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Purpose: Patient-report outcome measures (PROMs) have gained widespread support as a mechanism to improve healthcare quality. We aimed to map out key enablers and barriers influencing PROMs implementation strategies in routine clinical practice. Methods: An umbrella review was conducted to identify reviews exploring enablers and barriers related to the integration of PROMs in routine clinical practice from January 2000 to June 2023. Information on key enablers and barriers was extracted and summarised thematically according to the Theoretical Domains Framework. Results: 34 reviews met our criteria for inclusion. Identified reviews highlighted barriers such as limited PROMs awareness among clinicians and patients, perceived low value by clinicians and patients, PROMs that were too complex or difficult for patients to complete, poor usability of PROMs systems, delayed feedback of PROMs data, clinician concerns related to use of PROMs as a performance management tool, patient concerns regarding privacy and security, and resource constraints. Enablers encompassed phased implementation, professional training, stakeholder engagement prior to implementation, clear strategies and goals, 'change champions' to support PROMs implementation, systems to respond to issues raised by PROMs, and integration into patient pathways. No consensus favoured paper or electronic PROMs, yet offering both options to mitigate digital literacy bias and integrating PROMs into electronic health records emerged as important facilitators. Conclusions: The sustainable implementation of PROMs is a complex process that requires multicomponent organisational strategies covering training and guidance, necessary time and resources, roles and responsibilities, and consultation with patients and clinicians. [ABSTRACT FROM AUTHOR]

Purpose: This study describes the reporting of the preference-based health-related quality-of-life (HRQOL) instrument, the EQ-5D, and proposes strategies to improve reporting and reduce research waste. The EQ-5D is a validated instrument widely used for health economic evaluation and is useful for informing health policy.Methods: As part of a systematic review of papers reporting EQ-5D utility weights in patients with coronary artery disease, we noted the reasons data from some papers could not be reused in a meta-analysis, including whether health utility weights and sufficient statistical details were reported. Research waste was quantified using: (1) the percentage of papers and sample size excluded, and (2) researcher time and cost reviewing poorly reported papers.Results: Our search strategy found 5942 papers. At title and abstract screening 93% were excluded. Of the 379 full text papers screened, 130 papers reported using EQ-5D. Only 46% (60/130) of those studies reported utility weights and/or statistical properties enabling meta-analysis. Only 67% of included papers had reported EQ-5D in the title or abstract. A total sample size of 133,298 was excluded because of poor reporting. The cost of researcher time wasted estimated to be between $3816 and $13,279 for our review.Conclusions: Poor reporting of EQ-5D data creates research waste where potentially useful data are excluded from meta-analyses and economic evaluations. Poor reporting of HRQOL instruments also creates waste due to additional time spent reviewing papers for systematic reviews that are subsequently excluded.Recommendations: Studies using the EQ-5D should report utility weights with appropriate summary statistics to enable reuse in meta-analysis and more robust evidence for health policy. We recommend authors report the HRQOL instrument in the title or abstract in line with current reporting guidelines (CONSORT-PRO and SPIRIT-PRO Extensions) to make it easier for other researchers to find. Validated instruments should also be listed in the Medical Subject Headings (MeSH) to improve cataloguing and retrieval of previous research. [ABSTRACT FROM AUTHOR]

Purpose: Patient-reported outcome (PRO) measures are essential for assessing subjective patient experiences. Interactive voice response (IVR) data collection provides advantages for clinical trial design by standardizing and centralizing the assessment. Prior to adoption of IVR as a mode of PRO administration in the Testosterone Trials (TTrials), we compared IVR to paper versions of the instruments to be used.Methods: IVR versions of the FACIT-Fatigue scale and Psychosexual Daily Questionnaire, Question 4, were developed. In one pilot study, IVR versions of these scales were compared to paper versions in 25 men ≥ 65 years at each of two clinical sites. In another study, IVR versions of the SF-36 Vitality Scale (SF-36), Positive and Negative Affect Scale, and Patient Health Questionnaire were evaluated in comparison with previously validated paper versions in 25 men at two clinical sites. Both paper and IVR versions of each instrument were administered in counterbalanced order, and test-retest reliability was evaluated by repeated administration of the test. Bland-Altman plots were used to assess the degree of agreement. Test-retest correlations for each measure were also determined.Results: Satisfactory agreement was observed between IVR and paper versions of each study measure. Specifically, linear and highly positive associations were observed consistently across the study for IVR and paper versions of all study measures. These ranged from r = 0.91-0.99. Test-retest reliability for all measures was acceptable or better (r = 0.70-0.90).Conclusions: The IVR versions of TTrials endpoints in these two studies performed consistently well in comparison with paper versions. [ABSTRACT FROM AUTHOR]

Purpose: Patient-reported outcome (PRO) measures originally developed for paper administration are increasingly being administered electronically in clinical trials and other health research studies. Three published meta-analyses of measurement equivalence among paper and electronic modes aggregated findings across hundreds of PROs, but there has not been a similar meta-analysis that addresses a single PRO, partly because there are not enough published measurement equivalence studies using the same PRO. Because the SF-36(R) Health Survey (SF-36) is a widely used PRO, the aim of this study was to conduct a meta-analysis of measurement equivalence studies of this survey.Methods: A literature search of several medical databases used search terms for variations of "SF-36" or "SF-12" and "equivalence" in the title or abstract of English language publications. The eight scale scores and two summary measures of the SF-36 and SF-12 were transformed to norm-based scores (NBS) using developer guidelines. A threshold of within ± 2 NBS points was set as the margin of equivalence. Comprehensive meta-analysis software was used.Results: Twenty-five studies were included in the meta-analysis. Results indicated that mean differences across domains and summary scores ranged from 0.01 to 0.39 while estimates of agreement ranged from 0.76 to 0.91, all well within the equivalence threshold. Moderator analyses showed that time between administration, survey language, and type of electronic device did not influence equivalence.Conclusions: The results of the meta-analysis support equivalence of paper-based and electronic versions of the SF-36 and SF-12 across a variety of disease populations, countries, and electronic modes. [ABSTRACT FROM AUTHOR]

Purpose: The quality of patient-reported outcome (PRO) data can be compromised by non-response (NR) to scheduled questionnaires, particularly if reasons for NR are related to health problems, which may lead to unintended bias. The aim was to investigate whether electronic reminders and real-time monitoring improve PRO completion rate.Methods: The population-based study "Quality of life in Danish multiple myeloma patients" is a longitudinal, multicentre study with consecutive inclusion of treatment-demanding newly diagnosed or relapsed patients with multiple myeloma. Education of study nurses in the avoidance of NR, electronic reminders, 7-day response windows and real-time monitoring of NR were integrated in the study. Patients complete PRO assessments at study entry and at 12 follow-up time points using electronic or paper questionnaires. The effect of the electronic reminders and real-time monitoring were investigated by comparison of proportions of completed questionnaires before and after each intervention.Results: The first 271 included patients were analysed; of those, 249 (85%) chose electronic questionnaires. Eighty-four percent of the 1441 scheduled PRO assessments were completed within the 7-day response window and 11% after real-time monitoring, achieving a final PRO completion rate of 95%. A significant higher proportion of uncompleted questionnaires were completed after the patients had received the electronic reminder and after real-time monitoring.Conclusions: Electronic reminders and real-time monitoring contributed to a very high completion rate in the study. To increase the quality of PRO data, we propose integrating these strategies in PRO studies, however highlighting that an increase in staff resources is required for implementation. [ABSTRACT FROM AUTHOR]

