[Pharmaceutical Directive/Annex XII: Bedaquiline (assessment of an orphan drug after exceeding the turnover limit of €1 million)]

The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
Record ID 32018002123
English, German
Original Title: Arzneimittel-Richtlinie/Anlage XII: Bedaquilin (multiresistente pulmonale Tuberkulose) – 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: Bedaquiline as part of an appropriate combination therapy is approved for the treatment of multi-drug-resistant Mycobacterium tuberculosis (MDR-TB) in adult patients when an effective treatment regimen cannot otherwise be established due to resistance or intolerance. The benefit assessment of bedaquiline is based on the pivotal study C208. Study C208 is a Phase II, mulicenter, randomized, double-blind, placebo-controlled study evaluating the antibacterial activity, efficacy, and safety of bedaquiline as part of combination therapy (N = 160). Efficacy: With regard to morbidity, there are statistically significant effects in favor of the bedaquiline group in patients with MDR-TB, with a high potential for bias. Quality of life: No data were collected on health-related quality of life. Safety: Based on the available data, there are general uncertainties about the safety of bedaquiline because of the imbalance in terms of deaths that occurred in the bedaquiline arm compared with the placebo arm. The imbalance in deaths is unexplained. Further data on the safety profile of bedaquiline are being collected as part of an ongoing STREAM study.
Project Status: Completed
Year Published: 2019
Requestor: The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
English language abstract: An English language summary is available
Publication Type: Full HTA
Country: Germany
MeSH Terms
  • Tuberculosis, Multidrug-Resistant
  • Antitubercular Agents
  • Extensively Drug-Resistant Tuberculosis
  • Mycobacterium tuberculosis
  • Bedaquiline
  • Tuberculosis – Multidrug-Resistant
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/
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