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Preprint . 2025
License: CC BY
Data sources: Datacite
ZENODO
Preprint . 2025
License: CC BY
Data sources: Datacite
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Machine Learning Approaches for Accelerating Drug Discovery and Reducing Clinical Trial Failures.

Authors: Sudhir, Siddharth;

Machine Learning Approaches for Accelerating Drug Discovery and Reducing Clinical Trial Failures.

Abstract

This preprint reviews and synthesizes how modern machine learning is reshaping the drug discovery and clinical development pipeline, addressing two core bottlenecks in pharma R&D: extreme cost (often cited at multi-billion USD per approved drug) and high clinical trial failure rates. The paper surveys practical ML approaches across target identification, hit discovery, lead optimization, ADMET/toxicity prediction, de novo molecular design, and clinical trial risk modeling, highlighting where specific model families fit best, including graph neural networks for molecular property prediction and transformer-based architectures for molecule generation and sequence-driven tasks. A comparative evaluation is presented with reported gains such as improved hit identification performance, faster lead optimization cycles, stronger prediction of mid-stage (Phase II) trial failures, and robust toxicity prediction (e.g., AUC > 0.85) alongside generation of novel compounds with high synthetic accessibility. The manuscript also discusses real-world limitations: data quality, bias, interpretability, privacy/proprietary constraints, and regulatory acceptance, while outlining near-term and longer-term integration directions (e.g., federated learning, digital twins, automated labs, and quantum ML).

Keywords

FOS: Computer and information sciences, Medical and health sciences, Computer and information sciences, Pharmaceutical sciences, FOS: Medical and health sciences

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
0
Average
Average
Average
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