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Midostaurin for treating advanced systemic mastocytosis

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1 Recommendations

1.1 Midostaurin is not recommended, within its marketing authorisation, for treating advanced systemic mastocytosis (aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasms and mast cell leukaemia) in adults.

1.2 This recommendation is not intended to affect treatment with midostaurin that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

There is no standard treatment for advanced systemic mastocytosis. Current treatments include interferon alpha, pegylated interferon alpha, cladribine, imatinib, and treatments usually used for acute myeloid leukaemia. Midostaurin aims to treat the disease and its symptoms.

Evidence suggests that midostaurin is more effective than current treatments, but the evidence is uncertain. This is because midostaurin has been compared indirectly with current treatments using evidence from 1 unpublished study. Also, it is unlikely that higher quality comparative evidence will become available.

Midostaurin meets NICE's criteria for a life-extending treatment at the end of life. However, the cost-effectiveness estimates are much higher than what NICE normally considers a cost-effective use of NHS resources for end of life treatments. So, midostaurin is not recommended for routine use in the NHS. Because more evidence is unlikely to become available to help with the uncertainty, midostaurin is also not recommended for use within the Cancer Drugs Fund.