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 2020-05-01 to 2024-05-01.)

Article	Citations
Assessing the quality of studies in meta‐research: Review/guidelines on the most important quality assessment tools	71
To use or not to use propensity score matching?	54
The use of external controls: To what extent can it currently be recommended?	35
Principal stratum strategy: Potential role in drug development	34
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	21
Joint hypothesis testing of the area under the receiver operating characteristic curve and the Youden index	19
Adjusting win statistics for dependent censoring	18
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	17
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	14
Non‐proportional hazards in immuno‐oncology: Is an old perspective needed?	13
Estimating and comparing adverse event probabilities in the presence of varying follow‐up times and competing events	13
Standard and reference‐based conditional mean imputation	13
Bayesian optimal phase II clinical trial design with time‐to‐event endpoint	13
Delayed treatment effects, treatment switching and heterogeneous patient populations: How to design and analyze RCTs in oncology	13
Implementation of tripartite estimands using adherence causal estimators under the causal inference framework	12
Incorporating historical information to improve phase I clinical trials	12
Decision rules for identifying combination therapies in open‐entry, randomized controlled platform trials	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
TITE‐gBOIN: Time‐to‐event Bayesian optimal interval design to accelerate dose‐finding accounting for toxicity grades	11
Estimands in observational studies: Some considerations beyond ICH E9 (R1)	9
Estimand framework: Are we asking the right questions? A case study in the solid tumor setting	9
Defining estimands using a mix of strategies to handle intercurrent events in clinical trials	9
Sample size calculations for single‐arm survival studies using transformations of the Kaplan–Meier estimator	9

Estimands for overall survival in clinical trials with treatment switching in oncology	9
Ensuring exchangeability in data‐based priors for a Bayesian analysis of clinical trials	9
Methodological challenges in the analysis of recurrent events for randomised controlled trials with application to cardiovascular events inLEADER	9
Estimands in practice: Bridging the gap between study objectives and statistical analysis	9
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	8
Estimands in hematologic oncology trials	8
The detailed clinical objectives approach to designing clinical trials and choosing estimands	8
Imputation of missing covariate in randomized controlled trials with a continuous outcome: Scoping review and new results	7
Optimising the trade‐off between type I and II error rates in the Bayesian context	7
Prior distributions for variance parameters in a sparse‐event meta‐analysis of a few small trials	7
Estimation of treatment effects in short‐term depression studies. An evaluation based on the ICH E9(R1) estimands framework	7
Comparison of statistical methods for recurrent event analysis using pediatrics asthma data	6
Comparing Bayesian early stopping boundaries for phase II clinical trials	6
Counterfactual mediation analysis in the multistate model framework for surrogate and clinical time‐to‐event outcomes in randomized controlled trials	6
Assessing and communicating heterogeneity of treatment effects for patient subpopulations: The hardest problem there is	6
A Bayesian basket trial design accounting for uncertainties of homogeneity and heterogeneity of treatment effect among subpopulations	6
A weighted log‐rank test and associated effect estimator for cancer trials with delayed treatment effect	6
The power prior with multiple historical controls for the linear regression model	6
Estimands and inference in cluster‐randomized vaccine trials	6
Utilizing restricted mean duration of response for efficacy evaluation of cancer treatments	6
Covariate handling approaches in combination with dynamic borrowing for hybrid control studies	6
Improving the assessment of the probability of success in late stage drug development	6
Informed decision‐making: Statistical methodology for surrogacy evaluation and its role in licensing and reimbursement assessments	6
A robust permutation test for the concordance correlation coefficient	5
Estimating survival parameters under conditionally independent left truncation	5
An adaptive design for early clinical development including interim decision for single‐arm trial with external controls or randomized trial	5
TSNP: A two‐stage nonparametric phase I/II clinical trial design for immunotherapy	5
Left truncation in linked data: A practical guide to understanding left truncation and applying it using SAS and R	5
Assurance for clinical trial design with normally distributed outcomes: Eliciting uncertainty about variances	5
Progression‐free survival in oncological clinical studies: Assessment time bias and methods for its correction	5
Incorporating historical information to improve dose optimization design with toxicity and efficacy endpoints: iBOIN‐ET	5
Bayesian optimal phase II designs with dual‐criterion decision making	5
Operational characteristics of generalized pairwise comparisons for hierarchically ordered endpoints	4
Bayesian MCPMod	4
Conditional assurance: the answer to the questions that should be asked within drug development	4
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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
SCI: A Bayesian adaptive phase I/II dose‐finding design accounting for semi‐competing risks outcomes for immunotherapy trials	4
Sample size calculation for logrank test and prediction of number of events over time	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
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
Assessing goodness‐of‐fit for evaluation of dose‐proportionality	3
The promise and pitfalls of composite endpoints in sepsis and COVID‐19 clinical trials	3
Response‐adaptive trial designs with accelerated Thompson sampling	3

