The Short Form 6 Dimensions (SF-6D): Development and Evolution.

This paper considers the development and evolution of the short-form 6 dimensions (SF-6D), a generic preference-weighted measure consisting of a health classification with accompanying value set that was developed from one of the widest used health related quality of life measures, the SF-36 health survey. This enabled health state utility values to be directly generated from SF-36 and SF-12 data for a range of purposes, including to produce quality adjusted life years for use in economic evaluation of healthcare interventions across a range of different conditions and treatments. This paper considers the rationale for the development of the measure, the development process, performance and how the SF-6D has evolved since its conception. This includes the development of an updated version, SF-6D version 2 (SF-6Dv2), which was generated to deal with some criticisms of the first version, and now includes a standalone version for inclusion in studies without relying on use of SF-36 or SF-12. Valuation methods have also evolved, from standard gamble in-person interviews to online discrete choice experiment surveys. International work related to the SF-6Dv1 and SF-6Dv2 is considered. We also consider recommendations for use, highlighting key psychometric evidence and reimbursement agency recommendations.

Deriving a Preference-Weighted Measure for People with Hypoglycaemia from the Hypo-RESOLVE-QoL.

OBJECTIVE: Hypoglycaemia impacts the health-related quality of life (HRQoL) of people living with diabetes (PwD), and existing preference-weighted measures do not capture all important aspects. The study aimed to generate a preference-weighted measure capturing the HRQoL impact of hypoglycaemia in PwD. METHODS: Items for the health state classification system were selected from the hypoglycaemia-specific Hypo-RESOLVE QoL measure using: relevance in cognitive interviews, translatability, suitability for valuation, endorsement by patient advisors and experts, and psychometric performance in a large survey of PwD. Second, an online valuation survey using discrete choice experiment (DCE) with survival attribute was conducted with members of the UK public. DCE data was modelled using conditional logit analysis, and results scaled to produce preference weights for the classification system on a scale where 1 is equivalent to full health, 0 is equivalent to dead, and below zero is worse than dead. RESULTS: The health state classification system consists of eight items reflecting the factors of the Hypo-RESOLVE QoL (psychological, social and physical aspects). The valuation survey was completed by 1000 members of the UK public, representative for age and sex. Good understanding of DCE tasks was demonstrated. The item "do what I want to do in my life" had the largest preference weight, and "find it hard to stop thinking about my glucose levels" had the smallest. CONCLUSIONS: This study generated Hypo-RESOLVE QoL-8D, a preference-weighted measure capturing the HRQoL impact of hypoglycaemia in PwD, with UK general public preference-weights. The measure can be generated from Hypo-RESOLVE QoL data.

Development of a novel patient reported outcome measure for health-related quality of life in amyotrophic lateral sclerosis (PROQuALS): study protocol.

BACKGROUND: Patient reported outcome measures (PROMs) can be used to assess the impact of health conditions upon an individual's health-related quality of life (HRQoL). Whilst PROMs have been used to quantify the HRQoL impact of amyotrophic lateral sclerosis (ALS), existing instruments may not fully capture what matters to people living with ALS (plwALS) or be appropriate to be used directly to inform the cost-effectiveness of new treatments. This highlights a need for a new condition-specific PROM that can both capture what's important to plwALS and be used in economic evaluation. This study has two key aims: 1) to produce a novel PROM for measuring HRQoL in plwALS (PROQuALS). 2) to value a set of items from the novel PROM to generate an associated preference-weighted measure (PWM) that will enable utility values to be generated. METHODS: A mixed-methods study design will be conducted across three stages. Stage 1 involves concept elicitation and the generation of draft PROM content from a robust and comprehensive systematic review of HRQoL in ALS, with input from plwALS. Stage 2 consists of cognitive debriefing of the draft PROM content to ascertain its content validity (Stage 2a), followed by a psychometric survey (Stage 2b) to assess statistical performance. Evidence from Stage 2 will be used to make decisions on the final content and format of the novel PROM. Stage 3 will involve valuation and econometric modeling using health economics methods to generate preference weights, so a PWM derived from the novel PROM can be used in the cost-effectiveness analyses of treatments. Patient and clinical advisory groups will have critical, collaborative input throughout the project. DISCUSSION: The novel PROM will be designed to comprehensively assess important aspects of HRQoL to plwALS and to quantify HRQoL in terms of subjective impact. The PROQuALS measure will be available for use in research and healthcare settings. The associated PWM component will extend and enable the use of PROQuALS in cost-effective analyses of new treatments for ALS. TRIAL REGISTRATION: Not applicable.

