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Fedratinib for treating disease-related splenomegaly or symptoms in myelofibrosis

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1 Recommendations

1.1 Fedratinib is not recommended, within its marketing authorisation, for treating disease-related splenomegaly or symptoms of primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis in adults who have not had a Janus kinase (JAK) inhibitor or who have had ruxolitinib.

1.2 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 clinician consider it appropriate to stop.

Why the committee made these recommendations

Most people with higher risk myelofibrosis have ruxolitinib, and continue having it even if their disease does not fully respond, or stops responding. After ruxolitinib is stopped, people can have best available therapy, which includes chemotherapy, radiation therapy, splenectomy or red blood cell transfusion. The company proposes that fedratinib would only be used after ruxolitinib, which is narrower than the marketing authorisation for fedratinib.

Clinical trial evidence in this population suggests that fedratinib improves myelofibrosis symptoms and reduces spleen size. However, this evidence is uncertain because fedratinib was not compared with best available therapy and some people did not finish the trial. Fedratinib has only been compared indirectly with best available therapy using evidence from other trials. There is further uncertainty because of some differences between the trial populations in the indirect comparison.

Also, it is not clear how much longer people having fedratinib live compared with best available therapy, and which treatments would be used after fedratinib. These uncertainties have a large effect on the cost-effectiveness results.

Fedratinib does not meet NICE's criteria to be considered a life extending treatment at the end of life. Also, the cost-effectiveness estimates for fedratinib compared with best available therapy are higher than what NICE normally considers an acceptable use of NHS resources. It is unlikely that collecting more data in the Cancer Drugs Fund would resolve the uncertainties in the evidence. So, fedratinib is not recommended for routine use or through the Cancer Drugs Fund.