Machine learning and artificial intelligence research for patient benefit: 20 critical questions on transparency, replicability, ethics, and effectiveness

Machine learning, artificial intelligence, and other modern statistical methods are providing new opportunities to operationalise previously untapped and rapidly growing sources of data for patient benefit. Despite much promising research currently being undertaken, particularly in imaging, the literature as a whole lacks transparency, clear reporting to facilitate replicability, exploration for potential ethical concerns, and clear demonstrations of effectiveness. Among the many reasons why these problems exist, one of the most important (for which we provide a preliminary solution here) is the current lack of best practice guidance specific to machine learning and artificial intelligence. However, we believe that interdisciplinary groups pursuing research and impact projects involving machine learning and artificial intelligence for health would benefit from explicitly addressing a series of questions concerning transparency, reproducibility, ethics, and effectiveness (TREE). The 20 critical questions proposed here provide a framework for research groups to inform the design, conduct, and reporting; for editors and peer reviewers to evaluate contributions to the literature; and for patients, clinicians and policy makers to critically appraise where new findings may deliver patient benefit. Machine learning (ML), artificial intelligence (AI), and other modern statistical methods are providing new opportunities to operationalise previously untapped and rapidly growing sources of data for patient benefit. The potential uses include improving diagnostic accuracy,1 more reliably predicting prognosis,2 targeting treatments,3 and increasing the operational efficiency of health systems.4 Examples of potentially disruptive technology with early promise include image based diagnostic applications of ML/AI, which have shown the most early clinical promise (eg, deep learning based algorithms improving accuracy in diagnosing retinal pathology compared with that of specialist physicians5), or natural language processing used as a tool to extract information from structured and unstructured (that is, free) text embedded in electronic health records.2 Although we are only just …