OBJECTIVES: To evaluate the cost-effectiveness of pemigatinib compared to oxaliplatin-L-folinic-acid and fluorouracil plus active symptom control (mFOLFOX+ASC) and ASC alone for the treatment of patients with advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) rearrangement or fusion who have progressed on at least one line of prior systemic therapy in Greece.

METHODS: A partitioned survival model, with five health states, was locally adapted from a Greek payer perspective over a lifetime horizon. Clinical and safety data as well as utility values were extracted from the literature. A matching-adjusted indirect comparison of pemigatinib and mFOLFOX+ASC was performed. Reimbursable direct costs pertaining to drug acquisition, administration, monitoring, adverse events, and end of life were considered in the analysis. All cost inputs were indexed to 2023 euros. Primary outcomes were patients’ life years (LYs), quality-adjusted life years (QALYs), total costs and incremental cost-effectiveness ratios (ICERs) per QALY and LY gained. All future outcomes were discounted at 3.5% per annum. Sensitivity analyses were conducted.

RESULTS: Total lifetime cost per patient with pemigatinib, mFOLFOX+ASC and ASC alone were estimated to be €85,534, €2,537 and €1,010 respectively. In terms of health outcomes, pemigatinib was associated with 1.78 and 1.84 increment in LYs compared with mFOLFOX+ASC and ASC alone. Furthermore, pemigatinib appeared to yield more QALYs gained versus mFOLFOX+ASC and ASC alone (1.66 vs 0.44 and 0.41, respectively), resulting in ICERs of €69,928 per QALY gained and €46,626 per LY gained versus mFOLFOX+ASC and €69,345 per QALY gained and €45,935 per LY gained compared to ASC alone. Sensitivity analyses confirmed the robustness of base-case results.

CONCLUSIONS: Pemigatinib, a therapy covering the unmet medical need of patients with previously treated locally advanced or metastatic CCA with an FGFR2 fusion or rearrangement, was estimated to be cost-effective compared with mFOLFOX+ASC and ASC alone in Greece.