Utility Estimates For Health States Research Articles

Cataloging health state utility estimates for Duchenne muscular dystrophy and related conditions

BackgroundDuchenne muscular dystrophy (DMD) is a genetic disease resulting in progressive muscle weakness, loss of ambulation, and cardiorespiratory complications. Direct estimation of health-related quality of life for patients with DMD is challenging, highlighting the need for proxy measures. This study aims to catalog and compare existing published health state utility estimates for DMD and related conditions.MethodsUsing two search strategies, relevant utilities were extracted from the Tufts Cost-Effectiveness Analysis Registry, including health states, utility estimates, and study and patient characteristics. Analysis One identified health states with comparable utility estimates to a set of published US patient population utility estimates for DMD. A minimal clinically important difference of ± 0.03 was applied to each DMD utility estimate to establish a range, and the registry was searched to identify other health states with associated utilities that fell within each range. Analysis Two used pre-defined search terms to identify health states clinically similar to DMD. Mapping was based on the degree of clinical similarity.ResultsAnalysis One identified 4,308 unique utilities across 2,322 cost-effectiveness publications. The health states captured a wide range of acute and chronic conditions; 34% of utility records were extrapolated for US populations (n = 1,451); 1% were related to pediatric populations (n = 61). Analysis Two identified 153 utilities with health states clinically similar to DMD. The median utility estimates varied among identified health states. Health states similar to the early non-ambulatory DMD phase exhibited the greatest difference between the median estimate of the sample (0.39) and the existing estimate from published literature (0.21).ConclusionsWhen available estimates are limited, using novel search strategies to identify utilities of clinically similar conditions could be an approach for overcoming the information gap. However, it requires careful evaluation of the utility instruments, tariffs, and raters (proxy or self).

Analyzing quality of life among people with opioid use disorder from the National Institute on Drug Abuse Data Share initiative: implications for decision making.

We aimed to estimate health state utility values (HSUVs) for the key health states found in opioid use disorder (OUD) cost-effectiveness models in the published literature. Data obtained from six trials representing 1,777 individuals with OUD. We implemented mapping algorithms to harmonize data from different measures of quality of life (the SF-12 Versions 1 and 2 and the EQ-5D-3L). We performed a regression analysis to quantify the relationship between HSUVs and the following variables: days of extra-medical opioid use in the past 30 days, injecting behaviors, treatment with medications for OUD, HIV status, and age. A secondary analysis explored the impact of opioid withdrawal symptoms. There were statistically significant reductions in HSUVs associated with extra-medical opioid use (-0.002 (95% CI [-0.003,-0.0001]) to -0.003 (95% CI [-0.005,-0.002]) per additional day of heroin or other opiate use, respectively), drug injecting compared to not injecting (-0.043 (95% CI [-0.079,-0.006])), HIV-positive diagnosis compared to no diagnosis (-0.074 (95% CI [-0.143,-0.005])), and age (-0.001 per year (95% CI [-0.003,-0.0002])). Parameters associated with medications for OUD treatment were not statistically significant after controlling for extra-medical opioid use (0.0131 (95% CI [-0.0479,0.0769])), in line with prior studies. The secondary analysis revealed that withdrawal symptoms are a fundamental driver of HSUVs, with predictions of 0.817 (95% CI [0.768, 0.858]), 0.705 (95% CI [0.607, 0.786]), and 0.367 (95% CI [0.180, 0.575]) for moderate, severe, and worst level of symptoms, respectively. We observed HSUVs for OUD that were higher than those from previous studies that had been conducted without input from people living with the condition.

Health state utilities associated with treatment burden in cystic fibrosis: a patient valuation study

BACKGROUNDWhile recent advancements in the treatment of cystic fibrosis (CF) have improved survival, reducing high levels of treatment burden remains a priority issue for many people with CF (pwCF). However, economic evaluations of novel interventions may fail to capture their impact on treatment burden due to a lack of suitable outcome measures. This study aimed to estimate health state utilities (HSUs), for changes in treatment burden associated with different CF treatments. RESEARCH QUESTIONWhat value do pwCF place on changes in treatment burden associated with intravenous antibiotic treatment of pulmonary exacerbations, inhaled medicines, and physiotherapy? STUDY DESIGNAND METHODS: Adults attending a specialist CF center were invited to participate in a web-based time trade-off interview. Participants valued their own health, and five health state vignettes describing varying levels of intensity of physiotherapy, inhaled, and IV antibiotic treatment. HSUs for additional instances of each treatment type were estimated using mixed effect linear regression models. RESULTSFifty one people with CF completed the interview (median age, 30 years; range, 19-66); 53% were female; mean FEV(1) % predicted, 65% [SD, 20%]).Mean utility scores for own health were very similar between the EQ-5D index value (0.81, SD 0.20) and the TTO value (0.82, SD 0.20), however limited concordance was observed at the individual level. Adjusted utility decrements associated with treatment burden were -0.037 (SE 0.008) for an additional annual IV antibiotic treatment, -0.029 (SE 0.014) for an additional daily physiotherapy session, and -0.019 (SE 0.013) for an additional daily inhaled medicine. INTERPRETATIONIncreasing treatment burden was associated with decreasing HSU values. The utility decrements associated with treatment burden changes suggest meaningful differences in health-related quality of life (HRQoL) for pwCF. These findings align with existing literature on the impact of treatment burden on HRQoL, and highlight the importance of considering treatment burden in economic evaluations of interventions in CF.

