Abstract
A patient-reported outcome (PRO) is any report on the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else. In this chapter methods of PRO interpretation are discussed, under the assumption that a PRO instrument has already evidenced validity and reliability , in order to lend meaning and import to PRO scores. Specifically, we focus on three ways to advance interpretation of PRO scores: anchor-based methods , distribution-based methods , and mediation models . Anchor-based approaches use a criterion measure that is clinically interpretable and appreciably correlated with the targeted PRO measure of interest. Distribution-based approaches use the statistical distribution of the data to gauge the meaning of PRO scores. Mediation models involve a multivariate approach to collectively and simultaneously examine the interrelationship of the PRO measure of interest with other variables. Throughout the chapter, concepts are illuminated with illustrative and real-life examples.
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Cappelleri, J.C., Bushmakin, A.G. (2018). Advancing Interpretation of Patient-Reported Outcomes. In: Peace, K., Chen, DG., Menon, S. (eds) Biopharmaceutical Applied Statistics Symposium . ICSA Book Series in Statistics. Springer, Singapore. https://doi.org/10.1007/978-981-10-7826-2_5
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