Hypothetical Health States Research Articles

A Spanish value set for the SF-6D based on the SF-12 v1.

This paper reports the first estimation of an SF-6D value set based on the SF-12 for Spain. A representative sample (n = 1020) of the Spanish general population valued a selection of 56 hypothetical SF-6D health states by means of a probability lottery equivalent (PLE) method. The value set was derived using both random effects and mean models estimated by ordinary least squares (OLS). The best model was chosen on the basis of its predictive ability assessed in terms of mean absolute error (MAE). The model yielding the lowest MAE (0.075) was that based on main effects using OLS. Pain was the most significant dimension in predicting health state severity. Comparison with the previous SF-6D (SF-36) model estimated for Spain revealed no significant differences, with a similar MAE (0.081). Nevertheless, the new SF-6D (SF-12) model predicted higher utilities than those generated by the SF-6D (SF-36) scoring algorithm (minimum value -0.071 vs -0.357). A value set for the SF-6D (SF-12) based on Spanish general population preferences elicited by means of a PLE technique is successfully estimated. The new estimated SF-6D (SF-12) preference-based measure provides a valuable tool for researchers and policymakers to assess the cost-effectiveness of new health technologies in Spain.

Assessing utilities for muscle-invasive bladder cancer-related health states

ObjectivesHow patients value functional outcomes against oncologic outcomes during decision-making for muscular-invasive bladder cancer (MIBC) remains unclear. We sought to quantify individuals’ preferences on a scale of 0 to 1, where 1 represents perfect health and 0 represents death. MethodsDescriptions of 6 hypothetical health states were developed. These included: Neoadjuvant chemotherapy followed by radical cystectomy with ileal conduit (IC) or with neobladder reconstruction (NB), Transurethral resection and chemotherapy/radiation (CRT), CRT requiring salvage cystectomy (SC), Recurrent/metastatic bladder cancer after local therapy (RMBC), and Metastatic bladder cancer (MBC). Descriptions consisted of diagnosis, treatments, adverse effects, follow-up protocol, and prognosis and were reviewed for accuracy by expert panel. Included individuals were asked to evaluate states using the visual analog scale (VAS) and standard gamble (SG) methods. ResultsFifty-four individuals were included for analysis. No score differences were observed between IC, NB, and CRT on VAS or SG. On VAS, SC (value = 0.429) was rated as significantly worse (P < 0.001) than NB (value = 0.582) and CRT (value = 0.565). However, this was not the case using the SG method. Both RMBC (VAS value = 0.178, SG value = 0.631) and MBC (VAS value = 0.169, SG value = 0.327) rated as significantly worse (P < 0.001) than the other states using both VAS and SG. ConclusionsWithin this sample of the general population, preferences for local treatments including IC, NB, and CRT were not found to be significantly different. These values can be used to calculate quality-adjusted life expectancy in future cost-effectiveness analyses.

Health Utilities of Bilateral Severe-to-Profound Hearing Loss with Assistive Devices.

Hearing loss is a common sensory disorder in newborns. Early intervention with assistive devices benefits children's auditory and speech performance. This study aimed to measure the health utilities of children with bilateral severe-to-profound hearing impairment with different assistive devices. The descriptions of four hypothetical health states were developed, and their utility values were obtained from healthcare professionals via the visual analogue scale (VAS) and time trade-off (TTO) methods. Thirty-seven healthcare professionals completed the TTO interview and were included in the analysis. The mean utility scores obtained via VAS were 0.31 for no assistive devices, 0.41 for bilateral hearing aids, 0.63 for bimodal hearing, and 0.82 for bilateral cochlear implants. As for the utility scores obtained via TTO, mean values were 0.60, 0.69, 0.81, and 0.90, respectively. None of the four groups had the same VAS- or TTO-elicited utility (p < 0.001). The post hoc test results showed that the difference was significant between any two groups (all p < 0.05). In conclusion, this study elicited health utility of bilateral hearing impairment with different assistive devices using the VAS and TTO methods. The utility values obtained provide critical data for future cost-utility analysis and health technology assessment.

