Ms Eibhlin Mulroe:

I am from the Irish Platform for Patients' Organisations, Science and Industry, IPPOSI. I wish to revert to the comment made by Deputy Sandra McLellan on cystic fibrosis and clinical trials. The product ivacaftor was used in clinical trials but is now on the market. Patients in Ireland had access to the clinical trials. This goes back to the comments made by Senator John Crown in the context of registries. We are in a position in Ireland to attract research on cancer and cystic fibrosis because we have a registry of patients. We have information on patients, both clinical and personal information, that is necessary to access trials. Registries are important in the context of research. I revert to the point made by Senator John Crown on specialist care and he is correct. The European Organisation for Rare Diseases, EURORDIS, recently conducted a survey of patients in Europe who were experiencing difficulty. A total of 2,000 patients responded to the survey, of whom 90 were Irish. They had the highest rate of issues in respect of access to specialist care. The issues did not pertain to access to treatment or drugs but access to specialist care. We have an issue in Ireland in that context.

I am thinking of people who have rare conditions, that is, the people I know and with whom I interact, as do Ms Avril Daly and Mr. Tony Heffernan. It is really difficult when one lives in a country in which one does not have health information and one does not have unique identifiers within the hospital system. A person with a rare condition who visits a hospital but then has to vist another the following week because he or she happens to be elsewhere is in trouble. While this is not a rare disease issue, the health information Bill is really important. Its implementation is even more important, that is, the adoption of unique identifiers to ensure all citizens will have their health information collected somewhere. While this is probably a discussion for another day, in the context of rare conditions, it matters.

Another issue that is missing is that of health economics in respect of orphan drugs, of which there are only 72 under the orphan drugs regulations. There are not that many of them, as treatments are not available for every rare condition. There are only a few treatments and where they are available, particularly ultra-orphans where one is talking about perhaps one in 50,000 people, such treatments costs are high. There are fewer people with such conditions, but the cost of developing the drug is the same. In these situations the system of assessment of these drugs is the same as for any other drug. It happens within the National Centre for Pharmacoeconomics and I acknowledge its process is transparent. We can see it and what happened with the health technology assessment, HTA, analysis. The problem is with what happens after this, namely, the decision. What was really good about the cystic fibrosis case was that the people involved communicated with the patient organisation. However, as has been seen within the past six months, a patient who is one of only two or three in the country has nowhere to go to talk to those who make the decisions. The people sitting around this table from both the Department of Health and the HSE have been working with us on how to involve the patient within that process because what then happens is such patients go public and to the media. While they are entitled to do this, we should have a process in place as this issue will come up repeatedly in a country in which budgets matter.