Statistics in Biopharmaceutical Research

Article	Citations
Statistical Issues and Recommendations for Clinical Trials Conducted During the COVID-19 Pandemic	63
The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations	40
Machine learning for clinical trials in the era of COVID-19	38
Efficient Adaptive Designs for Clinical Trials of Interventions for COVID-19	36
The Current Landscape in Biostatistics of Real-World Data and Evidence: Causal Inference Frameworks for Study Design and Analysis	31
Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group	31
Clinical Trials Impacted by the COVID-19 Pandemic: Adaptive Designs to the Rescue?	27
Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment	24
Challenges in Assessing the Impact of the COVID-19 Pandemic on the Integrity and Interpretability of Clinical Trials	22
Assessing the Impact of COVID-19 on the Clinical Trial Objective and Analysis of Oncology Clinical Trials—Application of the Estimand Framework	22
The Hazards of Period Specific and Weighted Hazard Ratios	19
The Current Landscape in Biostatistics of Real-World Data and Evidence: Clinical Study Design and Analysis	18
Use of Nonconcurrent Common Control in Master Protocols in Oncology Trials: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion	14
Statistical Models for Composite Endpoints of Death and Nonfatal Events: A Review	12
Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event	12
The Use of a Variable Representing Compliance Improves Accuracy of Estimation of the Effect of Treatment Allocation Regardless of Discontinuation in Trials with Incomplete Follow-up	11
Developing a Targeted Learning-Based Statistical Analysis Plan	11
Targeted Learning: Toward a Future Informed by Real-World Evidence	9
Rejoinder to Letter to the Editor “The Hazards of Period Specific and Weighted Hazard Ratios”	9
Quantifying Efficiency Gains of Innovative Designs of Two-Arm Vaccine Trials for COVID-19 Using an Epidemic Simulation Model	9
Deep Learning Based Multimodal Progression Modeling for Alzheimer’s Disease	9
Efficiency Comparison of Analysis Methods for Recurrent Event and Time-to-First Event Endpoints in the Presence of Terminal Events—Application to Clinical Trials in Chronic Heart Failure	8
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development	8
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	8
Reference-Based Multiple Imputation—What is the Right Variance and How to Estimate It	8

Type I Error Considerations in Master Protocols With Common Control in Oncology Trials: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion	7
Bayesian Approaches on Borrowing Historical Data for Vaccine Efficacy Trials	7
Hypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods	7
Using Surrogate Endpoints in Adaptive Designs with Delayed Treatment Effect	7
Properties of Two While-Alive Estimands for Recurrent Events and Their Potential Estimators	7
Under a Black Cloud Glimpsing a Silver Lining: Comment on Statistical Issues and Recommendations for Clinical Trials Conducted During the COVID-19 Pandemic	7
Bayesian Optimal Phase II Design for Randomized Clinical Trials	6
Editorial: Roles of Hypothesis Testing, p-Values and Decision Making in Biopharmaceutical Research	6
A Generalized Framework of Optimal Two-Stage Designs for Exploratory Basket Trials	6
The Strong Null Hypothesis and the MaxCombo Test: Comment on “Robust Design and Analysis of Clinical Trials with Nonproportional Hazards: A Straw Man Guidance form a Cross-Pharma Working Group.”	6
A Phase 3 Trial Analysis Proposal for Mitigating the Impact of the COVID-19 Pandemic	6
An Alternative Implementation of Reference-Based Controlled Imputation Procedures	6
Bayesian Hierarchical Modeling and Biomarker Cutoff Identification in Basket Trials	6
Statistical Observations on Vaccine Clinical Development for Pandemic Diseases	6
Missing Data Imputation With Baseline Information in Longitudinal Clinical Trials	6
Optimizing Graphical Procedures for Multiplicity Control in a Confirmatory Clinical Trial via Deep Learning	6
Embedding a COVID-19 group sequential clinical trial within an ongoing trial: lessons from an unusual experience	6
Incorporating Surrogate Information for Adaptive Subgroup Enrichment Design with Sample Size Re-Estimation	5
