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 ).

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.

Producing a preference-based quality of LIFE measure to quantify the impact of HYPOGLYCAEMIA on people living with diabetes: A mixed-methods research protocol.

BACKGROUND: Assessment of patient-reported outcome measures (PROMs), including quality of life (QoL), is essential in diabetes research and care. However, a recent review concluded that current hypoglycaemia-specific PROMs have limited evidence of validity, reliability and responsiveness for assessing the impact of hypoglycaemia on QoL in people living with diabetes. None of the PROMs identified could be used directly to inform the cost-effectiveness of treatments and interventions. There is a need for a new hypoglycaemia-specific QoL PROM, which can be used directly to inform economic evaluations. AIMS: This project has three aims: (a) To develop draft PROM content for measuring the impact of hypoglycaemia on QoL in adults with diabetes. (b) To refine the draft content using cognitive debriefing interviews and psychometrics. This will result in a condition-specific PROM that can be used to quantify the impact of hypoglycaemia upon QoL. (c) To generate a preference-based measure (PBM) that will enable utility values to be calculated for economic evaluation. METHODS: A mixed-methods, three-stage design is used: (a) Qualitative interviews will inform the draft PROM content. (b) Cognitive debriefing interview data will be used to refine the draft PROM content. The PROM will be administered in a large-scale survey to enable psychometric validation. Final item selection for the PROM will be informed by psychometric performance, translatability assessment and input from stakeholder groups. (c) A classification system will be generated, comprising a reduced number of items from the PROM. A valuation survey will be conducted to derive a value set for the PBM.

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.

A comprehensive qualitative framework for health-related quality of life in Duchenne muscular dystrophy.

PURPOSE: Duchenne muscular dystrophy (DMD) is a rare x-linked neuromuscular condition predominantly affecting boys and men. There is a paucity of research qualitatively detailing the lived experience of health-related quality of life (HRQoL) for people with DMD. The aim of this study was to identify a comprehensive framework for better understanding HRQoL in DMD. METHODS: Eighteen boys and men (aged 7 to ≥ 40 years) with DMD were recruited from charity Duchenne UK, a DMD support group, and 5 UK National Health Service Trusts. Semi-structured interviews were conducted using a topic guide informed by a review into HRQoL in DMD. Generic, preference-based, patient-reported outcome measures (PROs) were used as prompts. Interviews were audio recorded, transcribed verbatim and analysed using framework analysis. RESULTS: Thirty-seven themes were coded, within seven categories. Six categories were conceptualised as components of HRQoL (autonomy, daily activities, feelings and emotions, identity, physical aspects, social relationships) and one considered an input (healthcare, support, and environment). Three additional themes were used to code feedback on the generic PROs (CHU-9D, EQ-5D, HUI). Social relationships received most coverage in the data and was noted as an omission from the PROs. CONCLUSION: A 30-item framework for HRQoL in DMD has been developed, which was used as input into a new condition-specific HRQoL PRO and preference-based measure: the DMD-QoL. The data has value in its own right in highlighting the lived experience of HRQoL for people with DMD and as a barometer for assessing the content of HRQoL PROs for use in DMD.

Unmet support needs relating to hypoglycaemia among adults with type 1 diabetes: Results of a multi-country web-based qualitative study.

OBJECTIVE: Hypoglycaemic episodes and fear of hypoglycaemia can be burdensome for adults with type 1 diabetes. This study explored support needs relating to hypoglycaemia among adults with type 1 diabetes living in Denmark, Germany, the Netherlands and the United Kingdom. RESEARCH DESIGN AND METHODS: Respondents participated in a web-based qualitative study involving four open-ended questions that asked what they wished other people understood about hypoglycaemia and what other people could do differently to support them with hypoglycaemia. Responses were analyzed using reflexive thematic analysis. RESULTS: Participants were 219 adults with type 1 diabetes (mean ± SD age 39 ± 13 years; mean ± SD diabetes duration 20 ± 14 years). They described unmet needs relating to: (1) Clinical support, involving access to new diabetes technologies, training on hypoglycaemia prevention, personalised care and psychological support; (2) Practical support, involving family and friends better supporting them with hypoglycaemia management and prevention; (3) Education for other people, involving others becoming more informed about hypoglycaemia; and (4) An appreciation of the burden, involving others recognizing the experience and impact of episodes, and the burden of living with the risk of hypoglycaemia. CONCLUSIONS: Adults with type 1 diabetes report several unmet support needs relating to hypoglycaemia. Service delivery should be person-centred and prioritise the individual's support needs. Clinical conversations are needed to identify the individual's support needs and develop tailored support plans. People with diabetes and their family members should be offered hypoglycaemia-specific education and training.

