I acknowledge the families and people who live with rare diseases for their advocacy and their resilience. I am not going to go into individual diseases. It is welcome news, though, that givinostat has been approved. I pay particular tribute to the current Minister for Health, who has really taken on the issue around givinostat and is also looking at the reform of the accessibility for orphan drugs for people. I also pay tribute to Deputy Pádraig O'Sullivan, who has been at this for a number of years. It is sometimes a demoralising task. I commend also Senator Anne Rabbitte, and Senator Teresa Costello on her unstinting support for families who are living with boys with Duchenne dystrophy. I will just make a couple of points. We have to factor everything into these rare diseases. They are often chronic, progressive, debilitating diseases. We need to factor in the cost of care without the early intervention of these drugs. That is not to give an open chequebook to pharmaceutical firms that produce orphan drugs. While we are grateful that they take on the research and development of orphan drugs, by their nature they are exclusive to that condition, so they have a closed market and considerable leverage over states in terms of the cost of those drugs. Some of those drugs that are designated orphan drugs achieve wider applications and become very profitable for those companies, outside the rare disease piece. I think of drugs like rituximab and Avastin, some of which have applications in inflammatory diseases beyond the rare diseases, such as inflammatory arthritis and in the area of treatment of cancers. That needs to be balanced. We also need to understand that it cannot always be that the State has to just open the coffers. We have to balance it. There is a defined budget and we have to balance the needs of other people in the system. That is not taking away from the need to have a proper, efficient system of evaluation and bringing these drugs to patients who need them. As a small country, we lack the heft and leverage with these companies. We should be operating through the European Union to try to have better negotiating leverage. We need better-managed access to these drugs and early access schemes. We are not engaging properly in clinical trials, for a country that hosts so much pharma industry. That is a recurring theme. I would like to think that a ring-fenced 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. At the end of this, I want to remember the people who are suffering from these diseases and the families who live with them. We cannot imagine unless we walk in their shoes.

Sentiment score: 0.12