OBJECTIVES: Myasthenia gravis (MG) is a chronic autoimmune neurological disorder characterized by fatigable muscle weakness resulting from defective transmission at the neuromuscular junction. We assessed evidence gaps in the literature for the burden of disease and treatment of MG.

METHODS: A targeted review of literature published between May 4, 2013, and May 4, 2023 was conducted in PubMed, Embase, and the Cochrane Library using a predefined search strategy for articles describing the disease; epidemiology; clinical, humanistic, and economic burden; and treatment patterns of MG. Online searches were also conducted for health technology assessments, prescribing information, treatment guidelines and clinical trials.

RESULTS: We identified and reviewed 251 articles. Our analysis indicated that previous epidemiologic estimates may not reflect the current disease landscape. Symptoms and comorbidities of MG are well studied, but differences by autoantibody subtype, age of onset, and geography are not reported adequately. Various instruments have been used to assess the humanistic burden, including tools specific for MG, neurological disorders, psychological disorders, and fatigue, as well as generic instruments. However, detailed studies on differences across subgroups are needed. Studies reporting cost in MG vary due to differences in health care resources included in the analyses. The cost of an initial exacerbation in the United States exceeded $43,000, with additional MG-related events costing over $24,000. Recent developments in the therapeutic landscape necessitate updates to these analyses. Besides traditional non-specific immunosuppressive agents, recently available biologics (e.g., eculizumab, ravulizumab, efgartigimod, rozanolixizumab, zilucoplan) are increasingly used in MG to target specific components of the immune pathway. Therefore, treatment pattern studies are needed to assess the real-world effectiveness of emerging therapies.

CONCLUSIONS: We identified several gaps in the literature, including the need for comprehensive studies to advance our understanding of the epidemiology, humanistic and economic burden, as well as the evolving treatment pathways in MG.