Abstract Details

Title 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

Topic Autoimmune Neurology

Presentation(s) P7 - Poster Session 7 (8:00 AM-9:00 AM)

Poster/Presentation
Number 1-003

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.

Authors/Disclosures
Shamik Bhattacharyya, MD, FAAN (Brigham and Women's Hospital) PRESENTER	Dr. Bhattacharyya has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NeuroLambda. Dr. Bhattacharyya has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Bhattacharyya has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Bhattacharyya has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics. Dr. Bhattacharyya has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Continuum. Dr. Bhattacharyya has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wiley. Dr. Bhattacharyya has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Merck. The institution of Dr. Bhattacharyya has received research support from Alexion Pharmaceuticals. The institution of Dr. Bhattacharyya has received research support from National Institute of Health. The institution of Dr. Bhattacharyya has received research support from UCB. The institution of Dr. Bhattacharyya has received research support from Genentech. Dr. Bhattacharyya has received publishing royalties from a publication relating to health care. Dr. Bhattacharyya has received publishing royalties from a publication relating to health care.
Annie Kennedy	Ms. Kennedy has received personal compensation for serving as an employee of EveryLife Foundation for Rare Diseases. Ms. Kennedy has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant with Parent Project Muscular Dystrophy.
Jeffrey Yu, PhD (Alexion)	Dr. Yu has received personal compensation for serving as an employee of Alexion. Dr. Yu has stock in AstraZeneca.
Adrian Kielhorn	Adrian Kielhorn has received personal compensation for serving as an employee of Alexion. Adrian Kielhorn has stock in Alexion.
Foteini Tsotra, MSc	Miss Tsotra has received personal compensation for serving as an employee of WifOR Institute, Athens, Greece.
Diego Hernandez	Dr. Hernandez has received personal compensation for serving as an employee of WifOR.
Malina Müller, PhD	Mrs. Müller has received personal compensation for serving as an employee of WifOR Institute.

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