NHS decision to deny drug to boy with severe autism was “fundamentally flawed”

BMJ 2017; 358 doi: (Published 09 August 2017) Cite this as: BMJ 2017;358:j3822
  1. Clare Dyer
  1. The BMJ

A High Court judge has quashed a “fundamentally flawed” decision by the NHS to refuse funding for a “life changing” drug for a 7 year old boy with severe autism and phenylketonuria (PKU).

Mrs Justice Andrews said that NHS England had made “error upon error” in the steps it took in deciding not to fund sapropterin dihydrochloride (Kuvan) for the boy, “SB,” who displays violent and challenging behaviour and whose autism makes the usual dietary treatment for PKU hard to manage.

The judge quashed that decision, taken last December, and has sent the case back for reconsideration. But she warned against “raising hopes too high,” noting that it was still open to the panel to use its discretion to refuse the individual funding request (IFR), as long as it took the decision properly.

PKU is an inherited metabolic disorder that inhibits the ability to digest protein and prevents the body from breaking down an amino acid, phenylalanine, which builds up in the blood. Saikat Santra, SB’s doctor at Birmingham Children’s Hospital, told the IFR panel that the boy’s severe autism made it hard to maintain his blood phenylalanine levels consistently within a low enough range to avoid brain damage. Kuvan, which would help SB to metabolise protein, costs about £100 (€111; $130) a day.

SB’s father told the court that the 7 year old’s management had become more difficult because he would not take his supplements and had become more interested in protein based foods since going to school. If Kuvan were prescribed, SB would be able to get a proportion of vitamins and minerals from ordinary food.

Andrews said that the IFR panel had accepted that SB was clinically exceptional but that it had wrongly refused the request on the basis of insufficient evidence of the drug’s clinical effectiveness.

“If ‘clinical effectiveness’ is properly interpreted, the evidence that Kuvan is clinically effective is overwhelming,” she said. “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 purposes 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 to Dr Santra 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.”

The panel had decided that the evidence as to SB’s likely benefit from Kuvan was insufficient to embark on a cost-benefit analysis. “However, this decision was informed by error upon error, the most fundamental of which was that the panel misunderstood (and/or mischaracterised) what Dr Santra was saying about the clinical implications of SB’s inability to control his blood phenylalanine levels to levels falling consistently within the target range,” said Andrews.

She added, “Whilst it is open to an IFR panel to disagree with the requesting clinicians if they have valid reasons for doing so, a decision based on a misinterpretation of what the clinicians are saying will be fundamentally flawed, and so it is with this one.”

An NHS England spokesman said, “While the outcome of this case is limited to the particular circumstances of this funding request and does not have any wider implications for how NHS England makes decisions regarding the funding of treatment, we will of course take a further look at the IFR in question in the light of any further information provided to us.”

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