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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Detalhes bibliográficos

Principais autores:	Fianne Sips, Marco Virgolin, Giuseppe Pasculli, Federico Reali, Alessio Paris, Annette Janus, Yann Godfrin, Daniel Röshammar, Luca Marchetti, Jane Knöchel
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Wiley 2025-11-01
Colecção:	CPT: Pharmacometrics & Systems Pharmacology
Acesso em linha:	https://doi.org/10.1002/psp4.70096
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