Qualitative Review on Domains of Quality of Life Important for Patients, Social Care Users, and Informal Carers to Inform the Development of the EQ-HWB.

OBJECTIVES: To identify the themes to inform the content of a new generic measure, the EQ-HWB (EQ Health and Wellbeing), that can be used in economic evaluation across health, social care, and public health, based on the views of users and beneficiaries of these services including informal carers. METHODS: A qualitative review was undertaken. Systematic and citation searches were undertaken focusing on qualitative evidence of the impact on quality of life from reviews for selected health conditions, informal carers, social care users, and primary qualitative work used in the development of selected measures. A subset of studies was included in the review. Framework analysis and synthesis were undertaken based on a conceptual model. RESULTS: A total of 42 reviews and 24 primary studies were selected for inclusion in the review. Extraction and synthesis resulted in 7 high-level themes (with subthemes): (1) feelings and emotions (sadness, anxiety, hope, frustration, safety, guilt/shame); (2) cognition (concentration, memory, confusion, thinking clearly); (3) self-identity (dignity/respect, self-esteem); (4) "coping, autonomy, and control" relationships; (5) social connections (loneliness, social engagement, stigma, support, friendship, belonging, burden); (6) physical sensations (pain, discomfort, sleep, fatigue); and (7) activity (self-care, meaningful activities, mobility, communication, hearing, vision). Apart from physical sensations, most of the other themes and subthemes were relevant across both health and social care, including for informal carers. CONCLUSIONS: The findings from this broad review identified themes that go beyond health and that are relevant to patients, informal carers, and social care users. The themes and subthemes informed the domains for the EQ-HWB.

Generation, Selection, and Face Validation of Items for a New Generic Measure of Quality of Life: The EQ-HWB.

OBJECTIVES: This article aims to describe the generation and selection of items (stage 2) and face validation (stage 3) of a large international (multilingual) project to develop a new generic measure, the EQ-HWB (EQ Health and Wellbeing), for use in economic evaluation across health, social care, and public health to estimate quality-adjusted life-years. METHODS: Items from commonly used generic, carer, social care, and mental health quality of life measures were mapped onto domains or subdomains identified from a literature review. Potential terms and items were reviewed and refined to ensure coverage of the construct of the domains/subdomain (stage 2). Input on the potential item pool, response options, and recall period was sought from 3 key stakeholder groups. The pool of candidate items was tested in qualitative interviews with potential future users in an international face validation study (stage 3). RESULTS: Stage 2 resulted in the generation of 687 items. Predetermined selection criteria were applied by the research team resulting in 598 items being dropped, leaving 89 items that were reviewed by key stakeholder groups. Face validation (stage 3) tested 97 draft items and 4 response scales. A total of 47 items were retained and 14 were modified, whereas 3 were added to the candidate pool of items. This resulted in a 64-item set. CONCLUSIONS: This international multiculture, multilingual study with a common methodology identified many items that performed well across all countries. These were taken to the psychometric testing along with modified and new items for the EQ-HWB.

The EQ-HWB: Overview of the Development of a Measure of Health and Wellbeing and Key Results.

OBJECTIVES: Existing measures for estimating quality-adjusted life-years are mostly limited to health-related quality of life. This article presents an overview of the development the EQ-HWB (EQ Health and Wellbeing), which is a measure that encompasses health and wellbeing. METHODS: Stages: (1) Establishing domains through reviews of the qualitative literature informed by a conceptual framework. (2) Generation and selection of items to cover the domains. (3) Face validation of these items through qualitative interviews with 168 patients, social care users, general population, and carers across 6 countries (Argentina, Australia, China, Germany, United Kingdom, United States). (4) Extensive psychometric testing of candidate items (using classical, factor analysis, and item response theory methods) on > 4000 respondents in the 6 countries. Stakeholders were consulted throughout. RESULTS: A total of 32 subdomains grouped into 7 high-level domains were identified from the qualitative literature and 97 items generated to cover them. Face validation eliminated 36 items, modified 14, and added 3. Psychometric testing of 64 items found little difference in missing data or problems with response distribution, the conceptual model was confirmed except in China, and most items performed well in the item response theory in all countries. Evidence was presented to stakeholders in 2 rounds of consultation to inform the final selection of items for the EQ-HWB (25-item) and the short version of EQ-HWB (9-items). CONCLUSIONS: EQ-HWB measures have been developed internationally for evaluating interventions in health, public health, and social care including the impact on patients, social care users, and carers.

