Question to the Department of Health and Social Care:

To ask His Majesty's Government whether permanent funding will remain in place for the treatment of infantile and juvenile Batten disease.

The National Institute of Health and Care Excellence (NICE) published guidance in 2019 that recommended cerliponase alfa, brand name Brineura, for treating neuronal ceroid lipofuscinosis type 2, within a managed access agreement. This was due to the uncertainty in the evidence base, particularly around the long-term clinical benefits and assumptions about disease stabilisation.

During this period of managed access, cerliponase alfa has been available to eligible patients while further data was collected to address the clinical uncertainties. The NICE is now carrying out a new evaluation of cerliponase alfa to determine whether it can be recommended for routine National Health Service funding, taking account of the real-world evidence collected during the managed access period. If this evaluation shows that the treatment is a clinically effective and cost-effective use of NHS resources, it will be recommended for routine use in the NHS. All parties are working together to ensure a conclusion to the ongoing evaluation as swiftly as possible.