Health state utilities associated with attributes of treatments for hepatitis C.
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ABSTRACT: Background
Cost-utility analyses are frequently conducted to compare treatments for hepatitis C, which are often associated with complex regimens and serious adverse events. Thus, the purpose of this study was to estimate the utility associated with treatment administration and adverse events of hepatitis C treatments.Design
Health states were drafted based on literature review and clinician interviews. General population participants in the UK valued the health states in time trade-off (TTO) interviews with 10- and 1-year time horizons. The 14 health states described hepatitis C with variations in treatment regimen and adverse events.Results
A total of 182 participants completed interviews (50% female; mean age = 39.3 years). Utilities for health states describing treatment regimens without injections ranged from 0.80 (1 tablet) to 0.79 (7 tablets). Utilities for health states describing oral plus injectable regimens were 0.77 (7 tablets), 0.75 (12 tablets), and 0.71 (18 tablets). Addition of a weekly injection had a disutility of -0.02. A requirement to take medication with fatty food had a disutility of -0.04. Adverse events were associated with substantial disutilities: mild anemia, -0.12; severe anemia, -0.32; flu-like symptoms, -0.21; mild rash, -0.13; severe rash, -0.48; depression, -0.47. One-year TTO scores were similar to these 10-year values.Conclusions
Adverse events and greater treatment regimen complexity were associated with lower utility scores, suggesting a perceived decrease in quality of life beyond the impact of hepatitis C. The resulting utilities may be used in models estimating and comparing the value of treatments for hepatitis C.
SUBMITTER: Matza LS
PROVIDER: S-EPMC4646927 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:OBJECTIVES:Transfusion-dependent ?-thalassemia (TDT) is a genetic disease that affects production of red blood cells. Conventional treatment involves regular red blood cell transfusions and iron chelation, which has a substantial impact on quality of life. While potentially curative, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is associated with risk of complications, including graft-versus-host disease (GvHD). Gene addition therapy, a novel treatment approach, involves autologous transplantation of the patient's own genetically modified hematopoietic stem cells. The purpose of this study was to estimate utilities associated with treatment approaches for TDT. METHODS:General population respondents in England valued eight health state vignettes (developed with clinician, patient, and parent input) in time trade-off interviews. RESULTS:A total of 207 participants completed interviews (49.8% female; mean age?=?43.2 years). Mean (SD) utilities for the pre-transplant health states were 0.73 (0.25) with oral chelation and 0.63 (0.32) with subcutaneous chelation. Mean utilities for the transplant year were 0.62 (0.35) for gene addition therapy, 0.47 (0.39) for allo-HSCT, and 0.39 (0.39) for allo-HSCT with acute GvHD. Post-transplant utilities were 0.93 (0.15) for transfusion independent, 0.75 (0.25) for 60% transfusion reduction, and 0.51 (0.38) for chronic GvHD. Acute and chronic GvHD were associated with significant disutility (acute?=?-?0.09, p?<?0.0001; chronic?=?-?0.42, p?<?0.0001). CONCLUSIONS:Utilities followed expected patterns, with logical differences between treatment options for TDT and substantially greater utility for transfusion independence than for ongoing treatment involving transfusion and chelation. These utilities may be useful in cost-utility models estimating the value of treatments for TDT.
| S-EPMC7188724 | biostudies-literature
Project description:IntroductionSurgical site infections (SSIs) are among the most common and potentially serious complications after surgery. Staphylococcus aureus is a virulent pathogen frequently identified as a cause of SSI. As vaccines and other infection control measures are developed to reduce SSI risk, cost-utility analyses (CUA) of these interventions are needed to inform resource allocation decisions. A recent systematic review found that available SSI utilities are of "questionable quality." Therefore, the purpose of this study was to estimate the disutility (i.e., utility decrease) associated with SSIs.MethodsIn time trade-off interviews, general population participants in the UK (London, Edinburgh) valued health states drafted based on literature and clinician interviews. Health states described either joint or spine surgery, with or without an SSI. The utility difference between otherwise identical health states with and without the SSI represented the disutility associated with the SSI.ResultsA total of 201 participants completed interviews (50.2% female; mean age = 46.2 years). Mean (SD) utilities of health states describing joint and spine surgery without infections were 0.79 (0.23) and 0.78 (0.23). Disutilities of SSIs ranged from - 0.03 to - 0.32, depending on severity of the infection and subsequent medical interventions. All differences between corresponding health with and without SSIs were statistically significant (all p < 0.001).ConclusionThe preference-based SSI disutilities derived in this study may be used to represent mild and serious SSIs in CUAs assessing and comparing the value of vaccinations that may reduce the risk of SSIs.