Who should value children's health and how? An international Delphi study.

Valuing child health necessitates normative methodological decisions on whose preferences should be elicited and who should be imagined as experiencing impaired health. Formal guidance is limited and expert consensus unclear. This study sought to establish the degree of consensus among expert stakeholders on normative issues of who to ask and who should be imagined when valuing child health (7-17 years) to inform UK health technology assessment. Sixty-two experts (n = 47 in Round 2) from 18 countries participated in a modified, two-round online Delphi survey (Round 1: May-June 2023; Round 2: September-October 2023). Participants were expert stakeholders in child health valuation, including academics (n = 38); industry/consultancy representatives (including the charity/not-for-profit sector; n = 13); and UK policy/government representatives (n = 11). The Delphi survey was modified between rounds and consisted of 9-point Likert, categorical, multiple-choice, and free-text questions on normative issues in valuing child health. Responses were analysed descriptively and thematically. An a priori criterion of ≥75% agreement was established for formal consensus, while areas approaching consensus (≥70% agreement) and without consensus were identified as a future research primer. Consensus was observed that older adolescents (aged 16-17 years) and adults (18+ years) should be asked to value child health states. There was consensus that the former should think about themselves when valuing the health states and the latter should imagine a child of some form (e.g., imagining themselves as a child or another hypothetical child). However, no consensus was evident on what form this should take. Several other methodological issues also reached consensus. These findings are largely consistent with recent views elicited qualitatively from members of the public and other stakeholders on normative issues in valuing child health. The results mean that, contrary to what has been done in previous child health valuation studies, efforts should be made to involve both older adolescents (16+ years) and adults in child health valuation.

Development of a new health-related quality of life measure for people with diabetes who experience hypoglycaemia: the Hypo-RESOLVE QoL.

AIMS/HYPOTHESIS: Valid and reliable patient-reported outcome measures are vital for assessing disease impact, responsiveness to healthcare and the cost-effectiveness of interventions. A recent review has questioned the ability of existing measures to assess hypoglycaemia-related impacts on health-related quality of life for people with diabetes. This mixed-methods project was designed to produce a novel health-related quality of life patient-reported outcome measure in hypoglycaemia: the Hypo-RESOLVE QoL. METHODS: Three studies were conducted with people with diabetes who experience hypoglycaemia. In Stage 1, a comprehensive health-related quality of life framework for hypoglycaemia was elicited from semi-structured interviews (N=31). In Stage 2, the content validity and acceptability of draft measure content were tested via three waves of cognitive debriefing interviews (N=70 people with diabetes; N=14 clinicians). In Stage 3, revised measure content was administered alongside existing generic and diabetes-related measures in a large cross-sectional observational survey to assess psychometric performance (N=1246). The final measure was developed using multiple evidence sources, incorporating stakeholder engagement. RESULTS: A novel conceptual model of hypoglycaemia-related health-related quality of life was generated, featuring 19 themes, organised by physical, social and psychological aspects. From a draft version of 76 items, a final 14-item measure was produced with satisfactory structural (χ2=472.27, df=74, p<0.001; comparative fit index =0.943; root mean square error of approximation =0.069) and convergent validity with related constructs (r=0.46-0.59), internal consistency (α=0.91) and test-retest reliability (intraclass correlation coefficient =0.87). CONCLUSIONS/INTERPRETATION: The Hypo-RESOLVE QoL is a rigorously developed patient-reported outcome measure assessing the health-related quality of life impacts of hypoglycaemia. The Hypo-RESOLVE QoL has demonstrable validity and reliability and has value for use in clinical decision-making and as a clinical trial endpoint. DATA AVAILABILITY: All data generated or analysed during this study are included in the published article and its online supplementary files ( https://doi.org/10.15131/shef. DATA: 23295284.v2 ).

Exploring the Factors that Drive Clinical Negligence Claims: Stated Preferences of Those Who Have Experienced Unintended Harm.

