OOIR: Observatory of International Research

Papers

(The median citation count of Statistics in Biopharmaceutical Research is 1. 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 2021-11-01 to 2025-11-01.)

Article	Citations
Treatment Selection at Interim Analysis Based on a Correlated Early Endpoint in Adaptive Two-Stage Trials with a Primary Endpoint Based on Count Data	35
U-MET: Utility-Based Dose Optimization Approach for Multiple-Dose Randomized Trial Designs	23
DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials	23
Predicting Probability of Success for Phase III Trials via Propensity-Score-Based External Data Borrowing	16
The Current Landscape of HTA Framework and Key Challenges	14
Consistency Based Multiplicity Adjustment Approach—Multiple Doses in Phase III Studies	13
Modified Simon’s Two-Stage Design for Phase IIA Clinical Trials in Oncology—Dynamic Monitoring and More Flexibility	12
We Need Subject Matter Expertise to Choose and Identify Causal Estimands: Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	11
Joint Analysis of Longitudinal Ordinal Categorical Item Response Data and Survival Times with Cure Fraction	11
A Basket Trial Design Based on Power Priors	10
Missing Data Imputation for a Multivariate Outcome of Mixed Variable Types	10
Some Multiplicity Adjustment Procedures for Clinical Trials with Sequential Design and Multiple Endpoints	10
Design Strategy and Consideration for Oncology Dose-Optimization: An Industry Perspective	10
Bayesian and Frequentist Approaches to Rescuing Disrupted Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	10
Joint Analysis of Longitudinal Data and Zero-Inflated Recurrent Events	9
Augmenting the Control Arm of Randomized Trials by Incorporating Multiple External Data Sources Using Propensity Score Stratification and Data-Driven Mixture Prior	9
A Two-Stage Covariate-Adjusted Response-Adaptive Enrichment Design	9
Monitoring Ongoing Clinical Trials Under Fractional Brownian Motion With Drift	9
Some Group Sequential Trials from Industry over the Last 30 Years	9
Statistical Considerations and Challenges for Pivotal Clinical Studies of Artificial Intelligence Medical Tests for Widespread Use: Opportunities for Inter-Disciplinary Collaboration	9
Statistics in Biopharmaceutical Research Best Papers Award 2023	8
MT-Keyboard: A Bayesian Model-Assisted Interval Design to Account for Toxicity Grades and Types for Phase I Trials	8
A New Centralized Statistical Monitoring Method for Detecting Atypical Distribution of Qualitative Variables in Multicenter Randomized Controlled Trials	8
Backward Sequential Significance Testing in Survival Trials	8
Closing a Chapter: A Farewell from the Editor of Statistics in Biopharmaceutical Research	8

