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Can preclinical drug development help to predict adverse events in clinical trials?

Chi LH, Burrows AD, Anderson RL

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  • Journal Drug discovery today

  • Published 29 Aug 2021

  • Volume 27

  • ISSUE 1

  • Pagination 257-268

  • DOI 10.1016/j.drudis.2021.08.010

Abstract

The development of novel therapeutics is associated with high rates of attrition, with unexpected adverse events being a major cause of failure. Serious adverse events have led to organ failure, cancer development and deaths that were not expected outcomes in clinical trials. These life-threatening events were not identified during therapeutic development due to the lack of preclinical safety tests that faithfully represented human physiology. We highlight the successful application of several novel technologies, including high-throughput screening, organs-on-chips, microbiome-containing drug-testing platforms and humanised mouse models, for mechanistic studies and prediction of toxicity. We propose the incorporation of similar preclinical tests into future drug development to reduce the likelihood of hazardous therapeutics entering later-stage clinical trials.