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dc.contributor.authorLee, Jooyoung
dc.contributor.authorCook, Richard J. 15:01:30 (GMT) 15:01:30 (GMT)
dc.descriptionThis is the peer reviewed version of the following article: Jooyoung Lee and Richard J. Cook. On estimands arising from misspecified semiparametric rate-based analysis of recurrent episodic conditions. Statistics in Medicine (2019), 38 (25): 4977–4998 which has been published in final form at article may be used for non-commercial purposes in accordance with Wiley Terms and Conditions for Use of Self-Archived Versions.en
dc.description.abstractMarginal rate-based analyses are widely used for the analysis of recurrent events in clinical trials. In many areas of application, the events are not instantaneous but rather signal the onset of a symptomatic episode representing a recurrent infection, respiratory exacerbation, or bout of acute depression. In rate-based analyses, it is unclear how to best handle the time during which individuals are experiencing symptoms and hence are not at risk. We derive the limiting value of theNelson-Aalen estimator and estimators of the regression coefficients under a semiparametric rate-based model in terms of an underlying two-state process. We investigate the impact of the distribution of the episode durations, heterogeneity, and dependence on the asymptotic and finite sample properties of standard estimators. We also consider the impact of these features on power in trials designed to test intervention effects on rate functions. An application to a trial of individuals with herpes simplex virus is given for illustration.en
dc.description.sponsorshipThis research was supported by grants from the Natural Sciences and Engineering Research Council of Canada (RGPIN 155849) and the Canadian Institutes for Health Research (FRN 13887). Richard Cook is a Canada Research Chair in Statistical Methods for Health Research.en
dc.relation.ispartofseriesStatistics in Medicine;38(25)
dc.subjectintensity functionen
dc.subjectrate functionen
dc.subjectrecurrent episodesen
dc.titleOn estimands arising from misspecified semiparametric rate-based analysis of recurrent episodic conditionsen
dcterms.bibliographicCitationLee, J, Cook, RJ. On estimands arising from misspecified semiparametric rate‐based analysis of recurrent episodic conditions. Statistics in Medicine. 2019; 38: 4977– 4998.
uws.contributor.affiliation1Faculty of Mathematicsen
uws.contributor.affiliation2Statistics and Actuarial Scienceen

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