In a patient centred health service one might expect the definition of “exceptional” when referring to rationing decisions, to have input from the patient and their medical representative. But a case record of one or two does not prove a treatment is effective. NHSreality sees no reason why the product cannot be tried, but with provision that there is an assessment interval period, and that this is done by an independent third party. Lack of progress would mean treatment stopped. Real improvement would mean it continued. There cannot be many children with this form of double trouble.. If the treatment were cheap there would have been little argument, but it is expensive at £365,000 per year…. Now even cheap placebos such as Homeopathy are being threatened. Prince Charles will be disappointed. Perhaps he can save the health services from implosion.
Phenylketonuria is the disease which is found by the heel prick in babies (Guthrie Test). it is reliable and allows treatment of a rare condition, preventing brain damage. It is an inherited inability to metabolize phenylalanine which, if untreated, causes brain and nerve damage.
Autism is a completely different condition, and to have both has to be exceptional by any normal use of the English Language. Unfortunately, in the UK health systems, English Language is distorted in a manner which has demotivated and disengaged two of the most altruistic and motivated professions: Medicine and Nursing.
The family of a seven-year-old boy with a condition that could cause severe brain damage are challenging an NHS decision to deny him a life-changing drug, at the High Court.
David, not his real name, is on a severely restricted diet, as his rare condition means that too much protein could cause permanent brain injury.
He also has autism – so controlling his diet is “a nightmare” for his parents.
The case could have huge implications for NHS drug provision, say lawyers…..David’s NHS consultant wants him to have a drug called Kuvan, which would help him metabolise protein.
But the drug costs £100 a day, and the NHS will pay for it only in exceptional cases.
An individual funding request for David was refused by NHS England on the grounds that his case was not sufficiently exceptional, and the family began legal proceedings to challenge that decision.
…But late last year, the NHS changed tack, agreeing that David’s case was exceptional, instead refusing funding on the grounds that the drug’s clinical and cost effectiveness were not established.
David’s consultant believes Kuvan would reduce the danger David faces and make his life much easier.
“It’s a basic right to be able to eat, and he’s being denied that right at this moment,” his mother said.
“With Kuvan, he could access more food and wouldn’t have to have as many supplements. It’s his quality of life really and our lives as a family as well.”
The family find the NHS’s continued refusal to fund the treatment “heartbreaking”.
“We just feel that they are totally ignoring the fact that there is a little boy at the centre of this,” said his father….
…”Funding is being squeezed, demand for care is going up, and that means that the NHS is having to take some really difficult decisions about what will and won’t be funded,” said Ms Robertson.
A spokeswoman for NHS England said that in order to make this sort of complex decision, “we must consistently apply our policies and consider the evidence presented to decide if the requested treatment will be clinically beneficial and cost effective”.
The spokeswoman said “it would not be appropriate for NHS England to discuss this case any further at this stage”.