What do we mean by validating a prognostic model?

abstract：:Risk prediction models play an important role in prevention and treatment of several diseases. Models that are in clinical use are often refined and improved. In many instances, the most efficient way to improve a successful model is to identify subgroups for which there is a specific biological rationale for improvem...

journal_title：Statistics in medicine

authors： Biswas S,Arun B,Parmigiani G

更新日期：2014-05-20 00:00:00

abstract：:We analyze data obtained from a study designed to evaluate training effects on the performance of certain motor activities of Parkinson's disease patients. Maximum likelihood methods were used to fit beta-binomial/Poisson regression models tailored to evaluate the effects of training on the numbers of attempted and su...

journal_title：Statistics in medicine

authors： Lora MI,Singer JM

更新日期：2008-07-30 00:00:00

abstract：:In the pharmaceutical industry, an assay method is considered validated if the accuracy and precision for an assay meet some acceptable limits. This paper discusses the assessment of assay precision in terms of the estimation of total variability of an assay from a one-way random effects model which is often considere...

journal_title：Statistics in medicine

authors： Chow SC,Tse SK

更新日期：1991-10-01 00:00:00

abstract：:Service on the Data Monitoring Committee of the CPEP (Calcium for Pre-eclampsia Prevention) has led us to four conclusions about clinical trials which we should like to present to this gathering of biostatisticians for their reactions: (i) meta-analyses of the pertinent published trials of the same therapy should alwa...

journal_title：Statistics in medicine

authors： Chalmers TC,Lau J

更新日期：1996-06-30 00:00:00

abstract：:Patient compliance (adherence) with prescribed medication is often erratic, while clinical outcomes are causally linked to actual, rather than nominal medication dosage. We propose here a hierarchical Markov model for patient compliance. At the first stage, conditional upon individual random effects and a set of indiv...

journal_title：Statistics in medicine

authors： Girard P,Blaschke TF,Kastrissios H,Sheiner LB

更新日期：1998-10-30 00:00:00

abstract：:It is valuable in many studies to assess both intrarater and interrater agreement. Most measures of intrarater agreement do not adjust for unequal estimates of prevalence between the separate rating occasions for a given rater and measures of interrater agreement typically ignore data from the second set of assessment...

journal_title：Statistics in medicine

authors： Lester Kirchner H,Lemke JH

更新日期：2002-06-30 00:00:00

abstract：:Statistical tests have been proposed for determining whether incident cases of adverse health effects are 'clustered' together. Several procedures, termed 'focused', specifically analyse disease surveillance data around pre-specified putative sources of environmental hazard. Little has been done to compare the perform...

journal_title：Statistics in medicine

authors： Waller LA,Lawson AB

更新日期：1995-11-15 00:00:00

abstract：:The objective of this report is to provide a basis to inform decisions about priorities for developing statistical research initiatives in the field of public health surveillance for emerging threats. Rapid information system advances have created a vast opportunity of secondary data sources for information to enhance...

journal_title：Statistics in medicine

authors： Rolka H,Burkom H,Cooper GF,Kulldorff M,Madigan D,Wong WK

更新日期：2007-04-15 00:00:00

abstract：:In repeated measures settings, modeling the correlation pattern of the data can be immensely important for proper analyses. Accurate inference requires proper choice of the correlation model. Optimal efficiency of the estimation procedure demands a parsimonious parameterization of the correlation structure, with suffi...

journal_title：Statistics in medicine

authors： Simpson SL,Edwards LJ,Muller KE,Sen PK,Styner MA

更新日期：2010-07-30 00:00:00

abstract：:A preference trial is a special form of cross-over trial where clinical conditions determine when patients change treatment, in a prescribed order. This can be modelled using a geometric distribution. The model can be simply fitted using standard logistic regression methodology. The procedure is applied to a trial stu...

journal_title：Statistics in medicine

authors： Lindsey JK,Jones B

更新日期：1996-02-28 00:00:00

abstract：:Spatial scan statistics are widely used for count data to detect geographical disease clusters of high or low incidence, mortality or prevalence and to evaluate their statistical significance. Some data are ordinal or continuous in nature, however, so that it is necessary to dichotomize the data to use a traditional s...

journal_title：Statistics in medicine

authors： Jung I,Kulldorff M,Klassen AC

更新日期：2007-03-30 00:00:00

abstract：:In many studies, interest lies in determining whether members of the study population will undergo a particular event of interest. Such scenarios are often termed 'mover-stayer' scenarios, and interest lies in modelling two sub-populations of 'movers' (those who have a propensity to undergo the event of interest) and ...

journal_title：Statistics in medicine

authors： O'Keeffe AG,Tom BD,Farewell VT

更新日期：2013-02-20 00:00:00

abstract：:We consider methods for selecting the joint specification of the mean and variance functions in statistical models for rates or counts. Based on analyses of diagnosis-specific hospital discharge rates in Michigan, we show that a Poisson model with an extra variance component for the systematic variation is superior to...

