Original Title: Arzneimittel-​Richtlinie/Anlage XII: Mexiletin

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: Mexiletin is approved for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic disorders. The benefit assessment of Mexiletin is based on the pivotal study MYOMEX (NCT02336477), a randomised, double-blind, placebo-controlled, multicentre, cross-over Phase III study to evaluate the efficacy and safety of Mexiletin in people with myotonia congenita (MC) and paramyotonia congenita (PC). The study included 26 people with MC and PC aged between 18 and 65 years. In the combined analysis of both treatment sequences, a statistically significant and clinically relevant decrease in VAS severity of muscle stiffness was observed. No significant differences in the incidence of muscle weakness and muscle blockage were observed between placebo and Mexiletin treatment. Pain and fatigue were significantly less common with Mexiletin treatment than with placebo. The potential for bias of all efficacy endpoints is considered unclear. For quality of life, a statistically significant effect in favour of Mexiletin was observed above the irrelevance threshold of 0.2 and -0.2 for hedges' g. The potential for bias of the quality-of-life endpoint INQoL is judged to be unclear. Within the MYOMEX study, 15 people (60%) had AE during the Mexiletin treatment and 9 people (36%) during the placebo treatment. A severe AE during Mexiletin treatment led to discontinuation of the study medication, SAE did not occur. There were no statistically significant differences in AE stratified by system organ class and preferred term. Overall, however, these analyses are only of limited value, as only a few events occurred due to the small number of individuals and short observation period.

Project Status: Completed

URL for project: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/438/#english

Year Published: 2019

URL for published report: https://www.g-ba.de/downloads/39-1464-3908/2019-08-01_AM-RL-XII_Mexiletin_D-427_EN.pdf

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

URL for additional information: https://www.g-ba.de/bewertungsverfahren/nutzenbewertung/438/#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/