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  • 1
    In: Pharmaceutical Statistics, Wiley, Vol. 16, No. 2 ( 2017-03), p. 122-132
    Abstract: Missing outcome data constitute a serious threat to the validity and precision of inferences from randomized controlled trials. In this paper, we propose the use of a multistate Markov model for the analysis of incomplete individual patient data for a dichotomous outcome reported over a period of time. The model accounts for patients dropping out of the study and also for patients relapsing. The time of each observation is accounted for, and the model allows the estimation of time‐dependent relative treatment effects. We apply our methods to data from a study comparing the effectiveness of 2 pharmacological treatments for schizophrenia. The model jointly estimates the relative efficacy and the dropout rate and also allows for a wide range of clinically interesting inferences to be made. Assumptions about the missingness mechanism and the unobserved outcomes of patients dropping out can be incorporated into the analysis. The presented method constitutes a viable candidate for analyzing longitudinal, incomplete binary data.
    Type of Medium: Online Resource
    ISSN: 1539-1604 , 1539-1612
    URL: Issue
    Language: English
    Publisher: Wiley
    Publication Date: 2017
    detail.hit.zdb_id: 2083706-9
    detail.hit.zdb_id: 2163550-X
    SSG: 15,3
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  • 2
    In: Statistics in Medicine, Wiley, Vol. 36, No. 8 ( 2017-04-15), p. 1210-1226
    Abstract: Non‐randomized studies aim to reveal whether or not interventions are effective in real‐life clinical practice, and there is a growing interest in including such evidence in the decision‐making process. We evaluate existing methodologies and present new approaches to using non‐randomized evidence in a network meta‐analysis of randomized controlled trials (RCTs) when the aim is to assess relative treatment effects. We first discuss how to assess compatibility between the two types of evidence. We then present and compare an array of alternative methods that allow the inclusion of non‐randomized studies in a network meta‐analysis of RCTs: the naïve data synthesis, the design‐adjusted synthesis, the use of non‐randomized evidence as prior information and the use of three‐level hierarchical models. We apply some of the methods in two previously published clinical examples comparing percutaneous interventions for the treatment of coronary in‐stent restenosis and antipsychotics in patients with schizophrenia. We discuss in depth the advantages and limitations of each method, and we conclude that the inclusion of real‐world evidence from non‐randomized studies has the potential to corroborate findings from RCTs, increase precision and enhance the decision‐making process. Copyright © 2017 John Wiley & Sons, Ltd.
    Type of Medium: Online Resource
    ISSN: 0277-6715 , 1097-0258
    URL: Issue
    RVK:
    Language: English
    Publisher: Wiley
    Publication Date: 2017
    detail.hit.zdb_id: 1491221-1
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