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Frequentist performance of Bayesian inference with response-adaptive designs
Bruno Lecoutre1, Gérard Derzko, Khadija Elqasyr
1ERIS, Laboratoire de Mathématique Raphaël Salem, CNRS and Université de Rouen, Avenue de l'Université, BP 12, 76801 Saint-Etienne-du-Rouvray, France. bruno.lecoutre@univ-rouen.fr
Bayesian inference in clinical trials offers a frequentist performance comparable to traditional methods. Response-adaptive designs show similar power to 1:1 randomization, with some designs minimizing treatment failures effectively.
Area of Science:
- Clinical Trials and Biostatistics
- Statistical Inference in Healthcare
Background:
- Response-adaptive designs offer an alternative to 1:1 randomization in clinical trials, adjusting treatment assignments based on prior outcomes.
- Minimizing treatment failures is a critical objective in controlled clinical trials.
Purpose of the Study:
- To evaluate the frequentist performance of Bayesian inference for response-adaptive designs in comparing two treatments.
- To assess Bayesian inference using metrics like coverage probabilities, power, and failure count minimization.
Main Methods:
- Considered several response-adaptive designs: Play-The-Winner (PW), Randomized Play-The-Winner (RPW), Drop-The-Loser, Generalized Drop-the-Loser, and Doubly adaptive Biased Coin Designs.
- Employed Bayesian inference with two independent Beta prior distributions.
- Assessed performance using frequentist criteria: coverage probabilities, statistical power, and minimization of treatment failures.
Main Results:
- Bayesian inference demonstrated favorable frequentist performance, comparable to existing frequentist procedures.
- The power of response-adaptive designs closely matched that of 1:1 randomized designs.
- Failure count reductions were generally modest, with notable exceptions for PW and Doubly adaptive Biased Coin designs under specific success rate conditions.
Conclusions:
- Bayesian inference provides a viable and effective frequentist approach for response-adaptive clinical trial designs.
- While power is comparable to 1:1 randomization, significant failure reduction is design- and condition-specific.
- Play-The-Winner, Generalized Drop-the-Loser, and Doubly adaptive Biased Coin Designs show potential for superior performance in certain scenarios, whereas RPW was least effective.
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