Martin Daly urges faster access to expensive drugs for rare diseases

Thank you very much for coming in this morning. Look, this is a difficult subject. We're trying to balance the cost-effectiveness of drugs against true and real-life human suffering, which is extremely depressing. And I suppose 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. And I suppose that is probably the key to it is there's a sense that this takes an inordinate length of time from the time of submission to the time of reimbursement. And there are a number of issues I suppose I'd like you maybe to comment on would be, one, the lack of early access programs in this country. That would be number one. I'd like your views on that. Also, two, we, for, I suppose we pride ourselves on being a wealthy country and being a sophisticated country in terms of health matters, but we are a laggard when it comes to clinical trials. We have a very poor clinical trial environment in this country. And that is one way internationally that people who require access to experimental drugs or drugs even that are in the process of being evaluated nationally would be through clinical trials. And then there are some drugs which I just, I'll ask you specifically about. We've had presentations from the muscular dystrophy Ireland in relation to Jovinistat. And then there are two other, there's one issue, like, for example, I can't understand how this works. I have someone who is a 54-year-old man who has severe advanced progressive osteoporosis. His life has been changed in the last four years, multiple fractures. He's gone from being a high-performing executive to someone who is disabled. And he can't get access to decenumab, prolia, because it's licensed this country for females only and osteoporosis. I can't get an answer after six months as to why a woman his age can get access to prolia. That's the trade name, decenumab, and he can't. And his condition is progressive and it is severely disabling. And I'd just like to ask specifically about, I have a young woman, mother of three, who develop a desmoid tumour during COVID, a little bit of delay in diagnosis because of COVID, but no one's fault in that, but Naira Gestat has been approved by the EMA, and it is available to people in France on compassionate grounds. She's a young mother, it's inoperable, she's tried chemotherapeutic options. And I suppose with three young children, is the cost-benefit analysis, does it include not just her life, but the life of her children and their quality of life because they're likely to lose their mother in the next few years if she doesn't get access. Non-operable tumour, she'll lose her whole hind quarter, and that simply, she would not survive surgery. Thank you. There were a number of points there. In relation to the submission of applications, the reality is that why are we lagging behind in terms of applications? And up to 40% of our rare disease drugs, there is no application for pricing reimbursement. And that has to come down to, in large part, finance. So companies will launch and apply for reimbursement in larger countries. Germany with 80 million, France with 70 almost, Italy with 60, and Spain with 50 million population. It's about money, to be fair about it. Not all of it, but a large part of it. Early access, you mentioned, that's an interesting one. And we have, through the Office of the Chief Clinical Officer, started to look at, and we're in discussions, in relation to early access. I agree, there is a role for early access. It's a question of, the devil is into detail in a lot of this. How would it, what drugs would go into an early access scheme? Essentially, we're looking at cancer initially. You know, who pays? How would you identify the drugs? Who identifies the drugs? The devil is into detail, as I say. But I agree with you. Early access definitely needs to be looked at, and it's a fair point. And we are looking at it. In relation to clinical trials, we've had problems with clinical trials over many, many years. You'll know it yourself, and we tried to do clinical trials in the past, and it proved difficult to do. I think it's getting better, but it is a very important area of access. In relation to Gevinostat, the drug for Duchenne muscular dystrophy, we were asked to review that initially. It received a marketing authorisation on the 6th of May or June, 6th of June. We were asked to look at it in August. We completed our initial review in nine days. We've met with the company since, and we're awaiting their assessment of the full dossier. And we will make sure that that's done quickly. I would say that, for example, for fenfluramine, which was for Travitz, as you'll know, we did that HTA in 54 days. So it can be done quickly, and we would strive to do that. Dinesiumab, I don't know the answer to that. I'm sure it's probably because of the application that was put into the HTA, but we can check that out for you. And the last drug, what was the last drug? Nyrogestat, gestat for desmoid tumours. Yeah, we haven't seen that at all, Martin, so I find it hard to comment on it. Okay, no, no, that's fine. Thank you. We'll check it. Thanks very much. I suppose just I have a minute left. This balancing exercise, I think, you know, between cost-effectiveness and benefit and the balance between the cost that's taken out of the rest of the health service. This is cost-effectiveness and benefit and the balance between the cost that's taken out of the rest of the health service. This is a huge challenge. It is, and that's exactly why we do this stuff, because the so-called opportunity cost. Because if you're going to spend millions of euros, and that's what we're talking about, in one area, then it's not available for somewhere else. And you know the challenges as well as I do in relation to many aspects across the health service. And so what we're trying to do is to make the best use of all the resources we can. So, for example, with the $142 million that won't be paid in relation to orphan drugs this year, that's available for somewhere else. It's not for me to decide where it should go, but that's the concept. You're absolutely right. It's the whole concept of opportunity cost. You could, if you want to, pay up front straight away, pay at the asking price, but there's a price to be paid for that. Do you think that some of the drug manufacturers have the public purse in some way by the throat? There is no relationship, very little relationship, between the price of a drug and the health outcomes that it delivers, particularly in the area of cancer and rare diseases. Thank you very much. That's very...