John Brassil (Kerry, Fianna Fail)
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I thank the witnesses for their presentation. I refer where we are as a country with regard to orphan drugs. The European Medicines Agency has designated 148 medicines as orphan drugs for rare diseases. Currently we reimburse 53 of those or 35%. The UK reimburses 68, Spain reimburses 75, Italy reimburses 84, France reimburses 116 and Germany reimburses 133. We are at 35%. Germany is best at 90%. Spain, a country whose economy is not going as well as ours, is at 50%. We are at the lower end for reimbursement for orphan drugs and drugs for rare diseases. The NCPE is aware of this. We have been fighting individual battles, over Respreeza for alpha-1 proteinase inhibitor deficiency, as Deputy Kelly mentioned, and Orkambi for CF. It seems that if the lobby is strong enough, or given the unfortunate and tragic deaths of two patients, the HSE takes notice.

As a result of my work as spokesperson on primary care I have tried to modify the Bill. This committee has stated that it needs to be modified. It is being resisted at all ends. That is the context of our dealings with the NCPE and the HSE in trying to establish a better model to reimburse more drugs and to give patients with rare diseases, who are as deserving of proper care as anyone else, some chance of either dealing with their symptoms or in some cases dramatically changing their lives.

The rare disease technical review committee was referenced. That is a welcome addition. The key point, which the witnesses have recognised, is that this group is reviewing the decision, not making it. Because it is reviewing a decision based on a flawed process, it will find it difficult to come up with a different decision. The 2013 Act needs to be amended. That is my focus and it should be that of the witnesses as well. Unless we amend the process by which these drugs are reviewed, we will be fighting an uphill battle.

Dr. Mitchell made reference to Sweden. Sweden's model is a based on quality-adjusted life year, QALYs, but it does not have an upper limit. The Swedish model allows for situations where maybe half a dozen patients are suffering from one condition, where one could not possibly get the type of data that is needed and meet the threshold of 45,000. Interestingly, in 2015, the proportion of health expenditure on medical goods was greater in Ireland than it was in Sweden.

Deputy Donnelly raised the issue of biosimilars. The IPU has identified a potential saving of €307 million on biosimilars alone over three years. I have met children suffering from spinal muscular atrophy. A drug called Spinraza has revolutionised treatment. Its total cost to the system would be approximately €9 million. Multiples of that cost are available.

We need to amend the Act to give some hope of orphan drugs being reviewed. If we do not do so, the companies will just stop applying, as Ms McGrath pointed out. They know the answer will be "No", so why would they waste time and resources applying for something when they are going to be refused? That will have a knock-on effect.

I met officials from Vertex, the company that makes Orkambi, recently. After all the battling, arguments, protests, marches and so on, that drug was reimbursed, including future iterations. I believe the next Orkambi, whatever it is called, is coming on-stream shortly without any debate. Vertex told me it is looking for other countries to consider reimbursement in the way the agreement was made in Ireland.