Cystic Fibrosis drug Orkambi rejected as not cost effective

A new €160,000 drug that cystic fibrosis (CF) campaigners have described as a “game-changer” has been rejected for Irish patients on cost effectiveness grounds.

Cystic Fibrosis Ireland said patients were dismayed by the recommendation against reimbursing the cost of the drug, Orkambi.

The recommendation by the National Centre for Pharmacoeconomics recommends that Orkambi should not be funded “at submitted cost” though it recognises the health benefits provided by the drug.

Price negotiations

CF Ireland said the recommendation opens the door to further price negotiations with the manufacturer, Vertex Pharmaceuticals.

It called on the Government to clarify if it will enter into further negotiations and called on Vertex to reduce significantly the price of Orkambi as part of these negotiations.

"We support a fairer deal for this drug, but it will be a travesty if this drug is not provided to our patients or if there is a significant delay in providing this drug," said CF Ireland chief executive Philip Watt.

About 500 people with CF have the potential to benefit from Orkambi in Ireland.