Health State Preference Values Research Articles

A national survey of Iranian general population to estimate a value set for the EQ-5D-5L.

The main aim of this study is to estimate a national value set of the EQ-5D-5L questionnaire for Iran. The composite time trade-off (cTTO) and discrete choice experiment (DCE) methods; and the protocol for EuroQol Portable Valuation Technology (EQ-PVT) were used to estimate the Iran national value set. 1179 face-to-face computer-assisted interviews were conducted with adults that were recruited from five Iran major cities in 2021. Generalized least squares, Tobit, heteroskedastic, logit, and hybrid models were used to analyze the data and to identify the best fitting model. According to the logical consistency of the parameters, significance levels and prediction accuracy indices of the MAE; a heteroscedastic censored Tobit hybrid model combining cTTO and DCE responses was considered as the best fitting model to estimate the final value set. The predicted values ranged from -1.19 for the worst health state (55555) to 1 for full health (11111), with 53.6% of the predicted values being negative. Mobility was the most influential dimension on health state preference values. The present study estimated a national EQ-5D-5L value set for Iranian policy makers and researchers. The value set enables the EQ-5D-5L questionnaire to use to calculate QALYs to assist the priority setting and efficient allocation of limited healthcare resources.

THU421 - A systematic literature review and meta-analysis of primary sources reporting health state preference values in chronic hepatitis B, C, and D

THU421 - A systematic literature review and meta-analysis of primary sources reporting health state preference values in chronic hepatitis B, C, and D

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.

Retraction Note to: The EQ-5D-5L Valuation Study in Egypt.

No value sets exist for either the EQ-5D-3L or the EQ-5D-5L in Egypt, despite local pharmacoeconomic guidelines recommending the use of the EQ-5D to derive utility. Most published Egyptian economic evaluation studies have used utility values from other published studies and systematic reviews. Our objective was to develop an Egyptian EQ-5D-5L value set using the international EuroQol standardized protocol (EQ-VT-2.1). Adult Egyptian participants were recruited from public places using multi-stratified quota sampling based on age, sex, and geographical distribution. Two elicitation techniques were applied: the composite time trade-off (cTTO) and discrete-choice experiments (DCEs). Before actual data collection, interviewers’ performance was assessed in a pilot phase. Data were modelled using generalized least square, Tobit, heteroskedastic, logit, and hybrid models, and the best fitting model was selected based on the value range between observed and predicted values, logical consistency of the parameters, significance level, and prediction accuracy. A total of 1378 interviews were conducted, of which 188 were excluded because they were incomplete or did not comply with protocol, 216 were pilot interviews, and 974 were included in the final analysis. The heteroskedastic model (model 4) based on the cTTO data was selected as the preferred model to generate the value set. Values ranged from − 0.93 for the worst health state (55555) to 1 for full health (11111), with 1136 (36.3%) of all predicted health states being worse than dead. Mobility had the largest impact on health state preference values. This is the first value set for the EQ-5D-5L based on social preferences obtained from a nationally representative sample in Egypt or any Arabic-speaking country. The value set can be used as a scoring system for economic evaluations and to improve the quality of health technology assessment in the Egyptian healthcare system.

HOUT-17. UTILITIES OF RARE CANCERS LIKE MALIGNANT PLEURAL MESOTHELIOMA AND GLIOBLASTOMA MULTIFORME - DO THEY COMPARE?

Abstract BACKGROUND Glioblastoma (GBM) is the most aggressive primary brain cancer in adults. Malignant pleural mesothelioma (MPM) is an invasive and generally fatal malignancy of the pleura mainly caused by exposure to asbestos fibers. TTFields treatment has been shown effective in both MPM and GBM with a comparable increase in median overall survival (OS). Differences in utility values however, could significantly influence assessment of cost-effectiveness for MPM and GBM, despite similar clinical efficacy. METHODS We reviewed the published literature adressing health state preference values or health utilities in both indications using a boolean search of the PubMed database. The results were compiled separately for both diseases and then compared to each other. RESULTS Estimates of health states preference values or utilities in GBM all refer to one single publication by the UK NICE. Healthy members of the NHS Value of Health Panel (VoHP) rated a total of nine descriptive health state scenarios using the standard gamble method for preference elicitation. Utilities for MPM were derived at individual patient level from the EQ-5 questionnaire collected during one trial. Other publications do not use specific mesothelioma heath state preference velues but assume applicability of utilities derived from a non small cell lung cancer (NSCLC) population. CONCLUSIONS GBM utilites use different health states for elicitation, while utilities used in MPM were elicited at patient level. GBM utilities can be used in a health state disease model but there are no similar published values for MPM. MPM utilities using the EQ-5 questionnaire however better describe individual utilities and their change during the course of disease. Despite similar costs and clinical efficacy of TTFields treatment for GBM and MPM, the difference in elicitation methods and sources of utilites may lead to significant disparities in cost effectiveness assessment and subsequent adoption of TTFields treatment in different indications.

