Psychometric Analysis of the Abdominal Score From the Diary for Irritable Bowel Syndrome Symptoms-Constipation Using Phase IIb Clinical Trial Data.

OBJECTIVES: The Diary for Irritable Bowel Syndrome Symptoms-Constipation (DIBSS-C) has been developed to assess the core signs and symptoms of irritable bowel syndrome with constipation (IBS-C). This article presents the psychometric evaluation of the DIBSS-C abdominal score. METHODS: Data for these analyses are from a multicenter phase IIb study in IBS-C patients (NCT02559206). Subjects completed a number of assessments via handheld electronic diary throughout the study. The analyses used the intent-to-treat population and were blinded to randomized treatment group. The analyses evaluated the reliability, validity, and responsiveness of the DIBSS-C abdominal score; identified an appropriate scoring algorithm; and determined thresholds for interpreting clinically meaningful changes at the individual level. RESULTS: The correlations between the DIBSS-C abdominal symptom items (ie, abdominal pain, discomfort, and bloating) were strong (>0.75). Cronbach's alpha for the abdominal symptom severity items was very strong (.94), indicating that the 3 abdominal symptom items produce a reliable score. The intraclass correlation coefficient for the abdominal score was 0.82, exceeding the threshold of 0.70 and indicating good test-retest reliability. Guyatt's responsiveness statistic values all exceeded the threshold for a large effect of 0.80, so the DIBSS-C abdominal score can be considered highly responsive to change. Triangulation across 3 sets of anchor-based analyses indicated that a threshold of -2.0 points on the abdominal score is an appropriate threshold for identifying meaningful change. CONCLUSIONS: Overall, this study provides evidence that the DIBSS-C abdominal score is valid, reliable, responsive to change, and interpretable for assessing treatment benefit in patients with IBS-C.

Assessing Asthma Symptoms in Adolescents and Adults: Qualitative Research Supporting Development of the Asthma Daily Symptom Diary.

BACKGROUND: Despite the widespread availability of patient-reported asthma questionnaires, instruments developed in accordance with present regulatory expectations are lacking. To address this gap, the Patient-Reported Outcome (PRO) Consortium's Asthma Working Group has developed a patient-reported asthma daily symptom diary (ADSD) for use in clinical research to assess outcomes and support medical product labeling claims in adults and adolescents with asthma. OBJECTIVES: To summarize the qualitative research conducted to inform the initial development of the ADSD and to provide evidence for content validity of the instrument in accordance with the Food and Drug Administration's PRO Guidance. METHODS: Research informing the initial development and confirming the content validity of the ADSD is summarized. This comprised a review of published qualitative research, semi-structured concept elicitation interviews (n = 55), and cognitive interviews (n = 65) with a diverse and representative sample of adults and adolescents with a clinician-confirmed diagnosis of asthma in the United States to understand the asthma symptom experience and to assess the relevance and understanding of the newly developed ADSD. RESULTS: From the qualitative literature review and concept elicitation interviews, eight core asthma symptoms emerged. These were broadly categorized as breathing symptoms (difficulty breathing, shortness of breath, and wheezing), chest symptoms (chest tightness, chest pain, and pressure/weight on chest), and cough symptoms (cough and the presence of mucus/phlegm). Conceptual saturation was achieved and differences in the experience of participants according to socio-demographic or clinical characteristics were not observed. Subsequent testing of the ADSD confirmed participant relevance and understanding. CONCLUSIONS: The ADSD is a new patient-reported asthma symptom diary developed in accordance with the Food and Drug Administration's PRO Guidance. Evidence to date supports the content validity of the instrument. Item performance, reliability, and construct validity will be assessed in future quantitative research.

Development of a patient-reported outcome instrument to assess complex activities of daily living and interpersonal functioning in persons with mild cognitive impairment: The qualitative research phase.

INTRODUCTION: As drug development research efforts move toward studying patients earlier in the course of Alzheimer's disease (AD), it is important to incorporate the patient's perspective into measurement of outcomes. METHODS: This article summarizes the qualitative work of the Patient-Reported Outcome Consortium's Cognition Working Group in the development of a new self-reported outcome measure in persons with mild cognitive impairment (MCI) due to suspected AD, herein referred to as MCI. RESULTS: The draft measure captures the patient's voice for two functional domains, complex activities of daily living and interpersonal functioning. DISCUSSION: This work represents a series of initial steps in the development of this rating scale. The next steps are to conduct psychometric analysis and evaluate the role of insight.

