NHS to provide treatment to children with rare nervous system condition

Children in England with Batten Disease – a rare, incurable illness which affects the nervous system and often leads to early death – are set to receive treatment on the NHS to slow the onset of sickness.

The announcement from NHS England comes after the health service struck a deal with drug manufacturers.

Batten Disease usually starts in childhood, and can cause seizures, visual impairment, mobility loss and early death. It is a very rare condition, with an estimated 25-40 children living with the condition in England.

Drug cerliponase alfa has been shown to extend the lives of youngsters who take it, and now NHS England has secured a deal with the manufacturer of the product to bring the treatment onto the healthcare service at a fair price which the NICE committee was able to recommend as an effective use of NHS resources.

There is currently no cure or life-extending treatments for Batten Disease, only symptom relief and supportive care.

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The deal to treat Batten Disease is the latest successful negotiation led by NHS England, which has seen new and life-changing drugs come on stream for patients in England, after treatments for rare cancers, sight loss, haemophilia, multiple sclerosis and a rare muscle wasting disease in children have all been approved within the past year.

Simon Stevens, NHS 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.”

Professor Stephen Powis, NHS national medical director, added: “This is important news for children and their families suffering from this devastating disease who can now have access to a life-changing treatment.”


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