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Introduction: Utility values offer a quantitative means to evaluate the impact of novel cancer treatments on patients' quality of life (QoL). However, the multiple methods available for valuing QoL present challenges in selecting the most appropriate method across different contexts. Areas covered: This review provides cancer clinicians and researchers with an overview of methods to value QoL for economic evaluations, including standalone and derived preference-based measures (PBMs) and direct preference elicitation methods. Recent developments are described, including the comparative performance of cancer-specific PBMs versus generic PBMs, measurement of outcomes beyond health-related QoL, and increased use of discrete choice experiments to elicit preferences. Recommendations and considerations are provided to guide the choice of method for cancer research. Expert opinion: We foresee continued adoption of the QLU-C10D and FACT-8D in cancer clinical trials given the extensive use of the EORTC QLQ-C30 and FACT-G in cancer research. While these cancer-specific PBMs offer the convenience of eliciting utility values without needing a standalone PBM, researchers should consider potential limitations if they intend to substitute them for generic PBMs. As the field advances, there is a greater need for consensus on the approach to selection and integration of various methods in cancer clinical trials. [ABSTRACT FROM AUTHOR]

Background: Understanding health state utilities of skin diseases is essential for health economic evaluations in an era of rising health care costs. Objective: To create a catalog of utility values of chronic skin diseases among Asians. Methods: This is a cross-sectional study of adults attending a dermatology outpatient clinic from February 2019 to March 2023 with one of the following skin diseases: (1) eczema, (2) psoriasis, (3) acne vulgaris, (4) chronic urticaria, (5) pigmentary disorders, (6) hair loss, (7) viral warts, (8) fungal infections, and (9) keloids. Demographic and socioeconomic information was collected. Health status and utilities (Dermatology Life Quality Index, Skindex-16, EuroQol-5 Dimension, visual analog scale, time-trade-off, and willingness-to-pay) were measured. Results: A total of 183 patients with a median age of 35.5 (21-77 years) years were included. Majority (76.9%) were Chinese, followed by Malays (11.5%). The time-trade-off utilities were lower than EuroQol-5 Dimension utilities across all disease severity and most skin diseases. Patients were willing to pay $740 USD or more for a hypothetical drug to cure conditions such as psoriasis, acne, hair loss, and keloids. Limitations: Limited sample size and ethnic representation. Conclusions: This study provides a catalog of utilities in skin diseases and highlights the strengths and challenges of different measures.

Background The VOYAGER PAD (Efficacy and Safety of Rivaroxaban in Reducing the Risk of Major Thrombotic Vascular Events in Subjects With Symptomatic Peripheral Artery Disease Undergoing Peripheral Revascularization Procedures of the Lower Extremities) trial compared rivaroxaban (2.5 mg twice a day) plus aspirin with aspirin alone in patients with symptomatic peripheral artery disease requiring endovascular or surgical limb revascularization, with 50% receiving clopidogrel background therapy. The New Drug Indication application includes benefit–risk assessments using clinical judgment to balance benefits against risks. During its review, the US Food and Drug Administration requested additional quantitative benefit–risk analyses with formal weighting approaches. Methods and Results Benefits and risks were assessed using rate differences between treatment groups (unweighted analysis). To account for clinical importance of the end points, a multi‐criteria decision analysis was conducted using health state utility values as weights. Monte Carlo simulations incorporated statistical uncertainties of the event rates and utility weights. Intent‐to‐treat and on‐treatment analyses were conducted. For unweighted intent‐to‐treat analyses, rivaroxaban plus aspirin would result in 120 (95% CI, −208 to −32) fewer events of the primary composite end point (per 10 000 patient‐years) compared with aspirin alone. Rivaroxaban caused an excess of 40 (95% CI, 8–72) Thrombolysis in Myocardial Infarction major bleeding events, which was largely driven by nonfatal, nonintracranial hemorrhage Thrombolysis in Myocardial Infarction major bleeding events. For weighted analyses, rivaroxaban resulted in the utility equivalent of 13.7 (95% CI, −85.3 to 52.6) and 68.1 (95% CI, 7.9–135.7) fewer deaths per 10 000 patient‐years (intent‐to‐treat and on‐treatment, respectively), corresponding to probabilities of 64.4% and 98.7%, respectively, that benefits outweigh risks favoring rivaroxaban per Monte Carlo simulation. Conclusions These analyses show a favorable benefit–risk profile of rivaroxaban therapy in the VOYAGER PAD trial, with findings generally consistent between the unweighted and weighted approaches.

Abstract Objective Rare genetic diseases of obesity typically present with hyperphagia, a pathologic desire to consume food. Cost‐utility models assessing the value of treatments for these rare diseases will require health state utilities representing hyperphagia. This study estimated utilities associated with various hyperphagia severity levels. Methods Four health state vignettes were developed using published literature and clinician input to represent various severity levels of hyperphagia. Utilities were estimated for these health states in a time trade‐off elicitation study in a UK general population sample. Results In total, 215 participants completed interviews (39.5% male; mean age 39.1 years). Mean (SD) utilities were 0.98 (0.02) for no hyperphagia, 0.91 (0.10) for mild hyperphagia, 0.70 (0.30) for moderate hyperphagia, and 0.22 (0.59) for severe hyperphagia. Mean (SD) disutilities were −0.08 (0.10) for mild, −0.28 (0.30) for moderate, and −0.77 (0.58) for severe hyperphagia. Conclusions These data show increasing severity of hyperphagia is associated with decreased utility. Utilities associated with severe hyperphagia are similar to those of other health conditions severely impacting quality of life (QoL). These findings highlight that treatments addressing substantial QoL impacts of severe hyperphagia are needed. Utilities estimated here may be useful in cost‐utility models of treatments for rare genetic diseases of obesity.

Purpose: Most studies regarding the association of obesity with health-related quality of life (HRQoL) have assessed obesity at only one or two time points. We aimed to examine the associations of life course body mass index (BMI) from childhood with health-related quality of life (HRQoL) in mid-adulthood. Methods: Data were from a cohort study of Australian children (n = 2254, mean baseline age 12.0 (2.0) years in 1985, 46.8% male). Weight and height were measured at baseline and measured or self-reported on average 20, 25, and 30 years later. Age and sex-standardised BMI-z score was calculated at each time point. Physical and mental HRQoL and health state utilities (HSUs) were measured by SF-12 and SF-6D at the last adult follow-up. Linear regression was used to examine the associations adjusting for age, sex, and childhood health status. Results: Higher BMI-z score in childhood (βadjusted − 1.39, 95% CI − 1.73 to − 1.05) and increasing BMI-z score from childhood to young adulthood (βadjusted − 1.82, 95% CI − 2.17 to − 1.46) and from young to mid-adulthood (βadjusted − 1.77, 95% CI − 2.28 to − 1.26) were associated with lower physical HRQoL in mid-adulthood. Similar results were found for mid-adulthood HSUs (βadjusted ranged − 0.006 to − 0.014, all P < 0.05). Only increasing BMI-z score from young to mid-adulthood significantly related to poorer mental HRQoL (βadjusted − 0.74, 95% CI − 1.29 to − 0.19) in mid-adulthood. Conclusion: High BMI from childhood to mid-adulthood had only modest associations with HRQoL and HSUs, with effects on physical HRQoL most apparent. [ABSTRACT FROM AUTHOR]

