Original Title: Arzneimittel-Richtlinie/Anlage XII: Bedaquilin (Neues Anwendungsgebiet: Multiresistente pulmonale Tuberkulose, 5 bis 11 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: This benefit assessment of bedaquilin (Sirturo) relates to the treatment of paediatric patients (aged 5 years to less than 12 years and weighing at least 15 kg) with multidrug-resistant pulmonary tuberculosis (MDR-TB) as part of an appropriate combination therapy when an effective treatment regimen cannot be otherwise composed due to resistance or intolerance. The benefit assessment is based on the pivotal study TMC207-C211 (C211), a single-arm, open-label, multicentre, phase II study investigating the pharmacokinetics, safety, tolerability and antibacterial activity of bedaquilin in combination with a standard therapy for the treatment MDR-TB in children and adolescents aged 0 months to < 18 years with confirmed or probable MDR-TB. At time of assessment, the study was ongoing with a planned study population of 60 subjects in 4 age cohorts. Treatment phase with bedaquilin lasted 24 weeks. All patients were followed up to 120 weeks after baseline. Primary endpoints include the evaluation of safety, tolerability and the pharmacokinetics of bedaquilin during 24 weeks of treatment and the derivation of a dose recommendation for each age cohort. The present assessment only considered cohort 2 (≥ 5 to < 12 years 1; n=15). The effect of bedaquilin on mortality in the population of patients aged ≥ 5 to < 12 years could not be assessed as no deaths were observed until the data cut on 10.01.2019. No valid data on patient-relevant outcomes of morbidity could be considered. For quality of life, no data was available. The severe and serious AE observed in the study refer to AE of the liver and liver-related abnormal laboratory values. The significance of the results is low due to the small sample size and the uncontrolled design of the study.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/676/#english

Year Published: 2021

URL for published report: https://www.g-ba.de/downloads/39-1464-5023/2021-09-16_AM-RL-XII_Bedaquilin_D-672_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/676/#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/