Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the potential merits of ensuring that a permanent access agreement is reached for Brineura as a treatment for patients with neuronal ceroid lipofuscinosis type 2 (CLN2).

The National Institute for Health and Care Excellence (NICE) is currently developing guidance for the National Health Service on whether cerliponase alfa, also known as Brineura, can be recommended for routine NHS funding, considering real-world evidence collected during a period of managed access. NICE and NHS England are actively engaging with the company, BioMarin, as well as other stakeholders in the development of the guidance to secure continued access for NHS to Brineura at a cost-effective price. These discussions are complex and given the nature of this evaluation and the potential impact on patients and their families, it is essential that they take the necessary care to reach the best possible outcome. All parties are also committed to providing updates to stakeholders, notwithstanding the necessity that discussions are undertaken in a confidential setting.