Paul Daly (Fianna Fail)
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I appreciate the attendance of the Minister of State, Deputy Helen McEntee, to answer my queries. The issue has been raised previously through a Dáil parliamentary question. The person in question, Charlotte Connolly from Mullingar, has been diagnosed with a rare blood disorder known as Degos.She is the only person in Ireland who has been diagnosed with the disease. There is a drug available, Soliris, which is produced by Alexion Pharma and which has been used in similar cases in America with a high degree of success. The Connolly family requests that she be gain access to this drug. In previous answers to parliamentary questions in the Dáil on this matter it was stated the drug was unlicensed for the treatment of Degos disease and that it was exorbitantly expensive. When Alexion Pharma was approached, it suggested she could gain access to the drug under the compassionate drug access scheme. When this issue was raised in the Dáil by Deputy Robert Troy, the then Minister for Health, Deputy Leo Varadkar, said he was unaware of such a scheme. Has the new Minister updated himself on it? Will he inform me of its availability and how it could be used? The drug is unlicensed for the treatment of Degos disease, but it has been recommended for use by the consultant neurologist at St. James's Hospital. Will the Minister progress the issue in a positive manner?

With this matter on my mind this morning, I was taken aback emotionally when entering Leinster House. Last week I could not get in because there were many people outside protesting and lobbying on behalf of the Irish hare. This morning there was no one outside for Charlotte Connolly. She is a human being and the eventual outcome of her condition is death. We have many discussions about various issues in the House. Yesterday I felt more comfortable in my role as agriculture spokesperson discussing the issue of farm safety and the potential saving of lives by taking action and spending money. Today I can actually identify an individual human being whose life could be saved by taking action. I hope I will not receive the copy-and-paste response received previously. Declan Connolly, Charlotte's husband, has written to the Minister since his appointment. He received an acknowledgement of just one letter and no response to the second. If it is not possible for the Minister to give me the answer that I would so love to hear this morning, could he, at least, meet the Connollys to progress the issue further down the line to come up with some solution?

Helen McEntee (Meath East, Fine Gael)
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I thank the Senator for raising this issue. I am taking this matter on behalf of the Minister for Health, Deputy Simon Harris, who sends his apologies for not being able to attend. I understand the type of issue the Senator is dealing with, as I have dealt with a similar one in my constituency. I know how difficult it is for the individual affected and their family. The Senator will appreciate, however, that I am not in a position to comment on the case of any individual patient, even when some personal details are already in the public domain.

The HSE, as the principal provider and budget holder in respect of public health services, is obliged to use the resources available in the most beneficial, effective and efficient manner to improve, promote and protect the health and welfare of the public. This requires it to make difficult decisions to ensure the finite resources at its disposal are used to best effect. Soliris 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 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 in Ireland and the European Medicines Agency in the European Union. It is not in the power of the Minister for Health to licence any pharmaceutical product, regardless of whether it has already been approved for use within the European Union. It is appropriate that such matters are dealt with by expert and impartial authorities established for this purpose.

In early 2015 the HSE decided to fund the provision of Soliris for sufferers of two specific conditions for which it had been licensed. At an individual cost of over €400,000 per patient per year, the drug is expected to cost the HSE approximately €8 million in 2016. Given these substantial costs, the director general of the HSE has put in place formal procedures to ensure each case in which Soliris is used is the subject of clear 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 the drug is approved by the European Medicines Agency, the manufacturer can, if it wishes, submit an application for pricing and reimbursement to the HSE.

It should be noted that there is no provision in legislation for the approval of compassionate access programmes for specific groups of patients with an unmet medical need. I trust the Senator will appreciate the position in this matter. Perhaps such a programme might be looked at further down the line.

I will raise with the Minister the fact that the Connolly family has written to him twice and ask him to respond.

Paul Daly (Fianna Fail)
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I note the response which is gut-wrenching. We are talking about a human life, but this is a copy-and-paste answer. I respectfully request the Minister to commit to meeting the Connolly family in person and explore avenues, be it the introduction of a compassionate drugs access scheme or otherwise. The family needs real, not copy-and-paste, answers. It is a family life that is on the line. I am emotional in addressing the issue. As a county councillor, I get great satisfaction in driving past a signpost on a road where I know I played a part in having it put there. I cannot even imagine what it would be like to help to save human life. Copy-and-paste answers do not wash. I respectfully request the Minister to meet the family in question to explore other avenues, if the drug in question is unlicensed and there is no compassionate drugs scheme in place. At least we should explore other avenues and give people some hope.

Helen McEntee (Meath East, Fine Gael)
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I take on board what the Senator said. I understand where he is coming from, as I have dealt with a similar matter. I cannot speak on behalf of the Minister, but I will bring it to his attention personally and ask him to come back to the Senator.