Original Title: Arzneimittel-Richtlinie/Anlage XII: Luspatercept (Myelodysplastische Syndrome (MDS))

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: Reblozyl is indicated for the treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. The benefit assessment is based on the pivotal study MEDALIST (ACE-536-MDS-001), a double-blind, randomized, phase III, controlled trial evaluating the efficacy and safety of reblozyl compared to placebo in patients with anaemia due to MDS with very low, low or intermediate risk according to IPSS-R, with ring sideroblasts and transfusion dependence. The primary treatment phase lasted 24 weeks. Study population included 229 patients (≥ 18 years) stratified by transfusion burden and IPSS-R risk and randomized in a 2:1 ratio to two treatment arms, reblozyl plus best supportive care (BSC) vs. placebo plus BSC with 153 and 76 patients, respectively. Primary endpoint was erythrocyte transfusion independence for at least eight continuous weeks during primary treatment phase. Results regarding mortality show no statistically significant difference in overall survival between reblozyl therapy compared with placebo. Although highly biased the physical function scale regarding quality of life showed as statistical significant disadvantage for Reblozyl compared to placebo. No difference was observed for hospitalisation between the two study arms. Patients taking reblozyl had a statistically significant higher chance of AE of any severity in SOC “Gastrointestinal disorders” and “Nervous system disorders” and in PT “Asthenia” and “Dizziness”, yet no clear conclusions can be drawn regarding safety and long-term safety based on missing information for this endpoint and the short observation period.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/572/#english

Year Published: 2021

URL for published report: https://www.g-ba.de/downloads/39-1464-4666/2021-01-21_AM-RL_XII_Luspatercept_MDS_D-561_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/572/#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/