Pharmaceutical Statistics

Article	Citations
An illness–death multistate model to implement delta adjustment and reference‐based imputation with time‐to‐event endpoints	960115767
Conditional power and information fraction calculations at an interim analysis for random coefficient models	876022323
Propensity score‐incorporated adaptive design approaches when incorporating real‐world data	858791986
Issue Information	842074418
Probability of success and group sequential designs	758329376
Assessing the quality of studies in meta‐research: Review/guidelines on the most important quality assessment tools	59
To use or not to use propensity score matching?	51
Principal stratum strategy: Potential role in drug development	33
The use of external controls: To what extent can it currently be recommended?	31
Controlling type I error rates in multi‐arm clinical trials: A case for the false discovery rate	21
A Bayesian approach in design and analysis of pediatric cancer clinical trials	20
The attributable estimand: A new approach to account for intercurrent events	18
Adjusting win statistics for dependent censoring	18
Joint hypothesis testing of the area under the receiver operating characteristic curve and the Youden index	18
Comparison of response adaptive randomization features in multiarm clinical trials with control	16
Win statistics (win ratio, win odds, and net benefit) can complement one another to show the strength of the treatment effect on time‐to‐event outcomes	16
How to choose a time zero for patients in external control arms	16
Assessing efficacy in important subgroups in confirmatory trials: An example using Bayesian dynamic borrowing	13
Bayesian optimal phase II clinical trial design with time‐to‐event endpoint	13
Improving interim decisions in randomized trials by exploiting information on short‐term endpoints and prognostic baseline covariates	12
Incorporating historical information to improve phase I clinical trials	12
Estimating and comparing adverse event probabilities in the presence of varying follow‐up times and competing events	12
Implementation of tripartite estimands using adherence causal estimators under the causal inference framework	12
Non‐proportional hazards in immuno‐oncology: Is an old perspective needed?	11
A novel equivalence probability weighted power prior for using historical control data in an adaptive clinical trial design: A comparison to standard methods	11

A critical review of graphics for subgroup analyses in clinical trials	11
Delayed treatment effects, treatment switching and heterogeneous patient populations: How to design and analyze RCTs in oncology	11
TITE‐gBOIN: Time‐to‐event Bayesian optimal interval design to accelerate dose‐finding accounting for toxicity grades	10
Defining estimands using a mix of strategies to handle intercurrent events in clinical trials	9
Standard and reference‐based conditional mean imputation	9
Estimands in practice: Bridging the gap between study objectives and statistical analysis	9
Sample size calculations for single‐arm survival studies using transformations of the Kaplan–Meier estimator	9
Decision rules for identifying combination therapies in open‐entry, randomized controlled platform trials	9
Estimands in observational studies: Some considerations beyond ICH E9 (R1)	8
Ensuring exchangeability in data‐based priors for a Bayesian analysis of clinical trials	8
Estimand framework: Are we asking the right questions? A case study in the solid tumor setting	8
The detailed clinical objectives approach to designing clinical trials and choosing estimands	8
Methodological challenges in the analysis of recurrent events for randomised controlled trials with application to cardiovascular events inLEADER	8
Flexible parametric copula modeling approaches for clustered survival data	8
A simulation‐free group sequential design with max‐combo tests in the presence of non‐proportional hazards	7
Estimands for overall survival in clinical trials with treatment switching in oncology	7
Imputation of missing covariate in randomized controlled trials with a continuous outcome: Scoping review and new results	7
Revisit of test‐then‐pool methods and some practical considerations	7
Comparison of statistical methods for recurrent event analysis using pediatrics asthma data	6
Improving the assessment of the probability of success in late stage drug development	6
Assessing and communicating heterogeneity of treatment effects for patient subpopulations: The hardest problem there is	6
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	6
Utilizing restricted mean duration of response for efficacy evaluation of cancer treatments	6
A weighted log‐rank test and associated effect estimator for cancer trials with delayed treatment effect	6
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials	6
Estimation of treatment effects in short‐term depression studies. An evaluation based on the ICH E9(R1) estimands framework	6
