TREATMENT PATTERNS AMONG PEOPLE NEWLY DIAGNOSED WITH NARCOLEPSY TYPE 1 IN THE UNITED STATES: A REAL-WORLD DATA STUDY
Author(s)
Stephen Crawford, MHS, PhD1, Brian Calingaert, MS2, Juliana Meyers, MA2, LaStella Sinclair, MS2, Amy Duhig, PhD3, Sean Candrilli, PhD2, Christine Ulbricht, MPH, PhD3.
1Takeda Development Center Americas, Inc., Cambridge, MA, USA, 2RTI Health Solutions, Research Triangle Park, NC, USA, 3Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA, USA.
1Takeda Development Center Americas, Inc., Cambridge, MA, USA, 2RTI Health Solutions, Research Triangle Park, NC, USA, 3Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA, USA.
Presentation Documents
OBJECTIVES: Despite therapeutic advances in narcolepsy type 1 (NT1), a disease characterized by excessive daytime sleepiness, cataplexy, and disrupted nighttime sleep, NT1 is still managed symptomatically. We provide a real-world characterization of individuals newly diagnosed with NT1 in the US.
METHODS: This was a retrospective observational study using Merative™ Marketscan® Research Databases from January 2017 to January 2025. The primary objectives were to describe demographics, clinical characteristics, and treatment patterns among commercially insured adults and Medicare Supplemental beneficiaries newly diagnosed with NT1. Treatment patterns including medication adherence (medication possession ratio [MPR]), persistence (in days), polypharmacy, treatment discontinuation, and treatment switching were assessed.
RESULTS: Individuals with incident NT1 were included (n=580; median [IQR] age: 33 [23-45] years; 71.7% female). Within 12 months of diagnosis, 87.2% (506/580) received narcolepsy-related treatments (NRTs: stimulants, antidepressants) or narcolepsy-indicated treatments (NITs: sodium oxybate, pitolisant, modafinil, armodafinil, and solriamfetol); 12.8% remained untreated. Over half (295/580) received NITs any time in the 12 months after their NT1 diagnosis index date, most commonly modafinil (24.1%) and armodafinil (15.7%). Mean MPR and persistence were 83.6% and 73.6 days for pitolisant, 79.3% and 71.9 for sodium oxybate, and 63.2% and 60.0 for modafinil. Polypharmacy was common: among all 506 treated individuals, 56.7% received ≥2 NRTs or NITs simultaneously; of the 295 individuals receiving NITs, 23.4% received ≥2 simultaneously. Among those with 12 months of follow-up after index NIT (n=259): the mean (SD) time to initiation of the index NIT was 71.8 (85.4) days; discontinuation of the index NIT reached 69.9% within 12 months after treatment initiation. Treatment switching occurred in 18.9% of individuals receiving NITs.
CONCLUSIONS: Findings indicate that treatment initiation is delayed after NT1 diagnosis, with frequent polypharmacy, switching, and high discontinuation rates within 12 months of diagnosis, highlighting substantial unmet treatment needs and the necessity for newer treatment options.
METHODS: This was a retrospective observational study using Merative™ Marketscan® Research Databases from January 2017 to January 2025. The primary objectives were to describe demographics, clinical characteristics, and treatment patterns among commercially insured adults and Medicare Supplemental beneficiaries newly diagnosed with NT1. Treatment patterns including medication adherence (medication possession ratio [MPR]), persistence (in days), polypharmacy, treatment discontinuation, and treatment switching were assessed.
RESULTS: Individuals with incident NT1 were included (n=580; median [IQR] age: 33 [23-45] years; 71.7% female). Within 12 months of diagnosis, 87.2% (506/580) received narcolepsy-related treatments (NRTs: stimulants, antidepressants) or narcolepsy-indicated treatments (NITs: sodium oxybate, pitolisant, modafinil, armodafinil, and solriamfetol); 12.8% remained untreated. Over half (295/580) received NITs any time in the 12 months after their NT1 diagnosis index date, most commonly modafinil (24.1%) and armodafinil (15.7%). Mean MPR and persistence were 83.6% and 73.6 days for pitolisant, 79.3% and 71.9 for sodium oxybate, and 63.2% and 60.0 for modafinil. Polypharmacy was common: among all 506 treated individuals, 56.7% received ≥2 NRTs or NITs simultaneously; of the 295 individuals receiving NITs, 23.4% received ≥2 simultaneously. Among those with 12 months of follow-up after index NIT (n=259): the mean (SD) time to initiation of the index NIT was 71.8 (85.4) days; discontinuation of the index NIT reached 69.9% within 12 months after treatment initiation. Treatment switching occurred in 18.9% of individuals receiving NITs.
CONCLUSIONS: Findings indicate that treatment initiation is delayed after NT1 diagnosis, with frequent polypharmacy, switching, and high discontinuation rates within 12 months of diagnosis, highlighting substantial unmet treatment needs and the necessity for newer treatment options.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE138
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Neurological Disorders