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Italian Population-Based Values of EQ-5D Health States
Abstract
Objective: To estimate a value set for the calculation of Italian-specific quality-adjusted life years (QALYs), based on preferences elicited on EuroQol five-dimensional (EQ-5D) questionnaire health states using the time trade-off technique. Methods: The revised standard Measurement and Valuation of Health protocol was followed. Twenty-five health states, divided into three groups and given to 450 subjects, were selected to obtain 300 observations per state. Subjects aged 18 to 75 years were recruited to be representative of the Italian general adult population for age, sex, and geographical distribution. To improve efficiency, face-to-face interviews were conducted by using the Computer Assisted Personal Interviewing approach. Several random effects regression models were tested to predict the full set of EQ-5D questionnaire health states. Model selection was based on logical consistency of the estimates, sign and magnitude of the regression coefficients, goodness of fit, and parsimony. Results: The model that satisfied the criteria of logical consistency and was more efficient includes 10 main effect dummy variables for the EQ-5D questionnaire domain levels and the D1 interaction term, which accounts for the number of dimensions at levels 2 or 3 beyond the first. This model has an R 2 of 0.389 and a mean absolute error of 0.03, which are comparable to or better than those of models used in other countries. The utility estimates after state 11111 range from 0.92 (21111) to −0.38 (33333). Italian utility estimates are higher than those estimated in the United Kingdom and Spain and used so far to assess QALYs and conduct cost-utility evaluations in Italy. Conclusions: A specific value set is now available to calculate QALYs for the conduction of health economic studies targeted at the Italian health care system.
... For example, an observational study used EQ-5D-3L in a large group of cancer patients treated in Italian hospitals (n = 802), obtaining a mean (± SD) EQ VAS score of 71.5 (± 17.38), i.e., 10 points lower than in this study for the general population (81.8 ± 13.5), and a mean (± SD) utility index value of 0.86 (± 0.13), compared with 0.93 (± 0.11) in our study population [50]. However, EQ-5D index values are not fully comparable since they were obtained using the 3L algorithm [51]. ...
... The mean EQ VAS score (81.8 ± 13.5) in this study is lower than the value (84.8 ± 13.8) obtained in the previous instrument version (EQ-5D-3L) Italian valuation study, which, however, had a younger study sample (mean age 46.6 ± 15.3) than in the current study (48.3 ± 16.1 years), since participants were recruited up to a maximum of 75 years [51]. Conversely, in a more recent survey conducted by telephone in Lombardy, the mean EQ VAS score was lower (78.2 ± 18.4) than in our study, as well as the mean EQ-5D-5L index value (0.915 ± 0.10) obtained using a mapping algorithm from 3L values [52]. ...
Objectives:
This study aimed to provide normative data obtained in response to the EQ-5D-5L questionnaire in Italy and compare this with data from other countries.
Methods:
A sample of the Italian adult population (aged ≥ 18 years) was recruited and interviewed online using videoconferencing software (Zoom) between November 2020 and February 2021. The distribution of answers was estimated as per the descriptive system of the EQ-5D-5L, and descriptive statistics were calculated for the EQ VAS score and EQ-5D-5L index value in the whole sample and relevant subgroups. An ordinary least square (OLS) regression was performed to evaluate the impact of sociodemographic variables on EQ-5D-5L results. Lastly, a comparison was made with EQ-5D-5L population norms of other countries. Data analysis was performed using Microsoft Excel and Stata 13.
Results:
Overall, 1182 people representative of the Italian population (2020) in terms of sex and geographical area responded to the survey. Of the 3125 potential EQ-5D-5L health states, only 106 (3.4%) were selected, and the '11111' and '11112' states were chosen by half of the participants. In terms of EQ-5D-5L dimensions, the frequency of any problems (from slight to extreme) associated with anxiety and depression was high among the very young (18-24 years, 56.0%) and in women of all ages (49.7%). The mean index value (± standard deviation [SD]) was 0.93 (± 0.11) for the entire sample and gradually decreased with age, moving from 0.95 (± 0.06) in the youngest group (18-24 years) to 0.91 (± 0.13) in the oldest age group (≥ 75 years). Similarly, the mean EQ VAS score (± SD) was 81.8 (± 13.5), and decreased from 87.0 (± 8.9) in the 18-24 years age group to 75.1 (± 16.4) among participants > 75 years of age. The existence of self-reported chronic conditions (e.g., cardiovascular disease), female sex, and social assistance recipiency were negatively associated with the EQ-5D index value, while the EQ VAS score was significantly lower in people with chronic conditions and aged > 55 years. Conversely, higher income levels had a positive impact on both the EQ-5D index value and the EQ VAS score. Lastly, both the EQ-5D index value and EQ VAS score in Italy were, on average, higher than in most European countries.
Conclusions:
EQ-5D-5L population norms provide useful insights into the health status of the Italian population and can be used as a reference for other surveys using the same instrument.
... Quality of Life was assessed by the standardized, generic EQ-5D questionnaire 11,12 . A summary weighted health utility score is calculated using country specific weights derived from a sample of the general population (in this case Italian time-trade-off values were used 13 ...
... To the best of our knowledge this was the first study in Italy to measure the impact of HZ on QoL by using the EQ-5D to measure disutility. We estimated a mean disutility of 0.134 during the first month of the HZ episode (equivalent to QALY loss of 0.011 per month) using the recently published Italian timetrade-off values 13 . This corresponds to losing approximately 4.1 days of perfect health per month of HZ. ...
Background
To estimate the health care resource utilisation, costs and impact on quality of life (QoL) of herpes zoster (HZ) and postherpetic neuralgia (PHN) in adults aged ≥50 years in Italy.
Methods
This was a prospective, observational, multi-centre, community-physician based surveillance study (NCT01772160) in Italy. Health related QoL data were collected using the EuroQoL-5 Dimension (EQ-5D) and zoster brief pain inventory (ZBPI) questionnaires. Both questionnaires were assessed at days 0 (HZ rash onset),15, 30, 60 and 90 for all patients and monthly thereafter for patients who developed PHN. Resource utilization was recorded for 3 months post HZ onset and for 9 months for PHN patients. Costs from both payer and societal perspectives were estimated and were composed of direct medical costs (general practitioner/specialist visits, procedures, hospitalisations, medications), work loss by patient/caregiver and transport costs.
Results
391 patients with HZ were included of whom 40 developed PHN. The mean ZBPI worst pain score was 5.7 at day 0, reducing to 2.6 at day 30 and 0.7 by day 90. Patients with PHN had a mean worst pain score of 5.7 at day 90. We estimated an overall disutility associated with HZ of 0.134. The mean cost per HZ patient from a payer/societal perspective was €153/298 respectively, and the mean cost per HZ patients who developed PHN was €176/426 respectively.
Conclusions
HZ is associated with impaired QoL and substantial health care resource use highlighting the need for preventive strategies. This could reduce the disease burden for the patient and health care system.
... Prior to developing our own CAPI app, we reviewed the literature to assess whether existing software could serve our purpose. We found 13 TTO studies that reported using CAPI software during in-person interviews: 7 studies used the EQ-VT [11,12,[17][18][19][20][21] and 6 did not describe the specific software [22][23][24][25][26][27]. Of these 6 studies, one [23] cited a previous study [28] as the source of the CAPI software. ...
... The previous study contained screenshots of the software but no technical details. Another 2 of the 6 studies reported using hard-copy visual aids to conduct the TTO task, with CAPI software reserved for data entry only [24,25]. A total of 22 other studies used Internet-based CAPI software, where participants logged onto websites and completed the TTO task on their own, without an interviewer present [22,. ...
BACKGROUND
The time trade-off (TTO) task is a method of eliciting health utility scores, which range from 0 (equivalent to death) to 1 (equivalent to perfect health). These scores numerically represent a person’s health-related quality of life. Software apps exist to administer the TTO task; however, most of these apps are poorly documented and unavailable to researchers.
OBJECTIVE
To fill the void, we developed an online app to administer the TTO task for a research study that is examining general public proxy health-related quality of life estimates for persons with Alzheimer’s disease. This manuscript describes the development and pretest of the app.
METHODS
We used Research Electronic Data Capture (REDCap) to build the TTO app. The app’s modular structure and REDCap’s object-oriented environment facilitated development. After the TTO app was built, we recruited a purposive sample of 11 members of the general public to pretest its functionality and ease of use.
RESULTS
Feedback from the pretest group was positive. Minor modifications included clarity enhancements, such as rearranging some paragraph text into bullet points, labeling the app to delineate different question sections, and revising or deleting text. We also added a research question to enable the identification of respondents who know someone with Alzheimer’s disease.
CONCLUSIONS
We developed an online app to administer the TTO task. Other researchers may access and customize the app for their own research purposes.
... Prior to developing our own CAPI app, we reviewed the literature to assess whether existing software could serve our purpose. We found 13 TTO studies that reported using CAPI software during in-person interviews: 7 studies used the EQ-VT [11,12,[17][18][19][20][21] and 6 did not describe the specific software [22][23][24][25][26][27]. Of these 6 studies, one [23] cited a previous study [28] as the source of the CAPI software. ...
... The previous study contained screenshots of the software but no technical details. Another 2 of the 6 studies reported using hard-copy visual aids to conduct the TTO task, with CAPI software reserved for data entry only [24,25]. A total of 22 other studies used Internet-based CAPI software, where participants logged onto websites and completed the TTO task on their own, without an interviewer present [22,. ...
... The EQ-5D-3L index score was computed using published Italian tariffs. 33 ...
... The EQ-5D-3L mean index score was 0.91 (SD = 0.12) when computed with published Italian EQ-5D tariffs. 33 This mean is slightly higher than most scores published previously for patients with T2D without complications, 34 likely because the Italian tariffs yield higher index scores than the more commonly used UK tariffs. ...
Objectives
Several glucagon-like peptide-1 receptor agonists are administered as weekly injections for treatment of type 2 diabetes (T2D). These medications vary in their injection processes, and a recent study in the UK found that these differences had an impact on patient preference and health state utilities. The purpose of this study was to replicate the UK study in Italy to examine preferences of an Italian patient sample, while allowing for comparison between utilities in the UK and Italy.
Materials and methods
Participants with T2D in Italy valued health states in time trade-off interviews. All health states had the same description of T2D, but differed in description of the treatment process. As in the original UK study, the first health state described an oral treatment regimen, while additional health states added a weekly injection. The injection health states differed in three injection-related attributes: requirements for reconstituting the medication, waiting during medication preparation, and needle handling.
Results
Interviews were completed by 238 patients (58.8% male; mean age = 60.2 years; 118 from Milan, 120 from Rome). The oral treatment health state had a mean (SD) utility of 0.90 (0.10). The injection health states had significantly (p < 0.0001) lower utilities, which ranged from 0.87 (requirements for reconstitution, waiting, and handling) to 0.89 (weekly injection with none of these requirements). Differences in health state utility scores suggest that each administration requirement was associated with a disutility (ie, negative utility difference): −0.006 (reconstitution), −0.006 (needle handling), −0.011 (reconstitution, needle handling), and −0.022 (reconstitution, waiting, needle handling).
Conclusion
Disutilities associated with the injection device characteristics were similar to those reported with the UK sample. Results suggest that injection device attributes may be important to some patients with T2D, and it may be useful for clinicians to consider these attributes when choosing medication for patients initiating these weekly treatments.
... With the EQ-5D, the respondents were asked about their HRQoL on the current day. The tool consists of two parts: I. a descriptive system (EQ-5D profile) consisting of three levels of severity per domain ('no problem', 'some/moderate problems', 'extreme problems/impossible to do') and II. a visual analogue scale (EQ-5D VAS) measuring the overall HRQoL, ranging from 0 (worst imaginable health state) to 100 (best imaginable health state) [10]. The results from the EQ-5D descriptive system can be convert to a utility index, measuring the value associated to the health state described by the patients with the EQ-5D domains. ...
... The results from the EQ-5D descriptive system can be convert to a utility index, measuring the value associated to the health state described by the patients with the EQ-5D domains. Utility index was calculated using the Italian specific social tariffs [10] and ranging from − 0.38 (severe problems in all EQ-5D domains) to 1.00 (no problem in all EQ-5D domains). ...
Background:
Autoimmune hepatitis, primary biliary cholangitis, and primary sclerosing cholangitis represent the three major autoimmune liver diseases (AILDs). Their management is highly specialized, requires a multidisciplinary approach and often relies on expensive, orphan drugs. Unfortunately, their treatment is often unsatisfactory, and the care pathway heterogeneous across different centers. Disease-specific clinical outcome indicators (COIs) able to evaluate the whole cycle of care are needed to assist both clinicians and administrators in improving quality and value of care. Aim of our study was to generate a set of COIs for the three AILDs. We then prospectively validated these indicators based on a series of consecutive patients recruited at three tertiary clinical centers in Lombardy, Italy.
Methods:
In phase I using a Delphi method and a RAND 9-point appropriateness scale a set of COIs was generated. In phase II the indicators were applied in a real-life dataset.
Results:
Two-hundred fourteen patients were enrolled and followed-up for a median time of 54 months and the above COIs were recorded using a web-based electronic medical record program. The COIs were easy to collect in the clinical practice environment and their values compared well with the available natural history studies.
Conclusions:
We have generated a comprehensive set of COIs which sequentially capture different clinical outcome of the three AILDs explored. These indicators represent a critical tool to implement a value-based approach to patients with these conditions, to monitor, compare and improve quality through benchmarking of clinical performance and to assess the significance of novel drugs and technologies. This article is part of a Special Issue entitled: Cholangiocytes in Health and Diseaseedited by Jesus Banales, Marco Marzioni, Nicholas LaRusso and Peter Jansen.
... Pediatr Blood Cancer based on a variety of generic questionnaires used to assess the Health related quality of life (HRQoL) of the patients and transform it into a utility value-based on the preferences that the general population reported to the different health states described by the questionnaires. [1,7] The generic quality of life questionnaire most recommended by HTA agencies to assess utility values is the EQ-5D; [4,7] however, other validated questionnaires are both used and accepted by HTAs agencies (e.g., the Health Utilities Index (HUI) and the SF 6D). ...
... Pediatr Blood Cancer based on a variety of generic questionnaires used to assess the Health related quality of life (HRQoL) of the patients and transform it into a utility value-based on the preferences that the general population reported to the different health states described by the questionnaires. [1,7] The generic quality of life questionnaire most recommended by HTA agencies to assess utility values is the EQ-5D; [4,7] however, other validated questionnaires are both used and accepted by HTAs agencies (e.g., the Health Utilities Index (HUI) and the SF 6D). ...