Aim: Electronic formats (ePROs) of paper-based patient-reported outcomes (PROs) should be validated before they can be reliably used. This review aimed to examine studies investigating measurement equivalence between ePROs and their paper originals to identify methodologies used and to determine the extent of such validation. Methods: Three databases (OvidSP, Web of Science and PubMed) were searched using a set of keywords. Results were examined for compliance with inclusion criteria. Articles or abstracts that directly compared screen-based electronic versions of PROs with their validated paper-based originals, with regard to their measurement equivalence, were included. Publications were excluded if the only instruments reported were stand-alone visual analogue scales or interactive voice response formats. Papers published before 2007 were excluded, as a previous meta-analysis examined papers published before this time. Results: Fifty-five studies investigating 79 instruments met the inclusion criteria. 53 % of the 79 instruments studied were condition specific. Several instruments, such as the SF-36, were reported in more than one publication. The most frequently reported formats for ePROs were Web-based versions. In 78 % of the publications, there was evidence of equivalence or comparability between the two formats as judged by study authors. Of the 30 publications that provided preference data, 87 % found that overall participants preferred the electronic format. Conclusions: When examining equivalence between paper and electronic versions of PROs, formats are usually judged by authors to be equivalent. Participants prefer electronic formats. This literature review gives encouragement to the further widespread development and use of ePROs. [ABSTRACT FROM AUTHOR]

Purpose: To assess the equivalence of patient-reported outcome (PRO) survey responses across Web, interactive voice response system (IVRS), and paper modes of administration.Methods: Postoperative colorectal cancer patients with home Web/e-mail and phone were randomly assigned to one of the eight study groups: Groups 1-6 completed the survey via Web, IVRS, and paper, in one of the six possible orders; Groups 7-8 completed the survey twice, either by Web or by IVRS. The 20-item survey, including the MSKCC Bowel Function Instrument (BFI), the LASA Quality of Life (QOL) scale, and the Subjective Significance Questionnaire (SSQ) adapted to bowel function, was completed from home on consecutive days. Mode equivalence was assessed by comparison of mean scores across modes and intraclass correlation coefficients (ICCs) and was compared to the test-retest reliability of Web and IVRS.Results: Of 170 patients, 157 completed at least one survey and were included in analysis. Patients had mean age 56 (SD = 11), 53% were male, 81% white, 53% colon, and 47% rectal cancer; 78% completed all assigned surveys. Mean scores for BFI total score, BFI subscale scores, LASA QOL, and adapted SSQ varied by mode by less than one-third of a score point. ICCs across mode were: BFI total score (Web-paper = 0.96, Web-IVRS = 0.97, paper-IVRS = 0.97); BFI subscales (range = 0.88-0.98); LASA QOL (Web-paper = 0.98, Web-IVRS = 0.78, paper-IVRS = 0.80); and SSQ (Web-paper = 0.92, Web-IVRS = 0.86, paper-IVRS = 0.79).Conclusions: Mode equivalence was demonstrated for the BFI total score, BFI subscales, LASA QOL, and adapted SSQ, supporting the use of multiple modes of PRO data capture in clinical trials. [ABSTRACT FROM AUTHOR]

In 2014, the European Medicines Agency (EMA) released for comment a draft reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies. A twelve-member International Society for Quality of Life Research (ISOQOL) taskforce was convened to coordinate the ISOQOL response. Twenty-one ISOQOL members provided detailed comments and suggestions on the paper: 81 % from academia and 19 % from industry. Taskforce members consolidated and further refined these comments and shared the recommendations with the wider ISOQOL membership. A final response was submitted to the EMA in November 2014. The impending publication of the EMA reflection paper presents a valuable opportunity for ISOQOL to comment on the current direction of EMA PRO guidance and strategy. The EMA paper, although focused on cancer, could serve as a model for using PROs in other conditions, as it provides a useful update surrounding some of the design issues common to all trial research including PRO endpoints. However, we believe there are a number of additional areas in need of greater consideration. The purpose of this commentary is therefore to highlight the strengths of this timely and potentially useful document, but also to outline areas that may warrant further discussion. [ABSTRACT FROM AUTHOR]

Purpose: Many paper-and-pencil (P&P) questionnaires have been migrated to electronic platforms. Differential item and test functioning (DIF and DTF) analysis constitutes a superior research design to assess measurement equivalence across modes of administration. The purpose of this study was to demonstrate an item response theory (IRT)-based DIF and DTF analysis to assess the measurement equivalence of a Web-based version and the original P&P format of the Four-Dimensional Symptom Questionnaire (4DSQ), measuring distress, depression, anxiety, and somatization.Methods: The P&P group (n = 2031) and the Web group (n = 958) consisted of primary care psychology clients. Unidimensionality and local independence of the 4DSQ scales were examined using IRT and Yen's Q3. Bifactor modeling was used to assess the scales' essential unidimensionality. Measurement equivalence was assessed using IRT-based DIF analysis using a 3-stage approach: linking on the latent mean and variance, selection of anchor items, and DIF testing using the Wald test. DTF was evaluated by comparing expected scale scores as a function of the latent trait.Results: The 4DSQ scales proved to be essentially unidimensional in both modalities. Five items, belonging to the distress and somatization scales, displayed small amounts of DIF. DTF analysis revealed that the impact of DIF on the scale level was negligible.Conclusions: IRT-based DIF and DTF analysis is demonstrated as a way to assess the equivalence of Web-based and P&P questionnaire modalities. Data obtained with the Web-based 4DSQ are equivalent to data obtained with the P&P version. [ABSTRACT FROM AUTHOR]

Purpose: To extend knowledge about measurement equivalence of depression measures assessed by tablet and paper-pencil administration, the present study evaluated the effect of mode of administration (MoA) on scale and item level for the Patient Health Questionnaire (PHQ-9) and the Aachen Depression Item Bank (ADIB) in elderly patients.Methods: Primary care patients (N = 193, ≥60 years) were assessed following a crossover design in Leipzig, Germany. All participants filled out the PHQ-9 and the ADIB in both MoAs under study. Effects of MoA were analyzed by intra-class correlation, mixed-effects regression, and differential item functioning (DIF). Additionally, detection rates between both MoAs were compared using receiver operating characteristics analysis compared to a diagnostic interview (SCID-I, N = 163).Results: No effect of MoA was found in the PHQ-9 on scale score or item level. Two ADIB items showed DIF according to MoA. In terms of discriminatory power, MoA did not influence detection rates of both instruments.Conclusions: In summary, our findings suggest that no severe effect of mode of administration on self-report assessments of depression should be expected. It can be concluded that tablets provide a valid way to electronically assess depressive symptoms in elderly patients. Yet changes in item presentation can influence the psychometric properties and require equivalence testing using sophisticated analyses on item level such as DIF. [ABSTRACT FROM AUTHOR]

Objectives: To ascertain the impact of home non-invasive ventilation (NIV) on the health-related quality of life (HRQL) of patients with moderate-to-severe chronic obstructive pulmonary disease (COPD). Design: Systematic review. Methods: A preliminary search of computerised databases (CINAHL, Medline, Clinical Key, Cochrane) was conducted in June 2021, without any limitations on publication date. Inclusion criteria focused on home NIV prescribed for patients with moderate-to-severe COPD. Identified papers were critically appraised for rigour and validity. Data were extracted, analysed, and a narrative synthesis completed. Results: The review included eight studies, including five randomised controlled trials. Variations in the HRQL scores meant that the data were difficult to collate. Nevertheless, the studies did indicate an overall improved HRQL for those using NIV at home. Conclusion: This systematic review determines that home NIV does positively impact the HRQL of those with COPD. However, the limited quality of primary studies highlights the need for more in-depth research in this area to bring about optimal standardisation of clinical practice in relation to the use of NIV at home. [ABSTRACT FROM AUTHOR]

