Application of the parallel line assay to assessment of biosimilar products based on binary endpoints.

abstract：:Patient reported outcome and observer evaluative studies in clinical trials and post-hoc analyses often use instruments that measure responses on ordinal-rating or Likert scales. We propose a flexible distributional approach by modeling the change scores from the baseline to the end of the study using independent beta...

journal_title：Statistics in medicine

authors： Zou KH,Carlsson MO,Quinn SA

更新日期：2010-10-30 00:00:00

abstract：:Recurrent event data are commonly encountered in health-related longitudinal studies. In this paper time-to-events models for recurrent event data are studied with non-informative and informative censorings. In statistical literature, the risk set methods have been confirmed to serve as an appropriate and efficient ap...

journal_title：Statistics in medicine

authors： Wang MC,Chiang CT

更新日期：2002-02-15 00:00:00

abstract：:Risk prediction procedures can be quite useful for the patient's treatment selection, prevention strategy, or disease management in evidence-based medicine. Often, potentially important new predictors are available in addition to the conventional markers. The question is how to quantify the improvement from the new ma...

journal_title：Statistics in medicine

authors： Uno H,Tian L,Cai T,Kohane IS,Wei LJ

更新日期：2013-06-30 00:00:00

abstract：:We describe rank-based approaches to assess principal stratification treatment effects in studies where the outcome of interest is only well-defined in a subgroup selected after randomization. Our methods are sensitivity analyses, in that estimands are identified by fixing a parameter and then we investigate the sensi...

journal_title：Statistics in medicine

authors： Lu X,Mehrotra DV,Shepherd BE

更新日期：2013-11-20 00:00:00

abstract：:In this commentary, we revisit Sir Austin Bradford Hill's seminal Alfred Watson Memorial Lecture in 1962 through the eyes of two practicing biostatisticians of the current era. We summarize some eternal takeaway messages from Hill's lecture regarding observations and experiments translated through the modern lexicon o...

journal_title：Statistics in medicine

authors： Dempsey W,Mukherjee B

更新日期：2021-01-15 00:00:00

abstract：:Clinical trials utilizing predictive biomarkers have become a research focus in personalized medicine. We investigate the effects of biomarker misclassification on the design and analysis of stratified biomarker clinical trials. For a variety of inference problems including marker-treatment interaction in particular, ...

journal_title：Statistics in medicine

authors： Liu C,Liu A,Hu J,Yuan V,Halabi S

更新日期：2014-08-15 00:00:00

abstract：:The purpose of this paper is to show that the sensitivity and specificity estimates obtained by 'discrepant analysis' are biased. Discrepant analysis is a widely used technique that attempts to provide estimates of sensitivity and specificity in the presence of an imperfect gold standard. Many researchers have applied...

journal_title：Statistics in medicine

authors： Hadgu A

更新日期：1997-06-30 00:00:00

abstract：:The World Health Organization and collaborating institutions in four developing countries have conducted a multi-centre randomized controlled trial, in which clinics were allocated at random to two antenatal care (ANC) models. These were the standard 'Western' ANC model and a 'new' ANC model consisting of tests, clini...

journal_title：Statistics in medicine

authors： Piaggio G,Carroli G,Villar J,Pinol A,Bakketeig L,Lumbiganon P,Bergsjø P,Al-Mazrou Y,Ba'aqeel H,Belizán JM,Farnot U,Berendes H,WHO Antenatal Care Trial Research Group.

更新日期：2001-02-15 00:00:00

abstract：:Case-control studies are usually defined to investigate risk factors for a single disease of interest. However, subsequent to data collection, investigators may wish to examine as an 'outcome' a variable that was an exposure in the original study. A naive analysis that disregards the sampling strategy that gave rise t...

journal_title：Statistics in medicine

authors： Reilly M,Torrång A,Klint A

更新日期：2005-12-30 00:00:00

abstract：:The statistical analysis of panel count data has recently attracted a great deal of attention, and a number of approaches have been developed. However, most of these approaches are for situations where the observation and follow-up processes are independent of the underlying recurrent event process unconditional or co...

journal_title：Statistics in medicine

authors： Li N,Zhao H,Sun J

更新日期：2013-07-30 00:00:00

abstract：:An observed confidence distribution is proposed as a measure of strength of evidence for practically equivalent efficacies of two treatments. The concept is independent of prior opinions about relevant sizes of a difference in efficacy. It also avoids retrospective power calculations for trials with missed recruitment...

journal_title：Statistics in medicine

authors： Mau J

更新日期：1988-12-01 00:00:00

abstract：:Relative survival provides a measure of the proportion of patients dying from the disease under study without requiring the knowledge of the cause of death. We propose an overall strategy based on regression models to estimate the relative survival and model the effects of potential prognostic factors. The baseline ha...

journal_title：Statistics in medicine

authors： Remontet L,Bossard N,Belot A,Estève J,French network of cancer registries FRANCIM.

