Costs and Healthcare Resource Utilization Associated With Generalized Myasthenia Gravis: A Systematic Literature Review
Author(s)
Rambabu Vatte, Masters in Pharmacy1, Mohit Kumar Bhutani, Masters in Pharmacy1, Nicholas Kirvassilis, MSc2, Rahul Khairnar, BS, MS, PhD3.
1Novartis Healthcare Private Limited, Hyderabad, India, 2Novartis Pharma AG, Basel, Switzerland, 3Novartis Pharmaceuticals Corporation, Jersey City, NJ, USA.
1Novartis Healthcare Private Limited, Hyderabad, India, 2Novartis Pharma AG, Basel, Switzerland, 3Novartis Pharmaceuticals Corporation, Jersey City, NJ, USA.
OBJECTIVES: To systematically review published evidence on costs and healthcare resource utilisation (HCRU) in generalised myasthenia gravis (gMG).
METHODS: A Health Technology Assessment (HTA) compliant systematic literature review (SLR) was conducted using Embase®, Medline® and NHS Economic Evaluation Databases to retrieve evidence published until April 2024. Key conferences (2021 onwards) and HTA websites were hand searched. Studies published in English involving adults with gMG that reported relevant direct and indirect costs, as well as HCRU, were included.
RESULTS: Overall, 29 studies from 38 publications were identified; majority were from the USA (n=11) or multinational settings (n=7). Most studies examined broader gMG populations (n=20), while three focused on acetylcholine receptor antibody-positive gMG and three compared refractory and non-refractory subgroups. In the USA, compared to individuals without disease, patients with refractory gMG incurred 10 times higher annual healthcare costs ($109,004 vs. $11,582; p<0.001). Additionally, refractory gMG costs were over four times higher than non-refractory gMG ($109,004 vs. $24,196; p<0.001). Newly diagnosed patients incurred higher all-cause costs ($26,419 per patient per year [PPPY]) compared to previously diagnosed patients ($24,941). Costs increased further with exacerbations ($43,734) and peaked around crisis events—$49,237 PPPY in the year before the event and $173,957 in the 12 months after. Higher HCRU was observed alongside increased cost burden in cases of new diagnosis, refractory gMG, and during exacerbations or crises. Newly diagnosed gMG patients had higher hospitalisations and longer stays (24%, 1.39 days) than previously diagnosed cases (19%, 0.99 days; p<0.001). Exacerbations and crises further raised hospitalisations (36%, 2.04 days; 100%, 15.38 days, respectively; p<0.001). Cost data for EU5, China, Japan, and serological subgroups were limited.
CONCLUSIONS: gMG poses a significant economic burden, especially in refractory cases and during crises. Further research is needed to fill evidence gaps in gMG serological subgroups and in regions such as Europe and Asia.
METHODS: A Health Technology Assessment (HTA) compliant systematic literature review (SLR) was conducted using Embase®, Medline® and NHS Economic Evaluation Databases to retrieve evidence published until April 2024. Key conferences (2021 onwards) and HTA websites were hand searched. Studies published in English involving adults with gMG that reported relevant direct and indirect costs, as well as HCRU, were included.
RESULTS: Overall, 29 studies from 38 publications were identified; majority were from the USA (n=11) or multinational settings (n=7). Most studies examined broader gMG populations (n=20), while three focused on acetylcholine receptor antibody-positive gMG and three compared refractory and non-refractory subgroups. In the USA, compared to individuals without disease, patients with refractory gMG incurred 10 times higher annual healthcare costs ($109,004 vs. $11,582; p<0.001). Additionally, refractory gMG costs were over four times higher than non-refractory gMG ($109,004 vs. $24,196; p<0.001). Newly diagnosed patients incurred higher all-cause costs ($26,419 per patient per year [PPPY]) compared to previously diagnosed patients ($24,941). Costs increased further with exacerbations ($43,734) and peaked around crisis events—$49,237 PPPY in the year before the event and $173,957 in the 12 months after. Higher HCRU was observed alongside increased cost burden in cases of new diagnosis, refractory gMG, and during exacerbations or crises. Newly diagnosed gMG patients had higher hospitalisations and longer stays (24%, 1.39 days) than previously diagnosed cases (19%, 0.99 days; p<0.001). Exacerbations and crises further raised hospitalisations (36%, 2.04 days; 100%, 15.38 days, respectively; p<0.001). Cost data for EU5, China, Japan, and serological subgroups were limited.
CONCLUSIONS: gMG poses a significant economic burden, especially in refractory cases and during crises. Further research is needed to fill evidence gaps in gMG serological subgroups and in regions such as Europe and Asia.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE286
Topic
Economic Evaluation, Study Approaches
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Neurological Disorders, Rare & Orphan Diseases