Abstract Details

Title Development of a Novel Patient-reported Outcome Measure of Functional Impacts for Narcolepsy Types 1 and 2

Topic Sleep

Presentation(s) P8 - Poster Session 8 (8:00 AM-9:00 AM)

Poster/Presentation
Number 4-003

Objective To develop a new patient-reported outcome (PRO) measure, the Functional Impacts of Narcolepsy Instrument (FINI), for evaluating treatment response in patients with narcolepsy type 1 (NT1) or type 2 (NT2).

Background Narcolepsy, a rare, chronic neurologic disorder affecting sleep-wake stability, is associated with detrimental effects on functioning, quality of life, and productivity. There is a need for a fit-for-purpose clinical outcome assessment that captures the functional impacts of narcolepsy in various clinical settings to complement the Narcolepsy Severity Scale.

Design/Methods Initial item generation was derived using concept elicitation (NT1, n=21), cognitive debriefing (NT1, n=20; NT2, n=15), and item debriefing with medical experts. A mixed-methods approach was used to determine the final PRO using two independent datasets: a cross-sectional, US observational NT1 study (n=126) and a global Phase 2 study (NT1/NT2, n=125).

Results Concept elicitation interviews were conducted to generate items deemed relevant by patients and medical experts. The original version was refined after cognitive debriefing with patients in two stand-alone studies yielding a 48-item pilot version. All items have a 7-day recall period on a 5-point Likert scale of frequency from “never” to “always” or severity from “not at all” to “very much.” Content and psychometric validation (including exploratory factor analysis) resulted in a final 28-item, 6-domain version (Tiredness, Cognitive Functioning, Cataplexy [for NT1], Social Activities, Everyday Activities, and Everyday Responsibilities) (FINI v1.0). Summary scores for each domain (6 for NT1, 5 for NT2) were calculated and transformed to a standardized scale from 0 (best health) to 100 (worst health).

Conclusions The FINI is a modular, domain-specific assessment of functional impacts that is fit-for-purpose for patients with NT1 (FINI) and NT2 (FINI-NT2). It demonstrates good content validity and psychometric methods support scoring and interpretation. The FINI can be used in clinical trials or clinical practice for functional recovery in patients with NT1 and NT2.

Authors/Disclosures
heather romero, PhD PRESENTER	Dr. romero has received personal compensation for serving as an employee of Takeda. Dr. romero has stock in Takeda.
Yulia Savva, PhD	Dr. Savva has stock in Takeda. Dr. Savva has stock in Thermo Fischer.
Stephen Crawford, PhD	Dr. Crawford has received personal compensation for serving as an employee of Takeda. Dr. Crawford has stock in Takeda.
Peter McPherson, DPsych (Clin)	Dr. McPherson has received personal compensation for serving as an employee of Clinical Outcomes Solutions.
Helen A. Doll, PhD	Dr. Doll has nothing to disclose.
Amy Howerter, PhD	Dr. Howerter has received personal compensation for serving as an employee of Clinical Outcomes Solutions.
Tara Symonds, PhD	Dr. Symonds has nothing to disclose.
Todd J. Swick, MD, FAAN (Takeda Pharmaceuticals)	Dr. Swick has received personal compensation for serving as an employee of Takeda Pharmaceuticals. Dr. Swick has stock in Takeda Pharmaceuticals.
Yves Dauvilliers, MD, PhD (Hopital Gui De Chaulliac)	Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for idorsia. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for JAZZ. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avadel. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alkermes. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Centessa. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bioprojet. Yves Dauvilliers, MD, PhD has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Harmony Bioscience.

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