The VISION trial showed durable activity of tepotinib in MET exon 14 (MET ex14) skipping non-small cell lung cancer. We analyzed health state utilities using patient-reported outcomes from VISION. 5-level version of EQ-5D (EQ-5D-5L) and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 responses were collected at baseline, every 6 to 12 weeks during treatment, and at the end of treatment and safety follow-up. EQ-5D-5L and European Organisation for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (QLU-C10D) utilities were derived using United States, Canada, United Kingdom, and Taiwan value sets, where available. Utilities were analyzed with linear mixed models including covariates for progression or time-to-death (TTD). Utilities were derived for 273/291 patients (EQ-5D-5L, 1545 observations; QLU-C10D, 1546 observations). Mean (± SD) US EQ-5D-5L utilities increased after tepotinib initiation, from 0.687 ± 0.287 at baseline to 0.754 ± 0.250 before independently assessed progression, and decreased post progression (0.704 ± 0.288). US QLU-C10D utilities showed similar trends (0.705 ± 0.215, 0.753 ± 0.195, and 0.708 ± 0.209, respectively). Progression-based models demonstrated a statistically significant impact of progression on utilities and predicted higher utilities pre versus post progression. TTD-based models showed statistically significant associations of TTD with utilities and predicted declining utilities as TTD decreased. Prior treatment (yes/no) did not significantly predict utilities in progression- or TTD-based models. Utilities for Canada, United Kingdom, and Taiwan showed comparable trends. In this first analysis of health state utilities in patients with MET ex14 skipping non-small cell lung cancer, who received tepotinib, utilities were significantly associated with progression and TTD, but not prior treatment. • Tepotinib is approved for treatment of advanced/metastatic MET exon 14 (MET ex14) skipping non-small cell lung cancer. Data on health state utilities in this population are lacking. • 5-level version of EQ-5D and European Organisation for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions utilities, derived from VISION trial patient-reported outcomes, increased from baseline during tepotinib treatment until progression. Utilities were significantly predicted by progression and time-to-death. • These analyses provide the first health state utility estimates for patients with MET ex14 skipping non-small cell lung cancer, which will populate cost-effectiveness models for tepotinib as a new treatment for these patients. [ABSTRACT FROM AUTHOR]

Objective: Rare genetic diseases of obesity typically present with hyperphagia, a pathologic desire to consume food. Cost‐utility models assessing the value of treatments for these rare diseases will require health state utilities representing hyperphagia. This study estimated utilities associated with various hyperphagia severity levels. Methods: Four health state vignettes were developed using published literature and clinician input to represent various severity levels of hyperphagia. Utilities were estimated for these health states in a time trade‐off elicitation study in a UK general population sample. Results: In total, 215 participants completed interviews (39.5% male; mean age 39.1 years). Mean (SD) utilities were 0.98 (0.02) for no hyperphagia, 0.91 (0.10) for mild hyperphagia, 0.70 (0.30) for moderate hyperphagia, and 0.22 (0.59) for severe hyperphagia. Mean (SD) disutilities were −0.08 (0.10) for mild, −0.28 (0.30) for moderate, and −0.77 (0.58) for severe hyperphagia. Conclusions: These data show increasing severity of hyperphagia is associated with decreased utility. Utilities associated with severe hyperphagia are similar to those of other health conditions severely impacting quality of life (QoL). These findings highlight that treatments addressing substantial QoL impacts of severe hyperphagia are needed. Utilities estimated here may be useful in cost‐utility models of treatments for rare genetic diseases of obesity. [ABSTRACT FROM AUTHOR]

This study aimed to quantify the association between the loss of health state utilities (LHSU) and obesity, considering different obesity categories. This is relevant to interventions economic evaluations and for public policy decision planning. Using data from the Chilean National Health Survey, this study uses linear regression models and counterfactual scenarios to calculate the prevalent burden, population averages, and total sum of LHSU attributable to obesity for the Chilean national level on people older than 15 years, year 2017. Adjustments for socioeconomic status and associated noncommunicable diseases (NCDs) are considered. Calculating the LHSU using these methods enables the approximation of loss of prevalent quality-adjusted life-years (QALYs). The raw obesity LHSU burden was 9.1% (95% uncertainty interval [UI] 5.1-13). When adjustment is considered, the LHSU attributable to obesity reaches 4.6% (95% UI 0.6-8.5) being responsible for 121 045 prevalent QALYs. Socioeconomic status adjusted analysis of higher body mass index (BMI, in kg/m2) categories of obesity shows a dose-response effect for LHSU, being the BMI ≥ 40 category with the highest population average of attributable LHSU (10.1; 95% UI 5.5-14.5, scale 0 [full health] to 100 [dead]). Burden for BMI ≥ 35 categories showed the biggest change after NCD adjustment. Obesity carries a significant burden of QALY loss. Policy decision-making addressing obesity should focus specially on the BMI ≥ 40 group. NCD comorbidity should be considered for policies addressing the BMI ≥ 35 group. • Studies show that the losses of health state utilities (LHSUs) associated to obesity are in the range 0.6 to 5.0 (anchored from 0 to 100; health to dead, respectively). • Adjusted, the burden of LHSU attributable to obesity is 4.6% (95% uncertainty interval 0.6-8.5). • The LHSU burden and the total prevalent quality-adjusted life-year losses form a "U" shape, where body mass index ≥ 40 and 30 to 35 kg/m2 categories show higher values. • These results support considering different strategies for tackling the obesity public health problem. • The methods used here can contribute to find new ways to make fruitful work with public cross-sectional information at national level. [ABSTRACT FROM AUTHOR]

Objectives. Health utility estimates from the current era of HIV treatment, critical for cost-effectiveness analyses (CEA) informing HIV health policy, are limited. We examined peer-reviewed literature to assess the appropriateness of commonly referenced utilities, present previously unreported quality-of-life data from two studies, and discuss future implications for HIV-related CEA. Methods. We searched a database of cost-effectiveness analyses specific to HIV prevention efforts from 1999 to 2016 to identify the most commonly referenced sources for health utilities and to examine practices around using and reporting health utility data. Additionally, we present new utility estimates from the Centers of Disease Control and Preventions Medical Monitoring Project (MMP) and the INSIGHT Strategies for Management of Anti-Retroviral Therapy (SMART) trial. We compare data collection time frames, sample characteristics, assessment methods, and key estimates. Results. Data collection for the most frequently cited utility estimates ranged from 1985 to 1997, predating modern HIV treatment. Reporting practices around utility weights are poor and lack details on participant characteristics, which may be important stratifying factors for CEA. More recent utility estimates derived from MMP and SMART were similar across CD4+ count strata and had a narrower range than pre-antiretroviral therapy (ART) utilities. Conclusions. Despite the widespread use of ART, cost-effectiveness analysis of HIV prevention interventions frequently apply pre-ART health utility weights. Use of utility weights reflecting the current state of the US epidemic are needed to best inform HIV research and public policy decisions. Improved practices around the selection, application, and reporting of health utility data used in HIV prevention CEA are needed to improve transparency.

This study estimated the "process utilities" of treatment options for patients with relapsed/refractory multiple myeloma (RRMM) in Japan using the time trade-off (TTO) method. Chimeric antigen receptor (CAR) T cell immunotherapy is available for patients with RRMM who are triple-class exposed (TCE) after treatment with immunomodulatory agents, proteasome inhibitors, and anti-CD38 monoclonal antibodies. However, the impact of available treatment options on health state utilities has not been well characterized, particularly in relation to process utilities. Eight vignettes of health states and daily activity restrictions related to each of the following RRMM therapies were prepared: no treatment, CAR T cell therapy with idecabtagene vicleucel (ide-cel), regular intravenous infusion, and oral administration. A face-to-face survey of healthy Japanese adults who were representative of the general population was conducted. The TTO method was used to evaluate each vignette and to generate utility scores for each treatment regimen. Three hundred and nineteen respondents participated in the survey (mean age: 44 years [range: 20–64]; female: 50%). Utility scores for no treatment, ide-cel, oral pomalidomide, and dexamethasone (Pd) therapy ranged from ∼0.7 to 0.8. Utility scores for regular intravenous infusion regimens ranged from 0.50 to 0.56. There was a difference of ∼0.2 between the utility scores for no treatment/ide-cel/oral administration and regular intravenous infusions. Differences in treatment administration across RRMM therapies showed a substantial impact on health state utilities. When quantifying the value of treatments, process utility gains should be considered as an independent factor in health technology assessments. [ABSTRACT FROM AUTHOR]