The clinical benefits of prophylaxis in patients with hemophilia are well-established and include the following: reduced bleeding episodes, prevention of joint damage, decreased inhibitor development, and improved health-related quality of life. However, the cost-effectiveness of prophylaxis is still not clear.
We reviewed the published hemophilia prophylaxis economic models focusing on utility assumptions.
We found six cost-utility studies that compared prophylaxis and on-demand regimens. These studies reported remarkably different results, using utility values based on different assumptions and data sources.
We suggest that cooperation among key stakeholders (clinicians, patient organizations and health-care decision makers) as a means of collecting evidence-based and experiential data to represent both the utility and the quality of life changes for patients with Hemophilia A who are treated with prophylaxis or receive on-demand treatments may represent a winning strategy with which to resolve the outstanding issues related to health technology assessments in the care of patients with hemophilia. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.
© 2015 Wiley Periodicals, Inc.
... EORTC QLU-C10D and EQ-5D-3L scores were calculated in accordance with the respective valuation studies. Here, we used the utility decrements of Australia [46,54], Canada [47,55], Italy [43,56], the Netherlands [44,57], Poland [43,58], and the United Kingdom [49,59]. ...
Cost-utility analysis generally requires valid preference-based measures (PBMs) to assess the utility of patient health. While generic PBMs are widely used, disease-specific PBMs may capture additional aspects of health relevant for certain patient populations. This study investigates the construct and concurrent criterion validity of the cancer-specific European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Utility-Core 10 dimensions (QLU-C10D) in non-small-cell lung cancer patients.
We retrospectively analysed data from four multicentre LUX-Lung trials, all of which had administered the EORTC Quality of Life Questionnaire (QLQ-C30) and the EQ-5D-3L. We applied six country-specific value sets (Australia, Canada, Italy, the Netherlands, Poland, and the United Kingdom) to both instruments. Criterion validity was assessed via correlations between the instruments’ utility scores. Correlations of divergent and convergent domains and Bland-Altman plots investigated construct validity. Floor and ceiling effects were assessed.
The comparison of the EORTC QLU-C10D and EQ-5D-3L produced homogenous results for five of the six country tariffs. High correlations of utilities (r > 0.7) were found for all country tariffs except for the Netherlands. Moderate to high correlations of converging domain pairs (r from 0.472 to 0.718) were found with few exceptions, such as the Social Functioning–Usual Activities domain pair (max. r = 0.376). For all but the Dutch tariff, the EORTC QLU-C10D produced consistently lower utility values compared to the EQ-5D-3L (x̄ difference from − 0.082 to 0.033). Floor and ceiling effects were consistently lower for the EORTC QLU-C10D (max. 4.67% for utilities).
The six country tariffs showed good psychometric properties for the EORTC QLU-C10D in lung cancer patients. Criterion and construct validity was established. The QLU-C10D showed superior measurement precision towards the upper and lower end of the scale compared to the EQ-5D-3L, which is important when cost-utility analysis seeks to measure health change across the severity spectrum.
... Country tariffs of both measures were calculated according to previously published algorithms (AUS (32,42 ), CAN (33,43 ), ITA (34,44 ), NLD (35,45 ), POL (34,46 ), UK (36, 47 )). ...
Objectives
The Quality of Life (QoL) Utility measure, QLU-C10D, is derived of the European Organisation for Research and Treatment of Cancer (EORTC) QoL Questionnaire, QLQ-C30. Based on the cancer-specific nature, the QLU-C10D is expected to be sensitive and responsive in lung cancer patients.
Methods
This retrospective analysis used data from four lung cancer multi-center trials. Clinical validity was assessed in comparison to a generic standard utility instrument, the EuroQoL Group´s EQ-5D-3L. Utilities for six country tariffs (Australia, Canada, Italy, the Netherlands, Poland, UK) were calculated at baseline and end of treatment. Country tariff pairs of both measures (k) were compared in terms of Relative Efficiency (RE) and difference in Effect Sizes (dES) of significant t-tests (Bonferroni-Holm correction) in 1) sensitivity to detect differences between performance status groups and 2) responsiveness to changes.
Results
ad 1) Sensitivity indices favored the QLU-C10D (k = 18, p ≤ 0.019; RE > 1.10; dES > 0.03). ad 2) Responsiveness indices of differences between adverse events groups favored the QLU-C10D (k = 6, p ≤ .006; RE > 1.0, dES > 0.02). Responsiveness indices of changes within clinically known groups (k = 78), largely favored QLU-C10D (k = 74, p ≤ .024; RE > 1.01; dES > 0.02). In summary, 96% of the comparative indices favored the QLU-C10D.
Conclusions
Our study confirms the clinical validity of the QLU-C10D in lung cancer patients. The QLU-C10D produced homogenous results across six country tariffs and detected differences/changes in alignment with clinical expectations. In most comparisons the QLU-C10D was more sensitive and responsive compared to the EQ-5D-3L.
... The EQ-5D was completed by the children or by their parents. The algorithm proposed by Scalone et al. [8] describes the translation of the EQ-5D-3L results in quality-adjusted life years (QALYs) specific to the Italian population. The second part of the questionnaire consists of a graduated scale from 0 to 100 (Visual Analogue Scale, VAS), on which the respondent indicated his/her perceived overall health status. ...
Purpose:
The aim of this study was to produce evidence on quality of life (QoL) among Italian growth hormone deficiency (GHD) children and adolescents treated with growth hormone (GH) and their parents.
Methods:
A survey was conducted among Italian children and adolescents aged 4-18 with a confirmed diagnosis of GHD and treated with GH therapy and their parents. The European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3L) and the Quality of Life in Short Stature Youth (QoLISSY) questionnaires were administered between May and October 2021 through the Computer-Assisted Personal Interview (CAPI) method. Results were compared with national and international reference values.
Results:
The survey included 142 GHD children/adolescents and their parents. The mean EQ-5D-3L score was 0.95 [standard deviation (SD) 0.09], while the mean visual analogue scale (VAS) score was 86.2 (SD 14.2); the scores are similar to those of a reference Italian population aged 18-24 of healthy subjects. As for the QoLISSY child-version, compared to the international reference values for GHD/ idiopathic short stature (ISS) patients, we found a significantly higher score for the physical domain, and lower scores for coping and treatment; compared to the specific reference values for GHD patients, our mean scores were significantly lower for all domains except the physical one. As for the parents, we found a significantly higher score for the physical domain, and a lower score for treatment; compared to reference values GHD-specific, we found lower score in the social, emotional, treatment, parental effects, and total score domains.
Conclusions:
Our results suggest that the generic health-related quality of life (HRQoL) in treated GHD patients is high, comparable to that of healthy people. The QoL elicited by a disease specific questionnaire is also good, and comparable with that of international reference values of GHD/ISS patients.
... In our population, QoL was found to be 85% (± 0.143), lower than the cut-off reported in literature of 89.7% from a general Spanish population [12] but close to other reported cut-off of 84.8 (+/-13.8)% from a multicenter cohort of general Italian population [13] representing all ages in a pre-pandemic period. ...
Background
Elderly people living with HIV show a significant prevalence of multimorbidity, polypharmacy and frailty that increase the risk of disability. Telehealth has been suggested as a new tool to monitor people living with HIV in the COVID era, but its effectiveness in elderly is unknown. The aim of this study was two-fold: to explore feasibility of a telephone interview and its capability to collect relevant geriatric outcomes.
Methods
Assessed health domains included comorbidities, falls, urinary incontinence, antiretroviral drugs exposure and comedications (polypharmacy), and the following patient reported outcomes: quality of life, intrinsic capacity, and resilience.
Results
214 (70.6%) answered and completed the interview. During confinement period, 57 (26.7%) of people switched antiretroviral therapy : 119 (55.4%) to dual therapy regimens and 95 (44.6%) to triple regimens. Prevalence of geriatric syndromes were falls in 31 cases (14.7%), urinary incontinence in 48 cases (22.7%) and polypharmacy 122 cases (57.2%). Mean Health-related Quality of Life score was mildly impaired (0,88%) with good concordance of Helath-related Quality of Life self-perception in a visual analogue scale (8/10) (r=+0.348; p <0.01). Intrinsic capacity depicted impaired functional ability in multiple domains (0.737 ) and Resilience (CD-RISC) was suboptimal (0.6).
Conclusions
A structured telephone call was feasible in elderly people living with HIV and allowed to collect clinically meaningful geriatric health domains when face-to-face visits are not needed or discouraged.
... For the estimation of the utility coefficients for the health states of the model, the Italian algorithm has been applied [40] to EuroQol 5D-5L data collected during the TRANS-FORM-2 study [14]. Values obtained with the EuroQol questionnaire were 0.899 (SD 0.075) for responders, 0.918 (SD 0.070) for remitters and 0.760 (SD 0.142) for non-responders (Table 1). ...
Aim:
Major depressive disorder is considered one of the most frequent diseases in the general population, and treatment-resistant depression (TRD) represents the subset with more significant clinical and social impact. Large, robust phase III studies have shown safety and efficacy of esketamine nasal spray plus SSRI/SNRI antidepressants (ADs) compared with SSRI/SNRI plus placebo nasal spray in patients with TRD. The main aim of this study was to perform a cost-utility analysis comparing esketamine plus ADs with ADs alone in TRD patients, from the societal perspective in Italy. A secondary analysis focused on the National Healthcare Service (NHS) perspective.
Methods:
A Markov multistate model has been developed to estimate quality-adjusted life years and economic outcomes of both treatment strategies over 5 years considering the initiation of esketamine in the different treatment lines, from 3 to 5 (3L-5L). The model has been populated with data from literature and real-world evidence. The analysis from the societal perspective considered direct healthcare costs and patients' productivity losses. In addition to the incremental cost-utility ratio (ICUR), the incremental net monetary benefit (INMB) has been calculated as (incremental benefit × WTP) - incremental cost and by applying a willingness-to-pay (WTP) of 50,000€/QALY. Deterministic and probabilistic sensitivity analyses have been performed to assess the robustness of the model results.
Results:
From the societal perspective, the ICUR ranged between 16,314€ and 22,133€ per QALY according to the different treatment lines, while it was over the threshold of 100,000€/QALY for the NHS perspective. The INMB was positive and ranged from 2259€ to 2744€ across treatment lines in the societal perspective; the INMB begins to occur earlier when moving towards subsequent lines of treatment (3.9 years for 3L, 3.6 years for 4L and 3.5 years for 5L). The analyses showed also that the advantage in terms of INMB is maintained for a wide range of societal preferences expressed by WTP thresholds, and in particular for values above 22,200€, 16,400€ and 17,100€ for 3L, 4L and 5L, respectively.
Conclusion:
The study showed that esketamine may be a cost-effective opportunity from the societal perspective for the management of patients with treatment-resistant depression. In the future, data collected from observational studies or registries, which can include the collection of productivity losses and also costs sustained by the patients, will be able to provide further evidence in order to improve the reliability of the model results.
... The results of the SF-12 questionnaire were evaluated according the Physical Component Summary (PCS) index, which reflects the physical health of the subjects, and the Mental Component Summary (MCS) index, regarding the mental health. The results of the EQ-5D questionnaire were analyzed globally, using the EQ-5D index score based on the values from the Italian population [38], but also focusing on single domains. The results were compared to the scores reported in the book by Szende et al. about the international Self-Reported Population Health, based on the EQ-5D [42]. ...
Background
Idiopathic normal pressure hydrocephalus (iNPH) is a potentially reversible disease. Surgical results have been well described in the literature, but only a few studies investigated the subjective outcome. This study aimed to investigate the patient’s expectations about surgery, the perceived improvement after treatment, and its impact on the quality of life (QoL).
Methods
A new dedicated survey was created to investigate subjectively different aspects of the treatment pathway of iNPH (diagnosis, symptoms, expectations from surgery, surgical operation, surgical results, and postoperative QoL), together with the SF-12 and EQ-5D as validated, standardized tools.
Results
Forty-five patients were included. Forty-three percent of cases received the diagnosis after at least 1 year, with symptoms worsening in 73%, and frustration in 93%. Reaching a diagnosis was important for 100% of patients, with high expectations from surgery; 86% of them hoped to return to a normal life. Seventy-two percent of patients reported a significant postoperative improvement (walking 68%, mood 57%). Memory and incontinence did not improve in 64% of cases. Subjectively, QoL improved in 72% of cases. The SF-12 score is comparable to controls >75 years, but lower than the 65–75 years group. The EQ-5D index was 0.66 (lower than those of the 65–75 years group = 0.823, and >75 years group = 0.724). Pain and discomfort, instead, were lower compared to the healthy population (43% vs 56%). The idea of having an implanted device and of long-term follow-up is not worrying for 80% of patients; approximately two-thirds of them reported a regained control of their lives.
Conclusions
The importance of early diagnosis and patients’ perspective, alongside clinical evaluation, is highlighted. The self-reported evaluations on symptoms and QoL, along with the balance between postoperative worries and benefits, should be discussed preoperatively with patients and relatives, and included postoperatively to comprehensively assess the surgical outcome.
... The converted Italian EQ-5D-3 L individual response data were then converted to utility weights using the most recent Italian model. 31 Utility estimates were adjusted for patient characteristics, study followup, the incidence of MI, stroke, TIMI bleeding events, and adverse events. Model health state utility values are presented in Table 3. ...
Aims
To conduct a health economic evaluation of ticagrelor in patients with type 2 diabetes and coronary artery disease, from a multinational payer perspective. Cost-effectiveness and cost-utility of ticagrelor was evaluated in the overall THEMIS (effect of Ticagrelor on Health Outcomes in diabEtes Mellitus patients Intervention Study) trial population and in the predefined patient group with prior percutaneous coronary intervention.
Methods and results
A Markov model was developed to extrapolate patient outcomes over a lifetime horizon. The primary outcome was incremental cost-effectiveness ratios (ICERs), which was compared with conventional willing to pay (WTP) thresholds (47,000 €/quality-adjusted life year [QALY] in Sweden, 30,000 €/QALY in other countries).
Treatment with ticagrelor resulted in QALY gains of up to 0.045 in the overall population and 0.099 in patients with PCI. Increased costs and benefits translated to ICERs ranged between €27,894 and €42,252/QALY across Sweden, Germany, Italy, and Spain in the overall population. In patients with prior PCI, estimated ICERs improved to €18,449, €20,632, €20,233, and €13,228/QALY in Sweden, Germany, Italy, and Spain, respectively, driven by higher event rates and treatment benefit.
