Measuring Health-Related Quality of Life in Amyotrophic Lateral Sclerosis: A Systematic Review and Conceptual Framework.

BACKGROUND AND OBJECTIVES: The assessment of health-related quality of life (HRQoL) in patients with amyotrophic lateral sclerosis (ALS) is heterogeneous and inconsistent. The objectives of this study were (1) to develop a comprehensive conceptual framework of HRQoL in ALS and (2) map the content of existing patient-reported outcome measures (PROMs) used in ALS to this novel framework. METHODS: Our model of HRQoL in ALS (Health-related Quality of life in Amyotrophic Lateral Sclerosis, QuALS) was developed from a systematic literature review and consultative input from key stakeholders (patients, carers, and health care professionals). Five electronic databases were searched in April 2022. Primary studies of any design that assessed HRQoL in ALS by using a multi-item PROM and/or qualitative methods were identified. Using an a priori framework, HRQoL themes were extracted and iteratively modified from the content of each PROM and qualitative study quotations identified in the literature. The conceptual framework was ratified by stakeholders with lived experience and clinical experts. The QuALS framework was used to map the content of identified PROMs and qualitative studies based on thematic coverage. RESULTS: QuALS covers 3 high-level domains of HRQoL (physical, psychological, and social functioning) and consists of 7 themes (Activities; Physical Health; Autonomy; Cognition; Feelings and Emotions; Self-identity; Relationships), characterized by 42 subthemes. Of 8,220 studies identified, 274 were included in the review that informed QuALS. In these studies, 111 PROMs were used to assess at least 1 aspect of HRQoL, and 11 studies used qualitative methods. Of the 3 high-level domains, physical functioning was the most commonly assessed, particularly within ALS-specific PROMs where almost one-quarter of PROMs exclusively assessed physical functioning. None of the PROMs or qualitative studies identified assessed all aspects of HRQoL in the QuALS framework. DISCUSSION: This study presents a new comprehensive conceptual framework of HRQoL in ALS (QuALS), informed by a robust systematic review of existing literature and stakeholder input, incorporating lived experience. QuALS provides a valuable resource for researchers and clinicians interested in taking a holistic approach to assessing and understanding the full impact of ALS on HRQoL and how this may be affected by treatments.

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 ).

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.

Fifteen-Year Outcomes after Monitoring, Surgery, or Radiotherapy for Prostate Cancer.

BACKGROUND: Between 1999 and 2009 in the United Kingdom, 82,429 men between 50 and 69 years of age received a prostate-specific antigen (PSA) test. Localized prostate cancer was diagnosed in 2664 men. Of these men, 1643 were enrolled in a trial to evaluate the effectiveness of treatments, with 545 randomly assigned to receive active monitoring, 553 to undergo prostatectomy, and 545 to undergo radiotherapy. METHODS: At a median follow-up of 15 years (range, 11 to 21), we compared the results in this population with respect to death from prostate cancer (the primary outcome) and death from any cause, metastases, disease progression, and initiation of long-term androgen-deprivation therapy (secondary outcomes). RESULTS: Follow-up was complete for 1610 patients (98%). A risk-stratification analysis showed that more than one third of the men had intermediate or high-risk disease at diagnosis. Death from prostate cancer occurred in 45 men (2.7%): 17 (3.1%) in the active-monitoring group, 12 (2.2%) in the prostatectomy group, and 16 (2.9%) in the radiotherapy group (P = 0.53 for the overall comparison). Death from any cause occurred in 356 men (21.7%), with similar numbers in all three groups. Metastases developed in 51 men (9.4%) in the active-monitoring group, in 26 (4.7%) in the prostatectomy group, and in 27 (5.0%) in the radiotherapy group. Long-term androgen-deprivation therapy was initiated in 69 men (12.7%), 40 (7.2%), and 42 (7.7%), respectively; clinical progression occurred in 141 men (25.9%), 58 (10.5%), and 60 (11.0%), respectively. In the active-monitoring group, 133 men (24.4%) were alive without any prostate cancer treatment at the end of follow-up. No differential effects on cancer-specific mortality were noted in relation to the baseline PSA level, tumor stage or grade, or risk-stratification score. No treatment complications were reported after the 10-year analysis. CONCLUSIONS: After 15 years of follow-up, prostate cancer-specific mortality was low regardless of the treatment assigned. Thus, the choice of therapy involves weighing trade-offs between benefits and harms associated with treatments for localized prostate cancer. (Funded by the National Institute for Health and Care Research; ProtecT Current Controlled Trials number, ISRCTN20141297; ClinicalTrials.gov number, NCT02044172.).

