15 citations · 63 across the 20 of their papers we have counts for
20 papers
Edgington's Combination Method for Two-Study Meta-Analysis: An Empirical Evaluation in 1226 Meta-Analyses
Samuel Pawel, Saverio Fontana, Jinyu Chen +6
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, rend…
Bayes Factor Group Sequential Designs
Samuel Pawel, Leonhard Held
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…
Balancing Evidentiary Value and Sample Size of Adaptive Designs with Application to Animal Experiments
Leonhard Held, Fadoua Balabdaoui, Saverio Fontana +1
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 n…
Stabilizing Thompson Sampling with Null Hypothesis Bayesian Response-Adaptive Randomization
Samuel Pawel, Leonhard Held
Response-adaptive randomization (RAR) methods can be used to adapt randomization probabilities based on accumulating data, aiming to increase the probability of allocating patients…
Combined P-value Functions for Compatible Effect Estimation and Hypothesis Testing in Drug Regulation
Samuel Pawel, Małgorzata Roos, Leonhard Held
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 f…
Assessing the replicability of RCTs in RWE emulations
Jeanette Köppe, Charlotte Micheloud, Stella Erdmann +2
Background: The standard regulatory approach to assess replication success is the two-trials rule, requiring both the original and the replication study to be significant with effe…
A comparison of combined p-value functions for meta-analysis
Leonhard Held, Felix Hofmann, Samuel Pawel
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 met…
Closed-Form Power and Sample Size Calculations for Bayes Factors
Samuel Pawel, Leonhard Held
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 a…
Mixture priors for replication studies
Roberto Macrì-Demartino, Leonardo Egidi, Leonhard Held +1
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 s…
The assessment of replicability using the sum of p-values
Leonhard Held, Samuel Pawel, Charlotte Micheloud
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 devel…
Beyond the Two-Trials Rule
Leonhard Held
The two-trials rule for drug approval requires "at least two adequate and well-controlled studies, each convincing on its own, to establish effectiveness". This is usually implemen…
Replication of "null results" -- Absence of evidence or evidence of absence?
Samuel Pawel, Rachel Heyard, Charlotte Micheloud +1
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".…
Simulating and reporting frequentist operating characteristics of clinical trials that borrow external information
Annette Kopp-Schneider, Manuel Wiesenfarth, Leonhard Held +1
Borrowing of information from historical or external data to inform inference in a current trial is an expanding field in the era of precision medicine, where trials are often perf…
Bayesian Approaches to Designing Replication Studies
Samuel Pawel, Guido Consonni, Leonhard Held
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…
Comparing Confidence Intervals for a Binomial Proportion with the Interval Score
Lisa J. Hofer, Leonhard Held
There are over 55 different ways to construct a confidence respectively credible interval (CI) for the binomial proportion. Methods to compare them are necessary to decide which sh…
Power priors for replication studies
Samuel Pawel, Frederik Aust, Leonhard Held +1
The ongoing replication crisis in science has increased interest in the methodology of replication studies. We propose a novel Bayesian analysis approach using power priors: The li…
Assessing replicability with the sceptical p-value: Type-I error control and sample size planning
Charlotte Micheloud, Fadoua Balabdaoui, Leonhard Held
We study a statistical framework for replicability based on a recently proposed quantitative measure of replication success, the sceptical -value. A recalibration is proposed to…
Normalized power priors always discount historical data
Samuel Pawel, Frederik Aust, Leonhard Held +1
Power priors are used for incorporating historical data in Bayesian analyses by taking the likelihood of the historical data raised to the power as the prior distribution for t…
The replication of equivalence studies
Charlotte Micheloud, Leonhard Held
Replication studies are increasingly conducted to assess the credibility of scientific findings. Most of these replication attempts target studies with a superiority design, but th…
Combining Evidence from Clinical Trials in Conditional or Accelerated Approval
Manja Deforth, Charlotte Micheloud, Kit C Roes +1
Conditional (European Medicines Agency) or accelerated (U.S. Food and Drug Administration) approval of drugs allow earlier access to promising new treatments that address unmet med…