Batten Disease Funding Crisis

Image credits: BBC

The lives of 36 children in the UK with the rare neurodegenerative CLN2 Batten disease hang in the balance as NHS England decides whether to continue funding for the life-changing enzyme therapy drug Brinuera. The drug, which costs £520,000 per child per year, has been shown to slow the progression of the disease, giving children a better quality of life. Without it, children with Batten disease will experience rapid decline in their ability to walk, talk, and see, leading to a painful death.

Parents of children with Batten disease are desperately hoping that NHS England will agree to continue funding for the drug, which has been provided under a Managed Access Agreement since 2019. The agreement is set to expire in May, and parents are worried that their children will be left to suffer without the treatment. The National Institute for Health and Care Excellence (NICE) has twice refused funding for the drug, but parents have been instrumental in challenging the decision and winning a Supreme Court battle.

The decision on whether to continue funding for Brinuera rests with a panel at NHS England, which is currently in commercial discussions with US pharmaceutical giant BioMarin, the manufacturer of the drug. Parents are concerned that the cost of the drug will be a major factor in the decision, and that their children's lives will be valued based on the cost of the treatment. The BDFA (Batten Disease Family Association) has described the treatment as "groundbreaking" and said that it has reduced patients' decline by 80%.

Parents of children with Batten disease have shared their stories of how the treatment has improved their children's quality of life. One mother, Phoebe, said that her daughter Flory's treatment has given her a "fantastic quality of life" and that she is still able to walk, communicate, and eat. Another mother, Anna, said that her daughter Beatrice's health is likely to deteriorate quickly if the NHS removes provision of the vital drug, and that she is worried about the impact on her daughter's life.

The timeline of events is as follows: * 2019: Brinuera is granted under a five-year Managed Access Agreement * 2024: The agreement is extended for six months * May 2025: The agreement is set to expire * Ongoing: Commercial discussions between NHS England and BioMarin * Ongoing: Parents and the BDFA campaign for continued funding for the drug The policy points to consider are: * The cost of the drug: £520,000 per child per year * The effectiveness of the drug: reduces patients' decline by 80% * The impact on children's quality of life: gives children a better quality of life * The decision-making process: NHS England panel decision based on commercial discussions with BioMarin Statistical data: * 36 children in the UK have Batten disease * 30-50 children in the UK are affected by CLN2 Batten disease * £522,722 per patient annually: the cost of a fortnightly 300mg dose of Brineura * 80%: the reduction in patients' decline due to the treatment * 10-12 years: the life expectancy for a child with Batten disease without treatment