Health utilities and willingness to pay in adult patients with coeliac disease in Hungary

BackgroundCoeliac disease (CD) is a life-long food-related disorder with a global prevalence of approximately 1%. Patients with CD often experience reduced health-related quality of life that could improve with a strict adherence to a gluten-free diet (GFD).ObjectivesTo obtain visual analogue scale (VAS), time trade-off (TTO) and willingness-to-pay (WTP) values amongst patients with CD.MethodsIn 2020–2021, a cross-sectional online survey was conducted amongst 312 adult CD patients in Hungary. Patients completed the Gastrointestinal Symptom Rating Scale (GSRS) and evaluated their current health and three hypothetical health state vignettes defined based on dietary adherence using VAS, conventional 10-year TTO and WTP. Multivariate regressions were used to explore the effect of patients’ demographic and clinical characteristics on utility and WTP values.ResultsMean VAS values for current health and ‘CD with strict adherence to GFD’, ‘CD with loose adherence to GFD’ and ‘CD without GFD’ hypothetical health states were 79.69 ± 18.52, 85.36 ± 16.18, 62.44 ± 19.91 and 36.69 ± 25.83, respectively. Corresponding mean TTO utilities were: 0.90 ± 0.19, 0.91 ± 0.20, 0.87 ± 0.23 and 0.76 ± 0.29. Mean annual WTP values for returning to full health were: €845 ± 1077, €648 ± 1002, €862 ± 1135 and €1251 ± 1496. Older age at diagnosis, male sex, more severe gastrointestinal symptoms (GSRS) and having comorbidities were associated with lower VAS and TTO or higher WTP values for current own health (p < 0.05).ConclusionThis is the first study to report TTO utilities for CD health states. Strict adherence to the GFD may result in substantial health gains in symptomatic patients. Utilities and WTP results can be used to estimate benefits of GFD in cost-utility and cost–benefit analyses.

Budget impact of introducing once-every-6-months paliperidone palmitate in US health care plans.

BACKGROUND: In the United States, most patients with schizophrenia have Medicaid coverage. Antipsychotic treatments are the cornerstone of schizophrenia management; most patients are treated with daily oral antipsychotics but struggle with medication adherence. Evidence suggests that medication adherence is inversely correlated with dosing frequency. Once-monthly paliperidone palmitate (PP) has been demonstrated to improve adherence compared with oral antipsychotics; transitioning to once-every-3-months PP (PP3M) further improved adherence. In 2021, once-every-6-months PP (PP6M) was approved by the US Food and Drug Administration to provide even longer between-dose intervals. Public health stakeholders who aim to improve medication adherence are interested in understanding how introducing PP6M to the formulary will impact the budget. OBJECTIVE: To evaluate the budget impact of introducing PP6M to the formulary from the perspectives of a hypothetical US multistate health care payer and state Medicaid programs using California, Georgia, and Ohio as examples. METHODS: The budget impact model was developed from a payer perspective, comparing the reference scenario (without PP6M in the market) with a new scenario (with PP6M). The study population included patients with schizophrenia who were eligible to receive PP6M. Market shares were assigned to the reference and new market scenarios. Efficacy was measured by the relative risk of relapse while receiving treatment. Adherence effects were included in the model and affected costs of treatment and relapse rates. A deterministic 1-way sensitivity analysis was performed. RESULTS: Base-case results for a multistate payer with 1 million members demonstrate that adding PP6M to the market results in total incremental plan-level costs ranging from $7,747 in year 1 to $11,501 in year 5. Increased drug costs were offset by administration and relapse cost savings ($105 and $881 in year 5, respectively). The average incremental cost per treated patient per year was stable at $180.06 for each year, and the incremental cost per member per month stayed below $0.01 for each year. The results of the model from the state-level Medicaid scenarios are broadly similar to those of the multistate base-case perspective. The 1-way sensitivity analysis demonstrated the model is most sensitive to the per-package costs of PP6M and PP3M, along with the proportion of patients fully adherent with PP3M. CONCLUSIONS: The budget impact of introducing PP6M as a treatment option is minimal. With the expected cost offsets from reduced administration and relapse costs due to adherence benefits, these results suggest that PP6M can be a viable treatment option from a clinical and a budgetary perspective. DISCLOSURES: This study was funded by Janssen Scientific Affairs, LLC. The study sponsor provided funds to Xcenda and ApotheCom for medical writing, editorial support, and submission of the manuscript. Hilary Phelps was an employee of Janssen Global Services, LLC, at the time of the development and finalization of the manuscript. Alex Keenan is an employee of Janssen Global Services, LLC, and holds stock in Johnson & Johnson, Inc. Dee Lin and Carmela Benson are employees of Janssen Scientific Affairs, LLC, and hold stock in Johnson & Johnson, Inc. Aditya Raju was an employee of Xcenda at the time of the development and finalization of the manuscript, and Danmeng Huang is an employee of Xcenda, a health care consulting firm that was contracted by Janssen Scientific Affairs, LLC. Chih-Yuan Cheng is an employee of Janssen NV.