Hypothetical versus experienced health state valuation: a qualitative study of adult general public views and preferences.

OBJECTIVES: Responses from hypothetical and experienced valuation tasks of health-related quality of life differ, yet there is limited understanding of why these differences exist, what members of the public think about them, and acceptable resolutions. This study explores public understanding of, opinions on, and potential solutions to differences between hypothetical versus experienced responses, in the context of allocating health resources. METHODS: Six focus groups with 30 members of the UK adult public were conducted, transcribed verbatim, and analysed using framework analysis. Participants self-completed the EQ-5D-5L, before reporting the expected consequences of being in two hypothetical EQ-5D-5L health states for ten years. Second, participants were presented with prior results on the same task from a public (hypothetical) and patient (experienced) sample. Third, a semi-structured discussion explored participants': (1) understanding, (2) opinions, and (3) potential resolutions. RESULTS: Twenty themes emerged, clustered by the three discussion points. Most participants found imagining the health states difficult without experience, with those aligned to mental health harder to understand. Participants were surprised that health resource allocation was based on hypothetical responses. They viewed experienced responses as more accurate, but noted potential biases. Participants were in favour of better informing, but not influencing the public. Other solutions included incorporating other perspectives (e.g., carers) or combining/weighting responses. CONCLUSION: Members of the UK public appear intuitively not to support using potentially uninformed public values to hypothetical health states in the context of health resource allocation. Acceptable solutions involve recruiting people with greater experience, including other/combinations of views, or better informing respondents.

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.

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.

Measuring quality of dying, death and end-of-life care for children and young people: A scoping review of available tools.

BACKGROUND: The circumstances and care provided at the end of a child's life have a profound impact on family members. Although assessing experiences and outcomes during this time is challenging, healthcare professionals have a responsibility to ensure high quality of care is provided. AIM: To identify available tools which measure the quality of dying, death and end-of-life care for children and young people; describe the content, and data on validity and reliability of existing tools. DESIGN: Scoping review was conducted following the Arksey and O'Malley methodological framework. DATA SOURCES: Four electronic databases (MEDLINE, EMBASE, CINAHL and PsycINFO) and grey literature were searched for studies published in English (January 2000-June 2021). A review of reference lists and citation searching was also undertaken. Tools needed to include a focus on the 'dying' phase of illness (defined as the last month of life). RESULTS: From 2078 articles, a total of 18 studies, reporting on 11 tools were identified. All tools were completed by primary caregivers or healthcare professionals as 'proxy' assessments; all except one was undertaken after death. Question items about quality of life and preparation for death were found in all tools; items relating to cultural aspects of care, grief and financial costs were less common. Only 6/11 had undergone psychometric testing within a paediatric palliative care setting. CONCLUSIONS: Future research should include ways to adapt, refine and improve existing tools. Assessing their wider application in different clinical and cultural settings and conducting further psychometric assessment represent areas of focus.

What Matters for Evaluating the Quality of Mental Healthcare? Identifying Important Aspects in Qualitative Focus Groups with Service Users and Frontline Mental Health Professionals.

BACKGROUND: Evaluating quality in mental healthcare is essential for ensuring a high-quality experience for service users (SUs). Policy-defined quality indicators, however, risk misalignment with the perspectives of SUs and mental healthcare professionals (MHPs). There is value in exploring how SUs and frontline MHPs think quality should be measured. OBJECTIVES: Our study objectives were to: (1) identify aspects that SUs and MHPs deem important for assessing quality in mental healthcare to help support attribute selection in a subsequent discrete choice experiment and (2) explore similarities and differences between SU and MHPs' views. METHODS: Semi-structured qualitative focus groups (n = 6) were conducted with SUs (n = 14) and MHPs (n = 8) recruited from a UK National Health Service Trust. A topic guide was generated from a review of UK policy documents and existing data used to measure quality in mental healthcare in England. Transcripts were analysed using a framework analysis. RESULTS: Twenty-one subthemes were identified, grouped within six themes: accessing mental healthcare; assessing the benefits of care; co-ordinated approach; delivering mental healthcare; individualised care; and role of the person providing care. Themes such as person-centred care, capacity and resources, and receiving the right type of care received more coverage than others. Service users and MHPs displayed high concordance in their views, with minor areas of divergence. CONCLUSIONS: We developed a comprehensive six-theme framework for understanding quality in mental healthcare from the viewpoint of the SU and frontline MHP, which can be used to help inform the selection of a meaningful set of quality indicators in mental health for research and practice.

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.

