NHS England will now offer a life-changing treatment for children with neuronal ceroid lipofuscinosis type 2 (CLN2 or Batten disease), a rare inherited condition that leads to an early death.
Cerliponase alfa, an enzyme replacement therapy, slows the decline of the disease and extends the patients’ lives. It is administrated directly into the brain via a surgically implanted permanent access device.
NHS England and the National Institute for Health and Care Excellence (NICE), in two separate statements published on Wednesday, said the manufacturer Biomarin agreed for a fair price deal with NHS England.
“This treatment shows great promise in slowing the progression of this devastating condition to allow children to enjoy normal childhood activities for longer which is so important,” said Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE.
NHS will rapidly offer the treatment to Batten disease patients not currently receiving treatment, by Christmas at the latest.
Batten disease is a very rare condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. The majority of the children with the disease live to between eight years and early adolescence. The average life expectancy is ten years.
Currently, it is estimated that around 30 to 50 children are living with this condition in the UK.
The recent successful negotiation is following the deals for new and life-changing treatments for rare cancers, sight loss, haemophilia, multiple sclerosis and rare muscle-wasting disease in children.
NHS chief executive Simon Stevens called the new deal as a reminder for companies to be flexible and realistic to succeed.
“This is another concrete step towards ensuring NHS patients with rare conditions get access to important new treatments,” Stevens said.
Health Secretary Matt Hancock said the deal is a great example of how the government can work with the industry to get treatments to patients as quickly as possible.
“I’m absolutely delighted this new treatment will be funded by the NHS, giving families dealing with the devastating impact of Batten disease renewed hope for a better quality of life for their child,” Hancock said.