Measuring carer quality of life in Duchenne muscular dystrophy: a systematic review of the reliability and validity of self-report instruments using COSMIN.

INTRODUCTION: Duchenne muscular dystrophy is a rare, progressive, life-limiting genetic neuromuscular condition that significantly impacts the quality of life of informal caregivers. Carer quality of life is measured using heterogeneous self-report scales, yet their suitability for Duchenne remains unclear. This review aimed to identify and evaluate the reliability and validity of quality of life instruments in Duchenne carers. MATERIALS AND METHODS: Systematic searches were conducted in Embase, MEDLINE, CINAHL, PsycINFO, Cochrane Library and Google Scholar. Full research articles reporting data on multiple-item self-report quality of life instruments in informal Duchenne carers were included. Extracted evidence was qualitatively synthesised and evaluated, including risk of bias, against the Consensus-based Standards for the selection of health Measurement Instruments. Duchenne carer collaborators (N = 17) helped rate the instruments' content validity. RESULTS: Thirty-one articles featuring thirty-two quality of life instruments were included. Content validity was rated as "inconsistent" based on very low quality evidence. For Duchenne carer collaborators, the best instrument was PedsQL Family Impact Module. Only one instrument had evidence for structural validity (rated "unsatisfactory") and measurement invariance (rated "satisfactory"). Instruments received "satisfactory" ratings for internal consistency and mixed ratings for construct validity and responsiveness. There was no evidence for reliability, measurement error, or criterion validity. DISCUSSION: Instruments used to measure Duchenne carer quality of life have limited and often inconsistent supportive psychometric evidence. Further work must investigate instruments' measurement properties in Duchenne carers and/or the development of new tools. In the interim, we recommend considering the PedsQL Family Impact Module based on Duchenne carer ratings.

The suitability of patient-reported outcome measures used to assess the impact of hypoglycaemia on quality of life in people with diabetes: a systematic review using COSMIN methods.

AIMS/HYPOTHESIS: It is generally accepted that hypoglycaemia can negatively impact the quality of life (QoL) of people living with diabetes. However, the suitability of patient-reported outcome measures (PROMs) used to assess this impact is unclear. The aim of this systematic review was to identify PROMs used to assess the impact of hypoglycaemia on QoL and examine their quality and psychometric properties. METHODS: Systematic searches (MEDLINE, EMBASE, PsycINFO, CINAHL and The Cochrane Library databases) were undertaken to identify published articles reporting on the development or validation of hypoglycaemia-specific PROMs used to assess the impact of hypoglycaemia on QoL (or domains of QoL) in adults with diabetes. A protocol was developed and registered with PROSPERO (registration no. CRD42019125153). Studies were assessed for inclusion at title/abstract stage by one reviewer. Full-text articles were scrutinised where considered relevant or potentially relevant or where doubt existed. Twenty per cent of articles were assessed by a second reviewer. PROMS were evaluated, according to COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines, and data were extracted independently by two reviewers against COSMIN criteria. Assessment of each PROM's content validity included reviewer ratings (N = 16) of relevance, comprehensiveness and comprehensibility: by researchers (n = 6); clinicians (n = 6); and adults with diabetes (n = 4). RESULTS: Of the 214 PROMs used to assess the impact of hypoglycaemia on QoL (or domains of QoL), seven hypoglycaemia-specific PROMS were identified and subjected to full evaluation: the Fear of Hypoglycemia 15-item scale; the Hypoglycemia Fear Survey; the Hypoglycemia Fear Survey version II; the Hypoglycemia Fear Survey-II short-form; the Hypoglycemic Attitudes and Behavior Scale; the Hypoglycemic Confidence Scale; and the QoLHYPO questionnaire. Content validity was rated as 'inconsistent', with most as '(very) low' quality, while structural validity was deemed 'unsatisfactory'. Other measurement properties (e.g. reliability) varied, and evidence gaps were apparent across all PROMs. None of the identified studies addressed cross-cultural validity or measurement error. Criterion validity and responsiveness were not assessed due to the lack of a 'gold standard' measure of the impact of hypoglycaemia on QoL against which to compare the PROMS. CONCLUSIONS/INTERPRETATION: None of the hypoglycaemia-specific PROMs identified had sufficient evidence to demonstrate satisfactory validity, reliability and responsiveness. All were limited in terms of content and structural validity, which restricts their utility for assessing the impact of hypoglycaemia on QoL in the clinic or research setting. Further research is needed to address the content validity of existing PROMs, or the development of new PROM(s), for the purpose of assessing the impact of hypoglycaemia on QoL. PROSPERO REGISTRATION: CRD42019125153.

Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL.

OBJECTIVES: This study generates a preference-based measure for capturing the quality of life of people with Duchenne muscular dystrophy (DMD) from a new measure of quality of life, DMD-QoL. METHODS: A health state classification system was derived from the DMD-QoL based on psychometric performance of items, factor analysis, and item response theory analysis. Preferences for health states described by the classification system were elicited using an online discrete choice experiment survey with life years as an additional attribute, from members of the UK general population (n = 1043). Discrete choice experiment data was modeled using a conditional fixed-effects logit model and utility estimates were directly anchored on the 1 to 0 full health-dead scale. RESULTS: The health state classification system has 8 dimensions: mobility, difficulty using hands, difficulty breathing, pain, tiredness, worry, participation, and feeling good about yourself. The standard model had mostly statistically significant coefficients and reflected the instrument's monotonic structure. However, 2 dimensions had inconsistent coefficients (where utility increased as health worsened) and a consistent model was estimated that merged adjacent inconsistent severity levels. The best state defined by the classification system has a value of 1 and the worst state has a value of -0.559. CONCLUSION: The modeled results enable DMD-QoL-8D utility values to be generated using DMD-QoL or DMD-QoL-8D data to generate QALYs for people with DMD. QALYs can then be used to inform economic models of the cost-effectiveness of interventions in DMD. Future research comparing the psychometric performance of DMD-QoL-8D to existing generic preference-based measures, including EQ-5D-5L, is recommended.

Criteria for item selection for a preference-based measure for use in economic evaluation.

Preference-based measures allow patients to report their level of health, and the responses are then scored using preference weights from a representative general population sample for use in cost utility analysis. The development process of new preference-based measures should ensure that valid items are selected to reflect the constructs of interest included in the measure and that are suitable for use in preference-elicitation exercises. Existing criteria on patient-reported outcome measures (PROMs) development were reviewed, and additional considerations were taken into account in order to generate criteria to support development of new preference-based measures. Criteria covering 22 different aspects related to item selection for preference-based measures are presented. These include criteria related to how items are phrased to ensure accurate completion, the coverage of items in terms of range of domains as well as focus on current outcomes and whether items are suitable for valuation. The criteria are aimed at supporting the development of new preference-based measures with discussion to ensure that even where there is conflict between criteria, issues have been considered at the item selection stage. This would minimize problems at valuation stage by harmonizing established criteria and expanding lists to reflect the unique characteristics of preference-based measures.

How to measure fluctuating impairments in people with MS: development of an ambulatory assessment version of the EQ-5D-5L in an exploratory study.

BACKGROUND: Health fluctuations even within a single day are typical in multiple sclerosis (MS), but are not captured by widely used questionnaires like the EQ-5D-5L. This exploratory study aimed to develop an ambulatory assessment (AA) version of the EQ-5D-5L (EQ-5D-AA) where patients rate their health on mobile phones multiple times per day over several days, and to assess its feasibility and face validity. METHODS: An initial EQ-5D-AA version was based on two patient focus groups. It was then tested and continuously developed in an iterative process: patients completed it over several days, followed by debriefing interviews. Findings were used to refine the EQ-5D-AA, with the resulting version being tested by the subsequent wave of patients until participants declared no need for changes anymore. Before and after the AA period, participants completed the standard paper-based EQ-5D-5L asking about 'today'. RESULTS: Focus group participants reported that their impairments often fluctuated between and within days. They regarded an AA with three assessments per day over seven days most appropriate; assessment should be retrospective to the previous assessment, but not all items should be assessed at each time point. Four waves of AA testing were conducted. Thirteen out of the 17 participants preferred the AA over standard assessment as they regarded it more informative, but not too burdensome. CONCLUSION: The newly developed one-week AA of the EQ-5D-5L captures within-day and day-to-day health fluctuations in people with MS. From the patients' perspective, it is a feasible and face valid way to provide important information beyond what is captured by the standard EQ-5D-5L.

Assessment of the psychometric properties and refinement of the Health and Self-Management in Diabetes Questionnaire (HASMID).