BACKGROUND: Better understanding of the factors that influence patients to make a financial claim for compensation is required to inform policy decisions. This study aimed to assess the relative importance of factors that influence those who have experienced a patient safety incident (PSI) to make a claim for compensation. METHOD: Participants completed an online discrete choice experiment (DCE) involving 10 single profile tasks where they chose whether or not to file a claim. DCE data were modelled using logistic, mixed logit and latent class regressions; scenario analyses, external validity, and willingness to accept were also conducted. RESULTS: A total of 1029 participants in the United Kingdom responded to the survey. An appropriate apology and a satisfactory investigation reduced the likelihood of claiming. Respondents were more likely to claim if they could hold those responsible accountable, if the process was simple and straightforward, if the compensation amount was higher, if the likelihood of compensation was high or uncertain, if the time to receive a decision was quicker, and if they used the government compensation scheme. Men are more likely to claim for low impact PSIs. DISCUSSION AND CONCLUSIONS: The actions taken by the health service after a PSI, and people's perceptions about the probability of success and the size of potential reward, can influence whether a claim is made. Results show the importance of giving an appropriate apology and conducting a satisfactory investigation. This stresses the importance around how patients are treated after a PSI in influencing the clinical negligence claims that are made.

Estimating the SF-6Dv1 Value Set for a Population-Based Sample in Lebanon.

OBJECTIVES: The SF-6Dv1 is a preference-based measure derived from the SF-36 for use in quality-adjusted life-year estimation for cost-utility analysis. Country-specific value sets for SF-6Dv1 are needed to reflect societal preferences but none are available for Lebanon and other Arabic countries. This study aimed to generate a value set for SF-6Dv1 for Lebanon and to compare results with the UK set. METHODS: A sample of 249 health states defined by the SF-6Dv1 were valued by a representative sample of 577 members of the Lebanon general population, using standard gamble. Several multivariate regression models at mean and individual level were fitted to estimate utilities for all SF-6Dv1 states with selection of best fitting models based on predictive ability, consistency, and model fit. The best fitting models were compared with those fitted in the UK study. RESULTS: Data from 553 eligible respondents providing 3308 valuations were used for the analysis. Lebanese values were consistently higher than UK values, indicating differences in preferences, and there were no negative values. The random effects model using only main effects was the best performing model. There were inconsistencies in 2 dimensions, thereby consistent models were estimated with values ranging from 0.367 to 1. The results are consistent with the UK results. CONCLUSIONS: This study provides the first population-based value set for SF-6Dv1 health states for Lebanon, making it possible to generate quality-adjusted life-years for cost-utility analysis studies. The potential for applications of a standardized utility measure is enormous both in Lebanon and all Arab countries.

Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD).

INTRODUCTION: Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B. METHODS: Seven adult and seven child health state vignettes describing ASMD were developed based on published literature, clinical trial results, and interviews with clinicians, patients with ASMD, and parents of children with ASMD. The health states were valued in time trade-off interviews with adult general population respondents in the UK. RESULTS: Interviews were completed with 202 participants (50.0% female; mean age = 41.3 years). The health state representing ASMD without impairment had the highest mean utility for both the adult and child health states (0.92/0.94), and severe ASMD had the lowest mean utility (0.33/0.45). Every child health state had a significantly greater utility than the corresponding adult health state. Differences between adult/child paired states ranged from 0.02 to 0.13. Subgroup analyses explored the impact of parenting status on valuation of child health states. DISCUSSION: Greater severity of ASMD was associated with lower mean utility. Results have implications for valuation of pediatric health states. The resulting utilities may be useful in cost-utility modeling estimating the value of treatment for ASMD.

Development and Valuation of a Preference-Weighted Measure in Age-Related Macular Degeneration From the Vision Impairment in Low Luminance Questionnaire: A MACUSTAR Report.