Improvement in the Analysis of Vaccine Adverse Event Reporting System Database	5
Statistical Considerations for Sequential Analysis of the Restricted Mean Survival Time for Randomized Clinical Trials	5
Partial Correlation Coefficient for a Study With Repeated Measurements	5
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”	5
Weighted Approach for Estimating Effects in Principal Strata With Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials	5
Nonproportional Hazards—An Evaluation of the MaxCombo Test in Cancer Clinical Trials	5
Good Data Science Practice: Moving Toward a Code of Practice for Drug Development	5
Predicting the COVID-19 Pandemic Impact on Clinical Trial Recruitment	5
Statistical Opportunities to Accelerate Development for COVID-19 Therapeutics	5
Radical Thinking: Scientific Rigor and Pragmatism	4
Comment on “Statistical Issues and Recommendations for Clinical Trials Conducted During the COVID-19 Pandemic”	4
Sample Size Calculation for “Gold-Standard” Noninferiority Trials With Fixed Margins and Negative Binomial Endpoints	4
Planning for the Next Pandemic: Ethics and Innovation Today for Improved Clinical Trials Tomorrow	4
Quantifying Replicability and Consistency in Systematic Reviews	4
Statistical Challenges in the Conduct and Management of Ongoing Clinical Trials During the COVID-19 Pandemic	4
OPTIM-ARTS—An Adaptive Phase II Open Platform Trial Design With Application to a Metastatic Melanoma Study	4
Discrete Time Multistate Model With Regime Switching for Modeling COVID-19 Disease Progression and Clinical Outcomes	4
Clinical Trial Drug Safety Assessment for Studies and Submissions Impacted by COVID-19	4
Application of Group Sequential Methods to the 2-in-1 Design and Its Extensions for Interim Monitoring	4
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
It’s the Selection’s Fault—Not the p-Values’: A Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	3
On Identification of the Principal Stratum Effect in Patients Who Would Comply If Treated	3
A Comparison of Different Approaches to Bayesian Hierarchical Models in a Basket Trial to Evaluate the Benefits of Increasing Complexity	3
Mitigating Study Power Loss Caused by Clinical Trial Disruptions Due to the COVID-19 Pandemic: Leveraging External Data via Propensity Score-Integrated Approaches	3
Clinical and Statistical Perspectives on the ICH E9(R1) Estimand Framework Implementation	3
Predictive Biomarker Identification for Biopharmaceutical Development	3
Assessing via Simulation the Operating Characteristics of the WHO Scale for COVID-19 Endpoints	3
Systematic Review of Published Meta-Analyses of Vaccine Safety	3
Sample Size Allocation in Multiregional Dose-Finding Study Using MCP-Mod	3
An Improved Sample Size Calculation Method for Score Tests in Generalized Linear Models	3
Estimands in Real-World Evidence Studies	3
Generalizing Clinical Trial Results to a Target Population	3

Applying Quantitative Approaches in the Use of RWE in Clinical Development and Life-Cycle Management	3
Selecting an Optimal Design for a Non-randomized Comparative Study: A Comment on “Some Considerations on Design and Analysis Plan on a Nonrandomized Comparative Study Utilizing Propensity Score Method	3
Quick Multiple Test Procedures and p-Value Adjustments	3
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”	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	3
Our Most Important Discovery: The Question	3
Simulation-Based Pharmacokinetics Sampling Design for Evaluating Correlates of Prevention Efficacy of Passive HIV Monoclonal Antibody Prophylaxis	3
Bayesian Change-Point Joint Models for Multivariate Longitudinal and Time-to-Event Data	3
Dynamic Monitoring of Ongoing Clinical Trials	2
Least Conservative Critical Boundaries of Multiple Hypothesis Testing in a Range of Correlation Values	2
Bayesian Design of Superiority Trials: Methods and Applications	2
Clinical Trial Drug Safety Assessment With Interactive Visual Analytics	2
Examples of Applying RWE Causal-Inference Roadmap to Clinical Studies	2
Appraising Minimum Effect of Standardized Contrasts in ANCOVA Designs: Statistical Power, Sample Size, and Covariate Imbalance Considerations	2
Methods for Informative Censoring in Time-to-Event Data Analysis	2
A Comparative Study of Bayesian Optimal Interval (BOIN) Design With Interval 3 + 3 (i3 + 3) Design for Phase I Oncology Dose-Finding Trials	2
