Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, with reference to the introduction of a cost-effectiveness threshold in the NICE highly specialised technologies appraisal process, what steps he is taking to ensure equity of access to treatment for different subgroups of patients with Duchenne muscular dystrophy.

The National Institute for Health and Care Excellence (NICE) evaluates all medicines referred to its highly specialised technologies (HST) programme, including medicines for Duchenne muscular dystrophy, consistently in accordance with its published methods and processes. In 2017, following public consultation, NICE introduced an assessment of cost-effectiveness into its HST methodology that provides a consistent and transparent mechanism for evaluating technologies while recognising the need for greater flexibility in the evaluation of very high cost drugs for the treatment of very rare diseases.