OBJECTIVES: This study generates VILL-UI (Vision Impairment in Low Luminance - Utility Index), a preference-weighted measure (PWM) derived from the VILL-33 measure for use in patients with age-related macular degeneration (AMD) and valued to generate United Kingdom and German preference weights. METHODS: A PWM consists of a classification system to describe health and utility values for every state described by the classification. The classification was derived using existing data collected as part of the MACUSTAR study, a low-interventional study on AMD, conducted at 20 clinical sites across Europe. Items were selected using psychometric and Rasch analyses, published criteria around PWM suitability, alongside instrument developer views and concept elicitation work that informed VILL-33 development. An online discrete choice experiment (DCE) with duration of the health state was conducted with the United Kingdom and German public. Responses were modeled to generate utility values for all possible health states. RESULTS: The classification system has 5 items across the 3 domains of VILL-33: reading and accessing information, mobility and safety, and emotional well-being. The DCE samples (United Kingdom: n = 1004, Germany: n = 1008) are broadly representative and demonstrate good understanding of the tasks. The final DCE analyses produce logically consistent and significant coefficients. CONCLUSIONS: This study enables responses to VILL-33 to be directly used to inform economic evaluation in AMD. The elicitation of preferences from both United Kingdom and Germany enables greater application of VILL-UI for economic evaluation throughout Europe. VILL-UI fills a gap in AMD in which generic preference-weighted measures typically lack sensitivity.

Measuring the overall performance of mental healthcare providers.

To date there have been no attempts to construct composite measures of healthcare provider performance which reflect preferences for health and non-health benefits, as well as costs. Health and non-health benefits matter to patients, healthcare providers and the general public. We develop a novel provider performance measurement framework that combines health gain, non-health benefit, and cost and illustrate it with an application to 54 English mental health providers. We apply estimates from a discrete choice experiment eliciting the UK general population's valuation of non-health benefits relative to health gains, to administrative and patient survey data for years 2013-2015 to calculate equivalent health benefit (eHB) for providers. We measure costs as forgone health and quantify the relative performance of providers in terms of equivalent net health benefit (eNHB): the value of the health and non-health benefits minus the forgone benefit equivalent of cost. We compare rankings of providers by eHB, eNHB, and by the rankings produced by the hospital sector regulator. We find that taking account of the non-health benefits in the eNHB measure makes a substantial difference to the evaluation of provider performance. Our study demonstrates that the provider performance evaluation space can be extended beyond measures of health gain and cost, and that this matters for comparison of providers.

Psychometric performance of the CFQ-R-8D compared to the EQ-5D-3L and SF-6D in people with cystic fibrosis.

OBJECTIVE: This study aimed to compare the psychometric performance of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions (CFQ-R-8D), a new, condition-specific, preference-based measure, with that of generic preference-based measures EQ-5D-3L and Short Form 6 dimensions (SF-6D). METHODS: Data from three trials of participants with CF aged ≥ 14 years who completed the CFQ-R and EQ-5D-3L or SF-6D were used. Analyses were undertaken to evaluate convergent validity based on correlations with CFQ-R domain scores. Known-group validity was assessed based on percent predicted forced expiratory volume in one second and pulmonary exacerbations. Responsiveness was based on correlation of change and sensitivity to change based on change in symptom severity. Effect sizes and standardized response means were estimated. RESULTS: CFQ-R-8D utilities and dimensions were strongly correlated with most of the overlapping CFQ-R domain scores (ρ > 0.5); EQ-5D-3L and SF-6D utilities and dimensions had moderate (ρ > 0.3) to strong correlations in dimensions capturing similar concepts. All measures showed evidence of known-group validity (P < 0.05). Change correlations were strong for CFQ-R-8D utilities and dimensions and CFQ-R, but they were moderate for SF-6D and mostly weak ((ρ > 0.1) for EQ-5D-3L. The SF-6D had the largest mean change over time and effect sizes, followed by CFQ-R-8D and then EQ-5D-3L. Neither CFQ-R-8D or SF-6D utility scores had ceiling effects (< 9% responses in full health) compared with those of EQ-5D-3L (61-62%). In participants classified as being in full health by EQ-5D-3L, CFQ-R-8D captured CF-specific health problems, particularly cough, abdominal pain, and breathing difficulty. CONCLUSIONS: The CFQ-R-8D reflected known-group differences and changes over time with stronger evidence of good psychometric performance than EQ-5D-3L and similar evidence as SF-6D. Additionally, the CFQ-R-8D captured more condition-specific symptoms than EQ-5D-3L or SF-6D, which are important determinants of health-related quality of life for people with CF.

The RETRIEVE Checklist for Studies Reporting the Elicitation of Stated Preferences for Child Health-Related Quality of Life.