BACKGROUND: The Health And Self-Management In Diabetes (HASMIDv1) questionnaire consists of 8 attributes, 4 about quality of life, and 4 about self-management. The overall aim of this study was to rigorously examine the psychometric properties of the HASMIDv1 questionnaire. METHODS: The study comprised two phases. Phase 1 identified items of the HASMIDv1 questionnaire that potentially required rewording through consultation with a patient involvement panel and two focus groups of people with diabetes. Phase 2 involved a cross-sectional longitudinal survey where HASMID, EQ-5D-5L, health, treatment and sociodemographic questions were administered using both paper and online versions to people with diabetes. Participants were asked to complete the survey again approximately 3 months later. Psychometric analyses were undertaken to examine floor and ceiling effects, item distributions, known group differences and internal consistency. Rasch analysis was undertaken to assess differential item functioning and disordered thresholds. RESULTS: Phase 1 derived five alternative wordings to items: Irritable, Affects Mealtimes, Daily Routine, Social Activities and Problem. Phase 2 achieved 2835 responses at time point 1 (n = 1944 online, n = 891 paper version) and 1243 at time point 2 (n = 533 online, n = 710 paper version). Overall the HASMID items performed well, though two alternative worded items (Irritable and Social Activities) provided additional information not fully captured by the original HASMID items. CONCLUSION: Psychometric evaluation and Rasch analysis were used in conjunction with expert opinion to determine the final questionnaire. The application of psychometric analyses or Rasch analysis alone to inform item selection would have resulted in different items being selected for the final instrument. The benefit of a combined approach has produced an instrument which has a broader evaluation of self-management. The final validated HASMID-10 is a short self-report PRO that can be used to evaluate the impact of self-management for people living with diabetes. HASMID-10 can be scored using total summative scores, with utility and monetary values also available for use in cost-utility and cost-benefit analyses.

Measuring quality of life in Duchenne muscular dystrophy: a systematic review of the content and structural validity of commonly used instruments.

Duchenne muscular dystrophy (DMD) is an inherited X-linked neuromuscular disorder. A number of questionnaires are available to assess quality of life in DMD, but there are concerns about their validity. This systematic review aimed to appraise critically the content and structural validity of quality of life instruments for DMD. Five databases (EMBASE, MEDLINE, CINAHL, PsycINFO, and Cochrane Library) were searched, with supplementary searches in Google Scholar. We included articles with evidence on the content and/or structural validity of quality of life instruments in DMD, and/or instrument development. Evidence was evaluated against the Consensus-based Standards for the selection of health Measurement INstruments (COSMIN) criteria. Fifty five articles featured a questionnaire assessing quality of life in DMD. Forty instruments were extracted and 26 underwent assessment. Forty-one articles contained evidence on content or structural validity (including 37 development papers). Most instruments demonstrated low quality evidence and unsatisfactory or inconsistent validity in DMD, with the majority not featuring direct validation studies in this population. Only KIDSCREEN received an adequate rating for instrument design and a satisfactory result for content validity based on its development, yet, like the majority of PROMs, the measure has not been directly validated for use in DMD. Further research is needed on the validity of quality of life instruments in DMD, including content and structural validity studies in this population.

PROM Validation Using Paper-Based or Online Surveys: Data Collection Methods Affect the Sociodemographic and Health Profile of the Sample.

OBJECTIVE: This study examines the impact of data collection method on the sociodemographic and health profile of samples of people with diabetes who complete either an online or postal patient-reported outcome measure (PROM) validation survey. METHODS: A longitudinal survey of people with diabetes was conducted using online and postal survey versions. The survey consisted of sociodemographic and health questions, a health and self-management PROM (Health and Self-Management in Diabetes [HASMID]), and 5-level version of EQ-5D. Dose adjustment for normal eating Online, Diabetes UK, and social media were used to recruit online survey participants. A panel of patients at a local National Health Service Trust was randomly allocated to participate in either survey version (two-thirds to postal version). Participants were asked to complete the survey again approximately 3 months later. RESULTS: A total of 2784 participants completed the survey (1908 online, 876 postal). The samples (online versus postal) differed; the online sample was younger, with a larger proportion of women and respondents with type 1 diabetes. There were significant differences in sociodemographic characteristics by type of diabetes across data collection mode. The proportion of respondents who responded again at point 2 was higher in the postal sample (525 postal, 698 online). CONCLUSION: The sociodemographic and health profile of samples of people with diabetes differed depending on whether they completed the online or postal survey. Differences are likely due to different recruitment methods and differences in those choosing to respond to different survey versions. Future PROM validation surveys should select data collection methods carefully because these can affect sample characteristics and results.