A Bayesian basket trial design accounting for uncertainties of homogeneity and heterogeneity of treatment effect among subpopulations	6
Estimands in hematologic oncology trials	6
Optimising the trade‐off between type I and II error rates in the Bayesian context	6
Prior distributions for variance parameters in a sparse‐event meta‐analysis of a few small trials	6
A robust permutation test for the concordance correlation coefficient	5
The power prior with multiple historical controls for the linear regression model	5
Assurance for clinical trial design with normally distributed outcomes: Eliciting uncertainty about variances	5
TSNP: A two‐stage nonparametric phase I/II clinical trial design for immunotherapy	5
Comparing Bayesian early stopping boundaries for phase II clinical trials	5
Critical appraisal of Bayesian dynamic borrowing from an imperfectly commensurate historical control	5
Progression‐free survival in oncological clinical studies: Assessment time bias and methods for its correction	5
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	5
Estimands and inference in cluster‐randomized vaccine trials	5
Return‐to‐baseline multiple imputation for missing values in clinical trials	4
MMRM vs joint modeling of longitudinal responses and time to study drug discontinuation in clinical trials using a “de jure” estimand	4
Generalized phase I‐II designs to increase long term therapeutic success rate	4
Conditional assurance: the answer to the questions that should be asked within drug development	4
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	4
Selection bias for treatments with positive Phase 2 results	4
Assessing safety at the end of clinical trials using system organ classes: A case and comparative study	4
A method for sample size calculation via E‐value in the planning of observational studies	4
Propensity score‐based methods for causal inference and external data leveraging in regulatory settings: From basic ideas to implementation	4
Estimating survival parameters under conditionally independent left truncation	4
Recurrent time‐to‐event models with ordinal outcomes	4

Eliciting judgements about dependent quantities of interest: The SHeffield ELicitation Framework extension and copula methods illustrated using an asthma case study	4
Operational characteristics of generalized pairwise comparisons for hierarchically ordered endpoints	4
Bayesian MCPMod	4
SCI: A Bayesian adaptive phase I/II dose‐finding design accounting for semi‐competing risks outcomes for immunotherapy trials	4
Left truncation in linked data: A practical guide to understanding left truncation and applying it using SAS and R	4
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Bayesian optimal phase II designs with dual‐criterion decision making	4
Comments on “Properties of the weighted log‐rank test in the design of confirmatory studies with delayed effects” by Jose Jimenez, Viktoriya Stalbovskaya, and Byron Jones. Pharm Stat. 18:287‐303, 2019	3
Incorporating historical two‐arm data in clinical trials with binary outcome: A practical approach	3
Encouragement of subgroup assessment by the FDA	3
Some design considerations incorporating early futility for single‐arm clinical trials with time‐to‐event primary endpoints using Weibull distribution	3
Bayesiansingle‐armphaseIItrial designs withtime‐to‐eventendpoints	3
Bayesian optimization design for dose‐finding based on toxicity and efficacy outcomes in phase I/II clinical trials	3
Parametric and nonparametric methods for confidence intervals and sample size planning for win probability in parallel‐group randomized trials with Likert item and Likert scale data	3
Sample size calculation for logrank test and prediction of number of events over time	3
Burden‐of‐illness vaccine efficacy	3
Response‐adaptive trial designs with accelerated Thompson sampling	3
Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab Phase 2 bioequivalence study	3
Bayesian accrual modeling and prediction in multicenter clinical trials with varying center activation times	3
Assessing goodness‐of‐fit for evaluation of dose‐proportionality	3
Combination of several matching adjusted indirect comparisons (MAICs) with an application in psoriasis	3
Identifying treatment effects using trimmed means when data are missing not at random	3
Adjusting for bias in the mean for primary and secondary outcomes when trials are in sequence	3
Bayesian adaptive randomization design incorporating propensity score‐matched historical controls	3
Potential impact of COVID‐19 on ongoing clinical trials: a simulation study with the neurological Yale Global Tic Severity Scale based on the CANNA‐TICS study	3
Novel concentration‐QTc models for early clinical studies with parallel placebo controls: A simulation study	3
A dose‐finding design for dual‐agent trials with patient‐specific doses for one agent with application to an opiate detoxification trial	3