BACKGROUND: Recent systematic reviews show varying methods for eliciting, modelling, and reporting preference-based values for child health-related quality-of-life (HRQoL) outcomes, thus producing value sets with different characteristics. Reporting in many of the reviewed studies was found to be incomplete and inconsistent, making them difficult to assess. Checklists can help to improve standards of reporting; however, existing checklists do not address methodological issues for valuing child HRQoL. Existing checklists also focus on reporting methods and processes used in developing HRQoL values, with less focus on reporting of the values' key characteristics and properties. We aimed to develop a checklist for studies generating values for child HRQoL, including for disease-specific states and value sets for generic child HRQoL instruments. DEVELOPMENT: A conceptual model provided a structure for grouping items into five modules. Potential items were sourced from an adult HRQoL checklist review, with additional items specific to children developed using recent reviews. Checklist items were reduced by eliminating duplication and overlap, then refined for relevance and clarity via an iterative process. Long and short checklist versions were produced for different user needs. The resulting long RETRIEVE contains 83 items, with modules for reporting methods (A-D) and characteristics of values (E), for researchers planning and reporting child health valuation studies. The short RETRIEVE contains 14 items for decision makers or researchers choosing value sets. CONCLUSION: Applying the RETRIEVE checklists to relevant studies suggests feasibility. RETRIEVE has the potential to improve completeness in the reporting of preference-based values for child HRQOL outcomes and to improve assessment of preference-based value sets.

Understanding UK public views on normative decisions made to value health-related quality of life in children: A qualitative study.

Developing methodology for measuring and valuing child health-related quality of life (HRQoL) is a priority for health technology agencies. One aspect of this is normative decisions that are made in child HRQoL valuation. This qualitative study aimed to better understand adult public opinion on the normative questions of whose preferences to elicit (adults, children, or both) and from which perspective (who should be imagined living in impaired health), when valuing child HRQoL. Opinions of the adult UK public (N = 32) were solicited using online semi-structured focus groups, featuring a breadth of age, sex, ethnicities, and responsibility for children under 18 years. Participants were provided with bespoke informational material on health state valuation and were probed for their views. Arguments for and against different positions were discussed. Data was analysed using framework analysis. Participants demonstrated near-to-universal agreement that children should be involved in valuation in some form, yet this should differ depending on age or maturity. There was strong support for approaches combining involvement from children and adults (e.g., their parents), especially for younger children. There was little intuitive support for the 'taxpayer argument' for asking taxpaying adults. In the context of greater involvement of children in valuation, most participants supported using an 'own' perspective. Most participants thought that valuation study participants should know the exercise is about valuing child health states for ethical reasons. Informed views from the UK public on who should be asked and with what perspective when valuing child HRQoL appear to differ from normative positions previously advocated by some health economists, such as prioritising the preferences of taxpaying adults. In contrast, the results suggest including adults and children in valuation, with the proviso that the children are of an appropriate age and level of maturity, and that an own perspective is used wherever possible.

UK Valuation of EQ-5D-5L, a Generic Measure of Health-Related Quality of Life: A Study Protocol.

OBJECTIVES: A high-quality and widely accepted UK EQ-5D-5L value set is urgently required to enable the latest version of EQ-5D scored using recent UK public preferences to inform policy including health technology assessments submitted to the National Institute for Health and Care Excellence. This article outlines the study protocol for the generation of a new EQ-5D-5L UK value set. METHODS: Twelve hundred interviews will be undertaken using the composite time trade-off elicitation technique for 102 health states (86 from the international EQ-5D-5L valuation protocol, plus 16 with best predictive performance in an extended design used in the Native American EQ-5D-5L valuation). The sample will be UK adults (age ≥18 years) proportionately representative across England, Wales, Scotland, and Northern Ireland, representative for age, sex, ethnicity, and socioeconomic group, with inclusion of participants with/without health problems. Participants will choose to be interviewed via videoconference (by Zoom) or in-person in a central venue. Data quality will be rigorously assessed. RESULTS: The value set will be generated using tobit random effects and heteroscedastic tobit models (with censoring at -1) using all data, excluding time trade-off values highlighted by participants as ones they would reconsider and data from interviewers failing protocol compliance. Quality and acceptance will be achieved by public involvement, regular Steering Group meetings, independent assessment of data quality at 4 time points, and final endorsement of data and analyses. CONCLUSION: This study will produce a UK value set for the EQ-5D-5L for use in prospective and retrospective data sets containing EQ-5D-5L data.

