Throckley mother's fight for Batten Disease drug to save 'beacon of hope' daughter

A mother from Newcastle says she worries every day about a decision by health bosses around her daughter's life-prolonging treatment.

Gail Rich's daughters Jessica and Nicole were both diagnosed with a rare and incurable neurodegenerative condition, commonly known as Batten Disease.

The family's battle for funding and access to the only drug that works in fighting the disease came too late for Nicole, who died in October last year.

Nicole's eight-year-old sister Jessica visits Newcastle's Royal Victoria Infirmary twice a week for infusions to prevent its development - but a decision on whether to keep the treatment on the NHS has been delayed.

Gail Rich, Jessica and Nicole's mother, told ITV Tyne Tees: "The mental burden is excruciating. Since we've lost Nicole, we know the value of time.

"We want to spend it loving our children and making memories with our children."

Having received early intervention treatment on the NHS, Nicole has not developed any symptoms.

The disease can cause childhood dementia, speech and language delays, heart problems and seizures.

On Wednesday 9 October, NICE - the body which assesses treatments for the NHS - said it will spend a further six months considering whether to fund it permanently.

Jessica and Nicole's mother Gail believes NICE’s failure to commit to providing Jessica's treatment is a sign they’ll pull the treatment, which would cause her to deteriorate rapidly.

NICE say it will decide on permanent use of the drug by 27 May 2025. Current patients will continue to receive treatment until then and new patients can also be assessed.

Gail says she spends every day worrying what will happen if the treatment ends. She said: "Nobody knows in May next year, is the treatment going to carry on? If it stops we all know what will happen.

"This treatment has given her a completely new pathway in life. She's a beacon of hope because it's never been seen before.

"Walking, talking, going to school, she was surfing the other week. She has now proven the worth and value of this treatment."

Liz Brownutt of the Batten Disease Family Association supports people like the Rich family. She told ITV Tyne Tees: "We're very dismayed that after five years of a managed access agreement that NICE hasn't issued any guidance about the outcome of its evaluations.

"We don't understand why six months is needed. It feels cruel."

NICE is yet to respond to our request for comment. In copy relating to the drug on its website, it says: "Discussions between NICE, NHS England and BioMarin so far have been constructive, leading to the agreement of the six-month extension, and will now continue.

"All parties are committed to providing updates to stakeholders, notwithstanding the necessity that discussions are undertaken in a confidential setting."

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