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Background The popularity of quality-adjusted life years (QALYs) has been resistant to concerns about validity and reliability. Utility-theoretic outcome equivalents are widely used in other areas of applied economics. Equivalence values can be derived for time, money, risk, and other metrics. These equivalence measures preserve all available information about individual preferences and are valid measures of individual welfare changes. Objective The objective of this study was to derive alternative generalized equivalence measures from first principles and illustrate their application in an empirical comparative-effectiveness example. Methods We specify a general-equilibrium model incorporating neoclassical utility functions, health production function, severity-duration preferences, and labor-market tradeoff function. The empirical implementation takes advantage of discrete-choice experiment methods that are widely accepted in other areas of applied economics and increasingly in health economics. We illustrate the practical significance of restrictive QALY assumptions using comparative-effectiveness results based on both QALYs and estimates of welfare-theoretic time-equivalent values for anti-tumor necrosis factor and prolonged corticosteroid treatments for Crohn’s disease in three distinct preference classes. Results The QALY difference between the two treatments is 0.2 months, while time-equivalent values range between 0.5 and 1.3 months for aggregate and class-specific differences. Thus, the QALY-based analysis understates welfare-theoretic values by 60%-85%. Conclusion These results suggest that using disease-specific equivalence values offer a meaningful alternative to QALYs to compare global outcomes across treatments. The equivalence values approach is consistent with principles of welfare economics and offers several features not represented in QALYs, including accounting for preference nonlinearities in disease severity and duration, inclusion of preference-relevant nonclinical healthcare factors, representing preferences of clinically-relevant patient subpopulations, and including utility losses related to risk aversion.

Objectives: With the internationalization of clinical trial programs, there is an increased need to translate and culturally adapt patient-reported outcome (PRO) measures. Although guidelines for good practices in translation and linguistic validation are available, the ISPOR Patient-Reported Outcomes Translation and Linguistic Validation Task Force identified a number of areas where they felt that further discussion around methods and best practices would be beneficial.The areas identified by the team were as follows: 1) the selection of the languages required for multinational trials; 2) the approaches suggested when the same language is required across two or more countries; and 3) the assessment of measurement equivalence to support the aggregation of data from different countries. Methods: The task force addressed these three areas, reviewed the available literature, and had multiple discussions to develop this report. Results: Decision aid tools have also been developed and presented for the selection of languages and the approaches suggested for the use of the same language in different countries. Conclusion: It is hoped that this report and the decision tools proposed will assist those involved with multinational trials to 1) decide on the translations required for each country; 2) choose the approach to use when the same language is spoken in more than one country; and 3) choose methods to gather evidence to support the pooling of data collected using different language versions of the same tool.

An initial review of the source text to be translated might allow one to anticipate translatability issues. Th is method was used to confi rm cultural acceptability of items such as “using a zipper” and “using a potty” (a device used to train a child to use a toilet). During cognitive debriefi ng of the Vineland-II Adaptive Behavior Scales [3], a series of probes were used to determine cultural appropriateness of the translated scales. Th e probes in this circumstance confi rmed the translated motions to be acceptable for the target country, but adaptation of the proper terminology for “potty” and the applicable corresponding motions was necessary.

BackgroundPatient-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.MethodsThe 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/RecommendationsAn 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.ConclusionsThe 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.

Objectives Patient-reported outcomes (PROs; self-report assessments) are increasingly important in evaluating medical care and treatment efficacy. Electronic administration of PROs via computer is becoming widespread. This article reviews the literature addressing whether computer-administered tests are equivalent to their paper-and-pencil forms. Methods Meta-analysis was used to synthesize 65 studies that directly assessed the equivalence of computer versus paper versions of PROs used in clinical trials. A total of 46 unique studies, evaluating 278 scales, provided sufficient detail to allow quantitative analysis. Results Among 233 direct comparisons, the average mean difference between modes averaged 0.2% of the scale range (e.g., 0.02 points on a 10-point scale), and 93% were within ±5% of the scale range. Among 207 correlation coefficients between paper and computer instruments (typically intraclass correlation coefficients), the average weighted correlation was 0.90; 94% of correlations were at least 0.75. Because the cross-mode correlation (paper vs. computer) is also a test–retest correlation, with potential variation because of retest, we compared it to the within-mode (paper vs. paper) test–retest correlation. In four comparisons that evaluated both, the average cross-mode paper-to-computer correlation was almost identical to the within-mode correlation for readministration of a paper measure (0.88 vs. 0.91). Conclusions Extensive evidence indicates that paper- and computer-administered PROs are equivalent.

14 CHAPTER 1: INTRODUCTION 16 I. BACKGROUND 16 II. STATEMENT OF THE PROBLEM 18 III. PURPOSE OF THE RESEARCH 20 IV. RESEARCH OBJECTIVES 20 V. RESEARCH HYPOTHESES 23 CHAPTER 2: LITERATURE REVIEW 24 I. HEALTH-RELATED QUALITY OF LIFE (HRQOL) ASSESSMENT 24 Importance of Measuring HRQoL 24 Conceptualizing HRQoL 26 Applications of HRQoL 27 HRQoL Measures 28 II. VISUAL ANALOG SCALES 30 Description 30 Characteristics of Visual Analog Scales and Issues Regarding its Use 31