UNDERSTANDING UNMET NEED IN THE CONTEMPORARY TREATMENT LANDSCAPE IN A EUROPEAN GENERALIZED MYASTHENIA GRAVIS POPULATION FROM BOTH PHYSICIAN AND PATIENT PERSPECTIVES: A REAL-WORLD STUDY
Author(s)
Jana Raab, MSc1, Sophi Tatlock, MA2, Hannah Connolly, MChem3, Beth Poirrette, MSc3, Amy Foster, BSc3, Brad Mason, BSc (Hons)4, Lara Ayala-Nunes, DSc, PhD4, Bethany Backhouse, MSc4, Mauminah Chaudry, MSc4, Gerard Harty, MSc1.
1Merck KGaA, Darmstadt, Germany, 2Merck Serono Ltd., Feltham, UK, an affiliate of Merck KGaA, Darmstadt, Germany, 3Adelphi Real World, Bollington, United Kingdom, 4Adelphi Values Patient-Centred Outcomes, Bollington, United Kingdom.
1Merck KGaA, Darmstadt, Germany, 2Merck Serono Ltd., Feltham, UK, an affiliate of Merck KGaA, Darmstadt, Germany, 3Adelphi Real World, Bollington, United Kingdom, 4Adelphi Values Patient-Centred Outcomes, Bollington, United Kingdom.
OBJECTIVES: Generalized myasthenia gravis (gMG) is a rare, chronic condition manifesting as muscle weakness and fatigue with unpredictable symptom exacerbations and myasthenic crises. With new therapies recently approved, it is essential to assess remaining disease burden and unmet need. Here, we characterize and identify unmet need and disease burden for gMG patients in Europe in the current treatment landscape.
METHODS: Physician-reported clinical characteristics, treatment and healthcare resource utilization (HCRU) data and patient-reported outcomes from the Adelphi gMG II Disease Specific Programme™ (DSP) were described. The DSP is a cross-sectional dataset of neurologists and their consulting gMG patients across Germany, Italy and France (January-November 2025).
RESULTS: Overall, n=66 physicians reported on n=428 patients. Mean (standard deviation [SD]) patient age was 52.3 (14.9) years and 52.3% were female. Reported mean (SD) time since gMG diagnosis was 3.7 (4.8) years at survey. Most patients (82.3%) were acetylcholine receptor autoantibody positive. At survey, 93.2% were prescribed treatment, with 16.0% prescribed neonatal Fc receptor inhibitors and 13.8% prescribed complement inhibitors. Mean (SD) MG-ADL score was 4.5 (3.3) and 57.5% were in MGFA class-II (mild). Since diagnosis of gMG, 31.1% patients had experienced ≥1 exacerbation of symptoms and 15.6% had experienced ≥1 myasthenic crisis. In the 12 months prior to survey, 15.3% of patients had ≥1 hospitalization and a mean (SD) of 5.3 (4.3) outpatient consultations. Of patients voluntarily self-reporting data (n=176), mean (SD) EQ-5D-5L score was 0.85 (0.16), MG-QoL 15r score was 8.9 (5.7) and FACIT-Fatigue score was 35.5 (9.1).
CONCLUSIONS: In this analysis, gMG patients reported impaired QoL and physicians reported frequent HCRU and clinical events indicating unmet needs remain. More research is needed to contextualize findings and enhance understanding as the treatment landscape evolves; further data are being collected and analyzed via a mixed methods approach combining updated DSP data and interviews with patients prescribed novel treatments.
METHODS: Physician-reported clinical characteristics, treatment and healthcare resource utilization (HCRU) data and patient-reported outcomes from the Adelphi gMG II Disease Specific Programme™ (DSP) were described. The DSP is a cross-sectional dataset of neurologists and their consulting gMG patients across Germany, Italy and France (January-November 2025).
RESULTS: Overall, n=66 physicians reported on n=428 patients. Mean (standard deviation [SD]) patient age was 52.3 (14.9) years and 52.3% were female. Reported mean (SD) time since gMG diagnosis was 3.7 (4.8) years at survey. Most patients (82.3%) were acetylcholine receptor autoantibody positive. At survey, 93.2% were prescribed treatment, with 16.0% prescribed neonatal Fc receptor inhibitors and 13.8% prescribed complement inhibitors. Mean (SD) MG-ADL score was 4.5 (3.3) and 57.5% were in MGFA class-II (mild). Since diagnosis of gMG, 31.1% patients had experienced ≥1 exacerbation of symptoms and 15.6% had experienced ≥1 myasthenic crisis. In the 12 months prior to survey, 15.3% of patients had ≥1 hospitalization and a mean (SD) of 5.3 (4.3) outpatient consultations. Of patients voluntarily self-reporting data (n=176), mean (SD) EQ-5D-5L score was 0.85 (0.16), MG-QoL 15r score was 8.9 (5.7) and FACIT-Fatigue score was 35.5 (9.1).
CONCLUSIONS: In this analysis, gMG patients reported impaired QoL and physicians reported frequent HCRU and clinical events indicating unmet needs remain. More research is needed to contextualize findings and enhance understanding as the treatment landscape evolves; further data are being collected and analyzed via a mixed methods approach combining updated DSP data and interviews with patients prescribed novel treatments.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO218
Topic
Clinical Outcomes, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Clinical Outcomes Assessment
Disease
Neurological Disorders, Rare & Orphan Diseases