Statistics in Biopharmaceutical Research

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	27
Predicting Probability of Success for Phase III Trials via Propensity-Score-Based External Data Borrowing	22
Considerations on Interim Evaluation of OS in Pivotal Oncology Trials	18
A Landscape Assessment of Key Evidence Needs in Study Design and Statistical Methodologies for HTA Submissions	18
DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials	16
U-MET: Utility-Based Dose Optimization Approach for Multiple-Dose Randomized Trial Designs	16
Some Multiplicity Adjustment Procedures for Clinical Trials with Sequential Design and Multiple Endpoints	14
Consistency Based Multiplicity Adjustment Approach—Multiple Doses in Phase III Studies	14
Missing Data Imputation for a Multivariate Outcome of Mixed Variable Types	13
Bayesian and Frequentist Approaches to Rescuing Disrupted Trials: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions	12
Joint Analysis of Longitudinal Ordinal Categorical Item Response Data and Survival Times with Cure Fraction	11
Modified Simon’s Two-Stage Design for Phase IIA Clinical Trials in Oncology—Dynamic Monitoring and More Flexibility	10
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”	10
A Cautionary Note on E-Values Using Biased Risk Ratio Approximations from Hazard Ratios	9
Design Strategy and Consideration for Oncology Dose-Optimization: An Industry Perspective	8
Joint Analysis of Longitudinal Data and Zero-Inflated Recurrent Events	8
The Current Landscape of HTA Framework and Key Challenges	8
Some Group Sequential Trials from Industry over the Last 30 Years	8
A Basket Trial Design Based on Power Priors	8
An Enhanced Doubly Robust Causal Estimator for Nonrandomized Trials With Binary Outcome	8
LORDs: Locally Optimal Restricted Designs for Phase I/II Dose-Finding Studies	7
Optimizing Pediatric Dose Finding: A Phase I/II Design Integrating Adult Data	7
A Bayesian Latent Subgroup Design for Basket Trials with Survival Endpoints	7
A Two-Stage Covariate-Adjusted Response-Adaptive Enrichment Design	7
Monitoring Ongoing Clinical Trials Under Fractional Brownian Motion With Drift	7

Augmenting the Control Arm of Randomized Trials by Incorporating Multiple External Data Sources Using Propensity Score Stratification and Data-Driven Mixture Prior	7
Statistical Considerations and Challenges for Pivotal Clinical Studies of Artificial Intelligence Medical Tests for Widespread Use: Opportunities for Inter-Disciplinary Collaboration	7
A New Centralized Statistical Monitoring Method for Detecting Atypical Distribution of Qualitative Variables in Multicenter Randomized Controlled Trials	6
Bayesian Hierarchical Model with Adaptive Similarity Evaluation of Treatment Effects in Oncology Basket Trials	6
Backward Sequential Significance Testing in Survival Trials	6
Statistics in Biopharmaceutical Research Best Papers Award 2023	6
Closing a Chapter: A Farewell from the Editor of Statistics in Biopharmaceutical Research	6
MT-Keyboard: A Bayesian Model-Assisted Interval Design to Account for Toxicity Grades and Types for Phase I Trials	6
Which Randomization Methods Are Used Most Frequently in Clinical Trials? Results of a Survey by the Randomization Working Group	5
Exact Matching as an Alternative to Propensity Score Matching	5
Pre-Specified Safety Analysis of OS Data for Trials in Indolent or Early-Stage Cancers	5
Effects of Allocation Method and Time Trends on Identification of the Best Arm in Multi-Arm Trials	5
Navigating Priorities, Statistical Rigor, and Decision-Making on Multiplicity Questions in the Context of the EU Joint Clinical Assessment	5
Novel Statistical Designs and Considerations to Support Diversity and Inclusion in Clinical Trials	5
Information Borrowing in Bayesian Clinical Trials: choice of Tuning Parameters for the Robust Mixture Prior	5
Estimating Treatment Efficacy Controlling for Subsequent Therapy Usage: Comparison of Multiple Methods and Application to Hematologic Oncology Trials	4
Statistical Thinking and Innovation with Global Impact—Special Issue for the 2023 Regulatory-Industry Statistics Workshop	4
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development	4
Bayesian and Frequentist Stratified Analysis of Treatment Effects with Survival Data in Comparative Trials	4
A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim Analysis When Slope Is the Primary Endpoint	4
A Primer of Quasi-Exact Tests for Randomized Clinical Trials with Binary Endpoints	4
Adaptive Endpoints Selection with Application in Rare Disease	4
Bias-Corrected Group Sequential Design in the Presence of Surrogate Endpoints with Application to PALM Trial	4
Rejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	4
Nonconcurrent Controls in Platform Trials: Can We Borrow Their Concurrent Observation Data?	4
A Propensity-Score Integrated Approach to Bayesian Dynamic Power Prior Borrowing	3
Comment on “Monitoring Overall Survival in Pivotal Trials in Indolent Cancers”	3
Statistical Innovation for Integrated Evidence in Medical Product Development	3
BOOST: An Adaptive Biomarker-Guided Simon Two-Stage Design with Explicit Control of Pooled and Subgroup Inference	3
Assessing the Commonly Used Assumptions in Estimating the Principal Causal Effect in Clinical Trials	3
An Alternative to Traditional Sample Size Determination for Small Patient Populations	3
A Comprehensive Bayesian Approach to Quantify and Compare Benefit-Risk of Medical Products	3
A Multi-Arm Multi-Stage Group Sequential Phase 2/3 Design with Dose Selection for Oncology Trials	3
The Use of Machine Learning in Regulatory Drug Safety Evaluation	3
Rejoinder to Commentaries on the “Monitoring OS in Pivotal Trials in Indolent Cancers” Article	3
OFGSD: Optimal Futility Stopping Rules for Group Sequential Design	3
Editor’s Note	3
Inverse Probability of Treatment Weighting: A Simple and Effective Approach to Covariate Adjustment for Survival Endpoints in Randomized Clinical Trials	3
The NISS Ingram Olkin Forum on Unplanned Clinical Trial Disruptions	3
Multiplicity Control in Clinical Trials with Adaptive Selection Followed by Group-Sequential Testing	3
Correction	3
Treatment-Control Comparisons in Platform Trials Including Non-Concurrent Controls	3
Comparison of Methods to Analyze Time-to-Event Endpoints When Treatment Effect is Delayed	3
Challenges of Modeling Biomarker Effects on Time-to-Event Outcomes	3
A Graphical Framework for Testing Hierarchically Structured Hypothesis Families	3
A Bayesian Design with Efficient Backfilling and Expansion to Accelerate Phase I/II Trials Accommodating Late-Onset Competing Risk Outcomes	3
Causal Machine Learning Approaches for Adjusting Treatment Switching in Clinical Trials	3
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
Random Effects Meta-Analysis of Contingency Tables with Complete and Partially Complete Data, with Application to COVID-19 Research	2
Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators	2

