New hope for boy denied 'life-changing' drug by NHS

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The parents of a boy with a rare genetic condition have won victory in a bitter High Court battle with the NHS.

Health bosses have refused to fund a “life-changing” drug the boy’s family believe would improve his quality of life.

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 case succeeded on the basis of one of the three grounds advanced – concerning the decision in relation to the clinical effectiveness of the drug.

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

She said the reason for turning down the initial application had been that Kuvan was not clinically effective and so overturned the decision.

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 decision “extremely frustrating, unfair and inhumane” and 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.

Additional reporting by PA

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