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Colorectal cancer (CRC) screening tests differ in benefits, harms, and processes, making individual informed decisions preference based. The objective was to analyze the preferences of insurees in Germany for characteristics of CRC screening modalities. A generic discrete choice experiment with 2-alternative choice sets and 6 attributes (CRC mortality, CRC incidence, complications, preparation, need for transportation, and follow-up; 3 levels each) depicting characteristics of fecal testing, sigmoidoscopy, and colonoscopy was generated. Participants completed 8 choice tasks. Internal validity was tested using a within-set dominated pair. Between June and October 2020, written questionnaires were sent to a stratified random sample (n = 5000) of 50-, 55-, and 60-year-old insurees of the AOK (Allgemeine Ortskrankenkasse) Lower Saxony, who had previously received an invitation to participate in the organized screening program including evidence-based information. Preferences were analyzed using conditional logit, mixed logit, and latent-class model. From 1282 questionnaires received (26% [1282 of 4945]), 1142 were included in the analysis. Approximately 42% of the respondents chose the dominated alternative in the internal validity test. Three heterogeneous preference classes were identified. Most important attributes were preparation (class 1; n = 505, 44%), CRC mortality (class 2; n = 347, 30%), and CRC incidence (class 3; n = 290, 25%). Contrary to a priori expectations, a higher effort was preferred for bowel cleansing (class 1) and accompaniment home (classes 1 and 2). Internal validity issues of choice data need further research and warrant attention in future discrete choice experiment surveys. The observed preference heterogeneity suggests different informational needs, although the underlying reasons remained unclear. • This discrete choice experiment on characteristics of colorectal cancer screening tests revealed significant heterogeneous preferences among German insurees. Three groups were identified: the largest group valued—unexpectedly—a process parameter the most, that is, comprehensive bowel preparation, whereas the other 2 groups focused on potential benefits, that is, either reduction in cancer-specific mortality or incidence. • The experiment included a within-set dominated pair for validity testing. A total of 42% chose the dominated alternative (type B respondents) and revealed significantly different preferences than type A respondents. The underlying reasons and individuals' considerations remain unknown. Internal validity issues of choice data warrant adequate attention in future discrete choice experiments. • Observed preference heterogeneity suggests different informational needs that are to be addressed by public health and healthcare services to support everyone regardless of their health literacy level in making individual informed decisions in the context of colorectal cancer screening by weighing tests' benefits, harms, and processes. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to compare discriminant validity evidence of 2 generic patient-reported outcome measures (PROMs), the Veterans RAND 12-Item Health Survey (VR-12) and level 5 of EQ-5D (EQ-5D-5L), for use in emergency departments (EDs).Methods: Data were obtained via a cross-sectional survey of 5876 patients in British Columbia (Canada) who completed a questionnaire after visiting an ED in 2018. We compared the extent to which the VR-12 and the EQ-5D-5L distinguished among groups of ED patients with different levels of comorbidity burden and self-reported physical and mental or emotional health status. Multivariable logistic regression was used to evaluate the ability of the 2 PROMs to identify patients presenting with a mental health (MH) condition.Results: All the measures produced small effect sizes (ESs) for discriminating comorbidity levels (R2 range: 0.00 [VR-12 mental component summary {MCS}] to 0.10 [VR-12 physical component summary score]). The EQ-5D visual analog scale offered the largest ES for discriminating self-reported physical health (R2 = 0.48), whereas the MCS, the VR-12 MH domain, and the EQ-5D-5L anxiety/depression dimension had the largest ESs for discriminating self-reported mental or emotional health (R2 = 0.42, 0.40, and 0.38, respectively). The MCS produced a medium ES (R2 = 0.42) along with the VR-12 utility score (R2 = 0.27) compared with the EQ-5D-5L index (R2 = 0.19). Having a MH condition was predominantly identified by the MCS (Pratt index = 0.56).Conclusions: The VR-12 PROM provides a more comprehensive measurement of MH than the EQ-5D-5L, which is important to inform healthcare service needs for patients who present in EDs with MH challenges. [ABSTRACT FROM AUTHOR]

To introduce the garbage class mixed logit (MIXL) model as a convenient alternative to manually screening and accounting for respondents with low data quality in discrete choice experiments. Garbage classes are typically used in latent class logit analyses to designate or identify group(s) of respondents with low data quality. Yet, the same concept can be applied to MIXL models as well. Based on a reanalysis of 4 discrete choice experiments that were originally analyzed using a standard MIXL model, it is shown that garbage class MIXL models can achieve the same effect as manually screening for (and excluding) respondents with low data quality based on the more commonly used root likelihood test, but with less effort and ambiguity. Including a garbage class in MIXL models removes the influence of respondents with a random choice pattern from the MIXL model estimates, provides an estimate of the number of low-quality respondents in the dataset, and avoids having to manually screen for respondents with low data quality based on internal or statistical validity tests. Although less versatile than the combination of standard MIXL estimates with separate assessments of data quality and sensitivity analyses, the proposed garbage class MIXL model provides an attractive alternative. [ABSTRACT FROM AUTHOR]

