REAL-WORLD TREATMENT PATTERNS, HEALTHCARE RESOURCE UTILIZATION, AND UNMET NEEDS IN GENERALIZED MYASTHENIA GRAVIS: A RETROSPECTIVE ANALYSIS OF GERMAN STATUTORY HEALTH INSURANCE CLAIMS
Author(s)
Wisam Karmous, PharmD1, Gonçalo Rodrigues, PhD2, Wim Noel, PhD3, Moritz Lehne, PhD4, Anja Cengia, MSc5, Federico Spandonaro, PhD6, Kostas Athanasakis, PhD7, Tom Denee, MBA, PharmD8.
1Johnson & Johnson, Paris, France, 2Johnson & Johnson, Madrid, Portugal, 3Johnson & Johnson, Beerse, Belgium, 4Cytel Inc., Berlin, Germany, 5Institute for Applied Health Research Berlin GmbH, Berlin, Germany, 6Dept. Economics and Finance, University of Roma Tor Vergata, Roma, Italy, 7Laboratory for Health Technology Assessment, University of West Attica, Attica, Greece, 8Johnson & Johnson, Breda, Netherlands.
1Johnson & Johnson, Paris, France, 2Johnson & Johnson, Madrid, Portugal, 3Johnson & Johnson, Beerse, Belgium, 4Cytel Inc., Berlin, Germany, 5Institute for Applied Health Research Berlin GmbH, Berlin, Germany, 6Dept. Economics and Finance, University of Roma Tor Vergata, Roma, Italy, 7Laboratory for Health Technology Assessment, University of West Attica, Attica, Greece, 8Johnson & Johnson, Breda, Netherlands.
OBJECTIVES: To describe real-world patterns, healthcare resource utilization (HCRU), and proxies of disease control among adults with generalized myasthenia gravis (gMG) receiving advanced therapies.
METHODS: Cohort study using German health insurance claims (InGef database; 2016-2024). Adults with gMG diagnosed by a neurologist and treated with advanced therapy were included (N=144). Baseline characteristics and prior treatments were assessed in the 12 months pre-index. During follow-up (from start of the advanced therapy to database end/death/switch), time-to-first event analyses assessed gMG exacerbations, rescue therapy (IVIg/PLEX/immuno-adsorption), high-dose corticosteroid use, and treatment switching. HCRU (all-cause and MG-related hospitalizations; outpatient visits) was summarized by therapy class (FcRn blockers approved at data cut: efgartigimod, rozanolixizumab; approved C5 inhibitors: eculizumab, ravulizumab, zilucoplan; and anti-CD20: rituximab).
RESULTS: Mean age was 56 years and 57% were female. During the 12-month pre-index period, 91% received pyridostigmine and 67% corticosteroids; 54% experienced ≥1 exacerbation and 43% received ≥1 rescue intervention. Across advanced therapies, off-label rituximab was most common (n=79), followed by FcRn blockers (n=47) and C5 inhibitors (n=44). During a median follow-up of 21 months, 63% had ≥1 exacerbation (median time to first 4.9 [4.2-6.5] months), highest with C5 inhibitors (~88-92%) and lowest with FcRn blockers (~49%). Post-index rescue therapy was required by 24% overall (~10% FcRn to ~32% anti-CD20) and high-dose corticosteroids by ~26% (similar across classes). Switching to another advanced therapy occurred most frequently among C5 inhibitor recipients. HCRU was high with 72% having ≥1 MG-related hospitalizations, and 83% having costs with MG outpatient visits; utilization was highest among C5 inhibitors (especially eculizumab) and anti-CD20 recipients.
CONCLUSIONS: In this claims analysis, gMG patients receiving advanced therapies had high prior treatment exposure and continued to experience frequent exacerbations, rescue therapy use, and high HCRU in routine care. These findings highlight unmet needs for sustained disease control and reduced healthcare burden in real-world gMG.
METHODS: Cohort study using German health insurance claims (InGef database; 2016-2024). Adults with gMG diagnosed by a neurologist and treated with advanced therapy were included (N=144). Baseline characteristics and prior treatments were assessed in the 12 months pre-index. During follow-up (from start of the advanced therapy to database end/death/switch), time-to-first event analyses assessed gMG exacerbations, rescue therapy (IVIg/PLEX/immuno-adsorption), high-dose corticosteroid use, and treatment switching. HCRU (all-cause and MG-related hospitalizations; outpatient visits) was summarized by therapy class (FcRn blockers approved at data cut: efgartigimod, rozanolixizumab; approved C5 inhibitors: eculizumab, ravulizumab, zilucoplan; and anti-CD20: rituximab).
RESULTS: Mean age was 56 years and 57% were female. During the 12-month pre-index period, 91% received pyridostigmine and 67% corticosteroids; 54% experienced ≥1 exacerbation and 43% received ≥1 rescue intervention. Across advanced therapies, off-label rituximab was most common (n=79), followed by FcRn blockers (n=47) and C5 inhibitors (n=44). During a median follow-up of 21 months, 63% had ≥1 exacerbation (median time to first 4.9 [4.2-6.5] months), highest with C5 inhibitors (~88-92%) and lowest with FcRn blockers (~49%). Post-index rescue therapy was required by 24% overall (~10% FcRn to ~32% anti-CD20) and high-dose corticosteroids by ~26% (similar across classes). Switching to another advanced therapy occurred most frequently among C5 inhibitor recipients. HCRU was high with 72% having ≥1 MG-related hospitalizations, and 83% having costs with MG outpatient visits; utilization was highest among C5 inhibitors (especially eculizumab) and anti-CD20 recipients.
CONCLUSIONS: In this claims analysis, gMG patients receiving advanced therapies had high prior treatment exposure and continued to experience frequent exacerbations, rescue therapy use, and high HCRU in routine care. These findings highlight unmet needs for sustained disease control and reduced healthcare burden in real-world gMG.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD67
Topic
Clinical Outcomes, Economic Evaluation, Real World Data & Information Systems
Topic Subcategory
Health & Insurance Records Systems
Disease
Biologics & Biosimilars, Neurological Disorders, Pediatrics, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)