1.1 Midostaurin monotherapy is recommended, within its marketing authorisation, as an option for treating aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm, or mast cell leukaemia in adults. It is recommended only if the company provides midostaurin according to the commercial arrangement.
Why the committee made these recommendations
There is no standard treatment for advanced systemic mastocytosis (aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm, or mast cell leukaemia). 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 this is uncertain because it was not compared directly with these. Also, better quality comparative evidence is unlikely to become available.
Midostaurin meets NICE's criteria for a life-extending treatment at the end of life, which means that higher cost-effectiveness estimates can be considered. This means that, despite the uncertainty about the clinical evidence, the cost-effectiveness estimates are within the range that NICE considers acceptable. So, midostaurin is recommended.