Testing the measurement equivalence of paper and interactive voice response system versions of the EORTC QLQ-C30.

PURPOSE: The objective of this study was to evaluate the measurement equivalence of an interactive voice response system (IVRS) version and the original paper-based version of the EORTC QLQ-C30. METHODS: The QLQ-C30 is a cancer-specific, health-related quality of life questionnaire consisting of nine multi-item scales (physical, role, emotional, cognitive and social functioning, fatigue, nausea/vomiting, pain, and quality of life) and six single item measures (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial problems). This study utilized a crossover design with subjects randomly assigned to one of two assessment orders: (1) paper then IVRS or (2) IVRS then paper. Equivalence between the two administration modes was established by comparing the 95% lower confidence interval (CI) of the intraclass correlation coefficients (ICCs) for each scale, with a critical value of 0.70. RESULTS: The ICCs for the nine multi-item scales were all above 0.79, ranging from 0.791 to 0.899 (ICC 95% lower CI range 0.726-0.865) and significantly different from our threshold reliability of 0.70. The ICCs for the six single items ranged from 0.689 to 0.896 (ICC 95% lower CI range 0.611-0.888). Two of the items, insomnia and appetite loss, were not statistically different from 0.70. When considered together, the per-protocol analysis results support the equivalence of the paper and IVRS versions of the QLQ-C30 for 13 of the 15 scores. CONCLUSION: This analysis provides evidence that the scores obtained from the IVRS version of the QLQ-C30 are equivalent to those obtained with the original paper version except for the insomnia and appetite loss items.

Measurement equivalence of interactive voice response and paper versions of the EQ-5D in a cancer patient sample.

OBJECTIVE: To assess the measurement equivalence of an interactive voice response (IVR) version of the EQ-5D with the original paper version. METHODS: Subjects were randomly assigned to: 1) paper then IVR, or 2) IVR then paper and asked to complete the questionnaire two days apart. The analyses tested mean differences (repeated measures analysis of variance) and reliability (intraclass correlation coefficient [ICC]). Equivalence of the means was established if the 95% confidence interval (CI) of the mean difference was within the minimally important difference interval: -0.035 to 0.035 for the EQ-5D index and -3 to 3 for the visual analog scale (EQ VAS). ICC adequacy was tested by comparing the ICC 95% lower CI with a critical value of 0.70. RESULTS: The analyses included 113 subjects for the index and 109 subjects for the EQ VAS. For the index, the adjusted means of the paper and IVR versions were 0.789 ± 0.016 and 0.798 ± 0. 017, respectively. The 95% CI of the mean difference was -0.024 to 0.006, within the equivalence interval. The ICC was 0.894 (95% lower CI 0.857), significantly greater than 0.70. For the EQ VAS, the adjusted means were 71.94 ± 1.87 for paper and 74.63 ± 1.79 for IVR. The 95% CI of the mean difference was -4.347 to -1.049, partially within the equivalence interval. The ICC was 0.887 (95% lower CI 0.840), significantly greater than 0.70. CONCLUSIONS: The results provide evidence that the EQ-5D scores on the IVR version were sufficiently equivalent to those obtained on the paper version.

Collection of Post-treatment PRO Data in Oncology Clinical Trials.

As patient-reported outcome (PRO) measures are being included more frequently in oncology clinical trials, regulatory and health technology assessment agencies have begun to request long-term, post-treatment PRO data to supplement traditional survival/progression endpoints. These data may be collected as part of cohort extension or registry studies to describe long-term outcomes of study participants after concluding their cancer treatment. While post-treatment PRO data may be expected to satisfy regulatory and payer expectations, significant practical barriers exist for the efficient incorporation of these data into oncology clinical trials, such as subject attrition, protocol deviations, and treatment crossover. The incorporation of post-treatment PRO assessments is a resource-intensive task requiring clear objectives for how the data will be analyzed and interpreted by both sponsors and regulators. Incorporating PRO data collection via electronic modalities (e.g., smartphone, web) may be a less expensive and more feasible option for incorporating long-term follow-up, reducing the frequency of manual study staff follow-up and expensive clinic visits. It is essential to include well-defined estimands for the statistical analysis, as well as to document limitations associated with the long-term follow-up data-collection approach. Analytical techniques will likely rely on descriptive and model-based statistics, and conclusions about treatment differences will likely be limited to preliminary findings of effectiveness (instead of efficacy). Finally, communications with health authorities and regulatory agencies regarding the LTFU study design and analysis should occur as early as possible to ensure that the PRO data to be collected offer an opportunity to properly evaluate the research question(s) of interest.

