arXiv subjects

Samuel Pawel

Publications and source records attributed to Samuel Pawel.

At least 19 recordsLinked to original sources

arXiv2026

Edgington's Combination Method for Two-Study Meta-Analysis: An Empirical Evaluation in 1226 Meta-Analyses

Two-study meta-analyses are common in evidence synthesis but pose major statistical challenges. With only two studies, the between-study variance cannot be reliably estimated, rendering standard random-effects methods unstable. Here, we investigate meta-analyses based on Edgington's p-value combination method as an alternative approach, applying it to 1226 two-study meta-analyses from the German Institute for Quality and Efficiency in Health Care (IQWiG). Like fixed-effect meta-analysis, Edgington's method is calibrated under homogeneity. However, it adapts confidence interval width to observed between-study discrepancy without requiring explicit heterogeneity estimation. In all of the examined meta-analyses, this leads to confidence intervals that contain both study-specific estimates but remain informative. Edgington's method agrees with fixed-effect meta-analysis on statistical significance (at two-sided $\alpha$ = 0.05) in 91% of all meta-analyses, but can give wider intervals when study results are discrepant and narrower intervals when results are highly consistent. Weighted extensions of Edgington's method shift point estimates toward the more precise study while preserving much of this adaptive behavior. We conclude that Edgington's method offers a principled and practically useful complement to existing approaches for two-study meta-analysis, occupying a middle ground between standard fixed-effect and random-effects approaches.

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arXiv2026

Extracting Bayesian Evidence from Frequentist p-Values

The $p$-value and the Bayes factor are measures of evidence that are often considered to be philosophically and mathematically incompatible: The $p$-value quantifies conflict between data and $H_0$ ("surprise"), whereas the Bayes factor quantifies the relative predictive accuracy of $H_0$ versus $H_1$ ("evidence"). We revisit Jeffreys's Approximate Bayes factor (JAB) -- a simple, largely overlooked approximation dating back to the 1930s -- which connects these two paradigms for objective hypothesis testing of the existence of an effect. Under a unit-information prior the approximation requires only the $p$-value and the effective sample size $n_\text{eff}$. We clarify the core assumptions and boundary conditions for the application of JAB and show across 704 published $t$-tests and 39 comparisons of proportions that JAB approximates objective Bayes factors remarkably well. The connection between $p$-values and JAB has a practical implication: The evidence implied by a $p$-value depends strongly on $n_\text{eff}$. Conventional verbal labels for $p$-values (e.g., "strong surprise" for .001 < $p$ < .01) correspond to similarly graded Bayes factors only around $n_\text{eff} \approx 8$; for larger samples the same $p$-value implies weaker evidence. In moderately sized to large samples, $p > .10$ can amount to moderate or even strong evidence for $H_0$. JAB offers a cheap, sample-size-sensitive supplement to $p$-values, computable from routinely reported statistics, that remains valid even under optional stopping.

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arXiv2026

Bayes Factor Group Sequential Designs

The Bayes factor, the data-based updating factor from prior to posterior odds, is a principled measure of relative evidence for two competing hypotheses. It is naturally suited to sequential data analysis in settings such as clinical trials and animal experiments, where early stopping for efficacy or futility is desirable. However, designing such studies is challenging because computing design characteristics, such as the probability of obtaining conclusive evidence or the expected sample size, typically requires computationally intensive Monte Carlo simulations, as no closed-form or efficient numerical methods exist. To address this issue, we extend results from classical group sequential design theory to sequential Bayes factor designs. The key idea is to derive Bayes factor stopping regions in terms of the z-statistic and use the known distribution of the cumulative z-statistics to compute stopping probabilities through multivariate normal integration. The resulting method is fast, accurate, and simulation-free. We illustrate it with examples from clinical trials, animal experiments, and psychological studies. We also provide an open-source implementation in the bfpwr R package. Our method makes exploring sequential Bayes factor designs as straightforward as classical group sequential designs, enabling experiments to rapidly design informative and efficient experiments.

