09:31, August 08 56 0 theguardian.com

2017-08-08 09:31:03
Court rules against NHS over drug funding for boy with rare condition

The parents of a boy with a rare genetic condition have won a high court victory in their battle over the NHS’s decision to refuse funding for a “life-changing” drug.

The seven-year-old, identified only as S, has severe autism and phenylketonuria (PKU), which inhibits his ability to digest protein.

His autism affects his ability to comply with the dietary restrictions which form the standard treatment for PKU, so his doctors sought funding for a drug called Kuvan.

His counsel, Ian Wise QC, told Mrs Justice Andrews that funding had been repeatedly refused by NHS England with differing reasons given, leaving S at risk of the consequences of poorly controlled PKU, including brain damage.

On Tuesday in London, the judge quashed the decision and remitted it for reconsideration.

She warned: “Whilst this judgment is bound to give rise to a degree of optimism, I must caution against raising hopes too high.

“The fact that this claim for judicial review has succeeded does not mean that there will necessarily be a favourable outcome to this IFR (individual funding request) application.”

The judge added: “However much one might hope that on the next occasion the panel will decide that the net additional expenditure of treating S with Kuvan would be justified, having regard to the likely clinical benefit of keeping his blood phenylalanine levels consistently within the range that would avoid his suffering any additional neurological impairment, thereby potentially enabling him to realise his maximum functioning potential, they could still lawfully decide to refuse funding.

“It is their decision, and their decision alone; and provided it is taken on the basis of the correct interpretation of the IFR policy, and a proper understanding of the case put before the panel and the supporting evidence, it will not be open to challenge.”

The case succeeded on the basis of one of the three grounds advanced, concerning the decision in relation to the drug’s clinical effectiveness.

The judge said: “If ‘clinical effectiveness’ is properly interpreted, the evidence that Kuvan is clinically effective is overwhelming.

“In my judgment, there is no room for a rational conclusion that Kuvan is not clinically effective or that the evidence of its clinical effectiveness – for the precise purpose for which it is sought to be used here – is insufficient.

“Given that the supposed absence of evidence of clinical effectiveness was the specific reason given … for turning down the application, that is such a material error that it suffices in and of itself to warrant quashing the decision and sending it back for reconsideration.”

S’s father told the court that his management had become more difficult over time because he would not take his supplements and had grown more and more interested in normal foods since going to school and seeing what other children were eating.

The only method the family could use to maintain his phenylalanine levels was to seriously reduce his food intake.

If prescribed Kuvan, S would be able to get a proportion of vitamins and minerals from ordinary food.

The boy’s father branded the NHS’s decision as “extremely frustrating, unfair and inhumane”.

“No family should have to fight with the NHS for over 18 months to ensure that their child is given proper treatment,” he said. “It is clear to me they do not want to fund this life-changing drug.”

Jenni Richards QC, for NHS England, said there was no basis for impugning on rationality grounds the judgments reached as to clinical and cost-effectiveness or equity of funding. The mainstay of PKU treatment was – and would remain, even with Kuvan – dietary control.

She said S’s consultant acknowledged that, in terms of neurological impairment, his overall development outcome would mostly be affected by the severity of his autism rather than his PKU and that Kuvan would not be expected to significantly alter or improve S’s behaviour.

Peter Todd, of Hodge Jones & Allen, who is representing S’s family, said they were “greatly heartened” by the ruling.

“Although no mandatory order for funding was made, it would be wholly extraordinary if the IFR panel was to, once again, find reason to not fund this life-changing drug for a child.

“My clients have felt increasingly disillusioned with NHS England, and frustrated by their constantly changing reasons to not fund the treatment.

“We hope that the panel will now take prompt action and secure the funding to ensure that S’s long-term health is no longer endangered.”

S’s father said: “We are very pleased that the court has ordered that NHS England reconsider their decision not to fund Kuvan for our son.

“It’s been a very difficult two years for our whole family and we are hoping that the funding will be granted soon, so that our son’s quality of life can be improved significantly.”