CHALLENGES FACED IN TRANSFERRING ECONOMIC EVALUATIONS TO MIDDLE INCOME COUNTRIES

Decision makers in middle-income countries are using economic evaluations (EEs) in pricing and reimbursement decisions for pharmaceuticals. However, whilst many of these jurisdictions have local submission guidelines and local expertise, the studies themselves often use economic models developed elsewhere and elements of data from countries other than the jurisdiction concerned. The objectives of this study were to describe the current situation and to assess the challenges faced by decision makers in transferring data and analyses from other jurisdictions. Experienced health service researchers in each region conducted an interview survey of representatives of decision making bodies from jurisdictions in Asia, Central and Eastern Europe, and Latin America that had at least 1 year's experience of using EEs. Representatives of the relevant organizations in twelve countries were interviewed. All twelve jurisdictions had developed official guidelines for the conduct of EEs. All but one of the organizations evaluated studies submitted to them, but 9 also conducted studies and 7 commissioned them. Nine of the organizations stated that, in evaluating EEs submitted to them, they had consulted a study performed in a different jurisdiction. Data on relevant treatment effect was generally considered more transferable than those on prices/unit costs. Views on the transferability of epidemiological data, data on resource use and health state preference values were more mixed. Eight of the respondents stated that analyses submitted to them had used models developed in other jurisdictions. Four of the organizations had a policy requiring models to be adapted to reflect local circumstances. The main obstacles to transferring EEs were the different patterns of care or wealth of the developed countries from which most economic evaluations originate. In middle-income countries it is commonplace to deal with the issue of transferring analyses or data from other jurisdictions. Decision makers in these countries face several challenges, mainly due to differences in current standard of care, practice patterns, or gross domestic product between the developed countries where the majority of the studies are conducted and their own jurisdiction.

Development of a Preference-Based Index From the National Eye Institute Visual Function Questionnaire–25

Understanding how individuals value health states is central to patient-centered care and to health policy decision making. Generic preference-based measures of health may not effectively capture the impact of ocular diseases. Recently, 6 items from the National Eye Institute Visual Function Questionnaire-25 were used to develop the Visual Function Questionnaire-Utility Index health state classification, which defines visual function health states. To describe elicitation of preferences for health states generated from the Visual Function Questionnaire-Utility Index health state classification and development of an algorithm to estimate health preference scores for any health state. Nonintervention, cross-sectional study of the general community in 4 countries (Australia, Canada, United Kingdom, and United States). A total of 607 adult participants were recruited from local newspaper advertisements. In the United Kingdom, an existing database of participants from previous studies was used for recruitment. Eight of 15,625 possible health states from the Visual Function Questionnaire-Utility Index were valued using time trade-off technique. A θ severity score was calculated for Visual Function Questionnaire-Utility Index-defined health states using item response theory analysis. Regression models were then used to develop an algorithm to assign health state preference values for all potential health states defined by the Visual Function Questionnaire-Utility Index. Health state preference values for the 8 states ranged from a mean (SD) of 0.343 (0.395) to 0.956 (0.124). As expected, preference values declined with worsening visual function. Results indicate that the Visual Function Questionnaire-Utility Index describes states that participants view as spanning most of the continuum from full health to dead. Visual Function Questionnaire-Utility Index health state classification produces health preference scores that can be estimated in vision-related studies that include the National Eye Institute Visual Function Questionnaire-25. These preference scores may be of value for estimating utilities in economic and health policy analyses.

Utility values for specific hepatic encephalopathy health states elicited from the general public in the United Kingdom.

Background and aimsTo elicit utility values for five health states corresponding to increasing severity of hepatic encephalopathy, from members of the general public in the UK. The health states studied were Conn grades 0, 1, 2, 3 and 4.MethodsInterviewer-administered time trade-off (TTO) and standard gamble (SG) utilities were elicited for the five health states from a random sample of 200 members of the general public in the UK, using health state descriptions validated by clinicians and members of the general public.ResultsRespondents’ mean age was 49.5 years and 49% were female. Mean utilities were 0.962 (TTO) and 0.915 (SG) for Conn grade 0; 0.912 (TTO) and 0.837 (SG) for Conn grade 1; 0.828 (TTO) and 0.683 (SG) for Conn grade 2; 0.691 (TTO) and 0.489 (SG) for Conn grade 3; and 0.429 (TTO) and 0.215 (SG) for Conn grade 4. The TTO and SG values between the five Conn grades were significantly different (p < 0.001). Additionally, the TTO value was significantly higher than the SG value for the corresponding state (p <0.0001).ConclusionThese findings quantify how different Conn grades and level of response to treatment may impact on the health-related quality of life of patients with hepatic encephalopathy. There were greater preference values for lower levels of disease, with the highest value associated with Conn grade 0. These health state preference values can be used to estimate the outcomes of different interventions for hepatic encephalopathy in terms of quality-adjusted life years.