| S-EPMC6652168 | biostudies-literature
Project description:IntroductionEarly cancer detection can significantly improve patient outcomes and reduce mortality rates. Novel cancer screening approaches, including multi-cancer early detection tests, have been developed. Cost-utility analyses will be needed to examine their value, and these models require health state utilities. The purpose of this study was to estimate the disutility (i.e., decrease in health state utility) associated with false-positive cancer screening results.MethodsIn composite time trade-off interviews using a 1-year time horizon, UK general population participants valued 10 health state vignettes describing cancer screening with true-negative or false-positive results. Each false-positive vignette described a common diagnostic pathway following a false-positive result suggesting lung, colorectal, breast, or pancreatic cancer. Every pathway ended with a negative result (no cancer detected). The disutility of each false positive was calculated as the difference between the true-negative and each false-positive health state, and because of the 1-year time horizon, each disutility can be interpreted as a quality-adjusted life-year decrement associated with each type of false-positive experience.ResultsA total of 203 participants completed interviews (49.8% male; mean age = 42.0 years). The mean (SD) utility for the health state describing a true-negative result was 0.958 (0.065). Utilities for false-positive health states ranged from 0.847 (0.145) to 0.932 (0.059). Disutilities for false positives ranged from - 0.031 to - 0.111 (- 0.041 to - 0.111 for lung cancer; - 0.079 for colorectal cancer; - 0.031 to - 0.067 for breast cancer; - 0.048 to - 0.088 for pancreatic cancer).ConclusionAll false-positive results were associated with a disutility. Greater disutility was associated with more invasive follow-up diagnostic procedures, longer duration of uncertainty regarding the eventual diagnosis, and perceived severity of the suspected cancer type. Utility values estimated in this study would be useful for economic modeling examining the value of cancer screening procedures.
| S-EPMC10884390 | biostudies-literature
Project description:The aim of the study was to obtain United Kingdom-based societal preferences for distinct stages of metastatic breast cancer (MBC) and six common toxicities. Health states were developed based on literature review, iterative cycles of interviews and a focus group with clinical experts. They described the burden of progressive, responding and stable disease on treatment; and also febrile neutropenia, stomatitis; diarrhoea/vomiting; fatigue; hand-foot syndrome (grade 3/4 toxicities) and hair loss. One hundred members of the general public rated them using standard gamble to determine health state utility. Data were analysed with a mixed model analysis. The study sample was a good match to the general public of England and Wales by demographics and current quality of life. Stable disease on treatment had a utility value of 0.72, with a corresponding gain of +0.07 following a treatment response and a decline by 0.27 for disease progression. Toxicities lead to declines in utility between 0.10 (diarrhoea/vomiting) and 0.15 (febrile neutropenia). This study underlines the value that society place on the avoidance of disease progression and severe side effects in MBC. This may be the largest preference study in breast cancer designed to survey a representative general public sample.
| S-EPMC2360509 | biostudies-other
Project description:BACKGROUND:Preferences for health states for Duchenne muscular dystrophy (DMD) are necessary to assess costs and benefits of novel therapies. Because DMD progression begins in childhood, the impact of DMD on health-related quality-of-life (HRQoL) affects preferences of both DMD patients and their families. The objective of this review was to synthesize published evidence for health state utility from the DMD patient and caregiver perspectives. METHODS:A systematic review was performed using MEDLINE and Embase, according to best practices. Data were extracted from studies reporting DMD patient or caregiver utilities; these included study and patient characteristics, health states considered, and utility estimates. Quality appraisal of studies was performed. RESULTS:From 888 abstracts, eight publications describing five studies were identified. DMD utility estimates were from preference-based measures presented stratified by ambulatory status, ventilation, and age. Patient (or patient-proxy) utility estimates ranged from 0.75 (early ambulatory DMD) to 0.05 (day-and-night ventilation). Caregiver utilities ranged from 0.87 (for caregivers of adults with DMD) to 0.71 (for caregivers of predominantly childhood patients). Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country. Utility estimates for health states within non-ambulatory DMD are under reported; nor were utilities for DMD-related health states such as scoliosis or preserved upper limb function identified. CONCLUSION:Published health state utilities document the substantial HRQoL impacts of DMD, particularly with disease progression. Additional research in patient utilities for additional health states, particularly in non-ambulatory DMD patients, is warranted.
