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Perspectives on Patient-Reported Outcomes

Content Validity and Qualitative Research in a Changing Clinical Trial Environment

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Abstract

Patient-reported outcomes (PROs) represent the voice of the patient in drug and device evaluation. As such, the outcomes selected for evaluation must be relevant to the patient, and the instruments used to capture them must have sound measurement properties. This paper discusses the role of qualitative research methods in assuring PRO clarity and content validity in a clinical trial environment characterized by evolving regulatory policy, new advances in technology and increasingly diverse samples within global clinical trials. Three important PRO measurement issues influenced by these changes are addressed: (i) endpoint selection and instrument development; (ii) the adaptation of instruments for electronic administration; and (iii) conceptual equivalence of measures across diverse samples. These are viewed as interrelated issues of content validity that can be addressed through the appropriate and rigorous application of qualitative research methods. Focus groups, cognitive debriefing, user acceptance testing and translation methodologies are discussed as ways to address and document the content validity of PRO instruments and ensure the empirical data representing the voice of the patient is sound.

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Acknowledgements

The authors are employed by the United BioSource Corporation (UBC), which provides consulting and other research services to pharmaceutical, device, government and non-government organizations. In this salaried position, the authors work with a variety of companies and organizations. They receive no payment or honoraria directly from these organizations for services rendered.

No sources of funding were used to assist in the preparation of this article.

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  1. United BioSource Corporation, 7101 Wisconsin Avenue, Suite 600, Bethesda, Maryland, 20814, USA

    Nancy Kline Leidy & Margaret Vernon

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  1. Nancy Kline Leidy
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  2. Margaret Vernon
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Correspondence to Nancy Kline Leidy.

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Leidy, N.K., Vernon, M. Perspectives on Patient-Reported Outcomes. Pharmacoeconomics 26, 363–370 (2008). https://doi.org/10.2165/00019053-200826050-00002

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