Back

LLM4TOP: An End-to-End framework based on Large Language Model for trial outcome prediction

Qian, L.; Lu, X.; Parvez, H.; Zhu, J.; Li, S.; Yang, Y.

2025-07-07 health informatics
10.1101/2025.07.06.25330956 medRxiv
Show abstract

Clinical trial outcome prediction involves estimating the probability of a trial successfully achieving its predefined endpoints. Current approaches primarily employ machine learning techniques that integrate diverse data modalities, including trial protocol descriptions, molecular structures of investigational drugs, and characteristics of target diseases. However, this field faces several critical challenges that hinder practical implementation. The preprocessing of heterogeneous clinical trial data requires extensive and complex transformation pipelines. Different data modalities demand specialized modeling architectures, complicating the development of unified prediction systems. Furthermore, the absence of privacy-preserving large language models capable of local deployment presents a significant barrier to clinical adoption, particularly given the sensitive nature of medical data. Addressing these challenges is essential for advancing reliable and clinically applicable prediction models. In the present study, we propose llm4top(large language model for trial outcome prediction) which is a framework designed specifically to streamline and standardize the process of Clinical trial outcome prediction using large language model based on Qwen3 [1]. By redefining the task of predicting clinical trial outcomes as a binary classification issue, the complete capabilities of large language models with 8B parameters are efficiently utilized. Findings reveal that the method attains a substantially higher degree of accuracy and precision in predicting clinical trial outcomes. The research underscores the automating and simplifies the complex process work and potential of large language models to significantly enhance the predictive accuracy of clinical trial outcomes, thereby facilitating more effective research efforts, drug development processes, and patient care strategies.

Matching journals

The top 5 journals account for 50% of the predicted probability mass.

50% of probability mass above

"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.