Original Title: Arzneimittel-Richtlinie/Anlage XII: Pemigatinib (Cholangiokarzinom mit FGFR2-Fusion oder FGFR2-Rearrangement, nach mindestens 1 Vortherapie)

Authors' objectives: The Federal Joint Committee [Gemeinsamer Bundesausschuss (G-BA)] has had the legal task of carrying out an (additional) benefit assessment for all newly approved drugs with new active ingredients immediately after market entry (§ 35a SGB V). The result of this assessment is the basis for deciding how much the statutory health insurance pays for a new drug with a new active ingredient. The G-BA was commissioned to carry out the benefit assessment through the Pharmaceuticals Market Reorganisation Act [Gesetz zur Neuordnung des Arzneimittelmarktes (AMNOG)]. In the context of the early benefit assessment of medicinal products containing new active substances, the following rules apply to orphan drugs: According to the legal requirements (§ 35a SGB V), the additional medical benefit of these drugs is already considered to be proven by the approval. The G-BA determines the extent of the additional benefit for orphan drugs that do not exceed a turnover of 50 million Euros in the last twelve calendar months, on the basis of the approval and the studies justifying the approval.

Authors' results and conclusions: Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy. The benefit assessment is based on the pivotal study FIGHT-202, an open-label, uncontrolled, multicentre phase II study evaluating the efficacy and safety of Pemazyre in patients with advanced/metastatic or unresectable cholangiocarcinoma who have received prior therapy. The assessment is based on a cohort of patients within the total study population, namely patients (≥ 18) with FGFR2 fusion or FGFR2 rearrangement according to genetically tested laboratory results. This cohort included 107 patients at data cut. All patients received Pemazyre in consecutive 21-day therapy cycles. Primary endpoint was the objective response rate (ORR) in patients with FGFR2 translocations based on centrally measured laboratory results. An external control study submitted with the dossier was not considered in the benefit assessment due to bias. An adequate comparative analysis on overall survival was not available. More than half of the patients died during the median follow-up period of 17.5 months. During the first 6 cycles of therapy, there were no strong mean or median changes in patient-reported symptoms. During the first 3 cycles, no strong mean or median changes could be reported in patient-reported health-related (EORTC QLQ-C30) and disease-specific (EORTC QLQ-BIL21) quality of life. Changes in quality of life between baseline and cycle 3 or 6 were small for all scales with less than 10 points, often less than 5 points. All patients experienced AE during treatment phase. Approximately two-thirds experienced severe AE and 40% serious AE. AE of special interest occurred in 8 of 10 participants. Due to the uncontrolled study design and high risk of bias, no conclusive evaluation the results can be made.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/677/#english

Year Published: 2021

URL for published report: https://www.g-ba.de/downloads/39-1464-5049/2021-10-07_AM-RL-XII_Pemigatinib_D-670_EN.pdf

Requestor: The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/677/#nutzenbewertung

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: Germany

Organisation Name: The Federal Joint Committee

Contact Address: Gutenbergstr. 13, 10587 Berlin, Germany

Contact Name: Fachberatung Medizin [Department of Medical Consultancy]

Contact Email: Fachberatung-Medizin@g-ba.de

Copyright: https://www.g-ba.de/sys/impressum/