Resource impact statement
NICE has recommended sapropterin as an option for treating hyperphenylalaninaemia that responds to sapropterin (response as defined in the summary of product characteristics) in people with phenylketonuria (PKU), only if they are:
- under 18 and a dose of 10 mg/kg is used, only using a higher dose if target blood phenylalanine levels cannot be achieved at 10 mg/kg
- aged 18 to 21 inclusive, continuing the dose they were having before turning 18 or at a maximum dose of 10 mg/kg
- pregnant (from a positive pregnancy test until birth).
It is estimated that there will be fewer than 100 people under 18 years old, fewer than 20 people aged between 18 and 21 years old, and around 5 pregnant women per year, eligible for treatment with sapropterin in England. Therefore it is unlikely that the resource impact of implementing this guidance will meet the threshold for a resource impact report and template to be produced.
Sapropterin has a discount that is commercial in confidence.
This technology is commissioned by NHS England. Providers are NHS hospital trusts.
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