Drug development in pediatric rare diseases is complicated by practical and ethical constraints on clinical trial design, stemming from small, highly heterogeneous, and vulnerable patient populations. Virtual patients (VPs) created with machine-learning (ML), mechanistically driven computational approaches, or hybrids thereof, have the potential to expedite and maximize the impact of trials. We discuss the potential of VPs to transform the efficiency and impact of clinical trials in pediatric rare diseases, based on adult and pediatric examples.
Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients / Sips, F., Virgolin, M., Pasculli, G., Reali, F., Paris, A., Janus, A., Godfrin, Y., Röshammar, D., Marchetti, L., Knöchel, J.. - In: CPT: PHARMACOMETRICS & SYSTEMS PHARMACOLOGY. - ISSN 2163-8306. - 14:11(2025), pp. 1739-1742. [10.1002/psp4.70096]
Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients
Giuseppe Pasculli;
2025
Abstract
Drug development in pediatric rare diseases is complicated by practical and ethical constraints on clinical trial design, stemming from small, highly heterogeneous, and vulnerable patient populations. Virtual patients (VPs) created with machine-learning (ML), mechanistically driven computational approaches, or hybrids thereof, have the potential to expedite and maximize the impact of trials. We discuss the potential of VPs to transform the efficiency and impact of clinical trials in pediatric rare diseases, based on adult and pediatric examples.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
