Large Language Models for Clinical Trial Protocol Assessments.

ABSTRACT: The purpose was to evaluate the utility of large language models (LLMs) for reviewing the statistical analysis plan (SAP) and pharmacokinetics-pharmacodynamics (PK-PD) components of clinical trial protocols. Clinical trial protocols and SAPs were obtained from clinicaltrials.gov for a testbed of 15 small-molecule drugs, biologics, and global antibiotic and public health interventions. The GPT-4o (ChatGPT) LLM was used to elicit study design attributes, relevant guidelines, and detailed SAP evaluations with prompts engineered to the persona of a regulatory expert. The SAP methodology was assessed against the Food and Drug Administration's (FDA) E9 Statistical Principles for Clinical Trials guidance. The SAP evaluation outputs were assessed in post hoc analyses with ChatGPT and Grok, based o

SUBMITTER: Shin E 

PROVIDER: S-EPMC12816432 | biostudies-literature | 2026 Feb

REPOSITORIES: biostudies-literature