Cystic fibrosis drug may be made available to patients

A new €160,000 drug that cystic fibrosis campaigners have described as a “game changer” is likely to be made available to those patients who will benefit from it, a key medical expert has said.

Prof Michael Barry, head of the National Centre for Pharmacoeconomics and an expert on the cost effectiveness of medicines, said there was no doubt that the new drug, known as Orkambi , worked for some patients.

However, he said the overall result of the clinical trial of the drug was probably less successful than the manufacturer and cystic fibrosis patient groups would have hoped.

He said the result of a breathing test of patients who had received the product was close to the point where, in the manufacturer’s own definition, it was considered to be “minimally effective”.

Prof Barry said 50 per cent of those receiving the new drug would do well, but others would do not so well.

Speaking on RTÉ Radio One's This Week programme, Prof Barry said the devil is in the detail when it comes to Orkambi.

However, he said the Health Service Executive (HSE) had a good history of making drugs available to patients who would benefit from them.

He said that he believed that people who would respond to the drug would get access to it.

Prof Barry said the price of Orkambi was about €160,000 per patient per year.

The main issue was the “opportunity cost” of this price, he said.

HSE plan

The HSE’s service plan for 2016 provided for new initiatives and developments worth about €90 million to be put in place in hospitals, general practice, mental health and cancer care, Prof Barry said.

He said that, if everyone with cystic fibrosis was to get the new drug at the current price, the cost would be about the same as the €90 million being made available for all the other new initiatives across the health service, he said.

Prof Barry said the National Centre for Pharmacoeconomics had had an initial look at the product and had recommended that it should go forward for a full assessment.

He said the centre was now awaiting the submission of a dossier from the drugs manufacturer, Vertex Pharmaceuticals, outlining the case for the State to reimburse the cost of the drug.