Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
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Patient-reported outcome (PRO) instruments are used to evaluate the effect of medical products on how patients feel or function. This article presents the results of an ISPOR task force convened to address good clinical research practices for the use of existing or modified PRO instruments to support medical product labeling claims. The focus of the article is on content validity, with specific reference to existing or modified PRO instruments, because of the importance of content validity in selecting or modifying an existing PRO instrument and the lack of consensus in the research community regarding best practices for establishing and documenting this measurement property. ⋯ Published evidence of the content validity of a PRO instrument for an intended application is often limited. Such evidence is, however, important to evaluating the adequacy of a PRO instrument for the intended application. This article provides an overview of key issues involved in assessing and documenting content validity as it relates to using existing instruments in the drug approval process.
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The goal of comparative effectiveness analysis is to examine the relationship between two variables, treatment, or exposure and effectiveness or outcome. Unlike data obtained through randomized controlled trials, researchers face greater challenges with causal inference with observational studies. Recognizing these challenges, a task force was formed to develop a guidance document on methodological approaches to addresses these biases. ⋯ Design of nonrandomized studies of comparative effectiveness face several daunting issues, including measurement of exposure and outcome challenged by misclassification and confounding. Use of causal diagrams and restriction are two techniques that can improve the theoretical basis for analyzing treatment effects in study populations of more homogeneity, with reduced loss of generalizability.
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Comparative Study
Mapping the cancer-specific EORTC QLQ-C30 to the preference-based EQ-5D, SF-6D, and 15D instruments.
To estimate models, via ordinary least squares regression, for predicting Euro Qol 5D (EQ-5D), Short Form 6D (SF-6D), and 15D utilities from scale scores of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30). ⋯ Evidence on the ability of QLQ-C30 scale scores to validly predict 15D and SF-6D utilities, and to a lesser extent, EQ-5D, has been provided. The modeling equations must be tried in future studies with larger and more diverse samples to confirm their appropriateness for estimating quality-adjusted life-year in cancer-patient trials including only the QLQ-C30.
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This study aimed to evaluate the length of stay (LOS), costs, and treatment consistency among patients hospitalized with community-acquired pneumonia (CAP) initially treated with intravenous (IV) moxifloxacin 400 mg or IV levofloxacin 750 mg. ⋯ In-hospital treatment of CAP with IV moxifloxacin 400 mg or IV levofloxacin 750 mg was associated with similar hospital LOS and costs in propensity-matched cohorts.
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One-year adjuvant trastuzumab therapy increases disease-free and overall survival in the adjuvant treatment of early HER2-positive breast cancer. This study aims to assess the long-term cost-effectiveness of adjuvant trastuzumab treatment in Beijing, Shanghai, and Guangzhou. ⋯ The results suggest that the 1-year adjuvant trastuzumab treatment is cost-effective. Both clinical and economic benefits were superior for the 1-year adjuvant trastuzumab treatment group compared with the standard adjuvant chemotherapy group.