228646 - Orphan Drugs (Answered)

Greg Mulholland
To ask the Secretary of State for Health, if he will take steps to commit to the referral of ultra-orphan drugs to NICE Highly Specialised Technologies; and if he will give a target date for this action.

George Freeman

We have referred a number of high cost, low volume technologies to the National Institute for Health and Care Excellence (NICE) for evaluation under its highly specialised technologies work programme. We will continue to do so for technologies that meet the specified criteria and where a NICE evaluation would add value. Only today, Ministers referred the drug Translarna (ataluren) for Duchenne muscular dystrophy, to NICE’s work programme.

Further information about the process by which topics are selected for referral to NICE’s highly specialised technologies work programme is available at: