Adaptive design strategies for ethical and efficient clinical trials using simulated annealing

We introduce a flexible adaptive design strategy for treatment allocation in clinical trials based on a randomized simulated annealing algorithm. The proposed approach provides a unified and modular framework for implementing a broad range of adaptive randomization objectives, including covariate-adaptive (CA), response-adaptive (RA), covariate-adjusted response-adaptive (CARA), and hybrid RA+CA and CARA+CA designs. The procedure accommodates multiple treatments, different outcome types, and any baseline covariate information available. Its implementation is compatible with both parametric and nonparametric predictive models; in this work, we use Bayesian additive regression trees to capture complex covariate-response relationships without committing to a low-dimensional parametric specification. The allocation rule combines a simulated-annealing-based recommendation with a fallback randomization step used to ensure positive assignment probabilities. In a finite stratified setting, we establish strong consistency of the resulting stratified sample mean estimators. Extensive simulation studies with homogeneous and heterogeneous treatment effects show that our proposal achieves a favorable trade-off between ethical allocation and statistical efficiency. A redesign of the ACTG 175 clinical trial further illustrates the practical advantages and flexibility of the proposed framework.

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Publication Details

Journal
Statistical Methods in Medical Research
Published
2026-09-14
DOI
https://doi.org/10.1177/09622802261488058
Primary Topic
Statistical Methods in Clinical Trials
Type
article
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article

Adaptive design strategies for ethical and efficient clinical trials using simulated annealing

Francesco Mariani, Marco Novelli, Rosamarie Frieri
Statistical Methods in Medical Research
Statistical Methods in Clinical Trials
article

Adaptive design strategies for ethical and efficient clinical trials using simulated annealing

Francesco Mariani, Marco Novelli, Rosamarie Frieri
article en

Abstract

We introduce a flexible adaptive design strategy for treatment allocation in clinical trials based on a randomized simulated annealing algorithm. The proposed approach provides a unified and modular framework for implementing a broad range of adaptive randomization objectives, including covariate-adaptive (CA), response-adaptive (RA), covariate-adjusted response-adaptive (CARA), and hybrid RA+CA and CARA+CA designs. The procedure accommodates multiple treatments, different outcome types, and any baseline covariate information available. Its implementation is compatible with both parametric and nonparametric predictive models; in this work, we use Bayesian additive regression trees to capture complex covariate-response relationships without committing to a low-dimensional parametric specification. The allocation rule combines a simulated-annealing-based recommendation with a fallback randomization step used to ensure positive assignment probabilities. In a finite stratified setting, we establish strong consistency of the resulting stratified sample mean estimators. Extensive simulation studies with homogeneous and heterogeneous treatment effects show that our proposal achieves a favorable trade-off between ethical allocation and statistical efficiency. A redesign of the ACTG 175 clinical trial further illustrates the practical advantages and flexibility of the proposed framework.

Statistical Methods in Medical Research
University of Bologna (IT)
Partnerships for the goals
Openalex Percentile: Top 8%
Statistical Methods in Clinical Trials
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Adaptive design strategies for ethical and efficient clinical trials using simulated annealing — Francesco Mariani, Marco Novelli, et al. · Statistical Methods in Medical Research (2026) | TGRS Research Map | TGRS