13 September 2019

NHSE announce funding for life-changing Batten Disease treatment

NHS England has announced plans to fund a drug treatment for a rare progressive neurological condition that affects children. Batten disease, which is currently incurable, is a form of childhood dementia that affects the nervous system, causing loss of sight and mobility, seizures and early death. Trials of the drug Cerliponase alfa (also known as Brineura) have shown it to be effective at slowing the onset of symptoms and extending the life expectancy of children with the condition.


The announcement comes in advance of a High Court trial in which two families were prepared to enter legal battle for access to the life-saving drug on behalf of their children with the condition. Previously, the National Institute for Health and Care Excellence (NICE) questioned Brineura’s value for money but has now come to an agreement with the drug manufacturer, Biomarin, on a reasonable price for producing it. NHS England expect to offer the drug to Batten disease patients awaiting treatment by December 2019.


Samantha Barber, CEO of the Batten Disease Family Association, a member organisation of The Neurological Alliance, said:


The decision by NICE to recommend Brineura for funding by NHSE is an historic step-forward for children with CLN2 type Batten disease. Brineura is an enzyme replacement therapy and is the first treatment for any form of Batten disease. It has been a much longer process than we anticipated and one that has been a rollercoaster of emotion. Our families have been determined and dignified during this time and it has been a personal honour to work with them to achieve this result.


This is a treatment for one type of Batten disease, and while we celebrate this huge achievement, all our community remember those children for whom this decision comes too late and those with other forms of the disease.


Sarah Vibert, Chief Executive of The Alliance, also welcomed the announcement, saying:

Batten disease is a rare condition that has an immeasurable impact on children and their families, who have had to deal with a long and excruciating wait for this announcement. It is a great relief to hear a deal has finally been agreed, and that Brineura will become available on the NHS for all those with CLN2 type Batten Disease who need it. This is a testament to the Batten Disease community’s hard work and perseverance in campaigning for this. I hope we will see more successful efforts to relieve the symptoms of other rare disease and neurological conditions for which there are currently no treatments available.


The prolonged battle to get Brineura funded by the NHS presents another example of a medicines access system that is not working for patients. It was not the efficacity of the medicine that was of concern, but its cost. The forthcoming NICE methods review is due to reassess many of NICE’s internal methods and processes, but it remains unclear whether any changes that would improve patient access to medicines deemed too costly will result.