Question to the Department of Health and Social Care:

To ask His Majesty's Government what assessment they have made of how the decision to limit Brineura access from January 2026 aligns with the Rare Diseases Framework; and what action they will take to ensure continued progress in rare disease treatment equity.

The National Institute for Health and Care Excellence (NICE) is currently updating its guidance on the use of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2, and has not yet published final guidance. Brineura is currently available for eligible patients via the managed access agreement.

Working under the UK Rare Diseases Framework, the Government is committed to improving the lives of those living with rare diseases. The framework sets out improving access to specialist care, treatments, and drugs as a priority, and health equity as a cross-cutting theme. As part of our work to address health inequities we commissioned a scoping review to further develop the evidence base which was published alongside the 2025 England Rare Diseases Action Plan. NHS England has developed a health inequalities toolkit to support people working within highly specialised services to improve equity in their everyday service delivery.