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Related Experiment Video

Updated: Apr 15, 2026

Evidence-based Knowledge Synthesis and Hypothesis Validation: Navigating Biomedical Knowledge Bases via Explainable AI and Agentic Systems
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Zero-shot interpretable biomedical literature appraisal with generative large language models.

Fangwen Zhou1, Muhammad Afzal2, Ashirbani Saha3

  • 1Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact, Faculty of Health Sciences, McMaster University, Hamilton, ON L8S 4K1, Canada.

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|April 14, 2026
PubMed
Summary

Generative Pre-trained Transformer (GPT) models like GPT-4o can automate randomized controlled trial (RCT) appraisal, showing performance comparable to specialized models when using full text. This AI-driven approach enhances transparency in critical appraisal.

Keywords:
GPTdeep learningevidence-based medicineexplainable AInatural language processingtext classification

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Area of Science:

  • Artificial Intelligence in Medicine
  • Biomedical Informatics
  • Clinical Trial Methodology

Background:

  • Automating the critical appraisal of randomized controlled trials (RCTs) is crucial for efficient knowledge synthesis.
  • Large language models (LLMs) offer potential for automating complex scientific text analysis.

Purpose of the Study:

  • To evaluate the performance of two decoder-based Generative Pre-trained Transformer (GPT) models (GPT-4o and GPT-3.5-mini) in automating RCT methodological appraisal.
  • To compare GPT models against a fine-tuned encoder-only BioLinkBERT model using various prompting strategies.

Main Methods:

  • A stratified random sample of 800 RCT articles was appraised.
  • Two prompting schemes were used: classifier (independent assessment) and verifier (validation of BioLinkBERT).
  • Assessments considered either title/abstract (TIAB) or full text, with performance measured against human assessments using Matthews correlation coefficient (MCC).

Main Results:

  • GPT-4o as a classifier using full text achieved an MCC of 0.429, comparable to BioLinkBERT (MCC 0.466).
  • GPT-4o as a verifier using full text showed similar performance (MCC 0.391).
  • GPT models provided transparent, criterion-specific justifications, but performance significantly decreased when using only TIAB (MCC ≤0.100).

Conclusions:

  • GPT-4o effectively automates RCT critical appraisal when full text is available, offering comparable performance to specialized models.
  • GPT models enhance interpretability and transparency through explicit justifications.
  • Fine-tuned models may complement GPTs when full texts are unavailable, and prompt optimization is key for clinical adoption.