Development of the Near Vision Presbyopia Task-based Questionnaire for use in evaluating the impact of presbyopia.

BACKGROUND: Presbyopia is a progressive condition that reduces the eye's ability to focus on near objects with increasing age. After a systematic literature review identified no existing presbyopia-specific patient-reported outcome (PRO) instruments meeting regulatory guidance, a new PRO instrument, the Near Vision Presbyopia Task-based Questionnaire (NVPTQ), was developed. RESULTS: To explore the patient experience with presbyopia, concept elicitation interviews were conducted with 20 presbyopic participants. The most frequently reported impacts were difficulty with reading menus/books/newspapers/magazines, reading on a cell phone/caller ID, and reading small print. Based on these results, a task-based PRO instrument (the NVPTQ) was developed instructing participants to complete four near-vision, paper-based reading tasks (book, newspaper, nutrition label, menu) under standardized settings, and subsequently assess their vision-related reading ability and associated satisfaction. The draft NVPTQ was cognitively debriefed with a sample of 20 presbyopes, which demonstrated that most participants interpreted the items as intended and endorsed the relevance of the concepts being assessed. After the qualitative research, the draft instrument was psychometrically tested using data from a Phase 2 study. Based on item-level analyses, all items in the NVPTQ demonstrated expected response option patterns and lacked substantial floor or ceiling effects. The reliability, validity, and responsiveness of the NVPTQ Performance and Satisfaction domain scores were assessed. All domains scores had large Cronbach's coefficient α values and good test-retest statistics, indicating that the scores are internally consistent and produce stable values over time. The pattern of correlations with a concurrent measure of visual functioning (National Eye Institute Visual Function Questionnaire 25) demonstrated that the NVPTQ domain scores were related to an alternative assessment of near-vision activities. The NVPTQ domain scores were able to distinguish between groups that were known to differ on the clinical outcome of uncorrected near visual acuity, supporting the construct validity of these scores. The NVPTQ domain scores showed evidence of responsiveness to change by being able to distinguish between groups defined as improved and not improved based on patient-reported and clinical outcomes. CONCLUSIONS: This research has resulted in a content-valid and psychometrically sound instrument designed to evaluate vision-related reading ability and satisfaction with vision-related reading ability. TRIAL REGISTRATION: ClinicalTrials.gov NCT02780115. Registered 23 May 2016, https://www.clinicaltrials.gov/ct2/show/NCT02780115?term=NCT02780115&draw=2&rank=1.

Development of the Presbyopia Impact and Coping Questionnaire.

