QUANTIFYING THE LIFETIME HEALTH AND SOCIETAL BENEFITS OF TIMELY DIAGNOSIS AND ACCESS TO APPROVED TREATMENTS IN ANTI-ACETYLCHOLINE RECEPTOR ANTIBODY-POSITIVE (ACHR-AB+) GENERALIZED MYASTHENIA GRAVIS (GMG) IN THE UNITED STATES
Author(s)
Chelsea Shugars, MD1, Annie Kennedy, BS2, Jeffrey C. Yu, MS, PhD3, Adrian Kielhorn, MBA3, Karen Yee, PhD3, Foteini Tsotra, MPH, MSc4, Diego Hernandez, PhD5, Malina Müller, BA, MA, PhD6.
1Erlanger Health System, Chattanooga, TN, USA, 2EveryLife Foundation for Rare Diseases, Washington, DC, USA, 3Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 4WifOR Institute, Athens, Greece, 5WifOR Institute, Berlin, Germany, 6WifOR Institute, Darmstadt, Germany.
1Erlanger Health System, Chattanooga, TN, USA, 2EveryLife Foundation for Rare Diseases, Washington, DC, USA, 3Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 4WifOR Institute, Athens, Greece, 5WifOR Institute, Berlin, Germany, 6WifOR Institute, Darmstadt, Germany.
OBJECTIVES: Diagnostic delays and access barriers to effective treatments in AChR-Ab+ gMG can increase disease burden for patients leading to societal productivity losses. A cohort modeling approach was used to quantify the lifetime health and societal benefits of timely diagnosis and access to approved treatments for AChR‑Ab+ gMG in the US.
METHODS: Disease course from symptom onset until death was modeled using changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) score to track the disease course. The lifetime health impacts (crises and exacerbations) and lifetime work/social productivity benefits for patients were compared for status quo (2-year duration from disease onset to diagnosis; 6 years from diagnosis to treatment with approved therapies) vs timely diagnosis (within 6 months of disease onset) with and without initiation of approved treatments within 6 months of diagnosis. Patients were assumed to receive usual care (steroidal/nonsteroidal immunosuppressive therapies) before approved treatments, and approved therapy use was modeled using a weighted basket approach.
RESULTS: Among patients with AChR-Ab+ gMG, MG-ADL score >5.0, and receiving approved therapies (assuming 27.6% of all patients eligible for approved treatments), timely diagnosis alone resulted in a 14% reduction in the number of crises (0.37 vs 0.43) and exacerbations (4.29 vs 4.99) compared with status quo. Timely diagnosis and earlier initiation of approved treatments resulted in a 51% reduction in the number of crises (0.21 vs 0.43) and exacerbations (2.46 vs 4.99), and 94.1% of these patients spent life-years with MG-ADL score ≤6.0 vs 0% in the status quo scenario. Societal benefit with timely diagnosis and earlier approved treatment initiation was $6674/patient/year, amounting to $85.9 million/year nationally or $1.7 billion/lifetime nationally.
CONCLUSIONS: These findings show that, although timely diagnosis alone can produce meaningful health benefits, timely diagnosis with early access to approved treatments could provide even greater health benefits and improve patients’ work and social productivity.
METHODS: Disease course from symptom onset until death was modeled using changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) score to track the disease course. The lifetime health impacts (crises and exacerbations) and lifetime work/social productivity benefits for patients were compared for status quo (2-year duration from disease onset to diagnosis; 6 years from diagnosis to treatment with approved therapies) vs timely diagnosis (within 6 months of disease onset) with and without initiation of approved treatments within 6 months of diagnosis. Patients were assumed to receive usual care (steroidal/nonsteroidal immunosuppressive therapies) before approved treatments, and approved therapy use was modeled using a weighted basket approach.
RESULTS: Among patients with AChR-Ab+ gMG, MG-ADL score >5.0, and receiving approved therapies (assuming 27.6% of all patients eligible for approved treatments), timely diagnosis alone resulted in a 14% reduction in the number of crises (0.37 vs 0.43) and exacerbations (4.29 vs 4.99) compared with status quo. Timely diagnosis and earlier initiation of approved treatments resulted in a 51% reduction in the number of crises (0.21 vs 0.43) and exacerbations (2.46 vs 4.99), and 94.1% of these patients spent life-years with MG-ADL score ≤6.0 vs 0% in the status quo scenario. Societal benefit with timely diagnosis and earlier approved treatment initiation was $6674/patient/year, amounting to $85.9 million/year nationally or $1.7 billion/lifetime nationally.
CONCLUSIONS: These findings show that, although timely diagnosis alone can produce meaningful health benefits, timely diagnosis with early access to approved treatments could provide even greater health benefits and improve patients’ work and social productivity.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
MSR9
Topic
Methodological & Statistical Research
Disease
SDC: Neurological Disorders, SDC: Rare & Orphan Diseases