What Industry Statisticians Should Know about Patient-Reported Outcomes to Support Label Claims
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*Joe Cappelleri, Pfizer 

Keywords: patient-reported outcomes, label claim, interpretation, responder analysis, validity, reliability, psychometrics

In December 2009 the Food and Drug Administration released its final guidance on patient-reported outcomes for use in medical product development to support labeling claims. The guidance is expected to engage industry statisticians on a regular basis and it is therefore important that industry statisticians know its contents and how to address them. To that end, this presentation highlights several statistical aspects of patient-reported outcomes to support label claims: general statistical considerations, study design, multiple endpoints and comparisons, composite endpoints, missing data, and measurement properties including validity, reliability, and interpretation.