Conclusion
Based on THEMIS results, ticagrelor plus aspirin compared with aspirin alone may be cost-effective in some European countries in patients with T2DM and CAD and no prior MI or stroke. Additionally, ticagrelor is likely to be cost-effective across European countries in patients with a history of PCI.
... QoL was assessed using the EuroQoL. Three layers, referring to the Italian valuation set, were used to derive utility from the scores (48)(49)(50)(51)(52)(53). ...
Objective:
People with Chronic Obstructive Pulmonary Disease (COPD) may suffer from anxiety, depression, low quality of life, and cognitive deficits that could play a role in their clinical conditions. These situations could be worsened during the adaptation process to a new treatment such as non-invasive ventilation (NIV), which is often rejected or inappropriately used. The study aimed to analyse the impact of a brief psychological support intervention on adherence to NIV among patients with COPD.
Methods:
A two-branch randomized controlled trial was conducted with 90 patients with COPD who had an indication for NIV. The experimental group received cognitive behavioural therapy (CBT) support, including counselling, relaxation and mindfulness-based exercises. Controls received standard care and watched educational videos. The course had been structured over 4 to 8 meetings at the hospital, at home and/or via telemedicine.
Results:
The psychological intervention was related to improvements in both adherence to NIV (F(304) = 19.054, p < .001) and quality of life (F(156) = 10.264, p = .002) after 8 meetings from baseline compared to the control group. Results indicated a significant change in the quality of life also over time (F(71.480) = 8.114, p = .006).
Conclusions:
The findings suggest that the psychological intervention is an appropriate treatment for acceptance of and adherence to NIV in COPD in clinical practice and highlight the importance of determining the underlying reasons for NIV use.Trial registration:ClinicalTrials.govidentifier NCT02499653.
... The average health utility of the Italian general population is 0.92 [32]. Patients in TFR were assumed to also have a health state utility of 0.92, as these patients approached a 'functional cure'. ...
Objective:
The aim of this study was to evaluate the cost effectiveness of second-line nilotinib versus dasatinib for the treatment of Philadelphia chromosome-positive chronic myeloid leukemia (CML-CP) patients who are intolerant or resistant to imatinib and can transition to treatment-free remission (TFR).
Methods:
A partitioned survival model was developed to compare the cost effectiveness of nilotinib versus dasatinib. The model was developed from the Italian healthcare payer perspective and included the following health states: on second-line tyrosine kinase inhibitor (TKI), off second-line TKI, accelerated phase/blastic crisis, TFR, and death. Progression-free and overall survival curves were derived from patient-level data that compared nilotinib and dasatinib as second-line therapy in CML-CP patients who were resistant or intolerant to imatinib. Drug costs, healthcare costs, and adverse event costs were based on real-world evidence and publicly available databases. Cost effectiveness was estimated over a 40-year time horizon. Scenario analyses were performed by adjusting time horizon, TFR parameters, costs, and utilities.
Results:
Second-line nilotinib resulted in greater time spent in TFR (0.91 life-years), increased quality-adjusted life-years (QALYs) (1.89), increased life-years (2.16), and decreased per-patient costs (- 38,760 €). Therefore, nilotinib was strongly dominant compared with dasatinib in the base-case analysis. Nilotinib remained strongly dominant in most scenario analyses including shorter time horizon, exclusion of TFR, and varying TKI drug costs.
Conclusions:
While the model showed that nilotinib treatment of imatinib-intolerant or resistant CML-CP patients was more effective and less costly than dasatinib treatment, there is considerable uncertainty in the findings.
... As reported by Ramos-Goni et al., interviewer effects were identified in many valuation studies [52]. In the CAPI process, a standardized procedure of valuation was embedded to facilitate self-administration, and should eliminate the potential interviewer bias [34,35,53,54]. Furthermore, the CAPI process has been successful in other research ways: assisting and simplifying. ...
Background:
Health-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.
Methods:
A 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.
Results:
Three 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.
Conclusions:
VAS 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.
... The EQ-5D-5L™ questionnaire (©EuroQol Group, Rotterdam, The Netherlands) was administered before treatment and thereafter at 1, 3 and 6 months in order to measure the QoL of our patients after the endoscopic or surgical treatment in the following areas of investigations (mobility, self-care activities, usual activities, pain/discomfort and anxiety/ depression) with each dimension graded into 5 levels (i.e., no problem/slight problem/moderate problem/severe problem/ extreme problem). The health state can therefore be defined by a 5-digit number by combining one level from each of the five dimensions and then converted into the EQ-5D-5L index values according to the data from the general Italian population matched for age (20). Furthermore, to help patients explain how good or bad their health state was, a visual analog scale (EQ-VAS™) with endpoints labelled "the best health you can imagine" graded 100 and "the worst health you can imagine" graded 0 was administered and the results were furtherly analyzed. ...
Background/aim:
A prospective randomized open label parallel trial, comparing the quality of life (QoL) after endoscopic placement of a self-expandable metal stent or primary tumor resection, in patients with stage IV colorectal cancer was performed.
Patients and methods:
Thirty-three patients affected with stage IV colorectal cancer and unresectable metastases were randomly assigned into two groups: Group 1 (16 patients), that underwent self-expandable metal stent positioning and Group 2 (17 patients), in which primary tumor resection was performed. Karnofsky performance scale and QoL assessment using the EQ-5D-5L™ questionnaire was administered before treatment and thereafter at 1, 3 and 6 months.
Results:
At 1 month, index values showed a statistically significant deterioration of the QoL in patients of Group 2 when compared to those of Group 1 (p=0.001; 95%CI=0.065-0.211) whereas, at 6 months, index values showed a statistically significant deterioration of the QoL in patients of Group 1 (p<0.025; 95%CI=0.017-0.238).
Conclusion:
QoL in patients affected with stage IV colorectal cancer has a bimodal fluctuation pattern: at 1-month it was better in patients that received stent, but at 6-months it was significantly better in patients submitted to surgical resection.
... The second part of the questionnaire consists of a visual analogue scale (VAS) measuring the overall GH, ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). To calculate the utility index from the EQ-5D-5L descriptive system, the responses in the descriptive system were converted into utilities using the mapping algorithm developed by van Hout et al. and applied on the Italian social tariffs (13,14). ...
Systemic sclerosis (SSc)-related Raynaud’s phenomenon (RP) and digital ulcers (DU) can impair health-related quality of life (HRQoL). The aim of our study was to estimate HRQoL in SSc patients treated with two different intravenous (IV) iloprost (ILO) regimens and in patients not treated with IV ILO. 96 consecutive SSc patients were enrolled in a pragmatic, prospective and non-randomized study, and divided into 3 groups: not requiring therapy with IV ILO (N=52), IV ILO once monthly (N=24) or IV ILO for 5 consecutive days every 3 months (N=20). Patients were followed up for three months. We assessed HRQoL using the generic preference-based questionnaire EQ-5D-5L. We conducted multiple regression analyses to estimate, in each treatment group, the mean general health (GH) and the mean utility index of the EQ-5D-5L, adjusting for possible confounders. The mean adjusted utility index and GH score, after three months’ follow-up, were not different in the three groups: IV ILO was able to make patients requiring IV ILO similar to those not requiring it. Moreover, there was no difference in this model between the two ILO regimens (1 day monthly vs 5 consecutive days every 3 months). The two different IV ILO regimens (the most appropriate regimen was decided according to patients’ characteristics and needs) were able to stabilize HRQoL in RP secondary to SSc non-adequately controlled by oral therapy.
... In Iran, the death scenario owing to religious beliefs might be more valuable than some severe or moderate scenarios. Our sample size was larger than the sample size of studies in Japan, 7 Portugal, 16 Australia, 15 Italy, 34 France, 26 and The Netherlands. 35 The sample size seems sufficient to obtain statistically significant coefficients with 95% confidence intervals. ...
Background:
The EuroQol 5-dimension (EQ-5D) is a standard instrument that is widely used for measuring health-related quality of life and quality-adjusted life years in economic evaluation of healthcare interventions.
Objective:
To estimate a preference valuation set for EQ-5D 3-level (3L) health states from the perspective of the general population in the capital of Iran.
Methods:
Eight hundred seventy adults aged ≥18 years were interviewed in Tehran (Iran's capital) from July to November 2013. The participants were selected by a stratified random sampling method and were interviewed face-to-face at their usual residence. Forty-two health states were selected and valued from the 243 states derived from the EQ-5D-3L instrument. Each respondent valued 11 health states using the time trade-off method. Generalized least squares regression with random effect was used to predict values for health states.
Results:
The analysis was performed for 846 respondents. The final model yielded the best fit for the time trade-off value at the individual level with an overall R2 of 0.45 and a mean absolute error of 0.214. The mean values for the 42 health states ranged from 0.934 for state 11121 to -0.142 for state 33333.
Conclusions:
This study provided for the first time a value set for calculating quality-adjusted life years from the EQ-5D instrument in Iran. The Iranian EQ-5D-3L value set slightly differs from the value sets of the UK and the United States.
... Quality of life was assessed with the visual-analogue scale (VAS) and the utility index from the EuroQol Group 5-Dimension Self-Report Questionnaire (EQ-5D). 20,21 Con- trary to what was planned in the study protocol, the Short Form 36 questionnaires were not used to assess quality of life because the number of questionnaires adequately completed was insufficient. ...
Background:
Evidence is scarce on the efficacy of long-term human albumin (HA) administration in patients with decompensated cirrhosis. The human Albumin for the treatmeNt of aScites in patients With hEpatic ciRrhosis (ANSWER) study was designed to clarify this issue.
Methods:
We did an investigator-initiated multicentre randomised, parallel, open-label, pragmatic trial in 33 academic and non-academic Italian hospitals. We randomly assigned patients with cirrhosis and uncomplicated ascites who were treated with anti-aldosteronic drugs (≥200 mg/day) and furosemide (≥25 mg/day) to receive either standard medical treatment (SMT) or SMT plus HA (40 g twice weekly for 2 weeks, and then 40 g weekly) for up to 18 months. The primary endpoint was 18-month mortality, evaluated as difference of events and analysis of survival time in patients included in the modified intention-to-treat and per-protocol populations. This study is registered with EudraCT, number 2008-000625-19, and ClinicalTrials.gov, number NCT01288794.
Findings:
From April 2, 2011, to May 27, 2015, 440 patients were randomly assigned and 431 were included in the modified intention-to-treat analysis. 38 of 218 patients died in the SMT plus HA group and 46 of 213 in the SMT group. Overall 18-month survival was significantly higher in the SMT plus HA than in the SMT group (Kaplan-Meier estimates 77% vs 66%; p=0·028), resulting in a 38% reduction in the mortality hazard ratio (0·62 [95% CI 0·40-0·95]). 46 (22%) patients in the SMT group and 49 (22%) in the SMT plus HA group had grade 3-4 non-liver related adverse events.
Interpretation:
In this trial, long-term HA administration prolongs overall survival and might act as a disease modifying treatment in patients with decompensated cirrhosis.
Funding:
Italian Medicine Agency.
... Using the EQ-5D descriptive system we calculated utility scores by means of an algorithm that uses population-based (social) values estimated in Italy [27]. ...
Abstract Background Failed back surgery syndrome (FBSS) represents one main cause of chronic neuropathic or mixed pain, functional disability and reduced Health Related Quality of Life (HRQoL). Spinal Cord Stimulation (SCS) can be a value for money option to treat patients refractory to conventional medical management (CMM). We estimated from real-world data: 1) the amount of reduced levels of HRQoL of target patients compared to general population, 2) the relationship between pain intensity, functional disability, and overall HRQoL, and 3) the improvement of patients’ health from SCS intervention, and 4) we give some insights and make some suggestions on the selection of a battery of patients’ reported health instruments for use in routine clinical practice. Methods At recruitment (before SCS) and every 6 months for 2 years after SCS a battery of questionnaires/tests were completed: the generic EQ-5D and SF-36 for HRQoL, the specific Numerical Rating Scale (NRS) to measure pain intensity, and Oswestry Disability Index (ODI) to measure functional disability. We conducted multilevel regression analyses to investigate the association of HRQoL with the NRS and ODI indexes; multiple regression analyses to compare EQ-5D data with those of the general population adjusted for age, sex and education, and statistical tests to compare the changes of HRQoL, NRS and ODI estimates at baseline with those measured during the follow-up. Results Eighty patients (40% male, mean age = 58 years) participated. HRQoL was significantly worse in the patients than in the corresponding general population. Pain, functional disability and HRQoL significantly related each other during follow-up, Significant improvements (p
... An Italian tariff developed for the 3-level version of the EQ-5D found that Italian valuations were higher, particularly for more severe health states. 27 Further research would be required to evaluate the implications for studies similar to these. ...
... Our literature search identified 14 valuation studies 7,[16][17][18][19][20][21][22][23][24][25][26][27][28] that reported the mean observed health utilities and their SD (or SE), see Table 1. ...
Background:
Resource-constrained countries have difficulty conducting large EQ-5D valuation studies, which limits their ability to conduct cost-utility analyses using a value set specific to their own population. When estimates of similar but related parameters are available, shrinkage estimators reduce uncertainty and yield estimators with smaller mean square error (MSE). We hypothesized that health utilities based on shrinkage estimators can reduce MSE and mean absolute error (MAE) when compared to country-specific health utilities.
Methods:
We conducted a simulation study (1,000 iterations) based on the observed means and standard deviations (or standard errors) of the EQ-5D-3L valuation studies from 14 counties. In each iteration, the simulated data were fitted with the model based on the country-specific functional form of the scoring algorithm to create country-specific health utilities ("naïve" estimators). Shrinkage estimators were calculated based on the empirical Bayes estimation methods. The performance of shrinkage estimators was compared with those of the naïve estimators over a range of different sample sizes based on MSE, MAE, mean bias, standard errors and the width of confidence intervals.
Results:
The MSE of the shrinkage estimators was smaller than the MSE of the naïve estimators on average, as theoretically predicted. Importantly, the MAE of the shrinkage estimators was also smaller than the MAE of the naïve estimators on average. In addition, the reduction in MSE with the use of shrinkage estimators did not substantially increase bias. The degree of reduction in uncertainty by shrinkage estimators is most apparent in valuation studies with small sample size.
Conclusion:
Health utilities derived from shrinkage estimation allow valuation studies with small sample size to "borrow strength" from other valuation studies to reduce uncertainty.