Katy Gallop,1 Sarah Acaster,1 Jane de Vries,2 Richard Browne,2 Robert Ryan,2 Sarah Baker,3 George Du Toit4 1Acaster Lloyd Consulting Ltd, London, UK; 2Aimmune Therapeutics, London, UK; 3Anaphylaxis Campaign, Farnborough, UK; 4Department of Paediatric Allergy, Division of Asthma, Allergy and Lung Biology, MRC & Asthma UK Centre in Allergic Mechanisms of Asthma, King’s College London, Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust, London, UKCorrespondence: Katy Gallop, Acaster Lloyd Consulting, 84 Theobalds Road, London, WC1X 8NL, UK, Tel +44 203 9781686, Email katy.gallop@acasterlloyd.com Robert Ryan, Aimmune Therapeutics, 10 Eastbourne Terrace, London, W2 6LG, UK, Email rryan@aimmune.comPurpose: This study aimed to estimate utility values for health states relating to oral immunotherapy (OIT) for peanut allergy (PA), for children with PA and their caregivers.Patients and Methods: Two methods were used: an online survey and structured interviews. Both methods assessed current utility/untreated PA (health-related quality of life, HRQoL) and HRQoL in different health states: “up-dosing phase of treatment”, “maintenance phase” and “able to tolerate 6– 8 peanuts if accidentally ingested”. The survey was conducted in individuals with and without experience of OIT; data collected included the EQ-5D-Y (child states) and EQ-5D-5L (caregiver states).Results: In total, 100 caregivers and 38 adolescents completed the treatment-naïve survey, a separate sample of 50 caregivers participated in structured interviews. Seven caregivers and two adolescents with experience of OIT for PA completed the survey. Data from the three samples were pooled, the mean utility values were untreated PA: 0.796 (child), 0.855 (caregiver); up-dosing: 0.711 (child), 0.806 (caregiver); maintenance: 0.821 (child), 0.849 (caregiver), tolerate 6– 8 peanuts: 0.859 (child), 0.884 (caregiver). The results show a gain in utility of 0.063 for children and 0.029 for caregivers between the untreated and tolerate 6– 8 peanuts health states.Conclusion: This study is the first to assess utilities relating to OIT for PA. The results show the potential benefit of OIT for individuals with PA and their caregivers and provide values for use in cost-effectiveness evaluation.Keywords: peanut allergy, oral immunotherapy, health-related quality of life, health state utilities, caregiver

Adam B Smith,1 Andria Hanbury,1 Jennifer A Whitty,2 Igor Beitia Ortiz de Zarate,3 Florence Hammes,3 Gérard de Pouvourville,4 Katharina Buesch5 1York Health Economics Consortium, University of York, York, UK; 2Norwich Medical School, University of East Anglia, Norwich, UK; 3PTC Therapeutics, Paris, France; 4Department of Economics, ESSEC, Paris, France; 5PTC Therapeutics, Zug, 6300, SwitzerlandCorrespondence: Adam B SmithYork Health Economics Consortium, Enterprise House, Innovation Way, University of York, York, YO10 5NQ, UKTel +44 1904 323620Fax +44 1904 323628Email adam.smith@York.ac.ukPurpose: Cost-effectiveness evaluations of interventions require health utility data. However, in medical conditions, such as aromatic L-amino acid decarboxylase (AADC) deficiency, this presents problems due to the rarity of the disease. The study aim therefore was to employ a discrete choice experiment (DCE) to generate health utilities for AADC deficiency.Methods: A previous literature review, clinician and parent interviews had identified six key AADC deficiency attributes: mobility, muscle weakness, oculogyric crises (OCG), feeding ability, cognitive impairment and screaming. A representative sample of the French general population was recruited. Participants rated 5 health state vignettes describing AADC deficiency using time-trade-off (TTO) and standard gamble (SG). Additionally, participants rated the worst health state using the Health Utility Index version 3 (HUI3). Subsequently, participants completed DCE 11 choice sets. Indirect DCE part-worth utilities were converted to health utilities using the anchors from the TTO, SG and HUI3.Results: The DCE was completed online by 1001 participants (50.9% female; mean age 45.7 years). Most participants (596, 59.5%) provided consistent responses to the repeated choice task. Five models were evaluated, and one preference reversal (“head control”/“sitting unaided”) was identified in all models. The rescaled utilities ranged from 0.3891 to 0.5577 (difference of 0.17 utilities) for TTO anchors corresponding to the worst (633233) and best (111111) health states. Health utilities ranged from 0.5534 to 0.7093 for the SG anchors. The disutility associated with a transition from “no problems walking” to “bedridden” was − 0.0533, whereas disutility of moving from “constant screaming” relative to “no screaming” was − 0.0248. The disutility associated with daily OCG was − 0.0167. Disutilities for the other attributes were small although there were exceptions.Conclusion: A DCE was used to derive health utilities for AADC deficiency. These health utilities will subsequently be used in an economic model evaluating an AADC deficiency intervention.Keywords: aromatic L-amino acid decarboxylase deficiency, discrete choice experiment, health state utilities, health utilities

Adam B Smith,1 Andria Hanbury,1 Jennifer A Whitty,2 Katharina Buesch3 1York Health Economics Consortium, University of York, York, UK; 2Norwich Medical School, University of East Anglia, Norwich, UK; 3PTC Therapeutics, Zug, 6300, SwitzerlandCorrespondence: Adam B SmithYork Health Economics Consortium, Enterprise House, Innovation Way, University of York, York, YO10 5NQ, UKTel +44 1904 323620Fax +44 1904 323628Email adam.smith@york.ac.ukPurpose: Deriving health utilities for rare medical conditions such as aromatic L-amino acid decarboxylase (AADC) deficiency poses challenges. The rarity of AADC deficiency and the fact that this genetic condition often presents in very young children means that robust utility values cannot be derived from the child or their parent/caregiver. Alternative approaches, eg, discrete choice experiments (DCE), are required in order to provide health utilities. The aim of the study was to generate health utilities for AADC deficiency using a DCE.Methods: The DCE was completed online by panel participants from a UK representative sample. The DCE comprised 6 AADC deficiency attributes (2– 6 levels): mobility, muscle weakness, oculogyric crises, feeding ability, cognitive impairment and screaming. These were identified from published literature, clinician input, parent interviews and expert opinion. Participants were presented with 10 choice sets specified using an orthogonal design, including a repeat task to evaluate choice consistency. Participants were presented with 5 health state vignettes prior to the DCE. These were used to elicit time trade-off (TTO) utilities. Multinomial logit models were estimated for the DCE data. The TTO utilities for the worst/best health states were used as anchors to convert indirect DCE part-worth utilities to health utilities.Results: A total of 1596 participants completed the DCE. The majority (70.7%) gave consistent responses to the repeated choice task; only 1.7% (27) always chose the same alternative for every choice set. Five models were evaluated. There was one preference reversal (“sitting unaided”/“standing with assistance”) occurring in all models; these two mobility level coefficients were set to be equal in the final model. Rescaled utilities ranged from 0.494 to 0.7279, corresponding to the worst (633233) and best (111111) health states.Conclusion: Health utilities were derived for AADC deficiency through a DCE. These will be used for a cost-effectiveness model of an AADC deficiency treatment.Keywords: AADC deficiency, discrete choice experiment, health state utilities, health utilities

Abstract Background Reference values are a helpful tool to facilitate comparisons of sampled values against a specified reference population. The aim is to describe the health profile and to provide visual analogue scale (EQ VAS) and utility reference values for the EQ-5D-5L from a normative sample of the general elderly population (65+) in Germany. Methods We analysed a sub-set of data from the German EQ-5D-5L valuation study using self-reported information based on EQ-5D-5L. We examined the share of respondents in each severity level per dimension as well as means, standard deviations (SD) and 95% confidence intervals for the index and EQ VAS values stratified by age groups and gender. Age was categorised in four groups (65–69, 70–74, 75–79 and > 79 years) to facilitate a more detailed examination of age-related health-related quality of life (HRQoL). Results The average index and EQ VAS scores were 0.84 (SD 0.22) and 73.2 (SD 18.5), respectively. In total, 21.4% reported no problems in all dimensions. With higher age, health problems were reported more frequently, which, in turn, lead to monotonically decreasing index and EQ VAS values. Overall, men reported fewer problems than women and this difference was largest beyond the age of 80. Conclusion HRQoL in the oldest old appears to be less stable and differs from the young elderly. However, the conventional age categorisation of earlier population norms studies seems to mask these differences. Hence, the more detailed provision of EQ-5D-5L reference values for the elderly population seems helpful for future German studies.

