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Orkambi’s cost too high for benefit offered, says NICE

17 June 2016  •  Author: Victoria White, Digital Content Producer

The National Institute for Health and Care Excellence (NICE) has said it will not be able to recommend Vertex Pharmaceuticals’ Orkambi (lumacaftor-ivacaftor) for treating cystic fibrosis.

orkambi

In March, NICE consulted on draft guidance which concluded that compared to the current standard of care, the benefit Orkambi offered did not justify its considerable cost. Although Orkambi was shown to reduce instances where people with cystic fibrosis are admitted to hospital, the benefits to lung function – one of the tests used to see how people are improving overall – appeared modest in the short term whilst the long-term benefits were uncertain.

Orkambi costs £104,000 per patient for every year of treatment. It is licensed to treat people who have a specific genetic defect known as the F508del mutation. There are around 2,750 people who would be eligible for treatment with Orkambi in England.

No cost-effective price proposal

In response to the public consultation, the Cystic Fibrosis Trust suggested that additional real-world data could be collected which would demonstrate that the treatment does offer significant benefits in the long-term. However the committee concluded that if the drug were to be made available to patients on this basis, the company would have to provide Orkambi to the NHS in a cost effective way. Vertex Pharmaceuticals did not put this proposal forward to the NICE appraisal committee to consider.

Commenting on the news: Professor Carole Longson, director of the NICE Centre for Health Technology Evaluation said: “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. 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.”

NICE has now issued further draft guidance to consultees who have the opportunity to appeal against the decision. If there are no appeals, the decision will be issued as final guidance in July.

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