More powerful randomization-based p-values in double-blind trials with non-compliance.

Abstract:

:Standard randomization-based tests of sharp null hypotheses in randomized clinical trials, that is, intent-to-treat analyses, are valid without extraneous assumptions, but generally can be appropriately powerful only with alternative hypotheses that involve treatment assignment having an effect on outcome. In the context of clinical trials with non-compliance, other alternative hypotheses can be more natural. In particular, when a trial is double-blind, it is often reasonable for the alternative hypothesis to exclude any effect of treatment assignment on outcome for a unit unless the assignment affected which treatment that unit actually received. Bayesian analysis under this alternative 'exclusion' hypothesis leads to new estimates of the effect of receipt of treatment, and to a new randomization-based procedure that has frequentist validity yet can be substantially more powerful than the standard intent-to-treat procedure. The key idea is to obtain a p-value using a posterior predictive check distribution, which includes a model for non-compliance behaviour, although only under the standard sharp null hypothesis of no effect of assignment (or receipt) of treatment on outcome. It is important to note that these new procedures are distinctly different from 'as treated' and 'per protocol' analyses, which are not only badly biased in general, but generally have very low power.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Rubin DB

doi

10.1002/(sici)1097-0258(19980215)17:3<371::aid-sim

subject

Has Abstract

pub_date

1998-02-15 00:00:00

pages

371-85; discussion 387-9

issue

3

eissn

0277-6715

issn

1097-0258

pii

10.1002/(SICI)1097-0258(19980215)17:3<371::AID-SIM

journal_volume

17

pub_type

杂志文章
  • Combining biomarker trajectories to improve diagnostic accuracy in prospective cohort studies with verification bias.

    abstract::In this paper, we develop methods to combine multiple biomarker trajectories into a composite diagnostic marker using functional data analysis (FDA) to achieve better diagnostic accuracy in monitoring disease recurrence in the setting of a prospective cohort study. In such studies, the disease status is usually verifi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8079

    authors: Li H,Gatsonis C

    更新日期:2019-05-20 00:00:00

  • An improved algorithm for outbreak detection in multiple surveillance systems.

    abstract::In England and Wales, a large-scale multiple statistical surveillance system for infectious disease outbreaks has been in operation for nearly two decades. This system uses a robust quasi-Poisson regression algorithm to identify abberrances in weekly counts of isolates reported to the Health Protection Agency. In this...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5595

    authors: Noufaily A,Enki DG,Farrington P,Garthwaite P,Andrews N,Charlett A

    更新日期:2013-03-30 00:00:00

  • A restricted mixture model for dietary pattern analysis in small samples.

    abstract::Multivariate finite mixture models have been applied to the identification of dietary patterns. These models are known to have many parameters, and consequently large samples are usually required. We present a special case of a multivariate mixture model that reduces the number of parameters to be estimated and seems ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.5336

    authors: Rita Gaio A,Costa JP,Santos AC,Ramos E,Lopes C

    更新日期:2012-08-30 00:00:00

  • Emerging and recurrent issues in drug development.

    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

    pub_type: 杂志文章,评审

    doi:10.1002/(sici)1097-0258(19990915/30)18:17/18<2301:

    authors: Anello C

    更新日期:1999-09-15 00:00:00

  • Estimating the stage-specific numbers of HIV infection using a Markov model and back-calculation.

    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

    pub_type: 杂志文章

    doi:10.1002/sim.4780110612

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

    更新日期:1992-04-01 00:00:00

  • Evaluating the cost-effectiveness of vaccination programmes: a dynamic perspective.

    abstract::Although there are many models which are used to calculate the health benefits (and thus the cost-effectiveness) of vaccination programmes, they can be divided into two groups: those which assume a constant force of infection, that is a constant per-susceptible rate of infection; and those which assume that the force ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19991215)18:23<3263::aid-s

    authors: Edmunds WJ,Medley GF,Nokes DJ

    更新日期:1999-12-15 00:00:00

  • Prospective epidemiological studies involving paired organs.

    abstract::Standard methods for analysing survival data or case-control data normally concern factors affecting a subject as a whole. However, in a study of a condition that might develop in one or both of a pair of bodily organs information on response and on covariates may be available for each separately. This information can...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780070509

    authors: Whitehead J,Dorse C

    更新日期:1988-05-01 00:00:00

  • Bayesian nonparametric areal wombling for small-scale maps with an application to urinary bladder cancer data from Connecticut.

    abstract::With increasingly abundant spatial data in the form of case counts or rates combined over areal regions (eg, ZIP codes, census tracts, or counties), interest turns to formal identification of difference "boundaries," or barriers on the map, in addition to the estimated statistical map itself. "Boundary" refers to a bo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7408

    authors: Guhaniyogi R

    更新日期:2017-11-10 00:00:00

  • R2: a useful measure of model performance when predicting a dichotomous outcome.

