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 a condition, known as phenylketonuria (PKU), which inhibits his ability to digest protein.

The boy’s 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 the court that funding had been repeatedly refused by NHS England with differing reasons given. He argued that this had left S at risk of the consequences of poorly controlled PKU, including brain damage.

On Tuesday in London, the judge quashed the decision to refuse funding for the drug and remitted it for reconsideration.

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

“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.

“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.”

NHS England had a policy, which was unchallenged, not to commission Kuvan because of insufficient evidence as to its medium and long term clinical effectiveness.

The mainstay of PKU treatment was – and would remain, even with Kuvan – dietary control.

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.

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.



World News


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