23 March 2016 - NICE has issued draft guidance which does not recommend Orkambi (lumacaftor-ivacaftor marketed by Vertex Pharmaceuticals) for treating cystic fibrosis.

The independent appraisal committee concluded that the cost of Orkambi was considerably higher than the current standard of care and it could not be considered a cost effective use of NHS resources.

There is currently no cure for cystic fibrosis and current treatments aim to manage individual symptoms.

Orkambi is licensed to treat people who have a specific genetic defect known as the F508del mutation. There are around 2,750 people in England with this genotype. It costs £104,000 per patient for every year of treatment. Most people suitable for Orkambi would also still remain on the current standard of care whilst taking it.

For more details, go to: https://www.nice.org.uk/News/Press-and-Media/nice-consults-on-draft-guidance-for-new-cystic-fibrosis-treatment