NICE has published draft guidance which confirms its earlier decision not to recommend cerliponase alfa (also called Brineura and made by Biomarin) for children with Batten disease - a very rare inherited condition affecting between 1 and 6 babies each year in the UK.

The decision comes at the end of year-long negotiations between the company and NHS England during which the company was unable to price the treatment at a level that would have addressed the problems highlighted during NICE's assessment of it.

In its earlier draft guidance, published last year, the independent committee had agreed that, although cerliponase alfa is not a cure for Batten disease (which is also called neuronal ceroid lipofuscinosis type 2), it is an important development for treating the condition and that it had shown substantial short-term benefits in slowing the rate at which it progresses.

However, in the absence of long-term evidence about its effectiveness in stabilising the disease and preventing death, and having taken all the health and non-health-related benefits of cerliponase alfa into account, the independent committee considered that the drug was not value for money within the context of a highly specialised service.

At that point the committee asked the company to engage with NHS England to develop a managed access agreement to address the long-term clinical and financial uncertainties, and to negotiate with NHS England terms that could allow the drug to be made available.

Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: "Given the significant burden this disease places on children with the condition and on their parents and carers, and the subsequent negative impact this can have on the quality of their lives, anything that can help to improve treatment is to be welcomed.

"We and NHS England have been very clear with the company about what would be needed in order for us to be able to recommend cerliponase alfa. However, despite being given ample opportunity to come up with a workable solution, regrettably the company has not been able to do so.

"The committee concluded that it is not possible within the NHS to manage complex components of the confidential commercial arrangement proposed by the company. Once these elements are excluded, the cost-effectiveness estimates for cerliponase alfa are far higher than NICE normally considers acceptable for highly specialised technologies. We are therefore left with no alternative but to conclude that cerliponase alfa cannot be recommended as a cost-effective use of NHS resources."

Batten disease is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. This results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.

Symptoms in children with the condition begin from around the age of 2 and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.

Children with Batten disease live to between 8 years and early adolescence; the average life expectancy is 10 years. It is estimated that in the UK there are around 30 to 50 children living with the condition.

The draft guidance is now with consultees, including the company, healthcare professionals and patient/carer organisations who have the opportunity to appeal against the draft recommendations.

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