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NHS agrees to fund drug for children with Batten disease

Decision comes weeks before families set to go to the High Court to challenge NHS England's reluctance to invest in a drug to help treat the rare degenerative disease

Decision comes weeks before families were set to go to the High Court to challenge NHS England's reluctance to invest in a drug for children with the rare degenerative disease

NHS England chief executive Simon Stevens welcomes the new deal with drug manufacturer Biomarin, which produces a drug cerliponase alfa which helps treat Batten disease.
NHS England chief executive Simon Stevens welcomed the new deal with drug
manufacturer Biomarin. Picture: Barney Newman

The NHS has agreed to fund a drug for children with a rare degenerative disease weeks before families were due to go to court.

Two sets of parents of children with Batten disease had been prepared to take their fight for the drug cerliponase alfa (marketed as Brineura) to the High Court. However, NHS England has announced that an agreement on price had been struck with the drug’s manufacturer Biomarin.

The National Institute for Health and Care Excellence had previously said it could not be certain the drug was value for money.

An estimated 25-40 children live with Batten disease in England

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.

NHS England said cerliponase alfa would be offered to patients not receiving treatment by Christmas at the latest.

Batten Disease Family Association chief executive Samantha Barber said: ‘While we recognise the efforts made on both sides to reach this agreement, the reality is that it has taken nearly two years to get to this point. The human cost of this delay and the anguish caused cannot be underestimated. 

‘We hope that efforts will be made to help others avoid this heartache in the future.’

Welcoming the decision, NHS England chief executive Simon Stevens said: ‘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.’


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