Random models for margins of a 2 x 2 contingency table and application to pharmacovigilance.

abstract：:In epidemiological studies we often want to learn about the direct effect of an exposure on an outcome, i.e. the effect that is not relayed by a specific intermediate variable. In the literature, there are two common definitions of direct effects; controlled and natural. When the intermediate variable and the outcome ...

journal_title：Statistics in medicine

authors： Sjölander A

更新日期：2009-02-15 00:00:00

abstract：:Disease incidence or disease mortality rates for small areas are often displayed on maps. Maps of raw rates, disease counts divided by the total population at risk, have been criticized as unreliable due to non-constant variance associated with heterogeneity in base population size. This has led to the use of model-ba...

journal_title：Statistics in medicine

authors： Stern HS,Cressie N

更新日期：2000-09-15 00:00:00

abstract：:To benefit Alzheimer's disease research, a central data co-ordinating centre (CDCC) is planned that will systematically collect data from 27 Alzheimer's disease centres (ADCs) located nationwide. This CDCC will combine, analyse and disseminate epidemiologic, demographic, clinical and neuropathological data to research...

journal_title：Statistics in medicine

authors： Cronin-Stubbs D,DeKosky ST,Morris JC,Evans DA

更新日期：2000-06-15 00:00:00

abstract：:Competing compartment models of different complexities have been used for the quantitative analysis of dynamic contrast-enhanced magnetic resonance imaging data. We present a spatial elastic net approach that allows to estimate the number of compartments for each voxel such that the model complexity is not fixed a pri...

journal_title：Statistics in medicine

authors： Sommer JC,Gertheiss J,Schmid VJ

更新日期：2014-03-15 00:00:00

abstract：:A statistical definition of surrogate endpoints as well as validation criteria was first presented by Prentice. Freedman et al. supplemented these criteria with the so-called proportion explained. Buyse and Molenberghs pointed to inadequacies of these criteria and suggested a new definition of surrogacy based on (i) t...

journal_title：Statistics in medicine

authors： Molenberghs G,Geys H,Buyse M

更新日期：2001-10-30 00:00:00

abstract：:We present some practical extensions and applications of a strategy proposed by Thall, Simon and Estey for designing and monitoring single-arm clinical trials with multiple outcomes. We show by application how the strategy may be applied to construct designs for phase IIA activity trials and phase II equivalence trial...

journal_title：Statistics in medicine

authors： Thall PF,Sung HG

更新日期：1998-07-30 00:00:00

abstract：:Prevalent sampling is frequently a convenient and economical sampling technique for the collection of time-to-event data and thus is commonly used in studies of the natural history of a disease. However, it is biased by design because it tends to recruit individuals with longer survival times. This paper considers est...

journal_title：Statistics in medicine

authors： Li S

更新日期：2017-04-15 00:00:00

abstract：:The use of data from multiple studies or centers for the validation of a clinical test or a multivariable prediction model allows researchers to investigate the test's/model's performance in multiple settings and populations. Recently, meta-analytic techniques have been proposed to summarize discrimination and calibra...

journal_title：Statistics in medicine

authors： Wynants L,Riley RD,Timmerman D,Van Calster B

更新日期：2018-05-30 00:00:00

abstract：:Recently, Wu and Follmann developed summary measures to adjust for informative drop-out in longitudinal studies where drop-out depends on the underlying true value of the response. In this paper we evaluate these procedures in the common situation where drop-out depends on the observed responses. We also discuss vario...

journal_title：Statistics in medicine

authors： Wu MC,Albert PS,Wu BU

更新日期：2001-01-15 00:00:00

abstract：:Recently, there has been much work on early phase cancer designs that incorporate both toxicity and efficacy data, called phase I-II designs because they combine elements of both phases. However, they do not explicitly address the phase II hypothesis test of H0 : p ≤ p0 , where p is the probability of efficacy at the ...

