The National Institute for Health and Care Excellence (NICE) is responsible for making decisions on the clinical and cost effectiveness of treatments. These can be very difficult decisions, and are made on the basis of very careful consideration of the evidence and public consultation. After considering the feedback from its consultation, NICE issued guidance in July 2016 which did not recommend Orkambi to treat cystic fibrosis. The decision was based on the evidence and price proposal provided by Vertex at the time of the appraisal.
The guidance will be reviewed again in 2019. This review can be brought forward where there is new evidence that is likely to impact on the current recommendations, or if the company puts forward a proposal that demonstrates their drug is cost-effective.