Protocol for estimating the willingness-to-pay-based value for a quality-adjusted life year to aid health technology assessment in India: a cross-sectional study

IntroductionTo ensure that the evidence generated by health technology assessment (HTA) is translated to policy, it is important to generate a threshold value against which the outcomes of HTA studies...

An experience- and preference-based EQ-5D-3L value set derived using 18 months of longitudinal data in patients who sustained a fracture: results from the ICUROS

IntroductionEQ-5D-3L preference-based value sets are predominately based on hypothetical health states and derived in cross-sectional settings. Therefore, we derived an experience-based value set from a prospective observational study.MethodsThe International Costs and Utilities Related to Osteoporotic fractures Study (ICUROS) was a multinational study on fragility fractures, prospectively collecting EQ-5D-3L and Time trade-off (TTO) within two weeks after fracture (including pre-fracture recall), and at 4, 12, and 18 months thereafter. We derived an EQ-5D-3L value set by regressing the TTO values on the ten impairment levels in the EQ-5D-3L. We explored the potential for response shift and whether preferences for domains vary systematically with prior impairment in that domain. Finally, we compared the value set to 25 other EQ-5D-3L preference-based value sets.ResultsTTO data were available for 12,954 EQ-5D-3L health states in 4683 patients. All coefficients in the value set had the expected sign, were statistically significant, and increased monotonically with severity of impairment. We found evidence for response shift in mobility, self-care, and usual activities. The value set had good agreement with the only other experience- and preference-based value set, but poor agreement with all hypothetical value sets.ConclusionsWe present an experience- and preference-based value set with high face validity. The study indicates that response shift may be important to account for when deriving value sets. Furthermore, the study suggests that perspective (experienced versus hypothetical) is more important than country setting or demographics for valuation of EQ-5D-3L health states.

Hypothetical versus experienced health state valuation: a qualitative study of adult general public views and preferences

ObjectivesResponses from hypothetical and experienced valuation tasks of health-related quality of life differ, yet there is limited understanding of why these differences exist, what members of the public think about them, and acceptable resolutions. This study explores public understanding of, opinions on, and potential solutions to differences between hypothetical versus experienced responses, in the context of allocating health resources.MethodsSix focus groups with 30 members of the UK adult public were conducted, transcribed verbatim, and analysed using framework analysis. Participants self-completed the EQ-5D-5L, before reporting the expected consequences of being in two hypothetical EQ-5D-5L health states for ten years. Second, participants were presented with prior results on the same task from a public (hypothetical) and patient (experienced) sample. Third, a semi-structured discussion explored participants’: (1) understanding, (2) opinions, and (3) potential resolutions.ResultsTwenty themes emerged, clustered by the three discussion points. Most participants found imagining the health states difficult without experience, with those aligned to mental health harder to understand. Participants were surprised that health resource allocation was based on hypothetical responses. They viewed experienced responses as more accurate, but noted potential biases. Participants were in favour of better informing, but not influencing the public. Other solutions included incorporating other perspectives (e.g., carers) or combining/weighting responses.ConclusionMembers of the UK public appear intuitively not to support using potentially uninformed public values to hypothetical health states in the context of health resource allocation. Acceptable solutions involve recruiting people with greater experience, including other/combinations of views, or better informing respondents.

