Microsimulation Model to Estimate the Clinical and Cost Burden of Adverse Events Related to Long-Term Oral Corticosteroid Usage in Autoimmune Diseases in the United States
Author(s)
John H. Stone, MD, MPH1, Peter Neumann, ScD2, Pushpa Narayanaswami, MD3, SYED RAZA, MBA, MSc4, David Proudman, MSc, MPH5, Arshya Feizi, PhD6, Rachel Katherine Meade, PhD5, Adrienne Kwok, MPH5, sydney ng, MPH7, Noam Kirson, PhD6, Glenn A. Phillips, PhD8.
1Massachusetts General Hospital, Boston, MA, USA, 2Tufts Medical Center, Boston, MA, USA, 3Harvard Medical School, Boston, MA, USA, 4Argenx, Milton Keynes, United Kingdom, 5Analysis Group, San Francisco, CA, USA, 6Analysis Group, Boston, MA, USA, 7Analysis Group, Los Angeles, CA, USA, 8Argenx, Boston, MA, USA.
1Massachusetts General Hospital, Boston, MA, USA, 2Tufts Medical Center, Boston, MA, USA, 3Harvard Medical School, Boston, MA, USA, 4Argenx, Milton Keynes, United Kingdom, 5Analysis Group, San Francisco, CA, USA, 6Analysis Group, Boston, MA, USA, 7Analysis Group, Los Angeles, CA, USA, 8Argenx, Boston, MA, USA.
Presentation Documents
OBJECTIVES: Oral corticosteroids (OCS) are often used for prolonged durations to manage autoimmune conditions, but are associated with important adverse events (AEs). This study aimed to quantify lifetime clinical and economic costs of AEs related to prolonged OCS use, relative to a hypothetical steroid-sparing treatment that reduces need for OCS. The model base-case focused on myasthenia gravis (MG). Chronic inflammatory demyelinating polyneuropathy, immune thrombocytopenic purpura, and lupus nephritis were also investigated.
METHODS: A lifetime-horizon patient-level microsimulation model with 3-month cycles was developed to assess costs and outcomes including quality-adjusted life-years (QALY). A long-term OCS regimen was compared with two steroid-sparing regimens (assuming clinical equivalence) consisting of: (i) 50% of standard OCS dose; and (ii) full-dose OCS for only 6 months. Distributions of typical OCS dose, patient age and sex were sourced from the literature. The model incorporated disutilities, mortality, and US costs associated with 12 chronic and short-term OCS-related AEs, including cardiovascular AEs, diabetes, pneumonia and fractures. Per-cycle risk of patients developing each AE depended on the current or cumulative OCS dose (assuming a 5-year dose accumulation window). Scenario and sensitivity analyses were conducted.
RESULTS: In MG, with base-case onset of OCS use at age 55, a 50% OCS dose-sparing regimen yielded lifetime QALY improvement of 0.56 (95%CI: 0.50 - 0.62) and per patient savings of USD$24,915 ($22,493 - $27,287). OCS use limited to 6 months resulted in per patient QALY gain of 1.24 (1.15 - 1.33) and savings of $38,480 ($35,122 - $42,015). Results were most sensitive to average age and to dose-related AE risks. Benefits were more substantial in populations with greater remaining life expectancy. Comparable findings were observed in other conditions.
CONCLUSIONS: A reduction in OCS exposure could substantially improve quality of life and reduce economic burden. Age at disease onset and cumulative dose of OCS are major determinants of burden.
METHODS: A lifetime-horizon patient-level microsimulation model with 3-month cycles was developed to assess costs and outcomes including quality-adjusted life-years (QALY). A long-term OCS regimen was compared with two steroid-sparing regimens (assuming clinical equivalence) consisting of: (i) 50% of standard OCS dose; and (ii) full-dose OCS for only 6 months. Distributions of typical OCS dose, patient age and sex were sourced from the literature. The model incorporated disutilities, mortality, and US costs associated with 12 chronic and short-term OCS-related AEs, including cardiovascular AEs, diabetes, pneumonia and fractures. Per-cycle risk of patients developing each AE depended on the current or cumulative OCS dose (assuming a 5-year dose accumulation window). Scenario and sensitivity analyses were conducted.
RESULTS: In MG, with base-case onset of OCS use at age 55, a 50% OCS dose-sparing regimen yielded lifetime QALY improvement of 0.56 (95%CI: 0.50 - 0.62) and per patient savings of USD$24,915 ($22,493 - $27,287). OCS use limited to 6 months resulted in per patient QALY gain of 1.24 (1.15 - 1.33) and savings of $38,480 ($35,122 - $42,015). Results were most sensitive to average age and to dose-related AE risks. Benefits were more substantial in populations with greater remaining life expectancy. Comparable findings were observed in other conditions.
CONCLUSIONS: A reduction in OCS exposure could substantially improve quality of life and reduce economic burden. Age at disease onset and cumulative dose of OCS are major determinants of burden.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE580
Topic
Clinical Outcomes, Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Neurological Disorders, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)