Purpose: The Comprehensive assessment of Acceptance and Commitment Therapy (CompACT) is a 23-item questionnaire measuring psychological flexibility, a quality of life protective factor. An 18-item version was recently produced. We assessed validity and reliability of CompACT, and equivalence of paper and electronic (eCompACT) versions in people with multiple sclerosis (PwMS) in Italy, Germany and Spain. Methods: We used confirmatory factor analysis and assessed CompACT-23 and CompACT-18 measurement invariance between the three language versions. We assessed construct validity (Spearman's correlations) and internal consistency (Cronbach's alpha). Test–retest reliability (intraclass correlation coefficient, ICC) and equivalence of paper and eCompACT (ICC and linear regression model for repeated measures) were assessed in subsamples of PwMS. Results: A total of 725 PwMS completed the study. The three-factor structure of the CompACT-23 showed poor fit (RMSEA 0.07; CFI 0.82; SRMR 0.08), while the fit of the CompACT-18 was good (RMSEA 0.05; CFI 0.93; SRMR 0.05). Configural and partial metric invariance were confirmed, as well as partial scalar invariance (reached when five items were allowed to vary freely). The CompACT-18 showed good internal consistency (all alpha ≥ 0.78); and test–retest reliability (all ICCs ≥ 0.86). Equivalence between paper and eCompACT was excellent (all ICCs ≥ 0.86), with no mode, order, or interaction effects. Conclusion: Results support using the refined CompACT-18 as a three-factor measure of psychological flexibility in PwMS. Paper and eCompACT-18 versions are equivalent. CompACT-18 can be used cross-culturally, but sub-optimal scalar invariance suggests that direct comparison between the three language versions should be interpreted with caution. [ABSTRACT FROM AUTHOR]

Purpose: To validate electronic versions of the Mini Pediatric and Pediatric Asthma Caregiver's Quality of Life Questionnaires (MiniPAQLQ and PACQLQ, respectively), determine completion times and correlate QOL of children and caregivers.Methods: A total of 63 children and 64 caregivers completed the paper and electronic MiniPAQLQ or PACQLQ. Agreement between versions of each questionnaire was summarized by intraclass correlation coefficients (ICC). The correlation between MiniPAQLQ and PACQLQ scores from child-caregiver pairs was assessed using Pearson's correlation coefficient.Results: There was no significant difference (mean difference = 0.1, 95% CI -0.1, 0.2) in MiniPAQLQ Overall Scores between paper (5.9 ± 1.0, mean ± SD) and electronic (5.8 ± 1.0) versions, or any of the domains. ICCs ranged from 0.89 (Overall) to 0.86 (Emotional Function). Overall PACQLQ scores for both versions were comparable (5.9 ± 0.9 and 5.8 ± 1.0; mean difference = 0.0; 95% CI -0.1, 0.2). ICCs ranged from 0.81 (Activity Limitation) to 0.88 (Emotional Function). The electronic PACQLQ took 26 s longer (95% CI 11, 41; p < 0.001). Few participants (3-11%) preferred the paper format. MiniPAQLQ and PACQLQ scores were significantly correlated (all p < 0.05) for Overall (r paper = 0.33, r electronic = 0.27) and Emotional Function domains (r paper = 0.34, r electronic = 0.29).Conclusions: These electronic QOL questionnaires are valid, and asthma-related QOL of children and caregivers is related. [ABSTRACT FROM AUTHOR]

Purpose: Spinal cord injury (SCI) is impairment of the spinal cord that adversely affects patients' health and quality of life (QoL). The aim of the study was to assess the quality of life (QoL) and related factors in patients with traumatic spinal cord injury in Middle Eastern countries. Methods: PubMed, Ovid Medline, Cumulative Index to Nursing and Allied Health Literature (CINAHL), EBSCOhost, psycINFO, and Google Scholar were systematically searched for eligible studies published in peer-reviewed journals up till October 2023. Results: Out of 1060 papers found in the literature, only 18 studies from Iran, Lebanon, and Turkey met the inclusion criteria. The majority of the studies employed the SF-36 scale to assess for QoL. In general, all SCI patients had reduced QoL scores when compared to the healthy population. Some of the factors such as age, education level, the level of injury, time since injury onset, marriage, and job opportunities were correlated with SCI subjects QoL. Our papers were assessed and found to be of both good and high quality. Conclusion: This review emphasizes the significant shortage of QoL studies among SCI patients in the Middle East countries and highlights the importance of improving the QoL of this marginalized population. This work should enhance the governments to establish rehabilitation centers, social and economic support systems, and mental health services to diminish complications arising from SCI. [ABSTRACT FROM AUTHOR]

Purpose: Strongly framed research questions are clear as to the population (P), the exposures or interventions (E/I), comparison groups (C), outcomes (O), time when relevant (T), and what the investigator wants to know. A solid framework sets up the measurement model, analysis, and anticipated results. The purpose of this study was to estimate the extent to which research questions in journals that focused on patient-reported outcome measures (PROM) and quality of life (QOL) are clear. Methods: All 440 research articles published in four PROM journals in 2020. excluding reviews, psychometric, and qualitative papers, were reviewed. Research questions were classified as: (i) adequately framed (ii) poorly framed; or (iii) unframed based on clarity criteria. Examples from each journal were presented and reframed to match results in the article. Results: Of 440 articles, 195 (44.3%) were classified as adequately framed; 230 (52.2%) as poorly framed; and 15 (3.4%) as unframed. There was heterogeneity across journals (Chi-square: 20.8; 6 df; p = 0.002). Only 29% were framed according to what the investigators wanted to know; 72% were framed like a "to do" list; and 6% were framed as a research agenda. Conclusion: Almost half of the questions were poorly framed or unframed a practice that could contribute to research wastage. Even "adequately framed" questions rarely stated what they wanted to know a priori, increasing the risk of biased reporting. Researchers, reviewers, and editors should encourage the use established frameworks for research questions. [ABSTRACT FROM AUTHOR]

Purpose: Technological advances in recent decades have led to the availability of new modes to administer patient-reported outcomes (PROs). To aid selecting optimal modes of administration (MOA), we undertook a systematic review to determine whether differences in bias (both size and direction) exist among modes.Methods: We searched five electronic databases from 2004 (date of last comprehensive review on this topic) to April 2014, cross-referenced and searched reference lists. Studies that compared two or more MOA for a health-related PRO measure in adult samples were included. Two reviewers independently applied inclusion and quality criteria and extracted findings. Meta-analyses and meta-regressions were conducted using random-effects models.Results: Of 5100 papers screened, 222 were considered potentially relevant and 56 met eligibility criteria. No evidence of bias was found for: (1) paper versus electronic self-complete; and (2) self-complete versus assisted MOA. Heterogeneity for paper versus electronic comparison was explained by type of construct (i.e. physical vs. psychological). Heterogeneity for self-completion versus assisted modes was in part explained by setting (clinic vs. home); the largest bias was introduced when assisted completion occurred in the clinic and follow-up was by self-completion (either electronic or paper) in the home.Conclusions: Self-complete paper and electronic MOA can be used interchangeably for research in clinic and home settings. Self-completion and assisted completion produce equivalent scores overall, although heterogeneity may be induced by setting. These results support the use of mixed MOAs within a research study, which may be a useful strategy for reducing missing PRO data. [ABSTRACT FROM AUTHOR]

Purpose: Low-grade glioma (LGG) patients may face health-related quality-of-life (HRQoL) impairments, due to the tumour, treatment and associated side-effects and prospects of progression. We systematically identified quantitative studies assessing HRQoL in adult LGG patients, for: aspects of HRQoL impacted; comparisons with non-cancer controls (NCC) and other groups; temporal trends; and factors associated with HRQoL. Methods: MEDLINE, CINAHL, Embase, PubMed, and PsycINFO were systematically searched from inception to 14th September 2021. Following independent screening of titles and abstracts and full-texts, population and study characteristics, and HRQoL findings were abstracted from eligible papers, and quality appraised. Narrative synthesis was conducted. Results: Twenty-nine papers reporting 22 studies (cross-sectional, n = 13; longitudinal, n = 9) were identified. Papers were largely good quality, though many excluded patients with cognitive and communication impairments. Comparators included high-grade gliomas (HGG) (n = 7); NCCs (n = 6) and other patient groups (n = 3). Nineteen factors, primarily treatment (n = 8), were examined for association with HRQoL. There was substantial heterogeneity in HRQoL instruments used, factors and aspects of HRQoL assessed and measurement timepoints. HRQoL, primarily cognitive functioning and fatigue, in adult LGG patients is poor, and worse than in NCCs, though better than in HGG patients. Over time, HRQoL remained low, but stable. Epilepsy/seizure burden was most consistently associated with worse HRQoL. Conclusion: LGG patients experience wide-ranging HRQoL impairments. HRQoL in those with cognitive and communication impairments requires further investigation. These findings may help clinicians recognise current supportive care needs and inform types and timings of support needed, as well as inform future interventions. [ABSTRACT FROM AUTHOR]