INTRODUCTION: Presbyopia is a progressive, age-related visual condition that is characterized by reduced ability to focus on near/close objects, causing impacts on individuals' daily function and health-related quality of life. The Presbyopia Impact and Coping Questionnaire (PICQ) is a new patient-reported outcome (PRO) instrument that assesses presbyopia impact and use of coping behaviors among presbyopic individuals. METHODS: To document the impacts of presbyopia and associated coping behaviors, concept elicitation (CE) interviews were conducted with 20 presbyopic participants. Results from the CE interviews were used to develop draft items for additional testing. Following item generation, the draft PICQ was cognitively debriefed with 20 participants. Data from a phase 2 controlled clinical trial were used for psychometric analyses of the PICQ. The PICQ was administered at site visits throughout a 28-day treatment period. Confirmatory factor analysis (CFA) methods were used to guide the development of the scoring algorithm. The reliability (internal consistency, test-retest), construct validity (convergent and discriminant validity, known-groups methods), and responsiveness (Guyatt's responsiveness statistic [GRS]) of the PICQ scores were evaluated. Finally, anchor-based and distribution-based methods were used to inform thresholds for interpreting meaningful within-patient change. RESULTS: CE interviews identified the important and relevant presbyopia-related impacts and coping behaviors and 22 items were drafted and cognitively debriefed. Following minor revisions and item addition/deletion, a version of the PICQ including 23 items was subjected to psychometric testing. The analysis sample included 151 participants. The CFA established two PICQ domain scores, Coping and Impact, on 0-to-4 scales that demonstrate good model fit (root mean square error of approximation = 0.06, comparative fit index = 0.98, Tucker-Lewis index = 0.98, standardized root mean square = 0.07). Cronbach's alphas for the Coping and Impact scores were 0.89 and 0.84, respectively. Test-retest intraclass correlation coefficients were 0.77 for Coping and 0.67 for Impact. The pattern of results assessing construct validity was acceptable for the PICQ Coping and Impact scores, with the magnitude of correlations and effect sizes generally meeting a priori expectations. The corresponding GRS effect sizes for the PICQ Coping scores were -1.23 (i.e., large) for Patient Global Impression of Change (PGIC) and -0.72 (i.e., medium) for uncorrected near visual acuity (UNVA). The GRS effect sizes for the PICQ Impact scores were -0.60 (i.e., medium) for PGIC and -0.35 (i.e., small) for UNVA. Across three sets of anchor-based analyses for interpreting individual-level change, a responder threshold of -1.00 was identified for both PICQ Coping and PICQ Impact scores. CONCLUSIONS: The totality of evidence from the qualitative and quantitative research establishes that the PICQ scores produced are valid and reliable measures of presbyopia impacts and coping behaviors that are important and relevant for assessing presbyopia treatment outcomes. CLINICALTRIALS. GOV IDENTIFIER: NCT02780115; date of registration May 19, 2016.

Agreement Among Paper and Electronic Modes of the EQ-5D-5L.

INTRODUCTION: While the EQ-5D-5L has been migrated to several electronic modes, evidence supporting the measurement equivalence of the original paper-based instrument to the electronic modes is limited. OBJECTIVES: This study was designed to comprehensively examine the equivalence of the paper and electronic modes (i.e., handheld, tablet, interactive voice response [IVR], and web). METHODS: As part of the foundational work for this study, the test-retest reliability of the paper-based, UK English format of the EQ-5D-5L was assessed using a single-group, single-visit, two-period, repeated-measures design. To compare paper and electronic modes, three independent samples were recruited into a three-period crossover study. Each participant was assigned to one of six groups to account for order effects. Descriptive statistics, mean differences (i.e., split-plot analysis of variance [ANOVA]), and intraclass correlation coefficients (ICCs) were calculated. RESULTS: The test-retest results showed mean differences near zero and ICC values > 0.90 for both the index and the EQ VAS scores. For the electronic comparisons, mean difference confidence intervals (CIs) for the EQ-5D index scores and EQ VAS scores reflected equivalence of the means across all modes, as the CIs were wholly contained inside the equivalence interval. Further, the ICC 95% lower CIs for the index and EQ VAS scores showed values above the thresholds for denoting equivalence across all comparisons in each sample. No significant mode-by-order interactions were present in any ANOVA model. CONCLUSIONS: Overall, our comparisons of the paper, screen-based, and phone-based formats of the EQ-5D-5L provided substantial evidence to support the measurement equivalence of these modes of data collection.

Recommendations on evidence needed to support measurement equivalence between electronic and paper-based patient-reported outcome (PRO) measures: ISPOR ePRO Good Research Practices Task Force report.

