NHS makes decision on funding of Brineura for Batten Disease

Some of you may remember these lovely children from my rare disease awareness campaign in February last year: Amelia and Ollie, brother and sister, who both have CLN2 type Batten disease.

At the time, they were waiting to see if the treatment (#Brineura), which is extending their life and allowing them a better quality of life, would be continued on the NHS. Can you imagine the torment their family has been through, waiting for a decision for all this time?

Well, that decision has arrived. NICE have approved the continued use for those already on treatment. This is wonderful news for these families, who finally have one less thing on their shoulders to worry about. 🥳

However, this decision comes with a sting in the tail. 🦂

"The committee took into account the condition's rarity, severity and the effect of cerliponase alfa (Brineura) on quality and length of life. But using the proposed price of the medicine, the most likely cost-effectiveness estimate is not within what NICE considers an acceptable use of NHS resources. So, cerliponase alfa is not recommended".

This decision means that no child born or diagnosed after December 2025 will have access to this drug. A devastating blow to the Batten community in the UK. It also has consequences for the campaign to add Battens to the newborn screening programme, as one of the criteria for inclusion on the panel includes the availability of effective treatments to provide better outcomes.

I hear those who say, "It's expensive", it is. It costs around 500,000 per child annually. There are currently around 35 - 50 children with CLN2 in the UK. But not all will be on treatment. IF ALL children were on treatment, the cost to the NHS would be 25 million annually.

If we compare this to the cost to the NHS relating to diabetes complications of £6.2 BILLION a year, it seems pretty insignificant (Diabetes UK).

The decision not to continue Brineura just doesn't make sense. This raises the ethical question of why companies and regulators can bring drugs to market for rare diseases without concrete measures in place to secure access to these drugs once the trials have been completed. This has also happened in other rare diseases, so this is not unique. We must to better for these communities.

Read the statement from the BDFA here: https://bdfa-uk.org.uk/news/bdfa-statement-response-nice-publication-draft-guidance-treatment-cln2