Using Age-Specific Values for Pediatric HRQoL in Cost-Effectiveness Analysis: Is There a Problem to Be Solved? If So, How?

Value sets for the EQ-5D-Y-3L published to date appear to have distinctive characteristics compared with value sets for corresponding adult instruments: in many cases, the value for the worst health state is higher and there are fewer values < 0. The aim of this paper is to consider how and why values for child and adult health differ; and what the implications of that are for the use of EQ-5D-Y-3L values in economic evaluations to inform healthcare resource allocation decisions. We posit four potential explanations for the differences in values: (a) The wording of severity labels may mean the worst problems on the EQ-5D-Y-3L are descriptively less severe than those on the EQ-5D-5L; (b) Adults may genuinely consider that children are less badly affected than adults by descriptively similar health issues. That is, for any given health problem, adult respondents in valuation studies consider children's overall health-related quality of life (HRQoL) on average to be higher than that for adults; (c) Values are being sought by eliciting adults' stated preferences for HRQoL in another person, rather than in themselves (regardless of whether the 'other person' concerned is a child); and (d) The need to elicit preferences for child HRQoL that are anchored at dead = 0 invokes special considerations regarding children's survival. Existing evidence does not rule out the possibility that (c) and (d) exert an upward bias in values. We consider the implications of that for the interpretation and use of values for pediatric HRQoL. Alternative methods for valuing children's HRQoL in a manner that is not 'age specific' are possible and may help to avoid issues of non-comparability. Use of these methods would place the onus on health technology assessment bodies to reflect any special considerations regarding child quality-adjusted life-year gains.

Discrete Choice Experiments in Health State Valuation: A Systematic Review of Progress and New Trends.

BACKGROUND: Discrete choice experiments (DCEs) are increasingly used in health state valuation studies. OBJECTIVE: This systematic review updates the progress and new findings of DCE studies in the health state valuation, covering the period since the review of June 2018 to November 2022. The review reports the methods that are currently being used in DCE studies to value health and study design characteristics, and, for the first time, reviews DCE health state valuation studies published in the Chinese language. METHODS: English language databases PubMed and Cochrane, and Chinese language databases Wanfang and CNKI were searched using the self-developed search terms. Health state valuation or methodology study papers were included if the study used DCE data to generate a value set for a preference-based measure. Key information extracted included DCE study design strategies applied, methods for anchoring the latent coefficient on to a 0-1 QALY scale and data analysis methods. RESULTS: Sixty-five studies were included; one Chinese language publication and 64 English language publications. The number of health state valuation studies using DCE has rapidly increased in recent years and these have been conducted in more countries than prior to 2018. Wide usage of DCE with duration attributes, D-efficient design and models accounting for heterogeneity has continued in recent years. Although more methodological consensus has been found than in studies conducted prior to 2018, this consensus may be driven by valuation studies for common measures with an international protocol (the 'model' valuation research). Valuing long measures with well-being attributes attracted attention and more realistic design strategies (e.g., inconstant time preference, efficient design and implausible states design) were identified. However, more qualitative and quantitative methodology study is still necessary to evaluate the effect of those new methods. CONCLUSIONS: The use of DCEs in health state valuation continues to grow dramatically and the methodology progress makes the method more reliable and pragmatic. However, study design is driven by international protocols and method selection is not always justified. There is no gold standard for DCE design, presentation format or anchoring method. More qualitative and quantitative methodology study is recommended to evaluate the effect of new methods before researchers make methodology decisions.

Randomised comparison of online interviews versus face-to-face interviews to value health states.

