budget the State has had.
That said, there is still a significant problem regarding the classification of rare diseases and the processes that surround the approval and reimbursement of orphan drugs. Under existing legislation, there is no differentiation between orphan medicinal drugs and mainstream drugs. It is abundantly clear that the orphan drugs used to treat rare diseases continuously struggle to secure reimbursement in Ireland. Juxtapose that with the position in other European states. The disheartening thing for me when I started my research on this was that Ireland is considered a laggard in European terms. That is the unfortunate situation we find ourselves in. Many countries in the eastern part of Europe are light years ahead of us in the provision of orphan drugs.
The reimbursement process is often protracted, public and pits patients against the State. Patients often have to take to the streets, lobby their public representatives or campaign for the approval of drugs. The purpose of this Bill is to amend the Health (Pricing and Supply of Medical Goods) Act 2013 to put in place a structure for the HSE to appropriately assess orphan medicinal drugs when making a relevant decision regarding an item for reimbursement. It will provide clear guidance to the HSE on appropriate use of the health technology assessment, HTA, guidelines when assessing an orphan drug, as well as establish new criteria for the HSE to consider when making a decision.
The provisions of the Bill are set out in four sections. This is a technical Bill. I do not want to get too technical, but section 1 sets out to define what an orphan drug is. Section 2 inserts the definition into the principal Act. Section 3 amends section 19 of the principal Act, which deals with the HSE decision-making process in adding medical items for reimbursement. It deals with issues around cost-effectiveness, the validity of the quality-adjusted life year, QALY, model and the role of the HTA. Section 4 establishes a new set of criteria for consideration of orphan medicinal drugs, mitigating against low quantitative data and guaranteeing the HSE would consider qualitative data presented on an orphan medicinal product for patients. Section 4 also deals with the concept of the State and pharma engaging in risk-sharing and obliges the State to be cognisant of what our European counterparts are engaged in.
I ask the Minister of State, Deputy Butler, to imagine what it feels like to be diagnosed with a rare disease and possibly be told there is no cure, treatment or way of curtailing the pain. I can only imagine the toll it would take on anyone in that position. Imagine being told there is a new drug or treatment that may help manage this pain, suppress symptoms or improve quality of life. Imagine the hope that availing of such a drug would bring to somebody diagnosed with a debilitating disease. Then - bang - the unfortunate reality hits that in Ireland it takes more than 1,000 days, on average, to potentially get access to that drug.
In response to recent parliamentary questions he tabled, my colleague, Deputy Michael Moynihan, received an outline of all drugs that have gone through the process and been signed off on by the HSE. Comparing these with public data available from the European Medicines Agency, EMA, and HSE websites, we see that for June 2020 to 2021, on average, orphan medicinal products took 1,361 days from securing EMA authorisation to being authorised in Ireland. The breakdown of that 1,361 wait is 426 days to make an application, followed by 429 days undergoing assessment under the so-called rapid review. At 429 days, I question its rapidity. A further 289 days are spent negotiating between the State and the pharma companies. Once a drug is approved, there are a further 217 days before it is implemented. In total, that is 1,361 days or 3.7 years. For somebody with a debilitating disease who could be experiencing great pain, that must be an excruciating wait.
Imagine how deflating and soul-destroying it must be for any human being to have a potentially life-changing drug at his or her fingertips, only to be most likely told he or she cannot be reimbursed for it because there is not enough data to support its use, it is in excess of the restrictive €45,000 QALY threshold or fails some other aspect of the health technology assessment. That is the scenario many people who suffer from rare diseases face. It is unconscionable that the State, which has responsibility for the health and well-being of its citizens, would block, refuse or defer any decision on approving a drug because of the arbitrary €45,000 figure. That is the archaic system presided over in this country.