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arXiv2025

The Bayesian optimal two-stage design for clinical phase II trials based on Bayes factors

Sequential trial design is an important statistical approach to increase the efficiency of clinical trials. Bayesian sequential trial design relies primarily on conducting a Monte Carlo simulation under the hypotheses of interest and investigating the resulting design characteristics via Monte Carlo estimates. This approach has several drawbacks, namely that replicating the calibration of a Bayesian design requires repeating a possibly complex Monte Carlo simulation. Furthermore, Monte Carlo standard errors are required to judge the reliability of the simulation. All of this is due to a lack of closed-form or numerical approaches to calibrate a Bayesian design which uses Bayes factors. In this paper, we propose the Bayesian optimal two-stage design for clinical phase II trials based on Bayes factors. The optimal two-stage Bayes factor design is a sequential clinical trial design that is built on the idea of trinomial tree branching, a method we propose to correct the resulting design characteristics for introducing a single interim analysis. We build upon this idea to invent a calibration algorithm which yields the optimal Bayesian design that minimizes the expected sample size under the null hypothesis. Examples show that our design recovers Simon's two-stage optimal design as a special case, improves upon non-sequential Bayesian design based on Bayes factors, and can be calibrated quickly, as it makes use only of standard numerical techniques instead of time-consuming Monte Carlo simulations. Furthermore, the design allows to ensure a minimum probability on compelling evidence in favour of the null hypothesis, which is not possible with other designs. As the idea of trinomial tree branching is neither dependent on the endpoint, nor on the use of Bayes factors, the design can therefore be generalized to other settings, too.

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arXiv2025

Single-Dataset Meta-Analysis For Many-Analysts And Multiverse Studies

Empirical claims often rely on one population, design, and analysis. Many-analysts, multiverse, and robustness studies expose how results can vary across plausible analytic choices. Synthesizing these results, however, is nontrivial as all results are computed from the same dataset. We introduce single-dataset meta-analysis, a weighted-likelihood approach that incorporates the information in the dataset at most once. It prevents overconfident inferences that would arise if a standard meta-analysis was applied to the data. Single-dataset meta-analysis yields meta-analytic point and interval estimates of the average effect across analytic approaches and of between-analyst heterogeneity, and can be supplied by classical and Bayesian hypothesis tests. Both the common-effect and random-effects versions of the model can be estimated by standard meta-analytic software with small input adjustments. We demonstrate the method via application to the many-analysts study on racial bias in soccer, the many-analysts study of marital status and cardiovascular disease, and the multiverse study on technology use and well-being. The results show how single-dataset meta-analysis complements the qualitative evaluation of many-analysts and multiverse studies.

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arXiv2025

Balancing Evidentiary Value and Sample Size of Adaptive Designs with Application to Animal Experiments

Reducing the number of experimental units is one of the three pillars of the 3R principles (Replace, Reduce, Refine) in animal research. At the same time, statistical error rates need to be controlled to enable reliable inferences and decisions. This paper proposes to adopt diagnostic likelihood ratios and the diagnostic odds ratio to statistical hypothesis tests and to adjust it for sample size to obtain a novel measure to quantify for the evidentiary value of one experimental unit. The experimental unit information index (EUII) is based on power, Type-I error and sample size, and has attractive interpretations both in terms of frequentist error rates and Bayesian posterior odds. We introduce the EUII in simple statistical test settings and show that its asymptotic value depends only on the assumed relative effect size under the alternative. We then extend the definition to adaptive designs where early stopping for efficacy or futility may cause reductions in sample size. Application to group-sequential designs show the usefulness of the approach when the goal is to maximize the evidentiary value of one experimental unit. A reanalysis of 2738 animal experiments with simulated results from (post-hoc) interim analyses illustrates the possible savings in sample size.

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arXiv2025

Living Synthetic Benchmarks: A Neutral and Cumulative Framework for Simulation Studies

Simulation studies are widely used to evaluate statistical methods. However, new methods are often introduced and evaluated using data-generating mechanisms (DGMs) devised by the same authors. This coupling creates misaligned incentives, e.g., the need to demonstrate the superiority of new methods, potentially compromising the neutrality of simulation studies. Furthermore, results of simulation studies are often difficult to compare due to differences in DGMs, competing methods, and performance measures. This fragmentation can lead to conflicting conclusions, hinder methodological progress, and delay the adoption of effective methods. To address these challenges, we introduce the concept of living synthetic benchmarks. The key idea is to disentangle method and simulation study development and continuously update the benchmark whenever a new DGM, method, or performance measure becomes available. This separation benefits the neutrality of method evaluation, emphasizes the development of both methods and DGMs, and enables systematic comparisons. In this paper, we outline a blueprint for building and maintaining such benchmarks, discuss the technical and organizational challenges of implementation, and demonstrate feasibility with a prototype benchmark for publication bias adjustment methods. We conclude that living synthetic benchmarks have the potential to foster neutral, reproducible, and cumulative evaluation of methods, benefiting both method developers and users.

