Original Title: Arzneimittel-​​​Richtlinie/Anlage XII: Ixazomib – Änderung der Befristung der Geltungsdauer

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: According to the marketing authorisation, Ixazomib in combination with Lenalidomide and Dexamethasone is indicated for the treatment of multiple myeloma in adult patients who have received at least one prior therapy. The pivotal study, C16010, is a randomised, double-blind, placebo-controlled, multicentre study to assess the efficacy and safety of Ixazomib in combination with Lenalidomide and Dexamethasone (Ixazomib+LenDex) compared to placebo in combination with Lenalidomide and Dexamethasone (placebo+LenDex) in adult patients with relapsed/refractory multiple myeloma who have received at least one prior therapy. Summary of efficacy: With low potential bias, there were no statistically significant differences between Ixazomib+LenDex and placebo+LenDex for the morbidity (BPI-SF and EQ-5D) and mortality (OS) considered. Based on two interim analyses, the overall survival results were based on 22% and 35% of planned events (n=486). A final assessment of the endpoint is possible at the time of the final analysis in 2020. The primary endpoint PFS is not considered in the benefit assessment. Quality of life summary: With a low potential for bias and taking into account the usability of the available results, the observed mean changes in quality of life on the pre-specified scales (EORTC QLQ-C30 and EORTC QLQ-MY20) do not show a significant difference between the treatment arms at any time point. Safety summary: With low potential for bias, there is no evidence of an increased adverse event profile with treatment with Ixazomib+LenDex compared with placebo+LenDex. Adverse events of special interest associated with increased risk from the intervention include skin and subcutaneous disorders and ocular disorders. See also Record ID 32018002474

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/275/#english

Year Published: 2019

URL for published report: https://www.g-ba.de/downloads/39-1464-3945/2019-09-05_AM-RL-XII_Ixazomib_D-272_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/275/#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/