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Parents of boy with rare condition win High Court battle over drug

A judge has ruled that a decision to refuse funding must now be reconsidered.

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.

The boy is being treated for a rare genetic condition (Lynne Cameron/PA)

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.

The judge cautioned against raising hopes (Peter Byrne/PA)

“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.”


From Belfast Telegraph