Advice
NICE is unable to make a recommendation about the use in the NHS of sebelipase alfa for treating lysosomal lipase deficiency that is not Wolman disease. The Wolman disease population was evaluated separately in NICE highly specialised technology guidance on sebelipase alfa (HST30). The cost effectiveness of the remaining non-Wolman population has not been demonstrated at this stage.
Information
If NHS organisations wish to consider sebelipase alfa for this indication, they should follow the advice on rational local decision making in the NHS Constitution for England and the NHS Commissioning Board and Clinical Commissioning Groups (Responsibilities and Standing Rules) Regulations 2012. This outlines the approach that should be taken when there is no NICE guidance.
NICE will review this decision if the company makes a submission in the remaining non-Wolman population.
ISBN: 978-1-4731-5882-5