journal_title：Statistics in medicine

authors： Bartroff J,Lai TL,Narasimhan B

更新日期：2014-07-20 00:00:00

abstract：:We extend the shared frailty model of recurrent events and a dependent terminal event to allow for a nonparametric covariate function. We include a Gaussian random effect (frailty) in the intensity functions of both the recurrent and terminal events to capture correlation between the two processes. We employ the penal...

journal_title：Statistics in medicine

authors： Zhangsheng Y,Liu L

更新日期：2011-09-30 00:00:00

abstract：:In many community-based studies on the incidence of dementia, a target population is screened and a subsample is clinically evaluated at baseline and follow-up. Incidence rates are affected by missed cases at both exams and this complicates the estimation of these rates. Recent work proposes a regression-based techniq...

journal_title：Statistics in medicine

authors： Izmirlian G,Brock D,White L

更新日期：2000-06-15 00:00:00

abstract：:For the analysis of time to event data in contraceptive studies when individuals are subject to competing causes for discontinuation, some authors have recently advocated the use of the cumulative incidence rate as a more appropriate measure to summarize data than the complement of the Kaplan-Meier estimate of discont...

journal_title：Statistics in medicine

authors： Farley TM,Ali MM,Slaymaker E

更新日期：2001-12-15 00:00:00

abstract：:Analyses to compare non-randomized groups are more and more common in both post hoc analyses of randomized clinical trials data and in analyses of long-term observational data. In such cases, it is quite likely that there are unknown or uncollected sources of heterogeneity in event rates. Research has shown that an un...

journal_title：Statistics in medicine

authors： Eberly LE

更新日期：2004-07-15 00:00:00

abstract：:The availability of large data sets together with the growth in power and storage capabilities of computers have made the analysis of the spatial distribution of disease rates an increasingly important tool in public health research. Use of existing geographic divisions or groupings tends to result either in unstable ...

journal_title：Statistics in medicine

authors： Morris RD,Munasinghe RL

更新日期：1993-10-01 00:00:00

abstract：:The stability-controlled quasi-experiment (SCQE) is an approach to study the effects of nonrandomized, newly adopted treatments. While covariate adjustment techniques rely on a "no unobserved confounding" assumption, SCQE imposes an assumption on the change in the average nontreatment outcome between successive cohort...

journal_title：Statistics in medicine

authors： Hazlett C,Maokola W,Wulf DA

更新日期：2020-12-10 00:00:00

abstract：:For the meta-analysis of controlled clinical trials with binary outcome a test statistic for testing an overall treatment effect is proposed, which is based on a refined estimator for the variance of the treatment effect estimator usually used in the random-effects model of meta-analysis. In simulation studies it is s...

journal_title：Statistics in medicine

authors： Hartung J,Knapp G

更新日期：2001-12-30 00:00:00

abstract：:Hierarchical regression analysis holds much promise for epidemiologic analysis, but has as yet seen limited application because of lack of easily used software and the relatively lengthy run times of preferred fitting methods (such as true maximum likelihood and Bayesian approaches). This paper compares three relative...

journal_title：Statistics in medicine

authors： Greenland S

更新日期：1997-03-15 00:00:00

abstract：:Correct specification of the inverse probability weighting (IPW) model is necessary for consistent inference from a marginal structural Cox model (MSCM). In practical applications, researchers are typically unaware of the true specification of the weight model. Nonetheless, IPWs are commonly estimated using parametric...

journal_title：Statistics in medicine

authors： Karim ME,Platt RW,BeAMS study group.

