Quantifying the Lifetime Health and Societal Benefits from Earlier Diagnosis and Access to Approved Targeted Immunotherapies in Anti-aquaporin-4 Antibody-positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD) in the United States
Shamik Bhattacharyya1, Annie Kennedy2, Jeffrey Yu3, Adrian Kielhorn3, Foteini Tsotra4, Diego Hernandez4, Malina Müller4
1Mass General Brigham, Harvard Medical School, 2EveryLife Foundation for Rare Diseases, 3Alexion, AstraZeneca Rare Disease, 4WifOR Institute
Objective:
Quantify, using a cohort modeling approach, the health and societal benefits of earlier diagnosis and approved targeted immunotherapy access for AQP4-Ab+ NMOSD in the US.
Background:
Patients with AQP4-Ab+ NMOSD frequently experience diagnostic delays and barriers to accessing approved immunotherapies, resulting in avoidable disease progression and societal productivity losses.
Design/Methods:
Disease course from symptom onset until death in a cohort of hypothetical patients with AQP4-Ab+ NMOSD was simulated, using relapses and changes on Expanded Disability Status Scale (EDSS) to track disease progression. Relapse rates for weighted baskets of usual care or approved immunotherapies were used. The lifetime health impacts (relapses and quality-adjusted life-years [QALYs]) and lifetime societal benefits (work/social productivity) were compared for status quo (4-year gap from disease onset to diagnosis; 2.3-year gap [while patients received usual care, including rituximab] from diagnosis to treatment with approved immunotherapies) vs scenarios with earlier diagnosis and/or earlier approved immunotherapy access.
Results:
Compared with status quo, diagnosis within 6 months of disease onset with no change in approved treatment timing resulted in a 5% reduction in relapses (3.72 vs 3.55) and a 4% improvement in QALYs (7.84 vs 8.16); both diagnosis and initiation of approved immunotherapy as first-line treatment within 6 months of disease onset resulted in a 27% reduction in relapses (3.72 vs 2.71) and 27% improvement in QALYs (7.84 vs 9.97), as well as more life-years with an EDSS score <6.0 (3.73 vs. 8.93). Societal benefit, calculated for 57% of patients with AQP4-Ab+ NMOSD on approved therapies, with timely diagnosis and earlier initiation of approved treatment, was $6,869/patient (or caregiver)/year, amounting to $18.2 million/year nationally or $364.3 million/lifetime nationally.
Conclusions:
These modeling data show that although earlier diagnosis alone can produce meaningful benefits, earlier diagnosis and timely access to approved immunotherapies may be even more beneficial and improve patients’ quality of life and work/social productivity.
Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.