I welcome the Minister, Deputy Leo Varadkar. I understand Senator Thomas Byrne is sharing time with Senator John Whelan.

I thank the Minister for coming to the Seanad and wish him the best. I will speak about a young man, Mr. John Duggan-----

I ask the Senator to refrain from naming people in the House.

The gentleman in question is here in the first seat beside the door on the front row of the Visitors Gallery. He is a young man from Bellewstown, County Meath and deserves the chance of a normal life; indeed, the chance of life. He was diagnosed with paroxysmal nocturnal haemoglobinuria, PHN, in 2010. It is an extremely rare blood disorder which is characterised by the breakdown of red blood cells, as I am sure the Minister will know only too well being a doctor. I understand about one third of patients with PHN die within five years. In John's case, PHN is resulting in him having blood transfusions every six weeks or so. He is lethargic and greatly fears blood clots. He describes his prognosis in his own words:

I am getting worse and worse. There is every chance I will have a heart attack or kidney failure. A blood clot is definitely coming and this is not a time for messing around.

There is a treatment available for PHN, as the Minister will know. It is an expensive treatment - I acknowledge it is expensive - called Soliris. The chemical name is eculizumab which has been shown to normalise life expectancy for those receiving it. As I understand it - this is part of the crux of the matter - ten patients in Ireland have already been prescribed Soliris, funded by the HSE, and I understand from contacts Mr. Duggan has had with some of them that it is working very well. Mr. Duggan and one other patient of whom I am aware - I am sure it is a person of whom Senator John Whelan is aware - have been refused it.

This strikes me as fundamentally wrong, unethical and unfair. How can the State arbitrarily decide that Mr. Duggan is different from those already being given the medicine by it? How are these decisions made?

May I quote Mr. Duggan's GP, Dr. Oliver Lynn?

I ask the Senator to refrain from naming people in the House because they are not here to defend themselves.

His name has already been mentioned. He said he would urge the HSE to consider providing for this young man the correct treatment before he reached catastrophic thrombosis. I will not name his consultant haematologists but will quote them. His consultant said that, given the overwhelming evidence that eculizumab was the only effective therapy for PHN and that the HSE already funded eculizumab therapy for ten patients, they believed there was a very strong moral and ethical argument the HSE should agree to fund eculizumab. I ask the Minister, his officials, the relevant body and the HSE to please reconsider the decision. Please give this man and the other person whom I know is affected the chance of life. Please do not tell him and his wife, Aileen, that there is no hope for them.

I am particularly heartened to see the Minister, Deputy Leo Varadkar. I am grateful to him for taking these questions from me and Senator Thomas Byrne. It is not unfair to say the Minister is regarded as one of the most forthright, upstanding and decent politicians of all time. His reputation and credibility precede him. I am not saying this, in any way, as a charm offensive, but I know that he is a straight-talker who will do anything that can, and should, be done. In that regard, we appeal for fairness, natural justice and basic equity of medical provision for the handful of people in the country who have been diagnosed with the rare blood disorder PNH, including Ms Mary Gorman from Ballinakill in my neighbourhood.

I have already explained the position to Senator Thomas Byrne. I ask the Senator to refrain from naming people in the House.

I respect that but Ms Gorman has travelled here today from County Laois to be with us. She has gone public having suffered in silence for the past two years in what has turned out to be the vain hope the HSE would address her medication and treatment, as recommended by her consultant team and haematology team at St. James's Hospital, the reputation of which is second-to-none in the world, as is the work being carried out there. It does not lightly prescribe and recommend this treatment which first became available to ten patients in this country, as Senator Thomas Byrne said, as far back as 2010. It is proving to be technically excellent and a life-saving intervention. There is no other way to put it. It is beyond me how we can continue to stand over making fish of one and flesh of another. There is no equity to it. Notwithstanding the moral bankruptcy of the exorbitant fees the pharmaceutical company which manufactures Soliris charges, the State has to intervene and make the best possible treatment available to Ms Mary Gorman and the handful of people who have this rare condition. We appeal to the Minister. One word from him to the HSE could resolve this issue. That is what we appeal for.

I thank the Senators for raising this important issue. As they will know, I cannot comment on individual cases and make no decisions on individual patients. As there are no patients' charts on my desk, my remarks relate to the issue at hand, not any individual patient.

The HSE has statutory responsibility for the decisions on pricing and reimbursement of medicinal products under the community drugs scheme in accordance with the provisions of the Health (Pricing and Supply of Medical Goods) Act 2013. This requires consideration of a range of statutory criteria to reimbursing any medicine, including clinical need, effectiveness, costs and the resources available to the HSE. The decisions on which medicines are reimbursed by the taxpayer are made on objective, scientific and economic grounds by the HSE on the advice of the National Centre for Pharmacoeconomics in St. James's Hospital. They are not political or ministerial decisions, nor should they be.

