Research News

Cystic fibrosis drug is not cost effective, says NICE

BMJ 2016; 353 doi: (Published 20 June 2016) Cite this as: BMJ 2016;353:i3409
  1. Anne Gulland
  1. London

A drug that would benefit nearly 3000 patients with cystic fibrosis should not be made available on the NHS, says the National Institute for Health and Care Excellence (NICE) in new guidelines.1

Although the drug lumacaftor-ivacaftor (Orkambi; Vertex Pharmaceuticals, Boston, MA, USA) was shown to reduce hospital admission and the use of antibiotics among people with cystic fibrosis, the short term benefits to lung function were modest and the long term benefits were uncertain, said NICE.

The drug costs £104 000 (€131 000; $147 000) for each patient for every year of treatment and is licensed for people with a specific genetic defect, the F508del mutation. Around 2750 people in the United Kingdom would be eligible for such treatment.

The Cystic Fibrosis Trust, responding to draft guidelines published in April, said that additional data could be collected that would demonstrate the drug’s long term effectiveness. However, the guideline committee said that, if the drug were to be made available to patients, then the manufacturer, Vertex Pharmaceuticals, would have to reduce the drug’s cost, which it did not do.

Carole Longson, director of the NICE Centre for Health Technology Evaluation, acknowledged that a new treatment option for people with cystic fibrosis was important. But she added, “For the benefits it offers, the cost of Orkambi is too high. 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.”

In its judgment NICE said that Vertex believed that not all of the evidence for the drug’s benefits had been considered by the expert committee. However, the committee said that the company had not presented any qualitative or quantitative evidence to support this belief.

The Cystic Fibrosis Trust said that, in the two years since NICE had conducted its appraisal, new data have shown that Orkambi almost halves the rate of decline in lung function in people with the disease over a two year period.2

Ed Owen, chief executive of the trust, said, “We are very disappointed by this decision. It demonstrates the weakness of the current system, and NICE has been unable to confidently predict the long term benefits of the drug.

“It is now vital that NHS England and Vertex Pharmaceuticals negotiate an agreement to provide access to those who need it. Further delay will cut lives short and we will not sit by watching this happen.”


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