journal_title：Statistics in medicine

authors： Wolfe RA,Petroni GR,McLaughlin CG,McMahon LF Jr

更新日期：1991-09-01 00:00:00

abstract：:In this article, we investigate procedures for comparing two independent Poisson variates that are observed over unequal sampling frames (i.e. time intervals, populations, areas or any combination thereof). We consider two statistics (with and without the logarithmic transformation) for testing the equality of two Poi...

journal_title：Statistics in medicine

authors： Ng HK,Tang ML

更新日期：2005-03-30 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 need for resource-intensive laboratory assays to assess exposures in many epidemiologic studies provides ample motivation to consider study designs that incorporate pooled samples. In this paper, we consider the case in which specimens are combined for the purpose of determining the presence or absence of a pool-w...

journal_title：Statistics in medicine

authors： Lyles RH,Tang L,Lin J,Zhang Z,Mukherjee B

更新日期：2012-09-28 00:00:00

abstract：:Determination of the equation that relates an ordered dependent variable to ordered independent variables is sought. One solution, non-parametric discriminant analysis (NPD), involves obtaining the best monotonic step function by means of a computer search procedure. Although one can use alternative selection criteria...

journal_title：Statistics in medicine

authors： Johnston B,Seshia SS

更新日期：1992-06-15 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：: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

abstract：:In record linkage studies, unique identifiers are often not available, and therefore, the linkage procedure depends on combinations of partially identifying variables with low discriminating power. As a consequence, wrongly linked covariate and outcome pairs will be created and bias further analysis of the linked data...

journal_title：Statistics in medicine

authors： Hof MH,Zwinderman AH

更新日期：2012-12-30 00:00:00

abstract：:Joint models initially dedicated to a single longitudinal marker and a single time-to-event need to be extended to account for the rich longitudinal data of cohort studies. Multiple causes of clinical progression are indeed usually observed, and multiple longitudinal markers are collected when the true latent trait of...

journal_title：Statistics in medicine

authors： Proust-Lima C,Dartigues JF,Jacqmin-Gadda H

更新日期：2016-02-10 00:00:00

abstract：:We discuss some of the statistical approaches to the design and analysis of phase I clinical trials in cancer. An attempt is made to identify the issues, particular to this type of trial, that should be addressed by an appropriate methodology. A brief review of schemes currently in use is provided together with our vi...

journal_title：Statistics in medicine

authors： O'Quigley J,Chevret S

更新日期：1991-11-01 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：:We consider recurrent events of the same type that occur during alternating restraint and non-restraint time periods. This research is motivated by a study on juvenile recidivism, where the probationers were followed for re-offenses during alternating placement periods and free-time periods. During the placement perio...

journal_title：Statistics in medicine

authors： Li X,Chen Y,Li R

更新日期：2017-02-20 00:00:00

abstract：:When designing a study to develop a new prediction model with binary or time-to-event outcomes, researchers should ensure their sample size is adequate in terms of the number of participants (n) and outcome events (E) relative to the number of predictor parameters (p) considered for inclusion. We propose that the mini...

journal_title：Statistics in medicine

authors： Riley RD,Snell KI,Ensor J,Burke DL,Harrell FE Jr,Moons KG,Collins GS

更新日期：2019-03-30 00:00:00

abstract：:Assessing the calibration of methods for estimating the probability of the occurrence of a binary outcome is an important aspect of validating the performance of risk-prediction algorithms. Calibration commonly refers to the agreement between predicted and observed probabilities of the outcome. Graphical methods are a...

journal_title：Statistics in medicine

authors： Austin PC,Steyerberg EW

更新日期：2019-09-20 00:00:00

abstract：:The asymptotic Pearson's chi-squared test and Fisher's exact test have long been the most used for testing association in 2x2 tables. Unconditional tests preserve the significance level and generally are more powerful than Fisher's exact test for moderate to small samples, but previously were disadvantaged by being co...

journal_title：Statistics in medicine

authors： Lydersen S,Fagerland MW,Laake P

更新日期：2009-03-30 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：:In behavioral, biomedical, and social-psychological sciences, it is common to encounter latent variables and heterogeneous data. Mixture structural equation models (SEMs) are very useful methods to analyze these kinds of data. Moreover, the presence of missing data, including both missing responses and missing covaria...

journal_title：Statistics in medicine

authors： Cai JH,Song XY,Hser YI

更新日期：2010-08-15 00:00:00

abstract：:The analysis of patient-reported outcomes or other psychological traits can be realized using the Rasch measurement model. When the objective of a study is to compare groups of individuals, it is important, before the study, to define a sample size such that the group comparison test will attain a given power. The Ras...

journal_title：Statistics in medicine

authors： Hardouin JB,Blanchin M,Feddag ML,Le Néel T,Perrot B,Sébille V

更新日期：2015-07-20 00:00:00