PND10 Conceptualization and Novel Budget Impact Analysis Framework for Treatments of Myasthenia Gravis

Myasthenia gravis (MG) is a rare chronic autoimmune neuromuscular disorder, which causes debilitating and potentially life-threatening exacerbations of muscle weakness. The objective this study was to develop novel United States (US) budget impact analysis framework assess innovative MG treatments. A targeted literature review conducted identify previous economic evaluations MG. Clinical experts were consulted ensure model components reflected current clinical practice understanding. US payers engaged provide feedback on the structure outputs, how optimize utility for informing formulary decision-making. Few analyses treatments identified in literature. Recent data direct pharmacy medical costs sparse. developed assessing an therapy, includes treatment-related offsets. Modeled include treatments, therapy adverse effects, myasthenic crisis events. acute inpatient, emergency department outpatient care as well temporary treatment with immunoglobulin or plasma exchange following exacerbations. recommended that comparators should all combinations conventional add-on therapies reflect individualized pathways observed practice. Conventional corticosteroids, non-steroidal immunosuppressive drugs (e.g. azathioprine, mycophenolate mofetil) acetylcholinesterase inhibitors. flexibility adapt market shares time horizons conduct alternative scenario analyses. Future capture both including those related disease effects. core essential full costs.