Public involvement in health outcomes research: lessons learnt from the development of the recovering quality of life (ReQoL) measures.

BACKGROUND: To provide a model for Public involvement (PI) in instrument development and other research based on lessons learnt in the co-production of a recently developed mental health patient reported outcome measure called Recovering Quality of Life (ReQoL). While service users contributed to the project as research participants, this paper focuses on the role of expert service users as research partners, hence referred to as expert service users or PI. METHODS: At every stage of the development, service users influenced the design, content and face validity of the measure, collaborating with other researchers, clinicians and stakeholders who were central to this research. Expert service users were integral to the Scientific Group which was the main decision-making body, and also provided advice through the Expert Service User Group. RESULTS: During the theme and item generation phase (stage 1) expert service users affirmed the appropriateness of the seven domains of the Patient Reported Outcome Measure (activity, hope, belonging and relationships, self-perception, wellbeing, autonomy, and physical health). Expert service users added an extra 58 items to the pool of 180 items and commented on the results from the face and content validity testing (stage 2) of a refined pool of 88. In the item reduction and scale generation phase (stage 3), expert service users contributed to discussions concerning the ordering and clustering of the themes and items and finalised the measures. Expert service users were also involved in the implementation and dissemination of ReQoL (stage 4). Expert service users contributed to the interpretation of findings, provided inputs at every stage of the project and were key decision-makers. The challenges include additional work to make the technical materials accessible, extra time to the project timescales, including time to achieve consensus from different opinions, sometimes strongly held, and extra costs. CONCLUSION: This study demonstrates a successful example of how PI can be embedded in research, namely in instrument development. The rewards of doing so cannot be emphasised enough but there are challenges, albeit surmountable ones. Researchers should anticipate and address those challenges during the planning stage of the project.

Recovering Quality of Life (ReQoL): a new generic self-reported outcome measure for use with people experiencing mental health difficulties.

BACKGROUND: Outcome measures for mental health services need to adopt a service-user recovery focus. Aims To develop and validate a 10- and 20-item self-report recovery-focused quality of life outcome measure named Recovering Quality of Life (ReQoL). METHOD: Qualitative methods for item development and initial testing, and quantitative methods for item reduction and scale construction were used. Data from >6500 service users were factor analysed and item response theory models employed to inform item selection. The measures were tested for reliability, validity and responsiveness. RESULTS: ReQoL-10 and ReQoL-20 contain positively and negatively worded items covering seven themes: activity, hope, belonging and relationships, self-perception, well-being, autonomy, and physical health. Both versions achieved acceptable internal consistency, test-retest reliability (>0.85), known-group differences, convergence with related measures, and were responsive over time (standardised response mean (SRM) > 0.4). They performed marginally better than the Short Warwick-Edinburgh Mental Well-being Scale and markedly better than the EQ-5D. CONCLUSIONS: Both versions are appropriate for measuring service-user recovery-focused quality of life outcomes. Declaration of interest M.B. and J.Co. were members of the research group that developed the Clinical Outcomes in Routine Evaluation (CORE) outcome measures.

Using a Discrete-Choice Experiment Involving Cost to Value a Classification System Measuring the Quality-of-Life Impact of Self-Management for Diabetes.

OBJECTIVES: To describe the use of a novel approach in health valuation of a discrete-choice experiment (DCE) including a cost attribute to value a recently developed classification system for measuring the quality-of-life impact (both health and treatment experience) of self-management for diabetes. METHODS: A large online survey was conducted using DCE with cost on UK respondents from the general population (n = 1497) and individuals with diabetes (n = 405). The data were modeled using a conditional logit model with robust standard errors. The marginal rate of substitution was used to generate willingness-to-pay (WTP) estimates for every state defined by the classification system. Robustness of results was assessed by including interaction effects for household income. RESULTS: There were some logical inconsistencies and insignificant coefficients for the milder levels of some attributes. There were some differences in the rank ordering of different attributes for the general population and diabetic patients. The WTP to avoid the most severe state was £1118.53 per month for the general population and £2356.02 per month for the diabetic patient population. The results were largely robust. CONCLUSIONS: Health and self-management can be valued in a single classification system using DCE with cost. The marginal rate of substitution for key attributes can be used to inform cost-benefit analysis of self-management interventions in diabetes using results from clinical studies in which this new classification system has been applied. The method shows promise, but found large WTP estimates exceeding the cost levels used in the survey.