This paper describes the Kidney Disease Quality of Life (KDQOL) Instrument (dialysis version), a self-report measure that includes a 36-item health survey as the generic core, supplemented with multi-item scales targeted at particular concerns of individuals with kidney disease and on dialysis (symptom/problems, effects of kidney disease on daily life, burden of kidney disease, cognitive function, work status, sexual function, quality of social interaction, sleep). Also included were multi-item measures of social support, dialysis staff encouragement and patient satisfaction, and a single-item overall rating of health. The KDQOL was administered to 165 individuals with kidney disease (52% female; 48% male; 47% White; 27% African-American; 11% Hispanic; 8% Asian; 4% Native American; and 3% other ethnicities), sampled from nine different outpatient dialysis centres located in Southern California, the Northwest, and the Midwest. The average age of the sample was 53 years (range from 22 to 87), and 10% were 75 years or older. Internal consistency reliability estimates for the 19 multi-item scales exceeded 0.75 for every measure except one. The mean scores for individuals in this sample on the 36-item health scales were lower than the general population by one-quarter (emotional well-being) to a full standard deviation (physical function, role limitations due to physical health, general health), but similar to scores for dialysis patients in other studies. Correlations of the KDQOL scales with number of hospital days in the last 6 months were statistically significant (p < 0.05) for 14 of the 19 scales and number of medications currently being taken for nine of the scales. Results of this study provide support for the reliability and validity of the KDQOL.

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.

The assessment of health-related quality of life (HR-QOL) is an essential element of healthcare evaluation. Hundreds of generic and specific HR-QOL instruments have been developed. Generic HR-QOL instruments are designed to be applicable across a wide range of populations and interventions. Specific HR-QOL measures are designed to be relevant to particular interventions or in certain subpopulations (e.g. individuals with rheumatoid arthritis). This review examines 7 generic HR-QOL instruments: (i) the Medical Outcomes Study 36-Item Short Form (SF-36) health survey; (ii) the Nottingham Health Profile (NHP); (iii) the Sickness Impact Profile (SIP); (iv) the Dartmouth Primary care Cooperative Information Project (COOP) Charts; (v) the Quality of Well-Being (QWB) Scale; (vi) the Health Utilities Index (HUI); and (vii) the EuroQol Instrument (EQ-5D). These instruments were selected because they are commonly used and/or cited in the English language literature. The 6 characteristics of an instrument addressed by this review are: (i) conceptual and measurement model; (ii) reliability; (iii) validity; (iv) respondent and administrative burden; (v) alternative forms; and (vi) cultural and language adaptations. Of the instruments reviewed, the SF-36 health survey is the most commonly used HR-QOL measure. It was developed as a short-form measure of functioning and well-being in the Medical Outcomes Study. The Dartmouth COOP Charts were designed to be used in everyday clinical practice to provide immediate feedback to clinicians about the health status of their patients. The NHP was developed to reflect lay rather than professional perceptions of health. The SIP was constructed as a measure of sickness in relation to impact on behaviour. The QWB, HUI and EQ-5D are preference-based measures designed to summarise HR-QOL in a single number ranging from 0 to 1. We found that there are no uniformly 'worst' or 'best' performing instruments. The decision to use one over another, to use a combination of 2 or more, to use a profile and/or a preference-based measure or to use a generic measure along with a targeted measure will be driven by the purpose of the measurement. In addition, the choice will depend on a variety of factors including the characteristics of the population (e.g. age, health status, language/culture) and the environment in which the measurement is undertaken (e.g. clinical trial, routine physician visit). We provide our summary of the level of evidence in the literature regarding each instrument's characteristics based on the review criteria. The potential user of these instruments should base their instrument selection decision on the characteristics that are most relevant to their particular HR-QOL measurement needs.

The D1 model best predicts the values for observed health states. The resulting preference weight estimates represent a significant enhancement of the EQ-5D's utility for health status assessment and economic analysis in the US.

Examining the patient's subjective experience in prospective clinical comparative effectiveness research (CER) of oncology treatments or process interventions is essential for informing decision making. Patient-reported outcome (PRO) measures are the standard tools for directly eliciting the patient experience. There are currently no widely accepted standards for developing or implementing PRO measures in CER. Recommendations for the design and implementation of PRO measures in CER were developed via a standardized process including multistakeholder interviews, a technical working group, and public comments. Key recommendations are to include assessment of patient-reported symptoms as well as health-related quality of life in all prospective clinical CER studies in adult oncology; to identify symptoms relevant to a particular study population and context based on literature review and/or qualitative and quantitative methods; to assure that PRO measures used are valid, reliable, and sensitive in a comparable population (measures particularly recommended include EORTC QLQ-C30, FACT, MDASI, PRO-CTCAE, and PROMIS); to collect PRO data electronically whenever possible; to employ methods that minimize missing patient reports and include a plan for analyzing and reporting missing PRO data; to report the proportion of responders and cumulative distribution of responses in addition to mean changes in scores; and to publish results of PRO analyses simultaneously with other clinical outcomes. Twelve core symptoms are recommended for consideration in studies in advanced or metastatic cancers. Adherence to methodologic standards for the selection, implementation, and analysis/reporting of PRO measures will lead to an understanding of the patient experience that informs better decisions by patients, providers, regulators, and payers.

This study provides U.S. population norms for self-reported health status on the EQ-5D, HUI2, and HUI3. Although these measures appeared to be valid and demonstrated similarities, health status assessed by these measures is not exactly the same.

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With the advent of highly active antiretroviral therapy (HAART), HIV-infected patients are living longer and are concerned not only with a treatment's ability to extend their life but also with the quality of the life they are able to lead. Regulatory authorities are also paying closer attention to the use of health-related quality-of-life (HR-QOL) measures in clinical trials and to the subsequent claims that are made based on the results. This paper reviews existing HR-QOL measures reported in the HIV/AIDS literature since 1990 and identifies those most worthy of consideration for use in future clinical trials.A comprehensive review following predefined selection criteria was conducted. Generic and HIV-targeted measures were assessed for content and practicality for the clinical trial setting. The generic measures were additionally reviewed for the ability to produce preference-based index scores and for the existence of normative general population data. Three generic and six HIV-targeted measures met these selection criteria and were then assessed more fully in terms of their development (HIV-targeted measures), psychometric properties and appropriateness for use in clinical trials.It was determined that each of the selected generic measures (i.e. Medical Outcomes Study [MOS] 36-Item Short Form Survey Instrument [SF-36], EQ-5D, Health Utilities Index [HUI]) could serve as a useful adjunct to an HIV-targeted measure in a trial. The Functional Assessment of HIV Infection (FAHI) and MOS-HIV health survey were deemed the two most appropriate HIV-targeted measures. Each of the measures can be self-administered in < or = 10 minutes and there was ample evidence of their excellent psychometric properties. However, they would not be optimal in all HIV-infected subgroups (e.g. treatment naive vs advanced; adolescents vs older adults) targeted for clinical trial interventions. Although there is no one best HR-QOL measure for use in HIV/AIDS clinical trials, based on our review criteria we identified three generic and two HIV-targeted candidate measures. However, these measures have their limitations and it is clear that greater consensus needs to develop regarding more effective and efficient approaches to HR-QOL measurement in HIV/AIDS clinical trials. Along with the increasingly complex HR-QOL measurement task resulting from changes in the HIV-infected population and shifts in the HR-QOL burden associated with HIV infection and its treatment over the past 25 years, it is increasingly important that HR-QOL outcomes become viable endpoints in HIV/AIDS clinical trials.