Development of a Novel Patient-reported Outcome Measure of Functional Impacts for Narcolepsy Types 1 and 2
Heather Romero1, Yulia Savva1, Stephen Crawford1, Peter McPherson2, Helen Doll2, Amy Howerter2, Tara Symonds2, Todd Swick1, Yves Dauvilliers3
1Takeda Development Center Americas, Inc., 2Clinical Outcomes Solutions, 3Sleep-Wake Disorders Center, Department of Neurology, Gui-de-Chauliac Hospital, University of Montpelier
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.
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