Abstract Background Hemophagocyti.c lymphohistiocytosis (HLH) is a rare and severe disorder characterized by abnormal activation of the immune system. Primary HLH causes prolonged fever, spleen and liver enlargement, and organ dysfunction, usually in infancy and early childhood and is fatal if left untreated. As effective treatment options emerge, such as emapalumab-lzsg, Health Technology Assessment bodies around the world will assess them in terms of cost-effectiveness. This study was designed to estimate quality of life weights (utilities) for such analyses. Methods Vignettes were developed describing HLH treatment related health states. Health states included active HLH, HLH plus neurological symptoms, receiving chemotherapy, undergoing stem cell transplant (SCT), graft versus host disease (GVHD), cure and end of life care. The vignettes were based on information from in depth interviews with clinical specialists; and qualitative research with four parents of children with primary HLH aged between 1 and 18 years old. The vignettes were then assessed in time trade off (TTO) interviews with members of the UK general public in one on one face to face interviews with trained, experienced interviewers. Preference data were analysed using the generalised estimating equations framework. Results Detailed qualitative data captured the substantial burden of this disease for young children. One hundred participants completed the TTO interviews. The utility score for Active HLH was estimated as 0.32 (95% CI, 0.24 to 0.39). Values for other states were HLH plus neurological symptoms (0.27, 95%CI 0.18–0.35), receiving chemotherapy (0.26, 95%CI 0.17–0.34), undergoing SCT (0.18, 95%CI 0.07–0.28), GVHD (0.07, 95%CI -0.04-0.17), cure (0.72, 95%CI 0.67–0.77) and end of life care (− 0.17, 95%CI -0.27- -0.07). Conclusions This study has estimated utility weights for seven different HLH related states which are based on detailed input from carers and physicians and have good face validity. There are few other options for collecting these data in an ultra-rare setting.

Purpose: Streptococcus pneumoniae infections remain a significant source of morbidity and mortality worldwide. The purpose of this review was to summarize the impact of pneumococcal disease on health state utilities (HSU) in the acute phase of illness.Methods: We searched MEDLINE, EMBASE, EconLit, the Health Technology Assessment Database, the National Health Economic Evaluation Database, and Tufts Cost-Effectiveness Registry (up to January 2020) for primary studies. Eligible studies elicited HSU estimates using preference-based instruments for the acute phase of infection of pneumococcal syndromes including acute otitis media, pneumonia/lower respiratory tract infections, bacteremia/sepsis, and meningitis. Two reviewers independently conducted screening, data extraction and quality appraisal.Results: We screened 10,178 studies, of which 26 met our inclusion criteria. Cohort sizes ranged from 8 to 2060 respondents. The most frequently studied syndrome was pneumonia (n = 17), followed by acute otitis media (n = 9), meningitis (n = 7) and bacteremia/sepsis (n = 4). Overall, each syndrome was associated with a substantial impact on HSU. Bacteremia/sepsis (range: - 0.331 to 0.992) and meningitis (range: - 0.330 to 0.977) were generally associated with the lowest HSU, followed by pneumonia (range: - 0.054 to 0.998) and acute otitis media (range: 0.064 to 0.970). HSU estimates varied considerably by treatment setting, elicitation method and type of respondent. The only study to compare pneumococcal infections to non-pneumococcal infections in the same population revealed significantly lower HSU estimates among pneumococcal infections.Conclusions: Pneumococcal syndromes are associated with decreased HSU estimates. Given the considerable heterogeneity in methods and source populations as well as study quality, care should be taken to select the most appropriate estimates. [ABSTRACT FROM AUTHOR]

Health state utilities associated with weight change are needed as inputs for cost-utility analyses (CUAs) examining the value of treatments for obesity and type 2 diabetes (T2D). Although some pharmaceutical treatments currently in development are associated with substantial weight loss, little is known about the utility impact of weight decreases greater than 10%. The purpose of this study was to estimate utilities associated with body weight decreases up to 20% based on preferences of individuals with obesity, with and without T2D. Health state vignettes were developed to represent respondents' own current weight and weight decreases of 2.5, 5, 10, 15, and 20%. Health state utilities were elicited in time trade-off interviews in two UK locations (Edinburgh and London) with a sample of participants with obesity, with and without T2D. Mean utility increases associated with each amount of weight decrease were calculated. Regression analyses were performed to derive a method for estimating utility change associated with weight decreases. Analyses were conducted with data from 405 individuals with obesity (202 with T2D, 203 without T2D). Utility increases associated with various levels of weight decrease ranged from 0.011 to 0.060 in the subgroup with T2D and 0.015 to 0.077 in the subgroup without T2D. All regression models found that the percentage of weight decrease was a highly significant predictor of change in utility (p <.0001). The relationship between weight change and utility change did not appear to be linear. Equations are recommended for estimating utility change based on the natural logarithm of percentage of weight decrease. Results of this study may be used to provide inputs for CUAs examining and comparing the value of treatments that are associated with substantial amounts of weight loss in patients with obesity, with or without T2D. [ABSTRACT FROM AUTHOR]

Abstract Objective Identify the most recent utility value estimates for cardiovascular disease (CVD) via systematic literature review (SLR) and explore trends in utility elicitation methods in the last 6 years. Methods This SLR was updated on January 25, 2018, and identified studies reporting utilities for myocardial infarction (MI), stroke, angina, peripheral artery disease (PAD), and any-cause revascularization by searching Embase, PubMed, Health Technology Assessment Database, and grey literature. Results A total of 375 studies reported CVD utilities (pre-2013 vs post-2013: MI, 38 vs 32; stroke, 86 vs 113; stable angina, 8 vs 9; undefined/unstable angina, 23 vs 8; PAD, 29 vs 13; revascularization, 54 vs 40). Median average utilities for MI, stroke, and revascularization increased over time (pre-2013 vs post-2013: MI, 0.71 vs 0.79; stroke, 0.63 vs 0.64; revascularization, 0.76 vs 0.81); angina and PAD showed a decrease in median values over time (stable angina, 0.83 vs 0.72; undefined/unstable angina, 0.70 vs 0.69; PAD, 0.76 vs 0.71). The proportion of utility estimates from trials increased across health states (pre-2013 vs post-2013: 22.5% vs 37.2%), as did the proportion of trials using the EuroQol Five Dimensions Questionnaire (EQ-5D; pre-2013 vs post-2013: 73.8% vs 91.4%). Use of methods such as the standard gamble, time trade-off, and Health Utilities Index has declined. Conclusions Health state utilities for cardiovascular health states have changed in the last 6 years, likely due to changes in the types of studies conducted, the patient populations evaluated, and possibly changing utility elicitation methods. The EQ-5D has been used more frequently.

Background: Understanding health state utilities of skin diseases is essential for health economic evaluations in an era of rising health care costs., Objective: To create a catalog of utility values of chronic skin diseases among Asians., Methods: This is a cross-sectional study of adults attending a dermatology outpatient clinic from February 2019 to March 2023 with one of the following skin diseases: (1) eczema, (2) psoriasis, (3) acne vulgaris, (4) chronic urticaria, (5) pigmentary disorders, (6) hair loss, (7) viral warts, (8) fungal infections, and (9) keloids. Demographic and socioeconomic information was collected. Health status and utilities (Dermatology Life Quality Index, Skindex-16, EuroQol-5 Dimension, visual analog scale, time-trade-off, and willingness-to-pay) were measured., Results: A total of 183 patients with a median age of 35.5 (21-77 years) years were included. Majority (76.9%) were Chinese, followed by Malays (11.5%). The time-trade-off utilities were lower than EuroQol-5 Dimension utilities across all disease severity and most skin diseases. Patients were willing to pay $740 USD or more for a hypothetical drug to cure conditions such as psoriasis, acne, hair loss, and keloids., Limitations: Limited sample size and ethnic representation., Conclusions: This study provides a catalog of utilities in skin diseases and highlights the strengths and challenges of different measures., Competing Interests: Dr Barbieri has received consulting fees from Dexcel pharma for work unrelated to the present submission. Drs Yew and Chen have no conflicts of interest., (© 2024 by the American Academy of Dermatology, Inc. Published by Elsevier Inc.)