更新日期：2007-05-10 00:00:00

abstract：:A personalized treatment strategy formalizes evidence-based treatment selection by mapping patient information to a recommended treatment. Personalized treatment strategies can produce better patient outcomes while reducing cost and treatment burden. Thus, among clinical and intervention scientists, there is a growing...

journal_title：Statistics in medicine

authors： Laber EB,Zhao YQ,Regh T,Davidian M,Tsiatis A,Stanford JB,Zeng D,Song R,Kosorok MR

更新日期：2016-04-15 00:00:00

abstract：:Health questionnaires are often built up from sets of questions that are totaled to obtain a sum score. An important consideration in designing questionnaires is to minimize respondent burden. An increasingly popular method for efficient measurement is computerized adaptive testing; unfortunately, many health question...

journal_title：Statistics in medicine

authors： Smits N,Finkelman MD

更新日期：2014-02-10 00:00:00

abstract：:In US states with small subpopulations, the observed mortality rates are often zero, particularly among young ages. Because in life tables, death rates are reported mostly on a log scale, zero mortality rates are problematic. To overcome the observed zero death rates problem, appropriate probability models are used. U...

journal_title：Statistics in medicine

authors： Voulgaraki A,Wei R,Kedem B

更新日期：2015-05-20 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：:A model is developed to estimate the effect of covariate imbalance on the size of a test of treatment efficacy in randomized clinical trials comparing two treatments when dispersion parameters are known. It is concluded that tests of homogeneity on the covariates should not be performed, that covariate imbalance is ju...

journal_title：Statistics in medicine

authors： Senn SJ

更新日期：1989-04-01 00:00:00

abstract：:A common but not necessarily logical requirement in drug development is that a 'limit of quantitation' be set for chemical assays and that observations that fall below the limit should not be treated as real data but should be labelled as below the limit and set aside for special treatment. We examine five of seven ap...

journal_title：Statistics in medicine

authors： Senn S,Holford N,Hockey H

更新日期：2012-12-30 00:00:00

abstract：:Data monitoring committees (DMCs) have become an increasingly common component of randomized clinical trials in recent years. As experience has accumulated, and more individuals and organizations have become involved in such activities, a variety of approaches to the operation of such committees has inevitably arisen....

journal_title：Statistics in medicine

authors： Ellenberg SS

更新日期：2001-09-15 00:00:00

abstract：:The back-calculation method has been used to estimate the number of HIV infections from AIDS incidence data in a particular population. We present an extension of back calculation that provides estimates of the numbers of HIV infectives in different stages of infection. We model the staging process with a time-depende...

journal_title：Statistics in medicine

authors： Longini IM Jr,Byers RH,Hessol NA,Tan WY

更新日期：1992-04-01 00:00:00

abstract：:Most phase I dose-finding methods in oncology aim to find the maximum-tolerated dose from a set of prespecified doses. However, in practice, because of a lack of understanding of the true dose-toxicity relationship, it is likely that none of these prespecified doses are equal or reasonably close to the true maximum-to...

journal_title：Statistics in medicine

authors： Chu Y,Pan H,Yuan Y

更新日期：2016-09-10 00:00:00

abstract：:Appropriate quantification of added usefulness offered by new markers included in risk prediction algorithms is a problem of active research and debate. Standard methods, including statistical significance and c statistic are useful but not sufficient. Net reclassification improvement (NRI) offers a simple intuitive w...

journal_title：Statistics in medicine

authors： Pencina MJ,D'Agostino RB Sr,Steyerberg EW

更新日期：2011-01-15 00:00:00

abstract：:Longitudinal designs are important in medical research and in many other disciplines. Complete longitudinal studies, in which each subject is evaluated at each measurement occasion, are often very expensive and motivate a search for more efficient designs. Recently developed statistical methods foster the use of inten...

journal_title：Statistics in medicine

authors： Helms RW

更新日期：1992-10-01 00:00:00

abstract：:The time-dependent change of HIV particle load, i.e. HIV dynamics, is likely to be controlled by a multitude of quantitative trait loci (QTL) that interact with each other as well as with various developmental and environmental factors in a coordinated manner. In this article, we have derived a new statistical model f...

journal_title：Statistics in medicine

authors： Wu S,Yang J,Wu R

更新日期：2006-11-30 00:00:00

abstract：:In clinical research, we are often interested in assessing how a biomarker changes with time, and whether it could be used as a surrogate marker when evaluating the efficacy of a new drug. However, when the longitudinal marker is correlated with survival, linear mixed models for longitudinal data may be inappropriate....

journal_title：Statistics in medicine

authors： Deslandes E,Chevret S

更新日期：2007-12-30 00:00:00

abstract：:Generalized linear models are often assumed to fit propensity scores, which are used to compute inverse probability weighted (IPW) estimators. To derive the asymptotic properties of IPW estimators, the propensity score is supposed to be bounded away from zero. This condition is known in the literature as strict positi...

journal_title：Statistics in medicine

authors： Molina J,Sued M,Valdora M

更新日期：2018-10-30 00:00:00

abstract：:We consider the design of both cohort and case-control studies in which an initial ('stage 1') sample of complete data on an error-free disease indicator (D), a correct ('gold standard') dichotomous exposure measurement (X) and an error-prone exposure measurement (Z) are available. We calculate the amount of additiona...

journal_title：Statistics in medicine

authors： Dahm PF,Gail MH,Rosenberg PS,Pee D

更新日期：1995-12-15 00:00:00

abstract：:Conditional power (CP) is the probability that the final study result will be statistically significant, given the data observed thus far and a specific assumption about the pattern of the data to be observed in the remainder of the study, such as assuming the original design effect, or the effect estimated from the c...

journal_title：Statistics in medicine

authors： Lachin JM

更新日期：2005-09-30 00:00:00

abstract：:Box-Cox transformation is investigated for regression models for left-censored data. Examples are provided using coronary calcification data from the Multi-Ethnic Study of Atherosclerosis and pharmacokinetic data of a nicotine nasal spray. ...

journal_title：Statistics in medicine

authors： Han C,Kronmal R

更新日期：2004-12-15 00:00:00

abstract：:Although the frequentist paradigm has been the predominant approach to clinical trial design since the 1940s, it has several notable limitations. Advancements in computational algorithms and computer hardware have greatly enhanced the alternative Bayesian paradigm. Compared with its frequentist counterpart, the Bayesi...

journal_title：Statistics in medicine

authors： Lee JJ,Chu CT

更新日期：2012-11-10 00:00:00