The promise and pitfalls of composite endpoints in sepsis and COVID‐19 clinical trials	3
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	2
A simulation‐based comparison of estimation methods for adaptive and classical group sequential clinical trials	2
Adjusting for covariates in analysis based on restricted mean survival times	2
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Evaluation of alternative confidence intervals to address non‐inferiority through the stratified difference between proportions	2
Resampling‐based stepwise multiple testing procedures with applications to clinical trial data	2
A natural lead‐in approach to response‐adaptive allocation for continuous outcomes	2
On kernel machine learning for propensity score estimation under complex confounding structures	2
Joint analysis of longitudinal measurements and survival times with a cure fraction based on partly linear mixed and semiparametric cure models	2
Improving early phase oncology clinical trial design: A case study	2
Statistical methods for handling missing data to align with treatment policy strategy	2
Should responder analyses be conducted on continuous outcomes?	2
Doubly‐robust methods for differences in restricted mean lifetimes using pseudo‐observations	2
A two‐stage adaptive clinical trial design with data‐driven subgroup identification at interim analysis	2
A comparison of reweighting estimators of average treatment effects in real world populations	2
An adaptive biomarker basket design in phase II oncology trials	2
Kernel hazard estimation for visualisation of the effect of a continuous covariate on time‐to‐event endpoints	2
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	2
The current state of Bayesian methods in nonclinical pharmaceutical statistics: Survey results and recommendations from the DIA/ASA‐BIOP Nonclinical Bayesian Working Group	2
Selection bias, investment decisions and treatment effect distributions	2
Incorporating covariates information in adaptive clinical trials for precision medicine	2
A Bayesian phase I/II design to determine subgroup‐specific optimal dose for immunotherapy sequentially combined with radiotherapy	2
Joint confidence region estimation on predictive values	2
Evaluating the time‐dependent predictive accuracy for event‐to‐time outcome with a cure fraction	2
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	2
An application of the mixed‐effects model and pattern mixture model to treatment groups with differential missingness suspected not‐missing‐at‐random	2
Flexible diagnostic measures and new cut‐point selection methods under multiple ordered classes	2
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	2
Propensity score matching and stratification using multiparty data without pooling	2
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	2
Natural cubic splines for the analysis of Alzheimer's clinical trials	2
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	2
The stratified win statistics (win ratio, win odds, and net benefit)	2
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	2
Heterogeneity in treatment effects across diverse populations	2
Using recurrent time‐to‐event models with multinomial outcomes to generate toxicity profiles	2
Score and deviance residuals based on the full likelihood approach in survival analysis	2
The use of local and nonlocal priors in Bayesian test‐based monitoring for single‐arm phase II clinical trials	2
Covariate handling approaches in combination with dynamic borrowing for hybrid control studies	2
Reflecting on Andy Grieve's influence and innovation: A personal perspective	2
Comparison of two treatments in the presence of competing risks	2
Empirical profile Bayesian estimation for extrapolation of historical adult data to pediatric drug development	1
Estimation of multivariate treatment effects in contaminated clinical trials	1
Extending the two‐stage single arm phase II clinical trial design to the delayed response scenario	1
Adaptive designs for IVPT data with mixed scaled average bioequivalence	1
Stability analysis using mixed models: A critique of tolerance interval methods and a probabilistic solution	1
A pragmatic adaptive enrichment design for selecting the right target population for cancer immunotherapies	1
Analysis of an incomplete binary outcome dichotomized from an underlying continuous variable in clinical trials	1
The design and analysis of vaccine trials for COVID‐19 for the purpose of estimating efficacy	1
Effects of duration of follow‐up and lag in data collection on the performance of adaptive clinical trials	1
A stochastically curtailed two‐arm randomised phase II trial design for binary outcomes	1
A meta‐analytic framework to adjust for bias in external control studies	1

Geometric approaches to assessing the numerical feasibility for conducting matching‐adjusted indirect comparisons	1
