A HEARTBROKEN family of a little girl with a rare terminal disease is this morning begging the NHS not to cut funding to potentially life-saving research.
Determined nine-year-old Maddison Crockford-Reid has such a rare condition – Sanfilippo Syndrome – that there is no cure.
Her devastated family have been struggling to come to terms with the fact she is unlikely to make it past her late teens since her shock diagnosis in August 2015.
But for the past few months, new clinical trials for a potential cure had given the family a much-needed glimmer of hope.
However, in a fresh blow the NHS and the National Institute for Health and Care Excellence (NICE) revealed they will no longer stump out cash for the most costly treatments, as of Saturday, April 1.
Maddi’s older sister, Jorden Evans, 21, said she and her loved ones had been left reeling by the news, saying it was a death sentence for Maddi.
Jorden, of Cheltenham Road, Paulsgrove, said: ‘We’re just shell-shocked. My mum is absolutely devastated and hasn’t stopped crying.
‘Having this funding is a massive glimmer of hope for both our families and families affected by rare diseases.
‘It just feels as though this tiny glimmer of hope has been snatched away from us.’
In the UK there are only 24 people affected by Sanflippo Syndrome, with a mere handful having the same ultra-rare version of the condition Maddi has.
Maddi illness means she will eventually lose the ability to walk, talk, see and hear – dealing a hammer blow to her loving family.
Jorden added: ‘This act by NHS England and NICE is traumatising to say the least.
‘It would leave families with absolutely no hope for their children.
‘In our personal circumstances, it would halt Maddison’s current clinical trial and would stop all future ‘cure’ trials from taking place.
‘This would effectively be leaving the disease to simply ‘take it course’ and thus leaving her no hope or chance of survival.’
The move will see an introduction of a £100,000 ‘quality-adjusted life year’ threshold for medicines evaluated as part of NICE’s highly specialised technologies programme, which assesses treatments for ultra-rare diseases like Maddi’s.
Campaigners say this threshold would stop the flow of expensive new medicines reaching patients most desperately in need, with most treatments costing upwards of £500,000.
However, charity leaders claim this is a small price to pay to give the few families in Britain, coping with ultra-rare illnesses, extra time with their loved ones.
Christine Lavery is the group chief executive of The Society for Mucopolysaccharide Diseases (MPS) which supports Maddi’s family.
In a scathing criticism, she branded the proposals an ‘abuse of human rights’.
She is now urging the people of Portsmouth to rally behind Maddi and contact their local MPs, calling for the proposals to be axed.
Speaking to The News last night, Mrs Lavery said: ‘To penalise these families and children for the misfortune to have these rare diseases is incomprehensible.
‘Being born with an ultra-rare disease, a disease affecting less than 110 people in England, is not a lifestyle choice; it is no-one’s fault; it happens albeit very rarely; it happened to my son. At that time, there was no treatment and Simon died aged seven years.
‘I can only imagine now how it might be to be faced with a child with an ultra-rare disease who could be treated with a highly-specialised medicine but is denied treatment on cost grounds.
‘The pain for the family of seeing their child condemned to death by Andrew Dillon, Chief Executive of NICE; Simon Stevens, Chief Executive of NHS England and the UK Government is unimaginable.’
She added: ‘This is an abuse of people’s human rights.’
Jorden said she is writing to Portsmouth North MP Penny Mordaunt, appealing for help.
She added she wants there to be a ‘pause’ to the funding proposals to allow the government to reconsider them.