Leo Varadkar (Dublin West, Fine Gael) | Oireachtas source

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.

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