Exploring the Issues of Valuing Child and Adolescent Health States Using a Mixed Sample of Adolescents and Adults.

Preferences for child and adolescent health states used to generate health state utility values can be elicited from adults, young adults, adolescents, or combinations of these. This commentary paper provides a critical overview of issues and implications arising from valuing child and adolescent health states using a novel approach of a mixed sample of adolescents and adults. The commentary is informed by critical analysis of normative, ethical, practical and theoretical arguments in the health state valuation literature. Discussion focusses upon adolescent empowerment, understanding and psychosocial maturity; ethical concerns; elicitation tasks; perspective; and selection of sample proportions across adolescents and adults. It is argued that valuation of child and adolescent health states by both adolescents and adults could involve all participants completing the same preference elicitation task using the same perspective (e.g. time trade-off imagining they are living in the health state), and all preferences being modelled to generate a combined value set that reflects both adolescent and adult preferences. It is concluded that the valuation of child and adolescent health states by a mixed adolescent and adult sample appears feasible and has the advantage that it includes some of the population who can potentially experience the health states, thus enabling adolescents to express their views around matters that may affect them, and the population that are taxpayers and voters. However, both the relative proportion of adults and adolescents to include in a valuation sample and the elicitation technique require careful consideration.

Valuing quality in mental healthcare: A discrete choice experiment eliciting preferences from mental healthcare service users, mental healthcare professionals and the general population.

High and sustained healthcare quality is important worldwide, though health policy may prioritise the achievement of certain aspects of quality over others. This study determines the relative importance of different aspects of mental healthcare quality to different stakeholders by eliciting preferences in a UK sample using a discrete choice experiment (DCE). DCE attributes were generated using triangulation between policy documents and mental healthcare service user and mental healthcare professional views, whilst ensuring attributes were measurable using available data. Ten attributes were selected: waiting times; ease of access; person-centred care; co-ordinated approach; continuity; communication, capacity and resources; treated with dignity and respect; recovery focus; inappropriate discharge; quality of life (QoL). The DCE was conducted online (December 2018 to February 2019) with mental healthcare service users (n = 331), mental healthcare professionals (n = 510), and members of the general population (n = 1018). Respondents' choices were analysed using conditional logistic regression. Relative preferences for each attribute were generated using the marginal rate of substitution (MRS) with QoL as numeraire. Across all stakeholders, being treated with dignity and respect was of high importance. A coordinated approach was important across all stakeholders, whereas communication had higher relative importance for healthcare professionals and service users and ease of access had higher relative importance for the general population. This implies that policy could be affected by the choice of whose preferences (service users, healthcare professionals or general population) to use, since this impacts on the relative value and implied ranking of different aspects of mental healthcare quality.

Transforming challenges into opportunities: conducting health preference research during the COVID-19 pandemic and beyond.

The disruptions to health research during the COVID-19 pandemic are being recognized globally, and there is a growing need for understanding the pandemic's impact on the health and health preferences of patients, caregivers, and the general public. Ongoing and planned health preference research (HPR) has been affected due to problems associated with recruitment, data collection, and data interpretation. While there are no "one size fits all" solutions, this commentary summarizes the key challenges in HPR within the context of the pandemic and offers pragmatic solutions and directions for future research. We recommend recruitment of a diverse, typically under-represented population in HPR using online, quota-based crowdsourcing platforms, and community partnerships. We foresee emerging evidence on remote, and telephone-based HPR modes of administration, with further studies on the shifts in preferences related to health and healthcare services as a result of the pandemic. We believe that the recalibration of HPR, due to what one would hope is an impermanent change, will permanently change how we conduct HPR in the future.

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.

Valuing child and adolescent health: a qualitative study on different perspectives and priorities taken by the adult general public.

BACKGROUND: Quantitative health preference research has shown that different "perspectives", defined here as who is imagined to be experiencing particular health states, impact stated preferences. This qualitative project aimed to elucidate this phenomenon, within the context of adults' valuation of child and adolescent health states. METHODS: Six focus groups with 30 members of the UK adult public were conducted between December 2019 and February 2020 and analysed using framework analysis. Each focus group had two stages. First, participants individually completed time trade-off tasks and a pairwise task (mirroring a discrete choice experiment without duration) for two EQ-5D-Y health states, assuming a series of perspectives: (a) themselves at current age; (b) another adult; (c) 10-year old child; (d) themselves as a 10-year old child. Second, a semi-structured discussion explored their responses. RESULTS: Participants' views were often heterogeneous, with some common themes. Qualitatively, participants expressed a different willingness to trade-off life years for a 10-year old child versus themselves or another adult, and this differed by the health profile and child imagined. The same health states were often viewed as having a different impact on utility for a 10-year old child than adults. Imagining a 10-year old child is difficult and there is variation in who is imagined. Participants found answering based on their own-adult perspective most acceptable. There were no strong preferences for prioritising child health over working-age adults' health. CONCLUSIONS: If an adult sample is used to value child- and adolescent-specific health states it is important to consider the perspective employed. Members of the adult public provide different responses when different perspectives are used due to differences in the perceived impact of the same health states. If adults are asked to imagine a child, we recommend that sampling is representative for parental status, since this can affect preferences.

Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL.

OBJECTIVE: Based on concerns about existing patient-reported outcome measures (PROMs) for assessing quality of life (QoL) in Duchenne muscular dystrophy (DMD), we describe the mixed methods development of a new QoL PROM for use in boys and men with DMD: the DMD-QoL. METHODS: The DMD-QoL was developed in 3 stages. First, draft items were generated from 18 semistructured qualitative interviews with boys and men with DMD, analyzed using framework analysis. Second, cognitive debriefing interviews with patients (n = 10), clinicians (n = 8), and patients' parents (n = 10) were undertaken, and a reduced item set was selected and refined. Third, psychometric data on the draft items from a cross-sectional online survey (n = 102) and stakeholder input from patients and patients' parents were used to produce the final questionnaire. Patient and public involvement and engagement was embedded throughout the process. RESULTS: From an initial draft of 47 items, a revised set of 27 items was produced at stage 2, and this set was further refined at stage 3 to generate the DMD-QoL, a 14-item QoL PROM. The DMD-QoL is designed for use from 7 years of age by proxy report and from 10 years of age by self-report or proxy report. The final measure showed good psychometric properties. CONCLUSION: The DMD-QoL is a new 14-item QoL PROM for boys and men with DMD, with demonstrable content and face validity.

The role of response domain and scale label in the quantitative interpretation of patient-reported outcome measure response options.

PURPOSE: Uncertainties exist in how respondents interpret response options in patient-reported outcome measures (PROMs), particularly across different domains and for different scale labels. The current study assessed how respondents quantitatively interpret common response options. METHODS: Members of the general public were recruited to this study via an online panel, stratified by age, gender, and having English as a first language. Participants completed background questions and were randomised to answer questions on one of three domains (i.e. loneliness (negatively phrased), happiness or activities (positively phrased)). Participants were asked to provide quantitative interpretations of response options (e.g. how many times per week is equal to "often") and to order several common response options (e.g. occasionally, sometimes) on a 0-100 slider scale. Chi-squared tests and regression analyses were used to assess whether response options were interpreted consistently across domains and respondent characteristics. RESULTS: Data from 1377 participants were analysed. There was general consistency in quantifying the number of times over the last 7 days to which each response option referred. Response options were consistently assigned a lower value in the loneliness than happiness and activities domains. Individual differences, such as age and English as a second language, explained some significant variation in responses, but less than domain. CONCLUSION: Members of the public quantify common response options in a similar way, but their quantification is not equivalent across domains or every type of respondent. Recommendations for the use of certain scale labels over others in PROM development are provided.

Care Starts at Home: Emotional State and Appeals to Altruism may Reduce Demand for Overused Health Services in the UK.

BACKGROUND: Overuse of unnecessary services, screening tests, and treatments is an ongoing problem for national health care systems. Overuse is at least partly driven by patient demand. PURPOSE: This study examined whether altering patients' emotional state and appealing to patient altruism would reduce demand for three commonly overused UK health services. METHODS: In an online experiment, 1,267 UK volunteers were randomized to anxiety, compassion, or neutral conditions before viewing three overuse vignettes. In each vignette, use of the health service was recommended against by the doctor and participants were further randomized to one of three altruism frames, emphasizing the impact of overuse on the self, the self and others locally, or the self and others nationally. Participants rated the likelihood that they would pursue the health service and, assuming that they did not, how long they would be willing-to-wait for it. RESULTS: Altruism frame had a small effect on intentions to use the health service. Those in the local or national (vs. self) frame were 4.7 and 6.1 percentage points, respectively, less likely to ask for the service. Emotion induction had no direct effect on outcomes. However, self-reporting higher levels of anxiety or compassion post-induction was associated with a small, greater likelihood in intentions to ask for the health service or willingness-to-wait, respectively. No interactions between frame and emotion were observed. CONCLUSIONS: As a low-cost initiative, emphasizing the benefits to the self and local or national communities could be embedded in appeals designed to appropriately reduce health care overuse in the UK.