INTRODUCTION: Health state valuation studies using composite time trade-off (cTTO) interviews have historically been conducted face-to-face. The COVID-19 pandemic forced disruptive innovation meaning a number of valuation studies conducted interviews via videoconference. These studies found online interviews feasible and acceptable; however, studies were not constructed to test the impact of online versus face-to-face interviews. This study builds on its sister study from the UK and aims to assess the acceptability and equivalence of in person face-to-face interviews with online interviews on cTTO valuation outcomes and on data quality. METHODS: Participants were recruited into a randomised equivalence study via an external research company. Consenting participants were randomly allocated to complete a cTTO interview face-to-face or online, using the same 10 EQ-5D-5L health states. Mean and distribution of the cTTO values, participant understanding, data quality, demographic characteristics, participant preference, participant engagement and participant feedback were all compared across interview mode. Statistical equivalence for cTTO values for each state was tested using two one-sided t-tests by mode. Finally, regression analysis was completed to assess the impacts of interview mode on cTTO value while controlling for demographic characteristics of the participants. RESULTS: Mean cTTO values were shown to be equivalent for mild health states and showed no significant difference for serious health states. The proportion of individuals who expressed an interest in the study but declined to arrange an interview after finding out their randomisation was significantly higher for the face-to-face (21.6%) than the online group (1.8%). No significant difference was found between groups for participant engagement, understanding or feedback nor for any indicators of data quality. CONCLUSION: Administrating interviews face to face or online did not appear to have a statistically significant impact on mean cTTO values. Offering both online and face-to-face interviews routinely allows all participants to select the most convenient option.

Valuing the EQ Health and Wellbeing Short Using Time Trade-Off and a Discrete Choice Experiment: A Feasibility Study.

OBJECTIVES: The EQ Health and Wellbeing Short (EQ-HWB-S) is a new generic measure that covers health and wellbeing developed for use in economic evaluation in health and social care. The aim was to test the feasibility of using composite time trade-off (cTTO) and a discrete choice experiment (DCE) based on an international protocol to derive utilities for the EQ-HWB-S and to generate a pilot value set. METHODS: A representative UK general population was recruited. Online videoconference interviews were undertaken where cTTO and DCE tasks were administered using EuroQol Portable Valuation Technology. Quality control (QC) was used to assess interviewers' performance. Data were modeled using Tobit, probit, and hybrid models. Feasibility was assessed based on the distribution of data, participants, and reports of understanding from the interviewer, QC and modeling results. RESULTS: cTTO and DCE data were available for 520 participants. Demographic characteristics were broadly representative of the UK general population. Interviewers met QC requirements. cTTO values ranged between -1 to 1 with increasing disutility associated with more severe states. Participants understood the tasks and the EQ-HWB-S states; and the interviewers reported high levels of understanding and engagement. The hybrid Tobit heteroscedastic model was selected for the pilot value set with values ranging from -0.384 to 1. Pain, mobility, daily activities, and sad/depressed had the largest disutilities, followed by loneliness, anxiety, exhaustion, control, and cognition in the selected model. CONCLUSIONS: EQ-HWB-S can be valued using cTTO and DCE. Further methodological work is recommended to develop a valuation protocol specific to the EQ-HWB-S.

Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised.

OBJECTIVES: Cystic fibrosis (CF) limits survival and negatively affects health-related quality of life (HRQOL). Cost-effectiveness analysis (CEA) may be used to make reimbursement decisions for new CF treatments; nevertheless, generic utility measures used in CEA, such as EQ-5D, are insensitive to meaningful changes in lung function and HRQOL in CF. Here we develop a new, CF disease-specific, preference-based utility measure based on the adolescent/adult version of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely used, CF-specific, patient-reported measure of HRQOL. METHODS: Blinded CFQ-R data from 4 clinical trials (NCT02347657, NCT02392234, NCT01807923, and NCT01807949) were used to identify discriminating items for a classification system using psychometric (eg, factor and Rasch) analyses. Thirty-two health states were selected for a time trade-off (TTO) exercise with a representative sample of the UK general population. TTO utilities were used to estimate a preference-based scoring algorithm by regression analysis (tobit models with robust standard errors clustered on participants with censoring at -1). RESULTS: A classification system with 8 dimensions (CFQ-R-8 dimensions; physical functioning, vitality, emotion, role functioning, breathing difficulty, cough, abdominal pain, and body image) was generated. TTO was completed by 400 participants (mean age, 47.3 years; 49.8% female). Among the regression models evaluated, the tobit heteroscedastic-ordered model was preferred, with a predicted utility range from 0.236 to 1, no logical inconsistencies, and a mean absolute error of 0.032. CONCLUSION: The CFQ-R-8 dimensions is the first disease-specific, preference-based scoring algorithm for CF, enabling estimation of disease-specific utilities for CEA based on the well-validated and widely used CFQ-R.