... First, the impact of fentanyl formulation on utility values was derived by Vissers et al. that estimated this data with the TTO method in a UK general population[17]. More reliable data could be collected using a recommended generic quality of life questionnaire as the EQ-5D[38]; however, at the moment of the analysis, the data provided by Vissers et al.[17]were the better available evidences documented with a well-established method[39]. Second, the health care resources included in the model for each fentanyl formulation were derived by the literature and based on the analgesic onset following the approach reported in a previous budget impact analysis[19]. ...
Breakthrough cancer Pain (BTcP) has a high prevalence in cancer population. Patients with BTcP reported relevant health care costs and poor quality of life. The study assessed the cost-effectiveness of the available Oral Fentanyl Formulations (OFFs) for BTcP in Italy. A decision-analytical model was developed to estimate costs and benefits associated with treatments, from the Italian NHS perspective. Expected reductions in pain intensity per BTcP episodes were translated into, percentage of BTcP reduction, resource use and Quality-Adjusted-Life-Years (QALYs). Relative efficacy, resources used and unit costs data were derived from the literature and validated by clinical experts. Probabilistic and deterministic sensitivity analyses were performed. At base-case analysis, Sublingual Fentanyl Citrate (FCSL) compared to other oral formulations reported a lower patient's cost (€1,960.8) and a higher efficacy (18.7% of BTcP avoided and 0.0507 QALYs gained). The sensitivity analyses confirmed the main results in all tested scenarios, with the highest impact reported by BTcP duration and health care resources consumption parameters. Between OFFs, FCSL is the cost-effective option due to faster reduction of pain intensity. However, new research is needed to better understand the economic and epidemiologic impact of BTcP, and to collect more robust data on economic and quality of life impact of the different fentanyl formulations. Different fentanyl formulations are available to manage BTcP in cancer population. The study is the first that assesses the different impact in terms of cost and effectiveness of OFFs, providing new information to better allocate the resources available to treat BTcP and highlighting the need of better data.
... The values or utilities are indicated on a scale on which death has a value of 0 and perfect health a value of 1, with negative values being possible. Validated country-specific adult value sets obtained via the EuroQol website were used, with the exception of Italy, for which values were taken from literature [26]. ...
Background
Severe haemophilia is associated with major psychological and economic burden for patients, caregivers, and the wider health care system. This burden has been quantified and documented for a number of European countries in recent years. However, few studies have taken a standardised methodology across multiple countries simultaneously, and sought to amalgamate all three levels of burden for severe disease. The overall aim of the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS) study was to capture the annualised economic and psychosocial burden of severe haemophilia in five European countries.
A cross-section of haemophilia specialists (surveyed between January and April 2015) provided demographic and clinical information and 12-month ambulatory and secondary care activity for patients via an online survey. In turn, patients provided corresponding direct and indirect non-medical cost information, including work loss and out-of-pocket expenses, as well as information on quality of life and adherence. The direct and indirect costs for the patient sample were calculated and extrapolated to population level.
Results
Clinical reports for a total of 1,285 patients were received. Five hundred and fifty-two patients (43% of the sample) provided information on indirect costs and health-related quality of life via the PSC. The total annual cost of severe haemophilia across the five countries for 2014 was estimated at EUR 1.4 billion, or just under EUR 200,000 per patient. The highest per-patient costs were in Germany (mean EUR 319,024) and the lowest were in the United Kingdom (mean EUR 129,365), with a study average of EUR 199,541. As expected, consumption of clotting factor replacement therapy represented the vast majority of costs (up to 99%). Indirect costs are driven by patient and caregiver work loss.
Conclusions
The results of the CHESS study reflect previous research findings suggesting that costs of factor replacement therapy account for the vast majority of the cost burden in severe haemophilia. However, the importance of the indirect impact of haemophilia on the patient and family should not be overlooked. The CHESS study highlights the benefits of observational study methodologies in capturing a ‘snapshot’ of information for patients with rare diseases.
... Quality of life was assessed with the visual-analogue scale (VAS) and the utility index from the EuroQol Group 5-Dimension Self-Report Questionnaire (EQ-5D). 20,21 Con- trary to what was planned in the study protocol, the Short Form 36 questionnaires were not used to assess quality of life because the number of questionnaires adequately completed was insufficient. ...
... In our study, mean EQ-5D utility scores for FXS adult patients were lower than the mean values observed in the general population in both France and Hungary, where references are available [45, 46]. In Italy, mean VAS score for adult FXS patients was lower than that measured in a sample representative of the Italian general population [47] and lower than the mean value for patients affected by HIV [48], similar to what was observed in Spain [49]. In Sweden, the mean utility score for adult FXS patients was lower than for patients suffering from a broad range of disorders, including diabetes, asthma, mental distress, hypertension, angina pectoris and neck/shoulder pain [50]. ...
Objective
To estimate the social/economic costs of fragile X syndrome (FXS) in Europe and to assess the health-related quality of life (HRQOL) of patients and caregivers.
Methods
A cross-sectional study was conducted in a sample of European countries. Patients were recruited through patients’ associations. Data on their resource use and absence from the labour market were retrospectively obtained from an online questionnaire. Costs were estimated by a bottom-up approach and the EuroQol-5 Domain (EQ-5D) questionnaire was used to measure patients’ and caregivers’ HRQOL.
Results
Five countries were included in the analysis. The mean annual cost of FXS per patient varied from €4951 in Hungary to €58,862 in Sweden. Direct non-healthcare costs represented the majority of costs in all countries but there were differences in the share incurred by formal and informal care among those costs. Costs were also shown to differ between children and adults. Mean EQ-5D utility score for adult patients varied from 0.52 in France (n = 42) to 0.73 in Hungary (n = 2), while for caregivers this score was consistently inferior to 0.87.
Conclusion
Our findings underline that, although its prevalence is low, FXS is costly from a societal perspective. They support the development of tailored policies to reduce the consequences of FXS on both patients and their relatives.
... QALYs combine length and quality of life, with the latter normally expressed as a value between 0 (dead) and 1 (perfect health). Societal preferences obtained in the general public typically underlie these values [2,3]. ...
A common approach to obtain health state valuations is the time-tradeoff (TTO) method. Much remains unknown regarding the influence of responder characteristics on TTO answers. The objective of this study is to increase understanding of the influence that beliefs regarding future health and death, as well as desires to witness certain life events, have on respondents’ health state valuations.
An online survey was designed, including three TTO questions using a 10 year timeframe. Moreover, respondents completed demographic questions, the Health-Risk Attitude Scale (HRAS), the Expectations Regarding Aging (ERA) questionnaire, questions about beliefs regarding future health (i.e. life expectancy) and death (i.e. fear of death, belief in life after death and opinion about euthanasia), and about important life events taking place within the TTO timeframe. Regression analyses were performed in order to assess the influence of these different variables.
One thousand sixty-seven respondents were included in the analyses. The following variables were significantly associated with years traded off: ERA mental health (decrease), ERA physical health (increase), HRAS (increase), support for euthanasia (increase), fear of death (decrease) and consideration of an important life event (decrease). The explained variance of the final model was low (0.08).
TTO responses may be influenced by considerations of future health, including life events and attitudes regarding health risks and death. Further investigation of TTO responses remains warranted.
... Quality of life was assessed with the visual-analogue scale (VAS) and the utility index from the EuroQol Group 5-Dimension Self-Report Questionnaire (EQ-5D). 20,21 Con- trary to what was planned in the study protocol, the Short Form 36 questionnaires were not used to assess quality of life because the number of questionnaires adequately completed was insufficient. ...
... It is not clear from their paper which social tariffs the authors used to obtain the utility indexes necessary to estimate QALYs. The authors collected HR-QOL data with the generic instrument EQ-5D-3L; hence, the Italian-specific social tariffs published in 2013 by Scalone et al. [2] are appropriate for converting the participants' responses into utility indexes. Previously, when a specific value set for Italy was missing, UK or Spanish sets were used to conduct health economic evaluations for the Italian healthcare system. ...
Spandonaro et al. [1] conducted an interesting cost-utility analysis to estimate the incremental cost/QALY (quality-adjusted life-year) ratio of biologic therapy for the treatment of chronic plaque psoriasis, adopting the perspective of the Italian National Health Service. The use of QALYs in the analysis allowed the calculation to include a parameter of effects measured in terms of health-related quality of life (HR-QOL), which is a component of health that is significantly compromised in patients with chronic plaque psoriasis.It is not clear from their paper which social tariffs the authors used to obtain the utility indexes necessary to estimate QALYs. The authors collected HR-QOL data with the generic instrument EQ-5D-3L; hence, the Italian-specific social tariffs published in 2013 by Scalone et al. [2] are appropriate for converting the participants’ responses into utility indexes. Previously, when a specific value set for Italy was missing, UK or Spanish sets were used to conduct ...
... Responses to these five dimensions are converted into one of 243 different EQ-5D health state descriptions, which range between no problems on all five dimensions (11111) and severe/extreme problems on all five dimensions (33333). The Italian populationbased values were used to convert patient responses to the health state classifier into a single index, which produces scores from 1 to −0.38 [32]. ...
Background
Over the last decade, significant progresses have been achieved in the development and validation of new tools for the evaluation of disease activity in axial spondyloarthritis (SpA). Despite they play a key role in the assessment of these patients, the calculation scores are relatively complex and difficult to be quickly assessed in the busy daily clinical practice.Objectives
To test the construct validity of the Simplified Ankylosing Spondylitis Disease Activity Score (SADSAS) to define disease activity and compare its internal and external responsiveness with the Ankylosing Spondylitis Disease Activity Score (ASDAS) and the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) in patients with axial SpA.Methods
The patient cohort comprised 397 consecutive axial SpA patients who had never been treated with tumor necrosis factor (TNF) blockers. Clinical and laboratory outcome assessments were performed at baseline, and at week 24. The following parameters were evaluated: BASDAI, ASDAS-CRP, ASDAS-ESR, and SASDAS. Construct convergent validity was evaluated by correlating SASDAS with ASDAS ERS/CRP, BASDAI, Bath Ankylosing Spondylitis Functional Index (BASFI) and EuroQol five-dimensional (EQ-5D) questionnaire. One hundred and fifty-six patients were observed longitudinally for 6 months. Responsiveness was assessed after six months of treatment with sulfasalazine (SSZ) or biologics. Internal responsiveness was evaluated by using the effect size (ES) and standardized response mean (SRM). External responsiveness was investigated by receiver operating characteristic (ROC) analysis. Change scores were compared by calculating paired t-test statistic for the difference.ResultsIn testing for convergent validity a strong correlations (p¿<¿0.0001) were observed between both SASDAS and ASDAS-ESR (r¿=¿0.835), and ASDAS-CRP (r¿=¿0.805). Strong correlations (p¿<¿0.0001) were also found between SASDAS and BASDAI score (r¿=¿¿0.886), SASDAS and BASFI scores (rho¿=¿0.588) and SASDAS and EQ-5D scores (rho¿=¿¿0.579). The cross-classification showed a significant overall agreement (defined as the percentage of observed exact agreements) for SASDAS vs ASDAS-ESR (weighted k¿=¿0.704) and for SASDAS vs ASDAS-CRP (k¿=¿0.661). The most efficient composite measure in detecting change was the ASDAS-CRP (ES 1.95 and SRM 0.97). The responsiveness of SASDAS was slightly higher to ASDAS-ESR with an ES of 1.62 and 1.33, and an SRM of 0.88 and 0.71, respectively. The BASDAI appear to be the less responsive (ES¿=¿0.93 and SRM¿=¿0.52). The area under ROC curve of the SASDAS gives similar results to those provided by ASDAS CRP/ESR. The score changes of all combinations were highly correlated (p¿<¿0.0001).Conclusions
The new SASDAS is a highly effective measure in assessing disease activity and it showed comparable internal and external responsiveness with respect to the ASDAS ESR/CRP response criteria in patients with axial SpA. SASDAS is easy to calculate and, therefore, appear suitable for clinical decision making, epidemiologic research, and clinical trials.
Background
Preference differences between countries and populations justify the use of country-specific value sets for the EQ-5D instruments. There are no clear criteria based on which the selection of value sets for countries without a national value set should be made. As part of the European PECUNIA project, this study aimed to identify factors contributing to differences in preference-based valuations and develop supra-national value sets for homogenous country clusters in Europe.MethodsA literature review was conducted to identify factors relevant to variations in the EQ-5D-3L/5L health state valuations across countries. Factors fulfilling the pre-specified criteria of validity, reliability, international feasibility and comparability were used to group 27 European Union member states, the European Free Trade Association countries and the UK. Clusters of countries were developed based on the frequency of their appearance in the same grouping. The supra-national value sets were estimated for these clusters from the coefficients of existing published valuation studies using the ordinary least-squares model.ResultsTen factors were identified from 69 studies. From these, five grouping variables: (1) culture and religion; (2) linguistics; (3) healthcare system typology; (4) healthcare system financing; and (5) sociodemographic aspects were derived to define the groups of homogenous countries. Frequency-based grouping revealed five cohesive clusters: English-speaking, Nordic, Central-Western, Southern and Eastern European.Conclusions
European countries were clustered considering variables that may relate to differences in health state valuations. Supra-national value sets provide optimised proxy value set selection in the lack of a national value set and/or for regional decision making.
Background
Real-world data on chemotherapy-naïve patients with metastatic castration-resistant prostate cancer (mCRPC) treated with abiraterone plus prednisone are limited, largely deriving from small retrospective studies.
Methods
ABitude is an Italian, observational, prospective, multicenter study of mCRPC patients receiving abiraterone plus prednisone in clinical practice. Chemotherapy-naïve mCRPC patients were consecutively enrolled at abiraterone start (February 2016 to June 2017) and are being followed for 3 years, with evaluation approximately every 6 months. Several clinical and patients reported outcomes were examined.
Results
In this second interim analysis, among 481 enrolled patients, 453 were evaluable for analyses. At baseline, the median age was 77 years and ~69% of patients had comorbidities (mainly cardiovascular diseases). Metastases were located mainly at bones and lymph nodes; 8.4% of patients had visceral metastases. During a median follow-up of 18 months, 1- and 2-year probability of radiographic progression-free survival were 73.9% and 56.2%, respectively; the corresponding rates for overall survival were 87.3% and 70.4%. In multivariable analyses, the number of bone metastases significantly affected radiographic progression-free survival and overall survival. During abiraterone plus prednisone treatment, 65% of patients had a ⩾50% prostate-specific antigen decline, and quality of life remained appreciably high. Among symptomatic patients according to the Brief Pain Inventory) (32%), scores significantly declined after 6 months of treatment. Overall, eight patients (1.7%) had serious adverse reactions to abiraterone.