Purpose: The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life-Core Questionnaire (QLQ-C30) is a widely used generic self-report measure of health-related quality of life (HRQOL) for cancer patients. However, no validated voice script for interviewer-led telephone administration was previously available. The aim of this study was to develop a voice script for interviewer administration via telephone. Methods: Following guidelines from the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) PRO Mixed Modes Good Research Practices Task Force, a randomised cross-over equivalence study, including cognitive debriefing, was conducted to assess equivalence between paper and telephone administration modes. Assuming an expected intraclass correlation coefficient (ICC) of 0.70 and a minimally acceptable level of 0.50, a sample size of 63 was required. Results: Cognitive interviews with five cancer patients found the voice script to be clear and understandable. Due to a protocol deviation in the first wave of testing, only 26 patients were available for analyses. A second wave of recruitment was conducted, adding 37 patients (n = 63; mean age 55.48; 65.1% female). Total ICCs for mode comparison ranged from 0.72 (nausea and vomiting, 95% CI 0.48–0.86) to 0.90 (global health status/QoL, 95% CI 0.80–0.95; pain, 95% CI 0.79–0.95; constipation, 95% CI 0.80–0.95). For paper first administration, all ICCs were above 0.70, except nausea and vomiting (ICC 0.55; 95% CI 0.24–0.76) and financial difficulties (ICC 0.60; 95% CI 0.31–0.79). For phone first administration, all ICCs were above 0.70. Conclusions: The equivalence testing results support the voice script's validity for administration of the QLQ-C30 via telephone. [ABSTRACT FROM AUTHOR]

Purpose: Assessment of patient-reported outcomes (PROs), such as health-related quality of life, has become an important component of healthcare that measures the impact of disease and medical treatment on patient health. Collecting PROs during point-of-care assessments and integrating them into the clinical setting, however, remains challenging. The objective of this pilot study was to evaluate the reliability, usability, and acceptability of point-of-care electronic PRO assessments implemented in a prostate cancer clinic.Methods: Fifty subjects completed paper-pencil and computerized formats of the Expanded Prostate Cancer Index Composite (EPIC), a validated, condition-specific QOL instrument, at separate times before treatment. Parallel-forms reliability was evaluated by comparing mean scores, variations in response distribution, and correlations between administration formats. Correlation coefficients of at least 0.70 were used for reliability testing. Differences between administration forms, indicating potential bias, were compared using the signed-rank test. A 6-item acceptability scale was also used to evaluate patient acceptability and satisfaction with the electronic format.Results: Mean scores and standard deviations were similar between the paper-pencil and electronic forms across all EPIC instrument domains, and no assessment bias was found. Each EPIC domain demonstrated a high reliability between administration formats (correlation coefficients: 0.70-0.98). The majority (>90 %) of respondents found that the computerized QOL format was user friendly and simple to use.Conclusions: Point-of-care computerized QOL assessments were reliable and acceptable to patients in this study, supporting the feasibility of PRO integration at the point-of-care in clinical settings. [ABSTRACT FROM AUTHOR]

Purpose: The number of patients with depressive disordered globally increased and affects people of all ages and countries and has a significant and negative impact on the quality of life (QoL). Depression if left untreated may lead to severe consequences. However, there are several types of effective treatments, but often patients need support from health staff to find suitable treatments. This study aims to examine the global trend of the publications as well as the development of interventions for depressing treatment. Methods: We download and analyzed 15,976 scientific research from the Web of Science from 1990 to 2018. A text mining based on Latent Dirichlet and terms' co-occurrence in titles and abstracts to identify hidden research topics and research landscapes. Results: We found that the number of papers related to non-pharmacological treatment (such as cognitive-behavioral, mindfulness, or family and social support) to improve the QoL of patients with depression has increased. The number of papers on this serious health issue in low–middle income countries (LMICs) was not as high as in high-income countries (HICs). Conclusion: It is necessary to increase support of the treatment of depression in LMICs as well as applied non-pharmacological interventions to patients with depression. [ABSTRACT FROM AUTHOR]

Purpose: In surveys and in research, proxies such as family members may be used to assess patient health-related quality of life. The aim of this research is to help cancer researchers select a validated health-related quality of life tool if they anticipate using proxy-reported data.Methods: Systematic review and methodological appraisal of studies examining the concordance of paired adult cancer patient and proxy responses for multidimensional, validated HRQOL tools. We searched PubMed, CINAHL, PsycINFO and perused bibliographies of reviewed papers. We reviewed concordance assessment methods, results, and associated factors for each validated tool.Results: A total of 32 papers reporting on 29 study populations were included. Most papers were cross-sectional (N = 20) and used disease-specific tools (N = 19), primarily the FACT and EORTC. Patient and proxy mean scores were similar on average for tools and scales, with most mean differences <10 points but large standard deviations. Average ICCs for the FACT and EORTC ranged from 0.35 to 0.62, depending on the scale. Few papers (N = 15) evaluated factors associated with concordance, and results and measurement approaches were inconsistent. The EORTC was the most commonly evaluated disease-specific tool (N = 5 papers). For generic tools, both concordance and associated factor information was most commonly available for the COOP/WONCA (N = 3 papers). The MQOL was the most frequently evaluated end-of-life tool (N = 3 papers).Conclusions: Proxy and patient scores are similar on average, but there is large, clinically important residual variability. The evidence base is strongest for the EORTC (disease-specific tools), COOP/WONCA (generic tools), and MQOL (end-of-life-specific tools). [ABSTRACT FROM AUTHOR]

Objective: To assess the validity of the e-OAKHQOL questionnaire and analyze whether the answers were affected by the form of administration (electronic vs. paper).Methods: Two samples of patients with knee osteoarthritis were constituted. The first was recruited by general practitioners. Patients could choose to respond to the electronic or paper version. The second included subjects who responded to the paper version and were matched with respondents to the electronic version in the first sample. The OAKHQOL questionnaire measures health-related quality of life in five dimensions (43 items): physical activity, mental health, pain, social functioning, and social support. Validity was assessed by the classical test theory (CTT) and a Rasch measurement model (partial credit model).Results: The electronic form was preferred by 471 (89.7%) patients: 345 were matched to respondents of the paper version. The percentage of missing responses was lower with the electronic than paper form (1.6 vs. 2.0%, p = .01). Rasch analysis revealed four items with underfitting. Internal consistency was excellent for physical activity (PSI = 0.96) and mental health (PSI = 0.93) but was slightly < 0.85 for the other dimensions. The top-down purification highlighted the significance of DIF by gender in the pain dimension and by form of questionnaire in the mental health dimension.Conclusion: CTT and Rasch analysis demonstrated acceptable measurement properties for the five dimensions of the e-OAKHQOL, so it may be a valuable alternative to the paper form for measuring HRQoL. [ABSTRACT FROM AUTHOR]

Introduction: Self-report is the gold standard for measuring children's health-related outcomes. Design of such measures is complex and challenging. This review aims to systematically appraise the evidence on recall period, response scale format, mode of administration and approaches needed to enable children and young people < 19 years to participate in valid and reliable self-reporting of their health outcomes. Method: PsycInfo, Medline, CINAHL and Embase were searched from 1 January 1990 to 15 March 2020, and citation searching undertaken in Scopus. Articles were included if they were primary research or case reports of ≥ 3 participants reporting the following: recall period, response scale selection, administration modality. Quality was assessed using QualSyst, and results synthesised narratively. This review was conducted and reported according to PRISMA guidelines. Results: 81 of 13,215 retrieved articles met the inclusion criteria. Children < 5 years old cannot validly and reliably self-report health outcomes. Face scales demonstrate better psychometric properties than visual analogue or Likert scales. Computerised and paper scales generally show equivalent construct validity. Children prefer computerised measures. Children ≤ 7 years old think dichotomously so need two response options. Those > 8 years old can reliably use a 3-point scale. Conclusion: The results of this review have both clinical and research implications. They can be used to inform appropriate choice of PROM for use with CYP in the clinical setting. We also give eight recommendations for future development of self-reported outcome measures for children and young people. [ABSTRACT FROM AUTHOR]