Testing the Equality of Proportions for Combined Unilateral and Bilateral Data Under Equal Intraclass Correlation Model	2
Combining the Target Trial and Estimand Frameworks to Define the Causal Estimand: An Application Using Real-World Data to Contextualize a Single-Arm Trial	2
Confidence and Prediction in Linear Mixed Models: Do Not Concatenate the Random Effects. Application in an Assay Qualification Study	2
The Impact of Major Events on Ongoing Noninferiority Trials, With Application to COVID-19	2
An Empirical Comparison of Segmented and Stochastic Linear Mixed Effects Models to Estimate Rapid Disease Progression in Longitudinal Biomarker Studies	2
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease	2
Noninferiority Clinical Trials With Binary Outcome: Statistical Methods Used in Practice	2
How Many Cohorts Should Be Considered in an Exploratory Master Protocol?	2
Statistical Inference on the Estimators of the Adherer Average Causal Effect	2
Bayesian Approaches for Handling Hypothetical Estimands in Longitudinal Clinical Trials With Gaussian Outcomes	2
Some Biostatistical Considerations About Real-World Data and Evidence in Clinical Studies, Especially for Regulatory Purposes	2
A Composite Endpoint for Treatment Benefit According to Patient Preference	2
Regulatory Guidance on Randomization and the Use of Randomization Tests in Clinical Trials: A Systematic Review	2
Novel Clinical Trial Designs and Statistical Methods in the Era of Precision Medicine	2
Testing and Interpreting the “Right” Hypothesis—Comment on “Non-proportional Hazards — An Evaluation of the MaxCombo Test in Cancer Clinical Trials”	2
Are p-values Useful to Judge the Evidence Against the Null Hypotheses in Complex Clinical Trials? A Comment on “The Role of p-values in Judging the Strength of Evidence and Realistic Rep	2
Analysis of Interval Censored Competing Risk Data via Nonparametric Multiple Imputation	2
What Can Be Achieved with the Estimand Framework?	2
An Extended Youden Design in Biological Assays	2
Combining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score	2
Multivariate Shared-Parameter Mixed-Effects Location Scale Model for Analysis of Intensive Longitudinal Data	2
Hierarchical Generalized Linear Models for Multiregional Clinical Trials	2
Post-Test Diagnostic Accuracy Measures of a Continuous Test With a Disease of Ordinal Multistages	2
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”: Is Real-World Evidence Real?	2
Waking up to p: Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	2
Bootstrap Cross-validation Improves Model Selection in Pharmacometrics	2
A Seamless Adaptive 2-in-1 Design Expanding a Phase 2 Trial for Treatment or Dose Selection Into a Phase 3 Trial	2
Statistical Inference Based on Accelerated Failure Time Models Under Model Misspecification and Small Samples	2
A Sequential Predictive Power Design for a COVID Vaccine Trial	2
Which Randomization Methods Are Used Most Frequently in Clinical Trials? Results of a Survey by the Randomization Working Group	2
A Note on the Promising Zone Approach in Adaptive Trial Design	2
Breaking the Bayesian Ice with Preclinical Discovery Biologists by Predicting Inadequate Animal Enrolment	2
Missing Data Imputation for a Multivariate Outcome of Mixed Variable Types	1
A Test of the Dependence Assumptions for the Simes-Test-based Multiple Test Procedures	1
Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	1
A Bayesian Decision-Theoretic Design for Simultaneous Biomarker-Based Subgroup Selection and Efficacy Evaluation	1
Comment on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	1
A Closer Look at the Kernels Generated by the Decision and Regression Tree Ensembles	1
Sample Size Determination for Stratified Phase II Cancer Trials With Monotone Order Constraints	1
Sequential Multiple Assignment Randomized Trials for COMparing Personalized Antibiotic StrategieS (SMART COMPASS): Design Considerations	1
A Novel Approach for Modeling Biphasic Dose–Response Curves	1
Optimal Spiking Experiment for Noninferiority of Qualitative Microbiological Methods on Accuracy With Multiple Microorganisms	1
Bayesian Borrowing Methods for Count Data: Analysis of Incontinence Episodes in Patients with Overactive Bladder	1
Comparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART)	1
Operational Experiences in China and Statistical Issues on the Conduct of Clinical Trials During the COVID-19 Pandemic	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
Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints	1
Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”	1
A Case Study of 2-Stage Seamless Adaptive Sample Size Re-Estimation Design with Efficacy Interim Analysis When Slope Is the Primary Endpoint	1
The Role of FDA CDER Statisticians in Response Efforts to the COVID-19 Pandemic	1
Statistical Considerations in Pediatric Cancer Trials: Report of American Statistical Association Biopharmaceutical Section Open Forum Discussions	1
Risk-Based Similarity Testing for Potency Assays Using MCMC Simulations	1
Randomization-Based Inference for Clinical Trials with Missing Outcome Data	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
Comment on “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
Efficient Multiple Imputation for Sensitivity Analysis of Recurrent Events Data with Informative Censoring	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
Accuracy and Safety of Novel Designs for Phase I Drug-Combination Oncology Trials	1
Three-Way Mixed Effect ANOVA to Estimate MRMC Limits of Agreement	1
A Simple Mantel–Haenszel Type Test for Noninferiority	1
Comment on “Good Data Science Practice: Moving towards a Code of Practice for Drug Development”	1
Comment on “Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group”: The Test Statistic Should Estimate Some Reasonable Meas	1
On the Implementation of Robust Meta-Analytical-Predictive Prior	1
Some Multiplicity Adjustment Procedures for Clinical Trials with Sequential Design and Multiple Endpoints	1

Statement on P-values	1
Bayesian Basket Trial Design Accounting for Multiple Cutoffs of an Ambiguous Biomarker	1
Teasing Out the Overall Survival Benefit With Adjustment for Treatment Switching to Multiple Treatments	1
Sequential Bayes Factors for Sample Size Reduction in Preclinical Experiments with Binary Outcomes	1
Linear Splines for Shelf Life Analysis of a Drug Product Stored in Hybrid Storage Conditions	1
Copula-Based Model for Incorporating Single-Agent Historical Data into Dual-Agent Phase I Cancer Trials	1
Validity of Tests for Time-to-Event Endpoints in Studies with the Pocock and Simon Covariate-Adaptive Randomization	1
The Choice of a Randomization Procedure in Survival Studies with Nonproportional Hazards	1
Statistical Considerations for Comparative Assessment of Quality Attributes	1
A Propensity-Score Integrated Approach to Bayesian Dynamic Power Prior Borrowing	1
Rejoinder to Letter to the Editor on “A New Principal Stratum Estimand Investigating the Treatment Effect in Patients Who Would Comply, If Treated With a Specific Treatment”	1
A Test for Treatment Effects Based on the Exact Distribution of an Ordinary Least-Square Estimator in Sequential Parallel Comparison Design	1
A Comparison of Randomization Methods for Multi-Arm Clinical Trials	1
DODII: Bayesian Dose Optimization Design for Randomized Phase II Trials	1
Statistical Issues and Challenges in Clinical Trials for COVID-19 Treatments, Vaccines, Medical Devices and Diagnostics	1
Comparison of Count Modeling Techniques for Estimating Environmental Monitoring Limits in Clean Rooms	1
A Flexible Analytical Framework for Reference-Based Imputation, Delta Adjustment and Tipping-Point Stress-Testing	1
Evaluation of an Adaptive Seamless Design for a Phase II/III Clinical Trial in Recurrent Events Data to Demonstrate Reduction in Number of Acute Exacerbations in Patients With Chronic Obstructive Pulm	1
Trigger Strategy in Repeated Tests on Multiple Hypotheses	1
Blinded Sample Size Re-Estimation in Comparative Clinical Trials With Overdispersed Count Data: Incorporation of Misspecification of the Variance Function	1
Current Issues in Analytical Similarity Assessment	1
Adaptive Endpoints Selection with Application in Rare Disease	1
Random Effects Models for Analyzing Mixed Overdispersed Binomial and Normal Longitudinal Responses With Application to Kidney Function Data of Cancer Patients	1
p-Values and Replicability: A Commentary on “The Role of p-Values in Judging the Strength of Evidence and Realistic Replication Expectations”	1