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Machine learning (ML) applications have received extensive attention in endocrinology research during the last decade. This review summarizes the basic concepts of ML and certain research topics in endocrinology and metabolism where ML principles have been actively deployed. Relevant studies are discussed to provide an overview of the methodology, main findings, and limitations of ML, with the goal of stimulating insights into future research directions. Clear, testable study hypotheses stem from unmet clinical needs, and the management of data quality (beyond a focus on quantity alone), open collaboration between clinical experts and ML engineers, the development of interpretable high-performance ML models beyond the black-box nature of some algorithms, and a creative environment are the core prerequisites for the foreseeable changes expected to be brought about by ML and artificial intelligence in the field of endocrinology and metabolism, with actual improvements in clinical practice beyond hype. Of note, endocrinologists will continue to play a central role in these developments as domain experts who can properly generate, refine, analyze, and interpret data with a combination of clinical expertise and scientific rigor.

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Most people are now familiar with the concepts of big data, deep learning, machine learning, and artificial intelligence (AI) and have a vague expectation that AI using medical big data can be used to improve the quality of medical care. However, the expectation that big data could change the field of medicine is inconsistent with the current reality. The clinical meaningfulness of the results of research using medical big data needs to be examined. Medical staff needs to be clear about the purpose of AI that utilizes medical big data and to focus on the quality of this data, rather than the quantity. Further, medical professionals should understand the necessary precautions for using medical big data, as well as its advantages. No doubt that someday, medical big data will play an essential role in healthcare; however, at present, it seems too early to actively use it in clinical practice. The field continues to work toward developing medical big data and making it appropriate for healthcare. Researchers should continue to engage in empirical research to ensure that appropriate processes are in place to empirically evaluate the results of its use in healthcare.

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