Martin Daly (Roscommon-Galway, Fianna Fail)

I thank the witnesses for coming in this morning. This is a difficult subject. We are trying to balance the cost-effectiveness of drugs against true and real-life human suffering, which is extremely depressing. At the end of the day, what we want is a fair system that is timely and that really needs an overview to see how we can improve access to expensive drugs for rare conditions.

That is the key to it. There is a sense that this takes an inordinate length of time from submission to reimbursement. There are a number of issues I would like the witnesses to comment on. One would be the lack of early access programmes in this country. That would be number one and I would like the witnesses' views on that. Second, we pride ourselves on being a wealthy and sophisticated country with regard to health matters but we are a laggard when it comes to clinical trials. We have a very poor clinical trial environment in this country. That is one way, internationally, where people who require access to experimental drugs or even drugs that are in the process of being evaluated nationally do so - through clinical trials.

There are some drugs I will ask the witnesses about specifically. We have had presentations from Muscular Dystrophy Ireland with regard to givinostat. There is also another issue and I cannot understand how this works. I know of a 54-year-old man with severe advanced progressive osteoporosis. His life has changed in the last four years. He has had multiple fractures. He has gone from being a high-performing executive to someone who is disabled and he cannot get access to denosumab, or Prolia, because it is licensed in this country for females only and osteoporosis. I cannot get an answer, after six months, as to why a woman his age can get access to Prolia - that is the trade name for denosumab - and he cannot. His condition is progressive and it is severely disabling.

I would like to ask specifically about a young woman, a mother of three, who developed a desmoid tumour during Covid. There was a little bit of a delay in diagnosis because of Covid but no one was at fault for that. Nirogacestat has been approved by the EMA and is available to people in France on compassionate grounds. She is a young mother and it is inoperable. She has tried chemotherapeutic options. With three young children, does the cost-benefit analysis include not just her life but the lives of her children and their quality of life because they are likely to lose their mother in the next few years if she does not get access? It is a non-operable tumour. She would lose her whole hind quarter and she would simply not survive surgery.