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arXiv2025

Stabilizing Thompson Sampling with Null Hypothesis Bayesian Response-Adaptive Randomization

Response-adaptive randomization (RAR) methods can be used to adapt randomization probabilities based on accumulating data, aiming to increase the probability of allocating patients to effective treatments. A popular RAR method is Thompson sampling, which randomizes patients proportionally to the Bayesian posterior probability that each treatment is the most effective. However, its high variability can also increase the risk of assigning patients to inferior treatments and lead to inferential problems such as confidence interval undercoverage. We propose a principled method based on Bayesian hypothesis testing to address these issues: We introduce a null hypothesis postulating equal effectiveness of treatments. Bayesian model averaging then induces shrinkage toward equal randomization probabilities, with the degree of shrinkage controlled by the prior probability of the null hypothesis. Equal randomization and Thompson sampling arise as special cases when the prior probability is one or zero, respectively. A simulation study demonstrates that the method can mitigate issues with Thompson sampling and has comparable statistical properties to Thompson sampling with common ad hoc modifications such as power transformation and probability capping. Under the null hypothesis and a normal model, the randomization probabilities are shown to converge asymptotically to equal randomization, unlike those of Thompson sampling. We implement the method in the free and open-source R package brar, enabling experimenters to easily perform null hypothesis Bayesian RAR and support more effective randomization of patients.

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arXiv2025

Combined P-value Functions for Compatible Effect Estimation and Hypothesis Testing in Drug Regulation

The two-trials rule in drug regulation requires statistically significant results from two pivotal trials to demonstrate efficacy. However, it is unclear how the effect estimates from both trials should be combined to quantify the drug effect. Fixed-effect meta-analysis is commonly used but may yield confidence intervals that exclude the value of no effect even when the two-trials rule is not fulfilled. We systematically address this by recasting the two-trials rule and meta-analysis in a unified framework of combined p-value functions, where they are variants of Wilkinson's and Stouffer's combination methods, respectively. This allows us to obtain compatible combined p-values, effect estimates, and confidence intervals, which we derive in closed-form. Additionally, we provide new results for Edgington's, Fisher's, Pearson's, and Tippett's p-value combination methods. When both trials have the same true effect, all methods can consistently estimate it, although some show bias. When true effects differ, the two-trials rule and Pearson's method are conservative (converging to the less extreme effect), Fisher's and Tippett's methods are anti-conservative (converging to the more extreme effect), and Edgington's method and meta-analysis are balanced (converging to a weighted average). Notably, Edgington's confidence intervals asymptotically always include the individual trial effects, while meta-analytic confidence intervals shrink to a point at the weighted average effect. We conclude that all of these methods may be appropriate depending on the estimand of interest. We implement combined p-value function inference for two trials in the R package twotrials, allowing researchers to easily perform compatible hypothesis testing and effect estimation.

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arXiv2025

Bayesian Power and Sample Size Calculations for Bayes Factors in the Binomial Setting

Bayesian design of experiments and sample size calculations usually rely on complex Monte Carlo simulations in practice. Obtaining bounds on Bayesian notions of the false-positive rate and power therefore often lack closed-form or approximate numerical solutions. In this paper, we focus on the sample size calculation in the binomial setting via Bayes factors, the predictive updating factor from prior to posterior odds. We discuss the drawbacks of sample size calculations via Monte Carlo simulations and propose a numerical root-finding approach which allows to determine the necessary sample size to obtain prespecified bounds of Bayesian power and type-I-error rate almost instantaneously. Real-world examples and applications in clinical trials illustrate the advantage of the proposed method. We focus on point-null versus composite and directional hypothesis tests, derive the corresponding Bayes factors, and discuss relevant aspects to consider when pursuing Bayesian design of experiments with the introduced approach. In summary, our approach allows for a Bayes-frequentist compromise by providing a Bayesian analogue to a frequentist power analysis for the Bayes factor in binomial settings. A case study from a Phase II trial illustrates the utility of our approach. The methods are implemented in our R package bfpwr.

