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.
Funded
on 04th April 2019
£21,241
pledged of £100,000 stretch target from 395 pledges
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.

Latest: Aug. 30, 2019

Update from the BDFA

Dear All

I am writing at a pivotal time for families of children with CLN2. This week there was a meeting of the NICE HST Committee. This is the fifth meeting about Brineura. Parent and clinician ex...

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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 4

Batten Disease Family Association

Aug. 30, 2019

Update from the BDFA

Dear All

I am writing at a pivotal time for families of children with CLN2. This week there was a meeting of the NICE HST Committee. This is the fifth meeting about Brineura. Parent and clinician experts were asked to attend the public part of the meeting to give their evidence. The emphasis was on a potential Managed Access Agreement, which would be the means of granting access to Brineura while continuing to collect more information about the treatment.

As you know, the main issue has been the cost of the treatment. The second part of the meeting dealt with the commercial aspects and we were not able to participate in that session.

Our experts gave their evidence thoroughly, clearly and from positions of deep knowledge and the BDFA is grateful to them all.

We will keep you updated.

Two of the families are taking court action against NICE. The date for their Judicial Review has been set for 16-17 October. More information is available here. We will be supporting them and all the families impacted by the whole process relating to Brineura.

It is never far from my thoughts that there are four families with children who have no access to the treatment currently. This waiting must be torture to them and I am sure all our hearts go out to them.

Lastly, while we all wait for news, it would be really useful that if you are talking about Brineura on social media channels, especially Twitter, please do use #Brineura. It's a useful way for us to be able to make sure we amplify everyone's voices. Thank you for your help.

We will, of course, let you know further news and thank you for your support.

All best wishes

Samantha Barber

BDFA CEO

Update 3

Batten Disease Family Association

Aug. 27, 2019

Breaking news - NICE HST Committee to discuss Brineura

Dear Friends

Over the summer work has continued apace to keep Brineura in the public eye, in the minds of Parliamentarians and to progress discussions between NHSE, NICE and the pharmaceutical company. We have been informed that Brineura will be discussed at the Highly Specialised Technology Committee this coming Thursday 29 August.

You can see the information from NICE on this page.

We will, of course, keep you informed about that meeting. In the meantime, thank you again for your support of this hugely important cause. 

Many thanks

The BDFA Team

Update 2

Batten Disease Family Association

May 24, 2019

We met the Secretary of State for Health and Social Care!

This week on Tuesday, two of our families met Matt Hancock, the Secretary of State for Health and Social Care. The meeting was arranged by Catherine McKinnell MP who has two constituents with CLN2. 

The meeting was meant to be for MPs only, but we were delighted when the families were invited to join the meeting. 

Mr Hancock spoke with the parents and children, asking about the impact of the treatment and its importance to the children. Whilst it is not in his powers to make a decision on Brineura, he can explore the barriers to reaching a positive decision and help to progress the conversation.

It was a positive meeting and the first time in a while that we have felt hopeful. We will keep you informed and keep doing all we can to get this decision reversed. Thank you for all your support.


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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