Pádraig O'Sullivan (Cork North Central, Fianna Fail)
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Last week, following the announcement of the retirement of the Chief Medical Officer, I noted that the list of the CMO's accomplishments included the publication of the previous national rare disease plan. The plan was published in 2014 and extended until 2018. Since then, the old national rare disease plan has not been replaced. That made me wonder what we have done in the past four years about the publication of a new plan. A new plan is important because there are approximately 300,000 people in Ireland who suffer from a rare disease.

Last year, the Minister for Health allocated the largest budget to rare diseases - €50 million - in the history of the State. That was very welcome. However, the difficulty is not only that 300,000 patients need to see the publication of the plan but also the startling statistic that, globally, 30% of all children who are unfortunate enough to have a rare disease, whether they contract it or it is genetic, will die by the age of five years. That is a scary statistic and an unfortunate truth. In this country we are laggards in our provision for those with rare diseases. I say that because our national rare disease plan is yet to be published, despite the fact that "publishing an updated national rare diseases plan" is a priority in the programme for Government. We are two years into the lifetime of this Government. Can we have an update on the publication of that plan?

Linked to that is the Mazars report that was supposed to look into the higher technical assessment, HTA, process for the reimbursement of orphan drugs for rare diseases. It was commissioned in 2017 and was submitted to the Department of Health in 2020. It has essentially sat on somebody's desk for the last two years, unpublished, at a cost to the State of €86,000. The Minister of State might not have the details to hand but I would love to know where the Mazars report is. It is incomprehensible that we would spend so much money on a report and leave it to gather dust. It will contain valuable insights into how we plan for rare diseases in the future. I would love to know what is in the report. It must be damning or there must be something coming that will require dramatic change because we seem to be afraid to publish it. I want to see what will happen with the Mazars report and would appreciate an update.

I have been interested in this area since I met some affected families about two years ago. I have spoken before of how difficult it is to sit in people's kitchens and listen to them say that if they do not get access to a drug, their health will seriously deteriorate and in many cases they will die. It is very hard to have those conversations. As I have said previously to the Minister for Health, the average waiting time in this country to approve a drug for a rare disease is 1,200 days. People wait nearly four years to be told whether they will have access to a potentially life-saving drug. Even in that time, it could take 400 days just to negotiate with a pharmaceutical company. Many of the delays in the area of rare diseases are administrative and bureaucratic. Surely we can do things an awful lot better.

Seán Fleming (Laois-Offaly, Fianna Fail)
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I thank Deputy Pádraig O’Sullivan for raising this matter, which is an area in which he has shown particular interest and knowledge. As he noted, having met people face-to-face, he understands the issue. It is important that the views of those who have first-hand experience of the need for orphan drugs are adequately reflected in Dáil Éireann.

All of us in this House fully appreciate how devastating a diagnosis of a rare disease can be for patients, families, and carers. I believe there is cross-party support for doing everything we can to help patients who live with these awful conditions on a daily basis. I know Deputy O'Sullivan has been a long-standing advocate in this area and I commend him on his recent orphan medicines Bill, which was widely supported in the House on its Second Reading. The Minister for Health looks forward to further positive engagements as this Bill progresses, as do I.

The Government's track record on both initiatives and funding for rare diseases speaks for itself. I will mention a few matters in the time available to me. Many of the recommendations of A National Rare Disease Plan for Ireland 2014–2018 have already been implemented, including the establishment of a national rare disease office incorporating the HSE national clinical programme for rare diseases. In 2021, the Minister met with all relevant stakeholders in this area, including the HSE, the national rare disease office and the rare diseases task force. The Minister secured the agreement of all parties for a number of priority areas for the coming period, including European reference networks, research and registries, access to services and medicines, diagnosis, and policy.

I am pleased to note that Governments have a long track record in supporting rare disease research through the Health Research Board, HRB. In the past ten years alone, the HRB has invested €14 million in rare disease research. On access to new medicines for the treatment of rare diseases, the Government has provided funding which has made a real and concrete difference. Budget 2021 allocated €50 million for the reimbursement of new drugs, enabling the HSE to approve 52 new medicines, 19 of which were for the treatment of rare diseases.

That includes a number of medicines that deal with innovative gene therapy treatments for spinal muscular atrophy following a joint assessment with Belgium and the Netherlands. Budget 2022 has allocated a further €30 million for the reimbursement of new medicines. I understand from the HSE that 27 new orphan drugs are currently undergoing a pricing and reimbursement assessment.