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arXiv2024

Handling Missingness, Failures, and Non-Convergence in Simulation Studies: A Review of Current Practices and Recommendations

Simulation studies are commonly used in methodological research for the empirical evaluation of data analysis methods. They generate artificial data sets under specified mechanisms and compare the performance of methods across conditions. However, simulation repetitions do not always produce valid outputs, e.g., due to non-convergence or other algorithmic failures. This phenomenon complicates the interpretation of results, especially when its occurrence differs between methods and conditions. Despite the potentially serious consequences of such "missingness", quantitative data on its prevalence and specific guidance on how to deal with it are currently limited. To this end, we reviewed 482 simulation studies published in various methodological journals and systematically assessed the prevalence and handling of missingness. We found that only 23% (111/482) of the reviewed simulation studies mention missingness, with even fewer reporting frequency (92/482 = 19%) or how it was handled (67/482 = 14%). We propose a classification of missingness and possible solutions. We give various recommendations, most notably to always quantify and report missingness, even if none was observed, to align missingness handling with study goals, and to share code and data for reproduction and reanalysis. Using a case study on publication bias adjustment methods, we illustrate common pitfalls and solutions.

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arXiv2024

A comparison of combined p-value functions for meta-analysis

P-value functions are modern statistical tools that unify effect estimation and hypothesis testing and can provide alternative point and interval estimates compared to standard meta-analysis methods, using any of the many $p$-value combination procedures available (Xie et al., 2011, JASA). We provide a systematic comparison of different combination procedures, both from a theoretical perspective and through simulation. We show that many prominent p-value combination methods (e.g. Fisher's method) are not invariant to the orientation of the underlying one-sided p-values. Only Edgington's method, a lesser-known combination method based on the sum of $p$-values, is orientation-invariant and still provides confidence intervals not restricted to be symmetric around the point estimate. Adjustments for heterogeneity can also be made and results from a simulation study indicate that Edgington's method can compete with more standard meta-analytic methods.

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arXiv2024

Closed-Form Power and Sample Size Calculations for Bayes Factors

Determining an appropriate sample size is a critical element of study design, and the method used to determine it should be consistent with the planned analysis. When the planned analysis involves Bayes factor hypothesis testing, the sample size is usually desired to ensure a sufficiently high probability of obtaining a Bayes factor indicating compelling evidence for a hypothesis, given that the hypothesis is true. In practice, Bayes factor sample size determination is typically performed using computationally intensive Monte Carlo simulation. Here, we summarize alternative approaches that enable sample size determination without simulation. We show how, under approximate normality assumptions, sample sizes can be determined numerically, and provide the R package bfpwr for this purpose. Additionally, we identify conditions under which sample sizes can even be determined in closed-form, resulting in novel, easy-to-use formulas that also help foster intuition, enable asymptotic analysis, and can also be used for hybrid Bayesian/likelihoodist design. Furthermore, we show how power and sample size can be computed without simulation for more complex analysis priors, such as Jeffreys-Zellner-Siow priors or non-local normal moment priors. Case studies from medicine and psychology illustrate how researchers can use our methods to design informative yet cost-efficient studies.

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arXiv2024

Mixture priors for replication studies

Replication of scientific studies is important for assessing the credibility of their results. However, there is no consensus on how to quantify the extent to which a replication study replicates an original result. We propose a novel Bayesian approach for replication studies based on mixture priors. The idea is to use a mixture of the posterior distribution based on the original study and a non-informative distribution as the prior for the analysis of the replication study. The mixture weight then determines the extent to which the original and replication data are pooled. Two distinct strategies are presented: one with fixed mixture weights, and one that introduces uncertainty by assigning a prior distribution to the mixture weight itself. Furthermore, it is shown how within this framework Bayes factors can be used for formal testing of relevant scientific hypotheses, such as tests on the presence or absence of an effect or whether the mixture weight equals zero (completely discounting the original data) or one (fully pooling with the original data). To showcase the practical application of the methodology, we analyze data from three replication studies. Our findings suggest that mixture priors are a valuable and intuitive alternative to other Bayesian methods for analyzing replication studies, such as hierarchical models and power priors. We provide the free and open source R package repmix that implements the proposed methodology.

