Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps he is taking to make Brineura accessible to sufferers of Batten disease.
The National Institute for Health and Care Excellence (NICE) is currently developing guidance on Brineura (cerliponase alfa) for treating neuronal ceroid lipofuscinosis type 2 through the highly specialised technology (HST) programme. 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 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 locally on whether to fund this treatment based on an assessment of the available evidence, and in consultation with the patient.