Preference values associated with stage III colon cancer and adjuvant chemotherapy

To elicit preference values for health states associated with Stage III colon cancer (CRC) and to explore the effect of neuropathy associated with current adjuvant treatment. We used time trade-off (TTO) techniques to elicit preferences from 49 CRC patients and 49 community members. We elicited preferences for 7 health states: remission; adjuvant therapy with no, mild, moderate, and severe neuropathy; metastatic stable; and metastatic progressive disease. Mean TTO values were adjusted for the covariates age, education, and current health. Patients' adjusted mean TTO value for remission was 0.83; adjuvant chemotherapy health states ranged from 0.48 to 0.61. Significant differences were observed for both patient and community groups between TTO for remission and all adjuvant health states (P < 0.001), and between adjuvant therapy with no neuropathy and metastatic health states (P < or = 0.001). Across all health states, patients' values were on average 0.12 higher than community members (P < 0.05). The findings highlight the trade-offs between the disutility of adjuvant treatment, the higher utility of remission, and the severe utility loss during metastatic disease. The preference values obtained from this study will be useful for informing patients' treatment decisions and payer cost-utility analyses of adjuvant treatment for colon cancer.

A Multinational Study of Health State Preference Values Associated with Chronic Myelogenous Leukemia

Objectives Chronic myelogenous leukemia (CML) is a progressive, largely fatal cancer. Emerging treatments may prolong life; however, these result in additional monetary costs. Accurate estimation of their economic impact requires reliable estimates on preferences for health states. The purpose was to estimate preference weights from the general population in four developed countries for standardized health states experienced by persons with CML. Methods Time trade-off preferences with a 10-year time horizon were elicited for CML-related health states using an interviewer-administered survey from convenience samples in Canada (n = 103), the United States (n = 74), the UK (n = 97), and Australia (n = 79). Standardized descriptions of seven CML-related health states (characterizing chronic, accelerated and blast phases, each with responding and nonresponding state, and adverse events of treatment) were derived in consultation with oncologists. Generalized linear models were used to estimate whether utilities, adjusted for age and sex, differed by country. Results The mean age of the sample (n = 357) was 45 years and 46% were male. Mean unadjusted preference values of CML-related health states ranged from 0.84 for “Chronic phase responding to treatment” to 0.21 for “Blast phase, not responding to treatment.” For each phase, preferences were lower for the nonresponding state. After adjustment for age and sex, considerable variability was observed in mean preference values between countries. Conclusion These data quantify the deteriorating impact of CML disease progression and the impact of nonresponse to treatment. The study results add to evidence from other disease areas that systematic differences exist in preference values between countries.

Preferences of Patients with Diabetes Mellitus for Inhaled versus Injectable Insulin Regimens

In clinical trials, patients have expressed greater satisfaction with inhaled human insulin (EXUBERA, Pfizer) than with injectable insulin. No studies to date have attempted to quantify the strength of preferences for these alternative routes of administration. To elicit health state preference values from people with diabetes mellitus for treatment with inhaled human insulin compared with injectable insulin. A patient preference study. Written descriptions were developed for five clinical scenarios: two for type 1 diabetes and three for type 2 diabetes. Each scenario required adjustment or initiation of insulin treatment because of poor glycaemic control. Two alternative insulin regimens were described for each scenario: injectable-only or inhaled human insulin to replace or reduce the number of daily injections. Equal efficacy was assumed within each of these scenario pairs.A total of 344 UK adults (66% male), 132 (mean age 49 years) with type 1 diabetes and 212 (mean age 63 years) with type 2 diabetes, rated scenario pairs corresponding to their own type of diabetes and rated their own health by time trade-off (TTO), by correspondence with EQ-5D health descriptions and on the EQ-5D visual analogue scale. Respondents stated their preference for, or indifference between, the injection-only or inhalation variant comprising each scenario pair. TTO utilities and EQ-5D utilities by UK community tariff were compared within each scenario pair, for the total sample rating, each scenario pair, and by subgroups of stated preference for each variant. A majority, ranging from 63% to 81% across the scenarios, preferred inhalation. Mean differences in TTO scores were 0.074, 0.076, 0.088, 0.053 and 0.043 for the five scenarios, respectively (p < 0.005 for all). Mean EQ-5D differences were 0.043, 0.029, 0.037, 0.020 and 0.021 for the five scenarios, respectively (p < 0.05 for scenarios 1 and 3), driven mainly by differences on the pain/discomfort dimension of the EQ-5D. Differences in favour of inhalation among those preferring inhalation, were greater than differences in favour of injections among those preferring injections. Mean self-rated health was similar between respondents with type 1 and type 2 diabetes, at 0.83 (TTO) and 0.75 (EQ-5D). The TTO was more sensitive than EQ-5D. Self-rated health by EQ-5D compared closely with reported values from the UK Prospective Diabetes Study (UKPDS). This study highlights the utility differences that people with diabetes perceive between the prospect of inhaled and injected routes of insulin administration, even under the assumption of no difference in efficacy. These differences are magnified when the comparison in utility scores is between the majority who prefer the inhaled route and the minority who prefer the injectable route.