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arXiv2024

A Bayes Factor Framework for Unified Parameter Estimation and Hypothesis Testing

The Bayes factor, the data-based updating factor of the prior to posterior odds of two hypotheses, is a natural measure of statistical evidence for one hypothesis over the other. We show how Bayes factors can also be used for parameter estimation. The key idea is to consider the Bayes factor as a function of the parameter value under the null hypothesis. This `support curve' is inverted to obtain point estimates (`maximum evidence estimates') and interval estimates (`support intervals'), similar to how P-value functions are inverted to obtain point estimates and confidence intervals. This provides data analysts with a unified inference framework as Bayes factors (for any tested parameter value), support intervals (at any level), and point estimates can be easily read off from a plot of the support curve. This approach shares similarities but is also distinct from conventional Bayesian and frequentist approaches: It uses the Bayesian evidence calculus, but without synthesizing data and prior, and it defines statistical evidence in terms of (integrated) likelihood ratios, but also includes a natural way for dealing with nuisance parameters. Applications to meta-analysis, replication studies, and logistic regression illustrate how our framework is of practical value for making quantitative inferences.

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arXiv2024

The assessment of replicability using the sum of p-values

Statistical significance of both the original and the replication study is a commonly used criterion to assess replication attempts, also known as the two-trials rule in drug development. However, replication studies are sometimes conducted although the original study is non-significant, in which case Type-I error rate control across both studies is no longer guaranteed. We propose an alternative method to assess replicability using the sum of p-values from the two studies. The approach provides a combined p-value and can be calibrated to control the overall Type-I error rate at the same level as the two-trials rule but allows for replication success even if the original study is non-significant. The unweighted version requires a less restrictive level of significance at replication if the original study is already convincing which facilitates sample size reductions of up to 10%. Downweighting the original study accounts for possible bias and requires a more stringent significance level and larger samples sizes at replication. Data from four large-scale replication projects are used to illustrate and compare the proposed method with the two-trials rule, meta-analysis and Fisher's combination method.

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arXiv2023

Replication of "null results" -- Absence of evidence or evidence of absence?

In several large-scale replication projects, statistically non-significant results in both the original and the replication study have been interpreted as a "replication success". Here we discuss the logical problems with this approach: Non-significance in both studies does not ensure that the studies provide evidence for the absence of an effect and "replication success" can virtually always be achieved if the sample sizes are small enough. In addition, the relevant error rates are not controlled. We show how methods, such as equivalence testing and Bayes factors, can be used to adequately quantify the evidence for the absence of an effect and how they can be applied in the replication setting. Using data from the Reproducibility Project: Cancer Biology, the Experimental Philosophy Replicability Project, and the Reproducibility Project: Psychology we illustrate that many original and replication studies with "null results" are in fact inconclusive. We conclude that it is important to also replicate studies with statistically non-significant results, but that they should be designed, analyzed, and interpreted appropriately.

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arXiv2022

Bayesian Approaches to Designing Replication Studies

Replication studies are essential for assessing the credibility of claims from original studies. A critical aspect of designing replication studies is determining their sample size; a too small sample size may lead to inconclusive studies whereas a too large sample size may waste resources that could be allocated better in other studies. Here, we show how Bayesian approaches can be used for tackling this problem. The Bayesian framework allows researchers to combine the original data and external knowledge in a design prior distribution for the underlying parameters. Based on a design prior, predictions about the replication data can be made, and the replication sample size can be chosen to ensure a sufficiently high probability of replication success. Replication success may be defined by Bayesian or non-Bayesian criteria, and different criteria may also be combined to meet distinct stakeholders and enable conclusive inferences based on multiple analysis approaches. We investigate sample size determination in the normal-normal hierarchical model where analytical results are available and traditional sample size determination is a special case where the uncertainty on parameter values is not accounted for. We use data from a multisite replication project of social-behavioral experiments to illustrate how Bayesian approaches can help design informative and cost-effective replication studies. Our methods can be used through the R package BayesRepDesign.

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