5 March 2020 - An 11-year-old girl with the rare disease phenylketonuria has lost a legal battle in the High Court over a decision by NICE to deny NHS access to the only approved drug in the UK.

The legal team representing Gail Cotter of Chelmsford in Essex challenged NICE in the courts over its decision to review BioMarin’s phenylketonuria therapy Kuvan (sapropterin) using its standard appraisal process, rather than its Highly Specialised Technology (HST) programme.

HST gives much more flexibility on cost when considering whether new drugs are cost-effective for NHS use, raising the threshold for cost-effectiveness from £30,000 per quality-adjusted life year (QALY) to £100,000.

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