BACKGROUND: Patient-reported outcomes (PROs) are the consequences of disease and/or its treatment as reported by the patient. The importance of PRO measures in clinical trials for new drugs, biological agents, and devices was underscored by the release of the US Food and Drug Administration's draft guidance for industry titled "Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims." The intent of the guidance was to describe how the FDA will evaluate the appropriateness and adequacy of PRO measures used as effectiveness end points in clinical trials. In response to the expressed need of ISPOR members for further clarification of several aspects of the draft guidance, ISPOR's Health Science Policy Council created three task forces, one of which was charged with addressing the implications of the draft guidance for the collection of PRO data using electronic data capture modes of administration (ePRO). The objective of this report is to present recommendations from ISPOR's ePRO Good Research Practices Task Force regarding the evidence necessary to support the comparability, or measurement equivalence, of ePROs to the paper-based PRO measures from which they were adapted. METHODS: The task force was composed of the leadership team of ISPOR's ePRO Working Group and members of another group (i.e., ePRO Consensus Development Working Group) that had already begun to develop recommendations regarding ePRO good research practices. The resulting task force membership reflected a broad array of backgrounds, perspectives, and expertise that enriched the development of this report. The prior work became the starting point for the Task Force report. A subset of the task force members became the writing team that prepared subsequent iterations of the report that were distributed to the full task force for review and feedback. In addition, review beyond the task force was sought and obtained. Along with a presentation and discussion period at an ISPOR meeting, a draft version of the full report was distributed to roughly 220 members of a reviewer group. The reviewer group comprised individuals who had responded to an emailed invitation to the full membership of ISPOR. This Task Force report reflects the extensive internal and external input received during the 16-month good research practices development process. RESULTS/RECOMMENDATIONS: An ePRO questionnaire that has been adapted from a paper-based questionnaire ought to produce data that are equivalent or superior (e.g., higher reliability) to the data produced from the original paper version. Measurement equivalence is a function of the comparability of the psychometric properties of the data obtained via the original and adapted administration mode. This comparability is driven by the amount of modification to the content and format of the original paper PRO questionnaire required during the migration process. The magnitude of a particular modification is defined with reference to its potential effect on the content, meaning, or interpretation of the measure's items and/or scales. Based on the magnitude of the modification, evidence for measurement equivalence can be generated through combinations of the following: cognitive debriefing/testing, usability testing, equivalence testing, or, if substantial modifications have been made, full psychometric testing. As long as only minor modifications were made to the measure during the migration process, a substantial body of existing evidence suggests that the psychometric properties of the original measure will still hold for the ePRO version. Hence, an evaluation limited to cognitive debriefing and usability testing only may be sufficient. However, where more substantive changes in the migration process has occurred, confirming that the adaptation to the ePRO format did not introduce significant response bias and that the two modes of administration produce essentially equivalent results is necessary. Recommendations regarding the study designs and statistical approaches for assessing measurement equivalence are provided. CONCLUSIONS: The electronic administration of PRO measures offers many advantages over paper administration. We provide a general framework for decisions regarding the level of evidence needed to support modifications that are made to PRO measures when they are migrated from paper to ePRO devices. The key issues include: 1) the determination of the extent of modification required to administer the PRO on the ePRO device and 2) the selection and implementation of an effective strategy for testing the measurement equivalence of the two modes of administration. We hope that these good research practice recommendations provide a path forward for researchers interested in migrating PRO measures to electronic data collection platforms.

Development of a Symptom-Based Patient-Reported Outcome Instrument for Functional Dyspepsia: A Preliminary Conceptual Model and an Evaluation of the Adequacy of Existing Instruments.

OBJECTIVES: The aim was to document, from the perspective of the empirical literature, the primary symptoms of functional dyspepsia (FD), evaluate the extent to which existing questionnaires target those symptoms, and, finally, identify any missing evidence that would impact the questionnaires' use in regulated clinical trials to assess treatment efficacy claims intended for product labeling. METHODS: A literature review was conducted to identify the primary symptoms of FD and existing symptom-based FD patient-reported outcome (PRO) instruments. Following a database search, abstracts were screened and articles were retrieved for review. The primary symptoms of FD were organized into a conceptual model and the PRO instruments were evaluated for conceptual coverage as well as compared against evidentiary requirements presented in the FDA's PRO Guidance for Industry. RESULTS: Fifty-six articles and 16 instruments assessing FD symptoms were reviewed. Concepts listed in the Rome III criteria for FD (n = 7), those assessed by existing FD instruments (n = 34), and symptoms reported by patients in published qualitative research (n = 6) were summarized in the FD conceptual model. Except for vomiting, all of the identified symptoms from the published qualitative research reports were also specified in the Rome III criteria. Only three of the 16 instruments, the Dyspepsia Symptom Severity Index (DSSI), Nepean Dyspepsia Index (NDI), and Short-Form Nepean Dyspepsia Index (SF-NDI), measure all seven FD symptoms defined by the Rome III criteria. Among these three, each utilizes a 2-week recall period and 5-point Likert-type scale, and had evidence of patient involvement in development. Despite their coverage, when these instruments were evaluated in light of regulatory expectations, several issues jeopardized their potential qualification for substantiation of a labeling claim. CONCLUSIONS: No existing PRO instruments that measured all seven symptoms adhered to the regulatory principles necessary to support product labeling. As such, the development of a new FD symptom PRO instrument is supported.