Health technology assessment agencies often prefer that utilities used to calculate quality-adjusted life years in cost-utility analyses (CUAs) are derived using standardized methods, such as generic preference-based measures completed by patients in clinical trials. However, there are situations when no standardized approach is feasible or appropriate for a specific medical condition or treatment that must be represented in a CUA. When this occurs, vignette-based methods are often used to estimate utilities. A vignette (sometimes called a "scenario," "health state description," "health state vignette," or "health state") is a description of a health state that is valued in a preference elicitation task to obtain a utility estimate. This method is sometimes the only feasible way to estimate utilities representing a concept that is important for a CUA. Consequently, vignette-based studies continue to be conducted and published, with the resulting utilities used in economic models to inform decision making about healthcare resource allocation. Despite the potential impact of vignette-based utilities on medical decision making, there is no published guidance or review of this methodology. This article provides recommendations for researchers, health technology assessment reviewers, and policymakers who may be deciding whether to use vignette-based methods, designing a vignette study, using vignette-based utilities in a CUA, or evaluating a CUA that includes vignette-based utilities. Recommendations are provided on: (A) when to use vignette-based utilities, (B) methods for developing vignettes, (C) valuing vignettes, (D) use of vignette-based utilities in models, and (E) limitations of vignette methods. [ABSTRACT FROM AUTHOR]

Background: Reference values are a helpful tool to facilitate comparisons of sampled values against a specified reference population. The aim is to describe the health profile and to provide visual analogue scale (EQ VAS) and utility reference values for the EQ-5D-5L from a normative sample of the general elderly population (65+) in Germany.Methods: We analysed a sub-set of data from the German EQ-5D-5L valuation study using self-reported information based on EQ-5D-5L. We examined the share of respondents in each severity level per dimension as well as means, standard deviations (SD) and 95% confidence intervals for the index and EQ VAS values stratified by age groups and gender. Age was categorised in four groups (65-69, 70-74, 75-79 and > 79 years) to facilitate a more detailed examination of age-related health-related quality of life (HRQoL).Results: The average index and EQ VAS scores were 0.84 (SD 0.22) and 73.2 (SD 18.5), respectively. In total, 21.4% reported no problems in all dimensions. With higher age, health problems were reported more frequently, which, in turn, lead to monotonically decreasing index and EQ VAS values. Overall, men reported fewer problems than women and this difference was largest beyond the age of 80.Conclusion: HRQoL in the oldest old appears to be less stable and differs from the young elderly. However, the conventional age categorisation of earlier population norms studies seems to mask these differences. Hence, the more detailed provision of EQ-5D-5L reference values for the elderly population seems helpful for future German studies. [ABSTRACT FROM AUTHOR]

Background: Hemophagocyti.c lymphohistiocytosis (HLH) is a rare and severe disorder characterized by abnormal activation of the immune system. Primary HLH causes prolonged fever, spleen and liver enlargement, and organ dysfunction, usually in infancy and early childhood and is fatal if left untreated. As effective treatment options emerge, such as emapalumab-lzsg, Health Technology Assessment bodies around the world will assess them in terms of cost-effectiveness. This study was designed to estimate quality of life weights (utilities) for such analyses. Methods: Vignettes were developed describing HLH treatment related health states. Health states included active HLH, HLH plus neurological symptoms, receiving chemotherapy, undergoing stem cell transplant (SCT), graft versus host disease (GVHD), cure and end of life care. The vignettes were based on information from in depth interviews with clinical specialists; and qualitative research with four parents of children with primary HLH aged between 1 and 18 years old. The vignettes were then assessed in time trade off (TTO) interviews with members of the UK general public in one on one face to face interviews with trained, experienced interviewers. Preference data were analysed using the generalised estimating equations framework. Results: Detailed qualitative data captured the substantial burden of this disease for young children. One hundred participants completed the TTO interviews. The utility score for Active HLH was estimated as 0.32 (95% CI, 0.24 to 0.39). Values for other states were HLH plus neurological symptoms (0.27, 95%CI 0.18–0.35), receiving chemotherapy (0.26, 95%CI 0.17–0.34), undergoing SCT (0.18, 95%CI 0.07–0.28), GVHD (0.07, 95%CI -0.04-0.17), cure (0.72, 95%CI 0.67–0.77) and end of life care (− 0.17, 95%CI -0.27- -0.07). Conclusions: This study has estimated utility weights for seven different HLH related states which are based on detailed input from carers and physicians and have good face validity. There are few other options for collecting these data in an ultra-rare setting. [ABSTRACT FROM AUTHOR]

Background: The VOYAGER PAD (Efficacy and Safety of Rivaroxaban in Reducing the Risk of Major Thrombotic Vascular Events in Subjects With Symptomatic Peripheral Artery Disease Undergoing Peripheral Revascularization Procedures of the Lower Extremities) trial compared rivaroxaban (2.5 mg twice a day) plus aspirin with aspirin alone in patients with symptomatic peripheral artery disease requiring endovascular or surgical limb revascularization, with 50% receiving clopidogrel background therapy. The New Drug Indication application includes benefit-risk assessments using clinical judgment to balance benefits against risks. During its review, the US Food and Drug Administration requested additional quantitative benefit-risk analyses with formal weighting approaches., Methods and Results: Benefits and risks were assessed using rate differences between treatment groups (unweighted analysis). To account for clinical importance of the end points, a multi-criteria decision analysis was conducted using health state utility values as weights. Monte Carlo simulations incorporated statistical uncertainties of the event rates and utility weights. Intent-to-treat and on-treatment analyses were conducted. For unweighted intent-to-treat analyses, rivaroxaban plus aspirin would result in 120 (95% CI, -208 to -32) fewer events of the primary composite end point (per 10 000 patient-years) compared with aspirin alone. Rivaroxaban caused an excess of 40 (95% CI, 8-72) Thrombolysis in Myocardial Infarction major bleeding events, which was largely driven by nonfatal, nonintracranial hemorrhage Thrombolysis in Myocardial Infarction major bleeding events. For weighted analyses, rivaroxaban resulted in the utility equivalent of 13.7 (95% CI, -85.3 to 52.6) and 68.1 (95% CI, 7.9-135.7) fewer deaths per 10 000 patient-years (intent-to-treat and on-treatment, respectively), corresponding to probabilities of 64.4% and 98.7%, respectively, that benefits outweigh risks favoring rivaroxaban per Monte Carlo simulation., Conclusions: These analyses show a favorable benefit-risk profile of rivaroxaban therapy in the VOYAGER PAD trial, with findings generally consistent between the unweighted and weighted approaches.

Background: Chronic hepatitis B (CHB) significantly impacts the health-related quality of life (HRQoL), but remains underexplored in the Thai population. Health state utilities (HSU) are indicators reflecting HRQoL which serve as fundamental inputs for economic evaluation analyses. This study aimed at assessing differences in HRQoL across five CHB stages in Thai patients, including non-cirrhotic CHB, compensated cirrhosis, decompensated cirrhosis, early-/intermediate-stage hepatocellular carcinoma (HCC) and advanced-/terminal-stage HCC., Methods: We conducted a cross-sectional study to collect HRQoL data from patients with CHB at five stages. The study included patients with CHB who were followed up at a super-tertiary care centre between March 2021 and February 2022. The participants completed the EQ-5D-5L questionnaire and provided demographic data. Disease stage and relevant data were obtained from medical records. HSU and Euroqol-visual analogue scale (EQ-VAS) scores, calculated using Thai-specific conversion coefficients, were assessed., Results: Among 422 patients, 236 did not have cirrhosis, 92 had compensated cirrhosis, 13 had decompensated cirrhosis, 55 had early-/intermediate-stage HCC, and 26 had advanced-/terminal-stage HCC. The HSU scores for non-cirrhotic, compensated cirrhosis, decompensated cirrhosis, early-/intermediate-stage HCC and advanced-/terminal-stage HCC were 0.95 ± 0.08, 0.89 ± 0.16, 0.79 ± 0.19, 0.89 ± 0.12 and 0.52 ± 0.39, respectively. Similarly, the EQ-VAS scores for various CHB stages were 83.56 ± 12.90, 80.48 ± 13.03, 68.76 ± 17.40, 79.00 ± 14.38 and 62.92 ± 20.62, respectively. A significant correlation ( r  =   0.469, P  <   0.001) was observed between the HSU and EQ-VAS scores. The disease progression led to a notable HSU decline, particularly in the advanced-/terminal-stage HCC group (regression coefficient: -0.436, P  <   0.001). The EQ-VAS scores indicated reduced quality of life in advanced liver disease., Conclusions: Later CHB stages compromise the HRQoL. Decompensated cirrhosis and advanced-/terminal-stage HCC profoundly affect physical health and quality of life, whereas patients with compensated cirrhosis and early-/intermediate-stage HCC report better HRQoL., Competing Interests: R. Chantrakul, P. Sripongpun, T. Pattarapuntakul, N. Chamroonkul, C Kongkamol, P. Phisalprapa and A. Kaewdech declare that there is no conflict of interest in this study., (© The Author(s) 2024. Published by Oxford University Press and Sixth Affiliated Hospital of Sun Yat-sen University.)

