Original Title: Arzneimittel-Richtlinie/Anlage XII: Idebenon – Verlängerung 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: Idebenone is approved for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). The benefit assessment of Idebenone is based on the pivotal RHODOS study, a multi-centre, double-blind, randomised, placebo-controlled, parallel phase II study, which evaluated the efficacy and safety of idebenone (3x daily 300mg) compared with placebo over 24 weeks. The study included 85 patients aged 14 to 65 with LHON. Endpoints investigated in the RHODOS study included the best improvement in visual acuity in one eye after 24 weeks (primary endpoint), the change in best visual acuity after 24 weeks of treatment, the change in visual acuity of the best eye at start of study after 24 weeks, colour contrast sensitivity, adverse events and quality of life. With respect to the visual acuity endpoints no statistically significant differences were found between the study arms after 24 weeks except within the endpoint regarding the change in colour contrast sensitivity. Whereas no statistically significant difference between the study arms regarding the perception of red-green (protan) was found, the perception of the colours yellow-blue (tritan) showed a statistically significant advantageous effect of idebenone compared to placebo (difference between the groups: −13.63 ± 5.05 (95% CI): [−23.61; −3.66]; p = 0.008). This was due to a decrease in colour confusion in the idebenone group and an opposite change after 24 weeks in the placebo group. However, the colours red-green and yellow-blue were measured only in one study centre. Results are therefore available for only one sub-group. In addition, the analysis is based on the number of eyes. The proportion of patients with an improvement in colour contrast sensitivity remains unclear. No usable data on quality of life were available and no statistically significant differences between the idebenone and placebo treated patient groups were found with regard to side effects.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/192/#english

Year Published: 2016

URL for published report: https://www.g-ba.de/downloads/39-1464-2527/2016-03-17_AM-RL-XII_Idebenon_D-191_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/192/#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/