Assessment of health state utilities in dermatology: an experimental time trade-off value set for the dermatology life quality index

BackgroundDermatology Life Quality Index (DLQI) scores are used in many countries as access and reimbursement criteria for costly dermatological treatments. In this study we examined how time trade-off (TTO) utility valuations made by individuals from the general population are related to combinations of DLQI severity levels characterizing dermatologically relevant health states, with the ultimate purpose of developing a value set for the DLQI.MethodsWe used data from an online cross-sectional survey conducted in Hungary in 2020 (n = 842 after sample exclusions). Respondents were assigned to one of 18 random blocks and were asked to provide 10-year TTO valuations for the corresponding five hypothetical health states. To analyze the relationship between DLQI severity levels and utility valuations, we estimated linear, censored, ordinal, and beta regression models, complemented by two-part scalable models accommodating heterogeneity effects in respondents’ valuation scale usage. Successive severity levels (0–3) of each DLQI item were represented by dummy variables. We used cross-validation methods to reduce the initial set of 30 dummy variables and improve model robustness.ResultsOur final, censored linear regression model with 13 dummy variables had R2 = 0.136, thus accounting for 36.9% of the incremental explanatory power of a maximal (full-information) benchmark model (R2 = 0.148) over the uni-dimensional model (R2 = 0.129). Each DLQI item was found to have a negative effect on the valuation of health states, yet this effect was largely heterogeneous across DLQI items, and the relative contribution of distinctive severity levels also varied substantially. Overall, we found that the social/interpersonal consequences of skin conditions (in the areas of social and leisure activities, work and school, close personal relationships, and sexuality) had roughly twice as large disutility impact as the physical/practical aspects.ConclusionsWe have developed an experimental value set for the DLQI, which could prospectively be used for quantifying the quality-adjusted life years impact of dermatological treatments and serve as a basis for cost-effectiveness analyses. We suggest that, after validation of our main results through confirmatory studies, population-specific DLQI value sets could be developed and used for conducting cost-effectiveness analyses and developing financing guidelines in dermatological care.

Development the societal preference-based utility value set for the Patient Health Questionnaire depression scale in Hungary

IntroductionDepression is associated with high impact on health-related quality of life (HRQoL). Health state valuations are used for cost-effectiveness analysis to provide results for health-policy interventions.ObjectivesThe study aims to estimate a population-based value set of depression described by Patient Health Questionnaire (PHQ-9). We intend to assess vignettes describing PHQ-9 health states to estimate utility valuesMethodsCurrent research elicited direct utility scores using time trade-off (TTO) method obtained from the Hungarian general population (N=2,000). TTO vignettes were created to describe hypothetical health states of depression based on the nine items of PHQ-9. The hypothetical health states were sorted orthogonally in 11 blocks, each containing 4 vignettes (combinations of no; mild; moderate; severe depression). All respondents valued the four health states of one randomly given block. Conventional TTO method was applied, using a 10-year timeframe, while the first iteration step was randomized to 1,3 and 5 year. Preference weights were estimated using regression model fitted to TTO utility results.ResultsAltogether 1,999 respondents valued overall 34 different health states. The mean age was 47.3 (16.9) years, the majority was female (57.2%). Nearly half of the respondents were secondary educated 45.4%, 27.3% higher educated and 27.2% completed primary school. The mean TTO utility of selected mild, moderate and severe depression was: 0.83; 0.82 and 0.77 respectively.ConclusionsOur results constitute the first population-based value set for PHQ-9. Utility scores give useful information for cost-effectiveness assessments. Estimates provide preference-based quality of life weights for the Hungarian population.DisclosureNo significant relationships.

People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study.

IntroductionThis patient preference study sought to quantify the preferences of people living with COPD regarding symptom improvement in the UK, USA, France, Australia and Japan.MethodsThe inclusion criteria were people living with COPD aged 40 years or older who experienced ≥1 exacerbation in the previous year with daily symptoms of cough and excess mucus production. The study design included: 1) development of an attributes and levels grid through qualitative patient interviews; and 2) implementation of the main online quantitative survey, which included a discrete choice experiment (DCE) to allow assessment of attributes and levels using hypothetical health state profiles. Preference weights (utilities) were derived from the DCE using hierarchical Bayesian analysis. A preference simulator was developed that enabled different health state scenarios to be evaluated based on the predicted patient preferences.Results1050 people living with moderate-to-severe COPD completed the survey. All attributes were considered important when patients determined their preferences in the DCE. In a health state preference simulation, two hypothetical health states (comprising attribute levels) with qualitatively equivalent improvements in A) cough and mucus and B) shortness of breath (SOB) resulted in a clear preference for cough and mucus improved profile. When comparing two profiles with C) daily symptoms improved and D) exacerbations improved, there was a clear preference for the daily symptoms improved profile.ConclusionsPeople living with moderate-to-severe COPD prefer to reduce cough and mucus production together over improvement of SOB and would prefer to reduce combined daily symptoms over an improvement in exacerbations.

