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NICE

NICE say NO to the use of Cerliponase Alfa for children with CLN2 (Late Infantile Batten disease)

BDFA Summary of NICE Decision

NICE ECD Report

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This decision means that children diagnosed with this devastating disease in England will not have access to this critical treatment which has been shown to slow down and in some cases halt the relentless progression of this disease. The BDFA, alongside families of children on this treatment, have seen how well it works. We have seen children retain their skills and abilities whilst their peers, who are not on treatment, have very sadly lost not just their skills and abilities but also their lives. This treatment has given much longed for hope to families were previously there was none. This hope has now been dashed by this cruel and inaccurately based decision.

Whilst NICE acknowledge that the treatment improves children’s quality of life and slows the deterioration, they question its long term effectiveness. Are we suggesting that clinical trials of treatments should run for many decades to enable this data to be collected or is the fact that children on this treatment continue to thrive and live life to the full after 3 years not sufficient? Data continues to be collected from children and this continues to show the positive effects of this treatment. Children are able to access the treatment in the USA and other European countries whilst the UK continues to lag behind providing treatment for this ultra-rare but devastating disease.

In making its decision, NICE also incorrectly took data from another form of Batten disease and attributed the long-term effects of that disease to CLN2 disease. We don’t have data yet to say how the treatment will work in 10 or 20 years time. What we do know is that it stabilises and in some cases improves life for these children when previously this diagnosis meant a relentless descent towards death.

Andrea West , Chief Executive of the Batten Disease Family Association (BDFA) said:
“We need everyone to help us challenge this unjust and inaccurate decision, we see on a daily basis that this is a treatment that works, we call on everyone to help us give hope to those children and families living with this devastating diagnosis, it has never been truer that together we WILL make a difference”

The BDFA campaign to respond to the NICE decision can be found on the website www.bdfa-uk.org.uk, the BDFA Facebook page or on Twitter.
For more information on how you can help please contact us at support@bdfa-uk.org.uk or 01252 416323

You can download this Briefing paper.

Background information

CLN2 (Late Infantile Batten disease) is a rare autosomal recessive neurological disease diagnosed in between 5-6 children in the UK each year. Children are born seemingly healthy
and develop normally for the first few years of life. Towards the age of 2 their development may slow, including their ability to talk. The first definite sign of the disease is seizures may be drops, vacant spells or motor seizures with violent jerking of the limbs and loss of consciousness. Seizures may be controlled by medicines for several months but always recur, becoming difficult to control. Children tend to become unsteady on their feet with frequent falls and gradually skills such as walking, playing and speech are lost. Children
become less able and gradually skills such as walking, playing and speech are lost. Children become less able, and increasingly dependent. By 4-5 years the children usually have myoclonic jerks of their limbs and head nods. They may have difficulties sleeping and become distressed around this time, often for no obvious reason. Vision is gradually lost and children become completely blind. By the age of 6 years, most will be completely dependent on families and carers for all of their daily needs. They will need a feeding tube and their arms and legs will become very stiff. Death usually occurs between the ages of 6 and 12 years.

CLN2 Information Here

Batten Disease Family Association (BDFA)
The BDFA is the only national UK Charity specifically set up to support and advocate for families facing the devastating diagnosis of Batten disease (a group of life-limiting neurological conditions) The BDFA also raises awareness of this rare set of diseases and directly funds research into potential treatments and ultimately a cure.

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