Development and validation of a generative AI-assisted medication-indication knowledge base

Abstract

Abstract Background Existing information resources about medicines and their indications have limited usefulness for health data analytics. The emerging potential of large language models (LLMs) to generate clinically accurate responses presents a novel opportunity to develop a comprehensive knowledge base of medicines and their clinical indications. Method Unique medications from the English Prescribing Dataset (EPD) were extracted and included in a fine-tuned prompt pipeline using the GPT-4 and MedCAT LLMs. The resulting database underwent clinical validation by three clinicians to calculate the precision for a sample of the knowledge base. This was followed by external validation using the participant reported indications included in the National Health and Nutrition Examination Survey (NHANES) dataset, a large-scale population health survey in the United States (US). Results 1,540 unique medications from the EPD were used to generate 10,853 unique medication-indication pairs. Initial threshold-based investigations of accuracy across LLM generated confidence scores for each pair revealed that a threshold of 0.75 was an optimal trade-off between error rate and knowledge base size. Common types of error which had to be addressed in the pipeline included duplications, alternative spellings and medical synonyms. A random subset of 465 medication-indication pairs was selected for clinical validation, of which 418 were assessed to be correct (a precision of 89.9%). External validation using the NHANES overall agreement of 84% (210 out of 250). Of the remaining 40, only 2 were valid indications omitted in the knowledge base, whilst the rest were judged by clinical reviewers to be off-licence indications (n=7) or appeared to be incorrectly reported by survey participants (n=31). Conclusion The AI-assisted medication-indication knowledge base demonstrated high precision and external validity. The coverage of off-licence indications adds to the potential of this knowledge base in various biomedical applications such as real-world evidence research, drug discovery and adverse event detection.

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