Patient-reported outcome (PRO) questionnaires considered in this paper contain multiple subscales, although not all subscales are equally relevant for administration in all target patient populations. A group of measurement experts, developers, license holders, and other scientific-, regulatory-, payer-, and patient-focused stakeholders participated in a panel to discuss the benefits and challenges of a modular approach, defined here as administering a subset of subscales out of a multi-scaled PRO measure. This paper supports the position that it is acceptable, and sometimes preferable, to take a modular approach when administering PRO questionnaires, provided that certain conditions have been met and a rigorous selection process performed. Based on the experiences and perspectives of all stakeholders, using a modular approach can reduce patient burden and increase the relevancy of the items administered, and thereby improve measurement precision and eliminate wasted data without sacrificing the scientific validity and utility of the instrument. The panelists agreed that implementing a modular approach is not expected to have a meaningful impact on item responses, subscale scores, variability, reliability, validity, and effect size estimates; however, collecting additional evidence for the impact of context may be desirable. It is also important to recognize that adequate rationale and evidence (e.g., of fit-for-purpose status and relevance to patients) and a robust consensus process that includes patient perspectives are required to inform selection of subscales, as in any other measurement circumstance, is expected. We believe that the considerations discussed within (content validity, administration context, and psychometric factors) are relevant across multiple therapeutic areas. [ABSTRACT FROM AUTHOR]

Purpose: The aim of this study was to assess the quality of the cross-cultural adaptations and psychometric properties of the Scale of Oral Health Outcomes for 5-year-old children (SOHO-5). Methods: A systematic search was performed in three databases, PubMed, SCOPUS, and Dentistry and Oral Science Source (EBSCO). Studies relating to the cross-cultural adaptation and psychometric properties of SOHO-5 were included. The quality of cross-cultural adaptation was assessed in five aspects including translation, synthesis, back-translation, expert committee review, and pretesting. The Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) checklist was used to assess the psychometric properties in seven domains including responsiveness, internal consistency, reliability, measurement error, and structural, construct, and cross-cultural validity. Results: The search identified 162 papers for screening; from these, 107 articles and 9 systematic reviews were excluded. 21 full papers were retrieved and 6 studies that met the inclusion criteria were included. Out of six, two reported all five aspects of cross-cultural adaptation procedures. Regarding the psychometric properties, none of the studies evaluated all aspects of psychometric properties. Almost all of the studies reported internal consistency (Cronbach's alpha ranging from 0.71 to 0.90), test–retest reliability (Intraclass Correlation Coefficient 0.46–0.98), and construct validity. None reported the cross-cultural validity and responsiveness of the measures. Conclusion: While these studies provided satisfactory results regarding the validity and reliability of SOHO-5, the majority of the included studies did not report all domains of the cross-cultural adaptations and psychometric properties. Overall, the quality of the cross-cultural adaptations and psychometric properties of the included studies is doubtful. Future research should follow the guidelines to assess all aspects of psychometric properties and adaptation procedures in cross-cultural settings. [ABSTRACT FROM AUTHOR]

Purpose: To evaluate the psychometric properties of common health-related quality-of-life instruments used post stroke and provide recommendations for research and clinical use with this diagnostic group. Methods: A systematic review of the psychometric properties of the five most commonly used quality-of-life measurement tools (EQ-5D, SF-36, SF-6D, AQoL, SS-QOL) was conducted. Electronic searches were performed in MEDLINE, CINAHL, and EMBASE on November 27th 2019. Two authors screened papers against the inclusion criteria and where consensus was not reached, a third author was consulted. Included papers were appraised using the COnsensus-based Standards for the selection of health status Measurement INstruments (COSMIN) checklist and findings synthesized to make recommendations. Results: A total of n = 50,908 papers were screened and n = 45 papers reporting on 40 separate evaluations of psychometric properties met inclusion criteria (EQ-5D = 19, SF-36 = 16, SF-6D = 4, AQoL = 2, SS-QOL = 4). Studies reported varied psychometric quality of instruments, and results show that psychometric properties of quality-of-life instruments for the stroke population have not been well established. The strongest evidence was identified for the use of the EQ-5D as a quality-of-life assessment for adult stroke survivors. Conclusions: This systematic evaluation of the psychometric properties of self-reported quality-of-life instruments used with adults after stroke suggests that validity across tools should not be assumed. Clinicians and researchers alike may use findings to help identify the most valid and reliable measurement instrument for understanding the impact of stroke on patient-reported quality of life. [ABSTRACT FROM AUTHOR]

Purpose: To compare the results from a simulated computerized adaptive test (CAT) for the 28-item Impact of Vision Impairment (IVI) questionnaire and the original paper-pencil version in terms of efficiency (main outcome), defined as percentage item reduction.Methods: Using paper-pencil IVI data from 832 participants across the spectrum of vision impairment, item calibrations of the 28-item IVI instrument and its associated 20-item vision-specific functioning (VSF) and 8-item emotional well-being (EWB) subscales were generated with Rasch analysis. Based on these calibrations, CAT simulations were conducted on 1000 cases, with 'high' and 'moderate' precision stopping rules (standard error of measurement [SEM] 0.387 and 0.521, respectively). We examined the average number of items needed to satisfy the stopping rules and the corresponding percentage item reduction, level of agreement between person measures estimated from the full IVI item bank and from the CAT simulations, and item exposure rates (IER).Results: For the overall IVI-CAT, 5 or 9.7 items were required, on average, to obtain moderate or high precision estimates of vision-related quality of life, corresponding to 82.1 and 65.4% item reductions compared to the paper-pencil IVI. Agreement was high between the person measures generated from the full IVI item bank and the IVI-CAT for both the high precision simulation (mean bias, - 0.004 logits; 95% LOA - 0.594 to 0.587) and moderate precision simulation (mean bias, 0.014 logits; 95% LOA - 0.828 to 0.855). The IER for the IVI-CAT in the moderate precision simulation was skewed, with six EWB items used > 40% of the time.Conclusion: Compared to the paper-pencil IVI instrument, the IVI-CATs required fewer items without loss of measurement precision, making them potentially attractive outcome instruments for implementation into clinical trials, healthcare, and research. Final versions of the IVI-CATs are available. [ABSTRACT FROM AUTHOR]

Purpose: This systematic review was performed to identify all relevant health-related quality of life (HRQoL) issues associated with COVID-19. Methods: A systematic literature search was undertaken in April 2020. In four teams of three reviewers each, all abstracts were independently reviewed for inclusion by two reviewers. Using a pre-defined checklist of 93 criteria for each publication, data extraction was performed independently by two reviewers and subsequently compared and discussed. If necessary, a third reviewer resolved any discrepancies. The search was updated in February 2021 to retrieve new publications on HRQoL issues including issues related to the long-term consequences of COVID-19. Results: The search in April 2020 identified 3342 potentially relevant publications, and 339 publications were selected for full-text review and data extraction. We identified 75 distinct symptoms and other HRQoL issues categorized into 12 thematic areas; from general symptoms such as fever, myalgia, and fatigue, to neurological and psychological issues. The updated search revealed three extra issues experienced during active disease and long-term problems with fatigue, psychological issues and impaired cognitive function. Conclusion: This first comprehensive systematic review provides a detailed overview of the wide range of HRQoL issues experienced by patients with COVID-19 throughout the course of the disease. It demonstrates the devastating impact of the disease and provides critically important information for clinicians, to enable them to better recognize the disease and to provide knowledge important for treatment and follow-up. The results provided the foundation for the international development of a COVID-19 specific patient-reported HRQoL questionnaire. [ABSTRACT FROM AUTHOR]