Test-Retest Reliability of an Interactive Voice Response (IVR) Version of the EORTC QLQ-C30.

OBJECTIVE: The objective of this study was to assess the test-retest reliability of an interactive voice response (IVR) version of the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30. METHODS: A convenience sample of outpatient cancer clinic patients (n = 127) was asked to complete the IVR version of the QLQ-C30 twice, 2 days apart. The QLQ-C30 is a 30-item, cancer-specific questionnaire composed of single-item and multi-item scales. The instrument has five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea/vomiting), and a global quality-of-life scale. The remaining single items assess dyspnea, appetite loss, insomnia, constipation, diarrhea, and financial problems. The analyses focused on intraclass correlation coefficients (ICCs), comparing the ICC 95 % lower confidence interval (CI) value with a critical value of 0.70. RESULTS: The ICCs for the nine multi-item scales were all above 0.69, ranging from 0.698 to 0.926 (ICC 95 % lower CI value range 0.588-0.895). All of the scales were significantly different from our threshold reliability of 0.70, with the exception of the cognitive functioning scale. The ICCs for the six single items ranged from 0.741 to 0.883 (ICC 95 % lower CI value range 0.646-0.835), and three of the six were statistically different from 0.70. The evidence supports the stability of 11 of the 15 scores obtained on the IVR version of the QLQ-C30 upon repeated measurement. CONCLUSION: The measurement equivalence of the IVR and paper versions of the QLQ-C30 has been reported elsewhere. This analysis provides evidence demonstrating adequate test-retest reliability of the IVR version for 11 of the QLQ-C30's 15 scores.

Considerations for Requiring Subjects to Provide a Response to Electronic Patient-Reported Outcome Instruments.

The increase in the use of electronic patient-reported outcome (ePRO) instruments has presented study teams with considerations not previously encountered with paper. Specifically, in an effort to minimize missing data, there is now the opportunity of requiring subjects to provide a response to an item before allowing the subject to proceed to the next item. While the ability to require subjects to respond to ePRO items would seem to guarantee a complete data set, it raises questions about the conditions under which it is appropriate to require subjects to respond to the items in an instrument. This article provides guidance on the circumstances under which allowing a subject to opt out of responding to ePRO items may be appropriate. Three main scenarios are discussed: (1) requiring subjects to complete all items in all the instruments in the study, (2) allowing subjects to opt out of at least some selective items that do not support key primary or secondary endpoints, and (3) allowing subjects to opt out of responding to any or all items in the study. For either of the 2 scenarios allowing the subject to opt out of responding to an item, the use of programmed edit checks is highly recommended to confirm that the subject intended to "skip" or "opt out of" the item. This ensures that, at the end of the study, the database contains an explicit data point indicating when a subject has actively decided to skip an item. While this article is focused on patient-reported outcomes, the issues raised could also apply to other clinical outcome assessments, such as clinician- and observer-reported outcomes.

Capturing Patient-Reported Outcome (PRO) Data Electronically: The Past, Present, and Promise of ePRO Measurement in Clinical Trials.

Patient-reported outcomes (PROs) are an important means of evaluating the treatment benefit of new medical products. It is recognized that PRO measures should be used when assessing concepts best known by the patient or best measured from the patient's perspective. As a result, there is growing emphasis on well defined and reliable PRO measures. In addition, advances in technology have significantly increased electronic PRO (ePRO) data collection capabilities and options in clinical trials. The movement from paper-based to ePRO data capture has enhanced the integrity and accuracy of clinical trial data and is encouraged by regulators. A primary distinction in the types of ePRO platforms is between telephone-based interactive voice response systems and screen-based systems. Handheld touchscreen-based devices have become the mainstay for remote (i.e., off-site, unsupervised) PRO data collection in clinical trials. The conventional approach is to provide study subjects with a handheld device with a device-based proprietary software program. However, an emerging alternative for clinical trials is called bring your own device (BYOD). Leveraging study subjects' own Internet-enabled mobile devices for remote PRO data collection (via a downloadable app or a Web-based data collection portal) has become possible due to the widespread use of personal smartphones and tablets. However, there are a number of scientific and operational issues that must be addressed before BYOD can be routinely considered as a practical alternative to conventional ePRO data collection methods. Nevertheless, the future for ePRO data collection is bright and the promise of BYOD opens a new chapter in its evolution.

