Martin Daly: Balancing access and costs for orphan drugs
Martin Daly addresses the Dail on access to treatments for rare diseases, welcoming the approval of Givinostat while warning about the rising costs and market power of orphan drugs. He calls for better managed access, EU bargaining leverage, more clinical trials and consideration of a ring-fenced fund for rare disease treatment.
Martin Daly opens by paying tribute to families living with rare diseases and to advocates and parliamentarians who have campaigned for better access. He welcomes the recent approval of Givinostat and applauds the Minister for Health and other deputies for their work on orphan drug reform.
Daly emphasises that many rare diseases are chronic, progressive and costly to manage, and that early drug intervention must be weighed against long-term care costs. He warns that orphan drugs often sit in a closed market with significant pricing leverage, and some later find wider, lucrative indications outside rare disease use.
He stresses that the state cannot simply open the coffers without balancing other health needs and calls for an efficient evaluation system. As a small country, Daly argues, Ireland needs greater negotiating strength through the European Union to secure fairer prices and better access terms.
Daly urges better managed access schemes and greater engagement in clinical trials, and suggests exploring a ring-fenced fund, potentially resourced from pharma tax revenues, to support treatment of rare diseases. He closes by asking colleagues to remember families living with these conditions and the human reality behind policy choices.
Acknowledging families and recent approvals
Martin Daly opens by paying tribute to families living with rare diseases and to advocates and parliamentarians who have campaigned for better access. He welcomes the recent approval of Givinostat and applauds the Minister for Health and other deputies for their work on orphan drug reform.
Cost, market dynamics and evaluation
Daly emphasises that many rare diseases are chronic, progressive and costly to manage, and that early drug intervention must be weighed against long-term care costs. He warns that orphan drugs often sit in a closed market with significant pricing leverage, and some later find wider, lucrative indications outside rare disease use.
Budget constraints and negotiating strategy
He stresses that the state cannot simply open the coffers without balancing other health needs and calls for an efficient evaluation system. As a small country, Daly argues, Ireland needs greater negotiating strength through the European Union to secure fairer prices and better access terms.
Proposals for access and funding
Daly urges better managed access schemes and greater engagement in clinical trials, and suggests exploring a ring-fenced fund, potentially resourced from pharma tax revenues, to support treatment of rare diseases. He closes by asking colleagues to remember families living with these conditions and the human reality behind policy choices.
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Transcript
First I just, Minister, first I just want to acknowledge the families who live with rare diseases and the people who live with rare diseases for their advocacy, their resilience and their advocacy for their conditions. And again, I'm not going to go into individual diseases. It is welcome news, though, that Ginna Stath has been approved. And I want to pay particular tribute to the current Minister for Health, who has really taken on the issue around Ginna Stath and is also looking at the reform of the accessibility for orphan drugs for people. And also pay tribute to Deputy Porica Sullivan, who has been at this for a number of years, and it is sometimes a demoralising task. Senator Anne Rabbitt also and Senator Teresa Costolo on her unstinting support for families who are living with boys with Duchenne's dystrophy. Look, I just want to make a couple of points. One, we've got to factor in everything into these rare diseases. They're often chronic, progressive, debilitating diseases and we need to factor in the cost of care without early intervention of these drugs. And that's not to give an open checkbook to pharmaceutical firms who produce orphan drugs, because whilst we are grateful that they take on the research and development of orphan drugs by their nature, they are exclusive to that condition. And so they have a closed market and considerable leverage over states in terms of the cost of those drugs. And some of those drugs that are designated orphan drugs often achieve wider applications and become very, very profitable for those companies outside the rare disease piece. I think of drugs like Rituximab and also Avastin, who had applications in inflammatory diseases beyond the rare diseases such as inflammatory arthritis and in the area of treatment of cancers. So that needs to be balanced. We also need to understand that it can't always just be about the state has to just open the coffers and say, we have to balance it. There is a defined budget and it can't be, we have to balance the needs of other people in the system. And that's not taking away from the need to have a proper efficient system of evaluation and bringing these drugs to patients who need them. And that means that I think as a small country, we lack the heft and leverage with these companies, and we should be operating through the European Union to try and have better negotiating leverage. We need better managed access to these drugs, early access schemes. We are not engaging properly in clinical trials for a country that hosts so much pharma industry here, and that is a recurring theme. And I would like to think that perhaps a fund, a ring fence fund could be achieved out of the tax revenues from some of the pharma companies that would be designated for the treatment of rare diseases. So I just want to, at the end of this, remember the families who are living, the people who are suffering from these diseases and the families who live with them, because we cannot imagine unless we walk in their shoes. Thank you.