Clinical efficacy and cost per improved outcome of treatments for generalized myasthenia gravis: evidence from a network meta-analysis and cost-per-responder analysis

Objective To indirectly compare the efficacy and value of biologic therapies for acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG) using a network meta-analysis (NMA) of pivotal trials and a cost-per-responder analysis.Methods A de novo NMA was performed using data from Phase III placebo-controlled trials of efgartigimod IV, inebilizumab, nipocalimab, ravulizumab, rozanolixizumab, and zilucoplan, including recently approved therapies. Outcomes included ≥3- and ≥5-point improvements, and continuous changes, in Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores. Treatment effects, ranking probabilities, and the surface under the cumulative ranking curve (SUCRA) values were estimated. Numbers needed to treat (NNTs) versus placebo were derived from NMA results. Cost per improved outcome (CPIO) was calculated using NNTs and 2026 US drug/administration costs.Results Most therapies demonstrated significantly greater efficacy than placebo across MG-ADL and QMG outcomes. Efgartigimod IV ranked highest across nearly all endpoints considering the probability of being the most effective and SUCRA values. It had the lowest NNTs for QMG improvement (2.18 [≥3-point]; 1.88 [≥5-point]), significantly lower than almost all treatments for QMG ≥3-point, and significantly lower than zilucoplan for QMG ≥5-point (p < 0.05). It had MG-ADL NNTs of 2.77 (at least 3-point) and 1.94 (at least 5-point), with the latter significantly lower than ravulizumab (4.68) and zilucoplan (4.78) (p < 0.05). Efgartigimod IV had the lowest CPIO across all QMG and MG-ADL thresholds, significantly lower than almost all other treatments (p < 0.05).Conclusions Among approved effective biologic therapies for gMG, efgartigimod IV offers a favorable combination of clinical efficacy and economic value.