Test-retest reliability of an interactive voice response version of the EQ-5D in a sample of cancer survivors.

BACKGROUND: Electronic data capture technologies, such as interactive voice response (IVR) systems, are emerging as important alternatives for collecting patient-reported outcome data. OBJECTIVE: The objective of this study was to assess the test-retest reliability of an IVR version of the EQ-5D. METHODS: Outpatient cancer clinic patients (n = 127) were asked to complete the IVR-based EQ-5D twice, 2 days apart. The analyses tested for mean differences (paired t-test) and test-retest reliability (intraclass correlation coefficient [ICC]) to assess measurement stability over time. Equivalence of the means was established if the 95% confidence interval (CI) was within the minimally important difference (MID) interval; namely -0.035 to 0.035 for the EQ-5D index and -3.0 to 3.0 for the visual analog scale (i.e. EQ VAS). Adequacy of the ICC was established by testing whether it differed from a value of 0.70. RESULTS: Both administrations were completed per protocol by 114 subjects (EQ-5D index) and 110 subjects (EQ VAS). For the EQ-5D index, the means (SD) of the first and second administrations were 0.871 (0.14) and 0.871 (0.15), respectively. The 95% CI of the mean difference was -0.013, 0.013, within the equivalence interval. The ICC was 0.876 (95% lower bound of 0.826) and was significantly different from 0.70. The EQ VAS means (SD) were 81.3 (17.5) and 80.8 (17.5), respectively. The 95% CI of the mean difference was -0.598, 1.617, within the equivalence interval. The EQ VAS ICC was 0.944 (95% lower bound of 0.919) and was significantly greater than 0.70. CONCLUSION: This analysis provides substantial evidence that the scores obtained from the IVR version of the EQ-5D are reliable upon repeated administrations.

Exploring household income as a predictor of psychological well-being among long-term colorectal cancer survivors.

PURPOSE: The purpose of this analysis was to determine the unique contribution of household income to the variance explained in psychological well-being (PWB) among a sample of colorectal cancer (CRC) survivors. METHODS: This study is a secondary analysis of data collected as part of the Health-Related Quality of Life in Long-Term Colorectal Cancer Survivors Study, which included CRC survivors with (cases) and without (controls) ostomies. The dataset included socio-demographic, health status, and health-related quality of life (HRQOL) information. HRQOL was assessed with the modified City of Hope Quality of Life (mCOH-QOL)-Ostomy questionnaire and SF-36v2. To assess the relationship between income and PWB, a hierarchical linear regression model was constructed combining data from both cases and controls. RESULTS: After accounting for the proportion of variance in PWB explained by the other independent variables in the model, the additional variance explained by income was significant (R (2) increased from 0.228 to 0.250; P = 0.006). CONCLUSIONS: Although the study design does not allow causal inference, these results demonstrate a significant relationship between income and PWB in CRC survivors. The findings suggest that for non-randomized group comparisons of HRQOL, income should, at the very least, be included as a control variable in the analysis.

Testing the measurement equivalence of paper and touch-screen versions of the EQ-5D visual analog scale (EQ VAS).

OBJECTIVES: This study examined the measurement equivalence of the original paper-based vertical format of the EQ-5D visual analog scale (EQ VAS) with a touch-screen computer-based horizontal format. METHODS: A total of 314 subjects were administered two modes of the EQ VAS in a randomized crossover design. One mode was the original paper-based 20 cm vertical EQ VAS; the other mode was touch-screen-based. Measurement equivalence was assessed by testing the 95% confidence interval of the mean differences from an equivalence threshold of -3 to +3 points on the VAS and evaluating the intraclass correlation coefficient (ICC). RESULTS: The adjusted mean (SE) EQ VAS score was 80.96 (0.87) on the paper and 79.59 (0.85) on the touch-screen. The mean (CI) difference between scores on the two formats was 1.37 with a confidence interval of 0.175-2.559, wholly contained within the equivalence interval. The ICC was 0.75, indicating acceptable agreement between the two modes. Almost a third (30.1%) of the respondents reported identical scores on both formats. CONCLUSION: These results provide evidence for the measurement equivalence of this EQ VAS touch-screen administration mode with the original paper mode.