Estimating the Sampling Distribution of Posterior Decision Summaries in Bayesian Clinical Trials
Bibliographic record
Abstract
Bayesian inference and the use of posterior or posterior predictive probabilities for decision making have become increasingly popular in clinical trials. The current practice in Bayesian clinical trials relies on a hybrid Bayesian-frequentist approach where the design and decision criteria are assessed with respect to frequentist operating characteristics such as power and type I error rate conditioning on a given set of parameters. These operating characteristics are commonly obtained via simulation studies. The utility of Bayesian measures, such as ``assurance", that incorporate uncertainty about model parameters in estimating the probabilities of various decisions in trials has been demonstrated recently. However, the computational burden remains an obstacle toward wider use of such criteria. In this article, we propose methodology which utilizes large sample theory of the posterior distribution to define parametric models for the sampling distribution of the posterior summaries used for decision making. The parameters of these models are estimated using a small number of simulation scenarios, thereby refining these models to capture the sampling distribution for small to moderate sample size. The proposed approach toward the assessment of conditional and marginal operating characteristics and sample size determination can be considered as simulation-assisted rather than simulation-based. It enables formal incorporation of uncertainty about the trial assumptions via a design prior and significantly reduces the computational burden for the design of Bayesian trials in general.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.060 | 0.248 |
| Meta-epidemiology (narrow) | 0.001 | 0.002 |
| Meta-epidemiology (broad) | 0.003 | 0.001 |
| Bibliometrics | 0.003 | 0.002 |
| Science and technology studies | 0.001 | 0.003 |
| Scholarly communication | 0.003 | 0.006 |
| Open science | 0.002 | 0.003 |
| Research integrity | 0.002 | 0.004 |
| Insufficient payload (model declined to judge) | 0.003 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".