    abstract::R2 has been criticized as a measure of model performance when predicting a dichotomous outcome, both because its value is often low and because it is sensitive to the prevalence of the event of interest. The C statistic is more widely used to measure model performance in a 0/1 setting. We use a simple parametric famil...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19990228)18:4<375::aid-sim

    authors: Ash A,Shwartz M

    更新日期:1999-02-28 00:00:00

  • Hierarchical nested trial design (HNTD) for demonstrating treatment efficacy of new antibacterial drugs in patient populations with emerging bacterial resistance.

    abstract::In the last decade or so, pharmaceutical drug development activities in the area of new antibacterial drugs for treating serious bacterial diseases have declined, and at the same time, there are worries that the increased prevalence of antibiotic-resistant bacterial infections, especially the increase in drug-resistan...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6233

    authors: Huque MF,Valappil T,Soon GG

    更新日期:2014-11-10 00:00:00

  • Modelling age-specific risk: application to dementia.

    abstract::We give up-to-date methods for estimating the age-specific incidence of a disease and for estimating the effect of risk factors. We recommend taking age as the basic time scale of the analysis; then, the hazard function can be interpreted as the age-specific incidence of the disease. This choice raises a delayed entry...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19980915)17:17<1973::aid-s

    authors: Commenges D,Letenneur L,Joly P,Alioum A,Dartigues JF

    更新日期:1998-09-15 00:00:00

  • Assessing the effect of interventions in the context of mixture distributions with detection limits.

    abstract::Many quantitative assay measurements of metabolites of environmental toxicants in clinical investigations are subject to left censoring due to values falling below assay detection limits. Moreover, when observations occur in both unexposed individuals and exposed individuals who reflect a mixture of two distributions ...

    journal_title:Statistics in medicine

    pub_type: 临床试验,杂志文章,随机对照试验

    doi:10.1002/sim.2079

    authors: Chu H,Kensler TW,Muñoz A

    更新日期:2005-07-15 00:00:00

  • Analyzing longitudinal data to characterize the accuracy of markers used to select treatment.

    abstract::With the increasing availability of detailed clinical information, there is optimism that treatment choices can be selectively directed to those individuals most likely to benefit. While standard clinical trials can establish whether a treatment appears to be effective on average, subsequent work is needed to determin...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6138

    authors: Sitlani CM,Heagerty PJ

    更新日期:2014-07-30 00:00:00

  • Bootstrap confidence intervals for medical costs with censored observations.

    abstract::Medical costs data with administratively censored observations often arise in cost-effectiveness studies of treatments for life-threatening diseases. Mean of medical costs incurred from the start of a treatment until death or a certain time point after the implementation of treatment is frequently of interest. In many...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1556

    authors: Jiang H,Zhou XH

    更新日期:2004-11-15 00:00:00

  • A general approach to evaluating the bias of 2-stage instrumental variable estimators.

    abstract::Unmeasured confounding is a common concern when researchers attempt to estimate a treatment effect using observational data or randomized studies with nonperfect compliance. To address this concern, instrumental variable methods, such as 2-stage predictor substitution (2SPS) and 2-stage residual inclusion (2SRI), have...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.7636

    authors: Wan F,Small D,Mitra N

    更新日期:2018-05-30 00:00:00

  • Posterior predictive model checks for disease mapping models.

    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

    pub_type: 杂志文章

    doi:10.1002/1097-0258(20000915/30)19:17/18<2377::aid-s

    authors: Stern HS,Cressie N

    更新日期:2000-09-15 00:00:00

  • Bayesian sensitivity analysis of incomplete data: bridging pattern-mixture and selection models.

    abstract::Pattern-mixture models (PMM) and selection models (SM) are alternative approaches for statistical analysis when faced with incomplete data and a nonignorable missing-data mechanism. Both models make empirically unverifiable assumptions and need additional constraints to identify the parameters. Here, we first introduc...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6302

    authors: Kaciroti NA,Raghunathan T

    更新日期:2014-11-30 00:00:00

  • The effect of non-differential outcome misclassification on estimates of the attributable and prevented fraction.

    abstract::This paper considers the effect of non-differential outcome misclassification on the population attributable fraction and the population prevented fraction. I examine the bias in the attributable and the prevented fraction derived from a risk ratio estimate as a function of the sensitivity and specificity of the outco...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780100308

    authors: Hsieh CC

    更新日期:1991-03-01 00:00:00

  • Bayesian predictive approach for inference about proportions.

    abstract::This paper investigates the Bayesian procedures for comparing proportions. These procedures are especially suitable for accepting (or rejecting) the equivalence of two population proportions. Furthermore the Bayesian predictive probabilities provide a natural and flexible tool in monitoring trials, especially for choo...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780140924

    authors: Lecoutre B,Derzko G,Grouin JM

    更新日期:1995-05-15 00:00:00

  • A sensitivity analysis for subverting randomization in controlled trials.

