FDA Modernization Act 2.0: transitioning beyond animal models with human cells, organoids, and AI/ML-based approaches
Abstract
. Introduction The discovery and generation of effective therapeutics to combat disease lies at the heart of biomedical research. Preclinical studies form the foundation of potential disease treatments, guiding their journey from scientific discovery to impactful patient outcomes. However, over the past two decades, preclinical research has been frequently plagued by the failure to replicate consistent results, costing an estimated $28 billion USD per year (1). Potential therapeutics from preclinical studies entering phase I trials only had a 10.4% approval rate between 2003 and 2014 (2) and an even lower 6% to 7% rate between 2011 and 2017 (3). The disappointing reality of promising preclinical findings that fail to translate into effective therapies has raised serious concerns within the scientific community (4). The cause of this failure is potentially elucidated in a 2015 retrospective analysis of four large biotech companies that showed the most common causes of termination in phase I and II clinical trials since 2003 are the lack of efficacy (60% of all trials) and toxicity (30%) (5). Given these insights and the emergence of advanced technologies that enable large-cohort, in vitro human testing, a pressing need to reassess our approaches to studying human diseases exists. Such changes are vital to facilitating the development of lifesaving therapeutics that can extend both health span and life span by more efficiently […] Viewpoint
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Evidence Hub reads this as a cohort study, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence.
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