CF drug may cost €230k per patient

Concerns have been raised that a new wonder drug for cystic fibrosis (CF) patients could be prohibitively expensive as manufacturers are seeking to charge up to €230,000 per patient for a one year supply of the pills.

Kalydeco — the first ever drug to treat the root causes of CF — was approved for usage by the European Medicines Agency in the summer.

Discussions between the health authorities and the drug manufacturer, Vertex, about cost, have been taking place.

The National Centre for Pharmaeconomics is now assessing its potential cost to the health budget.

Ireland has the highest proportion of people with CF in the world with the G551d mutation.

Globally, between 3% and 4% of people with CF carry the G551d gene.

Kalydeco is the first ever drug available that impacts on the underlying cause of CF for people that carry this particular gene mutation, which exists in about 10% of Ireland’s 1,200 CF sufferers.

Kalydeco is taken twice a day and is one of the first drugs designed to correct a specific genetic defect. Research showed how users’ lung strength increased by around 10% compared to patients taking a placebo.

Patients also had less infection and gained nearly seven pounds in weight, a significant increase for a group who have trouble putting on weight.

Áine Collins TD, who has a child with CF, called on the HSE to “play hard ball” with Vertex.

“We need the HSE to negotiate with the drug manufacturers, Vertex, to ensure that this new drug can be purchased at a reasonable price.

“It’s fantastic news that Kalydeco will soon be on the market, but it will only be of real use if it’s available to those who need it.”

CF is an inherited chronic disease that primarily affects the lungs and digestive system of about 1200 children and adults in the Ireland.

It is caused by a defective gene and its protein product that prevents cells from properly absorbing and excreting salt and water.

Earlier this year, Vertex executives warned Kalydeco would cost $294,000 (€230,000) for a one year supply, placing it among the most expensive prescription drugs sold in the US.

Speciality drugmakers are known to charge $300,000 or more for drugs that treat very small groups of patients.

The Cystic Fibrosis Association of Ireland are hosting information nights on Kalydeco and other new CF therapies in Limerick on Nov 14 and at a date to be yet confirmed in Cork.