Purpose: The Working Group undertook a critical, comprehensive synthesis of the response shift work to date. We aimed to (1) describe the rationale for this initiative; (2) outline how the Working Group operated; (3) summarize the papers that comprise this initiative; and (4) discuss the way forward. Methods: Four interdisciplinary teams, consisting of response shift experts, external experts, and new investigators, prepared papers on (1) definitions and theoretical underpinnings, (2) operationalizations and response shift methods, (3) implications for healthcare decision-making, and (4) on the published magnitudes of response shift effects. Draft documents were discussed during a two-day meeting. Papers were reviewed by all members. Results: Vanier and colleagues revised the formal definition and theory of response shift, and applied these in an amended, explanatory model of response shift. Sébille and colleagues conducted a critical examination of eleven response shift methods and concluded that for each method extra steps are required to make the response shift interpretation plausible. Sawatzky and colleagues created a framework for considering the impact of response shift on healthcare decision-making at the level of the individual patient (micro), the organization (meso), and policy (macro). Sajobi and colleagues are conducting a meta-analysis of published response shift effects. Preliminary findings indicate that the mean effect sizes are often small and variable across studies that measure different outcomes and use different methods. Conclusion: Future response shift research will benefit from collaboration among diverse people, formulating alternative hypotheses of response shift, and conducting the most conclusive studies aimed at testing these (falsification). [ABSTRACT FROM AUTHOR]

Purpose: Electronic patient-reported outcomes (ePROs) are increasingly being used for symptom monitoring during routine cancer care, but have rarely been evaluated in diverse patient populations. We assessed ePRO user experiences and perceived value among Black and White cancer patients.Methods: We recruited 30 Black and 49 White bladder and prostate cancer patients from a single institution. Participants reported symptoms using either a web-based or automated telephone interface over 3 months and completed satisfaction surveys and qualitative interviews focused on user experiences and value. Using a narrative mixed methods approach, we evaluated overall and race-specific differences in ePRO user experiences and perceived value.Results: Most participants selected the web-based system, but Blacks were more likely to use the automated telephone-based system than Whites. In satisfaction surveys, Whites more commonly reported ease in understanding and reporting symptoms compared with Blacks. Blacks more often reported that the ePRO system was helpful in facilitating symptom-related discussions with clinicians. During interviews, Blacks described how the ePRO helped them recognize symptoms, while Whites found value in better understanding and tracking symptoms longitudinally. Blacks also expressed preferences for paper-based ePRO options due to perceived ease in better understanding of symptom items.Conclusion: Electronic patient-reported outcomes are perceived as valuable for variable reasons by Black and White cancer populations, with greater perceived value for communicating with clinicians reported among Blacks. To optimize equitable uptake of ePROs, oncology practices should offer several ePRO options (e.g., web-based, phone-based), as well as paper-based options, and consider the e-health literacy needs of patients during implementation. [ABSTRACT FROM AUTHOR]

Purpose: Identify aspects of quality of life (QoL) important to Australian informal carers and explore how well the Adult Social Care Outcomes Toolkit for Carers, Care-related Quality of Life instrument and Carer Experience Scale capture these aspects in the Australian context. Methods: Online questionnaires were completed by Australian informal carers. Socio-demographics, open-ended questions: positive/negative aspects of caring and QoL aspects missing from the instruments, and ranking of the instrument domains was used to explore the content of the instruments. Instruments were scored using preference-weighted value sets (reported in another paper). Content analysis was used to analyse the open-ended responses. Chi-squared test looked at differences in domain importance. Descriptive analyses summarised all other information. Results: Eight themes were identified: Behaviour-mood of the care recipient, Caring responsibilities, Finances, Health, Own life, Perception of carers, Relationship with care recipient and Support. Many aspects of carer QoL mentioned as missing in the instruments appeared covered by the domains, of which all were reported as important. The highest ranked domain was relationship with the care recipient. The influence of the care recipient specific support, behaviour/mood and health on carer QoL appear absent in all instruments. Conclusion: The content of the three instruments appears relevant in an Australian setting. The influence of care recipient's health and well-being on carer QoL should be considered, along with spillover effects. A content and/or face validity analysis is required to confirm differences in item interpretation in Australian informal carers. [ABSTRACT FROM AUTHOR]

Aims: This paper aims to systematically identify reported health state utility values (HSUVs) in children and adolescents with mental health problems (MHPs) aged less than 25 years; to summarise the techniques used to elicit HSUVs; and to examine the psychometric performance of the identified multi-attribute utility instruments (MAUIs) used in this space. Methods: A systematic review was conducted following PRISMA guidelines. Peer-reviewed studies published in English, reporting HSUVs for children and adolescents with MHPs using direct or indirect valuation methods were searched in six databases. Results: We found 38 studies reporting HSUVs for 12 types of MHPs across 12 countries between 2005 and October 2021. Attention deficit hyperactivity disorder (ADHD) and depression are the most explored MHPs. Disruptive Behaviour Disorder was associated with the lowest reported HSUVs of 0.06 while cannabis use disorder was associated with the highest HSUVs of 0.88. Indirect valuation method through the use of MAUIs (95% of included studies) was the most frequently used approach, while direct valuation methods (Standard Gamble, Time Trade-Off) were only used to derive HSUVs in ADHD. This review found limited evidence of the psychometric performance of MAUIs used in children and adolescents with MHPs. Conclusion: This review provides an overview of HSUVs of various MHPs, the current practice to generate HSUVs, and the psychometric performance of MAUIs used in children and adolescents with MHPs. It highlights the need for more rigorous and extensive psychometric assessments to produce evidence on the suitability of MAUIs used in this area. [ABSTRACT FROM AUTHOR]

The poly (ADP-ribose) polymerase inhibitors (PARPi) have yielded significant clinical benefits as maintenance therapy in women with newly diagnosed and relapsed platinum-sensitive advanced ovarian cancer. These drugs were approved based on progression-free survival, the primary endpoint of their respective pivotal trials. Health-related quality of life (HRQoL) and/or patient-reported outcomes were included in these trials as a secondary exploratory endpoint. Nevertheless, many weaknesses in the analysis of HRQoL across these trials can be noticed. Heterogeneity and suboptimal HRQoL analysis in oncology trials contribute to misconceptions about this endpoint among oncologists and prevent quality of life as being an endpoint used for approvals. In this article, we discuss these HRQoL results from a methodological perspective and propose some solutions for improvement that could be used by regulatory and academic institutions running ovarian cancers trials. Notably, we suggest to measure and analyze HRQoL data after disease progression, to focus dedicated papers on the statistical analyses of HRQoL recommended by the SISAQOL consortium (linear mixed model for repeated measures and time-to-event approaches) and to communicate on available guidelines to ensure compliance with best international practices regarding the measurement and analysis of HRQoL. [ABSTRACT FROM AUTHOR]

Introduction: Through interviews with 148 older persons from four countries and in four languages, the content for a 17-item measure of active living was developed. The purpose of this paper is to present further evidence of the extent to which this new measure, Older Persons Active Living (OPAL), is "fit-for-purpose" for measuring the extent of active living at one point in time. Methods: A cross-sectional study was carried out on a population aged 65 + and living independently, drawn from a participant panel, HostedinCanada, sampling people from Canada, United States, United Kingdom, and Netherlands. The survey instrument comprised the OPAL questionnaire rated on importance and frequency, sociodemographics, and information on physical and mental function. The argument-based approach to validity framed the analyses. Logistic regression, structural equation modeling, ordinary least-squares regression, and correlation were used to generate estimates for parameters underpinning validity evidence. Results: A total of 1612 people completed the survey, 100 to 400 people across the 6 country-language strata. The proportion of people rating the items as extremely or quite important ranged from 60 to 90%, with no important differences between men and women and few differences between strata. A single-factor structure was supported. The ordinality of the response options justified an additive total score yielding a near normal distribution (mean: 33.1; SD: 11.5; range 0–51). Correlations with other measures of converging constructs were of moderate strength (~ 0.50), and differences across groups known to affect functioning and health were observed, suggesting a Miminal Important Difference (MID) of 6 out of 51. Conclusion: The results of this study provide evidence that the 17-item OPAL measure is fit for the purpose of estimating the extent to which older persons are living actively at one point in time. [ABSTRACT FROM AUTHOR]

