REAL-WORLD HEALTHCARE RESOURCE UTILIZATION AND ECONOMIC BURDEN OF NARCOLEPSY TYPE 1 IN THE US
Author(s)
David T. Plante, MD, PhD1, Samantha Floam, DMD2, Weiying Wang, MPH3, Junjun Cong, MS3, Leona Bessonova, PhD2.
1Department of Psychiatry, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA, 2Axsome Therapeutics, Inc., New York, NY, USA, 3KMK Consulting, Morristown, NJ, USA.
1Department of Psychiatry, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA, 2Axsome Therapeutics, Inc., New York, NY, USA, 3KMK Consulting, Morristown, NJ, USA.
OBJECTIVES: Narcolepsy is a chronic neurological disorder characterized by excessive daytime sleepiness, disrupted nighttime sleep, and REM sleep-associated symptoms, with cataplexy the key distinguishing clinical feature of narcolepsy type 1 (NT1). NT1-specific healthcare resource utilization (HCRU) and cost data are limited; this analysis described these outcomes among US adults using administrative claims data.
METHODS: This retrospective cohort study used the Merative™ MarketScan® Commercial and Medicare databases (Jan 1, 2021-Dec 31, 2024). Patients (aged ≥18 years) had ≥2 ICD-10 diagnoses from Jan 1, 2022-Dec 31, 2023 and 12-month continuous enrolment before and after the first qualifying diagnosis (index date). HCRU and costs were evaluated over the 12-month follow-up, including hospitalizations, emergency room (ER) and outpatient visits, and medical and pharmacy costs (inflation-adjusted to 2025 US dollars).
RESULTS: The NT1 cohort comprised 1707 adults (mean age 41.6 years; 70% female). During follow-up, 7% experienced ≥1 all-cause hospitalization, of which 11% had a 30-day readmission. Among those hospitalized, mean (SD) inpatient days were 6.6 (10.5) without 30-day readmission and 28.2 (32.1) with 30-day readmission. Overall, 27% of patients had ≥1 all-cause ER visit. Mean all-cause outpatient visits were 24.4 (24.1), and 23% of patients had ≥1 all-cause urgent care visit.
All-cause costs (medical + pharmacy) averaged $73,486 ($107,597) during follow-up, including $58,972 ($97,770) in mean pharmacy costs; mean prescription fills was 37.3 (32.8). Hospitalization costs averaged $40,641 ($123,597) among patients without 30-day readmission and $150,712 ($180,741) among those with 30-day readmission. ER visit costs averaged $4539 ($5803), and outpatient visit costs averaged $9747 ($17,940).
CONCLUSIONS: This claims analysis shows adults with NT1 incur substantial HCRU, including 30-day readmissions, ER, urgent care and other outpatient visits. Associated costs were substantial, driven by pharmacy expenditures and high-cost inpatient episodes, including lengthy readmissions. These results highlight prevailing unmet need for therapies that mitigate clinical and economic burden in NT1.
METHODS: This retrospective cohort study used the Merative™ MarketScan® Commercial and Medicare databases (Jan 1, 2021-Dec 31, 2024). Patients (aged ≥18 years) had ≥2 ICD-10 diagnoses from Jan 1, 2022-Dec 31, 2023 and 12-month continuous enrolment before and after the first qualifying diagnosis (index date). HCRU and costs were evaluated over the 12-month follow-up, including hospitalizations, emergency room (ER) and outpatient visits, and medical and pharmacy costs (inflation-adjusted to 2025 US dollars).
RESULTS: The NT1 cohort comprised 1707 adults (mean age 41.6 years; 70% female). During follow-up, 7% experienced ≥1 all-cause hospitalization, of which 11% had a 30-day readmission. Among those hospitalized, mean (SD) inpatient days were 6.6 (10.5) without 30-day readmission and 28.2 (32.1) with 30-day readmission. Overall, 27% of patients had ≥1 all-cause ER visit. Mean all-cause outpatient visits were 24.4 (24.1), and 23% of patients had ≥1 all-cause urgent care visit.
All-cause costs (medical + pharmacy) averaged $73,486 ($107,597) during follow-up, including $58,972 ($97,770) in mean pharmacy costs; mean prescription fills was 37.3 (32.8). Hospitalization costs averaged $40,641 ($123,597) among patients without 30-day readmission and $150,712 ($180,741) among those with 30-day readmission. ER visit costs averaged $4539 ($5803), and outpatient visit costs averaged $9747 ($17,940).
CONCLUSIONS: This claims analysis shows adults with NT1 incur substantial HCRU, including 30-day readmissions, ER, urgent care and other outpatient visits. Associated costs were substantial, driven by pharmacy expenditures and high-cost inpatient episodes, including lengthy readmissions. These results highlight prevailing unmet need for therapies that mitigate clinical and economic burden in NT1.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PT16
Topic
Economic Evaluation
Disease
SDC: Neurological Disorders, SDC: Rare & Orphan Diseases