There is no getting away from the fact the overriding factor in medicines being approved for reimbursement is the availability of Exchequer resources. This is a challenge for all of us over the coming years as more and more of these medicines become available, though often at very high asking prices. It is estimated about 97 new medicinal indications, including treatments for rare diseases, will receive market authorisation in Europe over the next five years. These new medicines alone will have the potential to add hundreds of millions to the State's annual bill for medicines, which is already in the region of €2.5 billion per year.

On the assessment process, the Deputy will know the Health (Pricing and Supply of Medical Goods) Act 2013 introduced a robust statutory framework for pricing and reimbursement decisions. Under the Act, the HSE has statutory responsibility for decisions on pricing and reimbursement of medicines. The 2013 Act does not provide for a separate rule set for assessing orphan drugs. The EU health technology assessment, HTA, regulation of December 2021 will enable co-operation on clinical evidence requirements for HTAs in clinical trial design, thereby supporting innovation and addressing unmet medical needs.

Pádraig O'Sullivan (Cork North Central, Fianna Fail)
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I thank the Minister of State for the response. I am conscious it is not his responsibility or area of expertise but there are some things I have an issue with in the response. One is that the HSE has approved 52 new medicines, including Zolgensma. Many of these new drugs were approved through the Benelux arrangement the Minister of State referenced in his response. He went on to say about 97 new medicinal indications including treatments for rare diseases will be approved by the EMA in Europe in the next five years but Ireland's track record in this is pitiful. Of the last 47 drugs approved by the EMA, only eight have been approved here. I do not say lightly that we are laggards among our European counterparts, but we are. All the facts point to it.

Later, the response the Minister of State has references the €45,000 quality assisted life year, QALI, threshold and the fact orphan drugs do not have a specific pathway for reimbursement. That is precisely what my Bill, which was introduced here a number of months ago, aims to achieve. It would provide a specific pathway for orphan drugs to be assessed. We must look at other countries. The Swedes can do it properly, as can the Scots and the Danes. What I advocate in my Bill is no different to what those countries have implemented, and it has not broken the banks. Specific to my legislation is a reference that any drug approvals for rare diseases would have to be done subject to budgetary constraints. It is there in black and white. What keeps coming from the Department of Health is that this will weaken our hand at the negotiating table with pharmaceutical companies or that it will cost the State an arm and a leg. That is nonsense. I have said it here before and have no problem reiterating it.

I get frustrated because the most difficult conversation I have ever had with a person was sitting at that kitchen table, as I referenced. The waiting times I referenced earlier are fact. They are from a parliamentary question put by Deputy Michael Moynihan a number of years ago. I gave the statistic at the start about the number of children with a rare disease aged under five years who die, which is 30%. Every year lost negotiating and prevaricating around the price of a drug means lives lost.

Seán Fleming (Laois-Offaly, Fianna Fail)
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I thank the Deputy for his reference to the note I circulated before I spoke, which deals with the HSE having sufficient scope from the legislation to consider the unique circumstances surrounding orphan drugs, such as small patient numbers. The evidence suggests these expensive orphan drugs are getting reimbursed even, in many cases, when they far exceed the National Centre for Pharmacoeconomics QALI of €45,000 the Deputy already mentioned in this contribution.

The pipeline for new medicines coming to market remains strong in Ireland and we are committed to funding access to new and innovative therapies, including those for rare diseases. Increasingly, products within the orphan drug designation have extremely high prices and budget impact. At this stage, one lesson we have learned from Covid is the importance of countries within the EU working together on the pricing, sourcing, contracting and placing of orders for these. Once they get regulatory approval in the different countries we have seen the value of this with respect to the delivery of drugs. It is something I have long called for and something we have learned from the pandemic. That principle should be extended into these areas as well.

On the Mazars report on the drug reimbursement process, this work stream was suspended to reallocate resources to support the pandemic response so I do not have a date for when it can be completed. The Deputy has previously mentioned online publication of the rare disease technology review committee meeting minutes and the HSE and Department support such publication with a view to improving transparency with respect to the committee.

On the issue of guaranteed weighting, which the Deputy has raised before, I understand the Department view is it is not possible at this time to commit to the assigning of guaranteed weighting for rare disease technology review committee recommendation in the assessment process.

I thank the Deputy. I will take the additional points he has raised directly back to the Minister at the first available opportunity.