Rónán Mullen (Independent) | Oireachtas source

I, too, thank the witnesses for their contributions. I wish to follow up on Senator Colm Burke's remarks. It appears that there is evidence of some type of scandal, moral or otherwise, here. We have the ironic situation that drugs that are sometimes manufactured here are available to people in other EU countries, and reimbursed by their governments, but they are not available to patients in Ireland. Can the witnesses explain what is at the root of the differentials? Ireland ranks extremely low compared with Germany, as Senator Colm Burke mentioned, France, England, Italy and Spain when one compares the access to orphan medical products. The figures show that 53 of the 148 licensed products are routinely available in Ireland. That is approximately one third. Vastly more of such treatments are available in Germany. Noting the irony that some of these drugs might well be manufactured in Ireland, what is the fundamental reason for that?

To move on from that, there is an EU regulation on orphan medicinal products. Is there a need to move to a European standard or a harmonisation where we accept drugs that are accepted, made available and funded in one member state for people who are in the very disadvantageous situation of having a rare disease? It is therefore much more difficult to do the types of tests for cost effectiveness involving these people.

Should we be moving towards a kind of harmonisation and an acknowledgement that if a medicine is available in other states, it ought to be available here? Through the State-funded National Treatment Purchase Fund, people are able to access treatments unavailable in Ireland. It should follow that if licensed, proven medical products are available to people in other European countries, they should be available here.

The failure to progress aspects of the national rare disease plan is at the root of this issue. The plan dates back to 2014 and envisages a pathway for the assessment of the orphan medicinal products. What is the reason for the delay? Could the witnesses give a timeline for the activation of relevant sections of the plan in order that this problem can be tackled?

It is not just a matter of fewer products being approved. There is also a much greater delay for people to access new orphan therapies. It takes 50% longer than for traditional medicines. That adds to the unfairness faced by those suffering from rare diseases who, in many cases, will have a very short lifespan.