Resource impact statement
NICE has recommended cipaglucosidase alfa plus miglustat as an option for treating late-onset Pompe disease in adults. It is recommended only if the company provides it according to the commercial arrangement.
We expect the resource impact of implementing the recommendations in England will be less than £5 million per year (or approximately £8,800 per 100,000 population, based on a population for England of 56.6 million people).
This is because the technology is an alternative treatment option to existing enzyme replacement therapies. The overall cost of treatment for this patient group will be similar and the population size is small.
Cipaglucosidase alfa and the other treatment options have discounts that are commercial in confidence. For enquiries about the patient access schemes, contact the manufacturers of these technologies.
This technology is commissioned by NHS England. Providers are NHS hospital trusts.
The payment mechanism for the technology is determined by the responsible commissioner and depends on the technology being classified as high cost.
This page was last updated: