Measuring patient-reported outcomes: moving beyond misplaced common sense to hard science

Interest in the patient's views of his or her illness and treatment has increased dramatically. However, our ability to appropriately measure such issues lags far behind the level of interest and need. Too often such measurement is considered to be a simple and trivial activity that merely requires the application of common sense. However, good quality measurement of patient-reported outcomes is a complex activity requiring considerable expertise and experience. This review considers the most important issues related to such measurement in the context of chronic disease and details how instruments should be developed, validated and adapted for use in additional languages. While there is often consensus on how best to undertake these activities, there is generally little evidence to support such accord. The present article questions these orthodox views and suggests alternative approaches that have been shown to be effective

Is the EQ-5D fit for purpose in asthma? Acceptability and content validity from the patient perspective

Background The increasing emphasis on patient-reported outcomes in health care decision making has prompted greater rigor in the evidence to support the instruments used. Acceptability and content validity are important properties of any measure to ensure it assesses the relevant aspects of the target concept. The purpose of this study was to evaluate the acceptability and content validity of the EQ-5D 5-Level (EQ-5D-5L) to assess the impact of asthma on patients’ lives. Methods Qualitative interviews were conducted with 40 adults with asthma in the United Kingdom. The first 25 interviews used cognitive-debriefing methods to assess the relevance and acceptability of the EQ-5D-5L and two asthma-specific measures for comparison: an asthma-specific, preference-based measure (the Asthma Quality of Life Utility Index–5 Dimensions) and an Asthma Symptom Diary. The final 15 interviews combined concept elicitation to identify patient-perceived asthma impact, and cognitive debriefing to assess relevance and acceptability of the EQ-5D-5L and the Asthma Symptom Diary. Cognitive-debriefing feedback on the content of the measures was collated and summarized descriptively. The concept-elicitation data were analyzed thematically. Results Participants were aged 20 to 57 years and 62.5% were female. Although some participants expressed positive opinions on aspects of the EQ-5D-5L, only the usual activities dimension was consistently considered relevant to participants’ asthma experiences. The mobility and self-care dimensions prompted strong negative reactions from some participants. Variations in interpretation of the mobility dimension and difficulties with multiple concepts in the pain/discomfort and anxiety/depression dimensions also were noted. Concepts reported by participants as missing included environmental triggers, asthma symptoms, emotions, and sleep. The EQ-5D-5L was the least preferred measure to describe the impact of asthma on participants’ lives. Participants reported shortness of breath and impact on activities as especially salient issues. Conclusions The content of the EQ-5D-5L was poorly aligned with the patient-perceived impact of asthma, and the measure failed to meet basic standards for acceptability and content validity as a measure to assess the impact of asthma from the patient perspective. The shortcomings identified raise concerns regarding the appropriateness of the EQ-5D in asthma and further evaluation is warranted

The Role Of Condition-Specific Preference-Based Measures In Health Technology Assessment

A condition-specific preference-based measure (CSPBM) is a measure of health related quality of life (HRQoL) that is specific to a certain condition or disease and that can be used to obtain the quality adjustment weight of the quality adjusted life year (QALY) for use in economic models. This article provides an overview of the role of CSPBMs, the development of CSPBMs, and presents a description of existing CSPBMs in the literature. The article also provides an overview of the psychometric properties of CSPBMs in comparison to generic preference-based measures (generic PBMs), and considers the advantages and disadvantages of CSPBMs in comparison to generic PBMs. CSPBMs typically include dimensions that are important for that condition but may not be important across all patient groups. There are a large number of CSPBMs across a wide range of conditions, and these vary from covering a wide range of dimensions to more symptomatic or uni-dimensional measures. Psychometric evidence is limited but suggests that CSPBMs offer an advantage in more accurate measurement of milder health states. The mean change and standard deviation can differ for CSPBMs and generic PBMs, and this may impact on incremental cost-effectiveness ratios. CSPBMs have a useful role in HTA where a generic PBM is not appropriate, sensitive or responsive. However due to issues of comparability across different patient groups and interventions, their usage in health technology assessment is often limited to conditions where it is inappropriate to use a generic PBM or sensitivity analyses

The extratropical transition of tropical cyclones. Part I: Cyclone evolution and direct impacts

Extratropical transition (ET) is the process by which a tropical cyclone, upon encountering a baroclinic environment and reduced sea surface temperature at higher latitudes, transforms into an extratropical cyclone. This process is influenced by, and influences, phenomena from the tropics to the midlatitudes and from themeso- to the planetary scales to extents that vary between individual events. Motivated in part by recent high-impact and/or extensively observed events such as NorthAtlanticHurricane Sandy in 2012 and western North Pacific Typhoon Sinlaku in 2008, this review details advances in understanding and predicting ET since the publication of an earlier review in 2003. Methods for diagnosing ETin reanalysis, observational, andmodel-forecast datasets are discussed.New climatologies for the eastern North Pacific and southwest Indian Oceans are presented alongside updates to western North Pacific and North Atlantic Ocean climatologies. Advances in understanding and, in some cases, modeling the direct impacts of ET-related wind, waves, and precipitation are noted. Improved understanding of structural evolution throughout the transformation stage of ET fostered in large part by novel aircraft observations collected in several recent ET events is highlighted. Predictive skill for operational and numerical model ET-related forecasts is discussed along with environmental factors influencing posttransition cyclone structure and evolution. Operational ET forecast and analysis practices and challenges are detailed. In particular, somechallenges of effective hazard communication for the evolving threats posed by a tropical cyclone during and after transition are introduced. This review concludes with recommendations for future work to further improve understanding, forecasts, and hazard communication

Construction of a traditional Chinese medicine syndrome-specific outcome measure: the Kidney Deficiency Syndrome questionnaire (KDSQ)

<p>Abstract</p> <p>Background</p> <p>Development of Traditional Chinese Medicine (TCM) syndrome-specific outcome measures is needed for the evaluation of TCM syndrome-specific therapies. We constructed a Kidney Deficiency Syndrome Questionnaire (KDSQ) for the evaluation of the common TCM syndromes Kidney-Yin Deficiency Syndrome (KDS-Yin) and Kidney-Yang Deficiency Syndrome (KDS-Yang) in middle-aged women with menopausal symptoms.</p> <p>Methods</p> <p>KDS-Yin and KDS-Yang were traditionally defined by expert opinion were validated by exploratory factor analysis (EFA) and structural equation modeling (SEM). Content validity was tested by EFA on a sample of 236 women from a seminar and SEM on another sample of 321 women from a postal survey. Other psychometric properties were tested on 292 women from the seminar at baseline and two systematically selected sub-samples: 54 who reported no changes in discomforts 11–12 days after the baseline and 31 who reported changes in discomforts 67–74 days after the baseline. All participants completed the KDSQ, the Greene Climacteric Scale and the standard 12-item Short Form Health Survey.</p> <p>Results</p> <p>The EFA and SEM established the measurement models of KDS-Yin and KDS-Yang supporting content validity of the KDSQ. Internal consistency was good (Cronbach’s Alpha >0.70). Construct validity was supported by theoretically-derived levels of correlation with the established external measures. Test–retest reliability was strong (ICC_agreement: KDS-Yin, 0.94; KDS-Yang, 0.93). The KDSQ was responsive to changes over time as tested by effect size and longitudinal validity.</p> <p>Conclusions</p> <p>The KDSQ was a valid and reliable measure for KDS-Yin and KDS-Yang in Hong Kong Chinese middle-aged women with menopausal symptoms.</p