PCR238 Estimating Health State Utilities (HSUS) for Aromatic L-Amino Acid Decarboxylase Deficiency (AADCD) in the United States

PCR238 Estimating Health State Utilities (HSUS) for Aromatic L-Amino Acid Decarboxylase Deficiency (AADCD) in the United States

Hypophosphatemia attenuates improvements in vitality after intravenous iron treatment in patients with inflammatory bowel disease

PurposeIron deficiency anemia is common in people with inflammatory bowel disease (IBD), causing deterioration in quality of life, which can be reversed by treatment that increases iron stores and hemoglobin levels. The present post hoc analyses estimate health state utility values for patients with IBD after treatment with ferric derisomaltose or ferric carboxymaltose and evaluate the health domains driving the changes.MethodsSF-36v2 responses were recorded at baseline and day 14, 35, 49, and 70 from 97 patients enrolled in the randomized, double-blind, PHOSPHARE-IBD trial (ClinicalTrials.gov ID: NCT03466983), in which patients with IBD across five European countries were randomly allocated to either ferric derisomaltose or ferric carboxymaltose. Changes in SF-36v2 scale scores and SF-6Dv2 health utility values were analyzed by mixed models.ResultsIn both treatment arms, SF-6Dv2 utility values and all SF-36v2 scale scores, except Bodily Pain, improved significantly (p = < 0.0001). The improvement in SF-6Dv2 utility values showed no significant treatment group difference. The improvement in utility values was completely explained by improvement in Vitality scores. Vitality scores showed significantly larger improvement with ferric derisomaltose versus ferric carboxymaltose (p = 0.026). Patients with the smallest decrease in phosphate had significantly larger improvements in Vitality scores at each time point (p = < 0.05 for all comparisons) and overall (p = 0.0006).ConclusionsUtility values improved significantly with intravenous iron treatment. Improvement in utility values was primarily driven by Vitality scores, which showed significantly greater improvement in the ferric derisomaltose arm. Smaller decreases in phosphate were associated with significantly higher Vitality scores, suggesting that quality of life improvement is attenuated by hypophosphatemia. The utility values can inform future cost-utility analysis.

The Hidden Toll of Psychological Distress in Australian Adults and Its Impact on Health-Related Quality of Life Measured as Health State Utilities.

Psychological distress (PD) is a major health problem that affects all aspects of health-related quality of life including physical, mental and social health, leading to a substantial human and economic burden. Studies have revealed a concerning rise in the prevalence of PD and various mental health conditions among Australians, particularly in female individuals. There is a scarcity of studies that estimate health state utilities (HSUs), which reflect the overall health-related quality of life in individuals with PD. No such studies have been conducted in Australia thus far. We aimed to evaluate the age-specific, sex-specific and PD category-specific HSUs (disutilities) in Australian adults with PD to inform healthcare decision making in the management of PD. Data on age, sex, SF-36/SF6D responses, Kessler psychological distress (K10) scale scores and other characteristics of N = 15,139 participants (n = 8149 female individuals) aged >15 years were derived from the latest wave (21) of the nationally representative Household, Income and Labor Dynamics in Australia survey. Participants were grouped into the severity categories of no (K10 score: 10-19), mild (K10: 20-24), moderate (K10: 25-29) and severe PD (K10: 30-50). Both crude and adjusted HSUs were calculated from participants' SF-36 profiles, considering potential confounders such as smoking, marital status, remoteness, education and income levels. The calculations were based on the SF-6D algorithm and aligned with Australian population norms. Additionally, the HSUs were stratified by age, sex and PD categories. Disutilities of PD, representing the mean difference between HSUs of people with PD and those without, were also calculated for each group. The average age of individuals was 46.130 years (46% male), and 31% experienced PD in the last 4 weeks. Overall, individuals with PD had significantly lower mean HSUs than those likely to be no PD, 0.637 (95% confidence interval [CI] 0.636, 0.640) vs 0.776 (95% CI 0.775, 0.777) i.e. disutility: -0.139 [95% CI -0.139, -0.138]). Mean disutilities of -0.108 (95% CI -0.110, -0.104), -0.140 (95% CI -0.142, -0.138), and -0.188 (95% CI -0.190, -0.187) were observed for mild PD, moderate PD and severe PD, respectively. Disutilities of PD also differed by age and sex groups. For instance, female individuals had up to 0.049 points lower mean HSUs than male individuals across the three classifications of PD. There was a clear decline in health-related quality of life with increasing age, demonstrated by lower mean HSUs in older population age groups, that ranged from 0.818 (95% CI 0.817, 0.818) for the 15-24 years age group with no PD to 0.496 (95% CI 0.491, 0.500) for the 65+ years age group with severe PD). Across all ages and genders, respondents were more likely to report issues in certain dimensions, notably vitality, and these responses did not uniformly align with ageing. The burden of PD in Australia is substantial, with a significant impact on female individuals and older individuals. Implementing age-specific and sex-specific healthcare interventions to address PD among Australian adults may greatly alleviate this burden. The PD state-specific HSUs calculated in our study can serve as valuable inputs for future health economic evaluations of PD in Australia and similar populations.

