We undertake here a comprehensive simulation study of the theoretical properties that we derive in a companion article devoted to the asymptotic study of adaptive group sequential designs in the case of randomized clinical trials (RCTs) with binary treatment, binary outcome and no covariate. By...

This article is devoted to the asymptotic study of adaptive group sequential designs in the case of randomized clinical trials (RCTs) with binary treatment, binary outcome and no covariate. By adaptive design, we mean in this setting a RCT design that allows the investigator to dynamically...

When cost data are collected in a clinical study, interest centers on the between-treatment difference in mean cost. When censoring is present, the resulting loss of information can be limited by collecting cost data for several pre-specified time intervals, leading to censored longitudinal cost...

Consider the problem of testing s null hypotheses simultaneously. In order to deal with the multiplicity problem, the classical approach is to restrict attention to multiple testing procedures that control the familywise error rate (FWE). The closure method of Marcus et al. (1976) reduces the...

With a binary response Y, the dose-response model under consideration is logistic in flavor with pr(Y=1 | D) = R (1+R)-1, R = λ0 + EAR D, where λ0 is the baseline incidence rate and EAR is the excess absolute risk per gray. The calculated thyroid dose of a person i is expressed as Dimes =...

Relative risks have become a popular measure of treatment effect for binary outcomes in randomized controlled trials (RCTs). Relative risks can be estimated directly using log binomial regression but the model may fail to converge. Alternative methods are available for estimating relative risks...

The paired availability design for historical controls postulated four classes corresponding to the treatment (old or new) a participant would receive if arrival occurred during either of two time periods associated with different availabilities of treatment. These classes were later extended to...

Dr. Pearl invites researchers to justify their use of principal stratification. This comment explains how the use of principal stratification simplified a complex mediational problem encountered when evaluating a smoking cessation intervention’s effect on reducing smoking withdrawal symptoms.

We investigate a competing risks model, using the specification of the Gompertz distribution for failure times from competing causes and the inverse Gaussian distribution for failure times from the cause of interest. The expectation-maximization algorithm is used for parameter estimation and the...

This work considers the estimation of the size N of a closed population using incomplete lists of its members. Capture histories are constructed by establishing the presence or the absence of each individual in all the lists available. Models for data featuring a heterogeneous catchability and...