Fedratinib is recommended as an option for treating disease-related splenomegaly or symptoms of primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis. It is recommended for adults, only if:
they have had ruxolitinib, and
momelotinib is unsuitable, and
the company provides fedratinib according to the commercial arrangement.
This recommendation is not intended to affect treatment with fedratinib 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 healthcare professional consider it appropriate to stop.
Why the committee made these recommendations
This evaluation reviews the evidence for fedratinib for treating disease-related splenomegaly or symptoms in myelofibrosis (NICE technology appraisal guidance 756). It also reviews new data collected as part of the managed access agreement.
Most people with higher risk myelofibrosis have ruxolitinib (a JAK inhibitor). After stopping ruxolitinib, people can have best available therapy, which includes hydroxycarbamide, other chemotherapies, androgens, splenectomy, radiation therapy, erythropoietin, and red blood cell transfusions. Some people with moderate to severe anaemia also have momelotinib. The company asked for fedratinib to be considered only for use after ruxolitinib and when momelotinib is unsuitable. This does not include everyone who it is licensed for.
Evidence from a clinical trial suggests that fedratinib reduces spleen volume and symptoms more than best available therapy. But it is not clear if people having fedratinib live for longer than people having best available therapy.
Because of uncertainty in the long-term clinical evidence, the cost-effectiveness estimates are uncertain. But the most likely estimates are within the range that NICE considers an acceptable use of NHS resources. So, fedratinib is recommended.