Midostaurin for untreated acute myeloid leukaemia

1.1 Midostaurin is recommended, within its marketing authorisation, as an option in adults for treating newly diagnosed acute FLT3-mutation-positive myeloid leukaemia with standard daunorubicin and cytarabine as induction therapy, with high-dose cytarabine as consolidation therapy, and alone after complete response as maintenance therapy. It is recommended only if the company provides midostaurin with the discount agreed in the patient access scheme.

Why the committee made these recommendations

Treatment for acute myeloid leukaemia is chemotherapy. Evidence from a randomised controlled trial shows that people taking midostaurin with chemotherapy live longer than people taking chemotherapy alone.

There is uncertainty about the cost effectiveness of midostaurin because of problems with the economic model. But with the most plausible model assumptions and the discounted price, the cost-effectiveness estimates of midostaurin plus chemotherapy compared with chemotherapy alone are within the range that NICE normally considers a cost-effective use of NHS resources, so midostaurin is recommended.