更新日期：2017-06-15 00:00:00

abstract：:Clinical prediction models (CPMs) can inform decision making about treatment initiation, which requires predicted risks assuming no treatment is given. However, this is challenging since CPMs are usually derived using data sets where patients received treatment, often initiated postbaseline as "treatment drop-ins." Th...

journal_title：Statistics in medicine

authors： Sperrin M,Martin GP,Pate A,Van Staa T,Peek N,Buchan I

更新日期：2018-12-10 00:00:00

abstract：:Adaptive designs have been proposed for clinical trials in which the nuisance parameters or alternative of interest are unknown or likely to be misspecified before the trial. Although most previous works on adaptive designs and mid-course sample size re-estimation have focused on two-stage or group-sequential designs ...

journal_title：Statistics in medicine

authors： Bartroff J,Lai TL

更新日期：2008-05-10 00:00:00

abstract：:The cost and efficiency of training clinical centre staff and of duplicate data entry in clinical trials is reviewed. Training is an essential component of quality assurance programmes and it is usually carried out at regular intervals in long-term clinical trials. Initial training of staff and regular retraining is i...

journal_title：Statistics in medicine

authors： Neaton JD,Duchene AG,Svendsen KH,Wentworth D

更新日期：1990-01-01 00:00:00

abstract：:Binary matched-pairs data occur commonly in longitudinal studies, such as in cross-over experiments. Many analyses for comparing the matched probabilities of a particular outcome do not utilize pairs having the same outcome for each observation. An example is McNemar's test. Some methodologists find this to be counter...

journal_title：Statistics in medicine

authors： Agresti A,Min Y

更新日期：2004-01-15 00:00:00

abstract：:The analysis of multivariate time-to-event (TTE) data can become complicated due to the presence of clustering, leading to dependence between multiple event times. For a long time, (conditional) frailty models and (marginal) copula models have been used to analyze clustered TTE data. In this article, we propose a gene...

journal_title：Statistics in medicine

authors： Tran TMP,Abrams S,Braekers R

更新日期：2020-05-30 00:00:00

abstract：:Causal inference for non-censored response variables, such as binary or quantitative outcomes, is often based on either (1) direct standardization ('G-formula') or (2) inverse probability of treatment assignment weights ('propensity score'). To do causal inference in survival analysis, one needs to address right-censo...

journal_title：Statistics in medicine

authors： Andersen PK,Syriopoulou E,Parner ET

更新日期：2017-07-30 00:00:00

abstract：:A new testing approach is described for improving statistical tests of independence in sets of tables stratified on one or more relevant factors in case of categorical (nominal or ordinal) variables. Common tests of independence that exploit the ordinality of one of the variables use a restricted-alternative approach....

journal_title：Statistics in medicine

authors： Iannario M,Lang JB

更新日期：2016-11-10 00:00:00

abstract：:The concept of broad sense agreement (BSA) has recently been proposed for studying the relationship between a continuous measurement and an ordinal measurement. They developed a nonparametric procedure for estimating the BSA index, which is only applicable to completely observed data. In this work, we consider the pro...

journal_title：Statistics in medicine

authors： Dai T,Guo Y,Peng L,Manatunga A

更新日期：2020-06-30 00:00:00

abstract：:A decision-theoretic framework is proposed for designing sequential dose-finding trials with multiple outcomes. The optimal strategy is solvable theoretically via backward induction. However, for dose-finding studies involving k doses, the computational complexity is the same as the bandit problem with k-dependent arm...

journal_title：Statistics in medicine

authors： Fan SK,Wang YG

更新日期：2006-05-30 00:00:00

abstract：:Identification of treatment selection biomarkers has become very important in cancer drug development. Adaptive enrichment designs have been developed for situations where a unique treatment selection biomarker is not apparent based on the mechanism of action of the drug. With such designs, the eligibility rules may b...

journal_title：Statistics in medicine

authors： Simon R,Simon N

更新日期：2017-11-20 00:00:00

abstract：:This paper reviews several emerging and recurrent issues relating to the drug development process. These emerging issues include changes to the FDA regulatory environment, internationalization of drug development, advances in computer technology and visualization tools, and efforts to incorporate meta-analysis methodo...

journal_title：Statistics in medicine

authors： Anello C

更新日期：1999-09-15 00:00:00