Pemigatinib (Pemazyre)

Abstract


 CADTH recommends that pemigatinib should not be reimbursed by public drug plans for the treatment of adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma (CCA) with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement.
 The clinical evidence reviewed by CADTH was not strong enough to show whether treatment with pemigatinib benefits patients with CCA. It is not known if pemigatinib would lead to better outcomes for patients compared to treatments that are currently available.
 Patients identified a need for treatments that improve tumour response, delay disease progression, and improve quality of life. It is not clear whether pemigatinib meets these needs.

Cost-effectiveness of pemigatinib as a second-line treatment for advanced intrahepatic cholangiocarcinoma with fibroblast growth factor receptor 2 fusions in Taiwan: from the evidence of the phase II trial and the perspective of Taiwan's National Health Insurance Administration

“…The model validation followed the Assessment of the Validation Status of Health-Economic decision models [ 26 ]. Our conceptual model followed the same approach adopted by NICE and CADTH [ 10 , 11 ]. We were unable to distinguish the mortalities attributed to the progressed state and the PF state due to the lack of individual-level data in the aforementioned trials.…”

“…Although the U.S. FDA accelerated the approvals of FGFR kinase inhibitors [ 4 , 6 , 7 ], high-priced pemigatinib has not been widely used or covered by third parties. Furthermore, the cost-effectiveness results of the National Institute for Health and Care Excellence (NICE) [ 10 ] and the Canada’s Drug and Health Technology Agency (CADTH) [ 11 ] were inconsistent. In Taiwan, the biomarker-driven regimen of pemigatinib and mFOLFOX recommended by the NCCN was not shown to be cost-effective compared to the current 5-FU regimen based on the pemigatinib unit price of NT$14,285, unless there was a 40% reduction in the proposed price from the manufacturer [ 12 ].…”