NICE Decision – 22.02.2019

We are very disappointed that NICE has published their decision not to recommend Cerliponase alfa, also known as Brineura, as a treatment for CLN2.

This is a dreadful blow to all those with children on treatment, those waiting for access to the treatment and to the families whose children have passed away without the treatment. This is not just disappointing to those families but for the whole Batten disease community.

You may want to read the decision document on the NICE website https://www.nice.org.uk/guidance/gid-hst10008/documents/html-content-3 . Please be aware that it contains a lot of detail about Batten disease that is upsetting to read.

The reasons stated for not recommending the treatment are uncertainty, lack of long-term evidence and cost. The way ‘cost-effectiveness’ has been calculated was changed in 2017, making it much harder for drugs for rare diseases, like Batten disease, to meet the required thresholds.

This is ‘new’ news and the BDFA is currently taking the advice we need to make sure that whatever we do next will achieve what we want: a favourable decision. We know that the treatment works and our children deserve this treatment. Our families and the team know that we will have your support.

We will keep you informed about how you can help us in the coming days. In the meantime, thank you for your support to date.

Together we will make a difference.