Purpose: The Adult Social Care Outcomes Toolkit for informal carers (ASCOT-Carer) can be used to assess long-term care-related quality of life (LTC-QoL) of adult informal carers of persons using LTC services. The ASCOT-Carer instrument has been translated into several languages, but preference weights reflecting the relative importance of different outcome states are only available for England so far. In this paper, we estimated preference weights for the German version of the ASCOT-Carer for Austria and investigated the value people place on different QoL-outcome states. Methods: We used data from a best–worst scaling (BWS) experiment and estimated a scale-adjusted multinomial logit (S-MNL) model to elicit preference weights for the ASCOT-Carer domain-levels. Data were collected using an online survey of the Austrian general population (n = 1001). Results: Top levels in the domains of 'Space and time to be yourself', 'Occupation' and 'Control over daily life' were perceived as providing the highest utility, and states with high needs in the same domains seen as particularly undesirable. 'Personal safety' was the only domain where levels were roughly equidistant. In all other domains, the difference between the top two levels ('ideal state' and 'no needs') was very small. Conclusion: The paper provides preference weights for the German version of ASCOT-Carer to be used in Austrian populations. Furthermore, the results give insight into which LTC-QoL-outcomes are seen as particularly (un)desirable, and may therefore help to better tailor services directed at informal carers and the persons they care for. [ABSTRACT FROM AUTHOR]

Background: Scale development is a complex activity requiring significant investments of time and money to produce evidence of a scale's ability to produce reliable scores and valid inferences. With increasing use of clinical outcome assessments (COAs) in medical product development, evidentiary expectations of regulatory bodies to support inferences are a key consideration. The goal of this paper is to demonstrate how existing methods in measurement science can be used to identify and fill evidence gaps when considering re-purposing an existing scale for a new use case (e.g., new patient population, altering the recall period), rather than creating a new COA tool. Methods: We briefly review select validity theory and psychometric concepts, linking them to the nomenclature in the COA/regulated space. Four examples (two in-text and two in online supplemental materials) of modifications are presented to demonstrate these ideas in practice for quality of life (QOL)-related measures. Results: Each example highlights the initial process of evaluating the desired validity claims, identifying gaps in evidence to support these claims, and determining how such gaps could be filled, often without having to develop a new measure. Conclusions: If an existing scale, with minimal modification or additional evidence, can be shown to be fit for a new purpose, considerable effort can be saved and research waste avoided. In many cases, a new instrument is simply unnecessary. Far better to recycle an "old" scale for a new use–with sufficient evidence that it is fit for that purpose–than to "buy" a new one. [ABSTRACT FROM AUTHOR]

Purpose: 'Bolt-on' dimensions are additional items added to multi-attribute utility instruments (MAUIs) such as EQ-5D that measure constructs not included in the core descriptive system. The use of bolt-ons has been proposed to improve the content validity and responsiveness of the descriptive system in certain settings and health conditions. EQ-5D bolt-ons serve a particular purpose and thus satisfy a certain set of criteria. The aim of this paper is to propose a set of criteria to guide the development, assessment and selection of candidate bolt-on descriptors. Methods: Criteria were developed using an iterative approach. First, existing criteria were identified from the literature including those used to guide the development of MAUIs, the COSMIN checklist and reviews of existing bolt-ons. Second, processes used to develop bolt-ons based on qualitative and quantitative approaches were considered. The information from these two stages was formalised into draft development and selection criteria. These were reviewed by the project team and iteratively refined. Results: Overall, 23 criteria for the development, assessment and selection of candidate bolt-ons were formulated. Development criteria focused on issues relating to i) structure, ii) language, and iii) consistency with the existing EQ-5D dimension structure. Assessment and selection criteria focused on face and content validity and classical psychometric indicators. Conclusion: The criteria generated can be used to guide the development of bolt-ons across different health areas. They can also be used to assess existing bolt-ons, and inform their inclusion in studies and patient groups where the EQ-5D may lack content validity. [ABSTRACT FROM AUTHOR]

There is a recognised need for reported national data that inform health policy, health professions, and consumers about the wellbeing of Australians with cancer and other chronic conditions. International initiatives have demonstrated the viability and benefits of utilising population-based cancer registries to monitor the prevalence and trajectory of health-related quality of life (HRQOL) outcomes among people with cancer. Establishing a similar level of monitoring in Australia would require timely access to health data collected by publicly funded, population-based cancer registries, and the capacity to link this information across jurisdictions. Combining information from different sources via data linkage is an efficient and cost-effective way to maximise how data are used to inform population health and policy development. However, linking health datasets has historically been highly restricted, resource-intensive, and costly in Australia due to complex and outdated legislative requirements, duplicative approval processes, and differing policy frameworks in each state and territory. This has resulted in significant research waste due to underutilisation of existing data, duplication of research efforts and resources, and data not being translated into decision-making. Recognising these challenges, from 2015 to 2017 the Productivity Commission investigated options for improving data availability and use in Australia, considering factors such as privacy, security, and intellectual property. The inquiry report recommended significant reforms for Australian legislation, including the creation of a data sharing and release structure to improve access to data for research and policy development purposes. This paper discusses (1) opportunities in HRQOL research enabled by data linkage, (2) barriers to data access and use in Australia and the implications for waste in HRQOL research, and (3) proposed legislative reforms for improving data availability and use in Australia. [ABSTRACT FROM AUTHOR]

Background: The growth in response-shift methods has enabled a stronger empirical foundation to investigate response-shift phenomena in quality-of-life (QOL) research; but many of these methods utilize certain language in framing the research question(s) and interpreting results that treats response-shift effects as "bias," "noise," "nuisance," or otherwise warranting removal from the results rather than as information that matters. The present project will describe the various ways in which researchers have framed the questions for investigating response-shift issues and interpreted the findings, and will develop a nomenclature for such that highlights the important information about resilience reflected by response-shift findings. Methods: A scoping review was done of the QOL and response-shift literature (n = 1100 articles) from 1963 to 2020. After culling only empirical response-shift articles, raters characterized how investigators framed and interpreted study research questions (n = 164 articles). Results: Of 10 methods used, papers using four of them utilized terms like "bias" and aimed to remove response-shift effects to reveal "true change." Yet, the investigators' reflections on their own conclusions suggested that they do not truly believe that response shift is error to be removed. A structured nomenclature is proposed for discussing response-shift results in a range of research contexts and response-shift detection methods. Conclusions: It is time for a concerted and focused effort to change the nomenclature of those methods that demonstrated this misinterpretation. Only by framing and interpreting response shift as information, not bias, can we improve our understanding and methods to help to distill outcomes with and without response-shift effects. [ABSTRACT FROM AUTHOR]

Purpose: People who have complex communication needs (CCN), and who use augmentative and alternative communication (AAC) to help them express themselves, can be difficult to engage in decision making about their healthcare. The purpose of this review was to identify what patient-reported outcome measures (PROMs) have been employed with people who use AAC. Of the tools identified, the review aimed to establish what conceptual frameworks were used and how the reports describe completion of the PROM.Methods: A systematic literature review was carried out. A pre-defined set of search terms was entered into five main health and education databases. Titles and abstracts were sifted for relevance. Full text papers were screened against inclusion/exclusion criteria. Data pertaining to the type and nature of the PROM used was extracted. Complementary data sources were analysed to construct a narrative synthesis of the papers identified.Results: Within 15 papers that met the review criteria, 25 PROMs were used with people who rely on AAC comprising of 15 separate measures. The conceptual frameworks for 12 of these tools were reported from which 62 items, or concepts being measured, were identified. Following synthesis of these items, 9 conceptual domains and 11 sub-domains were generated. Limited information was available about who completed the PROM nor how much, if any, support they received.Conclusions: No PROM that has been developed specifically for people who use AAC was identified by this review. Of the tools that have been used with people who use AAC, the concepts measured were broad and varied. The quality of reporting concerning who completed the PROM was limited, undermining the trustworthiness of many of the studies. [ABSTRACT FROM AUTHOR]

