Recently published articles have described criteria to assess qualitative research in the health field in general, but very few articles have delineated qualitative methods to be used in the development of Patient-Reported Outcomes (PROs). In fact, how PROs are developed with subject input through focus groups and interviews has been given relatively short shrift in the PRO literature when compared to the plethora of quantitative articles on the psychometric properties of PROs. If documented at all, most PRO validation articles give little for the reader to evaluate the content validity of the measures and the credibility and trustworthiness of the methods used to develop them. Increasingly, however, scientists and authorities want to be assured that PRO items and scales have meaning and relevance to subjects. This article was developed by an international, interdisciplinary group of psychologists, psychometricians, regulatory experts, a physician, and a sociologist. It presents rigorous and appropriate qualitative research methods for developing PROs with content validity. The approach described combines an overarching phenomenological theoretical framework with grounded theory data collection and analysis methods to yield PRO items and scales that have content validity.
ACKNOWLEDGMENTSThe individual contributions of Sorrel Wolowacz and Richard Willke are gratefully acknowledged. We thank all reviewers who commented during our forums at the ISPOR Milan and Amsterdam European Congresses. We especially thank the following individuals who reviewed drafts of the report and submitted written comments. Their feedback has both improved the manuscript and made it an expert consensus ISPOR task force report. Many thanks to Tony Ades, René Allard, Gouri Shankar Bhattacharyya, Lance Brannman, Michael Carter, David Cella, Akriti Chahar, Stephanie Yanjing Chen, Shiva Devarakonda, Salah Ghabri, Linda Gore Martin, Inigo Gorostiza, Thomas Grochtdreis, Michael Hagan, Nadine Hillock, Cynthia Holmes, Shrividya Iyer, Steve Kay, Jeanette Kusel, Ramanath KV, Dawn Lee, Joanna Le niowska, Sophia E. Marsh, Alan Martin, Nicholas Mitsakakis, Sharanya Murty, Nneka Onwudiwe, Guilhem Pietri, G.M. Rabiul Islam, Ghabri Salah, Mihail Samnaliev, Carsten Schousboe, Sarah Shingler, Fatema Turkistani, and Uday Venkat. Finally, many thanks to Theresa Tesoro for her assistance in developing this task force report. 2 AbstractEconomic evaluation conducted in terms of cost per Quality Adjusted Life Year (QALY) provides information that decision makers find useful in many parts of the world. Ideally, clinical studies designed to assess the effectiveness of health technologies would include outcome measures that are directly linked to health utility in order to calculate QALYs. Often this does not happen and, even where it does, clinical studies may be insufficient for a cost-utility assessment. Mapping can solve this problem. It uses an additional dataset to estimate the relationship between outcomes measured in clinical studies and health utility. This bridges the evidence gap between available evidence on the effect of a health technology in one metric and the requirement for decision makers to express it in a different one (QALYs). In 2014, ISPOR established a Good Practices for Outcome Research Task Force for mapping studies. This Task Force Report provides recommendations to analysts undertaking mapping studies, those that use the results in cost utility analysis, and those that need to critically review such studies. The recommendations cover all areas of mapping practice: the selection of datasets for the mapping estimation, model selection and performance assessment, reporting standards, and the use of results including the appropriate reflection of variability and uncertainty. This report is unique because it takes an international perspective, is comprehensive in its coverage of the aspects of mapping practice, and reflects the current state of the art.
Recently, mixed treatment comparisons (MTC) have been presented as an extension of traditional meta-analysis by including multiple different pairwise comparisons across a range of different interventions. MTC allow for indirect comparisons and can therefore provide very useful information for clinical and reimbursement decision-making in the absence of head-to-head data. In this article, we provide an introductory overview of MTC illustrated with example analyses of different drug treatments in rheumatoid arthritis using a continuous patient-reported end point. As a background, we start with an overview of the traditional meta-analyses for pairwise trials, and the difference between a traditional approach and a Bayesian approach. Next, the Bayesian MTC for continuous outcomes are presented. We finish with a discussion of how MTC can best be presented in order to maximize acceptance by target audiences, i.e., clinicians and market access decision-makers.
Cost-effectiveness models that present results in terms of cost per quality-adjusted life-year for health technologies are used to inform policy decisions in many parts of the world. Health state utilities (HSUs) are required to calculate the quality-adjusted life-years. Even when clinical studies assessing the effectiveness of health technologies collect data on HSUs to populate a cost-effectiveness model, which rarely happens, analysts typically need to identify at least some additional HSUs from alternative sources. When possible, HSUs are identified by a systematic review of the literature, but, again, this rarely happens. In 2014, ISPOR established a Good Practices for Outcome Research Task Force to address the use of HSUs in costeffectiveness models. This task force report provides recommendations for researchers who identify, review, and synthesize HSUs for use in cost-effectiveness models; analysts who use the results in models; and reviewers who critically appraise the suitability and validity of the HSUs selected for use in models. The associated Minimum Reporting Standards of Systematic Review of Utilities for Cost-Effectiveness checklist created by the task force provides criteria to judge the appropriateness of the HSUs selected for use in costeffectiveness models and is suitable for use in different international settings.
Significant improvements in function and health-related quality of life occurred in patients with RA during treatment with leflunomide or methotrexate. These findings were clinically meaningful and correlated with the ACR response status.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.