Self-esteem in patients with venous thromboembolism predicts time trade-off values for own health

BackgroundThe time trade-off (TTO) is a commonly used preference based method to assess health related values used in health economic analyses. Surprisingly little is known about the factors influencing the TTO. Since self-esteem is a predictor for health status measurements, and health status affects the TTO, we studied the relation between self-esteem and TTO values.MethodsData of 128 patients treated with vitamin K antagonists for venous thromboembolism on Short Form-36 (SF-36), Rosenberg self-esteem and patient characteristics were collected. TTO values were obtained for ‘current health’ and three chronic health states related to thrombosis, in face-to-face interviews with patients. Regression analyses were performed with the TTO as dependent variable. Analyses were performed in two groups; the complete sample, and traders only. Selected predictors were entered in four blocks: socio-demographic factors, medical-clinical factors, health status, and self-esteem.ResultsIn the complete sample (N = 128), bivariate regression analysis showed that self-esteem explained 14% of the variance in TTO values for current health (p < .000, N = 117). In traders, multivariate regression analysis showed a significant relationship between self-esteem and TTO values for current health. Self-esteem increased the variance explained (R2) by 8.8%, from 28.1 to 36.9%, (p = 0.01; N = 57). For hypothetical health states, the effect of self-esteem was weaker and mostly absent after controlling for selected variables.ConclusionsIn patients willing to trade-off time, higher self-esteem was associated with higher TTO values for own current health. Self-esteem explained an appreciable proportion of the variance in TTO values in traders. For hypothetical health states such associations were weak or absent.

Investigating 5-Level EQ-5D (EQ-5D-5L) Values Based on Preferences of Patients With Heart Disease

ObjectivesSeveral studies have shown that patients with heart disease value hypothetical health states differently from the general population. We aimed to investigate the health preferences of patients with heart disease and develop a value set for the 5-level EQ-5D (EQ-5D-5L) based on these patient preferences. MethodsPatients with confirmed heart disease were recruited from 2 hospitals in Singapore. A total of 86 EQ-5D-5L health states (10 per patient) were valued using a composite time trade-off method according to the international valuation protocol for EQ-5D-5L; 20-parameter linear models and 8-parameter cross-attribute level effects models with and without an N45 term (indicating whether any health state dimension at level 4 or 5 existed) were estimated. Each model included patient-specific random intercepts. Model performance was evaluated for out-of-sample and in-sample predictive accuracy in terms of root mean square error. The discriminative ability of the utility values was assessed using heart disease-related functional classes. ResultsA total of 576 patients were included in the analysis. The preferred model, with the lowest out-of-sample root mean square error, was a 20-parameter linear model including N45. Predicted utility values ranged from −0.727 for the worst state to 1 for full health; the value for the second-best state was 0.981. Utility values demonstrated good discriminative ability in differentiating among patients of varied functional classes. ConclusionsAn EQ-5D-5L value set representing the preferences of patients with heart disease was developed. The value set could be used for patient-centric economic evaluation and health-related quality of life assessment for patients with heart disease.

Time trade-off with someone to live for: impact of having significant others on time trade-off valuations of hypothetical health states

BackgroundThe TTO task involves giving up life years, i.e. living a shorter life, to avoid an undesirable health state. Despite being a hypothetical task, some respondents take other life factors into account when completing the task. This study explored the effect of having children and/or a partner on TTO valuations of hypothetical EQ-5D-5L health states in a valuation study of the general population.MethodsThe study used TTO data collected in a Norwegian EQ-5D-5L valuation study in 2019–2020, by one-to-one pc-assisted interviews following the EQ-VT protocol. We used regression modelling to determine the effect of significant others (having children or a partner) on disutility per health state from the TTO valuations.Results430 respondents were included [mean age 43.8 (SD 15.9) years, 58% female, 48% with children, 68% with a partner, 25% with neither children nor partner]. Having children and/or a partner was associated with lowered willingness to trade life years translating to higher elicited health state utilities (p < 0.01).ConclusionHaving significant others, or the lack of having significant others, was associated with respondents’ valuation of hypothetical health states using TTO, more so than traditional sampling variables such as age and sex. Inadequate representativeness in terms of having significant others could bias health state preference values in valuation studies.

