NHS to offer treatment for children with Batten Disease

Young people with Batten Disease will now be offered a drug to slow the onset of sickness, after NHS England and the manufacturer Biomarin agreed a fair price.

Batten Disease is an incurable illness which affects the nervous system, causing seizures, visual impairment, mobility loss and early death. It usually starts in childhood, with an estimated 25 – 40 children living with the condition in England.

The NHS will now be able rapidly to offer the cerliponase alfa treatment to sufferers not currently receiving treatment, by Christmas at the latest.

Simon Stevens, NHS England chief executive, said: “This is another concrete step towards ensuring NHS patients with rare conditions get access to important new treatments. Over recent months there’ve been a series of successful deals as NHS England works closely with the life sciences sector to make life changing new drugs available for haemophilia, MS, rare cancers and other conditions. Coming after extended negotiation, the new deal reached today is a reminder that in order to succeed, companies must be flexible and realistic, because the NHS in England cannot and will not simply write blank cheques at taxpayers’ expense.”

Event Diary

You are invited to this unique annual exhibition that brings together all the disciplines from the emergency services sector who are involved in prevention, response and recovery.