Characterizing the Experience of Excessive Daytime Sleepiness in Patients with Idiopathic Hypersomnia: Implications for Measuring Daytime Sleepiness with the Epworth Sleepiness Scale in a Clinical Trial (NCT05156047)
Author(s)
Siri Bolding, PhD1, Elizabeth Merikle, PhD1, Salvatore Insana, PhD2, Kelly Johnston, MPH1, Michelle Manuel, BS2, Katie Wilmsen, BS2, George Nomikos, MD, PhD2;
1Fortrea, Durham, NC, USA, 2Harmony Biosciences, Plymouth, PA, USA
1Fortrea, Durham, NC, USA, 2Harmony Biosciences, Plymouth, PA, USA
Presentation Documents
OBJECTIVES: Excessive daytime sleepiness (EDS) is the cardinal symptom of idiopathic hypersomnia (IH). Currently available objective measures of sleepiness are not fit-for-purpose to measure EDS in IH, and their value in evaluating treatment effectiveness is uncertain. The Epworth Sleepiness Scale (ESS) is the most frequently used patient-reported outcome measure of EDS in sleep disorder clinical practice/research. This qualitative study examined how the ESS content maps to IH patients’ experience with EDS and aligns with a Patient Global Impression of Severity (PGI-S) item for EDS.
METHODS: Concept elicitation interviews were conducted in a subset of patients (n=61) diagnosed with IH who participated in a clinical trial (Harmony Biosciences: NCT05156047 [N=214]). Qualitative researchers used a semi-structured interview guide with open-ended questions to elicit participants’ descriptions of EDS and their interpretations of the ESS and PGI-S. Transcripts were analyzed using applied thematic coding techniques.
RESULTS: Baseline characteristics of interviewed and non-interviewed participants were similar (female [80%], White/Caucasian [90%], severe EDS [mean ESS score=16.2]). Almost all participants experienced EDS (98%), and participant descriptions of their experience with dozing off mapped to the activities/situations on the ESS, including being a passenger in a car, in public places, talking to someone, lying down, and during sedentary activities. Participants described differentiating ESS response options by thinking about how the probability of falling asleep, or the amount of effort needed to maintain wakefulness, varied in each situation. Change in severity of EDS was associated with overall change in dozing off behavior.
CONCLUSIONS: This qualitative study demonstrates that ESS items map to descriptions of situations in which patients with IH experience EDS. IH participants were able to accurately interpret ESS item content and response options. This evidence supports the utility of the ESS as an endpoint measure for EDS in clinical trials and the PGI-S as an anchor measure.
METHODS: Concept elicitation interviews were conducted in a subset of patients (n=61) diagnosed with IH who participated in a clinical trial (Harmony Biosciences: NCT05156047 [N=214]). Qualitative researchers used a semi-structured interview guide with open-ended questions to elicit participants’ descriptions of EDS and their interpretations of the ESS and PGI-S. Transcripts were analyzed using applied thematic coding techniques.
RESULTS: Baseline characteristics of interviewed and non-interviewed participants were similar (female [80%], White/Caucasian [90%], severe EDS [mean ESS score=16.2]). Almost all participants experienced EDS (98%), and participant descriptions of their experience with dozing off mapped to the activities/situations on the ESS, including being a passenger in a car, in public places, talking to someone, lying down, and during sedentary activities. Participants described differentiating ESS response options by thinking about how the probability of falling asleep, or the amount of effort needed to maintain wakefulness, varied in each situation. Change in severity of EDS was associated with overall change in dozing off behavior.
CONCLUSIONS: This qualitative study demonstrates that ESS items map to descriptions of situations in which patients with IH experience EDS. IH participants were able to accurately interpret ESS item content and response options. This evidence supports the utility of the ESS as an endpoint measure for EDS in clinical trials and the PGI-S as an anchor measure.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR44
Topic
Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Neurological Disorders, SDC: Rare & Orphan Diseases