The Evolving Role of In Silico Clinical Trials in Drug Development: Applications, Regulatory Perspectives and Future Opportunities

Abstract

The process of drug development involves significant expenses, extended timelines, and high attrition rates, necessitating innovative strategies to enhance research efficiency. In Silico clinical trials (ISCTs) are innovative approaches that leverage computational modelling, virtual patient simulations, and artificial intelligence to assist in the generation of clinical evidence throughout the drug development process. Such strategies can help identify candidates, optimise dose, design clinical trials, and assess treatment efficacy without the need for extensive human trials. Model-informed drug development approaches have become increasingly recognised as an important complement to traditional clinical evidence by regulatory agencies, such as physiologically based pharmacokinetic models and quantitative systems pharmacology models. However, there are issues of model validation, data quality, transparency, and regulatory acceptance that still need to be addressed. New technologies like digital twins, generative artificial intelligence, and real-world evidence integration will increase the value of ISCTs in the future. Together, these developments make ISCTs as valuable resources for supporting efficient, evidence-based, and patient-centred drug development.