Pemigatinib for treating relapsed or refractory advanced cholangiocarcinoma with FGFR2 fusion or rearrangement
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1.1 Pemigatinib is not recommended, within its anticipated marketing authorisation, for treating locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after systemic therapy in adults.
1.2 This recommendation is not intended to affect treatment with pemigatinib 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
Current treatment for advanced cholangiocarcinoma with an FGFR2 fusion or rearrangement that has progressed after systemic therapy is symptom control, with or without modified folinic acid, 5-fluorouracil and oxaliplatin (mFOLFOX) chemotherapy.
Clinical evidence suggests that pemigatinib may be more effective than current treatments, but this is highly uncertain because the trial did not directly compare pemigatinib with symptom control or mFOLFOX. The results of an indirect comparison are very uncertain.
Pemigatinib meets NICE's criteria for a life-extending treatment at the end of life. But the most likely cost-effectiveness estimates are higher than what NICE considers a cost-effective use of NHS resources. Pemigatinib does not meet the criteria for inclusion in the Cancer Drugs Fund. So pemigatinib is not recommended.