Louise O'Reilly (Dublin Fingal, Sinn Fein) | Oireachtas source

I thank the witnesses for their evidence. I have a number of questions and I would imagine that none will shock or surprise the witnesses.

Following our engagement with Alpha One Foundation, specifically in respect of Respreeza, I have stated publicly that the families of the patients involved have been treated disgracefully. I would apportion blame equally to all involved in this. The patients and their families have been left in Limbo in a way that is actually quite cruel for them. They are in a situation whereby the drug they need is being provided on a compassionate basis. I do not think there is much compassion in global pharmaceuticals but that is the term that is used. On a compassionate basis that drug is available to them but they now do not have anyone to administer it, so they have the dual difficulty of the ticking time bomb, where compassionate access will run out and they know that but they also have a difficulty as to how it will be administered. That begs a broader question, which I will put to Mr. John Hennessy. In the situation that families or patients can by whatever means find the necessary funds to pay for the orphan drugs themselves, what is the involvement of the HSE regarding the administration of that drug? If a drug has been turned down for reimbursement for whatever reason, the HSE are making a statement that it will not have any involvement with it, but yet there are patients and their families who are really depending on these life saving and life altering drugs. What is the situation and could the witnesses comment specifically on the families that use Respreeza because I know they are going to be watching the committee proceedings? They have been let down on more than one occasion.

The broader questions are on the process of evaluation. Professor Barry alluded to this. The same process is used for orphan, ultra orphan and drugs such as Panadol. I am not a scientist but what strikes me is that there is a case to be made for orphan and ultra orphan drugs being assessed in a different way, the way they do in other countries and using a different assessment tool because clearly the numbers are not there. When we talk to families who are depending on these orphan drugs or the companies that are selling them, they will without exception tell us that the process that is used in this State is not appropriate for orphan drugs, or more specifically for ultra orphan drugs. The view of the witnesses on that issue would be very welcome.

Professor Barry alluded to the stakeholder involvement in the process. I have spoken to the stakeholders and it strikes me that they feel anything but involved. They feel very much excluded. The stakeholders are getting information from the media, information that is put into the public domain by the pharmaceutical companies. That is wholly unacceptable. Will Professor Barry provide more detail on his point of view as to how the stakeholders are accommodated? I do not purport to speak for them but I can repeat what they have said to me, which is that they feel anything but involved. They feel excluded.

With regard to Translarna and I know that my colleague, Senator Conway-Walsh will want to speak on this as well. Could Professor Barry outline briefly what is the difficulty with that drug in Ireland, if it is available in 22 other countries? When we discuss co-operation on an European level, there has to be some sort of standard. It strikes me as odd that a drug would meet the standards in one country but not in another, but a drug that meets the standard in 22 countries but does not meet our standard is strange. Will the witnesses outline that for us. I do not need to hear the areas where we have been leaders on this. We should focus on where we have been behind because we are clearly behind on this. I am aware that a High Court action is underway at present. I am sure that will give the witness the comfort of being able to say that he cannot say too much but I ask for an update on where that is at and when the outcome is going to be known. I know there may be a certain amount of estimation that has to go on because obviously it is the subject of proceedings. Will they give us an idea because families who are waiting are extremely anxious to know when they are likely to have an outcome. They are very disappointed that it had to come to this. The families concerned end up caught in the middle of this between the NCPE, the HSE on one side and the pharmaceutical companies on the other. There is a human story in the middle. For their sake the witnesses might be able to give us some idea when they see this matter coming to a conclusion.