The association between health-related quality of life and problem gambling severity: a cross-sectional analysis of the Health Survey for England

BackgroundProblem gambling can lead to health-related harms, such as poor mental health and suicide. In the UK there is interest in introducing guidance around effective and cost-effective interventions to prevent harm from gambling. There are no estimates of the health state utilities associated with problem gambling severity from the general population in the UK. These are required to determine the cost-effectiveness of interventions. This study aims to use an indirect elicitation method to estimate health state utilities, using the EQ-5D, for various levels of problem gambling and gambling-related harm.MethodsWe used the Health Survey for England to estimate EQ-5D-derived health state utilities associated with the different categories of the Problem Gambling Severity Index (PGSI), PGSI score and a 7-item PGSI-derived harms variable. Propensity score matching was used to create a matched dataset with respect to risk factors for problem gambling and regression models were used to estimate the EQ-5D-derived utility score and the EQ-5D domain score whilst controlling for key comorbidities. Further exploratory analysis was performed to look at the relationship between problem gambling and the individual domains of the EQ-5D.ResultsWe did not find any significant attributable decrements to health state utility for any of the PGSI variables (categories, score and 7-item PGSI derived harms variable) when key comorbidities were controlled for. However, we did find a significant association between the 7-item PGSI derived harms variable and having a higher score (worse health) in the anxiety/depression domain of the EQ-5D, when comorbidities were controlled for.ConclusionsThis study found no significant association between problem gambling severity and HRQoL measured by the EQ-5D when controlling for comorbidities. There might be several reasons for this including that this might reflect the true relationship between problem gambling and HRQoL, the sample size in this study was insufficient to detect a significant association, the PGSI is insufficient for measuring gambling harm, or the EQ-5D is not sensitive enough to detect the changes in HRQoL caused by gambling. Further research into each of these possibilities is needed to understand more about the relationship between problem gambling severity and HRQoL.

Cost-effectiveness analysis design for interventions to prevent children's oral disease.

In 2015, the National Institute of Dental and Craniofacial Research (NIDCR) launched the Multidisciplinary Collaborative Research Consortium to Reduce Oral Health Disparities in Children, supporting four randomized trials testing strategies to improve preventive care. A Coordinating Center provides scientific expertise, data acquisition and quality assurance services, safety monitoring, and final analysis-ready datasets. This paper describes the trials' economic analysis strategies, placing these strategies within the broader context of contemporary economic analysis methods. The Coordinating Center established a Cost Collaborative Working Group to share information from the four trials about the components of their economic analyses. Study teams indicated data sources for their economic analysis using a set of structured tables. The Group meets regularly to share progress, discuss challenges, and coordinate analytic approaches. All four trials will calculate incremental cost-effectiveness ratios; two will also conduct cost-utility analyses using proxy diseases to estimate health state utilities. Each trial will consider at least two perspectives. Key process measures include dental services provided to child participants. The non-preference-weighted Early Childhood Oral Health Impact Scale (ECOHIS) will measure oral health-related quality of life. All trials are measuring training, implementation, personnel and supervision, service, supplies, and equipment costs. Consistent with best practices, all four trials have integrated economic analysis during their planning stages. This effort is critical since poor quality or absence of essential data can limit retrospective analysis. Integrating economic analysis into oral health preventive intervention research can provide guidance to clinicians and practices, payers, and policymakers.

Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L

BackgroundThe progression of Duchenne muscular dystrophy (DMD) is characterized by loss of ambulation, respiratory insufficiency, cardiomyopathy, and early mortality. DMD profoundly impacts health-related quality-of-life (HRQoL). However, few health state utility data exist; published utilities tend to be derived from small samples for a limited number of health states and are often based on caregiver-reported patient health status. This study estimated utility values for varied clinical and functional health states in DMD, based on patient-reported health status.MethodsIndividuals with DMD in the US aged 12–40 years completed the EQ-5D (5-level) and Health Utilities Index (HUI) preference-based instruments. Based on responses to a clinical questionnaire, participants self-classified into functional health states according to level of lower and upper limb function, use of respiratory support, and presence of cardiomyopathy. Mean [standard deviation (SD)] utility and EQ-5D visual analogue scale (VAS) scores were estimated according to health state; and median (interquartile range) attribute levels calculated to understand which domains of health are most severely affected in DMD.ResultsOf 63 males with DMD, mean (SD) age was 19.8 (6.1) years and 11 (17.5%) were ambulatory. Mean (SD) utility values were 0.92 (0.08; HUI2), 0.84 (0.20; HUI3), and 0.84 (0.13; EQ-5D) for ambulatory patients without cardiomyopathy (n = 10). For non-ambulatory patients with moderately impaired upper limb function, night and daytime ventilation without cardiomyopathy, mean (SD) utilities were 0.49 (0.07) for the HUI2, 0.16 (0.15) for the HUI3 and 025 (0.14) for the EQ-5D. Mean (SD) VAS scores for the same health states were 91 (9) and 83 (21), respectively. In addition to impairments in mobility/ambulation, and self-care, attributes like usual activities and pain also showed notable effects of DMD.ConclusionsIn DMD, although a relationship between disease progression and HRQoL is observed, there is large variability in utility within functional health states, and across instruments. Utility values for less severe non-ambulatory health states described by level of upper limb function are novel. These utility values, derived based on direct patient feedback rather than from caregiver report, are relevant to individuals of varying functional statuses and augment scarce DMD-specific utility data.

NCOG-31. TUMOR TREATING FIELDS: REAL-WORLD SURVIVAL AND QUALITY OF LIFE OUTCOMES ON THE XCELSIOR PLATFORM

Abstract OBJECTIVE xCures operates a direct-to-patient, real-world evidence platform according to the nationwide observational research protocol XCELSIOR (NCT03793088), which aggregates medical data in an FDA-compliant electronic database. We used this platform to analyze advanced registry survival and Quality of Life (QoL) outcomes associated with the use of Tumor Treating Fields (TTFields) for treatment of high grade central nervous system (CNS) tumors. METHODS XCELSIOR participants with high grade CNS tumors were invited weekly to complete the EuroQol 5-Dimension (EQ-5D-5L) QoL questionnaire over the course of one year. A EuroQol-endorsed model was used to estimate health state utilities from patients’ EQ-5D responses. The participating patients’ clinical data was accessed from the XCELSIOR platform to enable study of clinical factors and survival outcomes. RESULTS 225 patients with high grade CNS tumors in total completed at least one EQ-5D questionnaire. 88 patients (39%) had ever used TTFields and 58 of these patients (26%) were currently using TTFields. Patients currently using TTFields had significantly higher average health state utility estimates compared to non-users (p=.00183), with a mean improvement in utility of .14 (SE=.04), indicating significantly higher overall QoL in TTFields users. Mortality events were recorded for 40 participating patients, 14 (35%) of which had used TTFields. From these 40 events the mean/median overall survival (OS) for patients who had used TTFields was 23.39/20.15 months vs 15.77/12.72 months for patients who had not used TTFields, showing significantly higher average OS in patients who had used TTFields (p=.04139). CONCLUSION The XCELSIOR platform has enabled the study of real-world clinical and QoL outcomes for patients with CNS tumors. The results show that use of TTFields is associated with improved survival as well as improved overall Quality of Life for patients in a real-world setting, corroborating results seen in trial settings as well as results seen in other real-world studies.

Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation

Background: Health state utilities are measures of health-related quality of life that reflect the value placed on improvements in patients’ health status and are necessary for estimation of quality-adjusted life-years. Health state utility data on Fabry disease (FD) are limited. In this study we used vignette (scenario) construction and valuation to develop health state utilities. Objectives: The aim of this study was to use vignette construction and valuation to estimate health state utility values suitable for inclusion in economic models of FD treatments. Methods: Health state vignettes were developed from semistructured qualitative telephone interviews with patients with FD and informed by published literature and input from an expert. Each vignette was valued in an online survey by members of the United Kingdom (UK) general population using the composite time trade-off (TTO) method, which aims to determine the time the respondent would trade to live in full health compared with each impaired health state. Results: Eight adults (50% women) with FD from the UK were interviewed. They were recruited via various approaches, including patient organizations and social media. The interviewees’ responses, evidence from published literature, and input from a clinical expert informed the development of 6 health state vignettes (pain, moderate clinically evident FD [CEFD], severe CEFD, end-stage renal disease [ESRD], stroke, and cardiovascular disease [CVD]) and 3 combined health states (severe CEFD + ESRD, severe CEFD + CVD, and severe CEFD + stroke). A vignette valuation survey was administered to 1222 participants from the UK general population who were members of an external surveying organization and agreed to participate in this study; 1175 surveys were successfully completed and included in the analysis. Responses to TTO questions were converted into utility values for each health state. Pain was the highest valued health state (0.465), and severe CEFD + ESRD was the lowest (0.033). Discussion: Overall, mean utility values declined as the severity of the vignettes increased, indicating that respondents were more willing to trade life-years to avoid a severe health state. Conclusions: Health state vignettes reflect the effects of FD on all major health-related quality-of-life domains and may help to support economic modeling for treatment of FD.

Estimation of Health State Utility Values in Fabry Disease Using Vignette Development and Valuation.

Background: Health state utilities are measures of health-related quality of life that reflect the value placed on improvements in patients' health status and are necessary for estimation of quality-adjusted life-years. Health state utility data on Fabry disease (FD) are limited. In this study we used vignette (scenario) construction and valuation to develop health state utilities. Objectives: The aim of this study was to use vignette construction and valuation to estimate health state utility values suitable for inclusion in economic models of FD treatments. Methods: Health state vignettes were developed from semistructured qualitative telephone interviews with patients with FD and informed by published literature and input from an expert. Each vignette was valued in an online survey by members of the United Kingdom (UK) general population using the composite time trade-off (TTO) method, which aims to determine the time the respondent would trade to live in full health compared with each impaired health state. Results: Eight adults (50% women) with FD from the UK were interviewed. They were recruited via various approaches, including patient organizations and social media. The interviewees' responses, evidence from published literature, and input from a clinical expert informed the development of 6 health state vignettes (pain, moderate clinically evident FD [CEFD], severe CEFD, end-stage renal disease [ESRD], stroke, and cardiovascular disease [CVD]) and 3 combined health states (severe CEFD + ESRD, severe CEFD + CVD, and severe CEFD + stroke). A vignette valuation survey was administered to 1222 participants from the UK general population who were members of an external surveying organization and agreed to participate in this study; 1175 surveys were successfully completed and included in the analysis. Responses to TTO questions were converted into utility values for each health state. Pain was the highest valued health state (0.465), and severe CEFD + ESRD was the lowest (0.033). Discussion: Overall, mean utility values declined as the severity of the vignettes increased, indicating that respondents were more willing to trade life-years to avoid a severe health state. Conclusions: Health state vignettes reflect the effects of FD on all major health-related quality-of-life domains and may help to support economic modeling for treatment of FD.

Health state utility estimation of Mycobacterium avium complex pulmonary disease using a time trade-off approach

AimsTo obtain appropriate health state utility values for cost-effectiveness analyses of new Mycobacterium avium complex pulmonary disease (MAC-PD) treatments. The impact of MAC-PD severity and symptoms on quality of life (QoL) also were quantified.MethodsA questionnaire describing four health states, MAC-positive severe, MAC-positive moderate, MAC-positive mild, and MAC-negative, was developed based on St. George’s Respiratory Questionnaire (SGRQ) Symptom and Activity scores from the CONVERT trial. The time trade-off (TTO) method with ping-pong titration procedure was used to estimate health state utilities. Regression analyses assessed the impacts of covariates.ResultsOf 319 Japanese adults (49.8% female, mean age 44.8 years), mean (95% CI) health state utility scores (MAC-positive severe, MAC-positive moderate, MAC-positive mild, and MAC-negative) were 0.252 (0.194–0.310), 0.535 (0.488–0.582), 0.816 (0.793–0.839), and 0.881 (0.866–0.896), respectively. MAC-negative state utility scores were significantly higher than MAC-positive severe (mean difference [95% CI], 0.629 [0.574–0.684]), MAC-positive moderate (0.346 [0.304–0.389]), and MAC-positive mild (0.065 [0.048–0.082]) scores (p < .001 each). Most participants would trade survival duration to avoid MAC-positive states (97.5% to avoid MAC-positive severe; 88.7% MAC-positive moderate; 61.4% MAC-positive mild). Regression analyses to investigate the impact of background characteristics showed similar utility differences between health states when not adjusted for covariates.LimitationsSome participant demographics differed from the general population; however, this did not impact utility differences among health states as regression analyses adjusting for demographics did not affect these differences. Similar investigations are needed among patients with MAC-PD and in other countries.ConclusionsThis study evaluating the impact of MAC-PD on utilities using the TTO method demonstrates that differences in utilities are dependent on the severity of respiratory symptoms and their impacts on daily activities and QoL. These results could contribute to a better quantification of the value of MAC-PD treatments and improve assessments of cost-effectiveness.

Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom.

Background: Late-onset Pompe disease (LOPD) is a rare, progressive neuromuscular condition typically characterized by weakness of skeletal muscles, including those involved in respiration and diaphragmatic dysfunction. Individuals with LOPD typically eventually require mobility and/or ventilatory support. Objectives: This study aimed to develop health state vignettes and estimate health state utility values for LOPD in the United Kingdom. Methods: Vignettes were developed for 7 health states of LOPD with states defined in terms of mobility and/or ventilatory support. Vignettes were drafted based on patient-reported outcome data from the Phase 3 PROPEL trial (NCT03729362) and supplemented by a literature review. Qualitative interviews with individuals living with LOPD and clinical experts were conducted to explore the health-related quality-of-life (HRQoL) impact of LOPD and to review the draft vignettes. Vignettes were finalized following a second round of interviews with individuals living with LOPD and used in health state valuation exercises with people of the UK population. Participants rated the health states using the EQ-5D-5L, visual analogue scale, and time trade-off interviews. Results: Twelve individuals living with LOPD and 2 clinical experts were interviewed. Following the interviews, 4 new statements were added regarding dependence on others, bladder control problems, balance issues/fear of falling, and frustration. One hundred interviews with a representative UK population sample were completed. Mean time trade-off utilities ranged from 0.754 (SD = 0.31) (no support) to 0.132 (SD = 0.50) (invasive ventilatory and mobility support-dependent). Similarly, EQ-5D-5L utilities ranged from 0.608 (SD = 0.12) to -0.078 (SD = 0.22). Discussion: The utilities obtained in the study are consistent with utilities reported in the literature (0.670-0.853 for nonsupport state). The vignette content was based on robust quantitative and qualitative evidence and captured the main HRQoL impacts of LOPD. The general public rated the health states consistently lower with increasing disease progression. There was greater uncertainty around utility estimates for the severe states, suggesting that participants found it harder to rate them. Conclusion: This study provides utility estimates for LOPD that can be used in economic modeling of treatments for LOPD. Our findings highlight the high disease burden of LOPD and reinforce the societal value of slowing disease progression.

Quality of Life with Late-Onset Pompe Disease: Qualitative Interviews and General Public Utility Estimation in the United Kingdom

Background: Late-onset Pompe disease (LOPD) is a rare, progressive neuromuscular condition typically characterized by weakness of skeletal muscles, including those involved in respiration and diaphragmatic dysfunction. Individuals with LOPD typically eventually require mobility and/or ventilatory support. Objectives: This study aimed to develop health state vignettes and estimate health state utility values for LOPD in the United Kingdom. Methods: Vignettes were developed for 7 health states of LOPD with states defined in terms of mobility and/or ventilatory support. Vignettes were drafted based on patient-reported outcome data from the Phase 3 PROPEL trial (NCT03729362) and supplemented by a literature review. Qualitative interviews with individuals living with LOPD and clinical experts were conducted to explore the health-related quality-of-life (HRQoL) impact of LOPD and to review the draft vignettes. Vignettes were finalized following a second round of interviews with individuals living with LOPD and used in health state valuation exercises with people of the UK population. Participants rated the health states using the EQ-5D-5L, visual analogue scale, and time trade-off interviews. Results: Twelve individuals living with LOPD and 2 clinical experts were interviewed. Following the interviews, 4 new statements were added regarding dependence on others, bladder control problems, balance issues/fear of falling, and frustration. One hundred interviews with a representative UK population sample were completed. Mean time trade-off utilities ranged from 0.754 (SD = 0.31) (no support) to 0.132 (SD = 0.50) (invasive ventilatory and mobility support–dependent). Similarly, EQ-5D-5L utilities ranged from 0.608 (SD = 0.12) to -0.078 (SD = 0.22). Discussion: The utilities obtained in the study are consistent with utilities reported in the literature (0.670-0.853 for nonsupport state). The vignette content was based on robust quantitative and qualitative evidence and captured the main HRQoL impacts of LOPD. The general public rated the health states consistently lower with increasing disease progression. There was greater uncertainty around utility estimates for the severe states, suggesting that participants found it harder to rate them. Conclusion: This study provides utility estimates for LOPD that can be used in economic modeling of treatments for LOPD. Our findings highlight the high disease burden of LOPD and reinforce the societal value of slowing disease progression.

