Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of whether NICE's guidance on the drug Orkambi supports his Department's approach of ensuring that NICE appropriately funds new, targeted technologies for the treatment of rare diseases.

Orkambi was appraised for the treatment of cystic fibrosis by the National Institute for Health and Care Excellence (NICE) in July 2016, but was not recommended. While it was clinically effective, the benefits were not sufficient to justify its considerable cost. NICE’s guidance is scheduled for review in July 2019.

NICE is the independent body responsible for developing authoritative, evidence-based guidance for the National Health Service on whether drugs and other treatments, including for rare diseases, represent an effective use of NHS resources. NICE’s methods and processes for the assessment of drugs have been carefully developed over time and are internationally respected. NICE continues to keep its procedures under periodic review to ensure that they remain effective.