Commentary on “Statistical Methodology Groups in the Pharmaceutical Industry”	2
Non-Inferiority Testing on the Accuracy of Two Qualitative Microbiological Methods on Paired Binary Data	2
Bayesian Estimation of Dynamic Treatment Regimens in a Partially Randomized Patient Preference, Sequential, Multiple-Assignment, Randomized Trial	2
Targeted Learning: Toward a Future Informed by Real-World Evidence	2
Rejoinder - Statistical Methodology Groups in the Pharmaceutical Industry #	2
Use of Common Control Arms Across Substudies in a Master Protocol via Ridge Estimation	2
A Rényi-Divergence-Based Family of Metrics for the Evaluation of Surrogate Endpoints in a Causal Inference Framework	2
Evaluating the Impact of Outcome Delay on the Efficiency of Two-Arm Group-Sequential Trials	2
Subgroup Analyses in Confirmatory Clinical Trials Using Bayesian Hierarchical Models	2
Balancing the Objectives of Statistical Efficiency and Allocation Randomness in Randomized Controlled Trials	2
The Role of Statistical Thinking in Biopharmaceutical Research	2
Prevalence Estimation in Finite Populations Adjusted for Misclassification	2
Performance of LTMLE in the Presence of Missing Data in Control-Matched Longitudinal Studies	2
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease	2
Dionne Price: An Esteemed Colleague, Inspirational Leader, Trailblazer Statistician and a Dear Friend	2
Chasing Shadows: How Implausible Assumptions Skew Our Understanding of Causal Estimands	2
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
Multiple Imputation with B-Splines (MI-B) in Clinical Trial Monitoring	1
Comparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART)	1
Bayesian Simultaneous Credible Intervals for Effect Measures from Multiple Markers	1
Remembering Gregory Campbell (1949–2023): An Accomplished Leader, Mentor, and Biostatistical Innovator	1
The Statistical and Practical Implications of Mis-Stratified Randomization in Clinical Trials	1
Modified Robust Meta-Analytic-Predictive Priors for Incorporating Historical Controls in Clinical Trials	1
Bayesian Dose Finding Model Using Ordinal Endpoints	1
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	1
Statistical Method for Threshold Value Determination of Diagnostic Biomarker in Noisy Data	1
A Modification of Location Commensurate Power Prior in Clinical Trials	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
Covariate-Adjusted Response Adaptive Designs for Competing Risk Survival Models	1
Evaluation of Statistical Methods for Estimating Rate Ratios for Recurrent Events in Clinical Trials	1
Beyond the Classical Type I Error: Bayesian Metrics for Bayesian Designs Using Informative Priors	1
Monitoring Overall Survival in Phase 3 Indolent Cancers: An Extended Framework and an Example	1
Assessment of Treatment Effect Heterogeneity for Multiregional Randomized Clinical Trials	1
Meta-Analysis of Moxifloxacin Concentration-QTc Effects with Application to Assay Sensitivity Assessment	1
Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints	1
Improved Trimmed Weighted Hochberg Procedures With Two Endpoints and Sample Size Optimization	1
A Statistical Method for Protocol Modifications With Heterogeneous Population Variances	1
Evaluation of Treatment Effect in Underrepresented Population in Cancer Trials: Discussion with International Regulators	1
Probability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development	1
Systematic Comparison of Bayesian Basket Trial Designs with Unequal Sample Sizes and Proposal of a New Method Based on Power Priors	1
An Accelerated Failure Time Non-Mixture Cure Model for Long-Term Survival Data with Application to Brain Cancer	1
Sample Size Calculation and Timing of Dose Selection in a Multiple-Dose Clinical Trial	1
Prognostic Covariate Adjustment in Non-Ideal Conditions: Limitations and Doubly Robust Alternatives	1
