Robert Troy (Longford-Westmeath, Fianna Fail)
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I thank the Ceann Comhairle for selecting this important topic today and I acknowledge the Minister's flexibility when I contacted his office earlier regarding this matter. I raise this issue because of a young lady from my constituency whom I know well. Back in 2011, she began to feel ill and to feel a numbness in her legs. Over the past number of years, she has gone through umpteen tests and examinations and many different diagnoses. Ultimately, she was diagnosed earlier this year with Degos disease. It is hard to believe that only a few short years ago this young woman ran the mini-marathon, and now she cannot walk without the aid of crutches. Her husband has had to give up work to act as a full-time carer. The family is more than complimentary of the level of care her consultant neurologist in St. James's Hospital, who has written requesting that the use of this drug be sanctioned in this woman's treatment, has provided. I will read some of the reply the consultant received from St. James's Hospital:

This is a rare life threatening disease and your patient has already suffered significant disability as a result of the disease process. Without effective treatment the likelihood is that your patient will have further central nervous system manifestation of disease such as minor, sub-massive or massive stroke, or may well develop bowel perforation, given that she appears to have endothelial manifestations on the serosal surface of her bowel. Either of these two complications would ultimately lead to her death.

It goes on to state:

[a]s you have reasonably identified, the manufacturer is not prepared to give you any information about "off-licence" use, or of any adverse effects that they may be aware of in this regard. Furthermore, the cost for this drug is prohibitive and would lead to significant adverse effect on the ability of St. James's Hospital to deliver care to very many other patients who are also the responsibility of the Hospital.

I speak quite sincerely to the Minister today. I believe he is a compassionate man. That is obvious from the career he chose in becoming a medical doctor. I have no doubt that he chose that career because he wanted to help people and save lives. Now that he is in an ideal position to help people and save lives, I ask him quite sincerely and genuinely to look at this case compassionately, with a view to sanctioning the care. When a consultant is making a recommendation for a treatment that has the potential to save the life of a lady who is only in her late 40s, we should make the necessary provisions available to try to save this young mother's life.

Leo Varadkar (Dublin West, Fine Gael)
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I thank the Deputy for raising this issue. The Deputy will appreciate that I am not in a position to comment on the case of any individual patient, even where some personal details are already in the public domain. I have no access to patient records and am not permitted, by law, to make decisions about individual cases.

Soliris, or eculizumab, is an ultra-orphan medicine manufactured by Alexion Pharma and licensed for the treatment of two rare blood disorders. The drug is not licensed for the treatment of Degos disease in Ireland, nor is it licensed by either the European Medicines Agency or the US Food and Drug Administration. The licensing of pharmaceutical medicines is a matter for the Health Products Regulatory Authority, formerly the Irish Medicines Board, in Ireland and for the European Medicines Agency in the EU. It is not in my power as Minister for Health to license any pharmaceutical product. It is appropriate that such matters are dealt with by expert and impartial authorities established for this purpose.

Decisions to use unlicensed medicines are made by health care professionals and their patients through evaluating the efficacy and safety of the medicine and its potential to achieve better patient outcomes. In situations where an individual hospital is asked to approve or support the use of an unlicensed drug, the hospital will rely on professional evaluation of evidence supplied by the treating clinician before making a decision based on that evaluation. In any such evaluation, the health care professionals considering the matter may not support funding the prescription of a particular drug.

Where medicines are used for an unlicensed purpose, this is ideally done as part of a clinical trial. As Minister, I have no role in setting up clinical trials or selecting patients to go on them, and nor should I. In relation specifically to Soliris, in early 2015 the HSE decided to fund the provision of this drug for sufferers of two specific conditions for which it is licensed. At an individual cost of over €400,000 per patient per year, this drug is expected to cost the taxpayer approximately €8 million in 2016. Given these substantial costs, the director general of the HSE has put in place formal procedures to ensure that each case in which Soliris is used is the subject of advance authorisation. These arrangements would not permit the drug to be used for the treatment of a condition beyond the terms of the marketing authorisation and where evidence of clinical benefit has not been demonstrated.

It is open to a drug's manufacturer at any time to submit an application to the European Medicines Agency to have a product licensed for use for a specific indication. Once that drug is approved by the European Medicines Agency, the manufacturer can, if it wishes, submit an application for pricing and reimbursement to the HSE. I hope this clarifies the matter for the Deputy.

Robert Troy (Longford-Westmeath, Fianna Fail)
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The Minister knows far better than I that this is an extremely rare disease. I believe there are only 40 or 50 sufferers worldwide and this lady is the only one in Ireland. The Minister said: "[d]ecisions to use unlicensed medicines are made by health care professionals and their patients through evaluating the efficacy and safety of the medicine and its potential to achieve better patient outcomes." The person who is treating this lady is recommending the use of this drug. The patient herself is willing to have this drug tested on her, because I understand six other people in the world have been treated by this. Three of the six are fully recovered, in two cases the disease has been left dormant, and in the final case the delay in administering the drug has resulted in death. My office contacted Alexium, the drug company, which has a base here in Dublin, and it advised of a compassionate drugs access scheme. I would love the HSE to accede to the request by consultant neurologist Dr. Siobhan Hutchinson and make the drug available. Failing that, at a minimum, can the HSE and the Minister and his Department support to the drugs company to fund this under the compassionate drugs access scheme?

Leo Varadkar (Dublin West, Fine Gael)
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I am advised that eculizumab has been used to treat a small number of Degos patients in highly experimental trials in the US. The results of these trials have been inconclusive, with some patients showing signs of improvement, some getting worse, and one patient dying while receiving treatment. There is no strong clinical evidence or research to support the use of this medicine for the treatment of Degos disease. It is not my decision as Minister to license or authorise medicines and under the HSE governance Act I am barred from making any directions in respect of an individual patient. From what I have read, the case for using this medicine seems very weak. If that changes or if new evidence emerges, it can be reconsidered by the relevant authorities.

I am not familiar with the compassionate access scheme. I will certainly undertake to examine how it works, but I imagine it would have to be based on some evidence that it would be effective in some way. I hear what the Deputy is saying about compassion. One thing we always have to consider in health care is opportunity cost and use of resources. Even in the richest country in the world, resources are limited and if one has €400,000, does one spend it on a drug for whose efficacy there is no evidence or on ambulance or maternity services? We know spending more money on things like ambulance and maternity services would definitely save lives.