| S-EPMC7028804 | biostudies-literature
Project description:Cost-effectiveness analysis depends on generalizable health-state utilities. Unfortunately, the available utilities for cirrhosis are dated, may not reflect contemporary patients, and do not capture the impact of cirrhosis symptoms. We aimed to determine health-state utilities for cirrhosis, using both the standard gamble (SG) and visual analog scale (VAS). We prospectively enrolled 305 patients. Disease severity (Child-Pugh [Child] class, Model for End-Stage Liver Disease with sodium [MELD-Na] scores), symptom burden (sleep quality, cramps, falls, pruritus), and disability (activities of daily living) were assessed. Multivariable models were constructed to determine independent clinical associations with utility values. The mean age was 57 ± 13 years, 54% were men, 30% had nonalcoholic steatohepatitis, 26% had alcohol-related cirrhosis, 49% were Child class A, and the median MELD-Na score was 12 (interquartile range [IQR], 8-18). VAS displayed a normal distribution with a wider range than SG. The Child-specific SG-derived utilities had a median value of 0.85 (IQR, 0.68-0.98) for Child A, 0.78 (IQR, 0.58-0.93) for Child B, and 0.78 (IQR, 0.58-0.93) for Child C. VAS-derived utilities had a median value of 0.70 (IQR, 0.60-0.85) for Child A, 0.61 (IQR, 0.50-0.75) for Child B, and 0.55 (IQR, 0.40-0.70) for Child C. VAS and SG were weakly correlated (Spearman's rank correlation coefficient, 0.12; 95% confidence interval, 0.006-0.23). In multivariable models, disability, muscle cramps, and MELD-Na were significantly associated with SG utilities. More clinical covariates were significantly associated with the VAS utilities, including poor sleep, MELD-Na, disability, falls, cramps, and ascites. Conclusion: We provide health-state utilities for contemporary patients with cirrhosis as well as estimates of the independent impact of specific symptoms on each patient's reported utility.
| S-EPMC7262287 | biostudies-literature
Project description:BackgroundTuberous sclerosis complex (TSC) is a rare multisystem disorder often associated with treatment-resistant epilepsy. Cost-effectiveness analysis for new antiseizure medications typically requires health state utilities (HSUs) that reflect the burden of a given condition.ObjectiveThis study aimed to estimate HSUs, with a focus on valuing the impact of seizure type and seizure frequency on health-related quality of life (HRQL) for patients with TSC and their caregivers.MethodsA targeted literature review and qualitative research with healthcare professionals and caregivers informed the development of health state vignettes describing the experience of living with TSC or caring for a child with TSC. Vignettes were evaluated in interviews with the UK general population using the time trade-off (TTO) method.ResultsSixteen vignettes were developed describing patient HRQL (n = 8) and caregiver HRQL (n = 8). Two hundred interviews were conducted via online video calls due to COVID-19 pandemic restrictions. Two hundred participants evaluated the patient (n = 100) and caregiver (n = 100) health state vignettes. Estimated utility scores varied consistently according to seizure type and seizure frequency. Patient TTO utility scores ranged between -0.234 (highest seizure frequency and multiple seizure types) and 0.725 (seizure-free state). Caregiver TTO utility scores ranged from 0.221 to 0.905.ConclusionsFindings highlight the substantial burden of living with TSC and caring for a child with TSC. Patient and caregiver burden was greater for generalised versus focal seizures. The burden was greatest for a combination of both seizure types and worsened with increasing seizure frequency.
