Cost of cystic fibrosis drug Orkambi too high for benefit offered, says NICE

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

In March, the medicines cost-effectiveness watchdog publicly consulted on draft guidance which concluded that, compared to the current standard of care, the benefit Orkambi offered did not justify its considerable cost.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 (~$149,000) 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.