Tomaras D1, Marshall S2, Forsythe A2
1Purple Squirrel Economics, Montreal, QC, Canada, 2Purple Squirrel Economics, New York, NY, USA

Presentation Documents

OBJECTIVES : MG is a debilitating chronic illness characterized by muscle weakness, potentially life-threatening exacerbations and high costs. We sought to further quantify the humanistic and economic burden by reviewing published evidence.

METHODS : Using PRISMA guidelines, two systematic literature reviews were conducted, one for quality of life (QOL) and one for economic studies, in key biomedical literature databases, Embase, Ovid Medline and Cochrane. Additionally, only studies published between 2009-2019 were identified.

RESULTS : A total of 957 abstracts from the QOL search and 521 from the economic search underwent screening. A total of 81 QOL and 41 economic studies were selected. A variety of QOL tools were used, including SF-36, EQ-5D, HADS, MG-ADL, MG-QOL15, QMG and MGC among others. The only study mapping utility values found a range of 0.94-0.20 across MGFA classifications, indicating a major QOL deterioration related to disease severity. Deterioration was further supported in other studies which found increased levels of chronic fatigue, sleep disturbances and anxiety/depression with MG. Refractory patients experienced significantly worse deteriorations across various scales. Employment status and medication adherence were also negatively affected. Of 41 economic studies, 32 were related to cost/healthcare resource use, while 9 described economic models. The average US cost in 2013 was $98,795 per MG hospitalization and the gross hospitalization cost in the HCUP database had risen more than 13-fold since 2003 totalling $546,834,101. Inpatient, outpatient and home costs were found to account for 27%, 23% and 23% of MG healthcare costs. A US claims database found annual costs to be approximately 4 times higher in refractory vs. non-refractory patients ($109,004 vs. $24,196, p<0.001), possibly related to a higher use of IVig/PLEX, costly therapies according to economic models.

CONCLUSIONS : Novel treatment strategies are necessitated to help control rising costs and alleviate the humanistic burden associated with MG, especially in refractory patients.

Conference/Value in Health Info

2020-05, ISPOR 2020, Orlando, FL, USA




Economic Evaluation, Patient-Centered Research

Topic Subcategory

Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes


Neurological Disorders, Rare and Orphan Diseases

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