PHARMACEUTICAL STATISTICS

Subgroup analysis and interpretation for phase 3 confirmatory trials: White paper of the EFSPI/PSI working group on subgroup analysis.

Planning and analyzing clinical trials with competing risks: Recommendations for choosing appropriate statistical methodology.

Reference‐based sensitivity analysis for time‐to‐event data

A flexible parametric survival model for fitting time to event data in clinical trials.

A comparison of subgroup identification methods in clinical drug development: Simulation study and regulatory considerations

Nonlinear mixed-effects models with misspecified random-effects distribution.

Performance characteristics of the adjusted r2 algorithm for determining the start of the terminal disposition phase and comparison with a simple r2 algorithm and a visual inspection method.

Nested combination tests with a time-to-event endpoint using a short-term endpoint for design adaptations.

Controlling type 1 error rate for sequential, bioequivalence studies with crossover designs.

Commentary on: Subgroup analysis and interpretation for phase 3 confirmatory trials: White Paper of the EFSPI/PSI working group on subgroup analysis by Dane, Spencer, Rosenkranz, Lipkovich, and Parke

Empowering phase II clinical trials to reduce phase III failures.

TITE-BOIN-ET: Time-to-event Bayesian optimal interval design to accelerate dose-finding based on both efficacy and toxicity outcomes.

Empirical evaluation of the implementation of the EMA guideline on missing data in confirmatory clinical trials: Specification of mixed models for longitudinal data in study protocols

Applications of Bayesian statistical methodology to clinical trial design: A case study of a phase 2 trial with an interim futility assessment in patients with knee osteoarthritis.

A simple test for the treatment effect in clinical trials with a sequential parallel comparison design and negative binomial outcomes.

Treatment policy estimands for recurrent event data using data collected after cessation of randomised treatment.

The win ratio: Impact of censoring and follow-up time and use with nonproportional hazards.

Multistate modeling of clinical hold in randomized clinical trials.

Design of experiments and the virtual PCR simulator: An online game for pharmaceutical scientists and biotechnologists

Criteria for reporting noncompartmental estimates of half-life and area under the curve extrapolated to infinity.

Lessons from meta-analyses of randomized clinical trials for analysis of distributed networks of observational databases.

Calculation of confidence intervals for a finite population size.

Tests for noninferiority trials with binomial endpoints: A guide to modern and quasi-exact methods for biomedical researchers.

Statistical considerations in a delayed-start design to demonstrate disease modification effect in neurodegenerative disorders.

A generalized Bayesian nonlinear mixed-effects regression model for zero-inflated longitudinal count data in tuberculosis trials.

Between a ROC and a hard place: Teaching prevalence plots to understand real world biomarker performance in the clinic.

Bayesian sequential integration within a preclinical pharmacokinetic and pharmacodynamic modeling framework: Lessons learned.

Integrating phase 2 into phase 3 based on an intermediate endpoint while accounting for a cure proportion-With an application to the design of a clinical trial in acute myeloid leukemia.

A flexible multi-domain test with adaptive weights and its application to clinical trials.

A comparative study of confidence intervals to assess biosimilarity from analytical data.

Assessing noninferiority: Evaluating efficacy of a new treatment without complete data.

Estimation of drug effect in radiographic progression for rheumatoid arthritis and psoriatic arthritis.

Bayesian sample-size determination methods considering both worthwhileness and unpromisingness for exploratory two-arm randomized clinical trials with binary endpoints.

Improvements to the Escalation with Overdose Control design and a comparison with the restricted Continual Reassessment Method.

A two-stage phase II clinical trial design with nested criteria for early stopping and efficacy.

Hazard ratio inference in stratified clinical trials with time-to-event endpoints and limited sample size.

Assessment of individual bioequivalence using sufficient bootstrap procedure.

Comment on Jaki et al., A proposal for a new PhD level curriculum on quantitative methods for drug development. Pharmaceutical Statistics 17 (5):593-606, Sep/Oct 2018, DOI: 10.1002/pst.1873.

Multivariate two-sample permutation tests for trials with multiple time-to-event outcomes.

Optimal designs for regional bridging studies using the Bayesian power prior method.

Controlling for confounding via propensity score methods can result in biased estimation of the conditional AUC: A simulation study.

Random effects models for estimation of the probability and time to progression of a continuous biomarker.

Statistical testing strategies for assessing treatment efficacy and marker accuracy in phase III trials.

Application of Bayesian analyses to doubly randomized delayed start, matched control designs to demonstrate disease modification.

Cancer immunotherapy trial design with delayed treatment effect.

Testing treatment-by-period interaction in four-period crossover trials.

Oncology phase II proof-of-concept studies with multiple targets: Randomized controlled trial or single arm?