The high cost of combination treatment lumacaftor/ivacaftor (Orkambi) means it has not been recommended for use in the NHS in England and Wales for cystic fibrosis (CF), draft guidance from the National Institute for Health and Care Excellence (NICE) says.
NICE says the product’s price – £104,000 per patient per year – is not justified by the benefits of the treatment.
Carole Longson, director of the NICE centre for health technology evaluation, says: “We know how important a new treatment option would be for people with CF, but for the benefits it offers, the cost of Orkambi is too high.”
The Cystic Fibrosis Trust has written an open letter, published on 17 June 2016, calling for the product’s manufacturer Vertex and NHS England to find a way to make the treatment available to patients.
The letter says NICE has not seen data which shows that the product reduced the rate of lung function decline over two years by almost half.
The charity adds that NICE is unsure of the treatment’s long-term value and has proposed that patients should be granted access to the drug and the long-term efficacy assessed via the Trust’s CF registry, which records health data on consenting people with CF in England, Wales, Scotland and Northern Ireland.
Ed Owen, chief executive of the Cystic Fibrosis Trust, says: “It is now vital that NHS England and Vertex sit down and negotiate a means by which this drug becomes available to those who need it while we put in place a system to measure the real-world impact through the CF registry.”
He adds: “We want to see meaningful discussions underway by the end of July  to design an access solution for Orkambi and we will not sit idly by if this does not materialise.”
NICE says that final guidance will be issued in July 2016, until which time consultees are able to appeal the decision. It adds that if the manufacturer submits a cost-effective proposal after that time, it will still be considered under rapid review.