UK NICE confirms it won't recommend use of Orkambi (lumacaftor + ivacaftor) for treating cystic fibrosis - Vertex Pharmaceuticals

The UK's National Institute for Health and Care Excellence has confirmed it will not be able to recommend Orkambi (lumacaftor + ivacaftor), from Vertex Pharmaceuticals, for treating cystic fibrosis. 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.

The committee discussed the clinical evidence from the TRAFFIC, TRANSPORT and PROGRESS trials. Although the acute improvement in ppFEV1 was modest, when combined with the improvement in rates of exacerbations, the clinical trials provide evidence that lumacaftor�ivacaftor may significantly improve the long-term outcome for patients. The committee concluded that longitudinal changes rather than acute changes in ppFEV1 were more clinically relevant for assessing long-term outcomes of cystic fibrosis, and both the observed and extrapolated benefits of lumacaftor�ivacaftor on ppFEV1 were taken into account in the company�s cost-effectiveness analysis.