Development of a disease-specific health utility score for chronic obstructive pulmonary disease from a discrete choice experiment patient preference study.

OBJECTIVES: While patient input to health technology assessment (HTA) has traditionally been of a qualitative nature, there is increasing interest to integrate quantitative evidence from patient preference studies into HTA decision making. Preference data can be used to generate disease-specific health utility data. We generated a health utility score for patients with chronic obstructive pulmonary disease (COPD) and consider its use within HTAs. METHODS: Based on qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance, and urinary incontinence. We employed a discrete choice experiment (DCE) and the random parameter logistic regression technique to estimate utility scores for all COPD health states. The relationship between patients' COPD health utility scores, self-perceived COPD severity, and EQ-5D-3L utility scores was analyzed, with data stratified according to disease severity and comorbidity subgroups. RESULTS: The COPD health utility score had face validity, with utility scores negatively correlated with patients' self-perceived COPD severity. The correlation between the COPD health utility scores and EQ-5D-3L values was only moderate. While patient EQ-5D-3L scores were impacted by comorbidities, the COPD health utility score was less impacted by comorbid conditions. CONCLUSIONS: Our COPD utility measure, derived from a DCE, provides a patient-centered health utility score and is more sensitive to the COPD health of the individual and less sensitive to other comorbidities. This disease-specific instrument should be considered alongside generic health-related quality of life instruments when valuing new COPD therapies in submissions to licensing and reimbursement agencies.

Health state utility and quality of life measures in patients with chronic myeloid leukemia in France.

PURPOSE: Tyrosine kinase inhibitors (TKIs) have dramatically improved the prognosis of chronic myeloid leukemia (CML). We aimed to assess health state utility and quality of life (QoL) in French patients with CML in real-life setting, to study the determinants of utility score and to compare health-related QoL values to general population norms. METHODS: We conducted a cross-sectional study in 412 patients with CML. Data were collected by electronic survey. Three patient-reported outcomes questionnaires were used: EORTC QLQ-C30, EORTC QLQ-CML24 and EuroQol EQ-5D-3L. Health state utility values were computed using the French value set. We computed deviations from reference norms from the general population. We studied the determinants of health utility score using multiple regression models. RESULTS: The mean utility score (SD) was 0.72 (0.25) in the chronic phase and 0.84 (0.21) in treatment-free remission, with marked variations by gender. Patients with CML had a deviation from the reference norm of -0.15 on average (SD: 0.25). In terms of QoL, social functioning, role functioning and cognitive functioning were notably impacted with a mean difference of -16.0, -13.1 and -11.7 respectively. Fatigue, dyspnea and pain were the symptoms with the highest deviation from general population norms (mean difference of 20.6, 14.0 and 8.3 respectively). In the multiple regression analysis, fatigue was the most important independent predictor of the utility score. CONCLUSION: Although TKIs prevent the disease from progressing and even allow remission without treatment, QoL in patients with CML is notably altered. The utility scores deteriorate with CML symptoms.

The impact of symptoms and comorbidity on health utility scores and health-related quality of life in small cell lung cancer using real world data.

PURPOSE: Small cell lung cancer (SCLC) is a highly fatal disease associated with significant morbidity, with a need for real-world symptom and health utility score (HUS) data. HUS can be measured using an EQ-5D-5L questionnaire, however most captured data is available in non-SCLC (NSCLC) only. As new treatment regimens become available in SCLC it becomes important to understand factors which influence health-related quality of life and health utility. METHODS: A prospective observational cohort study (2012-2017) of ambulatory histologically confirmed SCLC evaluated patient-reported EQ-5D-5L-derived HUS, toxicity and symptoms. A set of NSCLC patients was used to compare differential factors affecting HUS. Clinical and demographic factors were evaluated for differential interactions between lung cancer types. Comorbidity scores were documented for each patient. RESULTS: In 75 SCLC and 150 NSCLC patients, those with SCLC had lower mean HUS ((SCLC vs NSCLC: mean 0.69 vs 0.79); (p < 0.001)) when clinically stable and with progressive disease: ((SCLC mean HUS = 0.60 vs NSCLC mean HUS = 0.77), (p = 0.04)). SCLC patients also had higher comorbidity scores ((1.11 vs 0.73); (p < 0.015)). In multivariable analyses, increased symptom severity and comorbidity scores decreased HUS in both SCLC and NSCLC (p < 0.001); however, only comorbidity scores differentially affected HUS (p < 0.0001), with a greater reduction of HUS adjusted per unit of comorbidity in SCLC. CONCLUSION: Patients with advanced SCLC had significantly lower HUS than NSCLC. Both patient cohorts are impacted by symptoms and comorbidity, however, comorbidity had a greater negative effect in SCLC patients.

EQ-5D and SF-6D health utility scores in patients with spinal and bulbar muscular atrophy.

