Life Prolonging Drug Denied for Children with Life Limiting Disease

by Batten Disease Family Association

Life Prolonging Drug Denied for Children with Life Limiting Disease

by Batten Disease Family Association
Batten Disease Family Association
Case Owner
The Batten Disease Family Association (BDFA) is a small, national charity that supports and advocates for children and their families affected by Batten disease.
16
days to go
£20,751
pledged of £100,000 stretch target from 387 pledges
Pledge now
Batten Disease Family Association
Case Owner
The Batten Disease Family Association (BDFA) is a small, national charity that supports and advocates for children and their families affected by Batten disease.
Pledge now

This case is raising funds for its stretch target. Your pledge will be collected within the next 24-48 hours (and it only takes two minutes to pledge!)

Latest: March 28, 2019

Thank you from the BDFA

Thank you to everyone who has pledged towards our initial target so far. It has been immensely heartening to the families and the team to see your support in such a tangible way. We have just 8 day...

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The only drug ever for any form of Batten disease has not been recommended by NICE so is not available to children with CLN2 in England. It is available in 20 other countries including Wales. Batten disease is a form of childhood dementia and is terminal. The photos above are some of the children with CLN2 Batten disease.

Batten disease is a rare genetic condition and approximately 150 children have the disease in the UK each year. There is no cure and those with the disease either die as children or in early adulthood.

There are 14 forms of Batten disease. Each year five or six children are newly diagnosed with the CLN2 form. They enjoy a healthy start to life, reaching usual milestones but towards the age of two, development slows and a number of symptoms are presented. It is usual for seizures to be the first sign of the disease. Falls, loss of motor skills and speech loss compound the condition. Childhood dementia and blindness combine with these symptoms to make this a devastating disease. This means that families who looked forward to the usual upbringing of their child are now faced with the challenge of caring for their son or daughter whose deterioration means they become full time carers, watching their child die. Deterioration can be swift. Death usually occurs between the age of 6 and 12. Some families have to cope with more than one of their children having the condition.

On Friday 22 February 2019,  NICE announced that they would not fund a treatment called Brineura. This enzyme replacement therapy has shown to prolong life as well as improving the child’s quality of life. Our parents, not surprisingly, describe the treatment as miraculous.

Currently five children are on the clinical trial and six children have access to the treatment through a compassionate use scheme. We don’t know if these children will continue to receive this treatment given NICE’s decision. And there are other children who need the treatment who cannot receive it. It is hard to imagine how parents and other family members are coping with this situation.

WHY ARE WE RAISING FUNDS?

The effect on our families has been catastrophic and their emotions are a mixture of devastation, grief and anger. They had hope for their children having a better life. This hope has been snatched away from them.

The legal landscape is complex and BDFA is raising funds for expert legal advice on the options available to us and the families we support who are devastated by NICE’s decision. We are therefore seeking £20,000 for initial comprehensive advice and related costs. We will need more to continue our fight. Funds raised will pay our legal fees and associated costs, communication costs, and for our staff to take on this battle.

PLEASE CONTRIBUTE AND SHARE THIS PAGE NOW!

Your support will make a difference to the children living with CLN2 who need this treatment, improving their quality of life as well as giving their families more precious time together.

The BDFA is a small charity, we are determined to challenge NICE’s decision because we cannot comprehend how a drug, agreed to be effective should be denied to children in England when the drug is available in over 20 countries, including Wales.

We do not have reserves to enable us to take on this fight so your support is vitally important.

Our children deserve better from the health system that should be there to help them.

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Update 1

Batten Disease Family Association

March 28, 2019

Thank you from the BDFA

Thank you to everyone who has pledged towards our initial target so far. It has been immensely heartening to the families and the team to see your support in such a tangible way. We have just 8 days left until we need to reach our target and are just over half way to getting there.

We've had some really good interest in our cause. You may have seen a piece about the treatment and the NICE process in this week's Private Eye in the Medicine Balls section. It's great that Batten disease is getting this kind of coverage.

Equally important is that in the past week Batten disease and Brineura has been mentioned on Parliament, not once, but twice! The first time was in last week's debate on Rare Disease and NICE. The second time was a direct request for Matt Hancock to meet with one of our families. That request was made by their MP and we are absolutely delighted that Matt Hancock agreed to do so. We will keep you informed about this.

Obviously we need to reach our target and we're working on hard achieving that. Any help you can give would be great!

Many thanks again from all the families and the team at the Batten Disease Family Association

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