Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, pursuant to the Answer of 31 May 2018 to Question 146853, what assessment his Department has made of the effect of the introduction of a cost-effectiveness threshold into NICE's highly specialised technology programme on patients' access to new treatments for muscular dystrophy.

The changes to the National Institute for Health and Care Excellence’s (NICE) methodology for the development of highly specialised technology (HST) guidance were introduced following public consultation and create a framework that will enable truly transformative new drugs for patients with rare diseases to be made available to patients where companies are willing to set prices that fairly reflect the added benefit they bring. NICE is currently developing HST guidance on the use of eteplirsen for treating Duchenne muscular dystrophy.