Purpose: To describe health status and health state utilities measured by the EQ-5D-3L in a population-based sample of individuals aged 85 + in Germany, and to analyze associations with basic socio-demographic variables. Methods: Cross-sectional data from follow-up wave 7 (n = 761) of the German AgeCoDe Study were used. The EQ-5D-3L questionnaire was used to record problems in five health dimensions, its visual analogue scale (EQ VAS) was used to record self-rated health status, and the German EQ-5D-3L index was used to derive health state utilities. Results: Mean age of respondents was 88.9 years (SD 2.9; range 85 to 100), 67.4% were female. 81.9% reported problems in at least one of the EQ-5D dimensions, with 15.3% reporting extreme problems. Most frequent were problems with pain/discomfort (64.8%), followed by mobility (62.5%), usual activities (42.6%), self-care (28.2%), and anxiety/depression (20.5%). Mean EQ VAS score was 62.4 (SD 18.8), and mean EQ-5D index was 0.77 (SD 0.24). Multiple regression analysis showed associations of problem frequency in various EQ-5D dimensions with age, gender, living situation, marital status, and education. The EQ VAS score was negatively associated with age (β = − 0.56; p < 0.05) and female gender (β = − 3.49; p < 0.05). The EQ-5D index was negatively associated with not living in the community (β = − 0.10; p < 0.001) and being single (β = − 0.09; p < 0.05). Conclusions: The results show a substantially impaired health status of the oldest-old population. The data can be used for comparing health status of population groups as well as for health economic models. [ABSTRACT FROM AUTHOR]

Objectives: The present study aimed to provide normative data for the EQ-5D-5L questionnaire in Bulgaria, based on a nationally representative sample. Methods: Random sampling was used. In September 2018, a total of 1005 respondents (aged 18–89 years) completed the self-administered paper-based EQ-5D-5L questionnaire, including a visual analogue scale (EQ VAS). Health state utility index scores were derived using the directly measured Polish value set. Results: The study sample was representative of the general Bulgarian population in terms of age, sex, geographical region, educational level, social and professional status. Mean EQ-5D-5L and EQ VAS values decreased from 0.986 and 89.7 (age group 18–24 years) to 0.789 and 53.6 (age group ≥ 75 years), respectively. Perfect health (the "11,111" health state) was reported by half of the population (50.1%), more often by men than women (55.8% vs 44.9%). The most frequently reported complaints characterised pain/discomfort dimension (39.1%), followed by anxiety/depression (34.5%). Although the least commonly reported health limitations concerned the self-care dimension, their frequency (13.6%) was the highest among the seventeen identified EQ-5D-5L population norm studies. The mean severity index score for the whole study sample was 6.96. EQ-5D-5L index was higher in respondents from lower age groups and with a higher average income per household member. Conclusions: Bulgarian population norms, which were developed for the descriptive part of the EQ-5D-5L and EQ VAS, can be used as reference values. The availability of such normative data should encourage the use of the EQ-5D-5L questionnaire in health-related quality-of-life studies in Bulgaria. [ABSTRACT FROM AUTHOR]

Introduction: The Neuro-QoL is a standardized approach to assessing health-related quality of life in people with neurological conditions, including multiple sclerosis (MS). Item banks were developed with item response theory (IRT) methodology so items are calibrated along a continuum of each construct. The purpose of this study was to develop a preference-based scoring algorithm for the Neuro-QoL to derive utilities that could be used in economic modeling. Methods: With input from neurologists, 6 Neuro-QoL domains were selected based on relevance to MS and used to define health states for a utility elicitation study in the United Kingdom. General population participants and individuals with MS valued the health states and completed questionnaires (including Neuro-QoL short forms). The Neuro-QoL Utility Scoring System (NQU) was derived based on multi-attribute utility theory using data from the general population sample. Single-attribute disutility functions for 6 Neuro-QoL domains were estimated using isotonic regression with linear interpolation and then combined with a multiplicative model. NQU validity was assessed using MS participant data. Results: Interviews were completed with 203 general population participants (50.2% female; mean age = 45.0 years) and 62 participants with MS (62.9% female; mean age = 46.1 years). Mean (SD) NQU scores were 0.94 (0.06) and 0.82 (0.13) for the general population and MS samples, respectively. The NQU demonstrated known-groups validity by differentiating among subgroups categorized based on level of disability. The NQU demonstrated convergent validity via correlations with generic measures (0.66 and 0.63 with EQ-5D-5L and Health Utilities Index Mark 3, respectively; both P < 0.001). Discussion: With the NQU, utilities can be derived from any MS treatment group, subgroup, or patient sample who completes items from 6 Neuro-QoL domains. Because the Neuro-QoL is frequently used with MS patients, the NQU greatly expands the options for quantifying outcomes in cost-utility analyses conducted to inform allocation of resources for MS treatment. [ABSTRACT FROM AUTHOR]

Summary: Health state utilities (HSUs) are health economic metrics that capture and assess health‐related quality of life (HRQoL). They are essential in health‐economic evaluations when calculating quality‐adjusted life years. We investigated published studies reporting bariatric surgery‐related HSUs elicited through direct or indirect (multiattribute utility instrument [MAUI]) patient‐reported methods (PROSPERO registration number: CRD42019131725). Mean HSUs for different time points and HSU changes over time (where feasible) were meta‐analysed using random‐effects models. Of the 950 potentially relevant identified studies, n = 28 (2004–2018) qualified for data extraction, with n = 85 unique HSUs elicited mainly from the EQ‐5D (88%). Most (75%) studies were published after 2013. The follow‐up duration varied between studies and was often limited to 12 months. The pooled mean HSU was 0.72 (0.67–0.76) at baseline/presurgery (n = 18) and 0.84 (0.79–0.89) one‐year postsurgery (n = 11), indicating a 0.11 (0.09–0.14) utility unit increment. EQ‐5D showed the similar results. This positive difference can be partially explained by BMI and/or co‐morbidities status improvement. This study provides a valuable summary of HSUs to future bariatric surgery‐related cost‐utility models. However, more well‐designed higher‐quality bariatric‐related HSU studies are expected for future reviews to improve the available evidence. We suggest that researchers select an MAUI that is preferentially sensitive to the study population. [ABSTRACT FROM AUTHOR]

Objective: Identify the most recent utility value estimates for cardiovascular disease (CVD) via systematic literature review (SLR) and explore trends in utility elicitation methods in the last 6 years.Methods: This SLR was updated on January 25, 2018, and identified studies reporting utilities for myocardial infarction (MI), stroke, angina, peripheral artery disease (PAD), and any-cause revascularization by searching Embase, PubMed, Health Technology Assessment Database, and grey literature.Results: A total of 375 studies reported CVD utilities (pre-2013 vs post-2013: MI, 38 vs 32; stroke, 86 vs 113; stable angina, 8 vs 9; undefined/unstable angina, 23 vs 8; PAD, 29 vs 13; revascularization, 54 vs 40). Median average utilities for MI, stroke, and revascularization increased over time (pre-2013 vs post-2013: MI, 0.71 vs 0.79; stroke, 0.63 vs 0.64; revascularization, 0.76 vs 0.81); angina and PAD showed a decrease in median values over time (stable angina, 0.83 vs 0.72; undefined/unstable angina, 0.70 vs 0.69; PAD, 0.76 vs 0.71). The proportion of utility estimates from trials increased across health states (pre-2013 vs post-2013: 22.5% vs 37.2%), as did the proportion of trials using the EuroQol Five Dimensions Questionnaire (EQ-5D; pre-2013 vs post-2013: 73.8% vs 91.4%). Use of methods such as the standard gamble, time trade-off, and Health Utilities Index has declined.Conclusions: Health state utilities for cardiovascular health states have changed in the last 6 years, likely due to changes in the types of studies conducted, the patient populations evaluated, and possibly changing utility elicitation methods. The EQ-5D has been used more frequently. [ABSTRACT FROM AUTHOR]

