Original Title: Arzneimittel-Richtlinie/Anlage XII: Tisagenlecleucel (Neubewertung nach Fristablauf: Akute lymphatische B-Zell-Leukämie, rezidiviert / refraktär, 0 ≤ 25 Jahre)

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: Tisagenlecleucel is approved for the treatment of people with relapsed or refractory acute lymhoblastic leukemia. The benefit assessment of Tisagenlecleucel is based on the pivotal ELIANA study, a one-arm, open-label, and multicentric Phase II trial to investigate the safety and efficacy. Furthermore, two further clinical trials were assessed in the benefit assessment (ENSIGN; B2001X). An indirect comparison was rejected because of methodological shortcomings and limited comparability of study populations between clinical and real-world populations. Additionally, not all relevant confounders were taken into the model. Therefore it is not possible to draw conclusions on mortality and morbidity endpoints due to the missing control group. Side effects were mainly collected between the day of infusion and three months after infusion. Nearly every UE of special interest occurred in the trial, especially the Cytokine Release Syndrom. The safety assessment is limited due to differences in reporting between treatment phases and the missing control group.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/990/#english

Year Published: 2023

URL for published report: https://www.g-ba.de/downloads/39-1464-6478/2024-02-15_AM-RL-XII_Tisagenlecleucel_D-976_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/990/#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/