Estimating health state utilities in primary hyperoxaluria type 1: a valuation study

AimsQuantitative data on health state utilities in primary hyperoxaluria type 1 (PH1) are lacking. This study was conducted to estimate utility values in PH1 using 3 standard valuation techniques.Materials and methodsHealth state vignettes were developed with input from expert clinicians to describe different stages of chronic kidney disease (CKD) within the setting of PH1, along with a post-combined liver and kidney transplant (CLKT) health state ≥12 months following transplantation. The utility associated with living in each PH1 health state, as described by the vignettes, was evaluated by members of the UK general public using standard utility assessment techniques, including EQ-5D-5L, Visual Analog Scale, and Time Trade-Off.ResultsA similar pattern across the three valuation methods was observed. Utility values were roughly constant from CKD stage 1–3b and then dropped sharply from these states to CKD stage 4. Decreases in utility in the later stages of CKD (stages 4–5) were followed by a recovery in quality of life in the post-CLKT health state.LimitationsVignettes may not fully capture the burden of living with PH1.ConclusionsThis study serves as an informative source of data on how the burden of PH1 varies across disease stages of CKD and post-CLKT in patients with PH1. The study findings highlight the unique clinical features of PH1 compared with non-PH1-related CKD, such as the need for earlier and more intensive hemodialysis, the risk of systemic oxalosis, and the potential need for CLKT (as opposed to kidney-only transplant). The impact of PH1 on health-related quality of life, which worsens once hemodialysis is required and systemic disease manifestations occur, is captured in this study using quantitative health state utilities. These data provide an understanding of the impact of PH1 on health-related quality of life and will facilitate health economic evaluation of future treatments.

Patient preferences and health state utilities associated with the treatment process of antiretroviral therapy for people living with HIV

PurposePeople living with HIV (PLHIV) have reported challenges associated with daily oral antiretroviral therapy (ART), including missed doses, negative psychological impact, and difficulty remaining discreet while at home or traveling. Recently approved long-acting injectable (LAI) ART may help eliminate these concerns. The purpose of this study was to examine patient preferences and estimate health state utilities associated with oral and LAI treatment for ART.MethodsFour health state vignettes were developed based on published literature, clinician interviews, and a pilot study. All vignettes included the same description of HIV, but differed in treatment regimens: (A) single daily oral tablet, (B) two daily oral tablets, (C) injections once monthly, and (D) injections every two months. PLHIV in the UK reported their preferences and valued the health states in time trade-off utility interviews.ResultsThe sample included 201 PLHIV (83.1% male; mean age = 44.9y). The health states frequently selected as most preferable were D (n = 119; 59.2%) and A (n = 75; 37.3%). Utility differences among health states were relatively small, which is typical for treatment process utilities (mean utilities: A, 0.908; B, 0.905; C, 0.900; D, 0.910). Statistically significant differences in utility were found for one vs. two tablets and injections every month vs. every two months (p < 0.001). Participants’ quotations highlight the wide range of reasons for treatment process preferences.ConclusionsCurrent results indicate that many PLHIV would prefer LAI ART. The reported utilities may be useful in economic modeling comparing oral vs. LAI ART.

QOL-21. REAL-WORLD QUALITY-OF-LIFE AND HEALTH UTILITIES FOR PATIENTS WITH CNS CANCERS ON THE XCELSIOR PLATFORM

Abstract OBJECTIVE xCures operates a direct-to-patient, real-world evidence platform according to the nationwide observational research protocol XCELSIOR (NCT03793088), which aggregates medical data in an FDA-compliant electronic database. There is a relative scarcity of published results on real-world Quality-of-Life (QoL) outcomes and resulting health state utilities for patients with Central Nervous System (CNS) cancers. We sought to use the xCures platform to help address this issue. METHODS XCELSIOR participants were invited weekly to complete the EuroQol 5-Dimension (EQ-5D-5L) QoL survey. A EuroQol-endorsed model was used to estimate health state utilities from patient surveys. Survey results, utility estimates, and clinical information were analyzed to understand the influence of common mutations in glioma on patient reported outcomes. RESULTS 30 patients (21 glioblastoma, 4 oligodendroglioma, 2 diffuse midline glioma, 2 diffuse astrocytoma, 1 Schwannoma) completed at least one EQ-5D-5L survey over the course of 9 weeks. Health state utilities were positively correlated (p&amp;lt; .03) with Karnofsky Performance Score (KPS). The average utility for EGFR-mutated patients (N=6) was .578 (SD=.334) while for EGFR-wildtype patients (N=7) it was .747 (SD=.227). The average utility for MGMT-methylated patients (N=13) was .816 (SD=.139) while for non-methylated patients (N=10) it was .583 (SD=.379). The average utility for IDH-mutated patients (N=9) was .789 (SD=.153) while for non-mutated patients (N=17) it was .685 (SD=.324). The average utility for patients with a 1p/19q co-deletion (N=4) was .837 (SD=.126) while for those without a co-deletion (N=8) it was .744 (SD=.244). CONCLUSION The xCures platform has enabled the study of real-world QoL outcomes for patients with CNS cancers. This is the first report studying the influence of genetic mutations on QoL collected through patient reported outcomes. Future research aims to perform similar studies with larger number of patients.