A frequently used patient-reported outcome measure for assessing physical functioning in patients with hip problems is the 5-item Hip Disability and Osteoarthritis Outcome Score-Physical Function Shortform (HOOS-PS). Nevertheless, its content validity (whether this instrument adequately reflects the construct of physical functioning) is unknown. This study aimed to assess the content validity of the HOOS-PS. A quantitative and qualitative research approach was used. Physical functioning was defined as the ability to perform activities that require physical actions, ranging from self-care to more complex activities that require a combination of skills, often within a social context. Patients (n = 51) and experts (n = 25) completed questionnaires regarding the relevance, comprehensiveness, and comprehensibility of the HOOS-PS. Semistructured interviews (n = 5) explored issues identified in the quantitative data in more depth. Thematic content analysis was conducted using a coding frame. One of the 5 items was considered relevant to measure physical functioning. Comprehensiveness was considered insufficient by both patients and experts. Furthermore, comprehensibility was considered inadequate. Several items were found ambiguous or double barreled. Regarding interpretability, floor or ceiling effects were not found. This study showed concerns about the content validity of the HOOS-PS: most items are considered not relevant, the HOOS-PS is considered not comprehensive, and several items are considered not comprehensible. These findings challenge the applicability of the HOOS-PS in clinical practice, research, value-based healthcare, and benchmarking. • This study showed that the content validity of the Hip Disability and Osteoarthritis Outcome Score-Physical Function Shortform (HOOS-PS) in patients undergoing hip arthroplasty is problematic in 3 areas: (1) lack of relevance, (2) insufficient comprehensiveness, and (3) insufficient comprehensibility of the items. • Given that the HOOS-PS does not reflect physical functioning in total hip arthroplasty patients, this will challenge the applicability of the HOOS-PS in clinical practice, research, and benchmarking. • The lack of content validity in the observed data can subsequently lead to drawing incorrect conclusions. [ABSTRACT FROM AUTHOR]

This study aimed to perform a systematic review of published evidence on the psychometric properties of 3-level version of EQ-5D-Y and 5-level version of EQ-5D-Y (EQ-5D-Y-5L). A literature search on the MEDLINE, Embase, and EuroQol website (until June 2021) was conducted. Original studies on EQ-5D-Y psychometric properties such as feasibility, distribution properties (ceiling and floor effects), reliability (test-retest, interrater, intermodal), validity (known-groups, convergent), and responsiveness, published as full-text articles in English, were included. Studies on experimental EQ-5D-Y versions were excluded. The following data were pooled using random effects models: missing values, the ceiling effect, and correlations coefficients with other measures. A total of 47 studies (inclusive of 7 on EQ-5D-Y-5L) containing data from 45 310 children and 2690 proxy respondents representing 15 countries were included. These studies were characterized as being high quality according to the quality index. The most represented areas were school populations and musculoskeletal diseases and orthopedics. The EQ-5D-Y dimensions, EQ visual analog scale, and EQ index were reported in 89%, 77%, and 26% of studies, respectively. Most articles addressed validity (known-groups, n = 27; convergent, n = 21) and reliability (test-retest and interrater, n = 10 each). Convergent validity studies showed that, where the assessment of the child's functioning at school is required, EQ-5D-Y should be supplemented with other school-specific measures. This systematic review provides a summary of measurement properties and the psychometric performance of 3-level version of EQ-5D-Y and EQ-5D-Y-5L. The existing evidence supports using the EQ-5D-Y descriptive system and EQ visual analog scale in children and adolescent populations. Further research on test-retest reliability and the responsiveness of the EQ-5D-Y index obtained with child-specific value sets is needed. • The EQ-5D-Y is a generic, self-complete health status instrument, which was developed in 2010 to measure the health-related quality of life of children and adolescents. • A systematic review was conducted to identify published evidence on the psychometric properties (including validity, reliability, and responsiveness) of the 3-level version of EQ-5D-Y and 5-level version of EQ-5D-Y questionnaires. • We identify the research gaps, such as the evidence of reliability and responsiveness of the EQ-5D-Y index estimated based on child-specific value sets. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to identify the most commonly used internal validity tests in the discrete choice experiment (DCE) literature and establish their sensitivity and specificity.Methods: A structured literature review of recent DCE articles (2018-2020Q1) published in the health, marketing, transport economics, and environmental science literature was used to identify commonly used internal validity tests. The 2 most frequently used internal validity tests were incorporated in 4 new data collections. Respondent preferences in each application were summarized using a mixed logit model, which served as the benchmark for the subsequent sensitivity and specificity calculations. The performance of the internal validity tests was also compared with that of the root likelihood (RLH) test, which is a likelihood-based statistical validity test that is commonly used in marketing applications.Results: Dominant and repeated choice tasks were most commonly included in health-related DCE designs. Based on 4 applications, their specificity and sensitivity depend on the type of incorrect response pattern to be detected and on design characteristics such as the number of choice options per choice task and the number of internal validity tests as included in the experimental design. In all but one scenario, the performance of the dominant and repeated choice tasks was considerably worse than that of the RLH test.Conclusions: Dominant and repeated choice tasks are unreliable screening tests and costly in terms of statistical power. The RLH test, which is a statistical test that does not require additional choice tasks to be included in the DCE design, provides a more reliable alternative. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to present the face validity and psychometric stages performed in Spanish in Argentina, the only Spanish-speaking country of an international collaboration that undertook the construction of a new measure that can be used in economic evaluation across health, social care, and public health, the EQ EQ-HWB (EQ Health and Wellbeing). We also explored the relationship among 3-level version EQ-5D (EQ-5D-3L), 5-level version EQ-5D (EQ-5D-5L), and EQ-HWB.Methods: Face validity was based on semistructured face to face interviews of a purposive sample to explore translatability of language and concepts of 97 candidate items, translated into Argentina Spanish. The psychometric evaluation using an online panel assessed the psychometric properties of 64 items that were carried forward (floor and ceiling effects, item correlations, known-group differences in relevant prespecified subgroups by the international and local teams, exploratory and confirmatory factor analysis, and item response theory). EQ-5D-3L, EQ-5D-5L, and EQ-HWB correlations were explored.Results: In the face validity stage, 24 interviews with carers, general public, patients, and users of social services were included. Most items showed adequate face validity. In the psychometric assessment, 497 participants were recruited (64% reporting a long-term health condition). Most of the items showed adequate psychometrics in an Argentinian context. EQ-5D-3L and EQ-5D-5L had strong correlations, and EQ-HWB was moderately correlated to EQ visual analog scale. The Argentina team recommended 23 of the final 25 items.Conclusions: The assessment of Spanish items contributed to the overall development of EQ-HWB and helped inform the design of an internationally relevant 25-item and a short 9-item measure intended to be used in economic evaluations. [ABSTRACT FROM AUTHOR]