Identifying treatment effect heterogeneity in dose‐finding trials using Bayesian hierarchical models	1
A Bayesian adaptive design for biosimilar trials with time‐to‐event endpoint	1
The individual‐level surrogate threshold effect in a causal‐inference setting with normally distributed endpoints	1
A treatment‐specific marginal structural Cox model for the effect of treatment discontinuation	1
Improving early phase oncology clinical trial design: An opportunity for statisticians	1
Treatment effect measures under nonproportional hazards	1
Sample size re‐estimation for response‐adaptive randomized clinical trials	1
Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long‐term clinical endpoint	1
The utilities and pitfalls of stratified analysis in challenging situations	1
Enrollment forecast for clinical trials at the portfolio planning phase based on site‐level historical data	1
Bayesian hierarchical models for adaptive basket trial designs	1
From innovative thinking to pharmaceutical industry implementation: Some success stories	1
Bayesian adaptive linearization method for phase I drug combination trials with dimension reduction	1
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Confidence intervals for exposure‐adjusted rate differences in randomized trials	1
The importance of using ordinal scores for patient classification based on health‐related quality of life trajectories	1
“Super‐covariates”: Using predicted control group outcome as a covariate in randomized clinical trials	1
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	1
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Practical and robust test for comparing binomial proportions in the randomized phase II setting	1
Regulatory issues and the potential use of Bayesian approaches for early drug approval systems in Japan	1
Re‐randomization tests as sensitivity analyses to confirm immunological noninferiority of an investigational vaccine: Case study	1
A semi‐mechanistic dose‐finding design in oncology using pharmacokinetic/pharmacodynamic modeling	1
Utilizing Bayesian predictive power in clinical trial design	1
Statistical considerations in clinical trial design with event‐free survival as the primary efficacy endpoint	1
Setting the control limit at release for stability assurance	1
Statistical detection of synergy: New methods and a comparative study	1
Multiple imputation analysis of Miettinen‐Nurminen interval for difference in proportions	1
Should the two‐trial paradigm still be the gold standard in drug assessment?	1
Logistic retainment interval dose exploration design for Phase I clinical trials of cytotoxic agents	1
Sample size calculation for recurrent event data with additive rates models	1
Pharmacokinetic/pharmacodynamic data extrapolation models for improved pediatric efficacy and toxicity estimation, with application to secondary hyperparathyroidism	1
Two‐stage subgroup‐specific time‐to‐event (2S‐Sub‐TITE): An adaptive two‐stage time‐to‐toxicity design for subgroup‐specific dose finding in phase I oncology trials	1
Obituary: Sir David Cox	1
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	1
Cancer immunotherapy trial design with long‐term survivors	1
Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	1
Dose intra‐subject escalation to an event (DIETE): A new method for phase 1 dose‐finding utilizing systematic intra‐subject dose escalation with application to T‐cell engagers	1
Simulation‐based sequential design	1
AIDE: Adaptive intrapatient dose escalation designs to accelerate Phase I clinical trials	1
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	1
A note on confidence intervals for the restricted mean survival time based on transformations in small sample size	1
Statistical modeling approaches for the comparison of dissolution profiles	1
Power and sample size for GEE analysis of incomplete paired outcomes in 2 × 2 crossover trials	1
Empirical likelihood confidence interval for sensitivity to the early disease stage	1
Estimating the treatment effect for adherers using multiple imputation	1
Designing historical control studies with survival endpoints using exact statistical inference	1
Replenishing the pipeline: A quantitative approach to optimising the sourcing of new projects	1
A model selection criterion for clustered survival analysis with informative cluster size	1
Bivariate Bayesian hypothesis testing with missing data in components	1
Designing and analyzing clinical trials for personalized medicine via Bayesian models	1
Separation and the information theory surrogate evaluation approach: A penalised likelihood solution	1
Using marginal structural models to analyze the impact of subsequent therapy on the treatment effect in survival data: Simulations and clinical trial examples	1
Treatment allocation strategies for umbrella trials in the presence of multiple biomarkers: A comparison of methods	1