OOIR: Observatory of International Research

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.)

Article	Citations
Bayesian single‐to‐double arm transition design using both short‐term and long‐term endpoints	47
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	23
A Bayesian method for safety signal detection in ongoing blinded randomised controlled trials	17
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Issue Information	16
Conditional power and information fraction calculations at an interim analysis for random coefficient models	15
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Logistic retainment interval dose exploration design for Phase I clinical trials of cytotoxic agents	13
A semiparametric modeling approach for analyzing clinical biomarkers restricted to limits of detection	12
Inverse probability of censoring weighting for visual predictive checks of time‐to‐event models with time‐varying covariates	12
Bayesian optimal phase II designs with dual‐criterion decision making	11
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	11
Confirmatory efficacy testing for individual dose–placebo comparisons using serial gatekeeping procedure in dose‐finding trials with multiple comparison procedures–modeling	9
Issue Information	9
Assessing and communicating heterogeneity of treatment effects for patient subpopulations: The hardest problem there is	9
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 evaluation	9
Matrix decomposition in meta‐analysis for extraction of adverse event pattern and patient‐level safety profile	9
Evaluating response‐adaptive randomization procedures for recurrent events and terminal event data using a composite endpoint	9
A marginalized two‐part joint model for a longitudinal biomarker and a terminal event with application to advanced head and neck cancers	8
Frailty model with change points for survival analysis	8
Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power	8
Sample size re‐estimation for response‐adaptive randomized clinical trials	8
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Evaluation of a flexible piecewise linear mixed‐effects model in the analysis of randomized cross‐over trials	7
The Acute Stroke Therapy by Inhibition of Neutrophils study – Key features and impact	7

Identifying treatment effects using trimmed means when data are missing not at random	7
Using an early outcome as the sole source of information of interim decisions regarding treatment effect on a long‐term endpoint: The non‐Gaussian case	6
Generalizing Treatment Effect to a Target Population Without Individual Patient Data in a Real‐World Setting	6
T3 + 3: 3 + 3 Design With Delayed Outcomes	6
A comparative study of adaptive trial designs for dose optimization	6
Futility Interim Analysis Based on Probability of Success Using a Surrogate Endpoint	6
Reparametrized Firth's Logistic Regressions for Dose‐Finding Study With the Biased‐Coin Design	6
Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	5
Sample size calculation in clinical trials with two co‐primary endpoints including overdispersed count and continuous outcomes	5
Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation	5
Selection bias, investment decisions and treatment effect distributions	5
Using marginal structural models to analyze the impact of subsequent therapy on the treatment effect in survival data: Simulations and clinical trial examples	5
Sample size calculation for comparing two ROC curves	5
A Likelihood Perspective on Dose‐Finding Study Designs in Oncology	5
Estimating and comparing adverse event probabilities in the presence of varying follow‐up times and competing events	5
Multiple imputation analysis of Miettinen‐Nurminen interval for difference in proportions	4
A Bayesian optimal interval design for dose optimization with a randomization scheme based on pharmacokinetics outcomes in oncology	4
The utilities and pitfalls of stratified analysis in challenging situations	4
Estimation of treatment effects in short‐term depression studies. An evaluation based on the ICH E9(R1) estimands framework	4
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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 trials	4
Comparison of Prior Distributions for the Heterogeneity Parameter in a Rare Events Meta‐Analysis of a Few Studies	4
Pre‐Posterior Distributions in Drug Development and Their Properties	4
The Role of CMC Statisticians: Co‐Practitioners of the Scientific Method	4
Flexible Spline Models for Blinded Sample Size Reestimation in Event‐Driven Clinical Trials	4
Improved inference forMCP‐Modapproach using time‐to‐event endpoints with small sample sizes	4
Empirical likelihood confidence interval for sensitivity to the early disease stage	3
The detailed clinical objectives approach to designing clinical trials and choosing estimands	3
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	3
Issue Information	3
Confidence Intervals for the Risk Difference Between Secondary and Primary Infection Based on the Method of Variance Estimates Recovery	3
A conservative approach to leveraging external evidence for effective clinical trial design	3
Assessing the performance of group‐based trajectory modeling method to discover different patterns of medication adherence	3
Setting the control limit at release for stability assurance	3
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A meta‐analytic framework to adjust for bias in external control studies	3
Improving sample size recalculation in adaptive clinical trials by resampling	3
Issue Information	3
Combining evidence from clinical trials in conditional or accelerated approval	3
A dynamic power prior approach to non‐inferiority trials for normal means	3
Time‐to‐event estimands and loss to follow‐up in oncology in light of the estimands guidance	3
Improving early phase oncology clinical trial design: An opportunity for statisticians	3
Issue Information	3
On Some Modeling Issues in Estimating Vaccine Efficacy	3
Synergy evaluation of non‐normalizable dose–response data: Generalization of combination index for the linear effect of drugs	3
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	3
Re‐randomization tests as sensitivity analyses to confirm immunological noninferiority of an investigational vaccine: Case study	3
Issue Information	3
Information‐based group sequential design for post‐market safety monitoring of medical products using real world data	3
Type‐I‐error rate inflation in mixed models for repeated measures caused by ambiguous or incomplete model specifications	2

An adaptive biomarker basket design in phase II oncology trials	2
Optimal sample size allocation for two‐arm superiority and non‐inferiority trials with binary endpoints	2
Applying the Estimand Framework to Non‐Inferiority Trials	2
Weighted log‐rank test to compare two survival functions in the presence of dependent censoring	2
Potency Assay Variability Estimation in Practice	2
The individual‐level surrogate threshold effect in a causal‐inference setting with normally distributed endpoints	2
A note from the editors	2
Statistical modeling approaches for the comparison of dissolution profiles	2
CUSUMIN: A cumulative sum interval design for cancer phase I dose finding studies	2
A two‐stage drop‐the‐losers design for time‐to‐event outcome using a historical control arm	2
Improving early phase oncology clinical trial design: The case for finding the optimal biological dose	2
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials	2
Simulation‐based sample size calculations of marginal proportional means models for recurrent events with competing risks	2
Current developments of the estimand concept	2
A targeted simulation‐extrapolation method for evaluating biomarkers based on new technologies in precision medicine	2
Efficient Study Design and Analysis of Longitudinal Dose–Response Data Using Fractional Polynomials	2
Adjusting for covariates in analysis based on restricted mean survival times	2
Practical and robust test for comparing binomial proportions in the randomized phase II setting	2
Statistical considerations for design and analysis of stability, comparability and formulation tests	2
Rationale for the update algorithm of the graphical approach to sequentially rejective multiple test procedures	2
Improving the assessment of the probability of success in late stage drug development	2
A mixed effects model for analyzing area under the curve of longitudinally measured biomarkers with missing data	2
Identifying treatment effect heterogeneity in dose‐finding trials using Bayesian hierarchical models	2
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	2
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	2
Dynamic borrowing of historical controls adjusting for covariates in vaccine efficacy clinical trials	2
To Dilute or Not to Dilute: Nominal Titer Dosing for Genetic Medicines	2
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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
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Estimation and expected sample size in Simon's two‐stage designs that stop as early as possible	2
Left truncation in linked data: A practical guide to understanding left truncation and applying it using SAS and R	2
Digital twins and Bayesian dynamic borrowing: Two recent approaches for incorporating historical control data	2
A simulation‐free group sequential design with max‐combo tests in the presence of non‐proportional hazards	2