NICE (UK): recommended to treat systemic mastocytosis – Blueprint Medicines

Avapritinib is recommended, within its marketing authorisation, as an option for treating advanced systemic mastocytosis (including aggressive systemic mastocytosis, systemic mastocytosis with an associated haematological neoplasm and mast cell leukaemia) in adults. Avapritinib is only recommended if the company provides it according to the commercial arrangement.

Why the committee made this recommendation; Standard treatments for advanced systemic mastocytosis include midostaurin and cladribine. Midostaurin is used at first line when possible, with cladribine mostly being used at second line or later. Evidence from clinical trials suggests that avapritinib increases how long people have before their condition gets worse and how long they live. But avapritinib was not compared with any other treatments in these trials, so how it compares with them is uncertain. An indirect comparison suggests that avapritinib increases how long people live compared with midostaurin at first line and with cladribine at second or later lines.. Despite the uncertainty in the clinical-effectiveness evidence, the cost-effectiveness estimate for avapritinib is within the range that NICE considers an acceptable use of NHS resources. So, avapritinib is recommended.