Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps he has taken to improve access to treatments for metachromatic leukodystrophy.

Working under the UK Rare Diseases Framework, the Government is committed to improving the lives of those living with rare diseases, such as metachromatic leukodystrophy. One of the framework’s priorities is improving access to specialist care, treatments, and drugs. We remain committed to delivering under the framework and published the fourth England action plan on 28 February 2025.

The National Institute for Health and Care Excellence (NICE) makes evidence-based recommendations for the National Health Service on whether new medicines represent a clinically and cost-effective use of resources. NICE works closely with companies and the United Kingdom regulator with the aim of publishing guidance on new medicines as close as possible to the time of licensing so that patients can benefit from rapid access to clinically and cost-effective medicines. The NHS in England is legally required to fund medicines recommended in a NICE appraisal, usually within three months of final guidance.

In 2022, NICE recommended the world’s first gene therapy atidarsagene autotemcel, also known as Libmeldy, for treating metachromatic leukoystrophy, which is now available to eligible NHS patients in line with NICE’s recommendations.