Objectives: The development of measures such as the EQ-HWB (EQ Health and Wellbeing) requires selection of items. This study explored the psychometric performance of candidate items, testing their validity in patients, social carer users, and carers.Methods: Article and online surveys that included candidate items (N = 64) were conducted in Argentina, Australia, China, Germany, United Kingdom, and the United States. Psychometric assessment on missing data, response distributions, and known group differences was undertaken. Dimensionality was explored using exploratory and confirmatory factor analysis. Poorly fitting items were identified using information functions, and the function of each response category was assessed using category characteristic curves from item response theory (IRT) models. Differential item functioning was tested across key subgroups.Results: There were 4879 respondents (Argentina = 508, Australia = 514, China = 497, Germany = 502, United Kingdom = 1955, United States = 903). Where missing data were allowed, it was low (UK article survey 2.3%; US survey 0.6%). Most items had responses distributed across all levels. Most items could discriminate between groups with known health conditions with moderate to large effect sizes. Items were less able to discriminate across carers. Factor analysis found positive and negative measurement factors alongside the constructs of interest. For most of the countries apart from China, the confirmatory factor analysis model had good fit with some minor modifications. IRT indicated that most items had well-functioning response categories but there was some evidence of differential item functioning in many items.Conclusions: Items performed well in classical psychometric testing and IRT. This large 6-country collaboration provided evidence to inform item selection for the EQ-HWB measure. [ABSTRACT FROM AUTHOR]

Objectives: The Michigan Hand Questionnaire (MHQ) is a commonly used evaluation for hand problems, but previous work reports conflicting evidence regarding the subscale structures. Rasch analysis uses probabilistic modeling of items and responses: if scale items can be fit to the Rasch model, it provides evidence of construct validity and interval-level measurement for precise statistical estimates. We conducted Rasch analysis on the MHQ to evaluate model fit, unidimensionality of the subscales, bias across person factors, and conversion to interval metrics.Methods: We conducted a secondary Rasch analysis of MHQ data from 924 persons with thumb basal joint osteoarthritis using the RUMM2030 software. Modeling was based on responses for the most affected hand and person factors including age, sex, type of work, whether the dominant side was the most affected, and surgical status. The analysis plan followed the published recommendations for examinations of person and item fit, with iterative adjustments as required.Results: A total of 11 of the 37 items required rescoring to create orderly progression of scoring thresholds. Only the overall hand function and pain subscales could be fit to the Rasch model, demonstrating unidimensionality and good reliability of fit estimates. Dividing the activities of daily living subscale into unilateral and bilateral activities also allowed unilateral activities to fit the model. Persistent misfitting in other subscales suggested local dependency and response bias across multiple person factors.Conclusions: This Rasch analysis of the MHQ raises concerns regarding the validity and fundamental measurement properties of this widely used outcome evaluation when used as a summary score. [ABSTRACT FROM AUTHOR]

