转变儿科罕见病药物开发：加强虚拟患者的临床试验和监管证据。

IF 3 3区医学 Q2 PHARMACOLOGY & PHARMACY

CPT: Pharmacometrics & Systems Pharmacology Pub Date : 2025-08-04 DOI:10.1002/psp4.70096

Fianne Sips, Marco Virgolin, Giuseppe Pasculli, Federico Reali, Alessio Paris, Annette Janus, Yann Godfrin, Daniel Röshammar, Luca Marchetti, Jane Knöchel

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引用次数: 0

摘要

儿童罕见病的药物开发由于临床试验设计的实际和伦理限制而变得复杂，这些限制源于小的、高度异质的和脆弱的患者群体。使用机器学习（ML）、机械驱动的计算方法或其混合方法创建的虚拟患者（vp）有可能加速并最大化试验的影响。我们以成人和儿童为例，讨论了副总裁在改变儿科罕见病临床试验效率和影响方面的潜力。

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

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.

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