Burden and disutility of sleep disturbance and early morning OFF symptoms in people with advancing Parkinson’s disease: a vignette-based approach using the EQ-5D-5L

Parkinson’s disease (PD) is characterized by motor and non-motor symptoms that fluctuate as oral medication wears off, causing periods when symptoms return (“OFF” time) and periods of control (“ON” time). These fluctuations often occur at night and early morning. Sleep disturbance (SD) and early-morning OFF time (EMO) have been shown to decrease health-related quality of life (HRQoL) in people with PD (PwP). However, limited evidence exists on how these symptoms impact health state utility values (HSUVs) used in economic modeling. This study aimed to estimate the burden and disutility of SD and EMO in PwP through vignettes valued using the EQ-5D-5 L. An online survey was completed by adults (≥ 30 years) with self-reported PD diagnosis for ≥ 5 years and ≥ 2 h/day of OFF time, on oral PD medications, and residing in the US or UK. The survey included EQ-5D-5 L and EQ-VAS assessments for 4 vignettes—No SD or EMO, SD (without EMO), EMO (without SD), and Both SD and EMO—plus questions exploring the burden of SD and EMO. EQ-5D-5 L responses were converted into US utility values to estimate HSUVs and the disutility of SD and EMO. Seventy-five individuals completed the survey; 52.00% were male, and 78.66% resided in the US. The average age was 64.32 years, with a mean time since diagnosis of 9.82 years and an average OFF time of 3.91 h/day. In the previous week, 96.00% and 98.67% of respondents reported experiencing SD and EMO, respectively. The presence of either SD (HSUV: 0.796) or EMO (HSUV: 0.701) alone resulted in lower EQ-5D-5 L utilities, with the presence of both valued least (HSUV: 0.528), compared with when both SD and EMO were absent (HSUV: 0.911). EQ-VAS scores ranged from 81.01 for no symptoms to 48.41 for both. SD and EMO impose a significant burden on PwP and decrease HSUVs. As SD and EMO were associated with significant disutility, they should be incorporated into economic models to comprehensively assess PD treatment benefits. Raising awareness among PwP and healthcare providers and prioritizing treatments that minimize SD and EMO can reduce their burden and improve HRQoL for PwP. People with Parkinson’s (PD) often struggle with sleep disturbances (SD) and early morning OFF (EMO) symptoms, which can seriously affect their health-related quality of life (HRQoL). It is essential to comprehensively assess the value of PD treatments, and ignoring these symptoms in economic evaluations underestimates their true impact. Health Technology Assessment (HTA) bodies such as the National Institute for Health and Care Excellence, Haute Autorité de Santé, and Zorginstituut Nederland prefer HRQoL to be measured using the EQ-5D measure to generate health state utility values (HSUVs) for use in economic evaluations. The aim of this study was to estimate HSUVs associated with SD and EMO in people with advancing PD (aPD) through vignettes valued using the EQ-5D-5L. SD and EMO are highly prevalent in people with aPD (PwP) and negatively affect their HRQoL. We estimated the burden and the impact of SD and EMO on HRQoL to ensure a more comprehensive assessment of treatment value. Estimated HSUVs demonstrated the meaningful negative impact of SD and EMO on HRQoL, with EMO having greater impact than SD, as reflected particularly in the EQ-5D domains of mobility, self-care, and usual activities. Given the high prevalence of SD and EMO in PwP and their significant impact on HRQoL, it is essential to prioritize treatments that offer continuous symptom control throughout the entire day and night.