Unmet needs and gaps in the management of myasthenia gravis in Latin America: survey with experts and cost analysis

Background Generalized myasthenia gravis (gMG) is a rare autoimmune disease that manifests with fluctuating muscle weakness. Poor disease control may lead to myasthenic exacerbations and crises, increasing healthcare resource utilization (HCRU) and worsening quality of life. There is scant evidence on the patient profile, treatment patterns and HCRU for gMG in Latin America. We used a three-step approach comprising a targeted literature review, a modified Delphi panel involving experts’ individual response to a survey followed by country-level discussions, and a micro-costing analysis using a bottom-up costing approach to investigate the individual and societal impact of gMG in Argentina, Brazil, Colombia and Mexico. Results A qualitative assessment revealed several unmet needs and barriers, including disease awareness, a lack of specialized professionals, delayed diagnosis, limited access to tests and insufficient access to and coverage for innovative treatments. First therapy typically involves pyridostigmine and steroids; more advanced treatments are usually reserved for refractory patients. There was a clear perception of increased frequencies of exacerbations and crises across the treatment strategies. Depending on the country, estimates of HCRU ranged from a mean of 6 to 10 days of hospital stay for exacerbations and 9 to 13 days for crises, with mean stays in the intensive care unit of 4 to 5 days for exacerbations and 6 to 9 days for crises. Total yearly costs per country ranged from $10.7 M in Colombia to $122.7 M in Brazil. Costs per patient increased from 4 to 20 times, depending on the healthcare setting (public or private) and country, as patients move to later treatments. The estimated cost per exacerbation ranged in different countries from $3.3 to $11.8 thousand in the public sector and $4.3 to $12.1 thousand in the private sector. For crises, the corresponding estimated costs ranged from $ 5,000 to $ 26,800 and from $ 7,300 to $ 27,200. Conclusions This study indicates important unmet needs and barriers to adequate diagnosis and treatment of gMG in Latin American countries, suggesting that these patients are progressing across treatment without sufficient disease control, thus creating a substantial burden for healthcare systems.