CVBF embraces an innovative computational approach to advance research in paediatric rare diseases, involving the creation of virtual patient cohorts and the use of in silico trials. At the two-day conference “Il Ruolo del Farmacologo nell’applicazione delle Metodologie Innovative nella Ricerca Preclinica e Clinica Pediatrica” held on 21-22 March 2024 and hosted by the Società Italiana di Farmacologia (SIF) at the University of Trieste, CVBF presented an abstract on this groundbreaking methodology.

The abstract, titled “Generation of a Virtual Cohort of patients for paediatric rare diseases in silico trials,” was co-authored by a team of researchers including Giorgio Reggiardo, Arianna Bertolani, Claudia Pansieri, Mariagrazia Felisi, and Donato Bonifazi. It explores the concept of creating virtual representations of patient populations for rare diseases, enabling more efficient and effective in silico clinical trials.

Paediatric rare diseases have posed significant challenges for researchers due to limited patient populations and logistical obstacles hindering traditional clinical trial methods. By leveraging advanced computational techniques and data-driven approaches, CVBF aims to transform the way rare disease research is conducted.

Virtual cohorts are digital representations of patient populations, integrating diverse data sources such as electronic health records and patient registries. These virtual cohorts can then be utilized in computer simulations, known as in silico trials, to evaluate the safety and efficacy of potential therapeutic interventions without the need for large-scale physical trials.

Through this innovative approach, researchers can overcome the limitations of small patient populations and accelerate the development of treatments for children affected by rare diseases. The virtual cohorts enable the exploration of various scenarios, the identification of potential responders, and the optimization of treatment regimens within a controlled and cost-effective computational environment.

Through interdisciplinary collaborations and the integration of advanced computational techniques, CVBF is working towards a future where personalized and effective treatments become a reality for even the rarest of diseases.