Objectives: The study aimed to assess the reliability and validity of EQ-5D-5L-Y and to compare the performance of EQ-5D-5L-Y with EQ-5D-3L-Y in children and adolescents.Methods: The Spanish versions of the 3L and 5L of EQ-5D for youths, were administered to children and adolescents from the general population. Feasibility and reliability were determined for the EQ-5D-5L-Y. The EQ-5D-5L-Y and EQ-5D-3L-Y were evaluated in terms of ceiling effects, informativity, and correlations with other generic measurements of health-related quality of life.Results: A total of 714 healthy children and adolescents (10.7 ± 2.1 years old) from the general population participated in the study. Most of the sample reported full health status. The feasibility and reliability for the EQ-5D-5L-Y were acceptable, but the questionnaire showed a low convergent validity. Absolute informativity (Shannon index) showed a slight increase in all dimensions of the 5L compared with the 3L; nevertheless, there were only statistically significant differences between 5L and 3L in the dimension "feeling worried, sad, or unhappy" and also on the overall system. Relative informativity (Shannon evenness index) showed a decrease in the 5L compared with 3L for all dimensions, except for "looking after myself." Correlations with other health measurements, in both 3L and 5L, showed similar results to those observed in the international EQ-5D-3L-Y validation study.Conclusion: The results show that EQ-5D-5L-Y is feasible, consistent, and reliable, but there are minor differences in the ceiling effect and informativity between the EQ-5D-5L-Y and EQ-5D-3L-Y versions in the general population. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to develop and assess the content validity of a patient-reported outcomes (PROs) instrument to measure symptoms and impacts experienced by patients with active multiple myeloma (MM).Methods: The PRO instrument was developed using an iterative, mixed-methods approach. The list of concepts was generated based on a review of existing evidence (qualitative studies and literature) and post hoc psychometric evaluations of 2 PRO instruments in 3 clinical trials. A total of 30 adult patients with MM from the United States participated in hybrid concept elicitation/cognitive debriefing interviews to validate the content validity of the newly developed PRO instrument. Translatability assessment was completed in 8 languages.Results: The item generation process resulted in 17 symptom and 9 impact concepts for evaluation. The concept elicitation interviews and analysis were based on the first 25 participants; evidence of saturation was observed. The cognitive debriefing interviews and analysis were based on the last 23 participants across 4 waves of interviews. On the basis patient feedback, 10 items were removed, and 1 item was added to the PRO instrument. The translatability assessment resulted in 1 minor revision. The multiple myeloma symptom and impact questionnaire (MySIm-Q) includes 11 symptom and 6 impact concepts, organized within 8 hypothesized subdomains, with each concept measured using a 5-point verbal rating scale and a 7-day recall period.Conclusions: The MySIm-Q instrument was developed using rigorous and mixed methodology and with direct input from patients who received a diagnosis of MM. The MySIm-Q has good content validity and is culturally relevant for use in global clinical trials. [ABSTRACT FROM AUTHOR]

Objectives: This study aimed to synthesize and evaluate published evidence on the measurement properties of the EQ VAS, a component of all EQ-5D questionnaires.Methods: This systematic review followed the Consensus-Based Standards for the Selection of Health Measurement Instruments guidelines. Five electronic databases were searched for EQ-5D-3L and EQ-5D-5L validation articles published from January 1, 2009, to November 5, 2019. Evidence for construct validity, test-retest reliability, and responsiveness was extracted from individual studies before being aggregated for evaluation of the populations represented by the studied samples. Multivariable logistic meta-regression was conducted to explore the effects of potential covariates on construct validity.Results: A total of 50 articles containing 488 studies, using samples drawn from 12 different populations, were identified. Generally, the quality of evidence was high for construct validity studies (n = 397) but only moderate for both test-retest reliability studies (n = 21) and responsiveness studies (n = 70). "Sufficient" construct validity of EQ VAS was found in 8 of 12 populations, "sufficient" test-retest reliability was found in 3 of 11 populations, and "sufficient" responsiveness was found in 5 of 12 populations. Meta-regression analyses suggested that construct validity studies from the Asian-Pacific region were more likely to show a negative rating compared with studies from Europe and North America.Conclusion: The EQ VAS exhibits "sufficient" construct validity, "inconsistent" test-retest reliability, and "inconsistent" responsiveness across a broad range of populations. Additional studies are needed to explore the suboptimal validity of the EQ VAS in the Asian-Pacific region, whereas more high-quality validation studies are needed to assess its reliability and responsiveness. [ABSTRACT FROM AUTHOR]

Objectives: The standard quality-adjusted life year (QALY) model (SQM) assumes time-utility independence within constant health states and additive independence when health varies over time. The validity of SQM has been challenged through reported violations of these assumptions. An alternative approach that relaxes these assumptions is to assign a single valuation to an entire health profile: an integral assessment of disease severity over time. Here, we compare SQM with the annual profile model (APM) and test SQM for additive independence.Methods: Eighty-two respondents valued 6 episodic conditions, including 4 of short duration, with SQM and APM, using the time trade-off method. Inter-rater reliability was assessed using intraclass correlation coefficients. Face validity was tested by asking respondents how well they were able to imagine the health states under SQM and APM. We calculated SQM QALY values for a 1-year time period, allowing for a direct comparison with APM values. For the short-term conditions we expected higher QALY values for SQM, violating additive independence.Results: APM showed higher interrater reliability (intraclass correlation coefficient of 0.53 vs 0.18, respectively) and better face validity than SQM, with 6% (APM) vs 21% (SQM) of all respondents reporting difficulties. Additive independence of SQM was violated in 5 of the 6 conditions (including the 4 short duration health states), with higher QALY values under SQM (mean difference 0.04).Conclusion: The impact of short-term conditions is systematically underestimated under SQM when compared to a health profile model. APM is a less restrictive model and demonstrates better validity. [ABSTRACT FROM AUTHOR]

