Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, when he plans to make Cerliponase alfa, marketed as Brineura, available on the NHS for the treatment of ceroid lipofuscinosis type 2.

The National Institute for Health and Care Excellence (NICE) is currently developing highly specialised technology (HST) guidance on Brineura (cerliponase alfa) for treating neuronal ceroid lipofuscinosis type 2. Further information can be found at the following link:

https://www.nice.org.uk/guidance/indevelopment/gid-hst10008

Through its HST programme, NICE makes recommendations on whether very high cost drugs for very small numbers of patients represent an effective use of the National Health Service resources. NHS England is required to fund medicines recommended in NICE’s HST guidance, normally within three months of the publication of final guidance.

In the absence of positive guidance from NICE, it is for commissioners to make decisions on whether to fund this treatment based on an assessment of the available evidence.