Cystic Fibrosis Ireland has said that it is ‘dismayed' by a decision not to recommend funding of a groundbreaking drug, which could benefit around 500 people in Ireland with this inherited disease.

The National Centre for Pharacoeconomics (NCPE) is responsible for assessing medicines and medical technologies to see whether they are cost effective. The centre then makes a recommendation to the HSE on whether it should cover the cost of these treatments for patients. The final decision on reimbursement is made by the HSE.

The NCPE has been assessing the cost effectiveness of the drug, Orkambi, which could benefit around 500 people with cystic fibrosis (CF) in Ireland.

CF is Ireland's most common life-threatening inherited disease. Around one in 19 people are carriers of the CF gene and when two carriers have a child there is a one in four chance their child will be born with it. There are around 1,200 people in Ireland with CF and this country has the highest prevalence and the most severe types of the disease in the world.

The condition affects the glands, damaging many organs including the lungs, pancreas, digestive tract and reproductive system, impacting breathing, digestion and reproduction. It causes thick sticky mucus to be produced, blocking the bronchial tubes and preventing the body's natural enzymes from digesting food.

Many people with CF experience exacerbations of the condition, however Orkambi has been shown to reduce exacerbations and hospitalisations by up to 40% in those eligible to take it.

Almost half of people with CF are eligible to take this drug, which is aimed at those over the age of 12 with a specific genetic mutation.

However, the NCPE has issued its recommendation on the drug, stating that it is ‘not considered cost effective...and therefore is not recommended for reimbursement'.

Commenting on the decision, Philip Watt, CEO of Cystic Fibrosis Ireland, said that patients were ‘dismayed by the ruling'.

However, he noted that the NCPE's decision stated that the drug should not be funded ‘at the submitted price'. He believes this leaves the door open to further price negotiations.

Cystic Fibrosis Ireland is now calling on the Government to clarify whether it will enter into further negotiations and is also calling on the pharmaceutical company that manufactures the drug - Vertex - ‘to significantly reduce the price of Orkambi as part of these negotiations'.

It is understood that Vertex has said it is willing to negotiate on price.

"We support a fairer deal for this drug but it will be a travesty if it is not provided to our patients or if there is a significant delay in providing this drug," Mr Watt added.

For more information on CF and Cystic Fibrosis Ireland, click here