Objective: This review examined the psychometric performance of 4 generic child- and adolescent-specific preference-based measures that can be used to produce utilities for child and adolescent health.Methods: A systematic search was undertaken to identify studies reporting the psychometric performance of the Child Health Utility (CHU9D), EQ-5D-Y (3L or 5L), and Health Utilities Index Mark 2 (HUI2) or Mark 3 (HUI3) in children and/or adolescents. Data were extracted to assess known-group validity, convergent validity, responsiveness, reliability, acceptability, and feasibility. Data were extracted separately for the dimensions and utility index where this was reported.Results: The review included 76 studies (CHU9D n = 12, EQ-5D-Y-3L n = 20, HUI2 n = 26,HUI3 n = 43), which varied considerably across conditions and sample size. EQ-5D-Y-3L had the largest amount of evidence of good psychometric performance in proportion to the number of studies examining performance. The majority of the evidence related to EQ-5D-Y-3L was based on dimensions. CHU9D was assessed in fewer studies, but the majority of studies found evidence of good psychometric performance. Evidence for HUI2 and HUI3 was more mixed, but the studies were more limited in sample size and statistical power, which was likely to have affected performance.Conclusions: The heterogeneity of published studies means that the evidence is based on studies across a range of countries, populations and conditions, using different study designs, different languages, different value sets and different statistical techniques. Evidence for CHU9D in particular is based on a limited number of studies. The findings raise concerns about the comparability of self-report and proxy-report responses to generate utility values for children and adolescents. [ABSTRACT FROM AUTHOR]

Objectives: To compare the psychometric properties of the Adult Social Care Outcomes Toolkit for carers (ASCOT-Carer), the Carer Experience Scale (CES), and the Care-related Quality of Life (CarerQol) to inform the choice of instrument in future studies.Methods: Data were derived from a 2018 online survey of informal carers in Australia. Reliability was assessed via internal consistency (Cronbach alpha, α) and test-retest reliability (intraclass correlation coefficient, ICC) for respondents who self-reported no change in their quality of life as a carer over 2 weeks. Convergent validity was evaluated via predetermined hypotheses about associations (Spearman's rank correlation) with existing, validated measures. Discriminative validity was assessed based on the ability of the carer-related scores to distinguish between different informal care situations (Mann-Whitney U, Kruskal-Wallis one-way analysis of variance).Results: Data from 500 carers were analyzed. The ASCOT-Carer demonstrated a higher degree of internal consistency, possibly due to a unidimensional structure, and test-retest reliability than the CarerQol and CES (α = 0.87, 0.65, 0.59; ICC, 0.87, 0.67, 0.81, respectively). All 3 instruments exhibited convergent validity and detected statistically significant associations between carer-related scores and different informal care situations, except for the CarerQol-7D and sole carer status.Conclusions: The ASCOT-Carer, CarerQol, and CES performed reasonably well psychometrically; the ASCOT-Carer exhibited the best psychometric properties overall in this sample of Australian informal carers. Findings should be used in conjunction with consideration of research goals, carer population, targeted carer-related constructs, and prevailing perspectives on the economic evaluation to inform choice of instrument in future studies. [ABSTRACT FROM AUTHOR]

Objectives: Hospital comparisons to improve quality of care require valid and reliable quality indicators. We aimed to test the validity and reliability of 6 breast cancer indicators by quantifying the influence of case-mix and random variation.Methods: The nationwide population-based database included 79 690 patients with breast cancer from 91 Dutch hospitals between 2011 and 2016. The indicator-scores calculated were: (1) irradical breast-conserving surgery (BCS) for invasive disease, (2) irradical BCS for ductal carcinoma-in-situ, (3) breast contour-preserving treatment, (4) magnetic resonance imaging (MRI) before neo-adjuvant chemotherapy, (5) radiotherapy for locally advanced disease, and (6) surgery within 5 weeks from diagnosis. Case-mix and random variation adjustments were performed by multivariable fixed and random effect logistic regression models. Rankability quantified the between-hospital variation, representing unexplained differences that might be the result of the level of quality of care, as low (<50%), moderate (50%-75%), or high (>75%).Results: All of the indicators showed between-hospital variation with wide (interquartile) ranges. Case-mix adjustment reduced variation in indicators 1 and 3 to 5. Random variation adjustment (further) reduced the variation for all indicators. Case-mix and random variation adjustments influenced the indicator-scores of individual hospitals and their ranking. Rankability was poor for indicator 1, 2, and 5, and moderate for 3, 4, and 6.Conclusions: The 6 indicators lacked validity and/or reliability to a certain extent. Although measuring quality indicators may stimulate quality improvement in general, comparisons and judgments of individual hospital performance should be made with caution if based on indicators that have not been tested or adjusted for validity and reliability, especially in benchmarking. [ABSTRACT FROM AUTHOR]