Population-based utility scores for HPV infection and oropharyngeal squamous cell carcinoma among Indigenous Australians

BackgroundOropharyngeal squamous cell carcinoma (OPSCC) is associated with high mortality. Human papillomavirus (HPV) infection is a significant risk factor for OPSCC. Utilities are fundamental values representing the strength of individuals’ preferences for specific health-related outcomes. Our study aim was to work in partnership with Indigenous communities in South Australia to develop, pilot test and estimate utility scores for health states related to HPV, HPV vaccination, precursor OPSCC and its treatment, and early stage OPSCC among Indigenous Australians.MethodsDevelopment and pilot testing of hypothetical HPV and OPSCC health states, specifically through the lens of being Indigenous Australian, was conducted with an Indigenous Reference Group. Six health states were decided upon, with utility scores calculated using a two-stage standard gamble approach among a large convenience sample of Indigenous Australians aged 18+ years residing in South Australia. The rank, percentage of perfect health and utility score of each health state was summarised using means, and medians at 12 months and lifetime duration. Potential differences by age, sex and residential location were assessed using the Wilcox Rank Sum test.ResultsData from 1011 participants was obtained. The mean utility scores decreased with increasing severity of health states, ranging from 0.91–0.92 in ‘screened, cytology normal, HPV vaccination’ and ‘screened, HPV positive, endoscopy normal’, to less than 0.90 (ranging from 0.87–0.88) in lower grade conditions (oral warts and oral intraepithelial neoplasia) and less than 0.80 (ranging from 0.75–0.79) in ‘early stage throat cancer’. Higher utility scores were observed for ‘screened, cytology normal and HPV vaccination’ among younger participants (18–40 years), for ‘early stage invasive throat cancer’ among females, and for ‘oral intraepithelial neoplasia’ and ‘early stage invasive throat cancer’ among metropolitan-dwelling participants.ConclusionAmong a large sample of Indigenous Australians, utility for oral HPV infection and OPSCC decreased with severity of health states. Older participants, as well as males and those residing in non-metropolitan locations, had decreased utility for high-grade cytology and early invasive cancer states. Our findings are an important contribution to cost-utility and disease prevention strategies that seek to inform policies around reducing HPV infection and OPSCC among all Australians.

Evidence of a disability paradox in patient‐reported outcomes in haemophilia

IntroductionPeople with inherited and long‐term conditions such as haemophilia have been shown to adapt to their levels of disability, often reporting better quality of life (QoL) than expected from the general population (the disability paradox).AimTo investigate the disability paradox in people with haemophilia in the United States by examining preference differences in health state valuations versus the general population.MethodsWe conducted a discrete choice experiment including duration to capture valuations of health states based on patient‐reported preferences. Participants indicated their preferences for hypothetical health states using the EQ‐5D‐5L, where each participant completed 15 of the 120 choice tasks. Response inconsistencies were evaluated with dominated and repeated scenarios. Conditional‐logit regressions with random sampling of the general population responses were used to match the sample of patients with haemophilia. We compared model estimates and derived preferences associated with EQ‐5D‐5L health states.ResultsAfter removing respondents with response inconsistencies, 1327/2138 (62%) participants remained (177/283 haemophilia; 1150/1900 general population). Patients with haemophilia indicated higher preference value for 99% of EQ‐5D‐5L health states compared to the general population (when matched on age and gender). The mean health state valuation difference of 0.17 indicated a meaningful difference compared to a minimal clinically important difference threshold of 0.07. Results were consistent by haemophilia type and severity.ConclusionOur findings indicated the presence of a disability paradox among patients with haemophilia, who reported higher health states than the general population, suggesting the impact of haemophilia may be underestimated if general population value sets are used.

The estimation of health state utility values in rare diseases: overview of existing techniques.