Conclusions
Abiraterone plus prednisone is effective and safe for chemotherapy-naïve mCRPC patients in clinical practice.
Objectives:
The purpose of this study was to evaluate the current state of health economic evaluations (HEEs) submitted by pharmaceutical companies to the Italian Medicines Agency (AIFA) as part of their pricing and reimbursement (P&R) dossiers, and to explore potential future actions in order to enhance their quality.
Methods:
All company dossiers submitted from October 2016 to December 2018 were reviewed to select those containing pharmacoeconomic studies. The general characteristics of HEEs were described and their quality assessed based on a checklist adapted from Philips et al. (Review of guidelines for good practice in decision-analytic modelling in health technology assessment. Health Technol Assess. 2004;8: 1-158).
Results:
Of the 299 dossiers submitted to AIFA, 105 included one or more pharmacoeconomic studies, of which fifty-three were cost-effectiveness analyses. Overall, the compliance of the HEEs with the quality checklist was highly variable: some studies reached high methodological standards whereas others had serious flaws (mean 59.22 percent, range 19.35-90.32 percent). The main weaknesses were the unjustified exclusion of relevant alternatives, poor description and justification of model data and assumptions, and insufficient exploration of uncertainty and study validity. Non-homogeneity across studies was found in study perspectives, discount rates, methods for costing, estimating quality-adjusted life-years and conducting sensitivity analyses.
Conclusions:
Based on the results of this study, the recommended actions for increasing the quality of HEEs within reimbursement submissions in Italy are twofold: first, to set methodological standards for conducting and reporting HEEs; second, to strengthen the internal assessment process, also through the acquisition of companies' models and re-evaluation of results. These actions will hopefully provide greater contribution to the evidence-based P&R decision making.
Backgrounds: Contextual factors (CFs) have been recently proposed as triggers of placebo and nocebo effects in musculoskeletal pain. CFs encompass the features of the clinician (e.g. uniform), patient (e.g. expectations), patient–clinician relationship (e.g. verbal communication), treatment (e.g. overt therapy), and healthcare setting (e.g. design). To date, the researchers’ understanding of Italian patients’ knowledge about the role of CFs in musculoskeletal pain is lacking.
Objectives: The aim of this study was to investigate attitudes and beliefs of Italian patients with musculoskeletal pain about the use of CFs in clinical practice.
Methods: A national sample of Italian patients with musculoskeletal pain was recruited from 12 outpatient private clinics in Italy. An invitation to participate in an online survey was sent to patients: a) exhibiting musculoskeletal pain; b) aged 18–75; c) with a valid e-mail account; and d) understanding Italian language. Survey Monkey software was used to deliver the survey. The questionnaire was self-reported and included 17 questions and 2 clinical vignettes on the patients’ behavior, beliefs, and attitudes towards the adoption of CFs in clinical practice. Descriptive statistics and frequencies described the actual number of respondents to each question.
Results: One thousand one hundred twelve patients participated in the survey. Five hundred seventy-four participants were female (52%). The average age of patients was 41.7 ± 15.2 years. Patients defined CFs as an intervention with an unspecific effect (64.3%), but they believed in their clinical effectiveness. They identified several therapeutic effects of CFs for different health problems. Their use was considered ethically acceptable when it exerts beneficial psychological effects (60.4%), but it was banned if considered deceptive (51.1%). During clinical practice, patients wanted to be informed about the use of CFs (46.0%) that are accepted as an addition to other interventions to optimize clinical responses (39.3%). Moreover, patients explained the power of CFs through body–mind connections (37.1%).
Conclusion: Patients with musculoskeletal pain had positive attitudes towards the use and effectiveness of CFs when associated with evidence-based therapy. They mostly perceived the adoption of CFs in clinical practice as ethical.
Background: A consistent and comparative description of the burden of diseases, injuries and risk factors that cause them is an important input to health decision-making and planning processes. Objectives: The aim of this cross-sectional study was to compare the extent and pattern of variation in generic utility measures with respect to capturing the clinical nature of acute poisonings from the moment of onset until restoration of health after treatment. Patients and Methods: We measured the health status of patients admitted to the emergency medical toxicology ward of our teaching hospital, Mashhad, Iran. We measured their EuroQol both at admission and pre-discharge, and they were asked for time they traded-off for their current illness. Results: The study enrolled 82 patients (34 males, 48 females) admitted to the emergency medical toxicology department. A questionnaire was completed for all patients at the two time intervals. The results varied from 22222 to 33333 at initial management; at the time of discharge, this rating varied from 11111 to 11122. These significant changes occurred over just a few days. Conclusions: This study attempted to compare the course of acute poisonings with some other diseases to show how self-induced poisoning affects one’s health perception, and how this change takes place over a short time.
Background:
In measuring health utilities, the primary reason for selecting patients as a source for valuations is that they directly experience the impact of the disease.
Objectives:
Accordingly, the aim of this study was to examine the variation in generic utility measures with respect to acute poisonings by including a comparison between those subjects who had high intention and low intention to commit suicide.
Patients and methods:
We evaluated the responses of patients who had attempted suicide and were admitted to a toxicology ward. We used multiple methods, including TTO, VAS, and EQ-5D.
Results:
We reviewed the collected questionnaires of one hundred patients admitted to the medical toxicology ward of Emam Reza teaching hospital in Mashhad, Iran. Our results show that the mental state after an incomplete suicide attempt can present either a real desire for suicide or a desire for attention from relatives and rejection of life problems.
Conclusions:
This study demonstrates that the mental states associated with specific diseases should not be ignored in evaluating health states. Although there are benefits to relying on expert panels and the general population in evaluating various health states, attention to the particular health states of the patients (taking into account their associated mental well-being) should also be utilized.
EQ-5D-3L valuation studies continue to employ the MVH protocol or variants of MVH. One issue that has received attention is the selection of the states for direct valuation by respondents. Changes in the valuation subset have been found to change the coefficients of the utility function. The purpose of this study was to test the performance of valuation subsets based on orthogonal experiment designs. The design of the study also allowed a comparison of models based on raw or untransformed VAS values with values transformed at the level of the respondent and at the aggregate level.
Two different valuation subsets were developed based on orthogonal arrays. A VAS elicitation was undertaken with two groups of similar respondents and the resulting utility functions based on the valuations of the two different valuation subsets were compared using mean absolute errors between model and observed values, and by correlation with values in and out of sample. The impact of using untransformed versus VAS values transformed at the level of the individual and at aggregate level and the inclusion of a constant term in the utility functions were also investigated.
The utility functions obtained from the two valuation subsets were very similar. The models that included a constant and based on raw VAS values from the two valuation studies returned rank correlation coefficients of 0.994 and 0.995 when compared with respective observed values. MAEs of model values with observed values were 2.4% or lower for all models that included a constant term. Several models were developed and evaluated for the combined data (from both valuation subsets). The model that included the N3 term performed best.
The finding that two very different valuation subsets can produce strikingly similar utility functions suggests that orthogonal designs should be given some attention in further studies. The impact of rescaling VAS values at the level of the individual versus at aggregate level had minimal impact on the performance of the models when compared to models based on the raw VAS values.
To assess the cost-effectiveness and cost-utility of Spinal Cord Stimulation (SCS) in patients with failed back surgery syndrome (FBSS) refractory to conventional medical management (CMM).
We conducted an observational, multicenter, longitudinal ambispective study, where patients with predominant leg pain refractory to CMM expecting to receive SCS+CMM were recruited in 9 Italian centers and followed up to 24 months after SCS. We collected data on clinical status (pain intensity, disability), Health-Related Quality-of-Life (HRQoL) and on direct and indirect costs before (pre-SCS) and after (post-SCS) the SCS intervention. Costs were quantified in € 2009, adopting the National Health Service's (NHS), patient and societal perspectives. Benefits and costs pre-SCS versus post-SCS were compared to estimate the incremental cost-effectiveness and cost utility ratios.
80 patients (40% male, mean age 58 years) were recruited. Between baseline and 24 months post-SCS, clinical outcomes and HRQoL significantly improved. The EQ-5D utility index increased from 0.421 to 0.630 (p < 0.0001). Statistically significant improvement was first observed six months post-SCS. Societal costs increased from €6600 (pre-SCS) to €13,200 (post-SCS) per patient per year. Accordingly, the cost-utility acceptability curve suggested that if decision makers' willingness to pay per Quality-Adjusted-Life-Years (QALYs) was €60,000, SCS implantation would be cost-effective in 80% and 85% of cases, according to the NHS's and societal point of views, respectively.
Our results suggest that in clinical practice, SCS+CMM treatment of FBSS patients refractory to CMM provides good value for money. Further research is encouraged in the form of larger, long-term studies.
© 2015 International Neuromodulation Society.
Background: Health-related quality of life (HRQoL) is used as a measure to valuate healthcare
Background: Health-related quality of life (HRQoL) is used as a measure to valuate healthcare
interventions and guide policy making. The EuroQol EQ-5D is a widely used generic preference based
instrument to measure Health-related quality of life.
Objectives: The objective of this study was to develop a value set of the EQ-5D health states for
an Iranian population.
Methods: This study is the cross-sectional study of Iranian populations. Our sample from Iranian
populations consists out of 869 participants which are selected for this study using a stratified
probability sampling method. The sample was taken from individuals living in the city of
Tehran and was stratified by age and gender from July to November 2013. Respondents valued
13 health states using the visual analogue scale (VAS) of EQ-5D. Several fix effects regression
models were tested to predict the full set of health states. We selected the final model based on
the logical consistency of the estimates, sign and magnitude of the regression coefficients,
goodness of fit, and parsimony. We also compared predicted values with a value set from similar
studies in the UK and other countries.
Results: Our results show that the Health-related quality of life does not vary among socioeconomic
groups. Models at the individual level resulted in an additive model with all
coefficients statistically significant, R2 = 0.55, value of 0.75 for the best health state (11112) and
value of -0.074 for the worst health state (33333). The value set obtained for study sample
remarkably differs from those elicited in developed countries.
Conclusion: This study is the first estimate for EQ-5D value set based on VAS in Iran. Given
the importance of locally adapted value set the use of this value set can be recommended for
future studies in Iran and in the regions.
Keywords: Visual Analogue Scale, Quality of life, Preference-based health measures,
Population group, EQ-5D
Reliable and accurate mapping techniques that translate health-related quality-of-life data into EQ-5D index values are now in demand by researchers conducting economic evaluation of health care technologies. In this article, we present two commands (mrs2eq and oks2eq) that translate data from two widely used disease-specific instruments into EQ-5D index values and predicted probabilities of being at a particular level on each EQ-5D domain. mrs2eq conducts a response mapping approach to transform data from the stroke-specific modified Rankin scale into index values from the generic quality-of-life EQ-5D instrument. oks2eq uses a response mapping model to estimate EQ-5D index values based on patients’ responses to the Oxford Knee Score.
Since 1997, in order for a new drug to obtain its price and reimbursement in Italy, the negotiation between authorities and the pharmaceutical industry must include an economic evaluation. The economic evaluation study leads to price and reimbursement negotiations together with such other requirements as the price of the new drug in other countries, the impact on the domestic market in terms of public pharmaceutical expenditure, and the effects on the national economy (employment and investments).
Purpose:
To assess the performance of the EQ-5D-5L version compared with the standard EQ-5D-3L in a clinical setting targeted at patients with chronic hepatic diseases (CHDs).
Methods:
We introduced the 5L descriptive system into a cost-of-illness study involving patients with different CHDs. The patients completed a questionnaire including the two versions of the EQ-5D, together with other questions related to their condition. We tested the feasibility, the level of inconsistency, the redistribution properties among consistent responses, the ceiling effect, the discriminative power, and the convergent validity of the 5L compared with the 3L system.
Results:
A total of 1,088 valid patients were recruited: 62% male, 19-89 (median = 59) years old. Patients had chronic hepatitis from HCV (31.8%) or HBV infections (29.3%) or other causes (7.8%), 20.4% had cirrhosis, 11.9% underwent liver transplantation, and 7.8% had hepatic carcinoma. Less than 1% of EQ-5D-5L were returned blank, and 1.6% or less of missing values were calculated on the dimensions of the partially completed questionnaires. The proportion and weight of inconsistent responses (i.e., 3L responses that were at least two levels away from the 5L responses) was 2.9% and 1.2 on average, respectively. Regarding redistribution, 57-65% of the patients answering level 2 with the 3L version redistributed their responses to levels 2 or 4 with the 5L version. A relative 7% reduction of the ceiling effect was found. Furthermore, the absolute informativity increased but the relative informativity slightly decreased in every domain, and the convergent validity with the VAS improved.
Conclusions:
In a clinical setting involving CHD patients, the EQ-5D-5L was shown to be feasible and with promising levels of performance. Our findings suggest that the 5L performs better in at least some of the properties analyzed, and encourage further research to also test other psychometric properties of this new version of the EQ-5D.
Purpose
The aim of this study was to assess the measurement properties of the 5-level classification system of the EQ-5D (5L), in comparison with the 3-level EQ-5D (3L).
Methods
Participants (n = 3,919) from six countries, including eight patient groups with chronic conditions (cardiovascular disease, respiratory disease, depression, diabetes, liver disease, personality disorders, arthritis, and stroke) and a student cohort, completed the 3L and 5L and, for most participants, also dimension-specific rating scales. The 3L and 5L were compared in terms of feasibility (missing values), redistribution properties, ceiling, discriminatory power, convergent validity, and known-groups validity.
Results
Missing values were on average 0.8 % for 5L and 1.3 % for 3L. In total, 2.9 % of responses were inconsistent between 5L and 3L. Redistribution from 3L to 5L using EQ dimension-specific rating scales as reference was validated for all 35 3L–5L-level combinations. For 5L, 683 unique health states were observed versus 124 for 3L. The ceiling was reduced from 20.2 % (3L) to 16.0 % (5L). Absolute discriminatory power (Shannon index) improved considerably with 5L (mean 1.87 for 5L versus 1.24 for 3L), and relative discriminatory power (Shannon Evenness index) improved slightly (mean 0.81 for 5L versus 0.78 for 3L). Convergent validity with WHO-5 was demonstrated and improved slightly with 5L. Known-groups validity was confirmed for both 5L and 3L.
Conclusions
The EQ-5D-5L appears to be a valid extension of the 3-level system which improves upon the measurement properties, reducing the ceiling while improving discriminatory power and establishing convergent and known-groups validity.