Objectives: Formal evaluation of health states related to dysphonia have not been rigorously evaluated in affected patients. The objective of this project was to evaluate the health states of mild, moderate, and severe dysphonia using formal health state preference evaluation, and to compare these outcomes with the degree of voice handicap.Design: Prospective health state preference assessment.Methods: A convenience sample of patients presenting with voice complaints were enrolled from an academic voice center. Demographic and voice handicap index (VHI-10) data were obtained, and an assessment of preference for five health states (monocular blindness, binocular blindness, mild dysphonia, moderate dysphonia, and severe dysphonia) was performed. Utility scores were calculated on a scale from 0 (death) to 1 (perfect health). Analysis was performed with ANOVA testing with post-hoc comparisons and correlation statistics.Results: Of 209 assessments, 149 (75.6%) met quality criteria. Relative to monocular blindness (score 0.61 [CI 0.57-0.64]), moderate dysphonia (0.58 [0.54-0.62]) was rated equivalently, with severe dysphonia (0.33 [0.29-0.37]) ranking significantly worse and mild dysphonia (0.96 [0.95-0.98]) significantly better. Binocular blindness (0.18 [0.15-0.21]) was the worst-ranked health state. There was a weak inverse correlation of VHI-10 with dysphonia-related preference scores; with worsening reported voice handicap, scores decreased.Conclusion: This study demonstrated that dysphonia had a significant impact of quality of life, with moderate dysphonia ranking equivalently with monocular blindness. These numerical estimates may be used for ongoing research into the value and cost-effectiveness of medical, therapeutic, and surgical interventions for voice disorders.Level Of Evidence: 2c (outcomes research) Laryngoscope, 130:E177-E182, 2020. [ABSTRACT FROM AUTHOR]

Objective: The aims of this study were to examine current reporting standards of health state utilities (HSU) using a review of published cost-effectiveness analyses in cardiovascular disease and to explore the impact of variation in model inputs used in these on estimated quality-adjusted life-years (QALYs) and cost-effectiveness.Methods: Key health/economics bibliographic databases were searched to identify relevant articles published after 2014. Any narrative or values relating to the HSU used in the model were extracted and reviewed. The HSUs were systematically applied to an existing model to explore the influence of different values on QALYs and the incremental cost-effectiveness ratio.Results: Twenty-four peer-reviewed articles were identified. Only 2 studies referred to a literature review for the HSUs. Most (18 of 24) referenced previously published economic studies (as opposed to the original source) for at least 1 of the HSUs. Only 4 studies referenced the original sources and reported all of the HSUs accurately, and several did not provide all the HSUs. Little information was provided on the methods used to calculate QALYs, for example, the duration of time for acute HSUs, what the baseline HSU was, the method that was used to assign HSUs for subsequent different events, or how constant HSUs for clinical events were combined with age-adjusted baseline values. The huge differences in HSUs used in the studies produced substantial variations in the QALYs and incremental cost-effectiveness ratios generated from the cost-effectiveness model.Conclusion: Current standards are poor, and there is a need for greater transparency in reporting the HSUs used in cost-effectiveness models. [ABSTRACT FROM AUTHOR]

Aims: Health state utilities associated with weight change are needed for cost-utility analyses (CUAs) examining the value of treatments for type 2 diabetes and obesity. Previous studies have estimated the utility benefits associated with various amounts of weight reduction in the US and Europe, but preferences for weight change in Asian cultures may differ from these published values. The purpose of this study was to estimate utilities associated with reductions in body weight based on preferences of individuals with type 2 diabetes and obesity in Japan., Methods: Health state vignettes represented type 2 diabetes with respondents' own current weight and weight reductions of 2.5%, 5%, 7.5%, 10%, 12.5%, 15%, and 20%. Utilities were elicited in time trade-off interviews with a sample of respondents in Japan with type 2 diabetes and body mass index (BMI) ≥25 kg/m 2 (the cutoff for obesity in Japan)., Results: Analyses were conducted with data from 138 respondents (84.8% male; mean age = 58.0 years; mean BMI = 29.4 kg/m 2 ) from all eight regions of Japan. Utility gains gradually increased with rising percentage of weight reductions ranging from 2.5% to 15%. Weight reductions of 2.5% to 15% resulted in utility increases of 0.013 to 0.048. The health state representing a 20% weight reduction yielded a wide range of preferences (mean utility increase of 0.044). Equations are recommended for estimating utility change based on any percentage of weight reduction (up to 20%) in Japanese people with type 2 diabetes and obesity., Limitations: This study was conducted in a sample with limited representation of patients with BMI >35 kg/m 2 ( n  = 13) and relatively few women ( n  = 21)., Conclusion: Results may be used to provide inputs for CUAs examining the value of treatments that are associated with weight loss in patients with type 2 diabetes and obesity in Japan.

Background: Obesity is a major public health challenge and its prevalence has increased across the age spectrum from 1980 to date in most parts of the world including sub-Saharan Africa. Studies that derive health state utilities (HSUs) stratified by weight status to support the conduct of economic evaluations and prioritization of cost-effective weight management interventions are lacking in sub-Saharan Africa.Objectives: To estimate age- and sex-specific HSUs for Ghana, along with HSUs by weight status. Associations between HSUs and overweight and obesity will be examined.Study Design: Cross-sectional survey of the Ghanaian population.Methods: Data were sourced from the World Health Organization Study of Global AGEing and Adult Health (WHO SAGE), 2014 to 2015. Using a "judgment-based mapping" method, responses to items from the World Health Organization Quality-of-Life (WHOQOL-100) used in the WHO SAGE were mapped to EQ-5D-5L profiles, and the Zimbabwe value set was applied to calculate HSUs. Poststratified sampling weights were applied to estimate mean HSUs, and a multivariable linear regression model was used to examine associations between HSUs and overweight or obesity.Results: Responses from 3966 adults aged 18 to 110 years were analyzed. The mean (95% confidence interval) HSU was 0.856 (95% CI: 0.850, 0.863) for the population, 0.866 (95% CI: 0.857, 0.875) for men, and 0.849 (95% CI: 0.841, 0.856) for women. Lower mean HSUs were observed for obese individuals and with older ages. Multivariable regression analysis showed that HSUs were negatively associated with obesity (-0.024; 95% CI: -0.037, -0.011), female sex (-0.011; 95% CI: -0.020, -0.003), and older age groups in the population.Conclusions: The study provides HSUs by sex, age, and body mass index (BMI) categories for the Ghanaian population and examines associations between HSU and high BMI. Obesity was negatively associated with health state utility in the population. These data can be used in future economic evaluations for Ghana and sub-Saharan African populations. [ABSTRACT FROM AUTHOR]

Background Context: The US National Pain Strategy focused attention on high-impact chronic pain and its restrictions. Although many interventions have been studied for chronic low-back pain, results are typically reported for heterogeneous samples. To better understand chronic pain and target interventions to those who most need care, more granular classifications recognizing chronic pain's impact are needed.Purpose: To test whether chronic pain impact levels can be identified in chronic low-back pain clinical trial samples, examine the baseline patient mix across studies, and evaluate the construct validity of high-impact chronic pain.Study Design/setting: Descriptive analyses using 12 large study datasets.Patient Samples: Chronic low-back pain patients in nonsurgical, nonpharmacologic trials in the US, Canada, and UK.Outcome Measures: Preference-based health utilities from the SF-6D and EQ-5D, employment status and absenteeism.Methods: We used two logistic regression models to predict whether patients had high-impact chronic pain and whether the remainder had low- or moderate-impact chronic pain. We developed these models using two datasets. Models with the best predictive power were used to impute impact levels for six other datasets. Stratified by these estimated chronic pain impact levels, we characterized the case mix of patients at baseline in each dataset, and summarized their health-utilities and work productivity. This study was funded by a National Center for Complementary and Integrative Medicine grant. The authors have no potential conflicts of interest.Results: The logistic models had excellent predictive power to identify those with high-impact chronic pain. Although studies were all of chronic low-back pain patients, the baseline mix of patients varied widely. Across all datasets, utilities, and productivity were similar for those with high-impact chronic pain and worsened as chronic pain impact increased.Conclusions: There is a need to better categorize chronic pain patients to allow the targeting of optimal interventions for those with each level of chronic pain impact. [ABSTRACT FROM AUTHOR]