There are several techniques for estimating health state utility values, each of which presents pros and cons in the context of rare diseases (RDs). Direct approaches (e.g. standard gamble and time trade-off) may be too demanding for patients with RDs, since most of them affect young children or cause cognitive impairment. The alternatives are using "vignettes" that describe hypothetical health states for the general public, which may not reflect the heterogeneous manifestations of RDs, or multi-attribute utility instruments (i.e. indirect techniques), such as EQ-5D, which may be less sensitive in capturing the specificities of RDs. The "rule of rescue" approach is a promising alternative in RDs, since it prioritizes identifiable patients with life-threatening or disabling conditions. However, it raises measurement challenges and ethical issues. Furthermore, the literature reports on relevant implications of choosing a technique over others for health technology assessment, which should be considered in relation to individual RDs.

Elicitation of Norwegian EQ-5D-5L values for hypothetical and experience-based health states based on the EuroQol Valuation Technology (EQ-VT) protocol

IntroductionNorway is one of several European countries that lacks a national value set and scoring algorithm for the EuroQol five dimensions (EQ-5D). Recent studies have found differences between countries in...

Similar responses to EQ-5D-3L by two elicitation methods: visual analogue scale and time trade-off

BackgroundHealth-related quality of life (HRQoL) is often measured using EQ-5D-3L by the elicitation methods of visual analogue scale (VAS) and time trade-off (TTO). Although many countries have constructed both national VAS and TTO value sets, the fact that VAS and TTO value sets produces different values bewilders researchers and policymakers. The aim of this study is to explore certain conditions which could yield similar value sets using VAS and TTO.MethodsA homogeneous sample of medical school students was selected to value 18 hypothetical health states using VAS and TTO methods. The 18 hypothetical health states were produced by orthogonal design (L18, 2*3^7). The range of rescaled values was transformed into − 1 ~ 0 ~ 1. The investigations via different methods were carried out by computer-assisted personal interviewing with a wash-time interval of 72 h. Value sets for VAS and TTO were constructed using general least square regression models. Independent variables were composed of 10 dummy variables from 5 dimensions and including or omitting both constant and N3 terms.ResultsThree hundred thirteen medical students participated. The mean age was 21.03 ± 0.44 years and 56.2% were female. The four regression models (for each method with and without constant and N3 terms) were all statistically significant (P < 0.05) with high goodness-of-fit (Adj. R2 > 0.94 and MAE < 0.033). Differences between the coefficients of the 10 dummy variables corresponding to each model were all less than 0.059. Pearson correlation coefficients between observed means and predicted values exceeded 0.981. Fitted curves of VAS and TTO largely coincided.ConclusionsVAS and TTO can generate similar responses under certain conditions, suggesting that the two valuation methods could be equivalent intrinsically. The VAS method appears a more valid approach for valuation in the general population due to its greater simplicity and feasibility.

Estimation of population-based utility weights for health states of chronic kidney disease, dialysis and kidney transplantation.

This study aimed to determine the utility of chronic kidney disease (CKD)-related health states in the Korean general population by using the standard gamble (SG) method. Seven hypothetical CKD-related health states were developed and revised based on previous publications. Data from 242 individuals from the Korean general population were obtained via computer-assisted personal interviews and used to evaluate the CKD-related health states by using the visual analogue scale (VAS) and SG methods. Mean and median utility values were calculated for each health state. To assess the reliability of the valuation, some samples were retested and evaluated using intraclass correlation coefficients (ICC). In all health states, the utility weights from SG were higher than those from VAS. The difference in utility values between the two valuation methods ranged from 0.258 to 0.308. The utility value of CKD grade 3 was the highest and that of peritoneal dialysis (PD) was the lowest. The utility weights tended to decrease in accordance with the progression of CKD. CKD grade 3 was assigned a SG utility value of 0.878, whereas PD was assigned 0.507. The SG utility values of predialysis end-stage renal disease, haemodialysis, kidney transplantation (KT), and post-KT recovery states were 0.666, 0.547, 0.748 and 0.827, respectively. The mean ICC (SD) at the individual level was 0.746 (0.281) for the SG scores. Our results could be used to measure the burden of kidney disease or to evaluate the economic efficiency of CKD-related interventions.