Almost 3,000 people with cystic fibrosis will miss out on a ‘breakthrough’ treatment after the health watchdog turned it down for use on the NHS.

The National Institute for Health and Care Excellence (Nice) rejected the drug Orkambi, which has been shown in clinical trials to improve lung function and respiratory symptoms in people with the genetic condition.

Orkambi costs $259,000 per year per patient for every year of treatment.

“We know how important a new treatment option would be for people with cystic fibrosis; but for the benefits it offers, the cost of Orkambi is too high,” said professor Carole Longson, director of the Nice centre for health technology evaluation.

‘We can only recommend treatments when we are certain they are both clinically effective and represent good value for money. If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it.

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