    abstract::In some randomized controlled trials, subjects with a better prognosis may be diverted into the treatment group. This subverting of randomization results in an unobserved non-compliance with the originally intended treatment assignment. Consequently, the estimate of treatment effect from these trials may be biased. Th...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.715

    authors: Marcus SM

    更新日期:2001-02-28 00:00:00

  • Estimation of ROC curve with complex survey data.

    abstract::The receiver operating characteristic (ROC) curve can be utilized to evaluate the performance of diagnostic tests. The area under the ROC curve (AUC) is a widely used summary index for comparing multiple ROC curves. Both parametric and nonparametric methods have been developed to estimate and compare the AUCs. However...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.6405

    authors: Yao W,Li Z,Graubard BI

    更新日期:2015-04-15 00:00:00

  • Correcting for regression in assessing the response to treatment in a selected population.

    abstract::Previous work on the consequences of regression to the mean for the interpretation of responses to treatment is extended to the situation where the response measured is the proportional change in some variable. Methods for correcting for the bias are discussed. ...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4780060203

    authors: Curnow RN

    更新日期:1987-03-01 00:00:00

  • Modelling the geographical distribution of co-infection risk from single-disease surveys.

    abstract:BACKGROUND:The need to deliver interventions targeting multiple diseases in a cost-effective manner calls for integrated disease control efforts. Consequently, maps are required that show where the risk of co-infection is particularly high. Co-infection risk is preferably estimated via Bayesian geostatistical multinomi...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4243

    authors: Schur N,Gosoniu L,Raso G,Utzinger J,Vounatsou P

    更新日期:2011-06-30 00:00:00

  • Methods for the statistical analysis of binary data in split-mouth designs with baseline measurements.

    abstract::Many split-mouth trials are characterized by the pairing of site-specific outcome and baseline data within each segment of a subject's mouth. However when the response variable of interest is binary, methods of statistical analysis for this design are not well developed. In this paper we present several analytic appro...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.2782

    authors: Donner A,Zou GY

    更新日期:2007-08-15 00:00:00

  • Sequential designs for phase III clinical trials incorporating treatment selection.

    abstract::Most statistical methodology for phase III clinical trials focuses on the comparison of a single experimental treatment with a control. An increasing desire to reduce the time before regulatory approval of a new drug is sought has led to development of two-stage or sequential designs for trials that combine the defini...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.1362

    authors: Stallard N,Todd S

    更新日期:2003-03-15 00:00:00

  • There is no impact of exposure measurement error on latency estimation in linear models.

    abstract::Identification of the latency period for the effect of a time-varying exposure is key when assessing many environmental, nutritional, and behavioral risk factors. A pre-specified exposure metric involving an unknown latency parameter is often used in the statistical model for the exposure-disease relationship. Likelih...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.8038

    authors: Peskoe SB,Spiegelman D,Wang M

    更新日期:2019-03-30 00:00:00

  • Comparison of non parallel immunoassay curves resulting from mixtures of competing antigens.

    abstract::Relative potency is a measure that has been used for many years to summarize the comparison of dose-response curves in parallel line bioassays. When response curves for two preparations are not parallel the traditional definition of relative potency no longer applies. We review the concept of relative potency and show...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/(sici)1097-0258(19970530)16:10<1151::aid-s

    authors: Kaiser MS,Siev D

    更新日期:1997-05-30 00:00:00

  • A multiple imputation strategy for incomplete longitudinal data.

    abstract::Longitudinal studies are commonly used to study processes of change. Because data are collected over time, missing data are pervasive in longitudinal studies, and complete ascertainment of all variables is rare. In this paper a new imputation strategy for completing longitudinal data sets is proposed. The proposed met...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.740

    authors: Landrum MB,Becker MP

    更新日期:2001-09-15 00:00:00

  • Subgroup identification from randomized clinical trial data.

    abstract::We consider the problem of identifying a subgroup of patients who may have an enhanced treatment effect in a randomized clinical trial, and it is desirable that the subgroup be defined by a limited number of covariates. For this problem, the development of a standard, pre-determined strategy may help to avoid the well...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.4322

    authors: Foster JC,Taylor JM,Ruberg SJ

    更新日期:2011-10-30 00:00:00

  • An evaluation of phase I clinical trial designs in the continuous dose-response setting.

    abstract::Both traditional phase I designs and the increasingly popular continual reassessment method (CRM) designs select an estimate of maximum tolerable dose (MTD) from among a set of prespecified dose levels. Although CRM designs use an implied dose-response model to select the next dose level, in general it is neither assu...

    journal_title:Statistics in medicine

    pub_type: 杂志文章

    doi:10.1002/sim.903

    authors: Storer BE

    更新日期:2001-08-30 00:00:00