[Pharmaceutical Directive/Annex XII: Idebenone (reassessment after the deadline: Leber's Hereditary Optic Neuropathy)]

The Federal Joint Committee [Gemeinsamer Bundesausschuss] (G-BA)
Record ID 32018004246
English, German
Original Title: Idebenon (Lebersche hereditäre Optikusneuropathie) – Neubewertung nach Fristablauf
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: Idebenone is approved for the treatment of visual impairment in adolescent and adult patients with Leber's hereditary optic neuropathy. The benefit assessment is a reassessment after the deadline. The initial benefit assessment was conducted in 2016 on the basis of the double-blind, randomised, placebo-controlled study RHODOS. For the current benefit assessment, the RHODOS study was reassessed. In addition, the results of the PAROS study, a prospective, register-based, non-controlled safety study, and the LEROS study, a prospective, non-controlled intervention study, were considered. No deaths were observed in the RHODOS study. One death occurred in each of the PAROS and LEROS studies. In the morbidity category, only results on visual acuity are available. In the RHODOS study, only a statistically significant group difference in the response criterion CRR 0.2 in favour of idebenone was observed with a high risk of bias. Due to the lack of a valid comparison, no conclusions can be drawn from the LEROS study on the extent of the effectiveness of idebenone in regard to the heterogeneous disease characteristics and given the possibility of spontaneous regressions. Overall, only minor differences in the incidence of adverse events between the study arms were found in the RHODOS study, which occurred numerically to the disadvantage of idebenone. The overall risk of bias was judged to be low. Only a few severe AEs and SAEs were observed. Overall, the short study duration is a fundamental limitation of the RHODOS study. However, given the lack of a valid comparison, no conclusive assessment of the safety of idebenone can be made on the basis of the LEROS and PAROS studies.
Project Status: Completed
Year Published: 2022
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
  • Optic Nerve Diseases
  • Optic Atrophy, Hereditary, Leber
  • Ubiquinone
  • Antioxidants
  • Leber's hereditary optic neuropathy
  • adult
  • adolescent
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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