Background: The time trade-off (TTO) technique is commonly used to elicit health state utilities. Nevertheless, when the health states being valued are temporary, the TTO approach may be unsuitable. A variant of TTO- chained TTO-has been suggested to be used when the health states are temporary, but little research has been done on how chained TTO should be conducted.Objectives: To systematically review the use of chained TTO in valuing temporary health states.Methods: A systematic literature search was conducted using the following major databases: Ovid MEDLINE(R), Embase, EBM Reviews, and PsycINFO. Abstracts (full articles if necessary) were screened by two independent reviewers, with a third reviewer resolving any disagreements.Results: The resulting number of articles for review was low (n = 9). All the reviewed studies used face-to-face interviews, most had small sample sizes (<100), and all studies valued a small number of health states (<7), with time horizons typically ranging from 4 weeks to 1 year. All studies discussed methodological issues of using chained TTO, and some compared the results with those generated using other preference elicitation methods.Conclusions: Chained TTO appears to be feasible, consistent, and responsive and allows the valuation of temporary health states that would improve the efficiency and accuracy of decision making in health and health care. Nevertheless, the evidence is limited due to the low number of relevant studies in the literature. Further research is needed to examine the performance and validity of chained TTO compared with conventional TTO in the valuation of temporary health states. [ABSTRACT FROM AUTHOR]

Introduction Clinical trials have shown promising results for extended half-life factor VIII concentrates but little is known about individuals' valuation of haemophilia treatment attributes. Aim To assess patient/caregiver and population valuation of treatment attributes of prophylactic regimens for people with severe haemophilia A. Methods Members ≥16 years of the Swedish Haemophilia Society ( FBIS) and of a web-panel representative of the Swedish population were invited to participate in a web-survey investigating preferences for haemophilia treatment attributes using the Time Trade-Off methodology which ranks health states on a scale 0 (dead) to 1 (full health). All respondents assessed the same four treatment scenarios for severe haemophilia A, each described by three stylized attributes: injection interval (every 2nd or 5th day); participation in physical activity (Y/N); annual risk of bleed (1-2 or 5-6 bleeds). Results The survey had 1657 respondents (68% complete responses; 184/1233 from FBIS/web-panel gave informed consent; mean age 52 years, 51% men). Respondents from FBIS and from the web-panel had the same preference ranking of the four treatment scenarios, but members of FBIS consistently rated significantly higher health utilities; range 0.67-0.73 vs 0.54-0.60. Participation in physical activity implied +0.023 (95% confidence interval 0.015-0.030); a longer injection interval implied +0.038 (0.03; 0.45); and fewer bleeds implied +0.022 (0.015-0.029) utility points. Conclusions Patient/caregiver and population preferences indicate that treatment attributes such as frequency of injections and the possibility of participating in physical activity are important attributes impacting quality of life in addition to the control and prevention of bleeding episodes. [ABSTRACT FROM AUTHOR]

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., Competing Interests: D.H. reports consulting and speaker fees from Amicus Therapeutics, Chiesi, Freeline Therapeutics, Idorsia, Protalix BioTherapeutics, Sanofi Genzyme, and Takeda outside the submitted work. A.L., K.S., and L.L. are or were employees of PHMR, London, UK at the time the study took place. A.L. is a current employee of Decisive Consulting Ltd, London, UK. K.S. is a current employee of the National Institute for Health and Care Excellence, UK. G.D. and O.A. are employees of Takeda Development Center Americas, Inc. and stockholders of Takeda Pharmaceuticals Company Limited.

Background: In this analysis we report patients with advanced gastrointestinal stromal tumors (GIST) refractory to imatinib and sunitinib therapy as derived from the EuroQol-5D (EQ-5D) for progression-free (PF) and progressive disease health status. Methods: Data were analyzed from a phase III trial conducted at 57 hospitals in 17 countries (trial registration number, NCT01271712). Patients with advanced GIST were randomized (2:1) to receive blinded treatment using oral regorafenib 160 mg daily or placebo, plus best supportive care (BSC) in both groups, for the first 3 weeks of each 4-week cycle. EQ-5D-3L was administered on day 1 of each cycle before contact with their physician and before any study-related procedures. The effect of disease progression on the utility of EQ-5D was tested with paired-samples comparison and general linear mixed modeling (GLMM). Results: One hundred and eighty five patients [93 % of the intention-to-treat (ITT) population] completed 803 EQ-5D questionnaires: 77.7 % in progression-free (PF) state, 6.5 % at progression, 13.9 % following first progression, and 1.9 % after second progression. Mean baseline utility was 0.767 (SD 0.221) with no significant between-group differences for active treatment and BSC. The first post-progression health state was 0.647 (SD 0.343), suggesting significantly impaired health-related quality of life after confirmed disease progression showed a decrease of −0.120 (paired samples t test, p = 0.001). GLMM showed no effect of study treatment or cycle number on utility. Conclusions: We demonstrate a significant and clinically meaningful difference in health state utility values between PF and progression. Utility values remained stable over successive regorafenib cycles after controlling for disease status and treatment type. [ABSTRACT FROM AUTHOR]

The assessment of health-related quality of life (HRQL) via patient-reported outcomes has the potential to answer critical questions and improve the care of soft tissue sarcoma (STS). This review outlines the rationale for quality of life measures in sarcoma, and details various instrument types: disease- and anatomic-specific, provider-generated, generic HRQL and health state utilities. Prior usage in STS populations, relative advantages of specific patient-reported outcome measures and a framework for selecting appropriate measures are discussed. Uniform incorporation of validated HRQL measures in STS clinical research would further the understanding of patient wellbeing beyond traditional clinical measures, and more widespread use of health state utilities measures in particular has the potential to facilitate comparative effectiveness research. [ABSTRACT FROM AUTHOR]

Background With human papillomavirus (HPV) vaccination introduced in a number of countries, there is considerable interest in evaluating the cost-effectiveness of HPV testing as the primary cervical screening test in these settings. However, the availability of utility scores for these newer interventions is limited. Our aim in this paper is to present utility scores for HPV vaccination, HPV testing and cytology based screening states among women targeted for cervical screening. Methods We invited a random sample of women targeted for cervical screening (aged 20-69 years) living in Sydney, Australia, to participate in a face-to-face interview. Participants were asked to indicate preferences (rank and utility scores) for 10 hypothetical health states relating to HPV vaccination, cytology and primary HPV screening, cervical precursor disease and early stage cervical cancer. Preferences for hypothetical health states were measured through ranking then a two-stage standard gamble. Each participant's own health state was measured as a utility score using the EQ5D. Potential differences by age were assessed using the Wilcox Rank Sum test. Results A maximum of 276 women were contacted, of which 43 (mean age 49 years) agreed to be interviewed (15.6%). The overall health state of women as measured by the EQ5D was 0.86 (95%CI: 0.83-0.89). Of the 10 health states, the highest ranked were 'normal cytology' and 'HPV vaccination' (equal 1st). States involving an HPV positive result with a subsequent normal cytology or colposcopy were ranked below those for low grade cytological abnormalities with or without a subsequent colposcopic normal result (ranks 3-4 vs. 4-5). However, mean utility scores were broadly similar for all health states, except cervical cancer. No significant differences in scores were identified between age groups. Conclusion Our survey suggests health states relating to HPV testing are ranked below 'low grade cytology' disease abnormalities. However, this difference was minimal on the utility scale, as most values for health states were largely clustered. These results provide a preliminary set of non-clinic population-based utilities that may be used with other values to explore the economic implications of introducing HPV testing as a primary screening tool in the context of HPV vaccination. [ABSTRACT FROM AUTHOR]