Objectives: To improve quality in breast cancer care, large numbers of quality indicators are collected per hospital, but benchmarking remains complex. We aimed to assess the validity of indicators, develop a textbook outcome summary measure, and compare case-mix adjusted hospital performance.Methods: From a nationwide population-based registry, all 79 690 nonmetastatic breast cancer patients surgically treated between 2011 and 2016 in 91 hospitals in The Netherlands were included. Twenty-one indicators were calculated and their construct validity tested by Spearman's rho. Between-hospital variation was expressed by interquartile range (IQR), and all valid indicators were included in the summary measure. Standardized scores (observed/expected based on case mix) were calculated as above (>100) or below (<100) expected. The textbook outcome was presented as a continuous and all-or-none score.Results: The size of between-hospital variation varied between indicators. Sixteen (76%) of 21 quality indicators showed construct validity, and 13 were included in the summary measure after excluding redundant indicators that showed collinearity with others owing to strong construct validity. The median all-or-none textbook outcome score was 49% (IQR 42%-54%) before and 49% (IQR 48%-51%) after case-mix adjustment. From the total of 91 hospitals, 3 hospitals were positive (3%) and 9 (10%) were negative outliers.Conclusions: The textbook outcome summary measure showed discriminative ability when hospital performance was presented as an all-or-none score. Although indicator scores and outlier hospitals should always be interpreted cautiously, the summary measure presented here has the potential to improve Dutch breast cancer quality indicator efforts and could be implemented to further test its validity, feasibility, and usefulness. [ABSTRACT FROM AUTHOR]

Objectives: Carer quality-of-life (QoL) effects are recommended for inclusion in economic evaluations, but little is known about the relative performance of different types of QoL measures with carers. This study evaluated the validity and responsiveness of 3 care-related QoL measures (the Carer Experience Scale [CES], CarerQoL-7D, and ASCOT-Carer), 1 health-related QoL measure (the EQ-5D-5L), and 1 generic QoL measure (the ICECAP-A).Methods: Validity and responsiveness were assessed in a UK sample of informal carers of adults with dementia, stroke, mental illness, or rheumatoid arthritis. A questionnaire containing the 5 QoL measures was posted to carers identified through the Family Resources Survey (N = 1004). Hypotheses regarding the anticipated associations between constructs related to the QoL of carers were tested to investigate construct validity and responsiveness.Results: Each measure exhibited some level of construct validity. In general, larger effect sizes and stronger associations were detected for the ASCOT-Carer and ICECAP-A measures in the pooled sample and across all conditions. The 5 measures did not exhibit clear responsiveness to changes over a 12-month period in care recipient health status or hours of care provided per week.Conclusion: The results of this study provide initial evidence of the validity of care-related, health-related, and generic QoL (capability) measures in informal carers of adults with 4 highly prevalent conditions. Care-related measures were not always more sensitive to constructs associated with QoL of carers compared with generic measures. The performance of the ICECAP-A was comparable with that of the best-performing care-related measure, the ASCOT-Carer. [ABSTRACT FROM AUTHOR]

Background: The Institute for Medical Technology Assessment Productivity Cost Questionnaire (iPCQ) was recently developed to cover all domains of productivity costs; absenteeism, presenteeism and productivity costs related to unpaid work. The original iPCQ has not been tested with respect to neither content or construct validity, nor reliability, and there is no Norwegian version of the questionnaire.Objectives: To translate and cross-culturally adapt the iPCQ into Norwegian and to test its measurement properties among patients with musculoskeletal disorders.Methods: Translation and cross-cultural adaptation was conducted according to guidelines, and measurement properties were investigated using a cross-sectional design including a test-retest assessment. Patients with musculoskeletal disorders were recruited from secondary care. Data quality, content validity (10 patients evaluated comprehensibility, 2 researchers and 1 clinician evaluated relevance and comprehensiveness), construct validity (factor analysis, internal consistency, divergent hypothesis testing), and test-retest reliability (intraclass correlation coefficient two-way random average agreement, Cohen's unweighted kappa) were assessed.Results: In total, 115 patients with a mean age (SD) of 46 (9) years were included, and 62 responded to the retest. The questionnaire was feasible, with little missing data and no floor or ceiling effects. Content validity displayed good comprehensibility and relevance and sufficient comprehensiveness. Factor analysis revealed a 3-component solution accounting for 82% of the total variance; items loaded as expected and supported the original structure of the iPCQ. Internal consistency was acceptable for the 3 components of productivity cost, with an inter-item correlation ranging from 0.42 to 0.62. Further, a total of 91% of our hypotheses were verified. The intraclass correlation coefficient values ranged from 0.88 to 0.99 for all items except one; kappa ranged from 0.61 to 0.92, indicating overall good reliability of the questionnaire.Conclusions: The Norwegian iPCQ showed good measurement properties among patients with musculoskeletal disorders from secondary care in Norway. We therefore recommend the iPCQ as a useful tool for measuring productivity costs in patients with musculoskeletal disorders. [ABSTRACT FROM AUTHOR]