Is There Value in Obtaining a Patient’s Willingness to Pay for a Particular Anesthetic Intervention?

Is There Value in Obtaining a Patient’s Willingness to Pay for a Particular Anesthetic Intervention?

Feasibility, validity and test-retest reliability of scaling methods for health states: the visual analogue scale and the time trade-off.

The feasibility, validity and reliability of the Time Trade-Off (TTO) and Visual Analogue Scale (VAS) methods in obtaining preference values for health states were compared in a random sample of the Spanish population (n = 294). Respondents valued 43 EuroQol-5D health states in face-to-face interviews. Convergent validity was assessed by examining the relationship between values, and the effect of sociodemographic and health variables on values was used as a means of assessing construct validity. Test-retest reliability was analysed in a subgroup of 50 respondents, using the intraclass correlation coefficient (ICC) and generalisability theory. Rates of non-response and missing data were low on both methods, though the VAS took considerably less time to administer. VAS and TTO values correlated highly (r = 0.92), though there were differences in the ordering of health states between methods, and in the number of health states rated worse than death. VAS values were compressed into a considerably smaller valuation space than TTO values. Respondents in higher educational categories assigned higher TTO values to 12 health states. Mean ICCs (95% CI) at individual level were 0.90 (0.88-0.92) and 0.84 (0.81-0.87) for the VAS and TTO, respectively. Generalisability analysis showed variance due to time to be 0 for both methods. In conclusion, the VAS was more feasible and slightly more reliable than the TTO, whilst doubt can be cast on the degree of convergent validity existing between the two methods. The compression of VAS values means that the TTO is likely to discriminate better between health states, and it may have greater construct validity if results from larger samples confirm that there are genuine differences between sociodemographic subgroups.

Constructing health state preference values from descriptive quality of life outcomes: mission impossible?

Descriptive quality of life questionnaires are commonly administered in clinical trials, to evaluate outcomes from the patient's perspective alongside conventional clinical measures. When expressed in single index form as health state preference values (HSPVs), quality of life information is also relevant to economic evaluations. By combining HSPVs with survival information, quality adjusted life years (QALYs) may be derived for cost-utility analysis. Although HSPVs are rarely measured prospectively in cancer clinical trials, the UK Medical Research Council Cancer Therapy Committee recommends the routine administration of two specific quality of life questionnaires: the Rotterdam Symptom Checklist and the Hospital Anxiety Depression Scale. This study explores two potential methods for secondary derivation of HSPVs from these instruments, using data gathered in a clinical trial of two forms of radiotherapy for non-small cell cancer of the bronchus. The first method, secondary mapping to existing utility scales, was found to be infeasible from the above questionnaires. The second method used factor analysis to summarize the descriptive quality of life data collected through the questionnaires. This revealed five distinct factors prevalent in the trial population. Using these factors, simplified health state scenarios were developed from which direct measurement of HSPVs was feasible. As the resulting HSPVs and any QALYs that may be derived from them are cancer specific, their potential value in informing resource allocation would be limited to decisions within oncology services.

Pneumocystis Prophylaxis for All, Some, or No HIV-infected Infants Less than One Year of Age: A Decision Analysis Approach

Pneumocystis carinii pneumonia (PCP) is associated with significant mortality and morbidity among infants infected with human immunodeficiency virus (HIV). The preferred prophylaxis strategy for such infants is a subject of debate. Medical decision analysis was used to determine the preferred strategy for primary PCP prophylaxis among asymptomatic HIV-infected infants less than one year of age, and to determine the thresholds at which different variables influence decision making. Utility measures (health state preference values) were used to determine whether prophylaxis should be given to all, some or no infants. In this regard, some infants would receive prophylaxis if baseline CD4 counts are fewer than 1500 cells/mm(3). The results suggest that the preferred option is to give prophylaxis to all asymptomatic HIV-infected infants despite CD4 counts, if the risk of PCP is equal to or greater than 25%. However, if the risk of PCP is less than 25%, prophylaxis is recommended for those infants with CD4 counts of fewer than 1500 cells/mm(3). The results complement current guidelines regarding PCP prophylaxis for HIV-infected infants.