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How many patients could we save with LLM priors?

Created by
  • Haebom

Author

Shota Arai, David Selby, Andrew Vargo, Sebastian Vollmer

Outline

We present a novel framework for hierarchical Bayesian modeling of adverse events in multicenter clinical trials, leveraging large-scale language models (LLMs). Unlike data augmentation approaches that generate synthetic data points, this study derives parametric prior distributions directly from the model. Using pre-trained LLMs, we systematically derive informative prior distributions for the hyperparameters of the hierarchical Bayesian model, directly incorporating external clinical expertise into Bayesian safety modeling. Comprehensive temperature sensitivity analyses and rigorous cross-validation on real-world clinical trial data demonstrate that LLM-derived prior distributions consistently improve predictive performance compared to existing meta-analytic approaches. This methodology paves the way for more efficient and expert-informed clinical trial design, significantly reducing the number of patients required to achieve robust safety assessments and potentially transforming drug safety monitoring and regulatory decision-making.

Takeaways, Limitations

Takeaways:
Leveraging the LLM to present new possibilities for reducing the number of patients in clinical trials and increasing efficiency.
Development of an LLM-based Bayesian safety modeling framework that demonstrates improved predictive performance over existing meta-analyses.
Contribute to improving drug safety monitoring and regulatory decision-making processes.
Presenting ways to effectively integrate external expertise.
Limitations:
Dependence on the performance of the LLM. LLM bias or errors may affect the results.
Further research is needed to determine generalizability to real-world clinical settings.
There is a need to ensure transparency and explainability in the prior distribution extraction process using LLM.
Generalizability to various types of adverse events and clinical trial designs needs to be verified.
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