OBJECTIVE: This study assessed patient-reported health-related quality of life (HRQoL) using two generic preference-based measures in Chinese patients with spinal and bulbar muscular atrophy (SBMA) and identified demographic and clinical determinants of health utility scores in this population. METHODS: This study used cross-sectional data of 212 Chinese patients with SBMA who completed both the EQ-5D and SF-6D. Association between response to EQ-5D and SF-6D dimensions was examined using Spearman's correlation coefficient, and the association between the two utility scores was assessed using Pearson's correlation coefficient. The variations in utility scores across patients in different subgroups were compared using one-way ANOVA. Bland-Altman (B-A) plot was used to assess the agreement of utility scores between EQ-5D and SF-6D. A multivariate Tobit regression model was employed to estimate the association between utility scores and the presence of symptoms and chronic conditions. RESULTS: The mean utility scores for the EQ-5D and SF-6D were 0.54 and 0.56, respectively. The hypothesized correlation between the EQ-5D and SF-6D dimensions ranged from 0.31 to 0.58, and the correlation between their utility scores was 0.64. An acceptable agreement between EQ-5D and SF-6D utility scores was identified by B-A plot. Patients with chronic diseases, misdiagnosis, high financial burden, and several clinical symptoms were highly likely to report a low health utility score. CONCLUSIONS: This study is the first to investigate the HRQoL of patients with SBMA worldwide. The estimated health utility scores for EQ-5D and SF-6D can be utilized as baseline data for future cost-utility analyses of SBMA-related interventions.

Relation between health utility score and physical activity in community-dwelling ambulatory patients with stroke: a preliminary cross-sectional study.

Background The health utility score in patients with stroke relates to physical, psychological, and various other factors. However, the relationship between the health utility score in patients with stroke and objective physical activity has not been clarified. Objective To clarify the relation between the health utility score and objective physical activity in community-dwelling ambulatory patients with stroke. Design A cross-sectional study. Method Patients who received outpatient consultation from a stroke certified nurse after discharge were recruited. We assessed health-related quality of life with the EuroQoL 5-Dimension 3-Level questionnaire and calculated the health utility score. We measured the daily number of steps taken as the index of objective physical activity using an accelerometer. Results Twenty-two patients (72.7% men, 69.5 years old) were included. The health utility score was 0.78 ± 0.14. The physical activity value as indicated by the number of steps taken was 6276.3 ± 4640.7 steps. The health utility score showed a significant positive correlation with the number of steps taken (r = 0.466, p = 0.029). Conclusions The present study showed that the health utility score correlated significantly with objective physical activity in community-dwelling ambulatory patients with stroke. The more the patients with stroke walked, the higher their health utility score was. Further studies should assess other domains of health-related quality of life to comprehensively verify this relationship.

Quantifying Health Utilities in Patients Undergoing Stereotactic Body Radiation Treatment for Liver Metastases for Use in Future Economic Evaluations.

AIM: Stereotactic body radiation therapy (SBRT) is increasingly used as an option for those with liver metastases. In order to facilitate future economic impact of health technologies, health utility scores may be used. The EuroQOL-5D-3L (EQ-5D) preference-based healthy utility instrument was used to evaluate the impact of treatment with SBRT on health utility scores. MATERIALS AND METHODS: Between August 2013 and October 2014, 31 patients treated with 3-5 fractions of SBRT for liver metastases were enrolled in this study. The EQ-5D instrument was administered at baseline, during and up to 6 months post-SBRT. RESULTS: Mean EQ-5D score at baseline was 0.857, which remained stable across the entire study time period. Transient increases in difficulties with mobility (9.7% reported at baseline to 16.1% on the last day of treatment) and usual activities (3.2% reported at baseline to 34.5% on day two) were found during the course of treatment; these returned to baseline levels subsequently. The mean visual analogue score at baseline was 65.8 and remained unchanged throughout treatment and follow-up. CONCLUSIONS: The stability of health utility scores and problems reported by patients undergoing treatment indicate that SBRT for liver metastases does not impart a significant adverse effect on quality of life. These results may be used for future economic evaluation of SBRT.

Prediction of health preference values from CD4 counts in individuals with HIV.

BACKGROUND: A common measure of health benefit in technology assessments is the quality-adjusted life year, which incorporates health preference or utility scores. OBJECTIVE: To build and test a predictive model using CD4 counts to derive health preference scores. DESIGN: Predictive modeling. Setting. Ontario HIV Treatment Network Cohort Study. Measurement. The relationship between HUI3-derived health preference score and HIV health status measured by CD4 count was examined by a regression model. Additional independent variables considered included age, time since HIV diagnosis, AIDS-defining condition, sex, and education level. A polynomial regression model was fit to predict health preference scores. The final model was established using automated backwards stepwise variable elimination using the Akaike information criterion. Tenfold cross-validation was used to assess the model. RESULTS: Data from 841 participants were available. Mean age and time since diagnosis were 46.78 and 11.03 years, respectively. CD4 counts ranged from 2 to 995 cells per mm(3) with 267 (31.75%) individuals having less than 350 cells per mm(3). Mean HUI3 utility score was 0.72 and ranged from -0.25 to 1. The final model retained squared terms for CD4 counts, age, and time since HIV diagnosis and eliminated history of AIDS-defining condition and the nonsquared time since HIV diagnosis. Prediction error was assessed in 14 subgroups using the validation set. Two subgroups had mean prediction errors greater than 0.02. Limitations. All statistical models are limited by the data used to develop and test the model. The model estimates health utility scores primarily through CD4 counts. Therefore, the model may be inappropriate if noninfectious diseases are a significant factor. CONCLUSIONS: Results provide a model for predicting health preference values from CD4 counts.