In silico clinical trials for paediatric orphan diseases

In silico clinical trials provide an effective solution to the obstacles encountered in carrying out in vivo clinical trials. They have the potential to transform the approach of investigating and developing treatments for rare conditions, thereby enhancing the quality of life for individuals worldwide. To enhance existing treatment approaches, the utilization of mechanistic and datadriven modelling proves to be beneficial. These tools allow for the simulation and analysis of data obtained from in vivo clinical trials, as well as the categorization of subject populations. This technique has the capacity to completely transform the future of drug discovery, particularly for rare paediatric diseases.