Colm Burke (Cork North Central, Fine Gael) | Oireachtas source

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.

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