Fedratinib for treating disease-related splenomegaly or symptoms in myelofibrosis

1.1 Fedratinib is recommended for use within the Cancer Drugs Fund as an option for treating disease-related splenomegaly or symptoms of primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis in adults. It is recommended only if:

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 more restrictive than its marketing authorisation.

Clinical trial evidence for people who have stopped ruxolitinib 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 been compared indirectly with best available therapy using evidence from other studies. There is further uncertainty because of some differences between the trial populations in the indirect comparison.

Also, it is unclear how much longer people having fedratinib live compared with best available therapy, and this has a large effect on the cost-effectiveness results. There is also uncertainty around how many people would continue having fedratinib if their disease does not fully respond, or stops responding.

Fedratinib does not meet NICE's criteria to be considered a life-extending treatment at the end of life based on the evidence currently available. The cost-effectiveness estimates for fedratinib compared with best available therapy are uncertain because of limitations in the data. Because some of these estimates are higher than what NICE normally considers an acceptable use of NHS resources, fedratinib cannot be recommended for routine use in the NHS. Collecting more data on overall survival and treatment duration will reduce the uncertainty in the evidence. Therefore, fedratinib is recommended for use in the Cancer Drugs Fund.