Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what discussions he has had with the National Institute for Health and Care Excellence on securing long term access to cerliponase alfa.

Officials in the Department have had a number of discussions with colleagues in the National Institute for Health and Care Excellence (NICE) about the ongoing appraisal of cerliponase alfa (Brineura) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2). The Government understands the devastating impact of this condition on those affected, and how important the availability of effective new treatments is to patients and their families.

An agreement has now been reached between NHS England and the manufacturer of Brineura, BioMarin, that secures continued access for patients who have already started treatment with Brineura or who begin treatment before the end of this year.

NICE is, at present, unable to recommend Brineura for routine National Health Service funding for new patients in its draft guidance published in August. This decision comes after commercial negotiations between NHS England and BioMarin failed to secure a price for the treatment that properly reflects the limited evidence of its long-term effectiveness.

I recognise how disappointing this will be to all those affected, but I would like to underline that NICE has not yet concluded its appraisal. Stakeholders, including patient groups and the company, have had an opportunity to appeal the final draft guidance. If no appeal goes ahead, NICE expects to publish its final recommendations on cerliponase alfa in October.