Bayesian Hierarchical Model with Adaptive Similarity Evaluation of Treatment Effects in Oncology Basket Trials	7
Statistical Issues and Challenges in Clinical Trials for COVID-19 Treatments, Vaccines, Medical Devices and Diagnostics	7
Reference-Based Multiple Imputation—What is the Right Variance and How to Estimate It	7
Exact Matching as an Alternative to Propensity Score Matching	7
Which Randomization Methods Are Used Most Frequently in Clinical Trials? Results of a Survey by the Randomization Working Group	6
Effects of Allocation Method and Time Trends on Identification of the Best Arm in Multi-Arm Trials	6
Properties of Two While-Alive Estimands for Recurrent Events and Their Potential Estimators	6
Pre-Specified Safety Analysis of OS Data for Trials in Indolent or Early-Stage Cancers	6
Bayesian Basket Trial Design Accounting for Multiple Cutoffs of an Ambiguous Biomarker	6
A Comparison of Different Approaches to Bayesian Hierarchical Models in a Basket Trial to Evaluate the Benefits of Increasing Complexity	6
A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim Analysis When Slope Is the Primary Endpoint	5
A Primer of Quasi-Exact Tests for Randomized Clinical Trials with Binary Endpoints	5
Adaptive Endpoints Selection with Application in Rare Disease	5
Rejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	5
Editor’s Note	5
Statistical Thinking and Innovation with Global Impact—Special Issue for the 2023 Regulatory-Industry Statistics Workshop	5
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development	5
Treatment-Control Comparisons in Platform Trials Including Non-Concurrent Controls	5
Nonconcurrent Controls in Platform Trials: Can We Borrow Their Concurrent Observation Data?	5
Bias-Corrected Group Sequential Design in the Presence of Surrogate Endpoints with Application to PALM Trial	5
On Variance Estimation for the One-Sample Log-Rank Test	4
Rejoinder to Commentaries on the “Monitoring OS in Pivotal Trials in Indolent Cancers” Article	4
A Propensity-Score Integrated Approach to Bayesian Dynamic Power Prior Borrowing	4
Estimands for Continuous Longitudinal Outcomes in the Presence of Treatment Discontinuation—A Simulation Study in Hyperkalemia Treatments	4
The Use of Machine Learning in Regulatory Drug Safety Evaluation	4
A Multi-Arm Multi-Stage Group Sequential Phase 2/3 Design with Dose Selection for Oncology Trials	3
Bayesian Optimal Phase II Design for Randomized Clinical Trials	3
A Simulation Study on the Estimation of the Effect in the Hypothetical Scenario of No Use of Symptomatic Treatment in Trials for Disease-Modifying Agents for Alzheimer’s Disease	3
Performance of LTMLE in the Presence of Missing Data in Control-Matched Longitudinal Studies	3
The NISS Ingram Olkin Forum on Unplanned Clinical Trial Disruptions	3
Comment on “Monitoring Overall Survival in Pivotal Trials in Indolent Cancers”	3
Rejoinder to Letter to the Editor—Selecting an Optimal Design for a Nonrandomized Comparative Study: A Commentary on “Some Considerations on Design and Analysis Plan on a Nonrandomized Comparative Stu	3
An Alternative to Traditional Sample Size Determination for Small Patient Populations	3
Use of Common Control Arms Across Substudies in a Master Protocol via Ridge Estimation	3
Evaluating the impact of outcome delay on the efficiency of two-arm group-sequential trials	3
Correction	3
A Comprehensive Bayesian Approach to Quantify and Compare Benefit-Risk of Medical Products	3
Assessing the Commonly Used Assumptions in Estimating the Principal Causal Effect in Clinical Trials	3
A Rényi-Divergence-Based Family of Metrics for the Evaluation of Surrogate Endpoints in a Causal Inference Framework	3
Accuracy and Safety of Novel Designs for Phase I Drug-Combination Oncology Trials	3
Random Effects Meta-Analysis of Contingency Tables with Complete and Partially Complete Data, with Application to COVID-19 Research	2
A Modification of Location Commensurate Power Prior in Clinical Trials	2
Targeted Learning: Toward a Future Informed by Real-World Evidence	2
Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	2
Dionne Price: An Esteemed Colleague, Inspirational Leader, Trailblazer Statistician and a Dear Friend	2
A Composite Endpoint for Treatment Benefit According to Patient Preference	2
Vaccine Development during a Pandemic: General Lessons for Clinical Trial Design	2
Three-Way Mixed Effect ANOVA to Estimate MRMC Limits of Agreement	2
The Role of Statistical Thinking in Biopharmaceutical Research	2
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease	2

Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators	2
Monitoring Overall Survival in Phase 3 Indolent Cancers: An Extended Framework and an Example	2
Weighted Approach for Estimating Effects in Principal Strata With Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials	2
Eliciting the Discount Parameter in a Power Prior Method on the Basis of the Type I Error Consideration	2
Rejoinder to Commentaries on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	2
Trial Design with Win Statistics for Multiple Time-to-Event Endpoints with Hierarchy	2
Coping with Information Loss and the Use of Auxiliary Sources of Data: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	2
Balancing the Objectives of Statistical Efficiency and Allocation Randomness in Randomized Controlled Trials	2
Subgroup Analyses in Confirmatory Clinical Trials Using Bayesian Hierarchical Models	2
Chasing Shadows: How Implausible Assumptions Skew Our Understanding of Causal Estimands	2
Rejoinder: Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	2
Non-Inferiority Testing on the Accuracy of Two Qualitative Microbiological Methods on Paired Binary Data	2
Combined Tests Based on Restricted Mean Time Lost for Competing Risks Data	2
Modified Robust Meta-Analytic-Predictive Priors for Incorporating Historical Controls in Clinical Trials	2
Assessing Contribution of Treatment Phases through Tipping Point Analyses via Counterfactual Elicitation Using Rank Preserving Structural Failure Time Models	2
A Quantitative Bias Analysis to Assess Constancy Assumption in Noninferiority Trials Using Bayesian Hierarchical Models	2
Good Data Science Practice: Moving Toward a Code of Practice for Drug Development	2
Evaluation of current statistical methods for implementing Quality Tolerance Limits	1
A Bayesian Adaptive Umbrella Trial Design with Robust Information Borrowing for Screening Multiple Combination Therapies	1
Bayesian Shrinkage Estimation of Credible Subgroups for Count Data with Excess Zeros	1
Meta-Analysis of Moxifloxacin Concentration-QTc Effects with Application to Assay Sensitivity Assessment	1
An Unbiased Method to Approximate a Principal Estimand	1
Conditional and Unconditional Treatment Effects in Randomized Clinical Trials: Estimands, Estimation, and Interpretation	1
Type I Error Control of an Adaptive Endpoint Selection Procedure	1
Multiple Surrogates in the Meta-Analytic Setting for Normally Distributed Endpoints	1
Handling Intercurrent Events Through Hypothetical Strategy in Delayed-Start Designs	1
Incorporating Intermediate Endpoint in Two-Stage Design Decision Making	1
Beyond the Classical Type I Error: Bayesian Metrics for Bayesian Designs Using Informative Priors	1
Qualitative versus Quantitative Treatment-by-Subgroup Interaction in Equivalence Studies with Multiple Subgroups	1
Randomization-Based Inference for Clinical Trials with Missing Outcome Data	1
Comparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART)	1
Assessment of Treatment Effect Heterogeneity for Multiregional Randomized Clinical Trials	1
Probability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development	1
Shrinkage Estimation Methods for Subgroup Analyses	1
Editor’s Note	1
Editor’s Note: Special Section on Estimands, Design and Analysis of Clinical Trials with Time-to-Event Outcomes	1
Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes	1
Systematic Comparison of Bayesian Basket Trial Designs with Unequal Sample Sizes and Proposal of a New Method Based on Power Priors	1
Assessing the Harm and Benefit of Cancer Therapies with an Overall Survival Endpoint in Comparative Clinical Studies	1
Bayesian Simultaneous Credible Intervals for Effect Measures from Multiple Markers	1
Improved Trimmed Weighted Hochberg Procedures With Two Endpoints and Sample Size Optimization	1
Using Randomization Tests to Address Disruptions in Clinical Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	1
Bayesian Optimal Designs for Multi-Arm Multi-Stage Phase II Randomized Clinical Trials with Multiple Endpoints	1
The Statistical and Practical Implications of Mis-Stratified Randomization in Clinical Trials	1
A Statistical Method for Protocol Modifications With Heterogeneous Population Variances	1
Hybrid Control Design with Commensurate Prior Constructed Using Propensity Score-Matched External Controls: A Simulation Study	1
Combining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score	1
Joint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes	1
Adjusting for Time-Varying Treatment Switches in Randomized Clinical Trials: The Danger of Extrapolation and How to Address It	1
A Sequential Predictive Power Design for a COVID Vaccine Trial	1
Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints	1
A Method for Ensuring a Consistent Dose–Response Relationship Between An Entire Population and One Region in Multiregional Dose–Response Studies Using MCP-Mod	1
Quantifying Replicability and Consistency in Systematic Reviews	1
Comment on “Non-Proportional Hazards – an Evaluation of the MaxCombo Test in Cancer Clinical Trials” by the Cross-Pharma Non-Proportional Hazards Working Group	1
Good Data Science Practice: Moving toward a Code of Practice for Drug Development (Rejoinder)	1
Sample Size Calculation and Timing of Dose Selection in a Multiple-Dose Clinical Trial	1
Bayesian Design of Superiority Trials: Methods and Applications	1
Generalized Likelihood Ratios for Designing Dose Optimization Studies of Targeted Therapies	1
Remembering Gregory Campbell (1949–2023): An Accomplished Leader, Mentor, and Biostatistical Innovator	1
Comparison of g-estimation approaches for handling symptomatic medication at multiple timepoints in Alzheimer's Disease with a hypothetical strategy	1
Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	1
Covariate-Adjusted Response Adaptive Designs for Competing Risk Survival Models	1