Let us look at what our EU counterparts are doing. I will not compare us with France or Germany because they have far superior resources to us. I will make comparisons with countries like Sweden and Scotland. In Scotland, an ultra-orphan pathway was established, providing a distinct process for assessment for these rare conditions where qualitative data can be used in the absence of significant quantitative data. The Scots also provided for interim acceptance of some drugs. That acceptance and the subsequent reimbursement are conditional on the provision of further data and evidence over a specific period up to three years. Reimbursement is then provided on the basis that the drug in question clearly demonstrates its efficacy. Any drug that is not proved effective would not be the subject of reimbursement. This idea of risk-sharing between the state and the pharma industry means patients get quick access to potentially life-changing medications. If they do not work or something better comes along, and inevitability in this sphere many drugs come along to replace others, the state does not bear the burden of the cost. It is a clear example of the state and industry working together to provide for patients and improve health outcomes. This State, however, continues to apply the most rigid HTA criteria, which gives orphan drugs little or no chance of being approved if they exceed the arbitrary €45,000 threshold, as attested to by Professor Michael Barry, head of the National Centre for Pharmacoeconomics, in front of an Oireachtas health committee in 2019.
Since 2019, 31 drug indications have been approved by the EMA. Scotland has made 19 of these available; we have only made four of them available. The average time to approve those drugs in Scotland was 13 months; in Ireland, 21 months. These are significant differences in timelines considering the serious and often life-threatening diseases we are speaking about.
The assessment for reimbursement in Sweden is based on three core principals. One, everybody has a right to healthcare. Two, priority is given to patients with the greatest need and those most vulnerable. Third is cost-effectiveness. The big difference between Ireland and Sweden is that, while both use the QALY threshold and the Irish model employs the €45,000 threshold, the Swedes employ no such arbitrary figure. All drugs are assessed on their medical efficacy and the outcomes they deliver for patients, while still being cognisant of cost-effectiveness. An arbitrary figure is not used as a bazooka to disapprove a drug regardless of its level of efficacy.
Some critics of this Bill suggest that to remove the rigid QALY threshold we have in this State would somehow weaken our hand in any negotiations with big pharma. This is a fallacy that was created to detract from this Bill when brought forward previously by former Deputy, John Brassil. No doubt it is a myth that will be circulated again at some stage of this debate. It is stated implicitly in Part 4 that any drug must be assessed as to its potential or actual budget impact in any question being analysed. There is no magic money tree out there to approve all drugs all of the time, but it is possible to streamline legislation and guidelines on this issue in a way that gives patients hope but, more important, often gives them life-changing and life-saving treatments.
In regard to the first section, I undertook an awful lot of research over the past few months into this topic. I refer to the drugs Spinraza, Orkambi, Translarna, Zolgensma and to Onpattro, about which I raised an issue most recently and which the Minister of State has spoken about in previous Dáil debates on this matter. The bottom line is that is no way to conduct an assessment for any type of drug. Deputies and public representatives should not be hauled in here pleading and begging with the Minister of the day to approve one drug over another. There should be a clear transparent process with specific timelines. That evidently is currently not the case in light of the 1,300 plus days I referenced earlier in the reply to Deputy Michael Moynihan's parliamentary questions. That is a life sentence and, in many cases, a death sentence for people.
We need transparency in this regard and this Bill provides it. I am open to discussion and amendments of any description so long as the current system is overhauled and improved because clearly it is not working. Many people in the industry will say that such is the high threshold and, often, failure rates in Ireland in terms of this question in regard to orphan drugs that Ireland is one of the least attractive places for any pharmaceutical company to come and negotiate.
Ireland has entered into an arrangement with other small nations in the Beneluxa agreement. The logic behind that was that together a group of countries could compete and negotiate with big pharma companies in the same way as bigger nations such as France and Germany do. The group came together with the ambition of negotiating better prices for the State and, ultimately, getting drugs for patients. The Beneluxa group has been in place for two years and only one drug has been approved in that two years through that arrangement. To say that we will work with other EU partners is great, I am all about co-operation and working together and improving the case for the State and the case for patients, but the Beneluxa arrangement has implicitly failed in the past two years in that, to my knowledge, it got only one drug approved.
I acknowledge that the Minister of State is standing in for the Minister, Deputy Stephen Donnelly, as he has to attend the Cabinet sub-committee. In the past, there has been near universal agreement on this matter. It is something I am willing to work with other parties on. I am glad to see some of them represented here today. I believe we need to do more for patients. I would welcome the Minister of State's statement on the matter.