ReviewStatistics in medicine2023
Point estimation for adaptive trial designs II: Practical considerations and guidance.
Review in Statistics in medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 22 citations in OpenAlex.
- Confidence Intervals for Adaptive Trial Designs I: A Methodological Review.Statistics in medicine · 2025Pooled it
- Adaptive versus fixed designs in confirmatory clinical trials: centering the choice on ethics.Clinical trials (London, England) · 2026Article
- Design, simulate, refine: simulation-guided clinical trials for accelerated drug development.Nature reviews. Drug discovery · 2026Review
- Conditional Estimations for Seamless Phase II/III Clinical Trials Involving Multi-Stage Early Stopping.Statistics in medicine · 2026Article
- Bayesian Estimation of the Binomial Parameter in Adaptive Designs With Treatment Selection.Statistics in medicine · 2026Article
- A visual framework for classifying adaptive design clinical trials using the GATE frame and PICO terminology.Dialogues in health · 2026Review
- Confidence intervals and point estimates for treatment effects in adaptive enrichment designs.Statistical methods in medical research · 2026Article
- Article
- Clinical Trial Simulation: Planning With the OCTAVE Framework, Implementation and Validation Principles.Statistics in medicine · 2026Article
- Bayesian adaptive trial designs for evaluating low-risk programmatic changes for quality improvement in health services: a simulation study.BMC medical research methodology · 2026Article
- Response adaptive randomisation in clinical trials: Current practice, gaps and future directions.Statistical methods in medical research · 2025Article
- Evidence-Based Approaches to Quality Improvement: A Narrative Review of Integrating Bayesian Adaptive Trials Into Health Services.Journal of evaluation in clinical practice · 2025Review
- Confidence Intervals for Adaptive Trial Designs II: Case Study and Practical Guidance.Statistics in medicine · 2025Review
- Adjustment of Conditional Bias in Hazard Ratios for Group Sequential Testing of Progression-Free Survival and Overall Survival.Statistics in medicine · 2025Article
- Point estimation for adaptive trial designs II: Practical considerations and guidance.Statistics in medicine · 2023Review
- Point estimation for adaptive trial designs I: A methodological review.Statistics in medicine · 2023Review
- Adaptive treatment allocation and selection in multi-arm clinical trials: a Bayesian perspective.BMC medical research methodology · 2022Article
- A Fast, Flexible Simulation Framework for Bayesian Adaptive Designs-The R Package BATSS.Pharmaceutical statisticsArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 5 institutions in 2 countries.
Funding
Abstract
In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is desirable to report estimates of treatment effects that reduce or remove this bias. However, it may be unclear which of the available estimators are preferable, and their use remains rare in practice. This article is the second in a two-part series that studies the issue of bias in point estimation for adaptive trials. Part I provided a methodological review of approaches to remove or reduce the potential bias in point estimation for adaptive designs. In part II, we discuss how bias can affect standard estimators and assess the negative impact this can have. We review current practice for reporting point estimates and illustrate the computation of different estimators using a real adaptive trial example (including code), which we use as a basis for a simulation study. We show that while on average the values of these estimators can be similar, for a particular trial realization they can give noticeably different values for the estimated treatment effect. Finally, we propose guidelines for researchers around the choice of estimators and the reporting of estimates following an adaptive design. The issue of bias should be considered throughout the whole lifecycle of an adaptive design, with the estimation strategy prespecified in the statistical analysis plan. When available, unbiased or bias-reduced estimates are to be preferred.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.