Pharmaceutical Statistics

Papers
(The TQCC of Pharmaceutical Statistics is 2. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2021-03-01 to 2025-03-01.)
ArticleCitations
Bayesian single‐to‐double arm transition design using both short‐term and long‐term endpoints47
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy23
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials17
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Issue Information16
Conditional power and information fraction calculations at an interim analysis for random coefficient models15
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Logistic retainment interval dose exploration design for Phase I clinical trials of cytotoxic agents13
A semiparametric modeling approach for analyzing clinical biomarkers restricted to limits of detection12
Inverse probability of censoring weighting for visual predictive checks of time‐to‐event models with time‐varying covariates12
Bayesian optimal phase II designs with dual‐criterion decision making11
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments11
Evaluating response‐adaptive randomization procedures for recurrent events and terminal event data using a composite endpoint9
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling9
Issue Information9
Assessing and communicating heterogeneity of treatment effects for patient subpopulations: The hardest problem there is9
Using the Bayesian detection of potential risk using inference on blinded safety data (BDRIBS) method to support the decision to refer an event for unblinded evaluation9
Matrix decomposition in meta‐analysis for extraction of adverse event pattern and patient‐level safety profile9
Sample size re‐estimation for response‐adaptive randomized clinical trials8
A marginalized two‐part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers8
Frailty model with change points for survival analysis8
Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power8
Identifying treatment effects using trimmed means when data are missing not at random7
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Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials7
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact7
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint6
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design6
Using an early outcome as the sole source of information of interim decisions regarding treatment effect on a long‐term endpoint: The non‐Gaussian case6
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting6
T3 + 3: 3 + 3 Design With Delayed Outcomes6
A comparative study of adaptive trial designs for dose optimization6
Estimating and comparing adverse event probabilities in the presence of varying follow‐up times and competing events5
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study5
Sample size calculation in clinical trials with two co‐primary endpoints including overdispersed count and continuous outcomes5
Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation5
Selection bias, investment decisions and treatment effect distributions5
Using marginal structural models to analyze the impact of subsequent therapy on the treatment effect in survival data: Simulations and clinical trial examples5
Sample size calculation for comparing two ROC curves5
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology5
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes4
Multiple imputation analysis of Miettinen‐Nurminen interval for difference in proportions4
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology4
The utilities and pitfalls of stratified analysis in challenging situations4
Estimation of treatment effects in short‐term depression studies. An evaluation based on the ICH E9(R1) estimands framework4
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Simulating and reporting frequentist operating characteristics of clinical trials that borrow external information: Towards a fair comparison in case of one‐arm and hybrid control two‐arm trials4
Comparison of Prior Distributions for the Heterogeneity Parameter in a Rare Events Meta‐Analysis of a Few Studies4
Pre‐Posterior Distributions in Drug Development and Their Properties4
The Role of CMC Statisticians: Co‐Practitioners of the Scientific Method4
Flexible Spline Models for Blinded Sample Size Reestimation in Event‐Driven Clinical Trials4
Synergy evaluation of non‐normalizable dose–response data: Generalization of combination index for the linear effect of drugs3
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials3
Re‐randomization tests as sensitivity analyses to confirm immunological noninferiority of an investigational vaccine: Case study3
Issue Information3
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data3
Empirical likelihood confidence interval for sensitivity to the early disease stage3
The detailed clinical objectives approach to designing clinical trials and choosing estimands3
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior3
Issue Information3
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery3
A conservative approach to leveraging external evidence for effective clinical trial design3
Assessing the performance of group‐based trajectory modeling method to discover different patterns of medication adherence3
Setting the control limit at release for stability assurance3
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A meta‐analytic framework to adjust for bias in external control studies3
Improving sample size recalculation in adaptive clinical trials by resampling3
Issue Information3
Combining evidence from clinical trials in conditional or accelerated approval3
A dynamic power prior approach to non‐inferiority trials for normal means3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance3
Improving early phase oncology clinical trial design: An opportunity for statisticians3
Issue Information3
On Some Modeling Issues in Estimating Vaccine Efficacy3
A note from the editors2
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies2
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose2
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks2
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A targeted simulation‐extrapolation method for evaluating biomarkers based on new technologies in precision medicine2
Left truncation in linked data: A practical guide to understanding left truncation and applying it using SAS and R2
Adjusting for covariates in analysis based on restricted mean survival times2
A simulation‐free group sequential design with max‐combo tests in the presence of non‐proportional hazards2
Practical and robust test for comparing binomial proportions in the randomized phase II setting2
An adaptive biomarker basket design in phase II oncology trials2
Applying the Estimand Framework to Non‐Inferiority Trials2
Statistical considerations for design and analysis of stability, comparability and formulation tests2
Rationale for the update algorithm of the graphical approach to sequentially rejective multiple test procedures2
A mixed effects model for analyzing area under the curve of longitudinally measured biomarkers with missing data2
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials2
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials2
Statistical modeling approaches for the comparison of dissolution profiles2
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A two‐stage drop‐the‐losers design for time‐to‐event outcome using a historical control arm2
Response to the letter to the editor regarding our article ‘statistical methodology for highly variable compounds: A novel design approach for the ofatumumab phase 2 bioequivalence study’ https://doi.2
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials2
Current developments of the estimand concept2
Efficient Study Design and Analysis of Longitudinal Dose–Response Data Using Fractional Polynomials2
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Estimation and expected sample size in Simon's two‐stage designs that stop as early as possible2
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data2
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications2
Optimal sample size allocation for two‐arm superiority and non‐inferiority trials with binary endpoints2
Improving the assessment of the probability of success in late stage drug development2
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring2
Identifying treatment effect heterogeneity in dose‐finding trials using Bayesian hierarchical models2
Potency Assay Variability Estimation in Practice2
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET2
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines2
The individual‐level surrogate threshold effect in a causal‐inference setting with normally distributed endpoints2
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