(fashion) The NHS has finally struck a deal for a life-extending drug given to children battling a cruel disease that slowly robs them of their ability to walk, talk and breathe.
Health watchdogs earlier this year rejected cerliponase alfa – the only available medication to treat one type of Batten disease, saying it was too expensive.
But officials have now agreed a ‘fair price’ for the drug, marketed as Brineura, with its manufacturer following almost two years of fierce negotiations and just weeks before affected families were to appeal the decision in court.
It is estimated the drug costs around £500,000 per patient per year but the discounted price offered to the NHS has not been revealed following the U-turn.
Simon Stevens, chief executive of NHS England, today warned pharmaceutical firms that the health service ‘will not simply write blank cheques at taxpayers’ expense’.
Outraged families – including the parents of a boy once cradled by Prince Harry in hospital – launched legal action to get cerliponase alfa available (Oliver Carroll's plight touched the nation in 2016 when a striking image emerged of the six-year-old being hugged by the Prince - dubbed his 'Diana moment')
Amelia Carroll, Oliver's brother, who started the treatment before she developed any symptoms of Batten disease, is a happy, healthy little girl, pictured with Prince Harry in 2016
He said: ‘This is another concrete step towards ensuring NHS patients with rare conditions get access to important new treatments.
‘Coming after extended negotiation, the new deal reached today is a reminder that in order to succeed, companies must be flexible and realistic.’
Brineura is being offered as part of a managed access agreement (MAA) over the next four-and-a-half years. Evidence about its effectiveness will be collected in the meantime.
Outraged families – including the parents of a boy once cradled by Prince Harry in hospital – launched legal action in the wake of the initial decision by NICE.
The High Court last month announced it would hold a judicial review into legality of the decision. Lawyers fighting to overturn the decision claimed it was unlawful.
A full hearing in the High Court was scheduled to be held on 16 and 17 October. It is, however, currently unclear whether or not this will still go ahead.
Samantha Barber, chief executive of the Batten Disease Family Association, said the charity was ‘absolutely delighted’ with the decision to approve Brineura.
She said: ‘Today’s announcement will be a huge relief to all those families affected who have fought night and day for this outcome.
Caroline Day (left), mother of a three-year-old Connie who is struck down with Batten disease (right) told MailOnline how she fears a meeting tomorrow between officials and the drug manufacturer is just going to be a 'tick box exercise'
Batten disease is a rare genetically-inherited disease, caused by waste build-up in the cells which creates neurodegnerative effects including:Blindness Seizures Personality changes Dementia Loss of motor skills and the ability to walk, talk and communicate
Because it is a rare disease, it is often difficult to diagnosed and untested at birth.
There are 14,000 children worldwide that are diagnosed with the disorder.
The Batten Disease Family Association estimate that around up to 17 people are diagnosed with a form of the disease each year.
There are between 100 – 150 affected individuals currently living with Batten disease in the UK.
There is currently no known cure or treatment available for Batten disease.
‘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.
‘This human cost of this delay and the anguish caused cannot be underestimated and we hope that efforts will be made to help