The drug eculizumab, or Soliris to give it its brand name, is indicated for the treatment of patients with paroxysmal nocturnal haemoglobinuria. It is one of the most expensive drugs in the world.

I understand that in 2010 the HSE entered into an interim access with evidence development agreement, a sort of trial, between the company Alexion Pharma and St. James's Hospital to treat ten patients with this drug. The interim agreement was put in place with the expectation that evidence would emerge which would assist with the future decision on making this drug available.

The HSE received an application for the inclusion of eculizumab in the general medical services, GMS, and community drugs schemes. In accordance with agreed procedures, the National Centre for Pharmacoeconomics conducted a pharmacoeconomic evaluation of eculizumab. I have that evaluation from October 2013. It concludes that there is evidence that the medicine is a treatment for adults and patients with paroxysmal nocturnal haemoglobinuriam, that it reduces transfusion requirements and that there is weaker evidence that it reduces the risk of thrombosis, renal failure and mortality. Furthermore, evidence of clinical benefit in the treatment of patients with PNH is limited to patients with a history of transfusions. It also concluded that the total cost per patient per year of €437,247 would have a cumulative gross budget impact over five years estimated at €33 million. In addition, the manufacturer did not include an economic model as part of its submission and failed to demonstrate the cost effectiveness of the therapy. Consequently, the National Centre for Pharmacoeconomics was unable to recommend reimbursement of the product under the community drugs scheme.

The HSE has been engaging with the company for some time to arrive at a price that would assist it in its desire to fund this medicine for as many patients as possible within available resources. I am informed that engagement with the company has been concluded and the HSE is considering the outcome of that engagement. It is regrettable that to date the company has not been able to provide this drug at a more sustainable price for the HSE to reflect the clinical evidence. Nonetheless, the door is not closed to a new medicine and it remains open to the company to come back with new evidence, a new price, or both.

The HSE and I fully understand the concerns of patients about the availability of this drug and every effort is being made to achieve a satisfactory outcome. While I appreciate that some may take the view that the taxpayer should reimburse every licensed medicine for whatever the price the drug company demands, the better interests of the health service, the entire body of patients in the country and the taxpayer require that we only reimburse the most effective medicines and only do so at a fair price.

A brief question, please.

I will make a brief comment. There are significant differences between what the Minister read and what is contained in his script. He completely changed the meaning of one sentence and read out additional information. I am not complaining to him, but if one only had the script-----

I think it may state "now", but it should read "not".

Yes. The Minister also read another paragraph which put a bit more meat on the bones. I appreciate that he does not deal with individual cases, but there are so few such cases that they must come under consideration. If he is looking for evidence of a history of transfusions in a case, that is certainly the position in the case of the person about whom I spoke. The Minister stated the HSE was considering the outcome of the engagement. Does that mean that people should be awaiting a decision or has a decision been made? That is my follow-up question. I appeal to the Minister is the nicest possible way because I do not want to be political, critical or anything like that on an issue such as this that affects people's lives. I ask him to please look at it. There is a fundamental unfairness in that ten people have been given this drug. I do not know the terms of the agreement with the company for them, but they have been given the drug. They have the chance of life. I ask the Minister to, please, give it to the others who badly need it.

I thank the Minister for his reply. I was glad to hear him say the door was not closed. However, time is of the essence. The conduct of the pharmaceutical company is despicable in this regard. I urge the Minister to use his good offices to ask the HSE to engage with urgency on this and come to a positive conclusion with urgency. People's lives are at risk and we must help them if we can.

The HSE national drugs committee and the HSE as a body corporate are always open to pharmaceutical companies to make new or revised applications. Members may be interested to know that Alexion as a company has a revenue of €1.15 billion, had profits of €253 million and equity of €2.4 billion. Its chief executive officer is paid €12 million a year. In Belgium in 2011 government and opposition politicians alike alleged that the company was guilty of moral blackmail when it was discovered by De Standaard, a newspaper in that country, that it had hired a PR company to help a nine-year old child who had been denied the drug by the Belgian authorities.

The parents of the boy believed they were being helped by a patients' organisation and were not told that a PR company was behind it. That is the kind of thing we are dealing with in these cases.

There is no PR company in this case - none whatsoever.

There may not be, but there was in Belgium.

There is not.

I do not suggest there is in this case. This is a company that is very aggressive in the way it prices its medicines. The door is never closed to a new medicine. The company can present new evidence, a better price, or both. I call on the Seanad to stand behind the taxpayer in the interests of patients as a whole and support the HSE national drugs committee in its efforts to ensure a fair price for this medicine. Even though I have no direct role in this matter and do not make decisions under legislation, I am, of course, happy to discuss the matter in the Seanad and there will be a similar question in the Dáil tomorrow. However, I suggest to Senators that the Joint Committee on Health and Children might call in representatives of Alexion to help in a way that might be most effective by putting pressure on the company to do right by taxpayers and patients.