Background: The Symptoms of Major Depressive Disorder Scale (SMDDS) was expressly developed on the basis of qualitative data to directly incorporate patients' voices into evaluation of treatment benefit in major depressive disorder (MDD) clinical trials.Objectives: To collect quantitative data necessary to refine/optimize the SMDDS and document its psychometric properties.Methods: In this multicenter, observational study, participants with clinically diagnosed MDD completed questionnaires in 2 waves. Wave 1 was designed to refine the SMDDS using Rasch measurement evaluations and item reduction analyses. On a subset of wave 1 subjects, 7 to 12 months later, wave 2 further examined item performance and measurement properties. Exploratory factor analyses and assessments of construct validity and reliability (internal consistency and reproducibility) were completed.Results: Using wave 1 data (N = 315; females = 71%, white = 81%, mean age = 44 years), the SMDDS was revised from 36 to 16 items. The Rasch item threshold map indicated that all but 1 item (suicidal ideation) were appropriately ordered. The 207 wave 2 participants were 74% females, 82% white, with a mean age of 45 years. The exploratory factor analyses resulted in a single component (all standardized factor loadings >0.46). Cronbach α was 0.93 and the 7-day test-retest intraclass correlation coefficient (n = 93) was 0.84 (95% confidence interval 0.77-0.89). SMDDS scores discriminated between MDD severity levels.Conclusions: The 16-item SMDDS generated highly reliable scores with substantial evidence of construct validity. On the basis of the evidence of appropriate content validity and sound psychometric performance, the Food and Drug Administration qualified the SMDDS as an outcome measure to support exploratory efficacy endpoints in MDD clinical trials. [ABSTRACT FROM AUTHOR]

Background: New therapies in development for lowering low-density lipoprotein cholesterol, such as alirocumab, require administration by subcutaneous injections. There is a need to assess the acceptance of such treatments and their mode of administration.Objectives: To develop a novel patient-reported outcome measure, the Injection-Treatment Acceptance Questionnaire (I-TAQ), and assess its content validity using qualitative methods.Methods: Concepts generated from a literature and instrument review informed the initial drafting of 17 items in the I-TAQ, with item wording adapted from three existing instruments. Three rounds of qualitative interviews were conducted with 29 US-English speaking patients at high cardiovascular risk. Concept elicitation questioning was used to explore patients' treatment experiences followed by cognitive debriefing of the I-TAQ using "think-aloud" methods. Verbatim transcripts were analyzed using thematic analysis.Results: Qualitative analysis of concept elicitation data identified the following relevant concepts: perceived efficacy, side effects, self-efficacy, convenience, and overall acceptance. Seven (24%) patients discussed an initial fear of needles, but described this as subsiding with no impact on adherence. Five items were added after round one interviews, three of which were retained after round two testing in which two further items were added, forming the conceptually comprehensive 22-item I-TAQ. Patients demonstrated good understanding of item wording, instructions, response scales, and recall period.Conclusions: Successive rounds of in-depth interviews resulted in a treatment acceptance measure with strong content validity. Pending demonstration of its psychometric properties, the I-TAQ may prove to be a valuable measure of patients' perspectives toward being treated with low-density lipoprotein cholesterol-lowering therapies requiring subcutaneous injections. [ABSTRACT FROM AUTHOR]

We report a panel designed to open a dialog between pharmaceutical sponsors, regulatory reviewers, and other stakeholders regarding the use of social media to collect data to support the content validity of patient-reported outcome instruments in the context of medical product labeling. Multiple stakeholder perspectives were brought together to better understand the issues encountered in pursuing social media as a form of data collection to support content validity. Presenters represented a pharmaceutical sponsor of clinical trials, a regulatory reviewer from the Food and Drug Administration, and an online data platform provider. Each presenter shared its perspective on the advantages and disadvantages of using social media to collect this type of information. There was consensus that there is great potential for using social media for this purpose. There remain, however, unanswered questions that need to be addressed such as identifying which type of social media is most appropriate for data collection and ensuring that participants are representative of the target population while maintaining the advantages of anonymity provided by online platforms. The use of social media to collect evidence of content validity holds much promise. Clarification of issues that need to be addressed and accumulation of empirical evidence to address these questions are essential to moving forward. [ABSTRACT FROM AUTHOR]

Abstract: The importance of content validity in developing patient reported outcomes (PRO) instruments is stressed by both the US Food and Drug Administration and the European Medicines Agency. Content validity is the extent to which an instrument measures the important aspects of concepts that developers or users purport it to assess. A PRO instrument measures the concepts most significant and relevant to a patient''s condition and its treatment. For PRO instruments, items and domains as reflected in the scores of an instrument should be important to the target population and comprehensive with respect to patient concerns. Documentation of target population input in item generation, as well as evaluation of patient understanding through cognitive interviewing, can provide the evidence for content validity. Developing content for, and assessing respondent understanding of, newly developed PRO instruments for medical product evaluation will be discussed in this two-part ISPOR PRO Good Research Practices Task Force Report. Topics include the methods for generating items, documenting item development, coding of qualitative data from item generation, cognitive interviewing, and tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies. Part 1 covers elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument. Part 2 covers the instrument development process, the assessment of patient understanding of the draft instrument using cognitive interviews and steps for instrument revision. The two parts are meant to be read together. They are intended to offer suggestions for good practices in planning, executing, and documenting qualitative studies that are used to support the content validity of PRO instruments to be used in medical product evaluation. [Copyright &y& Elsevier]

