Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients

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 computat...

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Auteurs principaux:	Fianne Sips, Marco Virgolin, Giuseppe Pasculli, Federico Reali, Alessio Paris, Annette Janus, Yann Godfrin, Daniel Röshammar, Luca Marchetti, Jane Knöchel
Format:	Artigo
Langue:	Inglês
Publié:	Wiley 2025-11-01
Collection:	CPT: Pharmacometrics & Systems Pharmacology
Accès en ligne:	https://doi.org/10.1002/psp4.70096
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