Purpose: A paper reporting the development of the ICECAP-O was published in 2006. Since then, there has been increasing interest in the use of capability-based measures within health economics and the ICECAP-O has been suggested for use in economic evaluation by decision-making bodies in the Netherlands and UK.Methods: A systematic review of studies published between January 2006 and October 2018 which have assessed the psychometric properties of ICECAP-O or utilised the measure within economic evaluation.Results: Twenty-four studies explored the psychometric properties of ICECAP-O and 21 have utilised the measure within economic evaluation; one study reported psychometric properties as well as utilising the measure within economic evaluation. The ICECAP-O has good construct validity and responsiveness, but there is evidence of some issues relating to content validity. In the context of economic evaluation, the ICECAP-O has, to date, mainly been included as a secondary economic measure and the reporting of results is brief with minimal detail and often no discussion. Five of the economic evaluation studies combined scores from ICECAP-O with time, but each used different terminology to describe this result.Conclusion: Focus, in terms of publications, appears to have shifted now from assessment of psychometric properties to the utilisation of the ICECAP-O within economic evaluation. Further research is needed with respect to a decision-rule for the ICECAP measures. This additional research should also guide users in terms of appropriate analysis, terminology and presentation of results, which are in-keeping with the conceptual framework underpinning the ICECAP-O. [ABSTRACT FROM AUTHOR]

Purpose: Skeletal dysplasias are rare genetic disorders that are characterized by abnormal development of bone and cartilage. There are multiple medical and non-medical treatments for specific symptoms of skeletal dysplasias e.g. pain, as well as corrective surgical procedures to improve physical functioning. The aim of this paper was to develop an evidence-gap map of treatment options for skeletal dysplasias, and their impact on patient outcomes. Methods: We conducted an evidence-gap map to identify the available evidence on the impact of treatment options on people with skeletal dysplasias on clinical outcomes (such as increase in height), and dimensions of health-related quality of life. A structured search strategy was applied to five databases. Two reviewers independently assessed articles for inclusion in two stages: titles and abstracts (stage 1), and full text of studies retained at stage 2. Results: 58 studies fulfilled our inclusion criteria. The included studies covered 12 types of skeletal dysplasia that are non-lethal with severe limb deformities that could result in significant pain and numerous orthopaedic interventions. Most studies reported on the effect of surgical interventions (n = 40, 69%), followed by the effect of treatments on dimensions of health quality-of-life (n = 4, 6.8%) and psychosocial functioning (n = 8, 13.8%). Conclusion: Most studies reported on clinical outcomes from surgery for people living with Achondroplasia. Consequently, there are gaps in the literature on the full range of treatment options (including no active treatment), outcomes and the lived experience of people living with other skeletal dysplasias. More research is warranted to examine the impact of treatments on health-related quality-of-life of people living with skeletal dysplasias, including their relatives to enable them to make preference- and valued based decisions about treatment. [ABSTRACT FROM AUTHOR]

Purpose: Studies have found that many people who sustain an injury can experience adverse outcomes for a considerable time thereafter. Māori, the Indigenous peoples of Aotearoa me Te Waipounamu (New Zealand; NZ), are no exception. The Prospective Outcomes of Injury Study (POIS) found that almost three-quarters of Māori participants were experiencing at least one of a range of poor outcomes at two years post-injury. The aim of this paper was to estimate the prevalence, and identify predictors, of adverse health-related quality of life (HRQoL) outcomes in the POIS-10 Māori cohort, 12 years after participants sustained an injury. Methods: Interviewers reached 354 individuals who were eligible to participate in a POIS-10 Māori interview, to be conducted a decade after the last phase of POIS interviews (held 24 months post-injury). The outcomes of interest were responses to each of the five EQ-5D-5L dimensions at 12 years post-injury. Potential predictors (i.e., pre-injury sociodemographic and health measures; injury-related factors) were collected from earlier POIS interviews. Additional injury-related information was collected from administrative datasets proximate to the injury event 12 years prior. Results: Predictors of 12-year HRQoL outcomes varied by EQ-5D-5L dimension. The most common predictors across dimensions were pre-injury chronic conditions and pre-injury living arrangements. Conclusion: An approach to rehabilitation where health services proactively enquire about, and consider the broader aspects of, patient health and wellbeing throughout the injury recovery process, and effectively coordinate their patients' care with other health and social services where necessary, may help improve long-term HRQoL outcomes for injured Māori. [ABSTRACT FROM AUTHOR]

Introduction: The COVID-19 pandemic forced people to give up their daily routines and adjust to new circumstances. This might have affected health-related quality of life (HRQOL). We aimed to compare HRQOL during the first COVID-19 wave in 2020 to HRQOL before the pandemic and to identify determinants of HRQOL during the pandemic in Switzerland. Methods: We conducted a cross-sectional online survey during the pandemic (between May and July 2020; CoWELL sample; convenience sample). Before the pandemic (2015–2016), we had conducted a cross-sectional paper-based survey among a representative random sample of the Swiss general population (SGP sample). In both samples, we assessed physical and mental HRQOL (Short Form-36) and socio-demographic characteristics. In the CoWELL sample, we additionally assessed health- and COVID-19-related characteristics. Data were analysed using linear regressions. Results: The CoWELL sample included 1581 participants (76% women; mean age = 43 years, SD = 14 years) and the SGP sample 1209 participants (58% women, mean age = 49 years, SD = 15 years). Adjusted for sex, age, and education, the CoWELL sample reported higher physical HRQOL (PCS, +5.8 (95% CI: 5.1, 6.6), p < 0.001) and lower mental HRQOL (MCS, −6.9 (−7.8, −6.0), p < 0.001) than the SGP sample. In the CoWELL sample, especially persons with lower health literacy, who had no support network or who have had COVID-19, reported lower HRQOL. Discussion: Aspects unique to the COVID-19 pandemic affected HRQOL. Vulnerable persons such as those having had COVID-19, less support opportunities, and with lower health literacy are especially prone to impaired HRQOL during the COVID-19 pandemic. [ABSTRACT FROM AUTHOR]

Purpose: The collection and use of patient reported outcomes (PROs) in care-based child health research raises challenging ethical and logistical questions. This paper offers an analysis of two questions related to PROs in child health research: (1) Is it ethically obligatory, desirable or preferable to share PRO data collected for research with children, families, and health care providers? And if so, (2) What are the characteristics of a model best suited to guide the collection, monitoring, and sharing of these data? Methods: A multidisciplinary team of researchers, providers, patient and family partners, and ethicists examined the literature and identified a need for focus on PRO sharing in pediatric care-based research. We constructed and analyzed three models for managing pediatric PRO data in care-based research, drawing on ethical principles, logistics, and opportunities to engage with children and families. Results: We argue that it is preferable to share pediatric PRO data with providers, but to manage expectations and balance the risks and benefits of research, this requires a justifiable data sharing model. We argue that a successful PRO data sharing model will allow children and families to have access to and control over their own PRO data and be engaged in decision-making around how PROs collected for research may be integrated into care, but require support from providers. Conclusion: We propose a PRO data sharing model that can be used across diverse research settings and contributes to improved transparency, communication, and patient-centered care and research. [ABSTRACT FROM AUTHOR]

Introduction: A long-standing criticism of the QALY has been that it would discriminate against people in poor health: extending the lives of individuals with underlying health conditions gains fewer QALYs than extending the lives of 'more healthy' individuals. Proponents of the QALY counter that this only reflects the general public's preferences and constitutes an efficient allocation of resources. A pivotal issue that has thus far been overlooked is that there can also be negative QALYs. Methods and results: Negative QALYs are assigned to the times spent in any health state that is considered to be worse than dead. In a health economic evaluation, extending the lives of people who live in such states reduces the overall population health; it counts as a loss. The problem with this assessment is that the QALY is not based on the perspectives of individual patients—who usually consider their lives to be well worth living—but it reflects the preferences of the general public. While it may be generally legitimate to use those preferences to inform decisions about the allocation of health care resources, when it comes to states worse than dead, the implications are deeply problematic. In this paper, I discuss the (un)ethical aspects of states worse than dead and demonstrate how their use in economic evaluation leads to a systematic underestimation of the value of life-extending treatments. Conclusion: States worse than dead should thus no longer be used, and a non-negative value should be placed on all human lives. [ABSTRACT FROM AUTHOR]