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New HTA Decisions in England
August 2021
Drug name
PEMARYZE® (pemigatinib)
Incyte Corporation
Decision date
Therapeutic area
Therapeutic sub area
Liver cancers
Pemigatinib is indicated 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.
Decision Detail
It is recommended only if the company provides pemigatinib according to the commercial arrangement (simple discount patient access scheme).
The main clinical evidence for pemigatinib came from FIGHT-202, which was a phase 2, single-arm, non-randomised, open label study in people with advanced or surgically unresectable cholangiocarcinoma that had not responded to previous therapy. The results suggests that pemigatinib may be more effective than current treatments. The committee noted the uncertainty of this due to FIGHT-202 being a single-arm study. However, considering the rarity of this cancer, the committee acknowledged the difficulty in conducting studies for advanced chemorefractory cholangiocarcinoma and collecting robust comparative data. It concluded that, in the absence of direct evidence, indirect comparisons were needed to assess the relative effectiveness of pemigatinib compared with the comparators, and that the uncertainty is considered acceptable. In the absence of direct comparative evidence, the estimate of the relative treatment effect of pemigatinib compared with mFOLFOX+ASC and ASC alone was based on an unanchored matching adjusted indirect comparison of patient-level data from FIGHT-202 and data from ABC-06. ABC-06 was done in a different population to FIGHT-202 and did not report FGFR2 mutation status in either treatment group. The committee noted the lack of direct comparative evidence and the limitations of using a matching adjusted indirect comparison to compare the efficacy of pemigatinib with the comparators. However, it acknowledged that the company had identified all the available data to validate the survival estimates, given the rarity of the cancer. Using the committee's preferred assumptions for decision making, the ICER for pemigatinib was between £45,051 and £45,808 per QALY gained compared with mFOLFOX+ASC, and between £44,354 and £45,010 per QALY gained compared with ASC alone. The ICER value depended on whether the FIGHT 202 data were adjusted for the mFOLFOX+ASC or ASC alone data from ABC 06. Other scenarios resulted in higher ICERs. The committee considered the uncertainty in the clinical evidence but noted the rarity of the cancer being appraised. It concluded that the cost-effectiveness estimates for pemigatinib suggest it is an acceptable use of NHS resources for a life-extending treatment at the end of life. So pemigatinib was recommended for routine use in the NHS.