Análisis de decisión multicriterio sobre los tratamientos para la miastenia gravis generalizada en España

Objective: To quantify the relevance of criteria that could be impacting the treatment of generalized Myasthenia Gravis with nicotinic acetylcholine receptor antibodies (AChR Ab+ MGg) from a multi-stakeholder perspective in the Spanish context. Methods: The study followed the Multi-Criteria Decision Analysis good practice guidelines and the rare diseases framework: identify relevant evidence through literature review; determine the criteria relevance (weighting); assess the intervention (scoring); and calculate its added value. The study included the participation of a multidisciplinary panel (n=11) and considered the best current therapy (off-label use of rituximab) and two new agents (ravulizumab and efgartigimod alfa) as treatment options for adults with MGg AChR- Ab+. Results: MGg AChR-Ab+ was defined as a severe disease due to its debilitating and unpredictable symptoms, with a high impact on patients’ and caregivers’ quality of life. Participants identified several unmet needs in uncontrolled patients: improve patient stability and ensure treatment continuity. Experts perceived ravulizumab and efgartigimod alfa as two highly effective options with a greater degree of evidence than rituximab, which could have a positive impact on uncontrolled MGg AChR-Ab+ patients. Both treatments provide rapid control of symptoms with a manageable and acceptable safety profile. Efgartigimod alfa delivers pronounced responses and requires subsequent treatment cycles, while ravulizumab shows sustained improvements and a fixed dosing schedule. Conclusions: Study participants considered that both, ravulizumab and efgartigimod alfa, could provide value for the management of uncontrolled MGg AChR-Ab+ at different levels, being necessary to individualise decision-making according to the patient profile and specific needs.