This was a prospective observational study designed to evaluate direct and indirect costs and quality of life for patients with Crohn's disease in Italy from the perspectives of the National Health System and of society.
A total of 162 male and female subjects aged 18-70 years with Crohn's disease in the active phase and a Crohn's Disease Activity Index score ≥150 were included in the study. Subjects were recruited from 25 Italian centers on a consecutive basis. The study consisted of four visits undertaken every 6 months with a follow-up period of 18 months. The study started on September 1, 2006 and was completed on April 12, 2010. Multivariate analyses were carried out on demographic characteristics, treatment costs based on the prescribed daily dose, resource use and other cost parameters, and changes in quality of life using the EQ5D questionnaire.
Cost of illness per subject with Crohn's disease in Italy was estimated to be €15,521 per year, with direct costs representing 76% of total costs. Nonhealth care costs and loss of productivity accounted for 24% of total costs. Societal costs during the first months of enrolment were higher compared with costs in the final months of the study. Quality of life measured by the EQ-5D was 0.558 initially and then increased to 0.739, with a mean value of 0.677 during the enrolment period. The cost of illness was not correlated with age or gender.
The cost of illness was correlated with quality of life; Crohn's disease had a negative impact on subjects' quality of life, and higher costs corresponded to a lower quality of life as measured with the EQ5D. Drug treatment may improve quality of life and reduce hospitalization costs. Our results appear to be in line with the results of other international cost-of-illness studies.
The EQ-5D is a preference based instrument which provides a description of a respondent's health status, and an empirically derived value for that health state often from a representative sample of the general population. It is commonly used to derive Quality Adjusted Life Year calculations (QALY) in economic evaluations. However, values for health states have been found to differ between countries. The objective of this study was to develop a set of values for the EQ-5D health states for use in Canada.
Values for 48 different EQ-5D health states were elicited using the Time Trade Off (TTO) via a web survey in English. A random effect model was fitted to the data to estimate values for all 243 health states of the EQ-5D. Various model specifications were explored. Comparisons with EQ-5D values from the UK and US were made. Sensitivity analysis explored different transformations of values worse than dead, and exclusion criteria of subjects.
The final model was estimated from the values of 1145 subjects with socio-demographics broadly representative of Canadian general population with the exception of Quebec. This yielded a good fit with observed TTO values, with an overall R2 of 0.403 and a mean absolute error of 0.044.
A preference-weight algorithm for Canadian studies that include the EQ-5D is developed. The primary limitations regarded the representativeness of the final sample, given the language used (English only), the method of recruitment, and the difficulty in the task. Insights into potential issues for conducting valuation studies in countries as large and diverse as Canada are gained.
To derive EuroQol five-dimensional (EQ-5D) health states values from the Thai general population.
Forty-eight trained individuals successfully conducted interviews with a representative sample of 1409 respondents in 2007. A total of 12 sets of health states were used with one set allocated to each respondent. A respondent was requested to assign values for 11 states using the ranking and visual analogue scale methods and 10 states using the time trade-off method. The variables from the three existing models were used in model specifications and the best model was chosen on the basis of the extent of logical inconsistency in the estimated scores, predictive performance, parsimony, and sensitivity to changes in health.
Eighty-six health states were valued. The mean age of respondents was 44.6 years old. The highly consistent respondents tend to give higher scores for mild states and lower scores for severe states, compared with those given by the highly inconsistent respondents. The best model used variables from the Dolan 1997 study and estimated from the scores given by the respondents with fewer than 11 inconsistencies. The estimated scores are completely consistent, R(2) is 0.448. The second highest score was 0.766 given to state 11112 and the lowest score was -0.454 for state 33333.
Values for EQ-5D health states were estimated from the Thai general population. This is the first Thai generic health state value results to be used in evaluating health interventions in Thailand.
The validity, reliability and cross-country comparability of summary measures of population health (SMPH) have been persistently debated. In this debate, the measurement and valuation of nonfatal health outcomes have been defined as key issues. Our goal was to quantify and decompose international differences in health expectancy based on health-related quality of life (HRQoL). We focused on the impact of value set choice on cross-country variation.
We calculated Quality Adjusted Life Expectancy (QALE) at age 20 for 15 countries in which EQ-5D population surveys had been conducted. We applied the Sullivan approach to combine the EQ-5D based HRQoL data with life tables from the Human Mortality Database. Mean HRQoL by country-gender-age was estimated using a parametric model. We used nonparametric bootstrap techniques to compute confidence intervals. QALE was then compared across the six country-specific time trade-off value sets that were available. Finally, three counterfactual estimates were generated in order to assess the contribution of mortality, health states and health-state values to cross-country differences in QALE.
QALE at age 20 ranged from 33 years in Armenia to almost 61 years in Japan, using the UK value set. The value sets of the other five countries generated different estimates, up to seven years higher. The relative impact of choosing a different value set differed across country-gender strata between 2% and 20%. In 50% of the country-gender strata the ranking changed by two or more positions across value sets. The decomposition demonstrated a varying impact of health states, health-state values, and mortality on QALE differences across countries.
The choice of the value set in SMPH may seriously affect cross-country comparisons of health expectancy, even across populations of similar levels of wealth and education. In our opinion, it is essential to get more insight into the drivers of differences in health-state values across populations. This will enhance the usefulness of health-expectancy measures.
The valuation of health states is an integral part of economic evaluation studies. The source of these valuations (general public vs. patients) is surrounded by controversy. Health state values generated by the general public are often different compared with those of patients. General public values may not account for adaptation of the patients; patients' values potentially incorporate self-interest. Decisions on the appropriate source of health values ultimately depend on the specific decision-making context and objectives of the evaluation. Differences in valuations and implications for decision-making should be explicitly addressed. Further research should systematically identify circumstances in which public and patients' valuations diverge. There appears ground for development of methods that allow the simultaneous incorporation of patients and public preferences. Existing literature which discusses the issues around the health state valuation is summarized.
Health-related quality of life (HRQoL) in patients with systemic sclerosis (SSc), a chronic disabling disease associated to physical and psychological impairment, is often left behind in clinical practice and research. This is due to the use of tools that are not complete or mainly designed for the physical condition only. We tested EQ-5D, a valid, simple and brief questionnaire for HRQoL that has never been validated in SSc.
Thirty-three consecutive SSc patients referring to our Rheumatology Department and undergoing treatment have been asked to fulfill EQ-5D together with HAQ.
EQ-5D demonstrated good acceptability, feasibility and validity in patients affected by SSc. Conceptually equivalent domains of EQ-5D demonstrated a good correlation with HAQ correspondent domains.
We suggest the use of EQ-5D in SSc patients as a HRQoL measure in clinical practice, as well as an out come parameter in randomized clinical trials and/or in pharmaco-economic evaluations.
The aim of this survey study was to derive the societal values of the general public for the EuroQol EQ-5D. Using the same protocol as previously used in the United Kingdom, we compared the German values with the British. In face-to-face interviews a sample of 339 individuals in northern Germany valued 15 different health states from a sample of 36 states. Values were derived using the York MVH protocol for time trade-off (TTO) and a visual analogue scale (VAS). Values for all 243 health states of the EQ-5D were estimated by a regression model. The VAS values revealed close a resemblance to the British VAS results. German TTO values were higher than the British. This was especially the case for the worse health states. The results suggest that the TTO values are more related to national variables than values derived by VAS. The use of the TTO values of this investigation makes it possible to anticipate these cultural differences in studies carried out in Germany.
Currently, there is no EQ-5D value set for Poland. The primary objective of this study was to elicit EQ-5D Polish values using the time trade-off (TTO) method.
Face-to-face interviews with visitors of inpatients in eight medical centers in Warsaw, Skierniewice, and Puławy were carried out by trained interviewers. Quota sampling was used to achieve a representative sample of the Polish population with regard to age and sex. Modified protocol from the Measurement and Value of Health study was used. Each respondent ranked 10 health states and valued 4 health states using the visual analog scale and 23 using the TTO. Mean and variance stability tests were performed to determine whether using a larger number of health states per respondent would yield credible results. Modeling included random effects and random parameters models.
Between February and May 2008, 321 interviews were performed. Modeling based on 6777 valuations resulted in an additive model with all coefficients statistically significant, R(2) equal to 0.45, and value -0.523 for the worst possible health state. Means and variance did not differ significantly for states valued in the middle and at the end of the TTO exercise.
This is the first EQ-5D value set based on TTO in Central and Eastern Europe so far. Because the values differ considerably from those elicited in Western European countries, its use should be recommended for studies in Poland. Increasing the number of health states that each respondent is asked to value using TTO seems feasible and justifiable.
To present an episodic random utility model that unifies time trade-off and discrete choice approaches in health state valuation.
First, we introduce two alternative random utility models (RUMs) for health preferences: the episodic RUM and the more common instant RUM. For the interpretation of time trade-off (TTO) responses, we show that the episodic model implies a coefficient estimator, and the instant model implies a mean slope estimator. Secondly, we demonstrate these estimators and the differences between the estimates for 42 health states using TTO responses from the seminal Measurement and Valuation in Health (MVH) study conducted in the United Kingdom. Mean slopes are estimates with and without Dolan's transformation of worse-than-death (WTD) responses. Finally, we demonstrate an exploded probit estimator, an extension of the coefficient estimator for discrete choice data that accommodates both TTO and rank responses.
By construction, mean slopes are less than or equal to coefficients, because slopes are fractions and, therefore, magnify downward errors in WTD responses. The Dolan transformation of WTD responses causes mean slopes to increase in similarity to coefficient estimates, yet they are not equivalent (i.e., absolute mean difference = 0.179). Unlike mean slopes, coefficient estimates demonstrate strong concordance with rank-based predictions (Lin's rho = 0.91). Combining TTO and rank responses under the exploded probit model improves the identification of health state values, decreasing the average width of confidence intervals from 0.057 to 0.041 compared to TTO only results.
The episodic RUM expands upon the theoretical framework underlying health state valuation and contributes to health econometrics by motivating the selection of coefficient and exploded probit estimators for the analysis of TTO and rank responses. In future MVH surveys, sample size requirements may be reduced through the incorporation of multiple responses under a single estimator.
To evaluate costs, benefits and cost-effectiveness of anti-TNF agents in PsA patients with inadequate response to conventional treatment.
A total of 107 patients, from nine Italian rheumatology centres, with different forms of PsA were given anti-TNF treatment, mainly etanercept (87%). Information on resource use, health-related quality of life, disease activity, function and laboratory values were collected at baseline and through out the 12 months of therapy. Cost (expressed in euro 2007) and utility (measured by EuroQol) before and after anti-TNF therapy initiation were compared in order to estimate the incremental cost per quality-adjusted life year (QALY) gained, and cost-effectiveness acceptability curve was calculated.
At the end of 12 months, there was a significant increase in direct cost due to an increase of drug cost caused by TNF inhibitors that was only partially offset by the decrease in indirect cost. In the last 6 months of therapy, the direct cost increased by euro5052, the cost for the National Health System (NHS) by euro5044 and the social cost by euro4638. However, a gain of 0.12 QALY resulted in a cost per QALY gained of euro40 876 for the NHS and of euro37 591 for the society. The acceptability curve showed that there would be a 97% likelihood that anti-TNF therapy would be considered cost-effective at willingness-to-pay threshold of euro60 000 per QALY gained.
Cost-effectiveness ratios are within the commonly accepted willingness-to-pay threshold. These results need to be confirmed in larger samples of patients.
Iron chelation treatment (ICT) in beta-thalassemia major (beta-TM) patients undergoing blood transfusions can cause low satisfaction, low compliance, with possible negative consequences on treatment success, patients' wellbeing, and costs. The purpose was to estimate the societal burden attributable to beta-TM in terms of direct and indirect costs, health-related quality-of-life (HRQoL), satisfaction and compliance with ICT in patients undergoing transfusions and ICT.
The naturalistic, multicenter, longitudinal Italian-THAlassemia-Cost-&-Outcomes-Assessment (ITHACA) cost-of-illness study was conducted involving patients of any age, on ICT for at least 3 years, who were enrolled at 8 Italian Thalassemia Care Centers. Costs were estimated from the societal perspective, quantified with tariffs, prices, or net earnings valid in 2006.
One-hundred and thirty-seven patients were enrolled (median age = 28.3, 3-48 years, 49.6% male) and retrospectively observed for a median of 11.6 months. Mean direct costs were euro1242/patient/month, 55.5% attributable to ICT, 33.2% attributable to transfusions. Relevant quantity and quality of productivity was lost. Both physical and mental components of HRQoL were compromised. Little difficulties remembering to take ICT and positive satisfaction with the perceived effectiveness of therapy were declared, but not good levels of satisfaction with acceptance, perception of side effects and burden of ICT.
The management of beta-TM patients undergoing transfusions and ICT is efficacious, although costly, but overall benefits were not always perceived as optimal by patients. Efforts must be focused to improve patients' acceptance and satisfaction with their therapy; this would contribute to a better compliance and hence an increase in treatment effectiveness and patients' overall wellbeing, with expected improved allocation of human and economic resources.
In 1992, the Measurement and Valuation of Health (MVH) Group at the Centre for Health Economics conducted a study with Social and Community Planning Research (SCPR) comparing different methods of valuing health states (Dolan et al, 1993). A random sample of 335 members of the general population were interviewed in their own homes by specially trained interviewers. Each respondent was asked to value a series of health states using two different valuation methods – Standard Gamble (SG) and Time Trade-Off (TTO). Considerable time and energy went into the production of the protocols for the interviews. Standard methodology (derived primarily from research in Canada and the USA) for both the SG and TTO methods involves the use of specially designed boards and cards. He SG procedure typically uses a ‘probability wheel’ which allows different probabilities of health outcomes to be presented to the respondent, while the TTO typically uses a horizontal sliding scale which allows the length of time spent in a health state to be varied. SG and TTO boards based on the standard methodology were piloted as part of the MVH study and it was found that substantial modifications were required to simplify the material for both the interviewer and the respondent. In addition the standard boards were found to be too large and were difficult to operate. The substantive change made to the SG board during the course of the piloting was the use of a sliding scale rather than a wheel, and a new TTO board was designed so that both sides could be used – one for states considered better than death, and the other for states considered worse than death. A fundamental question arising from the pilot work was the advantage of using props such as boards and cards in the interview. To address this issue, an alternative method of administering the SG and TTO tasks was developed representing a significant departure from the standard methodology. In these modified procedures, the respondent was able to take a much more active role and in fact completed much of the valuation task by him/herself without the use of a board. All four methods performed very well in the main study, to the extent that no single method proved decisively superior to all others from an administrative point of view. Ultimately the choice of method was based on empirical grounds, with the result that the TTO ‘Props’ (with board and cards) was selected as the ‘best’ method for valuing health states in population surveys. Although the MVH Group is now concentrating on the TTO ‘Props’ method in further work, there are certain to be other researchers who want to use the SG method or the TTO in its self-completion form. Thud we want to ensure that all our methods are available to other interested parties in the field of health status measurement. The health states used in this study were based on the EuroQol descriptive system (kind et al, 1994), but these valuation procedures have a general application and can be used for any health state descriptive system. Being aware of the considerable work required in designing and piloting any new methods, we felt that it would be useful if other researchers were able to gain access to a detailed account of the procedures that we had developed. In order to maximise the availability of these designs, we have decided to supplement the initial report describing the piloting and interview design (Thomas and Thomson, 1992) with specific User Guides detailing the four valuation methods. Standard Gamble: Props and Self-completion Time Trade-Off: Props and Self-completion Revisions to the TTO Props method as a result of more recent survey work have also been included. We hope others will be able to pick up from where we have left off, either to make use of the methods in their current form or to modify them further as they wish. In either event we look forward to interest to hearing of the results.
