Development of a Conceptual Model of the Patient Experience with Idiopathic Hypersomnia: Patient Interviews from a Clinical Trial (NCT05156047)
Author(s)
Kelly Johnston, MPH1, Salvatore Insana, PhD2, Elizabeth Merikle, PhD1, Michelle Manuel, BS2, Katie Wilmsen, BS2, Carrie K. Presnall, MS1, Chelsea Carlson, PhD1, George Nomikos, MD, PhD2.
1Fortrea, Inc, Durham, NC, USA, 2Harmony Biosciences Management Inc., Plymouth Meeting, PA, USA.
1Fortrea, Inc, Durham, NC, USA, 2Harmony Biosciences Management Inc., Plymouth Meeting, PA, USA.
Presentation Documents
OBJECTIVES: Idiopathic hypersomnia (IH) is a rare neurological disorder characterized by excessive daytime sleepiness (EDS), sleep inertia, cognitive difficulties and compromised alertness. The extent to which IH symptom burden impacts day-to-day life is not fully understood. The goal of this study was to develop a conceptual model that depicts the IH patient experience, which may help inform the selection of endpoints for clinical trials that investigate treatments for IH.
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]). Trained qualitative researchers used a semi-structured interview guide with open-ended questions to elicit participants’ experience living with IH prior to enrollment in the study. Transcripts were analyzed using applied thematic coding techniques to identify salient symptoms of IH and their impact on daily life.
RESULTS: Baseline characteristics of interviewed and non-interviewed participants were similar (female [80%], White/Caucasian [90%], severe EDS [mean ESS score=16.2]). A wide spectrum of symptoms and impacts were spontaneously elicited. Most participants experienced multiple symptoms that had a negative effect on their daily function. Almost all participants described a difficulty staying awake or falling asleep during the day (98.3%) that disrupted their daily life (96.7%). Other prevalent symptoms were poor quality of wakefulness (81.6%), morning awakening problems (73.3%), sleep inertia (70.0%), and daytime tiredness (66.7%). Participants described the significant negative effect these symptoms had on their daily lives, including difficulty at work (71.2%) and interference with relationships and socialization (70.0%).
CONCLUSIONS: Results from these qualitative interviews helped to characterize the patient experience of living with IH. The health experiences described during the interviews informed the development of a conceptual model depicting the patient experience of living with IH. This conceptual model may help to guide the selection of endpoints for future clinical trials.
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]). Trained qualitative researchers used a semi-structured interview guide with open-ended questions to elicit participants’ experience living with IH prior to enrollment in the study. Transcripts were analyzed using applied thematic coding techniques to identify salient symptoms of IH and their impact on daily life.
RESULTS: Baseline characteristics of interviewed and non-interviewed participants were similar (female [80%], White/Caucasian [90%], severe EDS [mean ESS score=16.2]). A wide spectrum of symptoms and impacts were spontaneously elicited. Most participants experienced multiple symptoms that had a negative effect on their daily function. Almost all participants described a difficulty staying awake or falling asleep during the day (98.3%) that disrupted their daily life (96.7%). Other prevalent symptoms were poor quality of wakefulness (81.6%), morning awakening problems (73.3%), sleep inertia (70.0%), and daytime tiredness (66.7%). Participants described the significant negative effect these symptoms had on their daily lives, including difficulty at work (71.2%) and interference with relationships and socialization (70.0%).
CONCLUSIONS: Results from these qualitative interviews helped to characterize the patient experience of living with IH. The health experiences described during the interviews informed the development of a conceptual model depicting the patient experience of living with IH. This conceptual model may help to guide the selection of endpoints for future clinical trials.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR77
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Neurological Disorders, SDC: Rare & Orphan Diseases