Objective  To evaluate the efficacy, safety and economy of efgartigimod alfa in the treatment of generalized myasthenia gravis by rapid health technology assessment, and to provide evidence-based evidence for clinical rational drug use.

Methods  PubMed, Embase, Web of Science, Cochrane Library, CNKI, VIP, WanFang Data, SinoMed database and relevant databases of health technology assessment institutions were electronically searched to collect health technology assessment reports, systematic reviews/Meta-analysis and pharmacoeconomic literatures of efgartigimod alfa for the treatment of generalized myasthenia gravis from the inception to June 21, 2024. Two researchers screened the literature, extracted data, and evaluated the quality of the literature, summarised the finding and performed  qualitative descriptive analysis.

Results  A total of 6 literature were included, involving 5 systematic reviews/Meta-analysis and one pharmacoeconomic study. In terms of efficacy, efgartigimod alfa demonstrated a significant reduction in MG-ADL, QMGs, and MG-QOLs 15R compared to placebo among generalized myasthenia gravis patients, these differences were statistically significant (P<0.05). However, findings from different studies regarding comparisons with other biologics like batoclimab, ronzanolixizumab, and eculizumab yielded inconsistent conclusions. In terms of safety, the incidence of adverse events in adults generalized myasthenia gravis patients treated with efgartigimod alfa compared with that of the placebo was not statistically significant (P>0.05). However, compared with other biological agents, the conclusions drawn from each study were inconsistent. In terms of economics, efgartigimod alfa did not exhibit cost-effectiveness advantages over traditional therapies.

Conclusion  Efgartigimod alpha showed better efficacy than placebo in the treatment of generalized myasthenia gravis, but there was no definitive conclusion compared with other biological agents. In terms of safety, the incidence of ADE in adult generalized myasthenia gravis patients treated with efgartigimod alpha was not statistically different from that of the placebo group, and no definite conclusion could be drawn when compared with other biological agents. According to U.S. drug economic data, efgartigimod alpha does not have economic advantages over traditional therapies.

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