| S-EPMC8441242 | biostudies-literature
Project description:INTRODUCTION:Standard concentration (100 units/mL) mealtime insulin is frequently used to treat patients with type 1 (T1D) and type 2 diabetes (T2D). A more concentrated version of the medication (200 units/mL) has been available in Italy since 2016. This concentrated version is bioequivalent to the standard version and delivers the same amount of medication but in half the volume of liquid. The purpose of this study was to examine patient preferences and estimate health state utilities associated with standard and concentrated rapid-acting mealtime analog insulin. METHODS:Participants with T1D and T2D in Italy valued two health states in time trade-off interviews. The descriptions of diabetes and treatment in the two health states were identical, differing only in terms of insulin concentration (e.g., half as much liquid for the same dose, less effort needed to press the injection button, and fewer injection pens required with concentrated insulin). To ensure participants understood the health states, they were shown a short video illustrating the differences between concentrations. RESULTS:A total of 217 participants completed the interviews (49.8% male; mean age 56.1 years; 109 from Milan; 108 from Rome; 12.0% T1D; 88.0% T2D). When asked which health state they preferred, 98.2% responded the concentrated version, 0.9% said the standard version, and 0.9% had no preference. Mean [standard deviation (SD)] utilities rounded to three decimals were 0.892 (0.099) for the concentrated version and 0.884 (0.101) for the standard version. The mean (SD; p value) utility difference between the standard and concentrated rapid-acting insulin was 0.007 (0.019; p < 0.0001). CONCLUSIONS:Findings from this study provide insight into patient preferences associated with concentration of rapid-acting insulin. Although the difference in utility is small, patients consistently preferred the concentrated formulation over the standard insulin, and for some patients this difference had an impact on utility valuations. These results suggest that the concentration of rapid-acting insulin should be considered because it could affect treatment preference and quality of life. FUNDING:Eli Lilly and Company.
| S-EPMC6965595 | biostudies-literature
Project description:Background and purposeAn internet-based discrete choice experiment (DCE) was conducted to elicit preferences for a wide range of Dupuytren's contracture (DC)-related health states. An algorithm was subsequently developed to convert these preferences into health state utilities that can be used to assess DC's impact on quality of life and the value of its treatments.MethodsHealth state preferences for varying levels of DC hand severity were elicited via an internet survey from a sample of the UK adult population. Severity levels were defined using a combination of contractures (0, 45, or 90 degrees) in 8 proximal interphalangeal and metacarpophalangeal joints of the index, middle, ring, and little fingers. Right-handed, left-handed, and ambidextrous respondents indicated which hand was preferable in each of the 10 randomly-selected hand-pairings comparing different DC severity levels. For consistency across comparisons, anatomically precise digital hand drawings were used. To anchor preferences onto the traditional 0-1 utility scale used in health economic evaluations, unaffected hands were assigned a utility of 1.0 whereas the utility for a maximally affected hand (i.e., all 8 joints set at 90 degrees of contracture) was derived by asking respondents to indicate what combination of attributes and levels of the EQ-5D-5L profile most accurately reflects the impact of living with such hand. Conditional logistic models were used to estimate indirect utilities, then rescaled to the anchor points on the EQ-5D-5L.ResultsEstimated utilities based on the responses of 1,745 qualified respondents were 0.49, 0.57, and 0.63 for completely affected dominant hands, non-dominant hands, or ambidextrous hands, respectively. Utility for a dominant hand with 90-degree contracture in t h e metacarpophalangeal joints of the ring and little fingers was estimated to be 0.89. Separately, reducing the contracture of metacarpophalangeal joint for a little finger from 50 to 12 degrees would improve utility by 0.02.InterpretationDC is associated with substantial utility decrements. The algorithms presented herein provide a robust and flexible framework to assess utility for varying degrees of DC severity.
| S-EPMC3851672 | biostudies-literature
Project description:Background and objectivesNew treatments and interventions are in development to address clinical needs in heart failure. To support decision making on reimbursement, cost-effectiveness analyses are frequently required. A systematic literature review was conducted to identify and summarize heart failure utility values for use in economic evaluations.MethodsDatabases were searched for articles published until June 2019 that reported health utility values for patients with heart failure. Publications were reviewed with specific attention to study design; reported values were categorized according to the health states, 'chronic heart failure', 'hospitalized', and 'other acute heart failure'. Interquartile limits (25th percentile 'Q1', 75th percentile 'Q3') were calculated for health states and heart failure subgroups where there were sufficient data.ResultsThe systematic literature review identified 161 publications based on data from 142 studies. Utility values for chronic heart failure were reported by 128 publications; 39 publications published values for hospitalized and three for other acute heart failure. There was substantial heterogeneity in the specifics of the study populations, methods of elicitation, and summary statistics, which is reflected in the wide range of utility values reported. EQ-5D was the most used instrument; the interquartile limit for mean EQ-5D values for chronic heart failure was 0.64-0.72.ConclusionsThere is a wealth of published utility values for heart failure to support economic evaluations. Data are heterogenous owing to specificities of the study population and methodology of utility value elicitation and analysis. Choice of value(s) to support economic models must be carefully justified to ensure a robust economic analysis.
| S-EPMC7867520 | biostudies-literature