Abstract: The importance of content validity in developing patient reported outcomes (PRO) instruments is stressed by both the US Food and Drug Administration and the European Medicines Agency. Content validity is the extent to which an instrument measures the important aspects of concepts developers or users purport it to assess. A PRO instrument measures the concepts most relevant and important to a patient''s condition and its treatment. For PRO instruments, items and domains as reflected in the scores of an instrument should be important to the target population and comprehensive with respect to patient concerns. Documentation of target population input in item generation, as well as evaluation of patient understanding through cognitive interviewing, can provide the evidence for content validity. Part 1 of this task force report covers elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument. Building on qualitative interviews and focus groups used to elicit concepts, cognitive interviews help developers craft items that can be understood by respondents in the target population and can ultimately confirm that the final instrument is appropriate, comprehensive, and understandable in the target population. Part 2 details: 1) the methods for conducting cognitive interviews that address patient understanding of items, instructions, and response options; and 2) the methods for tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies. The task force report''s two parts are meant to be read together. They are intended to offer suggestions for good practice in planning, executing, and documenting qualitative studies that are used to support the content validity of PRO instruments to be used in medical product evaluation. [Copyright &y& Elsevier]

Objectives: The aim of this study is to assess the validity of a new condition-specific instrument measuring psychosocial consequences of abnormal screening mammography (PCQ-DK33). Methods: The draft version of the PCQ-DK33 was completed on two occasions by 184 women who had received an abnormal screening mammography and on one occasion by 240 women who had received a normal screening result. Item Response Theories and Classical Test Theories were used to analyze data. Construct validity, concurrent validity, known group validity, objectivity and reliability were established by item analysis examining the fit between item responses and Rasch models. Results: Six dimensions covering anxiety, behavioral impact, sense of dejection, impact on sleep, breast examination, andsexuality were identified. One item belonging to the dejection dimension had uniform differential item functioning. Two items not fitting the Rasch models were retained because of high face validity. A sick leave item added useful information when measuring side effects and socioeconomic consequences of breast cancer screening. Five “poor items” were identified and should be deleted from the final instrument. Conclusions: Preliminary evidence for a valid and reliable condition-specific measure for women having an abnormal screening mammography was established. The measure includes 27 “good” items measuring different attributes of the same overall latent structure—the psychosocial consequences of abnormal screening mammography. [ABSTRACT FROM AUTHOR]

This study undertook qualitative interviews with experienced health economists to test the usability, face validity and content validity of a newly developed RUM (ModRUM). Methods A purposeful sampling strategy was used to recruit health economists with recent experience of implementing RUMs. Health economists were sent two versions of ModRUM (core module and depth extension). [Extracted from the article]

A patient-reported outcomes measure (PROM) was developed to assess RSV symptom intensity and impact (RSV-iiiQ) in adults. Methods Initial draft RSV-iiiQ questions were adapted from the Influenza Intensity and Impact Questionnaire (Flu-iiQ: a well-developed and tested PROM to assess influenza severity), and then modified based on relevance to RSV infection in accordance with results from a literature review and clinical expert input. [Extracted from the article]

Duchenne muscular dystrophy (DMD) is a rare genetic, progressive life-limiting paediatric neuromuscular disorder. Full-text published articles containing evidence of content and/or structural validity of PROs assessing QoL in DMD, and/or articles on PRO development, were included. [Extracted from the article]

Fragile X Syndrome (FXS) is a rare pediatric disorder that causes a range of developmental issues. Anxiety, social avoidance, irritability, and social unresponsiveness/lethargy represent the core behavioral symptoms of FXS. [Extracted from the article]

Evaluate the acceptability, ceiling and floor effects, reliability and construct validity of the Brazilian version of the Treatment Satisfaction Questionnaire for Medication (TSQM - version 1.4) in patients with hypertension. Construct validity was evaluated by known group analysis to test if TSQM discriminates satisfaction between in different stages of hypertension, diagnoses of Left Ventricular Hypertrophy (LVH), medication adherence and occurrence of side effects and Convergent validity between patient satisfaction and medication adherence measures. [Extracted from the article]

The Treatment Satisfaction Questionnaire for Medication (TSQM) is a valid and reliable measure that is becoming the leading measure of treatment satisfaction. Although the content validity of the TSQM v1.4 has been assessed, the validity of the electronically migrated TSQM was recently asessed. The online approach for estimating face validity of the electronic migration seems to be a promising venue that will enable the interview of more patients rapidly, with the limitation that less data is collected per patient. [Extracted from the article]

Patient education tools (PETs) can help patients better understand their health condition, facilitate communication with clinicians, and improve condition management. Once developed, further qualitative research with patients and clinicians can validate PET content, ensuring comprehension and relevance in the intended population, also confirming clinical accuracy. Evidence-based PETs are valuable tools to aid patient-clinician communication and improve patient knowledge of their condition. [Extracted from the article]

The MP SAP-c v3 was designed to estimate the volume of menstrual bleeding through self-report, using pictograms of sanitary pads and tampons. The majority (90.0%) of women preferred the MP SAP-c v3 over the AH method for measuring HMB, with 76.7% considering the AH method to be "unhygienic" or "disgusting" and 36.7% considering the MP SAP-c v3 to be "easier". [Extracted from the article]