Rosalind Rabin, Frank de Charro, Agota Szende 1.1 Purpose of this booklet Governments and healthcare funders worldwide are making increasing use of economic evaluation to inform priority setting in health care. For various reasons, cost benefit analysis is usually rejected in favour of cost-effectiveness or cost-utility analyses, often involving the estimation of the incremental cost per Quality Adjusted Life Year (QALY) gained (Drummond et al, 2005). The estimation of QALYs gained requires valuations for all relevant health states on a scale anchored at 1 = Full health and 0 = Dead. The EQ-5D is widely used in this context and a number of value sets are available for all the health states generated by the EQ-5D descriptive system. These can be readily applied to health outcomes measured as EQ-5D profiles. EQ-5D has become one of the valuation approaches recommended by several reimbursement authorities and academic bodies in European countries (e.g. The Netherlands, Norway, Italy, Hungary, Poland, Portugal, UK), North America (e.g. Canada), and elsewhere (e.g. New Zealand). The EuroQol Group frequently receives requests for advice regarding EQ-5D valuation data. Those seeking to apply EQ-5D valuations in economic evaluation want to know about the availability of EQ-5D value sets and how they can obtain them. They also seek specific guidance about which of the available value sets they should use for their purposes.
One effect of rising health care costs has been to raise the profile of studies that evaluate care and create a systematic evidence base for therapies and, by extension, for health policies. All clinical trials and evaluative studies require instruments to monitor the outcomes of care in terms of quality of life, disability, pain, mental health, or general well-being. Many measurement tools have been developed, and choosing among them is difficult. This book provides comparative reviews of the quality of leading health measurement instruments and a technical and historical introduction to the field of health measurement, and discusses future directions in the field. This edition reviews over 100 scales, presented in chapters covering physical disability, psychological well-being, anxiety, depression, mental status testing, social health, pain measurement, and quality of life. An introductory chapter describes the theoretical and methodological development of health measures, while a final chapter reviews the current status of the field, indicating areas in which further development is required. Each chapter includes a tabular comparison of the quality of the instruments reviewed, followed by a detailed description of each instrument, covering its purpose and conceptual basis, its reliability and validity, alternative versions and, where possible, a copy of the scale itself. To ensure accuracy, each review has been approved by the original author of each instrument or by an acknowledged expert.
Objective
Factors influencing the choice between endovascular (endovascular aneurysm repair, EVAR) and open repair (OPEN) of abdominal aortic aneurysm (AAA) are of increasing interest. We quantified their importance among the different subjects involved in the treatment.Methods
Pre- and postoperative patients (pts), their relatives and vascular surgeons completed questionnaires evaluating six treatment characteristics: anaesthesia; recovery time to basic everyday activities; risk of re-intervention at 5 years (RR); complexity of follow-up; risk of major complications; and additional cost of intervention (AC). Through a discrete choice experiment, hypothetical scenarios of treatment were obtained and the relative importance (RI) of each characteristic was determined through a conditional logistic regression model.ResultsA total of 160 pts, 102 relatives and 30 surgeons from nine centres completed the questionnaires. Major complications and re-intervention risk were the most important characteristics (RI = 56.0% and 27.2%, respectively) for all the respondent categories. Pts and their relatives considered very important also a possible out-of-pocket AC. Recovery time and type of anaesthesia were among the least important characteristics, including hospital additional cost for surgeons. The different categories of respondents showed different opinions towards different treatment characteristics depending also on possible previous treatment.Conclusion
Preferences for AAA treatment characteristics differ between groups of involved subjects. Understanding individuals’ preferences could help in optimising treatment benefits.
Objectives
While a French language version of the EQ-5D exists, to date, there has been no French value set to accompany it. The objective of our study was then to derive the French TTO value set of the EQ-5D.
Established in 1987, the EuroQol Group initially comprised a network of international, multilingual and multi-disciplinary researchers from seven centres in Finland, the Netherlands, Norway, Sweden and the UK. Nowadays, the Group comprises researchers from Canada, Denmark, Germany, Greece, Japan, New Zealand, Slovenia, Spain, the USA and Zimbabwe. The process of shared development and local experimentation resulted in EQ-5D, a generic measure of health status that provides a simple descriptive profile and a single index value that can be used in the clinical and economic evaluation of health care and in population health surveys. Currently, EQ-5D is being widely used in different countries by clinical researchers in a variety of clinical areas. EQ-5D is also being used by eight out of the first 10 of the top 50 pharmaceutical companies listed in the annual report of Pharma Business (November/December 1999). Furthermore, EQ-5D is one of the handful of measures recommended for use in cost-effectiveness analyses by the Washington Panel on Cost Effectiveness in Health and Medicine. EQ-5D has now been translated into most major languages with the EuroQol Group closely monitoring the process.
Background
National EQ-5D value sets are developed because preferences for health may vary in different populations. UK values are lower than US values for most of the 243 possible EQ-5D health states. Although similar protocols were used for data collection, analytic choices regarding how to model values from the collected data may also influence national value sets. Participants in the UK and US studies assessed the same subset of 42 EQ-5D health states using the time trade-off (TTO) method. However, different methods were used to transform negative values to a range bounded by 0 and −1, and values for all 243 health states were estimated using two different regression models. The transformation of negative values is inconsistent with expected utility theory, and the choice of which transformation method to use lacks a theoretical foundation.
Objectives
Our objectives were to assess how much of the observed difference between the UK and US EQ-5D value sets may be explained by the choice of transformation method for negative values relative to the choice of regression model and the differences between elicited TTO values in the respective national studies (datasets).
Methods
We applied both transformation methods and both regression models to each of the two datasets, resulting in eight comparable value sets. We arranged these value sets in pairs in which one source of difference (transformation method, regression model or dataset) was varied. For each of these paired value sets, we calculated the mean difference between the two matching values for each of the 243 health states. Finally, we calculated the mean utility gain for all possible transitions between pairs of EQ-5D health states within each value set and used the difference in transition scores as a measure of impact from changing transformation method, regression model or dataset.
Results
The mean absolute difference in values was 1.5 times larger when changing the transformation method than when using different datasets. The choice of transformation method had a 3.2 times larger effect on the mean health gain (transition score) than the choice of dataset. The mean health gain in the UK value set was 0.09 higher than in the US value set. Using the UK transformation method on the US dataset reduced this absolute difference to 0.02. The choice of regression model had little overall impact on the differences between the value sets.
Conclusions
Most of the observed differences between the UK and US value sets were caused by the use of different transformation methods for negative values, rather than differences between the two study populations as reflected in the datasets. Changing the regression model had little impact on the differences between the value sets.
Objectives:
Cost-effectiveness analysis has been recommended by national health agencies worldwide. In the United Kingdom, the National Institute of Health and Clinical Excellence supports the use of generic health-related quality of life instruments such as EuroQol EQ-5D when quality-adjusted life-years are used to measure health benefits. Despite the urgent need for appropriate methodologies to improve the use of scarce resources in Latin American countries, little is known about how health is valued.
Methods:
A national population survey was conducted in the United States in 2002, based on a sample of 1603 non-Hispanic nonblacks and 1115 Hispanics. Participants provided time trade-off utilities for a subset of 42 EQ-5D health states. Hispanic respondents were grouped according to their language preferences (Spanish or English). Mean utilities were compared for each health state. A random-effects model was used to determine whether real population differences exist after adjusting for sociodemographic characteristics. A population value set for all 243 EQ-5D health states was developed using only the data from Spanish-speaking Hispanics.
Results:
Mean valuations differed slightly between non-Hispanic nonblacks and English-speaking Hispanics. Spanish-speaking Hispanics, however, tended to give higher valuations than non-Hispanic nonblacks (P < 0.05) corresponding to an average of 0.034 point. A regression model was developed for Spanish-speaking Hispanics with a mean absolute error of 0.031. Values estimated using this model show marked differences when compared with corresponding values estimated using the UK (N3) and US (D1) models.
Conclusion:
The availability of a Hispanic model for EQ-5D valuations represents a significant new option for decision-makers, providing a set of social preference weights for use in Latin American countries that presently lack their own domestic value set.
To estimate a EQ-5D value set for Malaysia by using time trade-off (TTO) and visual analogue scale (VAS) valuation methods.
TTO and VAS valuations were obtained from face-to-face surveys of a convenience sample of patients, caregivers, and health professionals conducted at nine government hospitals in 2004 and 2005. Forty-five EQ-5D questionnaire health states were valued, divided into five sets of 15 health states. Analysis was conducted by using linear additive regression models applying N3 and D1 specifications. Model selection was based on criteria of coefficient properties, statistical significance, and goodness of fit.
One hundred fifty-two respondents were interviewed, yielding 2174 TTO and 2265 VAS valuations. Respondents found TTO valuations to be more difficult than VAS valuations, and there were more inconsistencies in TTO valuations. All the independent variables in the models were statistically significant and consistent with expected signs and magnitude, except for the D1 specification modeled on TTO valuations. The N3 model provided the best fit for the VAS valuation data, with a mean absolute error of 0.032.
This study provides a Malaysian EQ-5D questionnaire value set that can be used for cost-utility studies despite survey limitations.
Cost-effectiveness analysis has been recommended by many national agencies around the world as a valid methodology to improve resource allocation within the health-care system. If the preferences of the society are taken into account in such a decision-making process, it is generally recommended that these values should be elicited by using a generic health-related quality-of-life instrument, such as the EuroQol five-dimensional (EQ-5D) questionnaire.
To estimate a set of social values for EQ-5D questionnaire based on the time trade-off valuation technique for use in Chile.
A valuation questionnaire was applied to a probabilistic sample of 2000 individuals, aged 20 years or older, living in the Metropolitan region. The fieldwork took place during October to November 2008. Utility weights for 42 health states were calculated directly by the application of time trade-off. Several random effect and ordinary least-squares regression models were fitted to these valuations to predict the full set of 243 health states generated by the EQ-5D system. The best model was chosen by applying criteria of parsimony, goodness of fit, and prediction capacity.
The selected regression model was robust and showed better predictive characteristics than others reported in similar studies conducted elsewhere. The chosen regression model showed a R(2) of 0.34, mean absolute error of 0.017, and high predictive capacity.
This study provides an EQ-5D social value set for domestic use in Chile. Our results differ from those reported in other countries, justifying the need to perform local studies that adequately reflect societal health preferences.
To estimate the prevalence of partly controlled and uncontrolled asthmatic patients, to evaluate quality of life and healthcare resource consumption.
Cross-sectional phase followed by a 12-month prospective phase. Asthma Control Test and the EQ-5D were used.
2853 adult patients recruited in 56 Hospital Respiratory Units in Italy were evaluated: 64.4% had controlled asthma, 15.8% partly controlled asthma and 19.8% were uncontrolled. The mean (SD) EQ-5D score was 0.86 (0.17) in controlled, 0.75 (0.20) in partly controlled and 0.69 (0.23) in uncontrolled patients (p<0.001 between groups). The number of patients requiring hospitalization or emergency room visits was lower in controlled (1.8% and 1.6%, respectively) than in partly controlled (5.1% and 11.5%) and uncontrolled (6.4% and 18.6%). A combination of an inhaled corticosteroid and a long-acting beta-2 agonist was the reported therapy by 56.0% of patients, with the rate of controlled asthma and improved quality of life being higher in patients on extrafine beclomethasone/formoterol compared to budesonide/formoterol (p<0.05) and fluticasone/salmeterol (p<0.05 for quality of life).
Asthma control is achieved in a good proportion of Italian patients. Differences may be detected in a real-life setting in favor of extrafine beclomethasone/formoterol combination.
Cost-utility analyses (CUAs) are increasingly common in Australia. The EuroQol five-dimensional (EQ-5D) questionnaire is one of the most widely used generic preference-based instruments for measuring health-related quality of life for the estimation of quality-adjusted life years within a CUA. There is evidence that valuations of health states vary across countries, but Australian weights have not previously been developed.
Conventionally, weights are derived by applying the time trade-off elicitation method to a subset of the EQ-5D health states. Using a larger set of directly valued health states than in previous studies, time trade-off valuations were collected from a representative sample of the Australian general population (n = 417). A range of models were estimated and compared as a basis for generating an Australian algorithm.
The Australia-specific EQ-5D values generated were similar to those previously produced for a range of other countries, but the number of directly valued states allowed inclusion of more interaction effects, which increased the divergence between Australia's algorithm and other algorithms in the literature.
This new algorithm will enable the Australian community values to be reflected in future economic evaluations.
In the original US valuation study of EQ-5D states, all worse-than-dead time trade-off responses (26% of the sample) were divided by 39 to increase the QALY estimates. This transformation has no theoretical justification and motivates this re-examination. Using the publically available dataset, we compared three alternative random utility models: instant (IRUM), angular (ARUM), and episodic (ERUM) models. Each leads to a distinct econometric estimator: mean ratio, ratio of means, and coefficient, respectively. IRUM suggests that 203 of the 243 EQ-5D states are worse-than-dead, which has little face validity compared to ARUM and ERUM (42 and 3 WTD states). ARUM and ERUM estimates are proportionally related such that losses in QALYs are approximately 37% larger under ARUM than ERUM. Compared to ERUM, economic evaluations using ARUM estimates emphasize quality of life, and this difference may influence policy decisions. Either ERUM or ARUM values sets are recommended over the original, transformed set.