Generalized Likelihood Ratios for Designing Dose Optimization Studies of Targeted Therapies	1
Editor’s Note: Special Section on Estimands, Design and Analysis of Clinical Trials with Time-to-Event Outcomes	1
Assessing the Harm and Benefit of Cancer Therapies with an Overall Survival Endpoint in Comparative Clinical Studies	1
Rejoinder to Commentaries on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	1
Harness the Power of Statistics, Data Science, and Digital Technology to Drive Patient-Focused Innovations Together - Special Issue for the 2024 Regulatory–Industry Statistics Workshop (RISW)	1
Phase 2 Design Considerations from a Program Perspective	1
Vaccine Development during a Pandemic: General Lessons for Clinical Trial Design	1
Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes	1
Assessing Contribution of Treatment Phases through Tipping Point Analyses via Counterfactual Elicitation Using Rank Preserving Structural Failure Time Models	1
An Integrated Framework Predicting Enrollment and Interim Analysis Timing in Clinical Trials with Recurrent Count Outcomes	1
Aligning Estimands to Strengthen the Credibility of Network Meta-Analysis: Implications for Indirect Treatment Comparisons	1
Randomization-Based Inference for Clinical Trials with Missing Outcome Data	1
Bayesian Shrinkage Estimation of Credible Subgroups for Count Data with Excess Zeros	1
Conditional and Unconditional Treatment Effects in Randomized Clinical Trials: Estimands, Estimation, and Interpretation	1
Statisticians at the Forefront of Health Technology Assessment: Aligning Regulatory and HTA Evidence Through Transdisciplinary Collaboration	1
A Closer Look at the Kernels Generated by the Decision and Regression Tree Ensembles	1
SBR Special Section “Overall Survival in Oncology Trials and Drug Development”	1
Good Data Science Practice: Moving toward a Code of Practice for Drug Development (Rejoinder)	1
Qualitative versus Quantitative Treatment-by-Subgroup Interaction in Equivalence Studies with Multiple Subgroups	1
Multiple Surrogates in the Meta-Analytic Setting for Normally Distributed Endpoints	1
Incorporating Intermediate Endpoint in Two-Stage Design Decision Making	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
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
Joint TITE-CRM: A Design for Dose Finding Studies for Therapies with Late-Onset Safety and Activity Outcomes	1
An Unbiased Method to Approximate a Principal Estimand	1
Commentary on “Statistical Methodology Groups in the Pharmaceutical Industry”	1
Eliciting the Discount Parameter in a Power Prior Method on the Basis of the Type I Error Consideration	1
Bayesian Optimal Designs for Multi-Arm Multi-Stage Phase II Randomized Clinical Trials with Multiple Endpoints	1
A Bayesian Adaptive Umbrella Trial Design with Robust Information Borrowing for Screening Multiple Combination Therapies	1
Doubly Robust Estimation of Desirability of Outcome Ranking (DOOR) Probability with Application to MDRO Studies	1
Shrinkage Estimation Methods for Subgroup Analyses	1
Trial Design with Win Statistics for Multiple Time-to-Event Endpoints with Hierarchy	1
Comparison of g-Estimation Approaches for Handling Symptomatic Medication at Multiple Timepoints in Alzheimer’s Disease with a Hypothetical Strategy	1
Type I Error Control of an Adaptive Endpoint Selection Procedure	1
Evaluation of Current Statistical Methods for Implementing Quality Tolerance Limits	1
Hybrid Control Design with Commensurate Prior Constructed Using Propensity Score-Matched External Controls: A Simulation Study	1
Adjusting for Time-Varying Treatment Switches in Randomized Clinical Trials: The Danger of Extrapolation and How to Address It	1

An Expected Score Approach to Ordinal Outcomes in a Bayesian, Response Adaptive, Randomized Trial

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