2021
DOI: 10.1007/s11336-021-09777-y
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Longitudinal Analysis of Patient-Reported Outcomes in Clinical Trials: Applications of Multilevel and Multidimensional Item Response Theory

Abstract: With decades of advance research and recent developments in the drug and medical device regulatory approval process, patient-reported outcomes (PROs) are becoming increasingly important in clinical trials. While clinical trial analyses typically treat scores from PROs as observed variables, the potential to use latent variable models when analyzing patient responses in clinical trial data presents novel opportunities for both psychometrics and regulatory science. An accessible overview of analyses commonly use… Show more

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Cited by 24 publications
(21 citation statements)
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References 36 publications
(62 reference statements)
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“…For the P method, expected a posteriori (EAP) estimates for θ were used for the computation. Item calibration and EAP scoring were conducted using flexMIRT (Cai, 2017).…”
Section: Estimation Of Classification Consistency and Accuracymentioning
confidence: 99%
“…For the P method, expected a posteriori (EAP) estimates for θ were used for the computation. Item calibration and EAP scoring were conducted using flexMIRT (Cai, 2017).…”
Section: Estimation Of Classification Consistency and Accuracymentioning
confidence: 99%
“…The response data were then calibrated to obtain item parameter estimates for the pool. Calibration was implemented using the MMLE-EM procedure with flexMIRT (Cai, 2017). The maximum number of cycles was 500; the convergence criterion was 1.00e-004; and there were 54 quadrature points between -4.0 and 4.0.…”
Section: Item Poolmentioning
confidence: 99%
“…Implementation of SC and FC resulted in differences and commonalities. Both SC and FC were implemented using the MMLE-EM procedure via flexMIRT (Cai, 2017). MMLE-EM control values used for calibrating the item pool were common to all calibration approaches.…”
Section: Simulationmentioning
confidence: 99%
“…Within each of the latent variable vectors ϑj and η ij , latent variables may be correlated, or a bifactor or testlet structure could be specified at each level. As with the single-level IRT models, MLIRT models can be expanded to include both a measurement model and a conditioning model (Cai & Houts, 2021).…”
Section: Approaches To Producing Scores In a Multisite Cluster Rctmentioning
confidence: 99%