Factors influencing the choice between endovascular (endovascular aneurysm repair, EVAR) and open repair (OPEN) of abdominal aortic aneurysm (AAA) are of increasing interest. We quantified their importance among the different subjects involved in the treatment.
Pre- and postoperative patients (pts), their relatives and vascular surgeons completed questionnaires evaluating six treatment characteristics: anaesthesia; recovery time to basic everyday activities; risk of re-intervention at 5 years (RR); complexity of follow-up; risk of major complications; and additional cost of intervention (AC). Through a discrete choice experiment, hypothetical scenarios of treatment were obtained and the relative importance (RI) of each characteristic was determined through a conditional logistic regression model.
A total of 160 pts, 102 relatives and 30 surgeons from nine centres completed the questionnaires. Major complications and re-intervention risk were the most important characteristics (RI = 56.0% and 27.2%, respectively) for all the respondent categories. Pts and their relatives considered very important also a possible out-of-pocket AC. Recovery time and type of anaesthesia were among the least important characteristics, including hospital additional cost for surgeons. The different categories of respondents showed different opinions towards different treatment characteristics depending also on possible previous treatment.
Preferences for AAA treatment characteristics differ between groups of involved subjects. Understanding individuals' preferences could help in optimising treatment benefits.
To develop a set of health state values based on EuroQol EQ-5D instrument for the Argentine general population.
Consecutive subjects attending six primary care centers in Argentina were selected based on quota sampling and were interviewed using the EuroQol Group protocol for measurement and valuation of health studies. Initially, the respondents were randomly assigned a unique card set; however, to improve efficiency, the subjects were later randomly assigned to one of three fixed sets of EQ-5D states. Using the visual analog scale (VAS) and time-trade off (TTO) responses for these states, we estimated a valuation model using ordinary least squares regression clustered by respondent. Predicted values for EQ-5D health states are compared with published values for the United States.
Six hundred eleven subjects were interviewed by 14 trained interviewers, rendering 6887 TTO and 6892 VAS responses. The model had an R(2) of 0.897 and 0.928 for TTO and VAS, respectively. The mean absolute difference between observed and predicted values was 0.039 for TTO and 0.020 for VAS, each showing a Lin's concordance coefficient more than 0.98. Argentine and US TTO-predicted values were highly correlated (Pearson's rho = 0.963), although the average absolute difference was clinically meaningful (0.06), rejecting the US values for nearly two-thirds of the states (62.8%). The Argentine population placed lower values on mild states and higher values on severe states.
This study provides an Argentine value set that could be used locally or regionally, with meaningful and significant differences with that of the United States. Health policy in Latin America must incorporate local values for sovereignty and validity.
This study establishes the South Korean population-based preference weights for EQ-5D based on values elicited from a representative national sample using the time trade-off (TTO) method.
The data for this paper came from a South Korean EQ-5D valuation study where 1307 representative respondents were invited to participate and a total of 101 health states defined by the EQ-5D descriptive system were directly valued. Both aggregate and individual level modeling were conducted to generate values for all 243 health states defined by EQ-5D. Various regression techniques and model specifications were also examined in order to produce the best fit model. Final model selection was based on minimizing the difference between the observed and estimated value for each health state.
The N3 model yielded the best fit for the observed TTO value at the aggregate level. It had a mean absolute error of 0.029 and only 15 predictions out of 101 had errors exceeding 0.05 in absolute magnitude.
The study successfully establishes South Korean population-based preference weights for the EQ-5D. The value set derived here is based on a representative population sample, limiting the interpolation space and possessing better model performance. Thus, this EQ-5D value set should be given preference for use with the South Korean population.
Health policy decisions should be based on national social preferences. In the absence of a set of Danish health preferences, patient outcome studies using the EQ-5D instrument have typically used UK health state valuations. This article describes the development of a Danish EQ-5D value set.
Regression modelling was based on Time Trade-Off (TTO) data derived from computer-assisted interviews conducted with 1,332 respondents from the Danish general population. Using a split-sample technique, 46 health states were directly valued by the respondents. Five different model types were tested and compared on statistical and theoretical grounds. Eleven different specifications were then tested for the chosen model type to identify the most appropriate model that had high explanatory power and parameters that were both consistent (positively signed) and statistically significant.
An additive random effects model was found to be superior to ordinary least squares, fixed effects, random coefficient and censored Tobit modelling approaches. From the 11 model specifications tested, the TTO3 model (main effects model, without an N3 factor) performed best and was used to generate a Danish set of health state preferences.
An additive random effects model appears to adequately generate a Danish set of EQ-5D health state preferences. The model has high explanatory power and produces consistent and significant parameters for EQ-5D dimensions and levels. It is recommended that this value set be used in Danish cost-utility studies using EQ-5D.
An important consideration when establishing priorities in health care is the likely effects that alternative allocations of resources will have on health-related quality-of-life (HRQoL). This paper reports on a large-scale national study that elicited the relative valuations attached by the general public to different states of health (defined in HRQoL terms). Health state valuations were derived using the time trade-off (TTO) method. The data from 3395 respondents were highly consistent, suggesting that it is feasible to use the TTO method to elicit valuations from the general public. The paper shows that valuations for severe health states appear to be affected by the age and the sex of the respondent; those aged 18-59 have higher valuations than those aged 60 or over and men have higher valuations than women. These results contradict those reported elsewhere and suggest that the small samples used in other studies may be concealing real differences that exist between population sub-groups. This has important implications for public policy decisions.
It has become increasingly common for preference-based measures of health-related quality of life to be used in the evaluation of different health-care interventions. For one such measure, The EuroQol, designed to be used for these purposes, it was necessary to derive a single index value for each of the 243 health states it generates. The problem was that it was virtually impossible to generate direct valuations for all of these states, and thus it was necessary to find a procedure that allows the valuations of all EuroQol states to be interpolated from direct valuations on a subset of these.
In a recent study, direct valuations were elicited for 42 EuroQol health states (using the time trade-off method) from a representative sample of the UK population. This article reports on the methodology that was adopted to build up a "tariff" of EuroQol values from this data.
A parsimonious model that fits the data well was defined as one in which valuations were explained in terms of the level of severity associated with each dimension, an intercept associated with any move away from full health, and a term that picked up whether any dimension in the state was at its most severe level.
The model presented in this article appears to predict the values of the states for which there are direct observations and, thus, can be used to interpolate values for the states for which no direct observations exist.
This article examines the twin concepts of the statistical significance and quantitative importance of observed differences in studies comparing medicines in terms of economic parameters such as cost-effectiveness and measures of health-related quality of life (HRQOL). Central to the design and interpretation of any comparative study, such as a randomised controlled trial, is some prior judgement about the order of magnitude of a difference that would make one switch from one therapy to another. Starting with current definitions of clinically important differences we argue by analogy that the importance of differences in HRQOL require a shift of focus from the physician to the patient for preferences and judgements concerning what is important to them. Whether an intervention offers sufficient value for money (cost effectiveness or cost utility) to warrant resources being reallocated to it is a collective decision requiring the input of public preferences about the relative importance of alternative therapies and health benefits. Ultimately, the importance of the health benefits offered by a new drug is revealed by societal willingness-to-pay. This may be stated implicitly through committees using cost-effectiveness 'league tables' for decision making, or explicitly by consumer surveys of willingness-to-pay in the context of cost-benefit analysis and stemming from the theoretical foundation of welfare economics.
The EQ-5D is a multiattribute health status measure that can be used to derive preference-based index scores for health-related quality-of-life (HRQOL) assessment. Valuations for EQ-5D health states using different techniques have been obtained in a number of countries. It is not clear how valuations from different countries compare. Using an ordinary least-squares regression, visual analog scale valuations for EQ-5D health states obtained in postal surveys in Finland and the United States were compared. The regression model estimates indicated that Finnish and U.S. respondents did provide different preference valuations for different levels of health. However, the country-specific differences were not large and depended on the dimension and the level of problem on that dimension. Differences in health-state valuations are unlikely to have important implications when using the EQ-5D in international studies.
Few studies have compared preference values for health states obtained in different countries. The present study compared Spanish and United Kingdom (UK) time trade-off values for EuroQol-5D health states. The same preference elicitation protocol was followed in both countries. Differences in values for 43 health states rated directly were analyzed using t tests, and regression coefficients generated by random effects modeling were compared by aggregating the 2 value sets and using dummy variables to analyze country effect by dimension and level of severity. For the milder health states, Spanish and UK value assignation was similar; for intermediate health states, Spanish values were both higher and lower than UK values, whereas for health states worse than death, UK values were generally higher than Spanish values. There were statistically significant differences (P < 0.01) in values for 34.9% of health states rated directly, and some preference reversals between countries. UK raters ascribed greater importance to dimensions of pain/discomfort and anxiety/depression, whereas Spanish raters placed more importance on functional dimensions of mobility and self-care. Further analysis is required to determine how these differences affect cost-effectiveness and cost-utility analyses.
Quality adjustment weights for quality-adjusted life years (QALYs) are available with the EQ-5D Instrument, which are based on a survey that quantified the preferences of the British public. However, the extent to which this British value set is applicable to other, especially non-European, countries is yet unclear. The objectives of this study are (a) to compare the valuations obtained in Japan and Britain, and (b) to explore a local Japanese value set. A diminished study design is employed, where 17 hypothetical EQ-5D health states are evaluated as opposed to 42 in the British study. The official Japanese version of the instrument and the Time Trade-Off method are used to interview 543 members of the public. The results are: firstly, the evaluations obtained in Japan and those from Britain differ by 0.24 on average on a [-1, +1] scale, and mean absolute error (MAE) in predicting the Japanese preferences with the British value set is 0.23. Secondly, comparable regressions suggest that the two peoples have systematically different preference structures (p<0.001 for 8 of 12 coefficients; F-test). Thirdly, using alternative models, the predictions are improved so that the local Japanese value set achieves MAE in the order of 0.01.
We sought to compare directly elicited valuations for EQ-5D health states between the US and UK general adult populations.
We analyzed data from 2 EQ-5D valuation studies where, using similar time trade-off protocols, values for 42 common health states were elicited from representative samples of the US and UK general adult populations. First, US and UK population mean valuations were estimated and compared for each health state. Second, random-effect models were used to compare the US and UK valuations while adjusting for known predictors of EQ-5D valuations (ie, age, sex, health state descriptors) and to investigate whether and how the valuations differ.
Population mean valuations of the 42 health states ranged from -0.38 to 0.88 for the United States and from -0.54 to 0.88 for the United Kingdom, with the US mean scores being numerically higher than the UK for 39 health states (mean difference: 0.11; range: -0.01 to 0.25). After adjusting for the main effects of known predictors, the average difference in valuations was 0.10 (P < 0.001). The magnitude of the difference in the US and UK valuations was not constant across EQ-5D health states; greater differences in valuations were present in health states characterized by extreme problems.
Meaningful differences exist in directly elicited TTO valuations of EQ-5D health states between the US and UK general populations. Therefore, EQ-5D index scores generated using valuations from the US general population should be used for studies aiming to reflect health state preferences of the US general public.
The EQ-5D is a brief, multiattribute, preference-based health status measure. This article describes the development of a statistical model for generating US population-based EQ-5D preference weights.
A multistage probability sample was selected from the US adult civilian noninstitutional population. Respondents valued 13 of 243 EQ-5D health states using the time trade-off (TTO) method. Data for 12 states were used in econometric modeling. The TTO valuations were linearly transformed to lie on the interval [-1, 1]. Methods were investigated to account for interaction effects caused by having problems in multiple EQ-5D dimensions. Several alternative model specifications (eg, pooled least squares, random effects) also were considered. A modified split-sample approach was used to evaluate the predictive accuracy of the models. All statistical analyses took into account the clustering and disproportionate selection probabilities inherent in our sampling design.
Our D1 model for the EQ-5D included ordinal terms to capture the effect of departures from perfect health as well as interaction effects. A random effects specification of the D1 model yielded a good fit for the observed TTO data, with an overall R of 0.38, a mean absolute error of 0.025, and 7 prediction errors exceeding 0.05 in absolute magnitude.
The D1 model best predicts the values for observed health states. The resulting preference weight estimates represent a significant enhancement of the EQ-5D's utility for health status assessment and economic analysis in the US.
The objective of this study was to estimate a Dutch EQ-5D tariff and to determine in a simulation study using the dataset of the original UK valuation study, the number of health states and respondents needed to estimate a reliable tariff. In all, 300 Dutch respondents directly valued 17 states compared to 3000 respondents and 42 states in the original MVH protocol. The results reaffirmed differences in health-related preferences between countries, justifying the estimation of national tariffs. The mean absolute error was 0.030. The design of this study is recommended for national EQ-5D valuation studies.
Utilities for health are measured on an interval scale, where 1 refers to full health and 0 refers to death. No theoretical lower boundary on the utilities for states worse than death exists. As a consequence, negative values receive greater weight in the calculation of mean utilities. To avoid this, negative values often are bound at -1.
The objective of this study was to compare the effect of 3 methods to bound negative values at -1 on the estimation of EQ-5D value sets: truncation, monotonic, and linear transformation.
Data of the Dutch EQ-5D valuation study were used. A total of 298 respondents directly valued 17 EQ-5D health states using the time trade-off (TTO) method. Random effects regression analysis was used to interpolate TTO values for all possible EQ-5D states. In the regression analysis the dependent variable is 1 minus the TTO value and the independent variables describe the health state. Two widely used models to estimate EQ-5D value were applied after truncation of negative values and monotonic and linear transformation of negative values. Both models also were estimated on medians.
Truncation of negative values gave the largest mean absolute error (MAE); the linear transformation resulted in the smallest MAE. When medians were used for estimation, the MAEs were comparable with the estimation on means.
The choice of a method to bound negative values is arbitrary and affects the resulting value set. For the estimation of EQ-5D value sets from a societal perspective the use of medians should be considered.
To estimate quality weights of EQ-5D health states with the time trade-off (TTO) method in the general population of South Korea.
A total of 500 respondents valued 42 hypothetical EQ-5D health states using the TTO and visual analog scale. The quality weights for all EQ-5D health states were estimated by a random effects model and compared with those from studies in other countries.
Overall estimated quality weights for all EQ-5D health states from this study were highly correlated with those from previous studies, but quality weights of individual states were substantially different from those of their corresponding states in other studies.
The Korean value set differed from value sets from other countries. Special caution is needed when a value set from one country is applied to another with a different culture.
Value sets for the EQ-5D-5L: a mapping approach
- B Vanhout
- Mf Janssen
- Ys Feng
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