Identifying treatment effects using trimmed means when data are missing not at random	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
Bayesian optimization design for dose‐finding based on toxicity and efficacy outcomes in phase I/II clinical trials	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
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
Adjusting for bias in the mean for primary and secondary outcomes when trials are in sequence	3
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	3
Score and deviance residuals based on the full likelihood approach in survival analysis	3
Statistical methodology for highly variable compounds: A novel design approach for the ofatumumab Phase 2 bioequivalence study	3
Statistical methods for handling missing data to align with treatment policy strategy	3
Incorporating covariates information in adaptive clinical trials for precision medicine	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
On kernel machine learning for propensity score estimation under complex confounding structures	2
Estimators for handling COVID‐19‐related intercurrent events with a hypothetical strategy	2
Using recurrent time‐to‐event models with multinomial outcomes to generate toxicity profiles	2
Improving early phase oncology clinical trial design: A case study	2
Should the two‐trial paradigm still be the gold standard in drug assessment?	2
An application of the mixed‐effects model and pattern mixture model to treatment groups with differential missingness suspected not‐missing‐at‐random	2
Win ratio approach for analyzing composite time‐to‐event endpoint with opposite treatment effects in its components	2
Quantification of follow‐up time in oncology clinical trials with a time‐to‐event endpoint: Asking the right questions	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
A two‐stage adaptive clinical trial design with data‐driven subgroup identification at interim analysis	2
Propensity score matching and stratification using multiparty data without pooling	2
Statistical modeling approaches for the comparison of dissolution profiles	2
The stratified win statistics (win ratio, win odds, and net benefit)	2
Incorporating historical controls in clinical trials with longitudinal outcomes using the modified power prior	2
Heterogeneity in treatment effects across diverse populations	2
Evaluation of alternative confidence intervals to address non‐inferiority through the stratified difference between proportions	2
The use of local and nonlocal priors in Bayesian test‐based monitoring for single‐arm phase II clinical trials	2
A natural lead‐in approach to response‐adaptive allocation for continuous outcomes	2
Joint analysis of longitudinal measurements and survival times with a cure fraction based on partly linear mixed and semiparametric cure models	2
Evaluating the time‐dependent predictive accuracy for event‐to‐time outcome with a cure fraction	2
Reflecting on Andy Grieve's influence and innovation: A personal perspective	2
Defining estimands for efficacy assessment in single arm phase 1b or phase 2 clinical trials in oncology early development	2
A MCP‐Mod approach to designing and analyzing survival trials with potential non‐proportional hazards	2
Vaccine clinical trials with dynamic borrowing of historical controls: Two retrospective studies	2
A simulation‐based comparison of estimation methods for adaptive and classical group sequential clinical trials	2
Comparison of two treatments in the presence of competing risks	2
Natural cubic splines for the analysis of Alzheimer's clinical trials	2
An adaptive biomarker basket design in phase II oncology trials	2
Resampling‐based stepwise multiple testing procedures with applications to clinical trial data	2
Adjusting for covariates in analysis based on restricted mean survival times	2
Statistical detection of synergy: New methods and a comparative study	2
Selection bias, investment decisions and treatment effect distributions	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
Treatment effect measures under nonproportional hazards	2
Should responder analyses be conducted on continuous outcomes?	2
Flexible diagnostic measures and new cut‐point selection methods under multiple ordered classes	2
Doubly‐robust methods for differences in restricted mean lifetimes using pseudo‐observations	2
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Estimation of a treatment policy estimand for time to event data using data collected post discontinuation of randomised treatment	2
A comparison of reweighting estimators of average treatment effects in real world populations	2
Empirical likelihood confidence interval for sensitivity to the early disease stage	2
Kernel hazard estimation for visualisation of the effect of a continuous covariate on time‐to‐event endpoints	2
Semi‐parametric accelerated failure‐time model: A useful alternative to the proportional‐hazards model in cancer clinical trials	2