P08.11.A A subgroup analysis of utilities from European patients using tumor treating fields for glioblastoma

Abstract Background Recently a large-scale, international survey was undertaken studying real-world Health-Related Quality of Life (HRQoL) outcomes for patients using Tumor Treating Fields (TTFields) for treatment of glioblastoma (GBM). It is the largest published HRQoL study in GBM to date (Palmer et al. 2021). The study issued the EuroQol 5-Dimension (EQ-5D) survey to 2,815 patients using TTFields therapy for GBM in the US (2,182 patients) and Europe (633 patients). Material and Methods We analyzed EQ-5D results reported from European patients using TTFields for GBM in the above study. We used a EuroQol-endorsed model (Andrade et al. 2020) to estimate health state utilities from these EQ-5D responses for application to the French population. A health state utility is a number that quantifies patient HRQoL, where 0 corresponds to dead, 1 corresponds to perfect health, and numbers in between 0 and 1 correspond to intermediate HRQoL. We additionally considered data on demographics, disease characteristics, and treatment details. Results We report final analysis of the 327 EQ-5D responses received from European patients, representing a response rate of 51.7% i.e. the majority of European patients surveyed. The median/mean age was 57.5/56 years. 36.5% of patients were female and 63.5% male. 64.5% of patients had not experienced disease progression while 35.5% were post-progression. 44% of patients reported no additional therapies were being used in addition to TTFields while 56% reported the current use of other therapies. The median/mean time-since-diagnosis was 16/21.4 months and the median/mean time-using-TTFields therapy was 10/14.3 months. The overall median/mean utility estimates were .871/.784. The median/mean utility estimates for patients who had not experienced disease progression were .901/.841, while those for patients who had experienced disease progression were .833/.701. The median/mean utilities for patients under 65 years of age were .872/.778 while for patients at or over 65 they were .855/.791. Univariate regression analyses show that greater than 15 months since diagnosis is correlated with improved utilities (p=.033). These analyses also show that greater than 10 months using TTFields therapy is correlated with improved utilities (p=.005). Multivariate analysis controlling for age, gender, disease progression, other therapies, and time-since-diagnosis still shows that longer time using TTFields is correlated with improved utilities (p=.004). Conclusion The high survey response rate and relatively high utility estimates for both progressed and non-progressed patients as well as patients both over and under 65 years of age indicates that many European patients with GBM using TTFields can expect good QoL. Results indicate that utilities for European patients with GBM are likely to improve with longer time using TTFields therapy.

An examination of machine learning to map non-preference based patient reported outcome measures to health state utility values.

Non‐preference‐based patient‐reported outcome measures (PROMs) are popular in health outcomes research. These measures, however, cannot be used to estimate health state utilities, limiting their usefulness for economic evaluations. Mapping PROMs to a multi‐attribute utility instrument is one solution. While mapping is commonly conducted using econometric techniques, failing to specify the complex interactions between variables may lead to inaccurate prediction of utilities, resulting in inaccurate estimates of cost‐effectiveness and suboptimal funding decisions. These issues can be addressed using machine learning. This paper evaluates the use of machine learning as a mapping tool. We adopt a comprehensive approach to compare six machine learning techniques with eight econometric techniques to map the Patient‐Reported Outcomes Measurement Information System Global Health 10 (PROMIS‐GH10) to the EuroQol five dimensions (EQ‐5D‐5L). Using data collected from 2015 Australians, we find the least absolute shrinkage and selection operator (LASSO) model out‐performed all machine learning techniques and the adjusted limited dependent variable mixture model (ALDVMM) out‐performed all econometric techniques, with the LASSO performing better than ALDVMM. The variable selection feature of LASSO was then used to enhance the performance of the ALDVMM in a hybrid model. Our analysis identifies the potential benefits and challenges of using machine learning techniques for mapping and offers important insights for future research.