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Practically Significant Method Comparison Protocols for Machine Learning in Small Molecule Drug Discovery

Jeremy R. Ash, Cas Wognum, Raquel Rodríguez-Pérez, Matteo Aldeghi, Alan C. Cheng, Djork-Arné Clevert, Ola Engkvist, Cheng Fang, Daniel Price, Jacqueline M. Hughes‐Oliver, W. Patrick Walters

2025Journal of Chemical Information and Modeling43 citationsDOIOpen Access PDF

Abstract

Machine Learning (ML) methods that relate molecular structure to properties are frequently proposed as in silico surrogates for expensive or time-consuming experiments. In small molecule drug discovery, such methods inform high-stakes decisions like compound synthesis and in vivo studies. This application lies at the intersection of multiple scientific disciplines. When comparing new ML methods to baseline or state-of-the-art approaches, statistically rigorous method comparison protocols and domain-appropriate performance metrics are essential to ensure replicability and ultimately the adoption of ML in small molecule drug discovery. This paper proposes a set of guidelines to incentivize rigorous and domain-appropriate techniques for method comparison tailored to small molecule property modeling. These guidelines, accompanied by annotated examples using open-source software tools, lay a foundation for robust ML benchmarking and thus the development of more impactful methods.

Topics & Concepts

Computer scienceIntersection (aeronautics)Drug discoveryBenchmarkingMachine learningIn silicoArtificial intelligenceSmall moleculeSet (abstract data type)Property (philosophy)Data miningTraining setChemical spaceSoftwareBaseline (sea)Protocol (science)Drug developmentComputational Drug Discovery MethodsMachine Learning in Materials ScienceStatistical Methods in Clinical Trials