Colm Burke (Cork North Central, Fine Gael)
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I thank the Minister of State for taking this matter. I know that health is not his area but I appreciate his taking this so late in the evening. All of those involved in the whole area of rare diseases, both families and those involved in developing new drugs, are concerned at worsening trends which suggest that rare disease patients in Ireland are being subjected to even longer waiting times before acquiring access to life-changing and life-saving orphan medicines in comparison with previous years. The latest EFPIA indicator survey for 2022 suggests that Irish patients are now waiting on average 877 days between approval from the European Medicines Agency and access to a new orphan medicinal product for rare diseases. This puts Ireland 31st out of 37 countries in Europe.

Ireland needs a system of reimbursement that is clear, predictable and time bound with fixed assessment criteria and one that also includes mechanisms that enable immediate early access to rare disease therapies. In 2018 the Oireachtas Joint Committee on Health published a report evaluating orphan drugs which called for a comprehensive review of the reimbursement evaluation process specifically for orphan medical products. Mazars was appointed by the Department of Health to conduct the report on rare diseases which was eventually published on 28 February 2023. Unfortunately, the report only examined the general reimbursement process as a whole and failed to consider the rare disease perspective as originally sought by the health committee. I was on that health committee in 2018 when we set out what needed to be implemented to make sure that these orphan drugs would be made available at an early date.

The Mazars report provides 17 recommendations which, while welcome, do not go far enough to address the issue of timely patient access to life-changing treatments and are too general in nature. Crucially, none of the recommendations involved a change in legislation. Despite the report producing that number of recommendations, very little changed.

I referred to the average waiting time in Ireland between approval by the European Medicines Agency for a drug and that orphan drug becoming available in Ireland as being 877 days. In Germany it is 45 days and in Austria it is 104 days. Therefore, we are way behind. This issue has been going on for over five years. I know the Minister has set up the process. We had the National Rare Disease Plan 2014 to 2018. That has been out of date for five years. I know the Minister is talking about a new national plan. However, when will that be delivered? When with the recommendations that already exist be put in place to deal with this whole issue of rare diseases?

Many people affected cannot get access to medication that is available in other European countries because it has been approved by the European Medicines Agency. This area should now be given priority.

Thomas Byrne (Meath East, Fianna Fail)
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Gabhaim buíochas leis an Teachta as an t-ábhar seo a ardú anocht. I think all of us fully appreciate how devastating a diagnosis of a rare disease can be for patients, families, and carers. I believe that there is cross-party support for doing everything we can to help patients who live with these conditions on a daily basis.

In February, on Rare Diseases Day, the Minister for Health announced a plan to develop a revised national rare disease plan, in line with the commitment in the programme for Government. Initial work for the new plan has commenced and a steering group to progress the new plan is being convened next week to begin to develop a successor to the previous national rare disease plan. The development of a successor plan will allow the steering group to consider areas of the previous plan that need to be further progressed, while also looking to the future needs of those patients and families living with a rare disease diagnosis.

An update on the current state of each of the 48 recommendations in the National Rare Diseases Plan 2014-2018 has been completed by the Department of Health. While a number of areas for improvement remain, various actions have been implemented from the 2014-2018 plan and significant resources invested in this area in recent years. Principal among those actions was the establishment of the national rare disease office, NRDO. The NRDO is the national rare disease co-ordination hub and the HSE's main contact and driver of rare disease initiatives and projects. The NRDO is committed to informing, supporting and empowering people affected by rare conditions, their families, caregivers and healthcare professionals.

The HSE national clinical programme for rare diseases published the HSE Model of Care for Transition from Paediatric to Adult Healthcare Providers in Rare Diseases in 2018 and the Model of Care for Rare Diseases in 2019. The work of the national clinical programme for rare diseases has now been mainstreamed into standard operations under the NRDO.

Furthermore, last year, the HSE was nominated as the national competent authority in an EU Joint Action of European Reference Networks for Rare Diseases, which enables greater co-ordination and sharing of best practices in key areas such as genetic testing. As a result of this collaboration, Ireland is now a member of 18 European reference networks, ERNs, on rare diseases. These ERNs include representation from five academic hospitals and three universities and are co-ordinated by the national rare disease office.

In terms of medicines for rare diseases, the Minister for Health during his term of office has significantly increased the level of funding available for new innovative medicines, including medicines for rare diseases - a combined €100 million in the last three budgets. More than 100 new medicines have been approved including 39 for orphan medicines that are used to treat rare diseases. The medicines have provided new treatments for rare diseases, including cystic fibrosis, multiple sclerosis, spinal muscular atrophy, Duchenne muscular dystrophy, and Leber’s hereditary optic neuropathy.

While the new national rare diseases plan is expected to be launched by quarter 3 of 2024, it will be dependent on the work of the steering group in the development of the plan.

Colm Burke (Cork North Central, Fine Gael)
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I thank the Minister of State. I fully appreciate the funding that has been made available. However, we need to remember the figure I gave at the start. I know very many new orphan drugs have been made available over the past two or three years. We need to remember that we are still talking about the average number of days from approval by the European Medicines Agency to being available here is 877 days. The Alliance of Rare Disease Companies and also the people who have a family member with a rare disease are looking for the appointment of a designated rare diseases liaison person within Department of Health, which is one of the recommendations. That is extremely important because everybody is dealing with a different person within the Department of Health. There is no one person they can talk to about it. That is one of the issues that is now being looked for.

The old national rare disease plan has now expired for over five years. There were 48 recommendations in that plan which were never implemented. The plan has already existed for five years. Many of the proposals in the 2014-18 plan were never implemented and would not require change of legislation to implement. I am asking that those recommendations would be looked at to see how they could be put in place.

The 2018 Oireachtas Joint Committee on Health report was quite specific on the avenue that the Department could go down to try to expedite the process for people with a rare disease. We need to remember that for somebody with a rare disease time is of the essence. If they cannot get access to medication, it will not help them get the appropriate treatment. We now need to go forward, look at the old plan and see what can be put in place without having to wait for the new plan but also progressing the new plan and putting in set timescales for the implementation of the terms of that new plan.

Thomas Byrne (Meath East, Fianna Fail)
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I thank the Deputy for providing the opportunity to discuss this matter and I acknowledge his work in the area. Many rare conditions are complex and the impact for patients living with them can be severe.

At times, it can be difficult to access appropriate medications and technology. The development of a successor to the previous national rare diseases plan is a key priority for the Department to ensure that there is a plan in place to drive improvements for rare disease patients and their families. As part of the development of the plan, a patient form will be established to ensure that voices of patients and families will be central to the development of policy. A steering group is being established to progress the development of the plan and will meet for the first time next week.

I once again thank the Deputy for raising this important issue and I assure him that the Government is fully committed to doing everything possible to help people living with a rare disease.

Colm Burke (Cork North Central, Fine Gael)
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Could I just ask the Minister of State about the issue I raised about a liaison person to be appointed? That would take out a lot of the uncertainty within the Department on that.