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1.2 This recommendation is not intended to affect treatment with elosulfase alfa that was started in the NHS before this guidance was published.
Why the committee made these recommendations
This guidance reviews the evidence for elosulfase alfa for treating MPS 4A, including new evidence collected as part of the managed access agreement (NICE highly specialised technology guidance HST2).
MPS 4A is rare and progressive, and has a significant effect on the quality of life of people with the condition, and their families and carers. It causes abnormalities in the joints and bones, respiratory symptoms, pain, fatigue and increasing dependence on a wheelchair. Current treatment options are limited.
Clinical trial evidence and data collected as part of the managed access agreement suggest that MPS 4A becomes stable in the long term with elosulfase alfa.
However, the company used the same limited economic model structure for the review that it had used for the original guidance. Also, its assumptions in model are not robust or plausible, particularly around:
using a model that relies on wheelchair use because it does not represent well enough how the condition progresses
making assumptions about treatment benefit rather than using the observed data
body weight because this is likely to change over time.
The long-term benefits of elosulfase alfa are not appropriately captured in the model. The extra health and quality-of-life benefits of elosulfase alfa are considered to be substantial. But the cost-effectiveness estimates are much higher than what NICE considers acceptable for highly specialised technologies. So, elosulfase alfa is not considered an appropriate use of NHS resources within the context of a highly specialised service, and cannot be recommended.