The NHS finally agrees to fund only known treatment for child killer disease - but it comes too late for tragic St Annes girl

Today's announcement comes too late for the family of little Saffa Shehzan, who died last year despite her parents' valiant efforts to win funding
Today's announcement comes too late for the family of little Saffa Shehzan, who died last year despite her parents' valiant efforts to win funding
Share this article
0
Have your say

The NHS has agreed to fund a drug for children with a rare degenerative disease weeks before families were due to go to court.

Two sets of parents of children with Batten disease had been prepared to take their fight for the drug cerliponase alfa – or Brineura – to the High Court.

NHS England announced earlier today that an agreement on price had been struck with manufacturer Biomarin.

The National Institute for Health and Care Excellence (Nice) had previously said it could not be certain the drug was value for money.

But the news comes too late for the family of one St Annes girl, who died despite her parents' valiant efforts to get funding from the NHS.

Saffa Shehzan, five, died last summer after being diagnosed with a rare form of Batten disease that took away her ability to walk and eat in 2017.

Her mum Saima and dad Majid fought to get Brineura approved in the UK, but were forced to put her on palliative care as her condition worsened. By the time she died, the youngster had been robbed of all her senses but one – her hearing.

Saima, 37, said at the time: “ The last few days, everybody got a chance to say goodbye in their own way. All her family was around. She could hear – she would have been aware of it. It worked out the way she would have wanted.”

Batten disease is an incurable illness which affects the nervous system, causing seizures, visual impairment, mobility loss and early death.

It usually starts in childhood, with an estimated 25 to 40 children living with the condition in England.

NHS England said Brineura would be offered to sufferers not currently receiving treatment by Christmas at the latest.

In February, the parents of a brother and sister with Batten disease said they were “devastated” their treatment would not be funded by the NHS. Ollie and Amelia Carroll, aged eight and six, from Cheshire, have the condition and their parents were given permission to seek a judicial review.

On their Facebook page on Wednesday, the parents said: “We are in utter shock right now... we sit in tears with the news that [Nice] have now recommended the only treatment for Batten disease to children in England.

“We are in talks with Nice as to what happens next. We thank you to everyone from the bottom of our hearts.”

Samantha Barber, chief executive of the Batten Disease Family Association, said: “Brineura offers real hope for these children and the priority now must be to ensure all those who need it get access as soon as possible.

While we recognise the efforts made on both sides to reach this agreement, the reality is that it has taken nearly two years to get to this point.

“The human cost of this delay and the anguish caused cannot be underestimated and we hope that efforts will be made to help others avoid this heartache in the future.

“For now, we are absolutely delighted with this decision, which is a critical step forward in improving the lives of children affected by this devastating condition.”

Simon Stevens, chief executive of NHS England, said: “Coming after extended negotiation, the new deal reached today is a reminder that in order to succeed, companies must be flexible and realistic, because the NHS in England cannot and will not simply write blank cheques at taxpayers’ expense.”